Cargando…

Reversal of life-threatening hepatopulmonary syndrome in Gaucher disease by imiglucerase enzyme replacement therapy

Advanced liver disease complicated by hepatopulmonary syndrome is a recognized complication of Gaucher disease. Macrophage-targeted, recombinant enzyme replacement therapy is effective in reversing clinical manifestations attributed to the accumulation of glycolipid-laden macrophages but it is not k...

Descripción completa

Detalles Bibliográficos
Autores principales:	Beshlawy, Amal El, Murugesan, Vagishwari, Mistry, Pramod Kumar, Eid, Khaled
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2019
Materias:	Case Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6606832/ https://www.ncbi.nlm.nih.gov/pubmed/31309038 http://dx.doi.org/10.1016/j.ymgmr.2019.100490

Ejemplares similares

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)

Review of the safety and efficacy of imiglucerase treatment of Gaucher disease
por: Elstein, Deborah, et al.
Publicado: (2009)

Imiglucerase in the treatment of Gaucher disease: a history and perspective
por: Deegan, Patrick B, et al.
Publicado: (2012)

Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial
por: Schiffmann, Raphael, et al.
Publicado: (2022)

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)

Enzyme replacement therapy with taliglucerase alfa: 36‐month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase
por: Pastores, Gregory M., et al.
Publicado: (2016)

Comparative Therapeutic Effects of Velaglucerase Alfa and Imiglucerase in a Gaucher Disease Mouse Model
por: Xu, You-Hai, et al.
Publicado: (2010)

Effects of imiglucerase on the growth and metabolism of Gaucher disease type I patients: a systematic review
por: Doneda, Divair, et al.
Publicado: (2013)

Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience
por: Tukan, Irina, et al.
Publicado: (2013)

Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy
por: Serratrice, Christine, et al.
Publicado: (2016)

Hepatopulmonary Fistula: a life threatening complication of hydatid disease
por: Gulamhussein, Mohamed Amirali, et al.
Publicado: (2015)

Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment
por: Weinreb, Neal J., et al.
Publicado: (2012)

Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3
por: Machaczka, Maciej, 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)

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

Osteonecrosis in Gaucher disease in the era of multiple therapies: Biomarker set for risk stratification from a tertiary referral center
por: Basiri, Mohsen, et al.
Publicado: (2023)

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)

Erratum to: Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment
por: Weinreb, Neal J., et al.
Publicado: (2013)

Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1
por: Stirnemann, Jérôme, et al.
Publicado: (2015)

Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series
por: Serratrice, Christine, et al.
Publicado: (2015)

The International Collaborative Gaucher Group GRAF (Gaucher Risk Assessment for Fracture) score: a composite risk score for assessing adult fracture risk in imiglucerase-treated Gaucher disease type 1 patients
por: Deegan, Patrick, et al.
Publicado: (2021)

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)

Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis
por: Mistry, Pramod K, et al.
Publicado: (2009)

The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158
por: Mistry, Pramod K., et al.
Publicado: (2023)

Gaucher Disease: Transcriptome Analyses Using Microarray or mRNA Sequencing in a Gba1 Mutant Mouse Model Treated with Velaglucerase alfa or Imiglucerase
por: Dasgupta, Nupur, et al.
Publicado: (2013)

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)

Improvement of bone disease by imiglucerase (Cerezyme) therapy in patients with skeletal manifestations of type 1 Gaucher disease: results of a 48-month longitudinal cohort study
por: Sims, KB, et al.
Publicado: (2008)

Cancer risk and gammopathies in 2123 adults with Gaucher disease type 1 in the International Gaucher Group Gaucher Registry
por: Rosenbloom, Barry E., et al.
Publicado: (2022)

A multicenter, open-label, phase III study of Abcertin in Gaucher disease
por: Lee, Beom Hee, et al.
Publicado: (2017)

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

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

Hormone-replacement Therapy for Hepatopulmonary Syndrome and NASH Associated with Hypopituitarism
por: Torii, Nobuyuki, et al.
Publicado: (2018)

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

THE HEPATOPULMONARY SYNDROME
por: NACIF, Lucas Souto, et al.
Publicado: (2014)

Hepatopulmonary syndrome
por: Raevens, Sarah, et al.
Publicado: (2022)

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

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)

The mutation spectrum in Indian patients with Gaucher disease
por: Bisariya, Vartika, et al.
Publicado: (2011)

Transjugular Intrahepatic Portosystemic Shunt for Refractory Ascites in Gaucher Disease
por: Adhyaru, Kunal, et al.
Publicado: (2022)

Hepatopulmonary syndrome: An update
por: Gandhi, Kejal D, et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_nor833omn7s0v65g6hh712a2im