Retrospective & Prospective Observational Study of Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Completed

• Conditions: Thrombocytopenia; Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP); Idiopathic Thrombocytopenic Purpura; Thrombocytopenic Purpura
• Interventions: Other: Retrospective Chart Review; Other: Patient-reported Outcome Questionnaires; Other: Physician Survey

• Registration Number: NCT00454857
• Lead Sponsor: Amgen

Brief Summary

This is a multi-center prospective observational descriptive study complemented by a retrospective chart review. Patients diagnosed with ITP and currently treated for ITP by a hematologist or hematologist-oncologist will be recruited from community-based clinics and academic/referral centers. They will be followed prospectively for a period of 12 months. At inception, participants' charts will also be reviewed from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever is less.

Detailed Description

Time Perspective: 12 months prospective chart review study with a retrospective chart review for a period up to 36 months from the start of the study, since the date of diagnosis for ITP.

Study Timeline

• Study Start: 2006-05
• Study First Submitted: 2007-03-29
• Study First Submitted QC: 2007-03-29
• Study First Posted: 2007-04-02
• Primary Completion: 2008-06
• Study Completion: 2008-08
• Results First Submitted: 2009-10-16
• Results First Submitted QC: 2010-04-23
• Results First Posted: 2010-05-12
• Status Verified: 2013-10
• Last Update Submitted: 2013-10-23
• Last Update Posted: 2013-11-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 326

Inclusion Criteria

• Has a diagnosis of ITP according to the American Society of Hematology guidelines (George et al., 1996).
• Is equal to or greater than 18 years of age.
• Is willing and able to complete a series of questionnaires.
• Before any study-specific procedure, the appropriate written informed consent must be obtained.

Exclusion Criteria

• Participated in clinical trial(s) during the past 36 months.
• Is considering participation in a clinical trial within the next 12 months.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with ITP	Physician Survey	Participants with ITP and currently treated for ITP were followed prospectively for a period of 12 months.
Patients with ITP	Retrospective Chart Review	Participants with ITP and currently treated for ITP were followed prospectively for a period of 12 months.
Patients with ITP	Patient-reported Outcome Questionnaires	Participants with ITP and currently treated for ITP were followed prospectively for a period of 12 months.

Primary Outcome Measures

Name	Time	Method
Number of Participants Utilizing Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Therapies for First-line Treatment	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).	The number of participants who received ITP therapies for first-line treatment during either the retrospective or prospective phases of the study.
The Number of Participants Utilizing ITP Therapies for Second-line Treatment.	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).	The number of participants who received ITP therapies for second-line treatment during either the retrospective or prospective phases of the study.
The Number of Participants Utilizing ITP Therapies for Third-line Treatment.	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).	The number of participants who received ITP therapies for third-line treatment during either the retrospective or prospective phases of the study.
The Number of Participants Utilizing ITP Therapies: Treatments With Unknown Starting Date.	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).	The number of participants who received ITP therapies for treatments with unknown starting date during either the retrospective or prospective phases of the study.
The Number of Participants Utilizing ITP Therapies for Fourth-line Treatment.	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).	The number of participants who received ITP therapies for fourth-line treatment during either the retrospective or prospective phases of the study.
The Number of Participants Utilizing ITP Therapies for Sixth-line Treatment.	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).	Assessed the number of participants who received ITP therapies for sixth-line treatment during either the retrospective or prospective phases of the study.
The Number of Participants Utilizing ITP Therapies for Fifth-line Treatment	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).	The number of participants who received ITP therapies for fifth-line treatment during either the retrospective or prospective phases of the study.
The Number of Participants Utilizing ITP Therapies for Seventh or Greater-line Treatment.	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).	The number participants who received ITP therapies as seventh or greater-line treatment during either the retrospective or prospective phases of the study.

Secondary Outcome Measures

Name	Time	Method
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase	12 months (prospective data collection 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.
Duration of Exposure to ITP Medication	12 months (prospective data collection phase)	Duration of exposure to each ITP medication measured from enrollment until the end of the 12-month data collection phase.
Number of Participants Requiring Splenectomy	12 months	The number of participants who required a splenectomy during the 12-month prospective phase of the study.
Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)	Baseline to month 12 during prospective data collection phase	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).
Change From Baseline to Month 12 in Treatment Satisfaction	Baseline to Month 12 during prospective data collection phase	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.
Change From Baseline to Month 12 in Health-related Quality of Life Assessed by EuroQol Visual Analog Scale (EQ-5D VAS)	Baseline to Month 12 during prospective data collection phase	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).