Challenges in the management of Haemophilia A
Haemophilia A is an inherited bleeding disorder where a person’s blood does not clot properly due to a lack or insufficient levels of a blood clotting protein known as factor VIII.
Learn more about Roche’s involvement in haemophilia.
Prof. Rebecca Kruse-Jarres welcomes delegates to The Hague and this symposium entitled "Understanding emicizumab▼: achieving safe and effective bleed control for people with haemophilia A"
Prof. Johnny Mahlangu discusses the safety profile of emicizumab▼ from the Phase 3 HAVEN and STASEY clinical trials, including data regarding thrombotic events and the management of breakthrough bleeds.
Dr Michael Callaghan outlines the emicizumab▼ efficacy data before outlining a case from his clinical practice.
Prof. Rebecca Kruse-Jarres discusses the association between adherence, breakthrough bleeding and subsequent joint damage with the aid of a case from her clinical practice.
Interactive discussion and question and answer session aiming to answer any outstanding questions following the emicizumab▼ safety and efficacy data presentations
This oral presentation reports the results of a safety evaluation of emicizumab in persons with acquired or congenital haemophilia A. Data for this evaluation was collated from post-market reports, expanded access programmes, compassionate use programmes and clinical trials and the analysis focuses on reports of thromboembolic and thrombotic microangiopathy events.
Improved treatments are needed for R/R NHL. Schuster and colleagues report updated clinical data from Group B (Cycle 1 step-up dosing) of the Phase I/Ib GO29781 study of mosunetuzumab in R/R indolent and aggressive NHL patients.
The aim of this study was to explore the effect of HEMLIBRA (emicizumab) prophylaxis on bone and joint health in adult/adolescent persons with hemophilia A without FVIII inhibitors
This analysis assessed whether fixed-duration venetoclax plus rituximab improves patient survival versus standard bendamustine-rituximab in the Phase III MURANO study (NCT02005471), when all patients had been off venetoclax treatment for median 22 months
Prophylaxis with intravenous FVIII replacement has been the standard of care for severe hemophilia A (HA). Emicizumab, a humanized bispecific FIXa- and FX-directed antibody, is approved for treatment of HA with or without inhibitors in the US. This study aimed to assess preferences for treatment characteristics for HA therapies in patients without current FVIII inhibitors and their caregivers using a discrete-choice experiment (DCE) survey.
Emicizumab has demonstrated efficacy in bleed prevention when administered subcutaneously 1.5 mg/kg once weekly, 3 mg/kg every two weeks (Q2W), and 6 mg/kg every four weeks (Q4W) in persons with haemophilia A (PwHA) with or without FVIII inhibitors. These dosing regimens were selected based on an exposure-repeated time-to-event model developed from bleed data collected in a phase I/II study in a small group of PwHA receiving weekly emicizumab doses of either 0.3, 1 or 3 mg/kg. This analysis presents the updated exposure–response relationship for bleeding events using a much larger database including data from PwHA from phase I/II and phase III studies.
The safety and efficacy of emicizumab has been demonstrated in persons with haemophilia A (PwHA) with or without FVIII inhibitors across all age groups in the HAVEN 1–4 studies. This presentation details the long-term efficacy and safety of emicizumab across the HAVEN 1–4 studies.
This is an ongoing multicenter phase I dose escalation trial investigating the safety, tolerability, pharmacokinetics (PK), biomarkers and antitumor activity of CD20-TCB in patients with heavily pre-treated NHL. Safety and efficacy data are presented
POLARIX (NCT03274492) will evaluate the efficacy, safety, pharmacokinetics, and patient-reported outcomes of 1.8mg/kg pola + chemoimmunotherapy (R-CHP) compared with SOC chemoimmunotherapy (R-CHOP) in previously untreated patients with intermediate- to high-risk CD20-positive DLBCL. This Trial in Progress presentation covers the study design, key inclusion/exclusion criteria, study endpoints and treatment schedule.
The phase 3 MabCute trial (NCT01461928) evaluated the efficacy and safety of prolonged R-SC maintenance after standard R-SC-based induction and maintenance in relapsed or refractory patients with indolent non-Hodgkin lymphoma. Primary analysis results are presented.
MIRROS is a randomized Phase III trial investigating the effects of idasanutlin (MDM2 antagonist) in combination with cytarabine versus placebo in combination with cytarabine in patients with relapsed or refractory AML. This Trial in Progress poster presents the MIRROS study design, including eligibility criteria, study endpoints, the futility interim analysis and enrollment status.
POLARIX (NCT03274492) will evaluate the efficacy, safety, pharmacokinetics, and patient-reported outcomes of 1.8mg/kg pola + chemoimmunotherapy (R-CHP) compared with SOC chemoimmunotherapy (R-CHOP) in previously untreated patients with intermediate- to high-risk CD20-positive DLBCL. This Trial in Progress poster presents the study design, key inclusion/exclusion criteria, study endpoints and treatment schedule.
The presentation reports the safety and efficacy results from the interim analysis of a phase I/II study of polatuzumab vedotin + obinutuzumab + lenalidomide in patients with relapsed/refractory follicular lymphoma. Interim safety and efficacy data are presented.