Children's Hospital Boston, Boston, MA, USA.
Pediatr Blood Cancer. 2012 Feb;58(2):216-20. doi: 10.1002/pbc.23112. Epub 2011 Jun 14.
Since pediatric immune thrombocytopenia (ITP) is relatively infrequent, comparisons among clinical studies are critical but have previously been limited by differences in terminology. In 2009, an international working group (IWG) developed consensus criteria to enhance comparability in future studies in adults and children.
We performed a retrospective medical record review of all pediatric ITP patients seen at a single children's hospital with a first visit between 2003 and 2010 and applied both historical (criteria(Hist) ) and IWG (criteria(IWG) ) ITP criteria to available clinical data.
Among the 505 patients seen for ITP over 7 years, 98% could be classified as "acute" or "chronic" ITP using the criteria(Hist) , while only 90.7% could be classified as "newly diagnosed," "persistent," or "chronic" ITP using the criteria(IWG) (P < 0.01). Only 33.7% met criteria(IWG) for severe ITP, whereas 77.4% met criteria(Hist) for severe ITP. A striking difference was that overall response to therapies was lower if the criteria(IWG) were used rather than the criteria(Hist) , particularly for IVIG (55.4% vs. 70%, P = 0.02) and rituximab (35.3% vs. 83.3% P = 0.05). Only 2 subjects (0.4%) met the criteria(IWG) for refractory ITP.
Most ITP patients could easily be classified using the 2009 criteria(IWG) . Limitations to applying the criteria(IWG) included absence of treatment response durations, incomplete definition of pediatric "refractory ITP," and exclusion of secondary ITP. Nevertheless, the criteria(IWG) were more clinically relevant given the reliance on definitions based on bleeding and their ability to be applied prospectively. The utility of using the criteria(IWG) within prospective trials remains to be determined.
由于儿科免疫性血小板减少症（ITP）相对少见，因此比较临床研究至关重要，但以前由于术语不同而受到限制。2009 年，一个国际工作组（IWG）制定了共识标准，以提高成人和儿童未来研究的可比性。
我们对 2003 年至 2010 年间在一家儿童医院就诊的所有儿科 ITP 患者进行了回顾性病历审查，并将历史（标准（Hist））和 IWG（标准（IWG））ITP 标准应用于可用的临床数据。
在 7 年期间因 ITP 就诊的 505 例患者中，98%可以使用标准（Hist）分类为“急性”或“慢性”ITP，而只有 90.7%可以使用标准（IWG）分类为“新诊断”、“持续性”或“慢性”ITP（P<0.01）。仅 33.7%符合标准（IWG）严重 ITP，而 77.4%符合标准（Hist）严重 ITP。一个显著的差异是，如果使用标准（IWG）而不是标准（Hist），总体治疗反应较低，尤其是对于 IVIG（55.4%对 70%，P=0.02）和利妥昔单抗（35.3%对 83.3%，P=0.05）。仅 2 例（0.4%）符合标准（IWG）难治性 ITP。
大多数 ITP 患者可以轻松使用 2009 年标准（IWG）进行分类。应用标准（IWG）的局限性包括缺乏治疗反应持续时间、儿科“难治性 ITP”的定义不完整以及排除继发性 ITP。尽管如此，鉴于基于出血的定义和前瞻性应用的能力，标准（IWG）更具临床相关性。在前瞻性试验中使用标准（IWG）的效用仍有待确定。
