Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Fanconi Anemia: Results of a Single Center with a Fludarabine-Based Conditioning Regimen
Abstract
(ISSN 2577-5820)
OBM Transplantation (ISSN 2577-5820) is an international peer-reviewed Open Access journal published quarterly online by LIDSEN Publishing Inc., which covers all evidence-based scientific studies related to transplantation, including: transplantation procedures and the maintenance of transplanted tissues or organs; assimilation of grafted tissue and the reconstitution of removed organs or parts of organs; transplantation of heart, lung, kidney, liver, pancreatic islets and bone marrow, etc. Areas related to clinical and experimental transplantation are also of interest.
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Special Issue
Allogeneic Stem Cell Transplantation in Fanconi Anemia and Pediatric Bone Marrow Failure
Submission Deadline: April 30, 2025 (Open) Submit Now
Guest Editor
Lamia Torjemane, PhD, Associate Professor
Hematology and Adult Transplant Service, National Bone Marrow Transplant Center, 1006, Tunis, Tunisia
Research Interests: Topics related to hematopoietic stem cell transplant
About This Topic
Allogeneic hematopoietic stem cell transplantation (alloHCT) is the only curative treatment of bone marrow failure (BMF) and hematologic complications of Fanconi anemia (FA). However, the intrinsic FA DNA-repair defect complicates alloHCT, because of reliance on alkylating agents and radiation in conditioning regimen, as well as increased of graft failure and graft-versus-host disease (GVHD). Our previous results during the first 10 years, with low-dose cyclophosphamide and busulfan as conditioning, we previously reported early graft failure (18%) and acute GVHD and chronic GVHD occurred in 23% and 13% of patients respectively.[1] Since 2005, we opted for a Fludarabine-based conditioning regimen ± rabbit antithymocyte globin (ATG).[2] The incidence of acute GVHD decreased to 12.5%. Graft failure was later, between 3 and 5 years, with lower incidence (8%). Long-term overall survival reached 82% at 10 years. However, the evolution remains affected by the risk of clonal evolution (acute leukemia and solid cancers) as well as renal complications (chronic renal failure) particularly in patients with renal malformations or a single kidney. The main risk factor affecting event-free survival is the patient's age at the time of transplantation.
Keywords
Fanconi anemia; Allogeneic hematopoietic stem cell transplantation; Fludarabine; Graft failure; graft-versus-host disease; Hematologic complications
References
1. L. Torjemane, S. Ladeb, T. Ben Othman, A. Abdelkefi, A. Lakhal, and A. Ben Abdeladhim. Bone Marrow Transplantation from Matched Related Donors for Patients with Fanconi Anemia Using Low-Dose Busulfan and Cyclophosphamide as Conditioning. Pediatr Blood Cancer 2005;46: 496–500.
2. Aker M, Varadi G, Slavin S, et al. Fludarabine-based protocol for human umbilical cord blood transplantation in children with Fanconi anemia. Journal of pediatric hematology/oncology. 1999;21(3):237–9.
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Publication
Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Fanconi Anemia: Results of a Single Center with a Fludarabine-Based Conditioning Regimenby
Sabrine Mekni
,
Lamia Torjemane
,
Malek Khayati
,
Dorra Belloumi
,
Rimmel Yosra Kanoun
,
Insaf Ben Yaiche
,
Rihab Ouerghi
,
Ines Turki
,
Saloua Ladeb
,
Tarek Ben Othman
and
Nour Ben Abdeljelil
Abstract 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 outcom [...] |
2023 | ||
CiteScore | SJR | SNIP |
0.6 | 0.179 | 0.17 |
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