Vexas syndrome successfully treated with canakinumab

Collantes‐Rodríguez, Cristina; Jiménez‐Gallo, David; Varga-Martínez, Raquel de la; Mora-López, Francisco; Garrastazul-Sánchez, María Paz; Linares‐Barrios, Mario

doi:10.1111/ddg.14933

Cited by 10 publications

(7 citation statements)

References 7 publications

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“…Canakinumab was reported to have controlled dermatologic inflammatory symptoms and may play a role when patients adversely react to anakinra. 55 However, a case report of a 68-year-old male with VEXAS showed immediate deterioration with elevation in inflammatory markers when anakinra was switched to canakinumab, suggesting caution should be used with this agent as well. 28 Another case reported on a 55-year-old male who acquired pneumonia that was refractory to treatment while being treated with a combination of prednisone, tocilizumab, canakinumab and etanercept, but when canakinumab and etanercept were discontinued, improvement was noted, suggesting respiratory status may be of particular concern in patient with VEXAS when considering treatment with canakinumab and/or etanercept.…”

Section: Role Of Biologic Agentsmentioning

confidence: 99%

“…Canakinumab has had an overall mixed response in VEXAS patients. Canakinumab was reported to have controlled dermatologic inflammatory symptoms and may play a role when patients adversely react to anakinra 55 . However, a case report of a 68‐year‐old male with VEXAS showed immediate deterioration with elevation in inflammatory markers when anakinra was switched to canakinumab, suggesting caution should be used with this agent as well 28 .…”

Section: Vexas Syndrome Treatmentsmentioning

confidence: 99%

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VEXAS syndrome: A review of cutaneous findings and treatments in an emerging autoinflammatory disease

Saad,

Patil,

Cruz

et al. 2024

Experimental Dermatology

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VEXAS (vacuoles, E1 enzyme, X‐linked, autoinflammatory and somatic mutation) syndrome is a novel autoinflammatory, late‐onset, disorder first identified in 2020. It is caused by mutations in the UBA1 gene. The most prominent clinical features reported by VEXAS patients are cutaneous and haematological, having characteristic skin features reported as the initial presenting findings of the disease. VEXAS is a severe and treatment‐resistant condition with high morbidity and mortality rates. Here, we examine all case reports and case series of VEXAS syndrome through March 2023 focusing on those presenting cutaneous manifestations. We discuss these manifestations and their reported treatment strategies. In many cases, it might be first suspected and diagnosed by dermatologists, highlighting their vital role in initiating timely multidisciplinary care.

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Section: Role Of Biologic Agentsmentioning

confidence: 99%

Section: Vexas Syndrome Treatmentsmentioning

confidence: 99%

VEXAS syndrome: A review of cutaneous findings and treatments in an emerging autoinflammatory disease

Saad,

Patil,

Cruz

et al. 2024

Experimental Dermatology

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“…62 Anti-IL1 therapies (anakinra, canakinumab) are commonly employed in patients with autoinflammatory diseases, however the outcomes in VEXAS have been mixed. While some series have reported clinical and biochemical response 17,63 others have noted difficulty in tolerance due to recurrent disease or severe cutaneous injection site reactions resulting in discontinuation. 2,24 We have observed similar results and, in our practice, tend to favor other cytokine directed therapies over IL-1 inhibitors.…”

Section: Uba1mentioning

confidence: 99%

VEXAS syndrome: Clinical, hematologic features and a practical approach to diagnosis and management

Koster,

Lasho,

Olteanu

et al. 2023

American J Hematol

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VEXAS (Vacuoles, E1 enzyme, X‐linked, Autoinflammatory, Somatic) syndrome is a newly identified disease caused by somatic alterations in UBA1 which produce a recalcitrant inflammatory state along with hematologic disturbances. Patients with VEXAS can have a wide spectrum of clinical symptoms and providers should be familiar with the heterogeneity of associated clinical features. While hematologic parameters may be generally non‐specific, peripheral blood features of macrocytosis, monocytopenia, and/or thrombocytopenia coupled with bone marrow vacuolization of erythroid or myeloid precursors should raise suspicion for this condition. Due to an increased mortality, prompt recognition and accurate diagnosis is paramount. Access to testing for confirmation of UBA1 variants is not yet universally available but clinicians should understand the current available options for genetic confirmation of this disease. Treatment options are limited due to lack of prospective clinical trials but cytokine directed therapies such as interleukin‐6 inhibitors and JAK–STAT inhibitors as well as hypomethylating agents such as azacitidine have shown evidence of partial effect. Though cases are limited, allogeneic stem cell transplantation holds promise for durable response and potential cure. The intent of this review is to outline the pathophysiology of VEXAS syndrome and to provide a practical approach to diagnosis and treatment.

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“…Treatment usually requires high doses of glucocorticoids, but recurrence during the tapering-off phase is common. Various systemic immunomodulatory agents (DMARDs, disease modifying antirheumatic drugs) have been tried for treating VEXAS syndrome with only moderate success; 30 better success has been reported for interleukin-1 receptor antagonists (anakinra), 8,13 anti-Interleukin-1β antibodies (canakinumab), 31 (also in combination with ciclosporin), 32 the interleukin-6 receptor antagonist tocilizumab, [33][34][35] the anti-IL-6 antibody siltuximab, 29 and also Janus kinase (JAK) inhibitors. In a retrospective analysis of 30 patients with VEXAS syndrome treated with JAK inhibitors, ruxolitinib emerged as the most effective agent: After six months of treatment, response rates up to 87% were reported.…”

Section: Prognosis and Treatment Optionsmentioning

confidence: 99%

VEXAS‐Syndrome, a newly described autoinflammatory systemic disease with dermatologic manifestations

Baur,

Stoevesandt,

Hueber

et al. 2023

J Deutsche Derma Gesell

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SummaryVEXAS syndrome is a recently identified autoinflammatory systemic disease caused by an acquired somatic mutation of the X‐linked UBA1 gene, the key enzyme of the first step of ubiquitylation. The acronym VEXAS stands for the characteristics Vacuoles, E1 enzyme, X‐linked, autoinflammatory and somatic. The disease occurs in advanced adulthood preferentially in men and is characterized by hematological, rheumatological and dermatological symptoms. The latter include neutrophil‐rich lesions reminiscent of Sweet's syndrome, erythema nodosum‐ and panniculitis‐like skin manifestations and recurrent polychondritis of the nose and auricles. The presence of cytoplasmic vacuoles in myeloid and erythroid precursors in the bone marrow is characteristic. In up to half of the cases, VEXAS syndrome is associated with myelodysplastic syndrome. Dermatologists should be familiar with the clinical picture, as skin symptoms are often the first indicator of the disease. Molecular diagnostics are essential for confirming the diagnosis and risk stratification of affected patients. In this minireview we provide an overview of the pathophysiology, diagnosis and therapy of VEXAS syndrome and illustrate its clinical picture with two own cases.

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Vexas syndrome successfully treated with canakinumab

Cited by 10 publications

References 7 publications

VEXAS syndrome: A review of cutaneous findings and treatments in an emerging autoinflammatory disease

VEXAS syndrome: A review of cutaneous findings and treatments in an emerging autoinflammatory disease

VEXAS syndrome: Clinical, hematologic features and a practical approach to diagnosis and management

VEXAS‐Syndrome, a newly described autoinflammatory systemic disease with dermatologic manifestations

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