Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia

Rachael F. Grace, Carolyn M. Bennett, A. Kim Ritchey, Michael Jeng, Courtney D. Thornburg, Michele P. Lambert, Michelle Neier, Michael Recht, Manjusha Kumar, Victor Blanchette, Robert J. Klaassen, George R. Buchanan, Margaret Heisel Kurth, Diane J. Nugent, Alexis A. Thompson, Kimo Stine, Leslie A. Kalish, Ellis J. Neufeld

Research output: Contribution to journalArticle

17 Citations (Scopus)

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 languageEnglish (US)
Pages (from-to)221-225
Number of pages5
JournalPediatric Blood and Cancer
Volume58
Issue number2
DOIs
StatePublished - Feb 1 2012
Externally publishedYes

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Idiopathic Thrombocytopenic Purpura
Steroids
Pediatrics
Platelet Count
Therapeutics
Registries
Rituximab
Inosine Triphosphate
Research Ethics Committees
Longitudinal Studies

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

Cite this

Grace, R. F., Bennett, C. M., Ritchey, A. K., Jeng, M., Thornburg, C. D., Lambert, M. P., ... Neufeld, E. J. (2012). Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia. Pediatric Blood and Cancer, 58(2), 221-225. https://doi.org/10.1002/pbc.23130
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. / Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia. In: Pediatric Blood and Cancer. 2012 ; Vol. 58, No. 2. pp. 221-225.
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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.",
author = "Grace, {Rachael F.} and Bennett, {Carolyn M.} and Ritchey, {A. Kim} and Michael Jeng and Thornburg, {Courtney D.} and Lambert, {Michele P.} and Michelle Neier and Michael Recht and Manjusha Kumar and Victor Blanchette and Klaassen, {Robert J.} and Buchanan, {George R.} and Kurth, {Margaret Heisel} and Nugent, {Diane J.} and Thompson, {Alexis A.} and Kimo Stine and Kalish, {Leslie A.} and Neufeld, {Ellis J.}",
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Grace, RF, Bennett, CM, Ritchey, AK, Jeng, M, Thornburg, CD, Lambert, MP, Neier, M, Recht, M, Kumar, M, Blanchette, V, Klaassen, RJ, Buchanan, GR, Kurth, MH, Nugent, DJ, Thompson, AA, Stine, K, Kalish, LA & Neufeld, EJ 2012, 'Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia', Pediatric Blood and Cancer, vol. 58, no. 2, pp. 221-225. https://doi.org/10.1002/pbc.23130

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 journalArticle

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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Grace RF, Bennett CM, Ritchey AK, Jeng M, Thornburg CD, Lambert MP et al. Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia. Pediatric Blood and Cancer. 2012 Feb 1;58(2):221-225. https://doi.org/10.1002/pbc.23130