Characteristics, outcome and response to therapy of multirefractory chronic immune thrombocytopenia (original) (raw)

Blood, 2016

Abstract

Refractory immune thrombocytopenia (ITP) was previously defined as lack of a minimum response to splenectomy and the requirement for long-term treatment to reduce the risk of significant bleeding events. We included, in this multicenter study, 37 patients with multirefractory ITP, defined as no response to splenectomy, rituximab, romiplostim and eltrombopag. As compared with an historical cohort of 183 ITP patients, matched on the calendar year of ITP diagnosis with a 5:1 ratio, patients with multirefractory ITP were more likely to have secondary ITP (OR 4.84, 95% CI [1.31-17.86], p=.018) and monoclonal gammopathy of undetermined significance (OR 5.94, 95% CI [1.08-32.48], p=.04). The median duration of ITP before being recognized as multirefractory was 78 months (range 6-450). The patients showed failure of a median of 10.5 prior treatment lines for ITP (range 6-15). At the end of follow-up (median 84 months [range 12-455]), only 1/14 patients achieved response with immunosuppressa...

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