Comparison between idiopathic and VEXAS-relapsing polychondritis: analysis of a French case series of 95 patients

Khitri, Mohamed-Yacine; Guédon, A.; Georgin-Lavialle, S.; Terrier, Benjamin; Saadoun, David; Séguier, J.; Besnerais, M. Le; Moreuil, Claire de; Denis, Guillaume; Gerfaud‐Valentin, Mathieu; Allain, Jean Sebastien; Maria, A.; Bouillet, Laurence; Grobost, Vincent; Galland, J.; Kosmider, Olivier; Dumont, Anaël; Devaux, Mathilde; Subran, Benjamin; Schmidt, Jean M.; Marianetti-Guingel, Paola; Audia, Sylvain; Palat, S.; Roux-Sauvat, Marielle; Jachiet, Vincent; Hirsch, Pierre; Fain, Olivier; Mékinian, A.

doi:10.1136/rmdopen-2022-002255

Cited by 59 publications

(79 citation statements)

References 42 publications

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“…All 5 primary studies [14][15][16][17][18] were non-randomized studies. Four of the studies [14][15][16][17] were retrospective cohort studies; 3 of these [15][16][17] were uncontrolled (i.e., single-arm cohort studies), and the fourth 14 included within-patient comparisons (i.e., the same patient may have received multiple lines of therapy) between outcomes associated with ruxolitinib, tocilizumab, and azacitidine, compared with corticosteroids. The fifth study 18 was a single-arm prospective clinical trial, in which the diagnosis of VEXAS was made retrospectively because the UBA1 mutation associated with VEXAS was discovered after the trial had been started.…”

Section: Methodsmentioning

confidence: 99%

“…In 1 study, 15 the study eligibility criteria aligned with the current report, while in the other 4 studies, 14,[16][17][18] the eligibility criteria were broader, yet the data were summarized for relevant subgroups.…”

Section: Methodsmentioning

confidence: 99%

“…All 5 studies [14][15][16][17][18] were published in 2021 14 or 2022, [15][16][17][18] with study periods ranging from July 2017 18 to February 2022 16 (where reported).…”

Section: Methodsmentioning

confidence: 99%

“…The characteristics of the 5 primary studies [14][15][16][17][18] are summarized in the subsequent sections, and additional details are provided in Appendix 2.…”

Section: Summary Of Study Characteristicsmentioning

confidence: 99%

“…All 5 primary studies [14][15][16][17][18] included sites in France. Of these, 1 was a multi-country study, 16 including patients treated in France, the US, and Portugal.…”

Section: Country Of Originmentioning

confidence: 99%

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Treatment Options for VEXAS Syndrome

Goring¹,

Horton²

2022

cjht

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VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a disorder caused by a genetic mutation, and is characterized by hematologic and autoinflammatory symptoms that are severe and progressive. It is typically seen in older men and is considered to be rare. Proposed treatments for VEXAS syndrome include DNA hypomethylating agents such as azacitidine, anti-IL6 monoclonal antibodies such as tocilizumab, and Janus kinase inhibitors such as ruxolitinib. We found 5 non-randomized studies on the effectiveness of the proposed treatments — 4 on azacitidine; 1 on ruxolitinib; and 1 on azacitidine, ruxolitinib, and tocilizumab. There are several limitations to the research studies, most notably that they included a small number of patients who were retrospectively identified as having VEXAS syndrome. Therefore, the effectiveness of tocilizumab, ruxolitinib, and azacitidine for treating VEXAS syndrome is uncertain. We did not find any evidence-based guidelines on the management of VEXAS syndrome.

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Section: Methodsmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

“…All 5 studies [14][15][16][17][18] were published in 2021 14 or 2022, [15][16][17][18] with study periods ranging from July 2017 18 to February 2022 16 (where reported).…”

Section: Methodsmentioning

confidence: 99%

“…The characteristics of the 5 primary studies [14][15][16][17][18] are summarized in the subsequent sections, and additional details are provided in Appendix 2.…”

Section: Summary Of Study Characteristicsmentioning

confidence: 99%

“…All 5 primary studies [14][15][16][17][18] included sites in France. Of these, 1 was a multi-country study, 16 including patients treated in France, the US, and Portugal.…”

Section: Country Of Originmentioning

confidence: 99%

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