Highlights of 2025 (Episode 1)

Is emicizumab finished? The FRONTIER2 trial of the novel bispecific antibody, denecimig (Mim8) for haemophilia A

On this episode, Pip and Rich discuss the FRONTIER2 [https://www.nejm.org/doi/10.1056/NEJMoa2517384], the phase 3 trial of Mim8 (denecimig) for prophylaxis in haemophilia A with or without inhibitors. They review the study design, efficacy and safety results, and consider where Mim8 may fit into the rapidly evolving haemophilia treatment landscape. They also explore the key unanswered question: does Mim8 offer any meaningful clinical advantage over emicizumab, or is its main benefit greater convenience through fixed-volume administration? ---------------------------------------- Hosted on Acast. See acast.com/privacy [https://acast.com/privacy] for more information.

Using AI to identify hospital acquired thrombosis

On this episode, Pip and Rich chat to Consultant Haematologist, Dr Kate Musgrave and Thrombosis Nurse Specialist, Vicky Marr about how they developed and implemented a natural language processing model that identifies cases of hospital acquired thrombosis. Implementing this strategy in their hospital in Newcastle-Upon-Tyne has led to saving thousands of hours of manually trawling radiology reports - enabling them to focus on what matters - interacting with patients. The tool is now available for implementation across the NHS. This is a must-listen for anyone wanting to improve efficiency in their hospital. Read more about the project here. [https://www.newcastle-hospitals.nhs.uk/news/newcastle-hospitals-using-artificial-intelligence-ai-to-identify-preventable-blood-clots/] ---------------------------------------- Hosted on Acast. See acast.com/privacy [https://acast.com/privacy] for more information.

Apixaban vs rivaroxaban: the trial we've all been waiting for

On this episode, Pip and Rich discuss the COBRRA trial: Castellucci et al. Bleeding Risk with Apixaban vs. Rivaroxaban in Acute Venous Thromboembolism. NEJM. 2026. [https://www.nejm.org/doi/full/10.1056/NEJMoa2510703] The trial showed a convincing reduction in clinically relevant bleeding in patients treated with apixaban (3.3% vs 7.1%) and no difference in efficacy. It's a compelling study but there were important exclusion criteria and as ever, there are some nuances beyond the abstract. This episode also includes an update on Pip's daughter's school science project where unwitting families were invited for dinner to find out whether there is, indeed, always time for pudding. ---------------------------------------- Hosted on Acast. See acast.com/privacy [https://acast.com/privacy] for more information.

ENERGIZE: Mitapivat in thalassaemia

Following its recent US FDA approval, on this episode, Pip and Rich discuss mitapivat, an allosteric activator of red-cell pyruvate kinase. They focus on ENERGIZE, a randomised controlled trial of mitapivat vs placebo (2:1) randomisation in people living with transfusion dependent thalassaemia. It met its primary endpoint of achieving significantly more patients reaching a haemoglobin rise of at least 1 g/L. However, Pip and Rich discuss the relevance of this surrogate endpoint as well as the small effects on fatigue and quality of life. Taher et al. Mitapivat in adults with non-transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial. Lancet. 2025 Jul 5;406(10498):33-42. doi: 10.1016/S0140-6736(25)00635-X. [https://www.thelancet.com/journals/lancet/article/PIIS0140-6736%2825%2900635-X/fulltext] ---------------------------------------- Hosted on Acast. See acast.com/privacy [https://acast.com/privacy] for more information.

Ianalumab for ITP: VAYHIT2

On this episode, Pip and Rich VAY-HIT2, a trial recently published in the New England Journal of Medicine [https://www.nejm.org/doi/abs/10.1056/NEJMoa2515168]. This study randomised patients with relapsed/refractory ITP after first line corticosteroids to eltrombopag + ianalumab or placebo.   As the thrombopoietin receptor agonists such as eltrombopag are often given for extensive periods, there's a lot of interest in giving additional treatment that can result in a relatively short course of treatment. Ianalumab is an anti-B-Cell Activating Factor (BAFF) Receptor monoclonal antibody.   VAY-HIT2 was a phase 3, randomised, double-blind trial, assigning, in a 1:1:1 ratio, adults with primary ITP and an insufficient response or a relapse after first-line glucocorticoid therapy to receive eltrombopag + ianalumab at a dose of 9 mg or 3 mg per kilogram of body weight or placebo once monthly for 4 months.   The trial did meet its primary endpoint but there's a lot to discuss, most importantly the quality of life end points - and the interpretation is not straight forward! Conflict of interest disclosure here: Pip has received travel expenses and payment for attending an advisory board for Novartis. Pip is also leading a Novartis-sponsored UK audit of the treatment of warm autoimmune haemolytic anaemia on behalf of HaemSTAR (www.haemstar.org/TRUTH [www.haemstar.org/TRUTH]). ---------------------------------------- Hosted on Acast. See acast.com/privacy [https://acast.com/privacy] for more information.