Table 1.
Schedule of assessments
| Procedures/items | Patients aged <18 years | Patients aged ≥18 years | ||||||||||||||
| Retrospective data | Informed consent and enrolment | Observation period | Retrospective data | Informed consent and enrolment | Observation period | |||||||||||
| M3* | M6* | Y1 | Y2 | Y3 | Y4 | Y5/end of observation | M3* | M6* | Y1 | Y3 | Y5/end of observation | |||||
| Informed consent | ○ | ○ | ||||||||||||||
| Demographic and baseline characteristics | ○ | ○ | ||||||||||||||
| Inclusion/exclusion criteria | ○ | ○ | ||||||||||||||
| Height, body weight | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○† | ○ | ○ | ○ | ○ | ○ | ○ |
| Tanner stage | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | |||||||
| Blood pressure | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ||
| Radiography (wrists, knees, long legs) | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○† | ||||||
| Radiography (sites of symptoms, spine) | ○ | ○ | ○ | ○ | ○ | ○ | ||||||||||
| CT | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ |
| MRI | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ |
| Renal ultrasonography | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ |
| Dental assessment | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ||
| Use of drugs for the underlying disease and its complications | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ | ○ |
| Bone mineral density | ○ | ○ | ○ | ○ | ○ | ○ | ||||||||||
| Active fractures (including pseudofractures) | ○ | ○ | Enter every new event as appropriate | ○ | ○ | Enter every new event as appropriate | ||||||||||
| Surgery for the underlying disease and its complications | ○ | ○ | Enter every operation as appropriate | ○ | ○ | Enter every operation as appropriate | ||||||||||
| Laboratory assessments (local): blood chemistry, urinalysis | ○ | ○ | Enter every test as appropriate | ○ | ○ | Enter every test as appropriate | ||||||||||
| Laboratory assessments (central): blood chemistry, urinalysis | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ||
| Complications of the underlying disease | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● |
| Sitting height, arm length, leg length | ● | ● | ● | ● | ● | ● | ● | ● | ||||||||
| Motor function | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ||||
| QOL | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ||||
| Loss of working/learning opportunities | ● | ● | ● | ● | ● | ● | ● | ● | ● | ● | ||||||
| Serious adverse event | ○ | Enter every event as appropriate | ○ | Enter every event as appropriate | ||||||||||||
●Items are specific for the study. ○Data to be entered/submitted if available at the medical institution.
*Patients who receive treatment with burosumab will undergo additional evaluations at 3 and 6 months.
†Data during the period of <18 years of age to be collected retrospectively if available at the medical institution.
QOL, quality of life; Y, year.