PMID- 38481741
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20240315
IS  - 1179-2736 (Print)
IS  - 1179-2736 (Electronic)
IS  - 1179-2736 (Linking)
VI  - 15
DP  - 2024
TI  - A Post-Authorization Safety Surveillance Study to Report Clinical Experience with 
      Purified Factor IX Concentrate in Pediatric Patients with Hemophilia B.
PG  - 113-122
LID - 10.2147/JBM.S425617 [doi]
AB  - INTRODUCTION: Purified factor IX (FIX) concentrate (IMMUNINE((R)), Takeda 
      Manufacturing Austria AG, Vienna, Austria) is indicated for the treatment and 
      prophylaxis of bleeding episodes in patients with congenital hemophilia B. Data 
      on the use of purified FIX concentrate in patients </=6 years old with congenital 
      hemophilia B are limited. AIM: Document real-world clinical experience with 
      purified FIX concentrate in routine practice for pediatric patients with 
      hemophilia B. METHODS: This prospective post-authorization safety surveillance 
      study enrolled patients </=6 years old with moderate or severe hemophilia B 
      (baseline FIX </=5%) who were prescribed purified FIX concentrate, as determined by 
      the treating physician. The planned observation period for each patient was 
      either 12 months or >/=50 exposure days, whichever occurred first. The primary 
      endpoints were the occurrence of treatment-related adverse events (AEs) and 
      serious AEs (SAEs), and inhibitor development. RESULTS: Thirteen male patients 
      (mean +/- standard deviation age, 3.80 +/- 1.76 years) enrolled and received >/=1 
      treatment with purified FIX concentrate. Thirty-two AEs were reported in 6 
      patients; 4 were SAEs. No AEs were considered related to purified FIX 
      concentrate. No patients developed inhibitory antibodies. Inhibitor testing was 
      not conducted in 2 patients. Eighteen bleeding episodes were treated with 
      purified FIX concentrate in 6 patients. Hemostatic efficacy was rated as either 
      "excellent" or "good" in all patients with an available rating. CONCLUSION: No 
      treatment-related AEs were reported, and purified FIX concentrate was shown to be 
      effective in treating and preventing bleeding episodes in pediatric patients </=6 
      years old with hemophilia B.
CI  - (c) 2024 Takeda Pharmaceuticals International AG.
FAU - Igrutinovic, Zoran
AU  - Igrutinovic Z
AUID- ORCID: 0000-0002-9857-6475
AD  - Clinic of Pediatrics, Department of Hemato-Oncology, University Clinical Center 
      of Kragujevac, Kragujevac, Serbia.
AD  - Faculty of Medical Sciences, Department of Pediatrics, University of Kragujevac, 
      Kragujevac, Serbia.
FAU - Hooimeijer, Helene Louise
AU  - Hooimeijer HL
AD  - Pediatric Hematology, Beatrix Children's Hospital, University Medical Center 
      Groningen, Groningen, the Netherlands.
FAU - Kentouche, Karim
AU  - Kentouche K
AD  - Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Jena, Jena, 
      Deutschland.
FAU - Botha, Jaco
AU  - Botha J
AD  - Takeda Pharmaceuticals International AG, Zurich, Switzerland.
FAU - Turecek, Peter L
AU  - Turecek PL
AUID- ORCID: 0000-0002-6161-1556
AD  - Baxalta Innovations GmbH, Part of Takeda, Vienna, Austria.
FAU - Kokot-Kierepa, Marta
AU  - Kokot-Kierepa M
AD  - Takeda Pharmaceuticals International AG, Zurich, Switzerland.
FAU - Gazda, Hanna T
AU  - Gazda HT
AD  - Takeda Development Center Americas, Inc., Cambridge, MA, USA.
LA  - eng
PT  - Journal Article
DEP - 20240305
PL  - New Zealand
TA  - J Blood Med
JT  - Journal of blood medicine
JID - 101550884
PMC - PMC10933513
OTO - NOTNLM
OT  - factor IX
OT  - hemophilia B
OT  - pediatrics
OT  - post-marketing product surveillance
OT  - surgery
COIS- Zoran Igrutinovic reports being an investigator for this post-authorization 
      safety surveillance study, which was funded by Baxalta Innovations GmbH, Vienna, 
      Austria, a Takeda company. Helene Louise Hooimeijer has no interests that might 
      be perceived as posing a conflict or bias for this study. Karim Kentouche reports 
      being an investigator for this post-authorization safety surveillance study, 
      which was funded by Baxalta Innovations GmbH, Vienna, Austria, a Takeda company. 
      Jaco Botha and Marta Kokot-Kierepa are employees of Takeda Pharmaceuticals 
      International AG, and Takeda stockholders. Peter L. Turecek is an employee of 
      Baxalta Innovations GmbH, a Takeda company, and a Takeda stockholder. Hanna T. 
      Gazda is an employee of Takeda Development Center Americas, Inc., a Takeda 
      company, and a Takeda shareholder. The authors report no other conflicts of 
      interest in this work.
EDAT- 2024/03/14 06:47
MHDA- 2024/03/14 06:48
PMCR- 2024/03/05
CRDT- 2024/03/14 04:13
PHST- 2023/07/21 00:00 [received]
PHST- 2024/01/29 00:00 [accepted]
PHST- 2024/03/14 06:48 [medline]
PHST- 2024/03/14 06:47 [pubmed]
PHST- 2024/03/14 04:13 [entrez]
PHST- 2024/03/05 00:00 [pmc-release]
AID - 425617 [pii]
AID - 10.2147/JBM.S425617 [doi]
PST - epublish
SO  - J Blood Med. 2024 Mar 5;15:113-122. doi: 10.2147/JBM.S425617. eCollection 2024.