ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP

Sponsor

Boston Children's Hospital (Other)

Overall Status

Completed

CT.gov ID

NCT01971684

Collaborator

Terrana ITP Research Fund (Other)

120

Enrollment

Locations

Duration (Months)

4.1

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Immune Thrombocytopenia	Drug: Second Line ITP agents

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:

To model factors that determine physician treatment decisions in selecting specific second line agents in pediatric ITP.
To assess patient reported outcomes with relation to specific second line pediatric ITP therapies.
To determine the comparative effectiveness of second line ITP treatments in terms of bleeding and platelet counts.

Secondary Objectives:

To describe phenotypic variation among patients with refractory ITP;
To assess side effects and complications related to specific treatments for refractory ITP;
To describe monitoring and follow up practices among pediatric hematologists with each second line agent;
To weight factors that physicians use when deciding to treat pediatric ITP patients with second line agents;
To determine whether physician perception of patient quality of life correlates with patient derived quality of life measures;
To measure the correlation between the ITP Bleeding Scale and the Bleeding Assessment Tool in refractory pediatric ITP patients.

Study Design

Study Type:

Observational

Actual Enrollment :

120 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Study Start Date :

Aug 1, 2013

Actual Primary Completion Date :

Apr 1, 2017

Actual Study Completion Date :

Apr 1, 2017

Arms and Interventions

Arm	Intervention/Treatment
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.	Drug: Second Line ITP agents The treating physicians will select the second line agent and clinical data will be collected. Other Names: rituximab 6-mercaptopurine mycophenolate mofetil sirolimus splenectomy romiplostim eltrombopag

Arm

Intervention/Treatment

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.

Drug: Second Line ITP agents

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

Other Names:

rituximab

6-mercaptopurine

mycophenolate mofetil

sirolimus

splenectomy

romiplostim

eltrombopag

Outcome Measures

Primary Outcome Measures

change from baseline in patient reported outcomes [Enrollment, 1 and 12 months]
Kids ITP Tool, Memorial Symptom Assessment Scale, Fatigue Scale
change from baseline in bleeding assessment [Enrollment, 1, 6, and 12 months]
ITP Bleeding Scale, Bleeding Assessment Tool
change from baseline in platelet count [over 1 year]

Secondary Outcome Measures

side effects and complications of treatments [1 year]

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Arkansas Children's Hospital	Little Rock	Arkansas	United States	72202
2	Mattel Children's Hospital	Los Angeles	California	United States	90095
3	Children's Hospital of Oakland	Oakland	California	United States	94609
4	Children's Hospital of Orange County	Orange	California	United States	92868
5	Lucile Packard Children's Hospital	Palo Alto	California	United States	94304
6	UC Davis Medical Center	Sacramento	California	United States	95817
7	UCSF School of Medicine	San Francisco	California	United States	94143
8	Colorado Children's Hospital	Denver	Colorado	United States	80045
9	Children's Healthcare of Atlanta	Atlanta	Georgia	United States	30322
10	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois	United States	60611
11	James Whitcomb Riley Hospital for Children	Indianapolis	Indiana	United States	46202
12	Boston Children's Hospital	Boston	Massachusetts	United States	02115
13	St. John Hospital & Medical Center	Detroit	Michigan	United States	48236
14	Goryeb Children's Hospital	Morristown	New Jersey	United States	07960
15	Columbia University Medical Center	New York	New York	United States	10032
16	New York-Presbyterian University Hospital of Columbia and Cornell	New York	New York	United States	10065
17	Duke University Medical Center	Durham	North Carolina	United States	27710
18	Cincinnati Children's Hospital	Cincinnati	Ohio	United States	45229
19	Nationwide Children's Hospital	Columbus	Ohio	United States	43205
20	Oregon Health and Sciences University	Portland	Oregon	United States	97239
21	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States	19104
22	Hasbro Children's Hospital	Providence	Rhode Island	United States	02903
23	St. Jude's Hospital	Memphis	Tennessee	United States	38105
24	University of Texas Southwestern Medical Center at Dallas	Dallas	Texas	United States	75235
25	Texas Children's Hospital	Houston	Texas	United States	77030
26	Seattle Children's	Seattle	Washington	United States	98105
27	McMaster Children's Hospital	Hamilton	Ontario	Canada	L8S 4K1
28	Children's Hospital of Eastern Ontario	Ottawa	Ontario	Canada	KIH8L1
29	U. de Montreal CHU St. Justine	Montreal	Quebec	Canada	H3T1C5