Effects of rare kidney diseases on kidney failure: a longitudinal analysis of the UK National Registry of Rare Kidney Diseases (RaDaR) cohort
Authors: Katie Wong, David Pitcher, Fiona Braddon, Lewis Downward, Retha Steenkamp, Nicholas Annear, Jonathan Barratt, Coralie Bingham, Constantina Chrysochou, Richard J Coward, David Game, Sian Griffin, Matt Hall, Sally Johnson, Durga Kanigicherla, Fiona Karet Frankl, David Kavanagh, Larissa Kerecuk, Eamonn R Maher, Shabbir Moochhala, Jenny Pinney, John A Sayer, Roslyn Simms, Smeeta Sinha, Shalabh Srivastava, Frederick W K Tam, Andrew Neil Turner, Stephen B Walsh, Aoife Waters, Patricia Wilson, Edwin Wong, Christopher Mark Taylor, Dorothea Nitsch, Moin Saleem, Detlef Bockenhauer, Kate Bramham, Daniel P Gale, Sharirose Abat, Shazia Adalat, Joy Agbonmwandolor, Zubaidah Ahmad, Abdulfattah Alejmi, Rashid Almasarwah, Nicholas Annear, Ellie Asgari, Amanda Ayers, Jyoti Baharani, Gowrie Balasubramaniam, Felix Kpodo, Tarun Bansal, Alison Barratt, Jonathan Barratt, Megan Bates, Natalie Bayne, Janet Bendle, Sarah Benyon, Carsten Bergmann, Sunil Bhandari, Coralie Bingham, Preetham Boddana, Sally Bond, Fiona Braddon, Kate Bramham, Angela Branson, Stephen Brearey, Vicky Brocklebank, Sharanjit Budwal, Conor Byrne, Hugh Cairns, Brian Camilleri, Gary Campbell, Alys Capell, Margaret Carmody, Marion Carson, Tracy Cathcart, Christine Catley, Karine Cesar, Melanie Chan, Houda Chea, James Chess, Chee Kay Cheung, Katy-Jane Chick, Nihil Chitalia, Martin Christian, Tina Chrysochou, Katherine Clark, Christopher Clayton, Rhian Clissold, Helen Cockerill, Joshua Coelho, Elizabeth Colby, Viv Colclough, Eileen Conway, H Terence Cook, Wendy Cook, Theresa Cooper, Richard J Coward, Sarah Crosbie, Gabor Cserep, Anjali Date, Katherine Davidson, Amanda Davies, Neeraj Dhaun, Ajay Dhaygude, Lynn Diskin, Abhijit Dixit, Eunice Doctolero, Suzannah Dorey, Lewis Downard, Mark Drayson, Gavin Dreyer, Tina Dutt, Kufreabasi Etuk, Dawn Evans, Jenny Finch, Frances Flinter, James Fotheringham, Lucy Francis, Daniel P Gale, Hugh Gallagher, David Game, Eva Garcia, Madita Gavrila, Susie Gear, Colin Geddes, Mark Gilchrist, Matt Gittus, Paraskevi Goggolidou, Christopher Goldsmith, Patricia Gooden, Andrea Goodlife, Priyanka Goodwin, Tassos Grammatikopoulos, Barry Gray, Megan Griffith, Steph Gumus, Sanjana Gupta, Patrick Hamilton, Lorraine Harper, Tess Harris, Louise Haskell, Samantha Hayward, Shivaram Hegde, Bruce Hendry, Sue Hewins, Nicola Hewitson, Kate Hillman, Mrityunjay Hiremath, Alexandra Howson, Zay Htet, Sharon Huish, Richard Hull, Alister Humphries, David P J Hunt, Karl Hunter, Samantha Hunter, Marilyn Ijeomah-Orji, Nick Inston, David Jayne, Gbemisola Jenfa, Alison Jenkins, Sally Johnson, Caroline A Jones, Colin Jones, Amanda Jones, Rachel Jones, Lavanya Kamesh, Durga Kanigicherla, Fiona Karet Frankl, Mahzuz Karim, Amrit Kaur, David Kavanagh, Kelly Kearley, Larissa Kerecuk, Arif Khwaja, Garry King, Grant King, Ewa Kislowska, Edyta Klata, Maria Kokocinska, Mark Lambie, Laura Lawless, Thomas Ledson, Rachel Lennon, Adam P Levine, Ling Wai Maggie Lai, Graham Lipkin, Graham Lovitt, Paul Lyons, Holly Mabillard, Katherine Mackintosh, Khalid Mahdi, Eamonn Maher, Kevin J Marchbank, Patrick B Mark, Sherry Masoud, Bridgett Masunda, Zainab Mavani, Jake Mayfair, Stephen McAdoo, Joanna Mckinnell, Nabil Melhem, Simon Meyrick, Shabbir Moochhala, Putnam Morgan, Ann Morgan, Fawad Muhammad, Shona Murray, Kristina Novobritskaya, Albert CM Ong, Louise Oni, Kate Osmaston, Neal Padmanabhan, Sharon Parkes, Jean Patrick, James Pattison, Riny Paul, Rachel Percival, Stephen J Perkins, Alexandre Persu, William G Petchey, Matthew C Pickering, Jennifer Pinney, David Pitcher, Lucy Plumb, Zoe Plummer, Joyce Popoola, Frank Post, Albert Power, Guy Pratt, Charles Pusey, Ria Rabara, May Rabuya, Tina Raju, Chadd Javier, Ian S D Roberts, Candice Roufosse, Adam Rumjon, Alan Salama, Moin Saleem, Richard Sandford, Kanwaljit S Sandu, Nadia Sarween, John A Sayer, Neil Sebire, Haresh Selvaskandan, Asheesh Sharma, Edward J Sharples, Neil Sheerin, Harish Shetty, Rukshana Shroff, Roslyn Simms, Manish Sinha, Smeeta Sinha, Kerry Smith, Lara Smith, Shalabh Srivastava, Retha Steenkamp, Ian Stott, Katerina Stroud, Pauline Swift, Justyna Szklarzewicz, Fred Tam, Kay Tan, Robert Taylor, Marc Tischkowitz, Kay Thomas, Yincent Tse, Alison Turnbull, A Neil Turner, Kay Tyerman, Miranda Usher, Gopalakrishnan Venkat-Raman, Alycon Walker, Stephen B Walsh, Aoife Waters, Angela Watt, Phil Webster, Ashutosh Wechalekar, Gavin I Welsh, Nicol West, David Wheeler, Kate Wiles, Lisa Willcocks, Angharad Williams, Emma Williams, Karen Williams, Deborah H Wilson, Patricia D Wilson, Paul Winyard, Edwin Wong, Katie Wong, Grahame Wood, Emma Woodward, Len Woodward, Adrian Woolf, David Wright
Journal: The Lancet (2024)

BackgroundIndividuals with rare kidney diseases account for 5–10% of people with chronic kidney disease, but constitute more than 25% of patients receiving kidney replacement therapy. The National Registry of Rare Kidney Diseases (RaDaR) gathers longitudinal data from patients with these conditions, which we used to study disease progression and outcomes of death and kidney failure.MethodsPeople aged 0–96 years living with 28 types of rare kidney diseases were recruited from 108 UK renal care facilities. The primary outcomes were cumulative incidence of mortality and kidney failure in individuals with rare kidney diseases, which were calculated and compared with that of unselected patients with chronic kidney disease. Cumulative incidence and Kaplan–Meier survival estimates were calculated for the following outcomes: median age at kidney failure; median age at death; time from start of dialysis to death; and time from diagnosis to estimated glomerular filtration rate (eGFR) thresholds, allowing calculation of time from last eGFR of 75 mL/min per 1·73 m2 or more to first eGFR of less than 30 mL/min per 1·73 m2 (the therapeutic trial window).FindingsBetween Jan 18, 2010, and July 25, 2022, 27 285 participants were recruited to RaDaR. Median follow-up time from diagnosis was 9·6 years (IQR 5·9–16·7). RaDaR participants had significantly higher 5-year cumulative incidence of kidney failure than 2·81 million UK patients with all-cause chronic kidney disease (28% vs 1%; p<0·0001), but better survival rates (standardised mortality ratio 0·42 [95% CI 0·32–0·52]; p<0·0001). Median age at kidney failure, median age at death, time from start of dialysis to death, time from diagnosis to eGFR thresholds, and therapeutic trial window all varied substantially between rare diseases.InterpretationPatients with rare kidney diseases differ from the general population of individuals with chronic kidney disease: they have higher 5-year rates of kidney failure but higher survival than other patients with chronic kidney disease stages 3–5, and so are over-represented in the cohort of patients requiring kidney replacement therapy. Addressing unmet therapeutic need for patients with rare kidney diseases could have a large beneficial effect on long-term kidney replacement therapy demand.FundingRaDaR is funded by the Medical Research Council, Kidney Research UK, Kidney Care UK, and the Polycystic Kidney Disease Charity.