2021 ASH annual meeting

Abstract

Two phase 3 trials investigated fitusiran, a subcutaneous investigational siRNA targeting antithrombin, as once-monthly 80 mg prophylaxis in patients with haemophilia A or B. The ATLAS-INH trial (NCT03417102) was presented by Guy Young (Children's Hospital Los Angeles and University of Southern California, Los Angeles, CA, USA). Eligible patients were male, aged 12 or more years, had developed inhibitors, and were receiving on-demand bypassing agents (BPA). 57 patients were randomly assigned 2:1 to fitusiran prophylaxis (n=38) or continuation of BPA (n=19). The annualised bleeding rate (ABR; primary endpoint) was 1·67 (95% CI 1·0–2·7) in the fitusiran group and 18·07 (10·6–30·8) in the BPA group (HR 0·09, 95% CI 0·04–0·19; p<0·0001). 25 (66%) patients in fitusiran group had zero treated bleeding events. Alok Srivastava (Christian Medical College, Vellore, India) presented the ATLAS A/B trial (NCT03417245) in patients with haemophilia A or B without inhibitors. Eligible patients were male, aged 12 years or older, and received previous treatment on-demand. Patients were randomly assigned 2:1 to fitusiran prophylaxis (n=80) or on-demand factor concentrates (n=40). The ABR was 3·1 (95%CI 2·3–4·3) in the fitusiran group and 31·0 (21·1–45·5) in the on-demand group (90% reduction, 95% CI 84–94; p<0·0001); 40 patients (51%) in the fitusiran group had no bleeds. In both trials, fitusiran also improved spontaneous and joint bleeds, and overall quality of life (secondary endpoints). One (2%) patient discontinued fitusiran in ATLAS-INH and 2 (3%) patients in the ATLAS A/B. A reduced dose of fitusiran is being evaluated in ongoing clinical studies. 63rd ASH Annual Meeting and ExpositionAddition of the anti-CD38 monoclonal antibody isatuximab to lenalidomide–bortezomib–dexamethasone (RVd) could be a new standard of care for patients with newly diagnosed multiple myeloma, according to results from the GMMG HD7 trial, presented by Hartmut Goldschmidt (University Hospital Heidelberg and National Center of Tumor Diseases, Heidelberg, Germany). In this phase 3 trial, patients with newly diagnosed, transplantation-eligible multiple myeloma were randomly assigned to RVd (n=329) or RVd plus intravenous isatuximab (n=331) and assessed for minimal residual disease negativity after induction. Full-Text PDF