Symptoms and General Pathology

All Conditions

Blood and Lymph Conditions

Neoplasms

Rare Diseases

Syndrome

Anemia

Thrombocytopenia

Preleukemia

Myelodysplastic Syndromes

Hematologic Diseases

Bone Marrow Diseases

Precancerous Conditions

Blood Platelet Disorders

Cytopenia

Anti-Infective Agents

All Drugs and Chemicals

Antineoplastic Agents

Anti-Bacterial Agents

Azacitidine

Lenograstim

Antibiotics, Antitubercular

Clotrimazole

Miconazole

Antiviral Agents

Antifungal Agents

Cc-486

Antimetabolites

Enzyme Inhibitors

Arm 1: Oral azacitidine tablets 300 mg daily (QD) + best supportive care (BSC) on days 1 through 21 of each 28-day treatment cycle.

Arm 2: Identically matching placebo tablets plus best supportive care on days 1 to 21 of each 28-day treatment cycle.

300 mg daily, days 1 to 21 of each 28-day treatment cycle

Oral Azacitidine

Identically matching placebo tablets on day 1 to 21 of each 28-day treatment cycle.

Placebo

BSC included and was not limited to packed RBC (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.

Best Supportiv Care (BSC)

Bristol-Myers Squibb

Evaluation of 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) lower risk myelodysplastic syndromes (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)

A Phase 3, Multicenter, Randomized, Double-blind Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo Plus Best Supportive Care in Subjects With Red Blood Cell Transfusion-dependent Anemia and Thrombocytopenia Due to IPSS Lower-risk Myelodysplastic Syndromes.

RBC transfusion (tfx) independence was defined as the absence of any RBC transfusion during any consecutive "rolling" 56 days within the treatment period. Participants who did not receive any RBC transfusion during a consecutive rolling 56 days (i.e., day 1 to day 56, day 2 to day 57) were considered as a 56-day RBC transfusion independent responder.

Duration of RBC transfusion independence was analyzed only for participants who achieved RBC transfusion independence of ≥ 56 days on treatment. Duration of RBC transfusion independence was defined as the time from the date transfusion independence is first observed (day 1 of a ≥ 56 days period without a transfusion) until the date the participants had a subsequently documented RBC transfusion. In the event a participant had more than one ≥56 days rolling periods which met the RBC independence criteria, the duration with the longest rolling period was used in the analysis. Participants who maintained RBC TI through the end of the treatment period were censored at the date of treatment discontinuation, death, or 1 day before the start of the subsequent MDS treatment (if any), whichever occurred first, or the particiapnts latest available assessment date in the database if the treatment was still on-going.

Time to RBC transfusion independence of ≥ 56 days was defined as the time between randomization and the date onset of transfusion independence was first observed (ie, Day 1 of 56 without any RBC transfusions).

A participant was considered as a RBC transfusion reduction responder if the participant had at least 4 units reduction in transfusion units over any consecutive 56 days period compared to the baseline transfusion units in 56 days.

RBC transfusion independence was defined as the absence of any RBC transfusion during any consecutive "rolling" 84 days within the treatment period. Participants who did not receive any RBC transfusion during a consecutive rolling 84 days (i.e., day 1 to day 84, day 2 to day 85) were considered as a 84-day RBC transfusion independent responder.

Duration of RBC transfusion independence was analyzed only for participants who achieved RBC transfusion independence of ≥ 84 days on treatment. Duration of RBC transfusion independence was defined as the time from the date transfusion independence is first observed (day 1 of a ≥ 84 days period without a transfusion) until the date the participants had a subsequently documented RBC transfusion. In case a participant had more than one ≥84 days rolling periods which met the RBC independence criteria, the duration with the longest rolling period was used in the analysis.

Time to RBC transfusion independence of ≥ 84 days was defined as the time between randomization and the date onset of transfusion independence was first observed (ie, Day 1 of 84 without any RBC transfusions).

Erythroid HI-E improvement was defined as a hemoglobin increase of ≥ 1.5 g/dL; or a reduction in units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a hemoglobin of ≤ 9.0 g/dL on treatment were counted in the RBC transfusion response evaluation.

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.

Platelet transfusion independence was defined as the absence of any platelet transfusion during any consecutive "rolling" 56 days during the treatment period, (ie, Day 1 to 56, Day 2 to 57, Days 3 to 58, etc.). Participants were considered platelet transfusion dependent at baseline if they had received ≥ 2 platelet transfusions during the 56 days immediately preceding randomization and had no consecutive 28-day period during which no platelet transfusions were administered.

Time to platelet transfusion independence was defined as the time between randomization and the first documented date of onset of transfusion independence (ie, Day 1 of 56 without any platelet transfusions).

Overall survial was defined as the time from randomization to death from any cause, and was calculated using randomization date and date of death, or date of last follow-up for censored participants. All subjects were followed until drop out (withdrawal of consent from further data collection or lost to follow-up), death, or study closure. Participants who dropped out or were alive at study closure (or at the time of the interim analysis) had their OS times censored at the time of last contact, as appropriate. Overall survival was assessed as an interim analysis at the time of the primary analysis.

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

Participants with a documented diagnosis of AML arising from previous MDS documented diagnosis.

Time to AML progression was defined as the time from the date of randomization until the date the subject has documented progression to AML. For participants who had progression to AML documented in MLL central lab report, the earliest sample collection date with the diagnosis of "s-AML arising from previous MDS" was used as the date to AML progression.

Clinically significant bleeding event was defined as: any intracranial or retroperitoneal bleed; bleeding requiring transfusions of \> 2 units of blood/blood products; bleeding associated with a decrease in hemoglobin of \> 2 g/dL; or bleeding from any site requiring transfusions of \> 2 units of blood.

A TEAE was defined as an adverse event that begins or worsens in intensity of frequency on or after the first dose of study drug through 28 days after last dose of study drug.

A serious adverse event (SAE) is any:

* Death;
* Life-threatening event;
* Any inpatient hospitalization or prolongation of existing hospitalization;
* Persistent or significant disability or incapacity;
* Congenital anomaly or birth defect;
* Any other important medical event The investigator determined the relationship of an AE to study drug based on the timing of the AE relative to drug administration and whether or not other drugs, therapeutic interventions, or underlying conditions could provide a sufficient explanation for the event. The severity of an AE was evaluated by the investigator according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) (Version 4.0) where Grade 1 = Mild, Grade 2 = Moderate, Grade 3 = Severe, Grade 4 = Life-threatening and Grade 5 = Death.

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.

The FACT-G and FACT-An score are summed to form the FACT-An total score. The FACT-An Trial Outcome Index (TOI) consists of the summation of a "summary scale" and includes the Physical Well-being, (PWB; 7 items; score range, 0-28), the Functional Well-being (7 items; score range, 0-28) and the Anemia subscale consisting of 20 items on the same five-point scale, with 13 of them measuring fatigue related symptoms (FS) and seven measuring non-FS. The FACT-An TOI has been demonstrated to be a sensitive indicator of clinical outcomes in a number of diseases including MDS. The Fact-TOI score ranges from 0 to 136. Higher scores on all scales of the Fact-An and subscales on the FACT-TOI reflect better quality of life or fewer symptoms.

The FACT-An is a 47-item, cancer-specific questionnaire consisting of a core 27-item general questionnaire (i.e., the Functional Assessment of Cancer Therapy-General \[FACT-G\]) The FACT-G measures the 4 domains on a 5-point scale ranging from 0 (not at all) to 4 (very much). The 4 domains are:

* Physical Well-being (PWB; 7 items; score range, 0-28),
* Social/Family Well-being (SWB; 7 items; score range, 0-28),
* Emotional Well-being (EWB; 6 items; score range, 0-24), and
* Functional Well-being (7 items; score range, 0-28). The FACT-G is a summation composed of a "summary scale" including the PWB, SWB, EWB and FWB. The FACT-G score range is from 0 to 108. For all summary scales including FACT-G, a higher score indicates better HRQoL or lower level of symptoms.

The FACT-G and the anemia subscale (AnS) are summed to form the FACT-An total score and the total score ranges from 0 to 188. The FACT-G measures the 4 domains on a 5-point scale ranging from 0 (not at all) to 4 (very much). The 4 domains are:

* Physical Well-being (PWB; 7 items; score range, 0-28),
* Social/Family Well-being (SWB; 7 items; score range, 0-28),
* Emotional Well-being (EWB; 6 items; score range, 0-24), and
* Functional Well-being (7 items; score range, 0-28). The AnS consists of 20 items on the same 5-point scale, with 13 of them measuring fatigue-related symptoms (FS) and 7 measuring non-FS. The AnS and FS scores can range from 0-80 and 0-52, respectively. For all domains and summary subscales, a higher score indicates better HRQoL or lower level of symptoms.

A clinically meaningful improvement or deterioration was defined by domain specific thresholds of change from baseline. The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being, social/family, emotional well being and Functional Well-Being and an additional 20-item anemia questionnaire that measures fatigue and 7 non-fatigue items. The scales are formatted on 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 HRQoL measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various 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 = the poorest QOL and 100 = the highest QOL.

The distribution (frequency and percentage) of the observed responses (i.e., "Not at all (0)," "A little bit (1)," "Somewhat (2)," "Quite a bit (3)," "Very much (4)," and missing) to Item GP-5 ("I am bothered by side effects of treatment" in the past seven days) of the FACT-An at each scheduled visit were summarized for each treatment group. The denominator for the percentage calculation per treatment group was based on the number of the FACT-An evaluable population at baseline. The distribution of change in responses (improved \[i.e., change score from 1 to 4\], no change \[0\], worsened by one level \[-1\], worsened by ≥2 levels \[-2 to -4\], and missing) from baseline at each post-baseline scheduled visit were summarized by treatment group.

The EQ-5D-3L is a generic, self-administered questionnaire that consists of 5 dimensions: mobility, self-care, pain, usual activities, and anxiety/depression. Each dimension has 3 levels of severity corresponding to no problems, some problems, and extreme problems. It also includes a Visual Analog Scale that recorded the respondent's self-rated health on a vertical, 0-100 scale, where 100 = Best imaginable health state and 0 = Worst imaginable health state. Distribution of the observed responses (i.e., no problems, moderate problems, severe problems, and missing) of the 5 dimensions at each visit was summarized per arm. The denominator for the percentage calculation per group was based on the number of the EQ-5D-3L evaluable population at baseline. The distribution of change in responses (i.e., improved \[by ≥1 level\], no change, worsened \[by ≥1 level\], and missing) from baseline are reported.

TThe EQ-5D-3L is a generic, self-administered questionnaire that consists of 5 dimensions: mobility, self-care, pain, usual activities, and anxiety/depression. Each dimension has 3 levels of severity corresponding to no problems, some problems, and extreme problems. It also includes a Visual Analog Scale that recorded the respondent's self-rated health on a vertical, 0-100 scale, where 100 = Best imaginable health state and 0 = Worst imaginable health state. Distribution of the observed responses (i.e., no problems, moderate problems, severe problems, and missing) of the 5 dimensions at each visit was summarized per arm. The denominator for the percentage calculation per group was based on the number of the EQ-5D-3L evaluable population at baseline. The distribution of change in responses (i.e., improved \[by ≥1 level\], no change, worsened \[by ≥1 level\], and missing) from baseline are reported.

The number of reasons for hospitalizations and hospital admissions during the treatment period were monitored and include those associated with: AEs, protocol-driven procedures, transfusions, non-protocol procedures, elective procedures or those associated with social, practical or technical reasons in the absence of AEs. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient.

The total number of days hospitalized due to any reason during the treatment period was monitored. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient.

The number of days hospitalized per total patient years. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient.

Celgene

Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo

Participants received 300 mg oral azacitidine tablets daily (QD) on days 1 to 21 of each 28-day treatment cycle and best supportive care (BSC) which included and was not limited to packed RBC (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.

Oral Azacitidine 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.

Placebo Plus Best Supportive Care

Total

Age, Continuous

Sex: Female, Male

Race/Ethnicity, Customized

The WHO classification recognizes eight subtypes of MDS that are distinguished by the percentage of myeloblasts, presence or absence of ringed sideroblasts (i.e., erythroid precursors with iron deposits surrounding the nucleus), presence of a monocytosis or a deletion 5q.

Myelodysplastic Syndrome (MDS) World Health Organization (WHO) 2008 Classification

The international prognostic scoring system (IPSS) is a standard for risk assessment in primary myelodysplastic syndromes (MDS) that categorizes prognoses taking into account cytogenetics, cytopenias, blasts and blood counts. The IPSS prognostic subgroups consist of low-, intermediate-1-, intermediate-2-, and high-risk groups. The scale is 0-3.5 at 0.5 increments. Scores of 0=Low; 0.5-1.0=Int-1; 1.5-2.0=Int-2; 2.5-3.5=High risk which corresponds to poorer prognosis.

International Prognostic Scoring System (IPSS)

The average red blood cell (RBC) transfusion units per 28 days is derived using transfusion records before randomization date to randomization date - 84 days (if enrolled under original protocol or protocol amendment 1), or to randomization date - 56 days (if enrolled under protocol 2).

Average Red Blood Cell Transfusion Requirement (units per 28 days)

Hemoglobin

Platelet Count

Participants with thrombocytopenia were defined by 2 platelet counts that were ≤ 75 × 10\^9/cells/L with a platelet measurement ≥ 21 days apart. For those who were platelet transfusion-dependent at baseline and did not achieve platelet transfusion independence (TI) ≥ 56 days (8 weeks) during study treatment were considered as non-responders. For participants who were not platelet transfusion-dependent at baseline, development of platelet transfusion dependence, ie, ≥ 2 platelet transfusions in any 56-day (8 week) period during study treatment and were considered worse outcome.

Platelet Transfusion Status

ECOG performance status is used to describe a patient's level of functioning in terms of their ability to care for themselves, daily activity, and physical ability (walking, working, etc.). The scale ranges from 0 to 5: 0 = Fully active, no restrictions; 1 = Restricted activity but ambulatory, able to carry out work of a light nature; 2 = Ambulatory and capable of all self-care but unable to carry out work activities; 3 = Capable to only limited self-care, confined to bed or chair more than 50% of waking hours; 4 = Completely disabled, no self-care, confined to bed or chair; 5 = Dead.

Eastern Cooperative Oncology Group (ECOG) Performance Status

The intent-to-treat (ITT) population included all participants who were randomized, regardless of whether they received treatment or not.

Anne McClain, Senior Manager

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.

Oral Azacitidine and Best Supportive Care

Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for ≥ 56 Days

Intent to Treat population. Participants who achieved RBC transfusion independence of ≥ 56 days on treatment.

Duration of RBC Transfusion Independence Among Participants Who Achieved RBC Transfusion Independence for at Least 56 Days

Participants who achieved a 56-day TI response. Responders in the intent to treat population.

Time to RBC Transfusion Independence for at Least 56 Days Among Participants Who Achieved RBC Transfusion Independence for at Least 56 Days

Intent to Treat population; includes participants who achieved RBC transfusion reduction of at least 4 units for at least 8 weeks.

Duration of RBC Transfusion Reduction for Participants Who Achieved RBC Transfusion Reduction of at Least 4 Units of RBCs for at Least 8 Weeks

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

Percentage of Participants Who Achieved Red Blood Cell Transfusion Independence for ≥ 84 Days

Intent to Treat population. Participants who achieved RBC transfusion independence for at least 84 days on treatment.

Duration of RBC Transfusion Independence Among Participants Who Achieved RBC Transfusion Independence for at Least 84 Days

Participants who achieved a 84-day TI response. Responders in the intent to treat population.

Time to RBC Transfusion Independence for at Least 84 Days Among Participants Who Achieved RBC Transfusion Independence for at Least 84 Days

HI-E Response

≥ 1.5 g/dL Hemoglobin Increase

RBC Transfusion Reduction

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

Percentage of Participants With an Erythroid Hematological Improvement (HI-E) Response According to 2006 IWG Criteria

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

ITT population; includes participants were were platelet transfusion dependent.

Percentage of Participants Who Achieved Platelet Transfusion Independence With a Duration of ≥ 8 Weeks (56 Days)

Intent to Treat population; includes participants who achieved platelet transfusion independence for at least 56 days.

Time to Platelet Transfusion Independence

Kaplan-Meier Estimate of Overall Survival (OS)

Complete Response (CR)

Partial Response

Marrow CR

Stable Disease (SD)

Disease Progression

Failure due to Death

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

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

Percentage of Participants Who Progressed to Acute Myeloid Leukemia (AML)

The intent-to-treat (ITT) population who progressed to AML.

Time to Progression to Acute Myeloid Leukemia (AML) Among Participants Who Progressed to AML

Percentage of Participants With Significant Bleeding Events

≥ 1 TEAE

≥ 1 TEAE Related to Study Drug

≥ 1 Serious TEAE

≥ 1 Serious TEAE Related to Study Drug

≥ 1 Grade (GR) 3-4 TEAE

≥ 1 Grade 3-4 TEAE Related to Study Drug

≥ 1 Grade (GR) 3-4 Serious TEAE

≥ 1 GR 3-4 Serious TEAE Related to Study Drug

≥ 1 TEAE Leading to Death

≥ 1 TEAE Related to Study Drug Leading to Death

≥ 1 TEAE Leading to Dose Reduction

≥ 1 TEAE Leading to Dose Interruption

≥ 1 TEAE Leading to Dose Interruption/Reduction

≥ 1 TEAE Leading to Treatment Discontinuation

The safety population includes all randomized participants who received at least one dose of study drug.

Number of Participants With Treatment Emergent Adverse Events (TEAE)

The 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.

Mean Change From Baseline in the Physical Well-Being Component of the Functional Assessment of Cancer Therapy-Anemia (FACT-An) Endpoints at Cycle 6

Mean Change From Baseline in the Social Well-Being Component of the Functional Assessment of Cancer Therapy-Anemia Instrument at Cycle 6

Mean Change From Baseline in the Emotional Well-Being Component of the Functional Assessment of Cancer Therapy-Anemia Instrument at Cycle 6

The FACT-Anemia evaluable population was defined as all ITT participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

Mean Change From Baseline in the Functional Well-Being Component of the FACT-An Instrument at Cycle 6

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.

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

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

Mean Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia Trial Outcome Index (FACT-An TOI) Summary Scale Within the FACT-An Instrument at Cycle 6

Mean Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia-General (FACT-G) Summary Scale Within the FACT-An Instrument at Cycle 6

Mean Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia-Total Score at Cycle 6

The common odds ratio and the 95% confidence interval (CI) were calculated using CMH tests, stratified by randomization stratification factors, to compare the odds of experiencing clinically meaningful improvement between CC-486 and placebo.

Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Physical Well-Being Domain Within the FACT-An Instrument at Cycle 6

Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Social Well-Being Domain Within the FACT-An Instrument at Cycle 6

Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Emotional Well-Being Domain Within the FACT-An Instrument at Cycle 6

Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Functional Well-Being Domain Within the FACT-An Instrument at Cycle 6

Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Anemia Subscale Domain Within the FACT-An Instrument at Cycle 6

Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline in the Fatigue Related Symptoms Subscale Domain Within the FACT-An Instrument at Cycle 6

Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline in the Functional Assessment of Cancer Therapy-Anemia Trial Outcome Index Subscale Domain Within the FACT-An Instrument at Cycle 6

The HRQoL evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score baseline visit and at least one post-baseline assessment visit.

Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline in the Functional Assessment of Cancer Therapy-Anemia-General Subscale Domain Within the FACT-An Instrument at Cycle 6

Percentage of Participants With a Clinically Meaningful Improvement (CMI) From Baseline in the Functional Assessment of Cancer Therapy Anemia-Total Score Domain Within the FACT-An Instrument at Cycle 6

Improved

No Change

Worsened by 1 Level

Worsened by 2 Levels

Missing

Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5

Worsened

The HRQoL evaluable population was defined as participants with a EQ-5D-3L health utility at baseline and at least one post-baseline assessment visit.

Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level (EQ-5D-3L) Mobility Dimension Responses at Cycle 6

Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level of Self-Care Dimension Responses at Cycle 6

Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level Usual Activities Dimension Responses at Cycle 6

Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Pain/Discomfort Dimension Responses at Cycle 6

Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Anxiety/Depression Dimension Responses at Cycle 6

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
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)	NCT01566695	Myelodysplastic...	Oral Azacitidin... Placebo Best Supportiv ...	18 Years -	Celgene
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)	NCT01566695	Myelodysplastic...	Oral Azacitidin... Placebo Best Supportiv ...	18 Years -	Celgene

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