Info
midostaurin
- FLT3 突變會導致 AML 病人的 → 預後 ✖ 不良
- midostaurin 成 為：全球第一個一線治療 FLT3 突變陽性 AML 的 → 標靶藥物。
- 台灣 FDA 也於 2018 年 4 月核准 midostaurin，核准的 → 適應症 為：
- 新確診 為：FLT3 突變陽性的 → AML 成年病人之標準前導 (daunorubicin 併用 cytarabine) 與鞏固性化療 （高劑量 cytarabine） 時合併使用。
Midostaurin Plus Chemotherapy in FLT3-Mutated Acute Myeloid Leukemia
Information
- Design: Phase 3, randomized, double-blind, placebo-controlled, multi-center
- Number of patients: 717
- Patients characteristics: Patients with newly diagnosed acute myeloid leukemia (AML) and a FLT3 mutation, 18 to 59 years of age
- Agent: Midostaurin plus standard chemotherapy vs placebo plus standard chemotherapy
- Treatment line: Frontline therapy
- Trial Name or NCT Number: NCT00651261
Comparison of two groups
| Endpoint | Midostaurin + Chemotherapy | Placebo + Chemotherapy |
|---|---|---|
| Overall Survival | Hazard ratio for death, 0.78 (one-sided P=0.009) | - |
| Event-Free Survival | Hazard ratio for event or death, 0.78 (one-sided P=0.002) | - |
Other findings
- The benefit of midostaurin was consistent across all FLT3 subtypes (ITD [high], ITD [low], and TKD)
- The rate of severe adverse events was similar in the two groups
- Allogeneic transplantation was allowed in both groups
Summary In this phase 3 trial, the addition of midostaurin, a multitargeted kinase inhibitor, to standard chemotherapy significantly prolonged overall and event-free survival in patients with acute myeloid leukemia (AML) and a FLT3 mutation. The benefit of midostaurin was consistent across all FLT3 subtypes, and the rate of severe adverse events was similar in both groups.
- 兩組常見的 → 副作用皆 為：
- 貧血、
- 嗜中性白血球低下、
- 血小板低下、
- 淋巴球減少 ↓ 、
- 嗜中性白血球低下合併發燒、
- 感染、
- 腹瀉、
- 低血鉀等，midostaurin 加標準化療組有較多貧血及皮疹的 → 副作用 (P<0.05) 。