Could Mineralocorticoid Receptor Antagonists Be Harmful in Some Patients With Heart Failure?
2026JACC. Heart failure
Testani JM, Cox ZL, Butler J
PubMedSOUDERTON, PA
Dr. Butler studies heart failure, particularly two types: heart failure with preserved ejection fraction (HFpEF) and heart failure with reduced ejection fraction (HFrEF). Her research includes innovative methods like machine learning to predict patient risks, new gene therapies to improve heart function, and the importance of medications like mineralocorticoid receptor antagonists for heart failure treatment. She also explores the relationships between obesity and heart health, examining how weight impacts heart failure outcomes and the effectiveness of obesity medications.
JACC. Heart failure
Testani JM, Cox ZL, Butler J
PubMedKnee surgery, sports traumatology, arthroscopy : official journal of the ESSKA
Balboni JM, Levine AR, Boggiano VJ, Webber KJ, Semelsberger SD +6 more
Plain English
This study looked at the best surgical treatments for Lisfranc injuries, which can be serious for elite athletes. It analyzed the results of 16 studies involving 406 athletes and found that a specific method called suture button fixation (SBF) led to better outcomes compared to open reduction internal fixation (ORIF) for unstable ligament injuries, with 95.5% of athletes showing improvement versus 89.4% for ORIF, and SBF having fewer complications (10.9% vs. 18.2%). The research also emphasized that treatment should be personalized for each athlete based on the type of injury.
Who this helps: This helps elite athletes at risk of Lisfranc injuries and their medical teams.
Journal of the American College of Cardiology
Aimo A, Butler J, De Michieli L, Mendieta G, Zannad F +1 more
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This paper looks at how biomarkers—measures found in blood or urine—are important for developing medications for heart, kidney, and metabolic diseases. The authors found that while many biomarkers exist, only a few are consistently used in clinical trials to decide the effectiveness of drugs. For example, they highlight that markers like albuminuria and natriuretic peptides sometimes do not predict the actual benefits of a treatment accurately.
Who this helps: This helps patients by ensuring that drug development is based on reliable measures of health improvement.
Journal of the American College of Cardiology
Butler J, Khan MS, Ferreira JP, Girerd N, Gulati M +5 more
Plain English
This research paper explores the prevention of heart failure, a serious condition affecting many people, with a lifetime risk of 1 in 4 for developing it. Although there are effective treatments for heart failure, once it occurs, patients face a high risk of death and complications. The study stresses the need for better strategies to prevent heart failure, particularly since there are currently no specific medications approved for this purpose and many cases arise from causes other than heart disease.
Who this helps: This helps patients at risk of heart failure and healthcare providers working on prevention strategies.
Journal of cachexia, sarcopenia and muscle
Anker SD, Ji L, Kindel T, Coats AJS, Ojji D +56 more
Plain English
This study created updated global guidelines for managing obesity, addressing the varied challenges that patients and healthcare providers face worldwide. It emphasizes using new therapies along with established care practices, recommending specific approaches based on individual patient needs and available resources. The goal is to improve patient outcomes while reducing hospital visits, especially in areas with limited healthcare resources.
Who this helps: This supports patients with obesity and healthcare providers around the world.
JAMA network open
Peltan ID, Groat D, Butler J, Bledsoe JR, Ofori-Atta BS +9 more
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This study looked at how quickly emergency department (ED) doctors give antibiotics to patients with sepsis, a serious infection, and whether faster treatment leads to giving unnecessary medications. Researchers analyzed data from nearly 10,000 patients and found that doctors' administration times varied significantly—averaging about 184 minutes—but this speed did not increase the rate of overtreatment. Understanding these patterns helps improve sepsis care without risking patients receiving unneeded antibiotics.
Who this helps: This benefits patients with sepsis and their doctors by refining treatment practices.
ESC heart failure
Biegus J, Tymków R, Butler J, Metra M, Chioncel O +8 more
Plain English
This study looked at the opinions of patients and doctors about a potential single-pill treatment for heart failure, which could combine multiple necessary medications into one pill. The results showed that 95% of doctors believe there is a real need for this type of treatment, and 88% feel it would be clinically useful. Among patients, 70% take six or more medications, and 83% would be willing to try this new single-pill solution if it didn’t cost extra, highlighting both groups' desire for simpler heart failure treatments.
Who this helps: This helps heart failure patients who struggle with managing multiple medications.
Urology case reports
Klein Nulend R, Lockhart K, Tree K, Tran LN, Matti B +7 more
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This study reports on a rare case of an aggressive prostate cancer in a 24-year-old man who initially experienced blood in his urine and difficulty urinating after an injury. Doctors found a large mass in his prostate, and while they couldn't reach a definite diagnosis, they think it resembles a very uncommon type of tumor. After receiving chemotherapy, surgery, and radiation, the patient has been cancer-free for 18 months, highlighting the need for careful evaluation and treatment of unusual prostate cancers in younger patients.
Who this helps: This benefits young patients with rare prostate tumors and their healthcare providers.
BMC cancer
Essa MEA, Noori H, Butler J, Ahmed AA
Plain English
This study looked at how the bacteria in our gut, called the gut microbiome, affects the success of immunotherapy treatments for patients with non-small cell lung cancer (NSCLC) and melanoma. Researchers found that people with a more diverse gut microbiome generally had better survival rates and treatment responses; for example, those with high diversity had a 48% lower risk of dying (overall survival) and a 42% lower risk of cancer progression (progression-free survival). In contrast, recent antibiotic use negatively impacted survival, making patients 72% more likely to die.
Who this helps: This research benefits patients with NSCLC and melanoma by highlighting how gut health could influence treatment outcomes.
European journal of heart failure
Greene SJ, Butler J, Fonarow GC
PubMedEuropean journal of heart failure
Ferreira JP, Zannad F, Butler J, Filippatos G, Vasques-Nóvoa F +4 more
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This study looked at how genitourinary infections, like urinary tract infections (UTIs) and mycotic genital infections (MGIs), affect patients with heart failure who are taking a medication called empagliflozin. Researchers followed nearly 10,000 patients over about 21 months and found that 7.8% had lower UTIs, while MGIs and severe infections like pyelonephritis were less common. Although empagliflozin raised the risk of UTIs and MGIs, having a UTI was linked to a higher risk of death, but the overall benefits of the medication remained strong, even for those who experienced infections.
Who this helps: This information benefits patients with heart failure and their doctors by informing them about potential risks and outcomes related to SGLT2 inhibitors.
The bone & joint journal
McCartney C, Moore D, McDonnell J, Storey R, Kepler C +2 more
Plain English
This study looked at the lack of clear guidelines for when professional rugby players can safely return to the game after having surgery for neck injuries. Researchers found that while some procedures, like spine fusion, have known risks, there isn't an agreed-upon timeline for when players can go back to playing. Establishing specific, comprehensive guidelines can help ensure player safety and prevent future injuries, which is crucial given the serious nature of these injuries.
Who this helps: This helps players, coaches, and medical staff involved in rugby.
European journal of heart failure
Ferreira JP, Fioretti F, Sumin M, Anker SD, Filippatos G +8 more
Plain English
This study looked at how levels of certain proteins in the blood relate to the heart's ability to pump blood, measured by a number called left ventricular ejection fraction (LVEF), in over 2,250 patients. The researchers found 297 proteins that showed some variation across different LVEF groups, but none of these differences were strong enough to be considered significant, suggesting that these proteins might not effectively indicate heart function. Understanding these findings is important as it helps clarify the role of blood proteins in heart health and may guide future research on better indicators for heart function.
Who this helps: This helps doctors and researchers better understand heart health indicators for patients with heart failure.
European journal of heart failure
Kereiakes DJ, Tedford RJ, Lindenfeld J, Gray W, Zahr F +12 more
Plain English
This study investigates a new medical device called the Edwards APTURE shunt, which is designed to help patients with certain types of heart failure, specifically those with preserved or mildly reduced heart function. The trial will involve about 100 patients who experience symptoms of heart failure and measures how safe the device is in 30 days and how it affects heart pressure after six months. This research matters because it could provide a new treatment option for heart failure patients who currently have limited options and high risks of hospitalization.
Who this helps: Patients suffering from heart failure with preserved or mildly reduced heart function.
European journal of heart failure
Díez J, González A, Kirabo A, Verdecchia P, Jafar TH +21 more
Plain English
This study focuses on how high blood pressure (hypertension) can lead to heart failure in people with hypertensive heart disease. The research shows that managing and treating high blood pressure effectively can prevent or delay heart failure, highlighting that timely intervention is crucial. It emphasizes the importance of recognizing and addressing hypertensive heart disease as a significant risk factor that is often overlooked in healthcare settings.
Who this helps: This helps patients with high blood pressure and hypertensive heart disease.
European journal of heart failure
Sharp A, Aw TC, Le TT, Phua SK, Richards AM +4 more
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This study looked at two blood markers, NT-proBNP and hsTnT, to see how well they can help categorize the severity of hypertensive heart disease (HHD) in people with high blood pressure who don’t yet show symptoms. Researchers followed 1,054 participants for about four and a half years, finding that those with higher levels of both markers had nearly 17 times the risk of serious heart problems compared to those with normal levels. This is important because it means doctors can use these markers to better stage heart disease severity and tailor treatments to reduce the risk of severe outcomes for patients.
Who this helps: This helps patients with high blood pressure and their doctors in managing heart disease risks.
European journal of heart failure
Siddiqi TJ, Khan MS, Fonarow GC, Butler J, Greene SJ
PubMedJACC. Asia
Khanna S, Gan GCH, Sindone AP, Tromp J, Butler J +3 more
Plain English
This study focuses on the growing problem of cardiometabolic diseases, like obesity and diabetes, especially in Asia. It found that more than one-third of deaths from heart-related issues are now due to these diseases, driven by unhealthy lifestyles and environmental factors like air pollution. Addressing these challenges is crucial, as it highlights the need for better healthcare access and preventive measures to keep people healthy.
Who this helps: Patients in Asia dealing with cardiometabolic conditions and healthcare providers looking to improve patient outcomes.
BJPsych bulletin
Deng Y, Butler J, Oram S
Plain English
This study looked at patients’ personal experiences of sexual violence while being treated in psychiatric hospitals. Researchers found that in six studies, patients often felt their concerns about harassment were dismissed by staff, leading to chronic fear and distress. The issues were worse in mixed-gender wards, where patients felt especially vulnerable due to inadequate safety measures and power imbalances. This matters because it highlights a critical need for improved safety practices and support for patients in these settings.
Who this helps: This helps patients in psychiatric care, particularly those in mixed-gender environments.
Journal of cardiac failure
Martyn T, Chedid El Helou M, Zalaquett Z, Rajendran J, Patolia H +9 more
Plain English
This study looked at how well certain heart failure treatments work in different types of heart failure patients. Researchers examined data from over 25,000 people and found that those who received more guideline-directed medical therapy (GDMT) had better outcomes, like fewer hospital visits and lower death rates. Specifically, the greatest improvements were seen in patients with a GDMT score of 6 or higher, especially for those with heart failure with preserved ejection fraction (HFpEF) and heart failure with mildly reduced ejection fraction (HFmrEF).
Who this helps: This research benefits patients with all types of heart failure by highlighting the importance of comprehensive treatment.
Nature cardiovascular research
Baudry G, Monzo L, Newton RAV, Li G, Petrie MC +9 more
Plain English
This study looked at how heart failure clinical trials often fail to include a diverse range of participants from different regions, particularly outside of North America and Europe. It found that most trials still recruit mainly from these areas, which limits the relevance of their findings for people in other regions. To address this, the researchers proposed better strategies for including participants from underrepresented areas, such as creating a standardized system for classifying regions and using tools to assess how well different regions are included in trials.
Who this helps: This benefits patients from diverse backgrounds who need treatments that are effective for their specific conditions.
European journal of heart failure
Ahmed AA, Lu F, Zhang S, Raman VK, Patel SS +20 more
Plain English
This study looked at how giving high doses of certain heart medications (ACE inhibitors) affects kidney failure risk in U.S. veterans with heart failure and reduced heart function. The results showed that patients on high doses of ACE inhibitors had an 18% lower risk of developing kidney failure and a 6% lower risk of dying after five years compared to those on lower doses. This is important because it suggests that higher doses of these medications can help protect the kidneys in patients with serious heart conditions, especially those with more advanced kidney disease.
Who this helps: Patients with heart failure and chronic kidney disease.
JACC. Heart failure
Ferrero C, Codina P, Lupón J, Ruiz-Cueto M, Zamora E +16 more
Plain English
This study looked at how heart failure patients with secondary mitral regurgitation (SMR) responded to optimized medical treatment over a year. The researchers found that after 12 months, 57.5% of patients with moderate or severe SMR improved to a mild or no SMR condition, significantly reducing their risk of death or hospitalization for heart failure. Those who continued to have moderate or severe SMR faced a much higher risk of dying or being hospitalized.
Who this helps: Heart failure patients and their doctors will benefit from these findings as they highlight the importance of effective medical therapy.
Journal of cardiac failure
Butler J, Rao I, Sauer AJ, Anker SD, Fioretti F +5 more
PubMedJournal of the American College of Cardiology
Platz E, Peikert A, Bozkurt B, Van der Schueren B, Butler J +4 more
Plain English
This paper looks at how new treatments for obesity can impact heart health and other serious conditions. Researchers found that recent treatments, called GLP-1 receptor agonists, have shown promise in reducing cardiovascular risks, which is important since obesity can lead to diseases like heart failure and diabetes. They argue that the way these new studies are designed needs to change to better assess how well these treatments work and suggest five key principles for future studies.
Who this helps: This helps patients struggling with obesity and related health issues, as well as the doctors treating them.
Journal of the American College of Cardiology
Khan MS, Borlaug BA, Butler J, Gomberg-Maitland M, Humbert M +5 more
Plain English
This study looks at pulmonary hypertension (high blood pressure in the lungs) that occurs with left heart disease, which is very common and can lead to serious health problems. Researchers found that traditional treatments for a different type of pulmonary hypertension don't help and could cause harm. New drugs and techniques are being tested, but progress has been slow due to challenges in finding suitable patients and measuring outcomes effectively.
Who this helps: This research benefits patients with left heart disease and their healthcare providers.
ESC heart failure
Fioretti F, Hibbert B, Eckman PM, Simard T, Labinaz M +14 more
Plain English
The study evaluated a new device that assists blood flow from the heart's left atrium to another vein, aiming to help patients with heart failure but preserved ejection fraction (HFpEF). Over three years, 76.3% of the 95 patients showed improvement in heart function symptoms and walking distance increased by an average of 39 meters. Additionally, the actual survival rate after three years was significantly better than expected, with 84.4% still alive compared to an anticipated 70.2%.
Who this helps: This benefits patients with heart failure, particularly those with preserved ejection fraction.
The Journal of the Association of Physicians of India
Anker SD, Ji L, Kindel T, Coats AJ, Ojji D +57 more
Plain English
This research paper outlines new guidelines for managing obesity with a focus on preventing and treating heart-related health issues. It highlights the importance of adapting treatment recommendations to fit different healthcare systems and patient needs, considering limitations like resources and access to care. The study emphasizes that these updated guidelines aim to improve patient outcomes and reduce hospital visits for people affected by obesity by offering tailored strategies based on the latest evidence.
Who this helps: These guidelines benefit patients struggling with obesity and healthcare providers around the world.
Journal of surgical case reports
Hines EM, Butler J, Aseervatham L
Plain English
This study looked at a rare type of liver tumor called mucinous cystic neoplasm (MCN-L) and a specific case that was initially misdiagnosed as a different condition. The researchers found that the tumor had burst into the bile ducts, causing complications like inflammation and stone formation. They emphasized that MCN-L can easily mimic other liver cysts, which makes a proper diagnosis challenging and important for effective treatment.
Who this helps: This helps doctors and patients by improving diagnosis and treatment approaches for liver tumors.
Journal of cardiac failure
Greene SJ, Butler J
PubMedEuropean journal of heart failure
Fioretti F, Zannad F, Barash I, Corda S, Ezekowitz JA +7 more
PubMedJACC. Heart failure
Chatur S, Meng T, Bobba G, John A, Pop-Busui R +4 more
PubMedJournal of clinical lipidology
Yadalam AK, Gold ME, Patel KJ, Razavi AC, Flowers A +11 more
Plain English
This research paper focuses on the problem of dyslipidemia, or abnormal lipid levels, in patients who have received a heart transplant, which affects up to 80% of them. It highlights that this condition can lead to severe heart complications, but treatments like statins can lower risks of heart disease, rejection of the transplant, and even death. Understanding how to manage lipid levels is crucial, especially as more patients survive after their transplants.
Who this helps: This study benefits heart transplant patients and their healthcare providers.
Health affairs scholar
Sharareh N, Castaneda G, Dalrymple R, Wambach C, Brintz B +7 more
Plain English
The study looked at why people who are struggling to afford food contact the 2-1-1 service for help. Researchers spoke with 30 callers in Utah and found that many sought assistance because they couldn’t access federal food programs, faced other challenges getting food, or had unexpected life changes. Callers recommended improving food assistance programs and better supporting emergency food services.
Who this helps: This information benefits people facing food insecurity and the organizations that support them.
JACC. Heart failure
Packer M, Ho J, Petrie MC, Schiattarella GG, Butler J +4 more
Plain English
Researchers studied the connection between excess fat, particularly in the abdominal area, and a specific type of heart failure known as heart failure with preserved ejection fraction (HFpEF). They found that this unhealthy fat not only contributes to HFpEF but also increases the risk of related conditions like high blood pressure and diabetes. Addressing excess fat is crucial because it plays a significant role in worsening heart and kidney health in many patients.
Who this helps: This helps patients struggling with heart failure and related health issues.
Journal of cardiac failure
Uchehara B, Felker GM, McVeigh T, Bowers MT, Gupta M +5 more
PubMedEuropean heart journal
Khan MS, Dawood MH, Handelsman Y, Anker SD, Stewart Coats AJ +2 more
Plain English
This study looked at how anti-obesity medications, particularly those targeting the glucagon-like peptide-1 receptor, affect both fat and muscle loss in patients. While these medications help people lose weight, they can also lead to muscle loss, which may increase the risk of frailty and heart disease. To combat this, researchers suggest combining these medications with resistance training and a higher protein diet to maintain muscle mass and improve overall health.
Who this helps: This benefits patients struggling with obesity and healthcare providers focused on cardiovascular health.
ESC heart failure
Savarese G, Basile C, Mebazaa A, Bayes-Genis A, Kang SM +4 more
Plain English
The study looked at real-world data on the medication Sacubitril/Valsartan (Sac/Val) for patients with heart failure and found that it significantly lowers the risk of dying from heart-related issues (10%-16% reduction) and hospitalizations due to heart failure (10%-38% reduction). Despite its benefits, only 15%-25% of patients are receiving the recommended doses of the drug, and the most common side effect is a drop in blood pressure. This matters because improving how often and effectively this medication is used could greatly reduce heart failure's impact on patients' lives and the healthcare system.
Who this helps: This helps patients with heart failure, doctors, and healthcare providers.
ESC heart failure
Fudim M, Kovacevic-Preradovic T, Zdravkovic M, Jovev S, Granov N +12 more
Plain English
This study looked at how improvements in heart function relate to patients’ overall health and ability to be active in people with advanced chronic heart failure. The researchers followed 65 patients over six months and found that their reported health scores and walking distance improvements moderately linked to heart function measurements, with correlations of 0.39. This matters because understanding these relationships can help doctors better assess treatment responses and tailor therapies for their patients.
Who this helps: This helps patients with chronic heart failure by providing insights for doctors on how to measure treatment effectiveness.
Journal of the American College of Cardiology
Khan MS, Javaid SS, Petrie MC, Zieroth S, Anker SD +1 more
Plain English
This study examined the connection between obesity and a specific type of heart failure known as heart failure with reduced ejection fraction (HFrEF). It found that many patients with HFrEF are also overweight or obese, but there hasn't been much research focused on this group, which makes understanding their treatment needs challenging. The research highlights that while being overweight is often linked with better outcomes in HFrEF patients, losing weight could still help manage the disease more effectively by influencing how it progresses.
Who this helps: This helps patients with heart failure, particularly those who are also struggling with obesity.
European journal of heart failure
Böhm M, Abdullakutty J, Bauersachs J, Bavendiek U, Bayes-Genis A +34 more
Plain English
This research paper focuses on the use of specific heart failure medications called mineralocorticoid receptor antagonists (MRAs), like spironolactone and eplerenone, which are essential for treating heart failure with reduced ejection fraction (a condition where the heart doesn't pump blood effectively). The findings highlight that despite these drugs significantly reducing mortality and hospital visits, many eligible patients are not receiving them—often due to doctors' fears of side effects like high potassium levels. The paper provides clear guidelines on how to use these medications more effectively, aiming to enhance patient care and reduce the burden of heart failure.
Who this helps: This benefits patients with heart failure and their healthcare providers.
European journal of heart failure
Henry TD, Chung ES, Alvisi M, Murray DR, Traverse JH +15 more
Plain English
This study looked at a new gene therapy called AB-1002, designed to help people with heart failure by improving heart muscle function. In a trial involving 30 to 50 participants across different groups, the therapy was administered to adults who have severe heart failure symptoms despite other treatments. Researchers will measure success based on heart function improvements, walking ability, and overall health after one year.
Who this helps: This research benefits patients with severe heart failure who have not responded well to standard treatments.
ESC heart failure
Fudim M, Van Empel V, Zehnder T, Sauty B, Esposito C +16 more
Plain English
This study focused on heart failure with preserved ejection fraction (HFpEF), a serious condition that affects many older adults. Researchers analyzed data from 1,208 patients to create machine learning models that predict the risk of death and hospitalization due to heart failure. They found that over two years, about 13% to 44% of patients were likely to be hospitalized, and 9% to 19% were at risk of death. The new models showed better accuracy in predicting these risks compared to existing methods.
Who this helps: This benefits patients with HFpEF, doctors treating them, and researchers looking for better treatment strategies.
BMC health services research
Bayes-Genis A, Butler J, Stawowczyk E, Turner M, Lewis RD +2 more
PubMedJournal of bone and joint infection
Egan KG, Duffy L, Fleming C, McGettrick P, Muldoon EG +9 more
Plain English
This study follows a 57-year-old woman who developed a serious infection caused by a type of bacteria after having weight loss surgery. Despite being on medication that weakens her immune system for a condition called polymyalgia rheumatica, she faced significant health challenges due to this infection, which spread to her spine. Understanding how to treat such infections is crucial since they can be complex and difficult to manage, especially in patients with weakened immune systems.
Who this helps: This information benefits doctors treating patients with similar infections, especially those on immunosuppressive therapy.
Journal of cardiac failure
Martinez OC, Greene SJ, Chinnakondepalli KM, Anker SD, Bozkurt B +11 more
Plain English
This study looked at how well patiromer, a medication that helps lower potassium levels in patients with heart failure, works in the real world. Among 2,558 patients, those taking patiromer saw their average potassium levels drop by 0.21 mEq/L, but only about 40% managed to increase their heart medications to optimal levels within two years. This matters because while patiromer can lower potassium, many patients still miss out on better heart medication, so more action is needed to improve their treatment.
Who this helps: This helps patients with heart failure who are at risk of high potassium levels.
JACC. Advances
Gouda P, Allyn N, Belin R, Borentain M, Butler J +14 more
Plain English
Researchers came together to discuss the urgent need for improvements in obesity medications, focusing on how to better define obesity, improve access to treatments, and ensure fair care for all patients. They highlighted issues like high costs and limited availability of these medications, which lead to inequalities in healthcare. A clear plan was proposed to tackle these challenges, including coming up with standardized measures of obesity beyond just weight and creating a roadmap for collaboration in obesity research.
Who this helps: This helps patients struggling with obesity and healthcare providers seeking better treatment options.
Journal of cardiac failure
Greene SJ, Patel S, Chinnakondepalli K, Anker SD, Böhm M +10 more
PubMedAnnals of clinical biochemistry
Rodney M, Rankin K, Butler J, Thompson S, Florkowski CM +1 more
Plain English
The study looked at a case of a 47-year-old woman who experienced high potassium levels (up to 8.9 mmol/L, while normal is 3.5-5.2 mmol/L) for two years without any clear explanation. Researchers found that her high potassium readings were not real but were due to a genetic condition called familial pseudohyperkalaemia, which causes potassium to leak out of red blood cells under certain conditions. Recognizing this condition is crucial because mistaking it for a real health issue could lead to dangerous treatments that lower potassium levels unnecessarily.
Who this helps: This research benefits patients with familial pseudohyperkalaemia and healthcare providers interpreting their test results.
Journal of health economics and outcomes research
Turner-Bowker DM, Butler J, Egan S, Weinstein DA, Rodriguez-Buritica DF +5 more
Plain English
Adults with glycogen storage disease type Ia — a condition requiring strict dietary management to keep blood sugar stable — were interviewed at multiple time points during a gene therapy trial to capture their lived experience of treatment. Most reported reduced reliance on cornstarch, improved physical and emotional functioning, and overall satisfaction with the therapy, though some experienced blood sugar instability during dietary transitions. The results highlight both the promise of gene therapy for this condition and the need for close dietary monitoring during treatment.
Physician data sourced from the NPPES NPI Registry . Publication data from PubMed . Plain-English summaries generated by AI. Not medical advice.