Even if identical diagnostics, treatment, and risk stratification are implemented, more adults will be stratified to high-risk therapy, which should be considered when comparing pediatric and adult outcomes.

Chronic cold agglutinin disease (CAD) is an acquired autoimmune hemolytic anemia. Previous therapeutic modalities, including alkylating cytostatics, interferon and prednisolone, have been disappointing. However, several case reports and small-scaled studies have demonstrated promising results after treatment with rituximab. We performed a phase II multicentre trial to investigate the effect of rituximab in CAD, including 20 patients studied from October 2002 until April 2003. Thirteen patients had idiopathic CAD and seven patients had CAD associated with a malignant B-cell lymphoproliferative disease. Rituximab was given in doses of 375 mg/m(2) at days 1, 8, 15 and 22. Sixteen patients were followed up for at least 48 weeks. Four patients were excluded after 8, 16, 23 and 28 weeks for reasons unrelated to CAD. Nine patients (45%) responded to the treatment, one with complete response (CR), and eight with partial response. Eight patients relapsed, one patient was still in remission at the end of follow-up. There were no serious rituximab-related side-effects. Our study confirms previous findings of a favourable effect of rituximab in patients with CAD. However, few patients will obtain CR and, in most patients, the effect will be transient.

Key Points• In newly diagnosed ITP, addition of rituximab to dexamethasone yields higher sustained response rates than dexamethasone alone.In this study, we report the results from the largest cohort to date of newly diagnosed adult immune thrombocytopenia patients randomized to treatment with dexamethasone alone or in combination with rituximab. Eligible were patients with platelet counts £25310/L with bleeding symptoms. A total of 133 patients were randomly assigned to either dexamethasone 40 mg/day for 4 days (n 5 71) or in combination with rituximab 375 mg/m 2 weekly for 4 weeks (n 5 62). Patients were allowed supplemental dexamethasone every 1 to 4 weeks for up to 6 cycles. Our primary end point, sustained response (ie, platelets ‡50310 9 /L) at 6 months follow-up, was reached in 58% of patients in the rituximab 1 dexamethasone group vs 37% in the dexamethasone group (P 5 .02). The median follow-up time was 922 days. We found longer time to relapse (P 5 .03) and longer time to rescue treatment (P 5 .007) in the rituximab 1 dexamethasone group. There was an increased incidence of grade 3 to 4 adverse events in the rituximab 1 dexamethasone group (P 5 .04). In conclusion, rituximab 1 dexamethasone induced higher response rates and longer time to relapse than dexamethasone alone. This study is registered at