Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy-Reference-Cited by-同舟云学术

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

Published:2017-04-27 Issue:17 Volume:129 Page:2375-2383
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Cox Timothy M.¹,Drelichman Guillermo²,Cravo Renata³,Balwani Manisha⁴,Burrow Thomas Andrew⁵,Martins Ana Maria⁶,Lukina Elena⁷,Rosenbloom Barry⁸,Goker-Alpan Ozlem⁹,Watman Nora¹⁰,El-Beshlawy Amal¹¹,Kishnani Priya S.¹²,Pedroso Maria Lucia¹³,Gaemers Sebastiaan J. M.¹⁴,Tayag Regina¹⁵,Peterschmitt M. Judith¹⁴

Affiliation:

1. Department of Medicine, University of Cambridge, Addenbrooke’s Hospital, Cambridge, United Kingdom;

2. Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina;

3. State Institute of Haematology “Arthur de Siqueira Cavalcanti,” Rio de Janeiro, Brazil;

4. Mount Sinai Hospital, New York, NY;

5. Cincinnati Children’s Hospital Medical Center, Cincinnati, OH;

6. Universidade Federal de São Paulo, São Paulo, Brazil;

7. National Research Center for Hematology, Moscow, Russia;

8. Cedars-Sinai/Tower Hematology Oncology, Beverly Hills, CA;

9. Lysosomal Disorders Unit, O&O Alpan, LLC, Fairfax, VA;

10. Hospital Ramos Mejia, Buenos Aires, Argentina;

11. Pediatric Hematology, Cairo University, Cairo, Egypt;

12. Department of Pediatrics, Duke University School of Medicine, Durham, NC;

13. Gastroenterology Department, Universidade Federal do Paraná, Curitiba, Brazil;

14. Sanofi Genzyme, Cambridge, MA; and

15. Prometrika LLC, Cambridge, MA

Abstract

Key Points Eliglustat treatment resulted in stable mean hemoglobin concentration, platelet count, and spleen and liver volumes for up to 4 years. Mean bone mineral density z scores also remained stable and were maintained in the healthy reference range for up to 4 years.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/129/17/2375/1400197/blood758409.pdf

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4. Osseous manifestations of adult Gaucher disease in the era of enzyme replacement therapy;Deegan;Medicine (Baltimore),2011

5. Revised recommendations for the management of Gaucher disease in children;Kaplan;Eur J Pediatr,2013

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