Sirolimus Therapy for Patients With Adult Polycystic Kidney Disease: A Pilot Study

Soliman, Amin R.; Ismail, Eman I.; Zamil, S.; Lotfy, A.

doi:10.1016/j.transproceed.2009.05.032

Cited by 15 publications

(9 citation statements)

References 9 publications

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“…152, 168 They improve immune response in old animals. 169 Rapamycin was used to treat insulinoma, 170, 171 polycystic kidney disease, 172 systemic sclerosis 173 and prevention of atherosclerotic in-stent restenosis. 130, 131, 132 Now is the turn of diabetic complications.…”

Section: Conclusion For Endocrinologistsmentioning

confidence: 99%

TOR-centric view on insulin resistance and diabetic complications: perspective for endocrinologists and gerontologists

2013

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This article is addressed to endocrinologists treating patients with diabetic complications as well as to basic scientists studying an elusive link between diseases and aging. It answers some challenging questions. What is the link between insulin resistance (IR), cellular aging and diseases? Why complications such as retinopathy may paradoxically precede the onset of type II diabetes. Why intensive insulin therapy may initially worsen retinopathy. How nutrient- and insulin-sensing mammalian target of rapamycin (mTOR) pathway can drive insulin resistance and diabetic complications. And how rapamycin, at rational doses and schedules, may prevent IR, retinopathy, nephropathy and beta-cell failure, without causing side effects.

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Section: Conclusion For Endocrinologistsmentioning

confidence: 99%

TOR-centric view on insulin resistance and diabetic complications: perspective for endocrinologists and gerontologists

2013

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“…A small benefit or no benefit in TKV change is exhibited from mTOR inhibitor use (Table 1). In the four sirolimus trials, excluding the Braun trial (32)(33)(34)(35), the change in GFR did not reach a statistically significant difference between sirolimus versus control groups. Sirolimus associated with significant side effects in all trials, including stomatitis, acne, increased infections, increased proteinuria, and hyperlipidemia.…”

Section: Mtor Pathway Inhibitionmentioning

confidence: 84%

“…Sirolimus associated with significant side effects in all trials, including stomatitis, acne, increased infections, increased proteinuria, and hyperlipidemia. In fact, dropout from the sirolimus group reached 29% in the Perico et al trial (32)(33)(34)(35). Walz et al evaluated everolimus over 24 months in a larger number of participants across a wide range of disease stages (37).…”

Section: Mtor Pathway Inhibitionmentioning

confidence: 99%

Novel Treatments of Autosomal Dominant Polycystic Kidney Disease

Mahnensmith¹

2014

Clinical Journal of the American Society of Nephrology

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“…To date, the results of six clinical trials, five using sirolimus and one using everolimus, have been published. [108][109][110][111][112][113] The trial durations were short, with the longest trial being 24 months. Only one small study showed an improvement in renal function in patients treated with sirolimus.…”

Section: Inhibition Of Mtormentioning

confidence: 99%

Autosomal dominant polycystic kidney disease: genetics, epidemiology, and treatment

Reed-Gitomer¹

2014

AGG

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Abstract:Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially lethal genetic disorder, accounting for 2%-8% of end-stage renal disease worldwide. While development of renal cysts is the major characteristic of ADPKD, several disease-related complications contribute significantly to morbidity and mortality. Since introduction of renal replacement therapies, cardiovascular disease is the leading cause of death among ADPKD patients. Loss of renal function requiring renal replacement therapy occurs in 50% of patients by the age of 60 years. The results of recent large epidemiological studies in ADPKD have shown little progress in delaying onset of renal failure despite an improvement in patient mortality. Significant progress has been made over the past decade in elucidating the genetics of the disorder. Genetic testing is now available in most countries, and development of more reliable methods for prenatal diagnosis of ADPKD has increased the sensitivity of testing. While there are no approved drugs for treatment of ADPKD within the USA, the first agent targeting renal disease progression in this disorder was recently approved for clinical use in Japan. Furthermore, several additional drugs for treatment of ADPKD are currently under clinical investigation. Overall, this presents an optimistic future for new therapeutic interventions in this disease. This paper reviews the current knowledge related to the epidemiology, genetics, and treatment of ADPKD.

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Sirolimus Therapy for Patients With Adult Polycystic Kidney Disease: A Pilot Study

Cited by 15 publications

References 9 publications

TOR-centric view on insulin resistance and diabetic complications: perspective for endocrinologists and gerontologists

TOR-centric view on insulin resistance and diabetic complications: perspective for endocrinologists and gerontologists

Novel Treatments of Autosomal Dominant Polycystic Kidney Disease

Autosomal dominant polycystic kidney disease: genetics, epidemiology, and treatment

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