VEXAS syndrome: complete molecular remission after hypomethylating therapy

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

Abstract

The VEXAS syndrome, a genetically defined autoimmune disease, associated with various hematological neoplasms has been attracting growing attention since its initial description in 2020. While various therapeutic strategies have been explored in case studies, the optimal treatment strategy is still under investigation and allogeneic cell transplantation is considered the only curative treatment. Here, we describe 2 patients who achieved complete molecular remission of the underlying UBA1 mutant clone outside the context of allogeneic HCT. Both patients received treatment with the hypomethylating agent azacitidine, and deep molecular remission triggered treatment de-escalation and even cessation with sustained molecular remission in one of them. Prospective studies are necessary to clarify which VEXAS patients will benefit most from hypomethylating therapy and to understand the variability in the response to different treatment strategies.

Details

OriginalspracheEnglisch
Seiten (von - bis)993-997
Seitenumfang5
FachzeitschriftAnnals of hematology
Jahrgang103
Ausgabenummer3
PublikationsstatusVeröffentlicht - März 2024
Peer-Review-StatusJa

Externe IDs

PubMedCentral PMC10866742
Scopus 85182149422

Schlagworte

Schlagwörter

  • Humans, Antimetabolites, Antineoplastic, Prospective Studies, Myelodysplastic Syndromes/drug therapy, Azacitidine, Pathologic Complete Response, Skin Diseases, Genetic