To assess the efficacy and safety of rituximab in adults responding poorly to standard treatment for severe autoimmune thrombotic thrombocytopenic purpura.
Open-label prospective study. Outcomes in the survivors were compared to those of 53 historical survivors who were given therapeutic plasma exchange alone or with vincristine.
Hospitals belonging to the Reference Network for Thrombotic Microangiopathies in France.
Twenty-two adults with either no response or a disease exacerbation when treated with intensive therapeutic plasma exchange.
Add-on rituximab therapy, four infusions over 15 days.
One patient died despite two rituximab infusions. In the rituximab-treated patients, the time to a durable remission was significantly shortened (p = .03), although the plasma volume required to achieve a durable remission was not significantly different compared to the controls. Platelet count recovery occurred within 35 days in all 21 survivors, compared to only 78% of the historical controls (p < .02). Of the rituximab-treated patients, none had a relapse within the first year but three relapsed later on. In patients treated with rituximab, a rapid and profound peripheral B-cell depletion was produced, lasting for 9 months and correlating with higher a disintegrin and metalloproteinase with thrombospondin-13 activity and lower anti-a disintegrin and metalloproteinase with thrombospondin-13 antibody titers. These differences were no longer significant after 12 months. No severe side effects occurred.
Adults with severe thrombocytopenic purpura who responded poorly to therapeutic plasma exchange and who were treated with rituximab had shorter overall treatment duration and reduced 1-yr relapses than historical controls.
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From the Service d'Hématologie Clinique et de Thérapie Cellulaire (AF, J-PV), AP-HP, Groupe Hospitalier Pitié-Salpétrière, UPMC Univ Paris 6, Paris, France; Département d'Hématologie (MB, SM, PC), Service de Réanimation Médicale (BG), AP-HP, Hôpital Saint-Antoine, UPMC Univ Paris 6, Paris, France; Service d'Hématologie Biologique (AV), AP-HP, Hôpital Antoine Béclère, Clamart, et U770 INSERM, Université Paris-Sud 11, Le Kremlin-Bicêtre, France; Service d'Hémaphérèse (PP), Hôpital de la Conception, Marseille, France; Service de Néphrologie (FP), Hôpital Albert Calmette, Lille, France; INSERM UMR S 707 (MS), and UPMC, Univ Paris 06, Paris, France; Service d'Immunopathologie (LG), Service de Réanimation Polyvalente (EM, EA), AP-HP, Hôpital Saint-Louis, Université Paris 7 Denis Diderot, Paris, France; Service de Néphrologie (PV), Centre Hospitalier de Valenciennes, Valenciennes, France; Service d'Hématologie Clinique et de Thérapie Cellulaire (DB), CHU Dupuytren, Limoges, France; Service de Néphrologie (ER), AP-HP, Hôpital Tenon, UPMC Univ Paris 6, Paris, France; Service de Réanimation Polyvalente (J-PM), AP-HP, Hôpital Cochin, Université Paris 5, Paris, France; Service de Néphrologie (AW), Hôpital Maison Blanche, Reims, France; Service de Réanimation Médicale (KC), Hôpital Charles Nicolle, Rouen, France; Service de Néphrologie-Médecine Interne (GC, CP), Hôpital Sud, Amiens, France; Service de Néphrologie et Immunologie Clinique (JP), CHU Rangueil, Toulouse, France; Service Médecine Interne A (MH), Hôpital Hôtel-Dieu, Nantes, France.
* See also p. 317.
Supported, in part, by grants from the Etablissement Français du Sang (CS/2002/009) and the GIS-Institut des Maladies Rares (GIS MR0428).
This work was presented at the 50th Annual Meeting of the American Society of Hematology (Abstract #890) and the 2010 Congress of the French Society of Hematology.
The authors have not disclosed any potential conflicts of interest.
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