Author(s): Amanda Simpson, BBA, DGI Clinical; Jonathan Roberts, MD, Bleeding and Clotting Disorders Institute; Justin Stanley, BS, DGI Clinical; Kari Knox, BS, DGI Clinical; Kenneth Rockwood, MD, DGI Clinical; Michael Denne, PharmD, Takeda Pharmaceuticals, USA

Research Category: Quality of Life/Outcomes Research

Description:

Objectives:

Many outcome measures in hemophilia lack sensitivity and few are patient-centric. With patient input, our group developed GAS-H?m: a hemophilia-specific goal menu to facilitate Goal Attainment Scaling (GAS). GAS-H?m proved valid and feasible in a multicenter feasibility study (Roberts et al. Haemophilia 2018;4:e199-e206). We refined the menu, employing a multi-disciplinary research team, incorporating patient and clinician feedback. With this input from individuals affected by hemophilia, our goal was to produce a truly patient-centric tool, Goal-H?m. We evaluated whether Goal-H?m is patient-centric and relevant to the challenges faced by people with hemophilia.

Methods:

This patient/caregiver feedback process recruited 38 adults with hemophilia or caregivers of children with hemophilia (Table 1). Participants reviewed menu content for clarity, comprehensibility, comprehensiveness, and possible application in their lives. The focus group and one-on-one interviews were semi-structured, recorded, and transcribed verbatim. Transcripts were systematically analyzed in a framework analysis to extract emergent themes.

Summary:

Based on the participants' feedback, from the original menu, 14 goals were reduced, two expanded, two added, and three removed. Goal descriptions were simplified so that each concisely described just one problem area (Table 2). Most descriptions (554/635; 87.2%) were reported as 'clear and relevant' by all respondents (online survey). Participants from one-on-one interviews endorsed the menu's language for being patient-centric. They noted that goal-setting could encourage those with hemophilia to be proactive about their health. A common theme for clinic use was the patients' desire to review the menu prior to the GAS interview, preferably in the waiting room.

Conclusions:

Participants endorsed the clarity and relevance of the revised menu and expressed enthusiasm for further interaction with the tool, driven by the personalized approach it affords. Patient centered tools such as this have the potential to detect clinically meaningful changes for both clinicians and individuals affected by hemophilia, while improving patient outcomes.