PMID- 38376107
OWN - NLM
STAT- MEDLINE
DCOM- 20240221
LR  - 20240221
IS  - 1607-8454 (Electronic)
IS  - 1024-5332 (Linking)
VI  - 29
IP  - 1
DP  - 2024 Dec
TI  - Real-world long-term safety and effectiveness of turoctocog alfa in the treatment 
      of haemophilia A in Japan: results from a multicentre, non-interventional, 
      post-marketing study.
PG  - 2316540
LID - 10.1080/16078454.2024.2316540 [doi]
AB  - OBJECTIVES: To assess the safety and effectiveness of turoctocog alfa in 
      previously treated patients (PTPs) and previously untreated patients (PUPs) with 
      haemophilia A in a real-world setting in Japan. METHODS: This multicentre, 
      non-interventional, post-marketing study recruited patients with haemophilia A 
      who initiated treatment with turoctocog alfa from 18 sites (08/2014-12/2018). The 
      primary endpoint was adverse events (AEs) during the 2-year study period. 
      RESULTS: The safety and effectiveness analysis set included 39 patients. In 
      total, 13 (33.3%) patients reported >/=1 AE; incidence rate was 60.4 events/100 
      patient-years of exposure (PYE). Treatment was withdrawn in two cases: pruritus 
      in a PTP and factor VIII inhibitor development in a PUP. Inhibitor development 
      occurred in 2.6% of all patients, with an incidence rate of 3.8 events/100 PYE. 
      The rate of inhibitor development was 0%, 25% and 20% in PTPs, PUPs and PUPs with 
      severe type, respectively. The haemostatic success rate was 91.4% for 383 
      bleeding episodes and 85.7% for 14 surgeries. The negative binomial annualised 
      bleeding rate for the prophylaxis regimen was 6.19 episodes/year (95% CI, 
      3.69-10.38). The mean (SD) total consumption of turoctocog alfa (n = 34; 
      excluding FVIII inhibitors) was 5,382.6 (7,180.1) IU/kg/year/patient; consumption 
      was 4,133.1 (1,452.4) IU/kg/year/patient for prophylaxis. DISCUSSION: The 
      effectiveness and safety profiles were comparable to those observed in other 
      turoctocog alfa trials; effectiveness analysis and consumption were not affected 
      by treatment regimens. CONCLUSION: Long-term use of turoctocog alfa therapy in 
      clinical practice posed no newly identified safety issues and was effective for 
      prophylaxis and treatment of bleeds in patients with haemophilia A in Japan.
FAU - Nagao, Azusa
AU  - Nagao A
AD  - Department of Blood Coagulation, Ogikubo Hospital, Tokyo, Japan.
FAU - Deguchi, Ayumi
AU  - Deguchi A
AD  - Medical Affairs, Novo Nordisk Pharma Ltd., Tokyo, Japan.
FAU - Nogami, Keiji
AU  - Nogami K
AD  - Department of Pediatrics, Nara Medical University, Kashihara, Japan.
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
DEP - 20240220
PL  - England
TA  - Hematology
JT  - Hematology (Amsterdam, Netherlands)
JID - 9708388
RN  - 0 (recombinant factor VIII N8)
RN  - 9001-27-8 (Factor VIII)
SB  - IM
MH  - Humans
MH  - *Factor VIII/adverse effects
MH  - *Hemophilia A/drug therapy
MH  - Japan
MH  - Hemorrhage/prevention & control/chemically induced
OTO - NOTNLM
OT  - Factor VIII
OT  - Haemophilia A
OT  - Japan
OT  - inhibitors
OT  - post-marketing product surveillance
OT  - prophylaxis
OT  - turoctocog alfa
EDAT- 2024/02/20 12:52
MHDA- 2024/02/21 11:22
CRDT- 2024/02/20 08:23
PHST- 2024/02/21 11:22 [medline]
PHST- 2024/02/20 12:52 [pubmed]
PHST- 2024/02/20 08:23 [entrez]
AID - 10.1080/16078454.2024.2316540 [doi]
PST - ppublish
SO  - Hematology. 2024 Dec;29(1):2316540. doi: 10.1080/16078454.2024.2316540. Epub 2024 
      Feb 20.