Vesely Sara K, Perdue Jedidiah J, Rizvi Mujahid A, Terrell Deirdra R, George James N
University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma 73190, USA.
Ann Intern Med. 2004 Jan 20;140(2):112-20. doi: 10.7326/0003-4819-140-3-200402030-00012.
Treatment of chronic refractory idiopathic thrombocytopenic purpura is a dilemma because many patients have minimal symptoms, response to treatment is uncertain, and treatments may have serious adverse effects.
To determine the effectiveness of treatments for adult patients with idiopathic thrombocytopenic purpura who have not responded to splenectomy.
English-language reports from 1966 through 2003 that were retrieved from MEDLINE and Reference Update and bibliographies of retrieved articles.
Articles reporting 5 or more total patients were reviewed to select eligible patients. Patients were eligible for inclusion if they were more than 16 years of age, had idiopathic thrombocytopenic purpura for more than 3 months, had a previous splenectomy, and had a platelet count less than 50 x 10(9) cells/L.
Patients were assessed for platelet count response, bleeding complications, duration of follow-up, and death. Complete remission was defined as a normal platelet count with no treatment for more than 3 months and for the duration of follow-up.
90 articles with 656 patients treated with 22 therapies met selection criteria. Azathioprine, cyclophosphamide, and rituximab had the most reported complete responses, but they were reported in only 41 to 109 patients. Reported complete response rates ranged from 17% to 27%, but 36% to 42% of patients had no response with these 3 treatments. Most reports described only platelet count responses; bleeding outcomes were reported in only 63 patients (10%). Only 111 (17%) of the 656 eligible patients had pretreatment platelet counts of less than 10 x 10(9) cells/L. No treatment method was reported in more than 20 patients.
Evidence for the effectiveness of any treatment for patients with idiopathic thrombocytopenic purpura and persistent severe thrombocytopenia after splenectomy is minimal. Potentially effective treatments must be evaluated by randomized, controlled trials to determine both benefit and safety.
慢性难治性特发性血小板减少性紫癜的治疗是一个难题，因为许多患者症状轻微，治疗反应不确定，且治疗可能有严重不良反应。
确定对脾切除无反应的成年特发性血小板减少性紫癜患者的治疗效果。
1966年至2003年的英文报告，从MEDLINE、Reference Update及检索文章的参考文献中获取。
对报告患者总数达5例或更多的文章进行审查以选择符合条件的患者。符合纳入标准的患者需年龄超过16岁，患有特发性血小板减少性紫癜超过3个月，曾接受脾切除术，且血小板计数低于50×10⁹/L。
评估患者的血小板计数反应、出血并发症、随访时间及死亡情况。完全缓解定义为血小板计数正常且在超过3个月及随访期间无需治疗。
90篇文章涉及656例接受22种治疗的患者，符合选择标准。硫唑嘌呤、环磷酰胺和利妥昔单抗报告的完全缓解病例最多，但仅见于41至109例患者。报告的完全缓解率为17%至27%，但这3种治疗中有36%至42%的患者无反应。大多数报告仅描述了血小板计数反应；仅63例患者（10%）报告了出血结局。656例符合条件的患者中只有111例（17%）预处理血小板计数低于10×10⁹/L。没有一种治疗方法在超过20例患者中得到报告。
对于脾切除术后仍持续严重血小板减少的特发性血小板减少性紫癜患者，任何治疗方法有效性的证据都很少。必须通过随机对照试验评估潜在有效的治疗方法，以确定其益处和安全性。
