Related Experiment Video
Updated: Jan 17, 2026
Author Spotlight: Evaluating the Adjuvant Efficacy and Safety of Angong Niuhuang Pill in Viral Encephalitis Treatment
Published on: April 19, 2024
JAK Inhibitors for Treatment of VEXAS Syndrome: A Systematic Review of 186 Cases
Saeed Bahramian1, Patrick Fazeli2, Arezou Rafati3
1School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran.
Abstract:
Objectives: Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome is an autoinflammatory disease with a wide spectrum of manifestations and no standard treatment. Janus kinase inhibitors (JAK-I) are small-molecule drugs that affect many molecular pathways. We aim to investigate the safety and efficacy of JAK-I in the treatment of VEXAS syndrome. Methods: A systematic search was conducted using MeSH terms/keywords related to JAK-I and VEXAS syndrome through PubMed/Medline, Scopus, Web of Science, and Embase until July 6, 2025. Results: We included 29 articles: 8 cohort, 8 case series, and 13 case reports. Our study includes data for 186 cases. The mean age was 69.64 years, and 83.33% were male. The most frequent manifestations were skin lesions (64.51%), fever (64.51%), arthritis and arthralgia (61.29%), lung involvement (31.72%), and venous thrombosis (24.19%). In general, 33.87% had a complete response, and 29.57% had a partial response. Ruxolitinib was used in 117 patients. Thirty-four out of 117 (29.06%) experienced complete to partial remission. Tofacitinib was used in 31 patients. About 29% of them showed complete to partial remission. Baricitinib was used in 25 patients; 12% had complete remission, and 16% had partial remission. Upadacitinib was used in 13 patients, which led to a complete remission in 38.46%. Filgotinib was used in four patients, leading to partial remission in one case. Among all, 36.55% showed adverse effects. Of these, eight were on Ruxolitinib, two on Tofacitinib, two on Baricitinib, and three on Upadacitinib. Conclusion: JAK-I seems to be a promising treatment option with tolerable adverse effects for VEXAS syndrome.
Insights
Janus kinase inhibitors (JAK-I) show promise for treating Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome. This systematic review found JAK-I effective in managing VEXAS symptoms with generally tolerable side effects.
Area of Science:
- Immunology
- Rheumatology
- Hematology
Background:
- Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome is a rare, severe autoinflammatory disorder with diverse clinical manifestations.
- Current treatment options for VEXAS syndrome are limited and lack standardization.
- Janus kinase inhibitors (JAK-I) modulate inflammatory pathways and are being explored for VEXAS syndrome treatment.
Purpose of the Study:
- To systematically evaluate the safety and efficacy of Janus kinase inhibitors (JAK-I) in patients diagnosed with VEXAS syndrome.
- To synthesize current evidence on JAK-I treatment outcomes, including response rates and adverse events.
Main Methods:
- A comprehensive systematic literature search was performed across major databases (PubMed/Medline, Scopus, Web of Science, Embase) up to July 6, 2025.
- Inclusion criteria focused on studies reporting on JAK-I treatment for VEXAS syndrome, encompassing cohort studies, case series, and case reports.
- Data extraction included patient demographics, clinical manifestations, JAK-I used, treatment response, and adverse events.
Main Results:
- The review analyzed data from 186 VEXAS syndrome patients across 29 articles, with a mean age of 69.64 years and 83.33% males.
- Common manifestations included skin lesions (64.51%), fever (64.51%), arthritis/arthralgia (61.29%), and lung involvement (31.72%).
- Overall, 33.87% achieved complete response and 29.57% partial response; Ruxolitinib, Tofacitinib, Baricitinib, and Upadacitinib showed varying efficacy rates. Adverse effects were reported in 36.55% of patients.
Conclusions:
- Janus kinase inhibitors (JAK-I) represent a promising therapeutic avenue for VEXAS syndrome, demonstrating significant clinical response rates.
- The safety profile of JAK-I in VEXAS syndrome appears tolerable, with manageable adverse events observed.
- Further research and larger clinical trials are warranted to establish optimal JAK-I regimens and long-term outcomes for VEXAS syndrome.
More Related Videos
Related Concept Videos
01:18Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
01:28Chemotherapy-Induced Nausea and Vomiting: Neurokinin-1 Receptor Antagonists
01:28Treatment for Pulmonary Arterial Hypertension: Phosphodiesterase Inhibitors
Among the PDE5 inhibitors, sildenafil (Revatio) stands out as a competitive and selective inhibitor. It operates by elevating cellular levels of cGMP and augmenting signaling through the cGMP-PKG pathway, promoting vasodilation. Upon oral...
01:26Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers
TKIs, such as imatinib (Gleevec), are particularly effective in tackling the growth and mitogenic factors that become upregulated in PAH patients. These factors contribute to the...
01:28Esophageal Varices-II: Clinical Features and Management
In the initial assessment, a thorough review of the patient's medical history is vital to identify risk factors such as liver disease, alcohol...
01:23Treatment for Pulmonary Arterial Hypertension: Prostacyclin Receptor Agonists
These agonists bind to the IPR receptor situated on the plasma membrane of the pulmonary artery smooth muscle cells. This binding triggers a cascade of reactions known as the GS-AC-cAMP-PKA pathway. This pathway results in the relaxation of smooth muscle...