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Updated: Apr 8, 2026
Intracellular Phosphoflow Cytometry of Acute Myeloid Leukemia Patient-Derived Xenotransplants
Published on: June 6, 2025
Long-term follow-up of essential thrombocythemia patients treated with anagrelide: subgroup analysis according to
María J Mela Osorio1, Luciana Ferrari1, Nora P Goette2
1Clínica Médica , Instituto de Investigaciones Médicas Alfredo Lanari, Universidad de Buenos Aires, Buenos Aires, Argentina.
Background:
Anagrelide represents a treatment option for essential thrombocythemia, although its place in therapy remains controversial.
Aim:
To assess the impact of mutational status in response rates and development of adverse events during long-term use of anagrelide.
Methods:
We retrospectively evaluated 67 patients with essential thrombocythemia treated with anagrelide during 68 (4-176) months.
Results:
Mutational frequencies were 46.3%, 28.3%, and 1.5% for JAK2V617F, CALR and MPL mutations. Anagrelide yielded a high rate of hematologic responses, which were complete in 49.25% and partial in 46.25%, without differences among molecular subsets. The rate of thrombosis during treatment was one per 100 patient-years, without excess bleeding. Anemia was the major adverse event, 30.3% at 5-yr follow-up, being more frequent in CALR(+) (P < 0.05). Myelofibrotic transformation developed in 14.9% (12.9%, 21%, and 12.5% in JAK2V617F(+), CALR(+), and triple-negative patients, respectively, P = NS) and those treated >60 months were at higher risk, OR (95% CI) 9.32 (1.1-78.5), P < 0.01, indicating the need for bone marrow monitoring during prolonged treatment.
Conclusion:
Although CALR(+) patients were at higher risk of developing anemia, anagrelide proved effective among all molecular subsets, indicating that mutational status does not seem to represent a major determinant of choice of cytoreductive treatment among essential thrombocythemia therapies.
