Survey: AHP Patients and Caregivers Face High Disease Burden in the UK

A recent study, titled “Patient and caregiver experiences of living with acute hepatic porphyria in the UK: a mixed method study,” investigated the disease burden faced by AHP patients and those closest to them. It revealed that this patient population has a diverse array of experiences when it comes to symptoms, the severity of an episode, and treatment. It also illustrated the high burden that they face. You can find the full study in the Orphanet Journal of Rare Diseases.

About Acute Hepatic Porphyria

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.

About the Study

The study was conducted with the use of an online survey that was distributed and analyzed from January until April 2019. It focused on a range of factors, such as disease management, as well as their overall quality of life. Members of the British Porphyria Association took the survey, all of whom were invited to participate in a one-hour follow-up interview over the phone.

In the end, 38 patients and 10 caregivers completed the survey, and ten patients and three caregivers went on to the interview. The majority of respondents were female and either had or took care of someone with acute intermittent porphyria, the most common form of AHP. Results include:

  • 84% of patients had experienced an attack since being diagnosed
    • 26 patients reported an attack within the last two years, two patients never experienced an attack, and four were unsure if they ever had
  • Of the ten interviewed patients, there was a range in the severity of attacks
    • 100% reported pain, 70% reported changes in mental health, 70% reported gastrointestinal effects, and 60% reported muscle weakness and paralysis
  • In terms of management, 60% of patients reported managing their attacks at home
    • 63% utilize heme arginate either in combination with glucose therapy or by itself
    • 43% are not satisfied by existing treatments, but 42% are satisfied
      • Many patients expanded on their dissatisfaction, stating that low awareness plays a role
  • Looking at symptoms, 94% of patients who reported an attack stated that experience chronic symptoms in between episodes
    • These symptoms include emotional distress, fatigue, pain, and trouble sleeping
  • Patients who experienced three or more attacks within the span of a year report a heavier disease burden than patients who experienced fewer than three episodes
    • Interviews revealed that patients are impacted in their work, household chores, self-care, independence, mobility, and daily activities
  • Caregivers reported that their relationships, finances, and employment were impacted the most

These results clearly demonstrate an unmet medical need within the acute hepatic porphyria patient population. Further research is necessary to investigate the burden faced and how to lessen it, but this survey marks a good start.

Find the source article here.

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