Corinna L Schultz1, Nandita Mitra2, Marilyn M Schapira3, Michele P Lambert4. 1. Children's Hospital of Philadelphia, Philadelphia, Pennsylvania2currently with Alfred I. duPont Hospital for Children, Wilmington, Delaware. 2. Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia4Leonard Davis Institute of Health Economics, University of Pennsylvania, Philadelphia. 3. Leonard Davis Institute of Health Economics, University of Pennsylvania, Philadelphia5Perelman School of Medicine, University of Pennsylvania, Philadelphia6Center for Health Equity Research & Promotion, Philadelphia VA Medical Center, Philadelphia, Pennsy. 4. Children's Hospital of Philadelphia, Philadelphia, Pennsylvania5Perelman School of Medicine, University of Pennsylvania, Philadelphia.
Abstract
IMPORTANCE: In 2011, the American Society of Hematology (ASH) published updated guidelines for the management of childhood immune thrombocytopenia (ITP) recommending management with observation alone when there are mild or no bleeding symptoms, regardless of platelet count. Little is known about practice patterns of newly diagnosed ITP in the United States. OBJECTIVE: To understand the impact of management recommendations on practice patterns. DESIGN, SETTING, AND PARTICIPANTS: Retrospective medical record review in the Children's Hospital of Philadelphia, a large, urban, pediatric tertiary care hospital in Philadelphia, Pennsylvania. The study involved 311 pediatric patients with newly diagnosed ITP managed between January 1, 2007, and December 31, 2012. MAIN OUTCOMES AND MEASURES: Management type (observation alone vs pharmacotherapy) was determined via medical record review and electronic pharmacy data at diagnosis and within 6 months after diagnosis. RESULTS: Overall, 44.7% of patients were managed with observation alone at diagnosis, with a significant increase from 34.9% in 2007-2010 to 49.2% in 2011 (P < .02) and 71.1% in 2012 (P < .001). Of those treated, 99% were treated with intravenous immunoglobulin. In multivariable logistic regression, younger age (odds ratio, 0.92; 95% CI, 0.87-0.99), lower platelet count (odds ratio, 0.86; 95% CI, 0.83-0.89), and earlier period (2007-2010) of diagnosis (odds ratio, 0.17; 95% CI, 0.09-0.34) were significantly associated with increased odds of pharmacologic management. During 2010-2012, 20.8% of patients were also treated within 6 months after diagnosis. There was no significant difference by year or initial management type in those who received this later pharmacotherapy. Additionally, 19.6% of patients had documented bleeding symptoms beyond cutaneous bruising or petechiae at diagnosis. Intracranial hemorrhage at diagnosis was rare (0.6%). CONCLUSIONS AND RELEVANCE: We demonstrated a significant practice change in the management of newly diagnosed ITP at a pediatric care tertiary care hospital in the United States surrounding revision of the ASH management guidelines for childhood ITP. Our experience supports adoption of observation alone for a proportion of patients with newly diagnosed childhood ITP. This form of management did not lead to an increase in later treatment or an increase in delayed bleeding symptoms.
IMPORTANCE: In 2011, the American Society of Hematology (ASH) published updated guidelines for the management of childhood immune thrombocytopenia (ITP) recommending management with observation alone when there are mild or no bleeding symptoms, regardless of platelet count. Little is known about practice patterns of newly diagnosed ITP in the United States. OBJECTIVE: To understand the impact of management recommendations on practice patterns. DESIGN, SETTING, AND PARTICIPANTS: Retrospective medical record review in the Children's Hospital of Philadelphia, a large, urban, pediatric tertiary care hospital in Philadelphia, Pennsylvania. The study involved 311 pediatric patients with newly diagnosed ITP managed between January 1, 2007, and December 31, 2012. MAIN OUTCOMES AND MEASURES: Management type (observation alone vs pharmacotherapy) was determined via medical record review and electronic pharmacy data at diagnosis and within 6 months after diagnosis. RESULTS: Overall, 44.7% of patients were managed with observation alone at diagnosis, with a significant increase from 34.9% in 2007-2010 to 49.2% in 2011 (P < .02) and 71.1% in 2012 (P < .001). Of those treated, 99% were treated with intravenous immunoglobulin. In multivariable logistic regression, younger age (odds ratio, 0.92; 95% CI, 0.87-0.99), lower platelet count (odds ratio, 0.86; 95% CI, 0.83-0.89), and earlier period (2007-2010) of diagnosis (odds ratio, 0.17; 95% CI, 0.09-0.34) were significantly associated with increased odds of pharmacologic management. During 2010-2012, 20.8% of patients were also treated within 6 months after diagnosis. There was no significant difference by year or initial management type in those who received this later pharmacotherapy. Additionally, 19.6% of patients had documented bleeding symptoms beyond cutaneous bruising or petechiae at diagnosis. Intracranial hemorrhage at diagnosis was rare (0.6%). CONCLUSIONS AND RELEVANCE: We demonstrated a significant practice change in the management of newly diagnosed ITP at a pediatric care tertiary care hospital in the United States surrounding revision of the ASH management guidelines for childhood ITP. Our experience supports adoption of observation alone for a proportion of patients with newly diagnosed childhood ITP. This form of management did not lead to an increase in later treatment or an increase in delayed bleeding symptoms.