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Somatostatin analog therapy for severe polycystic liver disease: results after 2 years.

ABSTRACT: We showed in a randomized double-blinded placebo-controlled clinical trial that octreotide long-acting repeatable depot.® (OctLAR(®)) for 12 months reduces kidney and liver growth in autosomal dominant polycystic kidney patients with severe polycystic liver disease (PLD) and liver growth in patients with severe isolated PLD. We have now completed an open-label extension for one additional year to assess safety and clinical benefits of continued use of OctLAR for 2 years (O ? O) and examined drug effect in the placebo group who crossed over to OctLAR in Year 2 (P ? O).The primary end point was change in total liver volume (TLV) measured by magnetic resonance imaging (MRI); secondary end points were changes in total kidney volume (TKV) measured by MRI, glomerular filtration rate (GFR), quality of life (QOL), safety, vital signs and laboratory parameters.Forty-one of 42 patients received OctLAR (n = 28) or placebo (n = 14) in Year 1 and received OctLAR in Year 2 (maximum dose 40 mg). Patients originally randomized to placebo (P ? O) showed substantial reduction in TLV after treatment with OctLAR in Year 2 (?% -7.66 ± 9.69%, P = 0.011). The initial reduction of TLV in the OctLAR group (O ? O) was maintained for 2 years (?% -5.96 ± 8.90%), although did not change significantly during Year 2 (?% -0.77 ± 6.82%). OctLAR inhibited renal enlargement during Year 1 (?% +0.42 ± 7.61%) in the (O ? O) group and during Year 2 (?% -0.41 ± 9.45%) in the (P ? O) group, but not throughout Year 2 (?% +6.49 ± 7.08%) in the (O ? O) group. Using pooled analyses of all individuals who received OctLAR for 12 months, i.e. in Year 1 for O ? O patients and Year 2 for P ? O patients, average reduction in TLV was -6.08 ± 7.58% (P = 0.001) compared to net growth of 0.9 ± 8.35% in the original placebo group. OctLAR-treated individuals continued to experience improvements in QOL in Year 2, although overall physical and mental improvements were not significant during Year 2 compared to Year 1. Changes in GFR were similar in both groups.Over 2 years, OctLAR significantly reduced the rate of increase in TLV and possibly the rate of increase in TKV.

SUBMITTER: Hogan MC

PROVIDER: S-EPMC3433774 | biostudies-literature | 2012 Sep

REPOSITORIES: biostudies-literature

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Somatostatin analog therapy for severe polycystic liver disease: results after 2 years.

Hogan Marie C MC Masyuk Tetyana V TV Page Linda L Holmes David R DR Li Xujian X Bergstralh Eric J EJ Irazabal Maria V MV Kim Bohyun B King Bernard F BF Glockner James F JF Larusso Nicholas F NF Torres Vicente E VE

Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association 20120706 9

<h4>Background</h4>We showed in a randomized double-blinded placebo-controlled clinical trial that octreotide long-acting repeatable depot.® (OctLAR(®)) for 12 months reduces kidney and liver growth in autosomal dominant polycystic kidney patients with severe polycystic liver disease (PLD) and liver growth in patients with severe isolated PLD. We have now completed an open-label extension for one additional year to assess safety and clinical benefits of continued use of OctLAR for 2 years (O → O ...[more]

PMID: 22773240

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Project description:ObjectivesA number of randomised control trials (RCTs) investigating the effects of long-acting somatostatin analogues in autosomal dominant polycystic kidney disease (ADPKD) and polycystic liver disease (PLD) have been recently reported. We sought to evaluate all available RCTs investigating the efficacy of somatostatin analogues treatment in ADPKD and PLD.Data sourcesElectronic databases; Pubmed, Clincaltrials.gov and Cochrane Central Register of Controlled Trials ELIGIBILITY CRITERIA FOR SELECTING STUDIES: RCTs and randomised cross-over trials comparing the effects of somatostatin analogue treatment with controls in patients with ADPKD or PLD.Data extraction and synthesisData extraction and bias assessments were performed by two independent reviewers between January and May 2019. Outcomes assessed included estimated glomerular filtration rate (eGFR), total kidney volume (TKV), total liver volume (TLV), progression to end stage renal failure (ESRF) and adverse effects. Data were pooled using a random-effects model and reported as relative risk or mean difference with 95% CIs.ResultsMeta-analysis was performed of six RCTs or randomised cross-over trials and three secondary analyses. A total of 592 patients were included. Compared with controls, somatostatin analogue treatment significantly reduced TLV (mean difference -0.15 L, 95%?CI -0.26 to -0.03, p=0.01). There was no significant effect on TKV (mean difference -0.19 L, 95%?CI -0.50 to 0.12, p=0.23) or eGFR (mean difference 0.27?mL/min/1.73?m2, 95%?CI -2.03 to 2.57, p=0.82). There was no effect on progression to ESRF. Somatostatin analogues were associated with known adverse effects such as gastrointestinal symptoms.ConclusionsThe available RCT data show improvement in TLV with somatostatin analogue treatment. There was no benefit to TKV or eGFR in patients with ADPKD, while being associated with various side effects. Further studies are needed to assess potential benefit in reducing cyst burden in patients with PLD.

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Somatostatin analog therapy for severe polycystic liver disease: results after 2 years.

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Somatostatin analog therapy for severe polycystic liver disease: results after 2 years.

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