A report has been published by the European ADPKD Forum (EAF), an international, multidisciplinary panel of experts from the fields of nephrology, hepatology and genetics, as well as patient group representatives, dedicated to improving the health and quality of life of people with autosomal dominant polycystic kidney disease (ADPKD) – a chronic, progressive, inherited disease in which fluid-filled cysts grow in the kidneys and liver. (1)
The report, entitled ‘Translating Science into Policy to Improve ADPKD Care in Europe’, details the important role of healthcare professionals to improve the development and delivery of ADPKD care, and provides a comprehensive overview of the current management of ADPKD throughout Europe.
The publication highlights the variation of clinical standards throughout Europe (2) and identifies the need for greater education and support for non-specialist healthcare professionals to ensure the accurate and appropriate diagnosis and treatment of ADPKD and ADPKD-related symptoms. These include pain, infections of cysts and the urinary tract, kidney stones, blood in the urine, and abdominal distension. (3–8) ADPKD patients may also present with cardiovascular symptoms as they are at high risk of developing hypertension and cardiovascular disease. (1) The report also highlights that polycystic kidneys cause kidney failure in the majority of patients, (2,9) and that delaying the progression of the disease would improve patient outcomes and cost efficiencies for health services.
Commenting on the publication of the report, Dr Richard Sandford, Consultant Clinical Geneticist at Addenbrooke’s Hospital, Cambridge, and co-chair of the EAF, said: “The management of ADPKD is currently based on the general management of declining renal function and the symptoms associated with the condition. Disease specific treatments are not currently available. Care pathways are fragmented across the EU and specialist knowledge of the disease itself remains limited. Innovative, long-term solutions should be sought to improve patient outcomes, and meet the financial and societal challenges that ADPKD represents.”
The EAF Report is endorsed by two leading patient organisations PKD International and the Patients Network for Medical Research and Health (EGAN). The following recommendations, captured in the ‘Brussels Declaration on ADPKD’ which is being launched alongside the report as a stand-alone ‘EU policy action plan’, aim to help address the existing unmet needs and to promote access to high quality care for all patients with ADPKD in Europe:
- The implementation of tiered care: Governments should support the development of a nationally coordinated, tiered approach to ADPKD care in collaboration with experts, patient advocacy organisations and other stakeholders, in line with the European Commission (EC) policy priority to address health inequalities.
- The establishment of specialist ADPKD centres: An expanded European Reference Network of specialist ADPKD centres should be established to facilitate further research and harmonised, integrated, patient-centred care pathways, ensuring best practice and consistency of care.
- Supporting therapeutic innovation: The EC and national governments should support research to develop disease-modifying treatments with the potential to maintain quality of life, delay renal decline and improve life expectancy among patients, and to reduce the economic impact on healthcare systems.
- Prioritisation of prognosis: Governments and healthcare providers should support the routine implementation of methods to assess prognosis in ADPKD to inform clinical decision-making, research and innovation.
- Empowering patients: All stakeholders, including the European Commission, national governments and healthcare providers, should support efforts to better inform individual patients and families affected by ADPKD, and to involve patient organisations in policy making regarding healthcare planning and delivery related to ADPKD.
- Engaging patients in health technology assessment: Health Technology Assessment (HTA) organisations should seek to engage ADPKD patient organisations in their assessments in order to consider their unique knowledge about the impact of living with ADPKD, and their aspirations for new treatments, according to the HTA International Quality Standards for Patient Involvement in HTA.
As the most common inherited kidney disease, and one of the most common of all life-threatening inherited conditions, (1) ADPKD represents a significant clinical and economic challenge to healthcare professionals and services throughout Europe. Research highlights that the high cost of renal replacement therapy (RRT) associated with ADPKD – when dialysis treatment or a kidney transplant is required – is due to rise as the number of recipients increases and their life expectancy improves. (10) Data show that RRT costs approximately €1.5 billion per year for people with ADPKD across the EU. (10)
The condition also has a significant impact on the quality of life of people with the disease; people with ADPKD commonly suffer from acute and chronic pain, (3–8) and are more likely to experience anxiety and depression compared to the general population. (11–12) Furthermore, the findings of the largest ever survey of people with ADPKD in Europe detailed in the report, conducted among 730 people with the condition, highlighted the negative impact the disease has on families (77% of respondents), relationships (41%), sexual relationships (42%), social lives (33%) and the decision to have children (35%). (13)
Commenting on the publication of the report, Tess Harris said: “Increased collaboration between patient experts and healthcare professionals can further improve the care and management of the condition throughout Europe.”
ADPKD is an important cause of chronic kidney disease and kidney failure, affecting hundreds of thousands of people in Europe. It is responsible for up to one in ten of all patients needing dialysis or transplantation, corresponding to approximately 50,000 people across Europe. (10) This figure represents only a proportion of the total number of cases as many patients living with ADPKD have yet to develop kidney failure or remain undiagnosed.
The EAF is a multidisciplinary, international faculty of experts dedicated to improving the health and quality of life of people with ADPKD. The EAF was initiated by, and is solely supported financially by Otsuka Pharmaceutical Europe Ltd. Faculty members receive no fees in respect of their roles in the initiative. The contents of the EAF Report and the Brussels Declaration on ADPKD are the opinions of the EAF Faculty and do not necessarily represent those of Otsuka.
- Torres VE, et al. Autosomal dominant polycystic kidney disease. Lancet 2007;369:1287–301.
- Spithoven EM, et al. Analysis of data from the ERA-EDTA Registry indicates that conventional treatments for chronic kidney disease do not reduce the need for renal replacement therapy in autosomal dominant polycystic kidney disease. Kidney Int 2014 Dec;86(6):1244–52.
- Bajwa ZH, et al. Pain patterns in patients with polycystic kidney disease. Kidney Int 2004;66:1561–9.
- Oberdhan D, et al. Patient-reported pain in autosomal dominant polycystic kidney disease: initial concepts based on patient focus group discussions [Abs SA-PO283]. J Am Soc Nephrol 2013;24 Abs Suppl:692A.
- Oberdahn D, et al. Patient experience with pain related to autosomal dominant polycystic kidney disease (ADPKD) [Abs PUB285]. J Am Soc Nephrol 2014;25 Abs Suppl:960A .
- Miskulin DC, et al. Health-related quality of life in patients with autosomal dominant polycystic kidney disease and CKD Stages 1-4: a cross-sectional study. Am J Kidney Dis 2014;63:214–26.
- Heiwe S, et al. An evil heritage: interview study of pain and autosomal dominant polycystic kidney disease. Pain Manag Nurs 2009;10:134–41.
- Casteleijn NF, et al. A stepwise approach for effective management of chronic pain in autosomal-dominant polycystic kidney disease. Nephrol Dial Transplant 2014;29 suppl 4:iv142–53.
- Shaw C, et al. Epidemiology of patients in England and Wales with autosomal dominant polycystic kidney disease and end-stage renal failure. Nephrol Dial Transplant 2014; 29:1910–8.
- Spithoven EM, et al. Renal replacement therapy for autosomal dominant polycystic kidney disease (ADPKD) in Europe: prevalence and survival-an analysis of data from the ERA-EDTA Registry. Nephrol Dial Transplant 2014;29 (Suppl 4):iv15-iv25.
- Wijnands TF, et al. Evaluating health-related quality of life in patients with polycystic liver disease and determining the impact of symptoms and liver volume. Liver Int 2014;34:1578–83.
- de Barros BP, et al. Anxiety, depression, and quality of life in patients with familial glomerulonephritis or autosomal dominant polycystic kidney disease. J Bras Nefrol 2011;33:120–8.
- Otsuka Pharmaceutical Europe Ltd. Market research data, 2014.