ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP

NCT ID: NCT01971684

Last Updated: 2020-05-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

120 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-08-31

Study Completion Date

2017-04-30

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:

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.

Conditions

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Immune Thrombocytopenia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Refractory Pediatric ITP Patients

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

Intervention Type DRUG

The treating physicians will select the second line agent and clinical data will be collected.

Interventions

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Second Line ITP agents

The treating physicians will select the second line agent and clinical data will be collected.

Intervention Type DRUG

Other Intervention Names

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rituximab 6-mercaptopurine mycophenolate mofetil sirolimus splenectomy romiplostim eltrombopag

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
Minimum Eligible Age

1 Year

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Terrana ITP Research Fund

OTHER

Sponsor Role collaborator

Boston Children's Hospital

OTHER

Sponsor Role lead

Responsible Party

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Rachael Grace

Principal Investigator, PKD Natural History Study

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Arkansas Children's Hospital

Little Rock, Arkansas, United States

Site Status

Mattel Children's Hospital

Los Angeles, California, United States

Site Status

Children's Hospital of Oakland

Oakland, California, United States

Site Status

Children's Hospital of Orange County

Orange, California, United States

Site Status

Lucile Packard Children's Hospital

Palo Alto, California, United States

Site Status

UC Davis Medical Center

Sacramento, California, United States

Site Status

UCSF School of Medicine

San Francisco, California, United States

Site Status

Colorado Children's Hospital

Denver, Colorado, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

James Whitcomb Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

St. John Hospital & Medical Center

Detroit, Michigan, United States

Site Status

Goryeb Children's Hospital

Morristown, New Jersey, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

New York-Presbyterian University Hospital of Columbia and Cornell

New York, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Oregon Health and Sciences University

Portland, Oregon, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Hasbro Children's Hospital

Providence, Rhode Island, United States

Site Status

St. Jude's Hospital

Memphis, Tennessee, United States

Site Status

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

Seattle Children's

Seattle, Washington, United States

Site Status

McMaster Children's Hospital

Hamilton, Ontario, Canada

Site Status

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Site Status

U. de Montreal CHU St. Justine

Montreal, Quebec, Canada

Site Status

Countries

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United States Canada

References

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Grace RF, Shimano KA, Bhat R, Neunert C, Bussel JB, Klaassen RJ, Lambert MP, Rothman JA, Breakey VR, Hege K, Bennett CM, Rose MJ, Haley KM, Buchanan GR, Geddis A, Lorenzana A, Jeng M, Pastore YD, Crary SE, Neier M, Neufeld EJ, Neu N, Forbes PW, Despotovic JM. Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol. 2019 Jul;94(7):741-750. doi: 10.1002/ajh.25479. Epub 2019 Apr 29.

Reference Type RESULT
PMID: 30945320 (View on PubMed)

Grace RF, Despotovic JM, Bennett CM, Bussel JB, Neier M, Neunert C, Crary SE, Pastore YD, Klaassen RJ, Rothman JA, Hege K, Breakey VR, Rose MJ, Shimano KA, Buchanan GR, Geddis A, Haley KM, Lorenzana A, Thompson A, Jeng M, Neufeld EJ, Brown T, Forbes PW, Lambert MP. Physician decision making in selection of second-line treatments in immune thrombocytopenia in children. Am J Hematol. 2018 Jul;93(7):882-888. doi: 10.1002/ajh.25110. Epub 2018 May 6.

Reference Type RESULT
PMID: 29659042 (View on PubMed)

Other Identifiers

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P00008709

Identifier Type: -

Identifier Source: org_study_id

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