title: Stem cell transplantation for primary immunodeficiencies: the European experience.
abstract: OBJECTIVE
  Primary immunodeficiencies (PIDs) constitute a heterogeneous group of inherited disorders affecting the development and/or function of the immune system. This review focuses on the recent advances in hematopoietic stem cell transplantation (HSCT) for PIDs, as it remains the only potentially curative option for many of these diseases.
  
  
  RESULTS
  We report on the most recent HSCT European results and suggest some opportunities for better treatment of certain PIDs. Progress on gene therapy is also discussed, as it emerges as an interesting option for PIDs management.
  
  
  CONCLUSIONS
  Progress in the treatment of primary immune deficiency with HSCT requires a better understanding of the pathophysiology and specificity of each of these diseases, allowing us to determine the best options in terms of donor, conditioning regimen, modification of the allograft and immunosuppressive therapy. Alternative therapies - such as gene therapy - emerge as an interesting option for some PIDs.
authors.affiliation: aBiotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris bBiotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM cParis Descartes - Sorbonne Paris Cité University, Imagine Institute dINSERM UMR 1163, Laboratory of Human Lymphohematopoiesis eImmunology and Pediatric Hematology Department, Assistance Publique-Hôpitaux de Paris fCollège de France, Paris, France.
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana
authors.forename: Fabien
authors.initials: F
authors.lastname: Touzot
authors.forename: Despina
authors.initials: D
authors.lastname: Moshous
authors.forename: Bénédicte
authors.initials: B
authors.lastname: Neven
authors.forename: Stéphane
authors.initials: S
authors.lastname: Blanche
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
mesh_terms: D064591:Allografts; D017074:Common Variable Immunodeficiency / Q000276:immunology / Q000628:therapy*; D005060:Europe; D005062:European Union; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans