Effectiveness and cost-effectiveness of a patient-initiated botulinum toxin treatment model for blepharospasm and hemifacial spasm compared to standard care: study protocol for a randomised controlled trial.

BACKGROUND: Blepharospasm and hemifacial spasm are debilitating conditions that significantly impact on patient quality of life. Cyclical treatment with botulinum toxin injections offers temporary relief, but the duration of treatment efficacy is variable. The standard model of patient care defines routine fixed-time based scheduled treatment cycles which may lead to unnecessarily frequent treatment for some patients and experience of distressing symptoms in others, if symptoms return before the scheduled follow-up period. METHODS/DESIGN: A randomised controlled trial will compare a patient-initiated model of care, where patients determine botulinum toxin treatment timing, to the standard model of care in which care is scheduled by the clinical team. A sample of 266 patients with blepharospasm or hemifacial spasm will be recruited from Moorfields Eye Hospital (MEH), London. The trial will be accompanied by a mixed-methods evaluation of acceptability of the new service. Patients who meet eligibility criteria will be assessed at baseline and those in the intervention group will be provided with instructions on how to book their own treatment appointments. Patients in both groups will be followed up 3 and 9 months into the trial and all patients will be returned to usual care after 9 months to meet safety protocols. Primary outcome measures include disease severity (questionnaire), functional disability (questionnaire) and patient satisfaction with care (questionnaire). Secondary outcomes include disease-specific quality of life (questionnaire), mood (questionnaire), illness and treatment perceptions (questionnaire and semi-structured interviews), economic impact (questionnaire) and acceptability (questionnaire and semi-structured interviews). DISCUSSION: This trial will assess the effectiveness and cost-effectiveness of a patient-led care model for botulinum toxin therapy. If the new model is shown to be effective in reducing distress and disability in these populations and is found to be acceptable to patients, whilst being cost-effective, this will have significant implications for service organisation across the NHS. TRIAL REGISTRATION: UK Clinical Research Network (UKCRN) Portfolio 18660. Clinicaltrials.gov ID NCT102577224 (registered 29 October 2015).

Value-based neurosurgery: the example of microvascular decompression surgery.

OBJECT: Value of care is emerging as a promising framework to restructure health care, emphasizing the importance of reporting multiple outcomes that encompass the entire care episode instead of isolated outcomes specific to care points during a patient's care. The authors assessed the impact of coordinated implementation of processes across the episode of surgical care on value of neurosurgical care, using microvascular decompression (MVD) as an example. METHODS: This study is a retrospective review of consecutive cases involving patients with either trigeminal neuralgia or hemifacial spasm undergoing first-time MVD. Patients were divided into 2 groups: Group 1 included patients who underwent surgery between February 2008 and November 2009 and Group 2 included those who underwent surgery between January 2011 and October 2012. The authors collected data on outcome measures spanning the entire surgical episode of care according to the Outcome Measures Hierarchy. RESULTS: Forty-nine patients were included: 20 patients in Group 1 and 29 patients in Group 2. Thirty-one patients underwent MVD for trigeminal neuralgia and 18 for hemifacial spasm. A zero mortality rate and high degree of symptom resolution were achieved in both groups. Group 2 benefited from a reduction in the average total operating room time, a decrease in the mean and median postoperative length of hospital stay, a decrease in the mean length of stay on the floor, and a reduction in the rates of complications and readmissions. CONCLUSIONS: Comprehensive implementation of improvement processes throughout the continuum of care resulted in improved global outcome and greater value of delivered care. Enhanced-recovery perioperative protocols and diagnosis-specific clinical pathways are two avenues built around global care delivery that can help achieve an "optimal episode of surgical care" in every case.

Complementary therapies in hemifacial spasm and comparison with other movement disorders.

OBJECTIVES: We determined the prevalence, range and factors influencing the use of complementary therapy among hemifacial spasm patients and compared the patterns of use of complementary therapies across different movement disorders in a systematic pooled analysis of published literature. METHODS: A structured questionnaire was administered to 96 hemifacial spasm patients evaluating frequency of complementary therapy use, and factors influencing patients' decision to seek these therapies. We also performed a PubMed search of epidemiology studies on use of complementary therapies in movement disorders. RESULTS: Fifty-one per cent of patients had tried complementary therapies, of which 47% reported some perceived benefit and 4.1% informed their doctor. Acupuncture (71.4%) and facial massage (17.6%) were most commonly used. Complementary therapy use was associated with greater HFS severity. The mean cost of treatment was about $78 per month. We identified eight articles on use of complementary therapies in movement disorders; Parkinson's disease (5), Tourette syndrome (2) and dystonia (1). Twenty-five to 88% of patient had tried complementary therapies, of which 32-70% reported some benefit. Trials of acupuncture (2-63%) and massage (7-38%) were reported across the spectrum of movement disorders studied. Mean cost of complementary therapies varied from 43 to 102 USD per month. CONCLUSION: Complementary therapies are used by over 50% of HFS patients, and the use is correlated with severity of disease. Despite differences in race, culture and population demographics, acupuncture and massage are used by patients across the spectrum of movement disorders.

[Costs and efficacy of type A botulinum toxin for the treatment of essential blepharospasm and hemifacial spasm]. / Custos e eficácia da toxina botulínica tipo A no tratamento do blefaroespasmo essencial e espasmo hemifacial.

PURPOSE: To evaluate the costs and efficacy of type A botulinum toxin in the treatment of essential blepharospasm and hemifacial spasm. METHODS: Pacients with essential blepharospasm and hemifacial spasm had their files analyzed. All patients were treated with type A botulinum toxin (Dysport) between April 2002 and May 2004 at the Oculoplastic Clinics of "Santa Casa de São Paulo". Twenty-seven patients presented essential blepharospasm and 23 presented hemifacial spasm. Information about the patient's degree of satisfaction after treatment, complaints and personal costs were recorded by a questionnaire, and information about the costs of Dysport treatment were obtained at the administration department of "Santa Casa de São Paulo". Wilcoxon and Mann-Whitney tests were used for statistical analysis. RESULTS: 1- The annual treatment costs were R Dollars 1,239.32 for essential blepharospasm and R Dollars 661.72 for hemifacial spasm. 2- The patient's annual costs were R Dollars 145.48 for essential blepharospasm and R Dollars 6.07 for hemifacial spasm. 3- The hospital's annual costs for the treatment were R Dollars 1,095.84 for essential blepharospasm and R Dollars 535.65 for hemifacial spasm. 4- Dysport treatment is successful in both essential blepharospasm and hemifacial spasm. CONCLUSIONS: The costs of essential blepharospasm and hemifacial spasm treatment with Dysport are high, mainly because of the toxin price. On economic analysis of health, we can conclude that this procedure has an excellent cost-benefit ratio.

Custos e eficácia da toxina botulínica tipo A no tratamento do blefaroespasmo essencial e espasmo hemifacial / Costs and efficacy of type A botulinum toxin for the treatment of essential blepharospasm and hemifacial spasm

OBJETIVO: Avaliar os custos do tratamento para blefaroespasmo essencial e espasmo hemifacial com toxina botulínica tipo A (Dysport®), correlacionando-os com sua eficácia terapêutica. MÉTODOS: Análise de 50 prontuários de pacientes com blefaroespasmo essencial e espasmo hemifacial, submetidos à terapia com Dysport®, no período de abril de 2002 a maio de 2004 no setor de Oculo-Plástica da Santa Casa de São Paulo. Dos 50 pacientes, 27 apresentavam blefaroespasmo essencial e 23 espasmo hemifacial. Informações sobre grau de satisfação, queixas e custos pessoais foram obtidas mediante questionário. Os custos do medicamento e dos materiais foram pesquisados no almoxarifado e na farmácia da Santa Casa. Quanto ao custo das consultas, utilizou-se a tabela de pagamento do SUS. Para a estatística foram utilizados os testes de Wilcoxon e Mann-Whitney. RESULTADOS: O custo total anual do tratamento foi de R$ 1.239,32 para o blefaroespasmo essencial e R$ 661,72 para o espasmo hemifacial. Para o paciente, o custo anual foi de R$ 145,48 para o blefaroespasmo essencial e R$ 126,07 para o espasmo hemifacial. Para o hospital, o custo anual foi de R$ 1.095,84 para o blefaroespasmo essencial e R$ 535,65 para o espasmo hemifacial. O tratamento com Dysport® promoveu melhora funcional significativa nos dois grupos. CONCLUSÃO: O procedimento tem custo elevado, principalmente devido ao preço da toxina. Entretanto, pela análise econômica da saúde fica demonstrado que o procedimento possui excelente relação custo-benefício.

PURPOSE: To evaluate the costs and efficacy of type A botulinum toxin in the treatment of essential blepharospasm and hemifacial spasm. METHODS: Pacients with essential blepharospasm and hemifacial spasm had their files analyzed. All patients were treated with type A botulinum toxin (Dysport®) between April 2002 and May 2004 at the Oculoplastic Clinics of "Santa Casa de São Paulo". Twenty-seven patients presented essential blepharospasm and 23 presented hemifacial spasm. Information about the patient's degree of satisfaction after treatment, complaints and personal costs were recorded by a questionnaire, and information about the costs of Dysport® treatment were obtained at the administration department of "Santa Casa de São Paulo". Wilcoxon and Mann-Whitney tests were used for statistical analysis. RESULTS: 1- The annual treatment costs were R$ 1,239.32 for essential blepharospasm and R$ 661.72 for hemifacial spasm. 2- The patient's annual costs were R$ 145.48 for essential blepharospasm and R$ 6.07 for hemifacial spasm. 3- The hospital's annual costs for the treatment were R$ 1,095.84 for essential blepharospasm and R$ 535.65 for hemifacial spasm. 4- Dysport® treatment is successful in both essential blepharospasm and hemifacial spasm. CONCLUSIONS:. The costs of essential blepharospasm and hemifacial spasm treatment with Dysport® are high, mainly because of the toxin price. On economic analysis of health, we can conclude that this procedure has an excellent cost-benefit ratio.

[Long-term treatment with botulinum toxin: dosage, treatment schedules and costs]. / Langzeitbehandlung mit Botulinum Toxin: Dosierung, Behandlungsabstände und Kosten.

Local injection of botulinum toxin A (BTX) is considered as the treatment of choice for spasmodic torticollis (ST), blepharospasm (BS) and, with certain limitations, for hemifacial spasm (HFS). Long-term data from 89 patients with ST, 39 patients with BS and 15 patients with HFS were evaluated and subgroups of patients who received at least three (60 ST, 17 BS, 9 HFS), six (35 ST, 10 BS, 8 HFS) and nine (26 ST) re-injections were formed. These groups were investigated in regard of increases in the dosage of BTX and in regard of shorter treatment intervals in cases of repeated treatment. Annual drug costs were calculated from these data. Patients treated at least three times were asked to fill out a questionnaire concerning satisfaction and the quality of life after BTX treatment. Furthermore, the patients' "willingness to pay" for BTX treatment was investigated. Our calculations showed neither a significant increase in the dosage of BTX nor a significant reduction in the length of treatment intervals after 3 or 6 treatment sessions. Annual drug costs for BTX for the treatment of ST were 10,542 ATS, of BS 2847 ATS and of HFS 1029 ATS. 86.1% of patients with ST, 88.9% with BS and 100% with HFS were very satisfied or satisfied with the BTX treatment. In all 3 diagnostic groups, a significant mean improvement of quality of life was measured on a visual analogue scale (p < 0.001). We conclude that the injection of botulinum toxin A is a highly effective and cost effective treatment for ST, BS and HFS, and significantly increases the quality of life of all these patients.