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NCT03306264 · OUTCOME

MDS/CMML: Percentage of Participants With Red Blood Cell (RBC) and Platelet Transfusion Independence (TI)

Study of ASTX727 vs IV Decitabine in Participants With MDS, CMML, and AML · Source last updated 2024-08-27

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
Up to approximately 2.7 years

What was measured

Transfusion independence was defined as no transfusion for 56 consecutive days or more (84 and 112 days) after the first dose of study treatment while maintaining hemoglobin ≥8 grams/deciliter (g/dL) (RBC TI) or maintaining platelets ≥20×109/L (platelet TI).

Analysis population: Efficacy Analysis Set included all participants who received any amount of study treatment. 'Overall number of participants analyzed' signifies those who were evaluable for this outcome measure. 'Number analyzed' signifies those subjects who were evaluable for this outcome measure in specified category.

Groups in this outcome

MDS or CMML: ASTX727 or IV Decitabine

Participants with MDS or CMML received ASTX727 tablet, containing the fixed-dose combination of 35 mg decitabine and 100 mg cedazuridine, orally, once daily, on Days 1 to 5 in cycle 1 (each cycle = 28 days), followed by IV infusion of decitabine 20 mg/m\^2, once daily, on Days 1 to 5 in cycle 2 or the converse. A washout period of 23 days was maintained between the 2 cycles. From cycle 3, all participants enrolled in cycles 1 and 2 received ASTX727 tablet, once daily, on Days 1 to 5 of each 28-day cycle until disease progression, unacceptable toxicity, treatment discontinuation for other reasons, or withdrawal from the study.

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

Analysis denominator · Participants

GroupSource count
MDS or CMML: ASTX727 or IV Decitabine54

Reported measurements

RBC TI: ≥56 Days

Analysis denominator · Participants

GroupSource count
MDS or CMML: ASTX727 or IV Decitabine54
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
MDS or CMML: ASTX727 or IV Decitabine51.9Not reported37.865.7Not reported
RBC TI: ≥84 Days

Analysis denominator · Participants

GroupSource count
MDS or CMML: ASTX727 or IV Decitabine54
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
MDS or CMML: ASTX727 or IV Decitabine40.7Not reported27.655.0Not reported
RBC TI: ≥112 Days

Analysis denominator · Participants

GroupSource count
MDS or CMML: ASTX727 or IV Decitabine54
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
MDS or CMML: ASTX727 or IV Decitabine33.3Not reported21.147.5Not reported
Platelet TI: ≥56 Days

Analysis denominator · Participants

GroupSource count
MDS or CMML: ASTX727 or IV Decitabine12
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
MDS or CMML: ASTX727 or IV Decitabine50.0Not reported21.178.9Not reported
Platelet TI: ≥84 Days

Analysis denominator · Participants

GroupSource count
MDS or CMML: ASTX727 or IV Decitabine12
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
MDS or CMML: ASTX727 or IV Decitabine33.3Not reported9.965.1Not reported
Platelet TI: ≥112 Days

Analysis denominator · Participants

GroupSource count
MDS or CMML: ASTX727 or IV Decitabine12
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
MDS or CMML: ASTX727 or IV Decitabine33.3Not reported9.965.1Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        37.8
        upper Limit
        65.7
        value
        51.9
    denoms
    1. counts
      1. group Id
        OG000
        value
        54
      units
      Participants
    title
    RBC TI: ≥56 Days
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        27.6
        upper Limit
        55.0
        value
        40.7
    denoms
    1. counts
      1. group Id
        OG000
        value
        54
      units
      Participants
    title
    RBC TI: ≥84 Days
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        21.1
        upper Limit
        47.5
        value
        33.3
    denoms
    1. counts
      1. group Id
        OG000
        value
        54
      units
      Participants
    title
    RBC TI: ≥112 Days
  4. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        21.1
        upper Limit
        78.9
        value
        50.0
    denoms
    1. counts
      1. group Id
        OG000
        value
        12
      units
      Participants
    title
    Platelet TI: ≥56 Days
  5. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        9.9
        upper Limit
        65.1
        value
        33.3
    denoms
    1. counts
      1. group Id
        OG000
        value
        12
      units
      Participants
    title
    Platelet TI: ≥84 Days
  6. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        9.9
        upper Limit
        65.1
        value
        33.3
    denoms
    1. counts
      1. group Id
        OG000
        value
        12
      units
      Participants
    title
    Platelet TI: ≥112 Days
denoms
  1. counts
    1. group Id
      OG000
      value
      54
    units
    Participants
description
Transfusion independence was defined as no transfusion for 56 consecutive days or more (84 and 112 days) after the first dose of study treatment while maintaining hemoglobin ≥8 grams/deciliter (g/dL) (RBC TI) or maintaining platelets ≥20×109/L (platelet TI).
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants with MDS or CMML received ASTX727 tablet, containing the fixed-dose combination of 35 mg decitabine and 100 mg cedazuridine, orally, once daily, on Days 1 to 5 in cycle 1 (each cycle = 28 days), followed by IV infusion of decitabine 20 mg/m\^2, once daily, on Days 1 to 5 in cycle 2 or the converse. A washout period of 23 days was maintained between the 2 cycles. From cycle 3, all participants enrolled in cycles 1 and 2 received ASTX727 tablet, once daily, on Days 1 to 5 of each 28-day cycle until disease progression, unacceptable toxicity, treatment discontinuation for other reasons, or withdrawal from the study.
    id
    OG000
    title
    MDS or CMML: ASTX727 or IV Decitabine
param Type
NUMBER
population Description
Efficacy Analysis Set included all participants who received any amount of study treatment. 'Overall number of participants analyzed' signifies those who were evaluable for this outcome measure. 'Number analyzed' signifies those subjects who were evaluable for this outcome measure in specified category.
reporting Status
POSTED
time Frame
Up to approximately 2.7 years
title
MDS/CMML: Percentage of Participants With Red Blood Cell (RBC) and Platelet Transfusion Independence (TI)
type
SECONDARY
unit Of Measure
percentage of participants

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Preserved source evidence · Not medical advice