INTRODUCTION:Currently, no quality-of-life instrument exists that captures the full experience of the mental health outlook (MHO), a coreHEM core outcome, in people with haemophilia, including the potential transformational experience of receiving gene therapy. AIM:To describe the methods used to develop a content validated patient-reported outcome measure (PROM) that measures MHO for people with haemophilia. METHODS:A conceptual framework, developed from a literature/evidence review, was used to create an interview guide and draft a questionnaire. Males aged 15 or older with severe/moderate haemophilia were eligible to participate in concept elicitation or cognitive debriefing interviews. The conceptual framework was refined based on a thematic analysis of concept elicitation interviews and PROM items were developed from the conceptual framework. Cognitive debriefing sessions that prioritised relevance and understanding of the PROM were held in two rounds; items were updated iteratively. RESULTS:A conceptual framework with five domains (stigma, anxiety, depression, life interference and identity) was constructed from over 300 identified MHO concepts. Fifty-three participants took part in interviews. After 32 concept elicitation interviews, the framework was updated by including eight new sub-concepts and eliminating two. Updates to the questionnaire included items added or removed and improved wording. The final coreHEM MHO PROM has 26 questions in two sections (general mental health associated with haemophilia, and a gene therapy section). CONCLUSIONS:The instrument is content-validated and can be used as an exploratory outcome. MHO scores can be measured and compared to give more insight into patient quality of life.
BACKGROUND:A multistakeholder core outcome set created for asthma trials showed that asthma-specific quality of life (QoL) was a critically meaningful outcome. However, the definition and measurement methods were undetermined. The adverse effects (AEs) of corticosteroids may be a vital clinical trial outcome. Nevertheless, the AE burden from the patient perspective has not yet been elucidated in an asthma population. OBJECTIVE:To characterize patient burden of AEs in oral (OCS) and inhaled corticosteroids (ICS) and how this relates to QoL within an asthma population. METHODS:We used a convergent parallel mixed-methods design with quantitative surveys of known ICS and OCS AEs that were distributed through the Allergy & Asthma Network database, social channels, and the Asthma UK newsletter. Participants rated the AEs that were (1) most burdensome and (2) most desired to be eliminated. Qualitative interviews and focus groups were performed to better understand patient views on barriers reported in the quantitative data, and to identify patient-important barriers that were not a part of the quantitative survey. RESULTS:The 3 most burdensome AEs for OCS were bone mineral density, infectious complications, and weight gain, whereas weight gain was the most desired to be eliminated. The 3 most burdensome AEs for ICS were pneumonia, hoarse voice, and oral thrush, with concordant results for the most desired to be eliminated. In the focus groups, OCS AEs were concordant with quantitative findings. Focus groups identified unmeasured psychosocial effects, such as embarrassment. CONCLUSION:The most burdensome AEs may not be those that would cause patients to stop therapy. Furthermore, qualitative focus groups suggest a psychosocial burden associated with ICS, which needs further investigation.
This program will provide a foundation in the principles of conducting embedded pragmatic clinical trials (ePCTs) in health care systems for people living with dementia and their care partners with an emphasis on integrating health equity throughout all aspects of study design. This program consists of 13 online modules that introduce learners to the essential features of a dementia care pragmatic trial embedded in a clinical care setting. The anticipated time to completion is approximately 7 hours, and learners will receive a certificate for completion.
coreHEM, a core outcome set (COS) for hemophilia gene therapy (GT) was identified by multi-stakeholder consensus (Iorio, Haemophilia, 2018). When consistently measured, the COS will inform a value-based approach to evaluating GT. First steps to determining appropriate measurements for each outcome were previously reported (ISPOR 2018). One outcome, mental health (MH) outlook, was regarded as highly important to capture the potential transformational impact of GT, however no instruments that measured MH when receiving a novel treatment were identified. Suggestions were for an instrument with a summary score evaluating concepts that contribute to MH along with qualitative data to capture the impact of a potential cure. coreHEM has been mapped to a hemophilia value framework (O’Mahony, Haemophilia, 2018) and while broad overlap was reported, gaps were identified for the MH outlook outcome; the group encouraged prioritization of this outcome for data collection/publication (Skinner, EAHAD, 2022). coreHEM MH is a patient-reported outcome measure (PROM) development project initiated to provide a content-validated instrument that can be used to measure this outcome. A conceptual framework and interview guide for MH outlook in hemophilia were developed from a literature/evidence review. Concept elicitation interviews were used to identify concepts and refine the framework. PROM items were developed from priority concepts. Thirty-two people with hemophilia (PWH) participated in concept elicitation interviews. The framework includes five domains (anxiety, depression, stigma, life interference, and identity). The PROM is currently undergoing cognitive debriefing (expected n=30 PWH) with a focus on understandability and relevance of items. A content-validated instrument will soon be available for use as an exploratory outcome in hemophilia GT clinical trials and real-world settings. Its use will allow for collection of MH outlook data, meeting the needs of the value framework and helping fulfill the intentions of the COS.
