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Nationwide study of paroxysmal nocturnal hemoglobinuria in South Korea: paradox of eculizumab

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Abstract

Eculizumab is effective in managing patients with paroxysmal nocturnal hemoglobinuria (PNH). In South Korea, the financial support for eculizumab therapy is extended by the National Health Insurance Services (NHIS) only to patients with high-risk PNH for approximately 10 years. In this study, we performed a nationwide analysis of the real-world efficacy of eculizumab therapy in patients diagnosed with PNH between January 1, 2002, and December 31, 2016, by using the NHIS database. Patients treated with eculizumab (the eculizumab-treated group) exhibited a significantly higher survival rate than patients not treated with eculizumab (the eculizumab-untreated group), with 4-year survival rates after propensity score matching of 98.31% and 79.67%, respectively (p = 0.0489). The mean red blood cell (RBC) transfusion units per 12 months after eculizumab therapy were significantly lower than that before eculizumab therapy (5.75 units vs. 12.28 units, p < 0.0001). The median time for the first transfusion in the eculizumab-treated group was significantly longer than that in the eculizumab-untreated group. The 4-year transfusion-independence rate for the eculizumab-treated group was significantly higher than that for the eculizumab-untreated group (20.81% vs. 10.24%, p = 0.0078). There was no significant difference between the two groups in the incidence of new documented complications related to PNH. In conclusion, eculizumab therapy for patients with high-risk PNH may effectively improve the survival rate and reduce the transfusion requirement. Paradoxically, eculizumab-treated patients with severe PNH exhibit a higher survival rate than eculizumab-untreated patients with less severe PNH.

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Funding

This study was supported by grants from the Korean Health Technology R&D Project, Ministry of Health & Welfare, South Korea (grant number: HI14C2750).

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Author notes

  1. Ka-Won Kang and Hyemi Moon contributed equally to this work.

Authors and Affiliations

  1. Division of Hematology-Oncology, Department of Internal Medicine, Korea University College of Medicine, 73, Goryeodae-ro, Seongbuk-gu, Seoul, 02841, South Korea

    Ka-Won Kang, Byung-Hyun Lee, Min Ji Jeon, Eun Sang Yu, Dae Sik Kim, Se Ryeon Lee, Hwa Jung Sung, Chul Won Choi, Byung Soo Kim & Yong Park

  2. Department of Biostatistics, Korea University College of Medicine, Seoul, South Korea

    Hyemi Moon & Juneyoung Lee

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  1. Ka-Won Kang
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Correspondence to Yong Park.

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Conflict of interest

The authors declare that they have no competing interests.

Ethics approval

All procedures in studies involving human participants were performed in accordance with the ethical standards of the institutional and national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. The study was approved by the institutional review board of Korea University (No. KU-IRB-17-EX-253-A-1).

Consent to participate

As this study was conducted using anonymous patient data from the National Health Insurance Database in South Korea, the requirement for informed consent was waived by the institutional review board of Korea University (No. KU-IRB-17-EX-253-A-1).

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The original version of this article was revised: The original version of this article inadvertently contained a typographical error in the Abstract section. In the Abstract, the p-value demonstrating the statistically significant difference between the transfusion-independence rate for the eculizumab-treated group and the eculizumab-untreated group should be p = 0.0078, and not p = 0.078.

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Kang, KW., Moon, H., Lee, BH. et al. Nationwide study of paroxysmal nocturnal hemoglobinuria in South Korea: paradox of eculizumab. Ann Hematol 99, 1493–1503 (2020). https://doi.org/10.1007/s00277-020-04133-z

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  • DOI: https://doi.org/10.1007/s00277-020-04133-z

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