Class: Inhibits the FLT3 internal tandem duplication (ITD) and FLT3 tyrosine kinase domain (TKD).
Disease: Relapsed or refractory acute myeloid leukemia (AML) with an FLT3 mutation.
Dose: 120 mg orally daily.
trial (NCT02421939): 21% of the patients who received gilteritinib exhibited complete remission or complete remission with partial hematologic recovery.
Class: Hedgehog pathway inhibitor.
Disease: Adults over age 75 years with newly diagnosed AML and other medical comorbidities that preclude them from intensive chemotherapy.
Dose: The recommended dose is 100 mg orally continuously in 28-day cycles.
AE: QT prolongation and embryo-fetal toxicity
Phase 2trial (NCT01546038): 3.9-month overall survival advantage for glasdegib plus cytarabine, compared with cytarabine alone. Overall, 15% of the glasdegib plus low dose cytarabine arm achieved complete remission, compared with the 1% complete remission rate in patients who received cytarabine alone.
Iobenguane I 131 (Azedra)
Class: Radiopharmaceutical agent; induces cell death within the noradrenaline transporter.
Disease: Iobenguane scan–positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma
Dose: Initial intravenous dosimetric dose, followed by two therapeutic doses.
AE: Pancytopenia and elevated international normalized ratio (INR).
trial (NCT00874614): One-quarter of patients receiving this therapy had at least a 50% reduction in the dose and number of antihypertensives for at least 6 months; almost all patients had a tumor response.
Class: Small-molecule inhibitor of mutant isocitrate dehydrogenase (IDH1).
Disease: Refractory AML and an IDH1 mutation
Dose: 500 mg orally daily.
trial (NCT02074839): Overall response rate of 41.6% in patients who received ivosidenib, with a 30.4% rate of complete remission or complete remission with partial hematologic recovery.
Class: Oral tyrosine kinase inhibitor.
Disease: Advanced solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion.
Dose: 100 mg orally twice daily.
, , and trials (NCT02122913, NCT02637687, and NCT02576431): Patients who received larotrectinib had durable responses regardless of patient age, tumor type, and fusion status.
Lutetium Lu 177 dotatate (Lutathera)
Class: Radiolabeled somatostatin analogue.
Disease: Somatostatin receptor–positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs).
Dose: Intravenous infusion 7.4 GBq (200 mCi) every 8 weeks for a total of four doses.
trial (NCT01578239): 65% of adults who received lutetium Lu 177 showed improved progression-free survival at 20 months, compared with just 10.8% in the control group.