PMID- 32024988 OWN - NLM STAT- MEDLINE DCOM- 20210621 LR - 20210621 IS - 1476-5365 (Electronic) IS - 0268-3369 (Linking) VI - 55 IP - 7 DP - 2020 Jul TI - 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). PG - 1485-1490 LID - 10.1038/s41409-020-0809-5 [doi] AB - 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. FAU - AlDawsari, Ghuzayel AU - AlDawsari G AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. aldawsarig@rchsp.med.sa. AD - Internal Medicine Department, Royal Commission Health Service Program, AL-Lulu Road18, Jubial Industrial City, 11994, Saudi Arabia. aldawsarig@rchsp.med.sa. FAU - Elhaddad, Alaa AU - Elhaddad A AD - National Cancer Institute, Cairo University, Cairo, Egypt. FAU - El Fakih, Riad AU - El Fakih R AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Ben Othman, Tarek AU - Ben Othman T AD - Centre National de Greffe de la Moelle Osseuse, Tunis, Tunisia. FAU - Ahmed, Parvez AU - Ahmed P AD - Armed Forces Institute of Transplantation, Rawalpindi, Pakistan. FAU - Ghavamzadeh, Ardeshir AU - Ghavamzadeh A AD - Hematology, Oncology and SCT Research Center, Tehran University of Medical Sciences, Tehran, Iran. FAU - Bazarbachi, Ali AU - Bazarbachi A AD - American University of Beirut Medical Center, Beirut, Lebanon. FAU - Dasouki, Majed J AU - Dasouki MJ AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Fathy, Gamal AU - Fathy G AD - Nasser Institute for Research and Treatment, Cairo, Egypt. FAU - Alzahrani, Hazzaa AU - Alzahrani H AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Samra, Mohamed AU - Samra M AD - National Cancer Institute, Cairo University, Cairo, Egypt. FAU - Torjemane, Lamia AU - Torjemane L AD - Centre National de Greffe de la Moelle Osseuse, Tunis, Tunisia. FAU - Satti, Tariq Mahmood AU - Satti TM AD - Armed Forces Institute of Transplantation, Rawalpindi, Pakistan. FAU - Shaheen, Marwan AU - Shaheen M AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Alfraih, Feras AU - Alfraih F AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Ayas, Mouhab AU - Ayas M AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Alahmari, Ali AU - Alahmari A AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Alhayli, Saud AU - Alhayli S AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Nassar, Amr AU - Nassar A AD - National Cancer Institute, Cairo University, Cairo, Egypt. FAU - Abboud, Miguel AU - Abboud M AD - American University of Beirut Medical Center, Beirut, Lebanon. FAU - Abdelfattah, Raafat AU - Abdelfattah R AD - National Cancer Institute, Cairo University, Cairo, Egypt. FAU - El Solh, Hassan AU - El Solh H AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Hashmi, Shahrukh AU - Hashmi S AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Elhassan, Tusneem AU - Elhassan T AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Ahmed, Syed Osman AU - Ahmed SO AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. FAU - Aljurf, Mahmoud AU - Aljurf M AD - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. LA - eng PT - Letter PT - Multicenter Study DEP - 20200205 PL - England TA - Bone Marrow Transplant JT - Bone marrow transplantation JID - 8702459 SB - IM MH - Adolescent MH - Adult MH - Bone Marrow MH - *Fanconi Anemia/therapy MH - Female MH - *Graft vs Host Disease MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Male MH - Retrospective Studies MH - Transplantation Conditioning EDAT- 2020/02/07 06:00 MHDA- 2021/06/22 06:00 CRDT- 2020/02/07 06:00 PHST- 2019/03/12 00:00 [received] PHST- 2020/01/21 00:00 [accepted] PHST- 2019/11/12 00:00 [revised] PHST- 2020/02/07 06:00 [pubmed] PHST- 2021/06/22 06:00 [medline] PHST- 2020/02/07 06:00 [entrez] AID - 10.1038/s41409-020-0809-5 [pii] AID - 10.1038/s41409-020-0809-5 [doi] PST - ppublish SO - Bone Marrow Transplant. 2020 Jul;55(7):1485-1490. doi: 10.1038/s41409-020-0809-5. Epub 2020 Feb 5.