دورية أكاديمية
أعرض في EDS

New plasma protein C and protein S concentrate: A synergy for therapeutic purposes.

التفاصيل البيبلوغرافية
العنوان: New plasma protein C and protein S concentrate: A synergy for therapeutic purposes.
المؤلفون: Mori F; Department of Research and Innovation, Kedrion Biopharma, Bolognana, Italy., Angelini C; Department of Research and Innovation, Kedrion Biopharma, Bolognana, Italy., Farina C; Department of Research and Innovation, Kedrion Biopharma, Bolognana, Italy.
المصدر: Vox sanguinis [Vox Sang] 2024 Mar; Vol. 119 (3), pp. 193-202. Date of Electronic Publication: 2023 Nov 28.
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: Blackwell Science Country of Publication: England NLM ID: 0413606 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1423-0410 (Electronic) Linking ISSN: 00429007 NLM ISO Abbreviation: Vox Sang Subsets: MEDLINE
أسماء مطبوعة: Publication: 2001- : Oxford, UK : Blackwell Science
Original Publication: Basel : Karger
مواضيع طبية MeSH: Purpura Fulminans*/drug therapy , Purpura Fulminans*/genetics , Protein C Deficiency*/drug therapy, Infant, Newborn ; Humans ; Protein C/analysis ; Protein C/therapeutic use ; Protein S ; Plasma/chemistry
مستخلص: Background and Objectives: Deficiencies of protein C (PC) or protein S (PS) are rare diseases, characterized by mutations in the PC or PS genes, which encode plasma serine proteases with anti-coagulant activity. Severe PC or PS deficiencies manifest in early life as neonatal purpura fulminans, a life-threatening heamorrhagic condition requiring immediate treatment. First-line treatment involves replacement therapy, followed by maintenance with anti-coagulants. Replacement therapy with specific protein concentrates is currently only limited to PC, and therefore, a PC + PS concentrate represents a useful addition to therapeutic options, particularly for severe PS deficiency. Further, the production of a PC + PS concentrate from unused plasma fractionation intermediates would impact favourably on manufacturing costs, and consequently therapy prices for patients and health systems.
Materials and Methods: Several chromatographic runs were performed on the same unused plasma fractionation intermediates using different supports to obtain a PC/PS concentrate. The best chromatographic mediums were chosen, in terms of specific activity and recovery. A full process of purification including virus inactivation/removal and lyophilization steps was set up.
Results: The final freeze-dried product had a mean PC concentration of 47.75 IU/mL with 11% of PS, and a mean specific activity of 202.5 IU/mg protein, corresponding to over 12,000-fold purification from plasma.
Conclusion: The development of a novel concentrated PC/PS mixture obtained from a waste fraction of other commercial products could be used for its potential therapeutic role in the management of neonatal purpura fulminans pathology.
(© 2023 The Authors. Vox Sanguinis published by John Wiley & Sons Ltd on behalf of International Society of Blood Transfusion.)
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معلومات مُعتمدة: Kedrion Biopharma Inc
فهرسة مساهمة: Keywords: neonatal purpura fulminans; protein C deficiency; protein S deficiency; purification of factor C and S concentrate proteins; unused plasma
المشرفين على المادة: 0 (Protein C)
0 (Protein S)
تواريخ الأحداث: Date Created: 20231129 Date Completed: 20240314 Latest Revision: 20240314
رمز التحديث: 20240314
DOI: 10.1111/vox.13567
PMID: 38018260
قاعدة البيانات: MEDLINE
الوصف
تدمد:1423-0410
DOI:10.1111/vox.13567