Publications by authors named "Marianne Uguen"
Article Synopsis
- Crovalimab is a new C5 inhibitor that can be self-administered every four weeks and is being tested in a phase 3 trial against another treatment, eculizumab, for patients with paroxysmal nocturnal hemoglobinuria (PNH).
- The trial's focus shifted from efficacy to safety due to not meeting recruitment goals, and exploratory endpoints included various measures of patient health and satisfaction.
- Results showed that while both treatments had adverse events, crovalimab showed sustained effectiveness and 85% of patients preferred it over eculizumab, suggesting it might be a more manageable treatment option for long-term PNH care.
Pentraxin 2 (PTX-2; serum amyloid P component), a circulating endogenous regulator of the inflammatory response to tissue injury and fibrosis, is reduced in patients with myelofibrosis (MF). Zinpentraxin alfa (RO7490677, PRM-151) is a recombinant form of PTX-2 that has shown preclinical antifibrotic activity and no dose-limiting toxicities in phase I trials. We report results from stage 1 of a phase II trial of zinpentraxin alfa in patients with intermediate-1/2 or high-risk MF.
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- Recent advancements in pediatric oncology drug development have led to innovative therapies and clinical trial approaches, though new drug approvals remain slow.
- Regulatory changes in the US and Europe have increased the urgency for faster drug development tailored to pediatric patients with cancer.
- The iMATRIX platform showcases a new trial framework that studies multiple drugs for various pediatric tumor types simultaneously, with six studies currently conducted or planned to test different treatment options.
Background: Bleeding in people with hemophilia A can be life threatening, and intra-articular bleeds can result in joint damage. Most clinical studies focus on treated bleeds, while bleeds not treated with coagulation factor(s) (untreated bleeds) are underreported.
Objectives: We assessed the incidence of untreated bleeds during a noninterventional study (NIS) wherein people with hemophilia A, with or without factor VIII (FVIII) inhibitors, were managed according to standard practice.
Introduction: Persons with haemophilia A (PwHA) with factor (F)VIII inhibitors, including children, have impaired health-related quality of life (HRQoL). The HAVEN 2 study (NCT027955767) of paediatric PwHA with FVIII inhibitors demonstrated that subcutaneous emicizumab prophylaxis resulted in low annualizedbleed rates.
Aim: We assessed the impact of emicizumab prophylaxis on the HRQoL of children and their caregivers participating in HAVEN 2.
Emicizumab, a bispecific humanized monoclonal antibody, bridges activated factor IX (FIX) and FX to restore the function of missing activated FVIII in hemophilia A. Emicizumab prophylaxis in children with hemophilia A and FVIII inhibitors was investigated in a phase 3 trial (HAVEN 2). Participants, previously receiving episodic/prophylactic bypassing agents (BPAs), were treated with subcutaneous emicizumab: 1.
View Article and Find Full Text PDFBackground: HFE hemochromatosis is an inborn error of iron metabolism linked to a defect in the regulation of hepcidin synthesis. This autosomal recessive disease typically manifests later in women than men. Although it is commonly stated that pregnancy is, with menses, one of the factors that offsets iron accumulation in women, no epidemiological study has yet supported this hypothesis.
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