The peripheral blood mononuclear cells versus purified CD34+ cells transplantation in patients with angiitis-induced critical limb ischemia trial: 5-year outcomes and return to work analysis-a randomized single-blinded non-inferiority trial

Hao Liu; Tianyue Pan; Yifan Liu; Yuan Fang; Gang Fang; Xiaolang Jiang; Bin Chen; Zheng Wei; Shiyang Gu; Peng Liu; Weiguo Fu; Zhihui Dong

doi:10.1186/s13287-022-02804-4

The peripheral blood mononuclear cells versus purified CD34⁺ cells transplantation in patients with angiitis-induced critical limb ischemia trial: 5-year outcomes and return to work analysis-a randomized single-blinded non-inferiority trial

Stem Cell Res Ther. 2022 Mar 21;13(1):116. doi: 10.1186/s13287-022-02804-4.

Authors

Hao Liu^#^{1

2}, Tianyue Pan^#^{1

2}, Yifan Liu^#^{1

2}, Yuan Fang^{1

2}, Gang Fang^{1

2}, Xiaolang Jiang^{1

2}, Bin Chen^{1

2}, Zheng Wei³, Shiyang Gu³, Peng Liu³, Weiguo Fu^{4

5}, Zhihui Dong^{6

7}

Affiliations

¹ Departments of Vascular Surgery of Zhongshan Hospital, Fudan University, 180 Fenglin Road, Shanghai, 200032, China.
² National Clinical Research Center for Interventional Medicine, Shanghai, China.
³ Departments of Hematology of Zhongshan Hospital, Fudan University, Shanghai, China.
⁴ Departments of Vascular Surgery of Zhongshan Hospital, Fudan University, 180 Fenglin Road, Shanghai, 200032, China. fu.weiguo@zs-hospital.sh.cn.
⁵ National Clinical Research Center for Interventional Medicine, Shanghai, China. fu.weiguo@zs-hospital.sh.cn.
⁶ Departments of Vascular Surgery of Zhongshan Hospital, Fudan University, 180 Fenglin Road, Shanghai, 200032, China. dzh926@126.com.
⁷ National Clinical Research Center for Interventional Medicine, Shanghai, China. dzh926@126.com.

^# Contributed equally.

Abstract

Backgrounds: Patients with AICLI constitute a considerable proportion of NO-CLI patients and cannot be treated with surgical or endovascular treatment. Although cell therapy has shown satisfactory results in treating AICLI, research comparing the efficacy of treatment with the 2 kinds of cell products is rare. The aim of this study was to report the 5-year outcomes of a randomized single-blinded noninferiority trial (Number: NCT02089828) on peripheral blood mononuclear cells (PBMNCs) and purified CD34⁺ cells (PCCs) transplantation for treating angiitis-induced critical limb ischemia (AICLI).

Methods: A randomized single-blinded non-inferiority trial (Number: NCT02089828) was performed. Fifty patients were randomized 1:1 to the PBMNCs and PCCs groups. Efficacy outcomes, safety outcomes and patients' work conditions were analyzed. The primary efficacy outcomes included major amputation and total amputation over 60 months.

Results: During the 60-month follow-up, 1 patient was lost to follow-up, 1 died, and 2 underwent major amputation. The major amputation-free survival rate (MAFS) was 92.0% (95% confidence interval [CI] 82.0%-100.0%) in the PBMNCs group and 91.7% (95% CI 81.3%-100.0%) in the PCCs group (P = 0.980). Compared with the PCCs group, the PBMNCs group had a significantly higher 5-year new lesion-free survival rate (100.0% vs. 83.3% [95% CI 69.7-99.7%], P = 0.039). All patients lost their ability to work before transplantation, and the 5-year cumulative return to work (RTW) rates were 88.0% in the PBMNCs group and 76.0% in the PCCs group (P = 0.085).

Conclusion: The long-term follow-up outcomes of this trial not only demonstrated similar efficacy and safety for the 2 types of autoimplants but also showed a satisfactory cumulative RTW rate in AICLI patients who underwent cell transplantation.

Trial registration: ClinicalTrials.gov, number NCT02089828. Registered 14 March 2014, https://clinicaltrials.gov/ct2/show/record/NCT02089828 .

Keywords: CD34+; Cell transplantation; Critical limb ischemia.

Publication types

Randomized Controlled Trial
Research Support, Non-U.S. Gov't

MeSH terms

Cell Transplantation
Chronic Limb-Threatening Ischemia
Humans
Ischemia / therapy
Leukocytes, Mononuclear*
Return to Work
Treatment Outcome
Vasculitis*

Associated data

ClinicalTrials.gov/NCT02089828
ClinicalTrials.gov/NCT02089828