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Patient-centered benefit-risk assessment in duchenne muscular dystrophy.
Hollin, Ilene L; Peay, Holly L; Apkon, Susan D; Bridges, John F P.
Afiliação
  • Hollin IL; Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland, USA.
  • Peay HL; RTI International, Research Triangle Park, North Carolina, USA.
  • Apkon SD; Parent Project Muscular Dystrophy, Hackensack, New Jersey, USA.
  • Bridges JFP; Department of Rehabilitation Medicine, Seattle Children's Hospital and University of Washington, Seattle, Washington, USA.
Muscle Nerve ; 55(5): 626-634, 2017 05.
Article em En | MEDLINE | ID: mdl-27649378
INTRODUCTION: This study quantified caregiver and patient preferences for a therapeutic agent with demonstrated pulmonary benefits for Duchenne muscular dystrophy (DMD). Caregiver and patient differences were also explored. METHODS: A best-worst scaling survey (BWS) was administered to caregivers and patients. Across 9 profiles, respondents selected the best and worst attributes. Utility scores were estimated using mixed logistic regression. RESULTS: Respondents indicated greatest preference for therapies that maintain their current level of cough strength for 10 years or for 2 years. Preference scores for risks were low: 50% chance of diarrhea and 4 additional blood draws per year. CONCLUSION: There is a strong preference for pulmonary benefit and willingness to trade off risks and burden to achieve these benefits. In exchange for maintaining cough strength for 10 years, respondents were willing to tolerate high probabilities of diarrhea and additional blood draws. Muscle Nerve 55: 626-634, 2017.
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Participação do Paciente / Distrofia Muscular de Duchenne / Tomada de Decisão Clínica Tipo de estudo: Etiology_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Child / Female / Humans / Male Idioma: En Revista: Muscle Nerve Ano de publicação: 2017 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Participação do Paciente / Distrofia Muscular de Duchenne / Tomada de Decisão Clínica Tipo de estudo: Etiology_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Child / Female / Humans / Male Idioma: En Revista: Muscle Nerve Ano de publicação: 2017 Tipo de documento: Article País de afiliação: Estados Unidos