Improved quality of life in patients with no-option critical limb ischemia undergoing gene therapy with DVC1-0101

Takuya Matsumoto, Michiko Tanaka, Keiji Yoshiya, Ryosuke Yoshiga, Yutaka Matsubara, Kumi Horiuchi-Yoshida, Yoshikazu Yonemitsu, Yoshihiko Maehara, Takuya Matsumoto, Michiko Tanaka, Keiji Yoshiya, Ryosuke Yoshiga, Yutaka Matsubara, Kumi Horiuchi-Yoshida, Yoshikazu Yonemitsu, Yoshihiko Maehara

Abstract

Critical limb ischemia (CLI) has a poor prognosis and adversely affects patients' quality of life (QOL). Therapeutic angiogenesis may improve mobility, mortality, and QOL in CLI patients. However, the effectiveness of gene therapy on such patients' QOL is unknown. DVC1-0101, a non-transmissible recombinant Sendai virus vector expressing human fibroblast growth factor-2 gene, demonstrated safety and efficacy in a phase I/II study of CLI patients. We investigated the effects of DVC1-0101 on QOL in this cohort. QOL was assessed using the Short Form-36 health survey version 2 (SF-36) in 12 patients at pre-administration, 28 days, and 3, 6, and 12 months post-treatment. We examined differences between pre and post-administration QOL scores and correlations between QOL scores and vascular parameters. Patients demonstrated low baselines scores on every SF-36 dimension. Post-treatment scores showed significant improvements in physical functioning at 3 and 6 months (P < 0.05), role-physical at 3, 6, and 12 months (P < 0.05), bodily pain at 1, 3, 6, and 12 months (P < 0.05), vitality at 1, 6, and 12 months (P < 0.05), and physical component summary at 6 and 12 months (P < 0.05). DVC1-0101-based gene therapy may improve QOL in CLI patients over a 6-month period.

Conflict of interest statement

Dr. Yonemitsu was a previous member of the scientific advisory board of DNAVEC Corporation, which has developed DVC1-0101. The other authors declare that they have no conflicts of interest.

Figures

Figure 1. Differences in Short Form-36 health…
Figure 1. Differences in Short Form-36 health survey scores in 12 patients with critical limb ischemia compared with Japanese national standards.
The Short Form-36 health survey includes eight domains: physical functioning (PF); role-physical (RP); bodily pain (BP); general health (GH); vitality (VT); social functioning (SF); role-emotional (RE); and mental health (MH). Abbreviation: SD, standard deviation.
Figure 2. Overall summary measures of the…
Figure 2. Overall summary measures of the Short Form-36 health survey for all 12 patients with critical limb ischemia.
The eight domains of the Short Form-36 health survey can be aggregated into three summary measures: physical component summary (PCS); mental component summary (MCS); and role/social component summary (RCS).

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