Risk adopted allogeneic hematopoietic stem cell transplantation using a reduced intensity regimen for children with thalassemia major.

Hussein AA, Al-Zaben A, Ghatasheh L, Natsheh A, Hammada T, Abdel-Rahman F, Abu-Jazar H, Sharma S, Najjar R, Frangoul H
Pediatr Blood Cancer. 2013 60 (8): 1345-9

PMID: 23424175 · DOI:10.1002/pbc.24493

BACKGROUND - Patients with thalassemia in developing countries have limited access to safe transfusions, regular medical care and chelation therapy. Although allogeneic hematopoietic stem cell transplantation (HSCT) can offer a curative approach, there are limited data on the use of this procedure in developing countries.

PROCEDURE - Forty-four patients underwent a risk adopted HSCT from matched related family donor in Jordan. Thirty-one patients (7 Class 1 and 24 Class 2) underwent myeloablative conditioning (MAC) with busulfan (16 mg/kg), cyclophosphamide (200 mg/kg) and antithymocyte globulin (ATG). Thirteen patients all with Class 3, seven with hepatitis C received reduced intensity conditioning (RIC) with busulfan (8 mg/kg), fludarabine (175 mg/m(2)), total lymphoid irradiation (500 cGy) and ATG.

RESULTS - All patients had initial neutrophil and platelet engraftment. Secondary graft failure was observed in 2 (6%) patients receiving myeloablative HSCT and 3 (23%) patients receiving RIC. At a median follow up of 64 months (13-108), 43 of 44 patients are alive. The 5-year probability of overall survival (OS) was 97.8% for all patients, 96.8% for patients received MAC and 100% for patients received RIC. The 5-year probability of thalassemia-free survival was 86.4% for all patients, 90.3% and 77% for patients who received MAC and RIC, respectively.

CONCLUSION - Implementing a risk-adopted therapy in patient with thalassemia in Jordan can result in an excellent thalassemia free and OS, especially in those at highest risk.

Copyright © 2013 Wiley Periodicals, Inc.

MeSH Terms (26)

Adolescent Adult Antilymphocyte Serum beta-Thalassemia Busulfan Child Child, Preschool Cyclophosphamide Disease-Free Survival Female Follow-Up Studies Graft Rejection Graft Survival Hematopoietic Stem Cell Transplantation Humans Immunologic Factors Infant Male Myeloablative Agonists Retrospective Studies Risk Factors Survival Rate Tissue Donors Transplantation, Homologous Transplantation Conditioning Vidarabine

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