Prospective phase 1/2 study of rituximab in childhood and adolescent chronic immune thrombocytopenic purpura

被引:169
作者
Bennett, CM
Rogers, ZR
Kinnamon, DD
Bussel, JB
Mahoney, DH
Abshire, TC
Sawaf, H
Moore, TB
Loh, ML
Glader, BE
McCarthy, MC
Mueller, BU
Olson, TA
Lorenzana, AN
Mentzer, WC
Buchanan, GR
Feldman, HA
Neufeld, EJ
机构
[1] Childrens Hosp, Dana Farber Canc Inst, Div Hematol Oncol, Boston, MA 02115 USA
[2] Childrens Hosp, Dana Farber Canc Inst, Clin Res Program, Boston, MA 02115 USA
[3] Harvard Univ, Sch Med, Boston, MA 02115 USA
[4] Univ Texas, SW Med Ctr, Dept Pediat, Dallas, TX USA
[5] Cornell Univ, Weill Med Coll, New York, NY USA
[6] Baylor Coll Med, Houston, TX 77030 USA
[7] Emory Univ, Sch Med, Atlanta, GA USA
[8] St Johns Hosp, Van Eslander Canc Ctr, Detroit, MI USA
[9] Univ Calif Los Angeles, Mattel Childrens Hosp, San Francisco, CA USA
[10] Stanford Univ, Stanford, CA 94305 USA
关键词
D O I
10.1182/blood-2005-08-3518
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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/ mm(3)) during 4 consecutive weeks, starting in weeks 9 to 12, was achieved by 11 of 36 patients (31%, confidence interval [Cl], 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 thirddose 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.
引用
收藏
页码:2639 / 2642
页数:4
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