Abstract
Fanconi anemia (FA) is a rare, multi-organ cancer-prone inherited bone
marrow failure syndrome (IBMFS) associated with hematological
malignancies and solid tumors. The androgen therapy, hematopoietic
growth factors, hematopoietic stem cell transplantation (HSCT), and gene
therapy (GT), still in the clinical trial, are various treatments for
this disease. Here, we aimed to compare the advantages and disadvantages
of HSCT and GT in FA cures.
We perform an advanced electronic search of ”Fanconi anemia” AND
(genetics OR treatment OR HSCT OR GT OR Mosaicism), and ”Allo-HSCT” AND
(conditioning regimen OR complications OR GvHD OR infection OR cost)
MeSH terms in non-citation and citation databases. Besides, the gray
literature was searched too. This article will provide a summary of the
advantages and disadvantages of HSCT and GT of FA disease.
Our results show that GT has a good potential in FA treatments in the
future. Furthermore, it has higher advantages and fewer disadvantages in
comparison with HSCT.
Systematic Review Registration: CRD42021247364 ID on PROSPERO
database.
Keywords: Fanconi Anemia, Hematopoietic Stem Cell
Transplantation, Gene Therapy, Comparison.