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 journalArticle

12 Citations (Scopus)

Abstract

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)1-11
Number of pages11
JournalInternational Journal of Hematology
DOIs
Publication statusAccepted/In press - 2016 Jul 27

Fingerprint

Paroxysmal Hemoglobinuria
Marketing
Japan
Hemoglobins
Hemolysis
Safety
Meningococcal Infections
Kidney
Platelet Count
L-Lactate Dehydrogenase
eculizumab
Adrenal Cortex Hormones
Survival Rate
Maintenance
Clinical Trials
Survival
Therapeutics

Keywords

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

ASJC Scopus subject areas

  • Hematology

Cite this

Ninomiya, H., Obara, N., Chiba, S., Usuki, K., Nishiwaki, K., Matsumura, I., ... Kanakura, Y. (Accepted/In press). Interim analysis of post-marketing surveillance of eculizumab for paroxysmal nocturnal hemoglobinuria in Japan. International Journal of Hematology, 1-11. https://doi.org/10.1007/s12185-016-2065-4

Interim analysis of post-marketing surveillance of eculizumab for paroxysmal nocturnal hemoglobinuria in Japan. / Ninomiya, Haruhiko; Obara, Naoshi; Chiba, Shigeru; Usuki, Kensuke; Nishiwaki, Kaichi; Matsumura, Itaru; Shichishima, Tsutomu; Okamoto, Shinichiro; Nishimura, Jun ichi; Ohyashiki, Kazuma; Nakao, Shinji; Ando, Kiyoshi; Kanda, Yoshinobu; Kawaguchi, Tatsuya; Nakakuma, Hideki; Harada, Daisuke; Akiyama, Hirozumi; Kinoshita, Taroh; Ozawa, Keiya; Omine, Mitsuhiro; Kanakura, Yuzuru.

In: International Journal of Hematology, 27.07.2016, p. 1-11.

Research output: Contribution to journalArticle

Ninomiya, H, Obara, N, Chiba, S, Usuki, K, Nishiwaki, K, Matsumura, I, Shichishima, T, Okamoto, S, Nishimura, JI, Ohyashiki, K, Nakao, S, Ando, K, Kanda, Y, Kawaguchi, T, Nakakuma, H, Harada, D, Akiyama, H, Kinoshita, T, Ozawa, K, Omine, M & Kanakura, Y 2016, 'Interim analysis of post-marketing surveillance of eculizumab for paroxysmal nocturnal hemoglobinuria in Japan', International Journal of Hematology, pp. 1-11. https://doi.org/10.1007/s12185-016-2065-4
Ninomiya, Haruhiko ; Obara, Naoshi ; Chiba, Shigeru ; Usuki, Kensuke ; Nishiwaki, Kaichi ; Matsumura, Itaru ; Shichishima, Tsutomu ; Okamoto, Shinichiro ; Nishimura, Jun ichi ; Ohyashiki, Kazuma ; Nakao, Shinji ; Ando, Kiyoshi ; Kanda, Yoshinobu ; Kawaguchi, Tatsuya ; Nakakuma, Hideki ; Harada, Daisuke ; Akiyama, Hirozumi ; Kinoshita, Taroh ; Ozawa, Keiya ; Omine, Mitsuhiro ; Kanakura, Yuzuru. / Interim analysis of post-marketing surveillance of eculizumab for paroxysmal nocturnal hemoglobinuria in Japan. In: International Journal of Hematology. 2016 ; pp. 1-11.
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AU - Nishiwaki, Kaichi

AU - Matsumura, Itaru

AU - Shichishima, Tsutomu

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AU - Ohyashiki, Kazuma

AU - Nakao, Shinji

AU - Ando, Kiyoshi

AU - Kanda, Yoshinobu

AU - Kawaguchi, Tatsuya

AU - Nakakuma, Hideki

AU - Harada, Daisuke

AU - Akiyama, Hirozumi

AU - Kinoshita, Taroh

AU - Ozawa, Keiya

AU - Omine, Mitsuhiro

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