Purpura, Thrombocytopenic, Idiopathic
Conditions
Brief summary
Patients above age 18 with a first episode of immune thrombocytopenia are randomized 1:1 between 2-4 weeks of daily prednisone (1 mg/kg/d) with subsequent dose tapering (arm A) and six 3-week cycles of pulsed dexamethasone (0.6 mg/kg/d, days 1-4; arm B). The primary endpoint is duration of remission defined as platelets ≥50/nl.
Detailed description
Patients above the age of 18 years with a first episode of immune thrombocytopenia (ITP) are eligible for inclusion, except pregnant women and patients unable to tolerate glucocorticoids. Diagnosis is made and treatment requirement defined according to the 1996 practice guidelines of the American Society of Hematology. The primary endpoint is duration of remission defined as platelets ≥50/nl. Secondary endpoints are response rate, complete remission (platelets ≥150/nl), cumulative cortisol equivalent dose (potency of prednisone / dexamethasone relative to cortisol: x 4 / x 30) and adverse events. During the first week of treatment all patients receive prednisone at 1 mg/kg/d. This period is used to confirm the diagnosis and identify patients with ITP secondary to lymphoproliferative, autoimmune (systemic lupus erythematosus, antiphospholipid syndrome) or infectious diseases (human immunodeficiency, hepatitis C, cytomegalovirus infection). Patients are stratified by age (cut-off: 50 years), gender and primary versus secondary ITP and then randomized 1:1 between daily prednisone (arm A) and pulsed dexamethasone (arm B). 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. In arm B patients receive six 21-day courses of pulsed dexamethasone (0.6 mg/kg/d, days 1-4). Patients failing to achieve or maintain a remission are switched to the alternative treatment arm (A: no response after 4 weeks of prednisone at 1-2 mg/kg/d, loss of response, maintenance doses exceeding the above limits; B: no response after 2 cycles, loss of response).
Interventions
DRUGPrednisone
Continuous daily therapy
DRUGDexamethasone
4-day pulses every 3 weeks
Sponsors
University Hospital, Essen
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* First episode of ITP
Exclusion criteria
* Pregnancy * Glucocorticoid intolerance
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Remission Duration | 6 months | Percentage of patients in ongoing remission at 6 months |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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 | 12 |
| 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 | 14 |
| Total | 26 |
Baseline characteristics
| Characteristic | Arm B: Pulsed Dexamethasone | Total | Arm A: Daily Prednisone |
|---|---|---|---|
| Age, Continuous | 46 years | 44 years | 43 years |
| Race and Ethnicity Not Collected | — | 0 Participants | — |
| Region of Enrollment Germany | 13 Participants | 22 Participants | 9 Participants |
| Sex: Female, Male Female | 5 Participants | 10 Participants | 5 Participants |
| Sex: Female, Male Male | 9 Participants | 16 Participants | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 9 | 0 / 13 |
| other Total, other adverse events | 2 / 9 | 4 / 13 |
| serious Total, serious adverse events | 0 / 9 | 0 / 13 |
Outcome results
Primary
Remission Duration
Percentage of patients in ongoing remission at 6 months
Time frame: 6 months
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Arm A: Daily Prednisone | Remission Duration | 3 Participants |
| Arm B: Pulsed Dexamethasone | Remission Duration | 11 Participants |