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Results from a multicenter, retrospective study evaluating the efficacy of mesenchymal stromal cells (MSCs) in pediatric patients (aged ≤18 years; N = 102) with steroid-refractory graft-versus-host disease (SR-GvHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) were recently published in Bone Marrow Transplantation by Echecopar et al.
Key data: The 28-day overall response rate (ORR) was 76.5% (complete response [CR], 30.4%; partial response [PR], 46.1%). Patients receiving <1 × 10⁶ cells/kg per infusion had a 2-year overall survival (OS) of 22.6% compared with 59.8% for patients receiving ≥1 × 10⁶ cells/kg (p < 0.001). The only treatment-related complication was sepsis, an isolated case attributed to batch contamination; the patient recovered fully and responded to MSC therapy.
Key learning: MSC therapy demonstrated high response rates in pediatric SR-GvHD. Prospective studies are required to validate results to help standardize MSC production and administration in pediatric GvHD.
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