PMID- 36362492
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20221117
IS  - 2077-0383 (Print)
IS  - 2077-0383 (Electronic)
IS  - 2077-0383 (Linking)
VI  - 11
IP  - 21
DP  - 2022 Oct 24
TI  - Real-Life Experience with Oral Eliglustat in Patients with Gaucher Disease 
      Previously Treated with Enzyme Replacement Therapy.
LID - 10.3390/jcm11216265 [doi]
LID - 6265
AB  - Three types of enzyme replacement therapies (ERTs) and two substrate reduction 
      therapies (SRTs) are approved for symptomatic patients with type 1 Gaucher 
      disease (GD1). Eliglustat is the second SRT approved, yet the first to be 
      approved as first-line therapy for any adult patients with compatible CYP2D6 
      metabolizer genotype. Herein we report safety and efficacy data of the first 29 
      patients switched from ERT to eliglustat from the Gaucher Unit at Shaare Zedek 
      Medical Center (SZMC) between 07/2017 and 06/2022; the median (range) time on ERT 
      was 13 (0.66-30) years, and the median (range) time on eliglustat was 7 (1-52) 
      months. Most patients switched due to oral preference or sub-optimal response to 
      low-dose ERT. Twelve patients stopped eliglustat after a median (range) of 4 
      (1-18) months; 11 due to adverse events (AEs) and one due to personal request. 
      There were no drug-related serious AEs and no drug-related cardiac events. Most 
      AEs were mild and transient, mainly dyspepsia. Efficacy achievements were 
      reflected by maintaining stability. We concluded that switching from ERT to 
      eliglustat is safe if choosing the appropriate patients. Reassuring patients to 
      tolerate early AEs may reduce discontinuation. Following the response and 
      compliance to therapy is important to ensure long-term efficacy.
FAU - Istaiti, Majdolen
AU  - Istaiti M
AUID- ORCID: 0000-0001-7598-2828
AD  - Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
FAU - Becker-Cohen, Michal
AU  - Becker-Cohen M
AD  - Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
FAU - Dinur, Tama
AU  - Dinur T
AD  - Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
FAU - Revel-Vilk, Shoshana
AU  - Revel-Vilk S
AUID- ORCID: 0000-0001-9151-0337
AD  - Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
AD  - Faculty of Medicine, Hebrew University, Jerusalem 91120, Israel.
FAU - Zimran, Ari
AU  - Zimran A
AUID- ORCID: 0000-0003-0077-8608
AD  - Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
AD  - Faculty of Medicine, Hebrew University, Jerusalem 91120, Israel.
LA  - eng
PT  - Journal Article
DEP - 20221024
PL  - Switzerland
TA  - J Clin Med
JT  - Journal of clinical medicine
JID - 101606588
PMC - PMC9659144
OTO - NOTNLM
OT  - Gaucher disease
OT  - eliglustat
OT  - substrate reduction therapy
OT  - switch
COIS- The Gaucher Unit receives support from Sanofi/Genzyme for participation in the 
      ICGG registry, from Takeda-for GOS, and from Pfizer for TALIAS, and research 
      grants from Shire (now Takeda), Pfizer, Sanofi/Genzyme and Centogene. M.I. No 
      conflicts to declare. M.B.-C. No conflicts to declare. T.D. No conflicts to 
      declare. S.R.-V. received research grants, speaker fees, travel support and 
      advisory fee from Pfizer, Sanofi Genzyme, and Takeda. A.Z. Honoraria-Takeda, 
      Pfizer, BioEvents, consulting fees from Prevail Therapeutics and Takeda.
EDAT- 2022/11/12 06:00
MHDA- 2022/11/12 06:01
PMCR- 2022/10/24
CRDT- 2022/11/11 01:30
PHST- 2022/08/25 00:00 [received]
PHST- 2022/09/13 00:00 [revised]
PHST- 2022/10/19 00:00 [accepted]
PHST- 2022/11/11 01:30 [entrez]
PHST- 2022/11/12 06:00 [pubmed]
PHST- 2022/11/12 06:01 [medline]
PHST- 2022/10/24 00:00 [pmc-release]
AID - jcm11216265 [pii]
AID - jcm-11-06265 [pii]
AID - 10.3390/jcm11216265 [doi]
PST - epublish
SO  - J Clin Med. 2022 Oct 24;11(21):6265. doi: 10.3390/jcm11216265.