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Burosumab treatment of X-linked hypophosphatemia patients: interim analysis of the SUNFLOWER longitudinal, observational cohort study.
Michigami, Toshimi; Kang, Hee Gyung; Namba, Noriyuki; Ito, Nobuaki; Kubota, Takuo; Shintani, Ayumi; Kabata, Daijiro; Kanematsu, Masanori; Nishida, Yayoi; Fukumoto, Seiji; Ozono, Keiichi.
Afiliação
  • Michigami T; Department of Bone and Mineral Research, Osaka Women's and Children's Hospital, Osaka Prefectural Hospital Organization, Osaka 594-1101, Japan.
  • Kang HG; Department of Pediatric Nephrology, Seoul National University Children's Hospital, Seoul 03080, Republic of Korea.
  • Namba N; Division of Pediatrics and Perinatology, Faculty of Medicine, Tottori University, Tottori 683-8504, Japan.
  • Ito N; Division of Therapeutic Development for Intractable Bone Diseases, Graduate School of Medicine and Faculty of Medicine, The University of Tokyo, Tokyo 113-0033, Japan.
  • Kubota T; Department of Pediatrics, Graduate School of Medicine, Osaka University, Osaka 565-0871, Japan.
  • Shintani A; Department of Medical Statistics, Graduate School of Medicine, Osaka Metropolitan University, Osaka 545-8585, Japan.
  • Kabata D; Department of Medical Statistics, Graduate School of Medicine, Osaka Metropolitan University, Osaka 545-8585, Japan.
  • Kanematsu M; Medical Affairs Department, Kyowa Kirin Co., Ltd., Tokyo 100-0004, Japan.
  • Nishida Y; Medical Affairs Department, Kyowa Kirin Co., Ltd., Tokyo 100-0004, Japan.
  • Fukumoto S; Department of Diabetes and Endocrinology, Tamaki-Aozora Hospital, Tokushima 779-3125, Japan.
  • Ozono K; Center for Promoting Treatment of Intractable Diseases, ISEIKAI International General Hospital, Osaka 530-0052, Japan.
JBMR Plus ; 8(8): ziae079, 2024 Aug.
Article em En | MEDLINE | ID: mdl-39015507
ABSTRACT
X-linked hypophosphatemia (XLH) is a genetic disease that results in excessive FGF23, chronic hypophosphatemia, and musculoskeletal abnormalities, with affected patients experiencing symptoms such as bone pain, bone deformity, fracture, and pseudofracture. Burosumab is a fully human monoclonal antibody that binds to FGF23, improving lowered serum 1,25(OH)2D and phosphate levels in patients with XLH. There are insufficient data on the use of burosumab, its safety, and the outcomes of treated patients in a real-world setting. The SUNFLOWER (Study of longitUdinal observatioN For patients with X-Linked hypOphosphatemic rickets/osteomalacia in collaboration With Asian partnERs) study is an ongoing longitudinal, observational cohort study of patients with XLH in Japan and South Korea. Enrollment occurred between April 2018 and December 2020. This interim analysis compared the background characteristics of patients who received burosumab with those who did not, and assessed improvements in biomarkers, physical and motor function, health-related quality-of-life (HRQOL) and other patient-reported outcome (PRO) measures, as well as the safety of burosumab treatment in 143 Japanese patients from 15 institutions over 6 mo. The patients had a median [interquartile range] age of 17.5 [11.0, 38.8] yr and 98 (68.5%) were female. Among patients aged <18 and ≥18 yr, 40/73 (54.8%) and 25/70 (35.7%) received burosumab, respectively. More patients aged ≥18 who received burosumab had bone pain at baseline vs those not treated with burosumab (6/25, 24.0% vs 2/45, 4.4%, p=.021). Patients treated with burosumab had improved serum phosphate and 1,25(OH)2D levels; moreover, rickets severity and HRQOL/PRO measures, such as pain, appeared to improve over 6 mo of burosumab treatment, and no new safety concerns were identified. This study identified trends in the background characteristics of patients with XLH who receive burosumab in real-world clinical practice. Furthermore, the results support the use of burosumab therapy in real-world settings.
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Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2024 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2024 Tipo de documento: Article