Using an Online Modified Delphi Approach to Engage Patients andCaregivers in Determining the Patient-Centeredness of Duchenne MuscularDystrophy Care Considerations

摘要

To determine the patient-centeredness of endocrine and bonehealth Duchenne muscular dystrophy (DMD) care considerations using the RAND/PPMDPatient-Centeredness Method (RPM), which is a novel, online, modified-Delphiapproach to engaging patients and caregivers in clinical guideline development. We solicited input on the patient-centeredness of careconsiderations from 28 individuals with DMD and 94 caregivers, randomly assignedto 1 of 2 mixed panels. During a 3-round online modified-Delphi process,participants rated the importance and acceptability of 19 DMD careconsiderations (round 1), reviewed and discussed the initial results (round 2),and revised their original ratings (round 3). Patient-centeredness wasoperationalized as importance and acceptability of recommendations. Weconsidered a care consideration to be patient-centered if both panels deemed itimportant and acceptable. Ninety-five panelists (78%)participated in this study. Of these, 88 (93%) participated in round 1, 74 (78%)in round 2, and 56 (59%) in round 3. Panelists deemed 12 care considerations tobe patient-centered: 3 weight management, 3 bone health, 4 vertical growth, and2 puberty recommendations. Seven care considerations did not meetpatient-centeredness criteria. Common reasons were lack of evidence specific toDMD and concerns about insurance coverage, access to treatment, and patientsafety. Using the RPM, Duchenne families consideredmost care considerations to be patient-centered. Besides being clinicallyappropriate, these considerations are likely to be consistent with thepreferences, needs, and values of Duchenne families. While all relevant careconsiderations should be discussed during patient-provider encounters, thosethat did not meet patient-centeredness criteria in particular should becarefully considered as part of joint decision making between Duchenne familiesand their providers. HSRProj 20163126.