PMID- 37477863
OWN - NLM
STAT- MEDLINE
DCOM- 20230811
LR  - 20230811
IS  - 1865-3774 (Electronic)
IS  - 0925-5710 (Linking)
VI  - 118
IP  - 3
DP  - 2023 Sep
TI  - Interim analysis of post-marketing surveillance of ravulizumab for paroxysmal 
      nocturnal hemoglobinuria in Japan.
PG  - 311-322
LID - 10.1007/s12185-023-03625-8 [doi]
AB  - Ravulizumab is a long-acting C5 inhibitor available for treating paroxysmal 
      nocturnal hemoglobinuria (PNH). Post-marketing surveillance (PMS) was implemented 
      following its approval in September 2019 in Japan. We report safety data obtained 
      through to December 2021 for 218 patients and effectiveness data for 194 patients 
      (182 switched from eculizumab and 12 complement inhibitor-naive). Over a median 
      follow-up of 74.4 weeks, 193 adverse events (AEs) were reported in 66/218 
      patients (30.3%; incidence 72.73/100 patient-years). The two most frequent AEs 
      were anemia and pyrexia (each 3.01/100 patient-years). The incidence of serious 
      AEs was 36.93/100 patient-years. In patients who switched from eculizumab, 
      lactate dehydrogenase (LDH) and hemoglobin (Hb) levels were maintained over 
      26 weeks of ravulizumab treatment. In complement inhibitor-naive patients, LDH 
      decreased significantly and Hb increased significantly over 26 weeks of 
      ravulizumab treatment. These data for Japanese patients with PNH who were naive 
      to complement inhibitors and patients who switched from eculizumab show that the 
      safety and effectiveness of ravulizumab are consistent with the published 
      clinical trial data. However, transfusion independence was less likely in 
      patients with bone marrow failure. Further follow-up data from this PMS will help 
      to elucidate the long-term clinical safety and effectiveness of ravulizumab for 
      treating PNH.
CI  - (c) 2023. Japanese Society of Hematology.
FAU - Usuki, Kensuke
AU  - Usuki K
AUID- ORCID: 0000-0002-1216-4470
AD  - Department of Hematology, NTT Medical Center Tokyo, Tokyo, Japan. 
      kensuke.usuki@gmail.com.
FAU - Ikezoe, Takayuki
AU  - Ikezoe T
AD  - Department of Hematology, Fukushima Medical University, Fukushima, Japan.
FAU - Ishiyama, Ken
AU  - Ishiyama K
AD  - Department of Hematology, Kanazawa University Hospital, Kanazawa, Japan.
FAU - Kanda, Yoshinobu
AU  - Kanda Y
AD  - Department of Hematology, Jichi Medical University, Shimotsuke, Japan.
FAU - Gotoh, Akihiko
AU  - Gotoh A
AD  - Department of Hematology, Tokyo Medical University, Tokyo, Japan.
FAU - Hayashi, Hideo
AU  - Hayashi H
AD  - Alexion Pharma GK, Tokyo, Japan.
FAU - Shimono, Akihiko
AU  - Shimono A
AD  - Alexion Pharma GK, Tokyo, Japan.
FAU - Kitajima, Akiyo
AU  - Kitajima A
AD  - Alexion Pharma GK, Tokyo, Japan.
FAU - Obara, Naoshi
AU  - Obara N
AD  - Department of Hematology, University of Tsukuba, Tsukuba, Japan.
FAU - Nishimura, Jun-Ichi
AU  - Nishimura JI
AD  - Department of Hematology and Oncology, Osaka University Graduate School of 
      Medicine, Suita, Japan.
LA  - eng
PT  - Journal Article
DEP - 20230721
PL  - Japan
TA  - Int J Hematol
JT  - International journal of hematology
JID - 9111627
RN  - C3VX249T6L (ravulizumab)
RN  - 0 (Complement Inactivating Agents)
RN  - EC 1.1.1.27 (L-Lactate Dehydrogenase)
SB  - IM
MH  - Humans
MH  - *Hemoglobinuria, Paroxysmal/drug therapy
MH  - Japan/epidemiology
MH  - Complement Inactivating Agents/adverse effects
MH  - L-Lactate Dehydrogenase
MH  - Product Surveillance, Postmarketing
MH  - Hemolysis
OTO - NOTNLM
OT  - Paroxysmal nocturnal hemoglobinuria
OT  - Post-marketing surveillance
OT  - Ravulizumab
EDAT- 2023/07/21 13:15
MHDA- 2023/08/11 06:43
CRDT- 2023/07/21 11:12
PHST- 2023/03/09 00:00 [received]
PHST- 2023/06/12 00:00 [accepted]
PHST- 2023/06/06 00:00 [revised]
PHST- 2023/08/11 06:43 [medline]
PHST- 2023/07/21 13:15 [pubmed]
PHST- 2023/07/21 11:12 [entrez]
AID - 10.1007/s12185-023-03625-8 [pii]
AID - 10.1007/s12185-023-03625-8 [doi]
PST - ppublish
SO  - Int J Hematol. 2023 Sep;118(3):311-322. doi: 10.1007/s12185-023-03625-8. Epub 
      2023 Jul 21.