A roadmap to surgery in osteogenesis imperfecta: results of an international collaboration of patient organizations and interdisciplinary care teams.

Background and purpose - Involvement of patient organizations is steadily increasing in guidelines for treatment of various diseases and conditions for better care from the patient's viewpoint and better comparability of outcomes. For this reason, the Osteogenesis Imperfecta Federation Europe and the Care4BrittleBones Foundation convened an interdisciplinary task force of 3 members from patient organizations and 12 healthcare professionals from recognized centers for interdisciplinary care for children and adults with osteogenesis imperfecta (OI) to develop guidelines for a basic roadmap to surgery in OI.Methods - All information from 9 telephone conferences, expert consultations, and face-to-face meetings during the International Conference for Quality of Life for Osteogenesis Imperfecta 2019 was used by the task force to define themes and associated recommendations.Results - Consensus on recommendations was reached within 4 themes: the interdisciplinary approach, the surgical decision-making conversation, surgical technique guidelines for OI, and the feedback loop after surgery.Interpretation - The basic guidelines of this roadmap for the interdisciplinary approach to surgical care in children and adults with OI is expected to improve standardization of clinical practice and comparability of outcomes across treatment centers.

A review of the orthopedic interventions and functional outcomes among a cohort of 114 children with arthrogryposis multiplex congenita.

PURPOSE: Arthrogryposis multiplex congenita (AMC) refers to a large heterogeneous group of conditions involving joint contractures in two or more different areas of the body. Contractures can lead to decreased range of motion and strength, and affect ambulation and autonomy. The aim of this study was to describe the orthopedic interventions and functional outcomes of a large cohort of children with AMC followed in a pediatric orthopedic center. METHODS: A retrospective chart review of all children diagnosed with AMC followed at Shriners Hospital for Children - Canada (SHC) between January 1979 and July 2016 was conducted. One hundred twenty patients were identified, of whom six were excluded due to misdiagnosis or insufficient chart information. One hundred fourteen were retained. Patient demographics, AMC classification, comorbidities, operative and non-operative treatments received as well as community ambulation status, level of autonomy in self-care and transfers at latest follow-up were recorded. RESULTS: There were 54 males and 60 females with a mean age at last clinic visit of 10 years 3 months. Amyoplasia and distal arthrogryposis (DA) were equally represented in our sample, 47 (41.2%) and 49 (43.0%) participants respectively, with the category Other comprising the remaining 18 (15.8%) participants. Children with DA had less involvement of the proximal joints than those in the two other groups. Contractures and deformities of the foot and ankle were the most prevalent, affecting 91.5% with Amyoplasia, 85.7% with DA and 83.3% in the Other category. Contractures of the shoulder and elbow were more common among individuals with Amyoplasia and those categorized Other than those with DA. In terms of walking ability, 98% of participants with DA were independent ambulators. Walking ability varied among the Other participants. Similarly, most children with DA were independent in self-care and transfers at the most recent follow-up. CONCLUSION: The relatively large sample size of this study allowed for a better insight into the challenges associated with AMC management. These findings demonstrated the need for genetic testing to provide accurate diagnosis and classification, along with the use of standardized outcome tools to measure effectiveness of interventions. As AMC is rare, multi-site prospective studies are needed to improve research opportunities, develop functional measures specific to AMC and disseminate findings on a wider scale.

Vacuum bell treatment of pectus excavatum: An early North American experience.

PURPOSE: Conservative treatment of pectus excavatum with a vacuum bell device may be an attractive alternative to surgical repair. We describe an early North American experience with this device. METHODS: Prospectively maintained chest wall clinic registries from two institutions were reviewed to identify pectus excavatum patients ≤21 years treated with the vacuum bell from 2013 to 2017. Multivariate linear regression was used to compare mean improvements in deformity-depth and Haller Index between groups of patients based on age and usage metrics (hours/day and days/week). RESULTS: Thirty-one patients with a median age of 14 years received treatment with the device. Mean follow-up duration was 18 months. Median depth and Haller Index at treatment onset were 2.3 cm and 3.9, respectively. Improvements in deformity-depth were superior with device usage >2 h/day (p < 0.01) and daily use (p < 0.01). After adjusting for compliance, younger age of treatment onset was associated with greater improvement in Haller Index but not deformity depth. CONCLUSION: Our prospective early North American experience found the vacuum bell to be a potential alternative to surgical treatment for pectus excavatum. Longer usage periods in a daily frequency are associated with best results. TYPE OF STUDY: Treatment study; case series with no comparison group. LEVEL OF EVIDENCE: Level IV.

Functional Gains in Children With Spastic Hemiplegia Following a Tendon Achilles Lengthening Using Computerized Adaptive Testing-A Pilot Study.

PURPOSE: This pilot study evaluated the outcomes of tendon Achilles lengthening in 12 children (mean age: 11.2 years) with spastic hemiplegia. METHODS: Cerebral Palsy Computer Adaptive Tests, the timed up-and-go, the Gross Motor Function Measure, the Gillette Functional Assessment Questionnaire, and the Pediatric Outcomes Data Collection Instrument were administered at baseline and at 6, 12, and 24 months postsurgery. RESULTS: Significant improvement at the latest follow-up (12-24 months following surgery) was seen in all domains of the Cerebral Palsy Computer Adaptive Test: activity (P = .017), lower extremity (P = .005), global (P = .005), pain (P = .005), and fatigue (P = .028), as well as in the Gross Motor Function Measure-D domain (P = .02) and the mobility domain of the Pediatric Outcomes Data Collection Instrument (P = .04). CONCLUSION: These findings indicate that the tendon Achilles lengthening improved functional outcome in these children as measured by tests of physical function, walking speed, and activity performance.

Functional Outcome of Forearm Rodding in Children With Osteogenesis Imperfecta.

BACKGROUND: The impact of corrective forearm surgery on functional ability in children with osteogenesis imperfecta (OI) has not previously been reported. This study addresses this issue. METHODS: A retrospective chart review was conducted on 19 children with OI who underwent 22 corrective forearm procedures between 1996 and 2013. Functional ability was assessed preoperatively and every year postoperatively using the Pediatric Evaluation of Disability Inventory (PEDI). RESULTS: The mean PEDI self-care score increased by 6.8 (P=0.017) and the mean PEDI mobility score increased by 7.2 (P=0.020) at 1-year postsurgery. Functional gains were greater in moderate OI (types IV, V, and VI) than in severe OI (type III). Improved function was maintained in the majority of cases at a mean of 8.9 years postcorrection. CONCLUSIONS: Corrective forearm surgery in children with OI leads to improved functional ability. LEVEL OF EVIDENCE: Level IV.

Osteogenesis imperfecta.

Skeletal deformity and bone fragility are the hallmarks of the brittle bone dysplasia osteogenesis imperfecta. The diagnosis of osteogenesis imperfecta usually depends on family history and clinical presentation characterized by a fracture (or fractures) during the prenatal period, at birth or in early childhood; genetic tests can confirm diagnosis. Osteogenesis imperfecta is caused by dominant autosomal mutations in the type I collagen coding genes (COL1A1 and COL1A2) in about 85% of individuals, affecting collagen quantity or structure. In the past decade, (mostly) recessive, dominant and X-linked defects in a wide variety of genes encoding proteins involved in type I collagen synthesis, processing, secretion and post-translational modification, as well as in proteins that regulate the differentiation and activity of bone-forming cells have been shown to cause osteogenesis imperfecta. The large number of causative genes has complicated the classic classification of the disease, and although a new genetic classification system is widely used, it is still debated. Phenotypic manifestations in many organs, in addition to bone, are reported, such as abnormalities in the cardiovascular and pulmonary systems, skin fragility, muscle weakness, hearing loss and dentinogenesis imperfecta. Management involves surgical and medical treatment of skeletal abnormalities, and treatment of other complications. More innovative approaches based on gene and cell therapy, and signalling pathway alterations, are under investigation.

Success and duration of dynamic bracing for pectus carinatum: A four-year prospective study.

BACKGROUND: This study sought to establish factors that can prognosticate outcomes of bracing for pectus carinatum (PC). METHODS: Prospective data were collected on all patients enrolled in a dynamic bracing protocol from July 2011 to July 2015. Pressure of correction (POC) was measured at initiation of treatment, and pressure of treatment (POT) was measured pre- and post-adjustment at every follow-up visit. Univariate and Cox regression analysis tested the following possible determinants of success and bracing duration: age, sex, symmetry, POC, and POT drop during the first two follow-up visits. RESULTS: Of 114 patients, 64 (56%) succeeded, 33 (29%) were still in active bracing, and 17 (15%) failed or were lost to follow-up. In successful patients, active and maintenance bracing was 5.66±3.81 and 8.80±3.94months, respectively. Asymmetry and older age were significantly associated with failure. Multivariable Cox proportional hazard analysis of time-to-maintenance showed that asymmetry (p=0.01) and smaller first drop in POT (p=0.02) were associated with longer time to reach maintenance. CONCLUSIONS: Pressure of correction does not predict failure of bracing, but older age, asymmetry, and smaller first drop in pressure of treatment are associated with failure and longer bracing duration. LEVEL OF EVIDENCE: Prospective Study/Level of Evidence IV.

Safety and Efficacy of Botulinum Toxin A in Children Undergoing Lower Limb Lengthening and Deformity Correction: Results of a Double-blind, Multicenter, Randomized Controlled Trial.

BACKGROUND: Lengthening of the lower limb is a complex procedure in which pain management and complications such as pin-site infections and muscle contractures impact the family and affect the child's quality of life. As a result, the paralytic and antinociceptive actions of neurotoxins may be indicated in managing these complications; however, few studies have explored ways to improve outcomes after lengthenings. The objective of this study was to evaluate the safety and efficacy of botulinum toxin A (BTX-A) in children undergoing lower limb lengthenings and deformity correction. METHODS: Participants with a congenital or acquired deformity of the lower extremity requiring surgery to one limb were randomized to receiving either BTX-A as a single dose of 10 units per kilogram body weight, or an equivalent volume of saline solution. Pain, medication, quality of life, and physical function were assessed at different time-points. Adverse events were recorded in all participants. T test and χ tests were used to compare potential differences across both groups. RESULTS: Mean age of the 125 participants was 12.5 years (range, 5 to 21 y), and lengthenings averaged 4.2 cm. Maximum pain scores on day 1 postoperatively were lower in the BTX-A group (P=0.03) than in the placebo group, and remained significant favoring botox when stratifying by location of lengthening (femur vs. tibia). Clinical benefits for BTX-A were found for 3 quality of life domains at mid-distraction and end-distraction. When stratifying according to location of lengthening, there were significantly fewer pin-site infections in the tibia favoring botox (P=0.03). The amount of adverse events and bone healing indices were no different in both groups. CONCLUSIONS: The clinical differences in quality of life, the lower pain on the first postoperative day, and the lower number of pin-site infections in the tibia favoring BTX-A support its use as an adjunctive treatment to the lengthening process. The detailed analyses of pain patterns help inform families on the pain expectations during lower limb lengthenings. The amount of adverse events were no different in both groups, and bone healing rates were similar, indicating that the use of BTX-A in children undergoing limb lengthening and deformity correction is safe. LEVEL OF EVIDENCE: Level I.

Multidisciplinary Treatment of Severe Osteogenesis Imperfecta: Functional Outcomes at Skeletal Maturity.

OBJECTIVE: To determine the functional outcomes associated with long-term multidisciplinary treatment, intravenous bisphosphonate treatment, orthopedic surgery, and rehabilitation in children with severe osteogenesis imperfecta (OI) (diagnosed clinically as OI types III or IV). DESIGN: Retrospective study where outcomes were measured prospectively. SETTING: Pediatric orthopedic hospital. PARTICIPANTS: Adolescents (N=41; age range, 15-21y) with severe OI (OI type III: n=17; OI type IV: n=24) who had started therapy before the age of 6 years, had received treatment for at least 10 years, and had achieved final height. INTERVENTIONS: Intravenous bisphosphonate treatment, orthopedic surgery, and rehabilitation. MAIN OUTCOME MEASURE: Pediatric Evaluation of Disability Inventory. RESULTS: At the time of the last available follow-up examination, none of the individuals diagnosed with OI type III (most severely affected group) was able to ambulate without ambulation aids, whereas 20 (83%) patients with OI type IV were able to ambulate without ambulation aids. Regarding self-care, we specifically assessed 8 skills that we deemed essential for living independently (grooming; dressing; toileting; bed, chair, toilet, tub, and car transfers). Only 6 (35%) of the youths with OI type III were able to complete all 8 items, whereas 23 (96%) individuals with OI type IV managed to perform all tasks. Teens with OI type III often needed assistance for the transfer to toilet, tub, and car and for personal hygiene and clothing management associated with toileting, usually because of limitations in upper-extremity function. CONCLUSIONS: These observations suggest that further improvements in the functional status of the most severely affected children with OI are contingent on advances in the clinical management of upper-extremity issues.

Intravenous Bisphosphonate Therapy of Young Children With Osteogenesis Imperfecta: Skeletal Findings During Follow Up Throughout the Growing Years.

Cyclical intravenous bisphosphonate therapy is widely used to treat children with osteogenesis imperfecta (OI), but little is known about long-term treatment outcomes. We therefore reviewed 37 children with OI (OI type I, n = 1; OI type III, n = 14; and OI type IV, n = 22) who started intravenous bisphosphonate therapy before 5 years of age (median 2.2 years; range, 0.1 to 4.8 years), and who had a subsequent follow-up period of at least 10 years (median 14.8 years; range, 10.7 to 18.2 years), during which they had received intravenous bisphosphonate treatment (pamidronate or zoledronic acid) for at least 6 years. During the observation period, the mean lumbar spine areal bone mineral density Z-score increased from -6.6 (SD 3.1) to -3.0 (SD 1.8), and weight Z-score increased from -2.3 (SD 1.5) to -1.7 (SD 1.7) (p < 0.001 and p = 0.008). At the time of the last assessment, patients with OI type IV had significantly higher height Z-scores than a control group of patients matched for age, gender, and OI type who had not received bisphosphonates. Patients had a median of six femur fractures (range, 0 to 18) and five tibia fractures (range, 0 to 17) during the follow-up period. At baseline, 35% of vertebra were affected by compression fractures, whereas only 6% of vertebra appeared compressed at the last evaluation (p < 0.001), indicating vertebral reshaping during growth. Spinal fusion surgery was performed in 16 patients (43%). Among the 21 patients who did not have spinal fusion surgery, 13 had scoliosis with a curvature ranging from 10 to 56 degrees. In conclusion, long-term intravenous bisphosphonate therapy was associated with higher Z-scores for lumbar spine areal bone mineral density and vertebral reshaping, but long-bone fracture rates were still high and the majority of patients developed scoliosis.

Long-term functional benefits of selective dorsal rhizotomy for spastic cerebral palsy.

OBJECT: Large-scale natural history studies of gross motor development have shown that children with spastic cerebral palsy (CP) plateau during childhood and actually decline through adolescence. Selective dorsal rhizotomy (SDR) is a well-recognized treatment for spastic CP, but little is known about long-term outcomes of this treatment. The purpose of this study was to assess the durability of functional outcomes in a large number of patients through adolescence and into early adulthood using standardized assessment tools. METHODS: The authors analyzed long-term follow-up data in children who had been evaluated by a multidisciplinary team preoperatively and at 1, 5, 10, and 15 years after SDR. These evaluations included quantitative, standardized assessments of lower-limb tone (Ashworth Scale), Gross Motor Function Measure (GMFM), and performance of activities of daily living (ADLs) by the Pediatric Evaluation of Disability Inventory in children who had been stratified by motor severity using the Gross Motor Function Classification System (GMFCS). In addition, group-based trajectory modeling (GBTM) was used to identify any heterogeneity of response to SDR among these treated children, and to find which pretreatment variables might be associated with this heterogeneity. Finally, a chart review of adjunct orthopedic procedures required by these children following SDR was performed. RESULTS: Of 102 patients who underwent preoperative evaluations, 97, 62, 57, and 14 patients completed postoperative assessments at 1, 5, 10, and 15 years, respectively. After SDR, through adolescence and into early adulthood, statistically significant durable improvements in lower-limb muscle tone, gross motor function, and performance of ADLs were found. When stratified by the GMFCS, long-lasting improvements for GMFCS Groups I, II, and III were found. The GBTM revealed 4 groups of patients who responded differently to SDR. This group assignment was associated with distribution of spasticity (diplegia was associated with better outcomes than triplegia or quadriplegia) and degree of hip adductor spasticity (Ashworth score < 3 was associated with better outcomes than a score of 3), but not with age, sex, degree of ankle plantar flexion spasticity, or degree of hamstring spasticity. In a sample of 88 patients who had complete records of orthopedic procedures and botulinum toxin (Botox) injections, 52 (59.1%) underwent SDR alone, 11 (12.5%) received only Botox injections in addition to SDR, while 25 patients (28.4%) needed further lower-extremity orthopedic surgery after SDR. CONCLUSIONS: In the majority of patients, the benefits of SDR are durable through adolescence and into early adulthood. These benefits include improved muscle tone, gross motor function, and performance of ADLs, as well as a decreased need for adjunct orthopedic procedures or Botox injections. The children most likely to display these long-term benefits are those in GMFCS Groups I, II, and III, with spastic diplegia, less hip adductor spasticity, and preoperative GMFM scores greater than 60.

Effectiveness and safety of botulinum toxin type a in children with musculoskeletal conditions: what is the current state of evidence?

Children with musculoskeletal conditions experience muscle weakness, difficulty walking and limitations in physical activities. Standard treatment includes physiotherapy, casting, and surgery. The use of botulinum toxins appears as a promising treatment on its own, but usually as an adjunct to other treatment modalities and as an alternative to surgery. The objectives were to establish the evidence on the effectiveness, safety and functional outcome of BTX-A in children with musculoskeletal conditions. A literature search using five electronic databases identified 24 studies that met our inclusion criteria. Two randomized clinical trials were included; most studies were case studies with small sample sizes and no control group. Improvements in gait pattern, function, range of motion, reduction of co-contractions, and avoidance of surgical procedures were found following BTX-A injections. Adverse events were not reported in 10 studies, minor adverse events were reported in 13 children and there were no severe adverse events. Additional doses appear safe. BTX-A is a promising treatment adjunct in improving functional outcomes in children with musculoskeletal conditions. Future studies including larger samples, longer follow-up periods and a comparison group are required to provide evidence on the effectiveness and safety of this drug in children with musculoskeletal conditions.

Fassier-Duval femoral rodding in children with osteogenesis imperfecta receiving bisphosphonates: functional outcomes at one year.

PURPOSE: To examine the functional outcomes of children with osteogenesis imperfecta (OI) following initial Fassier-Duval (FD) rodding to the femur at 1 year, and to determine which factors are associated with change in gross motor function, ambulation, and functional performance. METHODS: Approval from our Institutional Review Board was obtained. A retrospective chart review identified 60 children (28 males, 32 females) with OI who underwent initial FD femoral rodding (101 rods) and who were receiving bisphosphonates. The mean age of the children was 3 years, 11 months at the initial femoral FD rodding. Two had type I OI, 30 type III, 27 type IV, and one type VI. The maximum length of follow-up was 4 years. Telescoping FD rods were used for the femurs, with surgeries performed one leg at a time, with a 1-week interval. The active range of motion (AROM) of the hips and knees in flexion was measured 4-5 weeks post-initial rodding. Outcomes on the Gillette Functional Assessment Questionnaire (FAQ) Ambulation Scale, the Gross Motor Function Measure (GMFM), and the Pediatric Evaluation of Disability Inventory (PEDI) were compared pre-operatively and at 1 year post-surgery using t-tests and multivariate linear regression. RESULTS: Pre-operatively, the mean FAQ score was 2.0, and this increased to 5.8 at 1 year post-surgery. Statistically significant improvements (P ≤ 0.05) were found on the FAQ, crawling, standing, walking and running, and total domains of the GMFM, and PEDI mobility and self-care from baseline to 1 year. The results from the multivariate linear regression indicate that older age (P = 0.0045) and higher weight (P = 0.0164) are significantly associated with lower scores in the self-care domain of the PEDI, and that OI type III compared to type IV is significantly associated (P = 0.0457) with greater improvement on the crawling domain of the GMFM. Higher weight was also associated (P = 0.0289) with lower scores in the standing domain of the GMFM, as well as with the total GMFM score (P = 0.0398). CONCLUSIONS: Our findings indicate that initial FD femoral rodding resulted in benefits in ambulation, gross motor function, self-care, and mobility for children with OI beyond physiological expectations due to developmental growth. FD rodding is a procedure which can improve the overall mobility in children with OI with significant femoral deformities.

Ambulation gains after knee surgery in children with arthrogryposis.

BACKGROUND: Arthrogryposis multiplex congenita is a rare congenital disorder associated with multiple musculoskeletal contractures that causes substantial morbidity. Knee involvement is commonly seen among children with arthrogryposis, with flexion contracture of the knee being the most frequent knee deformity. Knee flexion contractures in the pediatric population are particularly debilitating as they affect ambulation. Treatment for knee flexion contractures requires numerous orthopaedic procedures and an extensive follow-up period. The purpose of this study was to assess the effectiveness of orthopaedic procedures, namely distal femoral supracondylar extension osteotomy and/or Ilizarov external fixator, on the ambulation status of children with knee flexion contracture and whether any functional gains are maintained at the latest follow-up. METHODS: All children with arthrogryposis followed at our institution who had surgical correction for knee flexion contractures were included in this study. Fourteen patients were identified and their medical records were reviewed. The etiology for all patients was amyoplasia. The mean age at first surgery was 7.0 years (range, 2 to 16 y). The mean length of follow-up was 59.3 months (range, 12 to 117 mo). Contractures were treated with femoral extension osteotomy (n=8), Ilizarov external fixator (n=1), or both (n=5). Three patients earlier had posterior soft tissue releases, including hamstrings lengthenings, proximal gastrocnemius release, and release of posterior capsule. RESULTS: Preoperatively, 11 patients were nonambulatory, 2 patients were household ambulators, and 1 patient walked with orthoses in the community. There was an average of 1.8 knee surgeries done per patient, namely distal femoral extension osteotomy and/or Ilizarov external fixator. At the latest follow-up, 8 patients were ambulatory with technical aids (orthosis, walker, braces, or rollator walker), 2 patients were household ambulators, 1 patient used a wheelchair but was independent for transfers, and 3 patients remained nonambulatory. The mean flexion contracture before the first surgery was 63.7 ± 26.8 degrees. Postoperatively, the mean flexion contracture was 13.2 ± 16.7 degrees. At the latest follow-up, the mean flexion contracture was 34.0 ± 24.1 degrees. There were complications in 2 patients, including infected hardware which resolved with antibiotic treatment, and neurologic compromise which resolved on its own. CONCLUSIONS: Surgical correction of knee flexion deformities by distal femoral extension osteotomy and/or Ilizarov external fixator was effective in improving the ambulation status of children with arthrogryposis. At latest follow-up, the gradual loss of total arc of motion and the recurrence of knee flexion contractures did not limit the ambulatory gains achieved. LEVEL OF EVIDENCE: IV, Case series.

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.

A prospective evaluation of the WeeFIM in patients with cerebral palsy undergoing orthopaedic surgery.

PURPOSE: Although frequently used in pediatric rehabilitation settings, the WeeFIM has not been tested in surgical pediatric orthopaedic patients. METHODS: The WeeFIM was administered to patients with surgical cerebral palsy at defined intervals preoperatively and at both 6 and 12 months postoperatively. The age-adjusted change scores from baseline to follow-up were tested both parametrically and nonparametrically. RESULTS: Four hundred sixty-eight patients had baseline evaluations. There were 161 six-month follow-up assessments and 108 twelve-month follow-up assessments. The baseline WeeFIM was able to separate children with different patterns of cerebral palsy. Hemiplegic patients had higher scores than diplegic and tetraplegic patients. Overall age-adjusted scores were improved at both 6 (mean increase 2.0) and 12 months (mean increase 2.2). The instrument showed significant ceiling effects for diplegic and hemiplegic patients with lower or upper extremity surgery and limited responsiveness for lower extremity surgery in tetraplegic patients. Parametrically, it showed improvements in mobility for both rhizotomy and tetraplegic upper extremity surgery. Nonparametric tests were not significant for rhizotomy mobility improvement. CONCLUSIONS: Although the WeeFIM adequately reflects the severity of neurological involvement in pediatric orthopaedic patients with cerebral palsy, it has a significant ceiling effect in diplegic and hemiplegic patients limiting responsiveness and lacks content validity for tetraplegic patients. The instrument may have some use in tetraplegic patients with upper extremity surgery and in rhizotomy patients. We recommend against its general use for orthopaedic surgery in patients with cerebral palsy lower extremity or spine surgery and in hemiplegic patients with upper extremity surgery.

Impact of selective posterior rhizotomy on fine motor skills. Long-term results using a validated evaluative measure.

Suprasegmental effects following selective posterior rhizotomy have been frequently reported. However, few studies have used validated functional outcome measures to report the surgical results beyond 3 years. The authors analyzed data obtained from the McGill Rhizotomy Database to determine the long-term impact of lumbosacral dorsal rhizotomy on fine motor skills. The study population comprised children with debilitating spasticity who underwent SPR and were evaluated by a multidisciplinary team preoperatively, at 6 months and 1 year postoperatively. Quantitative standardized assessments of upper extremity function were obtained using the fine motor skills section of the Peabody Developmental Motor Scales (PDMS) test. Of 70 patients who met the entry criteria for the study, 45 and 25 completed the 3- and 5-year assessments, respectively. Statistical analysis demonstrated significant improvements in grasping, hand use, eye-hand coordination, and manual dexterity at 1 year after SPR. More importantly, all improvements were maintained at 3 and 5 years following SPR. This study supports that significant improvements in upper extremity fine motor function using the PDMS evaluative measure are present after SPR and that these suprasegmental benefits are durable.