A 30-Year Prospective Follow-Up Study Reveals Risk Factors for Early Death in Cartilage-Hair Hypoplasia.


Journal

Frontiers in immunology
ISSN: 1664-3224
Titre abrégé: Front Immunol
Pays: Switzerland
ID NLM: 101560960

Informations de publication

Date de publication:
2019
Historique:
received: 28 03 2019
accepted: 25 06 2019
entrez: 6 8 2019
pubmed: 6 8 2019
medline: 21 10 2020
Statut: epublish

Résumé

Cartilage-hair hypoplasia (CHH) is a skeletal dysplasia with combined immunodeficiency, variable clinical course and increased risk of malignancy. Management of CHH is complicated by a paucity of long-term follow-up data, as well as knowledge on prognostic factors. We assessed clinical course and risk factors for mortality in a prospective cohort study of 80 patients with CHH recruited in 1985-1991 and followed up until 2016. For all patients we collected additional health information from health records and from the national Medical Databases and Cause-of-death Registry. The primary outcome was immunodeficiency-related death, including death from infections, lung disease and malignancy. Standardized mortality ratios (SMRs) were calculated using national mortality rates as reference. Half of the patients (57%,

Identifiants

pubmed: 31379817
doi: 10.3389/fimmu.2019.01581
pmc: PMC6646460
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1581

Subventions

Organisme : Swedish Childhood Cancer Foundation
Pays : International

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Auteurs

Svetlana Vakkilainen (S)

Pediatric Research Center, Children's Hospital, University of Helsinki and HUS Helsinki University Hospital, Helsinki, Finland.
Folkhälsan Research Center, Institute of Genetics, Helsinki, Finland.
Research Program for Clinical and Molecular Metabolism, Faculty of Medicine, University of Helsinki, Helsinki, Finland.

Mervi Taskinen (M)

Pediatric Research Center, Children's Hospital, University of Helsinki and HUS Helsinki University Hospital, Helsinki, Finland.

Paula Klemetti (P)

Pediatric Research Center, Children's Hospital, University of Helsinki and HUS Helsinki University Hospital, Helsinki, Finland.

Eero Pukkala (E)

Faculty of Social Sciences, Tampere University, Tampere, Finland.

Outi Mäkitie (O)

Pediatric Research Center, Children's Hospital, University of Helsinki and HUS Helsinki University Hospital, Helsinki, Finland.
Folkhälsan Research Center, Institute of Genetics, Helsinki, Finland.
Research Program for Clinical and Molecular Metabolism, Faculty of Medicine, University of Helsinki, Helsinki, Finland.
Department of Molecular Medicine and Surgery, Karolinska Institutet, Clinical Genetics, Karolinska University Hospital, Stockholm, Sweden.
Department of Clinical Genetics, HUSLAB, Helsinki University Hospital, Helsinki, Finland.

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