Bennett Carolyn M, Rogers Zora R, Kinnamon Daniel D, Bussel James B, Mahoney Donald H, Abshire Thomas C, Sawaf Hadi, Moore Theodore B, Loh Mignon L, Glader Bertil E, McCarthy Maggie C, Mueller Brigitta U, Olson Thomas A, Lorenzana Adonis N, Mentzer William C, Buchanan George R, Feldman Henry A, Neufeld Ellis J
Division of Hematology/Oncology, Children's Hospital Boston, Boston, MA 02115, USA.
Blood. 2006 Apr 1;107(7):2639-42. doi: 10.1182/blood-2005-08-3518. Epub 2005 Dec 13.
We assessed safety and efficacy of rituximab in a prospective study of 36 patients, age 2.6 to 18.3 years, with severe chronic immune thrombocytopenic purpura (ITP). The primary outcome of sustained platelets above 50 x 10(9)/L (50,000/mm3) during 4 consecutive weeks, starting in weeks 9 to 12, was achieved by 11 of 36 patients (31%, confidence interval [CI], 16% to 48%). Median response time was 1 week (range, 1 to 7 weeks). Attainment of the primary outcome was not associated with age, prior pharmacologic responses, prior splenectomy, ITP duration, screening platelet count, refractoriness, or IgM reduction. First-dose, infusion-related toxicity was common (47%) despite premedication. Significant drug-related toxicities included third-dose hypotension (n = 1) and serum sickness (n = 2). Peripheral B cells were depleted in all subjects. IgM decreased 3.4% per week, but IgG did not significantly decrease. Rituximab was well tolerated, with manageable infusion-related side effects, but 6% of subjects developed serum sickness. Rituximab is beneficial for some pediatric patients with severe, chronic ITP.
我们在一项针对36例年龄在2.6至18.3岁的重度慢性免疫性血小板减少性紫癜（ITP）患者的前瞻性研究中评估了利妥昔单抗的安全性和有效性。在第9至12周开始的连续4周内，血小板持续高于50×10⁹/L（50,000/mm³）这一主要结局，在36例患者中有11例（31%，置信区间[CI]，16%至48%）实现。中位反应时间为1周（范围为1至7周）。主要结局的达成与年龄、既往药物反应、既往脾切除术、ITP病程、筛查时的血小板计数、难治性或IgM降低无关。尽管进行了预处理，但首次剂量输注相关毒性很常见（47%）。显著的药物相关毒性包括第三剂量时的低血压（n = 1）和血清病（n = 2）。所有受试者外周B细胞均被耗竭。IgM每周下降3.4%，但IgG无显著下降。利妥昔单抗耐受性良好，输注相关副作用可控，但6%的受试者出现了血清病。利妥昔单抗对一些重度慢性ITP儿科患者有益。
