Medication selection, health services outcomes, and cost trajectories for Medicaid beneficiaries with infantile spasms.

OBJECTIVE

There are three recommended first-line treatments for infantile spasms, adrenocorticotropic hormone (ACTH), oral corticosteroids, and vigabatrin, though non-standard treatments such as topiramate are sometimes selected. Is it uncertain how treatment selection influences health services outcomes.

METHODS

We conducted a retrospective cohort study of Medicaid beneficiaries newly diagnosed with infantile spasms from 2009-2010. We included infants with a new diagnosis of infantile spasms between age 2-9 months who filled ACTH (reference), prednisolone, vigabatrin, or topiramate prescriptions. Multivariable Cox proportional hazards regression compared time to first emergency department (ED) visit or hospitalization across treatment groups during 2 years of follow-up. Monthly costs for each treatment were examined in 6-month intervals and compared in a multivariable generalized linear model.

RESULTS

Among 256 children with infantile spasms, 116 received ACTH, 62 prednisolone, 15 vigabatrin, and 63 topiramate. The rate of ED visit or hospitalization per person-year did not differ significantly for prednisolone (0.9 [95 % CI 0.7-1.2]; adjusted hazard ratio [aHR] 0.84, 95 % CI 0.57-1.24), vigabatrin (0.8 [95 % CI 0.4-1.5]; aHR 0.91, 95% CI 0.45-1.84), or topiramate (1.7 [95 % CI 1.3-2.3]; aHR 1.15, 95 % CI 0.80-1.65), when compared to ACTH (1.1 [95 % CI 0.9-1.3]). The median payment for ACTH was $96,406 (interquartile range 70,742-138,476) during the first 6 months. The adjusted mean total payment in the first 6 months was 73% lower for prednisolone (95% CI -82, -61), 69% lower for vigabatrin (95% CI -84, -40), and 73% lower for topiramate (95% CI -82, -59). However, in subsequent 6-month intervals, costs associated with ACTH were not significantly different compared to other treatments.

SIGNIFICANCE: Compared to other treatments for infantile spasms, use of ACTH was associated with greater cost in the first 6 months of treatment, but not with reduced ED visits or hospitalizations. The cost effectiveness of ACTH depends on its relative clinical efficacy, and merits additional research.