ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP
- Conditions
- Immune Thrombocytopenia
- Interventions
- Drug: Second Line ITP agents
- Registration Number
- NCT01971684
- Lead Sponsor
- Boston Children's Hospital
- Brief 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 120
- 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
- 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Refractory Pediatric ITP Patients Second Line ITP agents Pediatric ITP patients, ages 1-18, starting a new second line ITP therapy, defined as not IVIG, steroids, anti-D, or aminocaproic acid.
- Primary Outcome Measures
Name Time Method change from baseline in patient reported outcomes Enrollment, 1 and 12 months Kids ITP Tool, Memorial Symptom Assessment Scale, Fatigue Scale
change from baseline in bleeding assessment Enrollment, 1, 6, and 12 months ITP Bleeding Scale, Bleeding Assessment Tool
change from baseline in platelet count over 1 year
- Secondary Outcome Measures
Name Time Method side effects and complications of treatments 1 year
Trial Locations
- Locations (29)
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
St. Jude's Hospital
🇺🇸Memphis, Tennessee, United States
James Whitcomb Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Seattle Children's
🇺🇸Seattle, Washington, United States
UCSF School of Medicine
🇺🇸San Francisco, California, United States
Oregon Health and Sciences University
🇺🇸Portland, Oregon, United States
Colorado Children's Hospital
🇺🇸Denver, Colorado, United States
St. John Hospital & Medical Center
🇺🇸Detroit, Michigan, United States
Mattel Children's Hospital
🇺🇸Los Angeles, California, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Children's Hospital of Oakland
🇺🇸Oakland, California, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Lucile Packard Children's Hospital
🇺🇸Palo Alto, California, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Goryeb Children's Hospital
🇺🇸Morristown, New Jersey, United States
New York-Presbyterian University Hospital of Columbia and Cornell
🇺🇸New York, New York, United States
University of Texas Southwestern Medical Center at Dallas
🇺🇸Dallas, Texas, United States
McMaster Children's Hospital
🇨🇦Hamilton, Ontario, Canada
Children's Hospital of Eastern Ontario
🇨🇦Ottawa, Ontario, Canada
U. de Montreal CHU St. Justine
🇨🇦Montreal, Quebec, Canada
Hasbro Children's Hospital
🇺🇸Providence, Rhode Island, United States