What are the indications, monitoring requirements, and discontinuation criteria for cyproterone acetate (antiandrogen) therapy?

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Cyproterone Acetate: Indications, Monitoring, and Discontinuation Criteria

Cyproterone acetate is indicated for prostate cancer, gender-affirming care in transgender women, and androgenic conditions such as hirsutism and acne, with monitoring required for hepatic function, adrenal suppression, and anemia, and should be discontinued if severe adverse effects occur. 1

Indications

Prostate Cancer

  • Used as a second-line hormonal therapy for androgen-sensitive metastatic prostate cancer in Europe and Canada 2, 1
  • Can reduce tumor flare when initiating LHRH agonist therapy for prostate cancer 1
  • Less effective than goserelin when used alone for delaying progression of metastatic prostate cancer 1

Gender-Affirming Care

  • Used as an antiandrogen component in hormone therapy for transgender women/transfeminine individuals 1, 2
  • Produces feminizing effects through dual mechanisms: competitive inhibition of androgen receptor binding and suppression of testosterone production 1, 3
  • Can be used alone initially or in combination with estrogens 3

Other Indications

  • Treatment of androgenic conditions in women:
    • Moderate to severe acne related to androgen sensitivity 4, 5
    • Hirsutism 6, 5
    • Polycystic ovary syndrome (PCOS) with hyperandrogenic symptoms 5
  • Sexual disorders in men requiring androgen suppression 7

Dosing

  • For prostate cancer: Used as second-line therapy 2
  • For gender-affirming care: Often used in combination with estradiol 3
  • For androgenic conditions in women: Often combined with ethinylestradiol (2mg CPA/0.035mg ethinylestradiol) 4, 6

Monitoring Requirements

Baseline Assessment

  • Complete blood count to establish baseline hemoglobin and hematocrit 2
  • Liver function tests 1
  • Adrenal function assessment 2
  • Blood pressure and weight 4

Ongoing Monitoring

  • Regular liver function tests due to risk of hepatotoxicity, including potential for fulminant hepatic failure 2, 1
  • Complete blood count to monitor for anemia 2
  • Adrenal function tests to assess for adrenal suppression 2
  • Blood pressure and weight monitoring 4
  • In patients receiving combination therapy with estrogens, monitor for:
    • Venous thromboembolism risk 2, 5
    • Cardiovascular effects 5

Frequency of Monitoring

  • Liver function tests: Every 3-6 months, more frequently during initial treatment 1
  • Complete blood count: Every 3-6 months 2
  • Adrenal function: Periodically throughout treatment 2

Adverse Effects

Common Adverse Effects

  • Fatigue and decreased energy 3, 7
  • Breast tenderness and gynecomastia (in approximately 20% of male patients) 3, 7
  • Emotional changes 3
  • Hot flushes 3

Serious Adverse Effects

  • Hepatotoxicity, including risk of fulminant hepatic failure 2, 1
  • Adrenal suppression 2
  • Anemia 2
  • When combined with estrogens, increased risk of venous thromboembolism 5

Discontinuation Criteria

Absolute Criteria for Discontinuation

  • Development of fulminant hepatic failure or severe hepatotoxicity 2, 1
  • Severe anemia 2
  • Significant adrenal suppression 2

Relative Criteria for Consideration of Discontinuation

  • Persistent moderate elevation of liver enzymes 1, 6
  • Development of venous thromboembolism when used in combination with estrogens 5
  • Intolerable side effects such as severe fatigue, depression, or other mood disturbances 7

Special Considerations

  • Contraindicated in patients with hereditary angioedema as it may worsen the condition 1
  • Use with caution in patients with pre-existing liver disease 1
  • When used for gender-affirming care, discontinuation may result in significant exacerbation of gender dysphoria, potential depression and/or anxiety 2
  • In prostate cancer treatment, cyproterone acetate shows fewer cardiovascular side effects compared to diethylstilbestrol 1

Treatment Response Assessment

  • For hirsutism and acne: Clinical improvement typically seen after 3-6 months of treatment 6
  • For gender-affirming care: Decreased facial and non-facial hair growth, possible breast development 3
  • For prostate cancer: Monitor PSA levels and clinical symptoms 2

References

Guideline

Cyproterone Acetate Therapeutic Applications and Considerations

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Research

Use of cyproterone acetate/ethinylestradiol in polycystic ovary syndrome: rationale and practical aspects.

The European journal of contraception & reproductive health care : the official journal of the European Society of Contraception, 2017

Research

Experience in the long-term treatment of patients with hirsutism and/or acne with cyproterone acetate-containing preparations: efficacy, metabolic and endocrine effects.

Experimental and clinical endocrinology & diabetes : official journal, German Society of Endocrinology [and] German Diabetes Association, 1995

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.

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