Drug updated on 3/28/2024
| Dosage Form | Capsule (oral: 25 mg) |
| Drug Class | Kinase inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- For the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation positive as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation.
- For the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).
Summary
- Midostaurin (Rydapt) is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation positive, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation.
- The drug also treats adult patients suffering from aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).
- Two systematic reviews/meta-analyses were reviewed which provided evidence on the effectiveness of midostaurin compared to other tyrosine kinase inhibitors in treating AML.
- One study found a significant reduction in relapse incidence after TKI therapy, showing an overall pooled risk ratio of 0.35, indicating a marked 65% reduced risk for relapse when using midostaurin as post-transplant maintenance therapy for FLT3-ITD positive AML patients.
- Another review assessing five different FLT3 inhibitors including midostaurin concluded that these drugs improved overall survival hazard ratio by 0.83 and event-free survival HR by 0.85; however, they increased relative risks of grade three and above vascular, dermatological, respiratory and hepatobiliary adverse events compared to control groups.
- Despite promising results supporting the use of midostaurin in treating AML patients carrying FLT3 mutations more data are needed to verify its optimum usage considering disease stage patient characteristics along with managing adverse effects while maintaining quality life standards among treated individuals according to one meta-analysis report reviewed here.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Rydapt (Midostaurin) Prescribing Information. | 2021 | Novartis Pharmaceuticals Corporation, East Hanover, NJ |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Midostaurin in Advanced Systemic Mastocytosis: A Systematic Review and Meta-analysis. | 2022 | American journal of therapeutics. |
| TKI maintenance after stem-cell transplantation for FLT3-ITD positive acute myeloid leukemia: a systematic review and meta-analysis. | 2021 | Frontiers in Immunology |
| Final clinical guidance report: midostaurin (Rydapt) for systemic mastocystosis. | 2020 | CADTH |
| FLT3 inhibitors in acute myeloid leukaemia: assessment of clinical effectiveness, adverse events and future research-a systematic review and meta-analysis. | 2020 | Systematic Reviews |
| Australian public assessment report: midostaurin. | 2019 | Australian Government: Department of Health |