D47.0
Conditions
Interventions
Group 1: a) Duration of data collection:
The study is designed to follow each patient up to a maximum of 24 months.
After consent is provided, patient data will be collected from Day –30 to Day –1, D
EOS). Study visits will include data collection on variables of effectiveness, QoL, and healthcare resource utilization, and reporting of safety (SAEs).
b) Patient Population:
This non-interventional
Sponsors
Blueprint Medicines Cooperation
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: Target Population: Adult patients (>18 years of age) with ISM that is not controlled with best supportive care, who are initiating treatment with commercial avapritinib according to the German approved label. Inclusion Criteria Patients are eligible to be included in the study only if all the following criteria apply: 1. Patient is starting avapritinib treatment at the healthcare practitioner (HCP)’s discretion as part of their routine care (for ISM with moderate to severe symptoms inadequately controlled with symptomatic treatment) and in accordance with approved Summary of Product Characteristics. 2. Patient is able to understand and communicate with the HCP and comply with the requirements of the study. 3. Patient is willing and able to provide authorization to use and disclose personal health information. 4. Patient is willing and able to participate in the collection of patient-reported data.
Exclusion criteria
Exclusion criteria: Exclusion Criteria Patients are to be excluded from the study if any of the following criteria apply: 1. Patient with a potential increased risk for intracranial hemorrhage including those with a history of vascular aneurysm, intracranial hemorrhage, cerebrovascular accident within the prior year, or severe thrombocytopenia. 2. Patient who has previously taken avapritinib as a commercial drug or as part of a clinical study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in Mastocytosis Control-Quality of Life (MC-QoL) after 6 months vs baseline | — |
Secondary
| Measure | Time frame |
|---|---|
| Socio-demographics, prevalence of comorbidities, and medications used for ISM Baseline serum tryptase and KIT D816V variant allele fraction (VAF) Prior medical history of anaphylaxis and patient-reported anaphylaxis rates over 12 months before study enrollment Percentage of patients who have been prescribed a life support kit (eg, Notfallkit) Change in Mastocytosis Activity Score (MAS) after 3, 6, 12, 18, and 24 months vs baseline Change in MC-QoL after 3, 6, 12, 18, and 24 months vs baseline Change in Mastocytosis Control Test (MCT) after 3, 6, 12, 18, and 24 months vs baseline Time to response as assessed by MAS Change in serum tryptase after 3, 6, 12, 18, and 24 months vs baseline Patient-reported anaphylactic rates during the study (starting at Day 1) requiring hospitalization, emergency room (ER) visit, epinephrine autoinjector use, or other medication Collect polypharmacy (intended as the use of multiple medications simultaneously; typically, 3 or more symptom-directed prescription drugs), ISM-related hospital visits, and ER visits Collect and record changes in ISM symptom-directed therapies (including rationale for change in therapy and change in dose) Reason for starting avapritinib, the rationale for dose modifications and study/treatment discontinuations will be captured | — |
Countries
Germany
Contacts
Public ContactFrank Schippers
ClinCompetence GmbH
Outcome results
None listed