دورية أكاديمية
Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm
| العنوان: | Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm |
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| المؤلفون: | Donatella Baronciani, Maddalena Casale, Lucia De Franceschi, Giovanna Graziadei, Filomena Longo, Raffaella Origa, Paolo Rigano, Valeria Pinto, Monia Marchetti, Antonia Gigante, Achille Iolascon, (on behalf of, & Chair of, the EHA Scientific Working Group on Red Cells and Iron), Gian Luca Forni |
| المصدر: | HemaSphere, Vol 5, Iss 5, p e555 (2021) |
| بيانات النشر: | Wiley, 2021. |
| سنة النشر: | 2021 |
| المجموعة: | LCC:Diseases of the blood and blood-forming organs |
| مصطلحات موضوعية: | Diseases of the blood and blood-forming organs, RC633-647.5 |
| الوصف: | This expert opinion originally developed by a panel of the Italian Society of Thalassemias and Hemoglobinopathies (SITE), reviewed and adopted by the European Hematology Association (EHA) through the EHA Scientific Working Group on Red Cells and Iron, has been developed as priority decision-making algorithm on evidence and consensus with the aim to identify which patients with transfusion-dependent beta-thalassemia (TDT) could benefit from a gene therapy (GT) approach. Even if the wide utilized and high successful allogeneic hematopoietic stem-cell transplantation provides the possibility to cure several patients a new scenario has been opened by GT. Therefore, it is important to establish the patients setting for whom it is priority indicated, particularly in the early phase of the diffuse use outside experimental trials conducted in high selected centers. Moreover, actual price, limited availability, and resources disposal constitute a further indication to a rational and progressive approach to this innovative treatment. To elaborate this algorithm, the experience with allogeneic transplantation has been used has a predictive model. In this large worldwide experience, it has been clearly demonstrated that key for the optimal transplant outcome is optimal transfusion and chelation therapy in the years before the procedure and consequently optimal patient’s clinical condition. In the document, different clinical scenarios have been considered and analyzed for the possible impact on treatment outcome. According to the European Medicine Agency (EMA) for the GT product, this expert opinion must be considered as a dynamic, updatable, priority-based indications for physicians taking care of TDT patients. |
| نوع الوثيقة: | article |
| وصف الملف: | electronic resource |
| اللغة: | English |
| تدمد: | 2572-9241 00000000 |
| Relation: | http://journals.lww.com/10.1097/HS9.0000000000000555; https://doaj.org/toc/2572-9241 |
| DOI: | 10.1097/HS9.0000000000000555 |
| URL الوصول: | https://doaj.org/article/baad1c5a23b04e5c90481f92caec740d |
| رقم الأكسشن: | edsdoj.baad1c5a23b04e5c90481f92caec740d |
| قاعدة البيانات: | Directory of Open Access Journals |
Full Text Finder | تدمد: | 25729241 00000000 |
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| DOI: | 10.1097/HS9.0000000000000555 |