Cost-effectiveness of Intrathecal Baclofen Therapy for the Treatment of Severe Spasticity Associated With Cerebral Palsy

Abstract

Spasticity is relatively common among children with cerebral palsy. This condition can be painful, can severely impair a child's ability to perform basic tasks, and can place an enormous emotional and financial burden on the family. Intrathecal baclofen delivered via an implantable pump is an effective treatment option for children unresponsive to oral medication and needing generalized motor control. However, the initial investment for the delivery device and its surgical placement can be a barrier to access. A cost-effectiveness analysis of intrathecal baclofen for adults in the British health care system concluded that intrathecal baclofen offered good value for the money. No similar analysis of intrathecal baclofen has been conducted in the context of the US health care system, and no study has specifically examined cost-effectiveness of intrathecal baclofen in a pediatric population. The aim of this article is to assess the cost-effectiveness of intrathecal baclofen among children with severe spasticity of cerebral origin who have not responded to less invasive treatments such as oral medications relative to alternative medical and surgical therapy. The authors used mathematical modeling and computer simulation to estimate the incremental cost per quality-adjusted life-year for identical cohorts of children treated with intrathecal baclofen or alternative therapy over a 5-year episode of treatment. Data on treatment costs representative of these children were derived from a health insurance claims database that included both commercial and Medicaid data. Utility values used to construct quality-adjusted life-years were obtained from a panel of expert clinicians who used the Health Utilities Index—2 to rate health states associated with the course of treatment. On average, intrathecal baclofen therapy increased the 5-year cost of treatment by $49 000 relative to alternative treatment. However, this was accompanied by an average gain of 1.2 quality-adjusted life-years. The net result was an incremental cost-effectiveness ratio of $42 000 per quality-adjusted life-year, a figure well within the $50 000 to $100 000 range that is widely accepted as offering good value for the money.

Keywords

spasticity baclofen cost-effectiveness

Get full access to this article

View all access options for this article.

References

Lance JW Symposium synopsis. In: Feldman RG , Young RR , Koella WP , eds. Spasticity: Disordered Motor Control. Chicago, Ill: Year Book; 1980:480-494.

Rawlins P. Patient management of cerebral origin spasticity with intrathecal baclofen . J Neurosci Nurs. 1998;30:32-35, 40-46.

Awaad Y. , Tayem H. , Munoz S. , et al. Functional assessment following intrathecal baclofen therapy in children with spastic cerebral palsy. J Child Neurol. 2003;18:26-34.

Bjornson KF , McLaughlin JF , Loeser JD , et al. Oral motor, communication, and nutritional status of children during intrathecal baclofen therapy: a descriptive pilot study. Arch Phys Med Rehabil. 2003;84:500-506.

Gormley ME Jr , Krach LE , Piccini L. Spasticity management in the child with spastic quadriplegia. Eur J Neurol. 2001;8:127-135.

Krach LE Pharmacotherapy of spasticity: oral medications and intrathecal baclofen. J Child Neurol. 2001;16:31-36.

Boyd RN , Hays RM Outcome measurement of effectiveness of botulinum toxin type A in children with cerebral palsy: an ICIDH-2 approach. Eur J Neurol. 2001;8:167-177.

Dralle D. , Muller H. , Zierski J. , et al. Intrathecal baclofen for spasticity. Lancet . 1985;2:1003.

Butler C. , Campbell S. Evidence of the effects of intrathecal baclofen for spastic and dystonic cerebral palsy. AACPDM Treatment Outcomes Committee Review Panel. Dev Med Child Neurol. 2000;42:634-645.

10.

Campbell SK , Almeida GL , Penn RD , et al. The effects of intrathecally administered baclofen on function in patients with spasticity. Phys Ther. 1995;75:352-362.

11.

Gilmartin R. , Bruce D. , Storrs BB , et al. Intrathecal baclofen for management of spastic cerebral palsy: multicenter trial. J Child Neurol. 2000;15:71-77.

12.

Albright AL , Gilmartin R. , Swift D. , et al. Long-term intrathecal baclofen therapy for severe spasticity of cerebral origin. J Neurosurg. 2003;98:291-295.

13.

Dan B. , Bouillot E. , Bengoetxea A. , et al. Effect of intrathecal baclofen on gait control in human hereditary spastic paraparesis. Neurosci Lett. 2000;280:175-178.

14.

Sgouros S. , Seri S. The effect of intrathecal baclofen on muscle co-contraction in children with spasticity of cerebral origin. Pediatr Neurosurg. 2002;37:225-230.

15.

Albright AL Intrathecal baclofen in cerebral palsy movement disorders. J Child Neurol. 1996;11(Suppl 1):S29-S35.

16.

Sampson FC , Hayward A. , Evans G. , et al. Functional benefits and cost/benefit analysis of continuous intrathecal baclofen infusion for the management of severe spasticity. J Neurosurg. 2002;96:1052-1057.

17.

Torrance GW Measurement of health state utilities for economic appraisal. J Health Econ. 1986;5:1-30.

18.

Torrance GW , Feeny DH , Furlong WJ , et al. Multiattribute utility function for a comprehensive health status classification system. Health Utilities Index Mark 2. Med Care. 1996;34:702-722.

19.

Torrance GW , Furlong W. , Feeny D. Health utility estimation. Expert Rev Pharmacoeconomics Res. 2002;2:99-108.

20.

Leidy NK , Revicki DA , Geneste B.

Recommendations for evaluating the validity of quality of life claims for labeling and promotion. Value

Health. 1999 ;2:113-127.

21.

Patrick DL , Chiang YP Measurement of health outcomes in treatment effectiveness evaluations: conceptual and methodological challenges. Med Care. 2000 ;38:1114-1125.

22.

Albright AL , Barron WB , Fasick MP , et al. Continuous intrathecal baclofen infusion for spasticity of cerebral origin. JAMA. 1993 ;270:2475-2477.

23.

Albright AL , Barry MJ , Fasick MP , et al. Effects of continuous intrathecal baclofen infusion and selective posterior rhizotomy on upper extremity spasticity . Pediatr Neurosurg. 1995;23:82-85.

24.

Almeida GL , Campbell SK , Girolami GL , et al. Multidimensional assessment of motor function in a child with cerebral palsy following intrathecal administration of baclofen. Phys Ther. 1997;77: 751-764.

25.

Armstrong RW , Steinbok P. , Cochrane DD , et al. Intrathecally administered baclofen for treatment of children with spasticity of cerebral origin. J Neurosurg. 1997;87:409-414.

26.

Campbell WM , Ferrel A. , McLaughlin JF , et al. Long-term safety and efficacy of continuous intrathecal baclofen. Dev Med Child Neurol. 2002;44:660-665.

27.

Gerszten PC , Albright AL , Barry MJ Effect on ambulation of continuous intrathecal baclofen infusion. Pediatr Neurosurg. 1997 ;27:40-44.

28.

Gerszten PC , Albright AL , Johnstone GF Intrathecal baclofen infusion and subsequent orthopedic surgery in patients with spastic cerebral palsy. J Neurosurg. 1998;88:1009-1013.

29.

Gooch JL , Oberg WA , Grams B. , et al. Complications of intrathecal baclofen pumps in children. Pediatr Neurosurg. 2003;39:1-6.

30.

Murphy NA , Irwin MC , Hoff C. Intrathecal baclofen therapy in children with cerebral palsy: efficacy and complications. Arch Phys Med Rehabil. 2002 ;83:1721-1725.

31.

Scheinberg A. , O'Flaherty S. , Chaseling R. , et al. Continuous intrathecal baclofen infusion for children with cerebral palsy: a pilot study. J Paediatr Child Health. 2001;37:283-288.

32.

Wiens HD Spasticity in children with cerebral palsy: a retrospective review of the effects of intrathecal baclofen. Issues Compr Pediatr Nurs. 1998;21:49-6133.

33.

Palisano R. , Rosenbaum P. , Walter S. , et al. Development and reliability of a system to classify gross motor function in children with cerebral palsy. Dev Med Child Neurol. 1997;39:214-223.

34.

Horsman J. , Furlong F. , Feeny D. , et al. The Health Utilities Index (HUI®): concepts, measurement properties and applications. Health Qual Life Outcomes. 2003;1:54.

35.

Furlong WJ , Feeny DH , Torrance GW , et al. The Health Utilities Index (HUI) system for assessing health-related quality of life in clinical studies. Ann Med. 2001;33:375-384.

36.

Feeny D. , Furlong W. , Torrance GW , et al. Multiattribute and single-attribute utility functions for the Health Utilities Index Mark 3 system. Med Care. 2002;40:113-128.

37.

US Department of Labor. Bureau of Labor Statistics Data: Consumer Price Index—All Urban Consumers . Washington, DC: US Department of Labor; 2005.

38.

Gajdosik CG , Cicirello N. Secondary conditions of the musculoskeletal system in adolescents and adults with cerebral palsy. Phys Occup Ther Pediatr. 2001;21:49-68.

39.

Morrell DS , Pearson JM , Sauser DD Progressive bone and joint abnormalities of the spine and lower extremities in cerebral palsy. Radiographics. 2002;22:257-268.

40.

Elfron B. , Tibshirani RJ An Introduction to the Bootstrap. New York, NY: Chapman & Hall; 1993.

41.

Griebsch I. , Coast J. , Brown J. Quality-adjusted life-years lack quality in pediatric care: a critical review of published cost-utility studies in child health. Pediatrics. 2005;115:e600-e614.

42.

Bichey BG , Hoversland JM , Wynne MK , et al. Changes in quality of life and the cost-utility associated with cochlear implantation in patients with large vestibular aqueduct syndrome . Otol Neurotol. 2002;23:323-327.

43.

Mahle WT , Forbess JM , Kirshbom PM , et al. Cost-utility analysis of salvage cardiac extracorporeal membrane oxygenation in children. J Thorac Cardiovasc Surg. 2005;129:1084-1090.

44.

Yount LE , Mahle WT Economic analysis of palivizumab in infants with congenital heart disease . Pediatrics. 2004;114:1606-1611.

45.

Krach LE , Kriel RL , Gilmartin RC , et al. Hip status in cerebral palsy after one year of continuous intrathecal baclofen infusion. Pediatr Neurol. 2004;30:163-168.

46.

Steinbok P. , Daneshvar H. , Evans D. , et al. Cost analysis of continuous intrathecal baclofen versus selective functional posterior rhizotomy in the treatment of spastic quadriplegia associated with cerebral palsy. Pediatr Neurosurg. 1995 ;22:255-264.

47.

Luce BR , Manning WG , Siegel JE , et al. Estimating costs in cost-effectiveness analysis. In: Gold MR , Siegel JE , Russell LB , et al, eds. Cost-effectiveness in Health and Medicine. New York, NY: Oxford University Press; 1996:176-213.