title: Bone marrow transplant cure for beta-thalassaemia major: initial experience from a developing country.
abstract: Between July 2001 and June 2007, 48 consecutive patients with beta-thalassaemia major received allogeneic haematopoietic stem cell transplants (allo HSCT) from human-leukocyte-antigen-matched siblings at the Armed Forces Bone Marrow Transplant Centre, Rawalpindi, Pakistan, using standard conditioning regimens. The median age of the patient cohort was 4 years (range, 1-14 years). Thirty-one patients were in risk class I, 11 in class II and six patients were in class III. Engraftment was achieved in all patients. Survival was calculated from the date of transplant to death or last follow-up. Major post-transplant complications encountered were acute graft versus host disease (Ac GvHD) (grades II-IV), 35.4%; chronic GvHD, 8.3%; haemorrhagic cystitis, 12.5%; veno-occlusive disease (VOD) of the liver, 6.2%; bacterial infections, 37.5%; fungal infections, 19%; cytomegalovirus (CMV) infection, 6.2%; herpes infection, 6.2%; and tuberculosis in 2% of patients. Graft rejection was observed in five patients. Three patients received second transplants. Mortality was observed in 20.8% of patients. Major fatal complications included GvHD, VOD, intracranial haemorrhage, septicaema, CMV disease and disseminated tuberculosis. Overall survival and disease-free survival were 79% and 75%, respectively, at 6 years post-HSCT.
authors.affiliation: Department of Haematology, Armed Forces Bone Marrow Transplant Centre, Rawalpindi, Punjab, Pakistan. dr_khalil58@yahoo.com
authors.forename: Khalil
authors.initials: K
authors.lastname: Ullah
authors.forename: Badshah
authors.initials: B
authors.lastname: Khan
authors.forename: Shahid
authors.initials: S
authors.lastname: Raza
authors.forename: Parvez
authors.initials: P
authors.lastname: Ahmed
authors.forename: Tariq M
authors.initials: TM
authors.lastname: Satti
authors.forename: Tariq
authors.initials: T
authors.lastname: Butt
authors.forename: Waheed Z
authors.initials: WZ
authors.lastname: Tariq
authors.forename: Muhammad K
authors.initials: MK
authors.lastname: Kamal
mesh_terms: D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003906:Developing Countries; D018572:Disease-Free Survival; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D007223:Infant; D008297:Male; D010154:Pakistan; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D017086:beta-Thalassemia / Q000628:therapy*