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Randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without G-CSF priming: G-CONCORD study protocol

INTRODUCTION: A better long-term quality of life after umbilical cord blood transplantation (CBT) is observed compared with transplants from other alternative donors, whereas graft failure and relapses after CBT are still major issues. To minimise graft failure and relapse after CBT, intensification...

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Autores principales: Terakura, Seitaro, Konuma, Takaaki, Tanaka, Masatsugu, Ozawa, Yukiyasu, Onizuka, Makoto, Nanno, Satoshi, Onishi, Yasushi, Aotsuka, Nobuyuki, Kondo, Tadakazu, Kawakita, Toshiro, Kato, Jun, Kobayashi, Takeshi, Nishida, Tetsuya, Yamaguchi, Takuhiro, Kuwatsuka, Yachiyo, Takahashi, Satoshi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7722372/
https://www.ncbi.nlm.nih.gov/pubmed/33277285
http://dx.doi.org/10.1136/bmjopen-2020-040467
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author Terakura, Seitaro
Konuma, Takaaki
Tanaka, Masatsugu
Ozawa, Yukiyasu
Onizuka, Makoto
Nanno, Satoshi
Onishi, Yasushi
Aotsuka, Nobuyuki
Kondo, Tadakazu
Kawakita, Toshiro
Kato, Jun
Kobayashi, Takeshi
Nishida, Tetsuya
Yamaguchi, Takuhiro
Kuwatsuka, Yachiyo
Takahashi, Satoshi
author_facet Terakura, Seitaro
Konuma, Takaaki
Tanaka, Masatsugu
Ozawa, Yukiyasu
Onizuka, Makoto
Nanno, Satoshi
Onishi, Yasushi
Aotsuka, Nobuyuki
Kondo, Tadakazu
Kawakita, Toshiro
Kato, Jun
Kobayashi, Takeshi
Nishida, Tetsuya
Yamaguchi, Takuhiro
Kuwatsuka, Yachiyo
Takahashi, Satoshi
author_sort Terakura, Seitaro
collection PubMed
description INTRODUCTION: A better long-term quality of life after umbilical cord blood transplantation (CBT) is observed compared with transplants from other alternative donors, whereas graft failure and relapses after CBT are still major issues. To minimise graft failure and relapse after CBT, intensification of conditioning by the addition of high-dose cytosine arabinoside (CA) and concomitant continuous use of granulocyte-colony stimulating factor (G-CSF) are reported to convey a significantly better survival after CBT in some retrospective studies. To confirm the effect of G-CSF plus CA combination, in addition to the standard conditioning regimen, cyclophosphamide (CY)/total body irradiation (TBI), we design a randomised controlled study comparing CA/CY/TBI with versus without G-CSF priming (G-CSF combined conditioned cord blood transplantation [G-CONCORD] study). METHODS AND ANALYSIS: This is a multicentre, open-label, randomised phase III study that aimed to compare G-CSF+CA/CY/TBI as a conditioning regimen for CBT with CA/CY/TBI. Patients with acute myeloid leukaemia or myelodysplastic syndrome, aged 16–55 years, are eligible. The target sample size is 160 and the registration period is 4 years. The primary endpoint is the 2-year disease-free survival rate after CBT. The secondary endpoints are overall survival, relapse, non-relapse mortality, acute and chronic graft-versus-host disease, engraftment rate, time to neutrophil recovery, short-term adverse events, incidence of infections and causes of death. This study employs a single one-to-one web-based randomisation between the with-G-CSF versus without-G-CSF groups after patient registration. Combination of high-dose CA and CY/TBI in both groups is used for conditioning. ETHICS AND DISSEMINATION: The study protocol was approved by the central review board, Nagoya University Certified Review Board, after the enforcement of the Clinical Trials Act in Japan. The manuscripts presenting data from this study will be submitted for publication in quality peer-reviewed medical journals. Study findings will be disseminated via presentations at national/international conferences and peer-reviewed journals. TRIAL REGISTRATION NUMBERS: UMIN000029947 and jRCTs041180059.
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spelling pubmed-77223722020-12-14 Randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without G-CSF priming: G-CONCORD study protocol Terakura, Seitaro Konuma, Takaaki Tanaka, Masatsugu Ozawa, Yukiyasu Onizuka, Makoto Nanno, Satoshi Onishi, Yasushi Aotsuka, Nobuyuki Kondo, Tadakazu Kawakita, Toshiro Kato, Jun Kobayashi, Takeshi Nishida, Tetsuya Yamaguchi, Takuhiro Kuwatsuka, Yachiyo Takahashi, Satoshi BMJ Open Haematology (Incl Blood Transfusion) INTRODUCTION: A better long-term quality of life after umbilical cord blood transplantation (CBT) is observed compared with transplants from other alternative donors, whereas graft failure and relapses after CBT are still major issues. To minimise graft failure and relapse after CBT, intensification of conditioning by the addition of high-dose cytosine arabinoside (CA) and concomitant continuous use of granulocyte-colony stimulating factor (G-CSF) are reported to convey a significantly better survival after CBT in some retrospective studies. To confirm the effect of G-CSF plus CA combination, in addition to the standard conditioning regimen, cyclophosphamide (CY)/total body irradiation (TBI), we design a randomised controlled study comparing CA/CY/TBI with versus without G-CSF priming (G-CSF combined conditioned cord blood transplantation [G-CONCORD] study). METHODS AND ANALYSIS: This is a multicentre, open-label, randomised phase III study that aimed to compare G-CSF+CA/CY/TBI as a conditioning regimen for CBT with CA/CY/TBI. Patients with acute myeloid leukaemia or myelodysplastic syndrome, aged 16–55 years, are eligible. The target sample size is 160 and the registration period is 4 years. The primary endpoint is the 2-year disease-free survival rate after CBT. The secondary endpoints are overall survival, relapse, non-relapse mortality, acute and chronic graft-versus-host disease, engraftment rate, time to neutrophil recovery, short-term adverse events, incidence of infections and causes of death. This study employs a single one-to-one web-based randomisation between the with-G-CSF versus without-G-CSF groups after patient registration. Combination of high-dose CA and CY/TBI in both groups is used for conditioning. ETHICS AND DISSEMINATION: The study protocol was approved by the central review board, Nagoya University Certified Review Board, after the enforcement of the Clinical Trials Act in Japan. The manuscripts presenting data from this study will be submitted for publication in quality peer-reviewed medical journals. Study findings will be disseminated via presentations at national/international conferences and peer-reviewed journals. TRIAL REGISTRATION NUMBERS: UMIN000029947 and jRCTs041180059. BMJ Publishing Group 2020-12-04 /pmc/articles/PMC7722372/ /pubmed/33277285 http://dx.doi.org/10.1136/bmjopen-2020-040467 Text en © Author(s) (or their employer(s)) 2020. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. http://creativecommons.org/licenses/by-nc/4.0/ http://creativecommons.org/licenses/by-nc/4.0/This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.
spellingShingle Haematology (Incl Blood Transfusion)
Terakura, Seitaro
Konuma, Takaaki
Tanaka, Masatsugu
Ozawa, Yukiyasu
Onizuka, Makoto
Nanno, Satoshi
Onishi, Yasushi
Aotsuka, Nobuyuki
Kondo, Tadakazu
Kawakita, Toshiro
Kato, Jun
Kobayashi, Takeshi
Nishida, Tetsuya
Yamaguchi, Takuhiro
Kuwatsuka, Yachiyo
Takahashi, Satoshi
Randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without G-CSF priming: G-CONCORD study protocol
title Randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without G-CSF priming: G-CONCORD study protocol
title_full Randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without G-CSF priming: G-CONCORD study protocol
title_fullStr Randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without G-CSF priming: G-CONCORD study protocol
title_full_unstemmed Randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without G-CSF priming: G-CONCORD study protocol
title_short Randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without G-CSF priming: G-CONCORD study protocol
title_sort randomised controlled trial of conditioning regimen for cord blood transplantation for adult myeloid malignancies comparing high-dose cytarabine/cyclophosphamide/total body irradiation with versus without g-csf priming: g-concord study protocol
topic Haematology (Incl Blood Transfusion)
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7722372/
https://www.ncbi.nlm.nih.gov/pubmed/33277285
http://dx.doi.org/10.1136/bmjopen-2020-040467
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