A national register based study examining the prevalence, comorbidities, healthcare resource utilisation and burden of illness of hereditary hypophosphatemia in Demark

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

Non-interventional study

• Non-interventional study design:
  Case-control

Study drug and medical condition

• Medical condition to be studied:
  Hereditary hypophosphataemic rickets

Population studied

• Age groups:
  • Preterm newborn infants (0 – 27 days)
  • Term newborn infants (0 – 27 days)
  • Infants and toddlers (28 days – 23 months)
  • Children (2 to < 12 years)
  • Adolescents (12 to < 18 years)
  • Adults (18 to < 46 years)
  • Adults (46 to < 65 years)
  • Adults (65 to < 75 years)
  • Adults (75 to < 85 years)
  • Adults (85 years and over)
• Estimated number of subjects:
  100

Study design details

• Main study objective:
  The objective of this study identify the prevalence and incidence of a confirmed diagnosis of hereditary hypophosphatemia.
• Outcomes:
  To identify the prevalence and incidence of a confirmed diagnosis of hereditary hypophosphatemia. To examine the natural history of a patient population with a confirmed diagnosis of hereditary hypophosphatemia (called the case population), in detail, to understand the life course of the disease and the use of healthcare resource and social benefits, key co-morbidities and other clinical outcomes and mortality across different age cohorts.
• Data analysis plan:
  This is a case-controlled study with a control population (the control population is the case population multiplied by 50 and matched by gender and year of birth). The two populations will be compared according to age groups for comorbidity, burden of disease and costs to the public. Data will be described according to the number of unique persons in each stratum of the subpopulation. Descriptive statistics will be calculated using Paired t-test, Wilcoxon signed ranks test, McNemars test, optionally time rank analysis as well as Kaplan-Meier estimator. Data will be presented as mean and standard deviation (SD) or median (interquartile range IQR or range) as appropriate. Number of events and percent of the population, as appropriate.