Juvenile Myelomonocytic Leukemia and other myelodysplastic/myeloproliferative neoplasms

Authors

  • Neysimelia Costa Villela Barretos Cancer Hospital
  • Roseane Vasconcelos Gouveia Hospital Samaritano, São Paulo, SP, Brazil
  • Simone de Castro Resende Franco Hospital da Criança de Brasília José de Alencar. Brasília, DF, Brazil
  • Gustavo Zamperlini Hospital Samaritano, São Paulo, SP, Brazil
  • Patrícia Shimoda Ikeuti Instituto de Oncologia Pediátrica IOP/GRAACC/UNIFESP, São Paulo, SP, Brazil.
  • Luiz Fernando Lopes Barretos Cancer Hospital, Barretos, SP, Brazil.

DOI:

https://doi.org/10.46765/2675-374X.2021v2n4p128

Abstract

Juvenile Myelomonocytic Leukemia (JMML) is a clonal hematopoietic disorder that usually occurs in early childhood, characterized by hyperactivation of the RAS signaling pathway. About 90% of patients have mutations in 1 of 5 genes (PTPN11, NRAS, KRAS, NF1, CBL) that define genetically and clinically distinct subtypes of the disease, with a highly variable clinical course. Allogeneic hematopoietic stem cell transplantation (HSCT) remains the therapy of choice for most patients with JMML, although children with CBL mutations and few of those with N-RAS mutations may have spontaneous resolution of hematologic abnormalities. The results of HSCT in patients with JMML have progressively improved over time, but relapse is still an important cause of treatment failure.

Author Biographies

Roseane Vasconcelos Gouveia, Hospital Samaritano, São Paulo, SP, Brazil

MD.

Physician at the Pediatric Bone Marrow Transplant Unit, Hospital Samaritano, São Paulo, SP, Brazil

Simone de Castro Resende Franco, Hospital da Criança de Brasília José de Alencar. Brasília, DF, Brazil

MD.

Physician at the Pediatric Bone Marrow Transplant Unit, Hospital da Criança de Brasília José de Alencar. Brasília, DF, Brazil.

Gustavo Zamperlini, Hospital Samaritano, São Paulo, SP, Brazil

MD.

Physician at the Pediatric Bone Marrow Transplant Unit, Hospital Samaritano, São Paulo, SP, Brazil

Patrícia Shimoda Ikeuti, Instituto de Oncologia Pediátrica IOP/GRAACC/UNIFESP, São Paulo, SP, Brazil.

MD.

Physician at the Pediatric Bone Marrow Transplant Unit, Instituto de Oncologia Pediátrica IOP/GRAACC/UNIFESP, São Paulo, SP, Brazil.

Luiz Fernando Lopes, Barretos Cancer Hospital, Barretos, SP, Brazil.

MD, PhD.

Chairman of the Brazilian Pediatric Myelodysplastic Syndrome Study Group.

Director of Barretos Chindren´s Cancer Center

Downloads

Published

11/30/2021

How to Cite

Costa Villela, N., Vasconcelos Gouveia, R. ., de Castro Resende Franco, S. ., Zamperlini, G. ., Shimoda Ikeuti, P. ., & Lopes, L. F. . (2021). Juvenile Myelomonocytic Leukemia and other myelodysplastic/myeloproliferative neoplasms. JOURNAL OF BONE MARROW TRANSPLANTATION AND CELLULAR THERAPY, 2(4), 128. https://doi.org/10.46765/2675-374X.2021v2n4p128

Most read articles by the same author(s)