Trial Outcomes & Findings for Retrospective & Prospective Observational Study of Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) (NCT NCT00454857)
NCT ID: NCT00454857
Last Updated: 2013-11-26
Results Overview
The number of participants who received ITP therapies for first-line treatment during either the retrospective or prospective phases of the study.
COMPLETED
326 participants
Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12).
2013-11-26
Participant Flow
Participant milestones
| Measure |
Patients With ITP
Patients diagnosed with Immune (Idiopathic) Thrombocytopenic Purpura (ITP) were followed prospectively for a period of 12 months.
|
|---|---|
|
Overall Study
STARTED
|
326
|
|
Overall Study
Completed Retrospective Chart Review
|
324
|
|
Overall Study
Completed ≥1 Observational Study Visit
|
319
|
|
Overall Study
COMPLETED
|
272
|
|
Overall Study
NOT COMPLETED
|
54
|
Reasons for withdrawal
| Measure |
Patients With ITP
Patients diagnosed with Immune (Idiopathic) Thrombocytopenic Purpura (ITP) were followed prospectively for a period of 12 months.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
15
|
|
Overall Study
Lost to Follow-up
|
16
|
|
Overall Study
Noncompliance
|
6
|
|
Overall Study
Death
|
5
|
|
Overall Study
Other
|
11
|
|
Overall Study
Reason unknown
|
1
|
Baseline Characteristics
Retrospective & Prospective Observational Study of Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Baseline characteristics by cohort
| Measure |
Patients With ITP
n=326 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
Age Continuous
|
53.4 Years
STANDARD_DEVIATION 18.1 • n=93 Participants
|
|
Sex: Female, Male
Female
|
195 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
131 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12).Population: All enrolled participants
The number of participants who received ITP therapies for first-line treatment during either the retrospective or prospective phases of the study.
Outcome measures
| Measure |
Patients With ITP
n=326 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
Number of Participants Utilizing Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Therapies for First-line Treatment
Corticosteroids
|
128 Participants
|
|
Number of Participants Utilizing Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Therapies for First-line Treatment
IVIG (Intravenous immunoglobulin)
|
37 Participants
|
|
Number of Participants Utilizing Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Therapies for First-line Treatment
Rituximab
|
3 Participants
|
|
Number of Participants Utilizing Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Therapies for First-line Treatment
Anti-D immunoglobulin
|
11 Participants
|
|
Number of Participants Utilizing Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Therapies for First-line Treatment
Splenectomy
|
74 Participants
|
|
Number of Participants Utilizing Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Therapies for First-line Treatment
Other
|
36 Participants
|
PRIMARY outcome
Timeframe: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12).Population: All enrolled participants
The number of participants who received ITP therapies for second-line treatment during either the retrospective or prospective phases of the study.
Outcome measures
| Measure |
Patients With ITP
n=326 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
The Number of Participants Utilizing ITP Therapies for Second-line Treatment.
Corticosteroids
|
98 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Second-line Treatment.
IVIG
|
54 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Second-line Treatment.
Rituximab
|
16 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Second-line Treatment.
Anti-D immunoglobulin
|
16 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Second-line Treatment.
Splenectomy
|
0 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Second-line Treatment.
Other
|
48 Participants
|
PRIMARY outcome
Timeframe: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12).Population: All enrolled participants
The number of participants who received ITP therapies for third-line treatment during either the retrospective or prospective phases of the study.
Outcome measures
| Measure |
Patients With ITP
n=326 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
The Number of Participants Utilizing ITP Therapies for Third-line Treatment.
Corticosteroids
|
47 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Third-line Treatment.
IVIG
|
50 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Third-line Treatment.
Rituximab
|
15 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Third-line Treatment.
Anti-D immunoglobulin
|
14 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Third-line Treatment.
Splenectomy
|
0 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Third-line Treatment.
Other
|
50 Participants
|
PRIMARY outcome
Timeframe: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12).Population: All enrolled participants
The number of participants who received ITP therapies for fourth-line treatment during either the retrospective or prospective phases of the study.
Outcome measures
| Measure |
Patients With ITP
n=326 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
The Number of Participants Utilizing ITP Therapies for Fourth-line Treatment.
Corticosteroids
|
26 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Fourth-line Treatment.
IVIG
|
28 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Fourth-line Treatment.
Rituximab
|
14 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Fourth-line Treatment.
Anti-D immunoglobulin
|
14 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Fourth-line Treatment.
Splenectomy
|
0 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Fourth-line Treatment.
Other
|
56 Participants
|
PRIMARY outcome
Timeframe: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12).Population: All enrolled participants
The number of participants who received ITP therapies for fifth-line treatment during either the retrospective or prospective phases of the study.
Outcome measures
| Measure |
Patients With ITP
n=326 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
The Number of Participants Utilizing ITP Therapies for Fifth-line Treatment
Corticosteriods
|
13 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Fifth-line Treatment
IVIG
|
28 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Fifth-line Treatment
Rituximab
|
10 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Fifth-line Treatment
Anti-D immunoglobulin
|
11 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Fifth-line Treatment
Splenectomy
|
0 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Fifth-line Treatment
Other
|
41 Participants
|
PRIMARY outcome
Timeframe: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12).Population: All enrolled participants
Assessed the number of participants who received ITP therapies for sixth-line treatment during either the retrospective or prospective phases of the study.
Outcome measures
| Measure |
Patients With ITP
n=326 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
The Number of Participants Utilizing ITP Therapies for Sixth-line Treatment.
Corticosteriods
|
16 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Sixth-line Treatment.
IVIG
|
25 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Sixth-line Treatment.
Rituximab
|
7 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Sixth-line Treatment.
Anti-D immunoglobulin
|
8 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Sixth-line Treatment.
Splenectomy
|
0 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Sixth-line Treatment.
Other
|
34 Participants
|
PRIMARY outcome
Timeframe: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12).Population: All enrolled participants
The number participants who received ITP therapies as seventh or greater-line treatment during either the retrospective or prospective phases of the study.
Outcome measures
| Measure |
Patients With ITP
n=326 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
The Number of Participants Utilizing ITP Therapies for Seventh or Greater-line Treatment.
Corticosteroids
|
30 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Seventh or Greater-line Treatment.
IVIG
|
33 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Seventh or Greater-line Treatment.
Rituximab
|
19 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Seventh or Greater-line Treatment.
Anti-D immunoglobulin
|
11 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Seventh or Greater-line Treatment.
Splenectomy
|
0 Participants
|
|
The Number of Participants Utilizing ITP Therapies for Seventh or Greater-line Treatment.
Other
|
41 Participants
|
PRIMARY outcome
Timeframe: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12).Population: All enrolled participants
The number of participants who received ITP therapies for treatments with unknown starting date during either the retrospective or prospective phases of the study.
Outcome measures
| Measure |
Patients With ITP
n=326 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
The Number of Participants Utilizing ITP Therapies: Treatments With Unknown Starting Date.
Corticosteroids
|
25 Participants
|
|
The Number of Participants Utilizing ITP Therapies: Treatments With Unknown Starting Date.
IVIG
|
4 Participants
|
|
The Number of Participants Utilizing ITP Therapies: Treatments With Unknown Starting Date.
Rituximab
|
0 Participants
|
|
The Number of Participants Utilizing ITP Therapies: Treatments With Unknown Starting Date.
Anti-D immunoglobulin
|
0 Participants
|
|
The Number of Participants Utilizing ITP Therapies: Treatments With Unknown Starting Date.
Splenectomy
|
4 Participants
|
|
The Number of Participants Utilizing ITP Therapies: Treatments With Unknown Starting Date.
Other
|
17 Participants
|
SECONDARY outcome
Timeframe: Baseline to month 12 during prospective data collection phasePopulation: The Patient Reported Outcome (PRO) Analysis Set includes all participants who completed at least one questionnaire during the prospective observation period.
The Immune Thrombocytopenic Purpura Patient Assessment Questionnaire (ITP-PAQ) was developed to assess disease-specific quality of life (QoL) in adults with ITP. It is a 44-item questionnaire that includes scales for physical health (symptoms, fatigue/sleep, bother, and activity), emotional health (psychological and fear), overall QoL, social activity, women's reproductive health, and work. Scores for each scale range from 0 (worst) to 100 (best).
Outcome measures
| Measure |
Patients With ITP
n=274 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Physical Health-Symptoms score
|
6.7 Units on a scale
Standard Deviation 17.1
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Physical Health - Fatigue score
|
7.2 Units on a scale
Standard Deviation 23.7
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Physical Health - Bother score
|
9.4 Units on a scale
Standard Deviation 22
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Physical Health - Activity score
|
9 Units on a scale
Standard Deviation 24.5
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Emotional Health - Psychological score
|
6.4 Units on a scale
Standard Deviation 21.2
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Emotional Health - Fear score
|
4 Units on a scale
Standard Deviation 16
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Quality of Life score
|
9.2 Units on a scale
Standard Deviation 21.9
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Social Quality of Life score
|
3.4 Units on a scale
Standard Deviation 15.5
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Woman's Reproductive Health (WRH) score
|
4.7 Units on a scale
Standard Deviation 17
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
WRH: Menstrual Symptoms score
|
5.9 Units on a scale
Standard Deviation 22.4
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Woman's Reproductive Health: Fertility score
|
4.2 Units on a scale
Standard Deviation 19
|
|
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)
Work Quality of Life score
|
3.8 Units on a scale
Standard Deviation 14
|
SECONDARY outcome
Timeframe: Baseline to Month 12 during prospective data collection phasePopulation: The Patient Reported Outcome (PRO) Analysis Set includes all subjects who completed at least one questionnaire during the prospective observation period.
The EQ VAS records the respondent's self-rated health on a vertical, visual analogue scale where the endpoints are labelled 'Best imaginable health state' (score = 100) and 'Worst imaginable health state' (score = 0).
Outcome measures
| Measure |
Patients With ITP
n=274 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
Change From Baseline to Month 12 in Health-related Quality of Life Assessed by EuroQol Visual Analog Scale (EQ-5D VAS)
|
4.1 Units on a scale
Standard Deviation 18.00
|
SECONDARY outcome
Timeframe: Baseline to Month 12 during prospective data collection phasePopulation: The Patient Reported Outcome (PRO) Analysis Set includes all participants who completed at least one questionnaire during the prospective observation period.
Participant satisfaction with treatment was measured using the Treatment Satisfaction Questionnaire for Medication (TSQM), an 11-item questionnaire providing scores on four scales - side effects, effectiveness, convenience and global satisfaction. TSQM Scale scores range from 0 to 100 with higher scores indicating more satisfaction with treatment.
Outcome measures
| Measure |
Patients With ITP
n=274 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
Change From Baseline to Month 12 in Treatment Satisfaction
Overall Satisfaction score
|
1.8 Units on a scale
Standard Deviation 19.40
|
|
Change From Baseline to Month 12 in Treatment Satisfaction
Efficacy score
|
2.8 Units on a scale
Standard Deviation 21.02
|
|
Change From Baseline to Month 12 in Treatment Satisfaction
Side Effects score
|
7.9 Units on a scale
Standard Deviation 20.79
|
|
Change From Baseline to Month 12 in Treatment Satisfaction
Convenience score
|
0.3 Units on a scale
Standard Deviation 17.74
|
SECONDARY outcome
Timeframe: 12 months (prospective data collection phase)Population: All enrolled participants who completed at least 1 observational study visit during the prospective phase.
The number of participants who received drug therapies for the treatment of ITP during the prospective phase of the study. Use of multiple medications by the same participants is possible.
Outcome measures
| Measure |
Patients With ITP
n=319 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
Anti-D
|
14 Participants
|
|
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
Azathioprine
|
14 Participants
|
|
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
Corticosteroids-IV (Intravenous)
|
7 Participants
|
|
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
Corticosteroids-Oral
|
49 Participants
|
|
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
Cyclophosphamide
|
4 Participants
|
|
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
Danazol
|
19 Participants
|
|
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
IVIG
|
42 Participants
|
|
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
Rituximab
|
25 Participants
|
|
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
Vincristine/Vinblastine
|
1 Participants
|
|
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
Other
|
43 Participants
|
SECONDARY outcome
Timeframe: 12 months (prospective data collection phase)Population: All enrolled participants who completed at least 1 observational study visit during the prospective phase. N=number of participants using each medication.
Duration of exposure to each ITP medication measured from enrollment until the end of the 12-month data collection phase.
Outcome measures
| Measure |
Patients With ITP
n=319 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
Duration of Exposure to ITP Medication
Azathioprine (N=13)
|
7.3 Months
Standard Deviation 5.18
|
|
Duration of Exposure to ITP Medication
Corticosteriods-Intravenous (N=7)
|
0.2 Months
Standard Deviation 0.41
|
|
Duration of Exposure to ITP Medication
Corticosteriods-Oral (N=37)
|
3.6 Months
Standard Deviation 3.78
|
|
Duration of Exposure to ITP Medication
Cyclophosphamide (N=4)
|
1.5 Months
Standard Deviation 1.59
|
|
Duration of Exposure to ITP Medication
Danazol (N=18)
|
7.5 Months
Standard Deviation 4.27
|
|
Duration of Exposure to ITP Medication
Intravenous immunoglobulin (N=41)
|
0.7 Months
Standard Deviation 2.00
|
|
Duration of Exposure to ITP Medication
Rituximab (N=24)
|
0.7 Months
Standard Deviation 0.44
|
|
Duration of Exposure to ITP Medication
Other (N=42)
|
6.6 Months
Standard Deviation 6.57
|
|
Duration of Exposure to ITP Medication
Anti-D immunoglobulin (N=14)
|
2.0 Months
Standard Deviation 3.90
|
|
Duration of Exposure to ITP Medication
Vincristine/Vinblastine (N=1)
|
0.0 Months
Standard Deviation NA
Only 1 participant with data
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: All enrolled participants who completed at least 1 observational study visit during the prospective phase.
The number of participants who required a splenectomy during the 12-month prospective phase of the study.
Outcome measures
| Measure |
Patients With ITP
n=319 Participants
Patients diagnosed with ITP were followed prospectively for a period of 12 months.
|
|---|---|
|
Number of Participants Requiring Splenectomy
|
15 Participants
|
Adverse Events
Overall Study
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Study Director
Amgen Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results aftercompletion. The Agreement permits Amgen a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Amgen may remove confidential information, but authors have final control and approval of publication content. For multicenter studies, the investigator agrees not to publish any results before the first multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER