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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
Show Affiliations
Authors Affiliations
  1. 1. Eurocord, Paris-Diderot University, Equipe d'Accueil 3518, Hospital Saint Louis, 1 Ave Claude Vellefaux, Paris, 75010, France
  2. 2. Monacord, International Observatory on Sickle Cell Disease, Centre Scientifique de Monaco, Monaco
  3. 3. Department of Pediatrics, Referral Center for Sickle Cell Disease, Centre Hospitalier Intercommunal Creteil, Paris XII University, Creteil, France
  4. 4. European Society for Blood and Marrow Transplantation Statistical Unit, Hospital Saint Antoine, Assistance Publique-Hopitaux de Paris, Paris, France
  5. 5. Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI, United States
  6. 6. Ribeirao Preto Medical School, University of Sao Paulo, Sao Paulo, Brazil
  7. 7. Hemato-Oncology Unit, Hospital Universitaire des Enfants Reine Fabiola, Universite Libre de Bruxelles, Brussels, Belgium
  8. 8. Cliniques Universitaires Saint Luc, Hemato-Oncology Unit, Brussels, Belgium
  9. 9. Centre for Haematology, Imperial College London, London, United Kingdom
  10. 10. Hematoimmunology, Hospital Robert Debre and Paris-Diderot University, Paris, France
  11. 11. Pediatric Hematology-Oncology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy
  12. 12. University of California San Francisco, Benioff Children's Hospital, Oakland, CA, United States
  13. 13. Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA, United States
  14. 14. Morgan Stanley Children's Hospital of New York, New York, NY, United States
  15. 15. Baylor College of Medicine, Houston, TX, United States
  16. 16. Children's Hospital of Michigan, Detroit, MI, United States
  17. 17. Pediatric Blood and Marrow Transplant Program, Duke University Medical Center, Durham, NC, United States
  18. 18. Adolescent and Young Adults Hematology Department, Hospital Saint-Louis, Paris, France
  19. 19. Department of Pediatric Hematology and Oncology and Research Unit, Equipe d'Accueil 3279, Aix-Marseille University, Timone Children's Hospital Marseille, Marseille, France
  20. 20. Imperial College London, Hammersmith Hospital, London, United Kingdom
  21. 21. Service D'hemato Oncologie Pediatrique, Centre Hospitalier Universitaire de Strasbourg, Strasbourg, France
  22. 22. Pediatric Hematology-Immunology Department, Hospital Necker-Enfants Malades, Assistance Publique-Hopitaux de Paris, Paris, France
  23. 23. Department of Pediatric Hematology and Oncology, University Hospital of Lyon, Lyon, France
  24. 24. Equipe d'Accueil 3829, Institut de Recherche et d'Innovation Biomedicale, Faculte de Medecine-Pharmacie, Rouen, France
  25. 25. Paediatric Haematology/Oncology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
  26. 26. Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France
  27. 27. Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hopitaux de Paris, INSERM, Paris, France
  28. 28. Paris Descartes-Sorbonne Paris Cite University, Imagine Institute, Paris, France
  29. 29. Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, Austria
  30. 30. Hospital Sirio-Libanes, Servico de Hematologia, Hemoterapia e Terapia Celular, Sao Paulo University, Sao Paulo, Brazil
  31. 31. Churchill Hospital, Oxford, United Kingdom
  32. 32. Department for Stem Cell Transplantation and Immunology, Clinic for Children and Adolescents, University Hospital Frankfurt, Goethe University, Frankfurt, Germany
  33. 33. Dipartimento di Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico, Ospedale Bambino Gesu, Rome, Italy
  34. 34. Dipartimento di Scienze Pediatriche, Universita di Pavia, Pavia, Italy
  35. 35. Department of Hematology and Cell Therapy, Hospital Saint Antoine, Paris, France
  36. 36. University Hospital Gent, Gent, Belgium
  37. 37. University Hospital Gasthuisberg, Leuven, Belgium
  38. 38. University of Liege, Liege, Belgium
  39. 39. Ste-Justine Hospital, Montreal, QC, Canada
  40. 40. Rigshospitalet, Copenhagen, Denmark
  41. 41. Hospital Saint Jacques, Besancon, France
  42. 42. Centre Hospitalier Universitaire Pellegrin Enfants, Bordeaux, France
  43. 43. Centre Hospitalier Universitaire Hospital Hotel Dieu, Clermont-Ferrand, France
  44. 44. Hospital La Tronche, Grenoble, France
  45. 45. CHU Jeanne de Flandre, Lille, France
  46. 46. University Hospital Department of Pediatric Hematology/Oncology, Lyon, France
  47. 47. Hospital La Timone, Marseille, France
  48. 48. Hospital Brabois, Nancy, France
  49. 49. Hospital Hotel Dieu, Nantes, France
  50. 50. La Pitie, Paris, France
  51. 51. Hospital Charles Nicolle, Rouen, France
  52. 52. Hospital Nord, Saint-Etienne, France
  53. 53. Hospital Hautepierre, Strasbourg, France
  54. 54. Universitate Klinikum Carl Gustav Carus, Dresden, Germany
  55. 55. University of Freiburg, Freiburg, Germany
  56. 56. Hannover Medical School, Dept. Pediatric Haematology/Oncology, Hannover, Germany
  57. 57. Eppendorf, Hamburg, Germany
  58. 58. Klinikum Grosshadern, Munich, Germany
  59. 59. Klinikum Nurnberg, Nurnberg, Germany
  60. 60. St. Sophia Hospital, Athens, Greece
  61. 61. King Hussein Cancer Centre, Amman, Jordan
  62. 62. Shariati Hospital Teheran, Iran
  63. 63. Rambam Medical Center, Haifa, Israel
  64. 64. Hadassah University Hospital, Jerusalem, Israel
  65. 65. Schneider Childrens Medical Center, Petach-Tikva, Israel
  66. 66. Edmond and Lily Safra Children's Hospital, Tel-Hashomer, United States
  67. 67. S.Orsola-Malpighi, Bologna, Italy
  68. 68. Ospedale Ferrarotto, Catania, Italy
  69. 69. Instituto G. Gaslini, Genova, Italy
  70. 70. Ospedale San Raffaele, Milano, Italy
  71. 71. Ematologia Pediatrica, Napoli, Italy
  72. 72. Clinica di Oncoematologia Pediatrica, Padova, Italy
  73. 73. Ospedale Civile, Pescara, Italy
  74. 74. Hospital Guglielmo da Saliceto, Piacenza, Italy
  75. 75. Roma-Policlinico Tor Vergata and Transplant Network, Roma, Italy
  76. 76. Universita La Sapienza, Roma, Italy
  77. 77. Ospedale Infantile Regina Margherita, Torino, Italy
  78. 78. Istituto Burlo Garofolo, Trieste, Italy
  79. 79. Malicka-University Hospital, Leiden, Netherlands
  80. 80. University of Benin, Benin, Nigeria
  81. 81. Russian Childrens Hospital, Moscow, Russian Federation
  82. 82. Hospital Vall d'Hebron, Barcelona, Spain
  83. 83. Hospital Gregorio Maranon, Madrid, Spain
  84. 84. Nino Jesus Hospital, Madrid, Spain
  85. 85. Hospital Morales Meseguer, Murcia, Spain
  86. 86. Medi-Clinic Constantiaberg, Cape Town, South Africa
  87. 87. Pretoria East Hospital, Pretoria, South Africa
  88. 88. Sahlgrenska Hospital, Goteborg, Sweden
  89. 89. University Hospital, Linkoping, Sweden
  90. 90. Karolinska Hospital, Stockholm, Sweden
  91. 91. Hopitaux Universitaires Pediatric Department, Geneve, Switzerland
  92. 92. Universitats Kinderspital Eleonorenstiftung, Zurich, Switzerland
  93. 93. Cebeci Hospital, Ankara, Turkey
  94. 94. Gaziantep University, Gaziantep, Turkey
  95. 95. Istanbul University, Istanbul, Turkey
  96. 96. Erciyes Universitesi, Kapadokya Kayseri, Turkey
  97. 97. Birmingham Childrens Hospital, Birmingham, United Kingdom
  98. 98. Royal Free Hospital and School of Medicine, London, United Kingdom
  99. 99. St. George, London, United Kingdom
  100. 100. St. Marys Hospital, Division of Paediatrics, London, United Kingdom
  101. 101. NHS Trust, Manchester, United Kingdom
  102. 102. Royal Hallamshire, Sheffield, United Kingdom
  103. 103. Vanderbilt University, Nashville, TN, United States
  104. 104. St. Jude Children's Research Hospital, Memphis, TN, United States
  105. 105. Children's National Medical Center, Washington, DC, United States
  106. 106. Cohen Children's Medical Center of New York, New York, NY, United States
  107. 107. Children's Hospital of Wisconsin, Milwaukee, WI, United States
  108. 108. Cook Children's Medical Center, Fort Worth, TX, United States
  109. 109. Nationwide Children's Hospital, Columbus, OH, United States
  110. 110. University of California, San Francisco, CA, United States
  111. 111. Children's Hospital of Los Angeles, CA, United States
  112. 112. Hackensack University Medical Center, Hackensack, NJ, United States
  113. 113. University of Mississippi Medical Center, Jackson, MS, United States
  114. 114. Children's Medical Center, Dallas, TX, United States
  115. 115. Johns Hopkins Oncology Center, Baltimore, MD, United States
  116. 116. Medical University of South Carolina, Charleston, SC, United States
  117. 117. City of Hope National Medical Center, Duarte, CA, United States
  118. 118. Philadelphia Children's Hospital, Philadelphia, PA, United States
  119. 119. University of North Carolina Hospitals, Chapel Hill, NC, United States
  120. 120. Oklahoma University Medical Center, Oklahoma City, OK, United States
  121. 121. University of Alabama Birmingham, Birmingham, AL, United States
  122. 122. Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, United States

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.
2. Applied Stem Cell Research in Sickle Cell Disease, Comprehensive Hematology and Stem Cell Research: Volume 1-5 (2024)
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