2023-11-09 08:30:01
Each year, around 87,000 new cases of myelodysplastic syndromes are recorded around the world, a rare form of blood cancer. On October 24, 2023, the Food and Drug Administration (FDA) approved a new treatment for myelodysplastic syndrome patients with a particular genetic mutation. New hope for treatment for patients.
Myelodysplastic syndromes, rare blood cancers
THE syndromes myélodysplasiques are a rare form of blood cancer. They occur when stem cells present in the bone marrow are affected by genetic mutations. They then no longer produce a sufficient quantity of functional and healthy blood cells:
- Red blood cells;
- White blood cells;
- Blood platelets.
Therapies once morest myelodysplastic syndromes are few in number and vary depending on the type of mutation involved. Of the 87,000 new cases of myelodysplastic syndromes diagnosed worldwide each year, approximately 3.6% have an IDH1 mutation, which affects the enzyme isocitrate dehydrogenase-1. Around 1 in 3 patients will see their myelodysplastic syndrome progress to acute myeloid leukemia.
Finally a targeted treatment for certain myelodysplastic syndromes
For patients suffering from myelodysplastic syndrome with an IDH-1 mutation, there was until now no targeted treatment. But on October 24, 2023, the FDA authorized a new drug, ivosidenib, for the US market. targeted treatment of relapsed or refractory myelodysplastic syndrome with an IDH-1 mutation. Ivosidenib is an isocitrate dehydrogenase inhibitor drug. It was previously approved in the USA for the treatment of newly diagnosed, relapsed or refractory acute myeloid leukemia and locally advanced or metastatic cholangiocarcinoma. This time, treatment with this drug is possible from the stage of myelodysplastic syndrome, before a possible progression to acute leukemia. A major breakthrough for patients.
To know ! Cholangiocarcinoma is a malignant tumor of the bile ducts in the liver or gallbladder.
For patients to be eligible for this new treatment, the IDH-1 mutation must have been confirmed by a diagnostic test also approved by the FDA. This therapeutic advance for a rare form of blood cancer follows the publication of the results of a clinical study carried out on 18 adult patients suffering from relapsed or refractory myelodysplastic syndrome with an IDH-1 mutation. The drug was administered orally at a dose of 500 mg per day continuously for 28-day cycles until cancer progression, the occurrence of toxicity requiring treatment cessation or bone marrow transplantation.
Treat patients before progression to acute leukemia
Ultimately, 7 of the 18 patients in the study showed partial or complete remission of myelodysplastic syndrome thanks to treatment, with an average duration of remission between 1.9 and 80.8 months. Among patients requiring regular blood transfusions, 67% no longer needed them thanks to treatment. The side effects observed were the same as those encountered in acute myeloid leukemia, namely diarrhea, constipation, nausea, muscle and joint pain, fatigue, cough, rash and rhythm disturbances. cardiac.
In France, the drug ivosidenib has benefited since April 2023 early access authorization in dual therapy with azacitidine, in the treatment of newly diagnosed acute myeloid leukemia with an IDH-1 mutation, not eligible for intensive chemotherapy and other available therapies. It is also authorized as monotherapy in the treatment of advanced or metastatic cholangiocarcinoma with an IDH-1 mutation, following a first line of treatment. For the moment, it has no indication at the myelodysplastic syndrome stage.
Estelle B., Doctor of Pharmacy
Sources
– HIGHLIGHTS OF PRESCRIBING INFORMATION. FDA. www.accessdata.fda.gov. Accessed October 30, 2023.
– TIBSOVO – IVOSIDENIB SERVIER (ivosidenib) Acute myeloid leukemia.www.has-sante.fr. Accessed October 30, 2023.
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