Midostaurin monotherapy (advanced systemic mastocytosis, 1L alternative; D816V- or avapritinib-contraindicated)
Deterministic view of the source YAML entity. Clinical authority remains with the cited source IDs and reviewer sign-off state.
Knowledge-base record for audit. Provisional clinical content: this is not a prescription. An oncologist must verify original sources, applicability and review status. Full rules and constraints are in the YAML.
| Components | DRUG-MIDOSTAURIN |
|---|
| ID | REG-MIDOSTAURIN-ADVSM-1L |
|---|---|
| Type | Regimen |
| Status | reviewed 2026-04-27 | pending_clinical_signoff |
| Diseases | None declared |
| Sources | SRC-NCCN-SM-2025 |
Notes
D-RIVE / CPKC412D2201 (Gotlib NEJM 2016): midostaurin 100 mg BID in advanced SM ORR ~28% (per modified Valent / Cheson criteria; ORR ~60% by IWG-MRT-ECNM-modified). Pre-avapritinib standard-of-care. Now alternative when avapritinib is contraindicated (platelets <50 ×10⁹/L, prior intracranial hemorrhage, cognitive concerns) or inaccessible, OR for the rare KIT D816V-NEGATIVE advanced SM where avapritinib lacks activity. Significant nausea/vomiting toxicity — ~30% require dose reduction. NB: imatinib is CONTRAINDICATED in KIT D816V (steric clash); imatinib is reserved for the rare D816V- negative variant FIP1L1-PDGFRA myeloid neoplasm with eosinophilia (separate WHO entity).
Used By
Indication
IND-ADVSM-1L-MIDOSTAURIN- IND-ADVSM-1L-MIDOSTAURIN