Treatment of severe forms of LPS-responsive beige-like anchor protein deficiency with allogeneic hematopoietic stem cell transplantation

Markus G Seidel; Katrin Böhm; Figen Dogu; Austen Worth; Adrian Thrasher; Benoit Florkin; Aydan İkincioğulları; Anke Peters; Shahrzad Bakhtiar; Marie Meeths; Polina Stepensky; Isabelle Meyts; Svetlana O Sharapova; Laura Gámez-Díaz; Lennart Hammarström; Stephan Ehl; Bodo Grimbacher; Andrew R Gennery; Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation

doi:10.1016/j.jaci.2017.04.023

Treatment of severe forms of LPS-responsive beige-like anchor protein deficiency with allogeneic hematopoietic stem cell transplantation

J Allergy Clin Immunol. 2018 Feb;141(2):770-775.e1. doi: 10.1016/j.jaci.2017.04.023. Epub 2017 May 10.

Authors

Markus G Seidel¹, Katrin Böhm², Figen Dogu³, Austen Worth⁴, Adrian Thrasher⁴, Benoit Florkin⁵, Aydan İkincioğulları³, Anke Peters⁶, Shahrzad Bakhtiar⁷, Marie Meeths⁸, Polina Stepensky⁹, Isabelle Meyts¹⁰, Svetlana O Sharapova¹¹, Laura Gámez-Díaz¹², Lennart Hammarström¹³, Stephan Ehl¹⁴, Bodo Grimbacher¹², Andrew R Gennery¹⁵; Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation

Affiliations

¹ Research Unit of Pediatric Hematology and Immunology, Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Gwraz, Graz, Austria. Electronic address: markus.seidel@medunigraz.at.
² Research Unit of Pediatric Hematology and Immunology, Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Gwraz, Graz, Austria.
³ Department of Pediatric Immunology and Allergy, Ankara University School of Medicine, Ankara, Turkey.
⁴ Institute of Child Health, University College London, London, United Kingdom.
⁵ Immuno-Hémato-Rhumatologie Pédiatrique, Service de Pédiatrie, CHR Citadelle, Liege, Belgium.
⁶ Department of Pediatrics and Adolescent Medicine, Medical Center, University of Freiburg, Freiburg, Germany.
⁷ Division for Pediatric Stem-Cell Transplantation and Immunology, University Hospital Frankfurt, Frankfurt/Main, Germany.
⁸ Childhood Cancer Research Unit, Department of Women's and Children's Health and Clinical Genetics Unit, Department of Molecular Medicine and Surgery, and Center for Molecular Medicine, Karolinska Institutet, Karolinska University Hospital Solna, Stockholm, Sweden.
⁹ Department of Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
¹⁰ Department of Pediatric Immunology, Microbiology and Immunology, University Hospitals Leuven, KU Leuven, Leuven, Belgium.
¹¹ Research Department, Belarusian Research Center for Pediatric Oncology, Hematology and Immunology, Minsk Region, Belarus.
¹² Center for Chronic Immunodeficiency (CCI), Medical Center, University of Freiburg, Freiburg, Germany.
¹³ Division of Clinical Immunology, Department of Laboratory Medicine, Karolinska Institutet at Karolinska University Hospital Huddinge, Stockholm, Sweden.
¹⁴ Department of Pediatrics and Adolescent Medicine, Medical Center, University of Freiburg, Freiburg, Germany; Center for Chronic Immunodeficiency (CCI), Medical Center, University of Freiburg, Freiburg, Germany.
¹⁵ Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.

PMID: 28502825
DOI: 10.1016/j.jaci.2017.04.023

No abstract available

Publication types

Clinical Trial
Letter
Multicenter Study
Research Support, Non-U.S. Gov't

MeSH terms

Adaptor Proteins, Signal Transducing / deficiency*
Allografts
Child
Child, Preschool
Common Variable Immunodeficiency / genetics*
Common Variable Immunodeficiency / immunology*
Common Variable Immunodeficiency / pathology
Common Variable Immunodeficiency / therapy*
Female
Hematopoietic Stem Cell Transplantation*
Humans
Infant
Male

Substances

Adaptor Proteins, Signal Transducing
LRBA protein, human