Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.
Dogs with haemophilia A or haemophilia B exhibit spontaneous bleeding comparable with the spontaneous bleeding phenotype that occurs in humans with severe haemophilia. The phenotypic and genotypic characteristics of haemophilic dogs have been well-described, and such dogs are suitable for testing prophylactic protein replacement therapy and gene transfer strategies. In dogs with haemophilia, long-term effects on spontaneous bleeding frequency (measured over years) can be used as an efficacy endpoint in such studies. Although complete correction of coagulopathy has not been achieved, published data show that prophylactic factor replacement therapy and gene transfer can markedly reduce the frequency of spontaneous bleeding in haemophilic dogs. Further studies are currently ongoing.
Nichols
T C
TC
Francis Owen Blood Research Laboratory, Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC 27516-3114, USA. tnichols@med.unc.edu
Raymer
R A
RA
Franck
H W G
HW
Merricks
E P
EP
Bellinger
D A
DA
DeFriess
N
N
Margaritis
P
P
Arruda
V R
VR
Kay
M A
MA
High
K A
KA
D000818:Animals; D004285:Dogs; D005164:Factor IX / Q000627:therapeutic use*; D015316:Genetic Therapy / Q000379:methods; D006467:Hemophilia A / Q000628:therapy*; D002836:Hemophilia B / Q000628:therapy*; D006470:Hemorrhage / Q000517:prevention & control*
D005164:Factor IX