PMID- 33303707
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220419
IS  - 2287-979X (Print)
IS  - 2288-0011 (Electronic)
IS  - 2287-979X (Linking)
VI  - 55
IP  - 4
DP  - 2020 Dec 31
TI  - Safety and effectiveness of Rixubis in patients with hemophilia B: a real-world, 
      prospective, postmarketing surveillance study in South Korea.
PG  - 246-252
LID - 10.5045/br.2020.2020225 [doi]
AB  - BACKGROUND: Rixubis (recombinant factor IX, nonacog gamma) is indicated for the 
      control and prevention of bleeding episodes, perioperative management, and 
      routine prophylaxis in hemophilia B patients. This real-world, postmarketing 
      surveillance study aimed to evaluate the safety and effectiveness of Rixubis in 
      adult and pediatric hemophilia B patients in South Korea. METHODS: This 
      prospective, observational, multicenter study (clinicaltrials.gov identifier: 
      NCT029 22231) was conducted in hemophilia B patients between April 2015 and April 
      2019, who were observed for up to 6 months after the initiation of Rixubis 
      treatment. Safety was evaluated based on the number and severity of adverse 
      events (AEs) and serious AEs (SAEs). Hemostatic effectiveness was assessed by 
      physicians and patients by using a four-point scale and rated as excellent, good, 
      fair, or no response based on treatment type. RESULTS: In all, 58 patients were 
      enrolled from four centers by seven physicians during the study period. The 
      safety and effectiveness analysis sets included 57 and 54 patients, respectively. 
      Overall, 11 AEs were reported in eight patients (14.0%), of which three were SAEs 
      and occurred in three patients (5.3%). All 11 AEs were reported as unexpected and 
      mild in severity, with no anaphylactic reaction, and 10 AEs (90.9%) resolved. The 
      majority of AEs (10) were unrelated to Rixubis. Of the 142 hemostatic 
      effectiveness assessments, 123 (86.6%) were reported as good or excellent. 
      CONCLUSION: Rixubis demonstrated an acceptable safety and effectiveness profile 
      in the treatment of bleeding, perioperative management, and prophylaxis in 
      hemophilia B patients in a real-world setting in South Korea.
FAU - Choi, Eun Jin
AU  - Choi EJ
AD  - Department of Pediatrics, Daegu Catholic University Medical Center, Daegu, Korea.
FAU - Hwang, Tai Ju
AU  - Hwang TJ
AD  - Korea Hemophilia Foundation Clinic, Gwangju, Korea.
FAU - Choi, Yong Mook
AU  - Choi YM
AD  - Korea Hemophilia Foundation Clinic, Seoul, Korea.
FAU - Kim, Hugh Chul
AU  - Kim HC
AD  - HughChul Kim Internal Medicine Clinic, Seoul, Korea.
FAU - Yoo, Myung Chul
AU  - Yoo MC
AD  - Sunchun Chung Hospital, Seoul, Korea.
FAU - Song, Haylee
AU  - Song H
AD  - Hematology, Growth and Emerging Markets, Takeda Pharmaceutical International, 
      Singapore.
FAU - Badejo, Kayode
AU  - Badejo K
AD  - Global Patient safety Evaluation, Takeda Pharmaceutical Company Ltd, Cambridge, 
      MA, USA.
LA  - eng
SI  - ClinicalTrials.gov/NCT00000029
SI  - ClinicalTrials.gov/NCT00000029
PT  - Journal Article
PL  - Switzerland
TA  - Blood Res
JT  - Blood research
JID - 101605247
PMC - PMC7784123
OTO - NOTNLM
OT  - Bleeding
OT  - Effectiveness
OT  - Hemophilia B
OT  - Rixubis
OT  - Safety
OT  - South Korea
COIS- Authors' Disclosures of Potential Conflicts of Interest Haylee Song and Kayode 
      Badejo are full-time employees of Takeda. Haylee Song and Kayode Badejo own 
      Takeda stocks. The other authors have no potential conflicts of interest to 
      disclose.
EDAT- 2020/12/12 06:00
MHDA- 2020/12/12 06:01
PMCR- 2020/12/31
CRDT- 2020/12/11 05:51
PHST- 2020/08/31 00:00 [received]
PHST- 2020/10/30 00:00 [accepted]
PHST- 2020/12/12 06:00 [pubmed]
PHST- 2020/12/12 06:01 [medline]
PHST- 2020/12/11 05:51 [entrez]
PHST- 2020/12/31 00:00 [pmc-release]
AID - br.2020.2020225 [pii]
AID - BR-55-246 [pii]
AID - 10.5045/br.2020.2020225 [doi]
PST - ppublish
SO  - Blood Res. 2020 Dec 31;55(4):246-252. doi: 10.5045/br.2020.2020225.