ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP

Study Overview
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Brief Summary
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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
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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: 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. Secondary Objectives: 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.

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

Conditions
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Immune Thrombocytopenia

Intervention / Treatment
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* Drug: Second Line ITP agents


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Refractory Pediatric ITP Patients<br>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<br>* The treating physicians will select the second line agent and clinical data will be collected.<br>* Other names: eltrombopag;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| change from baseline in patient reported outcomes | Kids ITP Tool, Memorial Symptom Assessment Scale, Fatigue Scale | Enrollment, 1 and 12 months | 
| change from baseline in bleeding assessment | ITP Bleeding Scale, Bleeding Assessment Tool | Enrollment, 1, 6, and 12 months | 
| change from baseline in platelet count |  | over 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| side effects and complications of treatments |  | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ITP, idiopathic thrombocytopenic purpura, bleeding score, platelet count, health-related quality of life, HRQL, Purpura, Purpura, Thrombocytopenic, Purpura, Thrombocytopenic, Idiopathic, Blood Coagulation Disorders, Hematologic Diseases, Hemorrhage, Skin Manifestations, Thrombocytopenia, Blood Platelet Disorders, Immune System Diseases, Hemorrhagic Disorders, Autoimmune Diseases, Therapeutic Uses