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Comparison of conditioning regimens for autologous stem cell transplantation in children with acute myeloid leukemia: A nationwide retrospective study in Japan.
Pediatr Blood Cancer. 2019 01; 66(1):e27459.PB

Abstract

BACKGROUND

Indications for hematopoietic stem cell transplantation (HSCT) have decreased with the improvement in chemotherapy for pediatric acute myeloid leukemia (AML) in the last decade. We conducted reevaluation of autologous HSCT (AHSCT) to compare myeloablative conditioning (MAC) regimens for pediatric AML without the need for consideration of toxicities caused by allogeneic immune reactions.

PROCEDURE

This retrospective study analyzed the clinical outcomes of 220 children with AML who underwent consecutive AHSCT between 1989 and 2002 in Japan by the national prospective registry. The transplantation outcomes of various conditioning regimens were compared.

RESULTS

The median follow-up period of the survivors was 160 months. The clinical outcomes of busulfan + cyclophosphamide ± etoposide or busulfan + melphalan regimens were significantly superior compared with other busulfan-based and total body irradiation-based regimens (leukemia-free survival [LFS]: 68% vs 42% and 55%, P = 0.001; overall survival [OS]: 74% vs 49% and 61%, P < 0.001). Multivariate analysis showed that busulfan + cyclophosphamide ± etoposide and busulfan + melphalan regimens were independent favorable factors for LFS (hazard ratio: 0.46; P < 0.001) and OS (hazard ratio: 0.40; P < 0.001) compared with the other busulfan-based regimen, and both age 2 years or older and advanced stage at AHSCT were independent poor predictors for LFS and OS, simultaneously.

CONCLUSION

Busulfan + cyclophosphamide ± etoposide and busulfan + melphalan regimens exhibited superior antileukemic effects compared with other BU-based myeloablative regimens.

Authors+Show Affiliations

Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan.Department of Hematology and Oncology, Kobe Children's Hospital, Kobe, Japan.Department of Pediatrics, Fujita Health University School of Medicine, Aichi, Japan.Department of Pediatrics, Kyoto City Hospital, Kyoto, Japan.Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan.Department of Pediatrics, National Kyushu Cancer Center, Fukuoka, Japan.Department of Pediatrics, Yokohama City University Hospital, Yokohama, Japan.Department of Pediatrics, Osaka City University Graduate School of Medicine, Osaka, Japan.Department of Hematopoietic Stem Cell Transplantation, National Cancer Center Hospital, Tokyo, Japan.Division of Hematology/Oncology, Kanagawa Children's Medical Center, Yokohama, Japan.Department of Pediatrics, Osaka University Graduate School of Medicine, Suita, Japan.Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.Department of Pediatrics, Osaka University Graduate School of Medicine, Suita, Japan.Department of Hematology/Oncology, Osaka Women's and Children's Hospital, Izumi, Japan.Japanese Data Center for Hematopoietic Cell Transplantation, Nagoya, Japan. Department of Healthcare Administration, Nagoya University Graduate School of Medicine, Nagoya, Japan.No affiliation info available

Pub Type(s)

Comparative Study
Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

30270523

Citation

Sakaguchi, Hirotoshi, et al. "Comparison of Conditioning Regimens for Autologous Stem Cell Transplantation in Children With Acute Myeloid Leukemia: a Nationwide Retrospective Study in Japan." Pediatric Blood & Cancer, vol. 66, no. 1, 2019, pp. e27459.
Sakaguchi H, Muramatsu H, Hasegawa D, et al. Comparison of conditioning regimens for autologous stem cell transplantation in children with acute myeloid leukemia: A nationwide retrospective study in Japan. Pediatr Blood Cancer. 2019;66(1):e27459.
Sakaguchi, H., Muramatsu, H., Hasegawa, D., Kudo, K., Ishida, H., Yoshida, N., Koh, K., Noguchi, M., Shiba, N., Tokimasa, S., Fukuda, T., Goto, H., Miyamura, T., Nakazawa, Y., Hashii, Y., Inoue, M., & Atsuta, Y. (2019). Comparison of conditioning regimens for autologous stem cell transplantation in children with acute myeloid leukemia: A nationwide retrospective study in Japan. Pediatric Blood & Cancer, 66(1), e27459. https://doi.org/10.1002/pbc.27459
Sakaguchi H, et al. Comparison of Conditioning Regimens for Autologous Stem Cell Transplantation in Children With Acute Myeloid Leukemia: a Nationwide Retrospective Study in Japan. Pediatr Blood Cancer. 2019;66(1):e27459. PubMed PMID: 30270523.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Comparison of conditioning regimens for autologous stem cell transplantation in children with acute myeloid leukemia: A nationwide retrospective study in Japan. AU - Sakaguchi,Hirotoshi, AU - Muramatsu,Hideki, AU - Hasegawa,Daiichiro, AU - Kudo,Kazuko, AU - Ishida,Hiroyuki, AU - Yoshida,Nao, AU - Koh,Katsuyoshi, AU - Noguchi,Maiko, AU - Shiba,Norio, AU - Tokimasa,Sadao, AU - Fukuda,Takhiro, AU - Goto,Hiroaki, AU - Miyamura,Takako, AU - Nakazawa,Yozo, AU - Hashii,Yoshiko, AU - Inoue,Masami, AU - Atsuta,Yoshiko, AU - ,, Y1 - 2018/09/30/ PY - 2018/05/13/received PY - 2018/08/01/revised PY - 2018/08/20/accepted PY - 2018/10/3/pubmed PY - 2019/10/23/medline PY - 2018/10/2/entrez KW - acute myeloid leukemia KW - autologous bone marrow transplantation KW - autologous peripheral blood stem cell transplantation KW - myeloablative conditioning regimen SP - e27459 EP - e27459 JF - Pediatric blood & cancer JO - Pediatr Blood Cancer VL - 66 IS - 1 N2 - BACKGROUND: Indications for hematopoietic stem cell transplantation (HSCT) have decreased with the improvement in chemotherapy for pediatric acute myeloid leukemia (AML) in the last decade. We conducted reevaluation of autologous HSCT (AHSCT) to compare myeloablative conditioning (MAC) regimens for pediatric AML without the need for consideration of toxicities caused by allogeneic immune reactions. PROCEDURE: This retrospective study analyzed the clinical outcomes of 220 children with AML who underwent consecutive AHSCT between 1989 and 2002 in Japan by the national prospective registry. The transplantation outcomes of various conditioning regimens were compared. RESULTS: The median follow-up period of the survivors was 160 months. The clinical outcomes of busulfan + cyclophosphamide ± etoposide or busulfan + melphalan regimens were significantly superior compared with other busulfan-based and total body irradiation-based regimens (leukemia-free survival [LFS]: 68% vs 42% and 55%, P = 0.001; overall survival [OS]: 74% vs 49% and 61%, P < 0.001). Multivariate analysis showed that busulfan + cyclophosphamide ± etoposide and busulfan + melphalan regimens were independent favorable factors for LFS (hazard ratio: 0.46; P < 0.001) and OS (hazard ratio: 0.40; P < 0.001) compared with the other busulfan-based regimen, and both age 2 years or older and advanced stage at AHSCT were independent poor predictors for LFS and OS, simultaneously. CONCLUSION: Busulfan + cyclophosphamide ± etoposide and busulfan + melphalan regimens exhibited superior antileukemic effects compared with other BU-based myeloablative regimens. SN - 1545-5017 UR - https://www.unboundmedicine.com/medline/citation/30270523/Comparison_of_conditioning_regimens_for_autologous_stem_cell_transplantation_in_children_with_acute_myeloid_leukemia:_A_nationwide_retrospective_study_in_Japan_ L2 - https://doi.org/10.1002/pbc.27459 DB - PRIME DP - Unbound Medicine ER -