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Adapted from Whyte et al, 2014. Kaplan-Meier plot of survival (time from birth to death). Overall survival was 27%. The probability of survival (95% CI) was 42% (0.277, 0.550), 31% (0.189, 0.444), 29% (0.170, 0.421), and 27% (0.152, 0.398) at 1, 2, 3, and 4 years, respectively, after which it remained constant (therefore, figure has been truncated).4
Data from a noninterventional, retrospective chart review study designed to understand the natural history of 48 patients ≤5 years of age with severe perinatal- and infantile-onset HPP. Patients included in the study were those diagnosed with HPP based on at least one of the following: serum biomarker levels (below-normal ALP and above-normal pyridoxal 5‘-phosphate [PLP] or phosphoethanolamine [PEA]), below-normal ALP and radiographic abnormalities, or genetic analysis of the ALPL gene. Additionally, onset of HPP must have occurred prior to 6 months of age based on signs that included at least one of the following: respiratory compromise, rachitic chest deformity, and/or vitamin B6–responsive seizures.4
Lifetime fracture prevalence is lower in the general population than in patients with hypophosphatasia4,11,16,17,e
bData from a noninterventional, retrospective chart review study designed to understand the natural history of 48 patients ≤5 years of age with severe perinatal- and infantile-onset HPP. Patients included in the study were those diagnosed with HPP based on at least one of the following: serum biomarker levels (below-normal alkaline phosphatase and above-normal PLP or PEA), below-normal alkaline phosphatase and radiographic abnormalities, or genetic analysis of the ALPL gene. Additionally, onset of HPP must have occurred prior to 6 months of age based on signs that included at least one of the following: respiratory compromise, rachitic chest deformity, and/or vitamin B6–responsive seizures.4
cData from a retrospective, multinational, noninterventional natural history study of childhood HPP in patients 5 to 15 years of age (N=32).18
dCombined data from the HPP Impact Patient Survey/HPP Outcomes Survey Telephone (HIPS/HOST), an Internet questionnaire and telephone survey that queried demographics, HPP-related illness history, disease progression, and health-related quality of life. One hundred twenty-five adults participated.7
eData from a self-reported survey of a nationally representative general population sample of 45,293 individuals in England, plus a special boost sample of 10,111 drawn from the ethnic minority population.17
fData from HIPS, an Internet questionnaire that queried demographics, HPP-related illness history, disease progression, and health-related quality of life. Eighty-nine adults participated.7
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