ESPE2024 Free Communications Bone, Growth Plate and Mineral Metabolism (6 abstracts)
Analysis of baseline data from the SUNFLOWER longitudinal, observational cohort study of patients with XLH: relationship between various complications and QOL
Noriyuki Namba 1 , Nobuaki Ito 2 , Toshimi Michigami 3 , Hee Gyung Kang 4 , Takuo Kubota 5 , Osamu Miyazaki 6 , Ayumi Shintani 7 , Daijiro Kabata 7 , Yayoi Nishida 8 , Seiji Fukumoto 9 & Keiichi Ozono 10
1Division of Pediatrics and Perinatology, Tottori University Faculty of Medicine, Tottori, Japan. 2Division of Therapeutic Development for Intractable Bone Diseases, Graduate School of Medicine and Faculty of Medicine, The University of Tokyo, Tokyo, Japan. 3Department of Bone and Mineral Research, Osaka Women’s and Children’s Hospital, Osaka Prefectural Hospital Organization, Osaka, Japan. 4Division of Pediatric Nephrology, Department of Pediatrics, Seoul National University Children’s Hospital, Seoul, South Korea. 5Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan. 6Department of Radiology, National Center for Child Health and Development, Tokyo, Japan. 7Department of Medical Statistics, Graduate School of Medicine, Osaka Metropolitan University, Osaka, Japan. 8Medical Affairs Department, Kyowa Kirin Co., Ltd., Tokyo, Japan. 9Tamaki-Aozora Hospital, Tokushima, Japan. 10Center for Promoting Treatment of Intractable Diseases, ISEIKAI International General Hospital, Osaka, Japan.
X-linked hypophosphatemia (XLH) is characterized by excess FGF 23, hypophosphatemia, skeletal deformities, and growth impairment. Due to the lack of large-scale and long-term observational studies, not enough evidence has been accumulated to gain consensus on optimal care. A longitudinal, observational cohort study (SUNFLOWER) was initiated in Japan and South Korea to clarify the course of XLH; delineate its physical, mental, and financial burdens; and collect information on treatment. Here, we report cross-sectional data at the time of enrollment and assess the relationship between various complications and quality of life (QOL). 77 children (mean age, 9.7 years; 67.5% female) and 70 adults (mean age, 37.6 years; 65.7% female) were enrolled. Motor function assessments utilized the 6-minute walk test (6MWT) for patients aged ≥5 years to <18 years; the Timed Up and Go Test (TUGT) and grip strength for patients aged ≥18 years. QOL was evaluated using the revised faces pain scale (FPS-R; <18 years), the 10-item short-form health survey for children (SF-10; <18 years), the brief pain inventory (BPI; ≥18 years), and the Western Ontario and McMaster Universities osteoarthritis index (WOMAC; ≥18 years). Children appeared to have low pain levels (mean FPS-R, 1.3). In contrast, adults reported mild-to-moderate pain (mean BPI pain severity, 2.02). SF-10 demonstrated a score below normal for physical functioning in children. In adults, WOMAC indicated the presence of pain, stiffness, and decreased physical function. Children demonstrated significant relationship between SF-10 physical score and ectopic ossification and related symptoms (P = 0.008) and surgery (P = 0.037) as well as between SF-10 psychosocial score and hyperparathyroidism (P = 0.029) and surgery (P = 0.021), in the analyses with proportional-odds logistic regression models. Adults demonstrated significant relationship between BPI and hypertension (worst, P = 0.017; least, P = 0.022; average, P = 0.002), height (Z-score) (average, P = 0.048; pain interference, P = 0.04), and TUGT (worst, P = 0.002; least, P = 0.004; average, P = 0.005; now, P = 0.004; pain interference, P = 0.022). There were also significant relationships between WOMAC pain scores and surgery (P = 0.005), TUGT (P = 0.003), and grip strength (P = 0.044). Significant relationship between WOMAC stiffness scores and TUGT (P = 0.007), WOMAC physical function scores and surgery (P < 0.001) and TUGT (P = 0.004) were also observed. These data spotlight areas where most effort should be focused on in order to maximize QOL in XLH patients.
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