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Hum Gene Ther. 2015 Sep;26(9):614-21. doi: 10.1089/hum.2015.023. Epub 2015 Jul 30.

High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline.

Author information

1
1 Department of Neurology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
2
2 Wellstone Muscular Dystrophy Cooperative Research Center, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
3
3 Department of Pediatrics, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
4
4 Department of Anesthesiology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
5
5 Department of Radiology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.

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.

PMID:
25953425
PMCID:
PMC4575535
DOI:
10.1089/hum.2015.023
[Indexed for MEDLINE]
Free PMC Article

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