Observational cohort study of long-term outcomes of liver transplantation in haemophilia.
factor
gene therapy
hemophilia
liver disease
liver transplantation
outcomes research
quality of life
Journal
Haemophilia : the official journal of the World Federation of Hemophilia
ISSN: 1365-2516
Titre abrégé: Haemophilia
Pays: England
ID NLM: 9442916
Informations de publication
Date de publication:
18 Dec 2023
18 Dec 2023
Historique:
revised:
23
10
2023
received:
15
09
2023
accepted:
27
11
2023
medline:
19
12
2023
pubmed:
19
12
2023
entrez:
19
12
2023
Statut:
aheadofprint
Résumé
Gene therapy is now a reality for individuals with haemophilia, yet little is known regarding the quality-of-life impact of factor correction. As few data exist, and recognizing the analogy to liver transplantation (OLTX), we identified OLTX+ and OLTX- men in the ATHNdataset to compare post-OLTX factor VIII and IX on quality of life (QoL) by Haem-A-QoL and PROMIS-29. OLTX- were matched to OLTX+ by age, race, and haemophilia type and severity. Deidentified demographic data, including post-transplant factor levels, genotype and target joint disease were analysed by descriptive statistics. Haem-A-Qol and PROMIS-29 were compared in OLTX+ and OLTX- by student's t-test and univariate regression models. Of 86 people with haemophilia A (HA) or haemophilia B (HB) cared for at 10 haemophilia treatment centers (HTCs), 21 (24.4%) OLTX+ and 65 (75.6%) OLTX- were identified. OLTX+ and OLTX- had a similar frequency of target joint disease (p = .806), HA genotypes, null versus non-null (p = .696), and HIV infection (p = .316). At a median 9.2 years post-OLTX, median FVIII, .63 IU/mL [IQR 0.52-0.97] and FIX, .91 IU/mL [IQR .63-1.32], Haem-A-QoL, PROMIS-29, and HOT scores were comparable. Severe HA/HB had lower post-OLTX 'dealing with haemophilia' scores (p = .022) and higher 'sports and leisure' (p = .010) and 'view of yourself' scores (p = .024) than OLTX+ non-severe participants. Non-caucasian OLTX+ had significantly lower scores in sports and leisure (p = .042), future expectations (p = .021) and total score (p = .010). Nine years after OLTX, QoL is comparable to OLTX-, but significantly better in OLTX+ with severe than non-severe disease and in caucasians than non-caucasians.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : BioMarin Pharmaceutical External Research Program
Organisme : BioMarin Pharmaceutical
Organisme : HRSA Subaward Region III Federal Hemophilia Treatment Centers
ID : 230HC MC24050-11-00
Organisme : HRSA Subaward Region III Federal Hemophilia Treatment Centers
ID : 3209610516-P
Organisme : PA-DOH State Support of Hemophilia Center of Western Pennsylvania
Organisme : Pennsylvania Department of Health
ID : SAP #4100079797
Informations de copyright
© 2023 John Wiley & Sons Ltd.
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