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Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia
Author(s) -
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.
Publication year - 2012
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.23130
Subject(s) - rituximab , medicine , immune thrombocytopenia , univariate analysis , refractory (planetary science) , platelet , multivariate analysis , immunology , lymphoma , physics , astrobiology
Background 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. Methods 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. Results 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. Conclusion 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. Pediatr Blood Cancer 2012; 58: 221–225. © 2011 Wiley Periodicals, Inc.