Activities and participation in young adults with osteogenesis imperfecta.

The objective of this cross-sectional study was to compare the activities and participation in the domains of mobility, self-care, domestic life and social functioning in young adults according to osteogenesis imperfecta (OI) type. Fifty-four former OI patients were invited to participate and were sent a structured questionnaire. Twenty-four patients (mean age: 25.0 years, SD: 2.6 years) with OI types I (n=7), III (n=7), IV (n =8) and V (n=2) completed the questionnaire. Participants with OI type I reported full independence, and only few respondents with OI types IV and V reported some limitations in mobility and domestic life activities. Young adults with OI type III had significantly lower activity scores in aspects of mobility and domestic life and lower levels of participation in employment, sporting activities and transportation. Participation in leisure and social interactions were not different across OI types. Young adults with more severe types of osteogenesis imperfecta have greater activity limitations and participation restrictions. Our findings indicate the importance of promoting and facilitating involvement in meaningful activities and roles in young adults with moderate to severe forms of OI.

Botulinum toxin type A injection in alleviating postoperative pain and improving quality of life in lower extremity limb lengthening and deformity correction: a pilot study.

BACKGROUND: The Ilizarov technique is commonly used for lengthening and deformity corrections of the lower limbs in children. Postoperative pain can be significant, affecting quality of life and functional mobility, and often requiring prolonged medication use. Several studies have investigated the antinociceptive actions of botulinum toxin type A (BtX-A), yet evidence for its use in this population is limited. The objectives were to (1) establish the feasibility of a randomized clinical trial in children undergoing limb lengthening or deformity correction and (2) provide preliminary evidence of the beneficial effects of BtX-A in this population. METHODS: Fifty-two patients with a mean age of 13.7 years (range, 5 to 21 y) were randomized to receive either BtX-A or an equivalent volume of sterile saline solution (placebo group), as a single dose during the surgical procedure. Pain, medication use, quality of life, and functional mobility outcomes were assessed in all patients. Adverse events were reported for all patients and classified as minor or major. RESULTS: Differences between groups did not reach statistical significance; however, pain at mid-distraction was found to be slightly lower in the BtX-A group, as compared with the placebo group. Patients in the BtX-A group used less parenteral pain medication in the first 4 days after the surgery, had higher quality of life scores at 3 of the 5 time points assessed, and slightly higher functional mobility scores. All adverse events were expected complications of the lengthening process. No event was considered to be a serious adverse event related to the BtX-A injection itself. There was a trend toward fewer major adverse events in the BtX-A group. CONCLUSIONS: This pilot study established the feasibility of a randomized controlled trial design for in this population. Its findings indicate that BtX-A injections appear to be safe and effective for reducing pain and improving the quality of life and functional mobility of children undergoing lengthening or deformity corrections of the lower limbs. A larger-scale study is currently underway to confirm these preliminary findings.

Safety and efficacy of botox injection in alleviating post-operative pain and improving quality of life in lower extremity limb lengthening and deformity correction.

BACKGROUND: Distraction osteogenesis is the standard treatment for the management of lower limb length discrepancy of more than 3 cm and bone loss secondary to congenital anomalies, trauma or infection. This technique consists of an osteotomy of the bone to be lengthened, application of an external fixator, followed by gradual and controlled distraction of the bone ends. Although limb lengthening using the Ilizarov distraction osteogenesis principle yields excellent results in most cases, the technique has numerous problems and is not well tolerated by many children. The objective of the current study is to determine if Botulinum Toxin A (BTX-A), which is known to possess both analgesic and paralytic actions, can be used to alleviate post-operative pain and improve the functional outcome of children undergoing distraction osteogenesis. METHODS/DESIGN: The study design consists of a multi centre, randomized, double-blinded, placebo-controlled trial. Patients between ages 5-21 years requiring limb lengthening or deformity correction using distraction will be recruited from 6 different sites (Shriners Hospital for Children in Montreal, Honolulu, Philadelphia and Portland as well as DuPont Hospital for Children in Wilmington, Delaware and Hospital for Sick Children in Toronto, Ont). Approximately 150 subjects will be recruited over 2 years and will be randomized to either receive 10 units per Kg of BTX-A or normal saline (control group) intraoperatively following the surgery. Functional outcome effects will be assessed using pain scores, medication dosages, range of motion, flexibility, strength, mobility function and quality of life of the patient. IRB approval was obtained from all sites and adverse reactions will be monitored vigorously and reported to IRB, FDA and Health Canada. DISCUSSION: BTX-A injection has been widely used world wide with no major side effects reported. However, to the best of our knowledge, this is the first time BTX-A is being used under the context of limb lengthening and deformity correction. TRIAL REGISTRATION: NCT00412035.

Effect of intravenous pamidronate therapy on functional abilities and level of ambulation in children with osteogenesis imperfecta.

OBJECTIVE: To evaluate the functional abilities and the level of ambulation during pamidronate therapy in children with moderate to severe osteogenesis imperfecta. STUDY DESIGN: Functional abilities, ambulation, and grip force were assessed in 59 patients (mean age, 6.1 years; range, 0.5-15.7 years; 30 girls) during 3 years of pamidronate treatment. Functional skills (mobility and self-care) were both assessed by using the Pediatric Evaluation of Disability Inventory. Ambulation level was assessed by using the modified Bleck score. For 48 patients, results after 3 years of pamidronate treatment could be matched to those of patients with similar age and disease severity who had not received pamidronate. RESULTS: Mobility and self-care scores increased during the study period (+43% and +30%, respectively). The average ambulation score changed from 0.8 to 1.9. Maximal isometric grip force increased by 63%. Mobility and ambulation scores and grip force measures were significantly higher than in patients who had not received pamidronate. The difference in self-care scores did not reach significance. CONCLUSION: This study suggests that cyclical pamidronate treatment improves mobility, ambulation level, and muscle force in children with moderate to severe osteogenesis imperfecta.