PMID- 28440004
OWN - NLM
STAT- MEDLINE
DCOM- 20180319
LR  - 20180319
IS  - 1365-2516 (Electronic)
IS  - 1351-8216 (Linking)
VI  - 23
IP  - 4
DP  - 2017 Jul
TI  - Safety and effectiveness of room temperature stable recombinant factor VIIa in 
      patients with haemophilia A or B and inhibitors: Results of a multinational, 
      prospective, observational study.
PG  - 575-582
LID - 10.1111/hae.13227 [doi]
AB  - INTRODUCTION: A room temperature stable formulation of recombinant activated 
      factor VII (NovoSeven((R)) ), allowing convenient storage and therefore improved 
      treatment access, has been developed. Bioequivalence to the previous NovoSeven((R)) 
      was demonstrated in healthy humans, leading to European approval (2008). Although 
      no confirmed cases of neutralising antibodies to rFVIIa in patients with 
      haemophilia A or B have been observed with the original formulation, changes in 
      formulation or storage condition may alter immunogenicity. AIM: SMART-7 was 
      designed to investigate the safety of NovoSeven((R)) in a real-world setting in 
      patients with haemophilia A or B with inhibitors. METHODS: Study medication was 
      not provided by the sponsor, and treatment was at the discretion of the treating 
      physician, in accordance with the local label. Patient baseline information was 
      collected at enrolment. Information on safety, drug exposure and bleeding 
      episodes was collected and FVII antibody screening was encouraged at baseline and 
      performed at the investigator's discretion. RESULTS: Fifty-one patients were 
      enrolled and 31 completed the study. Forty-one adverse events (AEs) were reported 
      in 23 patients; 25 AEs in 14 patients were serious. No thromboembolic events were 
      observed. Although four cases of reduced therapeutic response were reported, FVII 
      antibody screening was negative. Forty-eight patients experienced 618 bleeding 
      episodes and 93.4% of 609 evaluated bleeds were stopped by treatment. Of the 538 
      bleeding episodes treated with NovoSeven((R)) monotherapy, 94.2% stopped by end of 
      treatment. CONCLUSION: Data collected during the SMART-7 study revealed no 
      treatment-related safety issues and no FVII-binding antibodies for patients 
      treated with NovoSeven((R)) under real-world conditions.
CI  - (c) 2017 John Wiley & Sons Ltd.
FAU - Kavakli, K
AU  - Kavakli K
AUID- ORCID: 0000-0002-4910-2142
AD  - Department of Hematology, Ege University Children's Hospital, Izmir, Turkey.
FAU - Demartis, F
AU  - Demartis F
AD  - Careggi Agenzia per l'Emofilia, A.O.U.C. Azienda Ospedaliero-Universitaria, 
      Firenze, Italy.
FAU - Karimi, M
AU  - Karimi M
AD  - Hematology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran.
FAU - Eshghi, P
AU  - Eshghi P
AD  - Pediatric Congenital Hematologic Disorders Research Center, Shahid Beheshti 
      University of Medical Sciences, Tehran, Iran.
FAU - Neme, D
AU  - Neme D
AD  - Fundacion de la Hemofilia, Buenos Aires, Argentina.
FAU - Chambost, H
AU  - Chambost H
AD  - Service d'Hematologie Oncologie Pediatrique, La Timone, APHM, Marseille, France.
AD  - Inserm, UMR 1062, Faculte de Medecine, Aix-Marseille Universite, Marseille, 
      France.
FAU - Sommer, L
AU  - Sommer L
AD  - Biostatistics Biopharm, Global Development, Novo Nordisk A/S, Soborg, Denmark.
FAU - Zak, M
AU  - Zak M
AD  - Medical and Science Haemophilia, Global Development, Novo Nordisk A/S, Soborg, 
      Denmark.
FAU - Benson, G
AU  - Benson G
AD  - Northern Ireland Haemophilia Comprehensive Care Centre, Belfast City Hospital, 
      Belfast, UK.
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
PT  - Observational Study
DEP - 20170424
PL  - England
TA  - Haemophilia
JT  - Haemophilia : the official journal of the World Federation of Hemophilia
JID - 9442916
RN  - 0 (Recombinant Proteins)
RN  - AC71R787OV (recombinant FVIIa)
RN  - EC 3.4.21.21 (Factor VIIa)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Child
MH  - Child, Preschool
MH  - Drug Stability
MH  - Factor VIIa/*adverse effects/pharmacology/*therapeutic use
MH  - Female
MH  - Hemophilia A/complications/*drug therapy
MH  - Hemophilia B/complications/*drug therapy
MH  - Hemorrhage/complications
MH  - Humans
MH  - Infant
MH  - Internationality
MH  - Male
MH  - Middle Aged
MH  - Recombinant Proteins/adverse effects/pharmacology/therapeutic use
MH  - *Safety
MH  - *Temperature
MH  - Treatment Outcome
MH  - Young Adult
OTO - NOTNLM
OT  - antibodies
OT  - haemophilia A
OT  - haemophilia B
OT  - real world
OT  - recombinant FVIIa
EDAT- 2017/04/26 06:00
MHDA- 2018/03/20 06:00
CRDT- 2017/04/26 06:00
PHST- 2017/02/09 00:00 [accepted]
PHST- 2017/04/26 06:00 [pubmed]
PHST- 2018/03/20 06:00 [medline]
PHST- 2017/04/26 06:00 [entrez]
AID - 10.1111/hae.13227 [doi]
PST - ppublish
SO  - Haemophilia. 2017 Jul;23(4):575-582. doi: 10.1111/hae.13227. Epub 2017 Apr 24.