Difficult‐to‐treat primary immune thrombocytopenia in adults: Prevalence and burden. Results from the CARMEN‐France registry

• Published in: British journal of haematology 2024-04, Vol.204 (4), p.1476-1482
• Main Authors: Moulis, Guillaume, Rueter, Manuela, Duvivier, Aymeric, Mahévas, Matthieu, Viallard, Jean‐François, Comont, Thibault, Chèze, Stéphane, Audia, Sylvain, Ebbo, Mikaël, Terriou, Louis, Lega, Jean‐Christophe, Jeandel, Pierre‐Yves, Hemim, Ines, Bozzi, Sylvie, Daak, Ahmed, Okada, Hikaru, Bonnotte, Bernard, Michel, Marc, Lapeyre‐Mestre, Maryse, Godeau, Bertrand
• Format: Article
• Language: English
• Subjects: Adaptive immunology; Adult; Benzoates - adverse effects; difficult‐to‐treat; epidemiology; France - epidemiology; Hematology; Human health and pathology; Humans; Hydrazines - adverse effects; Idiopathic thrombocytopenic purpura; immune thrombocytopenia; Immunology; infection; Life Sciences; Patients; Prevalence; Prospective Studies; Purpura, Thrombocytopenic, Idiopathic - chemically induced; Purpura, Thrombocytopenic, Idiopathic - epidemiology; Purpura, Thrombocytopenic, Idiopathic - therapy; Receptors, Fc; Recombinant Fusion Proteins; refractory; Registries; Santé publique et épidémiologie; Thrombopoietin - adverse effects; Thrombosis
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/bjh.19288

Summary The aim of this study was to assess the prevalence and the burden of difficult‐to‐treat primary ITP (pITP), defined by the need for another ITP treatment after romiplostim and eltrombopag. Adult patients were selected in the prospective, real‐world CARMEN‐France registry up to December 2021. Out of 821 adult patients with pITP, 29 had difficult‐to‐treat ITP (3.5%; 95% confidence interval [CI]: 2.3%–4.8% in total; 7.6%; 95% CI: 4.9%–10.2% of patients needing ≥2nd line treatment). The 3‐year cumulative incidence of bleeding, infection and thrombosis was 100%, 24.1% and 13.8% respectively. The median cumulative duration of hospital stays was 31 days (median follow‐up: 30.3 months).