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Marie C Hogan1, Tetyana V Masyuk, Linda Page
1Department of Internal Medicine, Division of Nephrology and Hypertension, Mayo Clinic, Rochester, MN, USA. hogan.marie@mayo.edu
Background:
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).
Methods:
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.
Results:
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 (Δ% -7.66 ± 9.69%, P = 0.011). The initial reduction of TLV in the OctLAR group (O → O) was maintained for 2 years (Δ% -5.96 ± 8.90%), although did not change significantly during Year 2 (Δ% -0.77 ± 6.82%). OctLAR inhibited renal enlargement during Year 1 (Δ% +0.42 ± 7.61%) in the (O → O) group and during Year 2 (Δ% -0.41 ± 9.45%) in the (P → O) group, but not throughout Year 2 (Δ% +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.
Conclusion:
Over 2 years, OctLAR significantly reduced the rate of increase in TLV and possibly the rate of increase in TKV.
Insights
Octreotide long-acting repeatable depot (OctLAR) significantly reduced liver and kidney growth in autosomal dominant polycystic kidney disease patients over two years. Continued treatment maintained these benefits, improving quality of life.
Area of Science:
- Nephrology
- Hepatology
- Pharmacology
Background:
- Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterized by kidney and liver cyst development.
- Polycystic liver disease (PLD) can lead to significant morbidity and reduced quality of life in ADPKD patients.
- Octreotide long-acting repeatable depot (OctLAR) has shown potential in managing cyst growth in ADPKD.
Purpose of the Study:
- To assess the safety and clinical benefits of two-year OctLAR treatment in ADPKD patients with severe PLD.
- To evaluate the efficacy of OctLAR in patients initially receiving placebo and then switching to OctLAR.
- To determine the long-term effects of OctLAR on total liver volume (TLV) and total kidney volume (TKV).
Main Methods:
- A randomized, double-blinded, placebo-controlled trial with a one-year open-label extension.
- Magnetic resonance imaging (MRI) was used to measure TLV and TKV.
- Secondary endpoints included glomerular filtration rate (GFR), quality of life (QOL), and safety parameters.
Main Results:
- OctLAR treatment for two years significantly reduced TLV, with benefits maintained from year one.
- Patients switching from placebo to OctLAR showed a substantial reduction in TLV in the second year.
- OctLAR demonstrated a trend towards inhibiting renal enlargement, particularly in the crossover group.
Conclusions:
- Two-year OctLAR treatment effectively reduces liver volume and potentially kidney volume in ADPKD patients.
- OctLAR is safe and well-tolerated, with sustained improvements in quality of life.
- OctLAR represents a promising therapeutic option for managing severe PLD in ADPKD.
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