title: Risk adopted allogeneic hematopoietic stem cell transplantation using a reduced intensity regimen for children with thalassemia major.
abstract: 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.
  
  
  METHODS
  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.
  
  
  CONCLUSIONS
  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.
authors: Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center, Amman, Jordan. aahmed@khcc.jo
authors: Ayad Ahmed
authors: AA
authors: Hussein
authors: Abdulhadi
authors: A
authors: Al-Zaben
authors: Lubna
authors: L
authors: Ghatasheh
authors: Abeer
authors: A
authors: Natsheh
authors: Tuka
authors: T
authors: Hammada
authors: Fawzi
authors: F
authors: Abdel-Rahman
authors: Husam
authors: H
authors: Abu-Jazar
authors: Shanta
authors: S
authors: Sharma
authors: Rula
authors: R
authors: Najjar
authors: Haydar
authors: H
authors: Frangoul
mesh_terms: D000293:Adolescent; D000328:Adult; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D002066:Busulfan / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000209:etiology / Q000401:mortality / Q000628:therapy; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage; D007223:Infant; D008297:Male; D019653:Myeloablative Agonists / Q000008:administration & dosage; D012189:Retrospective Studies; D012307:Risk Factors; D015996:Survival Rate; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives; D017086:beta-Thalassemia / Q000401:mortality* / Q000628:therapy*
chemical_list: D000961:Antilymphocyte Serum; D007155:Immunologic Factors; D019653:Myeloablative Agonists; D003520:Cyclophosphamide; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine