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Riociguat in patients with early diffuse cutaneous systemic sclerosis (RISE-SSc): randomised, double-blind, placebo-controlled multicentre trial

2020· article· en· W3016646661 on OpenAlexaff
Dinesh Khanna, Yannick Allanore, Christopher P. Denton, Masataka Kuwana, Marco Matucci‐Cerinic, Janet Pope, Tatsuya Atsumi, Radim Bečvář, László Czirják, É. Hachulla, Tomonori Ishii, Osamu Ishikawa, Sindhu R. Johnson, Ellen De Langhe, Chiara Stagnaro, Valeria Riccieri, Elena Schiopu, Richard M. Silver, Vanessa Smith, Virginia Steen, Wendy Stevens, Gabriella Szűcs, Marie‐Elise Truchetet, Melanie Wosnitza, Kaisa Laapas, Janethe de Oliveira Pena, Zhen Yao, Frank Kramer, Oliver Distler

Bibliographic record

VenueAnnals of the Rheumatic Diseases · 2020
Typearticle
Languageen
FieldMedicine
TopicSystemic Sclerosis and Related Diseases
Canadian institutionsToronto Western HospitalUniversity of TorontoUniversity Health NetworkMount Sinai HospitalWestern University
FundersChugai PharmaceuticalGenentechMedacAstellas PharmaOno PharmaceuticalEisaiSanofiBayer HealthCareBristol-Myers SquibbEli Lilly and CompanyCSL BehringAlexion PharmaceuticalsImmune Tolerance NetworkGlaxoSmithKlineBayerDaiichi-SankyoPfizer
KeywordsMedicineRiociguatDouble blindPlaceboScleroderma (fungus)Double blindedPlacebo-controlled studyRandomized controlled trialInternal medicinePathologyPulmonary hypertensionChronic thromboembolic pulmonary hypertension

Abstract

fetched live from OpenAlex

OBJECTIVES: Riociguat is approved for pulmonary arterial hypertension and has antiproliferative, anti-inflammatory and antifibrotic effects in animal models of tissue fibrosis. We evaluated the efficacy and safety of riociguat in patients with early diffuse cutaneous systemic sclerosis (dcSSc) at high risk of skin fibrosis progression. METHODS: In this randomised, double-blind, placebo-controlled, phase IIb trial, adults with dcSSc of <18 months' duration and a modified Rodnan skin score (mRSS) 10-22 units received riociguat 0.5 mg to 2.5 mg orally three times daily (n=60) or placebo (n=61). The primary endpoint was change in mRSS from baseline to week 52. RESULTS: At week 52, change from baseline in mRSS units was -2.09±5.66 (n=57) with riociguat and -0.77±8.24 (n=52) with placebo (difference of least squares means -2.34 (95% CI -4.99 to 0.30; p=0.08)). In patients with interstitial lung disease, forced vital capacity declined by 2.7% with riociguat and 7.6% with placebo. At week 14, average Raynaud's condition score had improved ≥50% in 19 (41.3%)/46 patients with riociguat and 13 (26.0%)/50 patients with placebo. Safety assessments showed no new signals with riociguat and no treatment-related deaths. CONCLUSIONS: Riociguat did not significantly benefit mRSS versus placebo at the predefined p<0.05. Secondary and exploratory analyses showed potential efficacy signals that should be tested in further trials. Riociguat was well tolerated.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.001
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Randomized trial · Consensus signal: Randomized trial
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.015
Threshold uncertainty score0.972

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.001
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0020.001
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.047
GPT teacher head0.264
Teacher spread0.217 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designRandomized trial
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations107
Published2020
Admission routes1
Has abstractyes

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