Longitudinal reliability of outcome measures in patients with Duchenne muscular dystrophy.

INTRODUCTION: The definition of reliable outcome measures is of increasing interest in patients with Duchenne muscular dystrophy (DMD). METHODS: In this retrospective study, we analyzed the longitudinal reliability of clinical and radiological endpoints in 29 ambulant patients with DMD. Clinical outcome measures included motor function measure (MFM) and timed function tests, while quantitative MRI data were mean fat fraction (MFF) and T2 relaxation time of thigh muscles. Statistical analysis was based on 3-, 6-, and 12-month follow-up data. RESULTS: Quantitative MRI using the MFF was the most sensitive and powerful marker of disease progression with a sample size of four at 1-year follow-up, followed by the D1 domain of MFM (standing and transfer function) with a sample size of 12. DISCUSSION: Our data support the longitudinal design of clinical trials over at least 12 months and the combinational use of clinical and radiological surrogate outcome measures.

Parents' vaccination information seeking, satisfaction with and trust in medical providers in Switzerland: a mixed-methods study.

OBJECTIVES: The aim of this study was to better understand parental trust in and satisfaction with information sources and medical providers regarding decision making about childhood vaccines. SETTING: The study was part of a Swiss national research programme investigating vaccine hesitancy and underimmunisation. PARTICIPANTS: We conducted qualitative interviews with 37 providers and 30 parents, observed 34 vaccination consultations, and then conducted quantitative surveys with 130 providers (both complementary and alternative medicine (CAM) oriented and biomedically oriented) and 1390 parents. MAIN OUTCOME MEASURES: Participants' vaccination information sources used in their decision-making process, parents' trust in and satisfaction with these sources and providers. RESULTS: Based on the Parent Attitudes about Childhood Vaccines scale, we considered 501 parents as vaccine-hesitant (VH) and 889 parents as non-VH. Whereas both groups mentioned providers as the most trusted source of information, VH-parents were less likely to mention paediatricians (N=358 (71%) vs N=755 (85%)) and public health authorities (N=101 (20%) vs N=333 (37%)) than non-VH-parents. VH-parents were more likely to have consulted another provider (N=196 (39%) vs N=173 (19%)) than non-VH-parents, to express less satisfaction with both their primary (N=342 (82%) vs N=586 (91%)) and other providers (N=82 (42%) vs N=142 (82%)) and less trust in their primary (N=368 (88%) vs N=632 (98%)) and other providers (N=108 (55%) vs N=146 (84%)). VH-parents were less likely to be satisfied with their biomedical primary provider than non-VH-parents (100 (69%) vs 467 (91%)). However, when the primary provider was CAM-oriented, there were similar levels of satisfaction among both groups (237 (89%) VH-parents vs 118 (89%) non-VH-parents). All differences were significant (p<0.05). CONCLUSIONS: While the provider remains the main information source, VH parents turn to additional sources and providers, which is likely related to VH parents being rather dissatisfied with and distrusting in obtained information and their provider. ETHICS: The local ethics committee (Ethikkommission Nordwest- und Zentralschweiz, EKNZ; project ID number 2017-00725) approved the study.