Real-Life Outcome After Gene Replacement Therapy for Spinal Muscular Atrophy: A Multicenter Experience.

Background: Onasemnogene abeparvovec-xioi (OA) has been available since 2019 as a gene replacement therapy for individuals with spinal muscular atrophy (SMA) under age two years. We aim to expand upon the sparse knowledge about its safety and clinical efficacy.

Methods: The clinical outcome data of all the individuals with SMA who were treated with gene therapy in four tertiary hospitals in Israel were retrieved and analyzed.

Diagnosis and treatment of phrenic nerve hourglass constriction in patients with Parsonage-Turner syndrome.

Phrenic nerve injury can occur anywhere along its course and clinically results in diaphragm paralysis. Although most patients with Parsonage-Turner syndrome and phrenic nerve dysfunction improve without treatment, some patients do not recover spontaneously. In these cases, an initial autoimmune response produces scarring along the affected nerve(s).

Technical Aspects of Lung Volume Reduction Surgery Including Anesthetic Management and Surgical Approaches.

As palliative treatment, lung volume reduction surgery can be offered to a selected subset of chronic obstructive pulmonary disease patients. Careful adherence to established inclusion and exclusion criteria is critical to achieve good outcomes. The evolution of surgical techniques toward minimally invasive approaches has improved outcomes.

Extracorporeal Carbon Dioxide Removal in the Treatment of Status Asthmaticus.

Objectives: Venovenous extracorporeal carbon dioxide removal may be lifesaving in the setting of status asthmaticus.

Design: Retrospective review.

Setting: Medical ICU.

Increasing Volume of Bone Marrow Biopsies by Radiology Providers: Evaluation of Trends by Physician Specialty and Practice Setting.

Purpose: The aim of this study was to evaluate trends in bone marrow biopsies performed in the United States by physician specialty and practice setting.

Methods: The CMS Medicare Physician Supplier Procedure Summary database was queried from 2005 to 2016 for bone marrow biopsies and aspirations (BMBs). Data were categorized according to the largest subspecialty groups (medicine, surgery, radiology, pathology, and other) and encounter setting (office, inpatient hospital, and outpatient hospital).

Percutaneous Vertebral Augmentation for Vertebral Compression Fractures: National Trends in the Medicare Population (2005-2015).

Study Design: Retrospective analysis of Medicare data OBJECTIVE.: To analyze trends of vertebral augmentation in the elderly Medicare population in the context of evolving evidence and varied medical society opinions.

Summary Of Background Data: Percutaneous vertebral augmentation offers a minimally invasive therapy for vertebral compression fractures.

ACR Appropriateness Criteria Suspected Lower Extremity Deep Vein Thrombosis.

Suspected lower extremity deep venous thrombosis is a common clinical scenario which providers seek a reliable test to guide management. The importance of confidently making this diagnosis lies in the 50% to 60% risk of pulmonary embolism with untreated deep vein thrombosis and subsequent mortality of 25% to 30%, balanced with the risks of anticoagulation. The ACR Appropriateness Criteria Expert Panel on Vascular Imaging reviews the current literature regarding lower extremity deep venous thrombosis and compared various imaging modalities including ultrasound, MR venography, CT venography, and catheter venography.

ACR Appropriateness Criteria Imaging of Mesenteric Ischemia.

Mesenteric ischemia is an uncommon condition resulting from decreased blood flow to the small or large bowel in an acute or chronic setting. Acute ischemia is associated with high rates of morbidity and mortality; however, it is difficult to diagnose clinically. Therefore, a high degree of suspicion and prompt imaging evaluation are necessary.

Lung volume reduction surgery in the post-National Emphysema Treatment Trial era.

Lung volume reduction surgery (LVRS) as means to improve the pulmonary function and quality of life of patients with chronic obstructive pulmonary disease (COPD) can be traced back to the 1950's and early work by Otto Brantigan. Joel Cooper revived this concept with pioneering work in the 1990's. His work, along with others, led to the National Emphysema Treatment Trial (NETT) which demonstrated a quality of life and survival benefit for certain subsets of patients with emphysema.

Hemodialysis Access Maintenance in the Medicare Population: An Analysis Over a Decade of Trends by Provider Specialty and Site of Service.

Purpose: To evaluate annual national trends in hemodialysis access maintenance procedures in the Medicare population by specialty and setting.

Methods: Medicare Physician Supplier Procedure Summary Master Files between 2005 and 2015 were analyzed for procedure codes of hemodialysis access angiography and percutaneous thrombectomy. Using physician specialty codes, component procedure volume for endovascular services were queried for radiology, medicine, and surgery.

Management of Surge in Extracorporeal Membrane Oxygenation Transport.

Background: Transporting patients receiving extracorporeal membrane oxygenation (ECMO) support is safe and reliable with a dedicated program and established management protocols. As our program has grown, our teams have had to adapt to manage surges in transport volume while maintaining patient safety. We assessed the outcomes at peak use of our ECMO transport services during surges.

Assessing social values for California's efforts to reduce the overuse of unnecessary medical care.

Background: A partnership of large health-care purchasers created a workgroup to reduce the overuse of harmful and wasteful medical care in California.

Objective: Employ a civic engagement process to identify the social values important to the public in considering different strategies to reduce overuse.

Intervention: Use of deliberation techniques for 3 case examples that explore possible strategies: physician oversight, physician compensation, increased patient cost-sharing or taking no definitive action.

ACR Appropriateness Criteria Penetrating Neck Injury.

In patients with penetrating neck injuries with clinical soft injury signs, and patients with hard signs of injury who do not require immediate surgery, CT angiography of the neck is the preferred imaging procedure to evaluate extent of injury. Other modalities, such as radiography and fluoroscopy, catheter-based angiography, ultrasound, and MR angiography have their place in the evaluation of the patient, depending on the specific clinical situation and question at hand. The ACR Appropriateness Criteria are evidence-based guidelines for specific clinical conditions that are reviewed annually by a multidisciplinary expert panel.

ACR Appropriateness Criteria Imaging of Deep Inferior Epigastric Arteries for Surgical Planning (Breast Reconstruction Surgery).

Breast cancer is the most common malignancy in women in the United States. Breast reconstruction surgery is a commonly used therapy for patients with breast cancer. The technique for the deep inferior epigastric perforator flap uses a preserved rectus muscle, which decreases donor site morbidity.

Setting Boundaries: Public Views on Limiting Patient and Physician Autonomy in Health Care Decisions.

We obtained and qualitatively analyzed input from more than nine hundred citizens during seventy-six public deliberation sessions about patient and physician autonomy in decision making, setting health care boundaries, and the tensions among competing social values. Generally, participants resisted interference with the patient-physician relationship and believed strongly in the freedom of patient and physician to control individual medical decisions. However, during deliberation participants identified two situations where boundaries and regulations in health care were more acceptable: protecting people from harm and allocating limited resources.

ACR Appropriateness Criteria Vascular Claudication-Assessment for Revascularization.

Vascular claudication is a symptom complex characterized by reproducible pain and weakness in an active muscle group due to peripheral arterial disease. Noninvasive hemodynamic tests such as the ankle brachial index, toe brachial index, segmental pressures, and pulse volume recordings are considered the first imaging modalities necessary to reliably establish the presence and severity of arterial obstructions. Vascular imaging is consequently used for diagnosing individual lesions and triaging patients for medical, percutaneous, or surgical intervention.

Radiofrequency and microwave ablation in combination with transarterial chemoembolization induce equivalent histopathologic coagulation necrosis in hepatocellular carcinoma patients bridged to liver transplantation.

Background: Bridging therapy plays an increasingly important role in the management of patients with hepatocellular carcinoma (HCC) awaiting liver transplantation (LT). Combination therapy with drug-eluting bead transarterial chemoembolization (DEB-TACE) and percutaneous thermal ablation, such as radiofrequency ablation (RFA) or microwave ablation (MWA), has shown success at prolonging survival and bridging patients to LT. However, few studies have evaluated the two combination therapy regimens head-to-head at a single institution, and fewer have compared histopathology.

ACR Appropriateness Criteria Clinically Suspected Pulmonary Arteriovenous Malformation.

Pulmonary arteriovenous malformations are often included in the differential diagnosis of common clinical presentations, including hypoxemia, hemoptysis, brain abscesses, and paradoxical stroke, as well as affecting 30% to 50% of patients with hereditary hemorrhagic telangiectasia (HHT). Various imaging studies are used in the diagnostic and screening settings, which have been reviewed by the ACR Appropriateness Criteria Vascular Imaging Panel. Pulmonary arteriovenous malformation screening in patients with HHT is commonly performed with transthoracic echocardiographic bubble study, followed by CT for positive cases.

Understanding An Informed Public's Views On The Role Of Evidence In Making Health Care Decisions.

Policy makers and practitioners increasingly believe that medical evidence plays a critical role in improving care and health outcomes and lowering costs. However, public understanding of the role of evidence-based care may be different. Public deliberation is a process that convenes diverse citizens and has them learn about and consider ethical or values-based dilemmas and weigh alternative views.

The safety, efficacy, and durability of lung-volume reduction surgery: A 10-year experience.

Objectives: The National Emphysema Treatment Trial (NETT) validated the efficacy of lung-volume reduction surgery (LVRS) in selected patients with emphysema; however, concerns about the safety and durability of the operation have limited its clinical application. We evaluated our experience with LVRS, for the time period since approval was given by the Centers for Medicare and Medicaid Services, with respect to surgical morbidity and mortality, early and late functional outcomes, and long-term survival.

Methods: Retrospective analysis was performed on 91 patients for whom consent was obtained for bilateral LVRS at our institution between January 2004 and June 2014.