Rare kidney diseases encompass at least 150 different conditions, most of which are inherited. Although individual rare kidney diseases raise specific issues, as a group these rare diseases can have overlapping challenges in diagnosis and treatment. These challenges include small numbers of affected patients, unidentified causes of disease, lack of biomarkers for monitoring disease progression, and need for complex care. To address common clinical and patient issues among rare kidney diseases, the KDIGO Controversies Conference entitled, Common Elements in Rare Kidney Diseases, brought together a panel of multidisciplinary clinical providers and patient advocates to address five central issues for rare kidney diseases. These issues encompassed diagnostic challenges, management of kidney functional decline and progression of chronic kidney disease, challenges in clinical study design, translation of advances in research to clinical care, and provision of practical and integrated patient support. Thus, by a process of consensus, guidance for addressing these challenges was developed and is presented here.
Bibliographical noteFunding Information:
The financial impact of a chronic disease is considerable and long-lasting for patients and their caregivers. Although available financial support varies considerably throughout the globe, patient support organizations can be reliable sources of information. Some patient organizations also provide financial assistance programs to patients or grants to researchers.
SA declared having received consultancy fees from Biogen. DB declared having received consultancy fees from Raptor. SD declared having received consultancy fees from Baxalta, Pharmalink, and Shire. OD declared having received research support from Otsuka. LMG-W declared having received consultancy fees from Otsuka and research support from the National Institutes of Health (National Center for Advancing Translational Sciences and National Institute of Diabetes and Digestive and Kidney Diseases). JBK declared having received consultancy fees from Akebia. NVAMK declared having received speaker honoraria from Seinen Congres Event Management and Foundation Devenir. RDP declared having received consultancy fees from Genzyme, Mitsubishi Tanabe, Otsuka, Palladio, and Regulus; speaker honoraria from Otsuka Canada; and research support from Otsuka and the US Department of Defense. FS declared having received consultancy fees from Akebia, Alexion, Amgen, Bayer, Fresenius Medical Care, Otsuka, and Roche, and research support from Fresenius Medical Care. VET declared having research support from Otsuka. DCW declared having received consultancy fees from Akebia, Amgen, Boehringer Ingelheim, Bristol Myers Squibb, GSK, Janssen, Otsuka, UCB Celltech, and Vifor Fresenius Medical Care Renal Pharma; speaker honoraria from Amgen, Fresenius Medical Care, Janssen, Vifor Fresenius Medical Care Renal Pharma, and ZS Pharma; and research support from Australian National Health & Medical Research Council, British Heart Foundation, Healthcare Quality Improvement Partnership, Kidney Research UK, and the National Institute for Health Research. WCW declared having received consultancy fees from Akebia, AMAG Pharmaceuticals, Amgen, AstraZeneca, Bayer, Daichi Sankyo, Medtronic, Relypsa, and Vifor Fresenius Medical Care Renal Pharma. All the other authors declared no competing interests.
© 2017 International Society of Nephrology
- chronic kidney disease progression
- clinical trials
- genetic kidney diseases
- practical and integrated patient support
- translational care