Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults
Language English Country United States Media print
Document type Journal Article, Multicenter Study
PubMed
32614953
DOI
10.1182/blood.2020005590
PII: S0006-4971(20)61750-5
Knihovny.cz E-resources
- MeSH
- Granulomatous Disease, Chronic pathology therapy MeSH
- Child MeSH
- Adult MeSH
- Transplantation, Homologous MeSH
- Infant MeSH
- Middle Aged MeSH
- Humans MeSH
- Survival Rate MeSH
- Adolescent MeSH
- Young Adult MeSH
- Follow-Up Studies MeSH
- Child, Preschool MeSH
- Transplantation Conditioning MeSH
- Prognosis MeSH
- Retrospective Studies MeSH
- Hematopoietic Stem Cell Transplantation methods mortality MeSH
- Check Tag
- Child MeSH
- Adult MeSH
- Infant MeSH
- Middle Aged MeSH
- Humans MeSH
- Adolescent MeSH
- Young Adult MeSH
- Male MeSH
- Child, Preschool MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Multicenter Study MeSH
Chronic granulomatous disease (CGD) is a primary immunodeficiency resulting in life-threatening infections and inflammatory complications. Allogeneic hematopoietic cell transplantation (allo-HCT) can cure the disease, but the indication to transplant remains controversial. We performed a retrospective multicenter study of 712 patients with CGD who underwent allo-HCT transplantation from March 1993 through December 2018. We studied 635 children (aged <18 years) and 77 adults. Median follow-up was 45 months. Median age at transplantation was 7 years (range, 0.1-48.6). Kaplan-Meier estimates of overall survival (OS) and event-free survival (EFS) at 3 years were 85.7% and 75.8%, respectively. In multivariate analysis, older age was associated with reduced survival and increased chronic graft-versus-host disease. Nevertheless, OS and EFS at 3 years for patients ≥18 years were 76% and 69%, respectively. Use of 1-antigen-mismatched donors was associated with reduced OS and EFS . No significant difference was found in OS, but a significantly reduced EFS was noted in the small group of patients who received a transplant from a donor with a >1 antigen mismatch. Choice of conditioning regimen did not influence OS or EFS. In summary, we report an excellent outcome after allo-HCT in CGD, with low incidence of graft failure and mortality in all ages. Older patients and recipients of 1-antigen-mismatched grafts had a less favorable outcome. Transplantation should be strongly considered at a younger age and particularly in the presence of a well-matched donor.
Children's Cancer Research Institute St Anna Children's Hospital Vienna Austria
Clinic of Hematology University Hospital Zurich Switzerland
Department of Bone Marrow Transplantation Hadassah University Hospital Jerusalem Israel
Department of Hematology Royal Hospital for Children Glasgow United Kingdom
Department of Paediatric Hematology and Oncology University Hospital Motol Prague Czech Republic
Department of Pediatrics and Adolescent Medicine University Medical Center Ulm Ulm Germany
Department of Pediatrics UMC Utrecht University Utrecht Utrecht The Netherlands
Department Pediatric Hematology Oncology Hannover Medical University Hannover Germany
European Society for Blood and Marrow Transplantation Data Office Leiden Leiden The Netherlands
Great Ormond Street Hospital for Children London United Kingdom
Hospital Israelita Albert Einstein São Paulo Brazil
Instituto da Criança Hospital das Clínicas da Universidade de São Paulo São Paulo Brazil
Istituto di Ricovero e Cura a Carattere Scientifico Ospedale Pediatrico Bambino Gesù Rome Italy
Oncohematology and Bone Marrow Transplantation Unit Ospedale dei Bambini Brescia Italy
Pediatric BMT Centre Ege University Izmir Turkey
Pediatric Hematology Oncology Fondazione IRCCS Policlinico San Matteo Pavia Italy
Princess Maxima Center Utrecht The Netherlands
Unité d'Immunologie et d'Hématologie Hôpital Necker Paris France
Willem Alexander Kinderziekenhuis Leiden University Medical Center Leiden The Netherlands
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