PMID- 28755067
OWN - NLM
STAT- MEDLINE
DCOM- 20180710
LR  - 20181113
IS  - 1573-2592 (Electronic)
IS  - 0271-9142 (Print)
IS  - 0271-9142 (Linking)
VI  - 37
IP  - 6
DP  - 2017 Aug
TI  - Efficacy and Safety of Human Intravenous Immunoglobulin 10% (Panzyga(R)) in 
      Patients with Primary Immunodeficiency Diseases: a Two-Stage, Multicenter, 
      Prospective, Open-Label Study.
PG  - 603-612
LID - 10.1007/s10875-017-0424-4 [doi]
AB  - PURPOSE: To assess the efficacy and safety of panzyga(R) (intravenous 
      immunoglobulin 10%) in preventing serious bacterial infections (SBIs) in patients 
      with primary immunodeficiency diseases (PIDs), a prospective, open-label, 
      multicenter, phase 3 study and an open-label extension study were undertaken. 
      METHODS: Initially, the study drug (infusion rate </=0.08 mL/kg/min) was 
      administered at intervals of 3 or 4 weeks for 12 months, followed by 3 months of 
      panzyga(R) at infusion rates increasing from 0.08 to 0.14 mL/kg/min. The primary 
      endpoint in the main study was the rate of SBIs per patient-year on treatment. 
      Secondary outcomes included non-serious infections, work/school absence, episodes 
      of fever, quality of life, and adverse events (AEs). RESULTS: The main study 
      enrolled 51 patients (35% female, mean age 26.8 years), with 21 participating in 
      the extension study. The rate of SBIs per patient-year was 0.08 in the total 
      population; there were four SBIs in the 4-weekly treatment group (2/30 patients) 
      and none in the 3-weekly group (n = 21). Compared with 4-weekly treatment, 
      3-weekly treatment was associated with a higher rate of upper respiratory tract 
      infections (RTIs), ear infections, and work/school absences, but a lower rate of 
      lower RTIs and fever. Treatment was generally well tolerated; no AE led to 
      treatment withdrawal or death. CONCLUSIONS: Overall, the use of panzyga(R) in 
      patients with antibody-deficient PID was associated with a low rate of AEs and 
      was effective in preventing SBIs, exceeding US FDA and European Medicines Agency 
      recommendations for efficacy.
FAU - Borte, Michael
AU  - Borte M
AD  - Klinik fur Kinder- und Jugendmedizin, Klinikum St. Georg gGmbH, Leipzig, Germany. 
      Michael.Borte@sanktgeorg.de.
AD  - Immunodeficiency Centre Leipzig (IDCL), Hospital St. Georg gGmbH Leipzig, 
      Delitzscher Strasse 141, 04129, Leipzig, Germany. Michael.Borte@sanktgeorg.de.
FAU - Melamed, Isaac R
AU  - Melamed IR
AD  - IMMUNOe Research Center Centennial, Centennial, CO, USA.
FAU - Pulka, Grazyna
AU  - Pulka G
AD  - Klinika Alergologii Collegium Medicum, Uniwersytetu Jagiellonskiego, Krakow, 
      Poland.
FAU - Pyringer, Barbara
AU  - Pyringer B
AD  - Clinical Research and Development Department, Octapharma Pharmazeutika 
      Produktionsges.m.b.H, Vienna, Austria.
FAU - Knutsen, Alan P
AU  - Knutsen AP
AD  - Saint Louis University, St. Louis, MO, USA.
FAU - Ochs, Hans D
AU  - Ochs HD
AD  - Department of Pediatrics, University of Washington and Seattle Children's 
      Research Institute, Seattle, WA, USA.
FAU - Kobayashi, Roger H
AU  - Kobayashi RH
AD  - UCLA School of Medicine, Los Angeles, CA, USA.
FAU - Kobayashi, Ai Lan
AU  - Kobayashi AL
AD  - , Papillion, NE, USA.
FAU - Gupta, Sudhir
AU  - Gupta S
AD  - University of California, Irvine, CA, USA.
FAU - Strach, Magdalena
AU  - Strach M
AD  - Jagiellonian University Medical College, Krakow, Poland.
FAU - Smits, William
AU  - Smits W
AD  - The Allergy and Asthma Center, Fort Wayne, IN, USA.
FAU - Pituch-Noworolska, Anna
AU  - Pituch-Noworolska A
AD  - University Children Hospital, Krakow, Poland.
FAU - Moy, James N
AU  - Moy JN
AD  - Division of Pediatric Allergy/Immunology, Stroger Hospital of Cook County, 
      Chicago, IL, USA.
LA  - eng
PT  - Clinical Trial, Phase III
PT  - Journal Article
PT  - Multicenter Study
DEP - 20170729
PL  - Netherlands
TA  - J Clin Immunol
JT  - Journal of clinical immunology
JID - 8102137
RN  - 0 (Immunoglobulin G)
RN  - 0 (Immunoglobulins)
RN  - 0 (Immunologic Factors)
RN  - 0 (Panzyga)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Bacterial Infections/epidemiology/immunology/*therapy
MH  - Child
MH  - Child, Preschool
MH  - Female
MH  - Humans
MH  - Immunoglobulin G/*therapeutic use
MH  - Immunoglobulins/*deficiency
MH  - Immunologic Deficiency Syndromes/epidemiology/immunology/*therapy
MH  - Immunologic Factors/*therapeutic use
MH  - Immunotherapy/*methods
MH  - Male
MH  - Middle Aged
MH  - Prospective Studies
MH  - Quality of Life
MH  - Treatment Outcome
MH  - Young Adult
PMC - PMC5554470
OTO - NOTNLM
OT  - Primary immunodeficiency diseases
OT  - intravenous immunoglobulin
OT  - panzyga(R)
OT  - serious bacterial infections
COIS- M Borte's institution has received research grant support from CSL Behring, 
      Octapharma, and Baxalta, and he has participated in advisory boards for CSL 
      Behring and Octapharma. IR Melamed has received research grant support from CSL 
      Behring, Octapharma, Baxalta, and Bio Products Laboratories. B. Pyringer is an 
      employee of Octapharma, Vienna, Austria. AP Knutsen has acted as a consultant for 
      Baxalta, CSL, and Octapharma. HD Ochs has acted as consultant for Baxalta, CSL 
      Behring, and Octapharma. RH Kobayashi has acted as a consultant for Baxalta, CSL, 
      ADMA, and Octapharma. AL Kobayashi received grant support from Baxalta and 
      Octapharma. JN Moy has received fees as a consultant for Grifols, Prometic, 
      Octapharma, MacroCure, and Baxalta. G Pulka, S Gupta, M Strach, W Smits, and A 
      Pituch-Noworolska have no conflicts of interest to declare. Open Access for this 
      Article was funded by Octapharma.
EDAT- 2017/07/30 06:00
MHDA- 2018/07/11 06:00
PMCR- 2017/07/29
CRDT- 2017/07/30 06:00
PHST- 2017/03/14 00:00 [received]
PHST- 2017/07/18 00:00 [accepted]
PHST- 2017/07/30 06:00 [pubmed]
PHST- 2018/07/11 06:00 [medline]
PHST- 2017/07/30 06:00 [entrez]
PHST- 2017/07/29 00:00 [pmc-release]
AID - 10.1007/s10875-017-0424-4 [pii]
AID - 424 [pii]
AID - 10.1007/s10875-017-0424-4 [doi]
PST - ppublish
SO  - J Clin Immunol. 2017 Aug;37(6):603-612. doi: 10.1007/s10875-017-0424-4. Epub 2017 
      Jul 29.