Hamilton Amber A, Faitos Susan, Jones Gin, Kinsley Athina, Gupta Rupal Naik, Lewiecki E Michael
Patient advocate, NY, USA.
The XLH Network, Inc., Albany, NY 12206, USA.
J Endocr Soc. 2022 Jun 13;6(8):bvac086. doi: 10.1210/jendso/bvac086. eCollection 2022 Aug 1.
The rare genetic disorder X-linked hypophosphatemia (XLH) is often exclusively considered to impact children, and, as such, adult patients with XLH may receive inadequate care because their symptoms are not associated with XLH. However, studies have shown that XLH has long-term adverse health consequences that continue throughout adulthood requiring comprehensive lifelong care. Indeed, XLH impacts patients' whole body, whole life, and whole family. XLH does not just affect the bones; symptoms are chronic and progressive, worsening throughout adulthood, and the burden of XLH overflows into the lives of a patient's family, friends, peers, and colleagues. To ensure early recognition, comprehensive care, and adequate management of XLH, there are key steps that clinicians can incorporate into their daily practice. These include education, a multidisciplinary approach, open communication, and support. Clinician education on rare disorders such as XLH is critical, and healthcare professionals (HCPs) should ensure that patients and their caregivers have access to XLH-related information. As a whole-body disorder, XLH requires a coordinated approach to treatment across specialties. Frequent open communication among members of the healthcare team is needed to increase HCPs' knowledge about XLH, and open communication must extend to the patient as well to ensure the patient's concerns and needs are addressed and treatment is tailored to their specific individual needs. Multiple networks of support, including social and psychological support, should be offered to patients and their families. A basic understanding that XLH affects patients' whole bodies, whole lives, and whole families is the first step toward accomplishing improved patient care.
罕见的遗传性疾病X连锁低磷血症(XLH)通常仅被认为会影响儿童,因此,成年XLH患者可能得不到充分的治疗,因为他们的症状与XLH无关。然而,研究表明,XLH会产生长期的不良健康后果,这些后果在整个成年期都会持续存在,需要全面的终身护理。事实上,XLH会影响患者的全身、一生以及整个家庭。XLH不仅会影响骨骼;症状是慢性且渐进性的,在成年期会不断恶化,XLH的负担还会蔓延到患者的家人、朋友、同龄人及同事的生活中。为确保对XLH的早期识别、全面护理和适当管理,临床医生可以在日常实践中纳入一些关键步骤。这些步骤包括教育、多学科方法、开放沟通和支持。对XLH等罕见疾病的临床医生教育至关重要,医疗保健专业人员(HCPs)应确保患者及其护理人员能够获取与XLH相关的信息。作为一种全身性疾病,XLH需要各专科之间协调一致的治疗方法。医疗团队成员之间需要频繁进行开放沟通,以增加HCPs对XLH的了解,而且开放沟通必须延伸到患者,以确保患者的担忧和需求得到解决,治疗能够根据其具体个人需求进行调整。应向患者及其家人提供包括社会和心理支持在内的多种支持网络。认识到XLH会影响患者的全身、一生以及整个家庭是实现改善患者护理的第一步。