Successful mismatched hematopoietic stem cell transplantation for pediatric hemoglobinopathy by using ATG and post-transplant cyclophosphamide.
Bone Marrow Transplant
; 56(9): 2203-2211, 2021 09.
Article
en En
| MEDLINE
| ID: mdl-33941871
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 ß-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.
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
Trasplante de Células Madre Hematopoyéticas
/
Enfermedad Injerto contra Huésped
/
Hemoglobinopatías
Límite:
Child
/
Humans
Idioma:
En
Revista:
Bone Marrow Transplant
Asunto de la revista:
TRANSPLANTE
Año:
2021
Tipo del documento:
Article
País de afiliación:
Países Bajos