Original Article
Bone Marrow Transplantation for Sickle Cell Disease
N Engl J Med 1996; 335:369-376August 8, 1996DOI: 10.1056/NEJM199608083350601
- Abstract
Background
We investigated the risks and benefits of allogeneic bone marrow transplantation in children with complications of sickle cell disease.
Methods
Twenty-two children less than 16 years of age who had symptomatic sickle cell disease received marrow allografts from HLA-identical siblings between September 1991 and April 1995. The indications for transplantation included a history of stroke (n = 12), recurrent acute chest syndrome (n = 5), and recurrent painful crises (n = 5). Patients were prepared for transplantation with busulfan, cyclophosphamide, and antithymocyte globulin.
Results
Twenty of the 22 patients survived, with a median follow-up of 23.9 months (range, 10.1 to 51.0), and 16 patients had stable engraftment of donor hematopoietic cells. In three patients the graft was rejected and sickle cell disease recurred; in a fourth patient graft rejection was accompanied by marrow aplasia. In 1 of the 16 patients with engraftment, there was stable mixed chimerism. Two patients died of central nervous system hemorrhage or graft-versus-host disease. Kaplan–Meier estimates of survival and event-free survival at four years were 91 percent and 73 percent, respectively. Among patients with a history of acute chest syndrome, lung function stabilized; among patients with prior central nervous system vasculopathy who had engraftment, stabilization of cerebrovascular disease was documented by magnetic resonance imaging.
Conclusions
Allogeneic stem-cell transplantation can be curative in young patients with symptomatic sickle cell disease.
Media in This Article
Figure 1
Kaplan–Meier Estimates of Survival and Event-free Survival after Bone Marrow Transplantation in 22 Patients with Sickle Cell Disease.Table 1
Criteria for Eligibility for Transplantation in Children with Sickle Cell Disease.Article Activity
- Article
Transplantation of hematopoietic stem cells from HLA-identical siblings can be curative in several nonmalignant hematologic disorders, including aplastic anemia, β-thalassemia major, congenital immunodeficiency disorders, and certain inborn errors of metabolism.1-3 Pilot studies of bone marrow transplantation for the treatment of young patients with symptomatic sickle cell disease have demonstrated eradication of the underlying disease with low transplantation-related mortality.4-10 We undertook this study to determine whether these results could be reproduced in a multicenter trial.
Because the clinical course of sickle cell disease is highly variable and difficult to predict, we attempted to identify patients at risk for poor outcomes before extensive organ damage due to sickle cell disease occurred. This strategy was similar to that applied in patients with β-thalassemia major.11 We selected patients with debilitating clinical events, such as stroke, recurrent acute chest syndrome, and recurrent painful vaso-occlusive crises, which contribute to the high morbidity and early mortality among patients with sickle cell disease.12 We report here the results of allogeneic stem-cell transplantation in 22 children with symptomatic sickle cell disease.
Methods
Patients less than 16 years of age with symptomatic sickle cell disease (sickle cell anemia [hemoglobin genotype S/S], sickle cell–hemoglobin C disease [S/C], or sickle cell–β-thalassemia) and HLA-identical sibling donors (hemoglobin genotype A/A or A/S) were considered for marrow transplantation. Typing for HLA-A, HLA-B, and HLA-DR antigens was performed with a standard complement-dependent microcytotoxicity assay.13 All patients were required to meet the eligibility criteria outlined in Table 1Table 1
Criteria for Eligibility for Transplantation in Children with Sickle Cell Disease. 10,14; those with extensive end-organ damage (e.g., stage III or IV sickle lung disease or severe renal impairment) were excluded.15,16 Patients were enrolled at 15 centers in the United States and Europe (see the Appendix). The study was approved by the institutional review board of the Fred Hutchinson Cancer Research Center and by the institutional review boards or their equivalents at each of the collaborating sites. All patients or their parents or guardians gave written informed consent for their participation.A Data Safety and Monitoring Board was appointed by the National Heart, Lung, and Blood Institute to monitor the safety of patients, adherence to ethical standards, and progress of this investigation. The board consisted of five hematologists, a biostatistician, and two patient advocates. Quarterly reports were submitted, and annual progress reports were presented to the board by the principal investigators.
Fourteen patients were prepared for transplantation with a combination of busulfan (total dose, 14 mg per kilogram of body weight, administered every six hours for 16 doses over four days), cyclophosphamide (total dose, 200 mg per kilogram, administered in 4 daily doses of 50 mg per kilogram), and horse antithymocyte globulin (ATGAM; total dose, 90 mg per kilogram, administered in 3 daily doses of 30 mg per kilogram). Four patients received CAMPATH antibody (10 mg per kilogram each day for five days) in lieu of antithymocyte globulin.17 Patients 9, 10, and 11 received busulfan (total dose, 500 mg per square meter of body-surface area, in 16 doses over four days), cyclophosphamide (200 mg per kilogram, as above), and rabbit antithymocyte globulin (total dose, 20 mg per kilogram, in 4 daily doses of 5 mg per kilogram), and Patient 6 received busulfan (16 mg per kilogram, in 16 doses over four days), cyclophosphamide (200 mg per kilogram, as above), and antithymocyte globulin (80 mg per kilogram, in 4 daily doses) at the discretion of the investigators at the center. Since November 1994, the pharmacokinetics of busulfan have been monitored in North American patients to maintain adjusted steady-state blood concentrations of 400 to 600 ng per milliliter in order to lower the risks of graft rejection and toxic effects. On the basis of their blood concentrations, we calculated that Patients 13, 19, and 20 (who were 5.2, 3.2, and 12.2 years of age, respectively) received a total dose of 14 mg of busulfan per kilogram, and Patient 17 (who was 4.2 years old) received 16 mg per kilogram. Patients received either a combination of methotrexate and cyclosporine (21 patients) or cyclosporine and prednisone (1 patient) for the prevention of acute graft-versus-host disease (GVHD).18,19 Prophylaxis with cyclosporine was given for six months after transplantation. The definitions and methods of grading acute and chronic GVHD have been described previously.18,19
Before transplantation, six patients who were not receiving long-term transfusion therapy underwent a partial exchange transfusion to achieve a fraction of hemoglobin S in the blood of 30 percent or less. All patients who were seronegative for cytomegalovirus received seronegative blood products.20 After transplantation, patients received prophylactic broad-spectrum antibiotics intravenously until the number of circulating neutrophils reached 500 per cubic millimeter and oral penicillin for at least two years after transplantation.
In response to an increased incidence of neurologic complications after transplantation, the following guidelines were adopted in June 1993: anticonvulsant prophylaxis with phenytoin was initiated during the administration of busulfan and continued for six months after transplantation, hypertension was strictly controlled, magnesium deficiency was promptly rectified, and hemoglobin concentrations were maintained at 9 to 11 g per deciliter (5.6 to 6.8 mmol per liter) and platelet counts above 50,000 per cubic millimeter.14 In addition, cerebral magnetic resonance imaging (MRI) and magnetic resonance angiography were performed in all patients before transplantation and 180 and 365 days after transplantation. Measurements of pulmonary function (total lung capacity, forced vital capacity, residual volume, and the ratio of forced expiratory volume to forced vital capacity) with pulse oximetry, arterial-blood gas measurements, or both, were made annually.
Hematopoietic-cell chimerism was identified by in situ hybridization with a Y-chromosome–specific probe in cells from marrow and peripheral blood when the donor and recipient were mismatched for sex and by studies of restriction-fragment–length polymorphisms or tandem repeats in DNA when the donor and recipient were of the same sex. These analyses were performed 56 days, 84 days, and 1 year after transplantation.
Statistical Analysis
We used the Kaplan–Meier method to estimate survival and event-free survival (the events we studied were death, graft rejection, and recurrence of sickle cell disease).21 A cumulative incidence curve for graft rejection is also presented.22 Survival and graft rejection were evaluated in relation to each of the following factors with the log-rank test22: the patient's age and sex, the donor's sex, the genotypes of both patient and donor, the number of transfusions received, the incidence of red-cell alloimmunization, previous chelation therapy, the presence of hepatomegaly, and the serum ferritin concentration before transplantation. We assessed the associations between various factors in contingency tables by Fisher's exact test.23
Results
Characteristics of the Patients
Twenty-one patients with sickle cell anemia and one patient with sickle β+-thalassemia received marrow allografts from HLA-identical siblings between September 1991 and April 1995 at 15 collaborating transplantation centers. The 8 girls and 14 boys ranged in age from 3.3 to 13.9 years (median, 10.4) (Table 2Table 2
Characteristics of the 22 Patients before Transplantation.). The indications for transplantation included a history of stroke (12 patients), recurrent acute chest syndrome (5 patients), and recurrent painful episodes (5 patients). The pretransplantation hemoglobin values varied from 6.4 to 11.9 g per deciliter (4.0 to 7.4 mmol per liter), and the hemoglobin S fractions reflected the long-term transfusion therapy given to 16 patients.Cerebral MRI was performed in all 22 patients before transplantation. Of the 12 patients with a history of overt stroke, all but 1 (Patient 13) had evidence of cerebral infarction, vasculopathy, or both on neuroimaging (Table 3Table 3
Complications of Sickle Cell Disease and Therapy Received by the 22 Patients.). Among the remaining 10 patients, 2 (Patients 6 and 18) had evidence of a “silent” cerebral infarction on cerebral MRI. Pulmonary function, tested in 18 patients, was normal in 7; mildly-to-moderately or moderately restrictive pulmonary disease was present in 6 patients, mildly restrictive changes were present in 4 patients, and mild airway obstruction without restrictive changes was noted in 1 patient (Table 3). A history of recurrent acute chest syndrome was the chief indication for transplantation in five patients, and three others (Patients 2, 9, and 17) had previously had one or two episodes of the acute chest syndrome. In this subgroup, pulmonary function was impaired in four patients, normal in two, and not studied in two. Of the five for whom a history of recurrent painful episodes was an indication for transplantation (Patients 9, 10, 16, 21, and 22), the number of painful crises ranged from 3 to 13 per year; two of these patients also had osteonecrosis.Sixteen patients were receiving monthly red-cell transfusions before transplantation (12 for stroke, 3 for recurrent painful episodes, and 1 for the acute chest syndrome). Six of these 16 patients had red-cell alloantibodies, and 10 were receiving chelation treatment for iron overload (Table 3). The serum ferritin values in all 22 patients ranged from 58 to 6795 ng per milliliter (mean, 1542). Most of the 22 children had received frequent transfusions before transplantation, although 5 patients had received 10 or fewer units of red cells. Of the 16 patients who underwent serologic testing before transplantation, 3 had evidence of a previous viral hepatitis infection (hepatitis A in Patient 9 and hepatitis C in Patients 1 and 6). Liver biopsies were performed before transplantation in eight patients with a history of hepatitis or numerous transfusions. Four patients (Patients 13, 16, 21, and 22) had mild-to-moderate hemosiderosis without portal fibrosis, one (Patient 2) had minimal periportal inflammation, and two (Patients 5 and 6) had changes consistent with chronic hepatitis. Only one patient (Patient 20, who had a serum ferritin concentration of 6795 ng per milliliter) had evidence of portal fibrosis.
Clinical Course after Stem-Cell Transplantation
In all patients the absolute neutrophil count exceeded 500 per cubic millimeter by a median of 18 days after transplantation (range, 15 to 32). The platelet count rose above 20,000 per cubic millimeter, independently of transfusions, by a median of 26 days after transplantation (range, 11 to 40). Mild-to-moderate veno-occlusive disease of the liver was noted in two patients, who recovered without sequelae. Interstitial pneumonitis developed in Patient 15, who had no prior pulmonary complications. Bronchoscopy showed no infective cause, and he recovered uneventfully. Five patients had bacteremia, including one episode of Haemophilus influenzae bacteremia with pneumonia in Patient 9. Grade II or III acute GVHD developed in two patients. The patient with grade III acute GVHD subsequently died of chronic GVHD.
Neurologic Complications
After four of the first seven patients enrolled had neurologic events, including two episodes of intracranial hemorrhage,14 we adopted the prophylactic measures described above. Three of the next 15 patients had seizures, but none had intracranial hemorrhage. Patient 19 had seizures during busulfan treatment and was noted to have a subtherapeutic plasma phenytoin concentration during oral loading. Three months later he had graft rejection and subsequently had recurrent cerebral infarction when the hemoglobin S fraction rose to 60 percent. He later resumed long-term transfusion therapy. Seizures developed in Patient 22 during replacement of a central venous catheter, and Patient 20 had seizures after the reinstitution of cyclosporine therapy. Both patients recovered without sequelae.
Graft Rejection
Sustained engraftment with 85 percent or more donor-derived blood cells was demonstrated by tests for chimerism in 16 of the 20 surviving patients (Table 4Table 4
Results of Studies of Chimerism and Outcomes after Transplantation.). Sickle cell disease recurred in three patients, and graft failure occurred in a fourth. One of the 16 patients with engraftment had stable mixed chimerism.Patient 3 had evidence of rejection on day 164 and by eight months after transplantation had no detectable hemoglobin A. She remained asymptomatic for nearly two years until she had a recurrence of vaso-occlusive crises; treatment with hydroxyurea was initiated, and she is currently free of symptoms. In Patient 12, hemoglobin electrophoresis on day 156 showed 30 percent hemoglobin S. There was no evidence of donor cells in the peripheral blood; accordingly, long-term transfusion therapy was resumed, and the patient remains asymptomatic. In Patient 19, cytogenetic studies performed one month after transplantation showed 40 percent donor cells. In situ hybridization with a Y-chromosome–specific probe of marrow progenitor colonies revealed a disparity in the degree of myeloid and erythroid chimerism: 6 of 34 blast-forming unit–erythroid colonies were of donor origin (18 percent), whereas 6 to 8 percent of 600 nuclei of myeloid cells derived from a long-term marrow culture were scored as donor cells. Similar findings have been reported in patients with thalassemia who had mixed chimerism after marrow transplantation and in an animal model of thalassemia.24,25
Patient 20 had 70 percent donor-derived hematopoietic cells in the bone marrow and 90 percent donor cells in the peripheral blood on day 180, but marrow aplasia developed 270 days after transplantation and she required red-cell and platelet transfusions. Chimerism studies demonstrated more than 90 percent host cells, a result that was consistent with graft rejection. After treatment with antithymocyte globulin and cyclosporine was instituted, the cellularity of the marrow improved and the need for transfusions diminished. Recent chimerism studies (315 days after transplantation) demonstrated 10 percent donor cells in the marrow and 66 percent donor cells in the peripheral blood.
Patient 13 had stable mixed chimerism. The hemoglobin S value was 12 percent three months after transplantation. Thereafter, serial studies revealed 10 to 20 percent donor cells and 30 percent hemoglobin S in the blood for nearly two years. As of this writing, the patient has no symptoms of sickle cell disease.
Outcome
After a median of 23.9 months (range, 10.1 to 51.0), 20 patients were alive, 16 of whom had stable engraftment with donor hematopoietic cells. Kaplan–Meier estimates of survival and event-free survival at four years were 91 percent and 73 percent, respectively; the cumulative incidence of graft rejection was 18 percent (Figure 1Figure 1
Kaplan–Meier Estimates of Survival and Event-free Survival after Bone Marrow Transplantation in 22 Patients with Sickle Cell Disease.). One patient died three months after transplantation from an intracerebral hemorrhage,14 and another died one year after transplantation from complications related to chronic GVHD and obliterative bronchiolitis. No risk factors for rejection were identified in the four patients who had graft rejection. Busulfan concentrations in four patients were judged adequate for donor engraftment; two of these four (Patients 19 and 20) had graft rejection despite having steady-state busulfan concentrations of 429 and 565 ng per milliliter, respectively.26 The effects of marrow transplantation on end-organ function were examined in patients with engraftment who were followed for at least 12 months (Table 3). All eight survivors with a history of stroke and stable donor-cell engraftment were studied with serial cerebral MRI and magnetic resonance angiography. As of this writing, they have had no progression of cerebral disease. The two patients with “silent” cerebral infarction before transplantation have had no changes on cerebral MRI. Pulmonary function could be evaluated in four of five patients with recurrent episodes of the acute chest syndrome before transplantation. All these patients had stable pulmonary function. No patient with engraftment had vaso-occlusive crises after transplantation.
Discussion
In this collaborative multicenter trial we investigated the risks and benefits of allogeneic bone marrow transplantation in children with symptomatic sickle cell disease. Our findings indicate that transplantation of hematopoietic stem cells can cure sickle cell anemia. End-organ damage was stabilized in patients with sustained engraftment of donor cells, but evaluation of the ultimate effect of transplantation on such damage requires longer follow-up. Transfusions have been discontinued in 16 patients who had engraftment; all have remained free of disabling symptoms. These results are especially encouraging in view of the severity of sickle cell disease in these patients.
There was allograft rejection followed by a recurrence of sickle cell disease or marrow aplasia in 4 of the 22 patients, a result similar to the 12 percent rejection rate reported among children with β-thalassemia major who received HLA-matched marrow grafts from siblings.2 Graft rejection after transplantation of hematopoietic stem cells occurs in patients with hemoglobinopathy more frequently than in patients with other hematologic disorders, such as aplastic anemia, severe combined immune deficiency, and malignant conditions, in whom the rate of rejection after the transplantation of marrow from HLA-identical siblings is less than 3 percent.27,28 The cause of this difference is not understood. Transfusion-induced sensitization could be a contributing factor, because among patients with aplastic anemia, the incidence of graft rejection is lower when transplantation is performed before transfusions are given.29
The frequency of graft rejection appears to be lower among patients with β-thalassemia who undergo transplantation early in the course of the disease, before red-cell transfusions lead to iron overload or liver disease.11,30 Lucarelli et al. reported a graft-rejection rate of 4 percent and event-free survival of 94 percent among such patients,11 and a similar outcome has been reported in patients with sickle cell disease.7,9,31 Among 28 Belgian patients with sickle cell anemia in an earlier report, 3 had graft rejection (1 of whom underwent a successful second transplantation), 1 died from transplantation-related complications, and 25 survived with stable donor engraftment (event-free survival, 89 percent).9 More recently, a Belgian bone marrow–transplantation center has required more severe clinical disturbances as a criterion for transplantation than were previously required.32 Of 12 patients who met these strict criteria, 4 had graft rejection, 1 died suddenly four years after transplantation, and 7 have survived free of sickle cell disease (Ferster A: personal communication). Giardini et al. reported that of four patients with advanced symptomatic sickle cell anemia, one is alive after marrow grafting, whereas four of four patients with less advanced sickle cell–β0-thalassemia have survived with donor-derived hematopoiesis.33 These data suggest that patients who have not undergone long-term transfusion therapy and who do not have severe complications related to sickle cell disease are optimal candidates for stem-cell transplantation.
We observed one case of stable mixed chimerism (in Patient 13) in which the reduction in hemoglobin S was sufficient to render the patient asymptomatic. Stable donor–host chimerism was also reported in patients with thalassemia.24,34 In such patients the risk of graft rejection appeared highest when more than 30 percent residual host cells were present two months after transplantation, although in three patients stable mixed chimerism persisted for four to seven years.24 Our data confirm that erythropoiesis by a minority of engrafted donor cells can lead to a majority of circulating normal erythrocytes.35 This observation has implications for the design of less cytotoxic pretransplantation conditioning regimens, which may result in stable mixed chimerism.
The optimal timing of marrow transplantation in the course of sickle cell anemia remains uncertain, in part because of the unpredictable nature of the disease. We selected patients who appeared to have a high risk of severe morbidity and early death. Only 6 percent of patients with sickle cell anemia who were less than 16 years old and were followed in the collaborating centers met the criteria for transplantation specified in the study protocol.36 But by the time these patients were selected, many had such severe vasculopathy and organ damage that they were predisposed to adverse events after transplantation. Marrow transplantation might therefore be better if performed early in the course of sickle cell disease, especially in patients with neurovascular disease (silent stroke) or pulmonary disease.37
The long-term administration of hydroxyurea to stimulate the production of fetal hemoglobin and the transfusion of red cells to lower sickle hemoglobin levels are other therapies now available for the treatment of patients with symptomatic sickle cell disease.38,39 The evolving role of the transplantation of hematopoietic stem cells and the availability of other therapeutic options underscore the need for controlled clinical trials to investigate the risks and benefits of and the indications for therapeutic intervention in sickle cell disease.
Supported in part by grants from the National Heart, Lung, and Blood Institute (HL36444) and the National Cancer Institute (CA18221 and CA15704).
We are indebted to Beverly Torok-Storb, Ph.D., for performing studies of marrow-progenitor chimerism and to the physicians and nurses at the sickle cell and transplantation centers for their support of this study.
Source Information
From the Division of Clinical Research, Fred Hutchinson Cancer Research Center, Seattle (M.C.W., M.P., W.L., D.C.M., R.S., K.M.S.); the Departments of Pediatrics (M.C.W., D.C.M.) and Medicine (R.S., K.M.S.), University of Washington, Seattle; Emory University, Atlanta (J.R.E.); the Medical College of Wisconsin, Milwaukee (J.P.S.); the University of California, San Francisco (W.C.M.); Central Middlesex Hospital National Health Service Trust, London (S.C.D.); the University of Pennsylvania, Philadelphia (K.O.-F.); and Hôpital Henri Mondor, Creteil, France (F.B.).
Address reprint requests to Dr. Sullivan at the Fred Hutchinson Cancer Research Center, Division of Clinical Research, FB600, 1124 Columbia St., Seattle, WA 98104.
Investigators and centers participating in this study are listed in the Appendix.
Other contributing authors were George R. Buchanan, M.D., University of Texas Southwestern Medical Center, Dallas; Philip J. Darbyshire, M.B., Ch.B., Birmingham Children's Hospital National Health Service Trust, Birmingham, United Kingdom; Robertson Parkman, M.D., University of Southern California, Los Angeles; Roswitha Dickerhoff, M.D., University of Bonn, Augustin, Germany; Franklin O. Smith, M.D., Indiana University School of Medicine, Indianapolis; and Jean E. Sanders, M.D., Fred Hutchinson Cancer Research Center, Seattle.
Appendix
The following investigators and centers made up the collaborative study group: Atlanta — J.R. Eckman and J. Wingard, Emory University; Augustin, Germany — R. Dickerhoff and T. Klingebiel, University of Bonn; Birmingham, United Kingdom — P.J. Darbyshire, Birmingham Children's Hospital National Health Service Trust; Boston — J. Antin, E. Guinan, B. Gee, and O. Platt, Dana–Farber Institute, Children's Hospital, and Harvard University, and L. McMahon, Boston Comprehensive Sickle Cell Center; Chapel Hill, N.C. — E. Orringer and J. Wiley, University of North Carolina; Creteil, France — F. Bernaudin, M. Kuentz, and J.P. Vernant, Hôpital Henri Mondor; Dallas — G.R. Buchanan, Z.R. Rogers, and E. Sandler, University of Texas Southwestern Medical Center; Durham, N.C. — M.L. Graham, T. Kinney, and J. Kurtzberg, Duke University; Houston — K.W. Chan, University of Texas; Indianapolis — F.O. Smith and W. Rackoff, Indiana University School of Medicine; London — S.C. Davies and I.A.G. Roberts, Royal Postgraduate Medical School; Los Angeles — R. Parkman, D. Powars, and C. Lenarsky, University of Southern California; Milwaukee — B. Camitta and J.P. Scott, Medical College of Wisconsin; New Haven, Conn. — J. Rappeport, Yale University; Oakland, Calif. — R. Vichinsky and L. Styles, Children's Hospital of Oakland; Philadelphia — K. Ohene-Frempong and N. Bunin, University of Pennsylvania; St. Louis — D. Wall, St. Louis University; St. Petersburg, Fla. — A. Wayne and M. Klemperer, University of Southern Florida; Sao Paulo, Brazil — S. Brandalise and R. Pasquini, University of Campinas; San Francisco — W.C. Mentzer and M. Cowan, University of California, San Francisco; Seattle — K.M. Sullivan, M.C. Walters, D.C. Matthews, J.E. Sanders, and F.R. Appelbaum, Fred Hutchinson Cancer Research Center and University of Washington; Stanford, Calif. — M. Amylon, B. Glader, and K. Blume, Stanford University; Toronto — N.F. Olivieri, Hospital for Sick Children; Washington, D.C. — P. Dindorf and O. Castro, Children's Hospital National Medical Center, George Washington University, and Howard University.
- References
References
1
Parkman R . The application of bone marrow transplantation to the treatment of genetic diseases. Science 1986;232:1373-1378
CrossRef | Web of Science | Medline2
Lucarelli G ,Galimberti M ,Polchi P , et al. Bone marrow transplantation in patients with thalassemia. N Engl J Med 1990;322:417-421
Free Full Text | Web of Science | Medline3
Camitta BM ,Storb R ,Thomas ED . Aplastic anemia: pathogenesis, diagnosis, treatment, and prognosis. N Engl J Med 1982;306:645-52, 712
Full Text | Web of Science | Medline4
Johnson FL ,Look AT ,Gockerman J ,Ruggiero MR ,Dalla-Pozza L ,Billings FT III . Bone-marrow transplantation in a patient with sickle-cell anemia. N Engl J Med 1984;311:780-783
Full Text | Web of Science | Medline5
Vermylen C ,Robles EF ,Ninane J ,Cornu G . Bone marrow transplantation in five children with sickle cell anemia. Lancet 1988;1:1427-1428
CrossRef | Web of Science | Medline6
Sullivan KM ,Reid CD . Introduction to a symposium on sickle cell anemia: current results of comprehensive care and the evolving role of bone marrow transplantation. Semin Hematol 1991;28:177-179
Web of Science | Medline7
Ferster A ,De Valck C ,Azzi N ,Fondu P ,Toppet M ,Sariban E . Bone marrow transplantation for severe sickle cell anaemia. Br J Haematol 1992;80:102-105
CrossRef | Web of Science | Medline8
Johnson FL ,Mentzer WC ,Kalinyak KA ,Sullivan KM ,Abboud MR . Bone marrow transplantation for sickle cell disease: the United States experience. Am J Pediatr Hematol Oncol 1994;16:22-26
Medline9
Vermylen C ,Cornu G . Bone marrow transplantation for sickle cell disease: the European experience. Am J Pediatr Hematol Oncol 1994;16:18-21
Medline10
Beutler E, Sullivan KM. Bone marrow transplantation for sickle cell disease. In: Forman SJ, Blume KG, Thomas ED, eds. Bone marrow transplantation. Boston: Blackwell Scientific, 1994:840-8.
11
Lucarelli G ,Galimberti M ,Polchi P , et al. Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. N Engl J Med 1993;329:840-844
Free Full Text | Web of Science | Medline12
Platt O. The sickle syndromes. In: Handin RI, Lux SE, Stossel TP, eds. Blood: principles & practice of hematology. Philadelphia: J.B. Lippincott, 1995:1645-700.
13
Hopkins KA. Basic microlymphocytotoxicity test. In: Zachary AA, Teresi GA, eds. Laboratory manual. 2nd ed. Lenexa, Kans.: American Society for Histocompatibility and Immunogenetics, 1990:195-201.
14
Walters MC ,Sullivan KM ,Bernaudin F , et al. Neurologic complications after allogeneic marrow transplantation for sickle cell anemia. Blood 1995;85:879-884
Web of Science | Medline15
Powars D ,Weidman JA ,Odom-Maryon T ,Niland JC ,Johnson C . Sickle cell chronic lung disease: prior morbidity and the risk of pulmonary failure. Medicine (Baltimore) 1988;67:66-76
Web of Science | Medline16
Powars DR ,Elliott-Mills DD ,Chan L , et al. Chronic renal failure in sickle cell disease: risk factors, clinical course, and mortality. Ann Intern Med 1991;115:614-620
Web of Science | Medline17
Dyer MJS ,Hale G ,Hayhoe FGJ ,Waldman H . Effects of CAMPATH-1 antibodies in vivo in patients with lymphoid malignancies: influence of antibody isotype. Blood 1989;73:1431-1439
Web of Science | Medline18
Storb R ,Deeg HJ ,Whitehead J , et al. Methotrexate and cyclosporine compared with cyclosporine alone for prophylaxis of acute graft versus host disease after marrow transplantation for leukemia. N Engl J Med 1986;314:729-735
Full Text | Web of Science | Medline19
Chao NJ ,Schmidt GM ,Niland JC , et al. Cyclosporine, methotrexate, and prednisone compared with cyclosporine and prednisone for prophylaxis of acute graft-versus-host disease. N Engl J Med 1993;329:1225-1230
Free Full Text | Web of Science | Medline20
Bowden RA ,Sayers M ,Flournoy N , et al. Cytomegalovirus immune globulin and seronegative blood products to prevent primary cytomegalovirus infection after marrow transplantation. N Engl J Med 1986;314:1006-1010
Full Text | Web of Science | Medline21
Kaplan EL ,Meier P . Nonparametric estimation from incomplete observations. J Am Stat Assoc 1958;53:457-481
CrossRef | Web of Science22
Kalbfleisch JD, Prentice NL. The statistical analysis of failure time data. New York: John Wiley, 1980:79-81, 168-71.
23
Fisher LD, van Belle G. Biostatistics: a methodology for health sciences. New York: John Wiley, 1993:185-6.
24
Andreani M ,Manna M ,Lucarelli G , et al. Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood 1996;87:3494-3499
Web of Science | Medline25
van den Bos C ,Kieboom D ,Wagemaker G . Correction of murine β-thalassemia by partial bone marrow chimerism: selective advantage of normal erythropoiesis. Bone Marrow Transplant 1993;12:9-13
Web of Science | Medline26
Slattery JT ,Sanders JE ,Buckner CD , et al. Graft-rejection and toxicity following bone marrow transplantation in relation to busulfan pharmacokinetics. Bone Marrow Transplant 1995;16:31-42
Web of Science | Medline27
Schuening F ,Deeg HJ ,Bean MA ,Storb R . Prevention of graft failure in patients with aplastic anemia. Bone Marrow Transplant 1993;12:Suppl 3:S48-S49
Web of Science | Medline28
Anasetti C ,Amos D ,Beatty PG , et al. Effect of HLA compatibility on engraftment of bone marrow transplants in patients with leukemia or lymphoma. N Engl J Med 1989;320:197-204
Full Text | Web of Science | Medline29
Anasetti C ,Doney KC ,Storb R , et al. Marrow transplantation for severe aplastic anemia: long-term outcome in fifty “untransfused“ patients. Ann Intern Med 1986;104:461-466
Web of Science | Medline30
Andreani M ,Manna M ,Nesci S ,Fattorini P ,Graziosi G ,Lucarelli G . Persistence of mixed chimerism in class 3 thalassemic patients following BMT. Bone Marrow Transplant 1991;7:Suppl 2:75-75
Medline31
Bernaudin F ,Souillet G ,Vannier JP , et al. Bone marrow transplantation (BMT) in 14 children with severe sickle cell disease (SCD): the French experience. Bone Marrow Transplant 1993;12:Suppl 1:118-121
Web of Science | Medline32
Ferster A, Corazza F, Bujan W, et al. Patients with sickle cell anemia who present graft failure after bone marrow transplantation may develop increased levels of fetal hemoglobin associated with improved clinical presentation. In: Beuzard Y, Lubin B, Rosa J, eds. Sickle cell disease and thalassaemias: new trends in therapy. London: John Libbey, 1995:579-80.
33
Giardini C ,Galimberti M ,Lucarelli G , et al. Bone marrow transplantation in sickle-cell anemia in Peasaro. Bone Marrow Transplant 1991;7:Suppl 2:122-123
Medline34
Nesci S ,Manna M ,Andreani M ,Fattorini P ,Graziosi G ,Lucarelli G . Mixed chimerism in thalassemic patients after bone marrow transplantation. Bone Marrow Transplant 1992;10:143-146
Web of Science | Medline35
Kapelushnik J ,Or R ,Filon D , et al. Analysis of β-globin mutations shows stable mixed chimerism in patients with thalassemia after bone marrow transplantation. Blood 1995;86:3241-3246
Web of Science | Medline36
Walters MC, Patience M, Leisenring W, et al. Barriers to bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant (in press).
37
Piomelli S . Sickle cell diseases in the 1990s: the need for active and preventive intervention. Semin Hematol 1991;28:227-232
Web of Science | Medline38
Charache S ,Terrin ML ,Moore RD , et al. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. N Engl J Med 1995;332:1317-1322
Free Full Text | Web of Science | Medline39
Styles LA ,Vichinsky E . Effects of a long-term transfusion regimen on sickle cell-related illnesses. J Pediatr 1994;125:909-911
CrossRef | Web of Science | Medline
- Citing Articles (182)
Citing Articles
1
Julie A. Panepinto, Sylvia Torres, Cristiane B. Bendo, Timothy L. McCavit, Bogdan Dinu, Sandra Sherman-Bien, Christy Bemrich-Stolz, James W. Varni. (2013) PedsQL™ sickle cell disease module: Feasibility, reliability, and validity. Pediatric Blood & Cancer 60:8, 1338-1344
2
Chioma Oringanje, Eneida Nemecek, Oluseyi Oniyangi, Chioma Oringanje. Hematopoietic stem cell transplantation for people with sickle cell disease. In: Cochrane Database of Systematic Reviews. John Wiley & Sons, Ltd, 2013.
3
Jesse D. Vrecenak, Alan W. Flake. (2013) In utero hematopoietic cell transplantation—recent progress and the potential for clinical application. Cytotherapy 15:5, 525-535
4
Mari H. Dallas, Brandon Triplett, David R. Shook, Christine Hartford, Ashok Srinivasan, Joseph Laver, Russell Ware, Wing Leung. (2013) Long-Term Outcome and Evaluation of Organ Function in Pediatric Patients Undergoing Haploidentical and Matched Related Hematopoietic Cell Transplantation for Sickle Cell Disease. Biology of Blood and Marrow Transplantation 19:5, 820-830
5
C D Fitzhugh, R P Weitzel, M M Hsieh, O A Phang, C Madison, L Luznik, J D Powell, J F Tisdale. (2013) Sirolimus and post transplant Cy synergistically maintain mixed chimerism in a mismatched murine model. Bone Marrow Transplantation
6
Susanne Matthes-Martin, Anita Lawitschka, Gerhard Fritsch, Thomas Lion, Brigitte Grimm, Sabine Breuer, Heidrun Boztug, Susanne Karlhuber, Wolfgang Holter, Christina Peters, Milen Minkov. (2013) Stem cell transplantation after reduced-intensity conditioning for sickle cell disease. European Journal of Haematology 90:4, 308-312
7
Susan Hunt, Joseph Alisky. (2013) Inpatient Management of Sickle Cell Disease. Hospital Medicine Clinics 2:2, e247-e262
8
Jean-Hugues Dalle. (2013) Hematopoietic stem cell transplantation in SCD. Comptes Rendus Biologies 336:3, 148-151
9
R. Hladun, I. Elorza, T. Olivé, J.L. Dapena, A. Llort, J. Sánchez de Toledo, C. Díaz de Heredia. (2013) Resultados del trasplante de progenitores hematopoyéticos en hemoglobinopatías: talasemia maior y enfermedad drepanocítica. Anales de Pediatría
10
J Freed, J Talano, T Small, A Ricci, M S Cairo. (2012) Allogeneic cellular and autologous stem cell therapy for sickle cell disease: ‘whom, when and how’. Bone Marrow Transplantation 47:12, 1489-1498
11
J. Bolanos-Meade, E. J. Fuchs, L. Luznik, S. M. Lanzkron, C. J. Gamper, R. J. Jones, R. A. Brodsky. (2012) HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease. Blood 120:22, 4285-4291
12
F. Bernaudin, M. Kuentz. (2012) Haplo-BMT: cure or back to sickle cell?. Blood 120:22, 4276-4277
13
Michael Roth, Julie Krystal, Deepa Manwani, Catherine Driscoll, Rosanna Ricafort. (2012) Stem Cell Transplant for Children with Sickle Cell Anemia: Parent and Patient Interest. Biology of Blood and Marrow Transplantation 18:11, 1709-1715
14
Michael J. Kelly, Brian W. Pennarola, Angie Mae Rodday, Susan K. Parsons, . (2012) Health-related quality of life (HRQL) in children with sickle cell disease and thalassemia following hematopoietic stem cell transplant (HSCT). Pediatric Blood & Cancer 59:4, 725-731
15
Franco Locatelli, Daria Pagliara. (2012) Allogeneic hematopoietic stem cell transplantation in children with sickle cell disease. Pediatric Blood & Cancer 59:2, 372-376
16
Naynesh R. Kamani, Mark C. Walters, Shelly Carter, Victor Aquino, Joel A. Brochstein, Sonali Chaudhury, Mary Eapen, Brian M. Freed, Michael Grimley, John E. Levine, Brent Logan, Theodore Moore, Julie Panepinto, Suhag Parikh, Michael A. Pulsipher, Jane Sande, Kirk R. Schultz, Stephen Spellman, Shalini Shenoy. (2012) Unrelated Donor Cord Blood Transplantation for Children with Severe Sickle Cell Disease: Results of One Cohort from the Phase II Study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Biology of Blood and Marrow Transplantation 18:8, 1265-1272
17
Emily Riehm Meier, Jeffery L. Miller. (2012) Sickle Cell Disease in Children. Drugs1
18
Anthony T.W. Cheung, Joshua W. Miller, Maricel G. Miguelino, Wilson J. To, Jiajing Li, Xin Lin, Peter C. Chen, Sandra L. Samarron, Ted Wun, Theodore Zwerdling, Ralph Green. (2012) Exchange Transfusion Therapy and Its Effects on Real-time Microcirculation in Pediatric Sickle Cell Anemia Patients. Journal of Pediatric Hematology/Oncology 34:3, 169-174
19
Suhag Parikh, Paul Szabolcs. (2012) Reduced-Intensity Conditioning (RIC) in Children with Nonmalignant Disorders (NMD) Undergoing Unrelated Donor Umbilical Cord Blood Transplantation (UCBT). Biology of Blood and Marrow Transplantation 18:1, S53-S55
20
Lori C. Jordan, James F. Casella, Michael R. DeBaun. (2012) Prospects for primary stroke prevention in children with sickle cell anaemia. British Journal of Haematologyno-no
21
Pablo Bartolucci, Frédéric Galactéros. (2012) Clinical management of adult sickle-cell disease :. Current Opinion in Hematology 19:3, 149
22
Hematology. In: Conn's Current Therapy 2012. Elsevier, 2012:767-871.
23
Adam Kirton, Gabrielle deVeber. Cerebrovascular Disease in Children. In: Swaiman's Pediatric Neurology. Elsevier, 2012:1395-1436.
24
Misaki Wayengera. (2012) Zinc finger nucleases for targeted mutagenesis and repair of the sickle-cell disease mutation: An in-silico study. BMC Blood Disorders 12:1, 5
25
Elisabeth H. Javazon, Mohamed Radhi, Bagirath Gangadharan, Jennifer Perry, David R. Archer. (2012) Hematopoietic Stem Cell Function in a Murine Model of Sickle Cell Disease. Anemia 2012, 1-9
26
M. Hsieh, C. Fitzhugh, J. F. Tisdale. (2011) Response: young adults with sickle cell disease have more transplant options. Blood 118:16, 4492-4493
27
Abdullah H. Al Jefri. (2011) Advances in Allogeneic Stem Cell Transplantation for Hemoglobinopathies. Hemoglobin 35:5-6, 469-475
28
Lucio Luzzatto, Foluke Fasola, Léon Tshilolo. (2011) Haematology in Africa. British Journal of Haematology 154:6, 777-782
29
M. M. Hsieh, C. D. Fitzhugh, J. F. Tisdale. (2011) Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now. Blood 118:5, 1197-1207
30
M. S. Islam, P. Anoop. (2011) Current concepts in the management of stroke in children with sickle cell disease. Child's Nervous System 27:7, 1037-1043
31
Ruby Khoury, Miguel R Abboud. (2011) Stem-cell transplantation in children and adults with sickle cell disease: an update. Expert Review of Hematology 4:3, 343-351
32
G Lucarelli, J Gaziev, A Isgrò, P Sodani, K Paciaroni, C Alfieri, G De Angelis, M Marziali, M D Simone, C Gallucci, A Roveda, F Saltarelli, F Torelli, M Andreani. (2011) Allogeneic cellular gene therapy in hemoglobinopathies—evaluation of hematopoietic SCT in sickle cell anemia. Bone Marrow Transplantation
33
M E McPherson, D Hutcherson, E Olson, A E Haight, J Horan, K-Y Chiang. (2011) Safety and efficacy of targeted busulfan therapy in children undergoing myeloablative matched sibling donor BMT for sickle cell disease. Bone Marrow Transplantation 46:1, 27-33
34
Eileen N. Hansbury, William H. Schultz, Russell E. Ware, Banu Aygun. (2011) Bone marrow transplant options and preferences in a sickle cell anemia cohort on chronic transfusions. Pediatric Blood & Cancern/a-n/a
35
Javid Gaziev, Guido Lucarelli. (2010) Allogeneic Cellular Gene Therapy for Hemoglobinopathies. Hematology/Oncology Clinics of North America 24:6, 1145-1163
36
Julie A. Panepinto, Raymond G. Hoffmann, Nicholas M. Pajewski. (2010) The effect of parental mental health on proxy reports of health-related quality of life in children with sickle cell disease. Pediatric Blood & Cancer 55:4, 714-721
37
Michelle Y. Owens, James N. Martin. Sickle Cell Crisis. In: Critical Care Obstetrics. Wiley-Blackwell, 2010:391-399.
38
Amir Ali Hamidieh, Mahdi Jalili, Omid Khojasteh, Ardeshir Ghavamzadeh. (2010) First Report of Successful Stem Cell Transplantation in a Patient With Sickle Cell Hemoglobin D Disease. Journal of Pediatric Hematology/Oncology 32:5, 397-399
39
Ellen C. Ebert, Michael Nagar, Klaus D. Hagspiel. (2010) Gastrointestinal and Hepatic Complications of Sickle Cell Disease. Clinical Gastroenterology and Hepatology 8:6, 483-489
40
Kay Sin Tan. Coagulation Disorders in Stroke. In: International Neurology. Wiley-Blackwell, 2010:23-24.
41
Hanane A. Dahoui, Munya N. Hayek, Paul J. Nietert, Mariam T. Arabi, Samar A. Muwakkit, Raya H. Saab, Abdallah N. Bissar, Nidal M. Jumaa, Fadi S. Farhat, Ibrahim A. Dabbous, Fadi F. Bitar, Miguel R. Abboud. (2010) Pulmonary hypertension in children and young adults with sickle cell disease: Evidence for familial clustering. Pediatric Blood & Cancer 54:3, 398-402
42
Mark C. Walters, Karen Hardy, Sandie Edwards, Thomas Adamkiewicz, James Barkovich, Francoise Bernaudin, George R. Buchanan, Nancy Bunin, Roswitha Dickerhoff, Roger Giller, Paul R. Haut, John Horan, Lewis L. Hsu, Naynesh Kamani, John E. Levine, David Margolis, Kwaku Ohene-Frempong, Melinda Patience, Rupa Redding-Lallinger, Irene A.G. Roberts, Zora R. Rogers, Jean E. Sanders, J. Paul Scott, Keith M. Sullivan. (2010) Pulmonary, Gonadal, and Central Nervous System Status after Bone Marrow Transplantation for Sickle Cell Disease. Biology of Blood and Marrow Transplantation 16:2, 263-272
43
Hady Felfly, Marie Trudel. (2010) Successful correction of murine sickle cell disease with reduced stem cell requirements reinforced by fractionated marrow infusions. British Journal of Haematology 148:4, 646-658
44
Margaret L. MacMillan, Mark C. Walters, Eliane Gluckman. (2010) Transplant Outcomes in Bone Marrow Failure Syndromes and Hemoglobinopathies. Seminars in Hematology 47:1, 37-45
45
Hari Prabhakar, Carlton Haywood, Robert Molokie. (2010) Sickle cell disease in the United States: Looking back and forward at 100 years of progress in management and survival. American Journal of HematologyNA-NA
46
Hsieh , Matthew M. , Kang , Elizabeth M. , Fitzhugh , Courtney D. , Link , M. Beth , Bolan , Charles D. , Kurlander , Roger , Childs , Richard W. , Rodgers , Griffin P. , Powell , Jonathan D. , Tisdale , John F. , . (2009) Allogeneic Hematopoietic Stem-Cell Transplantation for Sickle Cell Disease. New England Journal of Medicine 361:24, 2309-2317
Free Full Text47
L. A. Verduzco, D. G. Nathan. (2009) Sickle cell disease and stroke. Blood 114:25, 5117-5125
48
V K Prasad, J Kurtzberg. (2009) Umbilical cord blood transplantation for non-malignant diseases. Bone Marrow Transplantation 44:10, 643-651
49
Dorothy I. Bulas. (2009) Transcranial Doppler: Applications in Neonates and Children. Ultrasound Clinics 4:4, 533-551
50
Marianne E. McPherson, Alan R. Anderson, Ann E. Haight, Paula Jessup, Marta-Inés Castillejo, Christopher D. Hillyer, Cassandra D. Josephson. (2009) Transfusion management of sickle cell patients during bone marrow transplantation with matched sibling donor. Transfusion 49:9, 1977-1986
51
Parameswaran Anoop, David H. Bevan, Suparno Chakrabarti. (2009) Usefulness and limitations of Bayesian network model as a mortality risk assessment tool in sickle cell anemia. American Journal of Hematology 84:5, 312-313
52
Javier Bolaños-Meade, Robert A Brodsky. (2009) Blood and marrow transplantation for sickle cell disease: overcoming barriers to success. Current Opinion in Oncology 21:2, 158-161
53
Julie A. Panepinto, Nicholas M. Pajewski, Lisa M. Foerster, Svapna Sabnis, Raymond G. Hoffmann. (2009) Impact of family income and sickle cell disease on the health-related quality of life of children. Quality of Life Research 18:1, 5-13
54
Chioma Oringanje, Eneida Nemecek, Oluseyi Oniyangi, Chioma Oringanje. Hematopoietic stem cell transplantation for children with sickle cell disease. In: Cochrane Database of Systematic Reviews. John Wiley & Sons, Ltd, 2009.
55
Lakshmanan Krishnamurti, Sandhya Kharbanda, Melinda A. Biernacki, Wandi Zhang, K. Scott Baker, John E. Wagner, Catherine J. Wu. (2008) Stable Long-Term Donor Engraftment following Reduced-Intensity Hematopoietic Cell Transplantation for Sickle Cell Disease. Biology of Blood and Marrow Transplantation 14:11, 1270-1278
56
Julie A. Panepinto, Nicholas M. Pajewski, Lisa M. Foerster, Raymond G. Hoffmann. (2008) The Performance of the PedsQL Generic Core Scales in Children With Sickle Cell Disease. Journal of Pediatric Hematology/Oncology 30:9, 666-673
57
Paul M. Armistead, Mehrdad Mohseni, Roslyn Gerwin, Emily C. Walsh, Masoud Iravani, Bahram Chahardouli, Shahrbano Rostami, Wandi Zhang, Donna Neuberg, John Rioux, Ardeshir Ghavamzadeh, Jerome Ritz, Joseph H. Antin, Catherine J. Wu. (2008) Erythroid-lineage–specific engraftment in patients with severe hemoglobinopathy following allogeneic hematopoietic stem cell transplantation. Experimental Hematology 36:9, 1205-1215
58
Demetri Merianos, Todd Heaton, Alan W. Flake. (2008) In Utero Hematopoietic Stem Cell Transplantation: Progress toward Clinical Application. Biology of Blood and Marrow Transplantation 14:7, 729-740
59
A.E. Fawibe. (2008) Managing acute chest syndrome of sickle cell disease in an African setting. Transactions of the Royal Society of Tropical Medicine and Hygiene 102:6, 526-531
60
Neena Kapoor. (2008) Graft-versus-host disease and immunosuppression. Immunologic Research 41:1, 34-44
61
Allison A King, Michael R DeBaun, Desiree A White. (2008) Need for cognitive rehabilitation for children with sickle cell disease and strokes. Expert Review of Neurotherapeutics 8:2, 291-296
62
C Oringanje, E Nemecek, O Oniyangi, Chioma Oringanje. Hematopoietic stem cell transplantation for children with sickle cell disease. In: Cochrane Database of Systematic Reviews. John Wiley & Sons, Ltd, 2008.
63
K. I. Ataga, W. R. Smith, L. M. De Castro, P. Swerdlow, Y. Saunthararajah, O. Castro, E. Vichinsky, A. Kutlar, E. P. Orringer, G. C. Rigdon, J. W. Stocker, . (2008) Efficacy and safety of the Gardos channel blocker, senicapoc (ICA-17043), in patients with sickle cell anemia. Blood 111:8, 3991-3997
64
M Bhatia, M C Walters. (2008) Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future. Bone Marrow Transplantation 41:2, 109-117
65
Timothy G. Givens. Sickle Cell Disease. In: Pediatric Emergency Medicine. Elsevier, 2008:898-904.
66
Laura S. Inselman. Pulmonary Manifestations of Systemic Disorders. In: Pediatric Respiratory Medicine. Elsevier, 2008:1053-1079.
67
Mitchell E. Horwitz, Ivan Spasojevic, Ashley Morris, Marilyn Telen, James Essell, Cristina Gasparetto, Keith Sullivan, Gwynn Long, John Chute, Nelson Chao, David Rizzieri. (2007) Fludarabine-Based Nonmyeloablative Stem Cell Transplantation for Sickle Cell Disease with and without Renal Failure: Clinical Outcome and Pharmacokinetics. Biology of Blood and Marrow Transplantation 13:12, 1422-1426
68
N. Matsuda, T. Shimizu, M. Yamato, T. Okano. (2007) Tissue Engineering Based on Cell Sheet Technology. Advanced Materials 19:20, 3089-3099
69
Matthew M. Hsieh, Saskia Langemeijer, Aisha Wynter, Oswald A. Phang, Elizabeth M. Kang, John F. Tisdale. (2007) Low-dose parenteral busulfan provides an extended window for the infusion of hematopoietic stem cells in murine hosts. Experimental Hematology 35:9, 1415-1420
70
Tom V. Adamkiewicz, Paul Szabolcs, Ann Haight, K. Scott Baker, Susan Staba, Amos Kedar, K. Y. Chiang, Lakshmanan Krishnamurti, Michael W. Boyer, Joan Kurtzberg, John E. Wagner, John R. Wingard, Andrew M. Yeager. (2007) Unrelated cord blood transplantation in children with sickle cell disease: Review of four-center experience. Pediatric Transplantation 11:6, 641-644
71
Jane Hankins, Pamela Hinds, Sara Day, Yvonne Carroll, Chin-Shang Li, Patricia Garvie, Winfred Wang. (2007) Therapy preference and decision-making among patients with severe sickle cell anemia and their families. Pediatric Blood & Cancer 48:7, 705-710
72
Julie A. Panepinto, Mark C. Walters, Jeanette Carreras, Judith Marsh, Christopher N. Bredeson, Robert Peter Gale, Gregory A. Hale, John Horan, Jill M. Hows, John P. Klein, Ricardo Pasquini, Irene Roberts, Keith Sullivan, Mary Eapen, Alina Ferster, . (2007) Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. British Journal of Haematology 137:5, 479-485
73
Paul S. Frenette, George F. Atweh. (2007) Sickle cell disease: old discoveries, new concepts, and future promise. Journal of Clinical Investigation 117:4, 850-858
74
Lakshmanan Krishnamurti. (2007) Hematopoietic cell transplantation for sickle cell disease: state of the art. Expert Opinion on Biological Therapy 7:2, 161-172
75
Claudia R Morris, Sylvia T Singer, Mark C Walters. (2007) Clinical hemoglobinopathies: iron, lungs and new blood. Current Opinion in Internal Medicine 6:1, 60-71
76
B. Eggleston, M. Patience, S. Edwards, T. Adamkiewicz, G. R. Buchanan, S. C. Davies, R. Dickerhoff, S. Donfield, S. A. Feig, R. H. Giller, A. Haight, J. Horan, L. L. Hsu, N. Kamani, P. Lane, J. E. Levine, D. Margolis, T. B. Moore, K. Ohene-Frempong, R. Redding-Lallinger, I. A. G. Roberts, Z. R. Rogers, J. E. Sanders, J. P. Scott, B. Sleight, A. A. Thompson, K. M. Sullivan, M. C. Walters, . (2007) Effect of myeloablative bone marrow transplantation on growth in children with sickle cell anaemia: results of the multicenter study of haematopoietic cell transplantation for sickle cell anaemia. British Journal of Haematology 136:4, 673-676
77
Krista L. Hillyer, James R. Eckman. Transfusion of the Hemoglobinopathy Patient. In: Blood Banking and Transfusion Medicine. Elsevier, 2007:523-538.
78
Mark I. Evans, Yuval Yaron, Jan Deprest, Alan W. Flake, Charles Kleinman, Michael R. Harrison. Fetal Therapy. In: Obstetrics: Normal and Problem Pregnancies. Elsevier, 2007:245-266.
79
Joseph Yang, Masayuki Yamato, Kohji Nishida, Takeshi Ohki, Masato Kanzaki, Hidekazu Sekine, Tatsuya Shimizu, Teruo Okano. (2006) Cell delivery in regenerative medicine: The cell sheet engineering approach. Journal of Controlled Release 116:2, 193-203
80
N Qureshi, B Lubin, M C Walters. (2006) The prevention and management of stroke in sickle cell anaemia. Expert Opinion on Biological Therapy 6:11, 1087-1098
81
Shigeyuki Nagata, Shinji Okano, Yoshikazu Yonemitsu, Kazunori Nakagawa, Yukihiro Tomita, Yasunobu Yoshikai, Mitsuo Shimada, Yoshihiko Maehara, Katsuo Sueishi. (2006) Critical Roles of Memory T Cells and Antidonor Immunoglobulin in Rejection of Allogeneic Bone Marrow Cells in Sensitized Recipient Mice. Transplantation 82:5, 689-698
82
Paul Woodard. Hematopoietic Stem-Cell Transplantation for Sickle Cell Disease. In: Pediatric Hematopoietic Stem Cell Transplantation. CRC Press, 2006:397-412.
83
Jeffrey A Switzer, David C Hess, Fenwick T Nichols, Robert J Adams. (2006) Pathophysiology and treatment of stroke in sickle-cell disease: present and future. The Lancet Neurology 5:6, 501-512
84
Richard A. Nash, Peter A. McSweeney, J. Lee Nelson, Mark Wener, George E. Georges, Amelia A. Langston, Howard Shulman, Keith M. Sullivan, Julie Lee, Gretchen Henstorf, Rainer Storb, Daniel E. Furst. (2006) Allogeneic marrow transplantation in patients with severe systemic sclerosis: Resolution of dermal fibrosis. Arthritis & Rheumatism 54:6, 1982-1986
85
B Horn, L-A Baxter-Lowe, L Englert, A McMillan, M Quinn, K DeSantes, M Cowan. (2006) Reduced intensity conditioning using intravenous busulfan, fludarabine and rabbit ATG for children with nonmalignant disorders and CML. Bone Marrow Transplantation 37:3, 263-269
86
Lakshmanan Krishnamurti. (2006) Commentary on ???Identification of Unrelated Cord Blood Units for Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease???. Journal of Pediatric Hematology/Oncology 28:1, 1-3
87
Samir K. Ballas. (2005) Pain Management of Sickle Cell Disease. Hematology/Oncology Clinics of North America 19:5, 785-802
88
Julie A. Panepinto, Kerry M. O'Mahar, Michael R. DeBaun, Fausto R. Loberiza, J. P. Scott. (2005) Health-related quality of life in children with sickle cell disease: child and parent perception. British Journal of Haematology 130:3, 437-444
89
Paul Woodard, Kathleen J. Helton, Raja B. Khan, Gregory A. Hale, Sean Phipps, Winfred Wang, Rupert Handgretinger, John M. Cunningham. (2005) Brain parenchymal damage after haematopoietic stem cell transplantation for severe sickle cell disease. British Journal of Haematology 129:4, 550-552
90
Marco Mielcarek, Theodore Gooley, Paul J. Martin, Thomas R. Chauncey, Bessie A. Young, Rainer Storb, Beverly Torok-Storb. (2005) Effects of race on survival after stem cell transplantation. Biology of Blood and Marrow Transplantation 11:3, 231-239
91
Julie A. Panepinto, David C. Brousseau, Cheryl A. Hillery, J. Paul Scott. (2005) Variation in hospitalizations and hospital length of stay in children with vaso-occlusive crises in sickle cell disease. Pediatric Blood & Cancer 44:2, 182-186
92
Charles T. Quinn, Scott T. Miller. (2004) Risk factors and prediction of outcomes in children and adolescents who have sickle cell anemia. Hematology/Oncology Clinics of North America 18:6, 1339-1354
93
Julie A Panepinto, Kerry M O???Mahar, Michael R DeBaun, Kimberly M Rennie, J Paul Scott. (2004) Validity of the Child Health Questionnaire for Use In Children With Sickle Cell Disease. Journal of Pediatric Hematology/Oncology 26:9, 574-578
94
Mark C. Walters. (2004) Sickle cell anemia and hematopoietic cell transplantation: When is a pound of cure worth more than an ounce of prevention?. Pediatric Transplantation 8, 33-38
95
Rainer Storb. (2004) History of pediatric stem cell transplantation. Pediatric Transplantation 8, 5-11
96
J.B. Schnog, M.R. Mac Gillavry, A.P. van Zanten, J.C.M. Meijers, R.A. Rojer, A.J. Duits, H. ten Cate, D.P.M. Brandjes. (2004) Protein C and S and inflammation in sickle cell disease. American Journal of Hematology 76:1, 26-32
97
Franco Locatelli, Piero De Stefano. (2004) New insights into haematopoietic stem cell transplantation for patients with haemoglobinopathies. British Journal of Haematology 125:1, 3-11
98
Robin L. Brey, Bruce M. Coull. Coagulation Abnormalities in Stroke. In: Stroke. Elsevier, 2004:707-724.
99
Krista L. Hillyer. Hemoglobinopathies. In: Handbook of Pediatric Transfusion Medicine. Elsevier, 2004:209-220.
100
Juan José Ortega. (2004) Anemias hemolíticas. Anales de Pediatría Continuada 2:1, 12-21
101
Robert C Atkins, Mark C Walters. (2003) Haematopoietic cell transplantation in the treatment of sickle cell disease. Expert Opinion on Biological Therapy 3:8, 1215-1224
102
Warren D Shlomchik. (2003) Antigen presentation in graft-vs-host disease. Experimental Hematology 31:12, 1187-1197
103
F Baron. (2003) Hematopoietic cell transplantation: five decades of progress. Archives of Medical Research 34:6, 528-544
104
Christiane Vermylen. (2003) Hematopoietic stem cell transplantation in sickle cell disease. Blood Reviews 17:3, 163-166
105
Baron , Frederic , Dresse , Marie-Françoise , Beguin , Yves , . (2003) Transmission of Chronic Myeloid Leukemia through Peripheral-Blood Stem-Cell Transplantation. New England Journal of Medicine 349:9, 913-914
Free Full Text106
Te-Chao Fang, Richard Poulsom. (2003) Cell-based therapies for birth defects: A role for adult stem cell plasticity?. Birth Defects Research Part C: Embryo Today: Reviews 69:3, 238-249
107
Britt E. Anderson, Jennifer McNiff, Jun Yan, Hester Doyle, Mark Mamula, Mark J. Shlomchik, Warren D. Shlomchik. (2003) Memory CD4+ T cells do not induce graft-versus-host disease. Journal of Clinical Investigation 112:1, 101-108
108
Britt E. Anderson, Jennifer McNiff, Jun Yan, Hester Doyle, Mark Mamula, Mark J. Shlomchik, Warren D. Shlomchik. (2003) Memory CD4+ T cells do not induce graft-versus-host disease. Journal of Clinical Investigation 112:1, 101-108
109
Efemwonkiekie W Iyamu, Toshio Asakura. (2003) Drug therapy for sickle cell disease. Expert Opinion on Therapeutic Patents 13:6, 807-813
110
Thomas R. Spitzer. (2003) The expanding applications of non-myeloablative stem cell transplantation. Pediatric Transplantation 7, 95-100
111
Persis J. Amrolia, Antonio Almeida, Sally C. Davies, Irene A. G. Roberts. (2003) Therapeutic challenges in childhood sickle cell disease Part 2: a problem-orientated approach. British Journal of Haematology 120:5, 737-743
112
Rajinder Prasad, Syed Hasan, Oswaldo Castro, Elliott Perlin, Kyungsook Kim. (2003) Long-Term Outcomes in Patients with Sickle Cell Disease and Frequent Vaso-Occlusive Crises. The American Journal of the Medical Sciences 325:3, 107-109
113
Persis J. Amrolia, Antonio Almeida, Christina Halsey, Irene A. G. Roberts, Sally C. Davies. (2003) Therapeutic challenges in childhood sickle cell disease Part 1: current and future treatment options. British Journal of Haematology 120:5, 725-736
114
Frederick R. Appelbaum. (2003) The Current Status of Hematopoietic Cell Transplantation. Annual Review of Medicine 54:1, 491-512
115
Rainer Storb. (2003) Allogeneic hematopoietic stem cell transplantation—Yesterday, today, and tomorrow. Experimental Hematology 31:1, 1-10
116
George E. Georges, Rainer Storb. (2003) Review of “Minitransplantation”: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation. International Journal of Hematology 77:1, 3-14
117
J.J. Ortega Aramburu. (2003) Anemia de células falciformes: una enfermedad emergente en España. Anales de Pediatría 58:2, 93-94
118
Michael E. Trigg. (2002) Milestones in the development of pediatric hematopoietic stem cell transplantation-50 years of progress. Pediatric Transplantation 6:6, 465-474
119
Winfred C. Wang. (2002) Pain at Home in Sickle Cell Disease: An Underrecognized Problem. Journal of Pediatric Hematology/Oncology 24:8, 610-612
120
Henry O. Ogedegbe, PhD, BB(ASCP)SC. (2002) Sickle Cell Disease: An Overview. Laboratory Medicine 33:7, 515-543
121
Mara Prengler, Steven G. Pavlakis, Isak Prohovnik, Robert J. Adams. (2002) Sickle cell disease: The neurological complications. Annals of Neurology 51:5, 543-552
122
Tammara L. Jenkins. (2002) Sickle Cell Anemia in the Pediatric Intensive Care Unit: Novel Approaches for Managing Life-threatening Complications. AACN Clinical Issues: Advanced Practice in Acute and Critical Care 13:2, 154-168
123
Nabil Saba, Thomas Flaig. (2002) Bone Marrow Transplantation for Nonmalignant Diseases. Journal of Hematotherapy & Stem Cell Research 11:2, 377-387
124
F. Baron, Y. Beguin. (2002) Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation. Journal of Hematotherapy & Stem Cell Research 11:2, 243-263
125
Samir K. Ballas. (2002) Sickle Cell Anaemia. Drugs 62:8, 1143-1172
126
Michael Schleuning, Oliver Stoetzer, Christine Waterhouse, Markus Schlemmer, Georg Ledderose, Hans-Jochem Kolb. (2002) Hematopoietic stem cell transplantation after reduced-intensity conditioning as treatment of sickle cell disease. Experimental Hematology 30:1, 7-10
127
G Vassiliou, P Amrolia, I.A.G Roberts. (2001) Allogeneic transplantation for haemoglobinopathies. Best Practice & Research Clinical Haematology 14:4, 807-822
128
Samir K Ballas. (2001) Sickle cell disease: Current clinical management. Seminars in Hematology 38:4, 307-314
129
Sharada A. Sarnaik, Samir K. Ballas. (2001) Molecular characteristics of pediatric patients with sickle cell anemia and stroke. American Journal of Hematology 67:3, 179-182
130
John Moore, P. Brooks. (2001) Stem cell transplantation for autoimmune diseases. Springer Seminars in Immunopathology 23:1-2, 193-213
131
William Reed, Elliott P. Vichinsky. (2001) Transfusion Therapy: A Coming-of-Age Treatment for Patients With Sickle Cell Disease. Journal of Pediatric Hematology/Oncology 23:4, 197-202
132
Horwitz , Mitchell E. , Barrett , A. John , Brown , Margaret R. , Carter , Charles S. , Childs , Richard , Gallin , John I. , Gress , Ronald E. , Holland , Steven M. , Linton , Gilda F. , Miller , Judi A. , Leitman , Susan F. , Read , Elizabeth J. , Schermerhorn , James , Malech , Harry L. , . (2001) Treatment of Chronic Granulomatous Disease with Nonmyeloablative Conditioning and a T-Cell–Depleted Hematopoietic Allograft. New England Journal of Medicine 344:12, 881-888
Free Full Text133
Carolyn C. Hoppe, Mark C. Walters. (2001) Bone marrow transplantation in sickle cell anemia. Current Opinion in Oncology 13:2, 85-90
134
R. Grant Steen, Kathleen J. Helton, Edwin M. Horwitz, Ely Benaim, Stephen Thompson, Laura C. Bowman, Robert Krance, Winfred C. Wang, John M. Cunningham. (2001) Improved cerebrovascular patency following therapy in patients with sickle cell disease: Initial results in 4 patients who received HLA-identical hematopoietic stem cell allografts. Annals of Neurology 49:2, 222-229
135
Carlo Brugnara. (2001) Therapeutic Strategies for Prevention of Sickle Cell Dehydration. Blood Cells, Molecules, and Diseases 27:1, 71-80
136
Graham R. Serjeant. (2001) The emerging understanding of sickle cell disease. British Journal of Haematology 112:1, 3-18
137
Charles H. Pegelow. (2001) Stroke in Children With Sickle Cell Anaemia. Paediatric Drugs 3:6, 421-432
138
Keith E. Stockerl-Goldstein, Karl G. Blume. A decade of progress in allogeneic hematopoietic cell transplantation: 1990–2000. Elsevier, 2001:1-59.
139
Frederic Shapiro. Epiphyseal Involvement with Metabolic, Inflammatory, Neoplastic, Infectious, and Hematologic Disorders. In: Pediatric Orthopedic Deformities. Elsevier, 2001:872-933.
140
Hui-yu Lung, Ilse S Meeus, Rona S Weinberg, George F Atweh. (2000) In Vivo Silencing of the Human γ-Globin Gene in Murine Erythroid Cells Following Retroviral Transduction. Blood Cells, Molecules, and Diseases 26:6, 613-619
141
Lia Gore, Peter A. Lane, Ralph R. Quinones, Roger H. Giller. (2000) Successful Cord Blood Transplantation for Sickle Cell Anemia From a Sibling Who Is Human Leukocyte Antigen-Identical: Implications for Comprehensive Care. Journal of Pediatric Hematology/Oncology 22:5, 437-440
142
Emin Kansu, Keith Sullivan. Late Complications of Hematopoietic Stem Cell Transplantation. In: Hematopoietic Stem Cell Transplantation. CRC Press, 2000:413-433.
143
Michael Neipp, Christina Kaufman, Marianne Bergheim, Beate Exner, Suzanne Ildstad. Requirements for Hematopoietic Stem Cell Engraftment and Graft Engineering. In: Hematopoietic Stem Cell Transplantation. CRC Press, 2000:87-110.
144
Yasushi Fuchimoto, Christene A. Huang, Kazuhiko Yamada, Akira Shimizu, Hiroshi Kitamura, R.B. Colvin, Vincent Ferrara, Michael C. Murphy, Megan Sykes, Mary White-Scharf, David M. Neville, David H. Sachs. (2000) Mixed chimerism and tolerance without whole body irradiation in a large animal model. Journal of Clinical Investigation 105:12, 1779-1789
145
Robert A. Brodsky, Michelle Petri, Richard J. Jones. (2000) HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR SYSTEMIC LUPUS ERYTHEMATOSUS. Rheumatic Disease Clinics of North America 26:2, 377-387
146
Richard D. Moore, Samuel Charache, Michael L. Terrin, Franca B. Barton, Samir K. Ballas, . (2000) Cost-effectiveness of hydroxyurea in sickle cell anemia. American Journal of Hematology 64:1, 26-31
147
Kenneth I. Ataga, Eugene P. Orringer. (2000) Renal abnormalities in sickle cell disease. American Journal of Hematology 63:4, 205-211
148
W. Reed, P. Walker, T. Haddix, H.A. Perkins. (2000) Acute anemic events in sickle cell disease. Transfusion 40:3, 267-273
149
Miller , Scott T. , Sleeper , Lynn A. , Pegelow , Charles H. , Enos , Laura E. , Wang , Winfred C. , Weiner , Steven J. , Wethers , Doris L. , Smith , Jeanne , Kinney , Thomas R. , . (2000) Prediction of Adverse Outcomes in Children with Sickle Cell Disease. New England Journal of Medicine 342:2, 83-89
Free Full Text150
Aimen F. Shaaban, Alan W. Flake. (1999) Fetal hematopoietic stem cell transplantation. Seminars in Perinatology 23:6, 515-523
151
Mark C. Walters. (1999) Bone Marrow Transplantation for Sickle Cell Disease. Journal of Pediatric Hematology/Oncology 21:6, 467-474
152
Timothy E Mitchell, Martin H Steinberg. (1999) Novel approaches to treatment of sickle cell anaemia. Expert Opinion on Investigational Drugs 8:11, 1823-1836
153
Oswaldo Castro. (1999) MANAGEMENT OF SICKLE CELL DISEASE: RECENT ADVANCES AND CONTROVERSIES. British Journal of Haematology 107:1, 2-11
154
Jennifer Knight, Thomas M. Murphy, Iley Browning. (1999) The lung in sickle cell disease. Pediatric Pulmonology 28:3, 205-216
155
Joseph Polcaro, Michael Y. Divon, Eric Bentolila, William K. Rashbaum, William D. Lyman. (1999) Transplantation of CD34 human cells into mice with severe combined immunodeficiency results in functional T cells 4 weeks after transplantation. American Journal of Obstetrics and Gynecology 181:1, 80-86
156
Rodney J. Folz. (1999) Mechanisms of Lung Injury after Bone Marrow Transplantation. American Journal of Respiratory Cell and Molecular Biology 20:6, 1097-1099
157
Michael Neipp, Beate G Exner, Dipen Maru, Marian Haber, James S Gammie, Si M Pham, Suzanne T Ildstad. (1999) T-cell depletion of allogeneic bone marrow using anti-αβTCR monoclonal antibody. Experimental Hematology 27:5, 860-867
158
David W. Emery, Fionnuala Morrish, Qiliang Li, George Stamatoyannopoulos. (1999) Analysis of gamma-Globin Expression Cassettes in Retrovirus Vectors. Human Gene Therapy 10:6, 877-888
159
BEATE G. EXNER, MICHELE A. DOMENICK, MARIANNE BERGHEIM, YVONNE M. MUELLER, SUZANNE T. ILDSTAD. (1999) Clinical Applications of Mixed Chimerism. Annals of the New York Academy of Sciences 872:1 HEMATOPOIETIC, 377-386
160
Parkman , Robertson , . (1998) The Future of Placental-Blood Transplantation. New England Journal of Medicine 339:22, 1628-1629
Full Text161
A. J. Duits, J. B. Schnog, L. R. Lard, A.W. Saleh, R. A. Rojer. (1998) Elevated IL-8 levels during sickle cell crisis. European Journal of Haematology 61:5, 302-305
162
Adams , Robert J. , McKie , Virgil C. , Hsu , Lewis , Files , Beatrice , Vichinsky , Elliott , Pegelow , Charles , Abboud , Miguel , Gallagher , Dianne , Kutlar , Abdullah , Nichols , Fenwick T. , Bonds , Duane R. , Brambilla , Donald , Woods , Gerald , Olivieri , Nancy , Driscoll , Catherine , Miller , Scott Wang , Winfred , Hurlett , Anne , Scher , Charles , Berman , Brian , Carl , Elizabeth , Jones , Anne M. , Roach , E. Steve , Wright , Elizabeth , Zimmerman , Robert A. , Waclawiw , Myron , . (1998) Prevention of a First Stroke by Transfusions in Children with Sickle Cell Anemia and Abnormal Results on Transcranial Doppler Ultrasonography. New England Journal of Medicine 339:1, 5-11
Free Full Text163
K. M. SULLIVAN, C. ANASETTI, M. HOROWITZ, P. A. ROWLINGS, E. W. PETERSDORF, P. J. MARTIN, R. A. CLIFT, M. C. WALTERS, T. GOOLEY, J. SIERRA, J. E. ANDERSON, J. BJERKE, M. SIADAK, M. E. D. FLOWERS, R. A. NASH, J. E. SANDERS, F. R. APPELBAUM, R. STORB, J. A. HANSEN. (1998) Unrelated and HLA-Nonidentical Related Donor Marrow Transplantation forThalassemia and Leukemia: A Combined Report from the Seattle Marrow Transplant Team and the International Bone Marrow Transplant Registrya. Annals of the New York Academy of Sciences 850:1 COOLEY'S ANEM, 312-324
164
RAINER STORB, CONG YU, H. JOACHIM DEEG, GEORGE GEORGES, HANS-PETER KIEM, PETER A. MCSWEENEY, RICHARD A. NASH, BRENDA M. SANDMAIER, KEITH M. SULLIVAN, JOHN L. WAGNER, MARK C. WALTERS. (1998) Current and Future Preparative Regimens for Bone Marrow Transplantation in Thalassemiaa. Annals of the New York Academy of Sciences 850:1 COOLEY'S ANEM, 276-287
165
Yutaka Niihara, Charles R. Zerez, Dean S. Akiyama, Kouichi R. Tanaka. (1998) Oral L-glutamine therapy for sickle cell anemia: I. subjective clinical improvement and favorable change in red cell NAD redox potential. American Journal of Hematology 58:2, 117-121
166
R. Grant Steen, Wilburn E. Reddick, Raymond K. Mulhern, James W. Langston, Robert J. Ogg, Andrea A. Bieberich, Peter B. Kingsley, Winfred C. Wang. (1998) Quantitative MRI of the brain in children with sickle cell disease reveals abnormalities unseen by conventional MRI. Journal of Magnetic Resonance Imaging 8:3, 535-543
167
The-Hung Bui, D. Rhodri E. Jones. (1998) Stem cell transplantation into the fetal recipient: challenges and prospects. Current Opinion in Obstetrics and Gynaecology 10:2, 105-108
168
Napier, Chapman, Forman, Kelsey, Knowles, Murphy, Williamson, Wood, Kinsey, Murphy, Pamphilon, Warwick. (1998) Guidelines on the clinical use of leucocyte-depleted blood components. Transfusion Medicine 8:1, 59-71
169
Santiago Otero Lopez–Cubero, Keith M. Sullivan, George B. McDonald. (1998) Course of Crohn's disease after allogeneic marrow transplantation. Gastroenterology 114:3, 433-440
170
C. Anthony Blau. (1998) 10 Current status of stem cell therapy and prospects for gene therapy for the disorders of globin synthesis. Baillière's Clinical Haematology 11:1, 257-275
171
Samir K. Ballas. (1998) 7 Sickle cell disease: clinical management. Baillière's Clinical Haematology 11:1, 185-214
172
Griffin P. Rodgers. (1998) 9 Pharmacological therapy. Baillière's Clinical Haematology 11:1, 239-255
173
W. Reed, MD, E. P. Vichinsky, MD. (1998) NEW CONSIDERATIONS IN THE TREATMENT OF SICKLE CELL DISEASE. Annual Review of Medicine 49:1, 461-474
174
Bunn , H. Franklin . (1997) Pathogenesis and Treatment of Sickle Cell Disease. New England Journal of Medicine 337:11, 762-769
Full Text175
Graham R Serjeant. (1997) Sickle-cell disease. The Lancet 350:9079, 725-730
176
Laurence E. Walsh, Bhuwan P. Garg. (1997) Ischemic strokes in children. The Indian Journal of Pediatrics 64:5, 613-623
177
M. de Montalembert, M. Belloy, F. Bernaudin, F. Gouraud, R. Capdeville, R. Mardini, N. Philippe, J. P. Jais, J. Bardakdjian, R. Ducrocq, M. Maier-Redelsperger, J. Elion, D. Labie, R. Girot. (1997) Three-Year Follow-Up of Hydroxyurea Treatment in Severely Ill Children with Sickle Cell Disease. Journal of Pediatric Hematology/Oncology 19:4, 313-318
178
Richard Payne. (1997) PAIN MANAGEMENT IN SICKLE CELL ANEMIA. Anesthesiology Clinics of North America 15:2, 305-318
179
Richard E. Harris, Clinton H. Joiner. (1997) Should children with hemoglobinopathies be offered unrelated-donor cord blood stem cell transplantation?. The Journal of Pediatrics 130:5, 689-690
180
Patrick Kelly, Joanne Kurtzberg, Elliott Vichinsky, Bertram Lubin. (1997) Umbilical cord blood stem cells: Application for the treatment of patients with hemoglobinopathies. The Journal of Pediatrics 130:5, 695-703
181
(1996) Bone Marrow Transplantation for Sickle Cell Disease. New England Journal of Medicine 335:24, 1845-1846
Free Full Text182
Platt , Orah S. , Guinan , Eva C. , . (1996) Bone Marrow Transplantation in Sickle Cell Anemia — The Dilemma of Choice. New England Journal of Medicine 335:6, 426-428
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