Willems Jana, Pechmann Astrid, Wider Sabine, Ambs Rita, Meyer Sylvia A N, Cascante Isabel, Sproß Joachim, Mund Annette, Farin-Glattacker Erik, Langer Thorsten
Section of Health Care Research and Rehabilitation Research, Institute of Medical Biometry and Statistics, Faculty of Medicine and Medical Center, University of Freiburg, Freiburg, Germany.
Department of Neuropediatrics and Muscle Disorders, Center for Pediatrics, Faculty of Medicine and Medical Center, University of Freiburg, Freiburg, Germany.
Front Pediatr. 2023 Sep 20;11:1212012. doi: 10.3389/fped.2023.1212012. eCollection 2023.
Spinal muscular atrophy (SMA) is a rare neuromuscular disease requiring various clinical specialists and therapists to provide care. Due to the disease's dynamic nature and the long distances between specialized centers and local providers, integrating care between disciplines can be challenging. Care that is inadequately integrated can compromise the quality of care and become a burden for patients and families. This trial aimed to improve the care of patients through a case management (CM) intervention.
We conducted an exploratory, controlled, two-arm trial with pre-, post-, and follow-up measures (process and outcome evaluation). Proof of efficacy based on statistical significance was not our primary study objective since we were investigating a rare disease. Primary outcomes were caregivers' HRQoL and caregiver-rated quality of care integration. Our secondary outcome was the children's HRQoL.
Questionnaires and semi-structured interviews yielded heterogeneous results depending on caregivers' level of experience and desire (or possibility) to delegate care tasks.
Despite differing perceptions, all participants supported the establishment of a care coordination model. We recommend CM immediately after diagnosis to provide the greatest benefit to families. We hope that our trial will support the further development of CM interventions that can be customized for specific diseases.
脊髓性肌萎缩症（SMA）是一种罕见的神经肌肉疾病，需要各类临床专家和治疗师提供护理。由于该疾病的动态特性以及专业中心与当地医疗服务提供者之间距离较远，跨学科整合护理可能具有挑战性。整合不充分的护理会影响护理质量，并给患者及其家庭带来负担。本试验旨在通过病例管理（CM）干预改善患者护理。
我们进行了一项探索性、对照、双臂试验，采用了干预前、干预后及随访措施（过程和结果评估）。由于我们研究的是一种罕见疾病，基于统计学显著性的疗效证明并非我们的主要研究目标。主要结局是照料者的健康相关生活质量（HRQoL）以及照料者评定的护理整合质量。次要结局是儿童的HRQoL。
根据照料者的经验水平以及委托护理任务的意愿（或可能性），问卷调查和半结构化访谈得出了不同的结果。
尽管存在不同看法，但所有参与者都支持建立护理协调模式。我们建议在确诊后立即进行病例管理，以便为家庭带来最大益处。我们希望我们的试验将支持可针对特定疾病进行定制的病例管理干预措施的进一步发展。
