According to an article published by Alnylam Pharmaceuticals, a recent survey has provided insight into the burden experienced by acute hepatic porphyria (AHP) patients. Titled the POWER (POrphyria Worldwide Patient Experience Research) study, this survey investigated the mental, emotional, and physical impacts that patients face.

About the Survey

92 AHP patients participated in the survey, all of whom spoke to the burdens that they and their inner circle face. Results include:

• Substantial emotional burden
  • Examples include depression and anxiety, ranging from moderate to severe
• Significant levels of pain
• Significant work impairment

These and further results will be presented at the American College of Gastroenterology (ACG) Annual Scientific Meeting 2021. Beyond this survey, data and insights from the Phase 3 ENVISION study will be presented as well. This study investigated GIVLAARI, focusing on its efficacy, safety, and the disease burdens faced by patients.

You can read more about the survey here, and more about the ENVISION study here and here.

About AHP

AHP falls under the larger group of porphyria, and it occurs when the body lacks an enzyme needed to produce heme, a part of hemoglobin. This leads to an accumulation of porphyrins and porphyrin precursors, which then results in oxygen depletion. Looking more specifically to APH, this disease pertains to the liver, and it can be further divided into subcategories as well. It is characterized by attacks on the organs in the large cavity of the torso and parts of the nervous system. These attacks cause symptoms such as muscle weakness, convulsions, anxiety, irritability, abdominal cramping, confusion, sensory loss, and auditory or visual hallucinations. In terms of triggers, stress, medications, alcohol, hormonal changes, a diet too low in calories, and infections can all bring on an attack. To treat these symptoms, doctors focus on pain management, reversing attacks, and preventing any neurological symptoms. Hemin is often given through an IV, as is glucose if necessary. In severe cases, one may require a liver transplant.