. Author manuscript; available in PMC: 2015 Aug 5.

Published in final edited form as: Nat Rev Gastroenterol Hepatol. 2014 Sep 30;11(12):750–761. doi: 10.1038/nrgastro.2014.155

Table 3.

Clinical trials in polycystic liver diseases

Study	Patient randomization	Treatment			Liver volume reduction
Study	Patient randomization	Dose	Duration	Route of administration	Liver volume reduction
*Octreotide (targets cAMP)*
Caroli et al. (2010)¹⁰⁰	ADPKD, n = 12 (5 treated, 7 placebo)	40 mg every 28 days	6 months	Intramuscular	4.5% (vs 0.9% increase in placebo)
Hogan et al. (2010)¹⁰¹	ADPKD and ADPLD, n = 42 (28 treated, 14 placebo)	40 mg every 28 (± 5) days	12 months	Intramuscular	4.95% (vs 0.92% increase in placebo)
Hogan et al. (2012)¹⁰²	Extension study: ADPKD and ADPLD, n = 41/42 (all received treatment)	40 mg every 28 (± 5) days	Additional 12 months (24 months total)	Intramuscular	No significant changes (−0.77%) after an additional year of therapy
*Lanreotide (targets cAMP)*
Temmerman et al. (2013)¹⁰⁵	ADPKD and ADPLD, n = 132 (106 treated, 26 placebo)	90 mg (n = 55) Or 120 mg (n = 51) every 28 days	6 months	Subcutaneous	1.4% (LAN 90 mg) 2.8% (LAN 120 mg) (vs 1.1% increase in placebo) LAN 90 mg had fewer adverse effects
van Keimpema et al. (2009)¹⁰³	ADPKD and ADPLD, n = 54 (28 treated, 27 placebo)	120 mg every 28 days	24 weeks	Subcutaneous	2.9% (vs 1.6% increase in placebo)
Chrispijn et al. (2012)¹⁰⁴	Extension study: ADPKD and ADPLD, n = 41/54 (all received treatment)	120 mg every 28 days	12 months	Subcutaneous	4%
*Sirolimus vs tacrolimus (targets mTOR)*
Qian et al. (2008)⁸⁵	ADPKD (sirolimus n = 7, tacrolimus n = 9)	5–10 mg daily (sirolimus) 3 mg twice day (tacrolimus)	Retrospective analysis 19.4 months	Oral	11.9% decrease with sirolimus vs 14.1% increase with tacrolimus
*Everolimus alone or in combination with octreotide (targets cAMP and mTOR)*
Chrispijn et al. (2013)⁸⁸	ADPKD and ADPLD, n = 44 (23 received octreotide and 21 received octreotide + everolimus)	40 mg octreotide every 4 weeks, 2.5 mg everolimus daily	48 weeks	Intramuscular (octreotide) and oral (everolimus)	3.5 ± 5.2% octreotide monotherapy vs 3.8 ± 4.7% in the octreotide/everolimus group (in response to octreotide, everolimus does not further reduce liver volume)

Clinical trial NCT01670110 testing pasireotide in severe polycystic liver disease is underway (Mayo Clinic).107 Abbreviations: ADPKD, autosomal dominant polycystic kidney disease; ADPLD, autosomal dominant polycystic liver disease; cAMP, cyclic adenosine monophosphate; mTOR, mammalian target of rapamycin.