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Cord Blood Stem Cells

Unrelated cord blood transplantation for thalassaemia: a single-institution experience of 35 patients

Bone Marrow Transplantation volume 47, pages 33–39 (2012)Cite this article

Abstract

Our study was designed to prospectively determine whether or not unrelated cord blood transplantation (CBT) can produce outcomes comparable to related donor transplantation for children with β-thalassaemia. In 35 patients, 40 transplants were performed between October 2003 and September 2009. HLA matching at enrolment was 6/6 (n=8), 5/6 (n=16), 4/6 (n=27), or 3/6 (n=1) by low-resolution HLA-A, -B, and high-resolution DRB1. These patients received non-manipulated grafts without ex vivo expansion or T-cell depletion. The median number of nucleated and CD34+ cells infused was 7.8 × 107/kg (range, 2.8–14.7 × 107/kg) and 4.0 × 105/kg (range, 1.7–19.9 × 105/kg), respectively. The 5-year OS and thalassaemia-free survival after the first transplant were 88.3 and 73.9%, respectively. The cumulative incidence of TRM at 2 years was 11.7%. Fourteen patients developed chronic skin GVHD. Thirty patients were alive and transfusion-independent with a Lansky performance score ⩾80% achieved between 6 and 76 months post transplant (median, 36 months). These data compare acceptably with the survival rates of related-donor BMT for thalassaemia and suggest that patients without an available HLA-compatible sibling but who have well-matched unrelated donors should also be considered for CBT.

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Acknowledgements

We are indebted to the patients and their carers for participating in the externally audited results. The authors acknowledge and thank the Department of Health, Executive Yuan in Taiwan, for helping to conduct and supervise the clinical trial of cord blood transplantation. This work was supported by grant CMRPG4A0031 from the Chang Gung Medical Research Program.

The material contained in this paper has been presented at the following meetings:

1. Jaing TH, Wang B, Gjertson D, Law P, Petz L, Chow R. Unrelated Cord Blood Transplantation (UCBT) for Transfusion-Dependent Thalassemia a CIBMTR Audited Retrospective Analysis of 30 Consecutive Patients from a Single Center. Session Type: Oral Session, Board #131. 50th ASH annual meeting; San Francisco, CA, USA; 6–9 December 2008.

2. Jaing TH, Wang B, Gjertson D, Law P, Petz L, Chow R. Unrelated Cord Blood Transplantation (UCBT) for Transfusion-Dependent Thalassemia a CIBMTR Audited Retrospective Analysis of 30 Consecutive Patients from a Single Center. CIBMTR 2009 BMT Tandem Meeting; Tampa, Florida, USA; 11–15 February 2009.

3. Jaing TH. Transplantation of Patients with Transfusion-dependent Thalassemia. 8th Annual International Cord Blood Transplantation Symposium: San Francisco, CA, USA; 3–5 June 2010.

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Authors and Affiliations

  1. Division of Hematology/Oncology, Department of Pediatrics, Chang Gung Children's Hospital, Chang Gung University, Taoyuan, Taiwan

    T-H Jaing, I-J Hung, C-P Yang & S-H Chen

  2. Division of Cardiology, Department of Pediatrics, Chang Gung Children's Hospital, Chang Gung University, Taoyuan, Taiwan

    H-T Chung

  3. Department of Public Health and Center of Biostatistics, College of Medicine, Chang Gung University, Taoyuan, Taiwan

    P-K Tsay

  4. Department of Nursing, Chang Gung Memorial Hospital, Taoyuan, Taiwan

    Y-C Wen

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Correspondence to T-H Jaing.

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Jaing, TH., Hung, IJ., Yang, CP. et al. Unrelated cord blood transplantation for thalassaemia: a single-institution experience of 35 patients. Bone Marrow Transplant 47, 33–39 (2012). https://doi.org/10.1038/bmt.2011.39

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