A breakthrough we still can't benefit from

Prof. Sebastian Giebel said among others:

• Acute myeloid leukemia (AML) is a very aggressive cancer.
• Without proper treatment, AML can lead to death even within a few or several weeks
• Treatment should be very intensive, aimed at achieving remission.
• Very intensive treatment (chemotherapy and bone marrow transplantation) is possible in younger people without additional health burden.
• Unfortunately, about half of AML patients are elderly, burdened people. So far, we have had few therapeutic tools with them. The most used and considered standard drug is azacitidine, but only a small percentage of patients is able to induce a response and maintain it in this drug.
• Recent years have shown that the prognosis of AML patients can be improved by the use of new therapies, including the targeted drug, venetoclax, which selectively inhibits the BCL2 gene product.
• A large clinical trial that compared the efficacy of azacitidine in combination with venetoclax to azacitidine alone showed that the group treated with both drugs achieved complete remission and in over 60% of patients, and the probability of survival in 2-year follow-up increased from 20% in in the azacitidine arm up to 40% in the combination arm.
• The combination of azacitidine and venetoclax has become the new world standard in the treatment of AML in this difficult group of patients [the elderly].
• Azacitidine + venetoclax therapy should be used in the first line of treatment, because then the chances of its effectiveness are the greatest.
• So far, such therapy is not covered by reimbursement in Poland. You can only apply for funding as part of the emergency access procedure to drug therapies.
• As the Polish Society of Hematologists and Transfusionists and the Polish Group for the Treatment of Leukemia in Adults, we expect that the reimbursement of this therapy will soon become a reality.