Trial Outcomes & Findings for Daily Prednisone Versus Pulsed Dexamethasone in Treatment-naïve Adult Patients With Immune Thrombocytopenia (NCT NCT02334813)

Results Overview

Percentage of patients in ongoing remission at 6 months

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

26 participants

Primary outcome timeframe

6 months

Results posted on

2017-06-09

Participant Flow

Participant milestones

Participant milestones
Measure	Arm A: Daily Prednisone During the first week of treatment patients in both arms receive prednisone at 1 mg/kg/d. In arm A prednisone is continued at 1 mg/kg/d for a second week. If platelets increase to ≥50/nl, its dose is reduced to \<25 mg/d by week 14 and \<7.5 mg/d by week 20 according to a step-wise reduction scheme provided in the protocol. In patients without response after 2 weeks of treatment, the prednisone dose is increased to 2 mg/kg/d for another 2 weeks, then tapered as described above. Prednisone: Continuous daily therapy	Arm B: Pulsed Dexamethasone During the first week of treatment patients in both arms receive prednisone at 1 mg/kg/d. In arm B patients subsequently receive six 21-day courses of pulsed dexamethasone (0.6 mg/kg/d, days 1-4). Dexamethasone: 4-day pulses every 3 weeks
Overall Study STARTED	12	14
Overall Study COMPLETED	9	13
Overall Study NOT COMPLETED	3	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm A: Daily Prednisone n=12 Participants During the first week of treatment patients in both arms receive prednisone at 1 mg/kg/d. In arm A prednisone is continued at 1 mg/kg/d for a second week. If platelets increase to ≥50/nl, its dose is reduced to \<25 mg/d by week 14 and \<7.5 mg/d by week 20 according to a step-wise reduction scheme provided in the protocol. In patients without response after 2 weeks of treatment, the prednisone dose is increased to 2 mg/kg/d for another 2 weeks, then tapered as described above. Prednisone: Continuous daily therapy	Arm B: Pulsed Dexamethasone n=14 Participants During the first week of treatment patients in both arms receive prednisone at 1 mg/kg/d. In arm B patients subsequently receive six 21-day courses of pulsed dexamethasone (0.6 mg/kg/d, days 1-4). Dexamethasone: 4-day pulses every 3 weeks	Total n=26 Participants Total of all reporting groups
Age, Continuous	43 years n=12 Participants	46 years n=14 Participants	44 years n=26 Participants
Sex: Female, Male Female	5 Participants n=12 Participants	5 Participants n=14 Participants	10 Participants n=26 Participants
Sex: Female, Male Male	7 Participants n=12 Participants	9 Participants n=14 Participants	16 Participants n=26 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment Germany	9 Participants n=12 Participants	13 Participants n=14 Participants	22 Participants n=26 Participants

PRIMARY outcome

Timeframe: 6 months

Percentage of patients in ongoing remission at 6 months

Outcome measures

Outcome measures
Measure	Arm A: Daily Prednisone n=9 Participants During the first week of treatment patients in both arms receive prednisone at 1 mg/kg/d. In arm A prednisone is continued at 1 mg/kg/d for a second week. If platelets increase to ≥50/nl, its dose is reduced to \<25 mg/d by week 14 and \<7.5 mg/d by week 20 according to a step-wise reduction scheme provided in the protocol. In patients without response after 2 weeks of treatment, the prednisone dose is increased to 2 mg/kg/d for another 2 weeks, then tapered as described above. Prednisone: Continuous daily therapy	Arm B: Pulsed Dexamethasone n=13 Participants During the first week of treatment patients in both arms receive prednisone at 1 mg/kg/d. In arm B patients subsequently receive six 21-day courses of pulsed dexamethasone (0.6 mg/kg/d, days 1-4). Dexamethasone: 4-day pulses every 3 weeks
Remission Duration	3 Participants	11 Participants

Adverse Events

Arm A: Daily Prednisone

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Arm B: Pulsed Dexamethasone

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Arm A: Daily Prednisone n=9 participants at risk During the first week of treatment patients in both arms receive prednisone at 1 mg/kg/d. In arm A prednisone is continued at 1 mg/kg/d for a second week. If platelets increase to ≥50/nl, its dose is reduced to \<25 mg/d by week 14 and \<7.5 mg/d by week 20 according to a step-wise reduction scheme provided in the protocol. In patients without response after 2 weeks of treatment, the prednisone dose is increased to 2 mg/kg/d for another 2 weeks, then tapered as described above. Prednisone: Continuous daily therapy	Arm B: Pulsed Dexamethasone n=13 participants at risk During the first week of treatment patients in both arms receive prednisone at 1 mg/kg/d. In arm B patients subsequently receive six 21-day courses of pulsed dexamethasone (0.6 mg/kg/d, days 1-4). Dexamethasone: 4-day pulses every 3 weeks
Metabolism and nutrition disorders Hypokalemia	0.00% 0/9	7.7% 1/13 • Number of events 1
Cardiac disorders Hypertension	11.1% 1/9 • Number of events 1	0.00% 0/13
Metabolism and nutrition disorders Hyperglycemia	0.00% 0/9	7.7% 1/13 • Number of events 1
Skin and subcutaneous tissue disorders Petechiae	11.1% 1/9 • Number of events 1	15.4% 2/13 • Number of events 2

Additional Information

Prof. Dr. Ulrich Dührsen

University Hospital Essen, Germany

Phone: +49 201 723

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place