Table 2.
For patients who are considering either vosoritide or limb surgery (where ‘limb surgery’ includes surgical lengthening, corrective osteotomy, or guided growth)
| Statement | Level of agreement, mean (SD) | Range | % agreement* |
|---|---|---|---|
|
Theme 1. Drivers and Goals. When considering combining vosoritide and limb surgery the goals are to … | |||
| Achieve a target height, arm span or upper limb length to improve daily activities | 9.47 (0.64) | 8–10 | 100% |
| Attain as much benefit as possible from all available therapies | 9.07 (1.71) | 4–10 | 93% |
| Minimise the total number of surgical interventions to increase height | 8.93 (1.62) | 5–10 | 87% |
| Achieve the maximum possible treatment duration of vosoritide, to maximise height and potential benefits beyond height† | 8.87 (1.77) | 5–10 | 87% |
| Achieve maximum height with a combination of vosoritide and surgical lengthening | 8.87 (1.25) | 7–10 | 100% |
| Improve functionality to achieve maximum age-related independence in personal hygiene and activities of daily living | 8.73 (1.58) | 5–10 | 93% |
| Achieve height gains in older children in whom vosoritide alone will not meet the target | 8.53 (1.68) | 5–10 | 87% |
| Improve functionality to enable participation in all age-related activities (e.g., sport, social activities) | 8.40 (1.59) | 6–10 | 80% |
| Explore the opportunity for deformities to be corrected faster by combining vosoritide with guided growth surgery | 8.40 (2.23) | 5–10 | 80% |
| Improve physical, emotional, and social domains | 8.27 (2.09) | 3–10 | 80% |
| Focus on functional improvement, not height | 8.07 (1.98) | 5–10 | 67% |
| Improve proportionality | 8.00 (1.77) | 5–10 | 73% |
| Explore the potential for reciprocal amplification of outcomes in the combination of vosoritide with surgical limb lengthening | 8.00 (1.85) | 4–10 | 80% |
| Achieve maximum growth possible with vosoritide first, thereby minimising the need for, or total number of surgical interventions needed to increase height | 7.40 (3.40) | 1–10 | 67% |
| Improve the self-esteem of the patient | 7.40 (2.87) | 1–10 | 67% |
| Explore opportunities for further benefits of vosoritide, beyond height gain | 7.33 (3.06) | 1–10 | 73% |
| Obtain a sufficient increase in the length of the tibia and femur with vosoritide to enable surgical lengthening with implantable, lengthening nails after fusion of the physis | 7.27 (2.91) | 1–10 | 67% |
|
Theme 2. Pre-treatment considerations. Considerations prior to initiation of combined vosoritide and limb surgery should include … | |||
| Treatment planning | |||
| Involvement of a multidisciplinary team in a specialist centre to follow up the patient | 9.67 (0.90) | 7–10 | 100% |
| Previous surgical limb lengthening is not a contraindication to receiving vosoritide | 9.67 (0.82) | 7–10 | 100% |
| Planning a treatment strategy based on age and pubertal stage | 9.60 (0.83) | 8–10 | 100% |
| Identification of short- and long-term goals, based on individualised treatment planning | 9.27 (1.16) | 7–10 | 100% |
| Management of patient/family expectations | 9.07 (1.62) | 5–10 | 87% |
| Adherence to pre-treatment protocols, as for any therapeutic or surgical approach | 9.00 (1.85) | 3–10 | 93% |
| Establishing the age-dependent therapeutic window for the use of vosoritide | 8.73 (2.28) | 3–10 | 87% |
| Imaging to exclude foramen magnum stenosis prior to surgical intervention | 8.60 (2.59) | 1–10 | 80% |
| Identification of the best subgroup of candidates for treatment | 8.40 (2.16) | 3–10 | 80% |
| Potential impact of vosoritide on limb surgery practices | |||
| Reviewing the need for surgical limb lengthening procedures in patients who achieve target height and proportionality | 9.60 (0.91) | 7–10 | 100% |
| Reducing the need for extensive, or multiple rounds of, surgical limb lengthening | 9.53 (1.36) | 5–10 | 93% |
| Assessing the feasibility of dual therapy given the different modes of action of vosoritide and surgical limb lengthening | 9.07 (1.44) | 6–10 | 87% |
| Obtaining an understanding of the different modes of action of surgical limb lengthening and vosoritide | 8.87 (1.46) | 5–10 | 93% |
| The reassessment of surgical limb lengthening strategies given the inclusion of vosoritide | 8.73 (2.40) | 1–10 | 93% |
| Data indicate that mice with achondroplasia showed significantly better new bone formation than wild type mice; there are some concerns over the suppression of FGFR3 signalling by vosoritide during limb lengthening | 7.20 (2.78) | 1–10 | 67% |
| That combined vosoritide and limb surgery could add stress to the physis | 5.80 (2.86) | 1–10 | 40% |
| Decision-making process | |||
| Discussion of the potential risks and benefits of all options with the patient and family as part of the decision-making process | 9.87 (0.35) | 9–10 | 100% |
| Supporting the decision-making process by providing factual and evidence-based information | 9.47 (1.19) | 6–10 | 93% |
| Making the decision to initiate vosoritide or surgical limb lengthening on an individual basis | 9.33 (1.18) | 7–10 | 100% |
| Understanding the safety of any combination of vosoritide and surgical limb lengthening prior to initiation of a dual approach | 9.07 (2.02) | 3–10 | 87% |
| Consideration of the emotional, time and economic burden for families of both approaches | 9.00 (2.33) | 1–10 | 93% |
| Involving at least the prescribing clinician, the orthopaedic surgeon, and a psychologist in the multidisciplinary team | 8.47 (2.64) | 1–10 | 80% |
| An informed, shared decision-making process, involving the patient (if age-appropriate)/family and a multidisciplinary team including a psychologist or psychiatrist | 8.33 (2.64) | 1–10 | 73% |
| Ensuring psychological support is available to the patient and family prior to and during either vosoritide therapy or surgical limb lengthening | 7.93 (2.99) | 1–10 | 80% |
|
Theme 3. Timing Considering the timing of a dual treatment approach, vosoritide should be … | |||
| Prescribed first, and surgical limb lengthening discussed with patients and families in an age-appropriate way, as needed to achieve treatment goal | 8.33 (2.53) | 1–10 | 80% |
| Prescribed as early as possible to achieve maximum effect and to reduce future surgical treatment and associated complications | 8.33 (2.47) | 1–10 | 80% |
| Considered in the case of patients waiting to receive surgical limb lengthening | 8.27 (1.87) | 3–10 | 93% |
| Prescribed prior to limb surgery, with the option to add surgical interventions once outcomes achieved with vosoritide can be assessed | 7.27 (3.15) | 1–10 | 73% |
| Initiated at any stage of the surgical limb lengthening process | 6.27 (2.84) | 1–10 | 40% |
|
Theme 4. Contraindications Combined vosoritide and limb surgery should NOT be considered if … | |||
| The patient and their family are not motivated to accept the treatments and necessary follow ups | 9.87 (0.52) | 8–10 | 100% |
| Any contraindications exist either for surgical interventions or medical therapy | 9.47 (1.13) | 6–10 | 93% |
| Family support is not in place | 8.93 (2.28) | 3–10 | 87% |
| There is a history of poor compliance | 8.80 (2.40) | 1–10 | 93% |
| It is difficult to assess growth potential remaining | 7.20 (2.81) | 1–10 | 60% |
| Ongoing surgical limb lengthening is in the distraction phase | 5.40 (3.48) | 1–10 | 53% |
|
Theme 4. Follow up Considerations for follow up should include … | |||
| A structured regime specific to the monitoring of both therapies | 9.53 (1.13) | 6–10 | 93% |
| Appropriate frequency of follow up according to local protocols | 9.33 (1.29) | 6–10 | 93% |
| Adherence to local protocols for surgical lengthening and vosoritide therapy | 9.27 (1.44) | 6–10 | 87% |
*% of advisors scoring ≥ 7; †within the indication of vosoritide (while physes are open)