Successful second allogeneic hematopoietic stem cell transplantation with azacitidine as bridging therapy for relapsed juvenile myelomonocytic leukemia

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  • OSHIRO Tokiko
    Department of Pediatrics, University of the Ryukyus Hospital
  • HYAKUNA Nobuyuki
    Japanese Red Cross Okinawa Blood Center
  • ABE Hitomi
    Department of Pediatrics, University of the Ryukyus Hospital
  • HAMADA Satoru
    Department of Pediatrics, University of the Ryukyus Hospital
  • NAKANISHI Koichi
    Department of Pediatrics, University of the Ryukyus Hospital Department of Child Health and Welfare, Graduate School of Medicine, University of the Ryukyus

Bibliographic Information

Other Title
  • Azacitidineによる橋渡しで2回の同種造血幹細胞移植を行った再発若年性骨髄単球性白血病
  • Azacitidine ニ ヨル ハシワタシ デ 2カイ ノ ドウシュ ゾウケツ カンサイボウ イショク オ オコナッタ サイハツ ジャクネンセイ コツズイタンキュウセイ ハッケツビョウ

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Description

<p>Hematopoietic cell transplantation (HCT) is the only curative therapy for juvenile myelomonocytic leukemia (JMML). Meanwhile, an established conventional chemotherapy before HCT remains unavailable. Studies have shown that azacitidine (AZA), which is a DNA methyltransferase inhibitor, is clinically effective for JMML as a bridging therapy for HCT; a prospective clinical trial in Japan is ongoing. Herein, we present a case of a patient with JMML who was administered AZA as bridging therapy for both first and second HCT. A 3-year-old boy with neurofibromatosis type 1 was administered with intravenous AZA (75 mg/m2/day for 7 days, intervals of 28 days, and four cycles) and received myeloablative HCT (unrelated bone marrow). When relapse occurred on day 123, four additional AZA therapy cycles were administered, and the patient received a second nonmyeloablative HCT (cord blood). After seven AZA therapy cycles as post HCT consolidation, hematological remission was sustained for 16 months after the second HCT. No severe adverse events occurred. AZA is effective for JMML as a bridging therapy for HCT and has robust cytoreductive potential despite the risk of relapse.</p>

Journal

  • Rinsho Ketsueki

    Rinsho Ketsueki 64 (3), 187-192, 2023

    The Japanese Society of Hematology

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