PMID- 33277961
OWN - NLM
STAT- MEDLINE
DCOM- 20210924
LR  - 20210924
IS  - 1365-2516 (Electronic)
IS  - 1351-8216 (Linking)
VI  - 27
IP  - 1
DP  - 2021 Jan
TI  - Safety and efficacy of nonacog alfa for the treatment of haemophilia B in 
      children younger than 6 years of age in a routine clinical care setting: the 
      EUREKIX registry study.
PG  - e60-e68
LID - 10.1111/hae.14215 [doi]
AB  - INTRODUCTION: European regulatory authorities request postmarketing safety and 
      efficacy data for factor IX (FIX) products. AIM: Collect additional clinical data 
      from routine nonacog alfa use in children aged <6 years with haemophilia B. 
      METHODS: The EUREKIX registry included retrospective and prospective data 
      collection phases. Safety was assessed via adverse drug reactions (ADRs)/adverse 
      events (AEs) and events of special interest (ESIs) as the primary objective; 
      efficacy was evaluated via annualised bleeding rates (ABRs). RESULTS: The 
      retrospective phase comprised 37 subjects. Of these, 25 had severe haemophilia B. 
      One subject experienced 2 ADRs; another experienced 4 ESIs of hypersensitivity. 
      Median ABR in subjects receiving a predominantly on-demand regimen (prophylaxis 
      <50% of time; n = 11) was 2.0; median ABR was 3.8 in those receiving 
      predominantly prophylactic treatment (prophylaxis >/=50% of time; n = 24). Joint 
      bleeding was infrequent (median ABR, 0.4; n = 35). The prospective phase included 
      26 subjects, with 17 continuing from the retrospective phase. A total of 20 
      subjects had severe haemophilia B. Three subjects experienced 7 treatment-related 
      AEs; 3 experienced 4 ESIs. Median ABR was 4.5 and 1.1 in subjects who received 
      predominantly on-demand (n = 5) or prophylactic treatment (n = 19), respectively; 
      the overall median ABR for joint bleeding events was 0.0. CONCLUSIONS: Overall, 
      nonacog alfa treatment effectively controlled bleeding events, with no new safety 
      signals identified. These data support the safety and efficacy of nonacog alfa in 
      routine clinical settings in children aged <6 years.
CI  - (c) 2020 John Wiley & Sons Ltd.
FAU - Liesner, Ri
AU  - Liesner R
AUID- ORCID: 0000-0001-5659-554X
AD  - Great Ormond Street Hospital for Children, London, UK.
FAU - Andersson, Nadine G
AU  - Andersson NG
AUID- ORCID: 0000-0001-6058-8350
AD  - Department for Thrombosis and Haemostasis, Skane University Hospital, Malmo, 
      Sweden.
FAU - Frisk, Tony
AU  - Frisk T
AD  - Karolinska University Hospital, Stockholm, Sweden.
FAU - Santagostino, Elena
AU  - Santagostino E
AUID- ORCID: 0000-0001-9639-6422
AD  - Foundation IRCCS Ca Granda, Maggiore Hospital Policlinico, Angelo Bianchi Bonomi 
      Haemophilia and Thrombosis Centre, Milan, Italy.
FAU - Schulz, Martin
AU  - Schulz M
AD  - Pfizer Pharma GmbH, Berlin, Germany.
FAU - Young, Lisa
AU  - Young L
AD  - Pfizer Innovative Health, Walton Oaks, UK.
FAU - Giordano, Paola
AU  - Giordano P
AD  - Department of Biomedical Science and Human Oncology, University of Bari "Aldo 
      Moro", Bari, Italy.
FAU - Tagliaferri, Annarita
AU  - Tagliaferri A
AUID- ORCID: 0000-0003-3529-6334
AD  - Regional Reference Center for Inherited Bleeding Disorders, University Hospital 
      of Parma, Parma, Italy.
LA  - eng
GR  - Pfizer/
PT  - Journal Article
DEP - 20201205
PL  - England
TA  - Haemophilia
JT  - Haemophilia : the official journal of the World Federation of Hemophilia
JID - 9442916
RN  - 9001-28-9 (Factor IX)
SB  - IM
MH  - Child
MH  - Factor IX/therapeutic use
MH  - *Hemophilia A
MH  - *Hemophilia B/drug therapy
MH  - Hemorrhage/etiology/prevention & control
MH  - Humans
MH  - Registries
MH  - Retrospective Studies
OTO - NOTNLM
OT  - Europe
OT  - factor IX
OT  - haematology
OT  - haemorrhage
OT  - observational study
OT  - paediatrics
EDAT- 2020/12/06 06:00
MHDA- 2021/09/25 06:00
CRDT- 2020/12/05 08:33
PHST- 2020/02/26 00:00 [received]
PHST- 2020/10/26 00:00 [revised]
PHST- 2020/11/09 00:00 [accepted]
PHST- 2020/12/06 06:00 [pubmed]
PHST- 2021/09/25 06:00 [medline]
PHST- 2020/12/05 08:33 [entrez]
AID - 10.1111/hae.14215 [doi]
PST - ppublish
SO  - Haemophilia. 2021 Jan;27(1):e60-e68. doi: 10.1111/hae.14215. Epub 2020 Dec 5.