Trial Outcomes & Findings for A Study to Assess Efficacy and Safety of Eltrombopag in Combination With a Short Course of Dexamethasone in Patients With Newly Diagnosed ITP (NCT NCT04346654)

Last Updated: 2025-05-16

Results Overview

Sustained response off treatment at 52 weeks is defined as maintenance of platelet count ≥ 30 × 10\^9/L after treatment discontinuation until Week 52 in the absence of bleeding events ≥ Grade II or use of any rescue medication at all visits until Week 52. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

26 participants

Primary outcome timeframe

Study treatment discontinuation until week 52

Results posted on

2025-05-16

Participant Flow

The study was conducted in 25 centers in Germany.

Participants had to abstain from using investigational/marketed drugs and taking herbal supplements prior to taking the first dose of study treatment.

Participant milestones

Participant milestones
Measure	Eltrombopag + Dexamethasone Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Overall Study STARTED	13	13
Overall Study Randomized, Not Treated	1	0
Overall Study COMPLETED	8	10
Overall Study NOT COMPLETED	5	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Eltrombopag + Dexamethasone Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Overall Study Adverse Event	2	0
Overall Study Non-response	1	1
Overall Study Withdrawal by Subject	2	0
Overall Study Lost to Follow-up	0	1
Overall Study Pregnancy	0	1

Baseline Characteristics

A Study to Assess Efficacy and Safety of Eltrombopag in Combination With a Short Course of Dexamethasone in Patients With Newly Diagnosed ITP

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eltrombopag + Dexamethasone n=13 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.	Total n=26 Participants Total of all reporting groups
Age, Continuous	60.6 Years STANDARD_DEVIATION 14.5 • n=5 Participants	45.3 Years STANDARD_DEVIATION 14.3 • n=7 Participants	53.0 Years STANDARD_DEVIATION 16.1 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	6 Participants n=7 Participants	12 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	7 Participants n=7 Participants	14 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian	12 Participants n=5 Participants	12 Participants n=7 Participants	24 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Study treatment discontinuation until week 52

Population: The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization.

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=13 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Percentage of Patients With Sustained Response Off Treatment at 52 Weeks	2 Participants	2 Participants

SECONDARY outcome

Timeframe: Study treatment discontinuation until week 52

Population: The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization.

Overall response after treatment at week 52 was defined as maintenance of platelet count ≥ 30 x 109/L and ≥ 2-fold increase of screening platelet count after treatment discontinuation in the absence of bleeding event ≥ Grade II and no rescue therapy at all visits until Week 52. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding.

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=13 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Percentage of Patients With Overall Response at Week 52	2 Participants	2 Participants

SECONDARY outcome

Timeframe: from last dose of study treatment until loss of response, approx. 52 weeks

Population: The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization and who had a response.

Duration of sustained response off treatment is defined as time of treatment discontinuation until platelet count \< 30 × 109/L or bleeding events ≥ Grade II or use of any rescue therapy. If sustained response remains, the interval was censored with the date of the last platelet assessment. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding.

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=2 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=2 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Duration of Sustained Response Off Treatment	49.1 Weeks Standard Deviation 0.0	45.7 Weeks Standard Deviation 2.8

SECONDARY outcome

Timeframe: By Week 4

Population: The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization.

Overall response by week 4 is defined as platelet count ≥ 30 × 109/L and ≥ 2 fold increase of screening platelet count and absence of bleeding and no rescue therapy within the first 4 weeks

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=13 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Overall Response by Week 4	10 Participants	11 Participants

SECONDARY outcome

Timeframe: By Week 4

Population: The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization.

Complete Response by week 4 is defined as platelet count ≥ 100 × 109/L and absence of bleeding and no rescue therapy until week 4.

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=13 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Complete Response by Week 4	8 Participants	9 Participants

SECONDARY outcome

Timeframe: Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53

Population: The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. Only patients who contributed data were included in the "number analyzed" per week. As not all participants contributed data for each visit the "overall number of participants analyzed" and the "number analyzed" per week differ.

Absolute change in platelet count from pre-treatment or screening to baseline (week 1) and from pre-treatment / screening to 2, 4, 13, 27 and 53 weeks. If pre-treatment was necessary before inclusion, platelet count data performed directly before pre-treatment were used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate).

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=12 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 27	217.8 G/L Standard Deviation 126.5	141.2 G/L Standard Deviation 94.7
Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 53	139.0 G/L Standard Deviation 88.0	141.7 G/L Standard Deviation 61.4
Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 1	32.0 G/L Standard Deviation 44.3	23.2 G/L Standard Deviation 48.0
Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 2	145.2 G/L Standard Deviation 151.9	80.7 G/L Standard Deviation 68.8
Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 4	192.5 G/L Standard Deviation 165.8	92.0 G/L Standard Deviation 68.7
Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 13	119.4 G/L Standard Deviation 115.9	143.0 G/L Standard Deviation 88.5

SECONDARY outcome

Timeframe: Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53

Relative change in platelet count from pre-treatment or screening to baseline (week 1) and from pre-treatment or screening to 2, 4, 13, 27, and 53 weeks. If pre-treatment was necessary before inclusion, platelet count data performed directly before pre-treatment were used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate).

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=12 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 1	347.9 Percentage change in platelet counts Standard Deviation 486.8	293.4 Percentage change in platelet counts Standard Deviation 418.4
Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 13	8050.6 Percentage change in platelet counts Standard Deviation 12277.8	4725.7 Percentage change in platelet counts Standard Deviation 8418.2
Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 27	13667.9 Percentage change in platelet counts Standard Deviation 15717.1	4381.1 Percentage change in platelet counts Standard Deviation 8195.2
Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 53	9392.3 Percentage change in platelet counts Standard Deviation 11476.0	5612.1 Percentage change in platelet counts Standard Deviation 8300.7
Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 2	5795.5 Percentage change in platelet counts Standard Deviation 9672.4	2028.2 Percentage change in platelet counts Standard Deviation 4382.0
Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points Week 4	11400.9 Percentage change in platelet counts Standard Deviation 16875.2	2865.4 Percentage change in platelet counts Standard Deviation 3805.4

SECONDARY outcome

Timeframe: Time from starting study treatment to achievement of complete response (up to 52 weeks)

Population: The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization.

Time to overall response is defined as time from starting study treatment to time of achievement of overall response. Overall response is defined as a platelet count ≥ 30 × 10\^9/L and ≥ 2 fold increase of baseline platelet count and absence of bleeding and no rescue therapy censored with the last visit date for patients not achieving overall response. Results of TOR are reported per Kaplan-Meier estimates.

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=13 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Time to Overall Response (TOR)	1.1 Weeks Interval 0.0 to 4.1	1.0 Weeks Interval 0.1 to 1.3

SECONDARY outcome

Timeframe: Time from starting study treatment to achievement of complete response (up to 52 weeks)

Population: The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization.

Time to complete response is defined as time from starting study treatment to time of achievement of complete response. Complete response is defined as a platelet count ≥ 100 × 109/L and absence of bleeding and no rescue therapy. Results of time to complete response are reported per Kaplan-Meier estimates.

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=13 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Time to Complete Response	1.1 Weeks Interval 0.1 to NA = upper limit of CI is inestimable because there were not enough events to calculate	2.1 Weeks Interval 0.6 to NA = upper limit of CI is inestimable because there were not enough events to calculate

SECONDARY outcome

Timeframe: Achievement of overall or complete response until loss of response (up to 52 weeks)

Population: The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. Patients who did not reach OR/CR were censored at time 0.

Duration of overall or complete response (CR) is defined as time of achievement of overall or CR until loss of overall or CR. The duration of CR was calculated from the date of onset of CR until platelet count \< 100 x 109/L, or bleeding events ≥ Grade II (assessed by the modified World Health Organization (WHO) Bleeding Scale) or use of any rescue therapy, whatever was earlier. Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding. Results of duration of overall and complete response are reported per Kaplan-Meier estimates.

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=13 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Duration of Overall Response (OR) and Complete Response (CR) Median duration of overall response	8.3 Weeks Interval 0.3 to NA = upper limit of CI is inestimable because there were not enough events to calculate	3.6 Weeks Interval 1.1 to NA = upper limit of CI is inestimable because there were not enough events to calculate
Duration of Overall Response (OR) and Complete Response (CR) Median duration of complete response	9.7 Weeks Interval 1.3 to NA = upper limit of CI is inestimable because there were not enough events to calculate	1.3 Weeks Interval 0.9 to 6.1

SECONDARY outcome

Timeframe: Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53

The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) instrument is a 13-item validated tool used to measure an individual's level of fatigue during usual daily activities over the past 7 days. Items are scored on a 0-4 response scale (4=not at all to 0=very much) where the total possible score ranges from 0-52 (all items are summed up to create the total score); A score of less than 30 indicates severe fatigue. The higher scores represent better HRQoL.

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=10 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=11 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire Change from BL to Week 13	0.3 scores on a scale Standard Deviation 8.3	0.3 scores on a scale Standard Deviation 2.8
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire Change from Baseline (BL) to Week 2	-4.2 scores on a scale Standard Deviation 9.6	-4.5 scores on a scale Standard Deviation 6.9
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire Change from BL to Week	1.0 scores on a scale Standard Deviation 4.4	-2.9 scores on a scale Standard Deviation 5.1
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire Change from BL to Week 5	1.2 scores on a scale Standard Deviation 5.0	-5.1 scores on a scale Standard Deviation 6.9
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire Change from BL to Week 27	-3.6 scores on a scale Standard Deviation 11.0	-3.1 scores on a scale Standard Deviation 7.7
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire Change from BL to Week 53	2.7 scores on a scale Standard Deviation 5.6	-3.4 scores on a scale Standard Deviation 5.4

SECONDARY outcome

Timeframe: Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53

SF36 questionnaire is a tool to measure health-related QoL. SF36 questionnaires (physical and mental score) were answered throughout the study and is a validated instrument with 36 questions to measure general physical and mental health status via assessment of 8 domains-Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, and Mental Health-over the past 4 weeks. The SF36 is scored using norm-based scoring procedures and scores ranging from 0-100; higher values indicate less impairment, a higher QoL. In addition to this SAP-planned scoring score, an alternative scoring for both the physical SF36 score and the mental SF36 were performed by QualityMetric (QM) Incorporated, an IQVIA business.

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=11 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=12 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) Change from Baseline (CBL) to Wk 2: Physical Score (PS)	1.9 scores on a scale Standard Deviation 4.6	-3.3 scores on a scale Standard Deviation 6.6
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 3: PS	2.5 scores on a scale Standard Deviation 5.8	-1.4 scores on a scale Standard Deviation 8.8
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 5: PS	1.5 scores on a scale Standard Deviation 6.2	-2.7 scores on a scale Standard Deviation 10.5
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 13: PS	3.5 scores on a scale Standard Deviation 9.9	0.3 scores on a scale Standard Deviation 8.0
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 27: PS	2.6 scores on a scale Standard Deviation 14.7	-0.9 scores on a scale Standard Deviation 10.8
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 53: PS	8.1 scores on a scale Standard Deviation 11.8	0.3 scores on a scale Standard Deviation 10.0
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 2: Mental Score (MS)	-0.8 scores on a scale Standard Deviation 6.1	-1.2 scores on a scale Standard Deviation 7.6
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 3: MS	-2.0 scores on a scale Standard Deviation 11.0	-1.6 scores on a scale Standard Deviation 9.7
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 5: MS	0.1 scores on a scale Standard Deviation 7.3	-1.7 scores on a scale Standard Deviation 11.5
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 13: MS	3.3 scores on a scale Standard Deviation 7.5	4.1 scores on a scale Standard Deviation 7.4
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 27: MS	-1.4 scores on a scale Standard Deviation 4.9	-2.7 scores on a scale Standard Deviation 7.4
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 53: MS	2.1 scores on a scale Standard Deviation 8.8	0.6 scores on a scale Standard Deviation 5.7
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 2: PS-QM	2.0 scores on a scale Standard Deviation 4.1	-3.3 scores on a scale Standard Deviation 6.0
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 3: PS-QM	3.4 scores on a scale Standard Deviation 6.4	-1.4 scores on a scale Standard Deviation 7.8
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 5: PS-QM	2.4 scores on a scale Standard Deviation 6.0	-2.7 scores on a scale Standard Deviation 8.5
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 1: PS-QM	4.1 scores on a scale Standard Deviation 8.5	0.3 scores on a scale Standard Deviation 7.6
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 27: PS-QM	3.6 scores on a scale Standard Deviation 12.4	-1.0 scores on a scale Standard Deviation 9.7
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) CBL to Week 53: PS-QM	6.2 scores on a scale Standard Deviation 10.7	0.2 scores on a scale Standard Deviation 9.4

SECONDARY outcome

Timeframe: Baseline up to 52 weeks

Population: The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization.

Incidence and severity of bleeding assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Incidence of bleeding: participants had at least one bleeding event. Severity of bleeding: bleeding event is from grade 2 and higher

Outcome measures

Outcome measures
Measure	Eltrombopag + Dexamethasone n=13 Participants Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 Participants Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Incidence and Severity of Bleeding Events Incidence of Bleeding events with severity: ≥ grade 3	2 Participants	1 Participants
Incidence and Severity of Bleeding Events Incidence of Bleeding events	10 Participants	12 Participants
Incidence and Severity of Bleeding Events Incidence of Bleeding events with severity: ≥ grade 2	6 Participants	2 Participants

Adverse Events

Eltrombopag + Dexamethasone

Serious events: 5 serious events

Other events: 11 other events

Deaths: 0 deaths

Dexamethasone

Serious events: 5 serious events

Other events: 13 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Additional Information

Clinical Disclosure Office

Novartis Pharmaceuticals

Phone: 862-778-3000

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 pooled data (i.e., data from all sites) in clinical trial or disclosure of trial results in their entirety.
Publication restrictions are in place

Restriction type: OTHER

Measure	Eltrombopag + Dexamethasone n=12 participants at risk Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment.	Dexamethasone n=13 participants at risk Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment.
Blood and lymphatic system disorders Aplastic anaemia	8.3% 1/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Blood and lymphatic system disorders Immune thrombocytopenia	16.7% 2/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Blood and lymphatic system disorders Iron deficiency anaemia	8.3% 1/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	7.7% 1/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Cardiac disorders Myocardial infarction	0.00% 0/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	7.7% 1/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Gastrointestinal disorders Oesophageal stenosis	8.3% 1/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Gastrointestinal disorders Pancreatitis	0.00% 0/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	7.7% 1/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
General disorders Vascular stent occlusion	0.00% 0/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	7.7% 1/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Infections and infestations Herpes zoster	8.3% 1/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Infections and infestations Pneumonia	8.3% 1/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Metabolism and nutrition disorders Hyperglycaemia	0.00% 0/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	7.7% 1/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) B-cell lymphoma	8.3% 1/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Breast cancer	8.3% 1/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Myelodysplastic syndrome	0.00% 0/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	7.7% 1/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Prostate cancer	0.00% 0/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	7.7% 1/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Renal and urinary disorders Renal colic	8.3% 1/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Renal and urinary disorders Ureterolithiasis	8.3% 1/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.00% 0/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	15.4% 2/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
Vascular disorders Haematoma	8.3% 1/12 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.	0.00% 0/13 • Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years. Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.