Background The evaluation of the current state and progression of the Idiopathic Inflammatory Myopathies (IIM) should include patient reported outcomes (PRO), as these are objective measures of subjective symptoms, they reflect the disease impact on patient’s life, and they can improve and facilitate the communication between health care provider and patient. These measures are widely used and applied in related rheumatic diseases, but they are not extensively utilized during the treatment and surveillance of IIM patients. PROs are increasingly recognized as important to comprehensive evaluation of human health by the WHO and other international and national organizations.
Objectives To review the known patient reported outcomes and their current application in evaluation of IIM patients and similar neuromuscular diseases with symptoms of muscle weakness.
Methods We searched MEDLINE and clinical trial registries through September, 2011. Search terms included “myositis”, “dermatomyositis”, “juvenile dermatomyositis”, “polymyositis”, “idiopathic inflammatory myopathies”, “inclusion body myositis”, “immune-mediated necrotizing myopathy”, “centronuclear myopathy”, “progressive muscular atrophy”, “spinal muscular atrophy”, “sarcopenia”, “Charcot-Marie-Tooth disease”, “muscular dystrophy”, “Duchenne muscular dystrophy”, “multiple sclerosis”, “myasthenia gravis”, “patient related outcome”, “patient reported outcome” and “assessment”. Non-patient-centered outcomes were excluded. All extrapolated data were categorically tabulated as domain and relevant instrument(s).
Results In total, 109 articles were reviewed as initially relevant and 36 articles were discarded from according to the exclusion criteria.
We discovered that the majority of patient-reported outcomes are generic, non-disease specific instruments. We found only two disease specific instrument for myositis which include the Myositis Activities Profile (MAP) and the IBM Functional Rating Scale (IBMFRS).
“Quality of life” SF-36, CHQ, AIMS-2, NHP, INQoL
Patient Global Assessment VAS 10cm scale
Physical function HAQ-DI, C-HAQ, HAP, MAP, MACTAR
Pain VAS 10 cm or 100mm scale
Fatigue MAF, POMS-F
Conclusions PRO development is a rapidly evolving discipline that requires new scores or validation of the existing scores in specific disease groups. Outcome measures are important as they play an important role in the treat- to- target approach. Only with their use, the evaluation of the efficacy of treatment can become more objective. At this time, these outcomes are used only in clinical trials and for research needs. The majority of the presented scores are lengthy and difficult to use in practical terms. To incorporate and apply these scores in daily clinical practice, it will be imperative to develop a feasible, short, but valid instrument that can be a mandatory part of the patient’s standard of care.
Disclosure of Interest None Declared