Background and Objective:
Standard outcome measures in hemophilia, such as the annual bleeding rate, have inherent limitations in both clinical practice and in research, including lack of both sensitivity and personalization. Goal attainment scaling (GAS) is a method for evaluating outcome that is patient-centered and sensitive to change in individuals and populations; collaborative goal setting may also be viewed through the biopsychosocial lens as a potential resource to strengthen disease-related coping. GAS has been used successfully in several disease areas. However, feasibility can be an issue. To address this, standardized goal attainment menus can be developed that preserve individualization, while allowing greater ease of use by non-experts. We initiated a process to develop a novel outcome measure in hemophilia, the GAS-Hem, by creating a goal attainment menu for patients living with hemophilia.
Methods:
We conducted 2 multidisciplinary workshops with participants from medicine, nursing, social work, and physical therapy (n=12). During the first workshop, we developed a list of goal areas, each with an associated set of descriptors of attainment levels. At the second workshop, a second group of experts (n=8) critically reviewed each goal to evaluate its importance and relevance for inclusion in the GAS-Hem.
Summary:
28 goal areas with associated descriptors of attainment levels were developed, covering 3 broad categories: ability to manage hemophilia, ability to recognize and treat complications, and ability to cope with the impact of hemophilia on daily life. Descriptors incorporated several key parameters for each goal (eg. skill level, desire for change, utilization of available resources) (Table).
Conclusions:
We developed a novel, patient- centered outcome measure for patients with hemophilia. A comprehensive, preliminary list of goal areas and attainment levels was successfully created. The next step will be to incorporate feedback from patients and families, after which a feasibility study will be conducted to evaluate content and construct validity and overall ease of use.
Table. Examples of Goal Areas and Descriptors
