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. 2024 Apr;204(4):1476-1482.
doi: 10.1111/bjh.19288. Epub 2024 Jan 24.

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

Guillaume Moulis  1   2 , Manuela Rueter  2 , Aymeric Duvivier  3 , Matthieu Mahévas  4 , Jean-François Viallard  5 , Thibault Comont  6 , Stéphane Chèze  7 , Sylvain Audia  8 , Mikaël Ebbo  9 , Louis Terriou  10 , Jean-Christophe Lega  11 , Pierre-Yves Jeandel  12 , Ines Hemim  3 , Sylvie Bozzi  3 , Ahmed Daak  13 , Hikaru Okada  14 , Bernard Bonnotte  8 , Marc Michel  4 , Maryse Lapeyre-Mestre  2   15 , Bertrand Godeau  4 ; CARMEN‐France Investigators Group
Affiliations

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

Guillaume Moulis et al. Br J Haematol. 2024 Apr.

Abstract

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).

Keywords: difficult‐to‐treat; epidemiology; immune thrombocytopenia; infection; refractory; thrombosis.

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References

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