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

• Published in: Pediatric blood & cancer 2013-08, Vol.60 (8), p.1345-1349
• Main Authors: Hussein, Ayad Ahmed, Al-Zaben, Abdulhadi, Ghatasheh, Lubna, Natsheh, Abeer, Hammada, Tuka, Abdel-Rahman, Fawzi, Abu-Jazar, Husam, Sharma, Shanta, Najjar, Rula, Frangoul, Haydar
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Antilymphocyte Serum - administration & dosage; beta-Thalassemia - mortality; beta-Thalassemia - therapy; Busulfan - administration & dosage; Child; Child, Preschool; Cyclophosphamide - administration & dosage; Disease-Free Survival; Female; Follow-Up Studies; Graft Rejection - etiology; Graft Rejection - mortality; Graft Rejection - therapy; Graft Survival; Hematology; Hematopoietic Stem Cell Transplantation; Humans; Immunologic Factors - administration & dosage; Infant; Male; Myeloablative Agonists - administration & dosage; Oncology; Pediatrics; Retrospective Studies; Risk Factors; risk stratification; Survival Rate; thalassemia major; Tissue Donors; Transplantation Conditioning - methods; Transplantation, Homologous; Vidarabine - administration & dosage; Vidarabine - analogs & derivatives
• ISSN: 1545-5009; 1545-5017
• 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/m2), 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.