SCD News Briefs

Blood sample

Sickle Cell Disease May Be Protective Against HIV Infection

Sickle cell disease (SCD) may confer some protection against human immunodeficiency virus (HIV) infection, reducing the risk of infection by 75% (odds ratio [OR], 0.25, r=-0.36, P <.001; I2=71.65), according to a recently published systematic review. Moreover, HIV virulence was reduced by 77% (OR, 0.23, r=-0.38, P <.001; I2=63.07). These findings are in agreement with…

DNA

Gene Editing Demonstrates Efficacy in Sickle Cell Disease via Optical Test

Clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing is substantially effective in sickle cell disease but may not completely eliminate vaso-occlusive activity, according to a case study published in Case Reports in Hematology.  This finding was based on the results of optical coherence tomography angiography (OCT-A), which was capable of detecting and measuring micro-occlusive…

woman in pain

Buprenorphine Shows Promise as Safe Alternative to Other Opioids for Pain in SCD

A new study has determined that buprenorphine, an opioid that is safer than high-dose full agonist opioid therapy, can be used in patients with sickle cell disease (SCD). The study, published in the American Journal of Hematology, employed a transitioning protocol developed by the researchers in 36 patients to convert from chronic opioid therapy to…

blood tranfusion

Risk Factors, Mitigation Strategies, and Management Recommendations in SCD

A new review discussing the risk factors, mitigation strategies, and management recommendations for patients with sickle cell disease (SCD) has been published in Transfusion and Apheresis Science. The authors of the review are Margo R. Rollins, MD, from Children’s Healthcare of Atlanta and Emory University School of Medicine, Department of Pediatrics, Aflac Cancer and Blood…

inflamed gallbladder

Gallbladder Disease Associated With Age and Disease Severity in Children With SCD

A new study has determined that gallbladder disease is associated with age and disease severity, but not sex, in children with sickle cell disease (SCD). The study, published in Pediatric Blood & Cancer, also found that neither age nor sex were associated with the rate of cholecystectomy in pediatric patients with both SCD and gallbladder…

CBD

New Study to Test Safety and Efficacy of a Cannabinoid in Sickle Cell Disease

A new clinical trial testing the safety and efficacy of dronabinol as a palliative agent in patients with sickle cell disease is now open. The double-blind, randomized, phase 2 study will assess the effects of dronabinol in treating pain, inflammation, and other complications in patients with sickle cell disease. The trial aims to recruit 60…

sickle cell disease treatment

Study Suggests New Method of Preventing Vaso-Occlusive Crises in Sickle Cell Disease

The lectin and alternative pathways both play a role in promoting inflammation and vaso-occlusion in sickle cell disease, according to a new study published in Translational Research. Inhibiting complement activation via these 2 pathways could therefore prevent vaso-occlusive crises in patients with sickle cell disease. The markers of complement activation are high in patients with sickle…

NORD banners

Rare Disease Patients and Advocate to Headline NORD Conference in October

Two rare disease patients and a rare disease advocate will be the keynote speakers at the National Organization for Rare Disorders (NORD) 2022 annual rare disease conference, being held at the Marriott Marquis in Washington, DC, on October 17 and 18, 2022.   NORD announced the keynote speakers as Brittany Clayborne, MS, PsyD, a post-transplant…

gene manipulation concept

First Patient Dosed With Graphite Bio’s Gene-Editing Therapy for SCD

Graphite Bio announced the dosing of the first patient with GPH101, now known as nulabeglogene autogedtemcel (nula-cel), in their phase 1/2 CEDAR trial for patients with sickle cell disease (SCD). The announcement, reported by news release, noted that the therapy is designed to definitively cure the disease by correcting the causative genetic mutation in SCD.…

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