NCT01566695 · OUTCOME

Mean Change From Baseline in the Anemia Subscale Within FACT-An Instrument at Cycle 6

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: MEAN
Unit: Units on a Scale
Interval / dispersion: Standard Deviation
Time frame: Baseline to Cycle 6 Day 1

What was measured

The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being (PWG), social/family (SWB), emotional well being (EWB) and Functional Well-Being (FWB) and an additional 20-item anemia questionnaire that measures fatigue associated items and 7 non-fatigue items. The scales are formatted on 1 to 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general health related quality of life (HRQoL), the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various therapeutic areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 indicates the poorest QOL and 100 denotes the highest QOL.

Analysis population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

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	42
Placebo Plus Best Supportive Care	49

Reported measurements

Source class 1

Values in Units on a Scale · Interval/dispersion: Standard Deviation
Group	Value	Spread	Lower	Upper	Comment
Oral Azacitidine and Best Supportive Care	2.9	11.81	Not reported	Not reported	Not reported
Placebo Plus Best Supportive Care	-0.6	10.39	Not reported	Not reported	Not reported

Source statistical analyses

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

group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.130
statistical Comment
The p-value was calculated based on a pooled 2-sample, 2-sided t-test for the difference in mean change from baseline between treatment groups.
statistical Method
t-test, 2 sided

Complete source fields

analyses

group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.130
statistical Comment
The p-value was calculated based on a pooled 2-sample, 2-sided t-test for the difference in mean change from baseline between treatment groups.
statistical Method
t-test, 2 sided

classes

categories
measurements
group Id
OG000
spread
11.81
value
2.9
group Id
OG001
spread
10.39
value
-0.6

denoms

counts
group Id
OG000
value
42
group Id
OG001
value
49
units
Participants

description

dispersion Type

Standard Deviation

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

MEAN

population Description

Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

reporting Status

POSTED

time Frame

Baseline to Cycle 6 Day 1

title

Mean Change From Baseline in the Anemia Subscale Within FACT-An Instrument at Cycle 6

type

SECONDARY

unit Of Measure

Units on a Scale

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice