Background. We previously showed in a prospective study that rituximab appears to be effective in some children and adolescents with severe chronic immune thrombocytopenia. Eleven of 36 patients achieved and maintained platelet counts over 50,000/mm3 within the first 12 weeks. These patients were followed for the next year. Methods. Platelet counts were monitored monthly and all subsequent bleeding manifestations and need for further treatment was noted. Results. Eight of the 11 initial responders maintained a platelet count over 150,000/mm3 without further treatment intervention. Three patients had a late relapse. One initial non-responder achieved a remission after 16 weeks, and two additional patients maintained platelet counts around 50,000/mm3 without the need for further intervention. Conclusions. Rituximab resulted in sustained efficacy with platelet counts of 50,000/mm 3 or higher in 11 of 36 patients (31%).
- Clinical trial
- Immune thrombocytopenia
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health