ITP^2: Initial Treatment of Patients With Immune Thrombocytopenic Purpura

• Sponsor: HealthCore-NERI (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00991939
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI) (NIH)
• Enrollment: 8
• Locations: 18
• Arms: 2
• Duration (Months): 37.9
• Patients Per Site: 0.4
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

This study will compare treatment with 3 courses of high-dose dexamethasone versus treatment with prednisone, for patients recently diagnosed with immune thrombocytopenic purpura (ITP). The primary hypothesis is that patients treated with high-dose dexamethasone will obtain a more durable remission than patients treated with prednisone.

Condition or Disease	Intervention/Treatment	Phase
Immune Thrombocytopenic Purpura	Drug: Dexamethasone USP Micronized; Drug: Prednisone	Phase 3

Detailed Description

ITP is a common disorder associated with significant morbidity. For more than 40 years it has been recognized that this disorder was responsive to corticosteroid therapy. As corticosteroids are easily obtainable and inexpensive, they have become the standard first-line therapy for adult patients with newly-diagnosed ITP. Generally, patients are treated with prednisone at a dose of approximately 1 mg/kg, or 60 mg/day, and once a response is obtained the daily dosage is gradually tapered. While approximately 70% of patients treated in this manner respond initially, most will relapse as the corticosteroid dose is lowered; ultimately only 15-20% of patients achieve a complete or partial remission of their ITP at an "acceptable" dose of prednisone. Recently, several studies have suggested that the use of high dose corticosteroids, specifically pulse dexamethasone, may be a more efficacious initial therapy for ITP, capable of causing a higher initial response rate and a significantly longer duration of remission despite a shorter course of initial therapy.

This study will compare treatment with 3 courses of high-dose dexamethasone versus treatment with prednisone, for patients recently diagnosed with immune thrombocytopenic purpura (ITP). The primary hypothesis is that patients treated with high-dose dexamethasone will obtain a more durable remission than patients treated with standard oral corticosteroids. This may reflect the ability of high dose corticosteroids to eradicate a sensitive pathogenic lymphoid clone that may be transiently susceptible to aggressive immunosuppressive therapy early in the course of disease.

Study Design

• Study Type: Interventional
• Actual Enrollment: 8 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Initial Treatment of Patients With Immune Thrombocytopenic Purpura: The ITP^2 Study
• Study Start Date: Jan 1, 2010
• Actual Primary Completion Date: Mar 1, 2013
• Actual Study Completion Date: Mar 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: High dose pulse dexamethasone	Drug: Dexamethasone USP Micronized; The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.
Active Comparator: Standard prednisone therapy	Drug: Prednisone; Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.

Outcome Measures

Primary Outcome Measures

1. The Percentage of Patients in Each Treatment Arm Who Remain Free of All ITP Therapy With a Platelet Count ≥ 50,000/μl From 60 Days Through 365 Days After Study Entry. [From 60 days through 365 days after study entry.]

Secondary Outcome Measures

1. The Percentage of Patients Who Remain Free of All ITP Therapy With a Platelet Count ≥ 150,000/μl From 60 Days Through 365 Days After Study Entry [From 60 days through 365 days after study entry]

2. The Percentage of Patients With Platelets ≥ 50,000/μl at 365 Days Who Are Off All Treatment, Have Received ≤ 2 Acute Therapeutic Interventions for Thrombocytopenia, and Whose Last Acute Therapeutic Intervention Occurred at Least 90 Days Before Day 365 [365 days after study entry]

3. The Percentage of Patients Who Remain Free of All ITP Therapy With a Platelet Count of ≥ 150,000 From 180 Through 365 Days After Study Entry [From 180 days through 365 days after study entry]

4. The Percentage of Patients Who Remain Free of All ITP Therapy With a Platelet Count of ≥ 50,000 From 180 Through 365 Days After Study Entry [From 180 days through 365 days after study entry]

5. The Percentage of Patients Receiving Acute Therapeutic Intervention During the First 60 Days After Study Entry [Through 60 days after study entry]

6. The Percentage of Patients Receiving Acute Therapeutic Intervention Beyond the First 60 Days After Study Entry [From 60 days through 365 days after study entry]

7. The Percentage of Platelet Counts ≥ 50,000/μl After Day 60 (If a Subject Receives an Acute Therapeutic Intervention, the Next Protocol-specified Platelet Count Will be Excluded From This Analysis, as it May be Influenced by the Intervention.) [From 60 days through 365 days after study entry]

8. The Percentage of Platelet Counts ≥ 150,000/μl After Day 60 (If a Subject Receives an Acute Therapeutic Intervention, the Next Protocol-specified Platelet Count Will be Excluded From This Analysis, as it May be Influenced by the Intervention.) [From 60 days through 365 days after study entry]

9. The Percentage of Patients Undergoing Splenectomy [Through 365 days after study entry]

10. Change in the Quality of Life From Randomization to Weeks 4, 8 and End of Study, Determined Using the SF-36 Health Survey [Weeks 4, 8, and 52 after study entry]

11. The Incidence and Severity of Bleeding as Defined by a Customized Bleeding Score [Through 365 days after study entry]

12. The Percentage of Patients Not Completing Study Therapy [49 days after study entry]

13. The Percentage of Patients With Severe Adverse Events Attributable to Steroid Therapy [Through 1 year after study entry]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 15 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Must meet criteria for a diagnosis of ITP as specified by ASH guidelines

• Must be within 30 days after diagnosis of ITP at the time of randomization (diagnosis of ITP starts with first platelet count ≤ 100,000/μl)

• Platelet count ≤ 30,000/μl at the time ITP is diagnosed, and/or at some time between the diagnosis of ITP and study entry

• Platelet count ≤ 150,000/μl at the time of randomization

• Age ≥ 15 years

• If bone marrow examination is available, it must be compatible with ITP

• Subjects, or their legal guardians, must have the ability to provide informed consent

Exclusion Criteria:

• Rituximab therapy or splenectomy for ITP or for any other cause within the previous 8 weeks.

• Known HIV infection

• Known HCV infection

• Known systemic lupus erythematosus

• Pregnancy or breastfeeding

• Insulin-requiring diabetes mellitus

• Previous exposure to prednisone for ITP at a dose ≥ 1.5 mg/kg prednisone/day for ≥ 1 week prior to study entry

• Ongoing use of treatments that are known to inhibit platelet function, e.g. aspirin

• Anything that in the opinion of the investigator is likely to interfere with participation in the study

• Persons previously randomized in the ITP^2 study

• Persons currently enrolled in other interventional clinical trials

• Exposure to thrombopoietic agent prior to study entry

• Previous exposure to dexamethasone for the treatment of ITP at a dose of 30 mg/day or greater for subjects < 60 kg or 40 mg/day or greater for subjects >= 60 kg for at least four days

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Tulane University	New Orleans	Louisiana	United States	70112
2	University of Maryland	Baltimore	Maryland	United States	21201
3	Johns Hopkins Hospital	Baltimore	Maryland	United States	21287
4	Massachusetts General Hospital	Boston	Massachusetts	United States	02114
5	Brigham & Women's Hospital	Boston	Massachusetts	United States	02115
6	Children's Hospital Boston	Boston	Massachusetts	United States	02115
7	Weill Medical College, Cornell University	New York	New York	United States	10021
8	University of North Carolina Hospitals	Chapel Hill	North Carolina	United States	27514
9	Duke University	Durham	North Carolina	United States	27710
10	Case Western Reserve University	Cleveland	Ohio	United States	44106
11	Cleveland Clinic Foundation	Cleveland	Ohio	United States	44195
12	The University of Oklahoma Health Sciences Center	Oklahoma City	Oklahoma	United States	73104
13	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States	19104
14	University of Pennsylvania	Philadelphia	Pennsylvania	United States	19104
15	University of Pittsburgh Presbyterian and Shadyside Hospital	Pittsburgh	Pennsylvania	United States	15213
16	University of Washington Medical Center	Seattle	Washington	United States	98195
17	Gundersen Clinic	La Crosse	Wisconsin	United States	54601
18	University of Wisconsin	Madison	Wisconsin	United States	53792

Sponsors and Collaborators

• HealthCore-NERI
• National Heart, Lung, and Blood Institute (NHLBI)

Investigators

• Principal Investigator: Susan F Assmann, PhD, HealthCore-NERI
• Principal Investigator: James Bussel, MD, Weill Medical College, Cornell University
• Principal Investigator: Alvin Schmaier, MD, Case Western Reserve University
• Principal Investigator: Jodi Segal, MD, Johns Hopkins University
• Principal Investigator: Terry Gernsheimer, MD, University of Washington
• Principal Investigator: Eliot Williams, MD, University of Wisconsin, Madison
• Principal Investigator: Ellis Neufeld, MD, Boston Children's Hospital
• Principal Investigator: Judith Lin, MD, Brigham and Women's Hospital
• Principal Investigator: Thomas Ortel, MD, Duke University
• Principal Investigator: David Kuter, MD, Massachusetts General Hospital
• Principal Investigator: Cindy Leissinger, MD, Tulane University
• Principal Investigator: Ann Zimrin, MD, University of Maryland
• Principal Investigator: Nigel Key, MD, University of North Carolina, Chapel Hill
• Principal Investigator: James George, MD, The University of Oklahoma
• Principal Investigator: Michele Lambert, MD, Children's Hospital of Philadelphia
• Principal Investigator: Joseph Kiss, MD, University of Pittsburgh
• Principal Investigator: Bruce Sachais, MD, PhD, University of Pennsylvania

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: HealthCore-NERI
• ClinicalTrials.gov Identifier: NCT00991939
• Other Study ID Numbers: 675; U01HL072268; HL072268; HL072033; HL072291; HL072196; HL072289; HL072248; HL072191; HL072299; HL072305; HL072274; HL072028; HL072359; HL072072; HL072355; HL072283; HL072346; HL072331; HL072290
• First Posted: Oct 8, 2009
• Last Update Posted: Feb 14, 2014
• Last Verified: Jan 1, 2014

• Keywords provided by HealthCore-NERI: Immune thrombocytopenic purpura; ITP; Dexamethasone; Prednisone
• Additional relevant MeSH terms: Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic; Dexamethasone; Prednisone

Study Results

Participant Flow

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized : The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone : Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Period Title: Overall Study
STARTED	5	3
COMPLETED	0	1
NOT COMPLETED	5	2

Baseline Characteristics

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy	Total
Arm/Group Description	Dexamethasone USP Micronized : The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone : Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.	Total of all reporting groups
Overall Participants	5	3	8
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%
Between 18 and 65 years	5 100%	1 33.3%	6 75%
>=65 years	0 0%	2 66.7%	2 25%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	37.2 (15.6)	62.5 (35.8)	46.7 (26.0)
Sex: Female, Male (Count of Participants)
Female	3 60%	1 33.3%	4 50%
Male	2 40%	2 66.7%	4 50%
Region of Enrollment (participants) [Number]
United States	5 100%	3 100%	8 100%

Outcome Measures

1. Primary Outcome

Title	The Percentage of Patients in Each Treatment Arm Who Remain Free of All ITP Therapy With a Platelet Count ≥ 50,000/μl From 60 Days Through 365 Days After Study Entry.
Time Frame	From 60 days through 365 days after study entry.

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized : The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone : Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	3	1
Number [percentage of subjects]	0	0

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	High Dose Pulse Dexamethasone, Standard Prednisone Therapy
	Comments	Four subjects were not included in this analysis because not enough data was available to assess the primary outcome at the time the study was terminated.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	1.00
	Comments
	Method	Fisher Exact
	Comments
Method of Estimation	Estimation Parameter	Difference of proportions
	Estimated Value	0
	Confidence Interval	(2-Sided) 95%; -0.975 to 0.708
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 0.58
	Estimation Comments	The estimated value is the proportion of success in the high dose pulse dexamethasone group minus the proportion of success in the standard prednisone group.

2. Secondary Outcome

Title	The Percentage of Patients Who Remain Free of All ITP Therapy With a Platelet Count ≥ 150,000/μl From 60 Days Through 365 Days After Study Entry
Time Frame	From 60 days through 365 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

3. Secondary Outcome

Title	The Percentage of Patients With Platelets ≥ 50,000/μl at 365 Days Who Are Off All Treatment, Have Received ≤ 2 Acute Therapeutic Interventions for Thrombocytopenia, and Whose Last Acute Therapeutic Intervention Occurred at Least 90 Days Before Day 365
Time Frame	365 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

4. Secondary Outcome

Title	The Percentage of Patients Who Remain Free of All ITP Therapy With a Platelet Count of ≥ 150,000 From 180 Through 365 Days After Study Entry
Time Frame	From 180 days through 365 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

5. Secondary Outcome

Title	The Percentage of Patients Who Remain Free of All ITP Therapy With a Platelet Count of ≥ 50,000 From 180 Through 365 Days After Study Entry
Time Frame	From 180 days through 365 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

6. Secondary Outcome

Title	The Percentage of Patients Receiving Acute Therapeutic Intervention During the First 60 Days After Study Entry
Time Frame	Through 60 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

7. Secondary Outcome

Title	The Percentage of Patients Receiving Acute Therapeutic Intervention Beyond the First 60 Days After Study Entry
Time Frame	From 60 days through 365 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

8. Secondary Outcome

Title	The Percentage of Platelet Counts ≥ 50,000/μl After Day 60 (If a Subject Receives an Acute Therapeutic Intervention, the Next Protocol-specified Platelet Count Will be Excluded From This Analysis, as it May be Influenced by the Intervention.)
Time Frame	From 60 days through 365 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

9. Secondary Outcome

Title	The Percentage of Platelet Counts ≥ 150,000/μl After Day 60 (If a Subject Receives an Acute Therapeutic Intervention, the Next Protocol-specified Platelet Count Will be Excluded From This Analysis, as it May be Influenced by the Intervention.)
Time Frame	From 60 days through 365 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

10. Secondary Outcome

Title	The Percentage of Patients Undergoing Splenectomy
Time Frame	Through 365 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

11. Secondary Outcome

Title	Change in the Quality of Life From Randomization to Weeks 4, 8 and End of Study, Determined Using the SF-36 Health Survey
Time Frame	Weeks 4, 8, and 52 after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

12. Secondary Outcome

Title	The Incidence and Severity of Bleeding as Defined by a Customized Bleeding Score
Time Frame	Through 365 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

13. Secondary Outcome

Title	The Percentage of Patients Not Completing Study Therapy
Time Frame	49 days after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

14. Secondary Outcome

Title	The Percentage of Patients With Severe Adverse Events Attributable to Steroid Therapy
Time Frame	Through 1 year after study entry

Outcome Measure Data

Analysis Population Description

Due to the same sample size at the time that the study was terminated, this endpoint was not analyzed.

Arm/Group Title	High Dose Pulse Dexamethasone	Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.	Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
Measure Participants	0	0

Adverse Events

Arm/Group Title	High Dose Pulse Dexamethasone		Standard Prednisone Therapy
Arm/Group Description	Dexamethasone USP Micronized: The dose for dexamethasone is 30 mg/day for patients < 60 kg and 40 mg/day for patients > 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.		Prednisone: Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients > 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients < 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.
All Cause Mortality
	High Dose Pulse Dexamethasone		Standard Prednisone Therapy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	High Dose Pulse Dexamethasone		Standard Prednisone Therapy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	3/5 (60%)		1/3 (33.3%)
Blood and lymphatic system disorders
Thrombocytopenia	2/5 (40%)	2	0/3 (0%)	0
Gastrointestinal disorders
Gingival bleeding	1/5 (20%)	1	0/3 (0%)	0
General disorders
Adverse drug reaction	1/5 (20%)	1	0/3 (0%)	0
Injury, poisoning and procedural complications
Laceration	0/5 (0%)	0	1/3 (33.3%)	1
Tibia fracture	0/5 (0%)	0	1/3 (33.3%)	1
Investigations
Occult blood	1/5 (20%)	1	0/3 (0%)	0
Nervous system disorders
Headache	1/5 (20%)	1	0/3 (0%)	0
Respiratory, thoracic and mediastinal disorders
Haemoptysis	1/5 (20%)	1	0/3 (0%)	0
Skin and subcutaneous tissue disorders
Increased tendency to bruise	1/5 (20%)	1	0/3 (0%)	0
Other (Not Including Serious) Adverse Events
	High Dose Pulse Dexamethasone		Standard Prednisone Therapy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	5/5 (100%)		3/3 (100%)
Blood and lymphatic system disorders
Anaemia vitamin B12 deficiency	1/5 (20%)	1	0/3 (0%)	0
Cardiac disorders
Palpitations	3/5 (60%)	5	0/3 (0%)	0
Ear and labyrinth disorders
Ear pain	1/5 (20%)	1	0/3 (0%)	0
Endocrine disorders
Cushingoid	1/5 (20%)	1	1/3 (33.3%)	2
Eye disorders
Conjunctival haemorrhage	1/5 (20%)	1	1/3 (33.3%)	1
Eye pain	1/5 (20%)	1	0/3 (0%)	0
Scleral haemorrhage	0/5 (0%)	0	1/3 (33.3%)	1
Vision blurred	5/5 (100%)	6	0/3 (0%)	0
Visual acuity reduced	1/5 (20%)	1	0/3 (0%)	0
Visual impairment	0/5 (0%)	0	1/3 (33.3%)	1
Gastrointestinal disorders
Abdominal pain	1/5 (20%)	1	1/3 (33.3%)	1
Anal haemorrhage	0/5 (0%)	0	1/3 (33.3%)	1
Diarrhoea	2/5 (40%)	2	0/3 (0%)	0
Gingival bleeding	0/5 (0%)	0	1/3 (33.3%)	1
Haemorrhoids	1/5 (20%)	1	0/3 (0%)	0
Mouth haemorrhage	2/5 (40%)	3	0/3 (0%)	0
Mouth ulceration	2/5 (40%)	2	0/3 (0%)	0
Nausea	2/5 (40%)	3	2/3 (66.7%)	3
Oral disorder	0/5 (0%)	0	1/3 (33.3%)	1
Vomiting	3/5 (60%)	3	0/3 (0%)	0
Dyspepsia	1/5 (20%)	1	0/3 (0%)	0
General disorders
Application site bleeding	1/5 (20%)	1	0/3 (0%)	0
Chest pain	3/5 (60%)	4	1/3 (33.3%)	1
Face oedema	1/5 (20%)	2	1/3 (33.3%)	2
Fat tissue increased	1/5 (20%)	1	2/3 (66.7%)	2
Fatigue	5/5 (100%)	8	3/3 (100%)	3
Feeling jittery	1/5 (20%)	1	0/3 (0%)	0
Malaise	1/5 (20%)	1	0/3 (0%)	0
Mucosal haemorrhage	1/5 (20%)	2	0/3 (0%)	0
Non-cardiac chest pain	1/5 (20%)	1	0/3 (0%)	0
Oedema	1/5 (20%)	1	0/3 (0%)	0
Pyrexia	1/5 (20%)	1	0/3 (0%)	0
Swelling	1/5 (20%)	1	0/3 (0%)	0
Thirst	4/5 (80%)	5	2/3 (66.7%)	3
Infections and infestations
Bronchitis	1/5 (20%)	3	0/3 (0%)	0
Influenza	1/5 (20%)	1	0/3 (0%)	0
Pharyngitis	1/5 (20%)	1	0/3 (0%)	0
Viral upper respiratory tract	0/5 (0%)	0	1/3 (33.3%)	1
Paronychia	1/5 (20%)	1	0/3 (0%)	0
Injury, poisoning and procedural complications
Compensatory sweating	1/5 (20%)	1	1/3 (33.3%)	1
Investigations
Occult blood	1/5 (20%)	1	0/3 (0%)	0
Metabolism and nutrition disorders
Abnormal weight gain	0/5 (0%)	0	1/3 (33.3%)	1
Appetite disorder	1/5 (20%)	1	0/3 (0%)	0
Dehydration	1/5 (20%)	2	0/3 (0%)	0
Increased appetite	4/5 (80%)	7	3/3 (100%)	3
Weight fluctuation	1/5 (20%)	1	0/3 (0%)	0
Musculoskeletal and connective tissue disorders
Back pain	3/5 (60%)	4	3/3 (100%)	4
Bone pain	0/5 (0%)	0	1/3 (33.3%)	2
Joint swelling	1/5 (20%)	1	1/3 (33.3%)	4
Muscular weakness	2/5 (40%)	2	2/3 (66.7%)	2
Pain in extremity	1/5 (20%)	1	0/3 (0%)	0
Nervous system disorders
Dizziness	1/5 (20%)	1	1/3 (33.3%)	1
Headache	2/5 (40%)	2	1/3 (33.3%)	1
Migraine	1/5 (20%)	1	0/3 (0%)	0
Poor quality sleep	2/5 (40%)	3	1/3 (33.3%)	1
Syncope	0/5 (0%)	0	1/3 (33.3%)	1
Tremor	1/5 (20%)	1	2/3 (66.7%)	3
Psychiatric disorders
Agitation	1/5 (20%)	2	0/3 (0%)	0
Anxiety	5/5 (100%)	8	2/3 (66.7%)	2
Depressed mood	1/5 (20%)	1	1/3 (33.3%)	1
Depression	2/5 (40%)	2	1/3 (33.3%)	1
Insomnia	3/5 (60%)	4	1/3 (33.3%)	1
Libido decreased	1/5 (20%)	1	0/3 (0%)	0
Mood swings	3/5 (60%)	5	2/3 (66.7%)	2
Nightmare	2/5 (40%)	2	2/3 (66.7%)	2
Restlessness	4/5 (80%)	4	2/3 (66.7%)	3
Renal and urinary disorders
Haematuria	1/5 (20%)	1	0/3 (0%)	0
Reproductive system and breast disorders
Breast enlargement	1/5 (20%)	1	0/3 (0%)	0
Male sexual dysfunction	0/5 (0%)	0	2/3 (66.7%)	2
Menorrhagia	1/5 (20%)	4	0/3 (0%)	0
Menstruation irregular	1/5 (20%)	3	0/3 (0%)	0
Respiratory, thoracic and mediastinal disorders
Cough	0/5 (0%)	0	1/3 (33.3%)	1
Dyspnoea	1/5 (20%)	1	0/3 (0%)	0
Epistaxis	3/5 (60%)	4	2/3 (66.7%)	3
Haemoptysis	1/5 (20%)	1	0/3 (0%)	0
Nasal congestion	1/5 (20%)	1	0/3 (0%)	0
Pulmonary congestion	1/5 (20%)	1	0/3 (0%)	0
Skin and subcutaneous tissue disorders
Alopecia	1/5 (20%)	1	1/3 (33.3%)	1
Blood blister	0/5 (0%)	0	2/3 (66.7%)	2
Dermatitis acneiform	1/5 (20%)	1	0/3 (0%)	0
Dry skin	5/5 (100%)	6	2/3 (66.7%)	3
Ecchymosis	0/5 (0%)	0	1/3 (33.3%)	3
Erythema	1/5 (20%)	1	1/3 (33.3%)	1
Hyperhidrosis	4/5 (80%)	6	0/3 (0%)	0
Increased tendency to bruise	5/5 (100%)	10	2/3 (66.7%)	4
Nail discomfort	0/5 (0%)	0	1/3 (33.3%)	2
Night sweats	0/5 (0%)	0	1/3 (33.3%)	3
Oil acne	1/5 (20%)	1	0/3 (0%)	0
Onycholysis	1/5 (20%)	1	0/3 (0%)	0
Petechiae	3/5 (60%)	4	2/3 (66.7%)	2
Rash	1/5 (20%)	1	1/3 (33.3%)	1
Seborrhoea	1/5 (20%)	1	0/3 (0%)	0
Skin discolouration	0/5 (0%)	0	1/3 (33.3%)	1
Skin disorder	0/5 (0%)	0	1/3 (33.3%)	1
Skin reaction	0/5 (0%)	0	1/3 (33.3%)	1
Swelling face	0/5 (0%)	0	1/3 (33.3%)	2
Urticaria	0/5 (0%)	0	1/3 (33.3%)	1
Skin discomfort	0/5 (0%)	0	1/3 (33.3%)	1
Surgical and medical procedures
Menstrual cycle management	1/5 (20%)	1	0/3 (0%)	0
Vascular disorders
Flushing	2/5 (40%)	2	2/3 (66.7%)	2
Hypertension	0/5 (0%)	0	1/3 (33.3%)	1

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.