Diaphragm paralysis; surgery or mechanical ventilation?

Rationale: The diaphragm is a dome-shaped muscle which separates the thoracic cavity from the abdomen. In patients with diaphragm paralysis the treatment (surgery versus non-invasive ventilation) is based on physician preference, not sound scientific evidence. Clearly studies are needed to guide a scientific decision making.
Objective: In this pilot study we will evaluate if patients are willing and able to participate in a randomized trial. Secondly this pilot study is also needed to know the clinical relevant effect of both therapies on EQ-5D_5L, the latter being the primary outcome. Finally, it will show us the costs of both therapies form a societal perspective.
Study design: open label, multi center randomized controlled trial
Study population: 20 participants >18 year and diagnosed with a unilateral of bilateral diaphragm paralysis resulting from phrenic nerve injury.
Intervention: 10 participants for surgical plication and 10 participants for nocturnal non-invasive ventilation.
Main study parameters/endpoints: The primary question of the pilot study is whether a randomized trial comparing surgery versus non-invasive ventilation is feasible. In our opinion a randomized study is feasible if at least 50% of the participants fulfilling the inclusions criteria are randomized in this pilot study.
The second goal of the pilot study is to describe the effect of both plication and NIV on the . primary endpoint being quality of life measured by the EQ-5D-5L questionnaire. Secondary endpoints are; the Medical Research Council (MRC) dyspnoea scale, the Diaphragmatic Paralysis Questionnaire, Borg dyspnoea score, Endurance Shuttle Walk Test (ESWT), spirometry in both sitting and supine position, a polysomnography an ergometry and transcutaneous measurement of carbon dioxide an oxygen saturation at night.
Nature and extent of the burden and risks associated with participation, benefit and group relatedness: after randomization the participants receives a treatment that was not the primary reason for referral which might be a barrier in participant recruitment. At the first outpatient visit a physical exam and an arterial blood gas analysis is performed. At baseline and 6 months after starting the therapy, quality of life questionnaires, an endurance shuttle walk test (ESWT), spirometry, ergometry, a polysomnography and transcutaneous monitoring of carbon dioxide an oxygen saturation at night will be performed. All these measurements are non-invasive and some are part of standard care.
Both treatments, non-invasive ventilation and surgical plication are considered as standard care, so participating in this study does not impose additional risks.

Primary question is whether the intended cost effectiveness / cost utility study is feasible. The study is feasible if at least 50% of the participants fulfilling the inclusion criteria are randomized in this pilot study.

At baseline and 6 months after starting the therapy: Quality of life measured by the EQ-5D-5L, the Medical Research Council (MRC) dyspnoea scale, the Diaphragmatic Paralysis Questionnaire, Borg dyspnoea score, Endurance Shuttle Walk Test (ESWT), spirometry in both sitting and supine position, a polysomnography an ergometry and transcutaneous measurement of carbon dioxide an oxygen saturation at night.
At two months a video call.

Intervention: Surgical diaphragm-plication
Comparator: Non-invasive mechanical ventilation