EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity

Bone Marrow Transplantation, vol. 56, pp. 2052–2062

Abstract

Inborn errors of immunity (IEI) are a group of rare heterogeneous diseases. Currently, more than 400 monogenetic IEI have been identified and increasingly a genetic diagnosis can be made in patients with an immune deficiency disorder [ 1 ]. Patients may present with a variety of clinical symptoms including a broad spectrum of infections, inflammatory manifestations, auto-immune phenomena and malignant diseases. Treatment by hematopoietic stem cell transplantation (HSCT) is increasingly successful [ 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 ] and the joint EBMT/ESID Inborn Errors Working Party (IEWP) has played a pivotal role designing and developing common HSCT guidelines, which have contributed to this success.

Authors 12

1. Arjan C. Lankester corresponding Aachen

   Leiden University Medical Center · Willem-Alexander Kinderziekenhuis

   Affiliation as printed

   Willem-Alexander Children's Hospital, Pediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, The Netherlands. a.lankester@lumc.nl

   Willem-Alexander Children’s Hospital, Pediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, The Netherlands

2. Michael H. Albert

   LMU Klinikum · Ludwig-Maximilians-Universität München

   Affiliation as printed

   Department of Pediatrics, Dr. von Hauner Children's Hospital, University Hospital, LMU, Munich, Germany

   Department of Pediatrics, Dr. von Hauner Children’s Hospital, University Hospital, LMU, Munich, Germany

3. Claire Booth

   Great Ormond Street Hospital · Great Ormond Street Hospital for Children NHS Foundation Trust · University College London

   Affiliation as printed

   Great Ormond Street (GOS) Hospital for Children NHS Foundation Trust and University College London GOS Institute of Child Health, London, UK

4. Andrew Richard Gennery

   Great North Children's Hospital · Newcastle University

   Affiliation as printed

   Translational and Clinical Research Institute, Newcastle University, and Paediatric Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne, UK

   Translational and Clinical Research Institute, Newcastle University, and Paediatric Haematopoietic Stem Cell Transplant Unit, Great North Children’s Hospital, Newcastle upon Tyne, UK

5. Tayfun Güngör

   University Children's Hospital Zurich

   Affiliation as printed

   Department of Hematology, Oncology, Immunology, Gene-therapy and Stem Cell Transplantation, and Children's Research Center (CRC), University Children's Hospital, ZurichZurich, Switzerland

   Department of Hematology, Oncology, Immunology, Gene-therapy and Stem Cell Transplantation, and Children’s Research Center (CRC), University Children’s Hospital, ZurichZurich, Switzerland

6. Manfred Hoenig

   University Hospital Ulm

   Affiliation as printed

   Department of Pediatrics, University Medical Center Ulm, Ulm, Germany

7. Emma C. Morris

   The Royal Free Hospital · University College London

   Affiliation as printed

   Institute of Immunity and Transplantation, University College London Hospitals NHS FT and Royal Free London Hospitals NHS FT, London, UK

8. Despina Moshous

   Hôpital Necker-Enfants Malades · Inserm · Université Paris Cité · Assistance Publique – Hôpitaux de Paris · Institut des Maladies Génétiques Imagine

   Affiliation as printed

   Institut Imagine, INSERM UMR1163, Laboratory of genomic Dynamics in the Immune system, Paris, France

   Unité d'immune-Hematologie et Rhumatologie Pédiatrique, Hôpital Necker Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France

   Université de Paris, Paris, France

   Unité d’immune-Hematologie et Rhumatologie Pédiatrique, Hôpital Necker Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France

9. Bénédicte Neven

   Hôpital Necker-Enfants Malades · Inserm · Université Paris Cité · Assistance Publique – Hôpitaux de Paris · Institut des Maladies Génétiques Imagine

   Affiliation as printed

   Institut Imagine, INSERM UMR1163, Laboratory of Immunogenetics of Pediatric Autoimmune Diseases, Paris, France

   Unité d'immune-Hematologie et Rhumatologie Pédiatrique, Hôpital Necker Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France

   Université de Paris, Paris, France

   Unité d’immune-Hematologie et Rhumatologie Pédiatrique, Hôpital Necker Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France

10. Ansgar Schulz

    University Hospital Ulm

    Affiliation as printed

    Department of Pediatrics, University Medical Center Ulm, Ulm, Germany

11. Mary Anne Slatter

    Great North Children's Hospital · Newcastle University

    Affiliation as printed

    Translational and Clinical Research Institute, Newcastle University, and Paediatric Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne, UK

    Translational and Clinical Research Institute, Newcastle University, and Paediatric Haematopoietic Stem Cell Transplant Unit, Great North Children’s Hospital, Newcastle upon Tyne, UK

12. Paul A. Veys

    Great Ormond Street Hospital · Great Ormond Street Hospital for Children NHS Foundation Trust · University College London

    Affiliation as printed

    Great Ormond Street (GOS) Hospital for Children NHS Foundation Trust and University College London GOS Institute of Child Health, London, UK