NCT01566695 · OUTCOME

Percentage of Participants With a Hematological Improvement Response in Platelets (HI-P) According to 2006 IWG Criteria

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: 95% Confidence Interval
Time frame: From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

What was measured

HI-P response was defined according to IWG 2006 criteria (Cheson, 2006) and as: 1. Absolute increase of ≥ 30 X 10\^9/L for participants\^ starting with \> 20 X 10\^9/L platelets; 2. Increase from \< 20 X 10\^9/L to \> 20 X 10\^9/L and by at least 100%. HI-P must have lasted at least 8 weeks.

Analysis population: The ITT population included all participants who were randomized, regardless of whether they received treatment or not.

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

Group	Source count
Oral Azacitidine and Best Supportive Care	107
Placebo Plus Best Supportive Care	109

Reported measurements

Source class 1

Values in Percentage of Participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Oral Azacitidine and Best Supportive Care	24.3	Not reported	16.2	32.4	Not reported
Placebo Plus Best Supportive Care	7.3	Not reported	2.4	12.2	Not reported

Source statistical analyses

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

ci Lower Limit
7.5
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
26.4
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.0007
param Type
Rate Difference
param Value
17.0
statistical Comment
Stratified by average BL RBC tfx needs: ≤4 units versus \>4 units; BL platelet tfx status: dependent or independent and ECOG PS: 0 to 1 vs 2
statistical Method
Stratified Mantel-Haenszel. Chi-squared

Complete source fields

analyses

ci Lower Limit
7.5
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
26.4
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.0007
param Type
Rate Difference
param Value
17.0
statistical Comment
Stratified by average BL RBC tfx needs: ≤4 units versus \>4 units; BL platelet tfx status: dependent or independent and ECOG PS: 0 to 1 vs 2
statistical Method
Stratified Mantel-Haenszel. Chi-squared

classes

categories
measurements
group Id
OG000
lower Limit
16.2
upper Limit
32.4
value
24.3
group Id
OG001
lower Limit
2.4
upper Limit
12.2
value
7.3

denoms

counts
group Id
OG000
value
107
group Id
OG001
value
109
units
Participants

description

dispersion Type

95% Confidence Interval

groups

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
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

The ITT population included all participants who were randomized, regardless of whether they received treatment or not.

reporting Status

POSTED

time Frame

From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

title

Percentage of Participants With a Hematological Improvement Response in Platelets (HI-P) According to 2006 IWG Criteria

type

SECONDARY

unit Of Measure

Percentage of Participants

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Preserved source evidence · Not medical advice