Prescription Of Therapy Research Articles

Elevated Risk of Thyroid Malignancy in Biological Males Taking Estrogen Hormone Therapy.

The role of estrogen in developing thyroid malignancy is poorly understood. Epidemiological studies have shown exogenous estrogen is associated with increased risk in females. Still, no studies to date have investigated this association among biological males undergoing estrogen hormone therapy. Retrospective cohort study. TriNetX Research Network. Biologically male patients prescribed estrogen with at least 5 years of follow-up were queried from the database. Rates of diagnosis of malignant neoplasm of the thyroid gland within 5 years of estrogen hormone therapy prescription were determined, and statistics were conducted within the TriNetX platform. We identified 6394 biologically male patients from 65 health care organizations prescribed estrogen hormone treatment. The average age was 44.4 years. When balanced for demographic factors as well as known risk factors for thyroid malignancy, the estrogen treatment cohort had a 0.64% risk for diagnosis of thyroid malignancy within 5 years, compared to a 0.27% risk among patients not taking estrogen (relative risk: 2.35, 95% confidence interval: 1.34-4.15, P = .002). We found a higher risk of developing thyroid cancer in biologically male patients prescribed estrogen hormone therapy. This is the first association found between estrogen and thyroid malignancy in this group.

Association between urate-lowering therapy and kidney failure in patients with chronic kidney disease.

Hyperuricemia is a hallmark of gout and a suspected risk factor for the progression of chronic kidney disease (CKD). However, the impact of urate-lowering therapy on CKD progression is subject to debate. The objective of the present study was to describe the prevalence of inappropriate urate-lowering therapy prescriptions and evaluate the association between urate-lowering therapy prescription and the progression of kidney disease in patients with CKD. CKD-REIN is a French, nationwide, prospective cohort of 3,033 nephrology outpatients with CKD (eGFR < 60mL/min/1.73 m2). Prescriptions of urate-lowering therapy drugs (allopurinol or febuxostat) were recorded prospectively. The appropriateness of each prescription was evaluated according to the patient's kidney function at baseline and during follow-up. Propensity score-matched, cause-specific Cox proportional hazards regression models were used to assess the association between incident urate-lowering therapy use and CKD progression (defined as the initiation of kidney replacement therapy (KRT) but also in other ways). At baseline, 987 of the 3009 patients included in this study (median age: 69; men: 66%) were receiving urate-lowering therapy; 396 of these 987 patients were receiving an inappropriate prescription with regard to their kidney function. During a 5-year follow-up period, 70% of the 396 urate-lowering therapy prescriptions remained inappropriate. In the propensity score-matched cohort (n = 674), 136 patients started KRT. Compared with non- urate-lowering therapy use, urate-lowering therapy use was not significantly associated with a slowing in CKD progression, regardless of the definition used (HRKRT 0.89, 95% CI 0.67-1.20). Our real-world data emphasized the lack of reassessment of urate-lowering therapy prescriptions in patients with CKD. Urate-lowering therapy was not associated with a slowing of CKD progression.

Neighborhood Social Vulnerability and Access to Expedited Partner Therapy Prescriptions: A Secret Shopper Audit Survey.

Sexually transmitted infections (STIs) disproportionately impact populations with higher social vulnerability. Expedited Partner Therapy (EPT), which allows the treatment of partners without requiring a medical visit, reduces STI reinfection rates and expands treatment access for underserved groups. However, EPT remains underutilized, particularly in the electronic prescription era, which introduces logistical complexities. Previous studies highlight low pharmacist awareness of EPT, but few have assessed its real world availability in pharmacies or how accessibility varies by socioeconomic subcomponent of the Social Vulnerability Index (SVI). This study aimed to evaluate pharmacist awareness and willingness to dispense electronic, nameless EPT prescriptions in New York City (NYC) pharmacies and examine how these outcomes vary by the socioeconomic factors of the pharmacy's location. A cross-sectional study of 347 randomly sampled NYC pharmacies was conducted using a telephone-based secret shopper approach. Research associates posed as patients seeking to fill hypothetical EPT prescriptions to assess pharmacist awareness, willingness to dispense, and insurance acceptability. Multivariable logistic regression models evaluated the association between EPT awareness and willingness with SVI, adjusting for pharmacy type, neighborhood location, and local chlamydia rates. Among surveyed pharmacies, 40% (134/335) of pharmacists were aware of EPT, and only 30% (100/333) were willing to fill nameless prescriptions. Non-chain pharmacies were significantly less likely to be aware of EPT compared to national chains (34% vs. 54%, p=0.02). The most common dispensing approach was filling prescriptions under the index patient's name (34%, 114/335), with most pharmacies (86%, 179/208) accepting insurance. Only 30% (100/333) of pharmacists were willing to dispense nameless EPT prescriptions. Most pharmacists' methods of dispensing EPT prescriptions did not follow NY state EPT guidelines (54%, n=113). The most frequent reasons for refusal included unfamiliarity with EPT (62%, 66/107) and the incorrect belief that patient names were legally required (28%, 30/107). Adjusted regression showed increased odds of awareness of EPT in areas with the highest socioeconomic SVI quartile compared to the lowest quartile (odds ratio 3.7; 95% CI 1.4-10.8), though willingness to fill prescriptions did not differ by SVI (p=0.35). Despite higher pharmacist awareness of EPT in more socioeconomically vulnerable areas, willingness to dispense nameless prescriptions remains low across NYC pharmacies. Independent pharmacies demonstrated particularly low awareness and engagement with EPT. These findings underscore the need for targeted pharmacist education, system-level interventions to streamline EPT dispensing, and enhanced training to ensure guideline adherence, particularly in high-need areas. Addressing these barriers could reduce STI disparities and improve public health outcomes.

АРТЕРИИТ ТАКАЯСУ: КЛИНИЧЕСКИЙ СЛУЧАЙ РАННЕЙ ДИАГНОСТИКИ В ОБЩЕЙ ВРАЧЕБНОЙ ПРАКТИКЕ

Summary. We describe a clinical case of asymptomatic Takayasu arteritis, which was timely suspected and diagnosed in an outpatient setting, in a 29-year-old female patient with anemic syndrome and accelerated ESR syndrome. The diagnostic examination revealed the absence of pulse in the left upper limb, a difference of more than 10 mm Hg in blood pressure measured on the right and left arms, stenosis of more than 80% of the first segment of the left subclavian artery according to duplex scanning of the extracranial sections of the brachiocephalic arteries, signs of Takayasu arteritis as revealed by computed tomography. The patient was hospitalized in the rheumatology department of the Grodno University Clinic with further confirmation of the diagnosis and prescription of immunosuppressive, anti-inflammatory and antiplatelet therapy.

Identifying Cladribine prescription pattern in MS: an Italian multicentre study.

Characterizing Cladribine tablets prescription pattern in daily clinical practice is crucial for optimizing multiple sclerosis (MS) treatment. To describe efficacy, safety profile and new disease-modifying therapy (DMT) prescriptions following Cladribine treatment. Independent retrospective cohort study in patients followed at six Italian MS centres. Patients diagnosed with relapsing MS (RMS) according to 2017 McDonald criteria, who initiated Cladribine between January 2019 and May 2023, were included. A generalized linear regression model was built for the outcome DMT after Cladribine course. Heatmaps were generated based on weighted pivot tables to visualize the proportion of patients requiring DMT post-Cladribine. A total cohort of 352 patients was enrolled, 134 naïve to any DMT, 218 switchers from other DMTs. The last DMT was an injectable first-line DMT for 48 (22%) patients, oral first-line DMT for 141 (64.7%) patients, SP1 inhibitor-Fingolimod for 23 (10.6%) patients, and Natalizumab for 6 (2.7%) patients. Overall, Cladribine was efficacious and well tolerated, 12% of patients required a new DMT prescription after a median time of 24 months. The regression model revealed that patients aged >40 years at Cladribine prescription had a 16% decrease in likelihood of receiving a new DMT. Heatmaps showed patients previously on Fingolimod had a lower rate (72.2%) of being free from therapy after Cladribine. In our multicentric real-world Italian study, Cladribine therapy is generally effective during the investigated follow-up period. Understanding key characteristics of patients responding best to Cladribine can help tailor therapeutic strategies for optimal outcomes.

Quality Assessment of a Medical Prescription for Antibiotic Therapy

Objective: Our study aimed to assess the quality of antibiotic prescriptions and identify potential opportunities for improvement in antibiotic use in our setting. Methodology: This is how we conducted a prospective cross-sectional descriptive study which concerned 404 prescriptions collected in the pharmacy of the HGR mwangeji Results: The most prescribed antibiotic families were beta lactams with 60.15%, followed by imidazoles with 11.88% and fluoroquinolones or 8.1%; they were more prescribed in INN with 70% in the following galenic forms: tablets 46.8% followed by bottles 17.1% and finally suspensions with 12.6% with an unspecified route of administration in 80.9%. The dosage, dose, total quantity and duration of treatment were specified respectively as follows: 86.9%; 62.4%; 98.3% and 80.4%. Most of the prescriptions bore the name and first name of the patients 82.9%; The age of the patient was indicated at 75.2%; Almost all of the prescriptions did not have the weight of the patient indicated (97%); Almost all of them bore the sex of the patient (97.6%); Not all prescribers wore the patient's size (100%). In 84.4% of cases, prescribers had indicated their name on the prescriptions; A large number of prescription slips did not bear the prescriber status 86.6%; Almost all prescribers had indicated their place of practice 99.5%; 97% of prescribers had not indicated their contact details on the prescriptions; The minority 18.6% of prescribers had not signed on the prescriptions; The order number is shown on the majority of prescription slips 83.7%. Conclusion: The results of this study remain relevant. They will help improve training programs for health workers by establishing interactive information for practitioners, through visits and intensive supervision by more experienced managers in the hierarchy, this would be likely to optimize the quality of practitioners' prescriptions ; so that they better master the principles of prescribing antibiotics . Keywords: Prescription, Antibiotics, evaluation, quality, medical, antibiotic therapy

Benchmarks for low back pain in general practice in Flanders: electronic audit of INTEGO

BackgroundLow back pain (LBP) is one of the most frequent reasons for encounter in general practice. Yet results from literature show adherence to clinical practice guidelines is low. Audit & feedback is a well-known strategy to improve adherence to guidelines. Benchmarking is an important step in the audit & feedback process. The objective of this study was to develop data-derived benchmarks for low back pain quality indicators.MethodsFour electronic health record extractable quality indicators were selected from an existing indicator set developed by CEBAM, an independent, multidisciplinary and interuniversity medical scientific institute in Belgium. Data from 2021-2022 from INTEGO, a general practice morbidity registry, were used to calculate benchmarks for the four quality indicators. The Achievable Benchmark of Care methodology was used to create benchmarks based on the performance of the 10% best-performing practices.ResultsThe following benchmarks were derived: 4.2% prescription for medical imaging, 12.7% prescription for opioids, 27.2% for prescription for non-steroidal anti-inflammatory drugs or acetaminophen, 37.7% prescription for physical therapy and 11.9% prescription for work absenteeism.ConclusionsBenchmarks for four electronic health record-extractable quality indicators have been established. They can be used for an electronic audit & feedback tool in primary practice in Flanders or other quality improvement initiatives.

Health Disparities and Hormone Therapy Prescribing for Early, Peri-, and Postmenopausal Women: A Scoping Review.

Menopause hormone therapy has improved the quality of life for perimenopausal and early postmenopausal women, yet women may not be prescribed or use this treatment option. The purpose of this study was to conduct a scoping review to assess whether health disparities existed in hormone therapy prescription rates and use for peri- and postmenopausal care based on demographic and clinical characteristics in real-world, observational studies conducted in the USA. A chief librarian conducted a search strategy from 1940 to 10/31/2023 in five bibliographic databases. One hundred eighty-four articles were found through database and handsearching. After removing duplicates and excluding articles that did not meet study criteria, 14 articles were included in this review. Data were independently extracted and charted using a data extraction form, which was developed based on the study aims. Reviewers met to confirm agreement and discuss disagreements. Sixteen health disparities, in menopause hormone therapy prescribing or use, were found in this scoping review. Differences between ethnic groups were found in 9 of 14 articles. Education level, menopause route, medical conditions, and practitioner specialization werein 4 of the 14 articles. The remaining 11 health disparities were categorized under chronic medical conditions and social constructs. Health disparities have existed for decades in menopause hormone therapy prescribing and use for peri- and postmenopausal women. Identification of health disparities for underserved groups can help reshape educational initiatives for practitioners in all relevant disciplines.

Prediction of radiological decision errors from longitudinal analysis of gaze and image features.

Medical imaging, particularly radiography, is an indispensable part of diagnosing many chest diseases. Final diagnoses are made by radiologists based on images, but the decision-making process is always associated with a risk of incorrect interpretation. Incorrectly interpreted data can lead to delays in treatment, a prescription of inappropriate therapy, or even a completely missed diagnosis. In this context, our study aims to determine whether it is possible to predict diagnostic errors made by radiologists using eye-tracking technology. For this purpose, we asked 4 radiologists with different levels of experience to analyze 1000 images covering a wide range of chest diseases. Using eye-tracking data, we calculated the radiologists' gaze fixation points and generated feature vectors based on this data to describe the radiologists' gaze behavior during image analysis. Additionally, we emulated the process of revealing the read images following radiologists' gaze data to create a more comprehensive picture of their analysis. Then we applied a recurrent neural network to predict diagnostic errors. Our results showed a 0.7755 ROC AUC score, demonstrating a significant potential for this approach in enhancing the accuracy of diagnostic error recognition.

Association of testosterone replacement therapy with atrial fibrillation and acute kidney injury.

Secondary analyses of the Testosterone Replacement Therapy for Assessment of Long-Term Vascular Events and Efficacy Response in Hypogonadal Men (TRAVERSE) trial revealed significantly higher rates of new-onset atrial fibrillation (AF) and acute kidney injury (AKI) in the testosterone replacement therapy (TRT) cohort. To validate the secondary findings of the TRAVERSE trial. We utilized the TriNetX Research Network to identify a cohort of men ages 45-80years old who met similar inclusion criteria to the TRAVERSE trial. We compared hypogonadal men (testosterone 100-300ng/dL) who had a prescription for topical testosterone therapy and men who did not. Propensity score matching was used to match patient populations. Kaplan Meier survival analysis was used to determine the relative risk of new-onset AF and AKI within 3years. New-onset AF and AKI within 3years. There were 2134 men included in each cohort after propensity score matching. Men on TRT had significantly lower testosterone (T) at the time of diagnosis compared to men not prescribed TRT (207 ± 66ng/dL vs 246 ± 140ng/dL, P< 0.001). Kaplan-Meier survival analysis showed a significantly increased risk of AKI among men on TRT (RR 1.53, 95% CI 1.07-2.18). However, TRT was not associated with a significantly increased risk of new-onset AF (RR 1.48, 95% CI 0.93-2.37). Hypogonadal men with underlying cardiovascular risk factors or pre-existing cardiovascular disease who receive TRT may be at increased risk of AKI after starting therapy. We evaluated a large global research database and utilized similar inclusion and exclusion to the TRAVERSE trial. However, our results are limited by the retrospective study design and reliance on documented claims data. Similar to the TRAVERSE trial, our study demonstrated an increased risk of AKI among men on TRT, but did not find increased risk of AF. However, further studies are required to validate these results.

Barriers to prescription of hormonal contraception and hormone replacement therapy in gynecological cancer survivors: Results of a survey and literature review.

The incidence of gynecological cancers in premenopausal women is increasing, highlighting issues related to Hormonal Contraception (HC) and Hormone Replacement Therapy (HRT). However, the presence of hormonal receptors in many gynecological cancers complicates HC and HRT prescriptions. To identify barriers experienced by gynecologists in prescribing HC and HRT to gynecological cancer survivors, with a secondary objective of conducting a literature review on the safety of these prescriptions. A nationwide survey was conducted among Portuguese gynecologists, including questions about their prescribing practices for HC and HRT in gynecological cancer survivors. For the narrative review, the authors searched MEDLINE, Embase, Cochrane Central Register of Controlled Trials, and SCOPUS from January 2019 to April 2024. The included studies encompassed gynecological cancer survivors using HC or HRT, detailing tumor histologic type and clinical outcomes. 185 gynecologists participated in the questionnaire: 151 general gynecologists (81.6 %) and 34 oncology gynecologists (18.4 %). Of these, 49.7 % and 55.1 % had prescribed HC and HRT, respectively. Cervical cancer had the highest prescription rate, followed by vulvar and vaginal cancer, with fewer prescriptions for ovarian/fallopian tube, endometrial, and uterine corpus (non-endometrial) cancers. Older age and specialization in gynecologic oncology significantly predicted HC and HRT prescriptions (p < 0.05). Uncertainty was the main reason for not prescribing HC/HRT. A narrative review confirmed the safety of prescribing for specific tumor subtypes. The survey findings highlight an occasionally unfounded apprehension regarding the use of HC and HRT among gynecological cancer survivors. This underscores the crucial need for enhanced education on these matters.

Implementation of a Guidelines-Based Lipid Reporting and Rates of Lipid Lowering Therapy Prescription

Implementation of a Guidelines-Based Lipid Reporting and Rates of Lipid Lowering Therapy Prescription

Trends and disparities in antiretroviral therapy prescription rates among US Medicare beneficiaries with HIV.

Our objective was to evaluate antiretroviral therapy (ART) prescription rates over time among US Medicare enrolees with HIV and to describe disparities in ART prescription and associated factors. We constructed successive cross-sectional cohorts including adult enrollees with HIV and fee-for-service coverage and Part D enrolment in US Medicare between 2007 and 2019. We calculated the percentage of receipt of any ART prescription (ART%) in a calendar year by sex, age, and original Medicare entitlement and calculated age-sex standardized ART% over time. We used multivariable logistic regression to assess the association between ART prescription and sociodemographic factors and chronic conditions by age strata (<65, ≥65 years) in 2019. ART% increased over time and was highest among people with HIV aged 50-64 years in 2019: 95% in males and 92% in females. Multivariable analysis showed that female sex was associated with less ART%, with odds ratios (ORs) of 0.65 (95% confidence interval [CI] 0.60-0.70) and 0.34 (95% CI 0.30-0.39), than male sex in those aged <65 and ≥65 years, respectively. The youngest and oldest enrollees had lower ART use (e.g., OR 0.43 [95% CI 0.34-0.54] for 18-29 vs. 50-64 years; OR 0.34 [95% CI 0.30-0.39] for ≥80 vs. 65-69 years). The top conditions associated with less ART included dementia and alcohol use disorder. Other factors included no Part D low-income subsidy, non-Hispanic white race, and Midwest residence. ART use increased over time in US Medicare enrollees. Non-Hispanic white, female, and the youngest and oldest enrollees received less ART. Multimorbidity, substance use, and dementia were associated with less ART use. Research to overcome these disparities is needed.

Amyloid cardiomyopathy: сlinical significance and diagnostic value of magnetic resonance imaging

The incidence and prevalence of amyloid cardiomyopathy (AC) has been increasing in recent years, and this diagnosis is often detected at the final stage. This determines late prescription of specific therapy and worsening prognosis of patients. Unfortunately, doctors are not sufficiently informed about the pathogenesis of AC and diagnostic methods. This circumstance leads to prolongation of time from the manifestation of the disease to the diagnosis. Despite the presence of a large number of AC masks, a number of specific clinical diagnostic markers called «red flags» helps to suspect AC. In addition, the development of diagnostic algorithms using non-invasive imaging methods can help not only in diagnosing AC at an early stage of the disease, but also in determining the type of amyloidosis, that determines the success of timely administration of disease-modifying therapy. The aim of this review is to analyze the possibility of using MRI criteria to attempt non-invasive typing of amyloidosis and to differentiate ATTR and AL AС.

Probability of Starting Two-Drug Regimen (2DR) vs. Three-Drug Regimen (3DR) in ART-Naïve and ART-Experienced Person with HIV (PWH) Across the First Wave of COVID-19 Pandemic.

Background: This study examined the impact of the COVID-19 lockdown on antiretroviral therapy (ART) prescriptions among persons living with HIV (PWH) in Italy. Methods: Data from the ICONA cohort included ART-naïve individuals who started ART between January 2019 and December 2022, and ART-experienced individuals who started new ART with HIV RNA ≤50 cps/mL from January 2016 to December 2022. The analysis focused on the proportion of PWH starting or switching to dual (2DR) versus triple (3DR) ART regimens. Comparisons were made using Chi-square and Kruskal-Wallis tests, with logistic regression (LR) to assess associations, adjusting for sex and age. Results: Among 2481 ART-naïve PWH, 17% were female, with a median age of 40. Using 2020 as the comparator (the lockdown year), the odds ratio (OR) from fitting a LR showed a reduced probability of prescribing 2DR both before and after 2020. The proportion of PWH starting 2DR was 9% in 2019, 18% in 2020, 13% in 2021, and 10% in 2022. Among 12,335 ART-experienced PWH, 20% were female, with a median age of 47. The proportion switching to 2DR rose from 24% in 2016 to 38% in 2020, 62% in 2021, and 65% in 2022, showing a >3-fold higher probability to be switched to 2DR instead of 3DR in recent years (2021-2022). Conclusions: For ART-naive PWH, 2DR initiation did not decrease during the 2020 lockdown but changed in the following years, possibly indicating shifts in clinical practice or resuming HIV services. For ART-experienced PWH, 2DR prescriptions increased significantly over time, especially for INSTI-based regimens.

Participation in Tobacco Cessation Programs Among Medicaid Managed Care Enrollees in Florida.

Background/Objectives: Tobacco use remains a significant public health issue, particularly among individuals with low incomes, including Medicaid recipients who often face multiple barriers to quitting. This study aimed to identify barriers, from the perspective of Medicaid managed care organizations (MCOs), influencing Medicaid recipient participation in tobacco cessation programs. Methods: Focus group interviews were conducted with Florida Medicaid MCOs to elicit processes for case identification, outreach, referral, program participation, and incentives. Answers were synthesized into themes. Results: Medicaid recipients were primarily identified through nicotine dependency claim codes or Health Risk Assessments (HRAs). Individuals were referred to state and local community tobacco cessation programs through text messaging and outreach by MCO case managers. The MCOs identified the following as barriers: primary care physicians (PCPs) with limited knowledge about cessation programs and pharmacologic treatments for nicotine dependence, low availability of health coaches, long wait times for entry into cessation programs, weak coordination between MCOs and cessation programs, and insufficient incentives for individuals for program participation. Suggested strategies to overcome barriers were continuing medical education (CME) for PCPs about tobacco cessation programs and prescription therapies, increasing the training of health coaches, more investment in quitlines, increasing data sharing between MCOs and cessation programs, and increasing incentives for individuals. Conclusions: These findings highlight the importance of engaging MCOs in discussions about policy and program improvements, as their insights can drive meaningful changes in how tobacco cessation and other preventive health programs are structured and implemented. Targeted interventions are needed to enhance tobacco cessation program participation among Medicaid recipients.

Hypertension in Adults With Diabetes in Southeast Asia: A Systematic Review.

Diabetes is one of the most pressing health issues in the Southeast Asian region, and hypertension has been commonly reported as a comorbidity in adults with diabetes. This systematic review aimed to synthesize evidence on theprevalence and management of hypertension in adults with diabetes in Southeast Asian countries. A literature search was conducted in Ovid MEDLINE and Embase Classic + Embase from database inception until March 15, 2024. Studies were included if (1) they were conducted in Southeast Asian countries, (2) the study populations were adults with diabetes, and (3) there was information related to hypertension or blood pressure (BP) in the study results. Of the 7486 abstracts found, 90 studies qualified for this review. Most studies reported a hypertension prevalence of 70% or higher (ranging from 29.4% to 93.4%). Despite this high prevalence, a substantial proportion of these populations did not receive adequate BP control, with most studies indicating a control rate of less than 40%. There was limited evidence on the prescription of antihypertensive therapies and medication adherence. There was a lack of studies from 4 of the 11 countries in the region. This review highlights that BP control in adults with diabetes remains a significant challenge in Southeast Asia. Given the ongoing epidemiological transition, and the increasing older population in this region who are likely to accumulate multiple chronic conditions complicating medication strategies, this review highlights the urgent need to improve BP management in those with diabetes.

Lebrikizumab (Ebglyss)

Canada’s Drug Agency (CDA-AMC) recommends that Ebglyss should not be reimbursed by public drug plans for the treatment of moderate to severe atopic dermatitis (AD) in adults and adolescents aged 12 years and older with a body weight of at least 40 kg, whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. Evidence from 3 clinical trials demonstrated that, in the short-term, Ebglyss treatment improved the severity of AD and reduced itch symptoms compared with placebo in adults and adolescents with moderate to severe AD. However, based on the evidence reviewed in the initial meeting and the reconsideration meeting, the Canadian Drug Expert Committee (CDEC) could not determine whether lebrikizumab would address the unmet needs of patients because of the uncertainty around the benefit of lebrikizumab versus appropriate comparators and in patients who received prior dupilumab or Janus kinase (JAK) inhibitor treatment. No evidence was submitted that directly compared Ebglyss to currently available treatments for AD. The indirect evidence submitted had limitations that impacted the certainty of the evidence, and it was unclear if the estimates were valid. The safety of Ebglyss relative to other treatments for AD is unknown because no comparative evidence was submitted. In addition, longer-term safety and efficacy was uncertain because of limitations of the study designs and analysis with the available evidence.

Prolonged catamnesis of a child with type I mucopolysaccharidosis, Gurler syndrome, receiving enzyme replacement therapy

Hereditary pathology makes up a significant part of the structure of childhood morbidity, disability and mortality. Mucopolysaccharidosis type I is a hereditary lysosomal accumulation disease, with an autosomal recessive type of inheritance. At birth, children with Gurler syndrome look normal, but in the future, they develop symptoms characteristic of mucopolysaccharidosis, and the first clinical manifestations of the disease are often hernias and hepatosplenomegaly. The presented clinical case demonstrates the difficulties of early diagnosis of mucopolysaccharidosis type I, which lead to a later prescription of enzyme replacement therapy and the inability to use a more effective method of therapy – hematopoietic stem cell transplantation, due to the development of serious manifestations of the cardiovascular system. While the positive effects of enzyme replacement therapy in the early stages of treatment may seem promising, it does not guarantee a favorable prognosis for the future.Conclusion. Currently, early diagnosis of mucopolysaccharidosis type I is difficult due to the lack of early manifestations. Often, the progression of clinical manifestations leads to disability, and in severe cases, to death. Therefore, it is important to have early diagnosis and appropriate treatment.

Abstract 4121624: Ambulatory Inotropic Support in Patients with Stage D Heart Failure Does Not Increase ICD Therapies

Background: Ambulatory inotropic infusions can be used as a palliative therapy (PT) or bridge to advanced therapies in end-stage heart failure (ESHF) patients, many of whom have implantable cardioverter defibrillators (ICDs). Research Questions: How often do patients receive ICD therapies after initiation of outpatient inotropic support and how often is defibrillation inactivated? Methods: We conducted a retrospective review of all patients discharged from an academic tertiary medical center with a new prescription for inotropic therapy between November 2017 and March 2023. Clinical data, Palliative Care consultation, and ICD interrogations before and after inotrope initiation were extracted from the electronic health record. Results: The cohort comprised 45 patients discharged on new inotropic support, including 22 (49%) with intent of PT and 23 (51%) as a bridge to advanced therapies (Figure). Among PT patients, 15 (68%) had an ICD in place with only 4 having ICD therapies deactivated at discharge. Only 2 additional PT patients had a documented discussion regarding ongoing ICD therapies. By 1 year, 4 (18%) PT patients were transitioned to hospice and 13 (59%) had died, with only 1 additional patient having ICD deactivation. Of 28 patients discharged on inotropic infusions with ICD therapies on (both PT and bridge patients), only 4 patients had ICD therapies turned off later and no ICD shocks were reported for the year following initiation of inotropic support. Conclusion: In a series of 45 patients initiated on inotropic support for PT or bridge to advanced therapies, the majority (77%) had an active ICD, but therapies were rarely delivered. Notably, there were very few documented discussions regarding ICD therapies in PT patients even with specialty Palliative Care consultation. In patients with ESHF initiated on inotropic support as PT, most patients chose to leave ICD therapies in place. For those patients, there was a low rate of ICD therapies delivered.