Publications by authors named "Jean-Thomas Giraud"
Objectives: Vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic (VEXAS) syndrome is an adult-onset autoinflammatory disease associated with somatic ubiquitin-like modifier-activating enzyme 1 (UBA1) mutations. We aimed to evaluate the efficacy and safety of targeted therapies.
Methods: Multicentre retrospective study including patients with genetically proven VEXAS syndrome who had received at least one targeted therapy.
Article Synopsis
- Thrombocytopenia in MDS and CMML:
- Despite being moderate in prevalence for low-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukaemia (CMML), thrombocytopenia poses significant risks for severe bleeding, and current treatment options remain limited.
- Efficacy of Eltrombopag (ELT):
- In a retrospective study involving 61 patients (50 with MDS and 11 with CMML), ELT demonstrated a platelet response in 77% of patients, with a median duration of response lasting about 8 months, and none of the patients who stopped treatment relapsed within a follow-up period.
- Safety and Comp