Albireo Announces Publication of the PICTURE Study Highlighting the Caregiver Impact of PFIC, a Rare and Devastating Children’s Liver Disease
BOSTON: Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, has announced the publication of the PICTURE study in Orphanet Journal of Rare Disease. A multinational, retrospective, cross-sectional study, PICTURE evaluated and quantified the impact that progressive familial intrahepatic cholestasis (PFIC) has on caregivers and found a significant caregiver-reported burden on health-related quality of life (HRQoL), impairment of daily activities, reduced sleep, impact on work productivity, career building challenges and relationship strain. The study illustrates for the first time that PFIC levies a substantial burden that extends beyond the individuals with the disease to those caring for them.
“PFIC is a devastating diagnosis. As a mother of a child with PFIC, I could not ask for a greater gift than being my daughter’s advocate. With that said, the PICTURE study reinforces what many of us intuitively know – the challenges of caregiving are ever present and often overwhelming and exhausting,” said Emily Ventura, Chief Executive Director of PFIC Network, co-author of the PICTURE study and caregiver. “From loss of sleep to stress on finances and relationships, PICTURE reinforces that we must recognise the burden this disease puts on families and the importance of providing resources for PFIC caregivers, while continuing to fund research to improve treatment and care.”
PFIC is a spectrum of rare, paediatric, genetic diseases of cholestasis, characterised by inadequate bile secretion, often requiring liver transplantation and leading to liver failure and early death. The rare nature of PFIC has presented challenges to understanding and quantifying its impact on the daily lives of patients and their caregivers. The PICTURE study is the first and largest global study to evaluate and quantify the health-related quality of life responses from PFIC caregivers. The study showed that while caregivers report feeling fulfilment from their caregiving responsibilities, they also reported measurable negative impacts on many important health-related quality of life measures and work productivity. Specifically, the study found that:
- PFIC substantially impacted caregivers’ quality of life
- 82% of caregivers reported a strain on their relationships.
- 86% of caregivers reported difficulty sleeping.
- Notably, caregivers in the PICTURE study reported a median quality of life score of 67.7%. This score is lower in comparison with quality of life reported in a separate study of caregivers for individuals with cystic fibrosis, a similarly rare, genetic, progressive and lifespan-shortening condition (median 84.7 and 89.2 for mothers and fathers, respectively).
- On average, PFIC caregivers reported high impairment in daily activities; more than half also experienced loss of work productivity
- 50% of caregivers reported an impact of their child’s PFIC on their career-building efforts, with 73% of these citing the prevention of either their career progression or working more hours.
- A third (36%) of caregivers reported missing an average of 13 workdays in the last three months, equating to 52 workdays lost in 12 months.
- Of those who were working in paid employment, 36% stopped work due to caregiving needs, missing an average of 2.8 years of employment during their career.
“The PICTURE study provides, for the first time, a look at what it’s really like for caregivers to live with the impact of this devastating, rare disease and the effect it has every day on families and adjacent communities,” said Ron Cooper, President and CEO of Albireo. “At Albireo, we are committed to being the gold standard partner in the fight against PFIC and the findings from the PICTURE study reinforce the importance of providing access to Bylvay, the first approved drug treatment for patients with PFIC.”
PFIC is a rare and devastating disorder affecting young children that causes progressive, life-threatening liver disease. Patients with PFIC have impaired bile flow, or cholestasis, and the resulting bile build-up in liver cells causes liver disease and symptoms such as intense itching, poor sleep and diminished quality of life. Albireo is committed to advancing research in PFIC and other rare cholestatic diseases. The Company recently received approval by the U.S. FDA for Bylvay, the first drug for the treatment of pruritus in all types of PFIC. In Europe, Bylvay is approved for the treatment of PFIC. The Company is working with the community and patient advocacy groups like the PFIC Network and Children’s Liver Disease Foundation to raise awareness of PFIC and support families managing the burden of this devastating disease.