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Bone marrow transplant cure for β-thalassaemia major: initial experience from a developing country

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Abstract

Between July 2001 and June 2007, 48 consecutive patients with β-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.

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Acknowledgment

We thank the Departments of Microbiology, Virology and Molecular Biology of the Armed Forces Institute of Pathology, Rawalpindi, Pakistan, for helpful contributions, technical assistance and discussions. We also thank the Armed Forces Institute of Transfusion, Rawalpindi, Pakistan, for generously providing us with blood products. We are extremely grateful to Mr. Haroon Rafique of this centre for helping us in statistical analysis and manuscript preparation.

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Authors and Affiliations

  1. Department of Haematology, Armed Forces Bone Marrow Transplant Centre, Rawalpindi, Punjab, 46000, Pakistan

    Khalil Ullah, Badshah Khan, Shahid Raza, Parvez Ahmed, Tariq M. Satti, Tariq Butt, Waheed Z. Tariq & Muhammad K. Kamal

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  1. Khalil Ullah
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  2. Badshah Khan
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  3. Shahid Raza
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  4. Parvez Ahmed
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  5. Tariq M. Satti
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  7. Waheed Z. Tariq
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  8. Muhammad K. Kamal
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Correspondence to Khalil Ullah.

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Ullah, K., Khan, B., Raza, S. et al. Bone marrow transplant cure for β-thalassaemia major: initial experience from a developing country. Ann Hematol 87, 655–661 (2008). https://doi.org/10.1007/s00277-008-0478-8

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  • DOI: https://doi.org/10.1007/s00277-008-0478-8

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