Reducing Polycystic Liver Volume in ADPKD

Abstract

Background and objectives: No medical treatment is available for polycystic liver disease, a frequent manifestation of autosomal-dominant polycystic kidney disease (ADPKD). In a prospective, randomized, double-blind, crossover study, 6 months of octreotide (40 mg every 28 days) therapy limited kidney volume growth more effectively than placebo in 12 patients with ADPKD.Design, setting, participants, & measurements: In this secondary, post hoc analysis of the above study, octreotide-induced changes in liver volumes compared with placebo and the relationship between concomitant changes in liver and kidney volumes were evaluated. Those analyzing liver and kidney volumes were blinded to treatment.Results: Liver volumes significantly decreased from 1595 ؎ 478 ml to 1524 ؎ 453 ml with octreotide whereas they did not appreciably change with placebo. Changes in liver volumes were significantly different between the two treatment periods (؊71 ؎ 57 ml versus ؉14 ؎ 85 ml). Octreotide-induced liver volume reduction was fully explained by a reduction in parenchyma volume from 1506 ؎ 431 ml to 1432 ؎ 403 ml. Changes in liver volumes were significantly correlated with concomitant changes in kidney volumes (r ‫؍‬ 0.67) during octreotide but not during placebo treatment. Liver and kidney volume changes significantly differed with both treatments (octreotide: ؊71 ؎ 57 ml versus ؉71 ؎ 107; placebo: ؉14 ؎ 85 ml versus ؉162 ؎ 114), but net reductions in liver (؊85 ؎ 103 ml) and kidney (؊91 ؎ 125 ml) volume growth on octreotide versus placebo were similar.Conclusions: Octreotide therapy reduces liver volumes in patients with ADPKD and is safe.

Medical and surgical treatment options for polycystic liver disease1

“…Liver volume decreased by 4.4% during octreotide administration, whereas it increased by 1.2% with placebo. 6 The volume-reducing effect of octreotide is not dependent on its formulation. Short-acting octreotide administered at a dose of 100 lg three times daily subcutaneously for 70-180 days in eight patients (seven ADPKD; one PCLD) resulted in a median reduction of liver volume by 3.0% 55 (Fig.…”

“…The common thinking is that the natural history of PLD is compatible with a continuous growth in number and size of cysts. Data from three recent trials [4][5][6] indicate that the annual growth of polycystic livers is $0.9%-3.2% (Fig. 5).…”

“…5 The fifth and sixth bar show that 6 months of octreotide give a reduction of liver volume of 4.0%, whereas it increases with 1.2% on placebo. 6 The last bar shows the results of a trial with octreotide given for 4.5 months. Liver volume reduces with 3.0%.…”

“…Answers might come from ongoing trials that evaluate the effect of a 3-year treatment. 6 Finally, whereas somatostatin analogs are well tolerated, the side-effect profile is less acceptable with mTOR inhibitors. 59,60 Conclusion PLD is a progressive disease, and a substantial minority of patients will develop severe symptoms.…”

Development and validation of a polycystic liver disease complaint-specific assessment (POLCA)

“…Using search terms referring to the disease, its treatment forms and aspects of health-related quality of life, 15 articles were identified for data extraction [4,5,7,8,[17][18][19][20][21][22][23][24][25][26][27].…”