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Fanconi Anemia Phenotypic and Transplant Outcomes' Associations in Iranian Patients Publisher



Ansari F1 ; Behfar M1, 2 ; Naji P1 ; Darvish Z3 ; Rostami T3 ; Mohseni R1 ; Alimoghaddam K4 ; Salajegheh P5 ; Ahadi B1 ; Mardani M1 ; Hamidieh AA1
Authors
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Authors Affiliations
  1. 1. Pediatric Cell and Gene Therapy Research Centre, Gene, Cell & Tissue Research Institute, Tehran University of Medical Sciences, Tehran, Iran
  2. 2. Pediatric Hematopoietic Stem Cell Transplant Department, Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran
  3. 3. Institute for Oncology, Hematology and Cell Therapy, Shariati Hospital, Tehran University of Medical Sciences, Tehran, Iran
  4. 4. Hematology-Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran
  5. 5. Department of Pediatric, Faculty of Medicine, Kerman University of Medical Sciences, Kerman, Iran

Source: Health Science Reports Published:2023


Abstract

Objectives: Fanconi anemia (FA) is a rare, heterogeneous, inherited disorder. Allogeneic hematopoietic stem cell transplantation (HSCT) represents the only therapeutic option to restore normal hematopoiesis. This study reports the outcomes of FA-HSCT patients and identifies factors, including clinical phenotype. Our team examined more than 95% of Iranian FA patients during the last decade. Study Design: One hundred and six FA patients (age range: 2–41) who underwent HSCT from March 2007 to February 2018 were enrolled. Clinical characteristics of genetic disease, pre-HSCT findings, HSCT indication, and long-term follow-up evaluated and recorded. Data were analyzed using SPSS 19.0. Results: The mean follow-up period for survivors was 36 months (range, 1–101). The 3-year overall survival (OS) and disease-free survival were 72.2% and 71.2%, respectively. The 3-year OS rate for patients with limited and extensive malformations was 78.8% and 56.6%, respectively (p = 0.025). Acute graft versus host disease incidence was 60.52% for patients with limited malformations versus 70% for patients with extensive ones (p = 0.49). Chronic graft versus host disease incidence for these two groups was 9.21% and 10%, respectively (p = 0.91). Conclusions: OS was not associated with each of the malformations singly; however, it was lower in the extensive group. The younger age of patients at the HSCT time leads to a higher OS. The differences in FA patients' outcomes and the various genotypes were probably related. These data provide a powerful tool for further studies on genotype–phenotype association with HSCT results. © 2023 The Authors. Health Science Reports published by Wiley Periodicals LLC.
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