Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm
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 a...
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2021-05-01
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doaj-baad1c5a23b04e5c90481f92caec740d2021-06-28T03:13:50ZengWolters KluwerHemaSphere2572-92412021-05-0155e55510.1097/HS9.0000000000000555202105000-00012Selecting β-thalassemia Patients for Gene Therapy: A Decision-making AlgorithmDonatella Baronciani0Maddalena Casale1Lucia De Franceschi2Giovanna Graziadei3Filomena Longo4Raffaella Origa5Paolo Rigano6Valeria Pinto7Monia Marchetti8Antonia Gigante9Achille Iolascon10(on behalf of, & Chair of, the EHA Scientific Working Group on Red Cells and Iron)Gian Luca Forni111 UOC Immunoematologia e Trasfusionale, AORMN, Pesaro-Fano, Italy2 Università degli Studi della Campania «Luigi Vanvitelli», Napoli, Italy3 Dipartimento di Medicina, Università di Verona & AOUI Verona, Italy4 Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico Malattie Rare, Milano, Italy5 Centro Microcitemie-Pediatria, AOU San Luigi Gonzaga, Orbassano (TO), Italy6 Ospedale Pediatrico Microcitemico “A.Cao,” A.O. “G.Brotzu,” Cagliari, Italy7 Divisione di Malattie Rare del Sangue e degli Organi Emopoietici, Ospedale V. Cervello, Palermo, Italy8 Centro della Microcitemia e Anemie Congenite, E.O. Ospedali Galliera, Genova, Italy9 Hematology Day Service, SOC Oncologia, Ospedale Cardinal Massaia, Asti, Italy10 Società Italiana Talassemie ed Emoglobinopatie (SITE), Roma, Italy11 Dept. of Molecular Medicine and Medical Biotechnology, University of Naples Federico II, Naples, Italy8 Centro della Microcitemia e Anemie Congenite, E.O. Ospedali Galliera, Genova, ItalyThis 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.http://journals.lww.com/10.1097/HS9.0000000000000555 |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
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 |
spellingShingle |
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 Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm HemaSphere |
author_facet |
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 |
author_sort |
Donatella Baronciani |
title |
Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm |
title_short |
Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm |
title_full |
Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm |
title_fullStr |
Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm |
title_full_unstemmed |
Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm |
title_sort |
selecting β-thalassemia patients for gene therapy: a decision-making algorithm |
publisher |
Wolters Kluwer |
series |
HemaSphere |
issn |
2572-9241 |
publishDate |
2021-05-01 |
description |
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. |
url |
http://journals.lww.com/10.1097/HS9.0000000000000555 |
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