Cargando…

Case report of unexpected gastrointestinal involvement in type 1 Gaucher disease: comparison of eliglustat tartrate treatment and enzyme replacement therapy

BACKGROUND: Gastrointestinal involvement in Gaucher disease is very rare, and appears to be unresponsive to enzyme replacement therapy (ERT). CASE PRESENTATION: Here, we describe identical twin, splenectomized, non-neuronopathic Gaucher patients on long-term ERT for 9 years, who complained of epigas...

Descripción completa

Detalles Bibliográficos
Autores principales:	Kim, Yoo-Mi, Shin, Dong Hoon, Park, Su Bum, Cheon, Chong Kun, Yoo, Han-Wook
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2017
Materias:	Case Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5433137/ https://www.ncbi.nlm.nih.gov/pubmed/28506293 http://dx.doi.org/10.1186/s12881-017-0403-x

Ejemplares similares

Profile of eliglustat tartrate in the management of Gaucher disease
por: Sechi, Annalisa, et al.
Publicado: (2016)

Profile of eliglustat tartrate in the management of Gaucher disease [Corrigendum]
Publicado: (2016)

Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease
por: Bennett, Lunawati L, et al.
Publicado: (2015)

Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease [Corrigendum]
Publicado: (2015)

Safety and efficacy of eliglustat combined to enzyme replacement therapy for lymphadenopathy in patients with Gaucher disease type 3
por: Lee, Ni-Chung, et al.
Publicado: (2022)

Real-Life Experience with Oral Eliglustat in Patients with Gaucher Disease Previously Treated with Enzyme Replacement Therapy
por: Istaiti, Majdolen, et al.
Publicado: (2022)

Real‐world effectiveness of eliglustat in treatment‐naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry
por: Mistry, Pramod K., et al.
Publicado: (2020)

Incremental biomarker and clinical outcomes after switch from enzyme therapy to eliglustat substrate reduction therapy in Gaucher disease
por: Kleytman, Nathaniel, et al.
Publicado: (2021)

Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial
por: Pleat, Rebecca, et al.
Publicado: (2016)

Budget Impact Analysis of Eliglustat for the Treatment of Gaucher Disease Type 1 in the United States
por: Nalysnyk, Luba, et al.
Publicado: (2018)

A Japanese Patient with Gaucher Disease Treated with the Oral Drug Eliglustat as Substrate Reducing Therapy
por: Komada, Naoto, et al.
Publicado: (2021)

Gaucher Disease Types I and III Responded Well to Substrate Reduction Therapy Using Eliglustat
por: Harai, Nozomi, et al.
Publicado: (2023)

Enzyme Replacement in Gaucher Disease
por: Beutler, Ernest
Publicado: (2004)

Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat
por: Kamath, Ravi S., et al.
Publicado: (2014)

The Clinical Efficacy of Imiglucerase versus Eliglustat in Patients with Gaucher's Disease Type 1: A Systematic Review
por: Nabizadeh, Azita, et al.
Publicado: (2018)

Clinical response to eliglustat in treatment-naïve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry
por: Ibrahim, Jennifer, et al.
Publicado: (2016)

Effects of paroxetine, ketoconazole, and rifampin on the metabolism of eliglustat, an oral substrate reduction therapy for Gaucher disease type 1
por: Vu, Lucie, et al.
Publicado: (2020)

P1588: LONG-TERM REAL-WORLD EFFECTIVENESS OF ELIGLUSTAT IN TREATMENT-NAÏVE AND SWITCH PATIENTS ENROLLED IN THE INTERNATIONAL COLLABORATIVE GAUCHER GROUP (ICGG) GAUCHER REGISTRY
por: Mistry, Pramod, et al.
Publicado: (2023)

Pregnancy outcome in women with Gaucher disease type 1 who had unplanned pregnancies during eliglustat clinical trials
por: Lukina, Elena, et al.
Publicado: (2020)

Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial
por: Lukina, Elena, et al.
Publicado: (2018)

Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1
por: Peterschmitt, M. Judith, et al.
Publicado: (2019)

Impact on bone microarchitecture and failure load in a patient with type I Gaucher disease who switched from Imiglucerase to Eliglustat
por: Sidhu, Karamjot, et al.
Publicado: (2020)

Suicidal attempt with eliglustat overdose
por: Nadler, Johannes, et al.
Publicado: (2022)

Outcomes after 18 months of eliglustat therapy in treatment‐naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial
por: Mistry, Pramod K., et al.
Publicado: (2017)

A Quantitative Systems Pharmacology Model of Gaucher Disease Type 1 Provides Mechanistic Insight Into the Response to Substrate Reduction Therapy With Eliglustat
por: Abrams, Ruth, et al.
Publicado: (2020)

P1541: PRELIMINARY RESULTS OF TWO YEARS FOLLOW-UP OF TYPE 1 GAUCHER DISEASE PATIENTS TREATED WITH ELIGLUSTAT IN TRAZELGA PROJECT.
por: Serrano-Gonzalo, I., et al.
Publicado: (2022)

Identification of a novel therapeutic target underlying atypical manifestation of Gaucher disease
por: Kim, Eun Na, et al.
Publicado: (2022)

Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results
por: Mistry, Pramod K., et al.
Publicado: (2021)

The GBA p.G85E mutation in Korean patients with non-neuronopathic Gaucher disease: founder and neuroprotective effects
por: Kim, Yoo-Mi, et al.
Publicado: (2020)

Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease
por: van Dussen, Laura, et al.
Publicado: (2014)

Effect of Substrate Reduction Therapy in Comparison to Enzyme Replacement Therapy on Immune Aspects and Bone Involvement in Gaucher Disease
por: Limgala, Renuka P., et al.
Publicado: (2020)

MiRNA Expression in Patients with Gaucher Disease Treated with Enzyme Replacement Therapy
por: Pawliński, Łukasz, et al.
Publicado: (2020)

Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry
por: Mistry, Pramod K., et al.
Publicado: (2017)

Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS)
por: Hughes, Derralynn A., et al.
Publicado: (2022)

Reversal of life-threatening hepatopulmonary syndrome in Gaucher disease by imiglucerase enzyme replacement therapy
por: Beshlawy, Amal El, et al.
Publicado: (2019)

Down-regulation of Regulatory T-cells in Children With Gaucher Disease Under Enzyme Replacement Therapy
por: Zahran, Asmaa M., et al.
Publicado: (2019)

Drug–Drug Interactions Of Amiodarone And Quinidine On The Pharmacokinetics Of Eliglustat In Rats
por: Wang, Qiong, et al.
Publicado: (2019)

Considerations for evaluating the effectiveness and long-term outcome of enzyme replacement therapy in Pompe disease
por: Cheon, Chong Kun
Publicado: (2019)

Total Synthesis of Eliglustat via Diastereoselective Amination of Chiral para-Methoxycinnamyl Benzyl Ether
por: Kong, Younggyu, et al.
Publicado: (2022)

Evaluation of the Nutritional Status of Gaucher Disease Type I Patients under Enzyme Replacement Treatment
por: Iaccarino Idelson, Paola, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_ot81q4r77sbqggah0g2h22j3mr