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Muscle biopsies in clinical trials for Duchenne muscular dystrophy - Patients' and caregivers' perspective.
Verhaart, Ingrid E C; Johnson, Alex; Thakrar, Sejal; Vroom, Elizabeth; De Angelis, Fernanda; Muntoni, Francesco; Aartsma-Rus, Annemieke M; Niks, Erik H.
Afiliación
  • Verhaart IEC; Duchenne Parent Project Netherlands, the Netherlands; Department of Human Genetics, Leiden University Medical Center, the Netherlands.
  • Johnson A; Duchenne UK, UK.
  • Thakrar S; Duchenne UK, UK.
  • Vroom E; Duchenne Parent Project Netherlands, the Netherlands.
  • De Angelis F; Parent Project Onlus, Italy.
  • Muntoni F; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, London, UK.
  • Aartsma-Rus AM; Department of Human Genetics, Leiden University Medical Center, the Netherlands.
  • Niks EH; Department of Neurology, Leiden University Medical Center, Leiden, the Netherlands. Electronic address: EHNiks@lumc.nl.
Neuromuscul Disord ; 29(8): 576-584, 2019 08.
Article en En | MEDLINE | ID: mdl-31378431
The number of clinical trials for Duchenne muscular dystrophy is increasing. Many trials require muscle biopsies, which involve an invasive surgical procedure. Little is known about short- and long-term impacts of muscle biopsies as perceived by patients and caregivers. Therefore a survey was held among patients and their caregivers who participated in trials involving muscle biopsies, in seven countries. Seventy-eight responses were received. Analysis revealed that many patients and parents had significant anxiety before the biopsy. The main concern of caregivers was the required general anaesthesia. In most cases biopsies caused pain and temporarily hampered daily activities. The main long-term impact was scarring, although large variation in size was reported. Seventy-nine percent of caregivers were little bothered and 21% were moderately or severely bothered by the scar. Willingness to consider another biopsy in future protocols was higher for open-label studies than for placebo-controlled trials. Caregivers stressed the importance of knowing the results of biopsy analyses; only a minority actually received this information. Recommendations are made on the informed consent procedure regarding risks and consequences of muscle biopsies, and communication of results. Furthermore, efforts should be made to minimise the impact of biopsies through pain management and by considering plastic surgery.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Dolor Postoperatorio / Biopsia / Aceptación de la Atención de Salud / Cicatriz / Músculo Esquelético / Distrofia Muscular de Duchenne / Prioridad del Paciente / Anestesia Tipo de estudio: Clinical_trials / Guideline Límite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: Neuromuscul Disord Asunto de la revista: NEUROLOGIA Año: 2019 Tipo del documento: Article País de afiliación: Países Bajos Pais de publicación: Reino Unido
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Dolor Postoperatorio / Biopsia / Aceptación de la Atención de Salud / Cicatriz / Músculo Esquelético / Distrofia Muscular de Duchenne / Prioridad del Paciente / Anestesia Tipo de estudio: Clinical_trials / Guideline Límite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: Neuromuscul Disord Asunto de la revista: NEUROLOGIA Año: 2019 Tipo del documento: Article País de afiliación: Países Bajos Pais de publicación: Reino Unido