Table 2.
Vosoritide follow-up protocol from the French Early Access Program [39]
Data elements of interest | Day 0 visit (Treatment start) |
Teleconsultation (Month 1) |
Month 3 | Month 6 and every 6 months | End of follow-up |
---|---|---|---|---|---|
Documentation of achondroplasia | √ | ||||
Demographics | √ | ||||
Medical and surgical history | √ | ||||
Clinical exam | √ | √ | √ | ||
Vital signs including blood pressure | √ | √ | √ | ||
Detailed anthropometric and morphological measurements |
√ | √ (annual) | |||
X-ray of left hand and one knee | √ (annual) | ||||
Tanner stages | √ | √ (annual) | |||
Medical and surgical events (incl. ACH-related interventions or investigations including blood test results) |
√ | √ | √ | ||
Administration, prescribing and adherence information for vosoritide | √ | √ | √ | √ | √ |
Visits healthcare professional | √ | √ | √ | ||
Adverse event collection | √ | √ | √ | √ | √ |
Concomitant and new medications | √ | √ | √ | √ | √ |
ACH achondroplasia