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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) Publisher Pubmed



Aldawsari G1, 8 ; Elhaddad A2 ; El Fakih R1 ; Ben Othman T3 ; Ahmed P4 ; Ghavamzadeh A5 ; Bazarbachi A6 ; Dasouki MJ1 ; Fathy G7 ; Alzahrani H1 ; Samra M2 ; Torjemane L3 ; Satti TM4 ; Shaheen M1 Show All Authors
Authors
  1. Aldawsari G1, 8
  2. Elhaddad A2
  3. El Fakih R1
  4. Ben Othman T3
  5. Ahmed P4
  6. Ghavamzadeh A5
  7. Bazarbachi A6
  8. Dasouki MJ1
  9. Fathy G7
  10. Alzahrani H1
  11. Samra M2
  12. Torjemane L3
  13. Satti TM4
  14. Shaheen M1
  15. Alfraih F1
  16. Ayas M1
  17. Alahmari A1
  18. Alhayli S1
  19. Nassar A2
  20. Abboud M6
  21. Abdelfattah R2
  22. El Solh H1
  23. Hashmi S1
  24. Elhassan T1
  25. Ahmed SO1
  26. Aljurf M1
Show Affiliations
Authors Affiliations
  1. 1. King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
  2. 2. National Cancer Institute, Cairo University, Cairo, Egypt
  3. 3. Centre National de Greffe de la Moelle Osseuse, Tunis, Tunisia
  4. 4. Armed Forces Institute of Transplantation, Rawalpindi, Pakistan
  5. 5. Hematology, Oncology and SCT Research Center, Tehran University of Medical Sciences, Tehran, Iran
  6. 6. American University of Beirut Medical Center, Beirut, Lebanon
  7. 7. Nasser Institute for Research and Treatment, Cairo, Egypt
  8. 8. Internal Medicine Department, Royal Commission Health Service Program, AL-Lulu Road18, Jubial Industrial City, 11994, Saudi Arabia

Source: Bone Marrow Transplantation Published:2020


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. © 2020, Springer Nature Limited.
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