Trial Outcomes & Findings for Efficacy of Eltrombopag to Improve Thrombocytopenia of MYH9-related Disease (NCT NCT01133860)

Results Overview

The primary endpoints were the achievement of a platelet count over 100 x10e9/L or at least 3 times the baseline value (major response), or at least twice the baseline value but less than major response (minor response). The overall response to therapy is reported. Platelet count was measured at the end of therapy (21 or 42 days, see study design) by phase-contrast microscopy.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

21 days and/or 42 days of therapy, 15 and 30 days after the end of therapy

Results posted on

2011-07-26

Participant Flow

Patients enrolled between January 2009 and January 2010 as outpatients in medical clinic

Participant milestones

Participant milestones
Measure	Eltrombopag Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
Overall Study STARTED	12
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy of Eltrombopag to Improve Thrombocytopenia of MYH9-related Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eltrombopag n=12 Participants Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	11 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	35.0 years STANDARD_DEVIATION 14.3 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Region of Enrollment Italy	12 participants n=5 Participants

PRIMARY outcome

Timeframe: 21 days and/or 42 days of therapy, 15 and 30 days after the end of therapy

The primary endpoints were the achievement of a platelet count over 100 x10e9/L or at least 3 times the baseline value (major response), or at least twice the baseline value but less than major response (minor response). The overall response to therapy is reported. Platelet count was measured at the end of therapy (21 or 42 days, see study design) by phase-contrast microscopy.

Outcome measures

Outcome measures
Measure	Eltrombopag n=12 Participants Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
Response to Drug Based on Platelet Count at the End of Therapy	91.6 percentage of participants Interval 61.5 to 99.7

SECONDARY outcome

Timeframe: 21 days and/or 42 days of therapy, 15 and 30 days after the end of therapy

The percentage of patients with bleeding diathesis (grade 1, i.e. cutaneous bleeding, or grade 2, i.e. mild blood loss, according to WHO bleeding score) was calculated at baseline and at the end of therapy. The results are expressed as the mean change in the percentage of patients with bleeding diathesis (95%CI).

Outcome measures

Outcome measures
Measure	Eltrombopag n=12 Participants Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
Bleeding Tendency Assessed by WHO Bleeding Score	66.7 participants Interval 31.7 to 100.0

SECONDARY outcome

Timeframe: 21 days and/or 42 days of therapy, 15 and 30 days after the end of therapy

All type of adverse events were registered.Results indicate the number of participants who experience a side effect of the drug.

Outcome measures

Outcome measures
Measure	Eltrombopag n=12 Participants Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
All Types of Adverse Events	2 number of participants

SECONDARY outcome

Timeframe: 21 days or 42 days of therapy

in vitro platelet function will be assessed in patients achieving a platelet count of 100 x10e9/L or more at the end of the therapy

Outcome measures

Outcome measures
Measure	Eltrombopag n=7 Participants Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
in Vitro Function of Platelets Produced During Therapy in Responding Patients	5 participants

Adverse Events

Eltrombopag

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Eltrombopag n=12 participants at risk Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
General disorders headache	16.7% 2/12 • Number of events 2 • 51 or 72 days, i.e. during the administration of Eltrombopag (21 or 42 days, see study design) and until 30 days after the end of treatment (see study design).
General disorders dry mouth	8.3% 1/12 • Number of events 1 • 51 or 72 days, i.e. during the administration of Eltrombopag (21 or 42 days, see study design) and until 30 days after the end of treatment (see study design).

Additional Information

Prof. Carlo Balduini

IRCCS Policlinico San Matteo Foundation

Phone: 0039.0382.502580

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place