Quality of life in children and adolescents with hemophilia A: A systematic review and meta-analysis

André Ferreira Azeredo-da-Silva; Bruna Stella Zanotto; Yukie Sato Kuwabara; Verónica Elizabeth Mata

doi:10.1016/j.rpth.2022.100008

Quality of life in children and adolescents with hemophilia A: A systematic review and meta-analysis

Res Pract Thromb Haemost. 2022 Dec 9;7(1):100008. doi: 10.1016/j.rpth.2022.100008. eCollection 2023 Jan.

Authors

André Ferreira Azeredo-da-Silva¹, Bruna Stella Zanotto², Yukie Sato Kuwabara³, Verónica Elizabeth Mata³

Affiliations

¹ HTAnalyze Consulting and Training, Porto Alegre, Brazil.
² National Institute for Health Technology Assessment (Instituto de Avaliacao de Tecnologias da Saude/Instituto Nacional de Ciencia e Tecnologia), Porto Alegre, Brazil.
³ F. Hoffmann-La Roche, São Paulo, Brazil.

Abstract

Background: Various instruments have been used to assess health-related quality of life (HRQoL) in children and adolescents with hemophilia A.

Objective: We systematically reviewed the literature to summarize HRQoL measurement instruments and outcomes in this population.

Methods: MEDLINE, Embase, Cochrane CENTRAL, and LILACS databases were searched. Studies published from 2010 to 2021, reporting HRQoL assessed by generic or hemophilia-specific instruments in individuals aged 0 to 18 years were included. Two independent reviewers performed screening, selection, and data abstraction. Data were meta-analyzed using the generic inverse variance method with the random-effects model for single-arm studies reporting instrument-specific mean total HRQoL scores. Prespecified subgroup meta-analyses were performed. Heterogeneity among studies was assessed using the I ² statistic.

Results: Six instruments were identified in 29 studies meeting the following inclusion criteria: 4 generic instruments (PedsQL [5 studies], EQ-5D-3L [3 studies], KIDSCREEN-52 [1 study], and KINDL [1 study]) and 2 hemophilia-specific instruments (Haemo-QoL [17 studies] and CHO-KLAT [3 studies]). The overall risk of bias was moderate to low. There was a substantial variability in the primary outcome (mean total HRQoL score) among studies using the same instrument (Haemo-QoL), with scores ranging from 24.10 to 89.58 on a scale from 0 to 100 (higher scores indicating higher HRQoL). Meta-regression with 14 studies using the Haemo-QoL questionnaire demonstrated that 79.34% (R ² ) of the observed 94.67% total heterogeneity (I ² ) was explained by the proportion of patients receiving effective prophylactic treatment.

Conclusion: HRQoL assessment in young people with hemophilia A is heterogeneous and context specific. The proportion of patients on effective prophylactic treatment is positively correlated with HRQoL. The review protocol was registered prospectively with PROSPERO (CRD42021235453).

Keywords: adolescent; child; hemophilia A; quality of life; systematic review.