Gene therapy in hemophilia A: a cost-effectiveness analysis-Reference-Cited by-同舟云学术

Gene therapy in hemophilia A: a cost-effectiveness analysis

Published:2018-07-24 Issue:14 Volume:2 Page:1792-1798
ISSN:2473-9529
Container-title:Blood Advances
language:en
Short-container-title:

Author:

Machin Nicoletta¹²,Ragni Margaret V.¹²^ORCID,Smith Kenneth J.¹³^ORCID

Affiliation:

1. Division of Hematology/Oncology, Department of Medicine, University of Pittsburgh, Pittsburgh, PA;

2. Hemophilia Center of Western PA, Pittsburgh, PA; and

3. Section of Decision Sciences and Clinical Systems Modeling, Division of Internal Medicine and Center for Research on Health Care Data Center, University of Pittsburgh, Pittsburgh, PA

Abstract

Key Points Gene therapy is cost-effective in severe hemophilia A compared with standard factor VIII prophylaxis. Over a 10-year time horizon, gene therapy cost $1M and resulted in 8.33 QALYs gained, whereas prophylaxis cost $1.7M and resulted in 6.62 QALYs gained.

Publisher

American Society of Hematology

Subject

Hematology

Link

http://ashpublications.org/bloodadvances/article-pdf/2/14/1792/881155/advances021345.pdf

Reference37 articles.

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4. Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens;Collins;J Thromb Haemost,2010

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