Drug updated on 12/11/2024
| Dosage Form | Capsules (Oral: 25 mg) |
| Drug Class | Kinase inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated 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
- Indicated for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).
Latest News
Summary
- This summary is based on the review of two systematic review(s)/meta-analysis(es). [1-2]
- Maintenance therapy after allogeneic haematopoietic stem cell transplantation (HSCT) in acute myeloid leukemia (AML) patients showed an improvement in overall survival (OS) with a hazard ratio (HR) of 0.61 (95% confidence interval (CI) 0.47-0.80) and relapse-free survival (RFS) with an HR of 0.51 (95% CI 0.40-0.66).
- FMS-like tyrosine kinase 3 (FLT3) inhibitors, including midostaurin, in AML patients demonstrated improved OS with an HR of 0.83 (95% CI 0.75-0.92, p = 0.0005), event-free survival (EFS) with an HR of 0.85 (95% CI 0.77-0.94, p = 0.002), and relapse-free survival (RFS) with an HR of 0.76 (95% CI 0.64-0.90, p = 0.001).
- In AML patients receiving maintenance therapy after allogeneic HSCT, there was no difference in the occurrence of grade 3/4 adverse events, infections, or graft versus host disease compared to control arms.
- FLT3 inhibitors in AML patients were associated with a higher risk of vascular, dermatological, respiratory, and hepatobiliary adverse events, though no significant difference in 60-day mortality was observed (relative risk (RR) = 1.04; 95% CI 0.77-1.40, p = 0.79).
- There is no population type or subgroups information available in the reviewed studies.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Rydapt (midostaurin) Prescribing Information. | 2023 | Novartis Pharmaceuticals Corporation, East Hanover, NJ |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Maintenance therapy after allogeneic hematopoietic transplant for acute myeloid leukemia: a systematic review and meta-analysis | 2021 | Acta Oncologica (Stockholm, Sweden) |
| FLT3 inhibitors in acute myeloid leukaemia: assessment of clinical effectiveness, adverse events and future research-a systematic review and meta-analysis | 2020 | Systematic Reviews |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Infectious complications of targeted drugs and biotherapies in acute leukemia. clinical practice guidelines by the european conference on infections in leukemia (ECIL), a joint venture of the european group for blood and marrow transplantation (EBMT), the european organization for research and treatment of cancer (EORTC), the international immunocompromised host society (ICHS) and the european leukemia net (ELN). | 2022 | Leukemia |
| Treatment of advanced systemic mastocytosis with midostaurin: practical guidance for optimal therapy and management. | 2022 | Mediterranean Journal of Hematology and Infectious Diseases |
| Acute myeloid leukemia. | 2019 | Alberta Health Services |
| Acute myeloid leukemia, version 3.2019, NCCN clinical practice guidelines in oncology. | 2019 | Journal of the National Comprehensive Cancer Network |