Table 5.
For patients already prescribed vosoritide where limb surgery is being considered, in addition to considerations in Sect. 2
| Statement | Level of agreement, mean (SD) | Range | % agreement* |
|---|---|---|---|
|
Theme 1. Drivers for adding limb surgery For patients already prescribed vosoritide, the goals of limb surgery are to … | |||
| Improve functional limitations | 9.67 (0.72) | 8–10 | 100% |
| Correct deformity | 9.40 (0.99) | 7–10 | 100% |
| Increase height, lower and upper limb length beyond that achieved with vosoritide alone | 9.20 (1.08) | 7–10 | 100% |
|
Theme 2: Pre-surgery considerations For patients already prescribed vosoritide, considerations prior to initiation of limb surgery should include … | |||
| Assessing the commitment of the patient/family to undergo limb surgery | 9.67 (0.72) | 8–10 | 100% |
| Assessing the motivation behind the patient/family’s perceived need for limb surgery | 9.53 (0.74) | 8–10 | 100% |
| Establishing the growth expectations of the individual patient | 9.47 (0.83) | 8–10 | 100% |
| Establishing the amount of lengthening needed to achieve treatment goals in a safe manner | 9.33 (1.18) | 6–10 | 93% |
| Assessment of the age at which limb surgery is initiated | 9.33 (1.29) | 6–10 | 93% |
| Adherence to a protocol of pre-surgery assessments | 9.20 (1.32) | 6–10 | 93% |
| Assessing patient age, pubertal stage, open physes and the length of the bone segments | 9.13 (1.36) | 6–10 | 93% |
| Providing estimates of achievable outcomes to patients, based on available data and knowledge. This information will inform decisions to undergo additional lengthening, or to wait until the effect of vosoritide therapy is known | 8.87 (1.85) | 3–10 | 93% |
| Selecting the most appropriate type of surgery based on the drivers for adding limb surgery (for example, deformity correction vs. guided growth vs. surgical limb lengthening) | 8.80 (2.37) | 1–10 | 93% |
| Imaging of the spinal canal and foramen magnum to exclude anaesthetic-relevant risk factors | 8.53 (2.47) | 1–10 | 87% |
| Ensuring the maximum target length can be gained | 7.73 (1.75) | 4–10 | 80% |
| Establishing the risk factors for inhibition of growth zone function | 7.73 (2.58) | 2–10 | 67% |
|
Theme 3: Timing/Age considerations For patients already prescribed vosoritide, considerations for the timing of initiation of limb surgery should include … | |||
| Individualised decision-making whether to cease vosoritide during surgery | 8.33 (2.58) | 2–10 | 80% |
| Establishing that the maximum growth has been achieved with vosoritide treatment (the least traumatic surgical options can then be considered) | 7.27 (2.96) | 1–10 | 67% |
| Whether there is sufficient residual growth | 5.87 (3.09) | 1–10 | 33% |
|
Theme 4: Contraindications For patients already prescribed vosoritide, limb surgery should NOT be considered if … | |||
| The patient demonstrates a lack of motivation for limb surgery | 9.27 (2.31) | 1–10 | 93% |
| The patient is not a suitable candidate for surgical limb lengthening | 9.00 (2.56) | 1–10 | 87% |
|
Theme 5: Follow up Considerations for follow up should include … | |||
| Collecting data on new bone generation† | 9.73 (0.59) | 8–10 | 100% |
| Adherence to local protocols for monitoring of both treatment options | 9.47 (1.13) | 6–10 | 93% |
*% of advisors scoring ≥ 7; † To assess whether vosoritide has an impact on the quality of the new bone growth