The Need for Institutional Policies for Innovative Therapy: Existing Approaches and Key Elements.

Objectives: Innovative therapy is common in many areas of medicine. Yet, it is unknown whether medical centers have policies to ensure innovative therapy is conducted appropriately.

Design: We contacted three informants at leading U.

Shades of grey: choice, control and capacity in alcohol-related brain damage.

Liaison psychiatrists have identified that conducting capacity assessments in general hospital patients with alcohol-related brain damage (ARBD) can be challenging. This educational article uses the fictitious case of a man with ARBD, alcohol dependence and significant self-neglect, focusing on assessment of his capacity to decide about moving into a care home on discharge. We provide an overview of clinical, legal and ethical literature relevant to decision-making and capacity assessment in individuals with ARBD, with the aim of guiding clinicians approaching complex capacity assessments.

Vulnerability in Biomedical Research: A Historical Reflection and Practical Implications for HIV Cure-Related Research.

The concept of vulnerability in bioethics was first referenced in 1979, when the Belmont Report highlighted the need for special consideration of certain populations in the application of its general principles of respect for persons, beneficence, and justice in research with human participants. Since then, a body of literature has emerged regarding the content, status, and scope, as well as ethical and practical implications of vulnerability in biomedical research. The social history of HIV treatment development has at various points reflected and actively influenced bioethics' debate on vulnerability.

Expanded phenotypic and hematologic abnormalities beyond bone marrow failure in MECOM-associated syndromes.

The MECOM gene encodes multiple protein isoforms that are essential for hematopoietic stem cell self-renewal and maintenance. Germline MECOM variants have been associated with congenital thrombocytopenia, radioulnar synostosis and bone marrow failure; however, the phenotypic spectrum of MECOM-associated syndromes continues to expand and novel pathogenic variants continue to be identified. We describe eight unrelated patients who add to the previously known phenotypes and genetic defects of MECOM-associated syndromes.

Clinical presentation and natural history of Barth Syndrome: An overview.

Barth Syndrome is a rare X-linked disorder caused by pathogenic variants in the gene TAFAZZIN, which encodes for an enzyme involved in the remodeling of cardiolipin, a phospholipid primarily localized to the inner mitochondrial membrane. Barth Syndrome is characterized by cardiomyopathy, skeletal myopathy, neutropenia, and growth abnormalities, among other features. In this review, we will discuss the clinical presentation and natural history of Barth Syndrome, review key features of this disease, and introduce less common clinical associations.

Temporal and anatomic relationship between superficial and deep vein thromboses in hospitalized children.

Background: Recent publications have increasingly demonstrated a link between superficial-vein thrombosis (SVT) and deep-vein thrombosis (DVT) in the adult population and have led to changes in SVT treatment considerations. A similar relationship between SVT and DVT in pediatric populations, however, is not currently well established.

Objectives: We sought to evaluate the temporal and anatomic relationship between SVT and DVT among pediatric inpatients in order to determine to what degree SVT is associated with DVT.

Current approaches to diagnostic testing in von Willebrand Disease.

Von Willebrand Disease (VWD) is considered the most common inherited bleeding disorder. It has multiple subtypes and a primary symptom of mucocutaneous bleeding. Some researchers in this field speculate that inherited disorders of platelet function may be as common but underdiagnosed due to the difficulty of accessing testing.

Should Neonatologists Give Opinions Withdrawing Life-sustaining Treatment?

An infant has a massive intracranial hemorrhage. She is neurologically devastated and ventilator-dependent. The prognosis for pulmonary or neurologic recovery is bleak.