ICON 1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia

Study ID
STU 092013-055

Cancer Related
No

Healthy Volunteers
No

Study Sites

  • Children’s Medical Center (Dallas, Plano, Southlake)

Contact
Anna Winborn
214-456-8185
anna.winborn@childrens.com

Principal Investigator
George Buchanan

Summary

The goal enrollment for the study is 120 individuals with refractory iTP, and longitudinal data will be collected for approximately 1 year for each new second line treatment. Boston Children's Hospital will be the data coordinating center.

Patients starting new second-line treatment and meeting other eligibility criteria will be enrolled at the time of the decision to start treatment. Physicians treating eligible patients will also give their consent to participate by completing the physician data form. if the treating physician chooses not to complete the data form, the patient is not eligible for the study.
Data forms will be completed as follows (see Figure 1 and Table 1):
D1a. Physician will complete forms at the following visits:
1. at study entry
2. 1 month (2-8 weeks) after study entry
3. 6 months (3-[Less Than]9 months) after study entry
4. 12 months (9-14 months) after study entry

D1b. Patients/Parents will complete forms at the following visits:
1. at study entry
2. 1 month (2-8 weeks) after study entry
3. 12 months (9-14 months) after study entry

D1c. Forms will be completed by study personnel at the following visits:
1. at study entry
2. 1 month (2-8 weeks) after study entry
3. 6 months (3-[Less Than]9 months) after study entry
4. 12 months (9-14 months) after study entry

The physician forms and parent forms do not require that the same individual fill out the form at each visit. if a patient on study fails a treatment and begins a new second-line treatment, the patient remains on study for one additional second line treatment. Forms administered at the time points above can be filled out for cycle 1 (study entry cycle) and for cycle 2 (new second-line treatments after the first at study entry). if a patient is starting a new second line agent, then the timeline cycle of the forms restarts; however, the cycle 2 forms are completed instead of the cycle 1 forms (Figure 1).

The bleeding assessment tool (BaT) will be piloted in a subset of the sites participating in the study to address secondary aim 6.

Participant Eligibility

Inclusion Criteria:
1. Immune Thrombocytopenia or Evans Syndrome (with immune neutropenia or direct coombs positivity, but without a history of or current evidence of autoimmune hemolytic anemia)
2. Ages >12 months to <18 years old
3. Starting a new second line therapy defined as any therapy except steroids, IVIG, anti-D globulin, or aminocaproic acid
4. Starting a single agent/treatment (monotherapy) second-line therapy (not including rescue therapies). Patients who are transitioning from one therapy to another may be included if the goal is to switch from one therapy to another.

Inclusion criteria for the physicians
1. Physician treating eligible, consented subjects are eligible to complete survey