Acute Attacks Research Articles

Efficacy of Adenosine Versus Verapamil in Terminating Acute Attacks of Paroxysmal Supraventricular Tachycardia: A Systematic Review.

Paroxysmal supraventricular arrhythmias are a group of common rhythm disturbances that are often prevalent, frequently recurrent, sporadic, andlife-threatening. These arrhythmias are precipitated by factors such as age, sex, and associated comorbidities. Typically, patients with paroxysmal arrhythmias are asymptomatic during evaluation, and the condition is often detected incidentally. Symptoms associated with these arrhythmias include palpitations, fatigue, light-headedness, chest discomfort, dyspnea, presyncope, and, less commonly, polyuria and serious psychological distress. In terms of treatment, common modalities include antiarrhythmic drug therapy and catheter ablation. When selecting drug therapy, factors such as comorbidities, patient-specific modifiers, preferences, follow-up frequency, and cost-effectiveness are taken into account. For long-term treatment, calcium channel blockers are often used instead of adenosine, while adenosine is preferred for acute attacks due to its higher efficacy. Comparatively, adenosine and verapamil are commonly used drugs in the emergency setting to treat paroxysmal supraventricular tachycardia (PSVT). Adenosine exhibits a faster onset of action, but adverse effects occur more commonly, whereas verapamil has a slower onset of action and a lower incidence of adverse effects. Wesearched for articles from PubMed, PubMed Central (PMC), and Science Direct, and these articles were reviewed systematically. After applying the search strategy to these databases, 195 articles were identified. Fourteen of these were finalized for review. The efficacy of adenosine versus verapamil in terminating acute attacks of PSVT is reviewed in our article.

Qufeng Xuanbi Formula inhibited benzo[a]pyrene-induced aggravated asthma airway mucus secretion by AhR/ROS/ERK pathway

Ethnopharmacological relevanceExcessive secretion of airway mucus may be an important pathological factor of air pollution-induced acute asthma attacks. Treatment of airway mucus hypersecretion improves asthma aggravated by air pollutants. Qufeng Xuanbi Formula (QFXBF) has been used to treat asthma for more than 30 years. However, whether QFXBF inhibits asthmatic mucus secretion exacerbated by air pollutants has not yet been established. Aim of the studyThis study aimed to evaluate the effect of QFXBF on airway mucus secretion and the mechanism of action in an air pollutant benzo[a]pyrene (BaP)-induced mouse model of aggravated asthma. Materials and methodsOvalbumin (OVA) and BaP co-exposure were used to establish the aggravated asthma model. The average enhanced pause (Penh), serum OVA-specific IgE, and changes in lung histopathology were determined. 16HBE cells exposed to BaP, treatment with QFXBF, arylhydrocarbon receptor (AhR) signal antagonist SR1, reactive oxygen species (ROS) antagonist NAC, or extracellular signal-regulated kinase (ERK1/2) signal antagonist U0126 were established to investigate the effect of QFXBF on BaP-induced mucus secretion and its target. The mRNA and protein expression levels of MUC5AC in the lung tissue and 16HBE cells were examined. We also studied the effect of QFXBF on ROS production. Finally, the protein expression of AhR, phospho-extracellular signal-regulated kinases (p-ERK1/2), and ERK1/2 in 16HBE cells and lung tissues was determined by western blotting. ResultsAdministration of QFXBF significantly alleviated the pathological symptoms, including Penh, serum OVA-specific IgE, and changes in lung histopathology in a BaP-induced mouse model of aggravated asthma. QFXBF inhibited MUC5AC expression in asthmatic mice and 16HBE cells exposed to BaP. ROS production, AhR expression, and ERK1/2 phosphorylation were significantly increased in BaP-induced asthmatic mice and 16HBE cells. Signaling pathway inhibitors StemRegenin 1 (SR1), NAC, and U0126 significantly inhibitedBaP-induced MUC5AC expression in 16HBE cells. SR1 reversed Bap-induced ROS production and ERK activation, and NAC inhibited Bap-induced ERK activation. In addition, QFXBF regulated AhR signaling, inhibited ROS production, reversed ERK activation, and downregulated mucus secretion to improve asthma aggravated by air pollutant BaP. ConclusionsQFXBF can ameliorate mucus secretion in BaP-induced aggravated asthmatic mice and 16HBE cells, and the specific mechanism may be related to the inhibition of the AhR/ROS/ERK signaling pathway.

Percutaneous Cholecystostomy to Manage a Hot Gallbladder: A Single Center Experience.

Objective A percutaneous cholecystostomy (PC) is a suitable option for treating acutely inflamed gallbladders. Its use has been postulated before for treating acute cholecystitis (AC), especially in elderly populations. The primary aim of our study is to analyze and present the positive results of PC as a bridge to laparoscopic cholecystectomy. Methods All patients who underwent PC at our hospital, Princess Royal University Hospital, King's College Hospital NHS Foundation Trust, London, GBR, from October 2020were reviewed using a retrospective approach. Results Our study comprises 123 patients, with 72 females (58.5%) and 51 males (41.4%). In our study, many patients had significant comorbidities, and some of them were categorized as high-risk due to their frailty and medical conditions. The majority of the patients were in American Society of Anaesthesiologists' (ASA) groups II and III (45, 61), respectively. Though hospital stays can depend on variable factors, in our experience, the mean hospital length ofstay was 12.7 days. In our study, 119 patients (96.8%) had the procedure through the interventional radiological approach, while only four patients had it through the laparoscopic approach. The transhepatic route for drainage was more commonly practiced at our center and was used in 108 patients. At the time of writing this article, 54 patients have already had a laparoscopic cholecystectomy (LC) done as an interval procedure after surpassing the acute attack of cholecystitis, while 42 patients are still awaiting their surgical procedure. Conclusion Our results show that PC is a viable option, especially in cases of AC that are not responding to conservative treatments. Our study has shown low complications and conversion rates after PC. We believe PC is a safe and effective tool for managing severe and refractory cases of AC.

Herbal medicine for external use in acute gouty arthritis: A PRISMA-compliant systematic review and meta-analysis.

Acute gouty arthritis is accompanied by severe pain during an acute attack. This systematic review aimed to evaluate the efficacy and safety of herbal medicines acting directly on the affected area of acute gouty arthritis for external use. An envelope search was performed using 4 electronic databases (CNKI, PubMed, EMBASE, Cochrane), resulting in 27 clinical studies from inception to February 2023. Randomized controlled trials on external use herbal medicines for acute gouty arthritis were considered. The assessed outcomes were total effective rate, uric acid level, pain score, and inflammatory factor levels such as erythrocyte sedimentation rate and C-reactive protein. Quality assessment and meta-analysis of the included randomized controlled trials were also performed. Twenty-seven randomized controlled trials with a total of 1951 participants were included in the meta-analysis. All assessed outcomes including pain, inflammation, and uric acid levels, indicated that the treatment effects in the external use herbal medicine group were significantly better than those of the western medicine control group. Of the 10 studies mentioning side effects, no side effects were reported in 4, and in the remaining 6, the incidence of complications in the intervention group was much lower than that in the control group. This systematic review and meta-analysis suggests that external use herbal medicines may be a safe and effective alternative for treatment of pain and symptoms of acute gouty arthritis. However, owing to the heterogeneity of interventions, outcomes, and regional bias, further high-quality clinical trials on this topic are needed to confirm the level of evidence.

The Stiff Joint: Comparative Evaluation of Monotherapy and Combination Therapy With Urate Lowering Agents in Managing Acute Gout.

Gout, an extremely painful form of arthritis, is triggered by the innate immune system's response to the accumulation of monosodium urate crystals in specific joints and surrounding tissues. This condition is characterized by recurring episodes of excruciating arthritis flares, interspersed with periods of disease quiescence. Over time, gout can result in disability, tophi formation, and severe pain. The treatment of gout is centered around two main objectives: alleviating inflammation and pain during acute gout attacksand long-term management to reduce serum urate levels and mitigate the risk of future attacks. Addressing inflammation and pain during acute attacks is often complicated by various factors, including underlying health conditions commonly associated with gout, such as hypertension, chronic kidney disease, cardiovascular disease, and diabetes mellitus. Moreover, gout patients are frequently older and have multiple coexisting health issues, necessitating complex medication regimens. Given the rising prevalence of gout and its associated comorbidities, there's a growing demand for improved treatment options. While existing treatments effectively manage gout in some patients, a significant portion, particularly those with comorbidities, face contraindications to these treatments and require alternative approaches. Innovative medications are required to enhance gout treatment, especially for individuals with concurrent health conditions. These considerations underscore the importance of reviewing both monotherapy and combination therapy approaches for acute gout treatment.

First Report of a Low-Frequency Mosaic Mutation in the Hydroxymethylbilane Synthase Gene Causing Acute Intermittent Porphyria.

Acute porphyrias are a group of monogenetic inborn errors of heme biosynthesis, characterized by acute and potentially life-threatening neurovisceral attacks upon exposure to certain triggering factors. Biochemical analyses can determine the type of acute porphyria, and subsequent genetic analysis allows for the identification of pathogenic variants in the specific gene, which provides information for family counselling. In 2017, a male Swiss patient was diagnosed with an acute porphyria while suffering from an acute attack. The pattern of porphyrin metabolite excretion in urine, faeces, and plasma was typical for an acute intermittent porphyria (AIP), which is caused by inherited autosomal dominant mutations in the gene for hydroxymethylbilane synthase (HMBS), the third enzyme in the heme biosynthetic pathway. However, the measurement of HMBS enzymatic activity in the erythrocytes was within the normal range and Sanger sequencing of the HMBS gene failed to detect any pathogenic variants. To explore the molecular basis of the apparent AIP in this patient, we performed third-generation long-read single-molecule sequencing (nanopore sequencing) on a PCR product spanning the entire HMBS gene, including the intronic sequences. We identified a known pathogenic variant, c.77G>A, p.(Arg26His), in exon 3 at an allelic frequency of ~22% in the patient's blood. The absence of the pathogenic variant in the DNA of the parents and the results of additional confirmatory studies supported the presence of a de novo mosaic mutation. To our knowledge, such a mutation has not been previously described in any acute porphyria. Therefore, de novo mosaic mutations should be considered as potential causes of acute porphyrias when no pathogenic genetic variant can be identified through routine molecular diagnostics.

Characteristics and Drug Utilization of Patients with Hereditary Angioedema in Italy, a Real-World Analysis.

This real-world analysis investigated the characteristics and treatment patterns of patients with hereditary angioedema (HAE) in Italy using the administrative data of health units across Italy. Patients were identified via exemption code or HAE-specific treatments (thus, all known forms, type I, II and, III, were included). The index date was that of first prescription of HAE treatments within the inclusion period (01/2010-06/2021) or of the date of exemption. The number of HAE patients included was 148 (43.2% male, mean age 43.3 years). Gastrointestinal disorders affected 36.5% patients, hypertension affected 28.4%, hypercholesterolemia affected 11.5%, and depression affected 9.5%. The frequent gastrointestinal involvement was further confirmed by the use of antiemetics and systemic antihistamines that doubled after the index date. Among patients enrolled by treatment (n = 125), n = 105 (84%) were receiving a treatment for acute attacks. This analysis provided insights into the characterization of patients with HAE and their management in Italian clinical practice, suggesting that an unmet therapeutic need could be present for such patients in terms of the clinical burden.

Porphyrin precursors and risk of primary liver cancer in acute intermittent porphyria: A case-control study of 188 patients.

Acute intermittent porphyria (AIP) is a rare hereditary metabolic disease characterized by acute attacks and accumulation of the porphyrin precursors 5-aminolevulinic acid (ALA) and porphobilinogen (PBG). Patients with AIP have a high risk of primary liver cancer (PLC). We aimed to assess the association between porphyrin precursor excretion and the risk for PLC in patients with AIP. We studied 48 patients with AIP who developed PLC between 1987 - 2015 and 140 age and sex matched controls with AIP but no PLC. Data on all available urinary PBG and ALA samples collected from 1975 until one year before PLC diagnosis were analyzed and compared between cases and controls using logistic regression. Porphyrin precursor excretion was higher in patients with PLC (PBG median 7.9 (IQR 4.4-21.9) mmol/mol creatinine) than in controls (3.8 (1.2-9.8)) (adjusted odds ratio 1.07, 95% confidence interval: 1.02-1.12). None of the 28 patients with all registered samples below the upper limit of normal (ULN) developed PLC, and only one of the 45 patients with all samples <2x ULN developed PLC. Among non-PLC controls, ALA and PBG levels decreased after age 50-60 while an increasing trend was observed after age 65 among those who developed PLC. Increased urinary porphyrin precursors are associated with a high risk of developing PLC. Patients with normal levels appear to have a low risk while high or increasing ALA and PBG after age 65 indicates high risk, which should be considered in surveillance decisions. This article is protected by copyright. All rights reserved.

Bradykinin-Mediated Angioedema Induced by Commonly Used Cardiovascular Drugs

ACE inhibitors, sartans, and sacubitril are among the most important drugs for the prevention of cardiovascular mortality and morbidity. At the same time, they are known to cause non-allergic bradykinin-mediated angioedema, a potentially fatal swelling of the mucosa and/or submucosa and deeper skin without signs of urticaria or pruritus, occurring mainly in the head and neck region. In contrast with hereditary angioedema, which is also mediated by bradykinin, angioedema triggered by these drugs is by far the most common subtype of non-allergic angioedema. The molecular mechanisms underlying this type of angioedema, which are discussed here, are not yet sufficiently understood. There are a number of approved drugs for the prevention and treatment of acute attacks of hereditary angioedema. These include inhibitors of bradykinin synthesis that act as kallkrein inhibitors, such as the parenterally applied plasma pool, and recombinant C1 esterase inhibitor, ecallantide, lanadelumab, and the orally available berotralstat, as well as the bradykinin receptor type 2 antagonist icatibant. In contrast, no diagnostic tools, guidelines, or treatments have yet been approved for the diagnosis and treatment of acute non-allergic drug-induced angioedema, although it is more common and can take life-threatening courses. Approved specific drugs and a structured diagnostic workflow are needed for this emergency diagnosis.

Epidemiology and risk factors associated with gout control among adult Asians: a real-world retrospective cohort study.

Gout is associated with significant morbidity and mortality, yet suboptimal gout control remains a problem globally. Identifying the risk factors associated with poor gout control among patients in primary care allows targeted interventions to improve their clinical management. This study aimed to determine the prevalence of poor gout control and its associated demographic and clinical factors among urbanized community-dwelling Asian patients. This retrospective study was based on data extracted from the electronic medical records of 8 public primary care clinics in Singapore. Patients with a diagnostic code of gout who had 2 or more visits between 1st January 2018 and 31st December 2019 were included in the analysis. Data extracted included: demographics, anthropological measurements, comorbidities, serum uric acid levels and medication prescription. A patient is defined to have poor gout control if they suffer two or more acute gout attacks within a year. Chi-Squared test was used for categorical parameters. For continuous variables, univariate logistic regression analysis was first performed. Significant factors (p ≤ 0.1) were then included in the logistics regression model to account for confounders. A total of 7,970 patients and 24,624 visits were included in the analysis. The prevalence of poorly controlled gout was 28.2% (n = 2,244/7,970); only 46.3% of them (n = 1,039/2,244) were prescribed allopurinol and 13.4% (n = 301/2,244) were taking doses ≥300 mg. Using logistic regression, factors associated with poor gout control were: male gender [adjusted OR (AOR) =1.66, p < 0.001], Malay ethnicity (AOR = 1.27, p = 0.007), congestive heart failure (AOR = 1.64, p = 0.037). Patients prescribed allopurinol (AOR = 1.52, p < 0.001), NSAIDs (AOR = 2.76, p < 0.001) and corticosteroids (AOR = 2.83, p < 0.001) were more likely to have poorly-controlled gout. Nearly 30% of patients had poor gout. Interventions should focus on male and Malay patients and those with congestive cardiac failure.

Update on the diagnosis and treatment of neuromyelitis optica spectrum disorders (NMOSD) - revised recommendations of the Neuromyelitis Optica Study Group (NEMOS). Part II: Attack therapy and long-term management.

This manuscript presents practical recommendations for managing acute attacks and implementing preventive immunotherapies for neuromyelitis optica spectrum disorders (NMOSD), a rare autoimmune disease that causes severe inflammation in the central nervous system (CNS), primarily affecting the optic nerves, spinal cord, and brainstem. The pillars of NMOSD therapy are attack treatment and attack prevention to minimize the accrual of neurological disability. Aquaporin-4 immunoglobulin G antibodies (AQP4-IgG) are a diagnostic marker of the disease and play a significant role in its pathogenicity. Recent advances in understanding NMOSD have led to the development of new therapies and the completion of randomized controlled trials. Four preventive immunotherapies have now been approved for AQP4-IgG-positive NMOSD in many regions of the world: eculizumab,ravulizumab - most recently-, inebilizumab, and satralizumab. These new drugs may potentially substitute rituximab and classical immunosuppressive therapies, which were as yet the mainstay of treatment for both, AQP4-IgG-positive and -negative NMOSD. Here, the Neuromyelitis Optica Study Group (NEMOS) provides an overview of the current state of knowledge on NMOSD treatments and offers statements and practical recommendations on the therapy management and use of all available immunotherapies for this disease. Unmet needs and AQP4-IgG-negative NMOSD are also discussed. The recommendations were developed using a Delphi-based consensus method among the core author group and at expert discussions at NEMOS meetings.

Knowledge and awareness of stroke in the United Arab Emirates: a cross-sectional study of the general population

Background: The study aimed to assess stroke knowledge among the general population in the United Arab Emirates (UAE) and determine the factors associated with stroke awareness among people. Methods: A cross-sectional study was conducted in the UAE between September and November 2021. The general population has self-administered an online bilingual survey (Arabic and English) distributed via social media platforms. The questionnaire covered general knowledge about stroke risk factors, consequences of stroke, and responding to somebody with acute stroke attack. Results: A total of 545 surveys were completed, with more than half of the participants being female (58.5%), married (55.4%) and employed (59.4%). The majority were less than 50 years old (90.5%) and had a university degree (71.0%). Many of the participants (70.8%) had a good general knowledge of stroke; however, around 20% of the participants were able to recognize all symptoms and risk factors of stroke. The most common sources of stroke-related information were the internet/social media (53.6%). Females were better able to correctly identify at least one stroke symptom and outcome than males (p=0.008). University education has significantly affected participants' ability to identify early stroke symptoms (p=0.001) correctly. In addition, diabetic people were more likely to recognize at least one stroke outcome than non-diabetic people (p=0.039). Conclusions: The knowledge of stroke was good among highly educated people and females. However, the awareness of all stroke risk factors, symptoms, consequences and risk factors was inadequate among the general population of the UAE. Thus, there is still a gap between recognition of the relevant stroke and taking action among people.

Efficacy of Aristolochia rotunda Linn in hyperuricemia: A randomised standard controlled study

Hyperuricemia (HU) is a health risk that may go undiagnosed and is on the rise in both developed and developing countries. Gout, the most common inflammatory arthritis characterized by painful, disabling acute attack, is widely known to be caused by hyperuricemia. Its prevalence ranges from 2.6% to 36% in different populations. The commonly used drugs for alleviating uric acid and gout have obvious side effects, so herbal therapeutic drugs are in high demand. The aim of the present study was to evaluate the efficacy and safety of Aristolochia rotunda Linn in Hyperuricemia. Present study was designed as a single-blind randomized standard controlled trial with 20 patients in each group. Participants in the test group were administered A.rotunda Linn 4gms in two divided doses in capsule form and the control group was given Febuxostat 40mg once daily after meals for 28 days. Participants were asked to follow up weekly for the assessment of subjective parameters. The objective parameter was assessed pre- and post-trial. The results were analysed statistically. After the intervention, the test and control groups showed a statistically significant reduction in serum uric acid p=0.021 and p<0.01 respectively, while the reduction in the control group was found to be more statistically significant than the test group (p=0.009). Subjective parameters also showed statistical significance at the end of the trial. This study shows that the A.rotunda Linn in a dose of 4gm for 28 days effectively lowers serum uric acid. The trial was registered in the clinical trial registry of India under CTRI No. CTRI//2020/02/031587.

Immediate analgesic effect of acupuncture intervention within 10 min during acute migraine attacks: protocol of a randomised controlled trial

IntroductionMigraine is a widespread neurological disorder characterised by recurrent moderate-to-severe headaches. These headaches can seriously affect patients’ daily life and work, especially during acute attacks when patients often need immediate...

Regular (up to 10 puffs 4-hourly) inhaled salbutamol should be prescribed at discharge after an asthma attack: myth or maxim?

Over the past 20 years, the concept of asthma weaning plans on discharge after an attack has crept into common practice, although the precise origin of these plans is unclear. High use of short-acting β2-agonists (SABAs) may result in tolerance to their bronchodilator effects, thus diminishing their efficacy, particularly when they are most needed at the time of an acute attack. Furthermore, key warning signs of a deterioration in asthma control may be masked and the weaning plan may encourage the over-use and over-reliance on SABAs. Side-effects from over-use may also occur, including lactic acidosis, downregulation of the β2-adrenoreceptor, increased allergen response and pro-inflammatory effects. The need for asthma education at discharge, a personal asthma action plan and vigilance about prescribing and ensuring adherence to maintenance therapy are definitely important. However, the current authors conclude that the benefit of prescribing regular salbutamol (up to 10 puffs every 4 h) at discharge after an acute asthma attack is a myth, and a very dangerous one.

Spontaneous resolution of acute gout: mechanisms and therapeutic targets

Gout is a common inflammatory arthritis that has been increasing in both prevalence and incidence. Consequently, management of refractory and chronic gout has been gaining attention. Onset of gout is...

Real-world experience of hereditary angioedema (HAE) in Mexico: A mixed-methods approach to describe epidemiology, diagnosis, and treatment patterns

Introduction and objectivesDue to the lack of structured and systematic information available, the aim of this study was to describe the epidemiology, diagnosis, healthcare processes, and treatment patterns of hereditary angioedema (HAE) in Mexico. To achieve this, different data sources were consulted regarding medical literature, structured health system databases, and angioedema-specialized physicians (AEP) opinion regarding HAE. Material and methodsA mixed methods approach was conducted in 4 phases: I) systematic literature review (SLR) and meta-analysis according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines; II) review of national health system (NHS) databases and systematic reports; III) physician survey; and IV) an epidemiologic model. ICD 10 D84.1 encoded records from the NHS were used to estimate the number of patients with HAE attended and treated during 2019. A survey was implemented to increase understanding of the clinical profile and treatments used. ResultsA prevalence rate of 0.9/50 000 inhabitants was estimated for 2019. In the same year, an estimated 317 HAE type 1 patients were recorded in the NHS, aged ≥11 years old. The most frequent clinical symptoms were cutaneous edema (67.5%) and abdominal pain (47.9%). A severe episode with laryngeal edema appeared in 27.5% of cases. Acute episodes were mainly moderate to severe (77.0%), with an annual per capita frequency of emergency visits of 7.6 patient-year (range 1–12/patient-year). The main reasons for hospitalization corresponded to laryngeal facial, tongue, and abdominal edemas, representing 73.3% of annual ICD 10 D84.1 reported hospitalizations. The main treatments that patients with HAE received were fresh frozen plasma for acute attacks and danazol for short-term prophylaxis (STP). ConclusionsDespite efforts to make HAE visible, according to this study, cases recognized and treated in the NHS represent only 16.6% of the estimated prevalence.

Very Rare Incident of Pediatric Onset Multiple Sclerosis: A Case Report

Background: Pediatric Onset Multiple Sclerosis (POMS) is a chronic inflammatory disease in the central nervous system that affects children under 18. Children with POMS experience gradual deterioration leading to increased disability and lower life expectancy [1,2]. The Purpose of the Study: to present a rare case of POMS with an onset at 42 months, emphasizing its significance due to its infrequency in this age group. The hope is to encourage the establishment of specific diagnostic and treatment guidelines for POMS in the near future. Material and Methods: The evolution of POMS in a 3.6-year-old child who was admitted multiple times to the Hospital of Mother and Child Health Care in the Republic of Moldova during acute MS attacks. Results: In September 2022, the child received a confirmed POMS diagnosis based on McDonald’s criteria (2017), which was later confirmed in April 2023 through OCB-CSF examination. The April 2023 MRI showed changes in demyelination foci with temporary clinical improvement compared to the previous examination after the last acute attack. The child is currently undergoing five days of plasmapheresis, followed by a tapering regimen of oral steroids. Ongoing MRI monitoring is recommended, with consideration of receiving Disease-Modifying Treatments (DMTs) in Romania, where they are approved. Conclusions: This rare case highlights the importance of raising awareness about POMS in children. Early detection and intervention could lead to the development of new diagnostic and treatment guidelines. However, the lack of access to DMT drugs for POMS treatment in the Republic of Moldova results in significantly lower survival rates compared to other countries.

Very Rare Incident of Pediatric Onset Multiple Sclerosis: A Case Report

Background: Pediatric Onset Multiple Sclerosis (POMS) is a chronic inflammatory disease in the central nervous system that affects children under 18. Children with POMS experience gradual deterioration leading to increased disability and lower life expectancy [1,2]. The Purpose of the Study: to present a rare case of POMS with an onset at 42 months, emphasizing its significance due to its infrequency in this age group. The hope is to encourage the establishment of specific diagnostic and treatment guidelines for POMS in the near future. Material and Methods: The evolution of POMS in a 3.6-year-old child who was admitted multiple times to the Hospital of Mother and Child Health Care in the Republic of Moldova during acute MS attacks. Results: In September 2022, the child received a confirmed POMS diagnosis based on McDonald’s criteria (2017), which was later confirmed in April 2023 through OCB-CSF examination. The April 2023 MRI showed changes in demyelination foci with temporary clinical improvement compared to the previous examination after the last acute attack. The child is currently undergoing five days of plasmapheresis, followed by a tapering regimen of oral steroids. Ongoing MRI monitoring is recommended, with consideration of receiving Disease-Modifying Treatments (DMTs) in Romania, where they are approved. Conclusions: This rare case highlights the importance of raising awareness about POMS in children. Early detection and intervention could lead to the development of new diagnostic and treatment guidelines. However, the lack of access to DMT drugs for POMS treatment in the Republic of Moldova results in significantly lower survival rates compared to other countries.

Patient-level indirect treatment comparison of lanadelumab versus pdC1-INH i.v. in hereditary angioedema patients: PATCH study.

Hereditary angioedema (HAE) is an autosomal dominant inherited disease in which patients suffer from local attacks primarily affecting skin and gastrointestinal tract, and sometimes even the upper respiratory tract leading to asphyxiation. Since head-to-head trials between authorized treatments are lacking, this study compares efficacy and safety of lanadelumab and intravenous plasma-derived C1-esterase inhibitor (pdC1-INH i.v.) in HAE patients on long-term prophylaxis by means of an indirect treatment comparison. Efficacy and safety of lanadelumab against pdC1-INH i.v. were analyzed in a fully prespecified indirect comparison based on individual patient data (n = 231) from the HELP and CHANGE clinical trials. Primary and secondary efficacy endpoints were compared using a generalized linear model for count data. Confounding variables were identified a priori via systematic literature research and validated by clinical experts. Adjustment of confounders was implemented using a conditional regression model. Lanadelumab showed a statistically significant improvement in reduction of HAE attack rates compared to pdC1-INH i.v. across multiple endpoints: Monthly attack rate of patients treated with lanadelumab was less than half compared to pdC1-INH i.v. (Rate ratio: 0.486; 95% CI: 0.253, 0.932). Monthly rate of laryngeal attacks was found to be five times lower for lanadelumab (Rate ratio: 0.2; 95% CI: 0.044, 0.915) and monthly rate of acute treated HAE attacks among lanadelumab patients was about one third of the attack rate of pdC1-INH i.v. patients (Rate ratio: 0.366; 95% CI: 0.185, 0.727). This study contributes to current knowledge in the treatment of HAE by indicating a statistically significant reduction of HAE attacks under lanadelumab compared to pdC1-INH i.v.