Randomized clinical trial of long-acting somatostatin for autosomal dominant polycystic kidney and liver disease.
There are no proven, effective therapies for polycystic kidney disease (PKD) or polycystic liver disease (PLD). We enrolled 42 patients with severe PLD resulting from autosomal dominant PKD (ADPKD) or autosomal dominant PLD (ADPLD) in a randomized, double-blind, placebo-controlled trial of octreotide, a long-acting somatostatin analogue. We randomly assigned 42 patients in a 2:1 ratio to octreotide LAR depot (up to 40 mg every 28+/-5 days) or placebo for 1 year. The primary end point was percent change in liver volume from baseline to 1 year, measured by MRI. Secondary end points were changes in total kidney volume, GFR, quality of life, safety, vital signs, and clinical laboratory tests. Thirty-four patients had ADPKD, and eight had ADPLD. Liver volume decreased by 4.95%+/-6.77% in the octreotide group but remained practically unchanged (+0.92%+/-8.33%) in the placebo group (P=0.048). Among patients with ADPKD, total kidney volume remained practically unchanged (+0.25%+/-7.53%) in the octreotide group but increased by 8.61%+/-10.07% in the placebo group (P=0.045). Changes in GFR were similar in both groups. Octreotide was well tolerated; treated individuals reported an improved perception of bodily pain and physical activity. In summary, octreotide slowed the progressive increase in liver volume and total kidney volume, improved health perception among patients with PLD, and had an acceptable side effect profile.
Hogan
Marie C
MC
Division of Nephrology and Hypertension, Department of Biomedical STatistics and Informatics, Mayo Clinic College of Medicine, 200 First Street SW, Rochester, MN 55905, USA. hogan.marie@mayo.edu
Masyuk
Tetyana V
TV
Page
Linda J
LJ
Kubly
Vickie J
VJ
Bergstralh
Eric J
EJ
Li
Xujian
X
Kim
Bohyun
B
King
Bernard F
BF
Glockner
James
J
Holmes
David R
DR
Rossetti
Sandro
S
Harris
Peter C
PC
LaRusso
Nicholas F
NF
Torres
Vicente E
VE
D000328:Adult; D000368:Aged; D004311:Double-Blind Method; D005260:Female; D005919:Glomerular Filtration Rate / Q000187:drug effects; D006801:Humans; D007668:Kidney / Q000187:drug effects / Q000473:pathology / Q000503:physiopathology; D008099:Liver / Q000187:drug effects / Q000201:enzymology / Q000473:pathology; D008107:Liver Diseases / Q000188:drug therapy* / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D015282:Octreotide / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D009929:Organ Size / Q000187:drug effects; D016891:Polycystic Kidney, Autosomal Dominant / Q000188:drug therapy* / Q000503:physiopathology; D013004:Somatostatin / Q000031:analogs & derivatives*; D016896:Treatment Outcome
D013004:Somatostatin; D015282:Octreotide