Table 2.
Newly approved drugs for the treatment of AML and their possible fields of application according to the SIE, SIES, GITMO criteria.
| Drug Name | Mechanism of Action | Indications | Applicability |
|---|---|---|---|
| Gilteritinib | FLT3 inhibition | Treatment of adult patients who have R/R AML with an FLT3 mutation (FDA) | Single agent in R/R fit/unfit patients |
| Gemtuzumab Ozogamicin | Anti-CD33 Targeted antibody | Combination therapy with daunorubicin and cytarabine for the treatment of patients aged 15 years and above with previously untreated, de novo CD33-positive acute myeloid leukemia (EMA) | Combination therapy in ND fit adult patients |
| Combination therapy with daunorubicin and cytarabine or single agent for the treatment of ND CD33-positive acute myeloid leukemia (AML) in adults and single agent for treatment of relapsed or refractory CD33-positive AML in adults and in pediatric patients 2 years and older (FDA) | Single agent in ND and R/R fit patients | ||
| Combination therapy in ND fit patients | |||
| CPX-351 | Cytotoxic drug (daunorubicin plus cytarabine liposomal formulation) | Newly diagnosed, therapy-related acute myeloid leukemia or AML with myelodysplasia-related changes (FDA, EMA) | Single agent in ND fit patients |
| Venetoclax | Bcl-2 inhibition | In combination with HMAs or low-dose cytarabine for the treatment of ND AML in adults who are age 75 years or older, or who have comorbidities that preclude the use of IC (FDA) | Combination therapy in ND unfit to IC patients |
| Ivosidenib | IDH1 inhibition | ND AML with a susceptible IDH1 mutation, who are at least 75 years old and are considered unsuitable to IC (FDA) | Single agent in ND unfit to IC patients |
| Enasidenib | IDH2 inhibition | Adult patients with R/R AML with a susceptible IDH2 mutation (FDA) | Single agent in fit/unfit to IC R/R patients |
| Midostaurin | FLT3 inhibition | In combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Midostaurin single-agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukemia who are FLT3 mutation-positive (FDA, EMA) | Combination therapy in ND fit patients |
| Glasdegib | Smoothened Inhibition (HH/GLI positive regulator) | In combination with low-dose cytarabine, for newly-diagnosed AML in patients who are 75 years old or older or who have comorbidities that preclude intensive induction chemotherapy (FDA) | Combination therapy in ND unfit to IC patients |
AML: Acute Myeloid Leukemia; SIE: Italian Society of Hematology; SIES: Italian Society of Experimental Hematology, GITMO: Italian Group for Bone and Marrow Transplantation.