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

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

<p>Abstract</p> <p>Background</p> <p>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.</p> <p>Methods/Design</p> <p>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.</p> <p>Discussion</p> <p>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.</p> <p>Trial Registration</p> <p>NCT00412035</p

Internal validation of STRmix™ – A multi laboratory response to PCAST

We report a large compilation of the internal validations of the probabilistic genotyping software STRmix™. Thirty one laboratories contributed data resulting in 2825 mixtures comprising three to six donors and a wide range of multiplex, equipment, mixture proportions and templates. Previously reported trends in the LR were confirmed including less discriminatory LRs occurring both for donors and non-donors at low template (for the donor in question) and at high contributor number. We were unable to isolate an effect of allelic sharing. Any apparent effect appears to be largely confounded with increased contributor number

Radiological and clinical findings following distraction osteogenesis of the lower limb in children with or without botox injection: A preliminary report

Context: Limb lengthening correction according to Ilizarov distraction osteogenesis (DO) usually yields excellent results. While pediatric orthopedic surgeons anecdotally report that botulinum toxin A (Botox) alleviates spasm and pain during DO, no study has examined the effects of this surgery and Botox injection on muscle morphology in children. Aims: (1) To evaluate the clinical and radiological effects of lower limb lengthening in children and (2) the effects of adjunctive Botox muscle injection. Setting and Design: This was a center and randomized control trial. Methods: Seven children underwent tibial (n = 3) or femoral lengthening (n = 4) and were randomized to receive either a Botox or sterile saline solution. Quality of life and functional mobility outcomes, muscle testing, and magnetic resonance imaging examination were obtained at baseline, 12-, and 24-month postoperative. Muscle measurements were acquired on axial T1-weighted images and included: (1) total cross-sectional area (TCSA), (2) functional cross-sectional area (FCSA, fat-free area), (3) ratio of FCSA to TCSA, and (4) asymmetry between sides. Results: Physical performance improved at 12- and 24-month postoperative. Muscle strength remained functional across the three time points. A decrease in TCSA and FCSA was observed at 12-month follow-up in the operated thigh of patients undergoing femur lengthening, but muscle mass was regained at 24 months. However, TCSA and FCSA remained lower in the operated femur at 24 months. Similar findings were also observed in patients who underwent tibial lengthening. Patients in the Botox group appeared to have a slightly greater decrease in muscle mass and increase in fatty infiltration in the operated leg. Conclusions: Children undergoing DO preserved their muscle strength and improved physical performance 2-year postoperative. Our results suggest that muscle size and muscle mass remained lower in the operated leg; a finding that was slightly more prominent in children who received a Botox injection

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-standing 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

Familial ventricular aneurysms and septal defects map to chromosome 10p15

Aims Although ventricular septal defects (VSD) are the most common congenital heart lesion, familial clustering has been described only in rare instances. The aim of this study was to identify genetic factors and chromosomal regions contributing to VSD

Consensus statement on physical rehabilitation in children and adolescents with osteogenesis imperfecta

On the occasion of the 13th International Conference on Osteogenesis imperfecta in August 2017 an expert panel was convened to develop an international consensus paper regarding physical rehabilitation in children and adolescents with Osteogenesis imperfecta. The experts were chosen based on their clinical experience with children with osteogenesis imperfecta and were identified by sending out questionnaires to specialized centers and patient organizations in 26 different countries. The final expert-group included 16 representatives (12 physiotherapists, two occupational therapists and two medical doctors) from 14 countries. Within the framework of a collation of personal experiences and the results of a literature search, the participating physiotherapists, occupational therapists and medical doctors formulated 17 expert-statements on physical rehabilitation in patients aged 0-18 years with osteogenesis imperfecta