Purpose. To determine the patient-centeredness of endocrine and bone
health Duchenne muscular dystrophy (DMD) care considerations using the RAND/PPMD
Patient-Centeredness Method (RPM), which is a novel, online, modified-Delphi
approach to engaging patients and caregivers in clinical guideline development.
Methods. We solicited input on the patient-centeredness of care
considerations from 28 individuals with DMD and 94 caregivers, randomly assigned
to 1 of 2 mixed panels. During a 3-round online modified-Delphi process,
participants rated the importance and acceptability of 19 DMD care
considerations (round 1), reviewed and discussed the initial results (round 2),
and revised their original ratings (round 3). Patient-centeredness was
operationalized as importance and acceptability of recommendations. We
considered a care consideration to be patient-centered if both panels deemed it
important and acceptable. Results. 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 to
be patient-centered: 3 weight management, 3 bone health, 4 vertical growth, and
2 puberty recommendations. Seven care considerations did not meet
patient-centeredness criteria. Common reasons were lack of evidence specific to
DMD and concerns about insurance coverage, access to treatment, and patient
safety. Conclusions. Using the RPM, Duchenne families considered
most care considerations to be patient-centered. Besides being clinically
appropriate, these considerations are likely to be consistent with the
preferences, needs, and values of Duchenne families. While all relevant care
considerations should be discussed during patient-provider encounters, those
that did not meet patient-centeredness criteria in particular should be
carefully considered as part of joint decision making between Duchenne families
and their providers. Study Registration: HSRProj 20163126.