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NCT00471497 · OUTCOME

Percentage of Participants With MMR at 12 Months Between All 3 Arms by Sokal Risk Group With Imputation

A Study of Imatinib Versus Nilotinib in Adult Patients With Newly Diagnosed Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) · Source last updated 2020-11-18

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
12 months

What was measured

MMR is defined as the percentage of participants in MMR (reduction of ≥ 3 logs in BCR-ABL transcripts compared to the standardized baseline established in IRIS, or ≤ 0.1% BCR-ABL/ABL % by international scale and measured by real-time quantitative polymerase chain reaction (RQ-PCR)) at 12 months.

Analysis population: Full analysis Set (FAS): Contained 846 randomized patients. Patients were analyzed according to the treatment they were randomized to, regardless of actual treatment received.

Groups in this outcome

Imatinib 400 mg QD

Patients randomized to this arm were to receive 400 mg imatinib once a day (QD). If the patient required a dose escalation from 400 mg/day, the patient was to receive 400 mg imatinib twice daily orally. Imatinib was taken with food and a large glass of water. All patients were to avoid grapefruit, star fruit, pomegranate and Seville oranges or juices and products containing these fruits during the study. In cases of vomiting doses were not to be repeated

Nilotinb 300 mg BID

Patients who were randomized to this arm were to receive nilotinib 300 mg twice a day (BID) by mouth each morning and evening approximately 12 hours apart. If the morning or evening dose was delayed for more than 4 hours, the patient was to skip this dose and resume dosing with the next dose as per the original schedule in order to prevent overdosing. No imatinib washout period was necessary prior to administration of nilotinib. Nilotinib was not to be taken with food. No food was to be consumed for at least 2 hours before the dose was taken and no additional oral intake other than water was to be consumed for at least one hour after the dose was taken. Patients were instructed to swallow capsules whole with a full 8 ounce glass of water, and not to chew them. All patients were to avoid grapefruit, star fruit, pomegranate and Seville oranges or juices and products containing these fruits. Vomited doses were not to be repeated.

Nilotinib 400 mg BID

Patients who were randomized to this arm were to receive nilotinib 400 mg twice a day (BID) by mouth each morning and evening approximately 12 hours apart. If the morning or evening dose was delayed for more than 4 hours, the patient was to skip this dose and resume dosing with the next dose as per the original schedule in order to prevent overdosing. No imatinib washout period was necessary prior to administration of nilotinib. Nilotinib was not to be taken with food. No food was to be consumed for at least 2 hours before the dose was taken and no additional oral intake other than water was to be consumed for at least one hour after the dose was taken. Patients were instructed to swallow capsules whole with a full 8 ounce glass of water, and not to chew them. All patients were to avoid grapefruit, star fruit, pomegranate and Seville oranges or juices and products containing these fruits. Vomited doses were not to be repeated.

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

Analysis denominator · Participants

GroupSource count
Imatinib 400 mg QD283
Nilotinb 300 mg BID282
Nilotinib 400 mg BID281

Reported measurements

Sokal risk group = Low

Analysis denominator · Participants

GroupSource count
Imatinib 400 mg QD104
Nilotinb 300 mg BID103
Nilotinib 400 mg BID103
Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Imatinib 400 mg QD26.0Not reported17.935.5Not reported
Nilotinb 300 mg BID40.8Not reported31.250.9Not reported
Nilotinib 400 mg BID53.4Not reported43.363.3Not reported
Sokal risk group = Interm.

Analysis denominator · Participants

GroupSource count
Imatinib 400 mg QD101
Nilotinb 300 mg BID101
Nilotinib 400 mg BID100
Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Imatinib 400 mg QD22.8Not reported15.032.2Not reported
Nilotinb 300 mg BID50.5Not reported40.460.6Not reported
Nilotinib 400 mg BID40.0Not reported30.350.3Not reported
Sokal risk group = High

Analysis denominator · Participants

GroupSource count
Imatinib 400 mg QD78
Nilotinb 300 mg BID78
Nilotinib 400 mg BID78
Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Imatinib 400 mg QD16.7Not reported9.226.8Not reported
Nilotinb 300 mg BID41.0Not reported30.052.7Not reported
Nilotinib 400 mg BID32.1Not reported21.943.6Not reported

Source statistical analyses

Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.

  1. ci Lower Limit
    2.1
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    27.5
    group Description
    (Low)
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    14.8
  2. ci Lower Limit
    14.6
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    40.2
    group Description
    (Low)
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    27.4
  3. ci Lower Limit
    15.0
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    40.4
    group Description
    (Intermediate)
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    27.7
  4. ci Lower Limit
    4.6
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    29.8
    group Description
    (Intermediate)
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    17.2
  5. ci Lower Limit
    10.7
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    38.1
    group Description
    (High)
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    24.4
  6. ci Lower Limit
    2.1
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    28.6
    group Description
    (High)
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    15.4
Complete source fields
analyses
  1. ci Lower Limit
    2.1
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    27.5
    group Description
    (Low)
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    14.8
  2. ci Lower Limit
    14.6
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    40.2
    group Description
    (Low)
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    27.4
  3. ci Lower Limit
    15.0
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    40.4
    group Description
    (Intermediate)
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    27.7
  4. ci Lower Limit
    4.6
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    29.8
    group Description
    (Intermediate)
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    17.2
  5. ci Lower Limit
    10.7
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    38.1
    group Description
    (High)
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    24.4
  6. ci Lower Limit
    2.1
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    28.6
    group Description
    (High)
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    OTHER
    param Type
    Difference in response rate
    param Value
    15.4
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        17.9
        upper Limit
        35.5
        value
        26.0
      2. group Id
        OG001
        lower Limit
        31.2
        upper Limit
        50.9
        value
        40.8
      3. group Id
        OG002
        lower Limit
        43.3
        upper Limit
        63.3
        value
        53.4
    denoms
    1. counts
      1. group Id
        OG000
        value
        104
      2. group Id
        OG001
        value
        103
      3. group Id
        OG002
        value
        103
      units
      Participants
    title
    Sokal risk group = Low
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        15.0
        upper Limit
        32.2
        value
        22.8
      2. group Id
        OG001
        lower Limit
        40.4
        upper Limit
        60.6
        value
        50.5
      3. group Id
        OG002
        lower Limit
        30.3
        upper Limit
        50.3
        value
        40.0
    denoms
    1. counts
      1. group Id
        OG000
        value
        101
      2. group Id
        OG001
        value
        101
      3. group Id
        OG002
        value
        100
      units
      Participants
    title
    Sokal risk group = Interm.
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        9.2
        upper Limit
        26.8
        value
        16.7
      2. group Id
        OG001
        lower Limit
        30.0
        upper Limit
        52.7
        value
        41.0
      3. group Id
        OG002
        lower Limit
        21.9
        upper Limit
        43.6
        value
        32.1
    denoms
    1. counts
      1. group Id
        OG000
        value
        78
      2. group Id
        OG001
        value
        78
      3. group Id
        OG002
        value
        78
      units
      Participants
    title
    Sokal risk group = High
denoms
  1. counts
    1. group Id
      OG000
      value
      283
    2. group Id
      OG001
      value
      282
    3. group Id
      OG002
      value
      281
    units
    Participants
description
MMR is defined as the percentage of participants in MMR (reduction of ≥ 3 logs in BCR-ABL transcripts compared to the standardized baseline established in IRIS, or ≤ 0.1% BCR-ABL/ABL % by international scale and measured by real-time quantitative polymerase chain reaction (RQ-PCR)) at 12 months.
dispersion Type
95% Confidence Interval
groups
  1. description
    Patients randomized to this arm were to receive 400 mg imatinib once a day (QD). If the patient required a dose escalation from 400 mg/day, the patient was to receive 400 mg imatinib twice daily orally. Imatinib was taken with food and a large glass of water. All patients were to avoid grapefruit, star fruit, pomegranate and Seville oranges or juices and products containing these fruits during the study. In cases of vomiting doses were not to be repeated
    id
    OG000
    title
    Imatinib 400 mg QD
  2. description
    Patients who were randomized to this arm were to receive nilotinib 300 mg twice a day (BID) by mouth each morning and evening approximately 12 hours apart. If the morning or evening dose was delayed for more than 4 hours, the patient was to skip this dose and resume dosing with the next dose as per the original schedule in order to prevent overdosing. No imatinib washout period was necessary prior to administration of nilotinib. Nilotinib was not to be taken with food. No food was to be consumed for at least 2 hours before the dose was taken and no additional oral intake other than water was to be consumed for at least one hour after the dose was taken. Patients were instructed to swallow capsules whole with a full 8 ounce glass of water, and not to chew them. All patients were to avoid grapefruit, star fruit, pomegranate and Seville oranges or juices and products containing these fruits. Vomited doses were not to be repeated.
    id
    OG001
    title
    Nilotinb 300 mg BID
  3. description
    Patients who were randomized to this arm were to receive nilotinib 400 mg twice a day (BID) by mouth each morning and evening approximately 12 hours apart. If the morning or evening dose was delayed for more than 4 hours, the patient was to skip this dose and resume dosing with the next dose as per the original schedule in order to prevent overdosing. No imatinib washout period was necessary prior to administration of nilotinib. Nilotinib was not to be taken with food. No food was to be consumed for at least 2 hours before the dose was taken and no additional oral intake other than water was to be consumed for at least one hour after the dose was taken. Patients were instructed to swallow capsules whole with a full 8 ounce glass of water, and not to chew them. All patients were to avoid grapefruit, star fruit, pomegranate and Seville oranges or juices and products containing these fruits. Vomited doses were not to be repeated.
    id
    OG002
    title
    Nilotinib 400 mg BID
param Type
NUMBER
population Description
Full analysis Set (FAS): Contained 846 randomized patients. Patients were analyzed according to the treatment they were randomized to, regardless of actual treatment received.
reporting Status
POSTED
time Frame
12 months
title
Percentage of Participants With MMR at 12 Months Between All 3 Arms by Sokal Risk Group With Imputation
type
PRIMARY
unit Of Measure
Percentage of participants

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Preserved source evidence · Not medical advice