Cost-effectiveness of adrenocorticotropic hormone versus oral steroids for infantile spasms.

Abstract

OBJECTIVE:

To compare the effectiveness and cost-effectiveness of adrenocorticotropic hormone (ACTH) and oral steroids as first-line treatment for infantile spasm resolution, we performed a systematic review, meta-analysis, and cost-effectiveness study.

METHODS:

A decision analysis model was populated with effectiveness data from a systematic review and meta-analysis of existing literature and cost data from publicly available prices. Effectiveness was defined as the probability of clinical spasm resolution 14 days after treatment initiation.

RESULTS:

We included 21 studies with a total of 968 patients. The effectiveness of ACTH was not statistically significantly different from that of oral steroids (.70, 95% confidence interval [CI] = .60-.79 vs. .63, 95% CI = .56-.70; p = .28). Considering only the three available randomized trials with a total of 185 patients, the odds ratio of spasm resolution at 14 days with ACTH compared to high-dose prednisolone (4-8 mg/kg/day) was .92 (95% CI = .34-2.52, p = .87). Adjusting for potential publication bias, estimates became even more favorable to high-dose prednisolone. Using US prices, the more cost-effective treatment was high-dose prednisolone, with an incremental cost-effectiveness ratio (ICER) of $333 per case of spasms resolved, followed by ACTH, with an ICER of $1 432 200 per case of spasms resolved. These results were robust to multiple sensitivity analyses and different assumptions. Prednisolone at 4-8 mg/kg/day was more cost-effective than ACTH under a wide range of assumptions.

SIGNIFICANCE:

For infantile spasm resolution 2 weeks after treatment initiation, current evidence does not support the preeminence of ACTH in terms of effectiveness and, especially, cost-effectiveness.

• Authors: Iván Sánchez Fernández, Marta Amengual-Gual, Marina Gaínza-Lein, Cristina Barcia Aguilar, Ann Marie Bergin, Christopher J Yuskaitis, Chellamani Harini
• Journal: Epilepsia (Epilepsia) Vol. 62 Issue 2 Pg. 347-357 (02 2021) ISSN: 1528-1167 [Electronic] United States
• PMID: 33417252 (Publication Type: Journal Article, Meta-Analysis, Research Support, Non-U.S. Gov't, Systematic Review)
• Copyright: © 2020 International League Against Epilepsy.

Chemical References

• Glucocorticoids
• Hormones
• Adrenocorticotropic Hormone
• Prednisolone

Topics

• Adrenocorticotropic Hormone (economics, therapeutic use)
• Cost-Benefit Analysis
• Decision Support Techniques
• Dose-Response Relationship, Drug
• Glucocorticoids (economics, therapeutic use)
• Hormones (economics, therapeutic use)
• Humans
• Infant
• Prednisolone (economics, therapeutic use)
• Spasms, Infantile (drug therapy, economics)
• Treatment Outcome