Cost effectiveness of buprenorphine vs. methadone for pregnant people with opioid use disorder.

OBJECTIVE: To assess the cost effectiveness of buprenorphine versus methadone in the management of opioid use disorder (OUD) during pregnancy. METHODS: We designed a decision-analytic model to evaluate the costs and outcomes associated with buprenorphine compared to methadone for pregnant people with OUD. We used a theoretical cohort of 22,400 pregnant people, which is an estimation of pregnancies affected by OUD per year in the United States. Outcomes included maternal retention in maintenance treatment, neonatal opioid withdrawal syndrome, preterm birth, fetal growth restriction, cerebral palsy, and maternal overdose in addition to cost and quality-adjusted life-years (QALYs). We used a willingness-to-pay threshold of $100,000/QALY. All model inputs were derived from the literature and varied in sensitivity analyses to assess the robustness of our baseline inputs. RESULTS: In our theoretical cohort, treatment of OUD with buprenorphine during pregnancy resulted in 2413 fewer cases of neonatal opioid withdrawal syndrome, 1089 fewer preterm births, 299 fewer cases of fetal growth restriction, 32 fewer stillbirths, and 13 fewer cases of cerebral palsy compared to methadone treatment. Despite lower rates of retention, buprenorphine treatment saved nearly 123 million healthcare dollars and resulted in 558 additional QALYs, making it the dominant strategy compared to methadone treatment. Our findings were robust over a wide range of assumptions. CONCLUSION: Our data suggest that buprenorphine should be considered a cost effective treatment option for OUD in pregnancy, as it is associated with improved neonatal outcomes compared to methadone despite the risk of treatment discontinuation.

Pediatric Intrathecal Baclofen Pumps: A Descriptive Analysis of Hospital Course and Associated Costs.

INTRODUCTION: The purpose of this study was to identify predictors of increased cost and postoperative length-of-stay (LOS) following intrathecal baclofen pump (ITBP) placement. METHODS: Patients were derived from the 2009/2012 kids' inpatient database. Inclusion criteria were selected for patients with ICD-9 codes 343.X (infantile cerebral palsy), 86.06 (infusion pump insertion), 03.90 (spinal catheter insertion), and elective hospitalizations. Nonparametric univariate analysis and subsequent gamma log-link general linear modeling were used to identify significant predictors of cost/LOS (p < 0.05). RESULTS: 529 unweighted patients (787 with survey weights applied) met criteria. Median LOS was 3.00 days, and median cost was USD 23,284. Following multivariate modeling, predictors of increased LOS (in days) included increased hospital ITBP volume (p = 0.027), small hospital size (+0.55, p = 0.004), device complications (+0.95, p < 0.001), procedural complications (+1.40, p < 0.001), additional procedures (+0.86, p < 0.001), electrolyte abnormalities (+3.74, p < 0.001), and neurological comorbidities (+1.60, p < 0.001). Factors associated with decreased LOS were paralysis (-0.53, p < 0.001), Northeastern hospital region (-0.55, p = 0.018), and investor-owned hospital status (-0.75, p = 0.001). Similarly, predictors of increased cost included race of Hispanic (+USD 1,156, p = 0.033) or "other" (+USD 2,158, p = 0.001), Northeast hospital region (+USD 4,120, p < 0.001), small (+USD 4,139, p < 0.001) or medium (+USD 3,368, p < 0.001) hospital sizes, additional procedures (+USD 1,649, p < 0.001), neurological comorbidities (+USD 3,222, p = 0.003), and increased LOS (p < 0.001). Factors associated with decreased cost included Western hospital region (-USD 1,594, p = 0.001), government hospitals (-USD 1,391, p = 0.019), and investor-owned hospitals (-USD 2,057, p = 0.021). CONCLUSION: This study found multiple variables associated with increased cost/LOS following ITBP placement. Broadly, this analysis demonstrates national trends associated with increased cost following ITBP placement.

Cost Comparison of Botulinum Toxin Injections Versus Surgical Treatment in Pediatric Patients With Cerebral Palsy: A Markov Model.

PURPOSE: The purpose of this study was to compare the cost-effectiveness of surgical release to botulinum toxin injections in the treatment of upper-extremity (UE) cerebral palsy (CP). METHODS: A Markov transition-state model was developed to assess the direct and indirect costs as well as accumulated quality-adjusted life-years associated with surgery (surgery group) and continuous botulinum toxin injections (botulinum group) for the treatment of UE CP in children aged 7 to 12 years. Direct medical costs were obtained from institutional billing departments. The number of parental missed workdays associated with each treatment was estimated and previously published regressions were used to calculate indirect costs associated with missed work. Total costs, cost-effectiveness, and incremental cost-effectiveness ratios were calculated. Incremental cost-effectiveness ratios and willingness to pay thresholds were used to make decisions regarding society's willingness to pay for the incremental cost of each treatment given the incremental benefit. RESULTS: The surgery group demonstrated lower direct, indirect, and total costs compared with the botulinum group. Direct costs were $29,250.50 for the surgery group and $50,596.00 for the botulinum group. Indirect costs were $9,467.46 for the surgery group and $44,428.60 for the botulinum group. Total costs were $38,717.96 for the surgery group and $95,024.60 for the botulinum group, a difference of $56,306.64. The incremental cost-effectiveness ratio was -$42,019.88, indicating that surgery is a less costly and more effective treatment and that botulinum injections fall outside the societal willingness to pay threshold. Excluding indirect costs associated with parental missed work during home occupational therapy did not have a significant impact on the model. CONCLUSIONS: Surgery is associated with lower direct, indirect, and total costs, as well as a greater number of accumulated quality-adjusted life-years. Surgery provides a greater benefit at a lower cost, which suggests that botulinum injections should be used sparingly in this population. Treatment with surgery could represent savings of $5.6 to $11.3 billion annually in the United States. TYPE OF STUDY/LEVEL OF EVIDENCE: Economic/Decision Analysis II.

Polypharmacy Among Privately Insured Adults with Cerebral Palsy: A Retrospective Cohort Study.

BACKGROUND: Adults with cerebral palsy (CP) have increased risk for developing various secondary chronic diseases, especially when they have other neurodevelopmental disabilities (NDDs). Multiple medications are likely prescribed to manage the greater morbidity-related burden for adults with CP; however, because health care delivery and care coordination is suboptimal for this population, adults with CP may have an increased risk for polypharmacy. To date, very little is known about the prescribing practices and extent of polypharmacy for adults with CP. OBJECTIVE: To determine the prevalence and adjusted odds of polypharmacy among adults with CP only and those with CP+NDDs, compared with adults without CP. METHODS: Data from 2017 Optum Clinformatics Data Mart, a U.S. private administrative database, was used for this retrospective cohort study. Diagnosis codes were used to identify adults (aged ≥ 18 years) with CP, NDDs (e.g., intellectual disabilities, epilepsy, and autism spectrum disorders), and 24 relevant morbidities. Polypharmacy was examined as 0-4 versus ≥ 5, 0-9 versus ≥ 10, and 0-14 versus ≥ 15 medications. Logistic regression estimated the OR and 95% CI of polypharmacy before and after adjusting for age, sex, region of residence, and multimorbidity (as 0, 1, 2, 3, 4-5, and ≥ 6 morbidities). Exploratory analyses were conducted to compare polypharmacy among young (18-40 years) and middle-aged (41-64 years) adults with CP only and CP + NDDs with elderly (≥ 65 years) adults without CP. RESULTS: Adults with CP only (n = 5,603) and CP + NDDs (n = 2,474) had higher unadjusted prevalence and adjusted OR for each polypharmacy definition compared with adults without CP (n = 9.0 million; e.g., ≥ 5 medications: adjusted OR for CP only = 1.38, 95% CI = 1.30-1.47; CP + NDDs: OR = 2.42, 95% CI = 2.20-2.67). Adults with CP+NDDs had higher unadjusted prevalence and adjusted OR of each polypharmacy definition compared with CP only. Compared with elderly without CP, the unadjusted prevalence of polypharmacy was lower for young adults with CP only (e.g., ≥ 5 medications: 60.2%, 43.8%), similar for young adults with CP+NDDs (e.g., ≥ 15 medications: 10.9%, 12.5%), and elevated for middle-aged CP only and CP + NDDs (e.g., ≥ 10 medications: 28.7%, 34.3%, 41.7%). CONCLUSIONS: Privately insured adults with CP only and CP + NDDs have an elevated prevalence of polypharmacy compared with adults without CP, even after accounting for multimorbidity. Importantly, adults aged 18-40 years with CP have a similar (CP + NDDs) prevalence of polypharmacy compared with the general geriatric population, with the prevalence increasing further for CP by middle age. DISCLOSURES: Whitney was supported by the University of Michigan Office of Health Equity and Inclusion Diversity Fund and the American Academy of Cerebral Palsy and Developmental Medicine. These funding sources had no role in the design or conduct of the study; collection, management, analysis, or interpretation of the data; preparation, review, or approval of the manuscript; or the decision to submit the manuscript for publication. The other authors have no conflicts of interest to disclose.

Use of Shear Wave Elastography for Quantitative Assessment of Muscle Stiffness After Botulinum Toxin Injection in Children With Cerebral Palsy.

OBJECTIVES: This study aimed to investigate the stiffness of the gastrocnemius (GC) muscle with acoustic radiation force impulse (ARFI) elastography after botulinum toxin-A (BTX-A) injection in children with spastic cerebral palsy (CP) and to examine the relationship between elastographic and clinical parameters. METHODS: This prospective randomized single-blind controlled clinical study included 49 lower extremities of 33 children with spastic CP. They were randomized into 2 groups: group 1 (n = 25 extremities in 17 children) received BTX-A injection and a home-based exercise program; group 2 (n = 24 extremities in 16 children) received only a home-based exercise program. Patients were evaluated in pretreatment and posttreatment periods in the first and third months with ARFI elastography, the Modified Ashworth Scale, Modified Tardieu Scale, Pediatric Functional Independence Measure, Gross Motor Function Classification System, and goniometric range of motion measurement of the ankle. RESULTS: A statistically significant difference was found in elastography of the GC muscle in group 1 only at the first month after treatment (P < .05). No statistical difference was found in elastography of the GC after treatment in group 2. According to the Modified Ashworth Scale, Modified Tardieu Scale, and ankle passive range of motion, group 1 showed significant improvements after treatment (P < .05). Also, there was a significant correlation between these clinical parameters and elastographic measurements (P < .05). CONCLUSIONS: According to the results of this study, the measurements from ARFI elastography combined with clinical parameters might be useful for evaluation of spasticity after BTX-A treatment in children with CP. Also, they might be useful in distinguishing patients who will benefit clinically, especially in the early stages of treatment.

The role of botulinum toxin in multimodal treatment of spasticity in ambulatory children with spastic Cerebral Palsy: extensive evaluation of a cost-effectiveness trial.

BACKGROUND: A cost-effectiveness trial (the Space Bop study) on the added value of botulinum toxin injections (BoNT-A) in the leg muscles, as part of a multimodal intervention for ambulatory children with spastic cerebral palsy in the context of a single distinct cycle of care was performed recently by our group. For a broad set of effect outcomes, we found that BoNT-A had no added value if children received comprehensive rehabilitation. However, this counterintuitive finding was met with scepticism. OBJECTIVE: Since several noteworthy facts and experiences were recorded during the course of the trial and the dissemination phase, the aim of this paper was to describe and discuss some crucial aspects of, and barriers to, the Space Bop study, related to context and perspective, design and results, as well as publication and implementation. METHODS: This paper discusses 5 issues: (i) the design, interpretation and presentation of previous research; (ii) the role of one's own clinical experience and interpretation; (iii) the aims of (BoNT-A) treatment; (iv) conflict of interest, role of industry, and the role of history; (v) optimal treatment modalities and dose-response relationships. CONCLUSION: Despite the unambiguous findings from the Space Bop study, several factors hindered acceptance of the results. Awareness of these factors is important when performing rehabilitation research and disseminating and implementing research findings.

Access to Intrathecal Baclofen Treatment for Children with Cerebral Palsy in European Countries: An SCPE Survey Reveals Important Differences.

AIM: The aim is to study access to intrathecal baclofen (ITB) for children with cerebral palsy (CP) in Europe, as an indicator of access to advanced care. METHODS: Surveys were sent to CP registers, clinical networks, and pump manufacturers. Enquiries were made about ITB treatment in children born in 1990 to 2005 by sex, CP type, level of gross motor function classification system (GMFCS) and age at the start of treatment. Access to ITB was related to the country's gross domestic product (GDP) and % GDP spent on health. RESULTS: In 2011 population-based data from Sweden, Norway, England, Portugal, Slovenia, and Denmark showed that 114 (3.4%) of 3,398 children with CP were treated with ITB, varying from 0.4 to 4.7% between centers. The majority of the children were at GMFCS levels IV-V and had bilateral spastic CP. In Sweden, dyskinetic CP was the most commonly treated subtype. Boys were more often treated with ITB than girls (p = 0.014). ITB was reported to be available for children with CP in 25 of 43 countries. Access to ITB was associated with a higher GDP and %GDP spent on health (p < 0.01). Updated information from 2019 showed remaining differences between countries in ITB treatment and sex difference in treated children was maintained. CONCLUSION: There is a significant difference in access to ITB for children with CP across Europe. More boys than girls are treated. Access to ITB for children with CP is associated with GDP and percent of GDP spent on health in the country.

To switch from Botox to Dysport in children with CP, a real world, dose conversion, cost-effectiveness study.

BACKGROUND AND OBJECTIVES: Children with cerebral palsy (CP) are routinely treated with botulinum toxin A (BoNT-A). Two non dose-equivalent and differently priced products, Botox and Dysport are used. Depending on the conversion one of the products is considerably cheaper. However, the dose conversion factors studied to date have varied widely and relevant studies have not included children. Our objective here was to compare the efficacy and health economics of the switch from Botox to Dysport in children with CP when conversion was set to 1:2. Specifically were these treatments perceived as equivalent in terms of efficacy, duration and side-effects and were the drug cost lowered by using Dysport. METHODS: This prospective, real-world, cost-effectiveness population-based observational study included all children with CP, (n = 159) mean age 9.4 years (SD, 4.3), in the larger Stockholm area who received BoNT-A between September 1, 2014, and December 31, 2015. Parents reported the efficacy, duration and side-effects of previous treatment while physicians reported doses and goals set by children and parents for the present treatment. Drug acquisition costs were provided by county administrators. RESULTS: In connection with 341 visits caregivers reported comparable effects of similar duration with these products, with few, similar and transient side-effects. The drug-cost per treatment was 4029 SEK for Botox and 2380 SEK in the case of Dysport. CONCLUSION: When Botox was replaced by a two-fold higher Unit dose of Dysport (conversion 1:2) parents perceived the treatment of their children with CP to be equally effective while the cost was 41% lower according to procured prices.

Longitudinal assessment of gait quality in children with bilateral cerebral palsy following repeated lower limb intramuscular Botulinum toxin-A injections.

BACKGROUND: Serial lower limb intramuscular Botulinum toxin-A (BoNT-A) injections are administered to children with bilateral spastic cerebral palsy (BCP) to reduce spasticity, improve walking and functional mobility, and delay the need for orthopaedic surgery. Gait quality is clinically assessed following BoNT-A with 2D video gait assessments (2DVGA) using the Edinburgh Visual Gait Score (EVGS). AIM: To determine the effect of three consecutive treatment cycles of lower limb intramuscular BoNT-A injections on gait quality using the EVGS in children with BCP by retrospectively reviewing repeated 2DVGA measures. METHODS AND PROCEDURES: Seventeen children with BCP and dynamic equinus (8 females and 9 males, age mean (SD), 4.0 (2.2) years, GMFCS I=2 and II=15) were included in the study after a retrospective audit of the records of the Queensland Children's Gait Laboratory (QCGL), Children's Health Queensland, Brisbane. The medical records of children who attended the QCGL between January 2001 and January 2016 were searched for eligibility. Children who had undertaken pre- and post-treatment 2DVGA for the first three lower limb BoNT-A treatment cycles (6 assessments) were reviewed using the EVGS. BoNT-A treatments were administered 7.7 (2.3) months apart and post-BoNT-A reviews occurred 12.6 (6.7) weeks after injection. Mixed-effects linear regression assessed the change from baseline to each subsequent assessment (p<0.05). OUTCOMES AND RESULTS: EVGS reduced significantly by a mean of 2.4 points from pre- to post-BoNT-A in the first treatment cycle (p=0.001). Compared to baseline, mean total EVGS reduced significantly during the second (pre-BoNT-A -1.7 (p=0.020), post BoNT-A -2.8 (p<0.001)) and third (pre-BoNT-A -2.6 (p=0.001), post BoNT-A -2.4 (p=0.002)) treatment cycles. There was no difference in EVGS between post-BoNT-A in the first treatment cycle and scores for the second and third treatment cycles. CONCLUSIONS AND IMPLICATIONS: Improvements in gait quality were statistically significant, but did not reach the EVGS smallest real difference value of 4 points. Repeated lower limb intramuscular BoNT-A injections to improve gait quality in children with BCP should be reconsidered.

Medication, rehabilitation and health care consumption in adults with cerebral palsy: a population based study.

OBJECTIVE: To evaluate medication, rehabilitation and healthcare consumption in adults with CP as a function of Gross Motor Function Classification System (GMFCS) level. DESIGN: Questionnaire-based cross-sectional study. SETTING: Brittany, a French county. SUBJECTS: Adults with cerebral palsy. INTERVENTIONS: Questionnaires relating to drugs, orthotic devices, mobility aids, rehabilitation and medical input were sent to 435 members of a unique regional French network dedicated to adults with cerebral palsy. The questionnaire was completed by the participant or a helper if necessary. RESULTS: Of the 282 responders, 7.8% had a GMFCS level of I, 14.2% II, 17.7% III, 29.1% IV and 31.2% V. Participants consumed a large amount of healthcare. Almost three-quarters took orally administered drugs, of which antispastic and antiepileptic drugs were among the most frequent. Nearly all patients had at least one type of rehabilitation, 87.2% had physiotherapy, 78% used at least one mobility aid and 69.5% used at least one orthotic device. The frequency of numerous inputs increased with GMFCS level. Specificities were found for each GMFCS level, e.g. participants with GMFCS level IV and V had a high level of medical input and a greater use of trunk-supporting devices, antireflux and laxative. Profiles could be established based on GMFCS levels. CONCLUSIONS: Adults with cerebral palsy use a large amount of drugs, mobility aids, orthotic devices, rehabilitation and medical input. Healthcare is targeted at cerebral palsy-related issues. GMFCS is a determinant of healthcare consumption and thus a useful tool for clinical practice to target care appropriately.

Instrumented assessment of the effect of Botulinum Toxin-A in the medial hamstrings in children with cerebral palsy.

This study examined the sensitivity of an instrumented spasticity assessment of the medial hamstrings (MEH) in children with cerebral palsy (CP). Nineteen children received Botulinum Toxin type A (BTX-A) injections in the MEH. Biomechanical (position and torque) and electrophysiological (surface electromyography, EMG) signals were integrated during manually-performed passive stretches of the MEH at low, medium and high velocity. Signals were examined at each velocity and between stretch velocities, and compared pre and post BTX-A (43 ± 16 days). Average change between pre and post BTX-A was interpreted in view of the minimal detectable change (MDC) calculated from previously published reliability results. Improvements greater than the MDC were found for nearly all EMG-parameters and for torque parameters at high velocity and at high versus low velocity (p<0.03), however large inter-subject variability was noted. Moderate correlations were found between the improvement in EMG and in torque (r=0.52, p<0.05). Biomechanical and electrophysiological parameters proved to be adequately sensitive to assess the response to treatment with BTX-A. Furthermore, studying both parameters at different velocities improves our understanding of spasticity and of the physiological effect of selective tone-reduction. This not only provides a clinical validation of the instrumented assessment, but also opens new avenues for further spasticity research.

Dysport (botulinum toxin type A) in routine therapeutic usage: a telephone needs assessment survey of European physicians to evaluate current awareness and adherence to product labeling changes.

BACKGROUND: Botulinum neurotoxin type A is a well-established treatment for a number of conditions involving muscle hyperactivity. Dysport (Ipsen Ltd, Wrexham, United Kingdom) is a botulinum neurotoxin type A preparation that has been available for a number of therapeutic uses for over 20 years in the European Union (EU). This survey was part of the EU botulinum toxin risk management plan to identify potential educational needs of injectors by collecting data on their routine practice administration of Dysport and their awareness of potential adverse events (AEs) that are included in the current product labeling. METHODS: Dysport-experienced injectors in 5 EU countries were surveyed via telephone about their experience of Dysport in patients with cervical dystonia, adult upper and lower limb spasticity, pediatric cerebral palsy, and blepharospasm/hemifacial spasm. RESULTS: The reconstitution dilution volume most often used was 2.5 mL per 500 U for all indications. The mean total dose ranged from 387 to 530 U for cervical dystonia, 508 to 773 U for upper limb spasticity, 600 to 832 U for lower limb spasticity, 375 to 700 U for pediatric cerebral palsy, and 54 to 213 U for blepharospasm/hemifacial spasm. The potential AEs most commonly mentioned by surveyed physicians were dysphagia for cervical dystonia, arm muscle weakness for upper limb spasticity, leg muscle weakness for lower limb spasticity, and pediatric cerebral palsy and ptosis for blepharospasm/hemifacial spasm. CONCLUSIONS: The results indicate that product-labeling recommendations are generally applied in clinical practice and that there is a good familiarity with potential AEs based on clinical condition. Nevertheless, the survey shows that experienced injectors do sometimes deviate from the manufacturers labeling recommendations, highlighting the importance of ongoing education.

Conflict of interest in the assessment of botulinum toxin A injections in patients with cerebral palsy: a systematic review.

BACKGROUND: The efficacy of using botulinum toxin A injections in cerebral palsy (CP) is controversial. The financial conflict of interest related to medical research can affect the conclusion of an evidence-based review. This study was performed to determine as to what proportion of studies on botulinum toxin A injections in patients with CP was sponsored by the industry and whether the assessments of botulinum toxin injection in CP were associated with industry support. METHODS: Studies were identified with a search of the PubMed database (January 1991 to November 2011). All prospective, comparative, English language studies on the use of botulinum toxin A injections in patients with CP were included. A total of 374 articles were screened, 128 potentially eligible full articles were retrieved, and 66 studies met our inclusion criteria. The funding sources of the articles were reviewed, and qualitative conclusions regarding the effect of botulinum toxin A injection were classified as being either favorable, neutral, or unfavorable. RESULTS: Of 66 eligible articles, 28 were funded by the industry, and 25 were not. The other 13 studies did not include information on the funding source. A significant association was observed between the funding source and qualitative conclusions (P=0.042). Fifteen (53.6%) of the 28 industry-sponsored studies had favorable conclusions, whereas only 5 (20%) of the 25 non-industry-sponsored studies had favorable conclusions. CONCLUSIONS: About half of studies on the effect of botulinum toxin A in CP were sponsored by the industry. This systematic review revealed that the qualitative conclusions in those studies are more favorable to the use of the botulinum toxin A than the non-industry-sponsored studies. Clinicians should be aware of an industry-related conflict of interest regarding reports on the efficacy of botulinum toxin A injections in patients with CP. LEVEL OF EVIDENCE: Level II-therapeutic study.

Comparative assessment of therapeutic response to physiotherapy with or without botulinum toxin injection using diffusion tensor tractography and clinical scores in term diplegic cerebral palsy children.

The present study was to compare the effects of combined therapy [botulinum (BTX) plus physiotherapy] with physiotherapy alone using diffusion tensor imaging (DTI) derived fractional anisotropy (FA) values of motor and sensory fiber bundles and clinical grade of the disability to see the value of BTX in term children with spastic diplegic cerebral palsy (CP). Clinically diagnosed 36 children participated in the study. All these children were born at term, and had no history of seizures. The study was randomly categorized into two groups: group I (n=18) - physiotherapy alone and group II (n=18) - physiotherapy plus BTX injection. Quantitative diffusion tensor tractography on all these children was performed on motor and sensory fiber bundles on baseline as well as after 6months of therapy. Motor function and clinical grades were also measured by gross motor function measures (GMFM) scale on both occasions. We observed significant change in FA value in motor and sensory fiber bundle as well as in GMFM scores at 6months compared to baseline study in both the groups. However, delta change and relative delta change in FA values of sensory and motor fiber bundle as well as GMFM score between group I and group II was statistically insignificant. We conclude that addition of BTX to physiotherapy regimen does not influence the outcome at 6months with similar insult in children with term diplegic spastic CP. This information may influence management of diplegic CP especially in developing countries, where BTX is beyond the reach of these children.

Botulinum toxin a treatment in children with cerebral palsy: its effects on walking and energy expenditure.

OBJECTIVE: This study aimed to evaluate the effects of botulinum toxin A (BTX-A) treatment on energy expenditure and ambulation in children with cerebral palsy (CP) and to determine if energy expenditure measurement could be considered a useful tool for evaluating the effectiveness of BTX-A treatment for CP. DESIGN: The study included 16 pediatric CP patients with equinus deformity who were capable of walking without assistance (group with CP). The children with CP were administered BTX-A injections into the gastrocnemius muscle. All the children underwent three-dimensional gait analysis and energy expenditure measurement before injection and 8 wks after injection, the results of which were compared with those of children without CP (control group). RESULTS: After BTX-A treatment, oxygen consumption in the group with CP was significantly lower than at pretreatment and was similar to that in the control group (P > 0.05). In the group with CP, post-BTX-A treatment ankle joint dorsiflexion angle at initial contact, mid-stance, and mid-swing improved significantly (P < 0.05). CONCLUSION: BTX-A injected into the gastrocnemius muscle in children with CP not only reduced spasticity and improved ankle range of motion and walking pattern but also reduced energy consumption, resulting in functional improvement. Energy expenditure measurement can be considered an objective quantitative tool for clinical evaluation of the functional outcome of therapeutic interventions such as BTX-A.

Botulinum toxin assessment, intervention and after-care for lower limb spasticity in children with cerebral palsy: international consensus statement.

Botulinum neurotoxin type-A (BoNT-A) has been used in association with other interventions in the management of spasticity in children with cerebral palsy (CP) for almost two decades. This consensus statement is based on an extensive review of the literature by an invited international committee. The use of BoNT-A in the lower limbs of children with spasticity caused by CP is reported using the American Academy of Neurology Classification of Evidence for therapeutic intervention. Randomized clinical trials have been grouped into five areas of management, and the outcomes are presented as treatment recommendations. The assessment of children with CP and evaluation of outcomes following injection of BoNT-A are complex, and therefore, a range of measures and the involvement of a multidisciplinary team is recommended. The committee concludes that injection of BoNT-A in children with CP is generally safe although systemic adverse events may occur, especially in children with more physical limitations (GMFCS V). The recommended dose levels are intermediate between previous consensus statements. The committee further concludes that injection of BoNT-A is effective in the management of lower limb spasticity in children with CP, and when combined with physiotherapy and the use of orthoses, these interventions may improve gait and goal attainment.

Botulinum neurotoxin: the ugly duckling.

This review presents a brief account of the most significant biological effects and clinical applications of botulinum neurotoxins, in a way comprehensive even for casual readers who are not familiar with the subject. The most toxic known substances in botulinum neurotoxins are polypeptides naturally synthesized by bacteria of the genus Clostridium. These polypeptides inhibit acetylcholine release at neuromuscular junctions, thus causing muscle paralysis involving both somatic and autonomic innervation. There is substantial evidence that this muscle-paralyzing feature of botulinum neurotoxins is useful for their beneficial influence on more than 50 pathological conditions such as spastic paralysis, cerebral palsy, focal dystonia, essential tremor, headache, incontinence and a variety of cosmetic interventions. Injection of adequate quantities of botulinum toxins in spastic muscles is considered as a highly hopeful procedure for the treatment of people who suffer from dystonia, cerebral palsy or have experienced a stroke. So far, numerous and reliable studies have established the safety and efficacy of botulinum neurotoxins and advocate wider clinical therapeutic and cosmetic applications.

Intrathecal baclofen therapy in children with intractable spastic cerebral palsy: a cost-effectiveness analysis.

In a Dutch national study, we recently established the effectiveness and safety of continuous intrathecal baclofen infusion (CITB) in children with intractable spastic cerebral palsy (CP). Because prospective studies on the cost-effectiveness of CITB in children with spastic CP are lacking, we conducted a cost-effectiveness analysis alongside our prospective national study. We compared the costs and health effects of CITB with those of standard treatment only, from the health care perspective for a 1-year period. Health effects were expressed in terms of a visual analogue scale for individual problems and quality-adjusted life years (QALYs). We included eight females and seven males, aged between 7 and 17 years (mean age 13y 8mo [SD 3y]). Eleven children had spastic CP and four had spastic-dyskinetic CP. One child was clsssified on the Gross Motor Function Classification System at Level III, two at Level IV, and 12 at Level V. CITB was more effective and more costly than standard treatment only. Gaining one QALY cost on average 32,737 euros. We conclude that based on the threshold-willingness to pay for one QALY in the Netherlands (80,000 euros), our results confirm the cost-effectiveness of CITB for carefully selected children with intractable spastic CP.