Vesely S, Buchanan G R, Cohen A, Raskob G, George J
Department of Epidemiology, University of Oklahoma Health Sciences Center, USA.
J Pediatr Hematol Oncol. 2000 Jan-Feb;22(1):55-61. doi: 10.1097/00043426-200001000-00011.
To assess current physician self-reported practices regarding initial management of childhood idiopathic thrombocytopenic purpura (ITP) and to determine physician self-reported willingness to participate in randomized clinical trials comparing different initial management strategies.
A questionnaire was mailed in November 1997 to all 720 members of the American Society of Pediatric Hematology/Oncology asking how they would diagnose and manage ITP in children 18 months, 5 years, and 15 years of age who were experiencing either dry purpura (cutaneous hemorrhage only) or wet purpura (active mucous membrane hemorrhage). Specific questions dealt with bone marrow aspiration, hospital admittance, treatment strategy, and specific doses of corticosteroids and intravenous immunoglobulin.
The response rate to the questionnaire was 57%. Most respondents indicated they usually perform a bone marrow aspirate when corticosteroids are to be prescribed and administer drug therapy to patients with newly diagnosed ITP with wet or dry purpura. Only 16% of respondents would administer no drug therapy to a child with dry purpura. Intravenous immunoglobulin (IVIG) was preferred to steroids, with anti-D immunoglobulin prescribed less frequently. Hospital admittance often was used for patients with dry purpura and usually recommended for patients with wet purpura. Most respondents expressed willingness to randomize patients with dry purpura to IVIG versus no therapy and those with wet purpura to IVIG versus prednisone as part of a randomized controlled clinical trial.
The self-reported care of the patient with ITP was influenced by the severity of presentation (dry versus wet purpura). Most physicians reported they would administer specific drug treatment in both scenarios. This survey illustrates the diverse diagnostic and management strategies currently used in childhood ITP. Because no one therapeutic approach is predominant and a scientific basis for decision making in childhood ITP has not been developed, future randomized trials are warranted. On the basis of these survey results, such trials are desired by most pediatric hematology/oncology specialists.
评估当前医生自我报告的关于儿童特发性血小板减少性紫癜（ITP）初始管理的做法，并确定医生自我报告的参与比较不同初始管理策略的随机临床试验的意愿。
1997年11月向美国儿科学血液学/肿瘤学会的所有720名成员邮寄了一份问卷，询问他们将如何诊断和管理18个月、5岁和15岁患有单纯性紫癜（仅皮肤出血）或湿性紫癜（有活跃的黏膜出血）的儿童ITP。具体问题涉及骨髓穿刺、住院、治疗策略以及皮质类固醇和静脉注射免疫球蛋白的具体剂量。
问卷的回复率为57%。大多数受访者表示，当开具皮质类固醇药物时，他们通常会进行骨髓穿刺，并对新诊断的患有单纯性或湿性紫癜的ITP患者进行药物治疗。只有16%的受访者不会对患有单纯性紫癜的儿童进行药物治疗。静脉注射免疫球蛋白（IVIG）比类固醇更受青睐，抗-D免疫球蛋白的处方频率较低。患有单纯性紫癜的患者经常住院，而患有湿性紫癜的患者通常建议住院。作为随机对照临床试验的一部分，大多数受访者表示愿意将患有单纯性紫癜的患者随机分为接受IVIG治疗与不治疗组，将患有湿性紫癜的患者随机分为接受IVIG治疗与泼尼松治疗组。
ITP患者的自我报告护理受到表现严重程度（单纯性紫癜与湿性紫癜）的影响。大多数医生报告说，在两种情况下他们都会进行特定的药物治疗。这项调查说明了目前在儿童ITP中使用的多种诊断和管理策略。由于没有一种治疗方法占主导地位，并且尚未建立儿童ITP决策的科学依据，因此未来有必要进行随机试验。根据这些调查结果，大多数儿科血液学/肿瘤学专家都希望进行此类试验。
