Lum, Su Han and Eikema, Dirk-Jan and Piepenbroek, Brian and Wynn, Robert F. and Samarasinghe, Sujith and Dalissier, Arnaud and Kalwak, Krysztof and Ayas, Mouhab and Hamladji, Rose-Marie and Yesilipek, Akif and Dalle, Jean-Hugues and Uckan-Cetinkaya, Duygu and Bierings, Marc and Kupesiz, Alphan and Halahleh, Khalid and Skorobogatova, Elena and Ozturk, Gulyuz and Faraci, Maura and Renard, Cecile and Evans, Pamela and Corbacioglu, Selim and Locatelli, Franco and Dufour, Carlo and Risitano, Antonio and de Latour, Regis Peffault (2024) Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia. BLOOD, 144 (12). pp. 1329-1342. ISSN 0006-4971, 1528-0020
Full text not available from this repository. (Request a copy)Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only established curative option for Fanconi anemia (FA)-associated - associated bone marrow failure (BMF)/aplastic anemia (AA) and acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS). We performed a retrospective multicenter study on 813 children with FA undergoing fi rst HSCT between 2010 and 2018. Median duration of follow-up was 3.7 years. Median age at transplant was 8.8 years (IQR, 6.5-18.1). Five-year overall survival (OS), event-free survival (EFS), and graft-versus-host disease (GVHD)-free, relapse-free survival (GRFS) were 83% (95% confi dence interval [CI], 80-86), 78% (95% CI, 75-81), and 70% (95% CI, 67-74), respectively. OS was comparable between matched family donor (MFD; n = 441, 88%) and matched unrelated donor (MUD; n = 162, 86%) and was superior to that of mismatched family donor (MMFD) or mismatched unrelated donor (MMUD; n = 144, 72%) and haploidentical donor (HID; n = 66, 70%; P < .001). In multivariable analysis, a transplant indication of AML/MDS (vs AA/BMF), use of MMFD/MMUD and HID (vs MFD), and fl udarabine-cyclophosphamide (FluCy) plus other conditioning (vs FluCy) independently predicted inferior OS, whereas alemtuzumab vs antithymocyte globulin was associated with better OS. Age >= 10 years was associated with worse EFS and GRFS. Cumulative incidences (CINs) of primary and secondary graft failure were 2% and 3% respectively. CINs of grade 3 to 4 acute GVHD and chronic GVHD were 12% and 8% respectively. The 5-year CIN of secondary malignancy was 2%. These data suggest that HSCT should be offered to patients with FA with AA/BMF at a younger age in the presence of a well-matched donor.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | BONE-MARROW-TRANSPLANTATION; T-CELL; CONDITIONING REGIMEN; EUROPEAN GROUP; ACUTE-LEUKEMIA; CHILDREN; CYCLOPHOSPHAMIDE; FLUDARABINE; BLOOD; IRRADIATION |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 21 Oct 2025 05:06 |
| Last Modified: | 21 Oct 2025 05:06 |
| URI: | https://pred.uni-regensburg.de/id/eprint/65603 |
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