X-Linked Hypophosphatemia Management in Adults: An International Working Group Clinical Practice Guideline.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Khan Aliya A, Ali Dalal S, Appelman-Dijkstra Natasha M, Carpenter Thomas O, Chaussain Catherine, Imel Erik A, Jan de Beur Suzanne M, Florenzano Pablo, Abu Alrob Hajar, Aldabagh Rana, Alexander R Todd, Alsarraf Farah, Beck-Nielsen Signe Sparre, Biosse-Duplan Martin, Cohen-Solal Martine, Crowley Rachel K, Dandurand Karel, Filler Guido, Friedlander Lisa, Fukumoto Seiji, Gagnon Claudia, Goodyer Paul, Grasemann Corinna, Grimbly Chelsey, Hussein Salma, Javaid Muhammad K, Khan Sarah, Khan Aneal, Lehman Anna, Lems Willem F, Lewiecki E Michael, McDonnell Ciara, Mirza Reza D, Morgante Emmett, Morrison Archibald, Portale Anthony A, Rhee Yumie, Rush Eric T, Siggelkow Heide, Tetradis Sotirios, Tosi Laura, Ward Leanne M, Guyatt Gordon, Brandi Maria Luisa
Аннотация
METHODS: The IWG, consisting of 43 members as well as methodologists and a patient partner, conducted 2 systematic reviews (SRs) and narrative reviews to address key areas. The SRs addressed the impact of burosumab compared to conventional therapy (phosphate and active vitamin D) or no therapy on patient-important outcomes in adults. They also evaluated conventional therapy compared to no therapy. GRADE methodology was applied to evaluate the certainty of evidence. Non-GRADED recommendations were made in the presence of insufficient evidence to conduct SRs. These guidelines have been reviewed and endorsed by several medical and patient societies and organizations.
RESULTS: The diagnosis of XLH is based on integrating clinical evaluation, laboratory findings confirming renal phosphate wasting (following exclusion of conditions mimicking XLH), and skeletal imaging. Fibroblast growth factor 23 measurement and DNA analysis are of value in the diagnosis, if available. Pathogenic or likely pathogenic variants in the PHEX gene are confirmatory but not necessary for the diagnosis. Management requires a multidisciplinary team knowledgeable and experienced in XLH. Effective medical therapy with burosumab can improve fracture and pseudofracture healing.
MAIN CONCLUSION: In adults with XLH and fractures or pseudofractures, burosumab is recommended over no therapy (strong recommendation, GRADEd). Additionally, burosumab is suggested as the preferred treatment compared to conventional therapy (conditional recommendation, GRADEd) in the absence of fractures or pseudofractures. If burosumab is not available, symptomatic adults should be treated with conventional therapy (Non-GRADEd recommendation).