OBServatory of Compassionate Use of IVOsidenib in France for Patients With Acute Myeloid Leukemia

This study focuses on a type of blood cancer called acute myeloid leukaemia (AML). In about 10-15% of people with AML, there's a specific change in their genes, called an IDH1 mutation. This mutation causes the cancer cells to behave differently and grow in unusual ways. A medication called ivosidenib (IVO) has been developed to target these specific mutated cells.

IVO has shown promising results in other studies, helping some patients with this particular type of AML. In places like the United States, it's already approved for certain AML patients with the IDH1 mutation. However, in Europe, IVO hasn't yet received full approval from the European Medicines Agency (EMA) due to needing more evidence.

Because of its potential benefits, IVO has been available in France through a special programme called 'compassionate use'. This allows patients to access promising medicines before they are widely approved. This study is looking back at the real-world experiences of French patients who received IVO through this programme. By carefully reviewing their medical records, researchers hope to learn more about how well IVO works and if it's safe for patients with AML and the IDH1 mutation, both when they are first diagnosed and when their cancer has returned or didn't respond to other treatments. This information could be very important for doctors and might help with future efforts to get IVO approved across Europe.