Dexamethasone plus rituximab yields higher sustained response rates than dexamethasone monotherapy in adults with primary immune thrombocytopenia
Francesco Zaja1,*, Michele Baccarani2, Patrizio Mazza3, Monica Bocchia4, Luigi Gugliotta5, Alfonso Zaccaria6, Nicola Vianelli2, Marzia Defina4, Alessia Tieghi5, Sergio Amadori7, Selenia Campagna7, Felicetto Ferrara8, Emanuele Angelucci9, Emilio Usala9, Silvia Cantoni10, Giuseppe Visani11, Antonella Fornaro12, Rita Rizzi13, Valerio De Stefano14, Francesco Casulli3, Marta Lisa Battista1, Miriam Isola15, Franca Soldano15, Enrica Gamba16 and Renato Fanin1
Abstract
Previous observational studies suggest that rituximab may be useful in the treatment of primary immune thrombocytopenia (ITP). This randomized trial investigated rituximab efficacy in previously untreated adult ITP patients with a platelet count 20 x 109/L. One hundred and three patients were randomly assigned to receive 40 mg/day dexamethasone for 4 days with or without 375 mg/m2 rituximab weekly for 4 weeks. Patients refractory to dexamethasone alone received salvage therapy with dexamethasone plus rituximab. Sustained response (i.e. platelet count 50 x 109/L at Month 6 after treatment initiation), evaluable in 101 patients, was higher in patients treated with dexamethasone plus rituximab (n=49) than in those treated with dexamethasone alone (n=52) (63% vs. 36 %, P= 0.004, 95% C.I.: [0.079-0.455]. Patients in the experimental arm showed increased incidences of grade 3-4 adverse events (10% vs. 2%, P=0.082, 95% C.I.: [-0.010-0.175]), but incidences of serious adverse events were similar in both arms (6% vs. 2%, P=0.284, 95% C.I.: [-0.035-0.119]). Dexamethasone plus rituximab was an effective salvage therapy in 56% of patients refractory to dexamethasone. The combination of dexamethasone and rituximab improved platelet counts compared to dexamethasone alone. Thus, combination therapy may represent an effective treatment option before splenectomy.
