Correspondence

Hematopoietic-Cell Transplantation for Chronic Granulomatous Disease

N Engl J Med 2001; 345:377-378August 2, 2001

Article

To the Editor:

In his editorial (March 22 issue)1 on the treatment of chronic granulomatous disease, Ezekowitz balances the risk of bone marrow transplantation against the benefits of prophylaxis with antimicrobial agents and interferon gamma and cites a recent study from our center. We reported on an unselected group of 21 patients in whom the disease had been diagnosed at our center since 1990, all of whom were alive and well at a mean age of 4.4 years.2 Since this study was published, two of our patients have died from infectious complications of chronic granulomatous disease, despite optimal antibacterial and antifungal prophylactic therapy. These cases highlight the fact that prophylaxis cannot completely replace the protective function of NADPH oxidase.

We agree that the search for better methods of transplantation or engraftment of genetically modified stem cells is important but disagree that transplantation from an HLA-matched related donor should be reserved for older patients with recurrent complications. The emerging evidence is that transplantation from an HLA-matched donor as a treatment for immunodeficiency is most successful in younger patients in whom end-organ damage has not occurred3,4 and in whom the risk of graft-versus-host disease is lower.

Adrian Thrasher, M.B., B.S., Ph.D.
David Goldblatt, M.B., Ch.B., Ph.D.
Institute of Child Health, London WC1N 1EH, United Kingdom
a.thrasher@ich.ucl.ac.uk

4 References

1. 1

   Ezekowitz RAB. What is the best way to treat inherited disorders? N Engl J Med 2001;344:926-927
   Full Text | Web of Science | Medline

2. 2

   Cale CM, Jones AM, Goldblatt D. Follow up of patients with chronic granulomatous disease diagnosed since 1990. Clin Exp Immunol 2000;120:351-355
   CrossRef | Web of Science | Medline

3. 3

   Horwitz ME, Barrett AJ, Brown MR, et al. Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft. N Engl J Med 2001;344:881-888
   Full Text | Web of Science | Medline

4. 4

   Thrasher AJ, Kinnon C. The Wiskott-Aldrich syndrome. Clin Exp Immunol 2000;120:2-9
   CrossRef | Web of Science | Medline

Citing Articles (3)

Citing Articles

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   Manuel Grez, Janine Reichenbach, Joachim Schwäble, Reinhard Seger, Mary C Dinauer, Adrian J Thrasher. (2011) Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma. Molecular Therapy 19:1, 28-35
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   Wen-I Lee, Ming-Ling Kuo, Jing-Long Huang, Syh-Jae Lin, Cheng-Jang Wu. (2005) Akhil Kakroo. Journal of Clinical Immunology 25:2, 162-173
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