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

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Prospective Observational Descriptive Study and Retrospective Chart Review of Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Conditions
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Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic Purpura

Intervention / Treatment
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* Other: Retrospective Chart Review
* Other: Patient-reported Outcome Questionnaires
* Other: Physician Survey


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients with ITP<br>Participants with ITP and currently treated for ITP were followed prospectively for a period of 12 months. | Other: Retrospective Chart Review<br>* Retrospective chart review for up to 36 months of enrollment date.<br>Other: Patient-reported Outcome Questionnaires<br>* Quality of lige questionnaires as well as treatment satisfaction questionnaires are required monthly. Includes QOL, ITP-PAQ (immune thrombocytopenic purpura - patient assessment questionnaire), EQ-5D, and TSQM (Treatment Satisfaction Questionnaire for Medication).<br>Other: Physician Survey<br>* Physician characteristics to be collected include demographics, years of practice, medical specialty or primary treating physician, and the referral process.<br>|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Thrombocytopenia, Chart Review, Platelet, Retrospective, Prospective, Non-interventional, Health Resource Utilization (HRU), Quality of Life (QOL), Immune Thrombocytopenic Purpura (ITP), Idiopathic Thrombocytopenic Purpura (ITP), Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Observational