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Hematopoietic stem cell transplantation in patients with Fanconi anemia: a report of late effects and psychosocial adjustment in life

Bone Marrow Transplantation volume 59, pages 141–143 (2024)Cite this article

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Fanconi anemia (FA) cells are characterized by genomic instability that predisposes the patients for malignancies [1,2,3]. Hematopoietic cell transplantation (HCT) is a widely used modality to restore normal hematopoiesis in these patients [4,5,6]. After HCT, however, all other organs in FA patients continue to be subject to the natural history of the disease and many patients suffer from long-term complications contributing to an altered quality of life.

We analyzed the data on 149 FA patients who underwent HCT at our center between January 1995 and December 2015. At the time of initiation of this study, 45 patients had died. Of the remaining 104, we could contact and interview 87. All had survived at least 2 years after HCT. All the patients in the cohort had received chemo-based conditioning. Radiation was added in 53 patients. Overall survival (OS) was our primary endpoint of interest, whereas the event was death from any cause. Survival was time from transplant to death. The median age at first HCT was 8.5 years. Ten patients had primary graft failure (eight of them died, and two had a second HCT). Eight had secondary graft failure (seven died during follow-up, and one was lost to follow-up). Of the remaining 131 patients, 30 died later during follow-up, and 101 patients were alive at the last follow-up. The cumulative incidence of acute GvHD was 20.8%, and that of chronic GvHD was 13.4% (Table 1). Chimerism data at the last follow-up were available for 64 patients. Fifty-nine (92.2%) exhibited 100%, one 99–95%, one 95–90%, and the remaining three had 90–50%.

Table 1 Transplant characteristics.
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The authors have full access to all the data related to this study. The data used in this work are considered confidential and can be provided in its raw format upon reasonable request to the corresponding author.

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Authors and Affiliations

  1. Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia

    Mouhab Ayas, Fatima Al-Hashim, Khawar Siddiqui, Abdullah Al-Jefri, Ali Al-Ahmari, Ibrahim Ghemlas, Hawazen Al-Saedi, Awatif Al-Anazi, Saadiya Khan & Donya Qattan

  2. Department of Oncology, King Faisal Specialist Hospital and Research Center, Jeddah, Saudi Arabia

    Amal Al-Seraihi

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  1. Mouhab Ayas
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Contributions

MA and KS conceived the idea. MA, FA-H and KS designed and performed the study. FA-H extracted the data and carried out the initial quality checks. MA and KS drafted the manuscript and did critical editing. FA-H assisted and supported in data collection while subsequent analysis with statistics was done by KS. MA supervised this manuscript preparation and writing, while all other authors contributed to critically reviewing the results and revising the manuscript.

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Correspondence to Mouhab Ayas.

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The authors declare no competing interests.

Ethical approval

This study was submitted to the Institutional Review Board of King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia, before initiation and was approved by the Research Advisory Committee through established procedures with Approval Number 2141033.

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Ayas, M., Al-Hashim, F., Siddiqui, K. et al. Hematopoietic stem cell transplantation in patients with Fanconi anemia: a report of late effects and psychosocial adjustment in life. Bone Marrow Transplant 59, 141–143 (2024). https://doi.org/10.1038/s41409-023-02133-x

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