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Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies.
van den Broek, Brigitte T A; Page, Kristin; Paviglianiti, Annalisa; Hol, Janna; Allewelt, Heather; Volt, Fernanda; Michel, Gerard; Diaz, Miguel Angel; Bordon, Victoria; O'Brien, Tracey; Shaw, Peter J; Kenzey, Chantal; Al-Seraihy, Amal; van Hasselt, Peter M; Gennery, Andrew R; Gluckman, Eliane; Rocha, Vanderson; Ruggeri, Annalisa; Kurtzberg, Joanne; Boelens, Jaap Jan.
Afiliación
  • van den Broek BTA; Blood and Marrow Transplantation Program.
  • Page K; Laboratory for Translational Immunology, and.
  • Paviglianiti A; Sylvia Toth Center for Multidisciplinary Follow Up After Hematopoietic Cell Transplantation, UMC Utrecht, Utrecht, The Netherlands.
  • Hol J; Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC.
  • Allewelt H; Eurocord, Paris, France.
  • Volt F; Eurocord, Paris, France.
  • Michel G; Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC.
  • Diaz MA; Eurocord, Paris, France.
  • Bordon V; AP-HM Hôpital de la Timone, Marseille, France.
  • O'Brien T; Hospital Infantil Universitario Niño Jesús, Madrid, Spain.
  • Shaw PJ; Blood and Marrow Transplantation Program, Universiteits Ziekenhuis Gent, Gent, Belgium.
  • Kenzey C; Sydney Children's Hospital, Sydney, Australia.
  • Al-Seraihy A; Children's Hospital at Westmead, Sydney, Australia.
  • van Hasselt PM; Eurocord, Paris, France.
  • Gennery AR; King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia.
  • Gluckman E; Sylvia Toth Center for Multidisciplinary Follow Up After Hematopoietic Cell Transplantation, UMC Utrecht, Utrecht, The Netherlands.
  • Rocha V; Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom; and.
  • Ruggeri A; Eurocord, Paris, France.
  • Kurtzberg J; Eurocord, Paris, France.
  • Boelens JJ; Eurocord, Paris, France.
Blood Adv ; 2(1): 49-60, 2018 01 09.
Article en En | MEDLINE | ID: mdl-29344584
Leukodystrophies (LD) are devastating inherited disorders leading to rapid neurological deterioration and premature death. Hematopoietic stem cell transplantation (HSCT) can halt disease progression for selected LD. Cord blood is a common donor source for transplantation of these patients because it is rapidly available and can be used without full HLA matching. However, precise recommendations allowing care providers to identify patients who benefit from HSCT are lacking. In this study, we define risk factors and describe the early and late outcomes of 169 patients with globoid cell leukodystrophy, X-linked adrenoleukodystrophy, and metachromatic leukodystrophy undergoing cord blood transplantation (CBT) at an European Society for Blood and Marrow Transplantation center or at Duke University Medical Center from 1996 to 2013. Factors associated with higher overall survival (OS) included presymptomatic status (77% vs 49%; P = .006), well-matched (≤1 HLA mismatch) CB units (71% vs 54%; P = .009), and performance status (PS) of >80 vs <60 or 60 to 80 (69% vs 32% and 55%, respectively; P = .003). For patients with PS≤60 (n = 20) or 60 to 80 (n = 24) pre-CBT, only 4 (9%) showed improvement. Of the survivors with PS >80 pre-CBT, 50% remained stable, 20% declined to 60 to 80, and 30% to <60. Overall, an encouraging OS was found for LD patients after CBT, especially for those who are presymptomatic before CBT and received adequately dosed grafts. Early identification and fast referral to a specialized center may lead to earlier treatment and, subsequently, to improved outcomes.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Adrenoleucodistrofia / Trasplante de Células Madre de Sangre del Cordón Umbilical / Leucodistrofia de Células Globoides / Leucodistrofia Metacromática Tipo de estudio: Clinical_trials / Etiology_studies / Guideline / Prognostic_studies / Risk_factors_studies Límite: Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Blood Adv Año: 2018 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Adrenoleucodistrofia / Trasplante de Células Madre de Sangre del Cordón Umbilical / Leucodistrofia de Células Globoides / Leucodistrofia Metacromática Tipo de estudio: Clinical_trials / Etiology_studies / Guideline / Prognostic_studies / Risk_factors_studies Límite: Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Blood Adv Año: 2018 Tipo del documento: Article Pais de publicación: Estados Unidos