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Anita Frisanco Oliveira1,2, Aline Tansini1,3, Thais Toledo1,3

  • 1Barretos Children´s Cancer Hospital, Barretos, Brazil.

British Journal of Haematology
|February 21, 2022
PubMed
Summary

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This summary is machine-generated.

5-Azacitidine treatment in juvenile myelomonocytic leukaemia (JMML) reduces myeloid progenitors and monocytes while increasing T lymphocytes, correlating with clinical response. Phenotypic aberrancies persisted, indicating a worse treatment outcome.

Area of Science:

  • Hematology
  • Pediatric Oncology
  • Immunology

Background:

  • Juvenile myelomonocytic leukaemia (JMML) is a rare pediatric malignancy.
  • 5-Azacitidine is used in JMML patients before stem cell transplantation.
  • Previous work characterized JMML immunophenotypic features at diagnosis.

Purpose of the Study:

  • To investigate immunophenotypic changes during 5-azacitidine treatment in JMML.
  • To correlate these immunophenotypic changes with clinical response.
  • To assess the feasibility of immunophenotyping in this context.

Main Methods:

  • Longitudinal immunophenotypic analysis of JMML patients.
  • Evaluation at diagnosis, and after 3 and 6 cycles of 5-azacitidine.
  • Correlation of immunophenotypic data with clinical response.
Keywords:
azacytidineimmunophenotypingjuvenile myelomonocytic leukaemiatreatment

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Main Results:

  • 5-Azacitidine treatment led to decreased CD34/CD117+ cells, B-cell progenitors, and monocytes.
  • Complete clinical response correlated with increased classical monocytes and T lymphocytes.
  • Persistent immunophenotypic aberrancies (e.g., CD7 in myeloid progenitors) were associated with poorer response and NF1 presence.

Conclusions:

  • Clinical response to 5-azacitidine in JMML is linked to reduced myeloid progenitors/monocytes and increased T lymphocytes.
  • Immunophenotyping is a feasible method for monitoring treatment.
  • Persistent aberrant phenotypes suggest potential for treatment resistance.