Trial Outcomes & Findings for A Phase 3, Multicenter, Randomized, Double-blind,Active-controlled, Parallel-group Trial With an Open-labelExtension Phase to Evaluate the Efficacy and Safety of OralE5501 Versus Eltrombopag, in Adults With Chronic ImmuneThrombocytopenia (Idiopathic Thrombocytopenic Purpura) (NCT NCT01433978)

Last Updated: 2018-02-06

Results Overview

Platelet responses to avatrombopag was evaluated using the platelet counts determined at local clinical laboratories. Only participants with non-missing data at both baseline and the relevant post-baseline visit are included in the change from baseline summary statistics. Standard deviation is not applicable for some of the categories, from Visit 14 to Visit 22, as the number of participants analyzed for that visit was 1 individual.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

24 participants

Primary outcome timeframe

Day 5, Day 8, Week 2, Week 3, Week 4, Week 6, Week 8, Week 10, Week 12, Week 14, Week 16, Week 18, Week 19, Week 20, Week 22, Week 23, Week 24, Week 25, Week 26

Results posted on

2018-02-06

Participant Flow

One screen-failed participant was randomized into the study in error, but not dosed.

Participant milestones

Participant milestones
Measure	Eltrombopag (Core Study) Eltrombopag was administered orally as 25 mg, 50 mg, or 75 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 50 mg eltrombopag once daily and they were allowed to have their dose titrated up (maximum dose of 75 mg eltrombopag) or down (minimum dose of 25 mg eltrombopag) depending on their response to study drug.	Avatrombopag (Core Study) Avatrombopag was administered orally as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg avatrombopag, once daily and they were allowed to have their dose titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on their response to study drug.	Avatrombopag (Open-label Extension Phase) Participants who met the eligibility requirements for the Open-label Extension (OLE) Phase or who discontinued the Core Study early because of lack of treatment effect were eligible to continue into the OLE Phase for up to 104 weeks of open-label avatrombopag therapy. Participants entering the OLE from the Core Study received a starting dose of open-label avatrombopag that was determined by the last dose of study drug at the End of Treatment (EOT) Visit (Visit 22) of the Core Study. Participants who discontinued the Core Study early because of lack of treatment effect and entered the OLE received open-label avatrombopag at a starting dose of 20 mg once daily of open-label avatrombopag.
Core Study STARTED	11	12	0
Core Study COMPLETED	0	1	0
Core Study NOT COMPLETED	11	11	0
Extension Phase STARTED	0	0	6
Extension Phase COMPLETED	0	0	0
Extension Phase NOT COMPLETED	0	0	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Eltrombopag (Core Study) Eltrombopag was administered orally as 25 mg, 50 mg, or 75 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 50 mg eltrombopag once daily and they were allowed to have their dose titrated up (maximum dose of 75 mg eltrombopag) or down (minimum dose of 25 mg eltrombopag) depending on their response to study drug.	Avatrombopag (Core Study) Avatrombopag was administered orally as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg avatrombopag, once daily and they were allowed to have their dose titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on their response to study drug.	Avatrombopag (Open-label Extension Phase) Participants who met the eligibility requirements for the Open-label Extension (OLE) Phase or who discontinued the Core Study early because of lack of treatment effect were eligible to continue into the OLE Phase for up to 104 weeks of open-label avatrombopag therapy. Participants entering the OLE from the Core Study received a starting dose of open-label avatrombopag that was determined by the last dose of study drug at the End of Treatment (EOT) Visit (Visit 22) of the Core Study. Participants who discontinued the Core Study early because of lack of treatment effect and entered the OLE received open-label avatrombopag at a starting dose of 20 mg once daily of open-label avatrombopag.
Core Study Adverse Event, Non-Fatal	0	1	0
Core Study Lack of Efficacy	5	1	0
Core Study Study Terminated by Sponsor	6	9	0
Extension Phase Adverse Event, Non-Fatal	0	0	1
Extension Phase Lack of Efficacy	0	0	1
Extension Phase Study Terminated by Sponsor	0	0	4

Baseline Characteristics

A Phase 3, Multicenter, Randomized, Double-blind,Active-controlled, Parallel-group Trial With an Open-labelExtension Phase to Evaluate the Efficacy and Safety of OralE5501 Versus Eltrombopag, in Adults With Chronic ImmuneThrombocytopenia (Idiopathic Thrombocytopenic Purpura)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eltrombopag (Core Study) n=11 Participants Eltrombopag was administered orally as 25 mg, 50 mg, or 75 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 50 mg eltrombopag once daily and they were allowed to have their dose titrated up (maximum dose of 75 mg eltrombopag) or down (minimum dose of 25 mg eltrombopag) depending on their response to study drug.	Avatrombopag (Core Study) n=12 Participants Avatrombopag was administered orally as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg avatrombopag, once daily and they were allowed to have their dose titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on their response to study drug.	Total n=23 Participants Total of all reporting groups
Age, Continuous	45.4 Years STANDARD_DEVIATION 20.09 • n=5 Participants	50.8 Years STANDARD_DEVIATION 23.04 • n=7 Participants	48.2 Years STANDARD_DEVIATION 21.36 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	7 Participants n=7 Participants	14 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	5 Participants n=7 Participants	9 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 5, Day 8, Week 2, Week 3, Week 4, Week 6, Week 8, Week 10, Week 12, Week 14, Week 16, Week 18, Week 19, Week 20, Week 22, Week 23, Week 24, Week 25, Week 26

Population: The Full Analysis Set (FAS) included all participants who were randomized into the study. Only participants with non-missing data at both baseline and the relevant post-baseline visit are included in the change from baseline summary statistics.

Outcome measures

Outcome measures
Measure	Eltrombopag (Core Study) n=11 Participants Eltrombopag was administered orally as 25 mg, 50 mg, or 75 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 50 mg eltrombopag once daily and they were allowed to have their dose titrated up (maximum dose of 75 mg eltrombopag) or down (minimum dose of 25 mg eltrombopag) depending on their response to study drug.	Avatrombopag (Core Study) n=12 Participants Avatrombopag was administered orally as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg avatrombopag, once daily and they were allowed to have their dose titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on their response to study drug.
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 3 (Day 5)	8.60 cells x 10^9/L Standard Deviation 17.312	12.85 cells x 10^9/L Standard Deviation 16.054
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 4 (Day 8)	32.50 cells x 10^9/L Standard Deviation 44.519	47.00 cells x 10^9/L Standard Deviation 59.690
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 5 (Week 2)	73.41 cells x 10^9/L Standard Deviation 79.885	171.71 cells x 10^9/L Standard Deviation 201.736
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 6 (Week 3)	67.20 cells x 10^9/L Standard Deviation 95.536	114.21 cells x 10^9/L Standard Deviation 117.172
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 7 (Week 4)	28.72 cells x 10^9/L Standard Deviation 38.437	108.79 cells x 10^9/L Standard Deviation 217.036
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 8 (Week 6)	57.21 cells x 10^9/L Standard Deviation 57.718	150.68 cells x 10^9/L Standard Deviation 134.902
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 9 (Week 8)	67.25 cells x 10^9/L Standard Deviation 46.055	121.31 cells x 10^9/L Standard Deviation 149.040
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 10 (Week 10)	92.67 cells x 10^9/L Standard Deviation 34.649	126.25 cells x 10^9/L Standard Deviation 90.602
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 11 (Week 12)	87.33 cells x 10^9/L Standard Deviation 72.616	185.10 cells x 10^9/L Standard Deviation 115.841
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 12 (Week 14 )	104.33 cells x 10^9/L Standard Deviation 77.114	159.38 cells x 10^9/L Standard Deviation 116.746
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 13 (Week 16)	47.75 cells x 10^9/L Standard Deviation 2.475	123.88 cells x 10^9/L Standard Deviation 124.474
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 14 (Week 18)	107.50 cells x 10^9/L Standard Deviation NA Standard deviation is not applicable.	46.50 cells x 10^9/L Standard Deviation 53.740
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 15 (Week 19)	13.50 cells x 10^9/L Standard Deviation NA Standard deviation is not applicable.	29.50 cells x 10^9/L Standard Deviation 22.627
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 16 (Week 20)	—	50.50 cells x 10^9/L Standard Deviation NA Standard deviation is not applicable.
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 18 (Week 22)	—	75.50 cells x 10^9/L Standard Deviation NA Standard deviation is not applicable.
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 19 (Week 23)	—	104.50 cells x 10^9/L Standard Deviation NA Standard deviation is not applicable.
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 20 (Week 24)	—	106.50 cells x 10^9/L Standard Deviation NA Standard deviation is not applicable.
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 21 (Week 25)	—	120.50 cells x 10^9/L Standard Deviation NA Standard deviation is not applicable.
Change From Baseline in Local Platelet Count for the 6 Month Treatment Period Visit 22 (Week 26)	—	58.50 cells x 10^9/L Standard Deviation NA Standard deviation is not applicable.

Adverse Events

Eltrombopag (Core Study)

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Avatrombopag (Core Study)

Serious events: 2 serious events

Other events: 11 other events

Deaths: 0 deaths

Avatrombopag (Extension Phase)

Serious events: 4 serious events

Other events: 16 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Eltrombopag (Core Study) n=11 participants at risk Eltrombopag was administered orally as 25 mg, 50 mg, or 75 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 50 mg eltrombopag once daily and they were allowed to have their dose titrated up (maximum dose of 75 mg eltrombopag) or down (minimum dose of 25 mg eltrombopag) depending on their response to study drug.	Avatrombopag (Core Study) n=12 participants at risk Avatrombopag was administered orally as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg avatrombopag, once daily and they were allowed to have their dose titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on their response to study drug.	Avatrombopag (Extension Phase) n=17 participants at risk Participants who met the eligibility requirements for the Open-label Extension (OLE) Phase or who discontinued the Core Study early because of lack of treatment effect were eligible to continue into the OLE Phase for up to 104 weeks of open-label avatrombopag therapy. Participants entering the OLE from the Core Study received a starting dose of open-label avatrombopag that was determined by the last dose of study drug at the End of Treatment (EOT) Visit (Visit 22) of the Core Study. Participants who discontinued the Core Study early because of lack of treatment effect and entered the OLE received open-label avatrombopag at a starting dose of 20 mg once daily of open-label avatrombopag.
Blood and lymphatic system disorders Idiopathic thrombocytopenic purpura	0.00% 0/11 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	8.3% 1/12 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	5.9% 1/17 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.
Hepatobiliary disorders Portal vein thrombosis	0.00% 0/11 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	8.3% 1/12 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	5.9% 1/17 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.
Infections and infestations Bronchitis Moraxella	0.00% 0/11 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	8.3% 1/12 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	5.9% 1/17 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.
Infections and infestations Influenza	0.00% 0/11 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	0.00% 0/12 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	5.9% 1/17 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.
Infections and infestations Thrombophlebitis Septic	0.00% 0/11 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	8.3% 1/12 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	5.9% 1/17 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Chronic lymphotic leukaemia	0.00% 0/11 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	0.00% 0/12 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	5.9% 1/17 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	0.00% 0/11 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	8.3% 1/12 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	5.9% 1/17 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.
Respiratory, thoracic and mediastinal disorders Pneumothorax	0.00% 0/11 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	8.3% 1/12 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.	5.9% 1/17 • Up to 34 Weeks in the Core Study (including Titration, Treatment, Dose Taper, and Follow-up for those that did not enter the Extension Phase) and up to 112 weeks in the Extension Phase (including Conversion, Maintenance, Dose Taper, and Follow-up). Treatment emergent adverse events were collected. Core Study Safety Analysis Set (SAS): All participants who received at least 1 dose of study drug and had at least 1 postdose safety assessment. OLE SAS: All participants who received at least 1 dose of avatrombopag (either in the Core or Extension Phase) and had a postdose safety assessment.

Other adverse events

Additional Information

Eisai Inc.

Eisai Call Center

Phone: 888-422-4743

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER