Emerging treatment approaches for VEXAS syndrome: a systematic review and meta-analysis DOI Creative Commons
Berkay Kılıç,

Efe Sacin,

Muhammet Kadir Tanin

et al.

Annals of Hematology, Journal Year: 2025, Volume and Issue: unknown

Published: April 27, 2025

Abstract VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a monogenic autoinflammatory disorder with significant morbidity and mortality. Numerous treatment options including azacitidine, JAK inhibitors, IL-6 anti-IL-1, anti-TNF agents have been proposed. However, no consensus on optimal algorithm has reached. This study aims to evaluate the efficacy safety of medical through meta-analysis existing data help establish clearer guidelines for managing VEXAS. The protocol was registered in PROSPERO (CRD42024590134). MEDLINE EMBASE were screened from inception until March 2025. We included patients who received or agents. primary outcome proportion complete responders. Partial response reported adverse events also evaluated. A total 16 studies 367 included. Concomitant myelodysplastic (MDS) 149 (40.6%) patients. Azacitidine resulted partial 67% [95% CI (0.56,0.77)] 73% (0.64,0.82)] cases, respectively. inhibitors produced 42% (0.33,0.52)] 79% (0.71,0.87)]. led 24% (0.15,0.32)] 72% (0.64,0.81)]. Adverse frequently observed. demonstrated MDS. may be viable options. Prospective clinical trials are needed further confirmation results.

Language: Английский

Emerging treatment approaches for VEXAS syndrome: a systematic review and meta-analysis DOI Creative Commons
Berkay Kılıç,

Efe Sacin,

Muhammet Kadir Tanin

et al.

Annals of Hematology, Journal Year: 2025, Volume and Issue: unknown

Published: April 27, 2025

Abstract VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a monogenic autoinflammatory disorder with significant morbidity and mortality. Numerous treatment options including azacitidine, JAK inhibitors, IL-6 anti-IL-1, anti-TNF agents have been proposed. However, no consensus on optimal algorithm has reached. This study aims to evaluate the efficacy safety of medical through meta-analysis existing data help establish clearer guidelines for managing VEXAS. The protocol was registered in PROSPERO (CRD42024590134). MEDLINE EMBASE were screened from inception until March 2025. We included patients who received or agents. primary outcome proportion complete responders. Partial response reported adverse events also evaluated. A total 16 studies 367 included. Concomitant myelodysplastic (MDS) 149 (40.6%) patients. Azacitidine resulted partial 67% [95% CI (0.56,0.77)] 73% (0.64,0.82)] cases, respectively. inhibitors produced 42% (0.33,0.52)] 79% (0.71,0.87)]. led 24% (0.15,0.32)] 72% (0.64,0.81)]. Adverse frequently observed. demonstrated MDS. may be viable options. Prospective clinical trials are needed further confirmation results.

Language: Английский

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