Outcome of hematopoietic stem cell transplantation (HCT) from HLA-matched related donor for Fanconi anemia (FA) in adolescents and adults: a retrospective study by Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT).

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2020
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Abstract
Hematopoietic Stem Cell Transplantation (HSCT) is the only potentially curative treatment option for the hematologic complications that occur in patients with Fanconi anemia (FA). In this study, we present a retrospective multicenter analysis from the Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT) of matched related donor HSCT for FA in adolescents and adults transplanted between 1988 and 2015. Forty-five patients received HSCT with a median age at transplant of 18 years, the interquartile range (IQR) (15-23.5); 25 (55.6%) patients were females and 20 (44.4%) were males. Conditioning regimen was fludarabine-based in 29 (64.4%) patients, irradiation-based in five (11.1%) patients, and the remaining patients received other combinations. Indication for HSCT was bone marrow failure in 39 (86.7%) and myelodysplastic syndrome in six (13.3%) patients. Stem cell source was bone marrow in 22 (48.9%), peripheral blood in 20 (44.4%), umbilical cord blood in one (2.2%), and combination of bone marrow and cord blood in two (4.4%) patients. Twenty-seven (60%) patients engrafted and five (11.1%) had primary engraftment failure. The median time to neutrophil engraftment was 14 days (range 10-21 days); median time for platelet engraftment was 17 days (10-33 days). The probability of developing grade II-IV acute GVHD for all patients was 7.0% and chronic GVHD 36.6%. No new malignancies were reported. The OS probability was 53.6% (95% CI, 38.3-68.9%) with a median follow-up of 13 months (95% CI, 1-240). Our HLA-matched related HSCT results in AYA patients with FA compare favorably with other reported international registry data.
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Authors AlDawsari, Ghuzayel;Elhaddad, Alaa;El Fakih, Riad;Ben Othman, Tarek;Ahmed, Parvez;Ghavamzadeh, Ardeshir;Bazarbachi, Ali;Dasouki, Majed J;Fathy, Gamal;Alzahrani, Hazzaa;Samra, Mohamed;Torjemane, Lamia;Satti, Tariq Mahmood;Shaheen, Marwan;Alfraih, Feras;Ayas, Mouhab;Alahmari, Ali;Alhayli, Saud;Nassar, Amr;Abboud, Miguel;Abdelfattah, Raafat;El Solh, Hassan;Hashmi, Shahrukh;Elhassan, Tusneem;Ahmed, Syed Osman;Aljurf, Mahmoud;
Journal bone marrow transplantation
Year 2020
DOI 10.1038/s41409-020-0809-5
URL
Keywords
formaldehyde DNA repair dna damage base excision repair icl nucleotide excision repair fanconi anemia crosslink dna–protein crosslink dpc environmental toxin fmd1 formaldehyde dehydrogenase interstrand crosslink ner replication fork

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