Latest news with #aHUS
BBC News
08-08-2025
- Health
- BBC News
Newcastle University kidney drug research 'may be life-changing'
Early withdrawal of a treatment for people with a rare kidney disease is possible without relapse, safer for patients and saves the NHS millions of pounds, researchers Haemolytic Uraemic Syndrome (aHUS) is a life-threatening condition caused by an uncommon defect in the immune system which, in the past, led to kidney failure.A study by Newcastle University has found most patients can stop taking the drug eculizumab after six months and their disease will not return. Prof Neil Sheerin, who led the study, said the findings had "the potential to significantly change the way we manage aHUS and this may be life-changing for some patients". Ending the use of the drug after six months could lead to NHS savings of £4.2m per patient over their lifetime, the university chiefs previously recommended patients stay on eculizumab for the rest of their lives although that leads to an increased risk of meningococcal sepsis. 'I feel like myself again' Twenty-eight people, aged two to 59, from England and Scotland and who had been on eculizumab for at least six months, took part in the patients had their treatment withdrawn. Only four participants' kidney disease returned and the remaining 24 have remained aHus-free to date, the university those taking part was Louise Percival, of York, who was diagnosed with aHus in 35-year-old said she had suffered several side effects, such as migraines, hair loss and breathlessness, before being taken off eculizumab during the described the findings as fantastic."When I was diagnosed with aHUS I felt overwhelmed as it was a huge burden to live with," she said. "It was life-changing as it's something that will never go away."I strongly disliked the eculizumab infusions as they had to be given religiously every two weeks, which impacted my holidays and social events."I'm a very active person, enjoying hiking and the gym, so the side effects and time needed for the treatment were detrimental to my quality of life."I have a fear of needles, so having a cannula put in my arm every fortnight was very distressing."All of the side effects disappeared and I feel like myself again." Prof Sheerin said when a patient initially started treatment for aHUS "they faced a lifetime of eculizumab and the dangers associated with it"."Now we have shown that many people can stop the treatment, freeing them from the burden of regular intravenous injections and removing risk of serious infection associated with the drug," he said."In addition, significant efficiency savings for the NHS of £110.4m over five years could be realised in this patient population through more targeted use of this high-cost treatment."The study was funded by the National Institute for Health and Care Research and published in The Lancet Regional Health Europe. Follow BBC North East on X, Facebook, Nextdoor and Instagram.
Yahoo
12-02-2025
- Health
- Yahoo
New Interactive Storybook Aims to Educate Children with Rare Kidney Disease into a World of Adventure and Understanding
The interactive fantasy e-book "Revealing the Secrets of aHUS" puts young children at the centre of the story as they explore and learn with their families about atypical Haemolytic Uraemic Syndrome (aHUS) LONDON, February 12, 2025--(BUSINESS WIRE)--Alexion, AstraZeneca Rare Disease has launched a new electronic storybook (e-book) to support children living with rare kidney disease, atypical Haemolytic Uraemic Syndrome (aHUS). The interactive book will help children understand their condition by translating complex medical terms into accessible information, as well as building awareness of the practical and emotional support available. Estimated to affect 0.4 - 0.5 people per million,1 aHUS is a genetic, ultra-rare disease, which can progressively damage vital organs, such as the kidneys, and can affect both children and adults.1 The interactive storybook is designed to support children diagnosed with aHUS and their families to better understand the condition. Developed in consultation with Kidney Care UK and the Newcastle National Renal Complement Therapeutics Centre, this immersive book, "Revealing the Secrets of aHUS", transforms the journey of diagnosis, subsequent treatment and care for young people with aHUS, into an accessible, interactive experience. By blending complex healthcare information with imaginative play, the storybook aims to demystify aHUS for children, offering clarity and support in a way that is fun and empowering. "In the UK, 3.5 million people live with a rare disease; it's estimated that 75% of those conditions affect children.2 At Alexion, we work in partnership with healthcare providers to support these young people, helping them better understand a complex disease like aHUS," said Deborah Richards, General Manager, Alexion, AstraZeneca Rare Disease. "Working with medical and patient advocacy partners, we're now able to provide children with a way to learn about their disease and importantly help them feel in control." Aimed at 6–10-year-olds, readers become the heroes of their own story. By personalising the main character – choosing their name, appearance, and outfit – children step into the shoes of the 'Guardian of Healthoria', embarking on a mission to restore a once-thriving land overrun by naughty pixies. Along the way, they battle mythical creatures, solve puzzles, and uncover magical artefacts, all while learning about their kidneys, the immune system, and how to help manage the impact of aHUS on their body and mental health. Readers are offered a mix of quests and challenges – riddles, mazes, and jigsaws – that encourage children to absorb key healthcare information while having fun. At each stage of their journey, young adventurers collect rewards and receive memorable recaps of what they have learned, making complex medical concepts easier to understand. Laurie Cuthbert, Director of Fundraising, Marketing and Communications at Kidney Care UK, said: "The children and families we support at the charity show the most incredible levels of resilience. Having a rare and complex condition like aHUS can be challenging to both live with and to understand which is why we're delighted to be involved with "Revealing the Secrets of aHUS". It gives children the chance to see themselves in a character and story as well as learning more about their condition. It also helps to create a space where parents and carers can learn more about aHUS together and how they can support their child to live with this complex condition." Available on a laptop, tablet, mobile phone and in print, there are four key areas of focus throughout the book as the character navigates their way through. This includes what to expect through the patient journey, how the kidneys are affected, learning about and dealing with their emotions and how to find support, and the impact aHUS can have on children's daily lives. "Navigating through a disease is hard for anyone, at any age, but it can be particularly tough for children to fully understand the implications and support available." Christine Maville, Nurse Consultant (aHUS) for Newcastle Hospitals' National Renal Complement Therapeutics Centre (NRCTC) said. "This book clearly explains the complexities of aHUS and how to cope with the challenges of living with a rare disease. It is a valuable resource, and hopefully one which can brighten and engage the imagination of children diagnosed with aHUS and remind them they are not alone." To learn more about aHUS and how to find support available for patients, visit To access the storybook visit: Alexion Alexion, AstraZeneca Rare Disease is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more people with rare disease around the world. It is headquartered in Boston, US. AstraZeneca AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca's innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit and follow the Company on social media @AstraZeneca. About aHUS Atypical haemolytic uraemic syndrome (aHUS) is an ultra-rare disease that affects both children and adults and can lead to potentially irreversible damage to kidneys and other vital organs, sudden or progressive kidney failure (requiring dialysis or transplant) and premature death.1 aHUS is characterised by inflammation and the formation of blood clots in small blood vessels throughout the body (thrombotic microangiopathy [TMA]) mediated by chronic, uncontrolled activation of the complement system, which is part of the body's immune system. aHUS is characterised by reduced platelet count (thrombocytopenia), microangiopathic haemolytic anaemia (as a result of haemolysis [destruction of red blood cells]) and acute kidney injury (AKI).3 Early and careful diagnosis of aHUS is critical, as many coexisting diseases and events are known or suspected to activate the complement cascade, and as patients may not necessarily present with the classic triad of thrombocytopenia, haemolytic anaemia and renal impairment or may have less severe renal involvement.3 Available tests can help distinguish aHUS from other haemolytic diseases with similar symptoms such as HUS caused by Shiga toxin-producing Escherichia coli (STEC-HUS) and thrombotic thrombocytopenic purpura (TTP).3 About Kidney Care UK Kidney Care UK is the UK's leading kidney patient support charity, providing practical, financial and emotional assistance for kidney patients and their families throughout the UK. We believe that no-one should face kidney disease alone; email support@ or call 0808 801 00 00. References NRCTC Annual Report (2022). Available at: [Last accessed February 2025] GOV UK (2021). UK Rare Diseases Framework. Available at: [Accessed: February 2025] Rare Check (2017) Atypical Hemolytic Uremic Syndrome. Available at: [Accessed: February 2025] View source version on Contacts Media enquiries: Alexion, AstraZeneca Rare Disease UKLucy Pritchard07876 289 Real ChemistryJames Watkins07917 904 171jawatkins@ Sign in to access your portfolio
