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. 2023 Jun 9:17:1393-1405.
doi: 10.2147/PPA.S391025. eCollection 2023.

Young XLH Patients-Reported Experience with a Supportive Care Program

Anya Rothenbuhler  1 , Iva Gueorguieva  2 , Lydia Lichtenberger-Geslin  3 , Christelle Audrain  1 , Sylvie Soskin  4 , Candace Bensignor  5 , Sylvie Rossignol  4 , Aurélia Bertholet-Thomas  6 , Lorelei Naudeau  7 , Justine Bacchetta  6 , Agnès Linglart  1   8
Affiliations

Young XLH Patients-Reported Experience with a Supportive Care Program

Anya Rothenbuhler et al. Patient Prefer Adherence. .

Abstract

Purpose: X-linked hypophosphatemia (XLH) is a rare, chronic, genetic condition characterized by renal phosphate wasting and abnormal bone and teeth mineralization. It represents a challenging and multifaceted disease that causes wide-ranging impacts on patients' lives. In this context, a scientific committee has designed a support initiative for patients treated for XLH: the aXess program. We sought to determine if a patient support program (PSP) could help XLH patients cope with their condition.

Methods: During the 12 months of participation in the aXess program, XLH patients were contacted by phone by a nurse to coordinate their treatment, ensure treatment adherence, and provide motivational interviews. A Pediatric QOL inventory was conducted on all participants at enrollment (D0), at month 6, and month 12.

Results: Altogether, a total of 59 patients were enrolled in the program. Most patients reported an improvement in QOL in all examined dimensions by month 12 (physical, emotional, social, and school, 85.4 ± 0.2 at month 12 versus 75.6 ± 0.3 at enrollment, p<0.05). Patients were very satisfied with the program, with a mean overall satisfaction score of 9.8 ± 0.6 (on a scale from 0 to 10) at month 6 and 9.2 ± 1.5 at month 12.

Conclusion: Our findings indicate that this program might improve the QOL for patients with chronic conditions such as XLH through patient education, therapy adherence, motivational interviews, and frequent follow-up. It links the home environment and overall illness management, bringing patients, families, and caregivers together.

Keywords: XLH; aXess support program; children; hypophosphatemia; nurses; patient support program; quality of life.

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Conflict of interest statement

JB, AR and LL report speaker and consulting fees from Kyowa Kirin. AL received fees through her institution for the Phase I/II trial and a research grant from Kyowa Kirin International. LN is an employee of Webhelp Medica. IG reports consulting fees from XLH registry advisory board, outside the submitted work. The authors report no other conflicts of interest in this work.

Figures

Figure 1
Figure 1
Flow chart. The outline of the selection and flow of XLH patients.
Figure 2
Figure 2
Mean change from baseline in PedSQL Generic Core Scales summary scores (Global and sub-domains Physical, Emotional, Social, School functioning) after 6 and 12 months aXess supports. *p < 0.05 for all changes from baseline.
Figure 3
Figure 3
Percentage of response to each PedSQL questionnaire item provided by XLH patients supported by aXess support program at DO, M6 and M12 - subgroups of patients initiating treatment and patients already receiving treatment.
See this image and copyright information in PMC

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