Efficacy and safety of lentivirus gene therapy in the correction of sickle cell disease

Abstract

Background and objective: Lentivirus gene therapy (LGT) is an emerging therapy for sickle cell disease (SCD), although its efficacy and safety are under evaluation in clinical trials. This review assessed the efficacy and safety of LGT in relation to hydroxyurea (HU). Materials and methods: A systematic review was conducted using The Preferred Reporting Items for Systematic Review and Meta-analysis protocol. Following a set of inclusion criteria, 10 studies were selected for quality assessment, extraction, and meta-analysis from 499 studies pooled from PubMed, ScienceDirect and Sematic Scholar. Data obtained were described and subjected to random effect meta-analysis using RevMan software. Results: There was a significant increase (p-value<0.00001) in haemoglobin (Hb) level after LGT and production of HbAT87Q and foetal haemoglobin (HbF). Clinical outcome decreased significantly, and no hospitalization was required following LGT. A significant age-related difference in the LGT outcome was observed. Mode 1 treatment had significantly higher (p=0.004) outcome compared to mode 2 treatment. There was a significant increase (p<0.00001) in treatment outcome in SCD patients treated with LGT compared to those treated with HU. Gastroenteritis and leucopenia were the most reported adverse effects. Conclusion: The review has demonstrated that LGT has a promising efficacy in the treatment of SCD although there are existing safety concerns.