Impact of minimal residual disease on the outcome of hematopoietic stem cell transplantation for childhood acute lymphoblastic leukemia within the FORUM trial.

ABSTRACT: In the randomized cohort of the international phase-III FORUM trial, which showed the superiority of total-body irradiation (TBI) over chemotherapy-based conditioning prior to hematopoietic stem cell transplantation (HSCT) for pediatric acute lymphoblastic leukemia (ALL), type of conditioning and remission phase, but not pre-HSCT minimal residual disease (MRD), were associated with outcome. We report the impact of MRD within the extended FORUM cohort. Patients (N=1,014) aged 4-21 years old, transplanted from a matched donor who had ≥1 MRD measurement prior to and/or 100 days and/or one year after HSCT were eligible. A threshold of 0.01% defined MRD positivity versus negativity. Prior to HSCT, 21% of patients were MRDpos. Three-year event-free survival (EFS) was 0.73 and 0.59 (P<0.001), and

SUBMITTER: Balduzzi A 

PROVIDER: S-EPMC12775767 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature