Physician and patient perspectives on the management of hereditary angioedema: a survey on treatment burden and needs.

Hereditary angioedema (HAE) is a rare disorder caused by genetic mutations that lead to recurrent episodes of swelling in various parts of the body. Prophylactic treatment is common for patients with HAE, and the therapeutic options have expanded in recent years. The current standard of care for prophylactic HAE therapies is subcutaneous treatment, which can be self-administered at home, greatly improving patient quality of life. As new therapies emerge, it is important for patients and physicians to discuss the risks and benefits associated with each treatment to develop an individualized approach to HAE management. We conducted surveys of patients with HAE and physicians who treat patients with HAE to identify prescribing trends for prophylactic HAE treatments and the impact that such treatments has on patients. Our results confirmed that newer, subcutaneous therapies are prescribed for HAE prophylaxis more frequently than other therapies in the United States and that treatment burdens still exist for patients with HAE. We found that physicians and patients were not always aligned on how treatment choices affect patients' lives, which may mean that there are opportunities for enhanced patient-physician dialog and shared decision-making in HAE management in the United States.

Hereditary angioedema patients would prefer newer-generation oral prophylaxis.

OBJECTIVE: To explore treatment preferences of patients with Hereditary Angioedema (HAE), a debilitating disorder characterized by potentially life-threatening, recurrent episodes of swelling, resulting in significant physical, emotional, and economic burden. With newer oral prophylactic treatments on the horizon, it is important to understand patients' preferences. METHODS: An online survey was conducted in 2018 among United States (US) adult patients diagnosed with Type I or II HAE. Respondents were recruited anonymously from online panels and social media. RESULTS: Online surveys were completed by 75 patients diagnosed with HAE by a healthcare provider, with a mean of 16.7 years since diagnosis. Most patients (64%) report taking at least one medication for prophylaxis of HAE attacks. While almost all patients surveyed agree it is important to take preventative medication as prescribed, over half (52%) of patients report HAE prophylactic treatment to be burdensome. Despite stating that they like their current medications, 98% of the prophylactic HAE medication users would prefer an oral treatment if available; almost all (96%) prophylaxis users agree that oral preventative medication would fit their life better than an injectable medication, with 67% of users citing convenience as the primary reason to try an oral preventative HAE medication. If a more convenient option were available, nearly all (96%) patients currently not treating their HAE prophylactically would feel encouraged to do so. CONCLUSIONS: Most patients with HAE would prefer a newer generation oral prophylactic medication that would decrease treatment burden and allow them to live fuller lives.

Performance evaluation of Xpert HBV viral load (VL) assay: Point-of-care molecular test to strengthen and decentralize management of chronic hepatitis B (CHB) infection.

INTRODUCTION: Estimation of hepatitis B (HBV) viral load (VL) is critical in hepatitis-B cascade-of-care and at present there is no point of care (POC) molecular assay available for the same. This study evaluated the performance of a new near point of care molecular assay Xpert HBV- VL assay against the approved assays. MATERIALS & METHODS: In this study 172 archived previously tested samples, were simultaneously re-tested for HBV DNA on 3 real-time PCR assays: Abbott Real-Time HBV, Roche TaqMan® HBV and Xpert HBV assay. RESULTS: Out of 172 samples, 119 were previously positive for HBV DNA with a median VL 4.46 (IQR: 1-8.76)log10IU/mL and 53 were HBV DNA negative. Genotyping could be done in 95 (79.8 %) samples and genotype D (83; 87.3 %) was the commonest type. The Xpert assay demonstrated a good correlation with Abbott (R2 = 0.94) and Roche (R2 = 0.96). On comparison, the mean difference with 95 % Confidence Interval of Xpert assay was -0.018 and -0.043 log10 IU/mL with Abbott and Roche assay, respectively. The overall sensitivity, specificity, negative predictive value, and positive predictive value of the Xpert assay was found 97.5 %, 100 %, 94.65 & 100 % respectively. CONCLUSION: Xpert HBV-VL assay which has a potential for near point of care molecular testing has shown excellent performance and found to be a reliable method for HBV DNA quantification.

Prevalence and correlates of antenatal depression among women registered at antenatal clinic in North India.

OBJECTIVE: Women pass through many stages throughout her lifetime. Among these phases, pregnancy is crucial phase. If women are not able to cope with this stress, it may lead to adverse outcomes of pregnancy. Early detection of possible depression in pregnant women may lead to decrease in incidence of depression and adverse outcomes of pregnancy. MATERIALS AND METHODS: The present study was done in an urban primary health center of east Delhi where antenatal and postnatal services are provided. Two hundred pregnant women who attended antenatal clinic for their antenatal checkup were included in the study. Edinburgh postnatal depression scale was used to diagnose possible depression. The presence of a statistically significant difference between possibility of depression in terms of various socioeconomic, obstetric, gender issues, life events, previous psychiatric history and family relationships was ascertained using Chi-square/Fisher's exact test. Logistic regression was carried out to determine important confounding variables. RESULTS: The mean age of participants was 25.32 ± 3.86 years. Of total 200 women, 42 (21%) women were found to be suffering from possible depression. The possibility of depression was found to be significantly higher in literate participants (P = 0.001) and in women who were married after 18 years of age (P = 0.016). Participants who wanted the present pregnancy and whose spouses were alcoholic were found to be associated significantly (P = 0.00). On applying logistic regression, age and abortion history was found to be significant. CONCLUSION: This study demonstrates that the prevalence of antenatal depression is high in developing countries, and universal screening of depression during antenatal and postnatal period is feasible along with other antenatal and postnatal services provided to them.

Cost- Effectiveness of Avatrombopag for the Treatment of Thrombocytopenia in Patients with Chronic Liver Disease.

BACKGROUND AND AIM: Thrombocytopenia is common in people with chronic liver disease, who frequently undergo invasive procedures. To minimize the risk of bleeding, prophylactic platelet transfusions have traditionally been used but carry many risks. The aim of this study was to evaluate the cost-effectiveness of avatrombopag compared with platelet transfusion and lusutrombopag as a treatment for thrombocytopenia in adult patients with chronic liver disease scheduled to undergo a medical procedure. METHODS: A decision-tree model was developed from a US payer perspective to capture acute events observed in phase 3 global randomized controlled clinical trials and, to support exploratory analyses, potential longer-term complications resulting from a major bleed or thromboembolic event. Treatment costs were taken from publicly available data sources. The interventions were evaluated in the overall trial populations and in subpopulations with higher and lower baseline platelet counts. Results were presented as incremental cost per platelet transfusion avoided. One-way and probabilistic sensitivity analyses were conducted. RESULTS: In the overall population, avatrombopag reduced the need for platelet transfusions and produced cost-savings compared with platelet transfusion (80% fewer prophylactic platelet transfusions, $4250 lower costs) and lusutrombopag (42% fewer prophylactic platelet transfusions, $5819 lower costs). Similar results were seen in both the higher and lower platelet count subpopulations. The one-way and probabilistic sensitivity analyses found that the use of avatrombopag is cost-saving with the incremental cost-effectiveness ratio in quadrant IV (decreased costs, prophylactic platelet transfusions avoided). CONCLUSION: The use of avatrombopag is expected to be cost-saving while reducing the need for prophylactic platelet transfusions compared with platelet transfusion and lusutrombopag.

The burden associated with thrombocytopenia and platelet transfusions among patients with chronic liver disease.

Background: Thrombocytopenia (TCP), a common complication of chronic liver disease (CLD), can cause uncontrolled bleeding during procedures. As such, CLD patients with TCP and platelet counts <50,000/µL often receive prophylactic platelet transfusions before invasive procedures. However, platelet transfusions are associated with clinical complications, which may result in increased healthcare utilization and costs.Objective: This retrospective database analysis describes the clinical and economic burden in CLD patients with TCP, CLD patients without TCP, and CLD patients with TCP who receive platelet transfusions.Methods: Adult CLD patients with or without TCP were identified in the IBM MarketScan Commercial Claims and Medicare Supplemental data from 1 January 2012 to 31 December 2015. CLD patients with or without TCP were propensity-score matched (1:1) for the analysis of annual healthcare utilization and costs. Platelet transfusions among CLD patients with TCP were identified using procedure codes.Results: Of the 601,626 patients with CLD, 8,292 (1.4%) patients with TCP were matched to patients without TCP. Among CLD patients with TCP, 981 (11.8%) patients received ≥1 platelet transfusions and met inclusion/exclusion criteria. Compared to patients without TCP, CLD patients with TCP had more complications, including higher prevalence of neutropenia (11.4% vs 2.9%) and bleeding events (21.4% vs 10.9%), greater resource utilization including greater average hospital admissions (1.2 vs 0.7, p < .01), greater average ER visits (2.1 vs 1.3, p < .01), higher average outpatient office visits (20.1 vs 18.4, p < .01), and higher average healthcare costs including total costs (p < .01), inpatient costs (p < .01), ER visit costs (p < .01), and outpatient office visit costs (p < .01). The mean annual total costs in CLD and TCP patients with platelet transfusions were $206,396.Conclusions: CLD patients with TCP, and particularly those who received platelet transfusions, experienced significantly greater clinical and economic burden compared to CLD patients without TCP. Safer and more cost-effective treatments to increase platelets are necessary.

Knowledge, attitude, and practice regarding the screening of cervical cancer among women in New Delhi, India.

OBJECTIVE: Cervical cancer is one of the major concerns of public health importance in today's world. It is a leading cause of mortality in women of reproductive age group worldwide, mainly in developing countries. Reduction in mortality and morbidity due to cervical cancer is possible through early detection and treatment. The major factors influencing the early detection of cervical cancer are knowledge regarding risk factors, screening, Pap smear, and symptoms among women. MATERIALS AND METHODS: The present cross-sectional study was carried out to assess the knowledge, attitude, and practice of women about the risk factors, symptoms, and prevention of cervical cancer. Data were obtained from 220 women who visited international trade fair using a pretested self-administered questionnaire. RESULTS: Only 75 study women (50.0%) had ever heard of cervical cancer. The knowledge regarding cervical cancer and its various domains was significantly higher in students and unmarried women. The foul-smelling vaginal discharge was the most common early symptom of cervical cancer according to most of the study women (26, 17.3%). Most of the study women (19, 12.7%) reported tobacco and smoking as the most common risk factor associated with cervical cancer. Only 39 women (26%) had ever heard of cervical cancer screening. Only 27 women (18.0%) ever had Pap smear done in the past and 87 women (58.0%) were willing to undergo cervical cancer screening is offered free of cost. CONCLUSION: The study demonstrates the lack of awareness in women regarding cervical cancer and its screening modalities. This necessitates spreading awareness regarding early symptoms and risk factors associated with cervical cancer for early detection and treatment initiation.

A retrospective analysis of health care utilization for patients with mitochondrial disease in the United States: 2008-2015.

BACKGROUND: Mitochondrial disease (MD) is a heterogeneous group of disorders characterized by impaired energy production caused by abnormal oxidative phosphorylation. Diagnosis of MD is challenging given the variability in how the disease can affect an individual's neurologic, cardiovascular, ophthalmologic, or gastroenterological systems. This study describes the health care utilization and cost in patients diagnosed with MD. METHODS: This study was a retrospective claims analysis based on data from the Truven Health Analytics MarketScan Database and Milliman's Consolidated Health Cost Guidelines Sources Database. For the purpose of this study the diagnosis of MD was defined by ICD-9-CM (prior to October 2015), and ICD-10-CM (October 2015 or later), and included patients identified between January 1, 2008 to December 31, 2015. ICD-9-CM code of 277.87 (disorders of mitochondrial metabolism) and the ICD-10-CM codes of E88.40, E88.41, E88.42 and E88.49 (mitochondrial metabolism disorders) were used as inclusive criteria. Patients were included if they had at least six months of exposure after the first MD-related claim occurrence, and either one MD claim in the inpatient setting OR two MD claims in an outpatient setting. Claims of MD patients are compared to those of a general insured total member population, as well as to those from multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS) patients. RESULTS: During the study period, 3825 patients between the ages of 0 and 15 (pediatric) and 4358 patients 16 years of age and greater (adult) were identified. Total allowed per member per month (PMPM) cost for pediatric patients was $4829 and $3100 for adults, compared with an average of $202 and $486, respectively, for the total member population. The greatest drivers of costs based on allowed claims came from inpatient, surgery, and prescription medications. In the adult population, MD imposes a PMPM cost burden that was comparable to that observed for multiple sclerosis ($3518) and ALS ($3460) patients. CONCLUSIONS: This retrospective claim study highlights the significant differences in the cost of medical care for MD patients compared to those of a general population. Mitochondrial disorders are associated with multisystem disease manifestations and a greater care and cost burden similar to other devastating neuromuscular diseases.

Cost estimate of platelet transfusion in the United States for patients with chronic liver disease and associated thrombocytopenia undergoing elective procedures.

AIMS: This study aimed to estimate the cost of platelet transfusion in patients with chronic liver disease (CLD)-associated thrombocytopenia undergoing an elective procedure in the United States. MATERIALS AND METHODS: The study was conducted in two parts: development of a conceptual framework identifying direct, indirect and intangible costs of platelet transfusion, followed by the estimation of the total cost of platelet transfusion in patients with CLD-associated thrombocytopenia before an elective procedure in the United States using the conceptual framework and cost data obtained from a literature search. The cost of the entire care required to raise a patient's platelet count before the procedure was considered. RESULTS: The final conceptual framework included the costs of generating the supply of platelets, the platelet transfusion itself, adverse events associated with platelet transfusion and refractoriness to platelet transfusion. When costs were accounted for in all the framework cost categories, the total direct cost of a platelet transfusion in a patient with CLD and associated thrombocytopenia was estimated to be in the range of $5258 to $13,117 (2017 US dollars) in the United States. The largest portion of costs was incurred by the transfusion event itself ($3723 to $4436) and the cost of refractoriness ($874 to $7578), which included the opportunity cost of a delayed procedure and subsequent platelet transfusions with human leukocyte antigen-matched platelets. LIMITATIONS AND CONCLUSIONS: Although we were unable to include all cost components identified in the conceptual framework in our total cost estimate, thus likely underestimating the true total cost, and despite the data gaps and challenges limiting our estimate of the full cost of a platelet transfusion in patients with CLD-associated thrombocytopenia undergoing an elective procedure in the United States, this study outlines a comprehensive conceptual framework for estimating the cost elements of a platelet transfusion in these patients.

Modernising ophthalmology teaching: introducing a live and interactive video link.

The Health Education Thames Valley (HETV) Ophthalmology training programme holds compulsory weekly afternoon teaching sessions in Oxford. Traditionally, trainees travelled considerable distances to attend this teaching. Commuting is a known stress factor and car use has environmental, monetary and health costs. To reduce trainee costs, travelling time and improve teaching experience, we introduced an interactive and live video link across HETV. Teaching sessions were broadcast live using free videolink software between 3 sites. New hardware cost £200 per site. Attendees completed weekly feedback questionnaires on the videolink over 9 months. Over this period, the deanery had 22 trainees with 12 working outside Oxford. Projected annual travel savings were calculated. On average 10.8 trainees (49.1%) completed weekly questionnaires: 5.1 (range:3-8) were trainees working outside Oxford and 5.6 trainees (range 3-10) working in Oxford. Attendee responses showed on average: 78.6% learnt as much as attending in person; 91.17% felt interaction through the videolink was adequate; and 94.6% remained keen on telecommuting. Of the trainees in Oxford, 26.5% felt that the videolink interfered with the teaching session. The average videolink quality rating was 3.73 (1:poor; 5:excellent). Annually, the videolink will save each trainee working outside Oxford an average of 2120 kilometres in travel and £594 in expenses. Most attendees felt the videolink was a valuable tool in delivering teaching sessions. It eliminates unnecessary journeys and travel related stress. Within HETV, a £600 initial investment for new hardware would save trainees approximately £7128 annually. There is potential in expanding the role of the videolink to allow guest speakers worldwide to easily contribute to teaching sessions, eliminating unnecessary travel. This model could be adapted to postgraduate training programmes nationally to improve trainee wellbeing by reducing travelling time and costs.

Should all elective knee radiographs requested by general practitioners be performed weight-bearing?

The aims of this study were to: [1] Assess the number of patients with suspected knee osteoarthritis that underwent repeat weight-bearing(WB) knee radiographs in the orthopaedic clinic following initial non-WB radiograph requested by their general practitioner (GP). [2] Confirm whether repeating WB knee views changed radiology reports. [3] Determine the number of London trusts with protocols for routinely performing WB views. A Retrospective cohort study of 1968 patients aged >40 years referred to a London teaching hospital for knee radiographs over 12 months. Radiographs were identified as WB/non-WB. Subsequent repeat WB views performed in those that went on to have an orthopaedic consultation were also documented. A consultant musculoskeletal radiologist reported both images. A proforma containing a likert scale of severity for commonly reported abnormalities in knee osteoarthritis and criteria from the Kellgren and Lawrence scale was used for reporting. London NHS Trusts were surveyed to identify if protocols were in place for performing WB views. A total of 1,968 patients underwent knee radiographs, of which 1922 (97.7%) had initial non-WB radiographs. Of the 56 patients in this group that underwent required repeat WB radiographs, joint space narrowing was reported as more severe on WB versus non-WB radiographs (p = 0.035). Only 54% of departments routinely performed WB radiographs. Few patients (2.3%) referred by GPs have WB radiographs requested. Some of those referred for a specialist opinion required repeat WB views. Nearly half of London hospitals do not routinely perform WB radiographs. This represents a significant financial burden to the NHS, increased radiation exposure and wasted patient/clinician time. We propose that all GP requested knee radiographs be performed as WB unless otherwise stated.

Duodenal eosinophilia and early satiety in functional dyspepsia: confirmation of a positive association in an Australian cohort.

BACKGROUND AND AIM: Functional dyspepsia (FD), defined by unexplained pain or discomfort centered in the upper abdomen, is common. Diagnosis and treatment of FD based on the symptom-based Rome criteria remains challenging. Recently, eosinophilia in the duodenum has been implicated in the pathophysiology of FD in adults, specifically increased eosinophils in early satiety and postprandial distress, but the association remains controversial. The aim of this study was to characterize upper gastrointestinal (GI) tract pathology, specifically duodenal eosinophilia, in an Australian cohort of patients with FD. METHODS: Patients prospectively referred for an upper GI endoscopy (n = 55; mean age, 49.6 years; 61.8% female) were stratified to FD cases (n = 33) and controls (n = 22) using Rome II criteria. All subjects completed a validated bowel symptom questionnaire. The eosinophil count per square millimeter in the duodenal bulb (D1) and second part (D2) was assessed and Helicobacter pylori status determined by gastric histology. Associations with clinical symptoms were assessed. RESULTS: Cases and controls were demographically similar. Duodenal eosinophilia was significantly increased in subjects experiencing early satiety (P = 0.01) and postprandial fullness (P = 0.001). This association was seen in D2 but not D1. Abdominal pain was associated with eosinophilia in both D1 (P = 0.02) and D2 (P = 0.005). Smoking was also associated with higher eosinophil counts in D2 (P = 0.007) and symptoms of early satiety (P = 0.02). CONCLUSIONS: Duodenal eosinophilia occurs in a subset of FD. The potential role of duodenal eosinophils in FD has implications for diagnosis and therapeutic trials.

Type 2 diabetes mellitus without vascular complications and ischemia modified albumin.

Ischemia modified albumin (IMA) is a proven cardiac marker but its role in type 2 diabetes mellitus without vascular complications has not been reported yet. Therefore, IMA was estimated in 60 newly diagnosed patients of type 2 diabetes mellitus and 30 healthy controls along with HbA1c and other investigations (to rule out vascular complications). There was no significant change in IMA levels in type 2 diabetic patients as compared to controls. No correlation could be found between IMA levels and HbA1c. We conclude that IMA levels are not affected in type 2 diabetes mellitus before the onset of vascular complications.