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

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

Research output: Contribution to journalArticle

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 = 0.078). 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.

Original languageEnglish
Pages (from-to)1493-1503
Number of pages11
JournalAnnals of Hematology
Volume99
Issue number7
DOIs
Publication statusPublished - 2020 Jul 1

Keywords

  • Eculizumab
  • Nationwide analysis
  • Paroxysmal nocturnal hemoglobinuria
  • Survival

ASJC Scopus subject areas

  • Hematology

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    Kang, K. W., Moon, H., Lee, B. H., Jeon, M. J., Yu, E. S., Kim, D. S., Lee, S. R., Sung, H. J., Choi, C. W., Kim, B. S., Lee, J., & Park, Y. (2020). Nationwide study of paroxysmal nocturnal hemoglobinuria in South Korea: paradox of eculizumab. Annals of Hematology, 99(7), 1493-1503. https://doi.org/10.1007/s00277-020-04133-z