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Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia.
Strahm, Brigitte; Loewecke, Felicia; Niemeyer, Charlotte M; Albert, Michael; Ansari, Marc; Bader, Peter; Bertrand, Yves; Burkhardt, Birgit; Da Costa, Lydie M; Ferster, Alina; Fischer, Alexandra; Güngör, Tayfun; Gruhn, Bernd; Hainmann, Ina; Kapp, Friedrich; Lang, Peter; Müller, Ingo; Schulz, Ansgar; Szvetnik, Amina; Wlodarski, Marcin; Noellke, Peter; Leblanc, Thierry; Dalle, Jean-Hugues.
Afiliação
  • Strahm B; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Loewecke F; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Niemeyer CM; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Albert M; Dr. von Hauner University Children's Hospital, Munich, Germany.
  • Ansari M; Hôpital Universitaire de Genève, Geneva, Switzerland.
  • Bader P; Division for Stem Cell Transplantation, Immunology and Intensive Care Medicine, Hospital for Children and Adolescents, University Hospital Frankfurt, Johann Wolfgang Goethe University, Frankfurt, Germany.
  • Bertrand Y; Institut d'Hématologie et Oncologie Pédiatrique, Hôpitaux Civils de Lyon et Université de Lyon, Lyon, France.
  • Burkhardt B; Department of Pediatric Hematology and Oncology, Children's University Hospital, Münster, Germany.
  • Da Costa LM; Assistance Publique-Hôpitaux de Paris, Hematology Diagnostic Laboratory, Robert Debré Hospital, Paris University, Laboratory of Excellence GR-Ex, Paris, France.
  • Ferster A; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels, Belgium.
  • Fischer A; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Güngör T; Department of Immunology, Hematology, Oncology and Stem Cell Transplantation, University Children's Hospital Zurich, Zurich, Switzerland.
  • Gruhn B; Department of Pediatrics, Jena University Hospital, Jena, Germany.
  • Hainmann I; Department of Pediatric Haematology and Oncology, Bonn University Hospital, Bonn, Germany.
  • Kapp F; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Lang P; Department of Pediatric Hematology and Oncology, University Hospital Tübingen, Tübingen, Germany.
  • Müller I; Division for Pediatric Stem Cell Transplantation and Immunology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
  • Schulz A; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany; and.
  • Szvetnik A; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Wlodarski M; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Noellke P; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Leblanc T; Robert Debré Hospital, Groupe Hospitalier, Assistance Publique-Hôpitaux de Paris Nord, Université de Paris, Paris, France.
  • Dalle JH; Robert Debré Hospital, Groupe Hospitalier, Assistance Publique-Hôpitaux de Paris Nord, Université de Paris, Paris, France.
Blood Adv ; 4(8): 1760-1769, 2020 04 28.
Artigo em Inglês | MEDLINE | ID: mdl-32343795
ABSTRACT
Diamond-Blackfan anemia (DBA) is a congenital pure red cell aplasia associated with congenital abnormalities and cancer predisposition. Allogeneic hematopoietic stem cell transplantation (HSCT) can correct the hematological phenotype and is indicated in transfusion-dependent patients. In 70 children reported to the German DBA and French HSCT registries, HSCT was performed from 1985 to 2017. Median age at HSCT was 5.5 years (range, 0.9-17.3 years). Two-thirds of patients (64%) were transplanted from a matched sibling donor (MSD), and most procedures were performed after the year 1999 (73%). Primary engraftment was achieved in all patients. One patient developed secondary graft failure. Cumulative incidence of acute graft-versus-host disease (GVHD) was 24% for °II-IV (95% confidence interval [CI], 16% to 37%) and 7% for °III-IV (95% CI, 3% to 17%); cumulative incidence of chronic GVHD was 11% (95% CI, 5% to 22%). The probability of chronic GVHD-free survival (cGFS) was 87% (95% CI, 79% to 95%) and significantly improved over time (<2000 68% [95% CI, 47% to 89%] vs ≥2000 94% [95% CI, 87% to 100%], P < .01). cGFS was comparable following HSCT from a MSD and an unrelated donor (UD). Of note, no severe chronic GVHD or deaths were reported following MSD-HSCT after 1999. The difference of cGFS in children transplanted <10 years of age compared with older patients did not reach statistical significance (<10 years 90% [95% CI, 81% to 99%] vs 10-18 years 78% [95% CI, 58% to 98%]). In summary, these data indicate that HSCT is efficient and safe in young DBA patients and should be considered if a MSD or matched UD is available. HSCT for transfusion dependency only must be critically discussed in older patients.
Texto completo: Disponível Coleções: Bases de dados internacionais Base de dados: MEDLINE Idioma: Inglês Revista: Blood Adv Ano de publicação: 2020 Tipo de documento: Artigo País de afiliação: Alemanha

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Texto completo: Disponível Coleções: Bases de dados internacionais Base de dados: MEDLINE Idioma: Inglês Revista: Blood Adv Ano de publicação: 2020 Tipo de documento: Artigo País de afiliação: Alemanha