Pentosan Polysulfate Maculopathy: Final Outcomes from a 4-Year Prospective Study of Disease Progression after Drug Cessation.

Purpose: To report the long-term disease course of pentosan polysulfate (PPS) maculopathy following drug cessation.

Design: Single-institution, prospective case series.

Methods: 23 eyes of 12 participants seen at the Emory Eye Center with a diagnosis of PPS maculopathy were included in our study.

A novel large multi-gene deletion in syndromic choroideremia.

Introduction: Caused by mutation or deletion of the gene, choroideremia is a rare X-linked recessive chorioretinal dystrophy characterized by progressive degeneration of the retinal pigment epithelium, photoreceptors, and the choriocapillaris. There are few published reports of choroideremia associated with complex syndromic phenotypes due to large or contiguous gene deletions.

Methods: Case report and review of literature.

Characteristics of Vitamin A Deficiency Retinopathy at a Tertiary Referral Center in the United States.

Purpose: To explore the risk factors and fundus imaging features of vitamin A deficiency retinopathy (VADR) in an academic tertiary referral center in Atlanta, GA, United States, and to propose guidance regarding diagnostic workup and management of affected patients.

Design: Single-center retrospective case series.

Subjects: Nine patients seen between 2015 and 2021 at the Emory Eye Center diagnosed with VADR.

Colopathy Associated with Pentosan Polysulfate Use.

Introduction: We describe a novel colopathy associated with pentosan polysulfate (PPS) use and measure the strength of the drug-disease association.

Methods: Two-part investigation. In the cohort study of individuals with a history of prior long-term PPS use, case histories were obtained and gastrointestinal disease course was followed with review of endoscopy records and histopathology specimens.

Helping families heal in substance use treatment: A qualitative study about the role of peer support specialists with client families.

Introduction: Peer support specialists (PSS) are people with previous psychiatric illness or substance use disorders who use their experience to support those facing similar hardships. PSS offer a range of beneficial outcomes to both the PSS and clients. The most immediate social connections to those seeking treatment are often their families, yet no PSS studies are inclusive of family involvement.

Immunoglobulin A vasculitis presenting as bilateral upper eyelid erythema: A case report and review of literature.

Immunoglobulin A vasculitis (IgAV) is the most common vasculitis of childhood characterized by petechial or purpuric rash, abdominal pain, arthralgia, and renal involvement. Ophthalmic manifestations of IgAV are uncommon. Herein, we describe a case of bilateral upper eyelid erythema presenting in a 6-year-old male, leading to a diagnosis of IgAV.

The Impact of Poorly Differentiated Histology on Immunotherapy in Advanced Gastrointestinal Cancers.

Introduction: Checkpoint inhibitors (CPI) have significantly improved survival among patients with various cancer types. Prior studies have shown a correlation between immune cell infiltration and poorly differentiated cancers. This study evaluated the impact of poorly differentiated histology on survival in patients with advanced gastrointestinal cancers treated with immunotherapy.

Phakomatous Choristomas: A Case Report and Review of Literature.

Purpose: To document a case of phakomatous choristoma (PC), a rare benign periocular tumor, and to review the literature on previously reported cases.

Methods: The authors describe a case of PC and its clinical, histopathological, immunohistochemical, and radiological features, and present findings from a comprehensive review of all previously reported cases of this rare pediatric tumor.

Results: This case report and review highlights the benign clinical nature of PC.

Heterogeneity of antidiabetic treatment effect on the risk of major adverse cardiovascular events in type 2 diabetes: a systematic review and meta-analysis.

Background: We explored whether clinically relevant baseline characteristics of patients with type 2 diabetes can modify the effect of glucagon-like peptide-1 receptor agonists (GLP-1 RA) or sodium-glucose cotransporter-2 inhibitors (SGLT-2i) on the risk of major adverse cardiovascular events (MACE).

Methods: We investigated Medline and EMBASE through June 2019. We included randomized clinical trials reporting the effect of GLP-1 RA or SGLT-2i on MACE in subgroups of patients with type 2 diabetes, identified through key baseline factors: established cardiovascular disease; heart failure; chronic kidney disease; uncontrolled diabetes; duration of diabetes; hypertension; obesity; age; gender and race.

Correction to: FDA and EMA Biosimilar Approvals.

This paper published with several formatting errors. They have been corrected and the paper has re-published.

Desmopressin and the risk of hyponatremia: A population-based cohort study.

Background: Desmopressin was approved by the Food and Drug Administration (FDA) in 1978 for use in diabetes insipidus and bleeding disorders, but it is also prescribed off-label for patients with nocturia. Quantifying the potential risks facing adult patients taking desmopressin has taken on added importance because a new intranasal formulation of desmopressin was approved by the FDA in 2017. Like the old formulation, the main active ingredient is desmopressin acetate, but the new formulation also contains an excipient designed to enhance absorption.

Surrogate Endpoints and Drug Regulation: What Is Needed to Clarify the Evidence.

The FDA's new table of surrogate endpoints used for drug approvals is an important step forward for overseeing the use of biomarkers in clinical trials. Nevertheless, we present several ways in which the table can be improved.

Challenges and Opportunities for Biomarker Validation.

Biomarkers can be powerful tools to guide diagnosis, treatment, and research. However, prudent use of bio-markers requires formal validation efforts. Although the data needed for biomarker validation has traditionally been hard to access, new research initiatives can ease this process.

Prevalence of Publicly Available Expanded Access Policies.

The Food and Drug Administration's expanded access program allows patients with serious or immediately life-threatening conditions to seek access to experimental drugs and treatments from their manufacturers. The 21st Century Cures Act of 2016 sought to increase the transparency of manufacturers' approaches to expanded access by requiring public listing of five key pieces of information about their expanded access programs: 1) relevant contact information, 2) procedures for making requests, 3) general criteria used to evaluate requests, 4) length of time anticipated to acknowledge receipt of requests, and 5) a reference to pertinent information on ClinicalTrials.gov.