PMID- 35243202
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20230328
IS  - 2475-0379 (Electronic)
IS  - 2475-0379 (Linking)
VI  - 6
IP  - 2
DP  - 2022 Feb
TI  - Safety and effectiveness of recombinant factor XIII-A(2) in congenital factor 
      XIII deficiency: Real-world evidence.
PG  - e12628
LID - 10.1002/rth2.12628 [doi]
LID - e12628
AB  - BACKGROUND: Regular factor XIII (FXIII) prophylaxis is standard treatment for 
      congenital FXIII A-subunit deficiency (FXIII-A CD). Recombinant factor XIII-A(2) 
      (rFXIII-A(2)) was extensively evaluated in the mentor trials. OBJECTIVE: To 
      assess real-world safety and treatment effectiveness of rFXIII-A(2) prophylaxis 
      from the mentor 6 trial. PATIENTS/METHODS: mentor 6 was a noninterventional, 
      postauthorization safety study investigating rFXIII-A(2) prophylaxis in FXIII-A 
      CD. rFXIII-A(2) treatment was observed for 2 to 6 years per patient. The primary 
      end point was documentation of adverse drug reactions (including anti-FXIII 
      antibody development). Secondary end points were serious adverse events (SAEs), 
      medical events of special interest (MESIs), and annualized bleeding rate (ABR). 
      RESULTS: Among 30 patients (mean age, 25.5 years), there were 44 adverse events 
      (AEs) (30 mild, 13 moderate, 1 severe). Eleven AEs were possibly/probably related 
      to rFXIII-A(2). Of four MESIs, two were unlikely related to rFXIII-A(2) 
      (accidental overdose, deep vein thrombosis), and two were possibly/probably 
      related (nonneutralizing anti-FXIII antibody, decreased therapeutic response). 
      All 10 SAEs were unlikely related to rFXIII-A(2). Over a follow-up of 75.4 
      patient-years, there were six treatment-requiring bleeds (all trauma-related with 
      no spontaneous bleeds), giving a treatment-requiring ABR of 0.066; five bleeds 
      were treated successfully with rFXIII-A(2). Eight of nine minor surgeries 
      performed during rFXIII-A(2) prophylaxis reported successful hemostatic outcomes 
      (one missing evaluation). CONCLUSIONS: These data confirm that rFXIII-A(2) 
      prophylaxis is well tolerated as long-term care. There were no spontaneous 
      bleeds, ABR was low, and rFXIII-A(2) successfully treated bleeds in patients 
      receiving rFXIII-A(2) prophylaxis.
CI  - (c) 2022 The Authors. Research and Practice in Thrombosis and Haemostasis published 
      by Wiley Periodicals LLC on behalf of International Society on Thrombosis and 
      Haemostasis (ISTH).
FAU - Poulsen, Lone Hvitfeldt
AU  - Poulsen LH
AD  - The Haemophilia Center Department of Hematology Aarhus University Hospital Aarhus 
      Denmark.
FAU - Kerlin, Bryce A
AU  - Kerlin BA
AUID- ORCID: 0000-0002-1756-8271
AD  - The Ohio State University College of Medicine Nationwide Children's Hospital 
      Columbus Ohio USA.
FAU - Castaman, Giancarlo
AU  - Castaman G
AUID- ORCID: 0000-0003-4973-1317
AD  - Center for Bleeding Disorders and Coagulation Department of Oncology Careggi 
      University Hospital Firenze Italy.
FAU - Molinari, Angelo Claudio
AU  - Molinari AC
AUID- ORCID: 0000-0002-8078-8402
AD  - Regional Reference Center for Hemorrhagic Diseases Giannina Gaslini Children's 
      Hospital Genoa Italy.
FAU - Menegatti, Marzia
AU  - Menegatti M
AUID- ORCID: 0000-0002-8527-7556
AD  - IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico Angelo Bianchi Bonomi 
      Hemophilia and Thrombosis Center Milan Italy.
FAU - Nugent, Diane
AU  - Nugent D
AD  - Children's Hospital of Orange County University of California at Irvine, and 
      Center for Inherited Blood Disorders Orange California USA.
FAU - Dey, Sohan
AU  - Dey S
AD  - Novo Nordisk Service Centre India Private Ltd Bangalore India.
FAU - Garly, May-Lill
AU  - Garly ML
AD  - Novo Nordisk A/S Soborg Denmark.
FAU - Carcao, Manuel
AU  - Carcao M
AUID- ORCID: 0000-0001-5350-1763
AD  - Division of Haematology/Oncology Department of Paediatrics and Child Health 
      Evaluative Sciences Research Institute, Hospital for Sick Children University of 
      Toronto Toronto Ontario Canada.
LA  - eng
PT  - Journal Article
DEP - 20220227
PL  - United States
TA  - Res Pract Thromb Haemost
JT  - Research and practice in thrombosis and haemostasis
JID - 101703775
PMC - PMC8882239
OTO - NOTNLM
OT  - factor XIII
OT  - long-term care
OT  - recombinant factor XIII-A2
OT  - safety
OT  - treatment effectiveness
EDAT- 2022/03/05 06:00
MHDA- 2022/03/05 06:01
PMCR- 2022/02/27
CRDT- 2022/03/04 05:43
PHST- 2021/05/25 00:00 [received]
PHST- 2021/10/06 00:00 [revised]
PHST- 2021/10/31 00:00 [accepted]
PHST- 2022/03/04 05:43 [entrez]
PHST- 2022/03/05 06:00 [pubmed]
PHST- 2022/03/05 06:01 [medline]
PHST- 2022/02/27 00:00 [pmc-release]
AID - S2475-0379(22)01153-0 [pii]
AID - RTH212628 [pii]
AID - 10.1002/rth2.12628 [doi]
PST - epublish
SO  - Res Pract Thromb Haemost. 2022 Feb 27;6(2):e12628. doi: 10.1002/rth2.12628. 
      eCollection 2022 Feb.