Weaver Meaghann S, Yuroff Alice, Sund Sarah, Hetzel Scott, Halanski Matthew A
Children's Hospital and Medical Center, Omaha, NE 68114, USA.
National Center for Ethics in Healthcare, Washington, DC 20420, USA.
Children (Basel). 2021 Jul 17;8(7):604. doi: 10.3390/children8070604.
The purpose of this study was to explore early changes in patient and family caregiver report of quality of life and family impact during the transitional period of nusinersen use. Communication; family relationships; physical, emotional, social, and cognitive functioning; and daily activities were measured using Pediatric Quality of Life modules (Family Impact Modules and both Patient and Proxy Neuromuscular-Specific Reports) pre- and post-nusinersen exposure. A total of 35 patients with SMA (15 Type 1, 14 Type 2, and 6 Type 3) were grouped according to nusinersen exposure. When analyzed as a whole cross-sectional clinical population, no significant differences were found between the initial and final surveys. Nusinersen therapy was associated with improved communication and emotional functioning in subsets of the population, particularly for patients on maintenance therapy for longer duration. Several unexpected potentially negative findings including increases in family resources and trends towards increases in worry warrant further consideration. Further research is warranted to explore the impact of novel pharmaceuticals on quality of life for children with SMA longitudinally to optimize clinical and psychosocial outcomes.
本研究的目的是探讨在使用诺西那生钠的过渡期内，患者及家庭照顾者报告的生活质量和家庭影响的早期变化。使用儿科生活质量模块（家庭影响模块以及患者和代理神经肌肉特异性报告）在诺西那生钠暴露前后对沟通、家庭关系、身体、情感、社会和认知功能以及日常活动进行测量。共有35例脊髓性肌萎缩症患者（15例1型、14例2型和6例3型）根据诺西那生钠暴露情况进行分组。作为一个整体横断面临床人群进行分析时，初始调查和最终调查之间未发现显著差异。诺西那生钠治疗与部分人群的沟通和情感功能改善相关，特别是对于接受维持治疗时间较长的患者。一些意外的潜在负面发现，包括家庭资源增加和担忧增加的趋势，值得进一步考虑。有必要进行进一步研究，纵向探索新型药物对脊髓性肌萎缩症儿童生活质量的影响，以优化临床和心理社会结局。
