PRO54 ECONOMIC BURDEN OF CARE AND TREATMENT OPTIONS FOR PATIENTS WITH RETT SYNDROME: TWO SYSTEMATIC LITERATURE REVIEWS

Shifting Burdens explores the process and features of mental patient deinstitutionalization as it occurred in America in the 1950s and 1960s. This paper examines the disillusionments American society had with mental institutions, such as faltering standards of care, staff failures, and inadequate treatment options. These issues resulted in the movement towards deinstitutionalization, resulting in the burden of care for displaced mental patients being shifted onto community homes and patients families. Shifting Burdens challenges the notion that deinstitutionalization at the time was a successful endeavour, as the question of care for mental patients continues to exist today.

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Economic burden of the Surgical Treatment of Adult Acquired Flatfoot in the US Population

Foot & Ankle Orthopaedics ◽

10.1177/2473011417s000180 ◽

2017 ◽

Vol 2 (3) ◽

pp. 2473011417S0001

Author(s):

Tyler Goodwin ◽

J. Benjamin Jackson

Keyword(s):

South Carolina ◽

Surgical Treatment ◽

Soft Tissue ◽

Economic Burden ◽

Healthcare Costs ◽

Treatment Options ◽

Incidence Rates ◽

White Female ◽

Small Scale ◽

Clinical Awareness

Category: Ankle, Midfoot/Forefoot, Population Health Introduction/Purpose: Numerous studies have been published on the treatment of adult acquired flatfoot deformity (AAFF). However, there has been little focus on it’s incidence, and economic burden in the general US population. This study updates and examines surgical incidence rates, demographic factors, and economic burden compared to our previous study that used data from South Carolina. Our previous study reported on data from 2004-2014 while this study reports on data from 2004-2014 using a nationwide database. Additionally, we evaluated the number and type(s) of surgeries performed for patients with a diagnosis of flatfoot who underwent surgery. Our hypothesis was that the surgical incidence of AAFF and its economic burden would increase given the aging population and improvements in surgical treatment options. Methods: The Nationwide Inpatient Sample (NIS) compiles patient data representing a 20% stratified sample of all hospital discharges nationwide. Along with demographics, diagnosis and procedural codes the NIS includes: admission type, length of stay, age, gender, race, Diagnostic Related Group, discharge status, primary expected payer, total charges, and physician specialty code. Bivariate descriptive statistics were utilized to analyze the data. Surgical incidence was calculated by assessing the number of surgical procedures compared to the number of lives covered by the Centers for Medicare and Medicaid Services (CMS). Demographics and medical comorbidities of patients who progressed to surgical treatment for AAFF were analyzed. The cost associated with the surgical care episode was calculated to determine the economic burden of the disease. Given the retrospective nature of the study, we utilized a regression analysis with multiple dependent variables to look for trends that could be analyzed in a larger cohort or prospective fashion. Results: In total, 160,795 patients underwent AAFF corrective surgery between 2004 and 2014. Patients who underwent surgery for AAFF were more likely to be white, female and in their 5th decade of life. Average surgical incidence during this time period was 5.12%. Patients were most likely to undergo a combination of soft tissue and fusion procedures, followed by soft tissue procedures alone and then fusion procedures in isolation. The total nationwide healthcare costs associated with patients who underwent surgery for AAFF exceeded 4 billion dollars. Conclusion: Our data demonstrates that there has been an increasing burden of disease for AAFF on society over the past 10 years. Patients undergoing surgery for AAFF contributed more than 4 billion dollars to national healthcare costs between 1994- 2014. Our study confirmed prior small scale studies of the population most at risk and demonstrated an increasing surgical incidence. The increase in incidence and burden parallel rising rates of diabetes and obesity seen nationwide, known risk factors for AAFF. We hope that this data will lead to increased patient education, clinical awareness and resource allocation for future study of AAFF disease prevention.

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What Is Known about the Clinical and Economic Burden of Refractory Chronic Lymphocytic Leukemia in Clinical Practice: A Review of the Literature.

Blood ◽

10.1182/blood.v114.22.4549.4549 ◽

2009 ◽

Vol 114 (22) ◽

pp. 4549-4549

Author(s):

Gwilym J Thompson ◽

Vincent Lévy ◽

Krista Payne ◽

Radek Wasiak ◽

Yvonne Lis

Keyword(s):

Prognostic Factors ◽

Clinical Practice ◽

Chronic Lymphocytic Leukemia ◽

Conference Proceedings ◽

Natural History ◽

Clinical Outcomes ◽

Economic Burden ◽

Treatment Options ◽

Lymphocytic Leukemia ◽

Review Articles

Abstract Abstract 4549 Introduction Chronic lymphocytic leukemia (CLL) is the most common adult haematological malignancy in the Western world. It remains incurable and follows an extremely variable clinical course with survival ranging from months to decades. In those who become refractory to fludarabine based therapy, median survival is less than 1 year. Methods To identify data related to the clinical and economic sequelae of patients in routine clinical care diagnosed with refractory CLL, a systematic literature search of MEDLINE and EMBASE was conducted for the period January 1999-July 2009. The search terms used included incidence, prevalence, natural history, characteristics, clinical management, clinical outcomes, use of health care resources and was focused on patients with fludarabine refractory CLL also refractory to alemtuzumab (fludarabine and alemtuzumab refractory) or less suitable for alemtuzumab due to bulky lymph nodes (bulky fludarabine refractory). Supplementary checks were made of reference lists, particularly in review articles. Relevant conference proceedings for the period January 2006-July 2009 were also examined. Results The search of MEDLINE and EMBASE identified 102 articles of which 26 reported on the more general populations of patients with haematological malignancies, 61 on CLL and 15 on refractory CLL. Of those articles reporting data in relation to CLL (n=61), 49 described patient characteristics and outcomes, prognostic factors, diagnostic and treatment options, natural history of the disease, epidemiology, patient management and specific treatment outcomes and 12 review articles addressed areas such as prognostic factors and treatment options. Of those reporting on refractory CLL (n=15), one was a review article of novel agents and 14 were studies evaluating outcomes following salvage treatment with various pharmaceutical agents and other therapeutic interventions but mainly in patients refractory to fludarabine. Only one US study had evaluated patients who were refractory to both fludarabine and alemtuzumab or were bulky fludarabine refractory, examining response and survival following a variety of salvage treatments (Tam et al. Leukemia and Lymphoma 2007;48:1931-9). The search of conference proceedings identified 27 abstracts, 19 of which evaluated patients with CLL reporting on a range of topics including natural history and survival, prognostic factors and outcomes achieved with existing and experimental pharmaceutical interventions. Only two abstracts specifically reported on patients who were refractory to both fludarabine and alemtuzumab or were bulky fludarabine refractory, examining response rates following administration of a novel pharmaceutical agent (European Haematology Association Meeting 2009, abstracts 0494 and 0919). Conclusions There is very little data from clinical practice on clinical outcomes and none on economic sequelae for patients with double refractory or bulky nodal refractory disease. The lack of such data is likely to hinder the achievement of better outcomes for patients and the evaluation of cost effectiveness for newer agents. This lack of data to inform clinical practice and decision making has prompted the initiation of an observational study in five European countries with the objectives of characterising the current patterns of care, survival outcomes and resource utilization in double refractory and bulky nodal refractory CLL patients in Europe. The study, based on a retrospective chart review of approximately 250 patients, is currently underway in France, Germany, Italy, Spain and the UK. It is anticipated that the results, planned to be available by the end of 2009, will help to identify unmet clinical needs, quantify the clinical and economic burden in this particular population and contribute to the development of new treatment guidelines. Disclosures: Lévy: GlaxoSmithKline: Consultancy. Payne:GlaxoSmithKline: Consultancy. Wasiak:GlaxoSmithKline: Consultancy. Lis:GlaxoSmithKline: Consultancy.

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The Burden of Illness of Congenital Adrenal Hyperplasia (CAH) in Adults: Results: of a Structured Literature Review

Journal of the Endocrine Society ◽

10.1210/jendso/bvab048.193 ◽

2021 ◽

Vol 5 (Supplement_1) ◽

pp. A96-A97

Author(s):

Matthew Stephen Woods ◽

Helen Coope ◽

Kamran Maskin ◽

Lotta Elisabet Parviainen ◽

John Porter ◽

...

Keyword(s):

Literature Review ◽

Congenital Adrenal Hyperplasia ◽

Economic Burden ◽

Burden Of Illness ◽

Treatment Options ◽

Adult Patients ◽

Cochrane Library ◽

Adrenal Crisis ◽

Adrenal Hyperplasia ◽

The Impact

Abstract Objectives: Congenital adrenal hyperplasia (CAH) is a group of rare autosomal-recessive disorders that arise from genetic deficiencies in key enzymes involved in cortisol synthesis. The burden of CAH has never been comprehensively reviewed; this literature review was conducted to summarise the existing burden of illness evidence available for these patients. Methods: A structured, comprehensive literature review was conducted to identify articles describing the burden and treatment landscape of CAH. Literature databases (MEDLINE, Embase, the Cochrane Library and EconLit), websites and conference proceedings were searched. Searches were performed in 2016 and updated in June 2020; eligible articles presented evidence for patients with CAH or paediatric patients with adrenal insufficiency (AI), for ≥1 topic of interest (epidemiology; natural history; clinical characteristics; humanistic, caregiver and economic burden; treatment options; or clinical guidelines). The evidence presented here focusses on the humanistic and economic burden of CAH in adults. Results: A total of 3,711 citations were identified and 336 were included; 84 references reported humanistic or economic burden data relevant to adult patients with CAH. 51 publications were identified reporting patient symptoms, comorbidities and cardiometabolic risk factors; 38 reporting on the impact of CAH on health-related quality of life (HRQL); 5 reporting patient views and 2 reporting economic burden associated with CAH. Compared to the general population, adult patients with CAH were found to be significantly shorter, have poorer bone health, increased levels of obesity, impaired male and female fertility, higher blood pressure and cholesterol levels, have more psychiatric and neurological disorders and have poorer cognitive performance. Adult patients with CAH were also found to have greater insulin resistance and higher levels of type 2 diabetes (T2D). CAH patients are also at risk of adrenal crisis, which contributes to excess mortality. The reported HRQL in adults with CAH varies, with increased impairment observed in more severe forms of CAH, and challenges due to living with a chronic disease impacting HRQL varying according to sex. “Sick day rules” where patients need to double or triple treatment doses, have a significant impact on patients’ HRQL and also have an impact on patients’ resource use, with a UK study reporting that CAH patients will implement these rules 171 times over their lifetime, and be hospitalised for adrenal crises on 11 occasions. CAH was also found to have a significant economic impact, with significantly higher annual healthcare costs compared to matched controls (p=0.007 for patients aged 18–40 years; p<0.001 for patients aged ≥40 years). Conclusions: This comprehensive review highlights that CAH in adults is associated with a significant humanistic and economic burden.

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Improving care and treatment options for women and girls with bleeding disorders

European Journal Of Haematology ◽

10.1111/ejh.12580 ◽

2015 ◽

Vol 95 ◽

pp. 2-10 ◽

Cited By ~ 25

Author(s):

Roshni Kulkarni

Keyword(s):

Treatment Options ◽

Bleeding Disorders ◽

Women And Girls ◽

Care And Treatment

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Positive aspects, negative aspects and challenges associated with stem cell therapy for COVID - 19: A Mini-Review

Current Stem Cell Research & Therapy ◽

10.2174/1574888x16666211102092039 ◽

2021 ◽

Vol 16 ◽

Author(s):

Basavraj Nagoba ◽

Ajay Gavkare ◽

Abhijit Rayate ◽

Sachin Mumbre

Keyword(s):

Financial Constraints ◽

Potential Role ◽

Treatment Options ◽

Short Term ◽

Safety And Efficacy ◽

Respiratory Involvement ◽

Literature Reviews ◽

Effective Option ◽

Cytokine Imbalance

: Like any other pandemic, the Covid-19 scenario has also demanded effective treatment options. The circumstances demand to utilize all the possible weapons in the armamentarium. There have been many issues regarding the short-term and long-term safety and efficacy of these options. Some options are like uncharted seas and these need a detailed and critical review with respect to safety, efficacy, feasibility and financial constraints. Mesenchymal stem cells (MSCs) therapy has been studied for many years for its potential role in diseases with complex pathogenesis. Its efficacy in controlling cytokine imbalance and immuno-modulatory properties is well proven. These effects are being extensively studied for potential extension of the benefits for an effective option for management of COVID-19 patients with severe respiratory involvement. In this mini-review, an attempt has been made to review positive aspects, negative aspects, and challenges influencing MSCs therapy in the management of COVID-19 disease. The results of various studies and literature reviews show that MSCs therapy can be considered as one of the potential options.

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Procreation Confounded

Birth Rights and Wrongs ◽

10.1093/oso/9780190675721.003.0010 ◽

2019 ◽

pp. 127-140

Author(s):

Dov Fox

Keyword(s):

Medical Care ◽

Injury Severity ◽

Treatment Options ◽

Health Conditions ◽

Medical Conditions ◽

Diagnostic Uncertainty ◽

Variable Expression ◽

Care And Treatment ◽

Relevant Factors

Procreation is confounded when clinical professionals misdiagnose, misrepresent, or switch reproductive cells or entities. These errors lead patients to initiate, continue, or terminate pregnancies in ways that thwart their efforts to have a child of one kind or another: How serious is that reproductive injury? Do its benefits outweigh its harms? What are the chances of it manifesting within certain windows of time and at varying levels of severity? How likely is it that misconduct is what caused procreation to be confounded? Is some other factor responsible in addition or instead? To what extent was genetic randomness or diagnostic uncertainty to blame? The seriousness of that harm depends on its foreseeable impact on people’s lives—injury severity is an objective inquiry that begins by asking what kind of child the plaintiffs wanted and why. The variable expression of medical conditions or other targeted traits ratchets up the guesswork to forecast how a defendant’s negligence that thwarted their prenatal selection can be expected to affect the plaintiff. But all this uncertainty needn’t keep courts from assessing how serious confounded procreation is in particular cases: Just because any such determination is bound to admit of some arbitrariness doesn’t mean injury severity can’t be worked out in a principled and systematic way. For health conditions, relevant factors include foreseeable implications for offspring lifespan, impairment, medical care, and treatment options. Courts should reduce dollar awards by however much confounded procreation can be expected to simultaneously benefit plaintiffs, depending on its associated reasons and repercussions.

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