PDF(Pubmed)
Abstract：
BACKGROUND: Primary ciliary dyskinesia (PCD) is a rare genetical disease with malfunction of the motile cilia leading to impaired muco-ciliary clearance in the respiratory tract. There is no cure for PCD, only supportive therapy aimed at minimizing the progression of the disease and improving the patient\'s quality of life (QoL). Physical activity (PA) is one of these recommended supportive therapies for people with PCD (pwPCD). However, there is no scientific evidence to support this recommendation. In addition, regular medical advice to increase PA remains largely ineffective in pwPCD.
METHODS: To test the main hypothesis, that an individualized and supported PA program leads to a better QoL 6 months after randomization (QoL-PCD questionnaire) compared to usual recommendation in pwPCD, 158 pwPCD aged 7 to 55 years are to be included in this multi-center randomized controlled trial (RCT). After the screening visit, a 1:1 randomization stratified by age group and FEV1 will be performed. A QoL-PCD questionnaire, motor test, and lung function will be carried out at regular intervals in both groups. PA is recorded in both groups using activity trackers during the study period. The main aim of the trial is to estimate the difference in the change of QoL between the groups after 6 months. Therefore, our full analysis set consists of all randomized patients and analysis is performed using the intention-to-treat principle. Statistical software R ( http://www.r-project.org ) is used. Ethical approvement without any reservations: RUB Bochum Ethics Committee (No. 23-7938; December 4, 2023). Recruitment start: March 2024.
CONCLUSIONS: Limitations result from the rarity of PCD with its broad disease spectrum and the large age range. These are reduced by stratified randomization and the measurement of the individual change in QoL as primary endpoint. In our view, only a PA program tailored to individual needs with close contact to trainers offers the chance to meet personal needs of pwPCD and to establish PA as a pillar of therapy in the long term. The study protocol explains all procedures and methods of recruitment, implementation of the study visits and intervention, measures for patient and data safety, and for minimizing risks and bias.
BACKGROUND: German Clinical Trials Register (DRKS) 00033030. Registered on December 7, 2023. Update 10 July 2024. STUDY PROTOCOL VERSION 10: Version 1.2; 12 June 2024.
METHODS: To test the main hypothesis, that an individualized and supported PA program leads to a better QoL 6 months after randomization (QoL-PCD questionnaire) compared to usual recommendation in pwPCD, 158 pwPCD aged 7 to 55 years are to be included in this multi-center randomized controlled trial (RCT). After the screening visit, a 1:1 randomization stratified by age group and FEV1 will be performed. A QoL-PCD questionnaire, motor test, and lung function will be carried out at regular intervals in both groups. PA is recorded in both groups using activity trackers during the study period. The main aim of the trial is to estimate the difference in the change of QoL between the groups after 6 months. Therefore, our full analysis set consists of all randomized patients and analysis is performed using the intention-to-treat principle. Statistical software R ( http://www.r-project.org ) is used. Ethical approvement without any reservations: RUB Bochum Ethics Committee (No. 23-7938; December 4, 2023). Recruitment start: March 2024.
CONCLUSIONS: Limitations result from the rarity of PCD with its broad disease spectrum and the large age range. These are reduced by stratified randomization and the measurement of the individual change in QoL as primary endpoint. In our view, only a PA program tailored to individual needs with close contact to trainers offers the chance to meet personal needs of pwPCD and to establish PA as a pillar of therapy in the long term. The study protocol explains all procedures and methods of recruitment, implementation of the study visits and intervention, measures for patient and data safety, and for minimizing risks and bias.
BACKGROUND: German Clinical Trials Register (DRKS) 00033030. Registered on December 7, 2023. Update 10 July 2024. STUDY PROTOCOL VERSION 10: Version 1.2; 12 June 2024.
摘要：
背景：原发性纤毛运动障碍（PCD）是一种罕见的遗传性疾病，其运动纤毛功能异常导致呼吸道粘膜纤毛清除受损。没有治疗PCD的方法,仅支持治疗旨在最大限度地减少疾病进展和改善患者生活质量（QoL）。体力活动（PA）是PCD（pwPCD）患者推荐的支持疗法之一。然而,没有科学证据支持这一建议.此外,定期增加PA的医疗建议在pwPCD中仍然无效。
方法：为了检验主要假设，与pwPCD中的通常建议相比，个性化和支持的PA计划在随机化后6个月（QoL-PCD问卷）导致更好的QoL，158名年龄在7至55岁之间的pwPCD将纳入这项多中心随机对照试验（RCT）。筛选访问后，a将按年龄组和FEV1进行1:1随机化分层。QoL-PCD问卷，电机测试,两组均定期进行肺功能检查。在研究期间使用活动跟踪器记录两组的PA。该试验的主要目的是估计6个月后组间QoL变化的差异。因此,我们的完整分析集由所有随机分组的患者组成,采用意向治疗原则进行分析.统计软件R(http：//www。r-project.org）使用。毫无保留的道德认可：RUB波鸿道德委员会（编号：23-7938；2023年12月4日）。招聘开始：2024年3月。
结论：局限性是由于PCD的罕见性，其疾病谱广，年龄范围大。通过分层随机化和测量作为主要终点的QoL的个体变化来减少这些。在我们看来,只有与培训师密切接触的针对个人需求量身定制的PA计划，才有机会满足pwPCD的个人需求，并将PA长期确立为治疗的支柱。研究方案解释了招募的所有程序和方法，实施研究访问和干预，患者和数据安全措施，以及最小化风险和偏见。
背景：德国临床试验注册（DRKS）00033030。2023年12月7日注册。2024年7月10日更新。研究协议第10版：1.2版；2024年6月12日。
方法：为了检验主要假设，与pwPCD中的通常建议相比，个性化和支持的PA计划在随机化后6个月（QoL-PCD问卷）导致更好的QoL，158名年龄在7至55岁之间的pwPCD将纳入这项多中心随机对照试验（RCT）。筛选访问后，a将按年龄组和FEV1进行1:1随机化分层。QoL-PCD问卷，电机测试,两组均定期进行肺功能检查。在研究期间使用活动跟踪器记录两组的PA。该试验的主要目的是估计6个月后组间QoL变化的差异。因此,我们的完整分析集由所有随机分组的患者组成,采用意向治疗原则进行分析.统计软件R(http：//www。r-project.org）使用。毫无保留的道德认可：RUB波鸿道德委员会（编号：23-7938；2023年12月4日）。招聘开始：2024年3月。
结论：局限性是由于PCD的罕见性，其疾病谱广，年龄范围大。通过分层随机化和测量作为主要终点的QoL的个体变化来减少这些。在我们看来,只有与培训师密切接触的针对个人需求量身定制的PA计划，才有机会满足pwPCD的个人需求，并将PA长期确立为治疗的支柱。研究方案解释了招募的所有程序和方法，实施研究访问和干预，患者和数据安全措施，以及最小化风险和偏见。
背景：德国临床试验注册（DRKS）00033030。2023年12月7日注册。2024年7月10日更新。研究协议第10版：1.2版；2024年6月12日。
