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High-dose dexamethasone as a replacement for traditional prednisone as the first-line treatment in children with previously untreated primary immune thrombocytopenia: a prospective, randomized single-center study

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Abstract

Immune thrombocytopenia (ITP) is one of the most common acquired immune-mediated bleeding disorders found in children. Prednisone is usually considered a first-line therapeutic agent for ITP in children. Yet, prolonged exposure to prednisone has been associated with certain side effects. This prospective randomized study comparatively assessed the efficacy and safety of short-course high-dose dexamethasone (HDD) and standard prednisone (PDN) as a first-line treatment for children with previously untreated primary ITP. Two hundred eleven children were randomized into the HDD (n = 110) and PDN (n = 101) groups. There was no difference in baseline characteristics between the two groups (p > 0.05). Early response rates were 92.7% and 93% (p = 0.923); initial response rates were 93.6% and 95% (p = 0.658) and durable response rates were 90% and 91% (p = 0.787) in the HDD and PDN groups, respectively. More remission patients in the HDD group compared with the PDN group (86.3% vs. 80.1%) at 12th month after treatment, yet no statistical difference was observed (p = 0.703). Bleeding events were 10.9% and 14.8% (p = 0.105), and bleeding score improvement rates were 78.2% and 76.2% (p = 0.284) in the HDD and PDN groups, respectively. Cushing’s disease, weight gain and infection rates were higher in the PDN group compared to the HDD group (80% vs. 10%, p = 0.001; 74.2% vs. 13.6%, p = 0.001; and 26% vs. 11.8%, p = 0.012) 1 month after treatment. HDD showed non-inferior efficacy and fewer glucocorticoid-related adverse effects compared with PDN. These findings indicated that HDD could be considered as a first-line treatment in children with previously untreated primary ITP, thus replacing standard PDN.

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Abbreviations

HDD:

High-dose dexamethasone

ITP:

Immune thrombocytopenia

PDN:

Prednisone

CR:

Complete response

R:

Response

NR:

No response

IWG:

International Working Group

R:

Response

NR:

No response

ASH:

The American Society of Hematology

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Acknowledgements

This work was supported by the National Natural Science Foundation of China (No. 81970111), the Beijing Natural Science Foundation of China (No. 7192064), the Special Fund of the Pediatric Medical Coordinated Development Center of Beijing Hospitals Authority (No. XTZD20180205), and the National Science and Technology Key Projects (No. 2017ZX0930402900).

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Authors and Affiliations

  1. Department of Hematology and Oncology, Beijing Children’s Hospital, Capital Medical University, Nanlishi Road No. 56, Xicheng District, Beijing, 100045, People’s Republic of China

    Jie Ma, Lingling Fu, Zhengping Chen, Hao Gu, Jingyao Ma & Runhui Wu

  2. Beijing Key Laboratory of Pediatric Hematology Oncology, National Center for Children’s Health, Beijing, 100045, China

    Jie Ma, Lingling Fu, Zhengping Chen, Hao Gu, Jingyao Ma & Runhui Wu

  3. Key Laboratory of Major Diseases in Children, Ministry of Education, National Key Discipline of Pediatrics (Capital Medical University), Beijing, 100045, China

    Jie Ma, Lingling Fu, Zhengping Chen, Hao Gu, Jingyao Ma & Runhui Wu

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  1. Jie Ma
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Correspondence to Runhui Wu.

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The authors declare no competing financial interests.

Ethical approval

This study was conducted in accordance with the Declaration of Helsinki and approved by the Institutional Review Board (IRB) of Beijing Children’s Hospital, Capital Medical University. All parents signed informed consent forms.

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Ma, J., Fu, L., Chen, Z. et al. High-dose dexamethasone as a replacement for traditional prednisone as the first-line treatment in children with previously untreated primary immune thrombocytopenia: a prospective, randomized single-center study. Int J Hematol 112, 773–779 (2020). https://doi.org/10.1007/s12185-020-02977-9

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  • DOI: https://doi.org/10.1007/s12185-020-02977-9

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