Table 2.

mTOR inhibition in human ADPKD

Drug dose levels (ng/ml)	Study design	Duration oftreatment	No of patients	Outcome	Adverse events	Ref
Sirolimus	Retrospective, Kidney transplant patients Sirolimus vs. no sirolimus	24–40 mo	7	Reduction in kidney volume	Not reported	[28]
Sirolimus (mean levels: 14.3)	Retrospective, Kidney transplant patients Sirolimus vs. Tacrolimus	19 mo	16	Reduction in liver cyst volume, trend towards reduction in kidney cyst volume	Increased LDL	[39]
Sirolimus 3 mg/d (trough levels: 10–15, SIRENA study)	Retrospective crossover study, Mean baseline eGFR 76 mL/min	1 y	21	TKV increased less and cyst volume stable on sirolimus	Increased total and LDL cholesterol and triglycerides. aphthous ulcers, acne, edema	[40]
Everolimus 5 mg/d (trough levels: 3–8)	Randomized double blind trial, Mean baseline eGFR 53–56 mL/min	2 y	433	Slowed increase in TKV at 1 y Did not slow progression of eGFR	Anemia, leukopenia, thrombocytopenia, stomatitis, hyperlipidemia, acne, angioedema	[41]
Sirolimus 2 mg/d (mean levels: 4.1–4.9)	Randomized open label trial, Mean baseline eGFR 91–92 mL/min	18 mo	100	No change in TKV or eGFR	Mucositis, diarrhea	[42]

TKV, total kidney volume.