Three-Year Outcomes of a Randomized, Double-Blind, Placebo-Controlled Study Assessing Safety and Efficacy of C1 Esterase Inhibitor for Prevention of Delayed Graft Function in Deceased Donor Kidney Transplant Recipients

Clin J Am Soc Nephrol. 2020 Jan 7;15(1):109-116. doi: 10.2215/CJN.04840419. Epub 2019 Dec 16.

Abstract

Background and objectives: Delayed graft function is related to ischemia-reperfusion injury and may be complement dependent. We previously reported from a randomized, placebo-controlled trial that treatment with C1 esterase inhibitor was associated with a shorter duration of delayed graft function and higher eGFR at 1 year. Here, we report longer-term outcomes from this trial.

Design, setting, participants, & measurements: This is a post hoc analysis of a phase 1/2, randomized, controlled trial enrolling 70 recipients of deceased donor kidney transplants at risk for delayed graft function (NCT02134314). Subjects were randomized to receive C1 esterase inhibitor 50 U/kg (n=35) or placebo (n=35) intraoperatively and at 24 hours. The cumulative incidence functions method was used to compare graft failure and death over 3.5 years. eGFR slopes were compared using a linear mixed effects model.

Results: Three deaths occurred among C1 esterase inhibitor-treated patients compared with none receiving placebo. Seven graft failures developed in the placebo group compared with one among C1 esterase inhibitor-treated recipients; the cumulative incidence of graft failure was lower over 3.5 years among C1 esterase inhibitor-treated recipients compared with placebo (P=0.03). Although no difference in eGFR slopes was observed between groups (P for group-time interaction =0.12), eGFR declined in placebo-treated recipients (-4 ml/min per 1.73 m2 per year; 95% confidence interval, -8 to -0.1) but was stable in C1 esterase inhibitor-treated patients (eGFR slope: 0.5 ml/min per 1.73 m2 per year; 95% confidence interval, -4 to 5). At 3.5 years, eGFR was 56 ml/min per 1.73 m2 (95% confidence interval, 42 to 70) in the C1 esterase inhibitor group versus 35 ml/min per 1.73 m2 (95% confidence interval, 21 to 48) in the placebo group, with an estimated mean eGFR difference of 21 ml/min per 1.73 m2 (95% confidence interval, 2 to 41 ml/min per 1.73 m2).

Conclusions: Treatment of patients at risk for ischemia-reperfusion injury and delayed graft function with C1 esterase inhibitor was associated with a lower incidence of graft failure.

Keywords: complement C1 inhibitor protein; complement c1s; death; delayed graft function; double-blind method; epidermal growth factor receptor; glomerular filtration rate; human EGFR protein; humans; incidence; ischemia-reperfusion; kidney transplantation; reperfusion injury; tissue donors.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Complement C1 Inhibitor Protein / adverse effects
  • Complement C1 Inhibitor Protein / therapeutic use*
  • Delayed Graft Function / etiology
  • Delayed Graft Function / mortality
  • Delayed Graft Function / physiopathology
  • Delayed Graft Function / prevention & control*
  • Double-Blind Method
  • Female
  • Glomerular Filtration Rate / drug effects*
  • Humans
  • Incidence
  • Kidney / drug effects*
  • Kidney / physiopathology
  • Kidney Transplantation* / adverse effects
  • Kidney Transplantation* / mortality
  • Male
  • Middle Aged
  • Reperfusion Injury / etiology
  • Reperfusion Injury / mortality
  • Reperfusion Injury / physiopathology
  • Reperfusion Injury / prevention & control*
  • Risk Assessment
  • Risk Factors
  • Time Factors
  • Treatment Outcome

Substances

  • Complement C1 Inhibitor Protein