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Sickle Cell Disease: An International Survey of Results of Hla-Identical Sibling Hematopoietic Stem Cell Transplantation Publisher Pubmed



Gluckman E1, 2 ; Cappelli B2 ; Bernaudin F3 ; Labopin M4 ; Volt F1, 2 ; Carreras J5 ; Simoes BP6 ; Ferster A7 ; Dupont S8 ; De La Fuente J9, 100 ; Dalle JH10 ; Zecca M11 ; Walters MC12 ; Krishnamurti L13 Show All Authors
Authors
  1. Gluckman E1, 2
  2. Cappelli B2
  3. Bernaudin F3
  4. Labopin M4
  5. Volt F1, 2
  6. Carreras J5
  7. Simoes BP6
  8. Ferster A7
  9. Dupont S8
  10. De La Fuente J9, 100
  11. Dalle JH10
  12. Zecca M11
  13. Walters MC12
  14. Krishnamurti L13
  15. Bhatia M14
  16. Leung K15
  17. Yanik G16
  18. Kurtzberg J17
  19. Dhedin N18
  20. Kuentz M3
  21. Michel G19, 47
  22. Apperley J20
  23. Lutz P21, 53
  24. Neven B22
  25. Bertrand Y23, 46
  26. Vannier JP24, 51
  27. Ayas M25
  28. Cavazzana M26, 27, 28
  29. Matthesmartin S29
  30. Rocha V1, 30, 31
  31. Elayoubi H1, 2
  32. Kenzey C1, 2
  33. Bader P32
  34. Locatelli F33, 34
  35. Ruggeri A1, 2, 35
  36. Eapen M5
  37. Bordon V36
  38. Labarque V37
  39. Pereira M38
  40. Bittencourt H39
  41. Petersen H40
  42. Deconninck E41
  43. Jubert C42
  44. Perrin J43
  45. Cahn JY44
  46. Bruno B45
  47. Bordigoni P48
  48. Mechinaud F49
  49. Vernant JP50
  50. Stephan JL52
  51. Suttorp M54
  52. Strahm B55
  53. Da Cunha CB56
  54. Garwer B57
  55. Rothmayer M58
  56. Wendelin K59
  57. Graphakos S60
  58. Tbakhi A61
  59. Naeimi N62
  60. Zuckerman T63
  61. Sharon PB64
  62. Yaniv I65
  63. Amos T66
  64. Prete A67
  65. Lo Nigro L68
  66. Lanino E69
  67. Faraci M69
  68. Ciceri F70
  69. Marktel S70
  70. De Simone G71
  71. Messina C72
  72. Bartolomeo PDI73
  73. Santarone S73
  74. Vallisa D74
  75. Bertaina A33
  76. Arcese W75
  77. Foa R76
  78. Berger M77
  79. Maximova N78
  80. Wallet S79
  81. Bazuaye GN80
  82. Maschan A81
  83. De Heredia CD82
  84. Bieler CB83
  85. Pato JR84
  86. Heras I85
  87. Trevor R86
  88. Abayomi K86
  89. Thomson J87
  90. Fasth A88
  91. Frodin U89
  92. Ljugman P90
  93. Ansari M91
  94. Gungor T92
  95. Unal E93
  96. Pehlivan M94
  97. Anak S95
  98. Ozturk G95
  99. Unal A96
  100. Lawson S97
  101. Keshani J98
  102. Drake A99
  103. Wynn R101
  104. Williams J102
  105. Jagsia M103
  106. Leung W104
  107. Abraham A105
  108. Sahdey I106
  109. Margolis D107
  110. Eames G108
  111. Horwitz E109
  112. Cowan M110
  113. Kapoor N111
  114. Rowley S112
  115. Megason G113
  116. Rogers Z114
  117. Bolanosmeade J115
  118. Hudspeth M116
  119. Rosenthal J117
  120. Olson T118
  121. Kassow K119
  122. Selby G120
  123. Haines H121
  124. Chaudhury S122

Source: Blood Published:2017


Abstract

Despite advances in supportive therapy to prevent complications of sickle cell disease (SCD), access to care is not universal. Hematopoietic cell transplantation is, to date, the only curative therapy for SCD, but its application is limited by availability of asuitable HLA-matched donor and lack of awareness of the benefits of transplant. Included in this study are 1000 recipients of HLA-identical sibling transplants performed between 1986 and 2013 and reported to the European Society for Blood and Marrow Transplantation, Eurocord, and the Center for International Blood and Marrow Transplant Research. The primary endpoint was event-free survival, defined as being alive without graft failure; risk factors were studied using a Cox regression models. Themedian age at transplantation was 9 years, and themedian follow-up was longer than 5 years. Most patients received amyeloablative conditioning regimen (n = 873; 87%); the remainder received reduced-intensity conditioning regimens (n = 125; 13%). Bonemarrow was the predominant stem cell source (n = 839; 84%); peripheral blood and cord blood progenitors were used in 73 (7%) and 88 (9%) patients, respectively. The 5-year event-free survival and overall survival were 91.4% (95% confidence interval, 89.6%-93.3%) and 92.9% (95% confidence interval, 91.1%-94.6%), respectively. Eventfree survival was lower with increasing age at transplantation (hazard ratio [HR], 1.09; P<.001) and higher for transplantations performed after 2006 (HR, 0.95; P = .013). Twenty-three patients experienced graft failure, and 70 patients (7%) died, with the most common cause of death being infection. The excellent outcome of a cohort transplanted over the course of 3 decades confirms the role of HLA-identical sibling transplantation for children and adults with SCD. © 2017 by The American Society of Hematology.
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