Overview

This trial is active, not recruiting.

Condition immune thrombocytopenia
Treatment second line itp agents
Sponsor Children's Hospital Boston
Collaborator Terrana ITP Research Fund
Start date August 2013
End date December 2015
Trial size 118 participants
Trial identifier NCT01971684, P00008709

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective
Arm
Pediatric ITP patients, ages 1-18, starting a new second line ITP therapy, defined as not IVIG, steroids, anti-D, or aminocaproic acid.
second line itp agents rituximab
The treating physicians will select the second line agent and clinical data will be collected.

Primary Outcomes

Measure
change from baseline in patient reported outcomes
time frame: Enrollment, 1 and 12 months
change from baseline in bleeding assessment
time frame: Enrollment, 1, 6, and 12 months
change from baseline in platelet count
time frame: over 1 year

Secondary Outcomes

Measure
side effects and complications of treatments
time frame: 1 year

Eligibility Criteria

Male or female participants from 1 year up to 18 years old.

Inclusion Criteria: - Immune Thrombocytopenia or Evans Syndrome - Ages > 12 months to <18 years - Starting a new second line therapy as defined as any therapy except IVIG, steroids, anti-D globulin, or aminocaproic acid - Starting a single agent/monotherapy Exclusion Criteria: - Evans Syndrome with a history of or current evidence of autoimmune hemolytic anemia - Unwillingness to be followed for 1 year - Physician providing care is unwilling to participate - Patient is starting multiple second line agents simultaneously

Additional Information

Official title ICON1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia
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.
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Children's Hospital Boston.