S1 Appendix -

(DOCX)</p

PRISMA table [41].

(DOCX)</p

Data from SBMC^b'*' and Aarohi^b'**' on types of POCT used during consultation.

Data from SBMCb'*' and Aarohib'**' on types of POCT used during consultation.</p

Comparison of the design of 2 randomised controlled trials for vasculitis: MYPAN versus MYCYC.

<p>^aMYPAN: Mycophenolate mofetil for childhood polyarteritis nodosa;</p><p>^bPAN: polyarteritis nodosa;</p><p>^cMYCYC: Mycophenolate mofetil versus cyclophosphamide for ANCA associated vasculitis;</p><p>^dANCA: anti neutrophil cytoplasmic antibodies;</p><p>^eMMF: Mycophenolate mofetil;</p><p>^fCYC: cyclophosphamide;</p><p>^gPVAS: Paediatric Vasculitis Activity Score;</p><p>^hBVAS: Birmingham Vasculitis Activity Score.</p><p>Comparison of the design of 2 randomised controlled trials for vasculitis: MYPAN versus MYCYC.</p

Expert prior opinion before introduction of the MYCYC data regarding 6-month remission rates using treatment with CYC or MMF for children with PAN.

<p><b>Reprinted from [<a href="http://www.plosone.org/article/info:doi/10.1371/journal.pone.0120981#pone.0120981.ref007" target="_blank">7</a>] under a CC BY license, with permission from the authors, original copyright 2014.</b> Prior opinion was that the most likely value for p_C was 0.7; 90% and 50% credibility intervals were (0.30, 0.91) and (0.50, 0.78), respectively. The effective sample size was 5 patients on CYC. The prior for p_M is derived from those for p_C and θ. It had mode = 0.65; 90% and 50% credibility intervals were (0.21, 0.90) and (0.41, 0.74), respectively.</p

Flow diagram illustrating the sequence of activities undertaken during the MYPAN prior elicitation meeting and the time allocated to each activity.

<p>Flow diagram illustrating the sequence of activities undertaken during the MYPAN prior elicitation meeting and the time allocated to each activity.</p

Influence of the MYCYC trial results on expert prior opinion regarding 6-month remission rates using treatment with CYC or MMF for children with PAN. Reprinted from [7] under a CC BY license, with permission from the authors, original copyright 2014.

<p><a href="http://www.plosone.org/article/info:doi/10.1371/journal.pone.0120981#pone.0120981.g004" target="_blank">Fig 4A</a>): Influence of MYCYC results on prior opinion for p_C. The modified prior distribution for p_C after considering the MYCYC results had mode = 0.74; 90% and 50% credibility intervals were (0.51, 0.86) and (0.63, 0.78), respectively. This level of certainty is equivalent to what would be obtained from a clinical trial involving 17 patients treated with CYC (effective sample size). <a href="http://www.plosone.org/article/info:doi/10.1371/journal.pone.0120981#pone.0120981.g004" target="_blank">Fig 4B</a>): Influence of MYCYC results on prior opinion for p_M. The modified prior for p_M after considering the MYCYC results had mode = 0.71; 90% and 50% credibility intervals were (0.45, 0.85) and (0.59, 0.76), respectively. <a href="http://www.plosone.org/article/info:doi/10.1371/journal.pone.0120981#pone.0120981.g004" target="_blank">Fig 4C</a>): Comparison of the final expert prior opinions for p_C and p_M incorporating the MYCYC data.</p

Effects of rare kidney diseases on kidney failure: a longitudinal analysis of the UK National Registry of Rare Kidney Diseases (RaDaR) cohort

Individuals 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.People 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).Between 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 Background Methods Findings Interpretation Funding</p