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December 8, 2025Blood

Is hydroxyurea treatment associated with an increased risk of malignancy in sickle cell disease? a propensity-matched analysis of 27,854 patients in the real world

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Authors

SKSyeda Ashna Fatima KamalNCNoemy Coreas

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Overview

Retrospective cohort study reveals hydroxyurea raises risks of hematological and solid malignancies in sickle cell disease, suggesting careful monitoring is needed.

Key Points

  • This study explores the relationship between hydroxyurea treatment and malignancy risk in sickle cell disease patients.
  • Conducted a retrospective cohort study using TriNetX research network data
  • Compared patients with sickle cell disease treated with hydroxyurea to matched controls
  • Utilized propensity score matching to balance demographic and clinical characteristics
  • Analyzed time-to-event outcomes with Kaplan–Meier and Cox proportional hazards models
  • Performed subgroup analyses based on age categories and transfusion status.
  • Hydroxyurea treated patients had significantly higher rates of hematological (HR 2.16) and solid tumors (HR 2.29) compared to controls
  • Patients aged 18–30 on hydroxyurea showed the highest risks for both types of malignancies (HR 4.01 for hematological, HR 4.21 for solid tumors)
  • Transfusion history correlated with a 3.7-fold increase in hematological malignancies and doubled the risk of solid tumors
  • Higher rates of mortality (1.8% vs. 2.6%) and hospitalizations (9.6% vs. 21.2%) were observed in hydroxyurea patients
  • Common malignancies included myelodysplastic syndrome and skin cancers.

Cite This Study

Kamal et al. (2025) studied this question.

synapsesocial.com/papers/69362f7d4fa91c937236e495https://doi.org/10.1182/blood-2025-1178
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Real-world effectiveness and safety of hydroxyurea in adults with hemoglobin SC disease: A retrospective cohort study in Quebec, Canada2025
  2. 2Hydroxyurea (hydroxycarbamide) use in adults with haemoglobin <scp>SC</scp> disease: A real‐world study in Quebec2026
  3. 3A65-05 Pulmonary Hypertension and Mortality in Adults With Sickle Cell Disease: Impact of Hydroxyurea Therapy vs. Chronic Transfusions2026
  4. 4Multiple Skin Cancers Associated With Hydroxyurea: Case Report and Review of Literature2025
  5. 5Association of hydroxyurea use and sickle cell retinopathy: A systematic review and meta-analysis2026