ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP
ICON1
ICON1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia
1 other identifier
observational
120
2 countries
29
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Aug 2013
Typical duration for all trials
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 1, 2013
CompletedFirst Submitted
Initial submission to the registry
October 21, 2013
CompletedFirst Posted
Study publicly available on registry
October 29, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2017
CompletedMay 21, 2020
May 1, 2020
3.7 years
October 21, 2013
May 19, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
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 Outcomes (1)
side effects and complications of treatments
1 year
Study Arms (1)
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.
Interventions
The treating physicians will select the second line agent and clinical data will be collected.
Eligibility Criteria
Pediatric refractory ITP patients starting on a new second line therapy
You may qualify if:
- 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 may not qualify 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Boston Children's Hospitallead
- Terrana ITP Research Fundcollaborator
Study Sites (29)
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
Mattel Children's Hospital
Los Angeles, California, 90095, United States
Children's Hospital of Oakland
Oakland, California, 94609, United States
Children's Hospital of Orange County
Orange, California, 92868, United States
Lucile Packard Children's Hospital
Palo Alto, California, 94304, United States
UC Davis Medical Center
Sacramento, California, 95817, United States
UCSF School of Medicine
San Francisco, California, 94143, United States
Colorado Children's Hospital
Denver, Colorado, 80045, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
James Whitcomb Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
St. John Hospital & Medical Center
Detroit, Michigan, 48236, United States
Goryeb Children's Hospital
Morristown, New Jersey, 07960, United States
Columbia University Medical Center
New York, New York, 10032, United States
New York-Presbyterian University Hospital of Columbia and Cornell
New York, New York, 10065, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Oregon Health and Sciences University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Hasbro Children's Hospital
Providence, Rhode Island, 02903, United States
St. Jude's Hospital
Memphis, Tennessee, 38105, United States
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, 75235, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Seattle Children's
Seattle, Washington, 98105, United States
McMaster Children's Hospital
Hamilton, Ontario, L8S 4K1, Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario, KIH8L1, Canada
U. de Montreal CHU St. Justine
Montreal, Quebec, H3T1C5, Canada
Related Publications (2)
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.
PMID: 30945320RESULTGrace 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.
PMID: 29659042RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Principal Investigator, PKD Natural History Study
Study Record Dates
First Submitted
October 21, 2013
First Posted
October 29, 2013
Study Start
August 1, 2013
Primary Completion
April 1, 2017
Study Completion
April 1, 2017
Last Updated
May 21, 2020
Record last verified: 2020-05
Data Sharing
- IPD Sharing
- Will not share