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Long-Term Outcomes of 176 Patients With X-Linked Hyper-Igm Syndrome Treated With or Without Hematopoietic Cell Transplantation Publisher Pubmed



De La Morena MT1 ; Leonard D1 ; Torgerson TR2 ; Cabralmarques O3 ; Slatter M4 ; Aghamohammadi A5 ; Chandra S6 ; Murguiafavela L7 ; Bonilla FA8 ; Kanariou M9 ; Damrongwatanasuk R10 ; Kuo CY11 ; Dvorak CC12 ; Meyts I13 Show All Authors
Authors
  1. De La Morena MT1
  2. Leonard D1
  3. Torgerson TR2
  4. Cabralmarques O3
  5. Slatter M4
  6. Aghamohammadi A5
  7. Chandra S6
  8. Murguiafavela L7
  9. Bonilla FA8
  10. Kanariou M9
  11. Damrongwatanasuk R10
  12. Kuo CY11
  13. Dvorak CC12
  14. Meyts I13
  15. Chen K14
  16. Kobrynski L15
  17. Kapoor N16
  18. Richter D17
  19. Digiovanni D18
  20. Dhalla F19
  21. Farmaki E20
  22. Speckmann C21
  23. Espanol T22
  24. Shcherbina A23
  25. Hanson IC24
  26. Litzman J25
  27. Routes JM26
  28. Wong M27
  29. Fuleihan R28
  30. Seneviratne SL29
  31. Small TN30
  32. Janda A31
  33. Bezrodnik L18
  34. Seger R32
  35. Raccio AG18
  36. Edgar JDM33
  37. Chou J34
  38. Abbott JK35
  39. Van Montfrans J36
  40. Gonzalezgranado LI37
  41. Bunin N38
  42. Kutukculer N39
  43. Gray P40
  44. Seminario G18
  45. Pasic S41
  46. Aquino V1
  47. Wysocki C1
  48. Abolhassani H5
  49. Dorsey M12
  50. Cunninghamrundles C42
  51. Knutsen AP43
  52. Sleasman J44
  53. Costa Carvalho BT45
  54. Condinoneto A46
  55. Grunebaum E7
  56. Chapel H19
  57. Ochs HD2
  58. Filipovich A6
  59. Cowan M12
  60. Gennery A4
  61. Cant A4
  62. Notarangelo LD47
  63. Roifman CM7

Source: Journal of Allergy and Clinical Immunology Published:2017


Abstract

Background X-linked hyper-IgM syndrome (XHIGM) is a primary immunodeficiency with high morbidity and mortality compared with those seen in healthy subjects. Hematopoietic cell transplantation (HCT) has been considered a curative therapy, but the procedure has inherent complications and might not be available for all patients. Objectives We sought to collect data on the clinical presentation, treatment, and follow-up of a large sample of patients with XHIGM to (1) compare long-term overall survival and general well-being of patients treated with or without HCT along with clinical factors associated with mortality and (2) summarize clinical practice and risk factors in the subgroup of patients treated with HCT. Methods Physicians caring for patients with primary immunodeficiency diseases were identified through the Jeffrey Modell Foundation, United States Immunodeficiency Network, Latin American Society for Immunodeficiency, and Primary Immune Deficiency Treatment Consortium. Data were collected with a Research Electronic Data Capture Web application. Survival from time of diagnosis or transplantation was estimated by using the Kaplan-Meier method compared with log-rank tests and modeled by using proportional hazards regression. Results Twenty-eight clinical sites provided data on 189 patients given a diagnosis of XHIGM between 1964 and 2013; 176 had valid follow-up and vital status information. Sixty-seven (38%) patients received HCT. The average follow-up time was 8.5 ± 7.2 years (range, 0.1-36.2 years). No difference in overall survival was observed between patients treated with or without HCT (P = .671). However, risk associated with HCT decreased for diagnosis years 1987-1995; the hazard ratio was significantly less than 1 for diagnosis years 1995-1999. Liver disease was a significant predictor of overall survival (hazard ratio, 4.9; 95% confidence limits, 2.2-10.8; P < .001). Among survivors, those treated with HCT had higher median Karnofsky/Lansky scores than those treated without HCT (P < .001). Among patients receiving HCT, 27 (40%) had graft-versus-host disease, and most deaths occurred within 1 year of transplantation. Conclusion No difference in survival was observed between patients treated with or without HCT across all diagnosis years (1964-2013). However, survivors treated with HCT experienced somewhat greater well-being, and hazards associated with HCT decreased, reaching levels of significantly less risk in the late 1990s. Among patients treated with HCT, treatment at an early age is associated with improved survival. Optimism remains guarded as additional evidence accumulates. © 2016 American Academy of Allergy, Asthma & Immunology
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