The ERA-EDTA Working Groups on Inherited Kidney Disorders and European Renal Best Practice have published a position statement on the use of tolvaptan in autosomal dominant polycystic kidney disease (ADPKD).
ADPKD is the most common hereditary kidney disorder, accounting for -10% of European patients on dialysis or living with a renal transplant. Approximately 70% of patients with ADPKD progress to end-stage renal disease (ESRD) at a median age of 58 years.
Until recently, treatment options for these patients have been very limited, but in 2015 the European Medicines Agency approved the use of the vasopressin V2 receptor antagonist tolvaptan to slow the progression of cyst development and renal insufficiency. Due to the fact that there is insufficient knowledge to produce evidence based clinical guidelines for the treatment of ADPKD, the ERA-EDTA has now developed provisional recommendations resulting from consensus of ADPKD experts and methodologists on how to implement tolvaptan into the treatment strategy of ADPKD. The recommendations have just been published in Nephrology Dialysis Transplantation (NDT), the leading nephrology journal in Europe.
“Even though consensus documents are inherently weak for producing recommendations for clinical practice, these treatment recommendations may be useful to doctors and patients”, explains Prof. Carmine Zoccali, NDT Editor-in-Chief. As he points out the medication has a number of limitations and side effects and it is probably only suitable for a minority of patients. “Besides, we know that mainly the patients with rapid disease progression might profit of tolvaptan treatment. And among them we have to identify those who are willing to cope with the side-effects of the therapy, namely excessive urination and an immensely increased fluid intake over 5 liter a day in the majority of patients, an effect of tolvaptan, which has an obvious impact on the everyday life of patients.”
The recommendations provide a hierarchical decision algorithm for evaluating disease progression and making the decision which ADPKD patients might be treated with tolvaptan.