VEXAS syndrome: complete molecular remission after hypomethylating therapy.
Ann Hematol
; 103(3): 993-997, 2024 Mar.
Article
in En
| MEDLINE
| ID: mdl-38214707
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.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Skin Diseases, Genetic
/
Myelodysplastic Syndromes
/
Antimetabolites, Antineoplastic
Type of study:
Observational_studies
/
Risk_factors_studies
Limits:
Humans
Language:
En
Journal:
Ann Hematol
Journal subject:
HEMATOLOGIA
Year:
2024
Document type:
Article
Affiliation country: