Di Majo, Benedetta Elena and Abouqateb, Mouad and Galimard, Jacques-Emmanuel and Dalissier, Arnaud and Lucchini, Giovanna and Versluys, Birgitta and Zubarovskaya, Ludmila and Mirci-Danicar, Oana and Jubert, Charlotte and Tewari, Sanjay and Wynn, Robert and Lawson, Sarah and Michel, Gerard and Balduzzi, Adriana and Hamladji, Rose-Marie and Sirvent, Anne and Al-Ahmari, Ali and Rialland, Fanny and Faraci, Maura and Ansari, Marc and Dalle, Jean-Hugues and Calvo, Charlotte and Rocha, Vanderson and Gluckman, Eliane and Kleinschmidt, Katharina and Corbacioglu, Selim and Kalwak, Krzysztof and Ruggeri, Annalisa (2025) Single unrelated umbilical cord blood versus unmanipulated haploidentical HCT using PTCy in pediatric AML: a retrospective study on behalf of the EBMT PDWP and CTIWP. BONE MARROW TRANSPLANTATION, 60 (12). pp. 1635-1641. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)Abstract
Hematopoietic stem cell transplantation (HCT) is the standard consolidation therapy for children with high-risk or relapsed acute myeloid leukemia (AML). In the absence of an human leukocyte antigen (HLA)-matched donor, both unrelated cord blood transplantation (UCBT) and haploidentical HCT with post-transplant cyclophosphamide (haplo-PTCy) serve as viable alternatives. This study analyzed outcomes of 254 pediatric AML patients (CR1/CR2) who underwent either single-unit UCBT (N = 127) without serotherapy or haplo-PTCy (N = 127) between 2011 and 2021. Propensity score weighting was applied to minimize baseline differences. With a median follow-up of 2.9 years, no significant differences were observed in overall survival, leukemia-free survival, relapse incidence, non-relapse mortality (NRM), or graft-versus-host disease (GVHD)-free relapse-free survival. However, UCBT recipients had a lower risk of chronic GVHD (7.1% vs. 19.8%; P = 0.019). These findings confirm comparable survival outcomes between UCBT and Haplo-PTCy. Given the higher risk of cGVHD in haplo-PTCy, further research is needed to refine GVHD prevention strategies while maintaining relapse-free survival. Both approaches remain safe and readily available alternatives for pediatric AML patients lacking matched donors.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | BONE-MARROW-TRANSPLANTATION; STEM-CELL TRANSPLANTATION; ACUTE LYMPHOBLASTIC-LEUKEMIA; HEMATOLOGIC MALIGNANCIES; POSTTRANSPLANT CYCLOPHOSPHAMIDE; SIBLING DONORS; CHILDREN; DIAGNOSIS; CRITERIA; TRIAL; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 26 Mar 2026 06:12 |
| Last Modified: | 26 Mar 2026 06:12 |
| URI: | https://pred.uni-regensburg.de/id/eprint/68010 |
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