Standard Of Care For Patients Research Articles

Photodynamic therapy of Bowen’s disease

Bowen’s disease is a form of non-invasive (in situ) squamous cell skin cancer localized in the non-genital area. Russian and European clinical guidelines include photodynamic therapy (PDT) in the standard of care for patients with Bowen’s disease. In the present review, the efficacy and safety profile of different PDT regimens for Bowen’s disease are analyzed according to the available literature data. PDT can be used to treat large Bowen’s disease lesions localized in areas of the body characterized by severe healing and in cases where surgery is not feasible. Analysis of the results of studies shows that PDT is superior in efficacy and cosmetic results to traditional local treatments such as 5-fluorouracil or cryotherapy. In all analyzed studies in patients with Bowen’s disease, PDT achieved a significant clinical effect. Complete regression of pathologic foci was achieved in 67-100% of patients. In studies evaluating the recurrence rate of Bowen’s disease after PDT, this value ranged from 2-28% with the range of 6-18% in most of the studies. Most often, 5-aminolevulinic acid and its methyl ester are used for PDT in Bowen’s disease. According to some researchers, aminolevulinic acid allows to achieve a more long-term positive clinical effect with a lower incidence of painful reactions during treatment

Multicenter Registry of Adenomas of the Pituitary and Related Disorders: Initial Description of Cushing Disease Cohort, Surgical Outcomes, and Surgeon Characteristics.

To address the lack of a multicenter pituitary surgery research consortium in the United States, we established the Registry of Adenomas of the Pituitary and Related Disorders (RAPID). The goals of RAPID are to examine surgical outcomes, improve patient care, disseminate best practices, and facilitate multicenter surgery research at scale. Our initial focus is Cushing disease (CD). This study aims to describe the current RAPID patient cohort, explore surgical outcomes, and lay the foundation for future studies addressing the limitations of previous studies. Prospectively and retrospectively obtained data from participating sites were aggregated using a cloud-based registry and analyzed retrospectively. Standard preoperative variables and outcome measures included length of stay, unplanned readmission, and remission. By July 2023, 528 patients with CD had been treated by 26 neurosurgeons with varying levels of experience at 9 academic pituitary centers. No surgeon treated more than 81 of 528 (15.3%) patients. The mean ± SD patient age was 43.8 ± 13.9 years, and most patients were female (82.2%, 433/527). The mean tumor diameter was 0.8 ± 2.7 cm. Most patients (76.6%, 354/462) had no prior treatment. The most common pathology was corticotroph tumor (76.8%, 381/496). The mean length of stay was 3.8 ± 2.5 days. The most common discharge destination was home (97.2%, 513/528). Two patients (0.4%, 2/528) died perioperatively. A total of 57 patients (11.0%, 57/519) required an unplanned hospital readmission within 90 days of surgery. The median actuarial disease-free survival after index surgery was 8.5 years. This study examined an evolving multicenter collaboration on patient outcomes after surgery for CD. Our results provide novel insights on surgical outcomes not possible in prior single-center studies or with national administrative data sets. This collaboration will power future studies to better advance the standard of care for patients with CD.

The Importance of Early Diagnosis and Intervention in Chronic Kidney Disease: Calls-to-Action from Nephrologists Based Mainly in Central/Eastern Europe.

Chronic kidney disease (CKD) has a global prevalence of 9.1-13.4%. Comorbidities are abundant and may cause and affect CKD. Cardiovascular disease strongly correlates with CKD, increasing the burden of both diseases. As a group of 15 clinical nephrologists primarily practicing in 12 Central/Eastern European countries, as well as Israel and Kazakhstan, herein we review the significant unmet needs for patients with CKD and recommend several key calls-to-action. Early diagnosis and treatment are imperative to ensure optimal outcomes for patients with CKD, with the potential to greatly reduce both morbidity and mortality. Lack of awareness of CKD, substandard indicators of kidney function, suboptimal screening rates, and geographical disparities in reimbursement often hamper access to effective care. Our key calls-to-action to address these unmet needs, thus improving the standard of care for patients with CKD, are the following: increase disease awareness, such as through education; encourage provision of financial support for patients; develop screening algorithms; revisit primary care physician referral practices; and create epidemiological databases that rectify the paucity of data on early-stage disease. By focusing attention on early detection, diagnosis, and treatment of high-risk and early-stage CKD populations, we aim to reduce the burdens, progression, and mortality of CKD.

Immunotherapy and Radiation Therapy Combinatorial Approaches in Hepatocellular Carcinoma.

Hepatocellular carcinoma (HCC), a prevalent and often fatal liver cancer, presents significant treatment challenges, especially in its advanced stages. This article delves into the promising approach of combining immunotherapy, particularly immune checkpoint inhibitors, with radiation therapy, a cornerstone of HCC management. Our review synthesizes current preclinical and clinical research, highlighting the potential synergistic effects of this combinational treatment. Emerging evidence suggests that this synergy enhances tumor control and improves patient survival rates. The combination leverages the localized, tumor-targeting ability of radiation therapy and the systemic, immune-boosting effects of immunotherapy, potentially overcoming the limitations inherent in each treatment modality when used separately. This integrative approach is especially promising in addressing the complex tumor microenvironment of HCC. However, the treatment landscape is nuanced, with challenges such as patient-specific response variability and potential resistance to therapies. Future research directions should focus on refining these combination strategies, tailoring them to individual patient profiles, and understanding the underlying mechanisms that govern the interaction between immunotherapy and radiation therapy. Such advancements could significantly improve HCC management, setting new standards for patient care and treatment efficacy.

Current Status and Future Directions of Image-Guided Adaptive Brachytherapy for Locally Advanced Cervical Cancer.

The standard of care for patients with locally advanced cervical cancer is definitive chemoradiation followed by a brachytherapy boost. This review describes the current status and future directions of image-guided adaptive brachytherapy for locally advanced cervical cancer. A systematic search of the PubMed and Clinicaltrials.gov databases was performed, focusing on studies published within the last 10 years. The search queried "cervical cancer [AND] image-guided brachytherapy [OR] magnetic resonance imaging (MRI) [OR] adaptive brachytherapy". The retroEMBRACE and EMBRACE-I trials have established the use of MRI as the standard imaging modality for brachytherapy application and planning. Quantitative imaging and radiomics have the potential to improve outcomes, with three ongoing prospective studies examining the use of radiomics to further risk-stratify patients and personalize brachytherapy. Another active area of investigation includes utilizing the superior soft tissue contrast provided by MRI to increase the dose per fraction and decrease the number of fractions needed for brachytherapy, with several retrospective studies demonstrating the safety and feasibility of three-fraction courses. For developing countries with limited access to MRI, trans-rectal ultrasound (TRUS) appears to be an effective alternative, with several retrospective studies demonstrating improved target delineation with the use of TRUS in conjunction with CT guidance. Further investigation is needed to continue improving outcomes for patients with locally advanced cervical cancer treated with image-guided brachytherapy.

Invited commentary on “The impact of bleomycin deficit on survival in Hodgkin's lymphoma patients: A retrospective study”

The article “The impact of bleomycin deficit on survival in Hodgkin's lymphoma patients: A retrospective study” have presented the experience of AVD chemotherapy regimen in newly diagnosed Hodgkin's lymphoma (HL) in a single center in Brazil. Though being a small retrospective study, results from this study have provided the medical community a real-world data on HL in Brazil. ABVD has remained the standard of care for patients of newly diagnosed HL both in early and advance stages. Newer targeted molecules have also come for use in novel combinations with existing drugs. However, in a situation of temporary scarcity of bleomycin due to lack of supply during 2017 in Brazil led to use of incomplete ABVD regimen without bleomycin, i.e. AVD for HL. However, Soldi et al. utilized the opportunity to retrospectively study if the omission of bleomycin leads to subnormal treatment or unwarranted effects.

Radiotherapy Combined with a Radiosensitizer for Bacillus Calmette-Guérin–unresponsive Non–muscle-invasive Carcinoma In Situ Bladder Cancer: An Open-label, Single-arm, Multicenter, Phase 2 European Organisation for Research and Treatment of Cancer Trial

Radical cystectomy with pelvic lymph node dissection and urinary diversion is the standard of care for patients with bacillus Calmette-Guérin (BCG)-unresponsive non–muscle-invasive bladder cancer (NMIBC). However, many patients are unwilling or unable to undergo such major surgery associated with high morbidity and a negative impact on quality of life. Chemoradiotherapy is an established treatment option for muscle-invasive bladder cancer. However, it has not been investigated adequately in NMIBC until now. The European Organisation for Research and Treatment of Cancer (EORTC) 2235 study (NCT06310369) is designed as a multicenter, prospective, international, phase 2 trial of moderate hypofractionated radiotherapy combined with a radiosensitizer in BCG-unresponsive NMIBC patients with carcinoma in situ (CIS) who are not eligible for or declined to undergo radical cystectomy. Patients who have received nadofaragene firadenovec or TAR-200 are eligible. The primary endpoint is the 6-mo complete response (CR) rate defined by the absence of CIS proven by a control biopsy of the bladder. The secondary endpoints include overall survival, progression-free survival, durability of CR, grade 3–4 adverse events rate, patients’ quality of life, and organ preservation rate. Patient summaryIntravesical instillation of bacillus Calmette-Guérin is the standard treatment of non–muscle-invasive, also coined as superficial, bladder cancer. In case the cancer recurs, even superficially, there is no other proven treatment than a radical cystectomy—the surgical removal of the bladder. Although the surgical technique has improved dramatically over the past few years, it remains contraindicated in patients with severe comorbidities. In addition, because it affects the quality of life, patients may reject this option. This study will assess the efficacy of external beam radiotherapy, a robust alternative to surgery in muscle-invasive bladder cancer. Radiotherapy will be administered 5 d a week for 4 wk. It will be associated with a “radiosensitizer,” an intravenous or oral drug, during the radiotherapy treatment. The study will measure the proportion of patients remaining recurrence free at 6 mo and thereafter. It will also evaluate the safety of the treatment and its impact on quality of life.

Prolonged interval to surgery following neoadjuvant chemoradiotherapy in locally advanced rectal cancer: A meta-analysis of randomized controlled trials

BackgroundLong-course neoadjuvant chemoradiotherapy (NCRT), followed by surgery after an interval of 6–8 weeks, represents standard of care for patients with locally advanced rectal cancer (LARC). Increasing this interval may improve rates of complete pathological response (pCR) and tumour downstaging. We performed a meta-analysis comparing standard (SI, within 8 weeks) versus longer (LI, after 8 weeks) interval from NCRT to surgery. MethodsPubMed, Embase, and Cochrane databases were searched up to 31 August 2022. Randomized controlled trials (RCTs) comparing SI with LI after NCRT for LARC were included. The primary endpoint was pCR rate. Secondary endpoints included rates of R0 resection, circumferential resection margin positivity (+CRM), TME completeness, lymph node yield (LNY), operative duration, tumour downstaging (TD), sphincter preservation, mortality, postoperative complications, surgical site infection (SSI) and anastomotic leak (AL). Random effects models were used to calculate pooled effect size estimates. ResultsFour RCTs encompassing 867 patients were included. There were 539 males (62.1%). LI was associated with a higher pCR rate (OR 0.61, 95%CI ​= ​0.39–0.95, p ​= ​0.03), and more TD (OR 0.60, 95%CI ​= ​0.37–0.97, p ​= ​0.04) compared to SI. However, there was no difference in rates of R0 resection (p ​= ​0.87), +CRM (p ​= ​0.66), sphincter preservation (p ​= ​0.26), incomplete TME (p ​= ​0.49), LNY (p ​= ​0.55), SSI (p ​= ​0.33), AL (p ​= ​0.20), operative duration (p ​= ​0.07), mortality (p ​= ​0.89) or any surgical complication (p ​= ​0.91). ConclusionsA LI to surgery after NCRT for LARC increases pCR and TD rates. Local recurrence or survival were not assessed due to unavailable data. We recommend deferring TME until after an interval of 8 weeks following completion of NCRT.

Combination tumor-treating fields treatment for patients with metastatic non-small cell lung cancer: A cost-effectiveness analysis.

Tumor-treating field (TTFields) was a novel antitumor therapy that provided significant survival for previously treated metastatic non-small cell lung cancer (mNSCLC). The consistency of the cost of the new treatment regimen with its efficacy was the main objective of the study. The primary parameters, derived from the Phase 3 LUNAR study, were collected to evaluate the cost and efficacy of TTFields plus standard-of-care (SOC) (immune checkpoint inhibitors [ICIs] and docetaxel [DTX]) or SOC in patients with mNSCLC by establishing a three-state Markov model over a 15-year time horizon. Primary outcome measures for this study included costs, life-years (LYs), quality-adjusted LYs (QALYs), and incremental cost-effectiveness ratios (ICERs). Sensitivity analyses were performed. The total costs of TTFields plus SOC, TTFields plus ICI, and TTFields plus DTX were $319,358, $338,688, and $298,477, generating 1.23 QALYs, 1.58 QALYs, and 0.89 QALYs, respectively. The ICERs of TTFields plus SOC versus SOC, TTFields plus ICI versus ICI, and TTFields plus DTX versus DTX were $613,379/QALY, $387,542/QALY, and $1,359,559/QALY, respectively. At willingness-to-pay (WTP) thresholds of $150,000/QALY, the probability of combination TTFields being cost-effective was 0%. In addition, TTFields plus SOC exhibited similar efficacy (1.12 QALYs and 1.14 QALYs) and costs ($309,822 and $312,531) in the treatment of squamous cell carcinoma (SCC) and non-squamous cell carcinoma (NSCC) populations. In the United States, TTFields plus SOC as second-line treatment was not a more cost-effective strategy for patients with mNSCLC. Of the analyzed regimens, TTFields plus ICI was associated with most significant health benefits.

How to treat cardiac dyssynchrony in heart failure with reduced ejection fraction

Abstract Cardiac resynchronization therapy (CRT) has become part of the standard of care for symptomatic patients with heart failure, reduced ejection fraction (HRrEF), and wide QRS, despite optimal medical therapy. Device technology, operator experience, and post-implant optimization on follow-up have significantly improved CRT response during the last decade. In parallel, conduction system pacing (CSP) has emerged as a new and potentially better tool for maintaining physiological ventricular activation in patients with antibradycardia pacing, as well as an alternative for dyssynchrony correction in HFrEF patients. The current review focuses on cardiac dyssynchrony pathophysiology and correction methods, clinical evidence on CRT and CSP as therapies for cardiac dyssynchrony, and the role of each according to clinical evidence and current guidelines for HFrEF treatment.

The need for standardized perioperative care for patients undergoing bariatric and metabolic surgery in the United Kingdom.

Enhanced recovery after surgery (ERAS) protocols are shown to improve patient outcomes and reduce length of hospital stay. However, there is currently limited consensus on the perioperative management of patients undergoing bariatric and metabolic surgery (BMS) in the United Kingdom. This study aims to survey the level of consistency in patient care undergoing BMS. Bariatric nurse specialists from 30 bariatric units completed an anonymised, online survey from 21 December 2022 to 21 February 2023. Most units (77%) have implemented a premade postoperative care bundle protocol including predetermined timing of oral intake (77%) and postoperative day 1 bloods (60%). 63% of units have also established pre-set analgesia and anti-emetic bundles. Date of discharge is variable, ranging from 1 day after surgery (50%) to a 'two night stay' protocol (33%) to within 4 days after surgery (17%). Most follow-up clinics are either led by dietitians (33%) or both bariatric nurse specialists and dietitians collaboratively (57%). Patients are usually established on solid food 6 weeks after surgery in 53% (16/30) units. Chemical venous thromboembolism (VTE) prophylaxis was either given on day of surgery postoperatively (60%), day before (20%) or after (17%) surgery. Our study shows significant variability of care throughout the surgical pathway, in the study population. The results suggest a need for consensus guidelines outlining the best-practice approach to managing patients undergoing BMS; due to the heterogeneity of the patient group, these guidelines should contain overarching generalisable recommendations that can then be tailored to individual patients.

Feasibility of Introducing a Prehabilitation Program into the Care of Gynecological Oncology Patients-A Single Institution Experience.

Prehabilitation is an upcoming strategy to optimize patient's functional capacity, nutritional status, and psychosocial well-being in order to reduce surgical complications and enhance recovery. This study aims to assess the feasibility of implementing a multimodal prehabilitation program into the standard care of gynecological oncology patients at an academic hospital in terms of recruitment, adherence, and safety, which were assessed by the number of patients eligible, recruitment rate, participation rate, and adherence to individual modalities. Data were derived from the F4S PREHAB trial, a single-center stepped-wedge trial implementing a multimodal prehabilitation program among various surgical specialties. All patients undergoing elective surgery as part of treatment for ovarian, uterine, and vulvar cancer at the Radboudumc, an academic hospital in The Netherlands, between May 2022 and September 2023 were considered eligible for the F4S PREHAB trial and, consequently, were included in this cohort study. The multimodal prehabilitation program comprised a physical exercise intervention, nutritional intervention, psychological intervention, and an intoxication cessation program. A total of 152 patients were eligible and approached for participation of which 111 consented to participate, resulting in a recruitment rate of 73%. Participants attended an average of six exercise sessions and adhered to 85% of possible training sessions. Respectively, 93% and 98% of participants adhered to the prescribed daily protein and vitamin suppletion. Ten participants were referred to a psychologist and completed consultations. Out of nine active smokers, two managed to quit smoking. A total of 59% adhered to alcohol cessation advice. No adverse events were reported. This study demonstrates that introducing a multimodal prehabilitation program into the standard care of gynecological oncology patients is feasible in terms of recruitment and adherence, with no serious adverse events.

ASSESSMENT OF KNOWLEDGE, ATTITUDE AND PRACTICES REGARDING BIOETHICS AMONG STUDENTS IN DENTAL INSTITUTES OF PUNJAB

This cross-sectional study aimed to evaluate the levels of knowledge, attitudes, and practices (KAP) regarding bioethics among students enrolled in dental institutes across Punjab, India. A sample size of 202 students, including those exposed to clinical settings, participated in the study. A structured questionnaire was used to assess participants understanding of bioethical principles, their attitudes towards ethical dilemmas commonly encountered in dental practice, and their adherence to ethical standards in clinical settings. The findings revealed varying levels of knowledge among students, with notable disparities in understanding specific bioethical principles. Attitudinal responses highlighted a generally positive disposition towards ethical decision-making, although some students demonstrated uncertainty in navigating complex ethical scenarios. Furthermore, the study uncovered variations in the implementation of ethical practices among students in clinical settings. These findings underscore the importance of integrating comprehensive bioethics education within dental curricula to foster ethical awareness, enhance moral reasoning skills, and promote ethical conduct among future dental practitioners. Addressing these gaps in knowledge, attitudes, and practices is crucial for the cultivation of a morally conscious dental workforce capable of upholding the highest ethical standards in patient care and professional conduct.

Limb salvage surgery for primary bone tumors: retrospective study in a tertiary care centre

Background: Limb salvage surgery is the standard of care for patients with primary bone tumors. The study retrospectively analyses a single center experience of use of endo-prosthesis for limb conservation in cases of primary bone tumors over a five-year period (2015-2020). Methods: Total of 34 histologically proven primary bone tumor were retrospectively studied which included 25 males and 9 females who received limb salvage surgery for bone tumors at department of surgical oncology, Rajiv Gandhi Government general hospital, Madras medical college, Chennai. Results: Median age at diagnosis was 23 years. Tumor localized to lower limb in 23 patients, and upper limb in 9 patients, pelvic bones in 3 cases. Tumors were malignant in 24 patients (70.4%) and benign in 10 patients (29.4%). The most common diagnosis was osteosarcoma17 patients (50%). The median resection length was 10 cm. High grade tumors (grade 2a and 2b and grade 3) was found in 14 cases (41.1%). 6 patients had prosthesis related complications. The mean follow-up was 5 years (range: 3-7). 27 (79.4%) patients of 34 were alive with the endo-prosthesis at the last follow-up. Conclusions: The custom mega prosthesis favored by us in most of the cases in limb sparing surgery for bone tumors results in satisfactory results in terms of local tumor control and limb function.

A Comprehensive Review on Chemistry Uses in Medicine and Medical Technology

Medicinal chemistry, firmly rooted in chemistry while incorporating elements of biological, medical, and pharmaceutical sciences, spearheads innovation in disease diagnosis, treatment, and prevention at the molecular level. Chemistry serves as a cornerstone for understanding the intricate interactions between medications and biological systems, guiding medicinal chemists in the precise design and synthesis of compounds tailored for specific activities and optimized therapeutic outcomes. Concurrently, nanotechnology, founded on chemical principles, presents promising avenues in advanced imaging, diagnostics, theranostic platforms, and drug delivery systems. The indispensable role of chemistry is further underscored by polymers, integral to medical applications such as orthopedics and cardiovascular devices, driving forward medical advancements. Analytical chemistry techniques play a critical role in drug analysis and biomarker identification, providing essential support for these endeavors. Additionally, radioactive metal ions play pivotal roles in therapy and diagnosis within nuclear medicine, as demonstrated by the innovative DOTA-TATE compound. These interdisciplinary advancements underscore the crucial role of chemistry in fostering innovation and elevating patient care standards within modern healthcare systems.

Assessment of Professionalism, Attitudes, and Behaviors in Practicing Physical Therapy

Background: The field of physiotherapy plays a crucial role within the healthcare landscape, particularly in the context of modern medical and surgical practices. Professional behavior, attitudes, and values are fundamental to the practice, influencing patient care and interprofessional collaboration. Recent studies have highlighted a need for continuous evaluation and improvement in these areas to enhance the overall quality of physiotherapy services. Objective: This study aimed to assess the current state of professionalism, attitudes, and behaviors among practicing physical therapists, identifying strengths and areas for development to inform future educational and professional development initiatives. Methods: A cross-sectional descriptive study was conducted among 120 physical therapists in Lahore, utilizing convenient sampling. The Professional Behavior Continuum, a self-reflective tool, was employed to evaluate professional behaviors in various clinical settings. Data were collected through questionnaires, including a consent form to ensure confidentiality. The analysis was performed using SPSS version 25, focusing on descriptive statistics such as frequency and percentage to quantify the extent of core professional values among participants. Results: The majority of participants were aged between 26-30 years (45%), predominantly male (70%), and had 1-5 years of work experience (65%). The study revealed positive perceptions of integrity (60% honesty, 65% personal responsibility), respect (65% confidentiality, 70% diversity), responsibility (75% punctual for learning, 60% cope with challenges), competence (80% responsibility for learning, 70% self-reflection), maturity (80% professional dress/language, 70% emotional stability), and communication (80% respectful, 70% empathy/compassion) among physical therapists. Conclusion: The findings indicate a generally positive outlook on professional behaviors, attitudes, and values among physical therapists, with notable strengths in areas such as responsibility, competence, and communication. However, the study also suggests the need for ongoing professional development and education to address areas of improvement and ensure the highest standards of patient care and professional practice.

Outcomes of rectal cancer patients who refuse surgery after incomplete clinical response to neoadjuvant therapy.

Total mesorectal excision (TME) remains the standard of care for patients with rectal cancer who have an incomplete response to total neoadjuvant therapy (TNT). A minority of patients will refuse curative intent resection. The aim of this study is to examine the outcomes for these patients. A retrospective cohort study of stage 1-3 rectal adenocarcinoma patients who underwent neoadjuvant chemoradiation therapy or TNT at a single institution. Patients either underwent TME, watch-and-wait protocol, or if they refused TME, were counseled and watched (RCW). Clinical outcomes and resource utilization were examined in each group. One hundred seventy-onepatients (Male 59%) were included with a median surveillance of 43 months. Twenty-ninepatients (17%) refused TME and had shortened overall survival (OS). Twelvepatients who refused TME converted to a complete clinical response (cCR) on subsequent staging with a prolonged OS. 92% of these patients had a near cCR at initial staging endoscopy. Increased physician visits and testing was utilized in RCW and WW groups. A significant portion of patients convert to cCR and have prolonged OS. Lengthening the time to declare cCR may be considered in select patients, such as those with a near cCR at initial endoscopic staging.

Taliglucerase alfa in the longterm treatment of children and adolescents with type 1 Gaucher disease: the Albanian experience.

Enzyme replacement therapy is already recognized as the gold standard of care for patients with Gaucher disease. Taliglucerase alfa is one of the three alternatives recommended for treatment of Gaucher disease in children and adults. This study aims to evaluate the long-term efficacy and safety of Taliglucerase alfa in children and adolescents with Type 1 Gaucher disease. Over a six-year period, we monitored the efficacy of continuous treatment in 10 patients by assessing various parameters, including hemoglobin concentration, platelet count, liver and spleen volume, bone mineral density, glucosylsphingosine level, chitotriosidase activity, and growth parameters. Safety was evaluated by immunogenicity and adverse event monitoring. The mean age of patients was 13.4 ± 3.6 years and the treatment duration was 60.24 ± 13.4 months. From baseline to end line the parameters change as follows: hemoglobin concentration improved from 12.7 (±1.3) to 14.6 (±1.5) and platelet count from 180 (±74) to 198 (±79). The spleen volume, was reduced by 46% (p = 0,007). The chitotriosidase activity decreased from 4,019.7 (±3,542.0) nmoles/ml/hr to 2,039.5 (±1,372.2) nmoles/ml/hr (46% reduction). Glucoylsphingosine level dropped from 119.2 (±70.4) ng/ml to 86.2 (±38.1) ng/ml, indicating a reduction of 28%. Bone mineral density Z-score, improved from -1.47 (±1.76) to -0.46 (±0.99) (69.7% reduction). Out of the 1,301 total administrations, our patients reported only 37 (2.8%) infusion-related adverse events which were mild and transitory. Taliglucerase alfa exhibits good efficacy and a safe profile in the treatment of children and adolescents with Type 1 Gaucher disease.

Cost Effectiveness of Dapagliflozin for Heart Failure Across the Spectrum of Ejection Fraction: An Economic Evaluation Based on Pooled, Individual Participant Data From the DELIVER and DAPA-HF Trials.

The sodium glucose cotransporter-2 inhibitors are guideline-recommended to treat heart failure across the spectrum of left ventricular ejection fraction; however, economic evaluations of adding sodium glucose cotransporter-2 inhibitors to standard of care in chronic heart failure across a broad left ventricular ejection fraction range are lacking. We conducted a US-based cost-effectiveness analysis of dapagliflozin added to standard of care in a chronic heart failure population using pooled, participant data from the DAPA-HF (Dapagliflozin and Prevention of Adverse Outcomes in Heart Failure) and DELIVER (Dapagliflozin Evaluation to Improve the Lives of Patients With Preserved Ejection Fraction Heart Failure) trials. The 3-state Markov model used estimates of transitional probabilities, effectiveness of dapagliflozin, and utilities from the pooled trials. Costs estimates were obtained from published sources, including published rebates in dapagliflozin cost. Adding dapagliflozin to standard of care was estimated to produce an additional 0.53 quality-adjusted life years (QALYs) compared with standard of care alone. Incremental cost effectiveness ratios were $85 554/QALY when using the publicly reported full (undiscounted) Medicare cost ($515/month) and $40 081/QALY, at a published nearly 50% rebate ($263/month). The addition of dapagliflozin to standard of care would be of at least intermediate value (<$150 000/QALY) at a cost of <$872.58/month, of high value (<$50 000/QALY) at <$317.66/month, and cost saving at <$40.25/month. Dapagliflozin was of at least intermediate value in 92% of simulations when using the full (undiscounted) Medicare list cost in probabilistic sensitivity analyses. Cost effectiveness was most sensitive to the dapagliflozin cost and the effect on cardiovascular death. The addition of dapagliflozin to standard of care in patients with heart failure across the spectrum of ejection fraction was at least of intermediate value at the undiscounted Medicare cost and may be potentially of higher value on the basis of the level of discount, rebates, and price negotiations offered. URL: https://www.clinicaltrials.gov; Unique identifiers: NCT01035255 & NCT01920711.

The management of pemphigus vulgaris and mucous membrane pemphigoid in a joint oral medicine and dermatology clinic: a five-year narrative review

Pemphigus disease and mucous membrane pemphigoid are autoimmune blistering diseases (AIBDs) which may involve both oral and extra-oral tissues. The Bristol Joint Oral Medicine and Dermatology Combined Clinic was set up in 2014, with the primary aim of improving the standard of care for patients with AIBDs. This interdisciplinary approach aimed to address the medical management challenges due to the multisite nature of these AIBDs.We present a narrative report of the clinical work undertaken within this clinic, focused on the management of this patient cohort within a five-year span (2017-2022). This report outlines the multisite nature of AIBDs and the range of topical and systemic treatments that were employed to achieve adequate disease control and optimise outcomes for patients. We reflect on the experiential benefits of this multidisciplinary clinic extended beyond immediate patient benefits to areas such as specialist training, both from a dermatologist's and oral physician's perspective.