Fig. 4

Proportion of pediatric patients with HPP experiencing an improvement, worsening, or no change in signs/symptoms compared with 5 years ago. This was assessed by HOST only (n = 15). Patients’ first HPP-related signs/symptoms were excluded from this analysis; respondents could report up to three signs/symptoms that manifested after their first HPP signs/symptoms. Other HPP signs/symptoms included recurrent respiratory tract infections, nephrocalcinosis, lack of appetite, difficulty in gaining weight, tight hamstrings/calves, short stature, growth disorders, low red blood cell count, and other dental/oral problems (abocclusion, malposition of teeth, dysfunction of the tongue). Data for any sign/symptom are the numbers of patients who reported an improvement, worsening, or no change in their additional signs/symptoms compared with 5 years prior to their participation in the survey in response to the free-text survey question “What additional symptoms related to HPP do you have today?”. ^aOne respondent did not indicate whether their signs/symptoms had improved or worsened, or were unchanged compared with 5 years ago. ^bTwo respondents did not indicate whether their signs/symptoms had improved, worsened, or were unchanged compared with 5 years ago. HOST, Hypophosphatasia Outcomes Study Telephone interview; HPP, hypophosphatasia