Efficacy and safety of daratumumab in multiresistant immune-mediated thrombotic thrombocytopenic purpura.

Autor: Weisinger J; Service d'hématologie, Centre de Référence des Microangiopathies Thrombotiques (CNR-MAT), Hôpital Saint Antoine, Assistance Publique-Hôpitaux de Paris and Sorbonne Université (AP-HP.6), Paris, France., Bouzid R; Service d'hématologie, Centre de Référence des Microangiopathies Thrombotiques (CNR-MAT), Hôpital Saint Antoine, Assistance Publique-Hôpitaux de Paris and Sorbonne Université (AP-HP.6), Paris, France.; INSERM Unité Mixte de Recherche (UMRS) 1138, Centre de Recherche des Cordeliers, Paris, France., Ranta D; Service d'Hématologie, Centre Hospitalier Universitaire de Nancy, Nancy, France., Woaye-Hune P; Centre Hospitalier Départemental Vendée, La Roche sur Yon, France., Cohen-Aubart F; Départment de Médecine Interne 2, Hôpital Pitié-Salpêtrière, Assistance Publique-Hôpitaux de Paris and Sorbonne Université (AP-HP.6), Paris, France., Gaible C; Néphrologie et Transplantation d'organes, Centre Hospitalier Universitaire Rangueil, Toulouse, France., Marjanovic Z; Service d'hématologie, Centre de Référence des Microangiopathies Thrombotiques (CNR-MAT), Hôpital Saint Antoine, Assistance Publique-Hôpitaux de Paris and Sorbonne Université (AP-HP.6), Paris, France., Corre E; Service d'hématologie, Centre de Référence des Microangiopathies Thrombotiques (CNR-MAT), Hôpital Saint Antoine, Assistance Publique-Hôpitaux de Paris and Sorbonne Université (AP-HP.6), Paris, France., Joly AC; Pharmacie, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris, Paris, France., Baylatry MT; Pharmacie, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris, Paris, France., Joly BS; Service d'hématologie, Centre de Référence des Microangiopathies Thrombotiques (CNR-MAT), Hôpital Saint Antoine, Assistance Publique-Hôpitaux de Paris and Sorbonne Université (AP-HP.6), Paris, France.; INSERM Unité Mixte de Recherche (UMRS) 1138, Centre de Recherche des Cordeliers, Paris, France.; Service d'Hématologie Biologique, Hôpital Lariboisière, Assistance Publique-Hôpitaux de Paris Nord, Université Paris Cité, Paris, France., Veyradier A; Service d'hématologie, Centre de Référence des Microangiopathies Thrombotiques (CNR-MAT), Hôpital Saint Antoine, Assistance Publique-Hôpitaux de Paris and Sorbonne Université (AP-HP.6), Paris, France.; INSERM Unité Mixte de Recherche (UMRS) 1138, Centre de Recherche des Cordeliers, Paris, France.; Service d'Hématologie Biologique, Hôpital Lariboisière, Assistance Publique-Hôpitaux de Paris Nord, Université Paris Cité, Paris, France., Coppo P; Service d'hématologie, Centre de Référence des Microangiopathies Thrombotiques (CNR-MAT), Hôpital Saint Antoine, Assistance Publique-Hôpitaux de Paris and Sorbonne Université (AP-HP.6), Paris, France.; INSERM Unité Mixte de Recherche (UMRS) 1138, Centre de Recherche des Cordeliers, Paris, France.
Jazyk: angličtina
Zdroj: British journal of haematology [Br J Haematol] 2024 Sep 04. Date of Electronic Publication: 2024 Sep 04.
DOI: 10.1111/bjh.19752
Abstrakt: The immunosuppressive treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP) in patients with intolerance or refractoriness to the B-cell depleting monoclonal antibody rituximab remains debated. Daratumumab, a plasma cell-directed monoclonal antibody targeting CD38, represents a therapeutic option, but data are scarce. The French Thrombotic Microangiopathies Reference Center conducted a nationwide survey on iTTP patients treated with daratumumab. Nine episodes from seven patients were identified. Treatment was administered for A Disintegrin And Metalloproteinase with ThromboSpondin-1 motifs, 13th member (ADAMTS13) relapses while patients were otherwise in clinical response (N = 8), or during the acute phase of the disease following rituximab intolerance (N = 1). Patients have received a median of three previous therapeutic lines. ADAMTS13 activity improved in eight cases following daratumumab administration, including three cases where ADAMTS13 normalized. ADAMTS13 relapses occurred in three patients; in two cases, retreatment with daratumumab was successful. Median ADAMTS13 relapse-free survival was not reached; 12-month ADAMTS13 relapse-free survival was 56%. Daratumumab-related adverse events occurred in five cases and were non-severe infusion-related reactions in all cases. These results suggest that daratumumab may be an effective treatment option for iTTP patients with intolerance or refractoriness to rituximab.
(© 2024 The Author(s). British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.)
Databáze: MEDLINE
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