Efficacy and safety of octanorm (cutaquig(®)) in adults with primary immunodeficiencies with predominant antibody deficiency: a prospective, open-label study

Authors: Latysheva, E., Rodina, Y., Sizyakina, L., Totolian, A., and Tuzankina, I.

Publication: Immunotherapy; April 2020

Affiliations: Immunopathology Department, National Research Center Institute of Immunology FMBA, 24 Kashirskoe Shosse, Moscow 115522, Russia; Immunology Department, Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology & Immunology, 1 Samory Mashela Street, Moscow 117997, Russia; Department of Clinical Immunology and Allergology, State Medical University, Rostov 344022, Russia; Laboratory of Molecular Immunology, St Petersburg Pasteur Institute, 14 Mira Street, St Petersburg 197101, Russia; Department of Clinical Immunology, Institute of Immunology & Physiology of the Ural Branch of the Russian Academy of Sciences, 106 Pervomayskaya Street, Yekaterinburg 620049, Russia.

Abstract: AIM: To evaluate efficacy and safety of octanorm (16.5% subcutaneous immunoglobulin) in adult patients with primary immunodeficiencies. PATIENTS & METHODS: Primary immunodeficiencies patients (18-70 years) previously treated with intravenous immunoglobulin were included in this Phase III study. Octanorm was administered subcutaneously once weekly over 8 months. End points included infections, adverse events and quality of life. RESULTS: 25 patients (mean age 35.2 years, female 60.0%) were recruited, 24 completed the study. Mean dose of octanorm was 0.11 g/kg/week. No serious bacterial infections occurred. Three patients (12.0%) had an adverse event (mild) assessed as related to octanorm. Both the mental and physical summary 36-item Short Form Health Survey scores were improved. CONCLUSION: Octanorm is effective, safe and improves quality of life. Clinical Trial registration number: NCT03988426.