Response to STI571 in chronic myelomonocytic leukemia with platelet derived growth factor beta receptor involvement:

V Pitini1, C Arrigo, D Teti

  • 1Oncologia Medica e Trapianto di Midollo Osseo, Pad. H 5 piano, Policlinico Universitario, Via Consolare Valeria, 98125, Messina, Italia.pitini@ciaoweb.it

Haematologica
|May 15, 2003
PubMed

Insights

STI571 effectively treated a patient with myeloproliferative disorders/myelodysplastic syndromes (MPD/MDS) and eosinophilia. This condition involved a specific chromosomal translocation, suggesting potential for STI571 in similar MPD/MDS cases.

Area of Science:

  • Oncology
  • Hematology
  • Molecular Biology

Background:

  • STI571 inhibits tyrosine kinases including ABL, c-kit, and Platelet Derived Growth Factor Receptor (PDGFBR).
  • Myeloproliferative disorders/myelodysplastic syndromes (MPD/MDS) can involve chromosomal translocations, such as t(5;12)(q33;p13).
  • This translocation activates the PDGFBR gene, encoding a receptor tyrosine kinase implicated in certain MPD/MDS subtypes.

Observation:

  • A patient diagnosed with MPD/MDS and eosinophilia presented with a t(5;12)(q33;p13) translocation.
  • The patient carried the specific chromosomal abnormality involving the PDGFBR gene.

Findings:

  • Treatment with STI571 at 400 mg daily resulted in a complete remission for the patient.
  • The patient maintained complete remission with an excellent performance status at the time of reporting.

Implications:

  • This case highlights the potential efficacy of STI571 in treating MPD/MDS associated with PDGFBR gene rearrangements.
  • Further clinical studies are warranted to confirm these promising results in a larger cohort of patients with similar genetic profiles.
  • Targeted therapy with STI571 may offer a new treatment avenue for specific MPD/MDS subgroups.

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