Interim analysis of post-marketing surveillance of eculizumab for paroxysmal nocturnal hemoglobinuria in Japan

Haruhiko Ninomiya, Naoshi Obara, Shigeru Chiba, Kensuke Usuki, Kaichi Nishiwaki, Itaru Matsumura, Tsutomu Shichishima, Shinichiro Okamoto, Jun ichi Nishimura, Kazuma Ohyashiki, Shinji Nakao, Kiyoshi Ando, Yoshinobu Kanda, Tatsuya Kawaguchi, Hideki Nakakuma, Daisuke Harada, Hirozumi Akiyama, Taroh Kinoshita, Keiya Ozawa, Mitsuhiro OmineYuzuru Kanakura

Research output: Contribution to journalArticlepeer-review

27 Citations (Scopus)


Data characterizing the safety and effectiveness of eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) are limited. We describe the safety and effectiveness of eculizumab in PNH patients enrolled in a post-marketing surveillance study. Types and frequencies of observed adverse events were similar to those reported in previous clinical trials and no meningococcal infection was reported. Effectiveness outcomes included the reduction of intravascular hemolysis, the change in hemoglobin (Hb) level, the withdrawal of transfusion and corticosteroids, the change of renal function, and overall survival. The effect of eculizumab on intravascular hemolysis was demonstrated by a reduction in lactate dehydrogenase levels at all measurements after baseline. Significant increases in Hb levels from baseline were also observed after 1 month’s treatment with eculizumab (p < 0.01). Of those who were transfusion-dependent at baseline, the median number of transfusions decreased significantly from 18 to 0 unit/year after 1 year of treatment with eculizumab (p < 0.001). An increase in Hb and a high rate of transfusion independence were observed, especially in patients with platelet count ≥150 × 109/L. Approximately 97 % of patients showed maintenance or improvement of renal function. Overall survival rate was about 90 % (median follow-up 1.9 years). These results suggest an acceptable safety profile and favorable prognosis after eculizumab intervention.

Original languageEnglish
Pages (from-to)548-558
Number of pages11
JournalInternational journal of hematology
Issue number5
Publication statusPublished - 2016 Nov 1


  • Eculizumab
  • Effectiveness
  • Paroxysmal nocturnal hemoglobinuria
  • Post-marketing surveillance
  • Safety

ASJC Scopus subject areas

  • Hematology


Dive into the research topics of 'Interim analysis of post-marketing surveillance of eculizumab for paroxysmal nocturnal hemoglobinuria in Japan'. Together they form a unique fingerprint.

Cite this