The effect of intensity of conditioning regimen on the outcome of HSCT in children with sickle cell disease

Background Allogeneic hematopoietic stem cell transplantation (HSCT) provides a cure for patients with sickle cell disease (SCD). This study describes the effect of conditioning regimen intensity on HSCT outcomes among children younger than 14 years with SCD. Methods Transplants from HLA‐matched rel...

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Bibliographic Details
Published in:Clinical transplantation 2022-11, Vol.36 (11), p.e14787-n/a
Main Authors: Alsultan, Abdulrahman, Abujoub, Rodina, Elbashir, Enas, Essa, Mohammed F.
Format: Article
Language:English
Subjects:
Adolescent
Anemia, Sickle Cell - etiology
Anemia, Sickle Cell - therapy
Child
Child, Preschool
children
conditioning
Graft vs Host Disease - etiology
Hematopoietic Stem Cell Transplantation - adverse effects
HSCT
Humans
Retrospective Studies
Saudi Arabia
sickle cell disease
Transplantation Conditioning - adverse effects
Unrelated Donors
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Summary:Background Allogeneic hematopoietic stem cell transplantation (HSCT) provides a cure for patients with sickle cell disease (SCD). This study describes the effect of conditioning regimen intensity on HSCT outcomes among children younger than 14 years with SCD. Methods Transplants from HLA‐matched related donors (MRD) and unrelated donors (MUD) using either myeloablative conditioning (MAC) regimens or reduced intensity conditioning (RIC) regimens were considered. Event‐free survival (EFS) was the primary endpoint. Secondary endpoints included overall survival (OS) and occurrence of GVHD. Results 48 SCD patients underwent HSCT, 45 (93.8%) patients had MRD, 1 (2.1%) had 9/10 related donor, and 2 (4.1%) had MUD. The median age at transplant was 8.6 years (range, 3.1–13.8). Conditioning regimens were myeloablative (MAC) in 41 (85.4%) patients and of reduced intensity in 7 (14.6%) patients. EFS at 2 years was 100% among MAC group compared to 29% in the RIC group (p 
ISSN:0902-0063
1399-0012
DOI:10.1111/ctr.14787