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.

Intrathecal Baclofen Therapy in Patients With Multiple Sclerosis: Improved Outcomes and Reduced Costs Through Identification of Catheter Malfunction.

INTRODUCTION: Two patients previously implanted with intrathecal Baclofen (ITB) pumps for management of intractable spasticity due to multiple sclerosis (MS) were referred to our center for ongoing management of their spasticity. Initial evaluation of these patients revealed high levels of spasticity in the presence of ITB doses 10 times the average daily dose of our other MS patients. CLINICAL FACTS: High doses of ITB required frequent clinical visits and result in high drug and procedure costs. Both patients' daily doses were greater than 1000 mcg/day resulting in clinical visits every 1-2 months with drug and procedure costs ranging from 16 to 23 thousand dollars annually based on Medicare national average pricing for physician's office. Of the 59 MS patients receiving ITB therapy at our institution, the mean, median, and mode daily doses for ITB are 184, 115, and 159 mcg/day, respectively. The high ITB doses in these patients and poor spasticity control raised suspicion for pump/catheter malfunction and prompted immediate troubleshooting. FINDINGS: One patient's catheter was found to be disconnected from the pump and the other's catheter tip was outside the intrathecal space. In both cases, the patients were not receiving the therapy. After pump/catheter replacement, both patients received excellent clinical benefits from ITB at significantly lower daily doses. This reduction in dose resulted in decreased frequency of medication refills (twice annually) which resulted in decreased cost of care (12-19 thousand dollars savings annually per patient). DISCUSSION: These cases illustrate the need for early ITB pump troubleshooting to identify catheter problems, improve efficacy, and avoid unnecessary healthcare costs.

Dissociable contributions of dorsal and ventral striatal regions on a rodent cost/benefit decision-making task requiring cognitive effort.

Cognitive effort is a ubiquitous process, yet surprisingly little is known about the brain mechanisms responsible for evaluating it. Here, we utilize the rat Cognitive Effort Task (rCET) to probe the striatum's role in deciding between options that vary in the amount of cognitive effort required for success. In the rCET, animals choose to perform either an easy trial, in which the attentional demand is low but the potential reward is small, or a difficult trial which is more attentionally demanding but can yield twice the sugar pellets. Twenty-six male Long Evans rats were trained on the rCET and the effects of pharmacologically inactivating the dorsomedial striatum (DMS) and core region of the nucleus accumbens were determined. Temporary inactivation of the DMS decreased all animals' choice of the high-effort, high-reward option, impaired attentional accuracy, and robustly increased premature responding without impairing general indices of motor ability. The DMS therefore appears necessary for the integration of cognitive signals required for optimal performance. In stark contrast, following temporary inactivation of the ventral striatum, subjects were fundamentally unable to perform the task, as reflected by a drastic decrease in the number of trials initiated and an increase in omitted responses. Together, these data suggest the striatum is likely part of a larger cortico-limbic-striatal network whose function is to optimize decisions requiring cognitive effort costs, at least in the attentional domain, and that striatal subregions have dissociable roles in the adjudication and application of this form of cognitive effort.

Safety reports on the off-label use of baclofen for alcohol-dependence: recommendations to improve causality assessment.

INTRODUCTION: The off-label use of high-dose baclofen (HDB) for alcohol-dependence has recently spread. However, HDB has been associated with numerous reports of adverse events (AEs). Pharmacovigilance reporting is supposed to differentiate AEs from adverse drug reactions (ADRs), for which the causality of the drug is determined using validated methods. AREAS COVERED: Since 2010, we found 20 publications on baclofen-related AEs in alcohol dependence, in Medline-referenced journals or national pharmacovigilance reports. We focused on whether these reports used causality algorithms, and provided essential elements for determining baclofen causality and excluding the involvement of alcohol and other psychoactive substances or psychotropic drugs. In half of the cases, no causality algorithm was used. Detailed information on baclofen dosing was found in 17 out of 20 (85%) articles, whereas alcohol doses were given only in 10 (50%) publications. Other psychoactive substances and psychotropic drugs were broached in 14 (70%) publications. EXPERT OPINION: future publications reporting suspected HDB-induced ADRs should use validated causality algorithms and provide sufficient amount of contextual information for excluding other potential causes. For HDB, the psychiatric history, and the longitudinal description of alcohol consumptions and associated doses of psychoactive substances or psychotropic medications should be detailed for every reported case.

Impact of antibiotic prophylaxis for intrathecal baclofen pump surgery in pediatric patients.

OBJECT This study explored antibiotic prophylaxis (AP) in pediatric patients undergoing intrathecal baclofen pump (ITBP) surgery and factors associated with perioperative AP compliance with clinical guidelines. METHODS Data were obtained from the Pediatric Health Information System. The study cohort comprised patients who underwent ITBP surgery within 3 days of admission, between July 1, 2004, and March 31, 2014, with a minimum prior screening period and follow-up of 180 days. Exclusion criteria were prior infection, antibiotic use within 30 days of admission, and/or missing financial data. Chi-square tests and multivariate logistic regressions were used to determine factors associated with compliance with AP guidelines in ITBP surgeries. RESULTS A total of 1,534 patients met the inclusion criteria; 91.5% received AP and 37.6% received dual coverage or more. Overall bundled compliance comprised 2 components: 1) perioperative antibiotic administration and 2) < 24-hour postoperative antibiotic course. The most frequently used antibiotics in surgery were cefazolin (n = 873, 62.2%) and vancomycin (n = 351, 25%). Documented bundled AP compliance rates were 70.2%, 62.0%, 66.0%, and 55.2% in West, South, Midwest, and Northeast regions of the US, respectively. Compared with surgeries in the Northeast, procedures carried out in the West (OR 2.0, 95% C11.4-2.9, p < 0.001), Midwest (OR 1.6, 95% C11.1-2.3, p = 0.007), and South (OR 1.5, 95% C11.1-2.0, p = 0.021) were more likely to have documented AP compliance. Black (OR 0.74, 95% CI 0.55-1.00, p = 0.05) and Hispanic (OR 0.63, 95% CI 0.47-0.86, p = 0.004) patients were less likely to have documented AP compliance in ITBP surgeries than white patients. There were no significant differences in compliance rate by age, sex, type of insurance, and diagnosis. AP process measures were associated with shorter length of stay, lower hospitalization costs, and lower 6-month rates of surgical infection/complication. One of the 2 noncompliance subgroups, missed preoperative antibiotic administration, was correlated with a significantly higher 6-month surgical complication/infection rate (27.03%) compared with bundled compliance (20.00%, p = 0.021). For the other subgroup, prolonged antibiotic use > 24 hours postoperatively, the rate was insignificantly higher (22.00%, p = 0.368). Thus, of direct relevance to practicing clinicians, missed preoperative antibiotics was associated with 48% higher risk of adverse complication/infection outcome in a 6-month time frame. Adjusted hospitalization costs associated with baclofen pump surgery differed significantly (p < 0.001) with respect to perioperative antibiotic practices: 22.83, 29.10, 37.66 (× 1000 USD) for bundled compliance, missed preoperative antibiotics, and prolonged antibiotic administration, respectively. CONCLUSIONS Significant variation in ITBP antibiotic prophylaxis was found. Documented AP compliance was associated with higher value of care, showing favorable clinical and financial outcomes. Of most impact to clinical outcome, missed preoperative antibiotics was significantly associated with higher risk of 6-month surgical complication/infection. Prolonged antibiotic use was associated with significantly higher hospital costs compared with those with overall bundled antibiotic compliance. Future research is warranted to examine factors associated with practice variation and how AP compliance is associated with outcomes and quality, aiming for improving delivery of care to pediatric patients undergoing ITBP procedures.

The need for and provision of intrathecal baclofen therapy for the management of spasticity in England: an assessment of the Hospital Episode Statistics database.

OBJECTIVES: The aim of this study was to evaluate changes in the uptake of intrathecal baclofen (ITB) following commissioning of this therapy by the National Health Service (NHS) England in April 2013. The specific objectives of this study were: (i) to explore the gap between the need for and the actual provision of ITB services; and (ii) to compare England figures with other European countries with comparable data available. SETTING: Data for ITB -related procedures were obtained from the Hospital Episode Statistics (HES) database from 2009/2010 to 2013/2014. PARTICIPANTS: Patients receiving ITB for the management of spasticity. RESULTS: The available data for implantation of ITB from 2009/2010 to 2013/2014 for the treatment of spasticity due to varied aetiologies show that there has not been an increase in uptake of this therapy. The estimated need for this treatment based on the incidence and prevalence of conditions susceptible to ITB therapy is between 4.6 and 5.7 per million population. Our analysis of the data available from the HES database showed that the actual number of implants is around 3.0 per million population. The same period 2009-2014 has seen an increase in the delivery of other neuromodulation techniques including spinal cord stimulation, deep brain stimulation and sacral nerve stimulation. CONCLUSIONS: There is a considerable gap between the need for and provision of ITB figures nationally. Additionally, within the same area, we have observed important differences in the ITB service delivery between the various trusts. The reasons for this can be multifactorial, including individual experience and opinions, organisational structures, resource and financial limitations. Further research analysing the efficacy and cost-effectiveness of this treatment in the UK might inform the development of Technology Appraisal Guidance for ITB, potentially leading to an improvement in service provision.

Medical cost impact of intrathecal baclofen therapy for severe spasticity.

OBJECTIVES: To evaluate the economic effects of intrathecal baclofen (ITB) for patients with severe spasticity based on costs of care before and after implantation of an intrathecal drug delivery system. MATERIALS AND METHODS: An actuarial projection of post-implant experience in the absence of ITB intervention was used to simulate a continued conventional medical management protocol (ITB-free) by assuming a reasonable trend rate based on health-care industry standards. Cost projections were developed over a 30-year time horizon at various reimplantation rates. The model was informed by retrospective analysis of commercial administrative claims data from 409 pediatric and adult spasticity patients who received a pump implant (ITB-experienced) within a 3-year service period (January 2006 to January 2009). Common indications associated with pump implant included multiple sclerosis (N = 124), cerebral palsy (N = 131), and spinal cord injury (N = 40). RESULTS: ITB was less costly than the conventional protocol over our baseline implantation cycle. Costs in the month of implant and in the year following were cumulatively $26,375 more than with the conventional protocol. However, ITB financial break-even occurs between the second and third years post-implant. The lifetime analysis indicates that savings for ITB are $8009 per patient per year compared with conventional therapy. Most of the savings are derived from reductions in inpatient admissions, physician office visits, and outpatient physiotherapy. CONCLUSIONS: The results suggest that spasticity patients receiving ITB would expect to experience a reduction in cumulative future medical costs relative to anticipated costs in the absence of a pump implant. This finding complements the existing literature on the cost-effectiveness of ITB.

Cost-effectiveness analysis of baclofen and chlordiazepoxide in uncomplicated alcohol-withdrawal syndrome.

OBJECTIVES: Benzodiazepines (BZDs) are the first-line drugs in alcohol-withdrawal syndrome (AWS). Baclofen, a gamma-aminobutyric acidB (GABAB) agonist, controls withdrawal symptoms without causing significant adverse effects. The objective of this study was to compare the cost-effectiveness of baclofen and chlordiazepoxide in the management of uncomplicated AWS. MATERIALS AND METHODS: This was a randomized, open label, standard controlled, parallel group study of cost-effectiveness analysis (CEA) of baclofen and chlordiazepoxide in 60 participants with uncomplicated AWS. Clinical efficacy was measured by the Clinical Institute Withdrawal Assessment for alcohol (CIWA-Ar) scores. Lorazepam was used as supplement medication if withdrawal symptoms could not be controlled effectively by the study drugs alone. Both direct and indirect medical costs were considered and the CEA was analyzed in both patient's perspective and third-party perspective. RESULTS: The average cost-effectiveness ratio (ACER) in patient's perspective of baclofen and chlordiazepoxide was Rs. 5,308.61 and Rs. 2,951.95 per symptom-free day, respectively. The ACER in third-party perspective of baclofen and chlordiazepoxide was Rs. 895.01 and Rs. 476.29 per symptom-free day, respectively. Participants on chlordiazepoxide had more number of symptom-free days when compared with the baclofen group on analysis by Mann-Whitney test (U = 253.50, P = 0.03). CONCLUSION: Both study drugs provided relief of withdrawal symptoms. Chlordiazepoxide was more cost-effective than baclofen. Baclofen was relatively less effective and more expensive than chlordiazepoxide.

Off-label baclofen prescribing practices among French alcohol specialists: results of a national online survey.

OBJECTIVE: To evaluate, among alcohol specialists belonging to the Société Française d'Alcoologie (SFA), i.e., the French Alcohol Society, the proportion of physicians who prescribed off-label baclofen for alcohol use disorders (AUDs). The secondary objective was to depict the features of individual prescribing and monitoring practices. METHODS: On-line survey among 484 French alcohol specialists. Physicians were asked whether they prescribed baclofen for AUDs. If they did not, the reasons for this choice were investigated. If they did, the features of the physician's prescribing practice were explored, including the number of patients treated, the mean and maximum doses, the monitoring precautions and the pharmacovigilance reporting. Participants were also asked about their empirical findings on HDB's efficacy and safety. RESULTS: In total, 302 physicians (response rate of 62.4%) participated in the survey. Data from 296 participants were analysed, representing 59.4% of all active prescribing physicians belonging to the SFA. HDB use was declared by 74.6% of participants (mean dose 109.5 ± 43.6 mg/d; maximum dose 188 ± 93.3 mg/d). However, 79.2% of prescribers had treated less than 30 patients, and 67.8% used HDB as a second-line medication. Although HDB was perceived as more efficacious than approved drugs by 54.3% of prescribers, it was also declared less safe by 62.8%. Nonetheless, 79.7% of prescribers had never filed any pharmacovigilance report. Non-prescribers (25.6%) were primarily deterred by the current lack of scientific data and official regulation. CONCLUSION: A majority of French alcohol specialists reported using HDB, although often on a limited number of their patients. HDB was considered efficacious but also potentially hazardous. Despite this, physicians reported minimal safety data to the health security system. While French health authorities are planning to draft a specific regulatory measure for framing off-label HDB prescribing practices, the sustained education of prescribers on spontaneous pharmacovigilance reporting should be enhanced.

Cost-effectiveness analysis of intrathecal baclofen therapy in Japan.

Intrathecal baclofen (ITB) therapy was approved for health insurance coverage in 2005 for the treatment of patients whose spasticity could not be adequately controlled by conventional therapy, and is currently being used to treat around 300 patients nationwide in Japan. Various reports have examined the efficacy and safety of ITB therapy, but no report has evaluated the patient quality of life and medical costs in Japan. A cost-utility analysis of ITB was conducted by time period in six severely spastic patients admitted to our university hospital between 2005 and 2010 for ITB therapy. The average cost of ITB therapy per quality-adjusted life year (QALY; number of years survival in perfect health) 5 years after surgery was 1,554,428 yen, below the 6 million yen willingness-to-pay threshold for 1 QALY. This study shows that ITB therapy in Japan is an outstanding treatment in medicoeconomic terms.

Assessing competency in spasticity management: a method of development and assessment.

This project endeavored to create an educational module including methodology to instruct physical medicine and rehabilitation residents in the evaluation and appropriate treatment of patients with spasticity and other sequelae of the upper motor neuron syndrome. It further sought to verify acquired competencies in spasticity management through objective evaluation methodology. A physical medicine and rehabilitation board-certified physician with 10 yrs clinical experience in spasticity management trained 16 residents using a standardized competency-based module. Assessment tools developed for this program address the basic competencies outlined by the Accreditation Council for Graduate Medical Education. The 16 residents successfully demonstrated proficiency in every segment of the evaluation module by the end of the Postgraduate Year 3 spasticity management rotation. Objective measures compared resident scores on an institution-specific standardized test administered before and after training. Resident proficiency in the skills and knowledge pertaining to spasticity management was objectively verified after completion of the standardized educational module. Validation of the assessment tool is evidenced by significantly improved postrotational institution-specific standardized test scores (mean pretest score, 61.1%; mean posttest score, 95.4%) as well as oral testing. In addition, the clinical development tool was validated by residents being individually observed performing skills and deemed competent by a board-certified physical medicine and rehabilitation physician specializing in spasticity management. The standardized educational module and evaluation methodology provide a potential framework for the definition of baseline competency in the clinical skill area of spasticity management.

Baclofen-loaded solid lipid nanoparticles: preparation, electrophysiological assessment of efficacy, pharmacokinetic and tissue distribution in rats after intraperitoneal administration.

Intrathecal baclofen administration is the reference treatment for spasticity of spinal or cerebral origin, but the risk of infection or catheter dysfunctions are important limits. To explore the possibility of alternative administration routes, we studied a new preparation comprising solid lipid nanoparticles (SLN) incorporating baclofen (baclofen-SLN). We used SLN because they are able to give a sustained release and to target the CNS. Wistar rats were injected intraperitoneally with baclofen-SLN or baclofen solution (baclofen-sol group) at increasing dosages. At different times up to 4 h, efficacy was tested by the H-reflex and two scales evaluating sedation and motor symptoms due to spinal lesions. Rats were killed and baclofen concentration determined in blood and tissues. Physiological solution or unloaded SLN was used as controls. After baclofen-SLN injection, the effect, consisting in a greater and earlier reduction of the H/M ratio than baclofen-sol group and controls, was statistically significant from a dose of 5 mg/kg and was inversely correlated with dose. Clinical effect of baclofen-SLN on both the behavioral scales was greater than that of baclofen-sol and lasted until 4th hour. Compared with baclofen-sol, baclofen-SLN produced significantly higher drug concentrations in plasma from 2nd hour until 4th hour with a linear decrement and in the brain at all times. In conclusion, our study demonstrated the efficacy of a novel formulation of baclofen, which exploits the advantages of SLN preparations. However, for clinical purposes, high baclofen concentrations in brain tissue and sedation may be unwanted effects, requiring further studies and optimization of dosages.

Intrathecal baclofen in multiple sclerosis: too little, too late?

The majority of patients with multiple sclerosis (MS) have symptoms of spasticity that increasingly impair function as the disease progresses. With appropriate treatment, however, quality of life can be improved. Oral antispasticity medications are useful in managing mild spasticity but are frequently ineffective in controlling moderate to severe spasticity, because patients often cannot tolerate the adverse effects of increasing doses. Intrathecal baclofen (ITB) therapy can be an effective alternative to oral medications in patients who have a suboptimal response to oral medications or who cannot tolerate dose escalation or multidrug oral regimens. ITB therapy may be underutilized in the MS population because clinicians (a) are more focused on disease-modifying therapies rather than symptom control, (b) underestimate the impact of spasticity on quality of life, and (c) have concerns about the cost and safety of ITB therapy. Delivery of ITB therapy requires expertly trained staff and proper facilities for pump management. This article summarizes the findings and recommendations of an expert panel on the use of ITB therapy in the MS population and the role of the physician and comprehensive care team in patient selection, screening, and management.

[A system of prescriptions without drug approval: example of baclofen]. / Un dispositif de prescriptions hors-AMM : exemple du baclofène.

Baclofen is an anti-spastic drug that acts as an agonist of GABA-B receptors. It also seems to decrease the appetence for alcohol (anti-craving effect), although this effect has not been certified by Authorities for drug approval in France (AMM). However, baclofen receives a great deal of demand by patients hoping to reduce their alcohol consumption. Nonetheless, the lack of AMM and the high doses of baclofen supposed to exert an anti-craving effect often discourage practitioners from prescribing this drug in current medical practice. Therefore, it is preferable for a drug like baclofen to be prescribed under specific regulations. As such, certain criteria similar to those required in clinical trials are necessary to protect patients as well as the prescribing doctors. The criteria that are proposed here are: the use of drugs without AMM approval as a last resort (all other treatments must have failed), the collegiate decision for the drug prescription, good knowledge of the potency of the drug as well as good record keeping of patients and proper supervision. The departments of addiction, pharmacology and pharmacovigilance of the University Hospital of Lille, France present here a medical process named "multidisciplinary consultations for resort treatments of addictions" (CAMTEA). This process is designed to meet all the above mentioned criteria and to allow the use of baclofen as an anti-craving drug in safest conditions. If this proves to be successful with baclofen, it is possible to extend the use of CAMTEA to other drugs without AMM approval in addictologic pathologies.

Clinical and neurophysiologic assessment of strength and spasticity during intrathecal baclofen titration in incomplete spinal cord injury: single-subject design.

BACKGROUND/OBJECTIVE: Spasticity after spinal cord injury (SCI) is commonly managed with oral and intrathecal baclofen (ITB), with less attention to the effects on voluntary motor control. Studies combining clinical and neurophysiologic assessments during dose optimization are rare. Study aims (a) systematically evaluate effects of varied doses of oral and ITB on clinical and neurophysiologic measures of strength and spasticity and (b) relate clinical and neurophysiologic findings. METHODS: A 41-year-old man with an incomplete T11-ASIA D SCI was studied during ITB titration. Spasticity and strength in the lower extremities were assessed clinically and neurophysiologically at 5 different daily dosages of baclofen: (a) 80 mg oral, (b) 80 mg oral/50 microg ITB, (c) 80 mg oral/125 microg ITB, (d) 30 mg oral/125 microg ITB, and (e) 125 microg ITB only. RESULTS: A dose-dependent change in the Ashworth score and lower limb motor score was observed during titration of oral and ITB. Whereas the Hoffman (H)-reflex was abolished after the introduction of ITB, the flexion withdrawal reflex approximated a dose-dependent pattern. Changes in the motor score and EMG during voluntary muscle activation were proportionally smaller than the corresponding changes in clinical and neurophysiologic measures of spasticity. Neurophysiologic assessment largely paralleled clinical findings. CONCLUSIONS: This single-subject study shows that the control of spasticity can be achieved without detrimental effects on strength in incomplete SCI and suggests the need for including strength testing in comprehensive clinical assessment of spasticity. The study shows convergent validity between clinical and neurophysiologic assessments during ITB dose titration. Adding neurophysiologic assessment to clinical assessment may provide objectivity and sensitivity and facilitate decision-making during ITB titration.

The incidence and management of tolerance in intrathecal baclofen therapy.

STUDY DESIGN: Retrospective study. OBJECTIVES: To study the incidence and management of tolerance in patients treated with intrathecal baclofen (ITB) therapy. SETTING: Department of neurology and neurosurgery, University Medical Center Groningen, The Netherlands. METHODS: Medical records of all patients who had received an implantable ITB pump at our clinic during 1991-2005 were reviewed. RESULTS: A total of 37 patients (representing 116 pump years) were included. Mean follow-up time was 38 months (range 3-120 months). Baclofen dose increased in the first 18 months after implantation (P<0.05), and then stabilized around a mean dose of 350 microg per day. Eight patients (22%) developed tolerance, defined as a dose increase of >100 microg per year. No predictive factors for development of tolerance could be determined. Three different treatment regimens for tolerant patients were analyzed. Altering the infusion mode from simple to complex continuous (n=6) had no effect on the development of tolerance. Pulsatile bolus infusion (n=1) and a drug holiday (n=2) were both effective in reducing the daily baclofen dose. Patients who needed surgical revision of the pump system because of mechanical failures (n=11) showed a significant dose decrease during the first month after revision, indicating that the preoperative dose increase most likely had been caused by the pump failure. Pump-related complications occurred once per 10.5 years of ITB treatment. Drug-related side effects had an annual risk of 13.8%. The reported events were mostly mild. CONCLUSIONS: ITB therapy is effective and safe, also in the long term and causes tolerance in only 22% of the treated patients.

Cost-effectiveness modeling of intrathecal baclofen therapy versus other interventions for disabling spasticity.

OBJECTIVE: To assess by simulation the cost-effectiveness of intrathecal baclofen (ITB) therapy compared with conventional medical treatments for patients with disabling spasticity and functional dependence caused by any neurological disease. METHODS: Two models were created to simulate therapeutic strategies for managing severe spasticity, one with and one without the use of ITB, to assess various treatment sequences over 2 years based on current medical practices in France. Successful treatment at each evaluation was defined as a combination of: (1) the increased patient and caregiver satisfaction as assessed by goal attainment scaling (GAS), and (2) a decrease of at least 1 point on the Ashworth score. Probabilistic sensitivity analyses were performed using 5000 Monte-Carlo simulations taking into account specific distribution curves for direct costs and effectiveness parameters in each treatment option. RESULTS: The model simulations suggest that including ITB as a first option strategy in the management of function of severely impaired patients with disabling spasticity results in a higher success rate (78.7% vs 59.3%; P < .001). In addition, the ITB therapy model revealed a lower cost (pound 59,391 vs pound 88,272; P < .001) and an overall more favorable cost-effectiveness ratio (pound 75,204/success vs pound 148,822/success; P < .001), compared with conventional medical management without ITB. CONCLUSION: Within the assumptions of our modeling, ITB therapy evaluated by a combination of treatment success criteria at 6-month intervals over a 2-year period may be a cost-effective strategy compared to conventional medical management alone.