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Hematopoietic stem cell transplantation for pediatric acute promyelocytic leukemia in Japan
Author(s) -
Yamamoto Shohei,
Tomizawa Daisuke,
Kudo Kazuko,
Hasegawa Daiichiro,
Taga Takashi,
Yanada Masamitsu,
Kondo Tadakazu,
Nakazawa Yozo,
Eto Tetsuya,
Inoue Masami,
Kato Koji,
Atsuta Yoshiko,
Ishida Hiroyuki
Publication year - 2020
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28181
Subject(s) - medicine , hematopoietic stem cell transplantation , acute promyelocytic leukemia , cumulative incidence , incidence (geometry) , transplantation , complete remission , sibling , pediatrics , surgery , gastroenterology , oncology , chemotherapy , retinoic acid , biochemistry , chemistry , physics , optics , gene , psychology , developmental psychology
Background The number of hematopoietic stem cell transplantation (HSCT) procedures performed for pediatric acute promyelocytic leukemia (APL) has decreased in the all‐trans retinoic acid (ATRA) era. Although HSCT is still widely adopted as part of salvage therapy for relapsed patients, there is no general consensus about the optimal transplant type (autologous [auto‐HSCT] or allogeneic HSCT [allo‐HSCT]). Procedures We retrospectively reviewed the clinical data of 95 childhood APL patients who underwent their first HSCT between 1990 and 2014. Of the 95 patients, 40 (42%), 41 (43%), and 3 (3%) underwent HSCT procedures after achieving their first complete remission (CR1), CR2, and CR3, respectively, and 11 (12%) underwent HSCT while in a non‐CR state. Results The non‐CR group exhibited significantly worse five‐year overall survival (5yOS) and disease‐free survival (5yDFS) (5yOS: 46%; 5yDFS: 46%) than the CR1 (5yOS: 80%; 5yDFS: 78%) and CR2 + CR3 groups (5yOS: 81%; 5yDFS: 76%) ( P = 0.013 and P < 0.01, respectively). Of the patients treated in CR2, no significant differences in 5yOS or the five‐year cumulative incidence of relapse (5yRI) were detected between the auto‐HSCT and allo‐HSCT groups (5yOS: 85%, vs 78%, P = 0.648; 5yRI: 9%, vs 11%, P = 0.828). Among the patients who underwent allo‐HSCT in CR2, those with matched sibling donors displayed a significantly higher 5yRI (33%) than those with other types of donors (0%, P = 0.015). Conclusions Even after relapsing, childhood APL can be cured with HSCT if CR is achieved. These findings demonstrate that achieving CR followed by HSCT is the preferred strategy for treating children with relapsed or refractory APL.