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Br J Haematol ; 170(2): 208-17, 2015 Jul.
Article in English | MEDLINE | ID: mdl-25855259

ABSTRACT

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 52 weeks (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 1 year, 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.


Subject(s)
Antibodies, Monoclonal, Murine-Derived/therapeutic use , Immunologic Factors/therapeutic use , Plasma Exchange , Purpura, Thrombotic Thrombocytopenic/therapy , Adult , Antibodies, Monoclonal, Murine-Derived/administration & dosage , Female , Humans , Immunologic Factors/administration & dosage , Male , Middle Aged , Purpura, Thrombotic Thrombocytopenic/diagnosis , Purpura, Thrombotic Thrombocytopenic/mortality , Recurrence , Rituximab , Time Factors , Treatment Outcome
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