[Advances in the treatment of juvenile myelomonocytic leukemia].

Zhongguo Dang Dai Er Ke Za Zhi

Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin 300020, China.

Published: November 2018

AI Article Synopsis

  • Juvenile myelomonocytic leukemia (JMML) is a rare, aggressive type of cancer in children that combines elements of myelodysplastic syndrome and myeloproliferative neoplasm.
  • Traditional chemotherapy is largely ineffective for treating JMML, making hematopoietic stem cell transplantation the only viable cure currently available.
  • Recent advancements in targeted therapies, particularly focusing on mutations in the Ras signaling pathway and demethylation treatment for certain genetic abnormalities, have shown promise in improving treatment outcomes.

Article Abstract

Juvenile myelomonocytic leukemia (JMML) is a rare chronic myeloid leukemia in children and has the features of both myelodysplastic syndrome and myeloproliferative neoplasm. It is highly malignant and has a poor treatment outcome. Children with JMML have a poor response to conventional chemotherapy. At present, hematopoietic stem cell transplantation is the only possible cure for this disease. In recent years, significant progress has been made in targeted therapy for mutant genes in the Ras signaling pathway and demethylation treatment of aberrant methylation of polygenic CpG islands. This article reviews the treatment and efficacy evaluation of JMML.

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7389026PMC
http://dx.doi.org/10.7499/j.issn.1008-8830.2018.11.016DOI Listing

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