Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis

IBM-Functional Rating Scale (IBM-FRS)

The IBM-FRS is a 10-item interviewer-administered patient-reported outcomes (PRO) measure that assesses physical function in patients with IBM. The IBMFRS includes items assessing lower body physical function, general physical function, the ability to perform certain activities of daily living, fine motor skills, and swallowing. On a Likert scale ranging from 0 (unable to perform activity) to 4 (normal), patients are asked to rate their difficulty in performing tasks relative to each concept before disease onset. Scores on the IBMFRS range from 0 to 40, with higher scores indicating better functioning.

IBM Physical Functioning Assessment (sIFA)

The sIFA is an 11-item self-administered PRO measure assessing physical function in patients with sIBM. This questionnaire will be completed by the patient on a numeric rating scale ranging from 0 (no difficulty) to 10 (unable to do), with a total score ranging from 0 to 100, where 100 indicates severe limitations. P atients will be asked to rate their difficulty with performing tasks involving lower and upper body physical function, general physical function, fine motor skills, and swallowing in the past 7 days.

Manual Muscle Testing

This tool assesses muscle strength using a 0 - 10-point scale. A set of 7 designated muscles is tested bilaterally plus axial (neck flexor) testing so that the potential MMT-8 score ranges from 0 - 150, where a higher score indicates better functioning. The designated muscles tested will be: * Neck flexors * Deltoid middle * Biceps brachii * Gluteus maximus or Iliopsoas * Gluteus Medius * Quadriceps * Wrist extensors * Ankle dorsiflexors

Hand Grip Strength

In this test, strength is measured in both the right and left hands separately with a hand grip dynamometer. Consistent angles are maintained for each patient across the shoulder, elbow, and wrist joints. Forearm and hand placement are standardized for each participant and the forearm is supported by a flat surface. Muscle strength for this test will be assessed in kilograms (kg).

Quadriceps Strength Test

In this test, strength is measured in both the right and left quadriceps with a dynamometer. Consistent 90-degree angles are maintained for each patient across the hip and knee joints in sitting position. Muscle strength for this test will be assessed in kilograms (kg).

Health Assessment Questionnaire Disability Index (HAQ/HAQ-DI)

This tool consists of 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities, each containing 2 to 3 questions. Scoring includes 0 (without any difficulty), 1 (with some difficulty), 2 (with much difficulty), and 3 (unable to perform). The score for each section is determined by its highest score. The HAQ-DI considers the subject's use of aids, devices, or assistance in the scoring algorithm for a disability category. If either "aids/devices" and/or "assistance from another person" are checked for a disability category, the score for this category is set to "2" (much difficulty), if the original score was "0" (no difficulty) or "1" (some difficulty). The HAQ-DI is then calculated by summing the adjusted categories scores and dividing by the number of categories answered. Scoring conventions are based on the Standard Disability Index of HAQ/HAQ-DI, using the 20 response items. A higher score indicates greater disability.