Optimizing hand-function patient outcome measures for inclusion body myositis

Area of Science:

• Neurology
• Rheumatology
• Clinical Trials

Background:

• Inclusion body myositis (IBM) is the most common acquired myopathy in individuals over 45.
• IBM is a slowly progressive and heterogeneous disorder, posing challenges for clinical trial efficacy measurement.
• The current Inclusion Body Myositis-Functional Rating Scale (IBM-FS) upper extremity domain may not detect changes until significant strength loss.

Purpose of the Study:

• To evaluate novel patient-reported outcome measures (PROMs) for upper extremity function in IBM.
• To compare the sensitivity of new PROMs and the IBM-FS to changes in grip and pinch strength.
• To identify more responsive outcome measures for IBM clinical trials.

Main Methods:

• A cross-sectional observational study was conducted with 83 IBM patients and 38 controls.
• Participants were recruited at The Myositis Association's 2019 Annual Patient Conference.
• New upper extremity function PROMs, adapted from NIH PROMIS, were assessed alongside pinch and grip strength.

Main Results:

• New IBM patient-reported outcome measures for hand function demonstrated a higher correlation with pinch and grip strength.
• The IBM-FS upper extremity domain showed less consistent changes compared to strength metrics.
• PROMs offer a potentially more sensitive assessment of upper extremity function in IBM.

Conclusions:

• Novel PROMs for hand function are more closely aligned with objective strength measures in IBM.
• These findings suggest that new PROMs may improve the ability to measure clinical trial efficacy in IBM.
• Further validation of these PROMs is recommended for use in IBM research and clinical practice.