Substance / Medication

Mitotane

Overview

Active Ingredient
mitotane
RxNorm CUI
7004

Indications

LYSODREN is indicated for the treatment of patients with inoperable, functional or nonfunctional, adrenocortical carcinoma (ACC).

Labeler: HRA Pharma Rare DiseasesUpdated: 2025-10-08T00:00:00.000ZFull label on DailyMed

Boxed Warning

FDA Black Box Warning

Patients treated with LYSODREN are at increased risk for developing adrenal crisis in the setting of shock, severe trauma or infection that may lead to death. If shock, severe trauma or infection occurs or develops, temporarily discontinue LYSODREN and administer exogenous steroids. Monitor patients

Contraindications

When this intervention should not be used

None.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:
The efficacy of adjuvant mitotane therapy and radiotherapy following adrenalectomy in patients with adrenocortical carcinoma: A systematic review and meta-analysis.
Tsuboi Ichiro, Kardoust Parizi Mehdi, Matsukawa Akihiro et al. · Urol Oncol · 2025
PMID: 39379210Meta-Analysis
Benefits of Adjuvant Mitotane after Resection of Adrenocortical Carcinoma: A Systematic Review and Meta-Analysis.
Tang Yongquan, Liu Zhihong, Zou Zijun et al. · Biomed Res Int · 2018
PMID: 29967789Meta-AnalysisFull text (PMC)
Short-term variation in plasma mitotane levels confirms the importance of trough level monitoring.
Kerkhofs T M A, Derijks L J J, Ettaieb M H T et al. · Eur J Endocrinol · 2014
PMID: 25201518RCT
Mitotane therapy in adrenocortical cancer induces CYP3A4 and inhibits 5α-reductase, explaining the need for personalized glucocorticoid and androgen replacement.
Chortis Vasileios, Taylor Angela E, Schneider Petra et al. · J Clin Endocrinol Metab · 2013
PMID: 23162091RCT
Effect of mitotane on pituitary corticotrophs in clinically normal dogs.
Taoda Takahiro, Hara Yasushi, Takekoshi Susumu et al. · Am J Vet Res · 2006
PMID: 16881851RCT
Impact of Adjuvant Radiotherapy and Mitotane on Survival in Localized Adrenocortical Carcinoma: A Retrospective Cohort Study.
Elmali Aysenur, Guler Ozan Cem, Ozyigit Gokhan et al. · Int J Urol · 2026
PMID: 41399138Observational
Does Adjuvant Mitotane Impact Cure Rates in Adrenocortical Carcinoma? Insights From the ICARO-GETTHI/SEEN Registry.
Carmona-Bayonas Alberto, Álvarez-Escolá Cristina, Ballester Navarro Inmaculada et al. · J Clin Endocrinol Metab · 2025
PMID: 39988973Observational
External validation of the S-GRAS score for predicting recurrence in patients with adrenocortical carcinoma: implications for adjuvant mitotane therapy.
Jimenez-Fonseca Paula, Álvarez-Escola Cristina, Ballester Navarro Inmaculada et al. · Eur J Endocrinol · 2025
PMID: 40810251Observational

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Mitotane (substance)
SNOMED CT
26288002
UMLS CUI
C0026256
RxNorm CUI
7004
Labeler
HRA Pharma Rare Diseases

Clinical Data

This intervention maps to 1 entities in the Ltrl knowledge graph.

1
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.