TY - JOUR
T1 - Single-arm non-blinded multicenter clinical trial on t-cell-replete haploidentical stem cell transplantation using low-dose antithymocyte globulin for relapsed and refractory pediatric acute leukemia
AU - Kada, Akiko
AU - Kikuta, Atsushi
AU - Saito, Akiko M.
AU - Kato, Koji
AU - Iguchi, Akihiro
AU - Yabe, Hiromasa
AU - Ishida, Hiroyuki
AU - Hyakuna, Nobuyuki
AU - Takahashi, Yoshiyuki
AU - Nagasawa, Masayuki
AU - Hashii, Yoshiko
AU - Umeda, Katsutsugu
AU - Matsumoto, Kimikazu
AU - Fujisaki, Hiroyuki
AU - Yano, Michihiro
AU - Nakazawa, Yozo
AU - Sano, Hideki
N1 - Funding Information:
FUNDING: This trial is supported by research grants from the Japan Agency for Medical Research and Development (Innovative Cancer Medicine Practical Research Program) “Multicenter non-blind single-arm clinical trial for T cell replete haploidentical stem cell transplant using low-dose antithymocyte globulin for pediatric relapsed and refractory leukemia” (17ck0106378h0001); the Japanese Society of Hematology Research Grant program “The effect of the maturation stage of T cells in graft on outcomes after pediatric haplo transplantation”; the Young Researchers program (Japan Society for the Promotion of Science) “Identification of novel biomarkers for engraftment syndrome after non-T-cell-depleted HLA semi-matching transplantation” (17K16274); the Fukushima prefecture nuclear disaster recovery fund subsidy; the Kaneka Corporation, based on contracts for physician-initiated clinical trials; and Grants-in-aid for Scientific Research-KAKENHI (C) (15K00067).
Funding Information:
This trial is supported by research grants from the Japan Agency for Medical Research and Development (Innovative Cancer Medicine Practical Research Program) ?Multicenter non-blind single-arm clinical trial for T cell replete haploidentical stem cell transplant using low-dose antithymocyte globulin for pediatric relapsed and refractory leukemia? (17ck0106378h0001); the Japanese Society of Hematology Research Grant program ?The effect of the matu-ration stage of T cells in graft on outcomes after pediatric haplo transplantation?; the Young Researchers program (Japan Society for the Promotion of Science) ?Identification of novel biomarkers for engraftment syndrome after non-T-cell-depleted HLA semi-matching transplantation? (17K16274); the Fukushima prefecture nuclear disaster recovery fund subsidy; the Kaneka Corporation, based on contracts for physician-initi-ated clinical trials; and Grants-in-aid for Scientific Research-KAKENHI (C) (15K00067).
Publisher Copyright:
© 2019, Kurume University School of Medicine. All rights reserved.
PY - 2019
Y1 - 2019
N2 - Although approximately 70% of pediatric hematological malignancies are curable, approximately 30% remain fatal. No standard treatment is available in patients showing relapse and those with refractory disease. Although different methods are adopted in different hospitals, its efficacy is extremely limited. In recent years, haploidentical stem cell transplantation, involving high-dose cyclophosphamide administration post-transplanta-tion, has been used, mainly in adults; however, its application is limited to removal of alloreactive T cells. Multicenter single-arm clinical trials of T-cell replete haploidentical stem cell transplantation (TCR-haplo-SCT) will be conducted in children with relapsed and refractory acute leukemia. After myeloablative conditioning using total body irradiation or busulfan, intensive graft versus host disease prophylaxis is administered, consisting of low-dose rabbit anti-human thymocyte globulin, tacrolimus, methotrexate, and prednisolone. An external control group is set up for the study. The treatment period is around 3 months, and the follow-up period is 2 years from transplantation completion. The aim of this study is to verify the efficacy and safety of TCR-haplo-SCT and present it as a new immune cell therapy for improving survival rate in children with relapsed and refractory acute leukemia.
AB - Although approximately 70% of pediatric hematological malignancies are curable, approximately 30% remain fatal. No standard treatment is available in patients showing relapse and those with refractory disease. Although different methods are adopted in different hospitals, its efficacy is extremely limited. In recent years, haploidentical stem cell transplantation, involving high-dose cyclophosphamide administration post-transplanta-tion, has been used, mainly in adults; however, its application is limited to removal of alloreactive T cells. Multicenter single-arm clinical trials of T-cell replete haploidentical stem cell transplantation (TCR-haplo-SCT) will be conducted in children with relapsed and refractory acute leukemia. After myeloablative conditioning using total body irradiation or busulfan, intensive graft versus host disease prophylaxis is administered, consisting of low-dose rabbit anti-human thymocyte globulin, tacrolimus, methotrexate, and prednisolone. An external control group is set up for the study. The treatment period is around 3 months, and the follow-up period is 2 years from transplantation completion. The aim of this study is to verify the efficacy and safety of TCR-haplo-SCT and present it as a new immune cell therapy for improving survival rate in children with relapsed and refractory acute leukemia.
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U2 - 10.2739/kurumemedj.MS663004
DO - 10.2739/kurumemedj.MS663004
M3 - Article
C2 - 34421094
AN - SCOPUS:85117142029
VL - 66
SP - 161
EP - 168
JO - Kurume Medical Journal
JF - Kurume Medical Journal
SN - 0023-5679
IS - 3
ER -