Clinical trial NCT01971684: ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP
Official title: ICON1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Immune Thrombocytopenia
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
Lead sponsor: Boston Children's Hospital
Sponsor class: OTHER
Collaborators: name: Terrana ITP Research Fund
First posted: 2013-10-29
Enrollment: 120
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pediatric refractory ITP patients starting on a new second line therapy

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: change from baseline in patient reported outcomes
timeFrame: Enrollment, 1 and 12 months
measure: change from baseline in bleeding assessment
timeFrame: Enrollment, 1, 6, and 12 months
measure: change from baseline in platelet count
timeFrame: over 1 year
Secondary outcomes:
measure: side effects and complications of treatments
timeFrame: 1 year
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Los Angeles
country: United States
facility: Mattel Children's Hospital
state: California
city: Oakland
country: United States
facility: Children's Hospital of Oakland
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: San Francisco
country: United States
facility: UCSF School of Medicine
state: California
city: Denver
country: United States
facility: Colorado Children's Hospital
state: Colorado
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: James Whitcomb Riley Hospital for Children
state: Indiana
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Detroit
country: United States
facility: St. John Hospital & Medical Center
state: Michigan
city: Morristown
country: United States
facility: Goryeb Children's Hospital
state: New Jersey
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: New York
country: United States
facility: New York-Presbyterian University Hospital of Columbia and Cornell
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Sciences University
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Providence
country: United States
facility: Hasbro Children's Hospital
state: Rhode Island
city: Memphis
country: United States
facility: St. Jude's Hospital
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center at Dallas
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's
state: Washington
city: Hamilton
country: Canada
facility: McMaster Children's Hospital
state: Ontario
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario
state: Ontario
city: Montreal
country: Canada
facility: U. de Montreal CHU St. Justine
state: Quebec
Study officials:
References:
citation: 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.
citation: 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.