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NCT03580655 · OUTCOME

Objective Response Rate

(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
NUMBER
Unit
Percentage of Participants
Interval / dispersion
95% Confidence Interval
Time frame
Baseline through Month 65

What was measured

The objective response rate was defined as the number of participants with a confirmed best response of morphologic complete remission, morphologic complete remission with partial recovery of peripheral blood counts, or morphologic partial remission by pure pathologic response (PPR) criteria. The PPR criteria are a modification of the mIWG-MRT-ECNM criteria that define deep responses where direct measure of the disease burden determined the response and focus on objective changes (bone marrow mast cell burden, serum tryptase, and complete blood count).

Analysis population: Pure Pathologic Response-evaluable Population: all participants who received ≥1 dose of avapritinib 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
Avapritinib107

Reported measurements

Source class 1
Values in Percentage of Participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Avapritinib73.8Not reported64.481.9Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        64.4
        upper Limit
        81.9
        value
        73.8
denoms
  1. counts
    1. group Id
      OG000
      value
      107
    units
    Participants
description
The objective response rate was defined as the number of participants with a confirmed best response of morphologic complete remission, morphologic complete remission with partial recovery of peripheral blood counts, or morphologic partial remission by pure pathologic response (PPR) criteria. The PPR criteria are a modification of the mIWG-MRT-ECNM criteria that define deep responses where direct measure of the disease burden determined the response and focus on objective changes (bone marrow mast cell burden, serum tryptase, and complete blood count).
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
NUMBER
population Description
Pure Pathologic Response-evaluable Population: all participants who received ≥1 dose of avapritinib 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
Objective Response Rate
type
SECONDARY
unit Of Measure
Percentage of Participants

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

Preserved source evidence · Not medical advice