Rationale: Patients with indolent or smoldering systemic mastocytosis can have severe disabling symptoms. Almost all patients have fatigue, a compromised quality of life, hampering normal functioning. Because this form of mastocytosis is not considered life-threatening, mast cell eradication has never been applied and patients receive only symptomatic therapy with histamine blockers. Midostaurin, a c-KIT inhibitor has shown activity regarding symptom control and decrease of malignant mast cells in patients with aggressive systemic mastocytosis (ASM) or mast cell leukemia
Name: Midostaurin,
Description: Midostaurin, twice daily 100 mg orally, continuously for 6 monthsType: DrugMidostaurin
Description: Percent change in the total score ("Sumscore") of all symptoms assessed by the Mastocytosis Symptom Assessment Form (MSAF) after 12 weeks.
Measure: Symptom Scoring Time: 12 weeksDescription: persistence of improvement symptom score at 6 months.
Measure: Persistence of improvements Time: 6 monthsDescription: Percent change in the mast cell burden (bone marrow infiltrate, skin infiltrate, serum tryptase levels) after 6 months.
Measure: Mast cell burden Time: 6 monthsDescription: Number and grading of Common Terminology Criteria adverse events during the 6 months of therapy.
Measure: Adverse events Time: 6 monthsSingle Group Assignment
There is one SNP
Number and grading of Common Terminology Criteria adverse events during the 6 months of therapy.. Inclusion Criteria: - Patients with Indolent Systemic Mastocytosis (ISM) or Smouldering Systemic Mastocytosis (SSM) according to the WHO criteria - Presence of the D816V c-KIT mutation - Serum tryptase > 20 mg/l - Serious mediator-related symptoms that cannot be controlled by H1 and H2 blocking drugs. --- D816V ---
- Any known other present malignancy, non-melanoma skin cancers excluded - History of malignancy within the last 5 years, non-melanoma skin cancers excluded - Any serious comorbidity interfering with therapy compliance and follow-up compliance - Pregnancy - Patients not willing or who are not able to comply with contraceptive measures Inclusion Criteria: - Patients with Indolent Systemic Mastocytosis (ISM) or Smouldering Systemic Mastocytosis (SSM) according to the WHO criteria - Presence of the D816V c-KIT mutation - Serum tryptase > 20 mg/l - Serious mediator-related symptoms that cannot be controlled by H1 and H2 blocking drugs. --- D816V ---