Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.

Hematologic response rate: percentage of subjects who met any of the International Working Group criteria: Platelet count: If platelet transfusion independent at baseline (BL): Transfusion independent and increase from BL by 20×10\^9/L or more; If platelet transfusion dependent at BL: No platelet transfusion requirement for 8 weeks; Hemoglobin: If red blood cells (RBC) transfusion independent at BL: transfusion independent and increase from BL by 15 g/L or more; If RBC transfusion dependent at BL: A decrease of at least 4 units in RBC transfusions in the post-treatment 8-week period (1 unit = RBC derived from 200 mL blood) or no RBC transfusion requirement for 8 weeks; Neutrophil count: In the absence of granulocyte colony stimulating factor (G-CSF) taken within 21 days preceding the blood sample collection: Increase from BL by 0.5×10\^9/L or more, or (if \< 0.5×10\^9/L at BL) increase by 100% or more. Patients who discontinued from the trial before week 26 were treated as non-responders.

Time frame: 6 months (Week 26)

Population: The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment. The patients who discontinued from the trial before Week 26 were treated as non-responders.

The amount of transfusion was defined as the sum of transfusion multiplied by the volume of transfusion.

Time frame: Baseline, Week 13, Week 26 and Week 52

Population: Participants in the FAS who were platelet transfusion dependent at baseline and who did not discontinue prior to the corresponding visit date (Week 13, 26 and 52).

The amount of RBC transfusion was defined as the sum of transfusion multiplied by the volume of transfusion.

Time frame: Baseline, Week 13, Week 26 and Week 52

Population: Participants in the FAS who were RBC transfusion dependent at baseline and who did not discontinue prior to the corresponding visit date (Week 13, 26 and 52).

Change (increase from baseline) in hemoglobin (in the absence of red blood cell (RBC) transfusion) were calculated according to the lab test results entered into the CRF that were summarized at each visit using descriptive statistics. Hemoglobin level (g/L) was assessed in hematology test.

Time frame: Baseline, Week 13, Week 26 and Week 52

Population: The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment. These were the patients in FAS with a valid assessment for the outcome measure at baseline and at Weeks 13, 26 and 52.

Change (increase from baseline) in neutrophil count (in the absence of granulocyte colony stimulating factor (G-CSF) and calculated according to the lab test results entered into the CRF that were summarized at each visit using descriptive statistics). Neutrophil count (×109/L) was assessed in hematology test.

Time frame: Baseline, Week 13, Week 26 and Week 52

Population: The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment. These were the patients in FAS with a valid assessment for the outcome measure at baseline and at Weeks 13, 26 and 52.

Change (increase from baseline) in platelet count (in the absence of platelet transfusion) were calculated according to the lab test results entered into the case report form (CRF) that were summarized at each visit using descriptive statistics. Platelet Count (×109/L) was assessed in hematology test.

Time frame: Baseline, Week 13, Week 26, Week 52

Population: The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment.~These were the patients in FAS with a valid assessment for the outcome measure at baseline and at Weeks 13, 26 and 52.

Duration of hematologic response (any response according to the response criteria for the primary endpoint). For subjects who responded, duration of response was defined as the number of weeks from the first date of hematological response until the first date of relapse or death. Only participants with at least two response assessments were included for the duration of hematologic response assessment.

Time frame: up to approx. 3.5 years

Population: Participants in the FAS who achieved a hematologic response. The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment and achieved a hematologic response.

For subjects receiving transfusion (platelets) at baseline, the frequency of platelet transfusion in each period (Baseline: 4 weeks before Day 1; Week 13, 26, 52: 4 weeks before each visit day) was summarized using descriptive statistics.

Time frame: Baseline, Week 13, Week 26 and Week 52

Population: Participants in the FAS who were platelet transfusion dependent at baseline and who did not discontinue prior to the corresponding visit date (Week 13, 26 and 52).

For subjects receiving transfusion (RBC (Red Blood Cell)) at baseline, the frequency of RBC transfusion in each period (Baseline: 8 weeks before Day 1; Week 13, 26, 52: 8 weeks before each visit day) was summarized using descriptive statistics.

Time frame: Baseline, Week 13, Week 26 and Week 52

Population: Participants in the FAS who were RBC transfusion dependent at baseline and who did not discontinue prior to the corresponding visit date (Week 13, 26 and 52).

Hematologic response rate: percentage of subjects who met any of the International Working Group criteria: Platelet count: If platelet transfusion independent at baseline (BL): Transfusion independent and increase from BL by 20×10\^9/L or more; If platelet transfusion dependent at BL: No platelet transfusion requirement for 8 wks; Hemoglobin: If red blood cells (RBC) transfusion independent at BL: transfusion independent and increase from BL by 15 g/L or more; If RBC transfusion dependent at BL: A decrease of at least 4 units in RBC transfusions in the post-treatment 8-week period (1 unit =RBC derived from 200 mL blood) or no RBC transfusion requirement for 8 wks; Neutrophil count: In the absence of granulocyte colony stimulating factor (G-CSF) taken within 21 days preceding the blood sample collection: Increase from BL by 0.5×10\^9/L or more, or (if \< 0.5×10\^9/L at BL) increase by 100% or more. Patients who discontinued from the trial before wks 13 & 26 were treated as non-responders.

Time frame: Week (Wk) 13, Week 52

Population: The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment.

Number of participants with clonal evolution with a normal karyotype at baseline including clonal evolution to PNH (Paroxysmal Nocturnal Hemoglobinuria), evolution to AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes).

Time frame: From Baseline up to approx. 2.9 years

Population: The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned.

AUCtau is the area under the curve calculated to the end of a dosing interval (tau) at steady-state (amount\*time\*volume-1). AUClast is the AUC calculated from time 0 to the time of the last quantifiable concentration. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag. The plasma concentrations were used to determine the PK characteristics of eltrombopag. Serial intensive PK blood samples were collected for the initial 25 mg/day dose to provide at least 12 participants with evaluable PK profiles at steady state.

Time frame: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14

Population: Participants in the pharmacokinetic analysis set (PAS) who had an evaluable pharmacokinetic (PK) profile at steady state.~The pharmacokinetic analysis set (PAS) included all patients who received at least one dose of eltrombopag and who had at least one evaluable PK sample.

Steady State (CLss/F) is the apparent systemic (or total body) clearance at steady state from plasma (volume/time) following drug administration. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag. The plasma concentrations were used to determine the PK characteristics of eltrombopag. Serial intensive PK blood samples were collected for the initial 25 mg/day dose to provide at least 12 participants with evaluable PK profiles at steady state.

Time frame: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14

Population: Participants in the pharmacokinetic analysis set (PAS) who had an evaluable PK profile at steady state and who had a valid value for the outcome measure. The PAS included all patients who received at least one dose of eltrombopag and who had at least one evaluable PK sample.

Cmax is the maximum (peak) observed plasma drug concentration after single dose administration (mass\*volume-1). Blood samples were collected from patients to assess the plasma concentrations of eltrombopag. The plasma concentrations were used to determine the PK characteristics of eltrombopag. Serial intensive PK blood samples were collected for the initial 25 mg/day dose to provide at least 12 participants with evaluable PK profiles at steady state.

Time frame: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14

Population: Participants in the pharmacokinetic analysis set (PAS) who had an evaluable pharmacokinetic (PK) profile at steady state.~The pharmacokinetic analysis set (PAS) included all patients who received at least one dose of eltrombopag and who had at least one evaluable PK sample.

Tmax is the time to reach maximum (peak) plasma drug concentration after single dose administration (time). Blood samples were collected from all patients to assess the plasma concentrations of eltrombopag. The plasma concentrations were used to determine the PK characteristics of eltrombopag. Serial intensive PK blood samples were collected for the initial 25 mg/day dose to provide at least 12 participants with evaluable PK profiles at steady state.

Time frame: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14

Population: Participants in the pharmacokinetic analysis set (PAS) who had an evaluable pharmacokinetic (PK) profile at steady state.~The pharmacokinetic analysis set (PAS) included all patients who received at least one dose of eltrombopag and who had at least one evaluable PK sample.

Ctrough is the pre-dose concentration at the end of dose interval (mass\*volume-1). Blood samples were collected from patients to assess the plasma concentrations of eltrombopag. The plasma concentrations were used to determine the PK characteristics of eltrombopag. Serial intensive PK blood samples were collected for the initial 25 mg/day dose to provide at least 12 participants with evaluable PK profiles at steady state. Sparse PK blood samples were collected for the other doses and in the rest of the patients for Ctrough assessment.

Time frame: Pre-dose sample on the 15th day after each new dose level was started

Population: The pharmacokinetic analysis set (PAS) included all patients who received at least one dose of eltrombopag and who had at least one evaluable PK sample.

Time to hematological response was defined as the time from the date of first study drug administration to the first hematological response. If a participant did not meet hematological response before or at the cutoff date, censoring was performed using the date of last assessment.

Time frame: Baseline to Week 26

Population: The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment and achieved a hematologic response.

Deaths were collected from the first dose until end of study, a maximum duration of up to 3.5 years. On-treatment deaths were collected within 30 days of the last dose, while post-treatment deaths were collected more than 30 days after the last dose for those who discontinued treatment early. Adverse events were collected from the first dose until 30 days after the last dose, also up to a maximum duration if 3.5 years. Adverse Events were not collected in the post-treatment period.

Time frame: On-treatment deaths: up to approx. 3.5 years, post-treatment deaths: up to approx. 3.5 years

Population: All enrolled participants.