Publication: Wild-type HIV infection after treatment with lentiviral gene therapy for β-thalassemia
Issued Date
2021-07-13
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ISSN
24739537
24739529
24739529
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2-s2.0-85110215198
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Mahidol University
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SCOPUS
Bibliographic Citation
Blood Advances. Vol.5, No.13 (2021), 2701-2706
Suggested Citation
Suradej Hongeng, Usanarat Anurathapan, Duantida Songdej, Angsana Phuphuakrat, Kesinee Jongrak, Geoffrey Parsons, Briana Deary, Melissa Bonner, Gabor Veres, Mohammed Asmal Wild-type HIV infection after treatment with lentiviral gene therapy for β-thalassemia. Blood Advances. Vol.5, No.13 (2021), 2701-2706. doi:10.1182/bloodadvances.2020003680 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/78036
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Title
Wild-type HIV infection after treatment with lentiviral gene therapy for β-thalassemia
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Abstract
Betibeglogene autotemcel (beti-cel) gene therapy (GT) for patients with transfusiondependent b-thalassemia uses autologous CD341 cells transduced with BB305 lentiviral vector (LVV), which encodes a modified β-globin gene. BB305 LVV also contains select HIV sequences for viral packaging, reverse transcription, and integration. This case report describes a patient successfully treated with beti-cel in a phase 1/2 study (HGB- 204; #NCT01745120) and subsequently diagnosed with wild-type (WT) HIV infection. From 3.5 to 21 months postinfusion, the patient stopped chronic red blood cell transfusions; total hemoglobin (Hb) and GT-derived HbAT87Q levels were 6.6 to 9.5 and 2.8 to 3.8 g/dL, respectively. At 21 months postinfusion, the patient resumed transfusions for anemia that coincided with an HIV-1 infection diagnosis. Quantitative polymerase chain reaction assays detected no replication-competent lentivirus. Next-generation sequencing confirmed WT HIV sequences. Six months after starting antiretroviral therapy, total Hb and HbAT87Q levels recovered to 8.6 and 3.6 g/dL, respectively, and 3.5 years postinfusion, 13.4 months had elapsed since the patient's last transfusion. To our knowledge, this is the first report of WT HIV infection in an LVV-based GT recipient and demonstrates persistent long-term hematopoiesis after treatment with beti-cel and the ability to differentiate between WT HIV and BB305-derived sequences.