The impact of clinical trial participation on quality of life and psychosocial well-being in children with Duchenne muscular dystrophy and their parents.

Clinical trials provide Duchenne muscular dystrophy (DMD) patients access to medication. Nevertheless, such involvement can impose certain burdens, as the protocol may entail strict adherence and additional demands. This study assessed the psychosocial functioning and quality of life in boys with DMD and their parents who participate in clinical trials. DMD families participating in clinical trials (n = 25) and families with DMD patients not involved in clinical trials (N = 18) were included. Questionnaires assessing psychosocial well-being and quality of life were completed by the participants and their parents. MANOVAs were employed to compare outcomes between groups. The results showed that mothers in the clinical trial group experienced significantly higher scores of somatic complaints. Fathers in the clinical trial group reported significantly fewer psychological issues compared to fathers from the other group. DMD patients participating in clinical trials reported a better overall and emotional quality of life compared to them not involved in clinical trials. This study suggests that clinical trial participation may have positive effects on quality of life and psychosocial outcomes. It highlights the importance of providing support and counseling throughout the clinical trial decision making process to minimize potential burden for both eligible and ineligible patients.

Illness Perceptions and Illness Identity in Adolescents and Emerging Adults With Neuromuscular Disorders.

BACKGROUND: Neuromuscular disorders (NMD) are intrusive medical conditions with implications for psychosocial development. OBJECTIVES: This paper explores illness perceptions and illness identity dimensions of youth with NMD. First, we compare illness identity outcomes and illness perceptions of NMD patients with a comparison group of adolescents with type 1 diabetes mellitus (DM). Second, we report about the relationships between NMD-related variables and illness perceptions and illness identity. METHODS: Scores on the Brief Illness Perception Questionnaire and the Illness Identity Questionnaire were compared between a group of NMD patients (N = 59; 12-22 years) and an age- and gender-matched group of DM patients (N = 118). NMD-related variables included time since diagnosis, prognosis, wheelchair use, and physical limitations. RESULTS: Youth with NMD scored significantly higher on two of the four illness identity dimensions than youth with DM. NMD patients reported significantly less positive illness perceptions, experienced more physical symptoms, and had a lower score on understanding of their illness. Within the NMD group, wheelchair-users have a better understanding of their disease than those who are not wheelchair-bound. CONCLUSIONS: The present study is the first to investigate illness identity and illness perceptions in NMD. More research is needed to provide insight in the identity formation process of the growing group of adolescents with NMDs.