Premium
Risk adopted allogeneic hematopoietic stem cell transplantation using a reduced intensity regimen for children with thalassemia major
Author(s) -
Hussein Ayad Ahmed,
AlZaben Abdulhadi,
Ghatasheh Lubna,
Natsheh Abeer,
Hammada Tuka,
AbdelRahman Fawzi,
AbuJazar Husam,
Sharma Shanta,
Najjar Rula,
Frangoul Haydar
Publication year - 2013
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.24493
Subject(s) - medicine , busulfan , thalassemia , fludarabine , cyclophosphamide , hematopoietic stem cell transplantation , regimen , surgery , transplantation , gastroenterology , chemotherapy
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. Pediatr Blood Cancer 2013;601345‐1349. © 2013 Wiley Periodicals, Inc.