Cross‐cultural development and psychometric evaluation of a patient‐reported health‐related quality of life questionnaire for adults with haemophilia

Abstract

Co-morbidities of haemophilia, such as arthropathy and blood-borne infections, can adversely affect the quality of life of adult patients with haemophilia. The purpose of this study was to develop and validate a haemophilia-specific health-related quality of life questionnaire for adults (HAEMO-QoL-A). Subjects with varying severities of haemophilia completed the HAEMO-QoL-A at baseline and 4 weeks. Other assessments included the SF-36 and Health Assessment Questionnaire - Functional Disability Index (HAQ-FDI). Two-hundred and twenty-one participants completed the 41-item HAEMO-QoL-A covering six domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact and Treatment Concerns) and four independent items. Internal consistency was good-to-excellent (Cronbach's alpha-range: 0.75-0.95). Test-retest reproducibility was good, with intraclass correlation coefficients >0.80 except for the Emotional Impact domain (0.79). Concurrent validity between the HAEMO-QoL-A total and subscale scores and all SF-36 subscale scores were generally good (correlations range: 0.13-0.87). Significant correlations between the HAEMO-QoL-A and the HAQ-FDI ranged from -0.14 to -0.69. There were non-significant correlations with the Treatment Concerns subscale and with the Worry subscale. The HAEMO-QoL-A discriminated significantly between adults with haemophilia by severity and HIV status. The Physical Functioning subscale discriminated between patients receiving prophylactic or on-demand therapy. The HAEMO-QoL-A is a valid and reliable instrument for assessing quality of life in haemophilia patients.

Effect of Acute Bleeding on Daily Quality of Life Assessments in Patients with Congenital Hemophilia with Inhibitors and Their Families: Observations from the Dosing Observational Study in Hemophilia

“…Hemophilia-specific tests may provide more sensitive analyses that can supplement the use of standard measures. There are only a few disease-specific QOL parameters, however, and most have been recently developed [23][24][25][26]. The hemophilia-specific health-related QOL questionnaire was originally made available for use among children and adolescents [23,25], and a separate version has been developed independently for adults [26].…”

BE EMPOWERED, A Specialty Pharmacy Education Program for Hemophilia B Patients, Impacts Adult Joint Bleeds and Pediatric Use of RICE

“…RICE is part of the recommended comprehensive approach to treating bleeding events and is included in the guidelines for the management of hemophilia. 7 Quality of life (QoL) was assessed at the beginning of the study in the baseline survey and at the end of the program in the follow-up survey. The hemophilia-specific QoL tool (HAEMO-QoL-A) was used for adults because this tool has demonstrated excellent internal consistency, high reliability, and construct validity.…”

Efficacy and safety of secondary prophylactic vs. on‐demand sucrose‐formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13‐month crossover study

“…Joint function was assessed by the clinician (in the absence of an acute bleeding episode) at baseline, month 6 and month 13 using the physical examination score of the Gilbert scale [17]. Patient QoL was assessed at baseline, month 6 and month 13 using the Haemo-QoL-A questionnaire, a validated hemophilia A-specific instrument covering six areas (physical functioning, role functioning, worry, consequences of bleeding, positive affect, and treatment concerns) [18]. Health economic data (e.g.…”