Combining thrombopoietin receptor agonists with immunosuppressive drugs in adult patients with multirefractory immune thrombocytopenia, an update on the French experience

被引:11
作者
Crickx, Etienne [1 ,2 ]
Ebbo, Mikael [3 ]
Riviere, Etienne [4 ,5 ]
Souchaud-Debouverie, Odile [6 ]
Terriou, Louis [7 ]
Audia, Sylvain [8 ]
Ruivard, Marc [9 ,10 ]
Asli, Bouchra [11 ]
Marolleau, Jean-Pierre [12 ]
Meaux-Ruault, Nadine [13 ]
Gerfaud-Valentin, Mathieu [14 ]
Audeguy, Philippe [15 ]
Hamidou, Mohamed [16 ]
Corm, Selim [17 ]
Delbrel, Xavier [18 ]
Fontan, Jean [19 ]
Lebon, Delphine [12 ]
Mausservey, Christelle [20 ]
Moulis, Guillaume [21 ,22 ]
Limal, Nicolas [1 ]
Michel, Marc [1 ]
Godeau, Bertrand [1 ]
Mahevas, Matthieu [1 ,23 ,24 ]
机构
[1] Univ Paris Est Creteil, Ctr Natl Reference Cytopenies Autoimmunes Adulte, Federat Hosp Univ TRUE InnovaT Rapy ImmUne Disord, Internal Med Dept,Hop Henri Mondor,AP HP, Creteil, France
[2] Univ Paris Cite, Lab Immunogenet Pediat Autoimmune Dis, Imagine Inst, INSERM,UMR U1163, Paris, France
[3] Aix Marseille Univ, Hop Timone, AP HP, Internal Med Dept, Marseille, France
[4] Univ Hosp Ctr Bordeaux, Haut Leveque Hosp, Internal Med & Infect Dis Unit, Pessac, France
[5] Univ Bordeaux, INSERM, U1034, Pessac, France
[6] CHU Poitiers, Dept Internal Med, Poitiers, France
[7] CHU Lille, Internal Med & Clin Immunol Dept, Ctr Reference Malad Autoimmunes Syst Rares Nord &, LIRIC,INSERM,U995, Lille, France
[8] Ctr Hosp Univ CHU Dijon Bourgogne, Internal Med & Clin Immunol Dept, Ctr Reference Constitutif Cytopenies Autoimmunes, Hop Francois Mitterrand, Dijon, France
[9] CHU Clermont Ferrand, Estaing Univ Hosp, Dept Internal Med, Clermont Ferrand, France
[10] Univ Clermont Auvergne, Inst Pascal, CNRS, CHU Clermont Ferrand, Clermont Ferrand, France
[11] Sauvegarde Clin, Dept Internal Med, Lyon, France
[12] CHU Amiens Picardie, Clin Hematol & Cellular Therapy Dept, Equipe Hematim, CURS,UPJV,EA4666, Amiens, France
[13] Ctr Hosp Univ Jean Minjoz, Dept Internal Med, Besancon, France
[14] Univ Claude Bernard Lyon1, Hosp Civils Lyon, Dept Internal Med, Hop Croix Rousse, Lyon, France
[15] Hop Prive Pasteur, Internal Med, Evreux, France
[16] CHU Nantes, Dept Internal Med, Nantes, France
[17] Medipole Savoie, Dept Clin Hematol, Challes Les Eaux, France
[18] Ctr Hosp Pau, Dept Internal Med, Pau, France
[19] CHU Besancon, Dept Clin Hematol, Besancon, France
[20] Ctr Hosp William Morey, Dept Internal Med, Chalon Sur Saone, France
[21] CHU Toulouse, Dept Internal Med, Toulouse, France
[22] CHU Toulouse, CIC 1436, Equipe PEPSS, Toulouse, France
[23] Univ Paris Est Creteil, Inst Necker Enfants Malades INEM, Univ Paris Cite, INSERM,U1151,CNRS,UMS 8253,ATIP,Avenir Team,AI2B, Creteil, France
[24] Univ Paris Est Creteil UPEC, INSERM, Equipe 2, U955, Creteil, France
关键词
combination therapy; immunosuppressive drug; ITP; TPO-RA; MYCOPHENOLATE-MOFETIL; CYCLOSPORINE-A; THERAPY; PURPURA; EFFICACY;
D O I
10.1111/bjh.18893
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Combining drugs could be an effective option for treating multirefractory ITP, that is, patients not responding to rituximab, thrombopoietin receptor agonists (TPO-RA) and splenectomy. We conducted a retrospective, multicenter, observational study including multirefractory ITP patients who received a combination of a TPO-RA and an immunosuppressive drug. We included 39 patients (67% women, median age 59 years [range 21-96]), with a median ITP duration of 57 months [3-393] and a median platelet count at initiation of 10 x 10(9)/L [1-35]. The combination regimen was given for a median duration of 12 months [1-103] and included eltrombopag (51%) or romiplostim (49%), associated with mycophenolate mofetil (54%), azathioprine (36%), cyclophosphamide (5%), cyclosporin (3%) or everolimus (3%). Overall, 30 patients (77%) achieved at least a response (platelet count >= 30 x 10(9)/L and at least doubling baseline during at least 3 months), including 24 complete responses (platelet count >100 x 10(9)/L during at least 3 months) with a median time to response of 30 days [7-270] and a median duration of response of 15 months [4-63]. Severe adverse event related to ITP treatment was observed in 31%. In conclusion, this study confirms that some patients with multirefractory ITP can achieve long lasting response with this combination.
引用
收藏
页码:883 / 889
页数:7
相关论文
共 26 条
[11]   Mycophenolate mofetil (MMF) for the treatment of steroid-resistant idiopathic thrombocytopenic purpura [J].
Hou, M ;
Peng, J ;
Shi, Y ;
Zhang, CQ ;
Qin, P ;
Zhao, CL ;
Ji, XB ;
Wang, XY ;
Zhang, MH .
EUROPEAN JOURNAL OF HAEMATOLOGY, 2003, 70 (06) :353-357
[12]   Cyclosporin A for the treatment of patients with chronic idiopathic thrombocytopenic purpura refractory to corticosteroids or splenectomy [J].
Kappers-Klunne, MC ;
van't Veer, MB .
BRITISH JOURNAL OF HAEMATOLOGY, 2001, 114 (01) :121-125
[13]   Sirolimus is effective for primary relapsed/refractory autoimmune cytopenia: a multicenter study [J].
Li, Hongmin ;
Ji, Jiang ;
Du, Yali ;
Huang, Yuzhou ;
Gu, Hao ;
Chen, Miao ;
Wu, RunHui ;
Han, Bing .
EXPERIMENTAL HEMATOLOGY, 2020, 89 :87-95
[14]   Effects of Rapamycin Combined with Low Dose Prednisone in Patients with Chronic Immune Thrombocytopenia [J].
Li, Jiaming ;
Wang, Zhaoyue ;
Dai, Lan ;
Cao, Lijuan ;
Su, Jian ;
Zhu, Mingqing ;
Yu, Ziqiang ;
Bai, Xia ;
Ruan, Changgeng .
CLINICAL & DEVELOPMENTAL IMMUNOLOGY, 2013,
[15]   Characteristics, outcome, and response to therapy of multirefractory chronic immune thrombocytopenia [J].
Mahevas, Matthieu ;
Gerfaud-Valentin, Mathieu ;
Moulis, Guillaume ;
Terriou, Louis ;
Audia, Sylvain ;
Guenin, Sophie ;
Le Guenno, Guillaume ;
Salles, Gilles ;
Lambotte, Olivier ;
Limal, Nicolas ;
Viallard, Jean-Francois ;
Cheze, Stephane ;
Tomowiak, Cecile ;
Royer, Bruno ;
Neel, Antoine ;
Debouverie, Odile ;
Hot, Arnaud ;
Durieu, Isabelle ;
Perlat, Antoinette ;
Cliquennois, Manuel ;
Deteix, Clemence ;
Michel, Marc ;
Godeau, Bertrand .
BLOOD, 2016, 128 (12) :1625-1630
[16]   Identifying and treating refractory ITP: difficulty in diagnosis and role of combination treatment [J].
Miltiadous, Oriana ;
Hou, Ming ;
Bussel, James B. .
BLOOD, 2020, 135 (07) :472-490
[17]  
Mishra K, 2021, AM J BLOOD RES, V11, P217
[18]   Multirefractory Primary Immune Thrombocytopenia in Adults: Prevalence and Burden. Results from the CarmenFrance Registry [J].
Moulis, Guillaume ;
Rueter, Manuela ;
Mahevas, Matthieu ;
Viallard, Jean-Francois ;
Comont, Thibault ;
Cheze, Stephane ;
Audia, Sylvain ;
Ebbo, Mikael ;
Terriou, Louis ;
Lega, Jean-Christophe ;
Jeandel, Pierre-Yves ;
Bonnotte, Bernard ;
Michel, Marc ;
Lapeyre-Mestre, Maryse ;
Godeau, Bertrand .
BLOOD, 2022, 140 :5557-5558
[19]   Sirolimus versus cyclosporine for the treatment of pediatric chronic immune thrombocytopenia: A randomized blinded trial [J].
Mousavi-Hasanzadeh, Morteza ;
Bagheri, Bahador ;
Mehrabi, Sarvenaz ;
Eghbali, Aygin ;
Eghbali, Aziz .
INTERNATIONAL IMMUNOPHARMACOLOGY, 2020, 88
[20]   American Society of Hematology 2019 guidelines for immune thrombocytopenia [J].
Neunert, Cindy ;
Terrell, Deirdra R. ;
Arnold, Donald M. ;
Buchanan, George ;
Cines, Douglas B. ;
Cooper, Nichola ;
Cuker, Adam ;
Despotovic, Jenny M. ;
George, James N. ;
Grace, Rachael F. ;
Kuehne, Thomas ;
Kuter, David J. ;
Lim, Wendy ;
McCrae, Keith R. ;
Pruitt, Barbara ;
Shimanek, Hayley ;
Vesely, Sara K. .
BLOOD ADVANCES, 2019, 3 (23) :3829-3866