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Nonmyeloablative stem cell transplantation with a haploidentical donor in a class 3 lucarelli severe thalassemia patient

Bone Marrow Transplantation volume 34, pages 271–272 (2004)Cite this article

Thalassemia is a chronic hemolytic disease of genetic origin. The burden of the disease is enormous, disrupting growth and quality of life. Although adequate blood transfusion and iron chelation early in life can minimize morbidities, this treatment modality is only palliative and has many problems. The ultimate goal should be aimed at cure; at present, this is achievable with allogeneic bone marrow transplantation (BMT) but feasibility is limited by scarcity of matched sibling donors. Preliminary results of allogeneic BMT with matched sibling donors in thalassemia patients revealed that children in class 3 Lucarelli had the worst outcome in terms of survival, disease-free survival, and rejection of 0.61, 0.53, and 0.16, respectively. These unsatisfactory results were largely due to a major increase in nonrejection mortality.1 Andreani et al2 reported that persistent mixed chimerism can be achieved and can accomplish sufficient hemoglobin levels in post-transplant thalassemia. This should make nonmyeloablative stem cell transplantation (NST) an interesting approach for thalassemia in order to overcome nonrejection mortality among class 3 patients.3 Indeed, we have already reported a successful NST in a class 3 Lucarelli severe thalassemia patient.4 However, the limited availability of matched sibling donors requires that alternative sources of donors be explored. We, therefore, investigated one approach with haploidentical donor in this group of patients. This approach has not been published before. The study was approved by the Ethics Committee of the Faculty of Medicine, Ramathibodi Hospital, Mahidol University and the patient consented to this procedure.

An 18-year-old girl with homozygous β-thalassemia had 97% haemoglobin F at age 7 months. She has received monthly blood transfusion since 7 months of age (a total of 200 times) and chelation therapy with desferrioxamine since 16 years of age. Prior to each blood transfusion, her hemoglobin level was 5–6 gm/dl. At the age of 7 years, she underwent splenectomy because of massive splenomegaly. She also had hepatomegaly. Prior to transplantation, the patient received a thorough physical examination and evaluation. Her serum ferritin was 3860 ng/ml. Biopsy of the liver revealed moderate to severe hepatic fibrosis and iron over load in the liver as defined by Sciot.5 The extents of hepatomegaly, portal fibrosis, and iron overload in the liver put her into the class 3 risk category as proposed by Lucarelli et al.1

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References

  1. Lucarelli G, Galimberti M, Polchi P et al. Bone marrow transplantation in patients with thalassemia. N Engl JMed 1990; 322: 417–421.

    Article  CAS  Google Scholar 

  2. 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.

    CAS  Google Scholar 

  3. Champlin R, Khouri I, Kornblau S et al. Reinventing bone marrow transplantation: reducing toxicity using nonmyeloablative, preparative regimens and induction of graft-versus-malignancy. Curr Opin Oncol 1999; 11: 87–95 (Transplantation 2000; 6: 309–320).

    Article  CAS  Google Scholar 

  4. Hongeng S, Chuansumrit A, Hathirat P et al. Full chimerism in nonmyeloablative stem cell transplantation in a β-thalassemia major patient (class 3 Lucarelli). Bone Marrow Transplant 2003; 30: 409–410.

    Article  Google Scholar 

  5. Muretto P, Angelucci E, Del Fiasco S, Lucarelli G . Reversal features of hepatic haemosiderosis and haemochromatosis in thalassemia after bone marrow transplantation. Prog Clin Biol Res 1989; 309: 299–314.

    CAS  PubMed  Google Scholar 

  6. Krishnamurti L, Blazar BR, Wagner JE . Bone marrow transplantation without myeloablation for sickle cell disease. N Engl J Med 2001; 344: 68.

    Article  CAS  Google Scholar 

  7. Gaziev D, Polchi P, Lucarelli G et al. Second marrow transplant for graft failure in patients with thalassemia. Bone Marrow Transplant 1999; 24: 1299–1306.

    Article  CAS  Google Scholar 

  8. Xun CQ, Thompson JS, Jennings CD et al. Effect of total body irradiation, busulfan-cyclophosphamide, or cyclophosphamide conditioning on inflammatory cytokine release and development of acute and chronic graft-versus-host disease in H-2-incompatible transplanted SCID mice. Blood 1994; 83: 2360–2367.

    CAS  Google Scholar 

  9. Aversa F, Tabilio A, Velardi A et al. Treatment of high-risk leukemia with T-cell-depleted stem cells from related donors with one fully mismatched HLA haplotype. N Engl J Med 1998; 339: 1186–1193.

    Article  CAS  Google Scholar 

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Authors and Affiliations

  1. Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Bangkok, Thailand

    S Hongeng & S Pakakasama

  2. Department of Internal Medicine, Faculty of Medicine, Ramathibodi Hospital, Bangkok, Thailand

    A Ungkanont & S Jootar

  3. Bone Marrow Transplant Program, Faculty of Medicine, Ramathibodi Hospital, Bangkok, Thailand

    S Hongeng, S Pakakasama, W Chaisiripoomkere, A Ungkanont & S Jootar

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  1. S Hongeng
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Hongeng, S., Pakakasama, S., Chaisiripoomkere, W. et al. Nonmyeloablative stem cell transplantation with a haploidentical donor in a class 3 lucarelli severe thalassemia patient. Bone Marrow Transplant 34, 271–272 (2004). https://doi.org/10.1038/sj.bmt.1704572

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