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Table 1 List of rheumatic conditions where composite responder endpoints containing at least one continuous component are used

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 8.3 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

  1. *Denotes a single dichotomized continuous variable