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- Title
Effect of Lanreotide on Kidney Function in Patients With Autosomal Dominant Polycystic Kidney Disease: The DIPAK 1 Randomized Clinical Trial.
- Authors
Meijer, Esther; Visser, Folkert W.; van Aerts, Rene M. M.; Blijdorp, Charles J.; Casteleijn, Niek F.; D'Agnolo, Hedwig M. A.; Dekker, Shosha E. I.; Drenth, Joost P. H.; de Fijter, Johan W.; van Gastel, Maatje D. A.; Gevers, Tom J.; Lantinga, Marten A.; Losekoot, Monique; Messchendorp, A. Lianne; Neijenhuis, Myrte K.; Pena, Michelle J.; Peters, Dorien J. M.; Salih, Mahdi; Soonawala, Darius; Spithoven, Edwin M.
- Abstract
<bold>Importance: </bold>Autosomal dominant polycystic kidney disease (ADPKD) is characterized by progressive cyst formation in both kidneys and loss of renal function, eventually leading to a need for kidney replacement therapy. There are limited therapeutic management options.<bold>Objective: </bold>To examine the effect of the somatostatin analogue lanreotide on the rate of kidney function loss in patients with later-stage ADPKD.<bold>Design, Setting, and Participants: </bold>An open-label randomized clinical trial with blinded end point assessment that included 309 patients with ADPKD from July 2012 to March 2015 at 4 nephrology outpatient clinics in the Netherlands. Eligible patients were 18 to 60 years of age and had an estimated glomerular filtration rate (eGFR) of 30 to 60 mL/min/1.73 m2. Follow-up of the 2.5-year trial ended in August 2017.<bold>Interventions: </bold>Patients were randomized to receive either lanreotide (120 mg subcutaneously once every 4 weeks) in addition to standard care (n = 153) or standard care only (target blood pressure <140/90 mm Hg; n = 152).<bold>Main Outcomes and Measures: </bold>Primary outcome was annual change in eGFR assessed as slope through eGFR values during the 2.5-year treatment phase. Secondary outcomes included change in eGFR before vs after treatment, incidence of worsening kidney function (start of dialysis or 30% decrease in eGFR), change in total kidney volume and change in quality of life (range: 1 [not bothered] to 5 [extremely bothered]).<bold>Results: </bold>Among the 309 patients who were randomized (mean [SD] age, 48.4 [7.3] years; 53.4% women), 261 (85.6%) completed the trial. Annual rate of eGFR decline for the lanreotide vs the control group was -3.53 vs -3.46 mL/min/1.73 m2 per year (difference, -0.08 [95% CI, -0.71 to 0.56]; P = .81). There were no significant differences for incidence of worsening kidney function (hazard ratio, 0.87 [95% CI, 0.49 to 1.52]; P = .87), change in eGFR (-3.58 vs -3.45; difference, -0.13 mL/min/1.73 m2 per year [95% CI, -1.76 to 1.50]; P = .88), and change in quality of life (0.05 vs 0.07; difference, -0.03 units per year [95% CI, -0.13 to 0.08]; P = .67). The rate of growth in total kidney volume was lower in the lanreotide group than the control group (4.15% vs 5.56%; difference, -1.33% per year [95% CI, -2.41% to -0.24%]; P = .02). Adverse events in the lanreotide vs control group included injection site discomfort (32% vs 0.7%), injection site papule (5.9% vs 0%), loose stools (91% vs 6.6%), abdominal discomfort (79% vs 20%), and hepatic cyst infections (5.2% vs 0%).<bold>Conclusions and Relevance: </bold>Among patients with later-stage autosomal dominant polycystic kidney disease, treatment with lanreotide compared with standard care did not slow the decline in kidney function over 2.5 years of follow-up. These findings do not support the use of lanreotide for treatment of later-stage autosomal dominant polycystic kidney disease.<bold>Trial Registration: </bold>ClinicalTrials.gov Identifier: NCT01616927.
- Publication
JAMA: Journal of the American Medical Association, 2018, Vol 320, Issue 19, p2010
- ISSN
0098-7484
- Publication type
journal article
- DOI
10.1001/jama.2018.15870