Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Durable responses for hemophilia A gene therapy

High K et al. ASH 2018, Abstract 487

Publish date:: February 6, 2019

Key clinical point: A single infusion of SPK-8011 reduced bleed and infusion rates in severe hemophilia A patients.

Major finding: The overall reduction in annualized infusion rate (AIR) and annualized bleeding rate (ABR) was 97% for each.

Study details: A phase 1/2 study of 12 patients

Disclosures: Spark Therapeutics sponsored the study. Dr. George reported equity ownership related to the University of Pennsylvania and consultancy for Pfizer.

Read the article here.

Citation:

High K et al. ASH 2018, Abstract 487.

This Week's Must Reads

Younger adults present with more advanced esophageal adenocarcinoma,

PDAC: Tumor reduction after neoadjuvant therapy may predict postsurgical survival,

Mortality risks rise with age, infections, but not inhibitor status in persons with non-severe hemophilia A,

‘Impressive’ local control with MRI-guided brachytherapy in cervical cancer,

U.S. mothers underestimate role breastfeeding plays in curbing breast cancer,

Must Reads in Bleeding Disorders

Mortality risks rise with age, infections, but not inhibitor status in persons with non-severe hemophilia A,

Hemochromatosis variants may confer 10-fold higher risk of liver cancer,

Hemophilia, von Willebrand disease do not increase postop complications for ACL reconstruction,

Mouth splints decrease risk of post–dental extraction bleeding in hemophilia,

Low VWF levels or blood group O not linked to intracerebral hemorrhage risk,