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Multicenter Cohort Study Comparing U.S. Management of Inpatient Pediatric Immune Thrombocytopenia to Current
Char M Witmer1, Michele P Lambert1, Sarah H O'Brien2
1Divisions of Hematology, Departments of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Insights
Pediatric immune thrombocytopenia (ITP) treatment guidelines recommend watchful waiting for patients without bleeding. However, most hospitalized children with ITP still receive medication, indicating a gap between guidelines and practice.
Area of Science:
- Pediatric Hematology
- Clinical Practice Guidelines
- Observational Medicine
Background:
- Recent guidelines for pediatric immune thrombocytopenia (ITP) advocate for an observational approach in patients without significant bleeding, irrespective of platelet count.
- This shift aims to reduce unnecessary interventions in children with ITP.
Purpose of the Study:
- To evaluate the impact of updated pediatric ITP guidelines on clinical practice patterns.
- To compare treatment approaches before and after the guideline changes in hospitalized pediatric ITP patients.
Main Methods:
- Retrospective analysis of the Pediatric Health Information Systems (PHIS) database from January 2008 to September 2014.
- Inclusion criteria: patients aged 6 months to 18 years with primary ITP diagnosis.
- Comparison of clinical management (pharmacologic therapy, bone marrow procedures, length of stay) between preguideline and postguideline admission periods.
Main Results:
- Over 4,900 pediatric ITP patients were analyzed; 93.4% received pharmacologic treatment, yet only 14.2% had documented significant bleeding.
- No significant change in pharmacologic treatment rates (92.9% vs. 94.1%) was observed post-guidelines.
- A decrease in bone marrow procedures (9.7% to 6.4%) and length of stay (2.3 to 2 days) was noted.
Conclusions:
- Despite evidence supporting watchful waiting for pediatric ITP, a high proportion of hospitalized patients without bleeding receive pharmacologic therapy.
- US inpatient practice patterns for pediatric ITP remain largely inconsistent with current guidelines.
- Further efforts are required to understand and bridge the discrepancy between ITP treatment guidelines and clinical practice.
Background:
Recent pediatric immune thrombocytopenia (ITP) guidelines have significantly altered and are encouraging an observational approach for patients without significant bleeding regardless of their platelet count.
Procedure:
This retrospective multicenter cohort study utilized the Pediatric Health Information Systems (PHIS) administrative database. Subjects were 6 months to 18 years of age, admitted to a PHIS hospital between January 1, 2008 and September 30, 2014, with a primary diagnosis code for ITP. International Classification of Disease, Ninth Revision, Clinical Modification Code (ICD-9-CM) discharge codes identified significant bleeding. Pharmaceutical billing codes identified the use of pharmacologic therapy for ITP. Clinical management during preguideline admissions (January 1, 2008 to August 31, 2011) was compared to postguideline admissions (September 1, 2011 to September 30, 2014).
Results:
A total of 4,937 subjects met inclusion criteria with a mean age of 6.2 (SD 5) years; 93.4% (4,613/4,937) received pharmacologic treatment for ITP but only 14.2% (699/4,937) had ICD-9-CM codes for significant bleeding; 11.5% (570/4,937) of subjects were readmitted. In comparing pre- versus postguideline time periods, the proportion of subjects receiving ITP pharmacologic treatment did not change (92.9% vs. 94.1%; P = 0.26). A decrease was found in the proportion of bone marrows performed (9.7% vs. 6.4%; P < 0.001) and length of stay (2.3 vs. 2 days; P < 0.001). The proportion of ITP admissions from 2012 to 2014 was modestly decreased when compared to 2008-2010 (12.9 vs. 14.5/10,000 PHIS admissions, P < 0.001).
Conclusions:
Despite guidelines and evidence that supports a watchful waiting approach for pediatric patients with ITP, a large proportion of inpatients without significant bleeding are still receiving pharmacologic therapy. Continued efforts are needed to address why inpatient U.S. practice patterns are so discrepant from current treatment guidelines.

