Table 1.
Core set items selected for 11 domains
| Agreement from round 3 Median (25th–75th percentile) |
||
|---|---|---|
| Skin: | ||
| 1 | Modified Rodnan skin score (range 0–51) | 9 (9–9) |
| 2 | Visual analogue scale (VAS)/Likert score of patient’s global assessment for skin activity* | 7 (5.5–9) |
| 3 | VAS/Likert score of doctor’s global assessment for skin activity* | 7 (6–9) |
| 4 | Durometer | 7 (6–8) |
| Musculoskeletal: | ||
| 1 | Tender joint count | 6 (5–8) |
| 2 | Tendon friction rubs assessed by the doctor* | 8 (7–9) |
| 3 | Serum creatinine phosphokinase, aldolase | 7 (6–9) |
| Cardiac: | ||
| 1 | (1)Cardiac echocardiogram with Doppler† | 9 (8–9) |
| 2 | (2)Right heart catheterisation‡ | 8 (6–9) |
| 3 | 6-min walk test‡ | 8 (7–9) |
| 4 | Borg dyspnoea instrument‡ | 7 (6–9) |
| Pulmonary: | ||
| 1 | (3)Pulmonary function testing | 9 (9–9) |
| 2 | (A)Validated measure of dyspnoea | 8 (7–9) |
| 3 | Breathing VAS from the Scleroderma Health Assessment Questionnaire (S-HAQ)23 | 7 (6–9) |
| 4 | High resolution computer tomography (HRCT) of the lungs: quantifiable scale‡ | 9 (7–9) |
| Renal: | ||
| 1 | Calculated creatinine clearance based on serum creatinine (Cockroft–Gault or Modification of Diet in Renal Disease (MDRD) formula) | 9 (7–9) |
| 2 | Pre-defined renal crisis (presence or absence) | 9 (8–9) |
| Gastrointestinal: | ||
| 1 | Body mass index (BMI) | 7 (6–9) |
| 4 | Validated gastrointestinal (GI) tract VAS scale (part of S-HAQ)23 or other SSc-validated GI questionnaire | 8 (7–9) |
| Health-related quality of life and function: | ||
| 1 | Health Assessment Questionnaire-Disability Index (HAQ-DI)24 | 9 (7–9) |
| 2 | VAS pain from the HAQ-DI24 | 7 (6–9) |
| 3 | Short form-36 (SF-36) version 225 | 8 (7–9) |
| Global health: | ||
| 1 | VAS/Likert patient global severity | 8 (6.5–9) |
| 2 | VAS/Likert doctor global severity | 8 (6–9) |
| 3 | (B)Scleroderma-related health transition by patient | 7 (6–9) |
| 4 | (C)Scleroderma-related health transition by doctor¶ | 7 (5–8) |
| Raynaud phenomenon: | ||
| 1 | Raynaud condition score26 | 8 (7–9) |
| 2 | VAS Raynaud (part of S-HAQ)23 | 8 (6.5–9) |
| Digital ulcers: | ||
| 1 | Active digital tip ulcer count on the volar surface | 9 (7–9) |
| 2 | VAS digital ulcer (part of S-HAQ)23 | 8 (6–9) |
| Biomarkers: | ||
| 1 | Acute phase reactant(s): erythrocyte sedimentation rate (ESR) and/or C-reactive protein (CRP) | 8 (6–9) |
Parameters to be measured for the items in the core set:
- Echocardiogram with Doppler: measure pulmonary artery systolic pressure, left and right ventricular systolic and diastolic parameters, pericardial effusion, and chamber size.
- Right heart catheterisation: pulmonary artery pressure, pulmonary vascular resistance, wedge pressure, cardiac output/cardiac index.
- Pulmonary function test: forced vital capacity (FVC), diffusion capacity (DLCO), total lung capacity (TLC).
- Validated dyspnoea questionnaires, eg, University of California San Diego (UCSD) Dyspnoea Questionnaire, St. George Respiratory Questionnaire, Mahler Dyspnoea Index.
- Scleroderma-related health transition by patient; compared to 1 year ago, how do you rate your overall scleroderma: much better, a little better, no change, a little worse, much worse?
- Scleroderma-related health transition by doctor; compared to 1 year ago, how do you rate your patient’s overall scleroderma: much better, a little better, no change, a little worse, much worse?
*
Items were based on Steering Committee and Scleroderma Clinical Trials Consortium (SCTC) consensus despite lack of full validation.
†
Standardised central reading mechanism strongly encouraged,
‡
if relevant to the study.