@article {150455,
	title = {Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.},
	journal = {Blood},
	volume = {129},
	year = {2017},
	month = {2017 04 27},
	pages = {2375-2383},
	abstract = {<p>In the phase 3 Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE), at 1 year, eliglustat was noninferior to imiglucerase enzyme therapy in maintaining stable platelet counts, hemoglobin concentrations, and spleen and liver volumes. After this primary analysis period, patients entered a long-term extension phase in which all received eliglustat. Duration on eliglustat ranged from 2 to 5 years, depending on timing of enrollment (which spanned 2 years), treatment group to which patients were randomized, and whether they lived in the United States when commercial eliglustat became available. Here we report long-term safety and efficacy of eliglustat for 157 patients who received eliglustat in the ENCORE trial; data are available for 46 patients who received eliglustat for 4 years. Mean hemoglobin concentration, platelet count, and spleen and liver volumes remained stable for up to 4 years. Year to year, all 4 measures remained collectively stable (composite end point relative to baseline values) in >=85\% of patients as well as individually in >=92\%. Mean bone mineral density z scores (lumbar spine and femur) remained stable and were maintained in the healthy reference range throughout. Eliglustat was well tolerated over 4 years; 4 (2.5\%) patients withdrew because of adverse events that were considered related to the study drug. No new or long-term safety concerns were identified. Clinical stability assessed by composite and individual measures was maintained in adults with Gaucher disease type 1 treated with eliglustat who remained in the ENCORE trial for up to 4 years. This trial was registered at <a href="http://www.clinicaltrials.gov" title="www.clinicaltrials.gov">www.clinicaltrials.gov</a> as $\#$NCT00943111.</p>
},
	keywords = {Administration, Oral, Adult, Bone Density, Enzyme Inhibitors, Enzyme Replacement Therapy, Female, Femur, Gaucher Disease, Glucosylceramidase, Humans, Liver, Lumbar Vertebrae, Male, Middle Aged, Platelet Count, Pyrrolidines, Recombinant Proteins, Spleen},
	issn = {1528-0020},
	doi = {10.1182/blood-2016-12-758409},
	author = {Cox, Timothy M and Drelichman, Guillermo and Cravo, Renata and Balwani, Manisha and Burrow, Thomas Andrew and Martins, Ana Maria and Lukina, Elena and Rosenbloom, Barry and Goker-Alpan, Ozlem and Watman, Nora and Amal El-Beshlawy and Kishnani, Priya S and Pedroso, Maria Lucia and Gaemers, Sebastiaan J M and Tayag, Regina and Peterschmitt, M Judith}
}
