Can hydroxyurea cause thrombocytopenia?

Medical Advisory BoardAll articles are reviewed for accuracy by our Medical Advisory Board
Educational purpose only • Exercise caution as content is pending human review
Article Review Status
Submitted
Under Review
Approved

Last updated: October 25, 2025View editorial policy

Personalize

Help us tailor your experience

Which best describes you? Your choice helps us use language that's most understandable for you.

Hydroxyurea and Thrombocytopenia

Yes, hydroxyurea can cause thrombocytopenia as a known side effect, particularly when used at doses required for clinical response in myeloproliferative disorders. 1

Mechanism and Evidence

  • Hydroxyurea is a cytoreductive agent that causes bone marrow suppression, which can affect all cell lines including platelets 2
  • The European LeukemiaNet and NCCN guidelines specifically identify thrombocytopenia as a recognized adverse effect of hydroxyurea therapy 1
  • Platelet counts <100 x 10^9/L at the lowest dose of hydroxyurea required to achieve clinical response is considered a criterion for hydroxyurea intolerance in polycythemia vera 1
  • Bone marrow depression is more likely in patients who have previously received radiotherapy or cytotoxic cancer chemotherapeutic agents 2

Clinical Significance

  • Thrombocytopenia is less common than leukopenia, which is generally the first and most common manifestation of hydroxyurea-induced myelosuppression 2
  • In patients with myeloproliferative disorders, the development of thrombocytopenia (platelet count <100 x 10^9/L) is considered a criterion for intolerance to hydroxyurea and may necessitate dose reduction or discontinuation 1
  • Recovery from hydroxyurea-induced myelosuppression, including thrombocytopenia, is usually rapid when therapy is interrupted 2

Monitoring Recommendations

  • Complete blood counts should be monitored at least weekly during initial hydroxyurea therapy and every 4-8 weeks once stabilized 3, 2
  • Evaluate hematologic status prior to and during treatment with hydroxyurea 2
  • Bone marrow function should not be markedly depressed before initiating hydroxyurea therapy 2

Management of Hydroxyurea-Induced Thrombocytopenia

  • If platelet count falls below 100 x 10^9/L, consider dose reduction or temporary discontinuation of hydroxyurea 1
  • Provide supportive care as needed for significant thrombocytopenia 2
  • For patients with persistent thrombocytopenia despite dose adjustments, consider alternative cytoreductive agents:
    • For polycythemia vera: interferon-alpha is the recommended second-line therapy 1, 3
    • For essential thrombocythemia: anagrelide or interferon-alpha are alternatives 3, 4

Special Considerations

  • In rare cases, hydroxyurea can cause hemolytic anemia rather than myelosuppressive anemia, which may complicate the clinical picture 5
  • Patients with beta-thalassemia treated with hydroxyurea may develop transient thrombocytopenia that resolves after temporary discontinuation 6
  • The risk of hydroxyurea-induced thrombocytopenia must be balanced against its proven benefits in reducing thrombotic complications in high-risk essential thrombocythemia patients 7, 8

Remember that while hydroxyurea can cause thrombocytopenia, it is generally well-tolerated and remains a first-line cytoreductive therapy for myeloproliferative disorders when indicated 1, 3.

References

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Guideline

Treatment of Thrombocytosis with Hydroxyurea

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

Have a follow-up question?

Our Medical A.I. is used by practicing medical doctors at top research institutions around the world. Ask any follow up question and get world-class guideline-backed answers instantly.