Clinical outcomes in hemophilia: Towards development of a core set of standardized outcome measures for research

Abstract

Introduction A lack of uniformity in the choice of outcome measurement in hemophilia care and research has led to studies with incomparable results. We identified a need to define core outcome measures for use in research and clinical care of persons with hemophilia. Objective To move toward a core set of outcome measures for the assessment of persons with hemophilia in research and practice. Methods A modified nominal groups process was conducted with an international group of hemophilia experts, including persons with hemophilia as follows. Step 1: item generation for all potential outcome measures. Step 2: survey where respondents voted on the relative importance and usefulness of each item. Steps 3/4: 2‐day meeting where attendees voted for items they valued, followed by open discussion and a second round of voting. Step 5: survey where respondents selected their top five items from those with >50% agreement at the meeting. Results The highest ranked items for the pediatric core set (% agreement) are treatment satisfaction (92.7%), joint health (83.3%), a measure of access to treatment (82.5%), a measure of treatment adherence (72.5%), and generic performance based physical function (72.1%). The highest ranked items for the adult core set (% agreement) are total bleeding events (88.1%), EuroQol five dimensions (85.4%), treatment adherence (82.1%), joint health (79.1%), and number/location of bleeds per unit time (78.6%). Conclusion This process generated a list of preferred outcome measures to consider for assessment in persons with hemophilia. This information now requires refinement to define optimal core sets for use in different clinical/research contexts.

Patient‐relevant health outcomes for hemophilia care: Development of an international standard outcomes set

“…Four panels were involved: the coordinating core team, a steering group, the Patients and Health Care Professionals Panel, and the International Academic Council (Table 1). The coordinating core team extracted lists of the definitions, health outcomes, and risk-adjustment variables from the literature search; earlier outcomes initiatives 13,14,16,20 ; ICHOM standard sets 19 ; and clinical practice. The steering group and Patients and Health Care Professionals Panel members individually voted for the most relevant health outcomes and risk adjustment variables before each web-based meeting.…”

“…Scoring of instruments led to the selection of the recommended outcome measurement instruments (Tables S3 and S9). 34 ; the Scientific and Standardization Committee/International Society on Thrombosis and Haemostasis definitions in hemophilia project group, 2 an expert review on tools for outcome measurement 15,16 and systematic reviews on the psychometric properties of hemophilia-specific instruments for joint health, activities and participation, and healthrelated quality of life. [24][25][26] Improving value for persons with hemophilia should be the overarching goal of health care delivery.…”

“…Outcome sets evolve over time and build on earlier outcome sets. [13][14][15][16] The recently published patient-relevant outcomes framework for hemophilia care 13 required broader validation by input from representatives and hemophilia care providers of persons with hemophilia . 17 Therefore, we assembled stakeholders including persons with hemophilia and their representatives, hemophilia care providers with expertise in various disciplines, and experts in value-based health care to identify a globally applicable standard set of patient-relevant health outcomes for all persons living with hemophilia.…”

“…Outcome sets evolve over time and build on earlier outcome sets 13‐16 . The recently published patient‐relevant outcomes framework for hemophilia care 13 required broader validation by input from representatives and hemophilia care providers of persons with hemophilia 17 .…”

Measuring the impact of hemophilia on families: Development of the Hemophilia Family Impact Tool (H‐FIT)

“…5 Recently, a group of hemophilia health care providers (HCPs) and other experts in the field agreed that measures of HRQoL were important to consider as an outcome measure that should be included in clinical trials. 6 There are several hemophilia-specific questionnaires that are currently used to measure HRQoL in boys aged <18 years with hemophilia; the Canadian Hemophilia Kids' Life Assessment Tool (CHO-KLAT) and the Quality of Life Assessment instrument for children with hemophilia are the two most commonly used and well-studied tools. 7 These tools have child self-report to measure HRQoL in children aged ≥7 years, and parent-proxy for children aged <7 years.…”