Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia.
Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia.
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DOI：
10.1002/pbc.23130
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发表时间：
2012-02
影响因子：
3.2
通讯作者：
Neufeld, Ellis J.
中科院分区：
文献类型：
--
作者：
Grace, Rachael F.;Bennett, Carolyn M.;Ritchey, A. Kim;Jeng, Michael;Thornburg, Courtney D.;Lambert, Michele P.;Neier, Michelle;Recht, Michael;Kumar, Manjusha;Blanchette, Victor;Klaassen, Robert J.;Buchanan, George R.;Kurth, Margaret Heisel;Nugent, Diane J.;Thompson, Alexis A.;Stine, Kimo;Kalish, Leslie A.;Neufeld, Ellis J.
儿童免疫性血小板减少症(ITP)的治疗选择是随意的，因为很少有研究能够确定治疗反应的预测因素。利妥昔单抗越来越多地成为那些对其他疗法无效的人的首选治疗方法。本研究的目的是评估单变量和多变量预测血小板计数对利妥昔单抗的反应。在当地IRB批准后，2004年1月至2010年10月期间，565名慢性ITP患者在北美慢性ITP纵向注册中心(NACIR)登记并符合这项研究的标准。治疗反应定义为利妥昔单抗治疗16周内和类固醇治疗14天内的治疗后血小板计数≥50,000/µL。治疗反应数据在登记时进行了回顾性收集，然后进行了前瞻性收集。利妥昔单抗治疗80例(14.2%)，总有效率为63.8%(51/80)。利妥昔单抗疗效的单变量相关因素包括继发性ITP的存在和对类固醇的阳性反应。在多变量分析中，激素反应与利妥昔单抗的反应有很强的相关性，OR6.8(95%CI2.0~23.0，P=0.002)。继发性ITP仍然是利妥昔单抗疗效的有力预测因素，OR5.6(95%可信区间1.1-28.6，P=0.04)。尽管87.5%对类固醇有反应的患者对利妥昔单抗有反应，但对类固醇呈阴性反应的48%的患者对利妥昔单抗有反应。在NACIR中，对类固醇的反应和继发性ITP的存在与对利妥昔单抗的反应有很强的相关性，这一发现以前没有在儿童或成人中报道。
Treatment choice in pediatric immune thrombocytopenia (ITP) is arbitrary, because few studies are powered to identify predictors of therapy response. Increasingly, rituximab is becoming a treatment of choice in those refractory to other therapies. The objective of this study was to evaluate univariate and multivariable predictors of platelet count response to rituximab. After local IRB approval, 565 patients with chronic ITP enrolled and met criteria for this study in the longitudinal, North American Chronic ITP Registry (NACIR) between January 2004 and October 2010. Treatment response was defined as a post-treatment platelet count ≥50,000/µl within 16 weeks of rituximab and 14 days of steroids. Treatment response data were captured both retrospectively at enrollment and then prospectively. Eighty (14.2%) patients were treated with rituximab with an overall response rate of 63.8% (51/80). Univariate correlates of response to rituximab included the presence of secondary ITP and a positive response to steroids. In multivariable analysis, response to steroids remained a strong correlate of response to rituximab, OR 6.8 (95% CI 2.0–23.0, P = 0.002). Secondary ITP also remained a strong predictor of response to rituximab, OR 5.6 (95% CI 1.1–28.6, P =0.04). Although 87.5% of patients who responded to steroids responded to rituximab, 48% with a negative response to steroids did respond to rituximab. In the NACIR, response to steroids and presence of secondary ITP were strong correlates of response to rituximab, a finding not previously reported in children or adults.
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影响因子：
5.1
作者：
Buchanan, GR;Adix, L
通讯作者：
Adix, L
影响因子：
2.4
作者：
DAVIS, PW;WILLIAMS, DA;SHAMBERGER, RC
通讯作者：
SHAMBERGER, RC
影响因子：
2.4
作者：
El-Alfy, MS;El-Tawil, MM;Shahein, N
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Shahein, N
影响因子：
20.3
作者：
Stasi, Roberto;Del Poeta, Giovanni;Arnadori, Sergio
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Arnadori, Sergio
影响因子：
20.3
作者：
Bennett, CM;Rogers, ZR;Neufeld, EJ
通讯作者：
Neufeld, EJ