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

被引：2

作者：

Moulis, Guillaume ^{[1
,2
,16
]}

Rueter, Manuela ^{[2
]}

Duvivier, Aymeric ^{[3
]}

Mahevas, Matthieu ^{[4
]}

Viallard, Jean-Francois ^{[5
]}

Comont, Thibault ^{[6
]}

Cheze, Stephane ^{[7
]}

Audia, Sylvain ^{[8
]}

Ebbo, Mikael ^{[9
]}

Terriou, Louis ^{[10
]}

Lega, Jean-Christophe ^{[11
]}

Jeandel, Pierre-Yves ^{[12
]}

Hemim, Ines ^{[3
]}

Bozzi, Sylvie ^{[3
]}

Daak, Ahmed ^{[13
]}

Okada, Hikaru ^{[14
]}

Bonnotte, Bernard ^{[8
]}

Michel, Marc ^{[4
]}

Lapeyre-Mestre, Maryse ^{[2
,15
]}

Godeau, Bertrand ^{[4
]}

机构：

[1] Toulouse Univ Hosp, Dept Internal Med, Toulouse, France

[2] Toulouse Univ Hosp, Clin Invest Ctr, Toulouse, France

[3] Sanofi, Chilly Mazarin, France

[4] Creteil Univ Hosp, Natl Referral Ctr Autoimmune Cytopenias, Dept Internal Med, Creteil, France

[5] Bordeaux Univ Hosp, Dept Internal Med, Bordeaux, France

[6] Toulouse Canc Univ Hosp, Dept Internal Med, Toulouse, France

[7] Caen Univ Hosp, Dept Hematol, Caen, France

[8] Dijon Univ Hosp, Dept Internal Med, Dijon, France

[9] Marseille Univ Hosp, Dept Internal Med, Marseille, France

[10] Lille Univ Hosp, Dept Internal Med, Lille, France

[11] Lyon Univ Hosp, Dept Internal Med, Lyon, France

[12] Nice Univ Hosp, Dept Internal Med, Nice, France

[13] Sanofi KK, Tokyo, Japan

[14] Sanofi, Cambridge, MA USA

[15] Toulouse Univ Hosp, Dept Clin Pharmacol, Toulouse, France

[16] Ctr Hosp Univ Toulouse, Serv Med Interne, Pl Docteur Baylac, F-31059 Toulouse 9, France

来源：

BRITISH JOURNAL OF HAEMATOLOGY | 2024年 / 204卷 / 04期

关键词：

difficult-to-treat; epidemiology; immune thrombocytopenia; infection; refractory; thrombosis;

D O I：

10.1111/bjh.19288

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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).

引用

页码：1476 / 1482

页数：7

共 37 条

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