Evidence map›Paper›PMID 33258553›Full record

ArticleArthritis & rheumatology (Hoboken, N.J.)2021

Study of Tofacitinib in Refractory Dermatomyositis: An Open-Label Pilot Study of Ten Patients.

Julie J Paik, Livia Casciola-Rosen, Joseph Yusup Shin, Jemima Albayda, Eleni Tiniakou, Doris G Leung, Laura Gutierrez-Alamillo, Jamie Perin, Liliana Florea, Corina Antonescu and 4 more

Registry-linked trialOpen access · greenAbstract read
In one paragraph

Article in Arthritis & rheumatology (Hoboken, N.J.), 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06857240 (Topical Ruxolitinib Cream for Refractory Cutaneous Dermatomyositis), which is not on this map. Cited by 79 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
79citing papers in PubMed, 4 pooled it
13.5field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT06857240 phase2recruitingnot on this mapstarted 2025, after this paper: background citation

Topical Ruxolitinib Cream for Refractory Cutaneous Dermatomyositis

TypeinterventionalSponsorThe Cleveland ClinicRan2025 to 2027Enrolled15ConditionsDermatomyositisArmsRuxolitinib Topical Cream
3 · Its place in the literature

Who cites it

79 citing papers in PubMed, 4 syntheses or guidelines pooled it, 151 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Guideline
  4. Pooled it
  5. Trial
  6. Trial
  7. New treatments in myositis on the horizon.EULAR rheumatology open · 2026
    Review
  8. Emerging therapies in idiopathic inflammatory myopathies.Journal of neuromuscular diseases · 2026
    Review
  9. Article
  10. Article
  11. Review
  12. Article
  13. Article
  14. Review
  15. Review
  16. Review
  17. Article
  18. Targeted Therapy for Idiopathic Inflammatory Myopathy.Journal of cachexia, sarcopenia and muscle · 2025
    Review
  19. Review
  20. Article

19 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

14 authors at 2 institutions in 1 country.

Julie J PaikJohns Hopkins University School of Medicine, Baltimore, Maryland.ORCID 0000-0001-8436-1601
Livia Casciola-RosenJohns Hopkins University School of Medicine, Baltimore, Maryland.ORCID 0000-0002-4172-1539
Joseph Yusup ShinJohns Hopkins University School of Medicine, Baltimore, Maryland.
Jemima AlbaydaJohns Hopkins University School of Medicine, Baltimore, Maryland.ORCID 0000-0002-8030-9504
Eleni TiniakouJohns Hopkins University School of Medicine, Baltimore, Maryland.ORCID 0000-0003-4749-870X
Doris G LeungJohns Hopkins University School of Medicine, Baltimore, Maryland.
Laura Gutierrez-AlamilloJohns Hopkins University School of Medicine, Baltimore, Maryland.
Jamie PerinJohns Hopkins University School of Medicine, Baltimore, Maryland.
Liliana FloreaJohns Hopkins University School of Medicine, Baltimore, Maryland.
Corina AntonescuJohns Hopkins University School of Medicine, Baltimore, Maryland.
Sherry G LeungJohns Hopkins University School of Medicine, Baltimore, Maryland.
Grazyna PurwinJohns Hopkins University School of Medicine, Baltimore, Maryland.
Andrew KoenigPfizer Inc., New York, New York.
Lisa Christopher-StineJohns Hopkins University School of Medicine, Baltimore, Maryland.ORCID 0000-0002-7150-7306
Johns Hopkins University · USPfizer (United States) · US

Funding

Sample Processing and Immunoassay Research CoreP30AR070254 · NIAMS · JOHNS HOPKINS UNIVERSITY · PI Antony Rosen · 2016 to 2026
$8.9M
Computational Methods to Characterize Alternative Splicing from Massive Collections of RNA-seq DataR01GM129085 · NIGMS · JOHNS HOPKINS UNIVERSITY · PI FLOREA, LILIANA D · 2019 to 2022
$1.5M
Magnetic resonance imaging and spectroscopy biomarkers for facioscapulohumeral muscular dystrophyK23NS091379 · NINDS · HUGO W. MOSER RES INST KENNEDY KRIEGER · PI LEUNG, DORIS G · 2015 to 2019
$885k
NIAMS NIH HHS P30 AR070254NIGMS NIH HHS R01 GM129085NIH HHS P30-AR070254NINDS NIH HHS K23 NS091379
6 · The paper itself

Abstract

objectiveThis open-label 12-week study was conducted to evaluate the efficacy and safety of tofacitinib, a JAK inhibitor, in treatment-refractory active dermatomyositis (DM).

methodsTofacitinib in extended-release doses of 11 mg was administered daily to 10 subjects with DM. Prior to treatment, a complete washout of all steroid-sparing agents was performed. The primary outcome measure was assessment of disease activity improvement based on the International Myositis Assessment and Clinical Studies group definition of improvement. Response rate was measured as the total improvement score according to the 2016 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) myositis response criteria. Secondary outcome measures included Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) scores, chemokine levels, immunohistochemical analysis of STAT1 expression in the skin, RNA sequencing analysis, and safety.

resultsAt 12 weeks, the primary outcome was met in all 10 subjects. Five (50%) of 10 subjects experienced moderate improvement in disease activity, and the other 50% experienced minimal improvement according to the 2016 ACR/EULAR myositis response criteria. The secondary outcome of the mean change in the CDASI activity score over 12 weeks was statistically significant (mean ± SD 28 ± 15.4 at baseline versus 9.5 ± 8.5 at 12 weeks) (P = 0.0005). Serum chemokine levels of CXCL9/CXCL10 showed a statistically significant change from baseline. A marked decrease in STAT1 signaling in association with suppression of interferon target gene expression was demonstrated in 3 of 9 skin biopsy samples from subjects with dermatomyositis. The mean ± SD level of creatine kinase in the 10 subjects at baseline was 82 ± 34.8 IU/liter, highlighting that disease activity was predominantly located in the skin.

conclusionThis is the first prospective, open-label clinical trial of tofacitinib in DM that demonstrates strong clinical efficacy of a pan-JAK inhibitor, as measured by validated myositis response criteria. Future randomized controlled trials using JAK inhibitors should be considered for treating DM.

Indexed as

AdultChemokine CXCL10Chemokine CXCL9DermatomyositisFemaleHumansImmunohistochemistryJanus Kinase InhibitorsMaleMiddle AgedMuscle, SkeletalPilot ProjectsPiperidinesProof of Concept StudyProspective StudiesPyrimidinesChemokine CXCL10Chemokine CXCL9CXCL10 protein, humanCXCL9 protein, humanJanus Kinase InhibitorsPiperidinesPyrimidinesSTAT1 protein, humanSTAT1 Transcription Factortofacitinib

Identifiers

PMID33258553
PMCPMC8084900
OpenAlexW3109127380

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.