OBJECTIVES: To assess the efficacy and safety of intravenous immunoglobulin (IVIG) 10% (Panzyga® ), a novel human normal IVIG 10%, in patients with chronic immune thrombocytopenia (ITP). BACKGROUND: First-line treatment options in ITP include IVIGs. METHODS: In this prospective, open-label, non-controlled, multicentre, phase III study, patients received a daily dose of IVIG 10% (1 g kg-1 body weight) for two consecutive days. The primary end point was clinical response rate; secondary end points included alternate response definitions, time to response, response duration, platelet counts, regression of bleeding and safety. RESULTS: Forty patients were enrolled (57·5% male, mean age 36·7 years); the full analysis set comprised 36 patients. A clinical response was seen for 29 of 36 patients (80·6%). Median time to response and response duration was 2 days and 14 days, respectively. IVIG 10% was well tolerated at a maximum infusion rate of 8 mg (kg min)-1 in all but one patient; adverse events were mainly mild to moderate in severity, and the most frequent was headache (42·5%). CONCLUSION: IVIG 10% is well tolerated even at a high infusion speed and induces a rapid platelet count increase, thus decreasing the bleeding rate and the severity of bleeding events. TRIAL REGISTRY: ClinicalTrials.gov record: NCT01349790.

Clinical Project Management Octapharma Pharmazeutika Produktionsges m b H Vienna Austria

Department of Internal Medicine Hematology and Oncology University Hospital Brno Brno Czech Republic

Immunhaematology Institut für Transfusionsmedizin Universitätsmedizin Berlin Berlin Germany

Klinik für Gynäkologie Campus Virchow Klinikum Charite Universitätsmedizin Berlin Berlin Germany

SPARK Validation Fund Berlin Health Innovations Berlin Germany

See more in PubMed

Audia, S. , Mahévas, M. , Samson, M. , Godeau, B. & Bonnotte, B. (2017) Pathogenesis of immune thrombocytopenia. Autoimmunity Reviews, 16, 620–632. PubMed

Buchanan, G.R. & Adix, L. (2002) Grading of hemorrhage in children with idiopathic thrombocytopenic purpura. Journal of Pediatrics, 141, 683–688. PubMed

Bussel, J.B. & Pham, L.C. (1987) Intravenous treatment with gammaglobulin in adults with immune thrombocytopenic purpura: review of the literature. Vox Sanguinis, 52, 206–211. PubMed

Bussel, J.B. , Eldor, A. , Kelton, J.G. et al (2004) IGIV‐C, a novel intravenous immunoglobulin: evaluation of safety, efficacy, mechanisms of action, and impact on quality of life. Thrombosis and Haemostasis, 91, 771–778. PubMed

Cines, D.B. , Bussel, J.B. , Liebman, H.A. & Luning Prak, E.T. (2009) The ITP syndrome: pathogenic and clinical diversity. Blood, 113, 6511–6521. PubMed PMC

Colovic, M. , Dimitrijevic, M. , Sonnenburg, C. , Suvajdzic, N. , Donfrid, M. & Bogdanovic, A. (2003) Clinical efficacy and safety of a novel intravenous immunoglobulin preparation in adult chronic ITP. The Hematology Journal, 4, 358–362. PubMed

Committee for Medicinal Products for Human Use (2010). Guideline on the Clinical Investigation of Human Normal Immunoglobulin for Intravenous Administration (IVIg) (EMA/CHMP/BPWP/94033/2007 rev. 2). November 3, 2014. http://www.ema.europa.eu, Accessed December 2018.

Dash, C.H. , Gillanders, K.R. , Stratford Bobbitt, M.E. , Gascoigne, E.W. & Leach, S.J. (2014) Safety and efficacy of Gammaplex® in idiopathic thrombocytopenic purpura (http://ClinicalTrials.gov‐‐NCT00504075). PLoS One, 9, e96600. PubMed PMC

Daw, Z. , Padmore, R. , Neurath, D. et al (2008) Hemolytic transfusion reactions after administration of intravenous immune (gamma) globulin: a case series analysis. Transfusion, 48, 1598–1601. PubMed

Debes, A. , Bauer, M. & Kremer, S. (2007) Tolerability and safety of the intravenous immunoglobulin Octagam: a 10‐year prospective observational study. Pharmacoepidemiology and Drug Safety, 16, 1038–1047. PubMed

, van der Meer, J.W. , , van Beem, R.T. , Robak, T. , Deptala, A. & Strengers, P.F. (2011) Efficacy and safety of a nanofiltered liquid intravenous immunoglobulin product in patients with primary immunodeficiency and idiopathic thrombocytopenic purpura. Vox Sanguinis, 101, 138–146. PubMed

Hooper, J.A. (2008) Intravenous immunoglobulins: evolution of commercial IVIG preparations. Immunology and Allergy Clinics of North America, 28, 765–778, viii. PubMed PMC

Imbach, P. , Barandun, S. , Baumgartner, C. , Hirt, A. , Hofer, F. & Wagner, H.P. (1981a) High‐dose intravenous gammaglobulin therapy of refractory, in particular idiopathic thrombocytopenia in childhood. Helvetica Paediatrica Acta, 36, 81–86. PubMed

Imbach, P. , Barandun, S. , D'Apuzzo, V. et al (1981b) High‐dose intravenous gammaglobulin for idiopathic thrombocytopenic purpura in childhood. The Lancet, 1, 1228–1231. PubMed

Julia, A. , Kovaleva, L. , Loria, S. et al (2009) Clinical efficacy and safety of Flebogammadif®, a new high‐purity human intravenous immunoglobulin, in adult patients with chronic idiopathic thrombocytopenic purpura. Transfusion Medicine, 19, 260–268. PubMed

Kerr, J. , Quinti, I. , Eibl, M. et al (2014) Is dosing of therapeutic immunoglobulins optimal? A review of a three‐decade long debate in Europe. Frontiers in Immunology, 5, 629. PubMed PMC

Lo, E. & Deane, S. (2014) Diagnosis and classification of immune‐mediated thrombocytopenia. Autoimmunity Reviews, 13, 577–583. PubMed

Michel, M. (2013) Immune thrombocytopenia nomenclature, consensus reports, and guidelines: what are the consequences for daily practice and clinical research? Seminars in Hematology, 50 (Suppl. 1), S50–S54. PubMed

Neunert, C. , Lim, W. , Crowther, M. , Cohen, A. , Solberg, L. Jr. & Crowther, M.A. (2011) The American Society of Hematology 2011 evidence‐based practice guideline for immune thrombocytopenia. Blood, 117, 4190–4207. PubMed

Neunert, C.E. , Buchanan, G.R. , Imbach, P. et al (2013) Bleeding manifestations and management of children with persistent and chronic immune thrombocytopenia: data from the Intercontinental Cooperative ITP Study Group (ICIS). Blood, 121, 4457–4462. PubMed

Newland, A.C. , Burton, I. , Cavenagh, J.D. , Copplestone, A. , Dolan, G. , Houghton, J. & Reilly, T. (2001) Vigam‐S, a solvent/detergent‐treated intravenous immunoglobulin, in idiopathic thrombocytopenic purpura. Transfusion Medicine, 11, 37–44. PubMed

Provan, D. , Stasi, R. , Newland, A.C. et al (2010) International consensus report on the investigation and management of primary immune thrombocytopenia. Blood, 115, 168–186. PubMed

Robak, T. , Salama, A. , Kovaleva, L. et al (2009) Efficacy and safety of Privigen®, a novel liquid intravenous immunoglobulin formulation, in adolescent and adult patients with chronic immune thrombocytopenic purpura. Hematology, 14, 227–236. PubMed

Robak, T. , Mainau, C. , Pyringer, B. , Chojnowski, K. , Warzocha, K. , Dmoszynska, A. , Straub, J. & Imbach, P. (2010) Efficacy and safety of a new intravenous immunoglobulin 10% formulation (octagam® 10%) in patients with immune thrombocytopenia. Hematology, 15, 351–359. PubMed

Salama, A. (2011) Current treatment options for primary immune thrombocytopenia. Expert Review of Hematology, 4, 107–118. PubMed

Varga, G. , Volková, Z. , Leibl, H. et al (2006) Efficacy and safety of the new intravenous immunoglobulin IGIV 10% in adults with chronic idiopathic thrombocytopenic purpura. Transfusion Medicine and Hemotherapy, 33, 509–514.

Zufferey, A. , Kapur, R. & Semple, J.W. (2017) Pathogenesis and therapeutic mechanisms in immune thrombocytopenia (ITP). Journal of Clinical Medicine, 6, e16. PubMed

Safety and Tolerability of Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy: Results of the ProCID Study

Randomized trial of three IVIg doses for treating chronic inflammatory demyelinating polyneuropathy

See more in PubMed

ClinicalTrials.gov
NCT01349790