Medical therapies for amyotrophic lateral sclerosis-related respiratory decline: an appraisal of needs, opportunities and obstacles.
Amyotroph Lateral Scler Frontotemporal Degener
; 23(1-2): 66-75, 2022 02.
Article
in English
| MEDLINE | ID: covidwho-1358405
ABSTRACT
A roundtable convened in July 2020 examined issues concerning respiratory support in amyotrophic lateral sclerosis (ALS), with reference to the potential for an early-phase orally administered medication that might either postpone the introduction of noninvasive ventilation (NIV) and/or enhance the benefits to be gained from it. Attention was also given to the impact of the COVID-19 pandemic on usual practice in the assessment and management of ALS-related respiratory difficulties. Implementation of NIV marks a step-change in clinical status for patients and a major increase in burden for caregivers. All means to ease this transition should be explored an oral therapy that supported respiratory function and patients' independence and sense of well-being would aid discussions to facilitate the eventual successful introduction of NIV. Assessment of a candidate oral therapy that might support respiratory function in ALS patients would be aided by the development of improved patient-reported outcome measures for robust quantification of treatment effect and quality of life. Such instruments could also be used to monitor patients' status during the COVID-19 pandemic, averting some of the risks of face-to-face assessment plus the patient burden and costs of traditional methods. Several oral candidate therapies have recently failed to meet their primary endpoints in clinical trials. However, understanding of the underlying physiology and appropriate trial design have grown and will inform future developments in this field.
Keywords
Full text:
Available
Collection:
International databases
Database:
MEDLINE
Main subject:
Respiratory Insufficiency
/
Noninvasive Ventilation
/
COVID-19
/
Amyotrophic Lateral Sclerosis
Type of study:
Prognostic study
/
Qualitative research
/
Randomized controlled trials
Topics:
Long Covid
Limits:
Humans
Language:
English
Journal:
Amyotroph Lateral Scler Frontotemporal Degener
Year:
2022
Document Type:
Article
Affiliation country:
21678421.2021.1920981
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