Flebogamma(®) 5 % DIF Intravenous Immunoglobulin for Replacement Therapy in Children with Primary Immunodeficiency Diseases.
The previous studies with Flebogamma(®) 5 % DIF intravenous immunoglobulin (IVIG) contained insufficient numbers of pediatric subjects to fully warrant a pediatric indication by the FDA. The objective of this study was to evaluate the efficacy, safety, and pharmacokinetics of Flebogamma® 5 % DIF for replacement therapy in children (age 2-16) with primary immunodeficiency diseases (PIDD).



IVIG was administered at eight clinical sites to 24 subjects with well-defined PIDD at a dose of 300-800 mg/kg every 21-28 days for 12 months. The pharmacokinetics endpoint in this study was the dose-adjusted increment of the serum IgG trough levels.



The calculated serious bacterial infection rate was 0.05/subject/year. The incidence of adverse events considered potentially related to IVIG during or within 72 h after completing an infusion was within the FDA guidance threshold of <40 % at each time point. Dose-adjusted incremental IgG levels remained approximately equal to or slightly greater than pre-study IgG levels (between 800 and 1000 mg/dL throughout) when the subjects were treated with IVIG therapy other than Flebogamma(®) DIF 5 % indicating no evidence of a different pharmacokinetic profile in this pediatric population if compared to those profiles in previous Flebogamma studies in predominately adult populations.



Flebogamma(®) 5 % DIF is efficacious and safe, has adequate pharmacokinetic properties, is well-tolerated, and maintains the profile of Flebogamma(®) 5 % for the treatment of children with primary humoral immunodeficiency diseases.
The Children's Hospital of Buffalo, Buffalo, NY, USA. markbal.aird@gmail.com.|Division of Allergy and Immunology, Department of Pediatrics, University of South Florida, 601 4th Street South, Saint Petersburg, FL, 3370, USA. markbal.aird@gmail.com.
Mark
M
Ballow
Grifols Biologicals Inc., Los Angeles, CA, USA.
Paul J
PJ
Pinciaro
Penn State University Hershey Medical Center, Hershey, PA, USA.
Timothy
T
Craig
Jeffrey Modell Diagnostic Center for Primary Immunodeficiencies, University of Miami, Miami, FL, USA.
Gary
G
Kleiner
Allergy and Immunology, Rush University Medical Center, Chicago, IL, USA.
James
J
Moy
Seattle Children's Research Institute and University of Washington, Seattle, WA, USA.
Hans D
HD
Ochs
Division of Allergy and Immunology, Department of Pediatrics, University of South Florida, 601 4th Street South, Saint Petersburg, FL, 3370, USA.
John
J
Sleasman
The Allergy and Asthma Center, Fort Wayne, IN, USA.
William
W
Smits
D000293:Adolescent; D001424:Bacterial Infections / Q000209:etiology; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics; D007153:Immunologic Deficiency Syndromes / Q000175:diagnosis / Q000188:drug therapy*; D008297:Male; D013997:Time Factors; D016896:Treatment Outcome
D016756:Immunoglobulins, Intravenous
Flebogamma® 5 % DIF; IVIG; Intravenous immune globulin; clinical trial; immunologic deficiency syndromes