From: Increasing power in the analysis of responder endpoints in rheumatology: a software tutorial
Condition | Endpoint | Response definition |
---|---|---|
Acute Gout | Proportion of patients who responded* | 1. sUA level of < 6.0 mg |
Ankylosing spondylitis | ASAS20 response | 1. 20% improvement and ≥ 10 units of change (on a 0–100 scale) in each of 3 domains 2. No worsening of a similar amount in the fourth domain (Components are physical function, pain, inflammation and patient’s global assessment) |
Idiopathic arthritis-associated uveitis | Best corrected visual acuity above threshold and no light perception | 1. Best-corrected visual acuity, thresholds ≤ 20/50, ≤ 20/200 2. No light perception 3. Estimate contribution of amblyopia, yes/no |
Juvenile arthritis | Response | 1. Improvement by 30% in at least 3 of: a. MD global assessment; b. parent or patient global assessment c. functional ability; d. number of joints with active arthritis; e. number of joints with limited range of motion; f. erthrocyte sedimentation rate |
Juvenile dermatomyositis | Responder index |  1. ≥ 4 point reduction from baseline in safety of estrogen in lupus national assessment (SELENA) systemic lupus erythematosus disease activity index (SLEDAI) score 2. No worsening (increase of <0.30 points from baseline) in physician's global assessment (PGA) 3. No new British Isles Lupus Assessment Group of SLE clinics (BILAG) A organ domain score or 2 new BILAG B organ domain scores compared with baseline |
Prevention of fracture in high-risk populations | Response | 1. Bone mineral density increase 2. Occurrence of new vertebral fractures |
Proliferative and membranous lupus renal disease | Urinary protein levels within normal range* | 1. Between 6 and 8.3Â g per deciliter (g/dL) |
Rheumatoid arthritis | ACR20 response |  1. ≥ 20% improvement in ACR score 2. Can be combined with additional requirements e.g. no additional medication |
Sarcopenia prevention | Occurrence of sarcopenia | Heterogeneity in precise definition, but severe sarcopenia defined by all of the following: 1. Low muscle strength (assessed with chair stand test or grip strength) 2. Low muscle quantity/quality 3. Low physical performance as assessed with gait speed test or short physical performance battery |
Sjogren's syndrome | Response | 1.  > 30% reduction in analog scales evaluating dryness, pain and fatigue |
Systemic lupus erythematosus | SRI responder index | 1. SLEDAI change e.g. ≤− 4 2. PGA change e.g. <0.3 3. No Grade A or more than one Grade B in BILAG |
Systemic sclerosis | SCP in normal range, no renal crisis | E.g  1. <3.0 mg/dl not drug related 2. No renal crisis |
Vasculitis disorders | Response/partial improvement* | 1. 50% improvement in disease activity score |