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Role of gene therapy in Fanconi anemia: A systematic and literature review with future directions

Medical College, Aga Khan University, Karachi, Pakistan. Electronic address: hashmi.shahrukh@mayo.edu. Medical College, Aga Khan University, Karachi, Pakistan. Division of Hematology, Brigham and Women's Hospital, Boston, MA, USA. Department of Genetics, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. Department of Adult Hematology and Stem Cell Transplantation, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN, USA.

Hematology/oncology and stem cell therapy. 2021
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Abstract
Gene therapy (GT) has been reported to improve bone marrow function in individuals with Fanconi anemia (FA); however, its clinical application is still in the initial stages. We conducted this systematic review, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, to assess the long-term safety and clinical outcomes of GT in FA patients. Electronic searches from PubMed, Web of Science, Cochrane Library, and Google Scholar were conducted and full texts of articles meeting our inclusion criteria were reviewed. Three clinical trials were included, with a total of nine patients and mean age of 10.7 ± 5.7 years. All patients had lentiviral-mediated GT. A 1-year follow-up showed stabilization in blood lineages, without any serious adverse effects from GT. A metaregression analysis could not be conducted, as very little long-term follow-up data of patients was observed, and the median survival rate could not be calculated. Thus, we can conclude that GT seems to be a safe procedure in FA; however, further research needs to be conducted on the longitudinal clinical effects of GT in FA, for a better insight into its potential to become a standard form of treatment.
Study details
Study Design : Systematic Review
Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine