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NCT03580655 · OUTCOME

Progression-free Survival (PFS)

(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis · Source last updated 2026-04-24

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
MEDIAN
Unit
Months
Interval / dispersion
95% Confidence Interval
Time frame
Baseline through Month 65

What was measured

PFS was defined as time from first dose to the time of initial documentation of confirmed PD or death due to any cause, whichever occurred first. Participants who had not progressed or died at the time of analysis were censored at the last response assessment that was SD or better.

Analysis population: Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by study steering committee review and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments, and had been on study for ≥6 cycles; had an end of study visit.

Groups in this outcome

Avapritinib

Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles.

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
Avapritinib83

Reported measurements

Source class 1
Values in Months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Avapritinib51.3Not reported38.7NAValues were non-estimable (insufficient number of participants with events).
Complete source fields
classes
  1. categories
    1. measurements
      1. comment
        Values were non-estimable (insufficient number of participants with events).
        group Id
        OG000
        lower Limit
        38.7
        upper Limit
        NA
        value
        51.3
denoms
  1. counts
    1. group Id
      OG000
      value
      83
    units
    Participants
description
PFS was defined as time from first dose to the time of initial documentation of confirmed PD or death due to any cause, whichever occurred first. Participants who had not progressed or died at the time of analysis were censored at the last response assessment that was SD or better.
dispersion Type
95% Confidence Interval
groups
  1. description
    Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles.
    id
    OG000
    title
    Avapritinib
param Type
MEDIAN
population Description
Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by study steering committee review and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments, and had been on study for ≥6 cycles; had an end of study visit.
reporting Status
POSTED
time Frame
Baseline through Month 65
title
Progression-free Survival (PFS)
type
SECONDARY
unit Of Measure
Months

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice