BIOMAT Database Logo BIOMAT Database Logo

Sorafenib in Hepatopulmonary Syndrome: A Randomized, Double-Blind, Placebo-Controlled Trial. 2019

Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.

The tyrosine kinase inhibitor sorafenib improves hepatopulmonary syndrome (HPS) in an experimental model. However, the efficacy and adverse effect profile in patients with HPS are unknown. We aimed to determine the effect of sorafenib on the alveolar-arterial oxygen gradient (AaPO2 ) at 3 months in patients with HPS. We performed a randomized, double-blind, placebo-controlled parallel trial of sorafenib in patients with HPS at 7 centers. A total of 28 patients with HPS were randomized to sorafenib 400 mg by mouth daily or a matching placebo in a 1:1 ratio. We found no statistically significant difference in the median change in AaPO2 from baseline to 12 weeks between the patients allocated to sorafenib (4.5 mm Hg; IQR, -3.8 to 7.0 mm Hg) and those allocated to placebo (-2.4 mm Hg; IQR, -4.8 to 8.2 mm Hg; P = 0.70). There was also no difference between the groups in terms of degree of intrapulmonary shunting by contrast echocardiography. Sorafenib significantly reduced circulating levels of angiogenic markers, including vascular endothelial growth factor receptors (P < 0.01) and TIE2-expressing M2 monocytes (P = 0.03), but it reduced the mental component scores of the Short Form 36 (P = 0.04), indicating a worse quality of life. In conclusion, sorafenib did not change the AaPO2 or other disease markers at 3 months in patients with HPS. Alternative antiangiogenic therapies or treatments targeting other pathways should be investigated.

UI MeSH Term Description Entries
D008297 Male Males
D008875 Middle Aged An adult aged 45 - 64 years. Middle Age
D009389 Neovascularization, Pathologic A pathologic process consisting of the proliferation of blood vessels in abnormal tissues or in abnormal positions. Angiogenesis, Pathologic,Angiogenesis, Pathological,Neovascularization, Pathological,Pathologic Angiogenesis,Pathologic Neovascularization,Pathological Angiogenesis,Pathological Neovascularization
D010919 Placebos Any dummy medication or treatment. Although placebos originally were medicinal preparations having no specific pharmacological activity against a targeted condition, the concept has been extended to include treatments or procedures, especially those administered to control groups in clinical trials in order to provide baseline measurements for the experimental protocol. Sham Treatment
D011788 Quality of Life A generic concept reflecting concern with the modification and enhancement of life attributes, e.g., physical, political, moral, social environment as well as health and disease. HRQOL,Health-Related Quality Of Life,Life Quality,Health Related Quality Of Life
D004311 Double-Blind Method A method of studying a drug or procedure in which both the subjects and investigators are kept unaware of who is actually getting which specific treatment. Double-Masked Study,Double-Blind Study,Double-Masked Method,Double Blind Method,Double Blind Study,Double Masked Method,Double Masked Study,Double-Blind Methods,Double-Blind Studies,Double-Masked Methods,Double-Masked Studies,Method, Double-Blind,Method, Double-Masked,Methods, Double-Blind,Methods, Double-Masked,Studies, Double-Blind,Studies, Double-Masked,Study, Double-Blind,Study, Double-Masked
D004452 Echocardiography Ultrasonic recording of the size, motion, and composition of the heart and surrounding tissues. The standard approach is transthoracic. Echocardiography, Contrast,Echocardiography, Cross-Sectional,Echocardiography, M-Mode,Echocardiography, Transthoracic,Echocardiography, Two-Dimensional,Transthoracic Echocardiography,2-D Echocardiography,2D Echocardiography,Contrast Echocardiography,Cross-Sectional Echocardiography,Echocardiography, 2-D,Echocardiography, 2D,M-Mode Echocardiography,Two-Dimensional Echocardiography,2 D Echocardiography,Cross Sectional Echocardiography,Echocardiography, 2 D,Echocardiography, Cross Sectional,Echocardiography, M Mode,Echocardiography, Two Dimensional,M Mode Echocardiography,Two Dimensional Echocardiography
D005260 Female Females
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000075082 Proof of Concept Study An empirical investigation which pertains to the development of prototypes or models that demonstrate the feasibility of novel concepts, ideas, principles, schema or their practical application. Proof of Concept Approach,Proof of Concept Demonstration,Proof of Concept Evaluation,Proof of Concept Experiment,Proof of Concept Investigation,Proof of Concept Model,Proof of Concept Prototype,Proof of Concept Research,Proof of Concept Studies,Proof of Concept Testing,Approach Proof, Concept,Approach Proofs, Concept,Concept Approach Proof,Concept Approach Proofs,Concept Demonstration Proof,Concept Evaluation Proof,Concept Evaluation Proofs,Concept Experiment Proof,Concept Experiment Proofs,Concept Investigation Proof,Concept Model Proof,Concept Model Proofs,Concept Prototype Proof,Concept Prototype Proofs,Concept Research Proof,Concept Research Proofs,Concept Study Proof,Concept Testing Proof,Concept Testing Proofs,Demonstration Proof, Concept,Evaluation Proof, Concept,Experiment Proof, Concept,Investigation Proof, Concept,Model Proof, Concept,Prototype Proof, Concept,Research Proof, Concept,Study Proof, Concept,Testing Proof, Concept,Testing Proofs, Concept

Related Publications

Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
A double-blind randomized placebo-controlled trial of sibutramine.
May 1996, Obesity research,
Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
Marfan Sartan: a randomized, double-blind, placebo-controlled trial.
August 2015, European heart journal,
Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
A randomized, double-blind placebo-controlled trial of iron in restless legs syndrome.
January 2000, European neurology,
Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
Randomized, double-blind, placebo-controlled trial of pergolide in restless legs syndrome.
December 1998, Neurology,
Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
A double-blind, randomized, placebo-controlled trial of paroxetine controlled-release in irritable bowel syndrome.
January 2009, Psychosomatics,
Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
Applicability of press needles to a double-blind trial: a randomized, double-blind, placebo-controlled trial.
June 2009, The Clinical journal of pain,
Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
Alpha lipoic acid in burning mouth syndrome--a randomized double-blind placebo-controlled trial.
March 2009, Journal of oral pathology & medicine : official publication of the International Association of Oral Pathologists and the American Academy of Oral Pathology,
Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
[Gabapentin for spasticity: a randomized, double-blind, placebo-controlled trial].
January 2005, Medicina clinica,
Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
Celecoxib Versus Placebo in Tonsillectomy: A Prospective, Randomized, Double-Blind Placebo-Controlled Trial.
October 2016, The Annals of otology, rhinology, and laryngology,
Steven M Kawut, and Susan S Ellenberg, and Michael J Krowka, and David Goldberg, and Hugo Vargas, and David Koch, and Tiffany Sharkoski, and Nadine Al-Naamani, and Alyson Fox, and Robert Brown, and Joshua Levitsky, and Jae K Oh, and Grace Lin, and Nianfu Song, and Carl Mottram, and Margaret F Doyle, and David E Kaplan, and Samir Gupta, and Michael B Fallon
A randomized, double-blind, placebo-controlled trial of pseudoephedrine in coryza.
July 2004, Clinical and experimental pharmacology & physiology,
Copied contents to your clipboard!