geoscience.net logo
+ Resolve Article
+ Follow Us
Follow on FacebookFollow on Facebook
Follow on TwitterFollow on Twitter

+ Translate
+ Subscribe to Site Feed
GeoScience Most Shared ContentMost Shared Content

Low-dose rapamycin (sirolimus) effects in autosomal dominant polycystic kidney disease: an open-label randomized controlled pilot study


, : Low-dose rapamycin (sirolimus) effects in autosomal dominant polycystic kidney disease: an open-label randomized controlled pilot study. Clinical Journal of the American Society of Nephrology 9(5): 881-888

The two largest studies of mammalian target of rapamycin inhibitor treatment of autosomal dominant polycystic kidney disease (ADPKD) demonstrated no clear benefit on the primary endpoint of total kidney volume (TKV) or on eGFR. The present study evaluated two levels of rapamycin on the 12-month change in (125)I-iothalamate GFR (iGFR) as the primary endpoint and TKV secondarily. In a 12-month open-label pilot study, 30 adult patients with ADPKD were randomly assigned to low-dose (LD) rapamycin (rapamycin trough blood level, 2-5 ng/ml) (LD group, n=10), standard-dose (STD) rapamycin trough level (>5-8 ng/ml) (STD group, n=10), or standard care (SC group, n=10). They were evaluated with iGFR and noncontrast computed tomography. Change in iGFR at 12 months was significantly higher in the LD group (7.7±12.5 ml/min per 1.73 m(2); n=9) than in the SC group (-11.2 ± 9.1 ml/min per 1.73 m(2); n=9) (LD versus SC: P<0.01). Change in iGFR at 12 months in the STD group (1.6 ± 12.1 ml/min per 1.73 m(2); n=8) was not significantly greater than that in the SC group (P=0.07), but it was in the combined treatment groups (LD+STD versus SC: P<0.01). Neither eGFR calculated by the CKD-Epidemiology Collaboration equation nor TKV (secondary endpoint) changed significantly from baseline to 12 months in any of the groups. On the basis of results of the mixed model, during the study, patients in the LD group had significantly lower trough blood levels of rapamycin (mean range ± SD, 2.40 ± 0.64 to 2.90 ± 1.20 ng/ml) compared with those in the STD group (3.93 ± 2.27 to 5.77 ± 1.06 ng/ml) (P<0.01). Patients with ADPKD receiving LD rapamycin demonstrated a significant increase in iGFR compared with those receiving standard care, without a significant effect on TKV after 12 months.

US$19.90

PMID: 24721888

DOI: 10.2215/CJN.02650313


Other references

Kyparissoudas D.S., 1989: Control of cydia molesta busck by mating disruption using isomate m pheromone dispensers in northern greece. Trials were carried out in 1986 and 1987 with the mating disruption method for the control of oriental fruit moth (Cydia molesta Busck) in peaches of Northern Greece. One or two applications during April-September of the synthetic pheromone Isomat...

Slade, D.A., 1960: Black rot an important disease of Kumaras. Black rot (Ceratostomella fimbriata) of kumara is increasing in severity in Auckland Province since the first occurrence in 1947. No N.Z. vars. are resistant and control can only be achieved by the use of clean seed, treatment of propagating beds...

Xiao, J-Ping.; Zhang, G-Xia.; Chen, L.; Sun, B-Gui.; Zhang, H-Xu.; Chen, L-Hong.; Yuan, L.; Hao, L.; Wang, D-Guang., 2017: Restless legs syndrome in maintenance hemodialysis patients: an epidemiologic survey in Hefei. To investigate the prevalence of restless legs syndrome (RLS) in maintenance hemodialysis (MHD) patients and its possible influencing factors. MHD patients were consecutively enrolled from five hemodialysis centers in Hefei. Clinical, demographics...

Kylin, Anders, 1966: The influence of photosynthetic factors and metabolic inhibitors on the uptake of phosphate in P-deficient Scenedesmus. The uptake of phosphate at pH 6.5 was determined in cells with an Initial P content of about 20% of that in normal cells, using an experimental period of 4 hr. In the absence of CO2, light increased the uptake with saturation around 14,000 erg/cm2...

Peterson, J.; Kerrison, P.; Green, G.; Bernich, S.; Caldwell, B.; Saewert, M.; Trust, C.; Klose, J.; Lee, S.R., 1999: Confirmatory assay for a hepatitis C core antigen screening ELISA Performance. Transfusion (Bethesda) 39(10 SUPPL ): 64S, Oct

Sarvaiya, R.T., 1978: Prawn fisheries of kutch with special reference to sukhper and lakhpat india. The annual prawn landing of Kutch was estimated to be 1021.5 tonnes during 1973-1974. Metapenaeus kutchensis (George etc.) was a major constituent in prawn landings at the Cherowari, Sukhper and Lakhpat areas of the Little Rann of Kutch. Female pe...

Nagasawa, T.; Takeuchi, K.; Yamada, H., 1991: Characterization of a new cobalt-containing nitrile hydratase purified from urea-induced cells of Rhodococcus rhodochrous J1. A new cobalt-containing nitrile hydratase was purified from extracts of urea-induced cells from Rhodococcus rhodochrous J1 in seven steps. At the last step, the enzyme was crystallized by adding ammonium sulfate. Nitrile hydratase was a 500-530-kD...

Saha, T.; Kand, D.; Talukdar, P., 2014: A colorimetric and fluorometric BODIPY probe for rapid, selective detection of H2S and its application in live cell imaging. A BODIPY-azide based colorimetric and fluorescence turn-ON probe for rapid, selective and sensitive detection of H2S is reported. The probe displayed a fast response time (10 min in HEPES and 30 s in serum albumin), 28-fold fluorescence enhancemen...

Higgins, T., 2016: Commentary. Clinical Chemistry 62(6): 810-810

Richardson, M.D.; Kilts, J.D.; Kwatra, M.M., 2004: Increased expression of Gi-coupled muscarinic acetylcholine receptor and Gi in atrium of elderly diabetic subjects. In an ongoing investigation of the effects of age on G protein-coupled receptor signaling in human atrial tissue, we have found that the density of atrial muscarinic acetylcholine receptor (mAChR) increases with age but reaches statistical signifi...