Five poster presentations provide greater understanding of patient profiles and real-world experiences of people living with XLH

五张海报展示可以更好地了解XLH患者的个人资料和现实经验

PRINCETON, N.J., Oct. 12, 2023 /PRNewswire/ -- Kyowa Kirin, Inc., an affiliate of Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company based in Japan, will share new research that advances understanding about the real-world experiences and clinical characteristics of children, adolescents, and adults living with X-linked hypophosphatemia (XLH), a rare genetic metabolic bone disease.

新泽西州普林斯顿，2023年10月12日/PRNewswire/-Kyowa Kirin，Inc.，Kyowa Kirin Co.，Ltd。（Kyowa Kirin，TSE:4151）的附属公司，该公司是一家总部设在日本的全球特种制药公司，将分享新的研究，促进对患有X连锁低磷血症（XLH）的儿童，青少年和成人的现实经验和临床特征的理解，一种罕见的遗传代谢性骨病。

The findings will be presented in a series of poster presentations at the American Society for Bone and Mineral Research (ASBMR) 2023 annual meeting, which takes place October 13-16 in Vancouver, BC, Canada..

这些发现将在10月13日至16日在加拿大卑诗省温哥华举行的美国骨与矿物研究学会（ASBMR）2023年会上发表一系列海报展示。。

'Individuals living with XLH face a lifelong struggle of managing their disease and progressively worsening morbidities, which negatively impact their life,' said Zhiyi Li, MA, MBA, Director of Health Economics and Outcomes Research at Kyowa Kirin North America. 'The breadth of the research we are presenting at ASBMR reflects our commitment to helping inform a more complete understanding of XLH and its' management.'.

Kyowa Kirin North America卫生经济与成果研究主任，马志毅说：“与XLH一起生活的个人面临着管理疾病和逐渐恶化的疾病的终身斗争，这对他们的生活产生了负面影响。”我们在ASBMR上进行的广泛研究反映了我们致力于帮助您更全面地了解XLH及其“管理”。

Kyowa Kirin will present the following posters at the ASBMR annual meeting:

Kyowa Kirin将在ASBMR年会上展示以下海报：

'Real-world characteristics and disease history of patients with X-linked hypophosphatemia treated with burosumab: a United States claims database study' (Poster #SAT-493)

'burosumab治疗X连锁低磷血症患者的真实世界特征和疾病史：美国索赔数据库研究'（海报＃SAT-493）

Lead author: Kathryn Dahir, MD, Vanderbilt University Medical Center

主要作者：范德比尔特大学医学中心医学博士Kathryn Dahir

Poster Session I; Saturday, October 14, 1:30 – 3:00 pm PDT

海报会议I；星期六，10月14日，下午1:30-3:00 PDT

'Clinical and treatment characteristics of pediatric and adult patients with familial hypophosphatemia compared with demographically matched controls' (Poster #SAT-519)

'与人口统计学匹配的对照组相比，儿童和成人家族性低磷血症患者的临床和治疗特征'（海报＃SAT-519）

Lead author: Zhiyi Li, MA, MBA, Kyowa Kirin North America

主要作者：Zhiyi Li，MA，MBA，Kyowa Kirin北美

Poster Session I; Saturday, October 14, 1:30 – 3:00 pm PDT

海报会议I；星期六，10月14日，下午1:30-3:00 PDT

'Association between work productivity and characteristics of adults with X-linked hypophosphatemia: an analysis of the XLH Disease Monitoring Program' (Poster #SAT-496)

“工作效率与X连锁低磷血症成人特征之间的关联：XLH疾病监测计划的分析”（海报＃SAT-496）

Lead author: Aliya Khan, MD, McMaster University

主要作者：麦克马斯特大学医学博士Aliya Khan

Poster Session I; Saturday, October 14, 1:30 – 3:00 pm PDT

海报会议I；星期六，10月14日，下午1:30-3:00 PDT

'Age at diagnosis of XLH amongst children with and without a family history: findings from the International XLH Registry' (Poster #SUN-500)

“有或没有家族史的儿童中XLH的诊断年龄：国际XLH注册中心的发现”（海报＃SUN-500）

Lead author: Suma Uday, MBBS, FRCPCH, PhD, Birmingham Women's and Children's Hospital (UK)

主要作者：Suma Uday，MBBS，FRCPCH，PhD，伯明翰妇幼医院（英国）

Poster Session II; Sunday, October 15, 1:30 – 3:00 pm PDT

Poster Session II; Sunday, October 15, 1:30 – 3:00 pm PDT

'A patient-centered and multi-stakeholder co-designed, mixed method, observational, prospective study protocol: Example of the adolescent experience of treatment for X-linked hypophosphatemia (XLH)' (Poster #SUN-496)

“以患者为中心和利益相关者共同设计，混合方法，观察性，前瞻性研究方案：X连锁低磷血症（XLH）治疗的青少年经验示例”（海报＃SUN-496）

Lead author: Vrinda Saraff, MD, Birmingham Women's and Children'sHospital (UK)

主要作者：Vrinda Saraff，医学博士，伯明翰妇女儿童医院（英国）

Poster Session II; Sunday, October 15, 1:30 – 3:00 pm PDT

Poster Session II; Sunday, October 15, 1:30 – 3:00 pm PDT

About X-linked hypophosphatemia

关于X连锁低磷血症

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

X连锁低磷血症是一种罕见的终生遗传性疾病，可影响儿童和成人的骨骼和肌肉。在XLH患者中，身体无法保持足够的磷，磷是骨骼健康的必需矿物质。这是由于过量的成纤维细胞生长因子23（FGF23）的产生，导致身体通过尿液释放过多的磷。

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 adults (osteomalacia)..

当磷水平过低（低磷血症）时，它会导致儿童（佝偻病）和成人成熟骨骼（骨软化症）的生长骨骼软化和减弱。。

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

在儿童中，XLH通常表现为弯曲的腿或膝盖弯曲。随着时间的流逝，骨骼变弱还可能导致腿部骨骼异常，生长延迟和身材矮小。在成年人中，XLH可能引起骨软化，骨折和假性骨折以及低磷血症。

About CRYSVITA® (burosumab-twza) Injection

关于CRYSVITA®（burosumab twza）注射液

CRYSVITA is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, which binds to and inhibits the biological activity of FGF23, the underlying cause of hypophosphatemia in XLH. By blocking FGF23, CRYSVITA helps to restore phosphorus reabsorption in the kidneys and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium..

CRYSVITA是Kyowa Kirin发现的重组全人单克隆IgG1抗体，可结合并抑制FGF23的生物学活性，FGF23是XLH中低磷血症的根本原因。通过阻断FGF23，CRYSVITA有助于恢复肾脏中磷的重吸收，并增加活性维生素D的产生，从而增强肠道对磷酸盐和钙的吸收。。

U.S. CRYSVITA Indication

U、 S.CRYSVITA指示

CRYSVITA is a fibroblast growth factor (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older.

CRYSVITA是一种成纤维细胞生长因子（FGF23）阻断抗体，适用于治疗6个月及以上的成人和儿童患者的X连锁低磷血症（XLH）。

Important Safety Information

重要的安全信息

CONTRAINDICATIONSCRYSVITA is contraindicated:

contraindicationsCrysvita是contraindicated：

In concomitant use with oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol) due to the risk of hyperphosphatemia.

由于高磷血症的风险，与口服磷酸盐和/或活性维生素D类似物（例如骨化三醇，帕立骨化醇，多塞钙化醇，钙化二醇）同时使用。

When serum phosphorus is within or above the normal range for age. In patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism.

当血清磷在正常年龄范围内或以上时。严重肾功能不全或终末期肾病患者，因为这些情况与矿物质代谢异常有关。

WARNINGS AND PRECAUTIONS

警告和注意事项

Hypersensitivity

过敏

Hypersensitivity reactions (e.g., rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment.

CRYSVITA患者有超敏反应（如皮疹，荨麻疹）。如果发生严重的超敏反应，请停止CRYSVITA并开始适当的治疗。

Hyperphosphatemia and Risk of Nephrocalcinosis

高磷血症和肾钙化的风险

Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient's serum phosphorus levels.

血清磷增加至正常上限以上可能与肾钙质沉着症风险增加有关。对于已经服用CRYSVITA的患者，可能需要根据患者的血清磷水平中断剂量和/或减少剂量。

Injection Site Reactions

注射部位反应

Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment.

CRYSVITA的施用可能导致局部注射部位反应。如果发生严重的注射部位反应并进行适当的治疗，请停止CRYSVITA治疗。

ADVERSE REACTIONS

不良反应

Pediatric Patients

儿科患者

Adverse reactions reported in 10% or more of CRYSVITA-treated pediatric XLH patients across three studies are: pyrexia (55%, 44%, and 62%), injection site reaction (52%, 67%, and 23%), cough (52%), vomiting (41%, 48%, and 46%), pain in extremity (38%, 46%, and 23%), headache (34% and 73%), tooth abscess (34%, 15%, and 23%), dental caries (31%), diarrhea (24%), vitamin D decreased (24%, 37%, and 15%), toothache (23% and 15%), constipation (17%), myalgia (17%), rash (14% and 27%), dizziness (15%), and nausea (10%)..

在三项研究中，CRYSVITA治疗的小儿XLH患者中有10%或更多的不良反应报告为：发热（55%，44%和62%），注射部位反应（52%，67%和23%），咳嗽（52%），呕吐（41%，48%和46%），四肢疼痛（38%，46%和23%），头痛（34%和73%），牙脓肿（34%，15%和23%），龋齿（31%），腹泻（24%），维生素D减少（24%，37%和15%），牙痛（23%和15%），便秘（17%），肌痛（17%），皮疹（14%和27%），头晕（15%）和恶心（10%）。。

Postmarketing experience reported in CRYSVITA-treated pediatric XLH patients: blood phosphorus increased.

CRYSVITA治疗的小儿XLH患者的上市后经验报告：血磷增加。

Adult Patients

成人患者

Adverse reactions reported in more than 5% of CRYSVITA-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain (15%), headache (13%), tooth infection (13%), restless legs syndrome (12%), vitamin D decreased (12%), dizziness (10%), constipation (9%), muscle spasms (7%), and blood phosphorus increased (6%)..

在一项研究中，超过5%的CRYSVITA治疗的成人XLH患者和至少2名超过安慰剂的患者报告的不良反应是：背痛（15%），头痛（13%），牙齿感染（13%），不安腿综合征（12%），维生素D减少（12%），头晕（10%），便秘（9%），肌肉痉挛（7%）和血磷增加（6%）。。

Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression.

椎管狭窄在XLH成人中很普遍，并且有脊髓压迫的报道。CRYSVITA疗法是否会加剧椎管狭窄或脊髓压迫尚不清楚。

USE IN SPECIFIC POPULATIONS

在特定人群中使用

There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-844-768-3544.

目前还没有关于CRYSVITA在孕妇中使用的数据来告知药物相关的不良发育结局风险。在整个怀孕期间应监测血清磷水平。在1-844-768-3544向Kyowa Kirin，Inc。报告怀孕情况。

There is no information regarding the presence of CRYSVITA in human milk or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition..

没有关于人乳中CRYSVITA的存在或CRYSVITA对产奶或母乳喂养婴儿的影响的信息。因此，应考虑母乳喂养的发育和健康益处，以及母亲对CRYSVITA的临床需求以及对CRYSVITA母乳喂养婴儿或潜在母体状况的任何潜在不利影响。。

PATIENT COUNSELING INFORMATION

患者咨询信息

Advise patients not to use any oral phosphate and/or active vitamin D analog products.

建议患者不要使用任何口服磷酸盐和/或活性维生素D类似物产品。

Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless legs syndrome induction or worsening of symptoms occur.

如果出现过敏反应，注射部位反应和不安腿综合征诱发或症状恶化，请指导患者联系他们的医生。

You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Kyowa Kirin, Inc. at 1-844-768-3544.

您可以在（800）FDA-1088或www.FDA.gov/medwatch向FDA报告副作用。您也可以在1-844-768-3544向Kyowa Kirin，Inc。报告副作用。

For important risk and use information, please see the full Prescribing Information for CRYSVITA.

有关重要风险和使用信息，请参阅CRYSVITA的完整处方信息。

About Kyowa Kirin

关于Kyowa Kirin

Kyowa Kirin strives to create and deliver novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company with a more than 70-year heritage, we apply cutting-edge science including expertise in antibody research and engineering, to address the needs of patients and society across multiple therapeutic areas including Nephrology, Oncology, Immunology/Allergy and Neurology.

Kyowa Kirin致力于创造和提供具有改变生命价值的新药。作为一家拥有70多年历史的日本全球特种制药公司，我们应用尖端科学，包括抗体研究和工程专业知识，满足患者和社会在肾脏病学，肿瘤学，免疫学等多个治疗领域的需求。/过敏和神经病学。

Across our four regions – Japan, Asia Pacific, North America and EMEA/International – we focus on our purpose, to make people smile, and are united by our shared values of commitment to life, teamwork/Wa, innovation, and integrity. You can learn more about the Kyowa Kirin North America at: https://kkna.kyowakirin.com/..

在我们的四个地区-日本，亚太地区，北美和EMEA/国际-我们专注于我们的目标，让人们微笑，并通过我们对生活，团队合作/华盛顿州，创新和诚信的共同价值观团结在一起。您可以在以下网址了解有关Kyowa Kirin北美的更多信息：https://kkna.kyowakirin.com/..

SOURCE Kyowa Kirin

来源Kyowa Kirin