Trial Outcomes & Findings for Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children (NCT NCT03939637)

Results Overview

To determine if the percentage of patients with a platelet response is significantly greater in patients with newly diagnosed ITP treated with eltrombopag than those treated with standard first-line treatments

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

122 participants

Primary outcome timeframe

12 weeks

Results posted on

2025-07-29

Participant Flow

Participant milestones

Participant milestones
Measure	Experimental: Eltrombopag Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive the experimental therapy (eltrombopag).	Comparator: Standard Therapy Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive standard therapy (investigator choice of glucocorticoids, IVIg, anti-D immunoglobulin).
Overall Study STARTED	81	41
Overall Study COMPLETED	78	40
Overall Study NOT COMPLETED	3	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Experimental: Eltrombopag Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive the experimental therapy (eltrombopag).	Comparator: Standard Therapy Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive standard therapy (investigator choice of glucocorticoids, IVIg, anti-D immunoglobulin).
Overall Study patient retrospectively deemed ineligible (did not have primary ITP)	2	0
Overall Study patient retrospectively deemed ineligible (Day 0 plt ct >30 x 10^9/L)	1	1

Baseline Characteristics

Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Experimental: Eltrombopag n=78 Participants Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive the experimental therapy (eltrombopag).	Comparator: Standard Therapy n=40 Participants Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive standard therapy (investigator choice of glucocorticoids, IVIg, anti-D immunoglobulin).	Total n=118 Participants Total of all reporting groups
Age, Continuous	7.9 years n=5 Participants	8.7 years n=7 Participants	8.2 years n=5 Participants
Sex: Female, Male Female	42 Participants n=5 Participants	18 Participants n=7 Participants	60 Participants n=5 Participants
Sex: Female, Male Male	36 Participants n=5 Participants	22 Participants n=7 Participants	58 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	15 Participants n=5 Participants	11 Participants n=7 Participants	26 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	56 Participants n=5 Participants	27 Participants n=7 Participants	83 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	7 Participants n=5 Participants	2 Participants n=7 Participants	9 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Race (NIH/OMB) White	59 Participants n=5 Participants	26 Participants n=7 Participants	85 Participants n=5 Participants
Race (NIH/OMB) More than one race	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	9 Participants n=5 Participants	7 Participants n=7 Participants	16 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: This study included 118 pediatric patients (median age 8y, 49% male), 78 randomized to eltrombopag and 40 to standard therapy.

To determine if the percentage of patients with a platelet response is significantly greater in patients with newly diagnosed ITP treated with eltrombopag than those treated with standard first-line treatments

Outcome measures

Outcome measures
Measure	Experimental: Eltrombopag n=78 Participants Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive the experimental therapy (eltrombopag).	Comparator: Standard Therapy n=40 Participants Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive standard therapy (investigator choice of glucocorticoids, IVIg, anti-D immunoglobulin).
Proportion of Patients With a Platelet Response	52 Participants	14 Participants

SECONDARY outcome

Timeframe: 1 year

Poor bleeding score (binary) at 1, 2, 3, 4 weeks, 12 weeks, and 1 year after study enrollment defined as World Health Organization (WHO) Bleeding Scale ≥ 2 or Modified Buchanan Scale ≥ 3

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 weeks

Population: Patients who received \>/= 1 dose of protocol-directed therapy were evaluable for secondary objectives.

The percentage of patients who received rescue therapy in the experimental (eltrombopag) arm vs the comparator (standard therapy) arm during the first 12 weeks of treatment

Outcome measures

Outcome measures
Measure	Experimental: Eltrombopag n=78 Participants Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive the experimental therapy (eltrombopag).	Comparator: Standard Therapy n=39 Participants Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive standard therapy (investigator choice of glucocorticoids, IVIg, anti-D immunoglobulin).
Cumulative Number of Rescue Therapies Required	13 Participants	15 Participants

SECONDARY outcome

Timeframe: 12 weeks

Population: patient required a rescue treatment during weeks 1-2 of study.

Platelet response (binary), defined as ≥ 3 of 4 weeks with platelets \>50 x109/L during weeks 6-12 of therapy, but patient required a rescue treatment during weeks 1-2 of study.

Outcome measures

Outcome measures
Measure	Experimental: Eltrombopag n=6 Participants Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive the experimental therapy (eltrombopag).	Comparator: Standard Therapy n=2 Participants Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive standard therapy (investigator choice of glucocorticoids, IVIg, anti-D immunoglobulin).
Platelet Response Among Patients Requiring Rescue Therapy During Weeks 1-2 of Study	4 Participants	0 Participants

SECONDARY outcome

Timeframe: 6 months

No further need for treatment (binary) after 12 weeks or 6 months of study

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Treatment response (binary endpoints) at 1 year defined as: * CR is defined as platelet count \>/= 150 x 10\^9/L * Primary Remission at 1 year is defined as CR at 1 year with no second-line agents required and \>/= 3 months after discontinuing most recent platelet active medication * Disease resolution at 1 year is defined as complete response (CR) at 1 year \>/= 3 months after discontinuing most recent platelet active medication. May have received a second-line therapy, excluding rituximab or splenectomy. * Disease stability at 1 year is defined as platelets \>/= 50 x 10\^9/L but \<150 x 10\^9/L \>/= 3 months after discontinuing most recent platelet active medication.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 52 weeks

Number of 2nd-line therapies in weeks 13-52

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Absolute change in percentage of CD4+25+Foxp3+ regulatory T cells from baseline at 12 weeks and 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Change in parent proxy-reported Kids ITP tool (KIT) overall scores from baseline at 1 week, 4 weeks, 12 weeks, and 1 year after study enrollment

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Total scale intensity ratings (continuous) from the Hockenberry Fatigue Scale-Parent (FS-P) at 1 week, 4 weeks, 12 weeks, and 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Serum iron, total iron binding capacity (TIBC), transferrin saturation, ferritin, mean corpuscular volume (MCV), and hemoglobin at 12 weeks, 6 months, and 1 year after study enrollment

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Safety evaluations as defined by: * Abnormal liver function tests (LFTs): ALT ≥ 3 x upper limit of normal (ULN) in patients with normal baseline ALT ≥ 3 x baseline or ≥ 5 x ULN (whichever is lower) in patients with abnormal baseline ALT ≥ 3 x ULN AND bilirubin ≥ 1.5 x ULN (\>35% direct) * Incidence of adverse events * Incidence of serious adverse events

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Time to response (platelets \>30x10\^9/L, and at least 2-fold increase in the baseline count and absence of bleeding) (IWG definition)

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Treatment response (platelets \>30x10\^9/L, and at least 2-fold increase in the baseline count and absence of bleeding) (IWG definition) at 12 weeks

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Time to platelet count \>100x10\^9/L and absence of bleeding (IWG definition)

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Treatment response (platelet count \>100x10\^9/L and absence of bleeding) (IWG definition) at 12 weeks

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Loss of treatment response (platelet count below 30x10\^9/L, or less than 2-fold increase in the baseline count or bleeding) (IWG definition) at any time during the study period after achieving response during the first 12 weeks

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Extreme thrombocytosis (platelets \>1 x10\^12/L)

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Change in child self-reported and parent impact KIT scores from baseline at 1 week, 4 weeks, 12 weeks, and 1 year after study enrollment

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Change in Hockenberry fatigue (FS-C, FS-A, FS-P) scores from baseline at 1 week, 4 weeks, 12 weeks, and 1 year after study enrollment

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Global Change Scale scores at 1 week, 4 weeks, 12 weeks, and 1 year after study enrollment

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Number of hospitalizations

Outcome measures

Outcome data not reported

Adverse Events

Experimental: Eltrombopag

Serious events: 6 serious events

Other events: 8 other events

Deaths: 0 deaths

Comparator: Standard Therapy

Serious events: 3 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Experimental: Eltrombopag n=78 participants at risk Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive the experimental therapy (eltrombopag).	Comparator: Standard Therapy n=39 participants at risk Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive standard therapy (investigator choice of glucocorticoids, IVIg, anti-D immunoglobulin).
Ear and labyrinth disorders Hemotympanum	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Skin and subcutaneous tissue disorders Hematoma	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Renal and urinary disorders Hematuria	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Nervous system disorders Intracranial hemorrhage	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Reproductive system and breast disorders Menorrhagia	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Renal and urinary disorders Urinary Tract Infection	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Immune system disorders Allergic Reaction	0.00% 0/78 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	2.6% 1/39 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Ear and labyrinth disorders Epistaxis	0.00% 0/78 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	5.1% 2/39 • Number of events 2 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)

Other adverse events

Other adverse events
Measure	Experimental: Eltrombopag n=78 participants at risk Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive the experimental therapy (eltrombopag).	Comparator: Standard Therapy n=39 participants at risk Patients ages 1 to \<18 years with newly diagnosed primary ITP, platelets \<30 x 10\^9/L, requiring pharmacological treatment, but without severe bleeding or need for rapid rise in counts, randomized to receive standard therapy (investigator choice of glucocorticoids, IVIg, anti-D immunoglobulin).
Blood and lymphatic system disorders Anemia	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Ear and labyrinth disorders Epistaxis	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Injury, poisoning and procedural complications Fall	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Nervous system disorders Headache	3.8% 3/78 • Number of events 3 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	7.7% 3/39 • Number of events 3 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Psychiatric disorders Suicidal ideation	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)
Respiratory, thoracic and mediastinal disorders Upper respiratory infection	1.3% 1/78 • Number of events 1 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)	0.00% 0/39 • 12 weeks (during which data was collected, and is now reported, in reference to the primary endpoint and/or primary outcome of the study -- proportion of patients achieving a durable platelet response of at least 3 of 4 platelet counts over 50 x10^9/L without rescue therapy during weeks 6-12 of study) Adverse Events of Grade 3 or higher during the first 12 weeks on study (as defined by the CTCAE v5.0) are reported for all patients who received at least one dose of treatment (n=117)

Additional Information

Amanda Grimes

Baylor College of Medicine/ Texas Children's Hospital

Phone: 832-822-4217

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place