Childhood ITP: A Single Institution Review of Treatment and Outcomes.

Immune thrombocytopenia purpura (ITP) is diagnosed in approximately 50 per 1 million children per year. Although thrombocytopenia can be associated with serious, life-threatening bleeding, the outcome for most patients with ITP is excellent. Approximately 80% of children with ITP will recover by 6 – 12 months from diagnosis. Large randomized, controlled trials have not been done to demonstrate the optimal management strategy, either for safety or efficacy. Some patients are managed with observation, given the benign ultimate outcome of most cases, but, because of existing or potential bleeding, the majority of patients with severe thrombocytopenia receive treatment with glucocorticoids, IVIG, or anti-D globulin. At our institution, we do not have a standardized strategy for management of patients with childhood ITP. We performed a 10-year retrospective review of our institutional experience with childhood ITP. The approximately 400 eligible patients were aged 0.5 to 18 years, and were managed between January 1996 and January 2006 at St. Louis Children’s Hospital or Washington University Pediatric Hematology/Oncology Clinic. Data from the medical records of these patients was collected and will be analyzed to determine the following primary endpoints:

1. Time to reach target platelet count, stratified by initial therapy type;

2. Rate of re-treatment and/or addition of second therapy over the first 28 days, stratified by initial therapy type and dosage;

3. Incidence of treatment-related toxicities,

4. Incidence of hemorrhage, anemia and/or transfusion, stratified by therapy choice,

5. Rates and duration of hospitalization,

6. Time to resolution of normal platelet count, stratified by initial therapy.

The management and outcome of patients without platelet recovery by 6 and 12 months (i.e. chronic ITP) and will be detailed. Results from this study will be used as a framework for developing institutional guidelines for management of childhood ITP.