How Activin A Became a Therapeutic Target in Fibrodysplasia Ossificans Progressiva.

Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder characterized by episodic yet cumulative heterotopic ossification (HO) of skeletal muscles, tendons, ligaments, and fascia. FOP arises from missense mutations in Activin Receptor type I (ACVR1), a type I bone morphogenetic protein (BMP) receptor. Although initial findings implicated constitutive activity of FOP-variant ACVR1 (ACVR1) and/or hyperactivation by BMPs, it was later shown that HO in FOP requires activation of ACVR1 by Activin A.

Garetosmab in Fibrodysplasia Ossificans Progressiva: Clinical Pharmacology Results from the Phase 2 LUMINA-1 Trial.

Here, we report the clinical pharmacology data from LUMINA-1 (NCT03188666), a Phase 2 trial that evaluated garetosmab (a monoclonal antibody against activin A) in patients with fibrodysplasia ossificans progressiva. Forty-four patients were randomly assigned to intravenous 10 mg/kg of garetosmab or placebo every 4 weeks in a double-blind 28-week treatment period, followed by a 28-week open-label treatment period with garetosmab, and subsequent open-label extension. Serum samples were obtained to assess pharmacokinetics (PK), immunogenicity, and bone morphogenetic protein 9 (BMP9).

Effect of GLP-1 agonists on weight loss in patients with polycystic ovary syndrome and obesity: A single-center study.

Background: Weight loss of >5% in patients with polycystic ovary syndrome and obesity (PCOS-O) is believed to improve underlying drivers of the syndrome. Weight loss facilitated by GLP-1 agonists in patients with PCOS-O is not well characterized. In this single-center retrospective study, we determined weight loss in patients with PCOS-O with GLP-1 monotherapy versus metformin.

Disseminated Herpes Simplex Virus-2 (HSV-2) as a Cause of Viral Hepatitis in an Immunocompetent Host.

BACKGROUND Herpes simplex virus-2 (HSV-2) affects nearly 1 in 5 adults in the United States. Complications such as viral hepatitis and dissemination are rare in immunocompetent hosts. In this report, we describe a case of viral hepatitis secondary to disseminated HSV-2 in an immunocompetent patient with recurrent fevers and elevated aminotransferases.

Outcomes of Kidney Transplant Recipients With Posttransplant Genitourinary Infectious Complications: A Single Center Study.

Objectives: Long-term outcomes of kidney transplant recipients with postoperative genitourinary tract infections are not well characterized. In this single center retrospective study, we aimed to investigate the long-term effects of early posttransplant genitourinary infections under a protocol that included 1 month of antibiotic prophylaxis on graft failure and patient outcomes.

Materials And Methods: Electronic medical records of 1752 recipients of kidney-alone transplant between January 2000 and December 2008 were reviewed.

Variation in practice patterns and outcomes across United Network for Organ Sharing allocation regions.

Background: The number of heart transplants performed is limited by organ availability and is managed by the United Network for Organ Sharing (UNOS). Efforts are underway to make organ disbursement more equitable as demand increases.

Hypothesis: Significant variation exists in contemporary patterns of care, wait times, and outcomes among patients undergoing heart transplantation across UNOS regions.

ACE inhibitor-associated intestinal angioedema in orthotopic heart transplantation.

Angiotensin-converting enzyme inhibitor induced angioedema commonly involves the head and neck area. We report a case of angiotensin-converting enzyme inhibitor induced intestinal angioedema in a heart transplant recipient on mTOR immunosuppression. A 36-year-old Caucasian woman with history of heart transplantation on sirolimus, tacrolimus and prednisone presented to the Emergency Department with abdominal pain, one day following lisinopril initiation.

The Impact of the Transition From Volume to Value on Heart Failure Care: Implications of Novel Payment Models and Quality Improvement Initiatives.

In response to wide variation in quality and outcomes as well as escalating health care costs, the U.S. health care system is moving away from a volume-based payment system to a quality- and value-based system.

Outcomes of Kidney Transplant Recipients With Percutaneous Ureteral Interventions: A Single-Center Study.

Background: Long-term outcomes of kidney transplantation recipients with percutaneous ureteral management of transplant ureteral complications are not well characterized.

Methods: Electronic records of 1753 recipients of kidney-alone transplant between January 2000 and December 2008 were reviewed. One hundred thirty-one patients were identified to have undergone percutaneous ureteral management, with placement of percutaneous nephrostomy tube or additional intervention (nephroureteral stenting and/or balloon dilation).

Long-Term Outcomes of Kidney Transplant Recipients with Bladder Dysfunction: A Single-Center Study.

Background: Long-term outcomes of kidney transplantation recipients with bladder dysfunction or prior bladder surgery are not well characterized.

Material And Methods: Electronic records of 1753 recipients of kidney-alone transplant between January 2000 and December 2008 were reviewed. We found that 1652 recipients had normal bladder function, 80 had bladder dysfunction, and 21 had bladder substitute or urinary diversion.

Sports participation with arachnoid cysts.

OBJECT There is currently no consensus on the safety of sports participation for patients with an intracranial arachnoid cyst (AC). The authors' goal was to define the risk of sports participation for children with this imaging finding. METHODS A survey was prospectively administered to 185 patients with ACs during a 46-month period at a single institution.

Radiation safety and spine surgery: systematic review of exposure limits and methods to minimize radiation exposure.

Background: Ionizing radiation is typically used during spine surgery for localization and guidance in instrumentation placement. Minimally invasive (MI) surgical procedures are increasingly popular and often require significantly more fluoroscopy, placing surgeons at risk for increased radiation exposure and radiation-induced complications. This study provides recommendations for minimizing risk of radiation-induced injury to spine surgeons and summarizes studies addressing radiation exposure in spine procedures.

Risk factors for the development of deformity in patients with spinal infection.

Object: Bacterial spinal infections are infrequent but may lead to significant morbidity and death. Apart from neurological complications, infections may also lead to bone destruction resulting in deformity of the spine. However, the incidence of spinal deformities and risk factors is not well characterized in the literature.

Perioperative characteristics and complications in obese patients undergoing anterior cervical fusion surgery.

In the USA, obesity rates have significantly increased in the last 15 years. Mirroring this trend, a large proportion of patients undergoing spinal surgery are obese. Concern exists for increased complications due to surgical challenges posed by obese patients and their often-prevalent comorbidities.

SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy.

Background: Therapies designed to decrease the level of SOD1 are currently in a clinical trial for patients with superoxide dismutase (SOD1)-linked familial amyotrophic lateral sclerosis (ALS).

Objective: To determine whether the SOD1 protein in cerebral spinal fluid (CSF) may be a pharmacodynamic marker for antisense oligonucleotide therapy and a disease marker for ALS.

Design: Antisense oligonucleotides targeting human SOD1 were administered to rats expressing SOD1G93A.