Lack of Efficacy and Safety of Eculizumab for Treatment of Antibody-Mediated Rejection Following Renal Transplantation

Sujung Heoa,b, Youngchan Parka,b, Nagyeom Leea,b, Yanghyeon Kima,b, Ye Na Kima,b, Ho Sik Shina,b*,

Yeonsoon Junga,b, Hark Rima,b, Helmut G. Rennke c, and Anil Chandraker d

a Renal Division, Department of Internal Medicine, Gospel Hospital, Kosin University College of Medicine, Busan, South Korea; 

b Transplantation Research Institute, Kosin University College of Medicine, Busan, South Korea; 

c Renal Pathology, Department of Pathology,Brigham & Women’s Hospital, Harvard Medical School, Boston, MA, USA; 

and d Transplantation Research Center, Renal Division, Brigham & Women’s Hospital, Harvard Medical School, Boston, Massachusetts, USA

ABSTRACT

Background. We evaluated the efficacy and safety of eculizumab in comparison with plasmapheresis

and intravenous immunoglobulin therapy in renal transplant recipients diagnosed with

antibody-mediated rejection (AMR).

Methods. This was a multicenter, open-label, prospective, randomized analysis. The patients

were randomized by therapy type (eg, eculizumab infusions or standard of care [SOC]: plasmapheresis/

intravenous immunoglobulin). The patients (ie, eculizumab arm: 7 patients, SOC arm: 4

patients) were evaluated for the continued presence of donor-specific antibodies (DSAs) and

C4d (staining on biopsy), as well as histologic evidence, using repeat renal biopsy after

treatment.

Results. The allograft biopsies revealed that eculizumab did not prevent the progression to

transplant glomerulopathy. Only 2 patients in the SOC arm experienced rejection reversal, and

no graft losses occurred in either group. After AMR treatment, the DSA titers generally

decreased compared to titers taken at the time of AMR diagnosis. There were no serious adverse

effects in the eculizumab arm.

Conclusions. Eculizumab alone cannot treat AMR effectively and does not prevent acute

AMR from progressing to chronic AMR or transplant glomerulopathy. However, it should be

considered as a potential alternative therapy because it may be associated with decreased DSA

levels.