Original Article
Bone Marrow Transplantation in Patients with Thalassemia
N Engl J Med 1990; 322:417-421February 15, 1990
- Abstract
- Abstract
We reviewed the results of transplantation of allogeneic marrow from HLA-identical donors in patients with β-thalassemia who were less than 16 years old.
Among the 222 consecutive patients who had received transplants since 1983, survival and event-free-survival curves leveled off about one year after transplantation, at 82 and 75 percent, respectively. Pretransplantation clinical characteristics were examined for their impact on survival, event-free survival, and the recurrence of thalassemia in the 116 consecutive patients who were treated with our current regimen, in use since June 1985. In a multivariate analysis, portal fibrosis and either the presence of hepatomegaly or a history of inadequate chelation therapy were significantly associated with reduced probabilities of survival and event-free survival.
The patients were divided into three classes on the basis of the presence of hepatomegaly or portal fibrosis (class 1 had neither factor, class 2 had one, and class 3 had both). For class 1 patients the three-year probabilities of survival, event-free survival, and recurrence were 94, 94, and 0 percent, respectively. For class 2 patients the probabilities were 80, 77, and 9 percent, and for class 3 patients 61, 53, and 16 percent.
We conclude that for patients under 16 years of age, transplantation of bone marrow from an HLA-identical donor offers a high probability of complication-free survival, particularly if they do not have hepatomegaly or portal fibrosis. (N Engl J Med 1990; 322:417–21.)
Media in This Article
Figure 1
Probabilities of Survival, Event-free Survival, and Recurrence in 222 Patients under 16 Years Old with Thalassemia Treated with Allogeneic Marrow Transplantation.Figure 2
Probabilities of Survival after Transplantation in 99 Patients with Thalassemia.Article Activity
- Article
HOMOZYGOUS β-thalassemia is a genetic disease characterized by the absent or defective production of beta-globin chains. It results in severe anemia and a consequent hyperstimulation of erythropoiesis and expansion of the bone marrow that can only be avoided by repeated red-cell transfusions. In this setting, the daily administration of deferoxamine is necessary to avoid severe iron overload.
The correction of this hematopoietic disorder by bone marrow transplantation was first demonstrated by Thomas et al. in a young patient who had not undergone transfusion.1 We have also reported our experience with patients with thalassemia who underwent transplantation after preparation with various regimens in early and advanced stages of the disease.2 3 4
In the present report we examine the results of marrow transplantation after preparation with busulfan and cyclophosphamide in 222 patients with thalassemia between the ages of 1 and 15 years.
Methods
Between January 27, 1983, and August 4, 1988, 222 patients between the ages of 1 and 15 underwent bone marrow transplantation for the treatment of homozygous β-thalassemia. A complete absence of beta-chain synthesis was demonstrated in 160 patients.
In 212 cases, donors and recipients were genotypically HLA-identical siblings. Ten patients received marrow from their parents, with whom they were phenotypically HLA identical at the A, B, C, and DR loci and mutually nonresponsive in mixed-leukocyte culture. Fifty-eight donors had major ABO incompatibility and 30 had minor incompatibility with the marrow recipients. When major ABO incompatibility existed, red cells were removed from the donor marrow by sedimentation with hydroxyethyl starch. The median dose of marrow cells was 4.1×108 per kilogram of body weight (range, 1.0 to 13.7). One hundred forty-one donors were heterozygous for β-thalassemia, and 81 were normal homozygotes.
All the patients were prepared for transplantation with a modification of the regimen described by Santos et al.5 Busulfan was administered orally at a dose of 3.5 mg per kilogram per day in three divided doses for four days (total dose, 14 mg per kilogram). This treatment was followed by 50 mg of cyclophosphamide per kilogram, administered intravenously on each of four successive days. Marrow was infused 36 hours after the last dose of cyclophosphamide. The date of infusion was designated day 0. Five different regimens were used to prevent acute graft-versus-host disease. One hundred sixteen patients received daily cyclosporine beginning two days before transplantation; these patients formed the population examined for prognostic indicators in this report. Twenty-six patients received the same cyclosporine regimen, together with cyclophosphamide (7.5 mg per kilogram) given intravenously on day 1 and methotrexate (10 mg per square meter of body-surface area) given intravenously on days 3, 6, and 11. For 55 patients the prophylaxis consisted of intravenous cyclophosphamide (7.5 mg per kilogram) on day 1, followed by intravenous methotrexate on days 3, 6, and 11 and then weekly through day 100. Ten patients received daily cyclosporine beginning nine days before transplantation. Fifteen patients received daily cyclosporine and deferoxamine (40 mg per kilogram) given intravenously by continuous infusion from 10 days before transplantation to day 60.
The daily dose of intravenous cyclosporine was reduced from 5 mg to 3 mg per kilogram five days after transplantation. The drug was given orally as soon as the patient could tolerate this route. The oral dose of cyclosporine was 12.5 mg per kilogram per day, which was reduced by 5 percent a week beginning on day 60. Cyclosporine prophylaxis was discontinued six months after transplantation in 29 patients and one year after transplantation in 138 patients.
All the patients were treated in positive-pressure isolation rooms and received nonabsorbable oral antibiotics and a low-bacteria diet. Systemic prophylaxis with amikacin and piperacillin was begun on the day before transplantation and continued until the granulocyte count exceeded 0.5×109 per liter and the patient was afebrile. Patients with persistent unexplained fever were treated with amphotericin B. All the blood products administered after transplantation were irradiated with 30 Gy. Acute and chronic graft-versus-host disease was graded according to the Seattle criteria.6 , 7
Cytogenetic analyses were performed on unstimulated marrow and phytohemagglutinin-stimulated peripheral blood when the donor and recipient were of opposite sexes. Globin-chain synthesis of marrow and peripheral-blood reticulocytes was examined by the incorporation of [3H]leucine followed by column chromatography.
Liver biopsy was performed in 180 patients before transplantation. Grading systems were established to record siderosis, chronic aggressive hepatitis, chronic persistent hepatitis, and portal fibrosis on liver biopsy. Three grades of severity (mild, moderate, and severe) were identified for each diagnostic category.8 Iron concentrations in the liver-biopsy specimens were determined by atomic-absorption spectrometry and expressed as micrograms per gram of dry tissue (normal values ranged between 300 and 1400 μg per gram).9
In evaluating the quality of chelation, achieving the subcutaneous infusion of 40 mg of deferoxamine per kilogram for 8 to 12 hours a day at least five days a week initiated not later than two years after red-cell transfusions began was considered to indicate good chelation. Failure to achieve this standard was categorized as poor chelation.
Survival distributions were estimated by the product-limit method10 and tested for equality by the Mantel–Cox and Breslow statistics. In estimating event-free survival, we identified rejection, the recurrence of thalassemia, and death as events. Multivariate analyses were performed with the Cox proportional-hazards regression model.11 Information about some covariates was not available for all patients (Table 1Table 1
Influence of Clinical Factors on Survival, Event-free Survival, and Rejection.*). When this was the case, separate analyses were performed to evaluate these covariates. When splenomegaly was entered into the model, data on patients who had undergone splenectomy were excluded. The reference date for calculating statistics was March 1, 1989.Informed consent was obtained from the parents of all the patients after the procedures and the risks involved in marrow transplantation were explained in detail. Explanations to the patients themselves varied in complexity according to their age. Particular emphasis was placed on the option of continuing conventional management with transfusions and deferoxamine.
Results
Figure 1Figure 1
Probabilities of Survival, Event-free Survival, and Recurrence in 222 Patients under 16 Years Old with Thalassemia Treated with Allogeneic Marrow Transplantation. shows the probabilities of survival, event-free survival, and rejection for all 222 patients who received transplants. The latest death was on day 376 after transplantation, and the latest rejection was on day 473. The survival and event-free-survival curves leveled off after about a year at 82 and 75 percent, respectively, and the longest-surviving patient is alive and disease-free nearly six years after transplantation. The five regimens used in these patients' transplantations were examined by univariate analysis for their influence on survival, and there were no statistically significant differences.The regimen currently in use was introduced in June 1985, and 116 patients had followed this regimen through August 1988. The outcome of transplantation in these patients was examined retrospectively in an attempt to devise a categorization scheme to identify prognostically informative groups.
The factors presented in Table 1 were examined for their influence on survival, event-free survival, and rejection. Three factors assessed at the time of transplantation were univariately associated with significantly reduced probabilities of survival and event-free survival. These factors were a poor quality of chelation (P = 0.01 and 0.005, respectively), the presence of hepatomegaly (P = 0.005 and 0.001), and the presence of portal fibrosis (P = 0.01 for both). The only factors univariately associated with an increased probability of rejection were hepatomegaly (P = 0.01), the presence of chronic aggressive hepatitis (P = 0.002), and the presence of splenomegaly (P = 0.03). The chelation, hepatomegaly, and portal fibrosis factors were submitted to multivariate analysis to determine their influence on survival. When hepatomegaly was entered into the model, the quality of chelation was no longer a significant influence. The presence of either hepatomegaly or portal fibrosis was significantly associated with a decreased probability of survival; the relative risks and 95 percent confidence intervals are shown in Table 2Table 2
Multivariate Analysis of Risk Factors.. The quality of chelation, the presence of hepatomegaly, and the presence of chronic aggressive hepatitis or portal fibrosis were submitted to multivariate analysis to determine their influence on event-free survival. Only hepatomegaly was identified as significantly influencing event-free survival (Table 2).Patients whose records were examined in the multivariate analysis were divided into three classes on the basis of the risk factors hepatomegaly and portal fibrosis. Twelve patients did not undergo liver biopsy, and the biopsy specimens from an additional five patients could not be interpreted. These 17 patients were therefore judged unclassifiable and were not assigned to classes. The 39 patients with neither hepatomegaly nor portal fibrosis were assigned to class 1, and the 24 patients with both hepatomegaly and portal fibrosis were assigned to class 3. The 36 patients with only one of the risk factors were assigned to class 2. Further categorizing the patients in class 2 on the basis of the severity of portal fibrosis did not reveal a subcategory useful for predicting survival. Table 3Table 3
Characteristics of Patients According to Class. shows the characteristics of patients in each class.Figures 2Figure 2
Probabilities of Survival after Transplantation in 99 Patients with Thalassemia. and 3Figure 3
Probabilities of Event-free Survival after Transplantation in 99 Patients with Thalassemia. show the probabilities of survival and event-free survival for these three classes. The three-year probabilities of survival, event-free survival, and rejection for the patients in class 1 were 94, 94, and 0 percent, respectively. For the patients in class 2, they were 80, 77, and 9 percent, and for those in class 3, they were 61, 53, and 16 percent. The probabilities for patients in class 1 were significantly different from those for class 2 or class 3 patients. Table 4Table 4
Mortality in Patients on the Current Regimen. shows the cause of death in all the patients treated with the current regimen classified according to the presence or absence of hepatomegaly and portal fibrosis.All 37 surviving patients in class 1 had Karnofsky scores of 100, and 2 had mild chronic graft-versus-host disease. Of the 29 survivors in class 2, 28 had Karnofsky scores of 100, and the other had a score of 90 (the score of 90 was given to patients whose pretransplantation thalassemia returned after the graft had been rejected). Three patients in class 2 had evidence of chronic graft-versus-host disease, mild in one patient and moderate in two. Twelve of the class 3 survivors had Karnofsky scores of 100, two had scores of 90, and one had a score of 70. Six of these patients had evidence of chronic graft-versus-host disease, which was mild in three, moderate in two, and severe in one.
Discussion
At the time of transplantation, all the patients in this study had complications due to thalassemia, transfusion therapy, or both. These complications varied in pattern and degree and were mainly related to liver damage. The specific toxic effects associated with allogeneic marrow transplantation interacted with the preexisting complications to influence the outcome of treatment. Any attempt to assess the influence of disease-related complications can be simplified by examining patients who received the same treatment regimen. Moreover, factors associated with the success of the current regimen are more useful than those associated with the success of discarded regimens.
The outcome in 116 patients who underwent transplantation with the current preparative regimen was examined to identify pretransplantation characteristics that affect survival, event-free survival, and rejection. The effectiveness with which chelation therapy had been delivered, the degree of hepatomegaly, and the detection of portal fibrosis on liver biopsy were factors significantly associated with survival and event-free survival. The quality of chelation therapy and the degree of hepatomegaly were closely associated. In the multivariate analysis, quality of chelation was removed from the model when hepatomegaly was entered. Forcing quality of chelation into the model before hepatomegaly had the reverse effect. It could not be concluded that either variable was independently more influential. The nature of the interplay between these factors and the success of marrow transplantation is unknown. The most likely explanation is that good chelation therapy improves survival after transplantation by reducing the prevalence of liver damage, but other explanations are possible. For example, both liver damage and poor chelation therapy may be consequences of poor medical care or economic circumstances.
We used the degree of hepatomegaly and the presence of portal fibrosis to divide patients into three classes according to the absence of both risk factors (class 1), the presence of one factor (class 2), or the presence of both (class 3). The patients in class 1 had better probabilities of survival and event-free survival than the patients in classes 2 and 3, and the difference was statistically significant. The patients in class 3 had lower probabilities of survival and event-free survival than those in class 2, although the difference was not statistically significant. Event-free survival was 94 percent in class 1, 77 percent in class 2, and 53 percent in class 3.
Although there was a significant increase in the median age from class 1 to class 3, age was not a significantly influential variable affecting survival and event-free survival in the univariate and multivariate analyses. It is likely that as liver damage increases, the proportion of patients who are good risks for transplantation will decrease with increasing age, but it is not clear how this should affect treatment strategies.
This study did not attempt to evaluate the role of chelation therapy in the treatment of thalassemia. However, there was a clear association between the inconsistent delivery of chelation therapy and the existence of liver damage before transplantation. Moreover, it is clear that in patients who respond well to chelation therapy and who have acceptable marrow donors, the probability of a cure through transplantation is very high and the risk of complications very low. The hazards of transplantation are greater in patients who have liver damage (possibly as a consequence of ineffective chelation therapy). Unfortunately, data on survival in such patients when they are given conventional treatment are not available, and comparing the results of transplantation with those of continued conventional therapy is not possible.
The aim of marrow transplantation in patients with thalassemia is to improve survival by reducing both morbidity and mortality. Disease-free survival is a desirable objective that cannot be obtained with other therapy. However, it is possible that survival in patients who were good risks for transplantation and who were receiving good chelation therapy would have been no worse if they had not received transplants. If this possibility is demonstrated, the issues of morbidity, quality of life, and cost will need to be addressed in a comprehensive study.
It is important to move the debate on marrow transplantation in the treatment of thalassemia from confrontation between transplantation and conventional therapy to the design of the best strategy for using both approaches.
Our study suggests topics for future efforts that may provide information useful in designing such a strategy. Longitudinal studies of the development of hepatomegaly and portal fibrosis in patients receiving transfusion and chelation therapy are needed. If these complications are found to develop slowly, patients can be carefully monitored and can undergo transplantation at the first sign of progression. Until this information is available, we believe that patients with thalassemia who have acceptable donors should receive transplants as soon as possible. The results of this study should be helpful in counseling families about the results they can expect from marrow transplantation.
Supported by a grant (1987) from the Regione Marche, Ancona; the Berloni Foundation against Thalassemia, Pesaro; the Italian Association against Leukemia, Pesaro Section; and the Italian Association against Cancer, Milan, all in Italy.
Source Information
From the Division of Hematology and the Center for Bone Marrow Transplantation of Muraglia (G.L., M.G., P.P., E.A., D.B., C.G., P.P., S.M.T.D.), the Service of Anatomical Pathology (P.M.), and the Data Processing Services (F.A.), Ospedale di Pesaro, Pesaro, Italy. Address reprint requests to Dr. Lucarelli at the Divisione Ematologica, Ospedale di Pesaro, 61100 Pesaro, Italy.
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