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

Lisa V.E. Oostenbrink, Emma S. Pool, Cornelia M. Jol-van der Zijde, Anja M. Jansen-Hoogendijk, Carly Vervat, Astrid G.S. van Halteren, Robbert G.M. Bredius, Frans J.W. Smiers, Maarten J.D. van Tol, Marco W. Schilham, Arjan C. Lankester, Alexander B. Mohseny

Onderzoeksoutput: Bijdrage aan tijdschriftArtikelpeer review

6 Citaten (Scopus)

Samenvatting

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 β-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.

Originele taal-2Engels
Pagina's (van-tot)2203-2211
Aantal pagina's9
TijdschriftBone Marrow Transplantation
Volume56
Nummer van het tijdschrift9
DOI's
StatusGepubliceerd - sep. 2021
Extern gepubliceerdJa

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