Tehran University of Medical Sciences

Science Communicator Platform

Stay connected! Follow us on X network (Twitter):
Share By
Allogeneic Hematopoietic Stem Cell Transplantation in Leukocyte Adhesion Deficiency Type I and Iii Publisher



Bakhtiar S1 ; Salzmannmanrique E1 ; Blok HJ2 ; Eikema DJ2 ; Hazelaar S2 ; Ayas M3 ; Toren A4 ; Goldstein G4 ; Moshous D5, 6, 7 ; Locatelli F8 ; Merli P8 ; Michel G9 ; Ozturk G10 ; Schulz A11 Show All Authors
Authors
  1. Bakhtiar S1
  2. Salzmannmanrique E1
  3. Blok HJ2
  4. Eikema DJ2
  5. Hazelaar S2
  6. Ayas M3
  7. Toren A4
  8. Goldstein G4
  9. Moshous D5, 6, 7
  10. Locatelli F8
  11. Merli P8
  12. Michel G9
  13. Ozturk G10
  14. Schulz A11
  15. Heilmann C12
  16. Ifversen M12
  17. Wynn RF13
  18. Aleinikova O14
  19. Bertrand Y15
  20. Tbakhi A16
  21. Veys P17
  22. Karakukcu M18
  23. Kupesiz A19
  24. Ghavamzadeh A20
  25. Handgretinger R21
  26. Unal E22
  27. Perezmartinez A23
  28. Gokce M24
  29. Porta F25
  30. Aksu T26
  31. Karasu G27
  32. Badell I28
  33. Ljungman P29
  34. Skorobogatova E30
  35. Yesilipek A31
  36. Zuckerman T32
  37. Bredius RRG33
  38. Stepensky P34
  39. Shadur B34
  40. Slatter M35
  41. Gennery AR35
  42. Albert MH36
  43. Bader P1
  44. Lankester A33

Source: Blood Advances Published:2021


Abstract

Type I and III leukocyte adhesion deficiencies (LADs) are primary immunodeficiency disorders resulting in early death due to infections and additional bleeding tendency in LAD-III. The curative treatment of LAD-I and LAD-III is allogeneic hematopoietic stem cell transplantation (allo-HSCT). In this retrospective multicenter study, data were collected using the European Society for Blood and Marrow Transplantation registry; we analyzed data from 84 LAD patients from 33 centers, all receiving an allo-HSCT from 2007 to 2017. The 3-year overall survival estimate (95% confidence interval [CI]) was 83% (74-92) for the entire cohort: 84% (75-94) and 75% (50-100) for LAD-I and LAD-III, respectively. We observed cumulative incidences (95% CI) of graft failure (GF) at 3 years of 17% (9%-26%) and grade II to IV acute graft-versus-host disease (aGVHD) at 100 days of 24% (15%-34%). The estimate (95% CI) at 3 years for GF- and GVHD-II to IV-free survival as event-free survival (EFS) was 56% (46-69) for the entire cohort; 58% (46-72) and 56% (23-88) for LAD-I and LAD-III, respectively. Grade II to IV acute GVHD was a relevant risk factor for death (hazard ratio 3.6; 95% CI 1.4-9.1; P 5.006). Patients' age at transplant $13 months, transplantation from a nonsibling donor, and any serological cytomegalovirus mismatch in donor-recipient pairs were significantly associated with severe acute GVHD and inferior EFS. The choice of busulfan- or treosulfan-based conditioning, type of GVHD prophylaxis, and serotherapy did not impact overall survival, EFS, or aGVHD. An intrinsic inflammatory component of LAD may contribute to inflammatory complications during allo-HSCT, thus providing the rationale for considering anti-inflammatory therapy pretreatment. © 2021 by The American Society of Hematology.
Other Related Docs
13. Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients With Fanconi Anemia, Mediterranean Journal of Hematology and Infectious Diseases (2016)