Using input from a variety of patient and caregiver sources, the patient engagement tool and patient-reported outcomes (PRO) instrument, GOAL-Hēm, was revised to be more patient-centric, thus enhancing content validity, according to the results of a study published in the journal Research and Practice in Thrombosis and Haemostasis.

“GOAL-Hēm has the potential to improve patient outcomes through enhanced patient engagement with treatment and treaters, improved detection of clinically meaningful change for both clinicians and people with hemophilia, and development of patient goal-setting and goal-attainment data for use in PRO research,” the authors wrote.

The validity and feasibility of GOAL-Hēm was previously demonstrated in a 12-week, prospective study. However, feedback from the feasibility study suggested there was a need to revise the GOAL-Hēm menu to make it more relevant to people with hemophilia.

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The study included 38 patients and caregivers who participated in either an online survey, a 60- to 90-minute individual interview, or a 3-hour focus group between April and June 2018. Participants were asked to rate goal title areas and descriptors as clear or unclear and relevant or not relevant. Those who participated in the interview or focus group were able to provide additional qualitative assessments and specific recommendations for changes.

At the time of the assessment, GOAL-Hēm included a total of 635 descriptors, comprising 19 goals with 228 descriptors for children and 29 goals with 407 descriptors for adults.

After feedback from all patient/caregiver sources, 58% of goals were modified and 10% were removed. Several goals were added to the menu, including 2 goals for children and 1 for adults. This translated to a total of 16 goals for children and 22 goals for adults on the revised GOAL-Hēm menu.

A number of changes were made to descriptors, with 45% removed and 30% modified, reducing the number of descriptors by 34% for children and 46% for adults. There were 42 new descriptors added, including 22 to the child menu and 20 to the adult menu. The revised GOAL-Hēm tool now includes 150 descriptors in the child menu and 218 in the adult menu.

The authors suggested that “using this tool can help refine and further shape hemophilia treatment center culture around the importance of eliciting and more clearly defining patients’ goals in developing and executing treatment plans.” They added that “GOAL-Hēm can bridge an important gap in our ability to measure and thereby improve patient-centric hemophilia care.”

Disclosures: This study was funded by Takeda Development Center Americas, Inc.


Roberts JC, Recht M, Gonzales SE, et al. Incorporating the patient voice and patient engagement in GOAL-Hēm: Advancing patient-centric hemophilia care. Res Pract Thromb Haemost. 2022;6:e12655. doi: 10.1002/rth2.12655