Use of hematopoietic stem cell transplantation registry data to challenge the drug lag

• Project/Area Number: 26860346
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Medical sociology
• Research Institution: Nagoya University
• Principal Investigator: Kuwatsuka Yachiyo 名古屋大学, 医学部附属病院, 病院助教 (00720509)
• Project Period (FY): 2014-04-01 – 2017-03-31
• Project Status: Completed (Fiscal Year 2016)
• Budget Amount: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000); Fiscal Year 2015: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000); Fiscal Year 2014: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
• Keywords: ドラッグラグ / レジストリデータ / 造血幹細胞移植 / 移植後合併症 / 難治性疾患 / 未承認・適応外薬 / 移植片対宿主病

Outline of Final Research Achievements

We aimed to investigate the use of the unapproved or off-label drugs for hematopoietic stem cell transplantation and analyzed the Japanese nation level registry data. (1) We retrospectively analyzed the data of 39144 patients who received allogeneic stem cell transplantation. Chemotherapeutic agent, immunosuppressive drugs, anti-invective drugs were extracted and coded. The annual trends in the number of patients who used each unapproved or off-label drugs were obtained. (2) We analyzed the data of umbilical cord blood transplant and unrelated bone marrow transplant in 272 adults with aplastic anemia, and reported the transplant outcomes with information of the use of the off-label drugs.

Research Products

• [Int'l Joint Research] Medical College of Wisconsin/University of North Carolina/Seattle Cancer Care Alliance(米国)
• [Int'l Joint Research] University of Manitoba/Princess Margaret Cancer Centre/BC Children's Hospital(カナダ)
• [Int'l Joint Research] University of Freiburg(ドイツ)
• [Int'l Joint Research] University of New Castle/Royal Melbourne Hospital(オーストラリア)
• [Int'l Joint Research] Federal University of Parana(ブラジル)
• [Int'l Joint Research]
• [Journal Article] Impact of T-cell chimerism on relapse after cord blood transplantation for hematological malignancies: Nagoya Blood and Marrow Transplantation Group study (2017)
  • Author(s): Yokohata E, Kuwatsuka Y, Ohashi H, Terakura S, Kawashima N, Seto A, Kurahashi S, Ozawa Y, Goto T, Imahashi N, Nishida T, Miyao K, Sakemura R, Kato T, Sawa M, Kohno A, Sao H, Iida H, Kiyoi H, Naoe T, Miyamura K, Murata M
  • Journal Title: Bone Marrow Transplant. Volume: in press Issue: 4 Pages: 612-614
  • DOI: 10.1038/bmt.2016.323
  • Peer Reviewed
• [Journal Article] Role of reduced-intensity conditioning allogeneic hematopoietic cell transplantation in older patients with de novo acute myeloid leukemia. (2017)
  • Author(s): Yamasaki S, Hirakawa A, Aoki J, Uchida N, Fukuda T, Ogawa H, Ohashi K, Kondo T, Eto T, Kanamori H, Okumura H, Iwato K, Ichinohe T, Kanda J, Onizuka M, Kuwatsuka Y, Yanada M, Atsuta Y, Takami A, Yano S.
  • Journal Title: Ann Hematol Volume: 96 Issue: 2 Pages: 289-297
  • DOI: 10.1007/s00277-016-2872-y
  • Peer Reviewed
• [Journal Article] A Comparison of Outcomes for Cord Blood Transplantation and Unrelated Bone Marrow Transplantation in Adult Aplastic Anemia. (2016)
  • Author(s): Kuwatsuka Y, Kanda J, Yamazaki H, Mori T, Miyamura K, Kako S, Uchida N, Ohashi K, Ozawa Y, Takahashi Y, Kato C, Iwato K, Ishiyama K, Kobayashi H, Eto T, Kahata K, Kato J, Miyamoto T, Kato K, Mori S, Atsuta Y, Kimura F, Kanda Y.
  • Journal Title: Biol Blood Marrow Transplant Volume: 22 Issue: 10 Pages: 1836-1843
  • DOI: 10.1016/j.bbmt.2016.07.002
  • Peer Reviewed / Acknowledgement Compliant
• [Journal Article] Small-molecule Hedgehog inhibitor attenuates the leukemia-initiation potential of acute myeloid leukemia cells. (2016)
  • Author(s): Fukushima N, Minami Y, Kakiuchi S, Kuwatsuka Y, Hayakawa F, Jamieson C, Kiyoi H, Naoe T.
  • Journal Title: Cancer Sci Volume: 107 Issue: 10 Pages: 1422-1429
  • DOI: 10.1111/cas.13019
  • NAID: 120005898583
  • Peer Reviewed / Int'l Joint Research
• [Journal Article] Quality control and assurance in hematopoietic stem cell transplantation data registries in Japan and other countries (2016)
  • Author(s): Kuwatsuka Y
  • Journal Title: International journal of hematology Volume: 103 Issue: 1 Pages: 20-24
  • DOI: 10.1007/s12185-015-1896-8
  • Peer Reviewed / Acknowledgement Compliant
• [Journal Article] Comparing Outcomes with Bone Marrow or Peripheral Blood Stem Cells as Graft Source for Matched Sibling Transplants in Severe Aplastic Anemia across Different Economic Regions (2016)
  • Author(s): Kumar R, Kimura F, Ahn KW, Hu ZH, Kuwatsuka Y, Klein JP, et al.
  • Journal Title: Biology of Blood and Marrow Transplantation Volume: 22 Issue: 5 Pages: 932-940
  • DOI: 10.1016/j.bbmt.2016.01.012
  • Peer Reviewed / Int'l Joint Research
• [Journal Article] GVHD after HLA-matched sibling BMT or PBSCT: Comparison of North American Caucasian and Japanese populations (2016)
  • Author(s): Kanda J, Brazauskas R, Hu Z, Kuwatsuka Y, Nagafuji K, Kanamori H, Kanda Y, Miyamura K, Murata M, Fukuda T, Sakamaki H, Kimura F, Seo S, Aljurf M, Yoshimi A, Milone G, Wood WA, Ustun C, Hashimi S, Pasquini M, Bonfim C, Dalal J, Hahn T, Atsuta Y, Saber W
  • Journal Title: Biol Blood Marrow Transplant Volume: 22 Issue: 4 Pages: 744-751
  • DOI: 10.1016/j.bbmt.2015.12.027
  • Peer Reviewed / Open Access / Int'l Joint Research / Acknowledgement Compliant
• [Journal Article] Influence of melphalan plus fludarabine-conditioning regimen in elderly patients aged >55 years with hematological malignancies. (2016)
  • Author(s): Miyao K, Sawa M, Kuwatsuka Y, Ozawa Y, Kato T, Kohno A, Sao H, Nishida T, Iida H, Naito K, Tsurumi H, Taji H, Mizuta S, Kusumoto S, Nakase K, Morishita Y, Kawashima N, Miyamura K, Murata M.
  • Journal Title: Bone Marrow Transplant Volume: 51 Issue: 1 Pages: 157-160
  • DOI: 10.1038/bmt.2015.235
  • Peer Reviewed
• [Presentation] 造血幹細胞移植における免疫抑制薬の未承認・適応外使用状況の検討：ドラッグ・ラグ解消に向けて (2017)
  • Author(s): 鍬塚八千代, 熱田由子, 平川晃弘, 内田直之, 土岐典子, 一戸辰夫, 井上雅美, 高梨美乃子, 岡本真一郎, 宮村耕一, 福田隆浩
  • Organizer: 第39回日本造血細胞移植学会総会
  • Place of Presentation: 島根県民会館（島根県松江市）
  • Year and Date: 2017-03-03
• [Presentation] AYA 世代の AML：内科の立場から (2016)
  • Author(s): 鍬塚八千代，清井仁
  • Organizer: 第78回日本血液学会学術集会
  • Place of Presentation: パシフィコ横浜（神奈川県横浜市）
  • Year and Date: 2016-10-15
  • Invited
• [Presentation] Cord blood and unrelated bone marrow transplant outcomes for adult aplastic anemia: JSHCT WG study (2015)
  • Author(s): 鍬塚八千代、山﨑宏人、諫田淳也、木村文彦、賀古真一、森毅彦、内田直之、大橋一輝、小澤幸泰、高橋義行、加藤千明、岩戸康治、加藤剛二、森慎一郎、熱田由子、宮村耕一、神田善伸
  • Organizer: 第77回日本血液学会学術集会
  • Place of Presentation: ホテル金沢（石川県金沢市）
  • Year and Date: 2015-10-16