MULTIsite feasibility of MUSIc therapy to address Quality Of Life in Sickle cell disease (MULTI-MUSIQOLS)

患有镰状细胞病（SCD）的个体在其一生中经常遭受多次急性疼痛危机。这些危机和身体并发症的累积影响导致青春期早期出现慢性疼痛综合征、心理健康状况和健康相关生活质量（HRQoL）受损，并持续到成年。在其2020年SCD疼痛管理指南中，美国血液学会承认药物方法的有效性有限，而音乐疗法（MT）等非药物综合方法的潜在益处。这些指南确定了研究的优先事项，包括确定哪些非药物治疗是最可接受的，并制定手动的，可访问的，并发展适当的干预慢性SCD疼痛。在我们之前的单中心可行性研究中，24名患有SCD和慢性疼痛的成人随机接受6次MT干预（n = 12）或等待列表对照（WLC）（n = 12）。我们在这项随机对照试验（RCT）中制定了筛选、招募、保留和电子数据收集的程序。入组率为89%，所有研究指标均已完成，与WLC参与者相比，MT参与者表现出100%的出勤率以及自我效能和HRQoL的改善。虽然这项试点随机对照试验的结果很有希望，但为了扩大干预措施的规模，为未来的多地点、务实、明确的随机对照试验做准备，还需要采取进一步的措施。此外，随着MT越来越多地适用于虚拟分娩，包括在患有SCD的成年人中，需要进行试验来研究混合（即，一个面对面的会议和五个虚拟会议）的方法来进行机器翻译。在本R 01中，我们建议检查一项多中心研究的可行性，该研究招募了（1）亲自MT（n=30）、（2）混合MT（n=30）和（3）混合健康教育（HealthEd）（n=30）。我们的提案包括加州尔湾大学的MULTI-MUSIQOLS数据协调中心和两个招募站点：大学医院/凯斯西储大学和Prisma Health/南卡罗来纳州大学。共有90例受试者将随机分配至现场MT、混合MT或混合健康教育（HealthED）（1：1：1分配），重点关注数据收集、参与者招募、干预措施实施和家庭实践的可行性。我们的具体目标是：目的1）进行一项可行性随机对照试验，以检查数据收集过程和干预措施（亲自MT、混合MT和混合HealthED）的总体和2个研究中心的实施情况;目的2）使用定量数据（研究记录、利益相关者调查）和定性数据（访谈）评价亲自MT、混合MT和混合HealthED干预措施的实施情况。成功进行拟议的，可行性随机对照试验将提供必要的框架，进行未来的，多地点，务实，随机对照试验的MT相比，HealthED。完成拟定的R 01和后续RCT（UG 3/UH 3）可提供关键证据，支持纳入MT作为SCD患者和其他类似人群的慢性疼痛干预。

Individuals with sickle cell disease (SCD) often endure multiple acute pain crises throughout their lives. The cumulative impact of these crises and physical complications contribute to the emergence of chronic pain syndromes, mental health conditions, and impaired health-related quality of life (HRQoL) in early adolescence that persist through adulthood. In their 2020 pain management guidelines for SCD, the American Society of Hematology acknowledged the limited effectiveness of pharmacologic approaches and the potential benefits of nonpharmacologic integrative approaches such as music therapy (MT). These guidelines identified research priorities including determining which nonpharmacologic therapies are most acceptable and developing manualized, accessible, and developmentally appropriate interventions for chronic SCD pain. In our prior single-site feasibility study, 24 adults with SCD and chronic pain were randomized to receive either an in-person 6-session MT intervention (n = 12) or waitlist control (WLC) (n = 12). We developed procedures for screening, recruitment, retention, and electronic data collection within this randomized controlled trial (RCT). The enrollment rate was 89%, all study measures were completed, and MT participants demonstrated 100% attendance as well as improvements in self-efficacy and HRQoL compared to WLC participants. While results from this pilot RCT are promising, to scale an intervention in preparation for a future, multi-site, pragmatic, definitive RCT, further steps are needed. Furthermore, with MT being increasingly adapted for virtual delivery, including among adults with SCD, trials are needed to investigate the feasibility of hybrid (i.e., one in-person and five virtual sessions) approaches to MT delivery. In this R01, we propose to examine the feasibility of a multi-site study enrolling (1) in-person MT (n=30), (2) hybrid MT (n=30), and (3) hybrid Health Education (HealthEd) (n=30). Our proposal includes the MULTI-MUSIQOLS Data Coordinating Center at University of California Irvine and two recruiting sites: University Hospitals/Case Western Reserve University and Prisma Health/University of South Carolina. A total of 90 subjects will be randomized to either in-person MT, hybrid MT, or hybrid Health Education (HealthED) (1:1:1 allocation) with a focus on feasibility of data collection, participant recruitment, delivery of interventions, and home practice. Our Specific Aims are: AIM 1) conduct a feasibility RCT to examine the data collection processes and intervention (in-person MT, hybrid MT, and hybrid HealthED) implementation overall and across 2 sites; and AIM 2) evaluate the implementation of the in-person MT, hybrid MT, and hybrid HealthED interventions using both quantitative data (study records, stakeholder surveys) and qualitative data (interviews). Successful conduct of the proposed, feasibility RCT will provide the necessary framework for conducting a future, multi-site, pragmatic, RCT of MT compared with HealthED. Completion of the proposed R01 and the subsequent RCT (UG3/UH3) could provide critical evidence to support inclusion of MT as a chronic pain intervention for individuals with SCD and other similar populations.