Cystic Kidney Disease: Drug Therapy

Therapeutic targets

Therapy recommendations

  • Antihypertensives in hypertension [see below Hypertension or chronic renal failure].
    • Inhibitors of the renin-angiotensin-aldosterone system (RAAS) may favorably affect the progression of renal failure:
      • ACE inhibitors (nephroprotection (“kidney protection”); first-line agents); and
      • Angiotension II receptor antagonists (angiotensin receptor blockers, ARBs; nephroprotection) in hypertensive diabetic and nondiabetic adults with chronic kidney disease and albuminuria (appearance of albumin in urine) of >300 mg/d.
  • Tolvaptan (oral; selective antagonist of V2 vasopressin receptor) – inhibition of progression of renal cyst growth in ADPKD and progression of renal failure; in PKD1 mutation, progression is significantly slowedDose: initially 45 mg morning and 15 mg evening (maximum dose 90 mg morning and 30 mg evening)Side effects: Tolvaptan is potentially liver toxic; regular determination of ALT(alanine aminotransferase) and AST(aspartate aminotransferase) and total bilirubin is required monthly for 18 months and regularly every 3 months thereafter.Other side effects include hyperuricemia (increased uric acid concentrations in the blood) and increased gout episodes, as well as polyuria (increased urine output: > 1.5-3 l/day) and polydipsia (increased sense of thirst that is associated with excessive fluid intake through drinking)
  • Therapy of renal insufficiency (kidney weakness) depending on the stage (see under renal insufficiency).
  • Therapy of urinary tract infection (see below cystitis / cystitis or pyelonephritis / lateral infection of the renal pelvis with involvement of the renal parenchyma (kidney tissue)).
  • Surgical removal of cysts – indicated only in cases of severe pain.
  • See also under “Further therapy”.

Further notes

  • In autosomal dominant polycystic kidney disease (ADPKD) in patients with CKD stages 1 to 3 (and increased risk of dialysis): tolvaptan (vasopressin antagonist); this may slow rapidly progressive disease (reduction in renal function decline of 32% over a three-year period)
  • In a one-year, multicenter, randomized, double-blind, placebo-controlled phase IIIb study REPRISE, tolvaptan was able to slow eGFR (estimated GFR; estimated glomerular filtration rate) decline even in the later stages of ADPKD