A phase-II sequential case-series study of all patients presenting to four plasma exchange centres with presumed relapsed/refractory thrombotic thrombocytopenic purpura treated with rituximab

作者：Clark William F^*; Rock Gail; Barth David; Arnold Donald M; Webert Kathyrn E; Yenson Paul R; Kelton John G; Li Lihua; Foley Steven R

来源：British Journal of Haematology, 2015, 170(2): 208-217.

DOI：10.1111/bjh.13408

• ADAMTS13
• plasma exchange
• rituximab
• thrombotic thrombocytopenic purpura
• thrombotic microangiopathy

The primary objective of this phase II study was to evaluate the efficacy of rituximab in the management of adult patients with physician-diagnosed presumed thrombotic thrombocytopenic purpura (TTP); relapsed or refractory. We conducted a multicentre study in four Canadian hospital-based apheresis units. Forty patients with presumed TTP (20 refractory and 20 relapsing) were sequentially enrolled and all received rituximab in a standardized manner. A complete response was documented in 14 of 19 refractory patients by week 8 and 15/16 were alive and in remission at 52weeks (one patient was lost to follow-up, one was a non-responder, and three died). Among relapsing patients, 16/18 had a complete response at week 8 and 18/18 at week 52 (one patient lost to follow-up and one withdrew). At 1year, all relapsing and 85% of refractory patients survived. Of 38/40 patients who had ADMATS13 testing at study entry, 13/19 refractory and 10/19 relapsing patients had ADAMTS13<10% (typical TTP); whereas 6/19 refractory and 9/19 relapsing cases had ADAMTS13>10% (other thrombotic microangiopathy; TMA). Refractory-typical TTP in contrast to refractory-other TMA and all relapsing patients treated with plasma exchange and rituximab, were less likely to be responsive and more likely to die or relapse.

• 出版日期2015-7