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.

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.

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.

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.

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.

Full Analysis Set. No data collected for this endpoint.

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

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.

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.

Full Analysis Set

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

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

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

Full Analysis Set

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).

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

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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

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.

Full Analysis Set.

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.

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

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.

Safety Population