A nationwide survey of late effects in survivors of juvenile myelomonocytic leukemia in Japan

Shuichi Ozono; Kazuo Sakashita; Nao Yoshida; Harumi Kakuda; Kenichiro Watanabe; Miho Maeda; Yasushi Ishida; Atsushi Manabe; Takashi Taga; Hideki Muramatsu

doi:10.1002/pbc.30126

A nationwide survey of late effects in survivors of juvenile myelomonocytic leukemia in Japan

Pediatr Blood Cancer. 2023 Feb;70(2):e30126. doi: 10.1002/pbc.30126. Epub 2022 Dec 10.

Authors

Shuichi Ozono^{1

2}, Kazuo Sakashita^{2

3}, Nao Yoshida^{2

4}, Harumi Kakuda^{2

5}, Kenichiro Watanabe^{2

6}, Miho Maeda^{2

7}, Yasushi Ishida^{2

8}, Atsushi Manabe^{2

9}, Takashi Taga^{2

10}, Hideki Muramatsu^{2

11}

Affiliations

¹ Department of Pediatrics and Child Health, Kurume University School of Medicine, Kurume, Japan.
² Japan Children's Cancer Group (JCCG), Tokyo, Japan.
³ Department of Hemato-Oncology, Nagano Prefectural Children's Hospital, Nagano, Japan.
⁴ Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
⁵ Department of Hemato-Oncology, Chiba Prefectural Children's Hospital, Chiba, Japan.
⁶ Department of Hemato-Oncology, Shizuoka Prefectural Children's Hospital, Shizuoka, Japan.
⁷ Department of Pediatrics, Nippon Medical School, Tokyo, Japan.
⁸ Department of Pediatrics, Ehime Prefectural Central Hospital, Ehime, Japan.
⁹ Department of Pediatrics, Graduate School of Medicine, Hokkaido University, Hokkaido, Japan.
¹⁰ Department of Pediatrics, Shiga Medical College, School of Medicine, Shiga, Japan.
¹¹ Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan.

PMID: 36495260
DOI: 10.1002/pbc.30126

Abstract

We conducted a cross-sectional study using a questionnaire to explore the late effects in survivors of allogenic hematopoietic stem cell transplantation (HSCT) for juvenile myelomonocytic leukemia (JMML). The attending pediatric hematologists/oncologists completed the questionnaires. Of the 30 survivors, approximately 83% showed more than one late effect. The identified late effects included endocrine, dental, skin, ophthalmologic, musculoskeletal, pulmonary, neurocognitive, and cardiovascular dysfunction. The prevalence of short stature, pulmonary, cardiovascular, and nephrological complications was significantly elevated among survivors who were 12 years or more lapsed after HSCT. Therefore, a multidisciplinary follow-up system for survivors of JMML is crucial.

Keywords: myeloablative regimen; short stature; stem cell transplantation.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Child
Cross-Sectional Studies
Disease Progression
Hematopoietic Stem Cell Transplantation* / adverse effects
Humans
Japan / epidemiology
Leukemia, Myelomonocytic, Juvenile* / epidemiology
Leukemia, Myelomonocytic, Juvenile* / therapy
Survivors