Effectiveness of once-daily high-dose ACTH for infantile spasms

Hodgeman, Ryan; Kapur, Kush; Paris, Ann; Marti, Candice; Can, Afra; Kimia, Amir A.; Loddenkemper, Tobias; Bergin, Ann M.; Poduri, Annapurna; Libenson, Mark H.; Lamb, Nathan; Jafarpour, Saba; Harini, Chellamani

doi:10.1016/j.yebeh.2016.03.025

Cited by 7 publications

(6 citation statements)

References 17 publications

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“…27 However, other studies have not confirmed these factors. 16,28 In the current study, we did not find a correlation between these factors and the short-term prognosis.…”

Section: Response To Treatmentcontrasting

confidence: 80%

EEG Biomarkers of Repository Corticotropin Injection Treatment

Tanritanir

Vieluf

Jafarpour

et al. 2021

Journal of Clinical Neurophysiology

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Hypsarrhythmia is one of the major diagnostic and treatment response criteria in infantile spasms (IS). The clinical and electrophysiological effect of repository corticotropin injection treatment on IS was evaluated using electrophysiological biomarkers.Methods: Consecutive infants (,24 months) treated with repository corticotropin injection for IS were included in this retrospective descriptive study. Inclusion criteria were (1) clinical IS diagnosis, (2) repository corticotropin injection treatment, and (3) consecutive EEG recordings before and after repository corticotropin injection treatment. Patients with tuberous sclerosis complex were excluded. Response to treatment was defined as freedom from IS for at least 7 consecutive days during the treatment and resolution of hypsarrhythmia. The authors defined "relapse" as the recurrence of seizures after an initial response. Electrophysiological biomarker assessment included evaluation of semiautomatic spike counting algorithm, delta power, and delta coherence calculation during non-REM sleep EEG.Results: One hundred fifty patients (83 males; 55%; median age of IS onset: 5.9 months) with complete data were included, including 101 responders (67%, 71 with sustained response, and 30 relapses). Fifty patients (33%) with complete EEG data also underwent advanced EEG analysis. Baseline delta coherence was higher in sustained responders than in nonresponders or patients who relapsed. Greater decreases in semiautomatic spike counting algorithm, delta power, and delta coherence were found in sustained responders compared with nonresponders or patients who relapsed.Conclusions: Repository corticotropin injection treatment was associated with a 67% response rate in patients with IS. Computational biomarkers beyond hypsarrhythmia may provide additional information during IS treatment, such as early determination of treatment response and outcome assessment.

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“…27 However, other studies have not confirmed these factors. 16,28 In the current study, we did not find a correlation between these factors and the short-term prognosis.…”

Section: Response To Treatmentcontrasting

confidence: 80%

EEG Biomarkers of Repository Corticotropin Injection Treatment

Tanritanir

Vieluf

Jafarpour

et al. 2021

Journal of Clinical Neurophysiology

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“…Existing literature does not support clinical superiority of ACTH over oral steroids. Even considering only the studies that used high‐dose (150 IU/m 2 /day) natural ACTH, there is no evidence to support that ACTH is clinically superior to oral steroids at day 14 (Modifying Effectiveness ) 5,12,40–42 …”

Section: Discussionmentioning

confidence: 99%

“…Even considering only the studies that used high-dose (150 IU/m 2 /day) natural ACTH, there is no evidence to support that ACTH is clinically superior to oral steroids at day 14 (Modifying Effectiveness S1). 5,12,[40][41][42] The most novel result in our study is the cost-effectiveness comparison. With the cost of a 14-day course of ACTH being more than 475 times more expensive than a 14-day course of oral prednisolone, it is not a surprise that oral prednisolone is more cost-effective than ACTH.…”

Section: Discussionmentioning

confidence: 99%

Cost‐effectiveness of adrenocorticotropic hormone versus oral steroids for infantile spasms

et al. 2021

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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.

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“…16,18 Results with such a high response rate could, however, not be replicated in a prospective, large national study by Knupp et al 20 In a more recent report from Boston Children's Hospital, outcome data with once-daily high-dosage practice were reported. 19 Forty of 57 children (70%) were spasm-free at day 14 after natural ACTH initiation. EEG showed the disappearance of hypsarrhythmia in all responders.…”

Section: Discussionmentioning

confidence: 96%