TY - JOUR
T1 - Prospective phase 1/2 study of rituximab in childhood and adolescent chronic immune thrombocytopenic purpura
AU - Bennett, Carolyn M.
AU - Rogers, Zora R.
AU - Kinnamon, Daniel D.
AU - Bussel, James B.
AU - Mahoney, Donald H.
AU - Abshire, Thomas C.
AU - Sawaf, Hadi
AU - Moore, Theodore B.
AU - Loh, Mignon L.
AU - Glader, Bertil E.
AU - McCarthy, Maggie C.
AU - Mueller, Brigitta U.
AU - Olson, Thomas A.
AU - Lorenzana, Adonis N.
AU - Mentzer, William C.
AU - Buchanan, George R.
AU - Feldman, Henry A.
AU - Neufeld, Ellis J.
PY - 2006/4/1
Y1 - 2006/4/1
N2 - 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 × 109/L (50 000/ mm3) during 4 consecutive weeks, starting in weeks 9 to 12, was achieved by 11 of 36 patients (31%, confidence interval [CI], 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 third-dose 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.
AB - 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 × 109/L (50 000/ mm3) during 4 consecutive weeks, starting in weeks 9 to 12, was achieved by 11 of 36 patients (31%, confidence interval [CI], 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 third-dose 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.
UR - http://www.scopus.com/inward/record.url?scp=33645515481&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=33645515481&partnerID=8YFLogxK
U2 - 10.1182/blood-2005-08-3518
DO - 10.1182/blood-2005-08-3518
M3 - Article
C2 - 16352811
AN - SCOPUS:33645515481
SN - 0006-4971
VL - 107
SP - 2639
EP - 2642
JO - Blood
JF - Blood
IS - 7
ER -