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

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.

SUBMITTER: Matsumoto T

PROVIDER: S-EPMC4945920 | biostudies-literature | 2016 Jul

REPOSITORIES: biostudies-literature

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

Matsumoto Takuya T Tanaka Michiko M Yoshiya Keiji K Yoshiga Ryosuke R Matsubara Yutaka Y Horiuchi-Yoshida Kumi K Yonemitsu Yoshikazu Y Maehara Yoshihiko Y

Scientific reports 20160715

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 ...[more]

PMID: 27418463

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Project description:ObjectiveAlthough statin therapy has been linked to fewer short-term complications after infrainguinal bypass, its effect on long-term survival remains unclear. We therefore examined associations between statin use and long-term mortality, graft occlusion, and amputation after infrainguinal bypass.MethodsWe used the Vascular Study Group of New England registry to study 2067 patients (71% male; mean age, 67 ± 11 years; 67% with critical limb ischemia [CLI]) who underwent infrainguinal bypass from 2003 to 2011. Of these, 1537 (74%) were on statins perioperatively and at 1-year follow-up, and 530 received no statin. We examined crude, adjusted, and propensity-matched rates of 5-year surviva1, 1-year amputation, graft occlusion, and perioperative myocardial infarction.ResultsPatients taking statins at the time of surgery and at the 1-year follow-up were more likely to have coronary disease (38% vs 22%; P < .001), diabetes (51% vs 36%; P < .001), hypertension (89% vs 77%; P < .001), and prior revascularization procedures (50% vs 38%; P < .001). Despite higher comorbidity burdens, long-term survival was better for patients taking statins in crude (risk ratio [RR], 0.7; P < .001), adjusted (hazard ratio, 0.7; P = .001), and propensity-matched analyses (hazard ratio, 0.7; P = .03). In subgroup analysis, a survival advantage was evident in patients on statins with CLI (5-year survival rate, 63% vs 54%; log-rank, P = .01) but not claudication (5-year survival rate, 84% vs 80%; log-rank, P = .59). Statin therapy was not associated with 1-year rates of major amputation (12% vs 11%; P = .84) or graft occlusion (20% vs 18%; P = .58) in CLI patients. Perioperative myocardial infarction occurred more frequently in patients on a statin in crude analysis (RR, 2.2; P = .01) but not in the matched cohort (RR, 1.9; P = .17).ConclusionsStatin therapy is associated with a 5-year survival benefit after infrainguinal bypass in patients with CLI. However, 1-year limb-related outcomes were not influenced by statin use in our large observational cohort of patients undergoing revascularization in New England.

Project description:BackgroundAlthough the mononuclear cell (MNC) transplantation could theoretically induce therapeutic angiogenesis in the patients with no-option critical limb ischemia (NO-CLI), the clinical responses to this approach are inconsistent among different clinical trials. The purpose of this study was to identify the prognostic factors of responders and develop a predictive nomogram to guide patient selection.MethodsWe retrospectively reviewed a consecutive NO-CLI cohort who received peripheral blood-derived transplantation in our center. The patients who survived and achieved complete remission of CLI at 6 months post-transplantation were defined as responders. Logistic regression models were used to screen and identify the prognostic factors based on which predictive nomogram was developed. A receiver operating characteristic (ROC) curve and a calibration curve were drawn to determine the discrimination level and predictive accuracy.ResultsThe study ultimately enrolled 103 patients, including 58 responders and 45 non-responders. Based on the multivariate regression analysis, age ≥ 50 years (odds ratio [OR] 0.201, P = 0.004), blood fibrinogen > 4 g/L (OR 0.176, P = 0.003), arterial occlusion above the knee/elbow (OR 0.232, P = 0.010), the transcutaneous pressure of oxygen (TcPO2) (OR 1.062, P = 0.006), and the Log total transplanted CD34+ cell count (OR 3.506, P = 0.046) were identified as independent prognostic factors of the responders in the nomogram. An area under the ROC curve of 0.851 indicated good discrimination, and the calibration curve of the predicted probability showed optimal agreement with that of the observed probability.ConclusionsAge, blood fibrinogen, arterial occlusion level, TcPO2, and the total transplanted CD34+ cell count were independent prognostic factors of the responders. A nomogram with high discrimination and accuracy was developed to provide individualized predictions.Trail registrationChiCTR, ChiCTR1800019401 . Registered 9 November 2018-Retrospectively registered.

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

Publications

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

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