Fibrodysplasia Ossificans Progressiva: What Have We Achieved and Where Are We Now? Follow-up to the 2015 Lorentz Workshop.

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare progressive genetic disease effecting one in a million individuals. During their life, patients with FOP progressively develop bone in the soft tissues resulting in increasing immobility and early death. A mutation in the ACVR1 gene was identified as the causative mutation of FOP in 2006. After this, the pathophysiology of FOP has been further elucidated through the efforts of research groups worldwide. In 2015, a workshop was held to gather these groups and discuss the new challenges in FOP research. Here we present an overview and update on these topics.

Cardiometabolic vigilance in COVID-19 and resource husbandry in resource-challenged times: Clinical practice- based expert opinion.

BACKGROUND AND AIMS: The ongoing pandemic of coronavirus disease 2019 (COVID-19) is rapidly evolving, thereby posing a profound challenge to the global healthcare system. Cardiometabolic disorders are associated with poor clinical outcomes in persons with COVID-19. Healthcare challenges during the COVID-19 pandemic are linked to resource constraints including shortage of Personal Protective Equipment's (PPE), laboratory tests and medication. In this context, a group of clinical experts discussed the endocrine and cardiology vigilance required in times of COVID-19. Further, the group proposed certain resource husbandry recommendations to be followed during the pandemic to overcome the constraints. METHOD: The clinical experts discussed and provided their inputs virtually. The expert panel included clinical experts comprising endocrinologists, Consultant Physicians and cardiologists from India. The panel thoroughly reviewed existing literature on the subject and proposed expert opinion. RESULTS: The expert panel put forward clinical practice-based opinion for the management of cardiometabolic conditions including diabetes mellitus and hypertension. As these conditions are associated with poor clinical outcomes, the expert panel recommends that these persons be extra-cautious and take necessary precautions during the ongoing pandemic. Further, experts also provided appropriate, affordable, available and accessible solution to the resource constraint situations in times of COVID-19 pandemic. CONCLUSION: The clinical expert opinion put forward in this article will serve as a reference for clinicians treating diabetes and cardiovascular disease during the COVID-19 pandemic.

The Most Influential Publications in Obstetric Anesthesiology, 1998-2017: Utilizing the Delphi Method for Expert Consensus.

BACKGROUND: There have been many advances in obstetric anesthesiology in the past 2 decades. We sought to create a list of highly influential publications in the field using the Delphi method among a group of obstetric anesthesiology experts to create an important educational, clinical, and research resource. METHODS: Experts in the field, defined as obstetric anesthesiologists selected to present the Gerard W. Ostheimer Lecture at the Society for Obstetric Anesthesia and Perinatology (SOAP) annual meeting within the past 20 years, were recruited to participate. The Delphi technique was used by administering 3 rounds of surveys. Participants were initially asked to identify the highly influential publications from the year they presented the Ostheimer lecture, in addition to the most influential publications from the time period overall. Highly influential publications were defined as those that changed traditional views, invoked meaningful practices, catalyzed additional research, and fostered ideas or practices that had durability over time. After each round of surveys, responses were collected and used as choices for subsequent surveys with the goal of obtaining group consensus. RESULTS: We determined expert consensus on 22 highly influential publications from 1998 to 2017. The focus of these publications ranged from disease entities, interventions, treatment methodologies, and complications. CONCLUSIONS: Key themes in the publications chosen included the reduction of maternal morbidity and mortality and refinements in the analgesic and anesthetic management of labor and delivery.

Experts' opinion on inositols in treating polycystic ovary syndrome and non-insulin dependent diabetes mellitus: a further help for human reproduction and beyond.

Introduction: This Experts' opinion provides an updated scientific support to gynecologists, obstetricians, endocrinologists, nutritionists, neurologists and general practitioners on the use of Inositols in the therapy of Polycystic Ovary Syndrome (PCOS) and non-insulin dependent (type 2) diabetes mellitus (NIDDM).Areas covered: This paper summarizes the physiology of Myo-Inositol (MI) and D-Chiro-Inositol (DCI), two important molecules present in human organisms, and their therapeutic role, also for treating infertility. Some deep differences between the physiological functions of MI and DCI, as well as their safety and intestinal absorption are discussed. Updates include new evidence on the efficacy exerted in PCOS by the 40:1 MI/DCI ratio, and the innovative approach based on alpha-lactalbumin to overcome the decreased therapeutic efficacy of Inositols in some patients.Expert opinion: The evidence suggests that MI, alone or with DCI in the 40:1 ratio, offers a promising treatment for PCOS and NIDDM. However, additional studies need to evaluate some still unresolved issues, such as the best MI/DCI ratio for treating NIDDM, the potential cost-effectiveness of reduced gonadotropins administration in IVF due to MI treatment, or the benefit of MI supplementation in ovulation induction with clomiphene citrate in PCOS patients.

Tackling the 3 Big Challenges Confronting Health Technology Assessment Development in Asia: A Commentary.

There has been continuous development in the field of health technology assessment (HTA) owing to the added value of HTA in supporting healthcare reimbursement decisions. Collaboration and engagement among countries in Asia has been carried out to share experiences and learning on the barriers and factors facilitating the implementation and use of HTA in policy making. A symposium on the topic of Health Technology Assessment (HTA): Selecting the Highest Value Care was held on January 10, 2019 at the National University of Singapore, during which 3 major challenges confronting HTA development in Asia were identified. The symposium also offered possible ways to overcome the challenges.

No Docs of All Trades: Ruling Reinforces Expert Witness Reforms.

Before Texas' landmark liability reforms passed in 2003, gray areas in the law often led to serious green for people who sued physicians.

Primary immunodeficiency diseases in lung disease: warning signs, diagnosis and management.

BACKGROUND: Pulmonary complications are common in primary immunodeficiency diseases (PID) and contribute to morbidity and mortality in these patients. However, their varied presentation and a general lack of awareness of PID in this setting make early diagnosis and treatment difficult. The aim of this study was to define the warning signs of PID in patients with respiratory manifestations, the necessary diagnostic tests, and the therapeutic management of both children and adults. METHODS: A review of the literature was performed, and 43 PID interdisciplinary specialists were consulted. RESULTS: This document identifies the pulmonary and extrapulmonary manifestations that should prompt a suspicion of PID, the immunological and respiratory tests that should be included in the diagnostic process according to the level of care, recommendations regarding the use of immunoglobulin replacement therapy according to the specific immunodeficiency, and the minimum recommended immunological and pulmonary monitoring in these patients. CONCLUSIONS: This document is the first to combine scientific evidence with the opinion of a broad panel of experts specializing in the treatment of patients with immunodeficiencies. It aims to provide a useful tool for all practitioners who are regularly involved in the management of these patients.

Ten important articles on noninvasive ventilation in critically ill patients and insights for the future: A report of expert opinions.

BACKGROUND: Noninvasive ventilation is used worldwide in many settings. Its effectiveness has been proven for common clinical conditions in critical care such as cardiogenic pulmonary edema and chronic obstructive pulmonary disease exacerbations. Since the first pioneering studies of noninvasive ventilation in critical care in the late 1980s, thousands of studies and articles have been published on this topic. Interestingly, some aspects remain controversial (e.g. its use in de-novo hypoxemic respiratory failure, role of sedation, self-induced lung injury). Moreover, the role of NIV has recently been questioned and reconsidered in light of the recent reports of new techniques such as high-flow oxygen nasal therapy. METHODS: We conducted a survey among leading experts on NIV aiming to 1) identify a selection of 10 important articles on NIV in the critical care setting 2) summarize the reasons for the selection of each study 3) offer insights on the future for both clinical application and research on NIV. RESULTS: The experts selected articles over a span of 26 years, more clustered in the last 15 years. The most voted article studied the role of NIV in acute exacerbation chronic pulmonary disease. Concerning the future of clinical applications for and research on NIV, most of the experts forecast the development of innovative new interfaces more adaptable to patients characteristics, the need for good well-designed large randomized controlled trials of NIV in acute "de novo" hypoxemic respiratory failure (including its comparison with high-flow oxygen nasal therapy) and the development of software-based NIV settings to enhance patient-ventilator synchrony. CONCLUSIONS: The selection made by the experts suggests that some applications of NIV in critical care are supported by solid data (e.g. COPD exacerbation) while others are still waiting for confirmation. Moreover, the identified insights for the future would lead to improved clinical effectiveness, new comparisons and evaluation of its role in still "lack of full evidence" clinical settings.

Opinions of Spanish pig producers on the role, the level and the risk to public health of antimicrobial use in pigs.

Antimicrobial use is a major driver of antimicrobial resistance and prescribers (physicians and veterinarians) and end users (patients, food producers and pet owners) are the cornerstones of this scenario. Intensive pig farming is a livestock activity that has a high antimicrobial use. This study is based on the opinions of pig producers. The study was done at national level and comprised two independent cross-sectional surveys using a questionnaire-based methodology and face-to-face interviews carried out between April and October, 2010. The collected data, which consisted of opinions of producers on 48 farrow-to-finish farms and 62 finisher farms, showed that, irrespective of farm type, pig producers in Spain have an imperfect knowledge of the main use of antimicrobials. Antimicrobials are perceived as valuable cost-effective tools for animal health and husbandry and there is little concern among pig producers about the harmful effects on public health of on-farm antimicrobial use.

NO-FAULT COMPENSATION FOR MEDICAL INJURIES: TRENDS AND CHALLENGES.

UNLABELLED: As an alternative to the tort or fault-based system, a no-fault compensation system has been viewed as having the potential to overcome problems inherent in the tort system by providing fair, speedy and adequate compensation for medically injured victims. Proponents of the suggested no-fault compensation system have argued that this system is more efficient in terms of time and money, as well as in making the circumstances in which compensation is paid, much clearer. However, the arguments against no-fault compensation systems are mainly on issues of funding difficulties, accountability and deterrence, particularly, once fault is taken out of the equation. Nonetheless, the no-fault compensation system has been successfully implemented in various countries but, at the same time, rejected in some others, as not being implementable. In the present trend, the no-fault system seems to fit the needs of society by offering greater access to justice for medically injured victims and providing a clearer "road map" towards obtaining suitable redress. This paper aims at providing the readers with an overview of the characteristics of the no fault compensation system and some examples of countries that have implemented it. METHODOLOGY: Qualitative Research-Content Analysis. RESULTS: Given the many problems and hurdles posed by the tort or fault-based system, it is questionable that it can efficiently play its role as a mechanism that affords fair and adequate compensation for victims of medical injuries. However, while a comprehensive no-fault compensation system offers a tempting alternative to the tort or fault-based system, to import such a change into our local scenario requires a great deal of consideration. There are major differences, mainly in terms of social standing, size of population, political ideology and financial commitment, between Malaysia and countries that have successfully implemented no-fault systems. Nevertheless, implementing a no-fault compensation system in Malaysia is not entirely impossible. A custom-made no-fault model tailored to suit our local scenario can be promising, provided that a thorough research is made, assessing the viability of a no-fault system in Malaysia, addressing the inherent problems and, consequently, designing a workable no-fault system in Malaysia.