NCT01971684

ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP

ICON1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 120 (actual)
• Sponsor: Boston Children's Hospital · Academic / Other
• Sex: All
• Age: 1 Year – 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to understand physician treatment decisions in selecting specific second line treatments in pediatric ITP and to determine the effectiveness of different second line ITP treatments. Eligible patients are those ages 1-18 years who are starting on a new second line treatment for ITP, defined as any treatment other than IVIG, steroids, anti-D globulin, or aminocaproic acid. Enrolled patients remain on the study for approximately one year.

Detailed description

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: Primary Objectives: 1. To model factors that determine physician treatment decisions in selecting specific second line agents in pediatric ITP. 2. To assess patient reported outcomes with relation to specific second line pediatric ITP therapies. 3. To determine the comparative effectiveness of second line ITP treatments in terms of bleeding and platelet counts. Secondary Objectives: 1. To describe phenotypic variation among patients with refractory ITP; 2. To assess side effects and complications related to specific treatments for refractory ITP; 3. To describe monitoring and follow up practices among pediatric hematologists with each second line agent; 4. To weight factors that physicians use when deciding to treat pediatric ITP patients with second line agents; 5. To determine whether physician perception of patient quality of life correlates with patient derived quality of life measures; 6. To measure the correlation between the ITP Bleeding Scale and the Bleeding Assessment Tool in refractory pediatric ITP patients.

Conditions

• Immune Thrombocytopenia

Interventions

Type	Name	Description
DRUG	Second Line ITP agents	The treating physicians will select the second line agent and clinical data will be collected.

Timeline

• Start date: 2013-08-01
• Primary completion: 2017-04-01
• Completion: 2017-04-01
• First posted: 2013-10-29
• Last updated: 2020-05-21

Locations

29 sites across 2 countries: United States, Canada