Publications by authors named "F Gaches"
Introduction: Gaucher disease type 1 (GD1) is a rare genetic lysosomal storage disorder. Eliglustat is a first-line oral therapy for adult patients with GD1. The aim of the ELIPRO (ELIglustat Patient Reported Outcomes) study was to assess real-world outcomes of eliglustat treatment for over 1 year in patients with GD1, with a focus on patient-reported outcomes (PROs), including treatment adherence.
View Article and Find Full Text PDFArticle Synopsis
- A study compared two ways of giving a medicine called tocilizumab to patients with a disease called Takayasu arteritis (TAK).
- They looked at 109 patients from different countries and found that both methods worked similarly well after 6 months, with about 69% showing improvement.
- However, patients who got tocilizumab as a shot under the skin had a higher chance of getting worse again compared to those who received it through an IV.
Introduction: An association of systemic sclerosis (SSc) with cryoglobulin and/or cryofibrinogenemia has been described. However, clinical, biological, morphological and prognostic implications are unknown. The objective of this study was to describe the phenotype and evaluate the prognosis of cryoglobulinemia and/or cryofibrinogenemia in the progression of SSc.
View Article and Find Full Text PDFBackground: Systemic sclerosis (SSc) is associated with a variability of mortality rates in the literature.
Objective: To determine the mortality and its predictors in a long-term follow-up of a bi-centric cohort of SSc patients.
Methods: A retrospective observational study by systematically analyzing the medical records of patients diagnosed with SSc in Toulouse University Hospital and Ducuing Hospital.
Article Synopsis
- This study evaluates the safety and effectiveness of TNF-α antagonists and tocilizumab in treating Takayasu arteritis (TAK) in 209 patients, primarily young women.
- Results show that both treatment options achieved a high rate of complete response (66% for TNF-α and 70% for tocilizumab) after 6 months, although older age was positively correlated with response while certain baseline symptoms were negatively associated.
- The incidence of treatment relapses and adverse effects was similar for both therapies, indicating that they are equally effective for managing refractory TAK over a median follow-up of 36 months.