The following questions will be addressed in the PARASOL study• Is the proposed study design feasible?,We will set up the study as a randomized pilot study to evaluate if patients are willing and able to participate in a randomized trial, i.e. are…
ID
Source
Brief title
PARASOL
Condition
- Other condition
Synonym
Health condition
Diaphragm paralysis
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Main study parameter/endpoint
The primary question is whether a randomized trial comparing surgery versus
non-invasive ventilation is feasible. The study is feasible if at least 50% of
the patients fulfilling the inclusion criteria are randomized in this pilot
study
Secondary outcome
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 and transcutaneous measurement of carbon dioxide an oxygen
saturation at night.
Background summary
The diaphragm is a dome-shaped muscle which separates the thoracic cavity from
the abdomen. It is the most important muscle of respiration innervated by the
phrenic nerves. While many diseases might interfere with its function(1) , in
the intended study we will focus on diaphragm paralysis due to phrenic nerve
injury. Two types of diaphragm paralysis can be distinguished: unilateral and
bilateral. Patients with unilateral paralysis perceive exertional dyspnea, have
an impaired exercise capacity and orthopnea(2). Patients with a bilateral
paralysis usually have more symptoms and might even develop respiratory
failure(3). In addition, all patients with a diaphragm paralysis may have poor
sleep quality, as the diaphragm is the only active respiratory muscle during
REM sleep(4). Currently, two treatment approaches for patients with diaphragm
paralysis are used in clinical practice: surgical diaphragm plication and
nocturnal non-invasive ventilation (NIV). Plication is a minimal invasive
surgical procedure that aims to stiffen the diaphragm and such limits
dysfunctional (paradoxical movement) excursions of the paralytic diaphragm. The
procedure is performed in ±70 patients per year in the Netherlands. NIV is a
non-invasive mode of positive pressure ventilatory assistance; through a facial
mask the ventilator supports patient breathing effort. Patients with diaphragm
paralysis use their ventilator mainly during night time, to improve quality of
sleep and such to reduce day time symptoms. In the Netherlands, home mechanical
ventilation is very well organized, as care is delivered by only 4 specialized
centers. NIV for diaphragm paralysis is started in around 50 patients yearly.
Currently, both plication and nocturnal NIV appear beneficial, are standard
care and are covered by health care insurance. However, it is unknown which
intervention is most beneficial from a patient perspective. For instance,
comparison on patient relevant outcome measures and complications between these
treatment approaches is unknown. In addition, patients with diaphragm paralysis
may develop severe symptoms, limiting daily activities including ability to
perform their professional work. To assess the overall impact of this a
detailed cost analysis is necessary to compare both treatments from a societal
perspective. A solid cost effectiveness / cost utility study will reveal which
therapy is the best option from a societal perspective.
This pilot study will be set up as a randomized pilot study to evaluate if
patients are willing and able to participate in this trial. Question is, are
participants willing to participate, are they willing to travel to the other
institute and are they able to comply with the follow-up measurements.
Participants referred to either one of the therapies might be biased towards
that therapy and the questions is whether well informed about both therapies,
they are willing to participate. To know what clinical effect of both therapies
is relevant the EQ-5D-5L is used(5). It is unknown whether there is a
significant difference on the outcome between both therapies. A search in trial
registries did not reveal any study with similar research questions. Based on
the outcomes of this pilot study we can develop an adequately powered
randomized controlled study. Due to the acute origin of a diaphragm paralysis
patients get suddenly severely impaired which is interfering enormously with
their lives. As this is often happening in middle aged patients they often have
to discontinue professional activities. This means that the potential impact of
this disorder is huge from patient and societal perspective and needs to be
assessed.
As both therapies are completely different for invasiveness, we need to compare
the side effects and possible complications. Possible complications of the
surgery are infection, bleeding and abdominal pain while the well know side
effects of ventilatory support are leakage of the mask, aerophagia and
a-synchrony between breathing pattern of the patient and the ventilator. As
this is also an important outcome of this pilot study participants will be
closely monitored from the start of therapy and there will be a telephone call
after 2 months
Study objective
The following questions will be addressed in the PARASOL study
• Is the proposed study design feasible?,
We will set up the study as a randomized pilot study to evaluate if patients
are willing and able to participate in a randomized trial, i.e. are patients
willing to participate, are they willing to travel to the other institute, are
they able to comply with the follow-up measurements. Patients referred to
either one of the therapies might be biased towards that therapy and the
question is whether with good information about both therapies, they are
willing to participate in a randomized controlled trial.
• What is the effect of plication or non-invasive mechanical ventilation on the
EQ-5D-5L?
The pilot study is needed as we do not know the clinical relevant effect of
both therapies on EQ-5D-5L. Moreover, it is unknown whether there is a
significant difference on the outcomes between both therapies. A search in
trial registries did not reveal any study with similar research questions as
the current proposal. Based on the outcomes of pilot study a power analysis
can be performed for the seminal study.
• What are the costs of both therapies from a societal perspective?
Due to the acute origin of a diaphragm paralysis patients get suddenly severely
impaired which is interfering enormously with their lives. As this is often
happening in middle aged patients they often have to discontinue professional
activities. This means that the potential impact of this disorder is huge from
patient and societal perspective needs to be assessed.
• What are the side effects - complications of both interventions?
As both therapies are completely different for invasiveness, we need to compare
the side effects and possible complications. Possible complications of the
surgery are infection, bleeding and abdominal pain while the well know side
effects of ventilatory support are leakage of the mask, aerophagia and
a-synchrony between breathing pattern of the patient and the ventilator. As
this is also an important outcome of this pilot study patients will be closely
monitored from the start of therapy and there will be a telephone call after 2
months
Study design
Open-label, multi center randomized controlled trial / pilot
Intervention
Surgical plication of the diaphragm
Mechanical Ventilation
Study burden and risks
The present study is the first to investigate either diaphragm plication or
nocturnal NIV. Both options are standard care and covered by health care
insurance. It is unknown which intervention is most beneficial from a patient
perspective. For instance, comparison on patient relevant outcome measures and
complications between these treatment approaches are unknown. In addition,
patients with diaphragm paralysis may develop severe symptoms, limiting daily
activities including ability to perform their professional work. To assess the
overall impact of this a detailed cost analysis is necessary to compare both
treatments from a societal perspective.
Hanzeplein 1
Groningen 9700 RB
NL
Hanzeplein 1
Groningen 9700 RB
NL
Listed location countries
Age
Inclusion criteria
• >18 years
• diagnosed with a unilateral or bilateral diaphragm paralysis based on
isolated phrenic nerve injury.
o Unilateral or bilateral diaphragm paralysis is defined as follows: complaints
of dyspnea and / or orthopnea combined with a drop in VC of more than 15% when
change from upright to supine position and a positive sniff test during
fluoroscopy or ultrasonography. A positive sniff test means that the diaphragm
stands still or even moves in cranial direction (paradoxical movement ) during
the sniff inspiratory maneuver.
• Ability to provide written consent
• Time between diagnosis and treatment should be at least 1 year
Exclusion criteria
• Patients diagnosed with a unilateral or bilateral diaphragm paralysis due to
a more systemic neurological or neuromuscular disorder like for example
Amyotrophic Lateral Sclerosis ,
• Hypercapnia during daytime (PaCO2 > 6.0 kPa)
• Radiotherapy of the thorax
• Contra indication for diaphragm surgery.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL78661.042.22 |
| Other | UMCG research 202100559 |