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NICE Recommends First Treatment for Mastocytosis

The National Institute for Health and Care Excellence (NICE) has published new draft guidance recommending midostaurin (Rydapt, Novartis) as a treatment for advanced systemic mastocytosis in adults.

There are three diverse subtypes of the rare haematological condition: aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasms and mast cell leukaemia. Symptoms are varied and can range from frequent and unexpected diarrhoea and vomiting to anaphylaxis.

Because of the individual nature of the disease and the diversity of the subtypes, there is no current standard treatment for advanced systemic mastocytosis. Instead, treatment is based on symptoms and can include a variety of therapies.

The new draft guidance means that around 170 patients will now be eligible for treatment with midostaurin, the first targeted therapy for advanced systemic mastocytosis to be available on the NHS. The treatment is taken orally twice a day and works by blocking multiple enzymes involved in the condition.

NICE said that clinical trial evidence suggests that midostaurin improves the overall survival of people with advanced systemic mastocytosis compared with several comparator treatments. There is also a substantial improvement in the quality of life for patients using midostaurin; however, evidence around its effectiveness is uncertain.

Therefore, midostaurin is considered a cost-effective use of NHS resources and is recommended for routine use on the NHS.

Meindert Boysen, Deputy Chief Executive and Director of the Centre for Health Technology Evaluation at NICE, said: “We are pleased to be able to recommend midostaurin as a treatment option for people with advanced systemic mastocytosis, despite the limitations in the clinical and comparative effectiveness evidence."

“The symptoms experienced by patients with this rare disease can be devastating and limiting. By recognising that patients with advanced systemic mastocytosis have a limited life expectancy without midostaurin, and by working closely with the company, we can support access to this innovative treatment.”

This article originally appeared on Univadis, part of the Medscape Professional Network.