ArticleInternational journal of molecular sciences2020
Biological and Clinical Changes in a Pediatric Series Treated with Off-Label JAK Inhibitors.
Article in International journal of molecular sciences, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 2 of them syntheses that pooled 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.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
20 citing papers in PubMed, 2 syntheses or guidelines pooled it, 31 citations in OpenAlex.
- JAK inhibitor therapy in CANDLE syndrome: a systematic review of clinical outcomes in 46 patients.European journal of pediatrics · 2026Pooled it
- Effectiveness and Safety of JAK Inhibitors in Autoinflammatory Diseases: A Systematic Review.Frontiers in medicine · 2022Pooled it
- JAK Inhibition inJIMD reports · 2026Article
- Distinct by Design: Unraveling the Unique Clinical and Transcriptomic Identity of Juvenile Scleromyositis Overlap Compared to Juvenile Systemic Sclerosis and Juvenile Dermatomyositis: Implications for Care and Pathogenesis.ACR open rheumatology · 2026Article
- Progress of biological agents and CAR cell therapy in the treatment of common autoimmune diseases in children.Frontiers in pediatrics · 2026Review
- Juvenile-onset Systemic Lupus Erythematosus: Recent Advances in Pathogenesis and Treatment.Current rheumatology reports · 2025Review
- New Challenging Systemic Therapies for Juvenile Scleroderma: A Comprehensive Review.Pharmaceuticals (Basel, Switzerland) · 2025Review
- COPA Syndrome-From Pathogenesis to Treatment.Diagnostics (Basel, Switzerland) · 2024Review
- Type I interferon pathway in pediatric systemic lupus erythematosus.World journal of pediatrics : WJP · 2024Review
- Tofacitinib for child-onset systemic lupus erythematosus.Frontiers in immunology · 2024Article
- Lupus Nephritis in Children: Novel Perspectives.Medicina (Kaunas, Lithuania) · 2023Review
- JAK Inhibition in Juvenile Idiopathic Arthritis (JIA): Better Understanding of a Promising Therapy for Refractory Cases.Journal of clinical medicine · 2023Review
- Novel heterozygousFrontiers in immunology · 2023Article
- A toddler with an unusually severe polyarticular arthritis and a lung involvement: a case report.BMC pediatrics · 2022Article
- Druggable monogenic immune defects hidden in diverse medical specialties: Focus on overlap syndromes.World journal of clinical pediatrics · 2022Review
- Off-label medication use in rare pediatric diseases in the United States.Intractable & rare diseases research · 2021Review
- The Pathogenesis, Molecular Mechanisms, and Therapeutic Potential of the Interferon Pathway in Systemic Lupus Erythematosus and Other Autoimmune Diseases.International journal of molecular sciences · 2021Review
- An Update on the Management of Childhood-Onset Systemic Lupus Erythematosus.Paediatric drugs · 2021Review
- Vasculitis, Autoimmunity, and Cytokines: How the Immune System Can Harm the Brain.International journal of environmental research and public health · 2021Review
- Emerging Place of JAK Inhibitors in the Treatment of Inborn Errors of Immunity.Frontiers in immunology · 2021Review
Corrections and comments
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Authors and funding
9 authors at 2 institutions in 1 country.
Funding
Abstract
Off-label use of medications is still a common practice in pediatric rheumatology. JAK inhibitors are authorized in adults in the treatment of rheumatoid arthritis, psoriatic arthritis and ulcerative colitis. Although their use is not authorized yet in children, JAK inhibitors, based on their mechanism of action and on clinical experiences in small series, have been suggested to be useful in the treatment of pediatric interferon-mediated inflammation. Accordingly, an increased interferon score may help to identify those patients who might benefit of JAK inhibitors. We describe the clinical experience with JAK inhibitors in seven children affected with severe inflammatory conditions and we discuss the correlation between clinical features and transcriptomic data. Clinical improvements were recorded in all cases. A reduction of interferon signaling was recorded in three out of seven subjects at last follow-up, irrespectively from clinical improvements. Other signal pathways with significant differences between patients and controls included upregulation of DNA repair pathway and downregulation of extracellular collagen homeostasis. Two patients developed drug-related adverse events, which were considered serious in one case. In conclusion, JAK inhibitors may offer a valuable option for children with severe interferon-mediated inflammatory disorders reducing the interferon score as well as influencing other signal pathways that deserve future studies.
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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.