PMID- 31730687
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220411
IS  - 2287-979X (Print)
IS  - 2288-0011 (Electronic)
IS  - 2287-979X (Linking)
VI  - 54
IP  - 3
DP  - 2019 Sep
TI  - Subgroup analysis of a phase 2/3 study of rurioctocog alfa pegol in patients with 
      severe hemophilia A: efficacy and safety in previously treated Korean patients.
PG  - 198-203
LID - 10.5045/br.2019.54.3.198 [doi]
AB  - BACKGROUND: The efficacy and safety of extended half-life, full-length, pegylated 
      recombinant factor VIII rurioctocog alfa pegol [BAX 855, ADYNOVATE (USA)/ADYNOVI 
      (Europe); Baxalta US Inc., a Takeda company, Lexington, MA, USA] was investigated 
      in previously treated Korean patients with severe hemophilia A (HA). METHODS: A 
      post hoc data analysis from the international, multicenter, phase 2/3 PROLONG-ATE 
      study of rurioctocog alfa pegol in patients with severe HA (NCT01736475) 
      determined annualized bleeding rates (ABRs) and rates of adverse events (AEs) in 
      Korean patients treated in this study. RESULTS: All 10 enrolled Korean patients 
      receiving rurioctocog alfa pegol (9 prophylaxis, 1 on-demand) completed the study 
      [median (range) age, 28.0 (12-50) yr; weight, 64.8 (45-90) kg; 8 patients had >/=1 
      target joint at screening]. Median (range) ABR was 1.9 (0.0-14.5) for patients on 
      prophylaxis and 62.2 for the patient receiving on-demand treatment. The 
      hemostatic efficacy of rurioctocog alfa pegol was rated "excellent" or "good" and 
      only single infusions were required per bleeding episode. ABRs improved in most 
      patients compared with prestudy values. No dose adjustments were required for 
      prophylaxis, and the dosing frequency was reduced in 8 patients, compared with 
      their previous prophylaxis regimen. No serious AEs were reported; all 9 
      nonserious AEs (in 3 patients) were mild in severity and unrelated to the study 
      treatment. CONCLUSION: This post hoc analysis of a small group of Korean patients 
      with severe HA indicated that rurioctocog alfa pegol was effective, and no 
      serious AEs were observed. For most patients, the dosing frequency was also 
      reduced compared with their previous regimen.
CI  - (c) 2019 Korean Society of Hematology.
FAU - You, Chur Woo
AU  - You CW
AD  - Eulji University Hospital, Daejeon, Korea.
FAU - Baek, Hee Jo
AU  - Baek HJ
AD  - Chonnam National University Hwasun Hospital, Chonnam National University Medical 
      School, Gwangju, Korea.
FAU - Park, Sang Kyu
AU  - Park SK
AD  - Ulsan University Hospital, Ulsan, Korea.
FAU - Park, Young Shil
AU  - Park YS
AD  - Kyung Hee University Hospital at Gangdong, Seoul, Korea.
FAU - Shin, Ho-Jin
AU  - Shin HJ
AD  - Pusan National University Hospital, Busan, Korea.
FAU - Engl, Werner
AU  - Engl W
AD  - Baxalta Innovations GmbH, a Takeda Company, Vienna, Austria.
FAU - Tangada, Srilatha
AU  - Tangada S
AD  - Baxalta US Inc., a Takeda Company, Cambridge, MA, USA.
LA  - eng
PT  - Journal Article
DEP - 20190925
PL  - Switzerland
TA  - Blood Res
JT  - Blood research
JID - 101605247
PMC - PMC6779944
OTO - NOTNLM
OT  - *BAX 855
OT  - *Factor VIII
OT  - *Hemophilia A
OT  - *Post hoc analysis
OT  - *Rurioctocog alfa pegol
COIS- Authors' Disclosures of Potential Conflicts of Interest: CWY, HJB, SKP, YSP, and 
      H-JS have no conflicts of interest to declare. WE is an employee of Baxalta 
      Innovations GmbH, a member of the Takeda group of companies. ST is an employee of 
      Baxalta US Inc., a member of the Takeda group of companies, and a Takeda stock 
      owner.
EDAT- 2019/11/16 06:00
MHDA- 2019/11/16 06:01
PMCR- 2019/09/01
CRDT- 2019/11/16 06:00
PHST- 2019/03/22 00:00 [received]
PHST- 2019/05/23 00:00 [revised]
PHST- 2019/07/31 00:00 [accepted]
PHST- 2019/11/16 06:00 [entrez]
PHST- 2019/11/16 06:00 [pubmed]
PHST- 2019/11/16 06:01 [medline]
PHST- 2019/09/01 00:00 [pmc-release]
AID - 10.5045/br.2019.54.3.198 [doi]
PST - ppublish
SO  - Blood Res. 2019 Sep;54(3):198-203. doi: 10.5045/br.2019.54.3.198. Epub 2019 Sep 
      25.