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Exploring the relationship between condition severity and health-related quality of life in people with haemophilia A across Europe: a multivariable analysis of data from the CHESS II study.
Ferri Grazzi, Enrico; Hawes, Charles; Camp, Charlotte; Hinds, David; O'Hara, Jamie; Burke, Tom.
Afiliación
  • Ferri Grazzi E; HCD Economics, Keckwick Lane, Daresbury, Cheshire, WA4 4FS, UK. enrico.ferrigrazzi@primeglobalpeople.com.
  • Hawes C; BioMarin Europe, London, UK.
  • Camp C; BioMarin Europe, London, UK.
  • Hinds D; BioMarin Pharmaceutical, San Rafael, CA, USA.
  • O'Hara J; HCD Economics, Keckwick Lane, Daresbury, Cheshire, WA4 4FS, UK.
  • Burke T; University of Chester, Chester, UK.
Health Qual Life Outcomes ; 22(1): 58, 2024 Jul 29.
Article en En | MEDLINE | ID: mdl-39075533
ABSTRACT

BACKGROUND:

Haemophilia A (HA; Factor VIII deficiency) is a congenital X-linked bleeding disorder characterized by trauma-related or spontaneous bleeding events, most notably arising within the intraarticular space and resulting in chronic inflammation and degeneration of affected joints. Endogenous clotting factor activity relative to normal levels determines the severity of HA symptoms, as mild (> 5-40%), moderate (1-5%), or severe (< 1%). Within the current environment of rapid evolution in HA management, we seek to understand the interplay of condition severity and health-related quality of life (HRQoL) to characterise and differentiate unmet needs among people with HA (PwHA).

METHODS:

A generalised linear regression model (GLM) was developed to explore the relationship between HA severity and EQ-5D-5 L index score from adult HA patients sampled in the "Cost of Haemophilia across Europe - a Socioeconomic Survey II" (CHESS II) cross-sectional, retrospective burden of illness study among adults with hereditary haemophilia A or B from eight European countries. HA patients of any severity with no active inhibitors during the 12 months prior to data capture and a completeEQ-5D-5 L response were included. A base GLM model was specified with covariates for demographic and clinical characteristics (age, body mass index, country, employment, HA severity, annual bleeding rate, problem joints, and chronic pain).

RESULTS:

Of 381 evaluable patients, 221 (58.0%) had severe HA, 96 (25.2%) had moderate HA, and 64 (16.8%) had mild HA. Among the covariates included in the GLM model and after controlling for haemophilia-related outcomes, a significant association was observed between mild HA and higher EQ-5D-5 L index score (average marginal effects, 0.084; p = 0.016) relative to severe HA. Patient country of residence and magnitude of HA-related chronic pain were also associated with significant differences in index scores, with the latter showing a negative relationship with HRQoL outcomes.

CONCLUSIONS:

Condition severity and chronic pain are significant predictors of HRQoL in PwHA. Durable bleeding protection and effective management of chronic pain have the potential to address unmet treatment needs in this population.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Calidad de Vida / Índice de Severidad de la Enfermedad / Hemofilia A Límite: Adolescent / Adult / Aged / Female / Humans / Male / Middle aged País/Región como asunto: Europa Idioma: En Revista: Health Qual Life Outcomes Asunto de la revista: SAUDE PUBLICA Año: 2024 Tipo del documento: Article País de afiliación: Reino Unido Pais de publicación: Reino Unido

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Calidad de Vida / Índice de Severidad de la Enfermedad / Hemofilia A Límite: Adolescent / Adult / Aged / Female / Humans / Male / Middle aged País/Región como asunto: Europa Idioma: En Revista: Health Qual Life Outcomes Asunto de la revista: SAUDE PUBLICA Año: 2024 Tipo del documento: Article País de afiliación: Reino Unido Pais de publicación: Reino Unido