Rituximab May Be a Useful Option in Children with Severe Chronic Immunthrombocytopenia (ITP)

Abstract 4692

This otherwise healthy 12-year-old girl presented in March, 2011 with a history of severe ITP that had been diagnosed in November, 2007. Since then, she had suffered from recurrent bruises, petechiae, epistaxis, persistent bleending after tooth-loss and during menstruation (menarche: April, 2010), and subsequent iron-deficiency. Severe headache of sudden onset turned out to be caused by intracranial hemorrhage (ICH) in December, 2009, and in September, 2010, respectively. CT-scans in September 2010 revealed 2 additional ICH which had occured without symptomps. Fortunately, she recovered without sequelae from these complications. The initial and the subsequent treatment consisted of immunoglobulie (IG) and prednisone (IV or orally) given in various doses and for various time-intervals, always followed by a rise in platelet count for short periods of time, and a subsequent decline, when the drugs were withdrawn. Both ICH occured in phases of watchful waiting, while platelet count were below 1000. Splenectomy was offered and repeatedly denied by the patient and her parents.

After the second ICH (Sept 2010) the patient received continuous weekly IG as prophylaxis of further ICH. Serious side-effects of prednisone in this patient (Cushing’s habitus, abdominal striae, insomnia and secondary depression) led to withdrawal of this drug. Since February 2011, the efficacy of IG was decreasing significantly, and the patient was referred to hematological practice for further treatment. We obtained the patient’s and her parents’ informed consent for a trial of 4 weekly doses rituximab (375mg/ square meter body surface after premedication with an antihistamine and 50mg prednisone). The infusions were given over a time-period of 5 weeks (May – June 2011) and were well tolerated. Platelet counts began to rise significantly after the 2. course and have remained above 200 000 since the beginning of July, 2011 without any other medication.

Through the long-term course in this patient yet has to be followed, we feel that Rituximab (though not licensed for ITP in Germany) has been a useful option in this heavily pretreated patient. Because of good clinical results from US trials, like cases confirm previous reports of B.U. Mueller and C.M. Benett (Pediatr. Blood Cancer 2009; 52:259 and Blood 2006; 107:2639) we are now able to perform this treatment.