PMID- 36151913
OWN - NLM
STAT- MEDLINE
DCOM- 20221227
LR  - 20230415
IS  - 1442-200X (Electronic)
IS  - 1328-8067 (Print)
IS  - 1328-8067 (Linking)
VI  - 64
IP  - 1
DP  - 2022 Jan
TI  - Real-world results with IgPro20 for hypo- or agammaglobulinemia in Japan.
PG  - e15362
LID - 10.1111/ped.15362 [doi]
LID - e15362
AB  - BACKGROUND: Subcutaneous immunoglobulin is one of the standard treatments for 
      hypogammaglobulinemia in primary immunodeficiencies (PID) worldwide. In Japan, 
      IgPro20 (Hizentra((R)) ; l-proline-stabilized 20% human subcutaneous 
      immunoglobulin) is approved for agammaglobulinemia or hypogammaglobulinemia due 
      to PID or secondary immunodeficiency (SID); however, its safety and effectiveness 
      has not previously been assessed in a real-world setting. METHODS: This 
      multicenter, open label post-marketing surveillance study was conducted between 
      January 2014 and March 2019. Patients who received IgPro20 due to PID or SID were 
      included after informed consent. Physicians completed a case report form for each 
      patient. Safety was determined from reported adverse events (AEs), adverse drug 
      reactions, and serious AEs (SAEs); effectiveness was assessed by infection rates 
      after the first IgPro20 dose. RESULTS: Of 85 patients receiving IgPro20 in the 
      safety analysis, 39 developed AEs (45.9%; PID n = 28, SID n = 11). At least one 
      adverse drug reaction was observed in 27 patients (31.8%; PID n = 21, SID n = 6), 
      and the most common were injection site reactions (n = 17, 20.0%). Four patients 
      (PID n = 3, SID n = 1) reported SAEs but two were unrelated to IgPro20 
      administration. The infection rate decreased from 0.54 per patient during the 
      6 months before IgPro20 to 0.39 per patient during IgPro20 treatment. Serious 
      bacterial infections occurred in six patients before IgPro20 (7.9%; PID n = 2; 
      SID n = 4) but in only one patient with SID during IgPro20 treatment (1.2%). 
      CONCLUSIONS: In Japan, IgPro20 was considered safe and effective among patients 
      with agammaglobulinemia or hypogammaglobulinemia due to PID or SID.
CI  - (c) 2022 The Authors. Pediatrics International published by John Wiley & Sons 
      Australia, Ltd on behalf of Japan Pediatric Society.
FAU - Imai, Kohsuke
AU  - Imai K
AUID- ORCID: 0000-0003-2132-8403
AD  - Department of Community Pediatrics, Perinatal and Maternal Medicine, Tokyo 
      Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Ishii, Tomonori
AU  - Ishii T
AD  - Clinical Research, Innovation and Education Center, Tohoku University Hospital, 
      Miyagi, Japan.
FAU - Nonoyama, Shigeaki
AU  - Nonoyama S
AD  - Department of Pediatrics, National Defense Medical College, Saitama, Japan.
FAU - Yasumi, Takahiro
AU  - Yasumi T
AD  - Department of Pediatrics, Kyoto University Graduate School of Medicine, Kyoto, 
      Japan.
FAU - Kanegane, Hirokazu
AU  - Kanegane H
AUID- ORCID: 0000-0002-8696-9378
AD  - Department of Child Health and Development, Tokyo Medical and Dental University 
      (TMDU), Tokyo, Japan.
FAU - Fukushima, Taku
AU  - Fukushima T
AD  - Medical Affairs, CSL Behring K.K., Tokyo, Japan.
FAU - Matsumaru, Masayuki
AU  - Matsumaru M
AD  - Medical Affairs, CSL Behring K.K., Tokyo, Japan.
FAU - Akasaki, Tetsushi
AU  - Akasaki T
AD  - Department of Pharmacovigilance, CSL Behring K.K., Tokyo, Japan.
FAU - Usui, Hideo
AU  - Usui H
AD  - Department of Pharmacovigilance, CSL Behring K.K., Tokyo, Japan.
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
PL  - Australia
TA  - Pediatr Int
JT  - Pediatrics international : official journal of the Japan Pediatric Society
JID - 100886002
RN  - 0 (Hizentra)
RN  - 0 (Immunoglobulins, Intravenous)
SB  - IM
MH  - Humans
MH  - *Agammaglobulinemia/drug therapy
MH  - Immunoglobulins, Intravenous/therapeutic use
MH  - *Immunologic Deficiency Syndromes/drug therapy
MH  - Injections, Subcutaneous
MH  - Japan
PMC - PMC10099597
OTO - NOTNLM
OT  - agammaglobulinemia
OT  - hypogammaglobulinemia
OT  - immunoglobulins
OT  - postmarketing product surveillance
OT  - subcutaneous injections
COIS- Kohsuke Imai received support from CSL Behring K.K. for development of the 
      present manuscript. Tomonori Ishii has participated on speaker bureaus for 
      GlaxoSmithKline, Chugai Pharmaceutical Co., Ltd., Janssen Pharmaceutical K.K., 
      Astellas Pharma Inc., and Asahi Kasei Pharma Corporation. Taku Fukushima, 
      Masayuki Matsumaru, Tetsushi Akasaki and Hideo Usui are employees of CSL Behring. 
      K.K. Shigeaki Nonoyama, Takahiro Yasumi and Hirokazu Kanegane declare no 
      conflicts of interest.
EDAT- 2022/09/25 06:00
MHDA- 2022/11/30 06:00
PMCR- 2023/04/13
CRDT- 2022/09/24 07:12
PHST- 2022/09/13 00:00 [revised]
PHST- 2022/06/20 00:00 [received]
PHST- 2022/09/14 00:00 [accepted]
PHST- 2022/09/25 06:00 [pubmed]
PHST- 2022/11/30 06:00 [medline]
PHST- 2022/09/24 07:12 [entrez]
PHST- 2023/04/13 00:00 [pmc-release]
AID - PED15362 [pii]
AID - 10.1111/ped.15362 [doi]
PST - ppublish
SO  - Pediatr Int. 2022 Jan;64(1):e15362. doi: 10.1111/ped.15362.