Shionogi’s Mulpleo recommended for NHS use across England, Wales and Scotland for chronic liver disease
Both the regulators governing NHS use of medicines in England, Wales and Scotland – the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) – have moved to recommend Shionogi’s Mulpleo (lusutrombopag) for the treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) undergoing planned invasive procedures.
The decision was based on evidence indicating that the therapy met all primary endpoints in two trials of 312 CLD patients with severe thrombocytopenia and a platelet count of <50,000/µL. In the first trial, 79.2% of participants receiving lusutrombopag required no platelet transfusion prior to the primary invasive procedure, compared with 12.5% with placebo; in a second trial, 64.8% of lusutrombopag patients achieved the same prior to the procedure or rescue therapy for bleeding within seven days of the procedure, compared to 29% receiving placebo.
Additionally, the trials revealed that platelet counts were sustained above 50,000/µL for a median of 20.9 days with lusutrombopag in patients not requiring platelet transfusion, compared to 9.5 days with placebo in patients who do require infusion.
The decision follows approval of the treatment in Europe back in February. It is estimated that around 600,000 in the UK are affected by a form of serious liver disease. Now, these patients will have an alternative treatment available via the NHS.
“Chronic liver disease is a huge burden in the UK; the number of deaths has increased by 400% since 1970 and it is now responsible for the highest number of premature deaths in 35-49 year olds,” commented Pamela Healy, Chief Executive of the British Liver Trust. “Many people with chronic liver disease require life-saving invasive procedures, so it is vital we have effective medicines to treat common complications such as severe thrombocytopenia (low platelet count), which can make operations too risky to undertake. Up until now, the only option for these patients has been a platelet transfusion, which often involves a hospital stay, so this new treatment which can be taken orally at home has the potential to really improve their quality of life”.
Dr Yiannis Kallis, Consultant Hepatologist and Gastroenterologist at the Royal London Hospital and the Royal Free Hospital London, also remarked: “Currently there are no available treatments for thrombocytopenia in patients with chronic liver disease besides supportive care and platelet transfusions. Thrombocytopenia is the most common haematological consequence of chronic liver disease and it can complicate or delay lifesaving invasive procedures. The NICE and SMC recommendations for lusutrombopag are fantastic news for both patients and clinicians as we urgently require new and effective treatments which will help enable these interventions to go ahead safely and on time.”
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