TY - JOUR
T1 - Use of unapproved or off-label drugs in Japan for the treatment of graft-versus-host disease and post-transplant viral infection
AU - Kuwatsuka, Yachiyo
AU - Atsuta, Yoshiko
AU - Hirakawa, Akihiro
AU - Uchida, Naoyuki
AU - Inamoto, Yoshihiro
AU - Najima, Yuho
AU - Ikegame, Kazuhiro
AU - Eto, Tetsuya
AU - Ozawa, Yukiyasu
AU - Ichinohe, Tatsuo
AU - Inoue, Masami
AU - Kimura, Takafumi
AU - Okamoto, Shinichiro
AU - Miyamura, Koichi
AU - Fukuda, Takahiro
N1 - Publisher Copyright:
© 2020, Japanese Society of Hematology.
PY - 2020/12
Y1 - 2020/12
N2 - Many drugs are used for unapproved indications in Japan for post hematopoietic stem cell transplant (HCT) complications. To investigate unapproved or off-label drug usage for graft-versus-host disease (GVHD) and virus infections after allogeneic HCT, we analyzed the data of Japanese HCT registry. Between 2006 and 2017, 39,941 adults and children received HCT for a variety of disease and their transplant data were captured in the registry. Among them, 14,687 and 8914 patients receiving treatment for acute and/or chronic GVHD, 24,828 patients with cytomegalovirus (CMV) infection or receiving therapies for CMV, and 4943 who received treatment for other viral infections were included in the analyses of off-label or unapproved drugs. For GVHD, mycophenolate mofetil was the most frequently used off-label drug, followed by beclomethasone, infliximab, and etanercept. For viral infections other than CMV, foscarnet was the most frequently used off-label drug. Cidofovir, which is not approved for use in Japan, was mainly used for adenovirus infection. This study demonstrated that numerous off-label and unapproved drugs have been used as key drugs for GVHD and post-transplant viral infection, and the real world date in the transplant registry may serve as an important asset to regulatory purposes.
AB - Many drugs are used for unapproved indications in Japan for post hematopoietic stem cell transplant (HCT) complications. To investigate unapproved or off-label drug usage for graft-versus-host disease (GVHD) and virus infections after allogeneic HCT, we analyzed the data of Japanese HCT registry. Between 2006 and 2017, 39,941 adults and children received HCT for a variety of disease and their transplant data were captured in the registry. Among them, 14,687 and 8914 patients receiving treatment for acute and/or chronic GVHD, 24,828 patients with cytomegalovirus (CMV) infection or receiving therapies for CMV, and 4943 who received treatment for other viral infections were included in the analyses of off-label or unapproved drugs. For GVHD, mycophenolate mofetil was the most frequently used off-label drug, followed by beclomethasone, infliximab, and etanercept. For viral infections other than CMV, foscarnet was the most frequently used off-label drug. Cidofovir, which is not approved for use in Japan, was mainly used for adenovirus infection. This study demonstrated that numerous off-label and unapproved drugs have been used as key drugs for GVHD and post-transplant viral infection, and the real world date in the transplant registry may serve as an important asset to regulatory purposes.
KW - Drug lag
KW - Graft-versus-host disease
KW - Post-transplant viral infection
KW - Registry
KW - Stem cell transplantation
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U2 - 10.1007/s12185-020-02972-0
DO - 10.1007/s12185-020-02972-0
M3 - Article
C2 - 32875487
AN - SCOPUS:85090113752
SN - 0925-5710
VL - 112
SP - 841
EP - 850
JO - International Journal of Hematology
JF - International Journal of Hematology
IS - 6
ER -