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Study finds JAK inhibitors and tocilizumab effective in VEXAS syndrome - Video

Published On 2023-11-10T09:30:00+05:30  |  Updated On 10 Nov 2023 9:30 AM IST
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Overview

A recent study presented at ACR Convergence 2023 has offered newfound hope for individuals battling VEXAS syndrome, a rare and often lethal autoimmune disorder resulting from a UBA1 gene mutation. The research demonstrates the superior effectiveness of JAK inhibitors (JAKi) and tocilizumab in managing VEXAS syndrome, in stark comparison to other targeted therapies.

VEXAS syndrome is a debilitating condition characterized by extensive inflammation affecting the skin, lungs, blood vessels, and joints. Typically, high-dose corticosteroids are employed in its management, but these treatments come with severe side effects and limited supporting data. Alarmingly, the five-year survival rate for VEXAS patients is strikingly low.

The study enrolled a predominantly male patient cohort from the French national VEXAS registry, spanning from November 2020 to August 2023. All patients had received at least one targeted therapy, with a significant portion undergoing JAKi ruxolitinib or the interleukin (IL)-6 inhibitor tocilizumab.

After three months, the findings revealed an overall response rate of 24% for JAK inhibitors, 32% for IL-6 inhibitors, 9% for IL-1 inhibitors, and 0% for TNF blockers and other targeted medications. At six months, the overall response rate for JAK inhibitors slightly increased to 30%, while IL-6 drugs dipped to 26%. Notably, the numbers of complete and partial responses were similar, and the withdrawal of corticosteroids exhibited comparable patterns.

A more pronounced contrast arose concerning treatment discontinuation during follow-up: 28% for JAKi (with a median delay of 7.2 months) versus 69% for IL-6 inhibitors (with a median delay of 5.1 months). Although serious side effects were almost double with IL-6 inhibitors, there were fewer fatalities compared to JAKi. This underscores the instrumental role of JAK inhibitors and tocilizumab in revolutionizing VEXAS syndrome treatment.

Reference: AMERICAN COLLEGE OF RHEUMATOLOGY

Speakers

Isra Zaman

B.Sc Life Sciences, M.Sc Biotechnology, B.Ed

Isra Zaman is a Life Science graduate from Daulat Ram College, Delhi University, and a postgraduate in Biotechnology from Amity University. She has a flair for writing, and her roles at Medicaldialogues include that of a Sr. content writer and a medical correspondent. Her news pieces cover recent discoveries and updates from the health and medicine sector. She can be reached at editorial@medicaldialogues.in.

Dr. Kamal Kant Kohli

Dr Kamal Kant Kohli-MBBS, DTCD- a chest specialist with more than 30 years of practice and a flair for writing clinical articles, Dr Kamal Kant Kohli joined Medical Dialogues as a Chief Editor of Medical News. Besides writing articles, as an editor, he proofreads and verifies all the medical content published on Medical Dialogues including those coming from journals, studies,medical conferences,guidelines etc. Email: drkohli@medicaldialogues.in. Contact no. 011-43720751
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