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← Systemic Mastocytosis with an Associated Myeloid Neoplasm

NCT03580655 · STUDY IN CONTEXT

(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocy

Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

ORR was defined as the percentage of participants with a confirmed best response of complete remission (CR), complete remission with partial recovery of peripheral blood counts (CRh), partial remission (PR), or clinical improvement (CI) by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other clinical and laboratory parameters.

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.

→

secondary · POSTED

The AdvSM-SAF is a 10-item questionnaire that assesses eight symptoms specific to AdvSM. All eight symptoms are scored on a scale of 0 (absence of symptoms) to 10 (more severe symptoms) (up to 80 points maximum). Each symptom contributes to the TSS equally. The TSS was generated based on average scores for each 7-day period. An increase in score from 0 (no symptoms) to 80 (worst symptoms represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an electronic diary (eDiary). Each cycle is 28 days long.

Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number of Participants Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoints.

→

secondary · POSTED

ORR was defined as the percentage of participants with a confirmed best response of CR, CRh, PR, or CI by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other clinical and laboratory parameters.

Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by local investigator 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. Here, 'Overall Number of Participants Analyzed' signifies those participants evaluable for this outcome measure.

→

secondary · POSTED

TTR was defined as the time from first dose to the time of initial evaluation of clinical improvement (CI) or better. Here, 'Overall Number of Participants Analyzed' signifies those participants with a confirmed best response of CR, CRh, PR, and CI by mIWG-MRT-ECNM criteria.

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.

→

secondary · POSTED

DOR was defined as the time from initial documentation of a CI or better to the time of initial documentation of confirmed progressive disease (PD) or death due to any cause, whichever occurred first. For responders who had not progressed or died at the time of analysis, DOR was censored at the last response assessment that was stable disease (SD) or better. Here, 'Overall Number of Participants Analyzed' signifies those participants with a confirmed best response of CR, CRh, PR, and CI by mIWG-MRT-ECNM criteria.

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.

→

secondary · POSTED

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.

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.

→

Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

Patient-reported symptom or function measure · Mean Change From Baseline in Advanced Systemic Mastocytosis-Symptom Assessment Form (AdvSM-SAF) Total Symptom Score (TTS)

Score direction: Lower is better, according to the source. Direction is never inferred from an instrument name.

release id
clinical-indications-draft-1
disease state id
ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
nct id
NCT03580655
qol key
qol-0001
source section id
NCT03580655-om-0001
source outcome index
1
instrument
Patient-reported symptom or function measure
instrument basis
matched 'symptom score' in the study's own wording
scale title
Mean Change From Baseline in Advanced Systemic Mastocytosis-Symptom Assessment Form (AdvSM-SAF) Total Symptom Score (TTS)
measure definition
The AdvSM-SAF is a 10-item questionnaire that assesses eight symptoms specific to AdvSM. All eight symptoms are scored on a scale of 0 (absence of symptoms) to 10 (more severe symptoms) (up to 80 points maximum). Each symptom contributes to the TSS equally. The TSS was generated based on average scores for each 7-day period. An increase in score from 0 (no symptoms) to 80 (worst symptoms represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an electronic diary (eDiary). Each cycle is 28 days long.
time frame
Baseline up to Month 6 (Cycle 6, Day 1)
analysis role
secondary
reporting status
POSTED
higher is better
0
direction quote
increase in score from 0 (no symptoms) to 80 (worst symptoms represents a worse
population note
Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number of Participants Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoints.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
Open the exact result →
Patient-reported symptom or function measure · Mean Change From Baseline in AdvSM-SAF Individual Symptom Scores (Severity) at Month 10

Score direction: Lower is better, according to the source. Direction is never inferred from an instrument name.

release id
clinical-indications-draft-1
disease state id
ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
nct id
NCT03580655
qol key
qol-0015
source section id
NCT03580655-om-0015
source outcome index
15
instrument
Patient-reported symptom or function measure
instrument basis
matched 'symptom score' in the study's own wording
scale title
Mean Change From Baseline in AdvSM-SAF Individual Symptom Scores (Severity) at Month 10
measure definition
The AdvSM-SAF is a 10-item questionnaire that assesses symptoms and functional domains specific to AdvSM. Individual symptom scores are generated based on average scores for each 7-day period. Eight symptoms are scored on a scale of 0 to 10 for severity. An increase in score represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an eDiary.
time frame
Baseline, Month 10
analysis role
secondary
reporting status
POSTED
higher is better
0
direction quote
increase in score represents a worse
population note
Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
Open the exact result →
Patient-reported symptom or function measure · Mean Change From Baseline in AdvSM-SAF Individual Symptom Scores (Episodes) at Month 10

Score direction: Lower is better, according to the source. Direction is never inferred from an instrument name.

release id
clinical-indications-draft-1
disease state id
ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
nct id
NCT03580655
qol key
qol-0016
source section id
NCT03580655-om-0016
source outcome index
16
instrument
Patient-reported symptom or function measure
instrument basis
matched 'symptom score' in the study's own wording
scale title
Mean Change From Baseline in AdvSM-SAF Individual Symptom Scores (Episodes) at Month 10
measure definition
The AdvSM-SAF is a 10-item questionnaire that assesses symptoms and functional domains specific to AdvSM. Individual symptom scores are generated based on average scores for each 7-day period. Two symptoms (vomiting and diarrhea) are scored for number of episodes. An increase in score represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an eDiary.
time frame
Baseline, Month 10
analysis role
secondary
reporting status
POSTED
higher is better
0
direction quote
increase in score represents a worse
population note
Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
Open the exact result →
EORTC QLQ · Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30)

Score direction: Not stated by the study. Direction is never inferred from an instrument name.

release id
clinical-indications-draft-1
disease state id
ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
nct id
NCT03580655
qol key
qol-0018
source section id
NCT03580655-om-0018
source outcome index
18
instrument
EORTC QLQ
instrument basis
matched 'eortc' in the study's own wording
scale title
Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30)
measure definition
Quality of life was assessed using EORTC QLQ-C30, which is a 30-item questionnaire that includes 5 functional domains (physical, cognitive, role, emotional, and social) and a global health status scale. Twenty-eight questions are scored from 1 (not at all) to 4 (very much), while the other 2 are scored from 1 (very poor) to 7 (excellent). The calculated average is standardized using a linear transformation to a standardized scale of 0 to 100, with a decrease in score representing a decrease in quality of life. Each cycle is 28 days long.
time frame
Baseline, Cycle 1, Day 15 and Day 1 of Cycle 2, Cycle 3, Cycle 5, Cycle 7, Cycle 9, Cycle 11, Cycle 14, and Cycle 17 (28 days/cycle)
analysis role
secondary
reporting status
POSTED
higher is better
Source null
direction quote
Source null
population note
Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
Open the exact result →
Full draft applicability and source-link record
trial
release id
clinical-indications-draft-1
disease state id
ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
nct id
NCT03580655
trial label
(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocy
applicability
narrower
applicability note
Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
provenance class
regulator-cited
discovery note
Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
outcome Count
6
harms Count
2
qol Count
4
outcomes
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0000
    source section id
    NCT03580655-om-0000
    source outcome index
    0
    endpoint
    Overall Response Rate (ORR) Based on Modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (mIWG-MRT-ECNM) Response Criteria
    endpoint definition
    ORR was defined as the percentage of participants with a confirmed best response of complete remission (CR), complete remission with partial recovery of peripheral blood counts (CRh), partial remission (PR), or clinical improvement (CI) by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other clinical and laboratory parameters.
    time origin
    Baseline through Month 65
    analysis role
    primary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    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.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0001
    source section id
    NCT03580655-om-0001
    source outcome index
    1
    endpoint
    Mean Change From Baseline in Advanced Systemic Mastocytosis-Symptom Assessment Form (AdvSM-SAF) Total Symptom Score (TTS)
    endpoint definition
    The AdvSM-SAF is a 10-item questionnaire that assesses eight symptoms specific to AdvSM. All eight symptoms are scored on a scale of 0 (absence of symptoms) to 10 (more severe symptoms) (up to 80 points maximum). Each symptom contributes to the TSS equally. The TSS was generated based on average scores for each 7-day period. An increase in score from 0 (no symptoms) to 80 (worst symptoms represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an electronic diary (eDiary). Each cycle is 28 days long.
    time origin
    Baseline up to Month 6 (Cycle 6, Day 1)
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number of Participants Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoints.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0002
    source section id
    NCT03580655-om-0002
    source outcome index
    2
    endpoint
    ORR Based on mIWG-MRT-ECNM Response Criteria as Assessed by Local Investigator
    endpoint definition
    ORR was defined as the percentage of participants with a confirmed best response of CR, CRh, PR, or CI by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other clinical and laboratory parameters.
    time origin
    Baseline through Month 19
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by local investigator 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. Here, 'Overall Number of Participants Analyzed' signifies those participants evaluable for this outcome measure.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  4. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0003
    source section id
    NCT03580655-om-0003
    source outcome index
    3
    endpoint
    Time-to-Response (TTR)
    endpoint definition
    TTR was defined as the time from first dose to the time of initial evaluation of clinical improvement (CI) or better. Here, 'Overall Number of Participants Analyzed' signifies those participants with a confirmed best response of CR, CRh, PR, and CI by mIWG-MRT-ECNM criteria.
    time origin
    Baseline through Month 65
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    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.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  5. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0004
    source section id
    NCT03580655-om-0004
    source outcome index
    4
    endpoint
    Duration of Response (DOR)
    endpoint definition
    DOR was defined as the time from initial documentation of a CI or better to the time of initial documentation of confirmed progressive disease (PD) or death due to any cause, whichever occurred first. For responders who had not progressed or died at the time of analysis, DOR was censored at the last response assessment that was stable disease (SD) or better. Here, 'Overall Number of Participants Analyzed' signifies those participants with a confirmed best response of CR, CRh, PR, and CI by mIWG-MRT-ECNM criteria.
    time origin
    Baseline through Month 65
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    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.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  6. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0005
    source section id
    NCT03580655-om-0005
    source outcome index
    5
    endpoint
    Progression-free Survival (PFS)
    endpoint definition
    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.
    time origin
    Baseline through Month 65
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    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.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
arms
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0000
    arm key
    OG000
    arm label
    Avapritinib
    regimen
    Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles.
    analyzed count
    83
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT03580655-om-0000#groups/OG000
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0001
    arm key
    OG000
    arm label
    Avapritinib
    regimen
    Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles.
    analyzed count
    92
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT03580655-om-0001#groups/OG000
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0002
    arm key
    OG000
    arm label
    Avapritinib
    regimen
    Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles.
    analyzed count
    32
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT03580655-om-0002#groups/OG000
  4. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0003
    arm key
    OG000
    arm label
    Avapritinib
    regimen
    Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles.
    analyzed count
    61
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT03580655-om-0003#groups/OG000
  5. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0004
    arm key
    OG000
    arm label
    Avapritinib
    regimen
    Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles.
    analyzed count
    61
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT03580655-om-0004#groups/OG000
  6. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    outcome key
    om-0005
    arm key
    OG000
    arm label
    Avapritinib
    regimen
    Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles.
    analyzed count
    83
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT03580655-om-0005#groups/OG000
harms
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    harm key
    EG000
    source section id
    NCT03580655-ae
    event group key
    EG000
    event group label
    Avapritinib (100 mg)
    serious affected
    2
    serious at risk
    2
    other affected
    2
    other at risk
    2
    frequency threshold
    5
    collection period
    Day 1 through Month 65
    event definition
    All reported safety data based upon the Safety Population: all participants who received ≥1 dose of avapritinib regardless of the starting avapritinib dose (N=107). All participants were treated with a starting dose of 200 mg with the exception of 2 participants who were treated with a starting dose of 100 mg.
    undenominated note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    harm key
    EG001
    source section id
    NCT03580655-ae
    event group key
    EG001
    event group label
    Avapritinib (200 mg)
    serious affected
    67
    serious at risk
    105
    other affected
    104
    other at risk
    107
    frequency threshold
    5
    collection period
    Day 1 through Month 65
    event definition
    All reported safety data based upon the Safety Population: all participants who received ≥1 dose of avapritinib regardless of the starting avapritinib dose (N=107). All participants were treated with a starting dose of 200 mg with the exception of 2 participants who were treated with a starting dose of 100 mg.
    undenominated note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
quality of life
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    qol key
    qol-0001
    source section id
    NCT03580655-om-0001
    source outcome index
    1
    instrument
    Patient-reported symptom or function measure
    instrument basis
    matched 'symptom score' in the study's own wording
    scale title
    Mean Change From Baseline in Advanced Systemic Mastocytosis-Symptom Assessment Form (AdvSM-SAF) Total Symptom Score (TTS)
    measure definition
    The AdvSM-SAF is a 10-item questionnaire that assesses eight symptoms specific to AdvSM. All eight symptoms are scored on a scale of 0 (absence of symptoms) to 10 (more severe symptoms) (up to 80 points maximum). Each symptom contributes to the TSS equally. The TSS was generated based on average scores for each 7-day period. An increase in score from 0 (no symptoms) to 80 (worst symptoms represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an electronic diary (eDiary). Each cycle is 28 days long.
    time frame
    Baseline up to Month 6 (Cycle 6, Day 1)
    analysis role
    secondary
    reporting status
    POSTED
    higher is better
    0
    direction quote
    increase in score from 0 (no symptoms) to 80 (worst symptoms represents a worse
    population note
    Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number of Participants Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoints.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    qol key
    qol-0015
    source section id
    NCT03580655-om-0015
    source outcome index
    15
    instrument
    Patient-reported symptom or function measure
    instrument basis
    matched 'symptom score' in the study's own wording
    scale title
    Mean Change From Baseline in AdvSM-SAF Individual Symptom Scores (Severity) at Month 10
    measure definition
    The AdvSM-SAF is a 10-item questionnaire that assesses symptoms and functional domains specific to AdvSM. Individual symptom scores are generated based on average scores for each 7-day period. Eight symptoms are scored on a scale of 0 to 10 for severity. An increase in score represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an eDiary.
    time frame
    Baseline, Month 10
    analysis role
    secondary
    reporting status
    POSTED
    higher is better
    0
    direction quote
    increase in score represents a worse
    population note
    Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    qol key
    qol-0016
    source section id
    NCT03580655-om-0016
    source outcome index
    16
    instrument
    Patient-reported symptom or function measure
    instrument basis
    matched 'symptom score' in the study's own wording
    scale title
    Mean Change From Baseline in AdvSM-SAF Individual Symptom Scores (Episodes) at Month 10
    measure definition
    The AdvSM-SAF is a 10-item questionnaire that assesses symptoms and functional domains specific to AdvSM. Individual symptom scores are generated based on average scores for each 7-day period. Two symptoms (vomiting and diarrhea) are scored for number of episodes. An increase in score represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an eDiary.
    time frame
    Baseline, Month 10
    analysis role
    secondary
    reporting status
    POSTED
    higher is better
    0
    direction quote
    increase in score represents a worse
    population note
    Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  4. release id
    clinical-indications-draft-1
    disease state id
    ds-systemic-mastocytosis-with-an-associated-myeloid-neoplasm
    nct id
    NCT03580655
    qol key
    qol-0018
    source section id
    NCT03580655-om-0018
    source outcome index
    18
    instrument
    EORTC QLQ
    instrument basis
    matched 'eortc' in the study's own wording
    scale title
    Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30)
    measure definition
    Quality of life was assessed using EORTC QLQ-C30, which is a 30-item questionnaire that includes 5 functional domains (physical, cognitive, role, emotional, and social) and a global health status scale. Twenty-eight questions are scored from 1 (not at all) to 4 (very much), while the other 2 are scored from 1 (very poor) to 7 (excellent). The calculated average is standardized using a linear transformation to a standardized scale of 0 to 100, with a decrease in score representing a decrease in quality of life. Each cycle is 28 days long.
    time frame
    Baseline, Cycle 1, Day 15 and Day 1 of Cycle 2, Cycle 3, Cycle 5, Cycle 7, Cycle 9, Cycle 11, Cycle 14, and Cycle 17 (28 days/cycle)
    analysis role
    secondary
    reporting status
    POSTED
    higher is better
    Source null
    direction quote
    Source null
    population note
    Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
setting source links

    Preserved source evidence · Not medical advice