A Prospective Non-Interventional Study to Describe the Effectiveness of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Patients With Indolent Systemic Mastocytosis and Symptoms That Are Not Adequately Controlled With Symptomatic Treatments in Real-World Settings

简要总结

主要结局

次要结局

• Socio-demographic Variable: Participant Age(Baseline (Day -30 to Day -1))
• Socio-demographic Variable: Participant Gender(Baseline (Day -30 to Day -1))
• Socio-demographic Variable: Participant Race(Baseline (Day -30 to Day -1))
• Socio-demographic Variable: Participant Ethnicity(Baseline (Day -30 to Day -1))
• Baseline Serum Tryptase and KIT D816V Variant Allele Fraction (VAF)(Baseline (Day -30 to Day -1))
• Change From Baseline in Mastocytosis Activity Score (MAS) at Months 3, 6, 12, 18, and 24(Baseline, Months 3, 6, 12, 18, and 24)
• Number of Participants With Prior Medical History of Anaphylaxis(Baseline (Day -30 to Day -1))
• Number of Participants with Comorbidity(Baseline up to Month 24)
• Number of Participants Initiating a New ISM-directed Medication, Discontinuing an ISM-directed Medication, and With Dose Increases or Decreases for ISM-directed Medications(Baseline up to Month 24)
• Number of Participants Prescribed a Life Support Kit(Baseline up to Month 24)
• Change From Baseline in MC-QoL Questionnaire Score at Months 3, 6, 12, 18, and 24(Baseline, Months 3, 6, 12, 18, and 24)
• Change From Baseline in Mastocytosis Control Test (MCT) Score at Months 3, 6, 12, 18, and 24(Baseline, Months 3, 6, 12, 18, and 24)
• Time to Response as Assessed by MAS(Baseline up to Month 24)
• Change From Baseline in Serum Tryptase at Months 3, 6, 12, 18, and 24(Baseline, Months 3, 6, 12, 18, and 24)
• Number of Participants With Anaphylaxis Requiring Hospitalization, Emergency Room (ER) Visit, Epinephrine Autoinjector Use, or Other Medication(Baseline up to Month 24)
• Number of Participants Meeting the Definition of Polypharmacy (Use of Multiple Medications Simultaneously; Typically, 3 or More Symptom-directed Prescription Drugs)(Baseline up to Month 24)
• Number of Participants With ISM-related Hospital Visits and ER Visits(Baseline up to Month 24)
• Number of Participants With Changes in ISM Symptom-directed Therapies(Baseline up to Month 24)
• Number of Participants With Dose Modifications and Study/Treatment Discontinuations(Baseline up to Month 24)