Children's Hospital of Philadelphia, Philadelphia, Pennsylvania2currently with Alfred I. duPont Hospital for Children, Wilmington, Delaware.
Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia4Leonard Davis Institute of Health Economics, University of Pennsylvania, Philadelphia.
JAMA Pediatr. 2014 Oct;168(10):e142214. doi: 10.1001/jamapediatrics.2014.2214. Epub 2014 Oct 6.
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.
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.
Management type (observation alone vs pharmacotherapy) was determined via medical record review and electronic pharmacy data at diagnosis and within 6 months after diagnosis.
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%).
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.
2011 年，美国血液学会（ASH）发布了儿童免疫性血小板减少症（ITP）管理的更新指南，建议在存在轻度或无出血症状时，无论血小板计数如何，单独观察即可进行管理。对于美国新诊断 ITP 的治疗模式知之甚少。
了解管理建议对治疗模式的影响。
设计、地点和参与者：回顾性病历审查，地点为宾夕法尼亚州费城的费城儿童医院，这是一家位于宾夕法尼亚州费城的大型城市儿科三级护理医院。该研究涉及 311 名新诊断为 ITP 的儿科患者，这些患者于 2007 年 1 月 1 日至 2012 年 12 月 31 日期间接受治疗。
通过病历审查和电子药房数据，在诊断时和诊断后 6 个月内确定治疗类型（单独观察与药物治疗）。
总体而言，44.7%的患者在诊断时接受单独观察治疗，与 2007-2010 年的 34.9%相比，2011 年显著增加至 49.2%（P<0.02），2012 年增加至 71.1%（P<0.001）。在接受治疗的患者中，99%接受了静脉注射免疫球蛋白治疗。多变量逻辑回归分析显示，年龄较小（优势比，0.92；95%置信区间，0.87-0.99）、血小板计数较低（优势比，0.86；95%置信区间，0.83-0.89）和诊断时期较早（2007-2010 年）（优势比，0.17；95%置信区间，0.09-0.34）与增加药物治疗的几率显著相关。在 2010-2012 年期间，也有 20.8%的患者在诊断后 6 个月内接受了治疗。在接受后续药物治疗的患者中，无论是当年还是初始治疗类型，均无显著差异。此外，在诊断时，有 19.6%的患者存在除皮肤瘀斑或瘀点以外的出血症状。诊断时颅内出血很少见（0.6%）。
我们在美国一家儿科护理三级护理医院中证明了新诊断的 ITP 治疗管理方面的重大实践变化，这与儿童 ITP 的 ASH 管理指南修订有关。我们的经验支持为一部分新诊断的儿童 ITP 患者采用单独观察的方法进行治疗。这种治疗方法并没有导致随后治疗的增加或延迟出血症状的增加。
