Retrospective analysis1/1/2023 ![]() Allogeneic hematopoietic stem cell transplantation for 27 children with juvenile myelomonocytic leukemia diagnosed based on the criteria of the International JMML Working Group. Manabe A, Okamura J, Yumura-Yagi K, Akiyama Y, Sako M, Uchiyama H, et al. Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial. Locatelli F, Nöllke P, Zecca M, Korthof E, Lanino E, Peters C, et al. Chronic myelomonocytic leukemia in childhood: a retrospective analysis of 110 cases. ![]() Niemeyer CM, Arico M, Basso G, Biondi A, Rajnoldi AC, Creutzig U, et al. Myelodysplasia and myeloproliferative disorders in childhood: an update. Future prospective studies should be conducted to develop combination therapies with AZA and other molecular targeted drugs for high-risk patients.Īricò M, Biondi A, Pui C. 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. The only serious non-hematological adverse event was hyperbilirubinemia (grades III–IV) observed in a patient who received AZA after a second HSCT. Therapeutic toxicity with AZA was mostly limited to hematological toxicity. All five patients tolerated AZA treatment, including four patients who received AZA after HSCT. All five patients were boys and their ages at the time of treatment were 21, 23, 24, 26, and 46 months, respectively. ![]() We retrospectively analyzed 5 patients who received a total of 12 cycles (median 2 cycles) of AZA treatment in Japan. Retrospective analysis series#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). Juvenile myelomonocytic leukemia (JMML) is a pediatric hematological malignancy with a poor prognosis. ![]()
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