Abstract
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.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 221-225 |
| Number of pages | 5 |
| Journal | Pediatric Blood and Cancer |
| Volume | 58 |
| Issue number | 2 |
| DOIs | |
| State | Published - Feb 1 2012 |
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All Science Journal Classification (ASJC) codes
- Pediatrics, Perinatology, and Child Health
- Hematology
- Oncology
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Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia. / 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.
In: Pediatric Blood and Cancer, Vol. 58, No. 2, 01.02.2012, p. 221-225.Research output: Contribution to journal › Article
TY - JOUR
T1 - Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia
AU - Grace, Rachael F.
AU - Bennett, Carolyn M.
AU - Ritchey, A. Kim
AU - Jeng, Michael
AU - Thornburg, Courtney D.
AU - Lambert, Michele P.
AU - Neier, Michelle
AU - Recht, Michael
AU - Kumar, Manjusha
AU - Blanchette, Victor
AU - Klaassen, Robert J.
AU - Buchanan, George R.
AU - Kurth, Margaret Heisel
AU - Nugent, Diane J.
AU - Thompson, Alexis A.
AU - Stine, Kimo
AU - Kalish, Leslie A.
AU - Neufeld, Ellis J.
PY - 2012/2/1
Y1 - 2012/2/1
N2 - 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.
AB - 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.
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U2 - 10.1002/pbc.23130
DO - 10.1002/pbc.23130
M3 - Article
VL - 58
SP - 221
EP - 225
JO - Pediatric Blood and Cancer
JF - Pediatric Blood and Cancer
SN - 1545-5009
IS - 2
ER -