Unmanipulated haploidentical hematopoietic stem cell transplantation for children with myelodysplastic syndrome

Abstract Myelodysplastic syndrome (MDS) is a heterogeneous group of clonal disorders and is rare in children. Allogeneic hematopoietic stem cell transplantation (HSCT) is commonly used in children with MDS with excess blasts and in patients with refractory cytopenia of childhood (RCC) associated with monosomy 7, complex karyotype, severe neutropenia, or transfusion dependence. We recruited 27 children with MDS who received haploidentical hematopoietic stem cell transplantation (haplo‐HSCT). At transplantation, 10 patients had RCC, 12 patients had advanced MDS (RAEB and RAEB‐T), and 5 patients had myelodysplasia‐related acute myeloid leukemia (MDR‐AML). All patients received granulocyte colony‐stimulating factor (G‐CSF)–mobilized bone marrow cells and peripheral blood stem cells. At a median follow‐up of 24.1 months (range: 2.0‐74.5 months) after HSCT, the estimated probabilities of 3‐year disease‐free survival (DFS) and overall survival (OS) were both 81.9% (95% CI, 66.8‐100.0%). The estimated 3‐year incidences of relapse (CIR) and non‐relapse mortality (NRM) were both 7.4% (95% CI, 1.2%‐21.4%). The 100‐day cumulative incidence of grade II–IV aGVHD was 52.6% (95% CI, 42.9‐62.3%), while that of grade III–IV aGVHD was 11.1% (95% CI, 5.1‐17.1%). The 3‐year cumulative incidences of overall and extensive cGVHD were 42.3% (95% CI, 19.8%‐57.5%) and 21.1% (95% CI, 2.5%‐63.2%), respectively. Univariate analysis showed that chronic GVHD significantly affected OS and DFS. Haploidentical HSCT may be an effective treatment option with easier donor availability for pediatric patients with MDS.