Trial Outcomes & Findings for A Study to Assess the Ability of Eltrombopag to Induce Sustained Response Off Treatment in Subjects With ITP (NCT NCT03524612)

Results Overview

Sustained response off treatment (SRoT) was defined as: reaching platelet count \>= 100×10\^9/L (complete response \[CR\]) and then maintaining platelet counts around 100×10\^9/L for 2 months (no counts below 70×10\^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count \>= 30×10\^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy until month 12.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

105 participants

Primary outcome timeframe

Month 12

Results posted on

2025-01-07

Participant Flow

This study was conducted in 32 investigative sites in 15 countries:1 (Austria), 3 (Brazil), 2 (Chile), 2 (France), 2 (Greece), 2 (Italy), 1 (Japan), 2 (Mexico), 1 (Oman), 2 (Russia), 7 (Spain), 1 (Switzerland), 3 (Turkey), 1 (UK), 2 (USA).

Participant milestones

Participant milestones
Measure	Eltrombopag Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Overall Study STARTED	105
Overall Study COMPLETED	63
Overall Study NOT COMPLETED	42

Reasons for withdrawal

Reasons for withdrawal
Measure	Eltrombopag Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Overall Study Lack of Efficacy	10
Overall Study Physician Decision (Patient non-compliant)	1
Overall Study Adverse Event	10
Overall Study Withdrawal by Subject	7
Overall Study Patient Decision (Patient Changed Her Address)	1
Overall Study Patient Decision (Personal Reasons)	1
Overall Study New therapy for study indication	6
Overall Study Death	3
Overall Study Lost to Follow-up	2
Overall Study Protocol Violation	1

Baseline Characteristics

A Study to Assess the Ability of Eltrombopag to Induce Sustained Response Off Treatment in Subjects With ITP

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eltrombopag n=105 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Age, Continuous	48.1 Years STANDARD_DEVIATION 19.39 • n=5 Participants
Sex: Female, Male Female	64 Participants n=5 Participants
Sex: Female, Male Male	41 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	5 Participants n=5 Participants
Race (NIH/OMB) Asian	2 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	2 Participants n=5 Participants
Race (NIH/OMB) White	95 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Time since initial diagnosis	366.37 Days STANDARD_DEVIATION 911.420 • n=5 Participants

PRIMARY outcome

Timeframe: Month 12

Population: Full Analysis Set, which included all patients who received at least one dose of eltrombopag.

Sustained response off treatment (SRoT) was defined as: reaching platelet count \>= 100×10\^9/L (complete response \[CR\]) and then maintaining platelet counts around 100×10\^9/L for 2 months (no counts below 70×10\^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count \>= 30×10\^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy until month 12.

Outcome measures

Outcome measures
Measure	Eltrombopag n=105 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Percentage of Participants With Sustained Response Off Treatment (SRoT) by 12 Months	30.5 Percentage of participants Interval 21.9 to 40.2

SECONDARY outcome

Timeframe: From last dose of eltrombopag to month 12

Population: Full Analysis Set

Sustained response off treatment (SRoT) was defined as: reaching platelet count \>= 100×10\^9/L (complete response \[CR\]) and then maintaining platelet counts around 100×10\^9/L for 2 months (no counts below 70×10\^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count \>= 30×10\^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy until month 12.

Outcome measures

Outcome measures
Measure	Eltrombopag n=105 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Median Duration of Sustained Response Off Treatment (SRoT) After Treatment Discontinuation for Participants With Sustained Response Off Treatment	33.3 Weeks Interval 4.0 to 51.0

SECONDARY outcome

Timeframe: From last dose of eltrombopag to relapse, assessed up to month 24

Population: Full Analysis Set. No data collected for this endpoint.

Sustained response off treatment (SRoT) was defined as: reaching platelet count \>= 100×10\^9/L (complete response \[CR\]) and then maintaining platelet counts around 100×10\^9/L for 2 months (no counts below 70×10\^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count \>= 30×10\^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy. Patients with SRoT until month 12 who entered follow-up and did not relapse by cut-off date/Month 24 were censored at the earliest of discontinuation date/death date/Month 24 platelet assessment date/cutoff date. Patients with SRoT until Month 12 patients who did not enter or do not yet have data in follow-up phase are censored at their Month 12 platelet assessment date.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From last dose of eltrombopag to month 24

Population: Full Analysis Set. No data collected for this endpoint.

Patients who tapered and discontinued successfully, or did not relapse/die by cutoff date /month 24 were censored at the earliest of discontinuation date/death date/month 24 platelet assessment date/cutoff date.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Month 15, 18, 21 and 24

Population: Full Analysis Set

Sustained response off treatment is defined as reach platelet count ≥ 100×10\^9/L (complete response \[CR\]) and then maintain platelet counts around 100×10\^9/L for 2 months (no counts below 70×10\^9/L) AND then taper off the drug until treatment discontinuation while, maintain platelet count ≥ 30×10\^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy

Outcome measures

Outcome measures
Measure	Eltrombopag n=105 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Percentage of Participants With Sustained Response Off Treatment Until Month 24 Month 15	21 Participants
Percentage of Participants With Sustained Response Off Treatment Until Month 24 Month 18	18 Participants
Percentage of Participants With Sustained Response Off Treatment Until Month 24 Month 21	14 Participants
Percentage of Participants With Sustained Response Off Treatment Until Month 24 Month 24	15 Participants

SECONDARY outcome

Timeframe: By 1 month

Population: Full Analysis Set

Early response is defined as reaching a platelet count \>= 50×10\^9/L at least once within the first month (month 1) without bleeding events and no rescue therapy.

Outcome measures

Outcome measures
Measure	Eltrombopag n=105 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Percentage of Participants With Early Response Within First Month	76.2 Percentage of participants Interval 66.9 to 84.0

SECONDARY outcome

Timeframe: Up to month 24

Population: Full Analysis Set

Recovery response is defined as platelet count \>=30×10\^9/L after eltrombopag is re-introduced, in case of loss of response (\< 30×10\^9/L and/or bleeding event) without bleeding events and no rescue therapy.

Outcome measures

Outcome measures
Measure	Eltrombopag n=105 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Percentage of Participants With Recovery Response in Case of Loss of Response During or After Tapering of Eltrombopag Until Month 24 Participants with loss of response	11.4 Percentage of participants Interval 6.0 to 19.1
Percentage of Participants With Recovery Response in Case of Loss of Response During or After Tapering of Eltrombopag Until Month 24 Participants with recovery response	6.7 Percentage of participants Interval 2.7 to 13.3

SECONDARY outcome

Timeframe: Baseline, month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

Relative change (%) is the absolute change divided by the platelet counts at baseline and multiplied by 100.

Outcome measures

Outcome measures
Measure	Eltrombopag n=104 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Relative Change From Baseline in Platelet Count Over Time 3 Months	1384.29 Percentage of change Standard Deviation 3383.702
Relative Change From Baseline in Platelet Count Over Time 6 Months	1313.10 Percentage of change Standard Deviation 3459.616
Relative Change From Baseline in Platelet Count Over Time 9 Months	1396.68 Percentage of change Standard Deviation 3238.680
Relative Change From Baseline in Platelet Count Over Time 12 Months	1464.95 Percentage of change Standard Deviation 3509.964
Relative Change From Baseline in Platelet Count Over Time 15 Months	1622.19 Percentage of change Standard Deviation 3712.156
Relative Change From Baseline in Platelet Count Over Time 18 Months	1664.90 Percentage of change Standard Deviation 3718.191
Relative Change From Baseline in Platelet Count Over Time 21 Months	2527.20 Percentage of change Standard Deviation 5098.612
Relative Change From Baseline in Platelet Count Over Time 24 Months	1691.68 Percentage of change Standard Deviation 3611.977
Relative Change From Baseline in Platelet Count Over Time end of treatment visit	1213.91 Percentage of change Standard Deviation 2952.719

SECONDARY outcome

Timeframe: From first time of reaching the level to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set

Platelet counts level is defined as having platelet counts \>=30×10\^9/L without bleeding events and no rescue therapy.

Outcome measures

Outcome measures
Measure	Eltrombopag n=105 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24 maintain platelet counts level from the first time of reaching that level to month 3	49.5 Percentage of participants Interval 39.6 to 59.5
Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24 maintain platelet counts level from the first time of reaching that level to month 6	38.1 Percentage of participants Interval 28.8 to 48.1
Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24 maintain platelet counts level from the first time of reaching that level to month 9	28.6 Percentage of participants Interval 20.2 to 38.2
Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24 maintain platelet counts level from the first time of reaching that level to month 12	28.6 Percentage of participants Interval 20.2 to 38.2
Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24 maintain platelet counts level from the first time of reaching that level to month 15	20.0 Percentage of participants Interval 12.8 to 28.9
Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24 maintain platelet counts level from the first time of reaching that level to month 18	17.1 Percentage of participants Interval 10.5 to 25.7
Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24 maintain platelet counts level from the first time of reaching that level to month 21	13.3 Percentage of participants Interval 7.5 to 21.4
Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24 maintain platelet counts level from the first time of reaching that level to month 24	14.3 Percentage of participants Interval 8.2 to 22.5
Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24 maintain platelet counts level from the first time of reaching that level to end of treatment visit	31.4 Percentage of participants Interval 22.7 to 41.2

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue©) is a 13- item questionnaire that assesses self-reported fatigue and its impact upon daily activities over the past 7 days. FACIT-fatigue is scored using a 4-point Likert scale. Items are scored as follows: 4 = Not At All; 3 = A Little Bit; 2 = Somewhat; 1 = Quite A Bit; 0 = Very Much, EXCEPT items #7 and #8 which are reversed scored. Score range from 0-52. A score of less than 30 indicates severe fatigue. The higher the score, the better the quality of life (less fatigue).

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire 3 Months	5.42 Unit on a scale Standard Deviation 9.700
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire 6 Months	6.15 Unit on a scale Standard Deviation 8.779
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire 9 Months	6.89 Unit on a scale Standard Deviation 9.291
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire 12 Months	7.90 Unit on a scale Standard Deviation 9.064
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire 15 Months	7.12 Unit on a scale Standard Deviation 11.720
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire 18 Months	8.87 Unit on a scale Standard Deviation 11.644
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire 21 Months	8.80 Unit on a scale Standard Deviation 11.112
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire 24 Months	8.69 Unit on a scale Standard Deviation 10.635
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire end of treatment visit	5.40 Unit on a scale Standard Deviation 10.116

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

FACT-Th6 instrument is used to measure worry/concern about bleeding and bruising, and the impact of this worry/concern on physical and social activity (Cella 2006). FACT-Th6 is a 6-item subset of the more detailed FACT-Th, which is an 18-item subscale of the validated FACT that specifically measures concerns related to thrombocytopenia in the past 7 days. The FACT-Th6 is scored using a 5-level Likert scale (0=not at all to 4=very much) and is calculated by summing scores for the 6-items; therefore, scores can range from 0-24, with higher scores representing better HRQoL

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire 3 Months	3.80 Unit on a scale Standard Deviation 5.130
Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire 6 Months	4.35 Unit on a scale Standard Deviation 6.312
Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire 9 Months	5.60 Unit on a scale Standard Deviation 6.114
Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire 12 Months	5.62 Unit on a scale Standard Deviation 6.505
Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire 18 Months	7.04 Unit on a scale Standard Deviation 7.654
Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire 21 Months	7.32 Unit on a scale Standard Deviation 7.351
Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire 24 Months	7.08 Unit on a scale Standard Deviation 7.048
Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire 15 Months	5.44 Unit on a scale Standard Deviation 8.949
Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire end of treatment visit	4.15 Unit on a scale Standard Deviation 6.631

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score 3 Months	7.55 Unit on a scale Standard Deviation 24.199
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score 6 Months	2.75 Unit on a scale Standard Deviation 24.636
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score 9 Months	3.82 Unit on a scale Standard Deviation 21.507
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score 12 Months	7.53 Unit on a scale Standard Deviation 25.547
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score 15 Months	1.96 Unit on a scale Standard Deviation 36.697
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score 18 Months	13.61 Unit on a scale Standard Deviation 26.662
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score 21 Months	3.00 Unit on a scale Standard Deviation 20.967
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score 24 Months	9.27 Unit on a scale Standard Deviation 29.090
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score end of treatment visit	5.19 Unit on a scale Standard Deviation 29.460

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score 3 Months	4.12 Unit on a scale Standard Deviation 18.894
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score 6 Months	5.39 Unit on a scale Standard Deviation 20.646
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score 9 Months	7.40 Unit on a scale Standard Deviation 17.990
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score 12 Months	7.62 Unit on a scale Standard Deviation 18.420
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score 15 Months	11.17 Unit on a scale Standard Deviation 20.470
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score 18 Months	14.78 Unit on a scale Standard Deviation 26.801
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score 21 Months	14.36 Unit on a scale Standard Deviation 25.541
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score 24 Months	13.92 Unit on a scale Standard Deviation 19.577
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score end of treatment visit	3.80 Unit on a scale Standard Deviation 18.122

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score 3 Months	6.74 Unit on a scale Standard Deviation 15.775
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score 6 Months	6.97 Unit on a scale Standard Deviation 18.149
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score 9 Months	9.78 Unit on a scale Standard Deviation 15.074
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score 12 Months	10.26 Unit on a scale Standard Deviation 16.125
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score 15 Months	7.08 Unit on a scale Standard Deviation 22.259
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score 18 Months	15.22 Unit on a scale Standard Deviation 22.334
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score 21 Months	14.32 Unit on a scale Standard Deviation 20.948
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score 24 Months	12.50 Unit on a scale Standard Deviation 17.103
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score end of treatment visit	6.82 Unit on a scale Standard Deviation 17.058

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score 3 Months	8.48 Unit on a scale Standard Deviation 23.842
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score 6 Months	10.90 Unit on a scale Standard Deviation 26.069
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score 9 Months	12.67 Unit on a scale Standard Deviation 28.074
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score 12 Months	14.31 Unit on a scale Standard Deviation 28.767
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score 15 Months	11.87 Unit on a scale Standard Deviation 37.469
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score 18 Months	19.35 Unit on a scale Standard Deviation 33.449
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score 21 Months	24.09 Unit on a scale Standard Deviation 31.983
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score 24 Months	16.54 Unit on a scale Standard Deviation 35.882
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score end of treatment visit	9.00 Unit on a scale Standard Deviation 29.376

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score 3 Months	4.42 Unit on a scale Standard Deviation 22.371
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score 6 Months	6.15 Unit on a scale Standard Deviation 23.270
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score 9 Months	8.89 Unit on a scale Standard Deviation 22.361
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score 12 Months	10.20 Unit on a scale Standard Deviation 23.260
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score 15 Months	4.86 Unit on a scale Standard Deviation 27.022
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score 18 Months	13.04 Unit on a scale Standard Deviation 26.211
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score 21 Months	16.29 Unit on a scale Standard Deviation 25.520
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score 24 Months	16.03 Unit on a scale Standard Deviation 20.128
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score end of treatment visit	7.25 Unit on a scale Standard Deviation 22.078

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score 3 Months	13.26 Unit on a scale Standard Deviation 22.958
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score 6 Months	9.43 Unit on a scale Standard Deviation 27.066
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score 9 Months	17.36 Unit on a scale Standard Deviation 24.165
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score 12 Months	19.94 Unit on a scale Standard Deviation 29.232
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score 15 Months	15.89 Unit on a scale Standard Deviation 37.454
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score 18 Months	22.55 Unit on a scale Standard Deviation 32.680
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score 21 Months	27.27 Unit on a scale Standard Deviation 33.604
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score 24 Months	29.81 Unit on a scale Standard Deviation 32.849
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score end of treatment visit	15.88 Unit on a scale Standard Deviation 33.332

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score 3 Months	6.06 Unit on a scale Standard Deviation 24.143
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score 6 Months	7.17 Unit on a scale Standard Deviation 26.951
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score 9 Months	10.00 Unit on a scale Standard Deviation 22.863
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score 12 Months	16.38 Unit on a scale Standard Deviation 26.408
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score 15 Months	14.58 Unit on a scale Standard Deviation 30.986
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score 18 Months	26.63 Unit on a scale Standard Deviation 24.514
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score 21 Months	20.45 Unit on a scale Standard Deviation 26.318
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score 24 Months	25.96 Unit on a scale Standard Deviation 22.617
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score end of treatment visit	11.18 Unit on a scale Standard Deviation 26.236

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score 3 Months	7.48 Unit on a scale Standard Deviation 18.418
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score 6 Months	6.45 Unit on a scale Standard Deviation 20.696
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score 9 Months	12.78 Unit on a scale Standard Deviation 20.982
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score 12 Months	14.33 Unit on a scale Standard Deviation 22.398
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score 15 Months	8.33 Unit on a scale Standard Deviation 26.237
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score 18 Months	12.23 Unit on a scale Standard Deviation 27.918
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score 21 Months	17.33 Unit on a scale Standard Deviation 26.087
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score 24 Months	11.06 Unit on a scale Standard Deviation 24.705
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score end of treatment visit	8.97 Unit on a scale Standard Deviation 22.503

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score 3 Months	3.46 Unit on a scale Standard Deviation 7.445
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score 6 Months	2.72 Unit on a scale Standard Deviation 8.165
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score 9 Months	3.92 Unit on a scale Standard Deviation 7.608
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score 12 Months	4.85 Unit on a scale Standard Deviation 8.946
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score 15 Months	4.37 Unit on a scale Standard Deviation 11.754
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score 18 Months	6.77 Unit on a scale Standard Deviation 9.950
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score 21 Months	6.65 Unit on a scale Standard Deviation 8.017
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score 24 Months	6.63 Unit on a scale Standard Deviation 9.627
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score end of treatment visit	3.30 Unit on a scale Standard Deviation 9.728

SECONDARY outcome

Timeframe: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life. It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS). The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100. Higher scores indicative of better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag n=85 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score 3 Months	2.34 Unit on a scale Standard Deviation 8.443
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score 6 Months	2.85 Unit on a scale Standard Deviation 9.451
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score 9 Months	4.52 Unit on a scale Standard Deviation 7.815
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score 12 Months	5.33 Unit on a scale Standard Deviation 8.719
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score 15 Months	3.28 Unit on a scale Standard Deviation 11.083
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score 21 Months	7.18 Unit on a scale Standard Deviation 11.143
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score 24 Months	6.87 Unit on a scale Standard Deviation 9.081
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score end of treatment visit	3.53 Unit on a scale Standard Deviation 9.002
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score 18 Months	7.12 Unit on a scale Standard Deviation 11.312

SECONDARY outcome

Timeframe: Baseline to end of treatment visit, assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set.

The GP5 is a single question used to assess the overall bothersomeness of treatment side effects. The GP5 is scored using a 5-point rating scale (0 = not at all; 1 = a little bit; 2 = somewhat; 3 = quite a bit; and 4 = very much), where lower scores reflect less bothersomeness from treatment side effects.

Outcome measures

Outcome measures
Measure	Eltrombopag n=105 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Percentage of Participants With Worst Post-baseline Value in Functional Assessment of Cancer Therapy-G (GP5) Rating 0	51 Participants
Percentage of Participants With Worst Post-baseline Value in Functional Assessment of Cancer Therapy-G (GP5) Rating 1	15 Participants
Percentage of Participants With Worst Post-baseline Value in Functional Assessment of Cancer Therapy-G (GP5) Rating 2	10 Participants
Percentage of Participants With Worst Post-baseline Value in Functional Assessment of Cancer Therapy-G (GP5) Rating 4	5 Participants
Percentage of Participants With Worst Post-baseline Value in Functional Assessment of Cancer Therapy-G (GP5) Missing	22 Participants
Percentage of Participants With Worst Post-baseline Value in Functional Assessment of Cancer Therapy-G (GP5) Rating 3	2 Participants

SECONDARY outcome

Timeframe: Baseline to month 12 (End of Treatment Visit for non-responders) and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders

Population: Full Analysis Set. Only participants with a value at both Baseline and post-baseline visit included.

The Treatment Satisfaction Questionnaire for Medication (TSQM-9) is a psychometric measure of a patient's satisfaction with medication. It consists of 3 subscales: effectiveness, convenience and global satisfaction. The scores were computed by adding items for each domain, i.e. 1 to 3 for effectiveness, 4 - 6 for convenience and 7 to 9 for global satisfaction. The lowest possible score (1 for each item and 3 for all 3 subscales) was subtracted from the composite score and divided by the greatest possible score range. The greatest range was (7-1) X 3 items = 18 for the effectiveness and convenience, and (5-1) x 3 items = 12 for global satisfaction. This provided a transformed score between 0 and 1 that was then multiplied by 100. A positive change from baseline indicates improvement.

Outcome measures

Outcome measures
Measure	Eltrombopag n=66 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Global Satisfaction change from BL at 12 months	17.78 Unit on a scale Standard Deviation 29.201
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Global Satisfaction change from BL at 24 months	17.69 Unit on a scale Standard Deviation 26.110
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Global Satisfaction change from BL at end of treatment visit	9.96 Unit on a scale Standard Deviation 33.966
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Effectiveness change from BL at 12 months	18.82 Unit on a scale Standard Deviation 33.809
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Effectiveness change from BL at 24 months	17.99 Unit on a scale Standard Deviation 29.913
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Effectiveness change from BL at end of treatment visit	14.14 Unit on a scale Standard Deviation 36.673
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Convenience change from BL at 12 months	17.69 Unit on a scale Standard Deviation 30.758
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Convenience change from BL at 24 months	19.05 Unit on a scale Standard Deviation 25.189
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Convenience change from BL at end of treatment visit	15.07 Unit on a scale Standard Deviation 30.253
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Total score change from BL at 12 months	54.29 Unit on a scale Standard Deviation 78.218
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Total score change from BL at 24 months	54.72 Unit on a scale Standard Deviation 75.463
Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9) Total score change from BL at end of treatment visit	39.17 Unit on a scale Standard Deviation 86.449

POST_HOC outcome

Timeframe: On-treatment deaths: Up to approximately 35 months. Post-treatment deaths: Up to approximately 35 months

Population: Safety Population

On-treatment deaths were reported from the date of first dose of eltrombopag to 30 days after the date of the last non-zero dose of eltrombopag for patients who discontinued on or before month 12 (i.e., non-responders). For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021; approximately 35 months), as a patient could restart treatment at any time during study. Post-treatment deaths were collected in the post treatment period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021; approximately 35 months). These are not considered Adverse Events.

Outcome measures

Outcome measures
Measure	Eltrombopag n=105 Participants Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.
All Collected Deaths On-treatment deaths	3 Participants
All Collected Deaths Post-treatment deaths	1 Participants
All Collected Deaths All deaths	4 Participants

Adverse Events

Eltrombopag (On-Treatment)

Serious events: 21 serious events

Other events: 64 other events

Deaths: 3 deaths

Eltrombopag (Post-treatment Follow-up)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: + 1 862 778 8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
Publication restrictions are in place

Restriction type: OTHER

Measure	Eltrombopag (On-Treatment) n=105 participants at risk Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of \>=100×10\^9/L (CR), after 1st line steroids had failed.	Eltrombopag (Post-treatment Follow-up) Deaths collected in the post- treatment follow-up period (starting from day 31 post- treatment). No AEs were collected during this period.
Blood and lymphatic system disorders Anaemia	6.7% 7/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Blood and lymphatic system disorders Thrombocytopenia	15.2% 16/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Gastrointestinal disorders Abdominal pain	6.7% 7/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Gastrointestinal disorders Diarrhoea	9.5% 10/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Gastrointestinal disorders Gingival bleeding	5.7% 6/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Gastrointestinal disorders Nausea	7.6% 8/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Gastrointestinal disorders Vomiting	5.7% 6/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
General disorders Asthenia	7.6% 8/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
General disorders Fatigue	5.7% 6/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Investigations Alanine aminotransferase increased	8.6% 9/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Investigations Aspartate aminotransferase increased	5.7% 6/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Investigations Blood alkaline phosphatase increased	6.7% 7/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Musculoskeletal and connective tissue disorders Arthralgia	6.7% 7/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Nervous system disorders Headache	21.9% 23/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.
Skin and subcutaneous tissue disorders Petechiae	10.5% 11/105 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.	— 0/0 • Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days for patients who discontinued on or before month 12. For patients ongoing at month 12, the on-treatment period lasted until the cut-off date for the primary analysis (22-Oct-2021), up to a maximum of 35 months).Deaths were collected in the post treatment follow up period from 31 days after last dose of study medication until the cut-off date for the primary analysis (22-Oct-2021). Any sign or symptom that occurred during the conduct of the trial and safety follow-up.