DOI: 10.1111/bjh.19288 ISSN: 0007-1048
Difficult‐to‐treat primary immune thrombocytopenia in adults: Prevalence and burden. Results from the CARMEN ‐France registry
Guillaume Moulis, Manuela Rueter, Aymeric Duvivier, Matthieu Mahévas, Jean‐François Viallard, Thibault Comont, Stéphane Chèze, Sylvain Audia, Mikaël Ebbo, Louis Terriou, Jean‐Christophe Lega, Pierre‐Yves Jeandel, Ines Hemim, Sylvie Bozzi, Ahmed Daak, Hikaru Okada, Bernard Bonnotte, Marc Michel, Maryse Lapeyre‐Mestre, Bertrand Godeau, - Hematology
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).