Trial Outcomes & Findings for A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS) (NCT NCT00961064)

NCT ID: NCT00961064

Last Updated: 2023-11-14

Results Overview

The primary endpoint was hematologic response at 16 or 20 weeks, defined as either: (1) an increase in platelet counts =20.000/uL or transfusion independence for a minimum of 8 weeks; (2) hemoglobin (Hb) increase of =1.5g/dL from baseline, or a reduction in red blood cells (RBC) transfusion of at least 50%; or (3) an increase in absolute neutrophil counts (ANC) of =0.5x109/L or by at least 100% in patients with a baseline ANC \<0.5x109/L.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 30 participants
• Primary outcome timeframe: 16 - 20 weeks
• Results posted on: 2023-11-14

Participant Flow

Participant milestones

Measure	Eltrombopag in Low to Int-2 Risk Myelodysplastic Syndrome (MDS) Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.
Initial Treatment (Weeks 1 - 20) STARTED	30
Initial Treatment (Weeks 1 - 20) COMPLETED	14
Initial Treatment (Weeks 1 - 20) NOT COMPLETED	16
Extended Access: Week 20 to Up to Year 5 STARTED	14
Extended Access: Week 20 to Up to Year 5 COMPLETED	7
Extended Access: Week 20 to Up to Year 5 NOT COMPLETED	7

Reasons for withdrawal

Measure	Eltrombopag in Low to Int-2 Risk Myelodysplastic Syndrome (MDS) Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.
Initial Treatment (Weeks 1 - 20) No Response	11
Initial Treatment (Weeks 1 - 20) Disease Progression	3
Initial Treatment (Weeks 1 - 20) Death	1
Initial Treatment (Weeks 1 - 20) Withdrawal by Subject	1
Extended Access: Week 20 to Up to Year 5 Disease Progression	3
Extended Access: Week 20 to Up to Year 5 Lost Response	1
Extended Access: Week 20 to Up to Year 5 Non-compliant	1
Extended Access: Week 20 to Up to Year 5 Withdrawal by Subject	2

Baseline Characteristics

A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)

Baseline characteristics by cohort

Measure	Eltrombopag in Low to Int-2 Risk Myelodysplastic Syndromes (MDS) n=30 Participants Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	15 Participants n=5 Participants
Age, Categorical >=65 years	15 Participants n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	21 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	29 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	4 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	19 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Low Risk Myelodysplastic Syndromes (MDS)	2 Participants n=5 Participants
Intermediate-1 Risk Myelodysplastic Syndromes (MDS)	27 Participants n=5 Participants
Intermediate-2 Risk Myelodysplastic Syndromes (MDS)	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 16 - 20 weeks

Population: The first 5 subjects enrolled were not counted toward the accrual target due to changes in protocol.

The primary endpoint was hematologic response at 16 or 20 weeks, defined as either: (1) an increase in platelet counts =20.000/uL or transfusion independence for a minimum of 8 weeks; (2) hemoglobin (Hb) increase of =1.5g/dL from baseline, or a reduction in red blood cells (RBC) transfusion of at least 50%; or (3) an increase in absolute neutrophil counts (ANC) of =0.5x109/L or by at least 100% in patients with a baseline ANC <0.5x109/L.

Outcome measures

Measure	Eltrombopag in Low to Int-1 Risk Myelodysplastic Syndrome (MDS) n=25 Participants Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.
Number of Participants With Response Between Weeks 16 and 20	11 Participants

SECONDARY outcome

Timeframe: 16 - 20 weeks

Changes in serum thrombopoietin level at 16 or 20 weeks

Outcome measures

Measure	Eltrombopag in Low to Int-1 Risk Myelodysplastic Syndrome (MDS) n=30 Participants Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.
Changes in Serum Thrombopoietin Level	2080 pg/mL Interval 71.0 to 4817.0

SECONDARY outcome

Timeframe: up to 5 years

Population: intention to treat analysis

Number of participants with progression to higher risk Myelodysplastic syndrome (MDS) as measured by International Working Group (IWG) 2006 criteria in participants With Low to Int-2 Risk Myelodysplastic Syndrome (MDS) following Eltrombopag.

Progression of disease per modified IWG criteria 2006 were considered: if patients with baseline bone marrow blasts of < 5% increase by ≥ 50% to >5% blasts; and either at least 50% decrease in granulocytes or platelets from maximum response, decreasing in Hb by 2g/dL or transfusion dependence. The acquisition of new cytogenetic abnormality without meeting other IWG criteria for progression was not considered disease progression.

Outcome measures

Measure	Eltrombopag in Low to Int-1 Risk Myelodysplastic Syndrome (MDS) n=30 Participants Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.
Number of Participants With Progression to Higher Risk Myelodysplastic Syndrome as Measured by International Working Group Criteria	6 Participants

SECONDARY outcome

Timeframe: up to 5 years

Population: Analysis included those participants that participated in extended access portion of study

Number of participants who achieved a robust response and discontinued Eltrombopag in extended access

A robust response is defined as: as platelets >50,000/μL, hemoglobin > 10 g/dL in the absence of transfusions, and neutrophils > 1,000 for more than 8 weeks.

Outcome measures

Measure	Eltrombopag in Low to Int-1 Risk Myelodysplastic Syndrome (MDS) n=14 Participants Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.
Number of Participants Who Achieved a Robust Response and Discontinued Eltrombopag	10 Participants

SECONDARY outcome

Timeframe: up to 5 years

Number of Participants with Grade 2 or Higher Bleeding Events defined by Common Terminology Criteria for Adverse Events (CTCAE) v. 4.0.

The NCI CTCAE is a descriptive terminology which can be utilized for Adverse Event (AE) reporting. A grading (severity) scale is provided for each AE term. The CTCAE displays Grades 1 through 5 with unique clinical descriptions of severity for each AE based on this general guideline. Grade 1 Mild is asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 is moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living (ADL)*. Grade 3 is severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL**. Grade 4 if life-threatening consequences; urgent intervention indicated. Grade 5 is death related to AE.

Outcome measures

Measure	Eltrombopag in Low to Int-1 Risk Myelodysplastic Syndrome (MDS) n=30 Participants Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.
Number of Participants With Grade 2 or Higher Bleeding Events Defined by Common Terminology Criteria for Adverse Events v. 4.0	5 Participants

Adverse Events

Eltrombopag in Low to Int-2 Risk Myelodysplastic Syndrome (MDS)

Serious events: 12 serious events

Other events: 28 other events

Deaths: 1 deaths

Serious adverse events

Measure	Eltrombopag in Low to Int-2 Risk Myelodysplastic Syndrome (MDS) n=30 participants at risk Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.
General disorders Death	3.3% 1/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Injury, poisoning and procedural complications Fall	3.3% 1/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Investigations Investigation	3.3% 1/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Nervous system disorders Nervous system disorder	3.3% 1/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Renal and urinary disorders Bladder mass	3.3% 1/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Surgical and medical procedures Hospitalisation	33.3% 10/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.

Other adverse events

Measure	Eltrombopag in Low to Int-2 Risk Myelodysplastic Syndrome (MDS) n=30 participants at risk Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.
Blood and lymphatic system disorders Febrile neutropenia	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
General disorders Oedema	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Blood and lymphatic system disorders Jaundice	13.3% 4/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Blood and lymphatic system disorders Ocular icterus	13.3% 4/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Blood and lymphatic system disorders Petechiae	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Cardiac disorders Dizziness	23.3% 7/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Cardiac disorders Dyspnoea	20.0% 6/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Cardiac disorders Oedema peripheral	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Eye disorders Blepharospasm	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Eye disorders Vision blurred	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Abdominal discomfort	13.3% 4/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Abdominal pain	16.7% 5/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Constipation	16.7% 5/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Diarrhoea	20.0% 6/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Faeces discoloured	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Flatulence	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Gastrooesophageal reflux disease	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Gingival bleeding	13.3% 4/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Nausea	20.0% 6/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Oropharyngeal pain	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Stomatitis	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Toothache	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Gastrointestinal disorders Vomiting	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
General disorders Asthenia	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
General disorders Chills	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
General disorders Fatigue	36.7% 11/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
General disorders Flank pain	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
General disorders Pain	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
General disorders Pyrexia	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Infections and infestations Bronchitis	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Infections and infestations Sinusitis	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Infections and infestations Upper respiratory tract infection	23.3% 7/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Infections and infestations Urinary tract infection	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Injury, poisoning and procedural complications Contusion	13.3% 4/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Investigations Haemoglobin decreased	30.0% 9/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Investigations Liver function test increased	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Investigations Neutrophil count decreased	23.3% 7/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Investigations Platelet count decreased	23.3% 7/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Musculoskeletal and connective tissue disorders Arthralgia	16.7% 5/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Musculoskeletal and connective tissue disorders Musculoskeletal stiffness	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Musculoskeletal and connective tissue disorders Pain in extremity	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Nervous system disorders Aphasia	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Nervous system disorders Headache	20.0% 6/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Nervous system disorders Insomnia	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Nervous system disorders Paraesthesia	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Psychiatric disorders Anxiety	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Psychiatric disorders Depression	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Renal and urinary disorders Chromaturia	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Renal and urinary disorders Haematuria	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Respiratory, thoracic and mediastinal disorders Cough	16.7% 5/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Skin and subcutaneous tissue disorders Pruritus	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Skin and subcutaneous tissue disorders Rash	13.3% 4/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Skin and subcutaneous tissue disorders Skin discolouration	10.0% 3/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Skin and subcutaneous tissue disorders Skin lesion	16.7% 5/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.
Surgical and medical procedures Sinus operation	6.7% 2/30 • Up to 5 years Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.

Additional Information

Bhavisha Patel, MD

National Heart Lung and Blood Institute

Phone: 301.402.3477

Email: bhavisha.patel@nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place