학술논문
Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with Betibeglogene Autotemcel (Beti-cel) Gene Therapy: Up to 9 Years of Follow-up
Document Type
Article
Author
Kwiatkowski, Janet L.; Olson, Timothy S.; Walters, Mark C.; Porter, John B.; Schneiderman, Jennifer; Hongeng, Suradej; Kulozik, Andreas; Cavazzana, Marina; Sauer, Martin G.; Thrasher, Adrian J.; Thuret, Isabelle; Lal, Ashutosh; Rasko, John E. J.; Yannaki, Evangelia; Ali, Shamshad; Tao, Gloria; Deora, Ami; Thakar, Himal L.; Colvin, Richard A.; Locatelli, Franco; Thompson, Alexis A.
Source
Blood; November 2023, Vol. 142 Issue: 1, Number 1 Supplement 1 p2480-2480, 1p
Subject
Language
ISSN
00064971; 15280020
Abstract
Introduction:Beti-cel is an approved, one-time gene therapy treatment for adult and pediatric patients with TDT. Beti-cel consists of autologous hematopoietic stem and progenitor cells transduced with the BB305 lentiviral vector encoding a modified β-globin gene (β A-T87Q), which produces functional hemoglobin (Hb) and addresses the underlying genetic cause of TDT. While transfusion independence (TI) remains a critical component for assessing patient response to gene therapy, other measures, including markers of iron overload, are important for evaluating the impact of therapy and management of disease burden over time. Previously, we demonstrated that iron markers stabilized in patients who achieved TI and stopped chelation therapy. Here, we report iron management outcomes in patients who completed either a phase 1/2 or phase 3 beti-cel parent study and subsequently enrolled in the long-term follow-up study.