PMID- 29077900 OWN - NLM STAT- MEDLINE DCOM- 20171226 LR - 20190107 IS - 1462-0332 (Electronic) IS - 1462-0324 (Print) IS - 1462-0324 (Linking) VI - 57 IP - 1 DP - 2018 Jan 1 TI - Patient acceptable symptom state in scleroderma: results from the tocilizumab compared with placebo trial in active diffuse cutaneous systemic sclerosis. PG - 152-157 LID - 10.1093/rheumatology/kex396 [doi] AB - OBJECTIVES: Patient acceptable symptom state (PASS) as an absolute state of well-being has shown promise as an outcome measure in many rheumatologic conditions. We aimed to assess whether PASS may be effective in active diffuse cutaneous SSc differentiating active from placebo. METHODS: Data from the phase 2 Safety and Efficacy of Subcutaneous Tocilizumab in Adults with Systemic Sclerosis (faSScinate) trial were used, which compared tocilizumab (TCZ) vs placebo over 48 weeks followed by an open-label TCZ period to 96 weeks. Three different types of PASS questions were evaluated at weeks 8, 24, 48 and 96, including if a current state would be acceptable over time as a yes vs no response and Likert scales about how acceptable a current state is if remaining over time. Additional outcomes assessed included modified Rodnan skin score, HAQ disability index (HAQ-DI), physician and patient global assessments on a visual analogue scale, CRP and ESR. RESULTS: The placebo group consisted of 44 patients and the TCZ group had 43 patients. At baseline, 33% achieved a PASS for all three PASS questions, with the proportion increasing to 69, 71 and 78%, respectively, at 96 weeks. Changes in PASS scores showed a moderately negative correlation with HAQ-DI and patient and physician global assessments visual analogue scales, which indicates expected improvements as PASS improved. The PASS question, 'Considering all of the ways your scleroderma has affected you, how acceptable would you rate your level of symptoms?' showed significant correlations with patient-reported outcomes and differentiating placebo vs TCZ at 48 weeks (P = 0.023). CONCLUSION: PASS may be used as a patient-centred outcome in SSc, especially as a 7-point Likert scale. Further validation is required to determine the utility as an outcome measure in trials and clinical practice. CI - (c) The Author 2017. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For Permissions, please email: journals.permissions@oup.com FAU - Arnold, Michael B AU - Arnold MB AD - Schulich School of Medicine and Dentistry, Western University, London, ON, Canada. FAU - Khanna, Dinesh AU - Khanna D AD - Rheumatology, University of Michigan Scleroderma Program, Ann Arbor, MI, USA. FAU - Denton, Christopher P AU - Denton CP AD - Rheumatology and Connective Tissue Diseases, University College London Medical School, London, UK. FAU - van Laar, Jacob M AU - van Laar JM AD - Rheumatology & Clinical Immunology, University of Utrecht, Utrecht, The Netherlands. FAU - Frech, Tracy M AU - Frech TM AD - Rheumatology, University of Utah, Veterans Affairs Medical Center, Salt Lake City, UT, USA. FAU - Anderson, Marina E AU - Anderson ME AD - Rheumatology, University of Liverpool and Aintree University Hospital, Liverpool, UK. FAU - Baron, Murray AU - Baron M AD - Rheumatology, Jewish General Hospital, Montreal, QC, Canada. FAU - Chung, Lorinda AU - Chung L AD - Medicine and Dermatology, Stanford University School of Medicine and Palo Alto VA Health Care System, Palo Alto, CA, USA. FAU - Fierlbeck, Gerhard AU - Fierlbeck G AD - Dermatology, University of Tubingen, Tubingen, Germany. FAU - Lakshminarayanan, Santhanam AU - Lakshminarayanan S AD - Rheumatology, University of Connecticut Health Center, Farmington, CT, USA. FAU - Allanore, Yannick AU - Allanore Y AD - Rheumatology, Paris Descartes University, Paris, France. FAU - Riemekasten, Gabriela AU - Riemekasten G AD - Rheumatology, Charite University Hospital, German Rheumatism Research Center, Berlin, Germany. FAU - Steen, Virginia AU - Steen V AD - Rheumatology, Georgetown University, Washington, DC, USA. FAU - Muller-Ladner, Ulf AU - Muller-Ladner U AD - Lehrstuhl fur Innere Medizin mit Schwerpunkt Rheumatologie, Justus-Liebig University Giessen, Kerckhoff Clinic, Bad Nauheim, Germany. FAU - Spotswood, Helen AU - Spotswood H AD - Roche Products Ltd, Welwyn Garden City, UK. FAU - Burke, Laura AU - Burke L AD - Roche Products Ltd, Welwyn Garden City, UK. FAU - Siegel, Jeffrey AU - Siegel J AD - Rheumatology and Rare Diseases, Genentech, South San Francisco. FAU - Jahreis, Angelika AU - Jahreis A AD - Rheumatology and Rare Diseases, Genentech, South San Francisco. FAU - Furst, Daniel E AU - Furst DE AD - Rheumatology, University of California, Los Angeles, CA, USA. FAU - Pope, Janet E AU - Pope JE AD - Schulich School of Medicine and Dentistry, Western University, London, ON, Canada. LA - eng PT - Clinical Trial, Phase II PT - Journal Article PT - Randomized Controlled Trial PT - Research Support, Non-U.S. Gov't PL - England TA - Rheumatology (Oxford) JT - Rheumatology (Oxford, England) JID - 100883501 RN - 0 (Antibodies, Monoclonal, Humanized) RN - 0 (Antirheumatic Agents) RN - I031V2H011 (tocilizumab) SB - IM MH - Adult MH - Antibodies, Monoclonal, Humanized/*therapeutic use MH - Antirheumatic Agents/*therapeutic use MH - Female MH - Humans MH - Male MH - Middle Aged MH - *Patient Reported Outcome Measures MH - *Patient Satisfaction MH - Scleroderma, Diffuse/*drug therapy/physiopathology MH - Treatment Outcome PMC - PMC5850780 OTO - NOTNLM OT - PASS OT - diffuse systemic sclerosis OT - outcome measure OT - patient acceptable symptom state OT - patient-reported outcomes OT - scleroderma OT - systemic sclerosis OT - tocilizumab EDAT- 2017/10/28 06:00 MHDA- 2017/12/27 06:00 PMCR- 2019/01/01 CRDT- 2017/10/28 06:00 PHST- 2017/01/28 00:00 [received] PHST- 2017/10/28 06:00 [pubmed] PHST- 2017/12/27 06:00 [medline] PHST- 2017/10/28 06:00 [entrez] PHST- 2019/01/01 00:00 [pmc-release] AID - 4565530 [pii] AID - kex396 [pii] AID - 10.1093/rheumatology/kex396 [doi] PST - ppublish SO - Rheumatology (Oxford). 2018 Jan 1;57(1):152-157. doi: 10.1093/rheumatology/kex396.