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Impact of treatment on thrombotic complications in AYA with ET and PV: A retrospective analysis

By Sheetal Bhurke

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Apr 28, 2025

Learning objective: After reading this article, learners will be able to cite a new clinical development in myeloproliferative neoplasms.


 

ET and PV are linked with thrombotic complications in older patients. However, data are limited in AYA patients. A retrospective analysis evaluating the impact of treatment on thrombotic risk and secondary MF progression in AYA with ET and PV was published by Beauverd et al. in Leukemia.1

The cohort included 278 AYA patients with ET and 70 patients with PV. The primary outcomes were TFS and MFS. Secondary outcomes included risk factors associated with thrombotic events and secondary MF.

 

Key learnings

The estimated 10- and 20-year TFS rates were 86.8% and 78.8% for the entire cohort, 86.9% and 80.0% for the ET cohort, and 84.4% and 76.3% for the PV cohort, respectively. 

The 10- and 20-year MFS rates were 95.2% and 81.3% for the entire cohort, 94.7% and 75.7% for the ET cohort, and 96.8% and 93.3% for the PV cohort, respectively.

CALR mutation (HR, 6.0; 95% CI, 2.3–16.1; p < 0.001) and thrombosis history (HR, 3.8; 95% CI, 1.3–11.4; p = 0.015) at diagnosis were associated with increased risk of secondary MF progression.

Elevated WBC count (HR, 2.7; 95% CI, 1.2–6.0; p = 0.012) and absence of splenomegaly at diagnosis (HR, 5.7; 95% CI, 1.2–26.0; p = 0.026) were associated with increased risk of thrombosis.

Treatment with IFN was associated with a lower risk of secondary MF progression compared with HU, ANA, or the absence of cytoreduction (p = 0.046).

The analysis suggests that AYA patients have similar rates of thrombosis and secondary MF to those of older patients. AYA patients treated with IFN showed no disease progression, supporting its potential as a first-line treatment in this younger population.  

Abbreviations: ANA, anagrelide; AYA, adolescent and young adult; CALR, calreticulin mutation; CI, confidence interval; ET, essential thrombocythemia; HR, hazard ratio; HU, hydroxycarbamide; IFN, interferon; MF, myelofibrosis; MFS, myelofibrosis-free survival; PV, polycythemia vera; TFS, thrombosis-free survival; WBC, white blood cell. 

References

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