Eliglustat in patients with Gaucher disease previously treated with enzyme replacement therapy: Real-life experience from Israel

被引：0
|
作者：
Istaiti, Majdolen [1 ]
Ruhrman-Shahar, Noa [2 ]
Cohen, Ian J. [2 ]
Hershkovitz, Tova [3 ]
Becker-Cohen, Michal [1 ]
Dinur, Tama [1 ]
Revel-Vilk, Shoshana [4 ]
Zimran, Ari [1 ]
机构：
[1] Shaare Zedek Med Ctr, Gaucher Unit, Jerusalem, Israel
[2] Beilinson Med Ctr, Raphael Recanati Genet Inst, Rabin Med Ctr, Gaucher Clin, Petah Tiqwa, Israel
[3] Rambam Hlth Care Campus, Genet Inst, Haifa, Israel
[4] Shaare Zedek Med Ctr, Pediat Hematol Oncol Unit, Gaucher Unit, Jerusalem, Israel
来源：
MOLECULAR GENETICS AND METABOLISM | 2022年 / 135卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2021.11.151
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
136
引用
收藏
页码：S61 / S61
页数：1
相关论文
共 50 条
  • [31] Enzyme replacement therapy in romanian patients with type 1 Gaucher disease
    Sido, P. Grigorescu
    Drugan, C.
    Cret, V.
    Al-Kzouz, C.
    Denes, C.
    Zimmermann, A.
    Coldea, C.
    CLINICAL THERAPEUTICS, 2007, 29 : S118 - S118
  • [32] Incremental biomarker and clinical outcomes after switch from enzyme therapy to eliglustat substrate reduction therapy in Gaucher disease
    Kleytman, Nathaniel
    Ruan, Jiapeng
    Ruan, Audrey
    Zhang, Bailin
    Murugesan, Vagishwari
    Lin, Haiqun
    Guo, Lilu
    Klinger, Katherine
    Mistry, Pramod K.
    MOLECULAR GENETICS AND METABOLISM REPORTS, 2021, 29
  • [33] ENZYME REPLACEMENT THERAPY IN TYPE 1 GAUCHER DISEASE: A SINGLE CENTER'S EXPERIENCE
    Mandala, E.
    Lafaras, C.
    Tsioni, K.
    Michelakaki, H.
    Koutalidou, S.
    Ilonidis, G.
    CLINICAL THERAPEUTICS, 2009, 31 : S209 - S210
  • [34] Case report of unexpected gastrointestinal involvement in type 1 Gaucher disease: comparison of eliglustat tartrate treatment and enzyme replacement therapy
    Kim, Yoo-Mi
    Shin, Dong Hoon
    Park, Su Bum
    Cheon, Chong Kun
    Yoo, Han-Wook
    BMC MEDICAL GENETICS, 2017, 18
  • [35] The analysis of the achievement of therapeutic goals in 45 polish and Lithuanian Gaucher disease patients treated with enzyme replacement therapy
    Kleinotiene, G.
    Tylki-Szymanska, A.
    CLINICAL THERAPEUTICS, 2007, 29 : S117 - S118
  • [36] IMMUNE DYSFUNCTION IN PATIENTS WITH GAUCHER DISEASE: IMPACT OF DISEASE SEVERITY AND ENZYME REPLACEMENT THERAPY
    Khalifa, A.
    Tantawy, A.
    Sherif, E.
    Sadek, A.
    Tiseer, N.
    HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 2010, 95 : 104 - 104
  • [37] Effect of enzyme replacement therapy on lipid profile in patients with Gaucher's disease
    Alfonso, P
    Cenarro, A
    Pérez-Calvo, JI
    Puzo, J
    Giralt, M
    Giraldo, P
    Pocoví, M
    MEDICINA CLINICA, 2003, 120 (17): : 641 - 646
  • [38] Enzyme replacement therapy and bony changes in Egyptian paediatric Gaucher disease patients
    El-Beshlawy, A
    Ragab, L
    Youssry, I
    Yakout, K
    El-Kiki, H
    Eid, K
    Mansour, IM
    Abd El-Hamid, S
    Yang, M
    Mistry, PK
    JOURNAL OF INHERITED METABOLIC DISEASE, 2006, 29 (01) : 92 - 98
  • [39] Enzyme replacement therapy with imiglucerase in Taiwanese patients with type I Gaucher disease
    Hsu, CC
    Chien, YH
    Lai, MY
    Hwu, WL
    JOURNAL OF THE FORMOSAN MEDICAL ASSOCIATION, 2002, 101 (09) : 627 - 631
  • [40] Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease
    Ben Turkia, Hadhami
    Gonzalez, Derlis E.
    Barton, Norman W.
    Zimran, Ari
    Kabra, Madhulika
    Lukina, Elena A.
    Giraldo, Pilar
    Kisinovsky, Isaac
    Bavdekar, Ashish
    Ben Dridi, Marie-Francoise
    Gupta, Neerja
    Kishnani, Priya S.
    Sureshkumar, E. K.
    Wang, Nan
    Crombez, Eric
    Bhirangi, Kiran
    Mehta, Atul
    AMERICAN JOURNAL OF HEMATOLOGY, 2013, 88 (03) : 179 - 184
12345