title: High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline.
abstract: We evaluated safety and feasibility of high-pressure transvenous limb perfusion in an upper extremity of adult patients with muscular dystrophy, after completing a similar study in a lower extremity. A dose escalation study of single-limb perfusion with 0.9% saline was carried out in nine adults with muscular dystrophies under intravenous analgesia. Our study demonstrates that it is feasible and definitely safe to perform high-pressure transvenous perfusion with 0.9% saline up to 35% of limb volume in the upper extremities of young adults with muscular dystrophy. Perfusion at 40% limb volume is associated with short-lived physiological changes in peripheral nerves without clinical correlates in one subject. This study provides the basis for a phase 1/2 clinical trial using pressurized transvenous delivery into upper limbs of nonambulatory patients with Duchenne muscular dystrophy. Furthermore, our results are applicable to other conditions such as limb girdle muscular dystrophy as a method for delivering regional macromolecular therapeutics in high dose to skeletal muscles of the upper extremity.
authors.affiliation: 1 Department of Neurology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.|2 Wellstone Muscular Dystrophy Cooperative Research Center, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
authors.forename: Zheng
authors.initials: Z
authors.lastname: Fan
authors.affiliation: 2 Wellstone Muscular Dystrophy Cooperative Research Center, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.|3 Department of Pediatrics, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.|4 Department of Anesthesiology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
authors.forename: Keith
authors.initials: K
authors.lastname: Kocis
authors.affiliation: 3 Department of Pediatrics, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.|4 Department of Anesthesiology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
authors.forename: Robert
authors.initials: R
authors.lastname: Valley
authors.affiliation: 1 Department of Neurology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.|2 Wellstone Muscular Dystrophy Cooperative Research Center, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
authors.forename: James F
authors.initials: JF
authors.lastname: Howard
authors.affiliation: 1 Department of Neurology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.|2 Wellstone Muscular Dystrophy Cooperative Research Center, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
authors.forename: Manisha
authors.initials: M
authors.lastname: Chopra
authors.affiliation: 5 Department of Radiology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
authors.forename: Yasheng
authors.initials: Y
authors.lastname: Chen
authors.affiliation: 5 Department of Radiology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
authors.forename: Hongyu
authors.initials: H
authors.lastname: An
authors.affiliation: 5 Department of Radiology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
authors.forename: Weili
authors.initials: W
authors.lastname: Lin
authors.affiliation: 2 Wellstone Muscular Dystrophy Cooperative Research Center, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.|3 Department of Pediatrics, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
authors.forename: Joseph
authors.initials: J
authors.lastname: Muenzer
authors.affiliation: 1 Department of Neurology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.|2 Wellstone Muscular Dystrophy Cooperative Research Center, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
authors.forename: William
authors.initials: W
authors.lastname: Powers
mesh_terms: D000328:Adult; D005260:Female; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D006801:Humans; D008297:Male; D008875:Middle Aged; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy*; D010477:Perfusion; D011312:Pressure; D012965:Sodium Chloride / Q000008:administration & dosage*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D012965:Sodium Chloride