Watts Raymond G
Division of Hematology-Oncology, University of Alabama at Birmingham and Children's Hospital of Alabama, Birmingham, AL 35233, USA.
Clin Pediatr (Phila). 2004 Oct;43(8):691-702. doi: 10.1177/000992280404300802.
Childhood idiopathic thrombocytopenic purpura (ITP) is a common disorder. However, single-institution, long-term, natural history data are limited. The objective of this paper is to review presenting features, response to therapy, and natural history of ITP treated at a single pediatric academic medical center. A retrospective chart review was made for all children (ages birth-18 years) diagnosed with ITP (ICD 287.3) and treated at the Childrens Hospital of Alabama/University of Alabama at Birmingham between 1993 and 2003. Four hundred nine patients were identified (49% male, 51% female; mean age: 5.85 years; range: 1 month-17 years). There was no seasonal variation of presentation. The mean platelet count was 19k (0-120k). Bone marrow aspiration (BMA) was performed in 72% but altered the diagnosis or therapy in no patient. Treatment consisted of corticosteroids in 256 (92% response), intravenous immunoglobulin (IVIG) in 125 (87% response), Win-Rho D in 58 (91% response), and no therapy in 71 (100% response). Response was defined as increase in platelet count to > 50k. There was no difference in response to any therapy. No patients died. One patient presented with a CNS hemorrhage at presentation, responded to therapy, and survived. Twenty-three of 409 patients (6%) experienced clinical bleeding requiring hospitalization or blood transfusion. Chronic ITP (persistence > 6 months) was noted in 99 patients (24%). Chronic patients presented at an older age (7.8 vs 5.2 years for acute only, p<0.001), and with higher platelet counts (27k vs 17k, p<0.001). The risk of chronic ITP was partially predicted by presenting platelet count > 50k and age > 10 years, or both; 50% of patients presenting with these features developed chronic ITP vs 24% overall rate. Splenectomy was curative in 30/31 (97%) patients. There was no postsplenectomy sepsis. Of 99 patients with chronic ITP, 25 responded to splenectomy, 37 resolved at a mean of 20.3 months after diagnosis (7-96 months), 36 had persistent mild thrombocytopenia (50k-125k), and 1 failed to respond to any treatment including splenectomy. Overall, 91% of cases resolved with therapy or observation. ITP is a common pediatric disease presenting at any age with low morbidity and mortality. Most cases can be managed by pediatricians without hematology referral. Several equally successful therapeutic options exist. Chronic cases present at an older age with higher platelet counts. Up to 50% of cases of chronic ITP will resolve with ongoing follow-up. The overall prognosis in childhood ITP is excellent.
儿童特发性血小板减少性紫癜（ITP）是一种常见疾病。然而，单机构的长期自然病史数据有限。本文的目的是回顾在一家儿科学术医疗中心接受治疗的ITP的临床表现、治疗反应及自然病史。对1993年至2003年间在阿拉巴马大学伯明翰分校儿童医院被诊断为ITP（国际疾病分类代码287.3）并接受治疗的所有儿童（年龄从出生至18岁）进行了回顾性病历审查。共识别出409例患者（49%为男性，51%为女性；平均年龄：5.85岁；范围：1个月至17岁）。发病无季节性差异。平均血小板计数为19k（0至120k）。72%的患者进行了骨髓穿刺，但没有患者的诊断或治疗因此改变。治疗方法包括：256例使用皮质类固醇（有效率92%），125例使用静脉注射免疫球蛋白（IVIG，有效率87%），58例使用温 - 罗D（有效率91%），71例未接受治疗（有效率100%）。有效定义为血小板计数增加至>50k。对任何治疗的反应均无差异。无患者死亡。1例患者在发病时出现中枢神经系统出血，经治疗后存活。409例患者中有23例（6%）经历了需要住院或输血的临床出血。99例患者（24%）被诊断为慢性ITP（持续时间>6个月）。慢性患者发病年龄较大（急性患者仅5.2岁，慢性患者为7.8岁，p<0.001），且血小板计数较高（27k对17k，p<0.001）。慢性ITP的风险部分可通过发病时血小板计数>50k和年龄>10岁，或两者兼有来预测；具有这些特征的患者中50%发展为慢性ITP，而总体发生率为24%。脾切除术对30/31（97%）的患者有效。无脾切除术后败血症。在99例慢性ITP患者中，25例对脾切除术有反应，37例在诊断后平均20.3个月（7至96个月）病情缓解，36例持续存在轻度血小板减少（50k - 125k），1例对包括脾切除术在内的任何治疗均无反应。总体而言，91%的病例通过治疗或观察得到缓解。ITP是一种常见的儿科疾病，可在任何年龄发病，发病率和死亡率较低。大多数病例可由儿科医生处理，无需转诊至血液科。有几种同样有效的治疗选择。慢性病例发病年龄较大，血小板计数较高。高达50%的慢性ITP病例通过持续随访可得到缓解。儿童ITP的总体预后良好。
