How rare kidney diseases contribute to kidney failure.
Rare kidney diseases are a collection of over 150 conditions, mostly inherited, with a prevalence of 60-80 cases per 100,000 individuals in Europe and the USA. Often, the diagnosis of these rare kidney diseases is delayed, leading a significant number of patients to progress to kidney failure, necessitating kidney replacement therapy. Despite the impact of these diseases, there is limited information on their progression rates.
A groundbreaking study conducted by Katie Wong and colleagues for the UK National Registry of Rare Kidney Diseases (RaDaR) consortium, recently published in The Lancet, provides crucial insights into this realm. This extensive study utilized data from the world's largest rare kidney disease registry, encompassing 27,285 UK patients aged 0-96 years with 28 different rare kidney diseases. The analysis revealed that the median age at diagnosis across the RaDaR population was 41 years, with considerable variation across different conditions, ranging from 2 years for cystinosis to 63 years for monoclonal gammopathy of renal significance.
Importantly, the study found high 5-year kidney failure rates in RaDaR participants, surpassing those in the general UK population with all-cause chronic kidney disease. The observed rates of kidney failure exceeded predictions by the Kidney Failure Risk Equation, indicating the severity of kidney complications in these rare diseases. However, there was notable heterogeneity in the median age at kidney failure among different rare disease groups.
Furthermore, Wong and colleagues reported that patients in RaDaR had a higher mortality rate compared to the general English and Welsh populations. Particularly, those with kidney failure had a significantly elevated mortality rate. Despite this, the overall survival rates among RaDaR patients were better than those in the general chronic kidney disease population. Notably, patients with rare kidney diseases on dialysis exhibited improved survival rates across all age groups compared to individuals with kidney failure from common causes like diabetes or hypertension.
The study's findings underscore the need for policymakers and clinicians to acknowledge the significant impact of rare kidney diseases on kidney failure. Contrary to common belief, rare kidney diseases, though constituting a smaller fraction of the chronic kidney disease population, account for a substantial portion of individuals requiring kidney replacement therapy due to their unique disease characteristics. Addressing the unmet need for effective therapies in rare kidney diseases could alleviate the long-term demand for kidney replacement therapy and mitigate the economic burden on healthcare systems.
The study calls for immediate action to overcome obstacles in developing treatments for rare kidney diseases, such as the lack of reliable biomarkers for disease monitoring and the clinical heterogeneity of these conditions. It emphasizes the importance of distinguishing patients with rare kidney diseases from those with more common causes of chronic kidney disease to enable early specialist referral and tailored treatments aimed at delaying disease progression.
Additionally, there is a crucial requirement for accurate tools to predict kidney failure risk in patients with rare kidney diseases, as the current Kidney Failure Risk Equation underestimates this risk for these individuals. The integration of big data analytics and machine learning algorithms from registries like RaDaR could offer valuable insights into rare kidney diseases and pave the way for personalized medicine in the future.
In conclusion, the findings from this study shed light on the substantial impact of rare kidney diseases on kidney failure and mortality rates. They emphasize the urgency of developing targeted therapies, improving diagnostic approaches, and enhancing predictive tools to optimize care for individuals with rare kidney diseases.
Source: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(24)00198-3/fulltext
A groundbreaking study conducted by Katie Wong and colleagues for the UK National Registry of Rare Kidney Diseases (RaDaR) consortium, recently published in The Lancet, provides crucial insights into this realm. This extensive study utilized data from the world's largest rare kidney disease registry, encompassing 27,285 UK patients aged 0-96 years with 28 different rare kidney diseases. The analysis revealed that the median age at diagnosis across the RaDaR population was 41 years, with considerable variation across different conditions, ranging from 2 years for cystinosis to 63 years for monoclonal gammopathy of renal significance.
Importantly, the study found high 5-year kidney failure rates in RaDaR participants, surpassing those in the general UK population with all-cause chronic kidney disease. The observed rates of kidney failure exceeded predictions by the Kidney Failure Risk Equation, indicating the severity of kidney complications in these rare diseases. However, there was notable heterogeneity in the median age at kidney failure among different rare disease groups.
Furthermore, Wong and colleagues reported that patients in RaDaR had a higher mortality rate compared to the general English and Welsh populations. Particularly, those with kidney failure had a significantly elevated mortality rate. Despite this, the overall survival rates among RaDaR patients were better than those in the general chronic kidney disease population. Notably, patients with rare kidney diseases on dialysis exhibited improved survival rates across all age groups compared to individuals with kidney failure from common causes like diabetes or hypertension.
The study's findings underscore the need for policymakers and clinicians to acknowledge the significant impact of rare kidney diseases on kidney failure. Contrary to common belief, rare kidney diseases, though constituting a smaller fraction of the chronic kidney disease population, account for a substantial portion of individuals requiring kidney replacement therapy due to their unique disease characteristics. Addressing the unmet need for effective therapies in rare kidney diseases could alleviate the long-term demand for kidney replacement therapy and mitigate the economic burden on healthcare systems.
The study calls for immediate action to overcome obstacles in developing treatments for rare kidney diseases, such as the lack of reliable biomarkers for disease monitoring and the clinical heterogeneity of these conditions. It emphasizes the importance of distinguishing patients with rare kidney diseases from those with more common causes of chronic kidney disease to enable early specialist referral and tailored treatments aimed at delaying disease progression.
Additionally, there is a crucial requirement for accurate tools to predict kidney failure risk in patients with rare kidney diseases, as the current Kidney Failure Risk Equation underestimates this risk for these individuals. The integration of big data analytics and machine learning algorithms from registries like RaDaR could offer valuable insights into rare kidney diseases and pave the way for personalized medicine in the future.
In conclusion, the findings from this study shed light on the substantial impact of rare kidney diseases on kidney failure and mortality rates. They emphasize the urgency of developing targeted therapies, improving diagnostic approaches, and enhancing predictive tools to optimize care for individuals with rare kidney diseases.
Source: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(24)00198-3/fulltext
Comments
Post a Comment