Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, 385A Glossop Road, Sheffield, S10 2HQ, UK.
BMC Neurol. 2012 Aug 16;12:74. doi: 10.1186/1471-2377-12-74.
Motor neurone disease (MND) is a devastating illness which leads to muscle weakness and death, usually within 2-3 years of symptom onset. Respiratory insufficiency is a common cause of morbidity, particularly in later stages of MND and respiratory complications are the leading cause of mortality in MND patients. Non Invasive Ventilation (NIV) is the current standard therapy to manage respiratory insufficiency. Some MND patients however do not tolerate NIV due to a number of issues including mask interface problems and claustrophobia. In those that do tolerate NIV, eventually respiratory muscle weakness will progress to a point at which intermittent/overnight NIV is ineffective. The NeuRx RA/4 Diaphragm Pacing System was originally developed for patients with respiratory insufficiency and diaphragm paralysis secondary to stable high spinal cord injuries. The DiPALS study will assess the effect of diaphragm pacing (DP) when used to treat patients with MND and respiratory insufficiency.
METHOD/DESIGN: 108 patients will be recruited to the study at 5 sites in the UK. Patients will be randomised to either receive NIV (current standard care) or receive DP in addition to NIV. Study participants will be required to complete outcome measures at 5 follow up time points (2, 3, 6, 9 and 12 months) plus an additional surgery and 1 week post operative visit for those in the DP group. 12 patients (and their carers) from the DP group will also be asked to complete 2 qualitative interviews.
The primary objective of this trial will be to evaluate the effect of Diaphragm Pacing (DP) on survival over the study duration in patients with MND with respiratory muscle weakness. The project is funded by the National Institute for Health Research, Health Technology Assessment (HTA) Programme (project number 09/55/33) and the Motor Neurone Disease Association and the Henry Smith Charity.
Current controlled trials ISRCTN53817913. The views and opinions expressed therein are those of the authors and do not necessarily reflect those of the HTA programme, NIHR, NHS or the Department of Health.
运动神经元病（MND）是一种破坏性疾病，会导致肌肉无力和死亡，通常在症状出现后 2-3 年内。呼吸功能不全是发病率的常见原因，特别是在 MND 的后期，呼吸并发症是 MND 患者死亡的主要原因。非侵入性通气（NIV）是目前治疗呼吸功能不全的标准疗法。然而，由于面罩接口问题和幽闭恐惧症等多种问题，一些 MND 患者无法耐受 NIV。在那些能够耐受 NIV 的患者中，最终呼吸肌无力会进展到间歇性/夜间 NIV 无效的地步。NeuRx RA/4 膈肌起搏系统最初是为因稳定的高脊髓损伤而导致呼吸功能不全和膈肌麻痹的患者开发的。DiPALS 研究将评估在治疗 MND 和呼吸功能不全患者时使用膈肌起搏（DP）的效果。
方法/设计：将在英国的 5 个地点招募 108 名患者参加该研究。患者将被随机分配接受 NIV（当前的标准护理）或在接受 NIV 的基础上接受 DP。研究参与者将需要在 5 个随访时间点（2、3、6、9 和 12 个月）完成结果测量，对于 DP 组的患者还需要进行一次额外的手术和术后 1 周的就诊。DP 组的 12 名患者（及其照顾者）也将被要求完成 2 次定性访谈。
该试验的主要目的将是评估在患有呼吸肌无力的 MND 患者的研究期间，膈肌起搏（DP）对生存的影响。该项目由英国国家卫生研究院、卫生技术评估（HTA）计划（项目编号 09/55/33）、运动神经元疾病协会和亨利·史密斯慈善机构资助。
当前对照试验 ISRCTN53817913。其中表达的观点和意见是作者的观点，不一定反映 HTA 计划、NIHR、NHS 或卫生部的观点。
