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Effectiveness and Safety of Different Once-Daily Doses of Adrenocorticotropic Hormone for Infantile Spasms

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Abstract

Introduction

Adrenocorticotropic hormone (ACTH) has been commonly used as a first-line treatment for infantile spasms (IS), but its optimal dose and duration are still unclear. This study is the largest retrospective cohort to document the therapeutic efficacy and tolerability for three gradient doses of ACTH in IU/kg/day units in Chinese patients.

Objective

The aim of our study was to elucidate the effectiveness and safety of three different low doses and duration of ACTH treatment for IS in China.

Methods

We conducted a retrospective, chart review of IS cases that were treated with biologic short-acting ACTH and followed up for at least 6 months at a single center in China between June 2010 and June 2016. In total, 200 children met the inclusion criteria. Cases were divided into three groups according to dosage (1, 1.1–1.9, and 2–4 IU/kg/day). Furthermore, we divided the 2–4 IU/kg/day group into 2–3 and 3.1–4 IU/kg/day subgroups. All groups were evaluated for response rates, relapse rates, and adverse effects.

Results

Electroclinical remission by day 14 occurred in 41.4% of infants given 2–4 IU/kg/day and 36.4% of infants given 1.1–1.9 IU/kg/day, compared with only 14.7% of patients given 1 IU/kg/day (p = 0.004 and 0.03, respectively). Prolonging ACTH treatment for up to 28 days improved response by 24% in all 200 infants. Overall, 73.9% of infants receiving 2–4 IU/kg/day responded, significantly higher than the 52.7% responding to 1.1–1.9 IU/kg/day and the 23.5% responding to 1 IU/kg/day (p < 0.01). There was no significant difference in the number of relapses or adverse effects in the three groups. Moreover, in the 2–4 IU/kg/day group, 74.7% of children receiving 2–3 IU/kg/day of ACTH responded, compared with 70% who responded to 3.1–4 IU/kg/day (p = 0.78).

Conclusions

ACTH at a dosage of 2–3 IU/kg/day is superior to 1.1–1.9 and 1 IU/kg/day dosages, is as good as a 3.1–4 IU/kg/day dosage in terms of response rate, and causes no more adverse effects or relapses than other dosages. In addition, prolonging the duration of ACTH treatment can improve response.

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Acknowledgements

The authors thank Prof. Michael V. Johnston at Johns Hopkins Medicine for the critical review of this manuscript.

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Authors and Affiliations

  1. Department of Dermatology, The Second Xiangya Hospital, Central South University, Changsha, China

    Jinghua Yin & Qianjin Lu

  2. Hunan Key Laboratory of Medical Epigenomics, Changsha, China

    Jinghua Yin & Qianjin Lu

  3. Department of Pediatrics, Xiangya Hospital, Central South University, Xiangya Road 87, Changsha, Hunan, China

    Fei Yin, Ying Wang, Fang He, Liwen Wu, Lifen Yang, Xiaolu Deng, Chen Chen & Jing Peng

  4. Hunan Intellectual and Developmental Disabilities Research Center, Changsha, China

    Fei Yin, Ying Wang, Fang He, Liwen Wu, Lifen Yang, Xiaolu Deng, Chen Chen & Jing Peng

Authors
  1. Jinghua Yin
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  5. Fang He
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Contributions

All authors participated in developing the concept and design of the study. JY and YW obtained data, performed statistical analyses, and prepared the figures. JY was responsible for drafting and revising the manuscript. JP provided significant manuscript revisions and approved the final manuscript as submitted. All authors approved the final manuscript as submitted and agree to be accountable for all aspects of the work.

Corresponding author

Correspondence to Jing Peng.

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Funding

This work was kindly supported by the National Natural Science Foundation of China (81371434), the National Natural Science Foundation of China (81370771), the Natural Science Foundation of Hunan province (2015JJ3151) and the Hunan Province Key Technology Support Program (2015SK20191).

Conflict of interest

Jinghua Yin, Qianjin Lu, Fei Yin, Ying Wang, Fang He, Liwen Wu, Lifen Yang, Xiaolu Deng, Chen Chen and Jing Peng have no conflict of interest to disclose.

Ethical approval

This study was approved by the research ethical committee of Xiangya Hospital, Central South University.

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Yin, J., Lu, Q., Yin, F. et al. Effectiveness and Safety of Different Once-Daily Doses of Adrenocorticotropic Hormone for Infantile Spasms. Pediatr Drugs 19, 357–365 (2017). https://doi.org/10.1007/s40272-017-0225-5

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  • DOI: https://doi.org/10.1007/s40272-017-0225-5

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