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NCT01566695 · OUTCOME

Percentage of Participants With a Hematologic Response According to the 2006 IWG Criteria for MDS

The Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo and Best Supportive Care in Subjects With Red Blood Cell (RBC) Transfusion-Dependent Anemia and Thrombocytopenia Due to International Prognostic Scoring System (IPSS) Low Risk Myelodysplastic Syndrome (MDS) · Source last updated 2025-01-07

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
Not reported
Time frame
Response was assessed every 3 cycles; up to the data cut-off date of 25 Jan 2019; median duration of exposure to oral azacitidine was 86.0 days and 119.0 days for placebo

What was measured

Hematologic response was defined as: • A complete response (CR): \<5% myeloblasts, and normal maturation of all cell lines; Peripheral blood (PB) shows: hemoglobin \>10 g/dL, neutrophils ≥1.0x10\^9/L, platelets ≥100x10\^9/dL, blasts (0%) • Partial Response (PR): same as CR bone marrow (BM) shows blasts decreased by ≥ 50% over pre-treatment but still \> 5%; Cellularity and morphology not relevant • Marrow CR: BM: ≤ 5% myeloblasts and decrease by ≥ 50% over pre-treatment PB • Stable disease (SD): failure to achieve at least PR, but no evidence of progression for \> 8 wks • Failure: death during treatment or disease progression • Disease Progression for those with: - Less than 5% blasts: ≥ 50% increase in blasts to \> 5% blasts - 5%-10% blasts:≥ 50% increase to \> 10% blasts - 10%-20% blasts:≥ 50% increase to \> 20% blasts - 20%-30% blasts ≥ 50% increase to \> 30% blasts Any of the following: - ≥ 50% decrease from maximum remission/response in granulocytes or platelets

Analysis population: Population includes participants with a CR, PR and mCR who had baseline bone marrow blasts \> 5%. ITT population.

Groups in this outcome

Oral Azacitidine and Best Supportive Care

Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.

Placebo Plus Best Supportive Care

Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.

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

Analysis denominator · Participants

GroupSource count
Oral Azacitidine and Best Supportive Care107
Placebo Plus Best Supportive Care109

Reported measurements

Complete Response (CR)
Values in Percentage of Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Oral Azacitidine and Best Supportive Care7.7Not reportedNot reportedNot reportedNot reported
Placebo Plus Best Supportive Care0Not reportedNot reportedNot reportedNot reported
Partial Response
Values in Percentage of Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Oral Azacitidine and Best Supportive Care0Not reportedNot reportedNot reportedNot reported
Placebo Plus Best Supportive Care0Not reportedNot reportedNot reportedNot reported
Marrow CR
Values in Percentage of Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Oral Azacitidine and Best Supportive Care23.1Not reportedNot reportedNot reportedNot reported
Placebo Plus Best Supportive Care4.2Not reportedNot reportedNot reportedNot reported
Stable Disease (SD)
Values in Percentage of Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Oral Azacitidine and Best Supportive Care2.8Not reportedNot reportedNot reportedNot reported
Placebo Plus Best Supportive Care30.3Not reportedNot reportedNot reportedNot reported
Disease Progression
Values in Percentage of Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Oral Azacitidine and Best Supportive Care62.6Not reportedNot reportedNot reportedNot reported
Placebo Plus Best Supportive Care46.8Not reportedNot reportedNot reportedNot reported
Failure due to Death
Values in Percentage of Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Oral Azacitidine and Best Supportive Care0.9Not reportedNot reportedNot reportedNot reported
Placebo Plus Best Supportive Care0.9Not reportedNot reportedNot reportedNot reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        value
        7.7
      2. group Id
        OG001
        value
        0
    title
    Complete Response (CR)
  2. categories
    1. measurements
      1. group Id
        OG000
        value
        0
      2. group Id
        OG001
        value
        0
    title
    Partial Response
  3. categories
    1. measurements
      1. group Id
        OG000
        value
        23.1
      2. group Id
        OG001
        value
        4.2
    title
    Marrow CR
  4. categories
    1. measurements
      1. group Id
        OG000
        value
        2.8
      2. group Id
        OG001
        value
        30.3
    title
    Stable Disease (SD)
  5. categories
    1. measurements
      1. group Id
        OG000
        value
        62.6
      2. group Id
        OG001
        value
        46.8
    title
    Disease Progression
  6. categories
    1. measurements
      1. group Id
        OG000
        value
        0.9
      2. group Id
        OG001
        value
        0.9
    title
    Failure due to Death
denoms
  1. counts
    1. group Id
      OG000
      value
      107
    2. group Id
      OG001
      value
      109
    units
    Participants
description
Hematologic response was defined as: • A complete response (CR): \<5% myeloblasts, and normal maturation of all cell lines; Peripheral blood (PB) shows: hemoglobin \>10 g/dL, neutrophils ≥1.0x10\^9/L, platelets ≥100x10\^9/dL, blasts (0%) • Partial Response (PR): same as CR bone marrow (BM) shows blasts decreased by ≥ 50% over pre-treatment but still \> 5%; Cellularity and morphology not relevant • Marrow CR: BM: ≤ 5% myeloblasts and decrease by ≥ 50% over pre-treatment PB • Stable disease (SD): failure to achieve at least PR, but no evidence of progression for \> 8 wks • Failure: death during treatment or disease progression • Disease Progression for those with: - Less than 5% blasts: ≥ 50% increase in blasts to \> 5% blasts - 5%-10% blasts:≥ 50% increase to \> 10% blasts - 10%-20% blasts:≥ 50% increase to \> 20% blasts - 20%-30% blasts ≥ 50% increase to \> 30% blasts Any of the following: - ≥ 50% decrease from maximum remission/response in granulocytes or platelets
groups
  1. description
    Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.
    id
    OG000
    title
    Oral Azacitidine and Best Supportive Care
  2. description
    Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
    id
    OG001
    title
    Placebo Plus Best Supportive Care
param Type
NUMBER
population Description
Population includes participants with a CR, PR and mCR who had baseline bone marrow blasts \> 5%. ITT population.
reporting Status
POSTED
time Frame
Response was assessed every 3 cycles; up to the data cut-off date of 25 Jan 2019; median duration of exposure to oral azacitidine was 86.0 days and 119.0 days for placebo
title
Percentage of Participants With a Hematologic Response According to the 2006 IWG Criteria for MDS
type
SECONDARY
unit Of Measure
Percentage of Participants

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Preserved source evidence · Not medical advice