ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP (ICON1)
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||ICON1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia|
- change from baseline in patient reported outcomes [ Time Frame: Enrollment, 1 and 12 months ] [ Designated as safety issue: No ]Kids ITP Tool, Memorial Symptom Assessment Scale, Fatigue Scale
- change from baseline in bleeding assessment [ Time Frame: Enrollment, 1, 6, and 12 months ] [ Designated as safety issue: No ]ITP Bleeding Scale, Bleeding Assessment Tool
- change from baseline in platelet count [ Time Frame: over 1 year ] [ Designated as safety issue: No ]
- side effects and complications of treatments [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
|Study Start Date:||August 2013|
|Estimated Primary Completion Date:||December 2016 (Final data collection date for primary outcome measure)|
Refractory Pediatric ITP Patients
Pediatric ITP patients, ages 1-18, starting a new second line ITP therapy, defined as not IVIG, steroids, anti-D, or aminocaproic acid.
Drug: Second Line ITP agents
The treating physicians will select the second line agent and clinical data will be collected.
The purpose of this observational study is to model factors that determine physician treatment decisions in selecting specific second line agents in pediatric ITP and to determine the comparative effectiveness of second line ITP treatments by bleeding measures, platelet counts, and patient reported outcome measures. This prospective observational, longitudinal, multicenter cohort study will aim to collect routine clinical care data, quality of life information from patients, and decision making data from clinicians at enrollment and at regular clinical intervals for at least one year. The primary and secondary objectives are as follows:
- To model factors that determine physician treatment decisions in selecting specific second line agents in pediatric ITP.
- To assess patient reported outcomes with relation to specific second line pediatric ITP therapies.
- To determine the comparative effectiveness of second line ITP treatments in terms of bleeding and platelet counts.
- To describe phenotypic variation among patients with refractory ITP;
- To assess side effects and complications related to specific treatments for refractory ITP;
- To describe monitoring and follow up practices among pediatric hematologists with each second line agent;
- To weight factors that physicians use when deciding to treat pediatric ITP patients with second line agents;
- To determine whether physician perception of patient quality of life correlates with patient derived quality of life measures;
- To measure the correlation between the ITP Bleeding Scale and the Bleeding Assessment Tool in refractory pediatric ITP patients.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01971684
|Contact: Rachael F Grace, MD, MMSc||617-919-2144||Rachael.Grace@childrens.harvard.edu|
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