Article
作者: Nakamura, Toru ; Fujimori, Yoshihiro ; Yoh, Kazunori ; Fukami, Kei ; Kobayashi, Kaoru ; Yamahara, Kenichi ; Tanizawa, Kyoko ; Yamakado, Koichiro ; Ioji, Tetsuya ; Suzuki, Hiroyuki ; Yamamoto, Hidekazu ; Kako, Makoto ; Ohtake, Takayasu ; Takashima, Tomoyuki ; Kako, Yasukazu ; Kaibori, Masaki ; Masuda, Atsutaka ; Uojima, Haruki ; Torimura, Takuji ; Nagafuji, Koji ; Kawaguchi, Takumi ; Fujita, Yasuyuki ; Enomoto, Hirayuki ; Yoshihara, Satoshi ; Koga, Hironori ; Nishiguchi, Shuhei ; Kobayashi, Shuzo ; Hazama, Takuma ; Tsukiyama, Toshitaka ; Kawamoto, Atsuhiko ; Iwamoto, Hideki
IntroductionIn this multicenter clinical study, we aimed to investigate the efficacy and safety of the transhepatic arterial administration of granulocyte-colony stimulating factor (G-CSF)-mobilized autologous peripheral blood (PB)-CD34+ cells compared with standard therapy in patients with decompensated cirrhosis type C.MethodsPatients were randomly assigned (2:1) to the CD34+ cell transplant (CD34+ cell) or standard-of-care (SOC) group and followed up for 52 weeks. The primary endpoints were the non-progression rate of Child-Pugh (CP) scores at 24 weeks post-enrollment and the safety of the protocol treatment.ResultsFourteen patients (CD34+ cell group: 10; SOC group: 4) were enrolled. CP scores at 24 weeks had a non-progression rate of 90% in the CD34+ cell group and 100% in the SOC group, with no significant difference between groups. Importantly, 4 out of 10 patients in the CD34+ cell group exhibited an improvement from decompensated to compensated cirrhosis, whereas all patients in the SOC group remained in decompensated cirrhosis. With regard to secondary endpoints, a trend toward increased serum albumin levels in the CD34+ cell group was noted. Serious adverse events (SAEs) occurred in three patients in the CD34+ cell group and in one patient in the SOC group. No causal relationship was observed between all SAEs and G-CSF, leukapheresis, or cell transplantation in the CD34+ cell group. No patients died and no hepatocellular carcinoma occurred within the study period.ConclusionsPB-CD34+ cell infusion therapy may have the potential to circumvent the decompensated stage of cirrhosis, thus avoiding the need for liver transplantation.