ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP
Condition: Immune Thrombocytopenia
MeSH terms: Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic
Study type: Observational

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.
    

Intervention name(s): Second Line ITP agents
Intervention type(s): Drug
Intervention description(s): The treating physicians will select the second line agent and clinical data will be collected.
Other intervention name(s): rituximab; 6-mercaptopurine; mycophenolate mofetil; sirolimus; splenectomy; romiplostim; eltrombopag
Intervention MeSH terms: Mycophenolic Acid; Sirolimus; Mercaptopurine
Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Pediatric refractory ITP patients starting on a new second line therapy
      
Keywords: 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