Lifelong Monitoring Research Articles

Long-term outcomes of active surveillance for low-risk papillary thyroid carcinoma: Progression patterns and tumor calcification.

Active surveillance (AS) for low-risk papillary thyroid carcinoma (PTC) is acknowledged as a valid management strategy. While older age is identified as a favorable factor for progression, long-term evidence is scarce and lifelong monitoring has been deemed essential. This study investigated progression patterns and tumor calcification under long-term AS and explored the possibility of ending follow-up. A total of 650 patients with low-risk PTC who chose AS were enrolled. Progression was defined as either tumor enlargement (≥3mm from initiation) or development of clinically apparent lymph node metastasis. The median observation period was 8years; 45.2% were under surveillance for ≥10years. Overall, 80 patients (12.3%) exhibited progression. Median age and observation period at the time of progression were 55 and 4years, respectively. Only 2 patients showed progression after 15years of follow-up and 5 patients showed progression after reaching 80years old. Among 71 patients experiencing tumor enlargement, surgery was performed immediately in 32 patients. The remaining 39 patients continued surveillance, but only 5 demonstrated ongoing enlargement thereafter. Of 40 surgeries due to progression, 36 were conducted within the first 10years. The degree of calcification correlated with age and observation periods. No progression occurred after the development of rim calcification. Progression during AS was extremely rare in older patients with long-term surveillance and in tumors with rim calcification. It may be feasible to consider ending scheduled surveillance visits for these patients. Instances of progression halting after enlargement are not uncommon.

Branching and nonbranching intracranial aneurysms in the presence of a persistent stapedial artery and an aberrant internal carotid artery assessed with computational hemodynamics: illustrative case.

The mechanisms underlying the initiation and progression of bifurcation versus lateral wall aneurysms are not well understood. Computational fluid dynamics (CFD) can improve the understanding of these mechanisms and can consequently help identify patients at higher risk for developing aneurysms and monitor them more closely. A 36-year-old man presented with a ruptured anterior communicating artery aneurysm, which was successfully treated with microsurgical clipping. Imaging also revealed a persistent stapedial artery with an elongated and tortuous posterior communicating artery (PComA). Fourteen years later, he was readmitted for a ruptured aneurysm on a PComA loop. CFD helped identify considerable collateral circulation due to the aberrant internal carotid artery (ICA). High flow rates trigger both types of aneurysms, but nuances exist in the hemodynamic mechanisms that drive their growth. Berry aneurysms and lateral wall aneurysms initially start due to a high flow rate, a common underlying cause. However, the formation of true sidewall aneurysms is primarily characterized by locally increased wall shear stress, while the development of berry aneurysms is mainly linked to high local blood pressure at arterial bifurcations. An aberrant ICA can lead to supraphysiological compensatory flow in the anterior and posterior circulation, increasing the risk of intracranial aneurysm formation at both branching and nonbranching sites, underscoring the need for lifelong monitoring. https://thejns.org/doi/10.3171/CASE24421.

Malignant arterial hypertension in a child with nonspecific aortoarteritis

The article presents a clinical case of malignant renovascular hypertension in a child, resulting from non-specific aortoarteritis. The disease initially manifested with abdominal pain and convulsions, leading to hospitalization. During the hospital stay, blood pressure readings were elevated to 180/100 mmHg and higher. Over the course of a year, the patient underwent pathogenetic (immunosuppressive), combined (triple) antihypertensive treatment, and symptomatic therapy. Due to the malignant nature of the hypertension, progression of organ damage, a high risk of complications and mortality, and the ineffectiveness of conservative treatment, surgical interventions were performed. The first stage involved polyposition abdominal aortography and stent-graft implantation in the abdominal aorta. Despite restored blood flow in the abdominal aorta, blood pressure remained elevated over the following three months, even with ongoing antihypertensive therapy. As a result, a nephrectomy was performed at the second stage. Postoperatively, the patient’s blood pressure significantly decreased to 130/90–120/80 mmHg. Antihypertensive therapy was continued to reach lower target blood pressure values. Non-specific aortoarteritis remains a prognostically unfavorable condition, even after reconstructive surgery, owning to its autoimmune nature, which suggests ongoing arterial inflammation even during clinical remission. This necessitates lifelong monitoring by a rheumatologist.

A Platform of Serious Video Games Co-built for the General Interest

In a context where training takes place both in initial courses and in lifelong continuing education, the use of games offers a way of promoting effective and sustainable learning: by its very nature, this medium is an open work in which players can become involved and give meaning to their learning. Ikigai - Games for Citizens offers a unique ecosystem for creating serious, expert video games and offering them free of charge to people who want to learn while having fun, as well as producing a set of learning-related data that can be used for research and customizing training courses. By bringing together universities, Grandes Ecoles, research laboratories, associations, and institutions, Ikigai is a collaborative consortium of general interest, implementing the co-construction of videogame services between scientific experts, video game professionals and educational engineers. Together, they are producing a centralized platform of serious games and associated services that can be used by teachers at a moderate cost by pooling resources: the building blocks of each game can be reused in other games developed. The consortium is committed to general interest and popular education by making the games available free of charge to members and the general public. The decentralized infrastructure set up by Ikigai, which respects privacy by design, means that game usage data can now be collected on a national scale, but conceptually it could extend much further. The data collected in compliance with the RGPD will eventually be used to draw up enriched user profiles to offer personalized courses, teaching recommendations and progress monitoring as close as possible to the learner, using AI in particular. Ikigai is also heavily involved in the construction of new digital standards in education, which are needed to ensure the interoperability of data for lifelong monitoring of learners. But also, for the digital education ecosystem, to guarantee a sovereign, efficient, coherent, and sustainable cloud on a European scale.

An unknown situs viscerum inversus totalis, accidentally discovered after computed tomography

Benign situs inversus totalis of the viscerum is often diagnosed accidentally, rarely in adults, and more frequently in children and neonates, affecting both sexes. In this report, a young female patient accidentally discovered a situs inversus totalis after computed tomography for acute abdominal pain. In this uncommon anatomical abnormality, the major visceral organs are reversed in the opposite direction. This report highlights the importance of being aware of and considering situs inversus in clinical practice, particularly when interpreting imaging findings and planning medical procedures. This is critical for differential diagnosis and comorbidities that may affect those patients. The cause of situs inversus totalis is still unknown; however, this condition is frequently asymptomatic, particularly in infants, and is sometimes associated with other syndromes. The patient arrived at the emergency department with left flank pain, nausea, and fever. In the first ultrasonography, a strange anatomy was suspected; thus, a contrasted computed tomography was performed. The patient had never had a computed tomography scan before. The identification of situs inversus totalis was unexpected and coincidental; the computed tomography images were carefully examined. In patients with chest or abdominal pain, clinicians may consider situs inversus totalis based on computed tomography, particularly if without clinical and imaging history. This knowledge can help in the differential diagnosis, avoiding unneeded interventions. Moreover, comorbidities that affect several systems, particularly cardiovascular and pulmonary systems, affect quite a few patients with situs inversus totalis, who require careful examination and lifelong monitoring.

Single Anastomosis Duodeno-Ileostomy with Sleeve Gastrectomy/Single Anastomosis Duodenal Switch (SADI-S/SADS) IFSO Position Statement-Update 2023.

The single anastomosis duodeno-ileostomy with sleeve gastrectomy/single anastomosis duodenal switch (SADI-S/SADS) has gained attention as an alternative to the traditional biliopancreatic diversion with duodenal switch (BPD-DS). In 2021, IFSO endorsed SADI-S/SADS as a safe and effective procedure, underscoring the necessity for long-term multidisciplinary care and randomized controlled trials (RCTs). A task force was established to conduct a systematic review of current evidence on SADI-S/SADS to guide clinical practice. A systematic review was conducted across three databases, focusing on studies examining SADI-S/SADS and its outcomes. A total of 93 studies were analyzed. SADI-S/SADS demonstrated efficacy in weight loss and medium-to-long-term control of type 2 diabetes mellitus (T2DM), along with positive outcomes regarding hypertension and hyperlipidemia. However, its impact on other comorbidities remains inconclusive. Frequent nutritional deficiencies were identified, particularly in fat-soluble vitamins, anemia, and hypoalbuminemia. Despite significant efforts, high-quality evidence on SADI-S/SADS remains scarce, prompting IFSO to advocate for increased registry participation, publication of long-term studies, and more RCTs. Lifelong supplementation and monitoring for nutritional deficiencies are recommended. The current position statement will be reviewed in 2 years.

Hematopoietic stem cell transplantation for primary immunodeficiency.

Primary immunodeficiencies, also commonly called inborn errors of immunity (IEI), are commonly due to developmental or functional defects in peripheral blood cells derived from hematopoietic stem cells. In light of this, for the past 50 years, hematopoietic stem cell transplantation (HSCT) has been used as a definitive therapy for IEI. The fields of both clinical immunology and transplantation medicine have had significant advances. This, in turn, has allowed for both an increasing ability to determine a monogenic etiology for many IEIs and an increasing ability to successfully treat these patients with HSCT. Therefore, it has become more common for the practicing allergist/immunologist to diagnose and manage a broad range of patients with IEI before and after HSCT. This review aims to provide practical guidance for the clinical allergist/immunologist on the basics of HSCT and known outcomes in selected forms of IEI, the importance of pre-HSCT supportive care, and the critical importance of and guidance for life-long immunologic and medical monitoring of these patients.

The use of artificial neural networks in studying the progression of glaucoma

In ophthalmology, artificial intelligence methods show great promise due to their potential to enhance clinical observations with predictive capabilities and support physicians in diagnosing and treating patients. This paper focuses on modelling glaucoma evolution because it requires early diagnosis, individualized treatment, and lifelong monitoring. Glaucoma is a chronic, progressive, irreversible, multifactorial optic neuropathy that primarily affects elderly individuals. It is important to emphasize that the processed data are taken from medical records, unlike other studies in the literature that rely on image acquisition and processing. Although more challenging to handle, this approach has the advantage of including a wide range of parameters in large numbers, which can highlight their potential influence. Artificial neural networks are used to study glaucoma progression, designed through successive trials for near-optimal configurations using the NeuroSolutions and PyTorch frameworks. Furthermore, different problems are formulated to demonstrate the influence of various structural and functional parameters on the study of glaucoma progression. Optimal neural networks were obtained using a program written in Python using the PyTorch deep learning framework. For various tasks, very small errors in training and validation, under 5%, were obtained. It has been demonstrated that very good results can be achieved, making them credible and useful for medical practice.

Long-Term Follow-up Results of Children with Urolithiasis Followed in Our Clinic

Title and Objective: Urolithiasis is a prevalent condition frequently observed in childhood within the Turkish population. The aim of this study was to evaluate the metabolic, radiological, and clinical features of pediatric patients with urolithiasis. Materials and Methods: Records of 158 children referred to the Pediatric Nephrology Department of Celal Bayar University between 2010 and 2020 with suspected urolithiasis and microlithiasis were retrospectively reviewed. The complaints and ages of the cases during hospital admission, their medical histories, and the location of the stones were determined. All patients underwent complete urine analysis, spot urine electrolytes, urine culture, serum electrolytes, kidney function tests, uric acid, albumin measurements, and urinary ultrasonography. 24-hour urine electrolytes were studied in patients capable of urine collection. Stone analysis using X-ray diffraction was performed on patients from whom stones were obtained. Results: Out of the individuals, 88 (55.7%) were male, and 70 (44.3%) were female, resulting in a male-to-female ratio of 1.25:1. The average age at the time of diagnosis was determined to be 89.82 ± 57.35 months. A family history of urolithiasis was reported in 108 (68.3%) patients, and 46 individuals (29%) were born from consanguineous marriages. At the time of diagnosis, 32 patients (20%) had a urinary tract infection. Stones were predominantly situated in the upper urinary system in 129 patients (81.6%), with 123 (77.8%) having unilateral stones and 35 (22.2%) having bilateral stones. Calcium oxalate stones were the most commonly observed (80%) in patients who underwent stone analysis. Hypercalciuria emerged as the most frequently identified urinary metabolic risk factor. At the end of the follow-up period, 14 patients experienced a recurrence, while 67 patients remained free of stones. Conclusion: Urolithiasis continues to be a significant concern among children in our nation. Due to the higher recurrence rate and more frequent underlying metabolic disorders in children with stone diseases compared to adults, metabolic assessment and stone analysis are recommended procedures, emphasizing the need for lifelong monitoring in these cases.

External validation of models to predict hepatocellular carcinoma in Hepatitis C Virus cured F3-F4 patients.

Several hepatocellular carcinoma (HCC) risk-models have been developed to individualise patient surveillance following sustained viral response (SVR) in Hepatitis C Virus patients. Validation of these models in different cohorts is an important step to incorporate a more personalised risk assessment in clinical practice. We aimed at applying these models to stratify the risk in our patients and potentially determine cost-saving associated with individualised HCC risk-stratification screening strategy. Patients with baseline F3-4 fibrosis treated with new oral direct-acting antivirals who had reached a SVR were regularly followed as part of the HCC surveillance strategy. Six models were applied: Pons, aMAP, Ioannou, HCC risk, Alonso and Semmler. Validation of the models was performed based on sensitivity and the proportion of patients labelled as "high risk". After excluding 557 with less than 3 fibrosis, 12 without SVR, 18 with a follow up (FU)<1year, 17 transplant recipients, 16 lost to FU and 31 with HCC at time of antiviral therapy, our cohort consisted of 349 F3-4 SVR patients. Twenty-three patients (6.6%) developed HCC after a median FU of 5.12years. The sensitivity of the different models varied between 0.17 (Semmler7noalcohol) and 1 (Alonso A and aMAP). The lowest proportion of high-risk patients corresponded to the Semmler-noalcohol model (5%). Sixty-three and 90% of the Alonso A and aMAP patients, respectively were labelled as high risk. The most reliable HCC risk-model applied to our cohort to predict HCC development is the Alonso model (based on fibrosis stage assessed by liver stiffness measurements or Fibrosis-4 index (FIB-4) at baseline and after 1year, and albumin levels at 1year) with a-100% sensitivity in detecting HCC among those at high risk and 63% labelled as high risk. The application of the model would have saved the cost of 1290 ultrasound no longer being performed in the 37% low-risk group. In our cohort, the Alonso A model allows the most reliable reduction in HCC screening resulting in safely stopping life-long monitoring in about a third of F3-F4 patients achieving SVR with DAAs.

A clinical case of a continuous course of ulcerative with extra-intestinal manifestations

Ulcerative colitis (UC) is one of the most serious and poor-studied problems of modern gastroenterology. This nosology is a diffuse inflammation of the intestinal mucosa, limited to the colon with a predominant lesion of the rectum and sigmoid colon. Due to the progressive nature of the disease, patients require lifelong drug therapy and monitoring. Extra-intestinal manifestations are often found in patients with UC and represent a complex problem that requires the close attention of specialists and the appointment of modern pathogenetically-based treatment, including genetically engineered biological drugs. This clinical observation reflects the importance of early diagnosis of UC with extra-intestinal manifestations – primary sclerosing cholangitis and arthropathy, as well as the effectiveness of treatment with ustekinumab.

Lichen Sclerosus: A Survey of Diagnosis and Management Among Pediatric Dermatologists and Gynecologists.

Lichen sclerosus (LS) is a chronic condition that warrants close follow-up due to the risk of scarring. The optimal long-term management of pediatric vulvar and perianal lichen sclerosus (PVPLS) is unknown. This study aimed to identify diagnostic, treatment, and maintenance regimens among pediatric dermatologists and pediatric/adolescent gynecologists, as well as assess provider confidence and desire for guidance on long-term PVPLS management. A cross-sectional 35-question survey was administered through the Pediatric Dermatology Research Alliance (PeDRA) and the North American Society for Pediatric and Adolescent Gynecology (NASPAG) between 7/13/2021 and 8/30/2021 to ascertain PVPLS diagnostic and management regimens. Most responders were attending-level pediatric/adolescent gynecologists (46%) and pediatric dermatologists (41%). Although 85% of participants felt completely or very confident in diagnosing PVPLS, the majority (86%) desired further management guidelines. While the initial treatment was similar among providers, maintenance regimens and follow-up varied considerably, with only 42% recommending lifelong monitoring despite potential persistence into adulthood. While initial treatment was similar among practitioners, there was variation by specialty in subsequent management and a lack of uniformity in long-term follow-up. Additional studies are needed to clarify the optimal management of PVPLS and to provide evidence-based guidelines regarding long-term follow-up.&nbsp; J Drugs Dermatol. 2024;23(6):450-455.&nbsp; &nbsp;&nbsp; doi:10.36849/JDD.8084.

Quality of life of people living with chronic hepatitis B: The role of social support system.

People living with chronic hepatitis B (PLCHB) are recommended to follow a lifelong monitoring regimen and face increased risk of liver cancer. Additionally, PLCHB frequently encounter stigma and discrimination, and relationship disruptions because of their chronic hepatitis B (CHB). Social support plays a key role in coping with chronic illnesses; however, this is inadequately assessed for PLCHB. This study aims to assess the physical, social, and mental impacts of living with CHB, the strategies PLCHB utilize to cope with their disease, and how social support-or lack of-impacts their journey with hepatitis B. The study was promoted through the Hepatitis B Foundation social media platforms, interested individuals filled-in a form expressing their interest to participate. The researcher conducted 24 telephone interviews in English, with PLCHB ≥18 years of age residing in the United States (U.S.) and Canada. Questions focused on the lived experiences of CHB and explored social support mechanisms that helped PLCHB. PLCHB experience a wide range of impacts (physical, social, and mental) that negatively affect their quality of life. Participants reported that receiving social support from their close network of individuals, hepatitis B community, or healthcare providers positively influenced their perspective on their future health and helped them adhere to treatment. The physical, social, and mental impacts of living with hepatitis B significantly affect the quality of life of PLCHB, calling for more research to document these impacts, and design integrated care models to address them. Social support appears to play an essential role in helping PLCHB cope with their disease and should be further studied.

Update on Lupus Nephritis

Lupus Nephritis (LN) is a common and severe manifestation of systemic lupus erythematosus (SLE), impacting up to 40% of SLE patients. Despite advancements in understanding the pathogenesis of LN, outcomes have not significantly improved since the early 2000s. LN patients face higher mortality, emphasizing the importance of achieving disease remission. Screening for nephritis involves regular monitoring, especially within the first 5 years of SLE diagnosis. Monitoring includes urinalysis, serum creatinine, and immune serology. Kidney biopsy remains the gold standard for LN diagnosis and classification, providing crucial information for treatment decisions. The standard of care involves hydroxychloroquine for all LN patients, with immunosuppressive treatments tailored to the histologic class. The recently approved medications, belimumab and voclosporin, offer additional therapeutic alternatives. Approximately 20% of LN patients exhibit features of thrombotic microangiopathy, warranting anticoagulation. Optimizing glucocorticoid dosing is recommended, favouring lower doses to minimize adverse effects. Lifelong monitoring is essential, as flares can occur at any point, emphasizing the need for continued immunosuppression. Given the lack of renal response in 30–60% of patients, the addition of combination therapies, such as calcineurin inhibitors or belimumab, should be considered. Duration of treatment is crucial, considering the progressive loss of podocytes and nephron function, which may lead to chronic kidney disease. Regular monitoring, maintenance immunosuppression, and lifestyle modifications contribute to preventing flares and improving long-term outcomes for LN patients.

Enfermedad crónica transmisible y no transmisible, y enfermedad cutánea del paciente migrante

The care of migrant patients includes initial screening and lifelong monitoring, highlighting the importance of preventing and tracking chronic, communicable and non-communicable diseases.The prevalence of hypertension, diabetes mellitus, dyslipidemia, and obesity varies by ethnicity, influenced by genetic factors, lifestyle, and socio-economic status. Preventive measures, health promotion, and risk factor identification are crucial.Chronic communicable diseases may manifest years after transmission, underscoring the necessity of primary care screening, especially for populations from endemic or high-risk areas.Imported skin lesions are a common reason for consultation among migrant and traveller patients. Their ethiology is varied, ranging from common conditions such as scabies, mycoses, and urticaria to tropical dermatoses like filariasis and leprosy.

Health-Seeking Behavior of Persons with Chronic Hepatitis B in Peri-Urban Ghana: Application of the Health Belief Model

Sub-Saharan African countries, including Ghana, are disproportionately affected by hepatitis B. In Ghana, the burden of hepatitis B is unevenly distributed, with the Brong Ahafo region having the highest prevalence (13.7%). Given that people with hepatitis B (PWHB) in Ghana have previously been found to have a lack of understanding of the impact of the infection, we sought to explore their health-seeking behaviour using the health belief model as an organising framework. A qualitative exploratory design was used. In total, 18 people were purposively selected for face-to-face interviews. The data was processed and analysed using QSR NVivo version 11.0 and the Braun and Clarke thematic analysis procedure. The belief that hepatitis B can cause liver cancer and death was the most important determinant of health seeking. Furthermore, access to accurate hepatitis B information, particularly information about availability of effective hepatitis B treatment, influenced a number of participants to seek formal care. However, the high cost of clinical monitoring and treatment to use herbal medicine, despite their concerns about the effectiveness of herbal medicines in managing hepatitis B. Given that hepatitis B information was a factor in health seeking, it is recommended that a hepatitis B awareness campaign focusing on the availability of hepatitis B treatment and where it can be obtained be carried out in the study area. Counselling PWHB at the point of diagnosis should highlight the relevance of life-long clinical monitoring. To remove financial barriers to hepatitis B care in Ghana, the government should include the cost of hepatitis B laboratory investigations and treatment in the health insurance scheme.

MIR145 Core Promoter Methylation in Pretreatment Cell-Free DNA: A Liquid Biopsy Tool for Muscle-Invasive Bladder Cancer Treatment Outcome.

The lack of personalized management of bladder cancer (BlCa) results in patients' lifelong post-treatment monitoring with invasive interventions, underlying the urgent need for tailored and minimally invasive health care services. On the basis of our previous findings on miR-143/145 cluster methylation in bladder tumors, we evaluated its clinical significance in pretreatment cell-free DNA (cfDNA) of patients with BlCa. Methylation analysis was performed in our screening cohort (120 patients with BlCa; 20 age-matched healthy donors) by bisulfite-based pyrosequencing. Tumor recurrence/progression for patients with non-muscle-invasive bladder cancer, and progression and mortality for patients with muscle-invasive bladder cancer (MIBC) were used as clinical end point events in survival analysis. Bootstrap analysis was applied for internal validation of Cox regression models and decision curve analysis for assessment of clinical benefit on disease prognosis. Decreased methylation of MIR145 core promoter in pretreatment cfDNA was associated with short-term disease progression (multivariate Cox: hazard ratio [HR], 2.027 [95% CI, 1.157 to 3.551]; P = .010) and poor overall survival (multivariate Cox: HR, 2.098 [95% CI, 1.154 to 3.817]; P = .009) of patients with MIBC after radical cystectomy (RC). Multivariate models incorporating MIR145 promoter methylation in cfDNA with tumor stage clearly ameliorated patients' risk stratification, highlighting superior clinical benefit in MIBC prognostication. Reduced pretreatment cfDNA methylation of MIR145 core promoter was markedly correlated with increased risk for short-term progression and worse survival of patients with MIBC after RC and adjuvant therapy, supporting modern personalized and minimally invasive prognosis. Methylation profiling of MIR145 core promoter in pretreatment cfDNA could serve as a minimally invasive and independent predictor of MIBC treatment outcome and emerge as a promising marker for blood-based test in BlCa.

The factors influencing compliance in post-EVAR surveillance regarding the perspectives of practitioners and patients

Aim: Lifelong monitoring is essential to detect aorta-related complications. Yet, patient non-compliance with surveillance programs poses a significant obstacle. We explored reasons for adherence by engaging in clear, patient-centred communication and analyzed factors contributing to non-compliance. Material and Methods: In 2019, our clinic conducted elective EVAR procedures on 50 patients. They were informed of annual surveillance, including hospital visits, starting with a first-month appointment post-discharge. Patients with no or only one follow-up were deemed non-compliant, while two to three follow-ups indicated moderate compliance, and four to seven follow-ups qualified as high compliance. We recorded each patient's age, education level and demographics. We retrospectively assessed the factors on their compliance level as our first outcome. The second was to explore whether the survey could enlighten any reasons from the patient's perspective. Results: The average age of our cohort was 68.8±8.45 years, with only three females. Non-attendance at follow-ups was observed in 8 patients (16%). Seventy percent (35 patients) complied with at least two appointments. Residing in the same city markedly increased compliance by 20 times (p&lt;0.001). Older age appeared to influence compliance positively, on the borderline significantly (p=0.057). The primary motivation for attendance was adherence to doctor recommendations (n=14), while the predominant reason for non-attendance was patients’ feeling well (n=29). Notably, patients with lower compliance than predicted more frequently cited COVID-19 as a deterrent (p=0.02). Conclusion: Residence within the same city positively influences follow-up compliance. The national databases might be helpful tools for enhancing the surveillance anywhere the patients live. The existing hypotheses by healthcare professionals about patient follow-up compliance are only partially corroborated. Incorporating the patient's perspective into these models may be helpful in comprehensively understanding the underlying factors.

Current and emerging gene therapies for haemophilia A and B.

After decades of stumbling clinical development, the first gene therapies for haemophilia A and B have been commercialized and have normalized factor (F)VIII and factor (F)IX levels in some individuals in the long term. Several other clinical programs testing adeno-associated viral (AAV) vector gene therapy are at various stages of clinical testing. Multiyear follow-up in phase 1/2 and 3 studies showed long-term and sometimes curative but widely variable and unpredictable efficacy. Liver toxicities, mostly low-grade, occur in the 1st year in at least some individuals in all haemophilia A and B trials and are poorly understood. Wide variability and unpredictability of outcome and slow decline of FVIII levels are a major disadvantage because immune responses to AAV vectors preclude repeat dosing, which otherwise could improve suboptimal or restore declining expression, while overexpression may predispose to thrombosis. Long-term safety outcomes will need lifelong monitoring because AAV vectors infused at high doses integrate into chromosomes at rates that raise questions about potential oncogenicity and necessitate vigilance. Alternative gene transfer systems employing gene editing and/or non-viral vectors are under development and promise to overcome some limitations of the current state of the art for both haemophilia A and B. AAV gene therapies for haemophilia have now become new treatment options but not universal cures. AAV is a powerful but imperfect gene transfer platform. Biobetter FVIII transgenes may help solve some problems plaguing gene therapy for haemophilia A. Addressing variability and unpredictability of efficacy, and delivery of gene therapy to ineligible patient subgroups may require different gene transfer systems, most of which are not ready for clinical translation yet but bring innovations needed to overcome the current limitations of gene therapy.

Imaging follow-up strategy after endovascular treatment of Intracranial aneurysms: A literature review and guideline recommendations.

Endovascular coil embolization is the primary treatment modality for intracranial aneurysms. However, its long-term durability remains of concern, with a considerable proportion of cases requiring aneurysm reopening and retreatment. Therefore, establishing optimal follow-up imaging protocols is necessary to ensure a durable occlusion. This study aimed to develop guidelines for follow-up imaging strategies after endovascular treatment of intracranial aneurysms. A committee comprising members of the Korean Neuroendovascular Society and other relevant societies was formed. A literature review and analyses of the major published guidelines were conducted to gather evidence. A panel of 40 experts convened to achieve a consensus on the recommendations using the modified Delphi method. The panel members reached the following consensus: 1. Schedule the initial follow-up imaging within 3-6 months of treatment. 2. Noninvasive imaging modalities, such as three-dimensional time-of-flight magnetic resonance angiography (MRA) or contrast-enhanced MRA, are alternatives to digital subtraction angiography (DSA) during the first follow-up. 3. Schedule mid-term follow-up imaging at 1, 2, 4, and 6 years after the initial treatment. 4. If noninvasive imaging reveals unstable changes in the treated aneurysms, DSA should be considered. 5. Consider late-term follow-up imaging every 3-5 years for lifelong monitoring of patients with unstable changes or at high risk of recurrence. The guidelines aim to provide physicians with the information to make informed decisions and provide patients with high-quality care. However, owing to a lack of specific recommendations and scientific data, these guidelines are based on expert consensus and should be considered in conjunction with individual patient characteristics and circumstances.