Cytarabine Monotherapy as Bridging Treatment for Hematopoietic Stem Cell Transplantation in Children with Juvenile Myelomonocytic Leukemia
- Author:
Woo Suck SUH
1
;
Mun Sung CHO
;
Jae Wook LEE
;
Pil Sang JANG
;
Nack Gyun CHUNG
;
Dae Chul JEONG
;
Bin CHO
;
Hack Ki KIM
Author Information
1. Department of Pediatrics, College of Medicine, The Catholic University of Korea, Seoul, Korea. cngped@catholic.ac.kr
- Publication Type:Original Article
- Keywords:
Juvenile myelomonocytic leukemia;
Cytarabine;
RAS pathway;
Mutation;
Hematopoietic stem cell transplantation
- MeSH:
Bone Marrow Cells;
Child;
Cohort Studies;
Cytarabine;
Fetal Hemoglobin;
Follow-Up Studies;
Hematopoietic Stem Cell Transplantation;
Hematopoietic Stem Cells;
Humans;
Leukemia;
Leukemia, Myelomonocytic, Juvenile;
Leukocyte Count;
Medical Records;
Monocytes;
Neurofibromatoses;
Retrospective Studies
- From:Clinical Pediatric Hematology-Oncology
2012;19(2):86-91
- CountryRepublic of Korea
- Language:Korean
-
Abstract:
BACKGROUND: Mutations leading to hyperactivation of the RAS pathway play a critical role in the pathogenesis of juvenile myelomonocytic leukemia (JMML). Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative therapy, and the role of anti-leukemic treatment prior to HSCT is still controversial. In this study, we analyzed the response of cytarabine monotherapy as a bridging therapy for HSCT in children recently diagnosed with JMML.METHODS: We retrospectively reviewed the medical records of patients with JMML at Seoul St. Mary's Hospital from December 2009 to April 2012.RESULTS: A total 7 patients with JMML were diagnosed and treated with chemotherapy and HSCT. At presentation, all patients showed hepatosplenomegaly and the median leukocyte count was 41.9x109/L (range, 34.3-85.0), median monocyte count was 5.6x109/L (range, 2.7-26.3) and median fetal hemoglobin (HbF) was 13.5% (range, 2.8-42.7). Karyotypic abnormalities in bone marrow cells were noted in 2 cases. Three patients had mutation of NRAS and 2 patients had mutation of NF1. One of the patients with NF1 mutations had characteristic clinical features and familial history of neurofibromatosis. All patients were treated with non-intensive sequential cytarabine chemotherapy (70 mg/m2/day, I.V., 4-12 days) before HSCT and achieved complete hematologic response. All patients underwent unrelated (N=2) or familial mismatched (N=5) HSCT, and all patients successfully engrafted. All patients, except one who relapsed, are alive with leukemia free, although the duration of follow-up is short.CONCLUSION: In our cohort of NRAS prevalent patients, non-intensive cytarabine monotherapy was effective as pre-transplant bridging treatment for JMML.
