Work

Azacitidine as therapy for VEXAS syndrome

Year: 2025
Type: article
Abstract: The key message of the article is the favorable outcome with an acceptable safety profile of this combination with a 1year PFS of 36.2% and 1-year OS of 66.0%.The multivariable analysis revealed that 2 factors (the presence of CCR4 gene mutation and mogamulizumab-associated adverse events) were associated with a better OS.Finally, the study met its initial goal, which was aimed to show a clinically significant improvement of PFS in older patients with CCR4-positive aggressive ATLL, compared with a historical prospective study.However, this study has several limitations that should be considered for follow-up studies.The initial strategy was to administrate CHOP every 2 weeks.However, only 20 (42.6%) received CHOP-14, among whom 12 (25.5%)completed 6 cycles, suggesting that CHOP-21 regimen seems to be more tolerable regimen.This study used response criteria published in 2009 that seem now outdated.10 Notably, this study has not included modern tools for response assessment, such as flow cytometry and/or positron emission tomography-computed tomography.Moreover, this study does not address the relevance of maintenance therapy.Indeed, despite a high response rate, the 1-year PFS was only 36.2%.Finally, as mentioned above, mogamulizumab is not registered for ATLL treatment in Europe and in the United States.Despite these caveats, this study brings new insights regarding the management of patients experiencing aggressive ATLL and who are not eligible for alloSCT.This population will increase as the median age at onset of ATLL in Japan has risen to nearly 70 years.This article will be of interest for clinicians who treat patients with ATLL and will help in designing new clinical studies.
Source: Blood
Institution: Mayo Clinic
Language: English
Field: Medicine
Open Access status: bronze
OpenAlex ID: W4414340618
FWCI: 1.795
Cites: 9
Cited by: 3
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