From Early Hypothesis to Clinical Data About Reducing Vaso-Occlusive Episodes with Arginine Therapy

At the 40th American Society of Hematology (ASH) Annual Meeting and Exposition investigators discussed an early hypothesis about Arginine (Arg) therapy as treatment for vaso-occlusive episdes (VOEs) in patients with sickle cell disease (SCD).

Almost 25 years later, Dr. Claudia Morris concluded 2022 by presenting phase 2 data at the 64th ASH Annual Meeting that demonstrated the therapy's benefit, and her original premise. Her team's phase 3 trial evaluating Arg is currently underway.

The primary outcome for the latest data was total parenteral opioid use in milligrams per kilo of IV morphine. Despite the unexpected interruptions of severe pain experienced by this patient population, treatments for acute pain related to sickle cell disease are lacking.

"Important secondary outcome measures included time to crisis resolution—which is a favorite outcome measure with the FDA when you're looking at a new drug, and wanting to move it forward in a regulatory fashion for sickle cell disease and acute pain in the emergency department," she explained in depth with HCPLive.

Morris is a professor of pediatrics and emergency medicine at Children's Healthcare of Atlanta, Research Director of Pediatric Emergency Medicine, in addition to serving as Director of Clinical & Translational Research at Emory University, and practices Emergency Medicine in the Pediatric Emergency Department.

The study population consisted of children and young adults aged 3-21 who presented to the emergency department and required IV opioid treatment for a VOE.

"What was interesting is, is that when we honed in on to the younger kids, it turns out that that change in opioid use increased to 80% in the placebo arm, when we looked at the younger kids under 17," Morris explained.

After this video, watch more of Dr. Claudia Morris's interview with HCPLive.