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Thalassaemia

Haematopoietic stem cell transplantation for thalassaemia major in Hong Kong: prognostic factors and outcome

Bone Marrow Transplantation volume 29, pages 101–105 (2002)Cite this article

Abstract

From August 1992 to August 1999, 44 patients received allogeneic haematopoietic stem cell transplantation in a single institution. The donors were HLA-identical siblings except for one who was a phenotypically matched father. Thirty-eight patients received bone marrow stem cells and the others received peripheral blood stem cells or umbilical cord blood (UCB). The mean age at transplant was 10.7 ± 5.1 years, ranging from 1.8 to 21 years. Patients received busulphan (16 mg/kg) and cyclophosphamide (150 to 200 mg/kg) as conditioning, and antithymocyte globulin was given to 42 patients to prevent graft rejection. All had engraftment except a patient who received a UCB transplant. Four patients died from early treatment-related mortality, and one died from interstitial pneumonitis 3 months after transplant. Two patients developed secondary graft rejection and both received a second transplant. Thirty-eight patients survived and all except one were transfusion independent. The 5-year overall and event-free survival rates were 86% and 82%, respectively. By multivariate stepwise Cox proportional hazard analyses, severe veno-occlusive disease (VOD) of liver and Pesaro class 3 features were the significant factors associated with survival. Patients aged more than 11 years were more inclined to develop VOD. In conclusion, haematopoietic stem cell transplantation should be performed early if an HLA identical sibling is available.

Bone Marrow Transplantation (2002) 29, 101–105. doi:10.1038/sj.bmt.1703340

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Acknowledgements

This work was supported in part by The Hong Kong Paediatric Bone Marrow Transplant Fund of the Chinese University of Hong Kong. We thank the nursing staff for providing dedicated care to the BMT patients.

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Authors and Affiliations

  1. Department of Paediatrics, Prince of Wales Hospital, The Chinese University of Hong Kong, Hong Kong

    CK Li, MMK Shing, KW Chik, V Lee, TF Leung & MP Yuen

  2. Centre for Clinical Trials and Epidemiological Research, Prince of Wales Hospital, The Chinese Universtiy of Hong Kong, Hong Kong

    AYK Cheung

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  1. CK Li
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Li, C., Shing, M., Chik, K. et al. Haematopoietic stem cell transplantation for thalassaemia major in Hong Kong: prognostic factors and outcome. Bone Marrow Transplant 29, 101–105 (2002). https://doi.org/10.1038/sj.bmt.1703340

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