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Successful HLA haploidentical HSCT with post-transplant cyclophosphamide in Wiskott–Aldrich syndrome

Bone Marrow Transplantation volume 52, pages 913–914 (2017)Cite this article

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Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for Wiskott–Aldrich syndrome (WAS). The survival rates of transplanted WAS patients using a matched related (MRD) or unrelated donor (MUD) range from 80 to 90%. Without HSCT, the median life expectancy is 15 years.1 However, the probability of finding a MRD or MUD is only 30 to 60%. Therefore, an alternative related, haploidentical donor should be considered for the patient who lacks a suitable matched donor. From the previous report, haploidentical HSCT in WAS patients yielded an unfavorable outcome.1 Currently, a haploidentical HSCT (haplo-HSCT) protocol using post-transplant cyclophosphamide (PT-Cy) for GvHD prophylaxis provides a more favorable outcome in patients with malignant and nonmalignant diseases.2 To the best of our knowledge, we herein report the first successful haplo-HSCT using PT-Cy in a WAS patient.

The patient was the first male term infant of a non-consanguineous family. He presented with generalized petechiae and intracranial hemorrhage. Initial investigation revealed a platelet count of 16.0 × 109/L and mean platelet volume of 6.6 fL. His thrombocytopenia required platelet transfusions at least twice a week. At the age of 2 months, he developed generalized eczema. He also had viral pneumonia and viral meningitis when 3 months old. Subsequently, he developed autoimmune hemolytic anemia and was treated with prednisolone. His peripheral blood examination revealed absolute lymphocyte counts as follows: CD3+ of 552/mm3, CD4+ of 470/mm3, CD8+ of 60/mm3, CD19+ of 650/mm3 and CD16+/56+ of 210/mm3. T-cell proliferation in response to phytohemagglutinin mitogen stimulation was normal. Ig levels were within normal range for his age. A WAS gene mutation at exon 10 (c.1093delG) was detected.

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Acknowledgements

This work has been supported by Ramathibodi Foundation and Mahidol University Research Grant.

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Authors and Affiliations

  1. Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

    P Kreetapirom, S Hongeng, W Manuyakorn, U Anurathapan, S Pakakasama & N Sirachainan

  2. Departments of Stem Cell Transplantation and Cellular Therapy, University of Texas MD Anderson Cancer Center, Houston, TX, USA

    B S Andersson

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  1. P Kreetapirom
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  2. S Hongeng
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  3. W Manuyakorn
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  4. U Anurathapan
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  5. S Pakakasama
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  6. N Sirachainan
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  7. B S Andersson
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Correspondence to S Hongeng or W Manuyakorn.

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Kreetapirom, P., Hongeng, S., Manuyakorn, W. et al. Successful HLA haploidentical HSCT with post-transplant cyclophosphamide in Wiskott–Aldrich syndrome. Bone Marrow Transplant 52, 913–914 (2017). https://doi.org/10.1038/bmt.2017.25

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