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← Myeloid Neoplasm

NCT02038036 · STUDY IN CONTEXT

Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.

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. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm. 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

Proportion of patients achieving Hct control at Week 28 was defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 28, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8. Phlebotomy eligibility was defined by: * Confirmed Hct \> 45% that is at least 3 percentage points higher than the Hct obtained at Baseline Or * Confirmed Hct \> 48% The confirmation occurred 2 to 14 days subsequent to the initial observation.

Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.

→

secondary · POSTED

Proportion of patients achieving a complete hematological remission at Week 28 was defined by: * Hct control at Week 28 defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 28, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8, and * WBC \< 10 x109/L at Week 28, and * Platelets ≤ 400 x 109/L at Week 28

Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.

→

secondary · POSTED

Proportion of patients achieving a Hct control at Week 52 was defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52, and no more than one phlebotomy eligibility occurring post randomization and prior to Week 8 \- Endpoint for Week 80 was defined, similarly.

Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.

→

secondary · POSTED

Proportion of patients achieving a complete hematological remission at Week 52, was defined by: * Hct control at Week 52, as defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52 with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8, and * White Blood Count (WBC) \< 10 x10\^9/L at Week 52, and * Platelets ≤ 400 x 10\^9/L at Week 52 * Endpoint for Week 80 was defined, similarly.

Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.

→

secondary · POSTED

Phlebotomy eligibility was defined by Confirmed Hct \> 45% that is at least 3 percentage points higher than the Hct obtained at Baseline Or Confirmed Hct \> 48%. The confirmation occurred 2 to 14 days subsequent to the initial observation.

Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.

→

secondary · POSTED

Hematocrit is the volume percentage of red blood cells (RBC) in the blood.

Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. Data after crossover for participants randomized to BAT are reported as separate outcome measure.

→

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 · Number of Participants Achieving a Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 28

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-myeloid-neoplasm
nct id
NCT02038036
qol key
qol-0009
source section id
NCT02038036-om-0009
source outcome index
9
instrument
Patient-reported symptom or function measure
instrument basis
matched 'symptom score' in the study's own wording
scale title
Number of Participants Achieving a Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 28
measure definition
Proportion of patients achieving a partial remission at Week 28, based on the ELN and IWG-MRT criteria, as defined by: * Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) score reduction of greater than or equal to 10 points from baseline to Week 28, and * Hct control defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 28, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8, and * WBC \< 10 x10\^9/L at Week 28, and * Platelets ≤ 400 x 10\^9/L at Week 28, and * No palpable spleen at Week 28, and * No hemorrhagic or thrombotic events, and * No transformation into post-PV myelofibrosis, myelodysplastic syndrome (IWG-MRT criteria) or acute leukemia (WHO criteria).
time frame
Week 28
analysis role
secondary
reporting status
POSTED
higher is better
Source null
direction quote
Source null
population note
Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.
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 · Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)

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-myeloid-neoplasm
nct id
NCT02038036
qol key
qol-0016
source section id
NCT02038036-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
Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
measure definition
The MPN-SAF TSS is a disease specific questionnaire comprised of 10 items that measures fatigue related to MPN disease and the severity of nine of the most prevalent associated symptoms. Each item is scored on a scale ranging from 0 (no fatigue/absent) to 10 (As bad as you can imagine/worst imaginable).The MPN-SAF TSS is computed as the average of the observed items multiplied by 10 to achieve a 0-to-100 scale. The MPN-SAF TSS thus has a possible score range of 0 to 100 where a decrease indicates improvement.
time frame
Baseline, Week 4, 8, 16, 28, 40, 52, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234 and 247
analysis role
secondary
reporting status
POSTED
higher is better
Source null
direction quote
Source null
population note
Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. Data after crossover for participants randomized to BAT are reported as separate outcome measure.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
Open the exact result →
EQ-5D · Change From Baseline in Score as Per European Quality of Life 5-Dimension 5-level (EQ-5D-5L) Questionnaire

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-myeloid-neoplasm
nct id
NCT02038036
qol key
qol-0018
source section id
NCT02038036-om-0018
source outcome index
18
instrument
EQ-5D
instrument basis
matched 'eq-5d' in the study's own wording
scale title
Change From Baseline in Score as Per European Quality of Life 5-Dimension 5-level (EQ-5D-5L) Questionnaire
measure definition
EQ-5D-5L is a standardized instrument for measuring health outcomes in a wide range of health conditions and treatments. It consists of visual analogue scale (EQ VAS) which records the respondent's self-rated health on a vertical, visual analogue scale where the endpoints are labeled 'Best imaginable health state' and 'worst imaginable health state'. The EQ VAS scores were anchored on 100 = the best health you can imagine and 0 = worst health you can imagine.
time frame
Baseline, Week 4, 8, 16, 28, 52, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234 and 247
analysis role
secondary
reporting status
POSTED
higher is better
Source null
direction quote
Source null
population note
Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. Data after crossover for participants randomized to BAT are reported as separate outcome measure.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
Open the exact result →
EQ-5D · Change From Baseline in EQ-5D-5L VAS, by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover

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-myeloid-neoplasm
nct id
NCT02038036
qol key
qol-0019
source section id
NCT02038036-om-0019
source outcome index
19
instrument
EQ-5D
instrument basis
matched 'eq-5d' in the study's own wording
scale title
Change From Baseline in EQ-5D-5L VAS, by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover
measure definition
EQ-5D-5L is a standardized instrument for measuring health outcomes in a wide range of health conditions and treatments. It consists of visual analogue scale (EQ VAS) which records the respondent's self-rated health on a vertical, visual analogue scale where the endpoints are labeled 'Best imaginable health state' and 'worst imaginable health state'. The EQ VAS scores were anchored on 100 = the best health you can imagine and 0 = worst health you can imagine.
time frame
Baseline (last assessment before cross over), Week 4, 8, 16, 24, 28, 52, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234 and 247 after cross-over
analysis role
secondary
reporting status
POSTED
higher is better
Source null
direction quote
Source null
population note
Cross-over set consisted of all participants randomized to the BAT arm, who crossed over and received at least one dose of ruxolitinib.
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-myeloid-neoplasm
nct id
NCT02038036
trial label
Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.
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. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm.
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
3
qol Count
4
outcomes
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0000
    source section id
    NCT02038036-om-0000
    source outcome index
    0
    endpoint
    Number of Participants Achieving Hematocrit (Hct) Control at Week 28
    endpoint definition
    Proportion of patients achieving Hct control at Week 28 was defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 28, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8. Phlebotomy eligibility was defined by: * Confirmed Hct \> 45% that is at least 3 percentage points higher than the Hct obtained at Baseline Or * Confirmed Hct \> 48% The confirmation occurred 2 to 14 days subsequent to the initial observation.
    time origin
    Week 28
    analysis role
    primary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.
    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-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0001
    source section id
    NCT02038036-om-0001
    source outcome index
    1
    endpoint
    Number of Participants Achieving a Complete Hematological Remission at Week 28
    endpoint definition
    Proportion of patients achieving a complete hematological remission at Week 28 was defined by: * Hct control at Week 28 defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 28, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8, and * WBC \< 10 x109/L at Week 28, and * Platelets ≤ 400 x 109/L at Week 28
    time origin
    Week 28
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.
    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-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0002
    source section id
    NCT02038036-om-0002
    source outcome index
    2
    endpoint
    Number of Participants Achieving a Hematocrit (Hct) Control at Week 52 and Week 80
    endpoint definition
    Proportion of patients achieving a Hct control at Week 52 was defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52, and no more than one phlebotomy eligibility occurring post randomization and prior to Week 8 \- Endpoint for Week 80 was defined, similarly.
    time origin
    Week 52 and 80
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the 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-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0003
    source section id
    NCT02038036-om-0003
    source outcome index
    3
    endpoint
    Number of Participants Achieving a Complete Hematological Remission at Week 52 and Week 80
    endpoint definition
    Proportion of patients achieving a complete hematological remission at Week 52, was defined by: * Hct control at Week 52, as defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52 with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8, and * White Blood Count (WBC) \< 10 x10\^9/L at Week 52, and * Platelets ≤ 400 x 10\^9/L at Week 52 * Endpoint for Week 80 was defined, similarly.
    time origin
    Week 52 and 80
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.
    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-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0004
    source section id
    NCT02038036-om-0004
    source outcome index
    4
    endpoint
    Number of Participants With Phlebotomies Over Time
    endpoint definition
    Phlebotomy eligibility was defined by Confirmed Hct \> 45% that is at least 3 percentage points higher than the Hct obtained at Baseline Or Confirmed Hct \> 48%. The confirmation occurred 2 to 14 days subsequent to the initial observation.
    time origin
    Baseline to Week 260
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.
    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-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0005
    source section id
    NCT02038036-om-0005
    source outcome index
    5
    endpoint
    Change From Baseline in Hematocrit (Hct) at Each Visit
    endpoint definition
    Hematocrit is the volume percentage of red blood cells (RBC) in the blood.
    time origin
    Baseline, Week 4, 8, 12, 16, 20, 24, 28, 40, 52, 66, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234, 247 and 260
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. Data after crossover for participants randomized to BAT are reported as separate outcome measure.
    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-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0000
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Ruxolitinib at a starting dose of 10 mg twice a day (bid). Dose was adjusted based on efficacy and safety parameters up to a maximum dose of 25 mg bid
    analyzed count
    74
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0000#groups/OG000
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0000
    arm key
    OG001
    arm label
    Best Available Therapy (BAT)
    regimen
    Best Available Therapy as selected by the investigator from: Hydroxyurea, Pegylated-Interferon (IFN/PEG-IFN), pipobroman, anagrelide, IMIDs, or observation. Participants randomized to BAT who did not respond by Week 28 were eligible to crossover and start treatment with ruxolitinib
    analyzed count
    75
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0000#groups/OG001
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0001
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Ruxolitinib at a starting dose of 10 mg twice a day (bid). Dose was adjusted based on efficacy and safety parameters up to a maximum dose of 25 mg bid
    analyzed count
    74
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0001#groups/OG000
  4. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0001
    arm key
    OG001
    arm label
    Best Available Therapy (BAT)
    regimen
    Best Available Therapy as selected by the investigator from: Hydroxyurea, Pegylated-Interferon (IFN/PEG-IFN), pipobroman, anagrelide, IMIDs, or observation. Participants randomized to BAT who did not respond by Week 28 were eligible to crossover and start treatment with ruxolitinib
    analyzed count
    75
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0001#groups/OG001
  5. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0002
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Ruxolitinib at a starting dose of 10 mg twice a day (bid). Dose was adjusted based on efficacy and safety parameters up to a maximum dose of 25 mg bid
    analyzed count
    74
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0002#groups/OG000
  6. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0002
    arm key
    OG001
    arm label
    Best Available Therapy (BAT)
    regimen
    Best Available Therapy as selected by the investigator from: Hydroxyurea, Pegylated-Interferon (IFN/PEG-IFN), pipobroman, anagrelide, IMIDs, or observation. Participants randomized to BAT who did not respond by Week 28 were eligible to crossover and start treatment with ruxolitinib
    analyzed count
    75
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0002#groups/OG001
  7. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0003
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Ruxolitinib at a starting dose of 10 mg twice a day (bid). Dose was adjusted based on efficacy and safety parameters up to a maximum dose of 25 mg bid
    analyzed count
    74
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0003#groups/OG000
  8. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0003
    arm key
    OG001
    arm label
    Best Available Therapy (BAT)
    regimen
    Best Available Therapy as selected by the investigator from: Hydroxyurea, Pegylated-Interferon (IFN/PEG-IFN), pipobroman, anagrelide, IMIDs, or observation. Participants randomized to BAT who did not respond by Week 28 were eligible to crossover and start treatment with ruxolitinib
    analyzed count
    75
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0003#groups/OG001
  9. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0004
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Ruxolitinib at a starting dose of 10 mg twice a day (bid). Dose was adjusted based on efficacy and safety parameters up to a maximum dose of 25 mg bid
    analyzed count
    74
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0004#groups/OG000
  10. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0004
    arm key
    OG001
    arm label
    Best Available Therapy (BAT)
    regimen
    Best Available Therapy as selected by the investigator from: Hydroxyurea, Pegylated-Interferon (IFN/PEG-IFN), pipobroman, anagrelide, IMIDs, or observation. Participants randomized to BAT who did not respond by Week 28 were eligible to crossover and start treatment with ruxolitinib
    analyzed count
    75
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0004#groups/OG001
  11. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0005
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Ruxolitinib at a starting dose of 10 mg twice a day (bid). Dose was adjusted based on efficacy and safety parameters up to a maximum dose of 25 mg bid
    analyzed count
    74
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0005#groups/OG000
  12. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    outcome key
    om-0005
    arm key
    OG001
    arm label
    Best Available Therapy (BAT)
    regimen
    Best Available Therapy as selected by the investigator from: Hydroxyurea, Pegylated-Interferon (IFN/PEG-IFN), pipobroman, anagrelide, IMIDs, or observation. Participants randomized to BAT who did not respond by Week 28 were eligible to crossover and start treatment with ruxolitinib
    analyzed count
    75
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT02038036-om-0005#groups/OG001
harms
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT02038036
    harm key
    EG000
    source section id
    NCT02038036-ae
    event group key
    EG000
    event group label
    Ruxolitinib
    serious affected
    34
    serious at risk
    74
    other affected
    73
    other at risk
    74
    frequency threshold
    5
    collection period
    Adverse events were collected from the day of first dose of study medication to the End of study (End of Treatment +30 days) visit which was Week 260 or prior (+30 days for Rux + crossover) and Week 80 or prior (+30 days for BAT only).
    event definition
    Any signs or symptoms that occured from the day of first dose of study medication to the End of study (End of Treatment +30 days) visit which was Week 260 or prior (+30 days for Rux + crossover) and Week 80 or prior (+30 days for BAT only).
    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-myeloid-neoplasm
    nct id
    NCT02038036
    harm key
    EG001
    source section id
    NCT02038036-ae
    event group key
    EG001
    event group label
    Best Available Therapy
    serious affected
    9
    serious at risk
    75
    other affected
    56
    other at risk
    75
    frequency threshold
    5
    collection period
    Adverse events were collected from the day of first dose of study medication to the End of study (End of Treatment +30 days) visit which was Week 260 or prior (+30 days for Rux + crossover) and Week 80 or prior (+30 days for BAT only).
    event definition
    Any signs or symptoms that occured from the day of first dose of study medication to the End of study (End of Treatment +30 days) visit which was Week 260 or prior (+30 days for Rux + crossover) and Week 80 or prior (+30 days for BAT only).
    undenominated note
    Source null
    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-myeloid-neoplasm
    nct id
    NCT02038036
    harm key
    EG002
    source section id
    NCT02038036-ae
    event group key
    EG002
    event group label
    All Crossover Patients
    serious affected
    23
    serious at risk
    58
    other affected
    56
    other at risk
    58
    frequency threshold
    5
    collection period
    Adverse events were collected from the day of first dose of study medication to the End of study (End of Treatment +30 days) visit which was Week 260 or prior (+30 days for Rux + crossover) and Week 80 or prior (+30 days for BAT only).
    event definition
    Any signs or symptoms that occured from the day of first dose of study medication to the End of study (End of Treatment +30 days) visit which was Week 260 or prior (+30 days for Rux + crossover) and Week 80 or prior (+30 days for BAT only).
    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-myeloid-neoplasm
    nct id
    NCT02038036
    qol key
    qol-0009
    source section id
    NCT02038036-om-0009
    source outcome index
    9
    instrument
    Patient-reported symptom or function measure
    instrument basis
    matched 'symptom score' in the study's own wording
    scale title
    Number of Participants Achieving a Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 28
    measure definition
    Proportion of patients achieving a partial remission at Week 28, based on the ELN and IWG-MRT criteria, as defined by: * Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) score reduction of greater than or equal to 10 points from baseline to Week 28, and * Hct control defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 28, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8, and * WBC \< 10 x10\^9/L at Week 28, and * Platelets ≤ 400 x 10\^9/L at Week 28, and * No palpable spleen at Week 28, and * No hemorrhagic or thrombotic events, and * No transformation into post-PV myelofibrosis, myelodysplastic syndrome (IWG-MRT criteria) or acute leukemia (WHO criteria).
    time frame
    Week 28
    analysis role
    secondary
    reporting status
    POSTED
    higher is better
    Source null
    direction quote
    Source null
    population note
    Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure.
    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-myeloid-neoplasm
    nct id
    NCT02038036
    qol key
    qol-0016
    source section id
    NCT02038036-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
    Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
    measure definition
    The MPN-SAF TSS is a disease specific questionnaire comprised of 10 items that measures fatigue related to MPN disease and the severity of nine of the most prevalent associated symptoms. Each item is scored on a scale ranging from 0 (no fatigue/absent) to 10 (As bad as you can imagine/worst imaginable).The MPN-SAF TSS is computed as the average of the observed items multiplied by 10 to achieve a 0-to-100 scale. The MPN-SAF TSS thus has a possible score range of 0 to 100 where a decrease indicates improvement.
    time frame
    Baseline, Week 4, 8, 16, 28, 40, 52, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234 and 247
    analysis role
    secondary
    reporting status
    POSTED
    higher is better
    Source null
    direction quote
    Source null
    population note
    Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. Data after crossover for participants randomized to BAT are reported as separate outcome measure.
    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-myeloid-neoplasm
    nct id
    NCT02038036
    qol key
    qol-0018
    source section id
    NCT02038036-om-0018
    source outcome index
    18
    instrument
    EQ-5D
    instrument basis
    matched 'eq-5d' in the study's own wording
    scale title
    Change From Baseline in Score as Per European Quality of Life 5-Dimension 5-level (EQ-5D-5L) Questionnaire
    measure definition
    EQ-5D-5L is a standardized instrument for measuring health outcomes in a wide range of health conditions and treatments. It consists of visual analogue scale (EQ VAS) which records the respondent's self-rated health on a vertical, visual analogue scale where the endpoints are labeled 'Best imaginable health state' and 'worst imaginable health state'. The EQ VAS scores were anchored on 100 = the best health you can imagine and 0 = worst health you can imagine.
    time frame
    Baseline, Week 4, 8, 16, 28, 52, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234 and 247
    analysis role
    secondary
    reporting status
    POSTED
    higher is better
    Source null
    direction quote
    Source null
    population note
    Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. Data after crossover for participants randomized to BAT are reported as separate 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-myeloid-neoplasm
    nct id
    NCT02038036
    qol key
    qol-0019
    source section id
    NCT02038036-om-0019
    source outcome index
    19
    instrument
    EQ-5D
    instrument basis
    matched 'eq-5d' in the study's own wording
    scale title
    Change From Baseline in EQ-5D-5L VAS, by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover
    measure definition
    EQ-5D-5L is a standardized instrument for measuring health outcomes in a wide range of health conditions and treatments. It consists of visual analogue scale (EQ VAS) which records the respondent's self-rated health on a vertical, visual analogue scale where the endpoints are labeled 'Best imaginable health state' and 'worst imaginable health state'. The EQ VAS scores were anchored on 100 = the best health you can imagine and 0 = worst health you can imagine.
    time frame
    Baseline (last assessment before cross over), Week 4, 8, 16, 24, 28, 52, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234 and 247 after cross-over
    analysis role
    secondary
    reporting status
    POSTED
    higher is better
    Source null
    direction quote
    Source null
    population note
    Cross-over set consisted of all participants randomized to the BAT arm, who crossed over and received at least one dose of ruxolitinib.
    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