Somatostatin in renal physiology and autosomal dominant polycystic kidney disease

Autosomal dominant polycystic kidney disease (ADPKD) is characterized by progressive cyst formation, leading to growth in kidney volume and renal function decline. Although therapies have emerged, there is still an important unmet need for slowing the rate of disease progression in ADPKD. High intracellular levels of adenosine 30 ,50 -cyclic monophosphate (cAMP) are involved in cell proliferation and fluid secretion, resulting in cyst formation. Somatostatin (SST), a hormone that is involved in many cell processes, has the ability to inhibit intracellular cAMP p

Establishing a core outcome measure for pain in patients with autosomal dominant polycystic kidney disease: a consensus workshop report

Background. Pain is the highest prioritized patient-reported outcome in people with autosomal dominant polycystic kidney disease (ADPKD) but remains infrequently and inconsistently measured in clinical trials and poorly managed in clinical setting. A recently completed systematic review of pain in ADPKD identified 26 different outcome measures. None of these measures were considered appropriate as core outcome measure, due to lack of patient-important dimensions, inadequate content, relatively long duration in completion time and limited evidence to support psychometric robustness. Methods. We convened an international Standardized Outcomes in Nephrology – Polycystic Kidney Disease (SONG-PKD) consensus workshop involving 21 patients/caregivers and 40 health professionals (clinicians, nurses, researchers, policy makers, and industry representatives) from 18 countries to discuss the identification or development of a core outcome measure for pain. Results. Four themes were identified highlighting fundamental issues for the measurement of pain in ADPKD: distressing and disrupting life participation, variability and ambiguity in defining pain, stigma, frustration and adaptation to pain, and ensuring validity and feasibility of pain measures. Conclusions. Existing measures were found to be insufficient in capturing pain as a core outcome and there was consensus on the need for a new validated measure, that is simple, succinct, and addresses the impact of pain on life participation. This measure will facilitate the appropriate prioritization of pain in all trials and guide clinical decision-making in people with ADPKD