Managing ITP in children - update 12-4-14

BACKGROUND: Treatment recommendations for childhood Immune Thrombocytopenia (ITP) in the United States have been a subject of much debate. In early 2011, the American Society of Hematology (ASH) updated their guidelines, recommending that children with ITP be managed with observation alone when there is mild or no bleeding symptoms regardless of platelet count, reserving hospitalization and pharmacologic treatment for those with significant bleeding. The objective of this study was to better understand the impact these recommendations have had on practice patterns at a large, urban, pediatric tertiary care hospital in the United States. METHODS: We examined data from patients aged 0-17 with newly diagnosed ITP seen in inpatient and outpatient locations at the Children's Hospital of Philadelphia (CHOP) between January 1, 2007 and December 31, 2012. A cohort was developed by querying the hospital data system for all patients treated 2007-2012 using the ICD-9 code for ITP (287.31). Chart review was used to exclude patients with alternative diagnosis for thrombocytopenia, diagnosis prior to the time of interest, and initial treatment elsewhere. We compared management strategies at diagnosis and retreatment rates in the first 6 months after the initial diagnosis. The primary outcome, management at diagnosis, was analyzed by logistic regression and chi square analysis. RESULTS: 502 unique patients were identified. After chart review, an evaluable cohort of 313 patients with newly diagnosed ITP remained. The most common reasons for elimination were an alternative diagnosis (n=41), diagnosis in a previous year (n=68), and initial treatment at another hospital (n=69). Of those with an alternative diagnosis, Evan's syndrome (n= and ALPS (n=5) were the most common reasons for elimination. New diagnoses per year ranged from 34-51 in 2007-2009 and 60-63 in 2010-2012. The mean platelet count and age at diagnosis were 16.9 x10^9/l and 6.22 years old, respectively. Distribution was skewed toward lower platelet count and younger age. Overall, 19.5% of patients were noted to have bleeding symptoms beyond bruising or petechiae. The most common bleeding symptoms at presentation were epistaxis (n=25), wet purpura (n=20), gastrointestinal bleeding (n=5), hematuria (n=4), and menstrual bleeding (n=4). Intracranial hemorrhage was rare (n=2, 0.6%). Overall, 55% of patients were managed with pharmacologic treatment at diagnosis. Of those treated, 98% were treated with IVIG. Additionally, the proportion of patients observed at diagnosis rose significantly during this time period from 34% of patients in 2007-2010 to 49.2% in 2011 (p<.02) and 72% in 2012 (p<0.001). Via logistic regression, younger age, lower platelet count, and year of diagnosis were significantly associated with increased odds of pharmacologic treatment. During 2010-2012, 21% of patients were also treated within 0.25-6 months after diagnosis, with no significant difference by year or initial management (p>0.5). The majority treated after diagnosis were due to “low platelet counts” (n=14), bruising/wet purpura (n=6), or parental anxiety/activity level (n=3). One child, initially treated with IVIG, presented with ICH secondary to trauma 1.5 months after diagnosis. CONCLUSIONS: Over time, a significantly increased proportion of patients were observed at diagnosis. Though we cannot prove causation, this change demonstrates a strong association between timing of ASH recommendations and increase in observation rates. As we increased the proportion of children being observed at diagnosis we did not see an increase in the proportion of children receiving treatment or those with significant bleeding symptoms in the following 6 months. Additionally, we found that 37% of patients originally identified via ICD-9 code did not meet our criteria for evaluation of ‘newly diagnosed ITP'. As there is a greater shift to observing patients, along with the development of outpatient infusion services, inpatient databases could become skewed in regard to ITP. Because of this, caution should be utilized in using ICD-9 code alone to identify new diagnoses, especially when chart review is not accessible. Future studies will be able to identify the changing national trend in admission and management and whether adoption of ASH guidelines is widespread.

ash.confex.com/ash/2013/webprogram/Paper59391.html

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.


Continuing evolution at JAMA pediatrics. [JAMA Pediatr. 2014]

www.ncbi.nlm.nih.gov/pubmed/25288142

Thank you for posting this study Sandi. It increases my confidence that this is the best strategy for us.
I want to re read it later when I have more time, but I am also encouraged in the relatively low incidence of ICH, since ( rightly or wrongly) that it one of my major concerns, considering my daughter is 17 and like most teenagers occasionally does some foolish things.
Can someone clarify for me - " wet" purpura are lesions to the gums, mouth or tongue? I am considering it pretty lucky that I don't know for sure what they are!

Yes, that is what wet purpura is...blood blisters.

I'm glad this was helpful to you.