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

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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT00454857

Enrollment

326

Registered

2007-04-02

Start date

2006-05-31

Completion date

2008-08-31

Last updated

2013-11-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Keywords

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

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.

Interventions

OTHERRetrospective Chart Review

Retrospective chart review for up to 36 months of enrollment date.

OTHERPatient-reported Outcome Questionnaires

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).

OTHERPhysician Survey

Physician characteristics to be collected include demographics, years of practice, medical specialty or primary treating physician, and the referral process.

Study design

Observational model

COHORT

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
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 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 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 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 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 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.
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.

Secondary

Measure	Time frame	Description
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).
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.
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).

Participant flow

Participants by arm

Arm	Count
Patients With ITP Patients diagnosed with ITP were followed prospectively for a period of 12 months.	326
Total	326

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Death	5
Overall Study	Lost to Follow-up	16
Overall Study	Noncompliance	6
Overall Study	Other	11
Overall Study	Reason unknown	1
Overall Study	Withdrawal by Subject	15

Baseline characteristics

Characteristic	Patients With ITP
Age Continuous	53.4 Years STANDARD_DEVIATION 18.1
Sex: Female, Male Female	195 Participants
Sex: Female, Male Male	131 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	0 / 0
serious Total, serious adverse events	0 / 0

Outcome results

Primary

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.

Time frame: 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).