Successful mismatched hematopoietic stem cell transplantation for pediatric hemoglobinopathy by using ATG and post-transplant cyclophosphamide

Autor: Anja M. Jansen-Hoogendijk, Astrid G. S. van Halteren, Robbert G. M. Bredius, Maarten J. D. van Tol, Alexander B. Mohseny, Lisa V E Oostenbrink, Emma S. Pool, Arjan C. Lankester, Cornelia M. Jol-van der Zijde, Frans J. Smiers, Carly Vervat, Marco W. Schilham
Rok vydání: 2021
Předmět:
Zdroj: Bone Marrow Transplantation. SPRINGERNATURE
Bone Marrow Transplantation
ISSN: 1476-5365
0268-3369
DOI: 10.1038/s41409-021-01302-0
Popis: The use of HLA-mismatched (un)related donors is historically associated with a higher incidence of transplant-related complications and mortality. However, the use of such donors may overcome the limited availability of HLA-matched donors for patients with beta-thalassemia major (TM) and sickle cell disease (SCD). We investigated hematopoietic stem cell transplantation (HSCT) outcomes of pediatric TM and SCD patients treated with a mismatched donor using a treosulfan-based conditioning in combination with ATG and post-transplant cyclophosphamide (PT-CY) and compared these results to the clinical outcome of patients treated by matched donor HSCT without PT-CY. Thirty-eight children (n = 24 HLA-identical or 10/10-matched donors; n = 14 HLA-mismatched donors), who received a non-depleted bone marrow graft were included. Event-free survival (EFS) and GvHD were not higher in the mismatched PT-Cy group as compared to the matched group. Moreover, despite delayed neutrophil engraftment (day +22 vs. +26, p = 0.002) and immune recovery in the mismatched PT-Cy group, this did not result in more infectious complications. Therefore, we conclude that in the absence of an HLA-identical or a matched unrelated donor, HSCT with a mismatched unrelated or haploidentical donor in combination with ATG plus PT-CY can be considered a safe and effective treatment option for pediatric hemoglobinopathy patients.
Databáze: OpenAIRE
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