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

AI Article Synopsis

  • HLA-mismatched unrelated donors can provide a solution for patients with β-thalassemia major (TM) and sickle cell disease (SCD) when HLA-matched donors are not available, despite historically higher transplant complications.
  • A study compared outcomes of pediatric TM and SCD patients receiving hematopoietic stem cell transplantation (HSCT) from mismatched donors using a specific conditioning regimen (treosulfan, ATG, and PT-CY) to those receiving matched donors without PT-CY.
  • Results showed that the mismatched donor group did not have higher event-free survival (EFS) or graft-versus-host disease (GvHD) rates compared to matched donors, indicating this method is

Article Abstract

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.

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Source
http://dx.doi.org/10.1038/s41409-021-01302-0DOI Listing

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