Quizartinib for AML

Quisartinib, designed specifically for FLT3-ITD-positive acute myeloid leukemia, has been characterized as a new option for resistant patients with no other suitable treatment options.

Quisartinib underwent a critical clinical trial of phase III QuANTUM-R. During these trials, this drug was evaluated in the monotherapy regimen of acute myeloid leukemia in 367 patients with FLT3-ITD mutation, resistant or with relapses after common first-line therapy. Quizartinib group showed a significant increase in overall survival compared with the group receiving standby chemotherapy (27% in the quisartinib group compared to 20% in chemotherapy options).

However, the FDA confirms the fact that the results are statistically significant. But they indicated a certain imbalance between the samples. For example, compared with the chemotherapy group, the quizartinib group had a higher percentage of patients who underwent hematopoietic stem cell transplantation.

The loss of Daiichi Sankyo may be beneficial to other pharmaceutical companies, as other pharmaceutical manufacturers are also involved in FLT3 inhibitors. For example, Novartis approved Rydapt (midostaurin), Astellas Pharma developed and tested gilteritinib (Xospata), Arog Pharmaceuticals is also completing the latest clinical studies of crenolanib.

Acute myeloid leukemia, which is also abbreviated as AML is a malignant disease of the hematopoietic system. The disease begins in the bone marrow. Our bone marrow produces different blood cells. When the bone marrow becomes ill, then this organ begins to “release” a huge amount of immature white blood cells instead of healthy ones.

Signs and symptoms

AML is characterized by many different signs and can be manifested differently in different patients. The main symptoms are symptoms of anemia:

  • fatigue;
  • pale skin;
  • shortness of breath;
  • loss of appetite.

Platelet deficiency is manifested by increased bleeding during cuts and bruises, nosebleeds, and “causeless” bruising. Often there are infections resistant to treatment since the patient has too few “normal” (mature, functional) white blood cells to fight them. Damage to the mucous membranes of the mouth and gastrointestinal tract, swelling of the gums may occur. Often, body temperature is increased, bone pains occur. Sometimes there are tumors from leukemic cells outside the bone marrow known as myelosarcoma (chloroma).

Most of the symptoms are associated with other diseases and not specific to AML. Before starting treatment, it is necessary to clarify the diagnosis based on laboratory tests, which are urgently made in healthcare institutions.