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Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
06:39

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome

Published on: October 3, 2018

Myelodysplastic syndromes in children: where are we today?

A T K Rau1, A K Shreedhara, S Kumar

  • 1Department of Pediatrics and Department of Pathology, M. S. Ramaiah Medical College, Bangalore, India.

Ochsner Journal
|October 11, 2012
PubMed

Insights

Pediatric myelodysplastic syndromes (MDSs) and myeloproliferative disorders (MPDs) are rare. Diagnosis involves genetic alterations and biological features, with stem cell transplant as the primary treatment.

Area of Science:

  • Hematology
  • Pediatric Oncology

Background:

  • Myelodysplastic syndromes (MDSs) and myeloproliferative disorders (MPDs) are uncommon in pediatric populations.
  • Current classifications delineate MPDs into MDS, juvenile myelomonocytic leukemia, and Down syndrome-associated myeloid leukemia.

Purpose of the Study:

  • To highlight the diagnostic complexities of pediatric MDSs and MPDs.
  • To emphasize the importance of biological features and genetic alterations beyond blast counts.
  • To review current and emerging therapeutic strategies.

Main Methods:

  • Review of current classification systems for MPDs.
  • Discussion of diagnostic criteria, including blast counts and genetic markers.
  • Overview of established and investigational treatments for pediatric MDSs/MPDs.

Main Results:

  • Blast count is a key but not definitive factor in differentiating leukemias from MDSs.
  • Genetic alterations are frequently identified in MDSs, aiding in diagnosis.
  • Hematopoietic stem cell transplant remains the primary curative option.

Conclusions:

  • Accurate diagnosis of pediatric MDSs/MPDs requires integrating clinical, biological, and genetic information.
  • Novel therapeutic agents show promise, particularly for adult populations, with ongoing trials.
  • Further research is needed to optimize treatment for children with these rare myeloid malignancies.
Keywords:
Bone marrow transplantcytogeneticsdysplasiamyelodysplastic syndrome

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