Background: Umbilical cord blood transplantation (UCBT) serves as a valuable alternative for pediatric patients without matched donors, particularly in urgent situations where timely transplantation is critical. While UCBT offers immunological benefits and broader donor availability, its clinical outcomes remain inconsistent, especially in non-malignant hematologic disorders. Objective: To evaluate clinical outcomes of UCBT in pediatric patients with malignant and non-malignant hematologic conditions, focusing on transplant-related complications and survival rates.
Methods: In this retrospective single-center study, we analyzed 14 pediatric patients (aged 0.7–11 years) who underwent allogeneic UCBT at Mofid Children’s Hospital between 2019 and 2024. Data were extracted from institutional registries and medical records, including patient demographics, disease classification, graft characteristics, conditioning regimens, engraftment status, incidence of graft-versus-host disease (GVHD), infections, and survival outcomes.
Results: Among the 14 patients, 85.7% had non-malignant disorders. Most received unrelated donor grafts, with HLA matching of 6/6 in 42.9%, 5/6 in 50%, and 4/6 in 7.1%. The median total nucleated cell dose was 4.95×10⁷/kg, and the median CD34⁺ cell dose was 1.7×10⁵/kg. Neutrophil engraftment was achieved in 42.9% of patients, with a median of 19 days. Primary graft failure occurred in 50% of patients, and secondary graft failure in 7.1%. Acute GVHD developed in 14.3% of cases, while no chronic GVHD was observed. CMV reactivation occurred in 42.9% of patients, and bacterial infections were reported in 57.1%. The overall survival rate was 42.9%, with transplant-related mortality accounting for 57.1% of cases, due to infections and graft failure.
Conclusion: UCBT remains a feasible therapeutic option for pediatric patients lacking matched donors, particularly in urgent or resource-constrained settings. However, the high incidence of graft failure and infection-related mortality highlights the need for improved graft selection strategies, personalized conditioning protocols, and optimized post-transplant care to enhance patient outcomes.
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Original Article |
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Pediatric Hematology & Oncology Received: 2025/10/28 | Accepted: 2025/11/15 | Published: 2025/10/8