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Preemptive rituximab prevents long-term relapses in immune-mediated thrombotic thrombocytopenic purpura.

Jestin, Matthieu; Benhamou, Ygal; Schelpe, An-Sofie; Roose, Elien; Provôt, François; Galicier, Lionel; Hié, Miguel; Presne, Claire; Poullin, Pascale; Wynckel, Alain; Saheb, Samir; Deligny, Christophe; Servais, Aude; Girault, Stéphane; Delmas, Yahsou; Kanouni, Tarik; Lautrette, Alexandre; Chauveau, Dominique; Mousson, Christiane; Perez, Pierre; Halimi, Jean-Michel; Charvet-Rumpler, Anne; Hamidou, Mohamed; Cathébras, Pascal; Vanhoorelbeke, Karen; Veyradier, Agnès; Coppo, Paul.
Blood; 132(20): 2143-2153, 2018 Nov 15.
Artigo em Inglês | MEDLINE | ID: mdl-30201758
Preemptive rituximab infusions prevent relapses in immune thrombotic thrombocytopenic purpura (iTTP) by maintaining normal ADAMTS13 activity. However, the long-term outcome of these patients and the potential adverse events of this strategy need to be determined. We report the long-term outcome of 92 patients with iTTP in clinical remission who received preemptive rituximab after identification of severe ADAMTS13 deficiency (activity <10%) during the follow-up. Thirty-seven patients had >1 iTTP episode, and the median cumulative relapse incidence before preemptive rituximab was 0.33 episode per year (interquartile range [IQR], 0.23-0.66). After preemptive rituximab, the median cumulative relapse incidence in the whole population decreased to 0 episodes per year (IQR, 0-1.32; P < .001). After preemptive rituximab, ADAMTS13 activity recovery was sustained in 34 patients (37%) during a follow-up of 31.5 months (IQR, 18-65), and severe ADAMTS13 deficiency recurred in 45 patients (49%) after the initial improvement. ADAMTS13 activity usually improved with additional courses of preemptive rituximab. In 13 patients (14%), ADAMTS13 activity remained undetectable after the first rituximab course, but retreatment was efficient in 6 of 10 cases. In total, 14 patients (15%) clinically relapsed, and 19 patients (20.7%) experienced benign adverse effects. Preemptive rituximab treatment was associated with a change in ADAMTS13 conformation in respondent patients. Finally, in the group of 23 historical patients with iTTP and persistently undetectable ADAMTS13 activity, 74% clinically relapsed after a 7-year follow-up (IQR, 5-11). In conclusion, persistently undetectable ADAMTS13 activity in iTTP during remission is associated with a higher relapse rate. Preemptive rituximab reduces clinical relapses by maintaining a detectable ADAMTS13 activity with an advantageous risk-benefit balance.
Selo DaSilva