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NCT01566695 · ADVERSE EVENTS

Adverse events

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.

Adverse events occurred during the study; they are not necessarily caused by treatment. Serious events are not interchangeable with severe/grade 3–4 events. These safety populations may differ from efficacy populations.

Collection time frame
Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months)
Other-event reporting threshold
5%

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

Participants affected / participants at risk, as reported
Safety groupSerious eventsOther eventsDeaths
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.

83 / 107107 / 10783 / 107
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.

69 / 109104 / 10986 / 109

Group identifiers: EG000: Oral Azacitidine and Best Supportive Care · EG001: Placebo Plus Best Supportive Care

Serious adverse events

Event terms and counts are source-reported; expand to read more. A reporting threshold may omit less frequent events.

  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      7
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      5
      num At Risk
      109
    term
    Anaemia
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Blood loss anaemia
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      2
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Bone marrow failure
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      29
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      9
      num At Risk
      109
    term
    Febrile neutropenia
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Haemolytic anaemia
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      2
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Neutropenia
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Pancytopenia
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      5
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Thrombocytopenia
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Acute coronary syndrome
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Acute myocardial infarction
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Angina pectoris
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Angina unstable

Other reported adverse events

Event terms and counts are source-reported; expand to read more. A reporting threshold may omit less frequent events.

  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      23
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      17
      num At Risk
      109
    term
    Anaemia
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      10
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      3
      num At Risk
      109
    term
    Leukopenia
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      52
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      16
      num At Risk
      109
    term
    Neutropenia
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      30
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      18
      num At Risk
      109
    term
    Thrombocytopenia
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      7
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      109
    term
    Atrial fibrillation
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      6
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Cardiac failure
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      16
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      14
      num At Risk
      109
    term
    Abdominal pain
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      51
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      24
      num At Risk
      109
    term
    Constipation
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      73
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      26
      num At Risk
      109
    term
    Diarrhoea
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      6
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Gastrooesophageal reflux disease
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      7
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      4
      num At Risk
      109
    term
    Gingival bleeding
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      4
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      6
      num At Risk
      109
    term
    Haemorrhoids
Complete source fields
description
Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
event Groups
  1. deaths Num Affected
    83
    deaths Num At Risk
    107
    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
    EG000
    other Num Affected
    107
    other Num At Risk
    107
    serious Num Affected
    83
    serious Num At Risk
    107
    title
    Oral Azacitidine and Best Supportive Care
  2. deaths Num Affected
    86
    deaths Num At Risk
    109
    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
    EG001
    other Num Affected
    104
    other Num At Risk
    109
    serious Num Affected
    69
    serious Num At Risk
    109
    title
    Placebo Plus Best Supportive Care
frequency Threshold
5
other Events
  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      23
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      17
      num At Risk
      109
    term
    Anaemia
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      10
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      3
      num At Risk
      109
    term
    Leukopenia
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      52
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      16
      num At Risk
      109
    term
    Neutropenia
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      30
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      18
      num At Risk
      109
    term
    Thrombocytopenia
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      7
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      109
    term
    Atrial fibrillation
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      6
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Cardiac failure
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      16
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      14
      num At Risk
      109
    term
    Abdominal pain
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      51
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      24
      num At Risk
      109
    term
    Constipation
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      73
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      26
      num At Risk
      109
    term
    Diarrhoea
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      6
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Gastrooesophageal reflux disease
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      7
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      4
      num At Risk
      109
    term
    Gingival bleeding
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      4
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      6
      num At Risk
      109
    term
    Haemorrhoids
serious Events
  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      7
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      5
      num At Risk
      109
    term
    Anaemia
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Blood loss anaemia
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      2
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Bone marrow failure
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      29
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      9
      num At Risk
      109
    term
    Febrile neutropenia
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Haemolytic anaemia
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      2
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Neutropenia
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Pancytopenia
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      5
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Thrombocytopenia
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Acute coronary syndrome
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Acute myocardial infarction
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Angina pectoris
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    26.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      107
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Angina unstable
time Frame
Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months)

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