Table 2 Completed randomized controlled trials on pharmacotherapy regimens for polycystic liver disease
Author (Trial Registry Code) | Study Design | Included Patients | Arms | Primary Outcome and Safety (Arm 1 in comparison with Arm 2) |
|---|---|---|---|---|
Keimpema et al. 2009 [96] (NCT00565097) | Randomized double-blind parallel assignment | 54 patients with PLD (ADPLD and ADPKD) from the Netherlands and Belgium | 1) Lanreotide-LAR 120 mg SC every 28 days for 24 weeks 2) Placebo | Imaging modality: CT TLV: -2.9% vs. + 1.6%, p-value < 0.01 No severe adverse events related to the intervention |
Hogan et al. 2010 [97] (NCT00426153) | Randomized double-blind parallel assignment | 42 patients with PLD (ADPLD and ADPKD) from the USA | 1) Octreotide-LAR 40 mg IM every 28 days for one year 2) Placebo | Imaging modality: MRI (CT in three patients) TLV: -4.95% vs. + 0.92%, p-value < 0.05 No serious adverse events related to the intervention |
Caroli et al. 2010 [98] (Not registered) (Post-hoc analysis) | Randomized double-blind cross-over assignment | 12 patients with PLD (ADPKD) from Italy | 1) Octreotide-LAR 40 mg IM every 28 days for six months 2) Placebo | Imaging modality: CT TLV change: -71 ± 57 mL vs. + 14 ± 85 mL, p-value < 0.05 |
Pisani et al. 2016 [99] (NCT02119052) (Post-hoc analysis) | Randomized single-blind parallel assignment | 27 patients with PLD (ADPKD) from Italy | 1) Octreotide-LAR 40 mg IM every 28 days for three years 2) Placebo | Imaging modality: MRI TLV change: -7.8% vs. + 6.1%, p-value < 0.01 Treatment-related serious adverse events: one asymptomatic cholelithiasis and one acute cholecystitis |
van Aerts et al. 2019[100] (NCT01616927) (Post-hoc analysis) | Randomized open-label parallel assignment | 175 patients with PLD (ADPKD) from the Netherlands | 1) Lanreotide-LAR 120 mg SC every 28 days for 120 weeks 2) Standard care | Imaging modality: MRI h-TLV: -1.99% vs. + 3.92%, p-value < 0.001 Serious adverse events: 30.1% vs. 12.2% |
Hogan et al. 2020[101] (NCT01670110) | Randomized double-blind parallel assignment | 48 patients with PLD (ADPLD and ADPKD) from USA | 1) Pasireotide-LAR 60 mg IM every 28 days for one year 2) Placebo | Imaging modality: MRI TLV: -3% vs. + 6%, p-value < 0.001 Serious adverse events: 12% vs. 13%, p-value = 0.91 |
Wijnands et al. 2018 [102] (NCT02048319) | Randomized double-blind parallel assignment | 34 patients who underwent aspiration sclerotherapy of a symptomatic dominant liver cyst (23 patients had PLD) from the Netherlands | 1) Pasireotide-LAR 60 mg IM two weeks before and two weeks after the aspiration sclerotherapy 2) Placebo | Imaging modality: ultrasonography Median cyst diameter reduction: 23.6% vs 21.8%, p-value = 0.96 Serious adverse events: 12% vs. 12% |
D'Agnolo et al. 2016 [103] (NCT02021110) | Randomized open-label parallel assignment | 34 patients with PLD (ADPLD and ADPKD) from the Netherlands and Spain | 1) UDCA oral in a dose of 15–20 mg/kg/day for 24 weeks 2) Standard care | Imaging modality: CT TLV change: + 4.6% vs. + 3.1%, p-value = 0.49 No serious adverse events related to the intervention |
Chrispijn et al. 2013 [104] (NCT01157858) | Randomized open-label parallel assignment | 44 patients with PLD (ADPLD and ADPKD) from the Netherlands | 1) Octreotide-LAR 40 mg IM every four weeks + everolimus 2.5 mg oral daily for 48 weeks 2) Octreotide-LAR 40 mg IM every four weeks for 48 weeks | Imaging modality: CT TLV change: -3.8% vs. -3.5%, p-value = 0.73 Serious adverse events: 14% vs. 9% |