The Impact Of Fibrodysplasia Ossificans Progressiva (Fop) on Patients and their Family Members: Results From an International Burden of Illness Survey

Plain Language Summary

What is this article about?

Fibrodysplasia ossificans progressiva (also known as FOP) is a very rare genetic condition. In FOP, bone forms in places where it would not normally, such as muscles, tendons, and ligaments. This leads to loss of movement over time for people with FOP. Currently, there is very little information on the relationship between the physical impact, quality of life impact, and economic impact of the condition on people with FOP and their families.

What was done?

To address this gap, the first international FOP burden of illness survey was done between January 18 and April 30, 2021. People with FOP, referred to as the ‘patient’ population, and their family members took part in the online survey. This was available across 15 countries and in 11 languages. Researchers used multiple assessments to measure patients’ movement and ability to carry out daily activities, quality of life for patients and family members, use of adaptive tools (also commonly referred to as living adaptations) by patients, and the impact of FOP on employment for patients and family members.

What were the results?

The survey received 463 responses in total (219 patients and 244 family members). The results show that older patients have greater loss of movement than younger patients, and this can have a negative impact on their quality of life. Also, as movement and the ability to carry out daily activities become more difficult, patients tend to rely on more living adaptations. There is also a negative impact of FOP on employment decisions. As a result, FOP may have a large financial impact that is highest for older patients and their families. There may also be a large financial impact on healthcare systems.

What do the results mean?

The results of this survey provide valuable information that can be used to improve care, resources, and support for people with FOP and their family members.

Clinical Trial Registration: NCT04665323 (ClinicalTrials.gov)

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Keywords::

• burden of illness
• economic impact
• fibrodysplasia ossificans progressiva
• FOP
• lay summary
• plain language summary
• quality of life
• rare diseases

This article is related to:

The impact of fibrodysplasia ossificans progressiva (FOP) on patients and their family members: results from an international burden of illness survey

Acknowledgments

The authors thank all participants involved in the study and the FOP study team which includes Michelle Davis, Executive Director of the IFOPA, Adam Sherman, former Research Director of the IFOPA, and the following FOP community advisors, who all contributed to the design of this study: Anna Belyaeva, Russia; Christopher Bedford-Gay, UK; Amanda Cali, USA; Julie Collins, Australia; Suzanne Hollywood, USA; Antoine Lagoutte, France; Moira Liljesthröm, Argentina; Karen Munro, Canada; Nancy Sando, USA. We thank Marin Wallace, Canada and Roger zum Felde, Germany for their contributions to this project prior to their passing. The authors would like to acknowledge the following national FOP associations for their valuable contributions: Fundación FOP (Argentina), FOP Brasil (Brazil), Canadian FOP Network, FOP France, FOP eV (Germany), FOP Italia Onlus (Italy), J-FOP (Japan), FOP Mexico, FOP Polska (Poland), FOP Russia, Korean FOP Overcome Family (KFOPOF; South Korea [Republic of Korea]), Asociación Española de Fibrodisplasia Osificante Progresiva (AEFOP; Spain), Svenska FOPföreningen (Sweden; members from Norway, Denmark, and Finland), and FOP Friends (United Kingdom). The survey was carried out by Engage Health (Eagan, Minnesota, USA). Translation of the survey was managed by the specialist vendor TransPerfect Life Sciences and validated by local affiliates of the Sponsor.

Financial & competing interests disclosure

MAM: Research investigator: Clementia/Ipsen, Regeneron; Non-paid consultant: BioCryst, Blueprint Medicines, Daiichi Sankyo, Incyte, Keros; Advisory board (all voluntary): IFOPA Registry Medical Advisory Board, Incyte, International Clinical Council on FOP; non-restricted educational fund from Excel and Catalyst sponsored by Ipsen; KST: Research funding from Clementia/Ipsen and Regeneron; MD: Member of the Rare Bone Disease Alliance Steering Committee and the Rare Bone Disease Summit Steering Committee; AC: Trustee of The Radiant Hope Foundation, Trustee of the Ian Cali FOP Research Fund/Penn Medicine, Co-founder and Advisory Board member Tin Soldiers Patient Identification Program, Executive Producer Tin Soldiers documentary, Past IFOPA Chairman of the Board, Executive Associate of the International Clinical Council on FOP (all voluntary); CBG: IFOPA Board Member, IFOPA Executive Committee Member, FOP Friends Chair and Trustee, International President’s Council Chair; ML: Co-founder and President of Fundación FOP, Argentine IPC Representative, IFOPA Research Committee member, past IFOPA Board member (all voluntary); SH: Committee member on the IFOPA LIFE Award Program, Committee member on the IFOPA Fundraising Committee; KC, EAB: Employees and shareholders of Ipsen; ASG: Employee of Atlanstat, contractor for Ipsen; JDW: Employee of Ipsen at the time of the study; FSK: Research investigator: Clementia/Ipsen, Regeneron; Advisory Board: IFOPA Registry Medical Advisory Board; Founder and Past-President of the International Clinical Council on FOP.