Efficacy and safety of a new intravenous immunoglobulin (Panzyga® ) in chronic immune thrombocytopenia
Jazyk angličtina Země Velká Británie, Anglie Médium print-electronic
Typ dokumentu klinické zkoušky, fáze III, časopisecké články, multicentrická studie
Grantová podpora
Octapharma
PubMed
30687970
PubMed Central
PMC6850321
DOI
10.1111/tme.12573
Knihovny.cz E-zdroje
- Klíčová slova
- ITP, IVIG, Panzyga®, immune thrombocytopenia, intravenous immunoglobulin, platelet count, safety,
- MeSH
- chronická nemoc MeSH
- dospělí MeSH
- idiopatická trombocytopenická purpura krev farmakoterapie MeSH
- imunoglobulin G aplikace a dávkování škodlivé účinky MeSH
- intravenózní imunoglobuliny aplikace a dávkování škodlivé účinky MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- počet trombocytů MeSH
- prospektivní studie MeSH
- senioři MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- mužské pohlaví MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- klinické zkoušky, fáze III MeSH
- multicentrická studie MeSH
- Názvy látek
- imunoglobulin G MeSH
- intravenózní imunoglobuliny MeSH
- Panzyga MeSH Prohlížeč
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
Zobrazit více v 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
Randomized trial of three IVIg doses for treating chronic inflammatory demyelinating polyneuropathy
ClinicalTrials.gov
NCT01349790