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Eligibility
for people ages 1 year to 18 years
Location
at San Francisco, California and other locations
Dates
study started
estimated completion:

Description

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.

Official Title

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

Details

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.

Keywords

Immune Thrombocytopenia 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 Mycophenolate mofetil 6-Mercaptopurine

Eligibility

You can join if…

Open to people ages 1 year to 18 years

  • 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

You CAN'T join if...

  • 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

Locations

  • Children's Hospital of Oakland
    Oakland, California, 94609, USA
  • Lucile Packard Children's Hospital
    Palo Alto, California, 94304, USA
  • UC Davis Medical Center
    Sacramento, California, 95817, USA
  • Mattel Children's Hospital
    Los Angeles, California, 90095, USA
  • Children's Hospital of Orange County
    Orange, California, 92868, USA

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Boston Children’s Hospital
ID
NCT01971684
Study Type
Observational
Last Updated
January 2017