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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
Show Affiliations
Authors Affiliations
  1. 1. Division for Stem Cell Transplantation, Immunology and Intensive Care Medicine, Department for Children and Adolescents Medicine, University Hospital Frankfurt, Frankfurt am Main, Germany
  2. 2. Department of Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, Netherlands
  3. 3. Department of Pediatric Hematology- Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia
  4. 4. Paediatric Haemato Oncology and BMT, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel-Hashomer, Israel
  5. 5. Pediatric Immunology, Hematology, and Rheumatology Unit, Hopital Necker Enfants Malades, Assistance Publique-Hopitaux de Paris (AP-HP), Paris, France
  6. 6. INSERM U1163, Institut Imagine, Paris, France
  7. 7. Paris-Descartes University, Sorbonne Paris Cite, Paris, France
  8. 8. Department of Pediatric Hematology/ Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ospedale Pediatrico Bambino Gesu, Sapienza University of Rome, Rome, Italy
  9. 9. Department of Pediatric Hematology, Immunology and Oncology, La Timone Children's Hospital, Assistance Publique-Hopitaux de Marseille (AP-HM)/Aix-Marseille University, Marseille, France
  10. 10. Pediatric BMT Unit, Acibadem University Atakent Hospital, Istanbul, Turkey
  11. 11. Department of Pediatrics, University Medical Center Ulm, Ulm, Germany
  12. 12. BMT Unit, Department of Hematology, Rigshospitalet, Copenhagen, Denmark
  13. 13. Blood and Marrow Transplant Unit, Department of Paediatric Haematology, Royal Manchester Children's Hospital, Manchester, United Kingdom
  14. 14. Belarussian Centre for Paediatric Oncology and Hematology, Minsk, Belarus
  15. 15. Institut d'Hematologie et d'Oncologie Pediatrique, Hospices Civils de Lyon and Universite Claude Bernard, Lyon, France
  16. 16. King Hussein Cancer Centre, Amman, Jordan
  17. 17. Great Ormond Street (GOS) Hospital for Children, NHS Foundation Trust, University College London, GOS Institute of Child Health/National Institute for Health Research (NIHR) GOS Hospital Biomedical Research Centre (BRC), London, United Kingdom
  18. 18. Division of Pediatric Hematology and Oncology, Department of Pediatrics, Erciyes University, Faculty of Medicine, Kayseri, Turkey
  19. 19. Department of Pediatric Hematology, Akdeniz University Medical School, Antalya, Turkey
  20. 20. ArdeshirGhavamzadeh, Hematology-Oncology and BMT Research, Shariati Hospital, Tehran, Iran
  21. 21. Department of Pediatrics, University Hospital Tuebingen, Tuebingen, Germany
  22. 22. Division of Pediatric Hematology-Oncology, Department of Pediatrics, Ankara University Faculty of Medicine, Ankara, Turkey
  23. 23. Hospital Infantil. Hematologia-Oncologia, Hospital Universitario La Paz, Madrid, Spain
  24. 24. Pediatric BMT Unit, Department of Pediatric Hematology and Oncology, Gaziosmanpasa Hospital, Yeniyuzyil University, Istanbul, Turkey
  25. 25. Oncohaematology and BMT Unit, Ospedale dei Bambini, Brescia, Italy
  26. 26. Pediatric Hematology, Ankara Child Health and Diseases Hematology Oncology Training and Research Hospital, Ankara, Turkey
  27. 27. Pediatric Stem Cell Transplantation Unit, Medical Park Goztepe Hospital, Istanbul, Turkey
  28. 28. Department of Pediatrics, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain
  29. 29. Centre for Allogeneic Stem Cell Transplantation, Karolinska University Hospital, Stockholm, Sweden
  30. 30. Department of Bone Marrow Transplantation, The Russian Children's Research Hospital, Moscow, Russian Federation
  31. 31. Pediatric Stem Cell Transplantation Unit, Medical Park Antalya Hospital, Antalya, Turkey
  32. 32. Department of Hematology and BMT, Rambam Medical Center, Haifa, Israel
  33. 33. Willem-Alexander Children's Hospital, Department of Pediatrics, Leiden University Medical Center, Leiden, Netherlands
  34. 34. Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Hospital, Jerusalem, Israel
  35. 35. Great North Children's Hospital, Royal Victoria Infirmary, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom
  36. 36. Dr. v. Hauner University Children's Hospital, Ludwig-Maximilians Universitat, Munich, Germany

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.