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Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Availability
Please visit this website for more information about the instrument: Primary Mitochondrial Myopathy Symptom Assessment
Classification
Supplemental: Mitochondrial Disease (Mito)
Short Description of Instrument
The Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) is a 10-item patient-reported outcome questionnaire that assesses tiredness at rest, tiredness during activities, muscle weakness at rest, muscle weakness during activities, balance problems, vision problems, abdominal discomfort, muscle pain, numbness, and headache over the previous 24 hours in Primary Mitochondrial Myopathy (PMM) patients.
Comments/Special Instructions
The PMMSA is a daily PRO assessment to evaluate symptom severity and is relevant to individuals with PMM in both clinical trials and observational research (Gwaltney et al., 2020).
Scoring and Psychometric Properties
Scoring: Each item in the PMMSA has a four-point verbal rating scale (VRS) ranging from 1-Not at all to 4-Severe. Each item's daily score is reflected by a 1 to 4 VRS where higher scores reflect more severe symptoms. The total score is calculated as the sum of the 10 items.
 
Psychometric Properties: The PMMSA Fatigue 4 summary score, made up of Items 1, (tiredness at rest), 2 (tiredness during activities), 3 (muscle weakness at rest), and 4 (muscle weakness during activities) demonstrated strong reliability and construct-related validity (Gwaltney et al., 2022).
Rationale/Justification
Strengths: The PMMSA is a useful tool to examine a PMM patient's perspective on the symptoms that negatively affect their quality of life and activities of daily living in clinical trials (Gwaltney et al., 2020).
 
Weaknesses: The full 10-item PMMSA is multidimensional and a total score may not be appropriate for this instrument (Gwaltney et al., 2022).
References
Key Reference:
Gwaltney C, Stokes J, Aiudi A, Mazar I, Ollis S, Love E, Espensen A, Shields AL. Development of a Patient-Reported Outcome Questionnaire to Evaluate Primary Mitochondrial Myopathy Symptoms: The Primary Mitochondrial Myopathy Symptom Assessment. J Clin Neuromuscul Dis. 2020 Dec;22(2):65-76.
 
Additional References:
Gwaltney C, Stokes J, Aiudi A, Mazar I, Ollis S, Love E, Karaa A, Houts CR, Wirth RJ, Shields AL. Psychometric performance of the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) in a randomized, double-blind, placebo-controlled crossover study in subjects with mitochondrial disease. J Patient Rep Outcomes. 2022 Dec 23;6(1):129.
 
Karaa A, Haas R, Goldstein A, Vockley J, Cohen BH. A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathy. J Cachexia Sarcopenia Muscle. 2020 Aug;11(4):909-8.
 
Mancuso M, McFarland R, Klopstock T, Hirano M; consortium on Trial Readiness in Mitochondrial Myopathies. International Workshop: Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adults. Consensus recommendations. 16-18 November 2016, Rome, Italy. Neuromuscul Disord. 2017 Dec;27(12):1126-1137.
 
Document last updated March 2024