Amyotrophic Lateral Sclerosis Clinical Trial
Official title:
Role of Non-invasive Ventilation in Amyotrophic Lateral Sclerosis: Volume Versus Pressure Mode
Non-invasive mechanical ventilation (NIV) has been increasingly used as a treatment of
chronic hypercapnic respiratory failure. Its use in patients affected by chronic obstructive
pulmonary disorders is still controversial, while most of the studies performed in
restrictive thoracic disorders (RTD), and in particular in neuromuscular patients, suggested
alleviation of the symptoms of chronic hypoventilation in the short term, and in two small
studies survival was prolonged.
In the terminal phase of the disease, when the respiratory muscles became weaker it is very
likely that the operators need to frequently adjust the level of inspiratory pressure in an
attempt to guarantee an adequate tidal volume, so that alveolar hypoventilation may be
avoided.
Theoretically the use of a volume assisted ventilation may overpass this problem of frequent
variations of the settings, since the provision of a fixed tidal volume may always guarantee
and adequate alveolar ventilation.
The primary aims of this multicenter randomized study are to evaluate the clinical efficacy,
the patients' tolerance and quality of life and the frequency of changing settings in a
group of patients with SLS and initial chronic respiratory failure undergoing long-term NIV
with Pressure Support Ventilation or Volume Assisted Ventilation.
Background
Non-invasive mechanical ventilation (NIV) has been increasingly used as a treatment of
chronic hypercapnic respiratory failure. Its use in patients affected by chronic obstructive
pulmonary disorders is still controversial, while most of the studies performed in
restrictive thoracic disorders (RTD), and in particular in neuromuscular patients, suggested
alleviation of the symptoms of chronic hypoventilation in the short term, and in two small
studies survival was prolonged. As a matter of fact a recent Cochrane review stated that
"long-term mechanical ventilation should be offered as a therapeutic option to patients with
chronic respiratory failure due to neuromuscular diseases". Recurrent episodes of nocturnal
desaturation especially during REM sleep may deeply influence the prognosis of patients
affect by these disorders (i.e. ALS), and they are often associated with hypercapnia, so
that the correction of nocturnal hypoventilation seems to be the major goal to achieve with
the ventilatory treatment.
A very recent randomized controlled trial show that in ALS without severe bulbar
dysfunction, NIV improves survival with maintenance of, and improvement in, quality of life,
including sleep quality. The survival benefit from NIV in this group is much greater than
that from currently available neuroprotective therapy, like riluzole.
Most of the studies performed in RTD disorders were performed using a pressure assisted mode
(i.e Pressure Support Ventilation), because of the supposed better tolerance of the patients
and easy of settings for the operators, even though this has never been scientifically
proven.
In the terminal phase of the disease, when the respiratory muscles became weaker it is very
likely that the operators need to frequently adjust the level of inspiratory pressure in an
attempt to guarantee an adequate tidal volume, so that alveolar hypoventilation may be
avoided.
Theoretically the use of a volume assisted ventilation may overpass this problem of frequent
variations of the settings, since the provision of a fixed tidal volume may always guarantee
and adequate alveolar ventilation.
The primary aims of this multicenter randomized study are to evaluate the clinical efficacy,
the patients' tolerance and quality of life and the frequency of changing settings in a
group of patients with SLS and initial chronic respiratory failure undergoing long-term NIV
with Pressure Support Ventilation or Volume Assisted Ventilation.
Aim of the Project
The primary aims of the project are to evaluate the clinical efficacy, the patients'
tolerance and quality of life and the frequency of changing settings in a group of patients
with SLS and initial chronic respiratory failure undergoing long-term NIV with Pressure
Support Ventilation or Volume Assisted Ventilation.
• Enrollment criteria: Patients with the diagnosis of ALS and one of the following signs: 1)
Vital Capacity < 50% predicted 2) a Maximal Inspiratory Pressure (MIP) < 60% predicted 3)
polygraphic signs of nocturnal hypoventilation with daytime symptoms.
- The patients will be randomized to receive NIV with one of the following modes: 1)
Pressure Support Ventilation with the inspiratory pressure set according to the patient's
tolerance in order to achieve an expired tidal volume > 6 ml/Kg and <8ml/Kg. The back-up
rate will be set at 10 breaths/min. 2) Volume assist ventilation with a back-up rate of 10
breaths/min and a tidal volume set to achieve an expired tidal volume > 6 ml/Kg and <8ml/Kg
;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Caregiver, Outcomes Assessor), Primary Purpose: Supportive Care
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