Results from XLH Community Impact Survey Underscore Urgency to Address Critical Gaps in Care

PR Newswire · 10/15 12:00
  • The XLH Network and Kyowa Kirin publish findings from community survey, accompanying white

    paper
  • New evidence shows wide-ranging impact of XLH, including finances, daily activities, and mental

    health
  • Survey insights will be shared at the NORD Breakthrough Summit in Washington, D.C., October

    20-22

PRINCETON, N.J., Oct. 15, 2024 /PRNewswire/ -- Kyowa Kirin, Inc., a wholly owned subsidiary of Kyowa Kirin Co. Ltd., along with The XLH Network, a non-profit patient advocacy organization, today announced the publication of Insights for Change: Findings from the XLH Community Impact Survey, an inside look at the experiences of people living with X-linked hypophosphatemia (XLH), a rare, progressive, genetic disease that impacts the bones and muscles in both children and adults.1

The white paper reveals the broader impact of XLH on U.S. patients and their families, including personal finances and out of pocket expenses; physical, mental, and social health; and the challenges faced when accessing expert care. The full survey data report and white paper are the result of a collaboration between Kyowa Kirin and The XLH Network and are now available along with other resources at XLHimpact.com.

"For generations, families living with XLH have struggled to explain the disease to many of the professionals involved in their care. Without the data to back them up, patients have often reported feeling dismissed, or that their concerns were not validated," said Susan Faitos, Executive Director of The XLH Network. "Research projects like this, which focus on the actual lived experience of someone with XLH, will lead to a greater understanding of XLH and facilitate meaningful discussions between XLHers and all those involved in their care. The XLH Network is proud to be a part of this project."

More than 85 adults with XLH and caregivers of children with XLH responded to the online survey, and 56 of these respondents completed additional follow-up phone interviews. The following key findings illustrate the ongoing challenges of living with XLH.

Access to Care: 

XLHers face unique challenges accessing care. Survey respondents rated their XLH doctors as fairly knowledgeable; however, they spent considerable time and money to access their doctors. Survey respondents reported traveling on average 180 miles roundtrip, taking over three hours just to get to and from their XLH doctor's office. These travel requirements proved to be particularly costly, amounting to on average $1,000 and as much as $15,000 in annual travel costs for survey respondents.

Financial Burden: 

The broad health implications of living with XLH are reflected in the wide array of expenses reported for direct XLH care and other expenses such as home modifications, dental procedures, and travel to doctor offices. Survey respondents indicated that they spent an average of 6% – and in one case as much as 40% – of their annual household income on out of pocket (OOP) expenses related to managing or living with XLH in the last year. In addition, over the past year, 61% of respondents paid OOP for dental care, with annual costs averaging $2,775.

Impact on Daily Activities:

The survey and white paper also document the considerable toll XLH has on an individuals' day-to-day and social life. When asked to identify the most burdensome impacts on their daily lives, XLHers commonly cited absenteeism, reduced productivity at work or school, and challenges with personal relationships. Some adult survey respondents noted that XLH restricts daily activities such as getting dressed, housework, driving, shopping, and cooking.

Mental Health Impact:

XLH can strain patients' and families' mental and emotional health resources. A majority of respondents – 83% of adults and 90% of children – reported at least one mental health issue related to their XLH. While most expressed concerns about their overall mental health, very few said they were seeing a mental health professional to address these concerns (13% of adults, 3% of children).

Transition of Care: 

Because of the lifelong, progressive nature of XLH, most XLHers are diagnosed during childhood but continue to experience symptoms into adulthood.1 Nevertheless, an overwhelming majority (94%) of adult XLHers who completed the survey reported they did not formally transition their care from a pediatrician to an adult healthcare professional (HCP). When asked for a reason, 14 of the 49 adults who were diagnosed before age 18, stated they were advised the disease "would go away over time."

"Kyowa Kirin is deeply committed to listening to and working with the XLH community to advance the understanding of XLH and the challenges this disease places on patients and their families," said Lauren Walrath, Vice President of Public Affairs at Kyowa Kirin North America. "As this survey makes clear, the needs of every XLHer are unique and while progress has been made, more work is needed to make care accessible and to ease the burden on families. We look forward to working with The XLH Network, healthcare providers, and other stakeholders on solutions that provide meaningful change at the community level."

About the XLH Community Impact Survey

The XLH Community Impact Survey is a collaboration between Kyowa Kirin and The XLH Network. The purpose of the survey was to quantify and deepen understanding of the lived experiences of adults, caregivers, and children with XLH in the United States. The survey evaluated the impact of XLH on personal finances and physical, mental, and social health, as well as access to expert quality care. Between November 2023 and February 2024, a total of 87 adults living with XLH and caregivers of children with XLH completed an online survey, with follow-up phone interviews conducted with 56 of the respondents.

Survey responses reflect respondent variation across key demographics such as age, geography, and insurance status, and may not reflect the experiences of all individuals with XLH. Adult males, young adults aged 18-44, and individuals from rural settings were under-represented among respondents. Engage Health designed, fielded, and conducted analysis of the survey with analytic and editorial support from SmithSolve and input from The XLH Network and Kyowa Kirin, Inc.

To learn more about XLH and the survey results, and to access additional resources, visit XLHimpact.com.

X-linked hypophosphatemia

X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults.1 In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health.1 This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine.1 When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in children and adults (osteomalacia).1

In children, XLH typically appears as bowed legs or knock knees.1 Over time, bone weakening can also lead to bone abnormalities in the legs, delayed growth, and short stature.1 In adults, XLH may cause osteomalacia, fractures and pseudo-fractures, and hypophosphatemia.1

About Kyowa Kirin

Kyowa Kirin aims to discover novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients affected by severe and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across Japan, North America, and EMEA/International.

You can learn more about Kyowa Kirin North America at: kkna.kyowakirin.com or LinkedIn: Kyowa Kirin Inc. U.S.

About The XLH Network

The XLH Network supports families and individuals living with X-Linked Hypophosphatemia (XLH). Our mission includes promoting awareness and education, providing opportunities for connection and community, and to foster the search for a cure. Our vision is early, accurate diagnosis; readily available, well-managed treatment; and healthy quality of life for those who are affected with XLH and related disorders.

COR-US-CRY-0096 October 2024

1Dahir K, et al. X-linked hypophosphatemia: a new era in management. J Endocr Soc. 2020;4(12):bvaa151.

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SOURCE Kyowa Kirin