TY  - JOUR
AU  - Mekni, Sabrine
AU  - Torjemane, Lamia
AU  - Khayati, Malek
AU  - Belloumi, Dorra
AU  - Kanoun, Rimmel Yosra
AU  - Yaiche, Insaf Ben
AU  - Ouerghi, Rihab
AU  - Turki, Ines
AU  - Ladeb, Saloua
AU  - Othman, Tarek Ben
AU  - Abdeljelil, Nour Ben
PY  - 2024
DA  - 2024/11/11
TI  - Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Fanconi Anemia: Results of a Single Center with a Fludarabine-Based Conditioning Regimen
JO  - OBM Transplantation
SP  - 226
VL  - 08
IS  - 04
AB  - Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) remains the only curative treatment for bone marrow failure (BMF) and hematologic complications of Fanconi anemia (FA). The evolution remains affected by the toxicity, the risk of graft failure, and clonal evolution. This study aimed to identify factors affecting outcomes in FA patients after allo-HSCT. We included FA patients who underwent allo-HSCT between January 2006 and December 2021. The conditioning regimen was cyclophosphamide/fludarabine (Cy/Flu) ± rabbit anti-thymocyte globulin (ATG). Bone marrow was the stem cell source from HLA-matched related donors in all transplants. Twenty-three patients, 19 with BMF and 4 with MDS/clonal evolution, were included. The median age was 11 years (5-39 years). Five patients (22%) received serotherapy with the conditioning regimen. Engraftment occurred in all patients without severe regimen-related toxicity. The 100-day cumulative incidence (CI) of grade II-IV acute GvHD (a GVHD) and 5-year CI of chronic GvHD (cGVHD) were 17% and 32%, respectively. The 5-year CI of late secondary graft failure was 10.5%. Two patients developed clonal evolution (AML; n = 2) and one patient developed nephrotic syndrome (n = 1). The 10-year overall survival (OS) and event-free survival (EFS) were 80% and 75%, respectively. There was a trend toward a better EFS in patients aged 
SN  - 2577-5820
UR  - https://doi.org/10.21926/obm.transplant.2404226
DO  - 10.21926/obm.transplant.2404226
ID  - Mekni2024
ER  -