Characteristics, outcome, and response to therapy of multirefractory chronic immune thrombocytopenia

被引：80

作者：

Mahevas, Matthieu ^{[1
]}

Gerfaud-Valentin, Mathieu ^{[1
,2
]}

Moulis, Guillaume ^{[3
,4
,5
]}

Terriou, Louis ^{[6
]}

Audia, Sylvain ^{[7
]}

Guenin, Sophie ^{[1
]}

Le Guenno, Guillaume ^{[8
]}

Salles, Gilles ^{[9
]}

Lambotte, Olivier ^{[10
,11
,12
]}

Limal, Nicolas ^{[1
]}

Viallard, Jean-Francois ^{[13
]}

Cheze, Stephane ^{[14
]}

Tomowiak, Cecile ^{[15
]}

Royer, Bruno ^{[16
]}

Neel, Antoine ^{[17
]}

Debouverie, Odile ^{[18
]}

Hot, Arnaud ^{[19
]}

Durieu, Isabelle ^{[20
]}

Perlat, Antoinette ^{[21
]}

Cliquennois, Manuel ^{[22
]}

Deteix, Clemence ^{[23
]}

Michel, Marc ^{[1
]}

Godeau, Bertrand ^{[1
]}

机构：

[1] Univ Paris Est Creteil, Hop Henri Mondor, AP HP,Serv Med Interne, Ctr Reference Cytopenies Autoimmunes Adulte, Creteil, France

[2] Univ Lyon 1, Hosp Civils Lyon, Hop Croix Rousse, Serv Med Interne, Lyon, France

[3] Ctr Hosp Univ CHU Toulouse, Serv Med Interne, Toulouse, France

[4] Univ Toulouse, INSERM, Unite Mixte Rech UMR 1027, Toulouse, France

[5] CHU Toulouse, Ctr Invest Clin 1436, Toulouse, France

[6] Univ Lille Nord France, Ctr Hosp Reg Univ Lille, Hop Claude Huriez, Serv Med Interne & Immunol Clin, Lille, France

[7] Hop Bocage Cent, CR INSERM 1098, Ctr Competences Cytopenies Autoimmunes Adulte, Serv Med Interne & Immunol Clin, Dijon, France

[8] CHU Clermont Ferrand, Hop Estaing, Serv Med Interne, Clermont Ferrand, France

[9] Univ Lyon 1, CNRS, UMR 5239, Serv Hematol,Ctr Hosp Lyon Sud,Hosp Civils Lyon, Pierre Benite, France

[10] Univ Paris 11, Hop Univ Paris Sud, AP HP, Serv Med Interne Immunol Clin, Le Kremlin Bicetre, France

[11] CEA, Inst Malad Emergentes & Therapies Innovantes DSV, UMR 1184, Div Immunovirol,IDMIT, Le Kremlin Bicetre, France

[12] Ctr Immunol Viral Infect & Autoimmune Dis, INSERM, U1184, Le Kremlin Bicetre, France

[13] CHU Bordeaux, Hop Haut Leveque, Serv Med Interne & Malad Infect, Pessac, France

[14] CHU Cote Nacre, Serv Hematol Clin, Caen, France

[15] CHU Poitiers, Serv Hematol, Poitiers, France

[16] CHU Amiens, Hop Sud, Serv Hematol Clin & Therapie Cellulaire, Amiens, France

[17] CHU Nantes, Serv Med Interne, Nantes, France

[18] CHU Poitiers, Serv Med Interne, Poitiers, France

[19] Univ Lyon 1, Hop Edouard Herriot, Hosp Civils Lyon, Serv Med Interne, Lyon, France

[20] Univ Lyon 1, Hop Lyon Sud, Hosp Civils Lyon, Serv Med Interne, Pierre Benite, France

[21] CHU Rennes, Hop Sud, Serv Med Interne, Pole Accueil Urgences Med Interne Geriat, Rennes, France

[22] Hop St Vincent de Paul, Grp Hosp Inst Catholique Lille, Serv Oncohematol, Lille, France

[23] Univ Grenoble 1, CHU Grenoble, Serv Hematol,Clin Univ Hematol, UMR 5525,CNRS, Grenoble, France

来源：

BLOOD | 2016年 / 128卷 / 12期

关键词：

LONG-TERM OUTCOMES; MYCOPHENOLATE-MOFETIL; CYCLOSPORINE-A; PURPURA; SPLENECTOMY; ITP; CLASSIFICATION; CRITERIA; ADULTS;

D O I：

10.1182/blood-2016-03-704734

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Refractory immune thrombocytopenia (ITP) was previously defined as lack of a minimum response to splenectomy and the requirement for long-term treatment to reduce the risk of significant bleeding events. In this multicenter study, we included 37 patients with multirefractory ITP, defined as no response to splenectomy, rituximab, romiplostim, and eltrombopag. As compared with a historical cohort of 183 ITP patients, matched on the calendar year of ITP diagnosis with a 5:1 ratio, patients with multirefractory ITP were more likely to have secondary ITP (odds ratio [OR], 4.84; 95% confidence interval [CI], 1.31-17.86; P = .018) and monoclonal gammopathy of undetermined significance (OR, 5.94; 95% CI, 1.08-32.48; P = .04). The median duration of ITP before being recognized as multirefractory was 78 months (range, 6-450). The patients showed failure of a median of 10.5 prior treatment lines for ITP (range, 6-15). At the end of follow-up (median, 84 months; range, 12-455), only 1/14 patients achieved response with immunosuppressant therapy alone. By contrast, 7/10 patients achieved response with a combination of immunosuppressant therapy and thrombopoietin-receptor agonists that lasted for a median of 15 months (range, 6-32). Throughout the course of ITP, 5/37 patients died, 3 with ITP (bleeding, n = 2; sepsis n = 1); 15 (40%) had at least 1 bacterial infection and 9 (24%) at least 1 episode of thrombosis. In conclusion, multirefractory ITP was associated with high morbidity and mortality. Combining an immunosuppressant therapy with thrombopoietin-receptor agonists may be a good strategy for management for these patients with severe disease.

引用

页码：1625 / 1630

页数：6

共 24 条

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