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. 2022 Feb;115(2):263-268.
doi: 10.1007/s12185-021-03248-x. Epub 2021 Oct 29.

A retrospective analysis of azacitidine treatment for juvenile myelomonocytic leukemia

Yuko Honda  1 , Hideki Muramatsu  2 , Yuka Nanjo  3 , Shinsuke Hirabayashi  4 , Toru Meguro  5 , Nao Yoshida  6 , Harumi Kakuda  7 , Shuichi Ozono  8 , Manabu Wakamatsu  2 , Hiroshi Moritake  9 , Masahiro Yasui  10   11 , Hideki Sano  12 , Atsushi Manabe  4 , Kazuo Sakashita  13
Affiliations

A retrospective analysis of azacitidine treatment for juvenile myelomonocytic leukemia

Yuko Honda et al. Int J Hematol. 2022 Feb.

Abstract

Juvenile myelomonocytic leukemia (JMML) is a pediatric hematological malignancy with a poor prognosis. Although several case series have been published describing hematological and molecular responses to azacitidine (AZA) treatment in patients with JMML, the efficacy and safety profile of AZA is not well investigated, especially in Asian children and children undergoing hematopoietic stem cell transplantation (HSCT). We retrospectively analyzed 5 patients who received a total of 12 cycles (median 2 cycles) of AZA treatment in Japan. All five patients were boys and their ages at the time of treatment were 21, 23, 24, 26, and 46 months, respectively. All five patients tolerated AZA treatment, including four patients who received AZA after HSCT. Therapeutic toxicity with AZA was mostly limited to hematological toxicity. The only serious non-hematological adverse event was hyperbilirubinemia (grades III-IV) observed in a patient who received AZA after a second HSCT. Two out of five patients treated with AZA achieved a partial response (PR), while three patients treated for post-transplant relapse did not have an objective response. Future prospective studies should be conducted to develop combination therapies with AZA and other molecular targeted drugs for high-risk patients.

Keywords: Azacitidine; DNA hypomethylating agents; Hematopoietic stem cell transplantation; Juvenile myelomonocytic leukemia.

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