Fecal microbiota transplantation for patients with steroid-resistant/dependent acute graft-versus-host disease of the gut

Kazuhiko Kakihana, Yuki Fujioka, Wataru Suda, Yuho Najima, Go Kuwata, Satoshi Sasajima, Iyo Mimura, Hidetoshi Morita, Daisuke Sugiyama, Hiroyoshi Nishikawa, Masahira Hattori, Yutaro Hino, Shuntaro Ikegawa, Keita Yamamoto, Takashi Toya, Noriko Doki, Koichi Koizumi, Kenya Honda and Kazuteru Ohashi

Key points

  • FMT was safely performed in SCT patients, with 3 CRs and 1 PR.

  • Temporal microbiota dynamics seem linked to gut condition and effector Treg cells also increased during response to FMT.


Increasing evidence indicates that the gut microbiota is closely associated with acute graft-versus-host disease (aGVHD) in stem cell transplantation (SCT). Fecal microbiota transplantation (FMT) could represent an alternative treatment option for aGVHD. However, FMT for SCT patients carries a potential risk of infection by infused microbiota because of the severely immunosuppressed status. We therefore conducted a pilot study to evaluate the safety of FMT in SCT. A total of 4 patients with steroid-resistant (n=3) or steroid-dependent gut aGVHD (n=1) received FMT. No severe adverse events attributed to FMT were observed. All patients responded to FMT, with 3 complete responses and 1 partial response. Temporal dynamics of microbiota seemed to be linked to the gut condition of patients and peripheral effector regulatory T cells also increased during response to FMT. FMT was safely performed in our patients and might offer a novel therapeutic option for aGVHD. This study is registered to the University Hospital Medical Information Network as #UMIN000015115 (

  • Submitted May 18, 2016.
  • Accepted July 18, 2016.