J 2019

Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig (R)]) in the Treatment of Patients With Primary Immunodeficiencies

KOBAYASHI, Roger H., Sudhir GUPTA, Isaac MELAMED, Fernando J. MANDUJANO, Ai Lan KOBAYASHI et. al.

Basic information

Original name

Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig (R)]) in the Treatment of Patients With Primary Immunodeficiencies

Authors

KOBAYASHI, Roger H. (840 United States of America, guarantor), Sudhir GUPTA (840 United States of America), Isaac MELAMED (840 United States of America), Fernando J. MANDUJANO (840 United States of America), Ai Lan KOBAYASHI (840 United States of America), Bruce RITCHIE (124 Canada), Bob GENG (840 United States of America), Thomas Prescott ATKINSON (840 United States of America), Syed REHMAN (840 United States of America), Eva TURPEL-KANTOR (40 Austria) and Jiří LITZMAN (203 Czech Republic, belonging to the institution)

Edition

Frontiers in Immunology, LAUSANNE, FRONTIERS MEDIA SA, 2019, 1664-3224

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30102 Immunology

Country of publisher

Switzerland

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

Impact factor

Impact factor: 5.085

RIV identification code

RIV/00216224:14110/19:00109451

Organization unit

Faculty of Medicine

DOI

UT WoS

000457623800001

Keywords in English

primary immunodeficiencies; immunoglobulins; antibodies; SCIG; infections; infusion site reactions

Tags

Tags

International impact, Reviewed
Změněno: 15/4/2019 15:23, Soňa Böhmová

Abstract

V originále

Introduction: Subcutaneously administered immunoglobulin (SCIG) is increasingly used to treat patients with primary immunodeficiencies (PIDs). Octanorm (marketed as cutaquig (R) in USA and Canada) is a new 16.5% solution of human SCIG, manufactured by a process based on that of the intravenous preparation (IVIG) octagam (R). Objectives: To investigate the efficacy, safety and tolerability of octanorm in a prospective, open-label, single-arm phase 3 study involving adult and pediatric patients with PIDs (NCT01888484; clinicaltrials. gov/ct2/show/NCT01888484). Methods: Patients who were previously treated with IVIG received a total of 64 weekly SCIG infusions, including 12 weekly infusions during the wash-in/wash-out period, followed by 52 weekly infusions during the evaluation period. Results: A total of 61 patients aged 2-73 years received 3,497 infusions of octanorm. The mean dose per patient was 0.175 g/kg/infusion. The mean calculated dose conversion factor from the patients' previous IVIG dose for octanorm was 1.37. No serious bacterial infections developed during the study. The rate of other infections per person-year during the primary observation period was 3.43 (upper 95% CI 4.57). All but one non-bacterial infection were mild or moderate in intensity. IgG trough levels were constant during the course of the study. Eleven patients (18.0%) experienced 14 mild or moderate systemic adverse events (AEs) related to octanorm. The rate of related AEs per infusion was 0.004. In 76.7% of infusions, no infusion site reactions were observed and only two (0.3%) reactions were deemed severe. The incidence of site reactions decreased with successive infusions. Conclusion: The new 16.5% SCIG octanorm was shown to be efficacious in preventing infections in PIDs, and was well tolerated.