Burden of disease
Among children and adolescents with hypophosphatasia (HPP):
Data adapted from Rush ET, et al. 2019. The study consisted of HPP Impact Patient Survey (HIPS; n=44) and HPP Outcomes Study Telephone interview (HOST; n=15).1 The 59 paediatric responders either reported by themselves or their carers reported. Survey questions were used to capture information on patient demographics, HPP-related medical history, mobility and health-related quality of life.1

* HIPS only.1

† Self-reported (or carer/family member-reported) inability to perform activities of daily living. Information in brackets is the reason given for the specific inability. Five patients (or carers/family members) did not respond to this part of the HIPS.1

‡ Data presented were assessed by both HIPS and HOST (n=59).1
Among children and adolescents with hypophosphatasia (HPP):
Among children and adolescents with hypophosphatasia (HPP):
* The Global HPP Registry is an observational, longitudinal, multinational long-term study collecting data on HPP diagnosis, history, clinical course, symptoms (including multisystemic aspects of disease) and burden of illness from patients who have a diagnosis of HPP.2 304 adults with a confirmed diagnosis of HPP and who met the analysis criteria were included in this analysis.2

† Of those with available data.3

‡ The HIPS consists of approximately 40 questions on demography, medical history and functional abilities (n=89).3 The HOST consists of approximately 37 questions covering demography, medical history and physical function (n=36).3 Across the two surveys, 125 patients were adults aged 18 years or older at the time of surveying.3
HPP has a high burden of disease and may cause severe limitations at every stage of life, drastically impacting health-related quality of life1–4
Rush ET, et al. Burden of disease in pediatric patients with hypophosphatasia: results from the HPP Impact Patient Survey and the HPP Outcomes Study Telephone interview. Orphanet J Rare Dis. 2019;14(1):201. Seefried L, et al. Burden of Illness in Adults With Hypophosphatasia: Data From the Global Hypophosphatasia Patient Registry. J Bone Miner Res. 2010;35(11):2171–2178. Weber TJ, et al. Burden of disease in adult patients with hypophosphatasia: Results from two patient-reported surveys. Metabolism. Metabolism. 2016;65(10):1522–1530. Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013;10 (Suppl 2):380–388.