Megestrol

Quick links to important sections

Megestrol

Treatment option:

Medically reviewed

Rosario Oropesa

Last updated on 22/12/2025

This page provides general, reference-level information compiled from official medical sources. It is not a substitute for professional medical advice, diagnosis, or treatment. For decisions about your health, please consult a qualified healthcare professional.

Overview of Megestrol

What is Megestrol? Overview and Quick Facts

Property Description
Active ingredient Megestrol acetate
Form Tablet and Oral Suspension (liquid)
Pharmacological class Progestin (Synthetic Hormonal Agent)
General purpose Hormonal modulation and Appetite stimulation
Origin Synthetic derivative of progesterone

What is Megestrol and What Type of Medicine is It?

Megestrol is a potent, prescription-only medicine whose active compound is Megestrol acetate, classified pharmacologically as a progestin and a hormonal agent. This substance is entirely synthetic, created as a chemical derivative of the naturally occurring steroid hormone progesterone. Its structure grants it high potency, an INN-level differentiation that sets it apart from less modified progestins. It is recognized as a progestational agent, confirming its established role in influencing hormone-sensitive processes.

As a progestin, Megestrol's primary action involves acting as a strong agonist on specific hormone receptors in the body. This mode of action is clinically recognized for providing high-level hormonal modulation when managing certain conditions related to hormone-sensitive tissues.

How is Megestrol Supplied and What is its General Purpose?

Megestrol acetate is manufactured for oral administration and is supplied in two primary dosage forms: a tablet and an oral suspension (liquid). The liquid form is a differentiating factor, often utilized because its finely divided, micronized particles are designed to increase oral bioavailability compared to a standard tablet, which means the body absorbs the active ingredient more effectively.

The general purpose of this medicine is dual: to manage certain hormone-sensitive conditions and to promote healthy weight gain by stimulating appetite. Its use as an appetite stimulant is a recognized benefit for improving nutritional status when a patient is experiencing significant, unintentional weight loss. This single-ingredient product's ability to influence both hormonal pathways and metabolic regulation is why it is prescribed in situations requiring either specific tissue control or robust nutritional support.

Regulatory References

  1. WHO

What side effects are possible with Megestrol?

Possible Side Effects and Safety Information

The official safety profile of Megestrol acetate is formally defined in government regulatory documents and is structured around its potent hormonal (progestin) activity, metabolic impact, and vascular risks. The classification of adverse reactions follows standard frequency categories, such as Very Common, Common, and Uncommon.


Official Adverse Reaction Scope

Component Description
System-Organ Classes Primarily affects Metabolism (e.g., weight gain, hyperglycemia), Vascular (e.g., thromboembolic events), Endocrine (e.g., adrenal suppression), and Gastrointestinal systems.
Common Reactions Commonly reported adverse events include diarrhea, nausea, flatulence, and asthenia (weakness), alongside weight gain and hot flashes.

Serious Adverse Reactions and Safety Constraints

The regulatory labeling highlights several serious adverse reactions. These include thromboembolic phenomena, such as Deep Vein Thrombosis (DVT) and Pulmonary Embolism (PE), which are associated with the drug's hormonal effects. Due to its intrinsic glucocorticoid activity, the medicine can cause Adrenal Suppression and, with chronic use, Overt Cushing’s Syndrome.

Specific Population-Specific Safety Considerations are defined in official documents: the medicine is contraindicated in pregnancy due to the potential for fetal harm. Caution is advised for older adults due to potential decreased renal function and for patients with pre-existing diabetes due to the risk of exacerbating hyperglycemia. Furthermore, adrenal insufficiency may occur upon withdrawal from chronic therapy, a key duration-related safety pattern documented in the label.

This structure ensures a clear, regulatory-defined distinction between the spectrum of documented adverse events and clinically significant risks.

Overdose and Emergency Response

Overdose and When to Seek Help — Official Regulatory Information

Official regulatory information regarding megestrol overdose is based on limited reports received through post-marketing experience.

Overdose scope

Feature Official Regulatory Statement
Documented Overdose Presentations Diarrhea, nausea, abdominal pain, shortness of breath, cough, unsteady gait, listlessness, and chest pain.
Physiological Systems Affected Gastrointestinal, Respiratory, Musculoskeletal/Neurological, Cardiovascular.
Dose-related or Exposure-related Factors Overdose reports are described as limited in post-marketing experience (FDA).
Emergency-Response Statements In case of overdose, appropriate supportive measures should be taken.
Antidote There is no specific antidote for megestrol overdose.

When Immediate Medical Help is Required

The regulatory guidance indicates that in the event of an overdose, a patient requires immediate medical evaluation for appropriate supportive measures. Individuals should contact the poison control helpline or seek emergency medical services if severe symptoms such as collapse, seizure, trouble breathing, or unconsciousness occur. The listed signs of overdose—including shortness of breath and chest pain—also warrant urgent professional medical attention. The official information does not classify the severity of overdose.

Therapeutic Uses of Megestrol

What Megestrol Treats: Main Uses and Benefits

In clinical practice, the medication is used in situations involving certain distressing symptoms related to both significant wasting and specific malignancies.

Megestrol is considered relevant in clinical settings marked by severe, unintended weight loss and profound anorexia, which are symptoms related to systemic imbalance and create noticeable physiological strain. It is commonly used to help manage these groups of symptoms that interfere with daily comfort, particularly AIDS-related cachexia. The medication supports the patient by helping to ease the severe physical strain caused by wasting, which is relevant for stimulating appetite and may assist with promoting an increase in body mass.

The primary therapeutic indications where Megestrol is relevant include the palliative treatment of advanced carcinoma of the breast or endometrium and the management of anorexia, cachexia, or unexplained significant weight loss in patients with AIDS.

“The medication supports nutritional management in wasting conditions and aids in the symptomatic management of specific advanced cancers.”

The medication is relevant in conditions where functional stability becomes affected, addressing both these therapeutic areas. For hormonal use, it offers supportive therapeutic benefit in these hormone-sensitive contexts, which may assist with maintaining a sense of stability when symptoms are more noticeable in these specific hormone-sensitive malignancies.


Quick Fact: Relief for Appetite Loss

Megestrol supports patients by addressing profound anorexia and severe weight loss, contributing to improved comfort and helping to ease the overall symptom burden associated with wasting syndromes.

Eligibility and Restrictions for Use

Eligibility Map: Who Can and Cannot Use Megestrol? — Official Regulatory Information

Populations for whom use is allowed (as stated in label):

  • Adult patients for the palliative treatment of advanced carcinoma of the breast or endometrium.
  • Adult patients with anorexia, cachexia, or unexplained significant weight loss in patients with Acquired Immunodeficiency Syndrome (AIDS).

Populations for whom use is contraindicated:

  • Patients with a history of hypersensitivity to megestrol acetate or any component of the formulation.
  • Patients with known or suspected pregnancy (FDA Pregnancy Category D).

Age-related eligibility rules:

  • Pediatric Use: Safety and effectiveness have not been established; use is generally not recommended in children.
  • Geriatric Use: Insufficient data are available from clinical studies. Caution is required due to the greater frequency of decreased hepatic, renal, or cardiac function in older adults; older adults should not usually take megestrol for appetite loss and weight loss.

Condition-specific eligibility rules:

  • Use requires caution in patients with a history of thromboembolic disease, impaired renal function, or diabetes mellitus.
  • Therapy for weight loss should only be instituted after treatable causes of weight loss (e.g., systemic infections, endocrine disease) are sought and addressed.

Pregnancy and lactation eligibility status (if explicitly documented):

  • Pregnancy: Contraindicated. Women of childbearing potential must be advised to use effective contraception during treatment.
  • Lactation: Nursing should be discontinued if the medicine is required for treatment due to the potential for adverse effects on the newborn.

Connection to the overall eligibility profile (2–4 sentences): Official regulatory documents define who can and cannot use the medicine by establishing absolute contraindications based on hypersensitivity and pregnancy status. Eligibility is constrained by age-group limitations, where safety is often not established in pediatric groups, and by comorbidity-based restrictions, where existing conditions like renal impairment or diabetes necessitate cautious use. This structure formally maps the permissible, restricted, and prohibited populations under the terms of government-approved labeling.

What should I know about interactions with other medicines?

Interactions with other medicines and products

Official regulatory documents detail specific interaction patterns for Megestrol acetate that relate to both pharmacokinetic and pharmacodynamic effects, establishing constraints on co-administration.


Documented Interaction Constraints

Category Interacting Medicine or Class Official Regulatory Statement
Formal Contraindication Dofetilide Co-administration is contraindicated due to Megestrol's inhibition of renal cationic secretion, which significantly increases Dofetilide plasma concentration.
Exposure Reduction Indinavir Co-administration results in a documented significant decrease in Indinavir systemic exposure (reduced AUC and Cmax).
Pharmacodynamic Antagonism Antidiabetic Agents (e.g., Insulin) Megestrol may diminish the therapeutic effect due to its progestin-related capacity to impair glucose tolerance.
Pharmacodynamic Potentiation Warfarin / Anticoagulants Megestrol may increase the effects of Warfarin. Close monitoring is often required for anticoagulants.
Formulation Caution Megace ES Oral Suspension The concentrated suspension is not equivalent to the conventional oral suspension on a mg-per-mg basis, requiring a formulation-specific administration caution.

These constraints define the official interaction profile. The most significant restriction is the formal contraindication with Dofetilide, which necessitates strict avoidance. Additionally, population-specific considerations note that Megestrol's effect of fluid retention may be exacerbated in patients with documented renal dysfunction.

Mechanism of Action

Megestrol is a synthetic progestin that exerts its primary action by selectively binding to progesterone receptors (PR) located in the nucleus of target cells. This binding initiates a complex signaling cascade that is central to its physiological effects. The strength of this binding interaction is a key determinant of its downstream activity.

The binding of Megestrol to the PR causes a change in the receptor's conformation, which allows the newly formed Megestrol-PR complex to translocate into the nucleus. Here, the complex acts as a transcription factor, modulating the expression of specific genes. This action alters the cellular environment, primarily by suppressing the synthesis and release of certain catabolic cytokines, such as tumor necrosis factor-alpha (TNF-alpha) and interleukin-6 (IL-6).

In addition to its effect on sex hormone receptors, Megestrol also exhibits weak glucocorticoid activity by potentially binding to glucocorticoid receptors. These dual effects—progestational and mild glucocorticoid—modulate key inflammatory and metabolic pathways. The overall physiological consequence of these actions is a modification of energy balance and a reduced inflammatory drive within the body's systems.

Dosage and Administration Information

How to Use Megestrol Acetate: Administration Guidelines

Megestrol acetate is administered exclusively by the oral route, available in both a tablet and an oral suspension (liquid) form. The administration pattern, including the total daily dose and frequency, is determined by the specific clinical indication, reflecting the two distinct applications of the medicine.


Dosing Regimens

Indication Standard Daily Dosing Pattern
Palliative Breast Cancer 160 mg/day, often given as 40 mg four times a day (QID) or as a single daily dose
Palliative Endometrial Cancer 40 mg to 320 mg/day in divided doses
Anorexia/Cachexia (AIDS-related) 800 mg/day (standard suspension) or 625 mg/day (concentrated suspension) as a single daily dose

Administration Requirements and Duration

For the liquid formulation, the bottle of oral suspension must be shaken vigorously before the dose is measured to ensure the correct concentration is administered. The concentrated suspension (e.g., 125 mg/mL) is specified to be taken without regard to meals.

It is important to note that the concentrated oral suspension and standard tablets are not equivalent on a milligram-to-milligram basis due to differences in how the drug is absorbed. For cancer palliative use, a minimum period of two months of continuous treatment is generally required for assessing the initial effect of the drug.

Additionally, dose selection involves caution in older adults and in patients with renal impairment, which often involves conservative, low-end dosing to account for potentially reduced organ function.

Recent Clinical Evidence

Research Evidence / Overview of Studies for Megestrol


Evidence for Use in Appetite Stimulation and Weight Gain for Cachexia/Anorexia

This section will summarize the available clinical trials and research findings that have investigated Megestrol's potential role in helping people with significant and unintentional weight loss due to conditions like AIDS or cancer. The studies reviewed will focus on the general outcomes related to nutritional status and body weight changes.

Megestrol was studied for its potential role in helping people with significant and unintentional weight loss, a condition often referred to as cachexia or anorexia. The research examined outcomes related to weight gain, measured changes in appetite, and overall quality of life. The studies monitored these effects over defined time intervals, with many focusing on episodes where symptoms become more noticeable due to short-term changes.

Research indicates patterns where weight gain was associated with the use of Megestrol compared to placebo. This weight gain was observed in some studies to be linked to an increase in appetite, as reported by patients themselves. These findings describe patterns observed in the studies conducted during periods where symptoms become more noticeable. However, the studies explored whether this weight gain represented primarily lean muscle mass or body fat, and findings were mixed regarding the composition of the weight gained.

While the data show patterns related to improvements in appetite and weight change in the observed populations, certainty remains low regarding the long-term durability of these effects. The research highlights changes measured during the study period, but follow-up durations were limited in many of the key studies. This means there is limited information for long-term outcomes regarding sustained nutritional improvement.


Evidence for Use in Advanced Breast Cancer

This section will present an overview of the key clinical studies, including randomized controlled trials, that have evaluated Megestrol in a research setting as a hormonal approach for advanced-stage, hormone-sensitive breast cancer. The summary will focus on outcomes related to tumor status and the time until disease progression was noted in the studies involving these specific patient populations.

Megestrol was evaluated in women with advanced breast cancer, particularly those whose tumors were classified as hormone-sensitive. The research explored its potential as an approach when other hormonal strategies had been tried or were not suitable. The trials examined outcomes related to systemic or functional imbalance by monitoring changes in tumor status and the time until disease progression was noted.

Studies report how measured outcomes evolved in the observed populations, and the data show patterns related to changes in tumor size (including slowing of growth or temporary shrinkage) in some patients. This effect was observed in some studies that focused on episodes where symptoms become more noticeable due to advanced disease. The research provides context on how the approach was associated with these outcomes in the groups studied.

The extent of the evidence varies across studies. Research focusing on episodes where symptoms become more noticeable may not capture the full range of long-term effects. The evidence is limited regarding whether Megestrol's measured outcomes are similar to those of other approaches. Comparative evidence is lacking in certain modern contexts.


Long-term Studies and Follow-up

This section summarizes what the existing research has reported regarding long-term patient outcomes after using Megestrol, including how durable any initial effects might be and any data available from extended-duration or follow-up studies. It will also highlight where the evidence is limited concerning the very long-term effects.

When considering extended use, studies contribute to understanding symptom patterns regarding outcomes reflecting daily functioning or activity level over longer periods. The available research was observed in trials that monitored patient-reported outcomes describing perceived discomfort and general health status over defined time intervals.

For both appetite stimulation and cancer research, long-term effects are not fully established. Follow-up durations were limited in much of the initial research, meaning there is limited information for long-term outcomes that would provide definitive clarity on the durability of the initial findings. The research provides insight into short-term changes, but data are still emerging about what happens years after the study period concludes.

This limitation means that while the findings describe group patterns, not personal outcomes, for short periods, the long-term trajectory may vary considerably among individuals. Research contributes to the broader evidence landscape by showing short-term effects, but more long-term studies are needed to fully understand the full course of effects.


Evidence in Special Populations

This section outlines what studies, if any, have specifically evaluated the effects of Megestrol in distinct groups of people, such as older adults, individuals with specific pre-existing health conditions (comorbidities), or other groups where evidence may be limited or requires separate consideration.

Research has explored the use of Megestrol in populations outside of the main clinical trials, such as older adults with conditions marked by functional limitations or conditions where symptoms may vary in intensity. Studies examined its use in these groups, monitoring outcomes related to systemic or functional imbalance.

In general, data for certain groups remain insufficient. For instance, while older adults were observed in some studies related to appetite, subgroup findings are uncertain regarding how effects or measured changes may differ compared to younger adults. Research into its use during pregnancy or for certain comorbid conditions like severe diabetes is extremely limited, and comparative evidence is lacking for many of these special circumstances.

Therefore, the results apply only to the populations studied in the primary clinical trials, and the research does not determine whether an individual in a special population will respond similarly.


What is Still Uncertain About Megestrol

This section synthesizes the main limitations, inconsistencies, or gaps found within the current body of research on Megestrol, clarifying the areas where more research is needed to better understand its overall effects and utility.

A primary uncertainty is the long-term impact on weight gain composition. While Megestrol was associated with weight gain in studies for cachexia, it is unclear how these changes relate to patient-reported outcomes describing perceived discomfort or overall survival over many years. This is a crucial gap because long-term effects are not fully established and follow-up durations were limited.

Additionally, the findings were mixed concerning the optimal timing or duration of its use for both indications, and the extent of the evidence varies across studies. For example, research exploring short-term symptom changes sometimes shows a pattern of response, but research exploring long-term patterns in conditions presenting with cycles of stability and flare-ups over a long period is still ongoing.

The research provides context but not individual predictions, and many questions remain regarding its use when combined with newer therapies. As research is ongoing, these limitations mean that the evidence highlights what is known — and what is still uncertain — about the full picture of Megestrol's effects.

Frequently Asked Questions (FAQ)

Common questions about Megestrol (FAQ)

Q: Why is Megestrol sometimes used for appetite?

A: Official documents note that the precise mechanism by which Megestrol exerts effects on appetite is currently unknown. However, studies and research have associated its use with affecting certain hormones and reducing inflammatory molecules in the body, which can influence metabolism and lead to weight changes.

Q: Does Megestrol affect blood sugar levels?

A: Yes, official safety information indicates that Megestrol can affect how the body handles glucose (sugar). There have been reports of new cases of diabetes, or a worsening of pre-existing diabetes (high blood sugar), in people using the drug chronically.

Q: Are there any long-term health concerns associated with using Megestrol?

A: Long-term use is associated with a specific risk noted in regulatory documents: secondary adrenal insufficiency. This means that upon stopping the drug after chronic use, the adrenal glands may not produce enough hormones, a condition for which official documents note a need for medical consideration.

Q: Are there age restrictions for who can use Megestrol?

A: According to official product information, safety and effectiveness have not been established for children, meaning its use is typically avoided in this age group. Use requires caution in older adults, generally due to the greater frequency of decreased kidney, liver, or heart function in this population.

Q: How is Megestrol usually discontinued after long-term use?

A: Regulatory documents emphasize that the possibility of adrenal insufficiency should be considered upon withdrawal from chronic use. Any patient stopping chronic therapy may require medical surveillance.

Q: Does Megestrol cause stomach upset or nausea?

A: Nausea, diarrhea, and vomiting are among the commonly reported side effects listed in the clinical trial data for Megestrol. These gastrointestinal effects are included in the official safety profile of the medicine.

Q: Can Megestrol interfere with lab tests?

A: Since Megestrol can impact glucose tolerance and may lead to new or worsening diabetes, it can indirectly interfere with lab tests. Specifically, it may affect the accuracy of blood tests used to monitor blood sugar levels.

Q: Does Megestrol have a generic version available?

A: Yes, Megestrol acetate is the generic name for the active ingredient. It is available under its generic name, as well as various brand names.

Q: Is Megestrol the same as the birth control pill?

A: Megestrol acetate is classified as a progestin, which is a type of synthetic hormonal agent derived from progesterone. While progestins are used in birth control, the official uses for this specific medicine are strictly limited to advanced cancer treatment and anorexia/cachexia, and not for contraception.

Q: Can people taking Megestrol still drive or operate machinery?

A: Official labeling lists nervous system adverse effects such as dizziness, confusion, and abnormal thinking as potential side effects. Such effects have the potential to impact a person's ability to safely perform tasks requiring complete alertness, such as driving or operating heavy machinery.

Q: How long does it usually take to see any effect from Megestrol?

A: For cancer treatment, a minimum period of two months of continuous treatment is established for assessing initial effectiveness. However, clinical studies focusing on appetite stimulation have reported that some patients experienced weight gain within three weeks of starting treatment.

Q: Is Megestrol a form of chemotherapy?

A: No, Megestrol is pharmacologically classified as a progestin, which is a hormonal agent. It works by affecting hormone receptors and inflammatory pathways in the body, and it is not classified as a traditional cytotoxic chemotherapy drug.

Q: Is it common to feel fatigued while taking Megestrol?

A: Fatigue (tiredness) is a reported adverse event listed in the clinical trial data for Megestrol. Additionally, asthenia (weakness) and lethargy are also listed in the product's official safety profile.

Q: Can Megestrol affect the liver or kidneys?

A: Enlarged liver (hepatomegaly) has been reported as an adverse event in some clinical data. Additionally, official labeling advises caution in patients who already have decreased renal (kidney) function.

Q: How does Megestrol compare to other appetite stimulants mentioned in research?

A: Official reviews of the evidence note that the full scope of direct comparisons between Megestrol and other appetite stimulants is limited. Studies do not provide definitive comparative data in certain modern medical contexts.

Q: What is the difference between Megestrol Acetate and other forms of Megestrol?

A: The drug is primarily available as Megestrol Acetate in different formulations (tablets and oral suspensions). Official labeling notes that the concentrated oral suspension and the standard formulations are not equivalent, and therefore cannot be substituted on a milligram-to-milligram basis due to differences in how the body absorbs them.

Q: Do patients typically need routine monitoring while on Megestrol?

A: Close surveillance or monitoring is typically indicated for any patient treated for recurrent or metastatic cancer. Monitoring is also advised for blood sugar changes and for signs of adrenal insufficiency.

Q: Can Megestrol cause changes to a person's skin or hair?

A: Adverse events listed on the product label include effects on the skin and hair. Specifically, rash, increased sweating, and alopecia (hair loss) have been reported.

Q: Does Megestrol cause swelling in the ankles or feet?

A: Edema (swelling) and peripheral edema (swelling of the extremities) are listed as adverse events in the product labeling.

Q: Is Megestrol used for weight loss in any capacity?

A: No, Megestrol acetate is indicated for the treatment of significant, unintentional weight loss associated with conditions like cancer and AIDS (anorexia and cachexia). It is not intended for use as a weight-loss product.

Q: What is the process for getting Megestrol if someone is deemed eligible?

A: Megestrol acetate is a prescription-only medicine (Rx only). Official guidance states that its use requires evaluation and prescribing by a licensed healthcare provider.

Q: Are headaches a commonly reported side effect of Megestrol?

A: Headache is listed as a reported adverse event in the clinical trial data for megestrol acetate, indicating it has been observed.

How should Megestrol be stored and disposed of?

Storage and Disposal Requirements

Megestrol acetate must be stored according to regulatory specifications to ensure its stability. The medication, in both tablet and oral suspension forms, should be maintained at controlled room temperature, specifically between 20 C and 25 C (68 F and 77 F). The oral suspension must be protected from freezing and also protected from light.

All forms must be kept in a tightly closed container and stored out of the sight and reach of children and pets.

Disposal of unused or expired megestrol should adhere to official instructions on the product label or utilize a local drug take-back program. It should not be flushed down the toilet or poured into a drain unless specific guidance is provided by a regulatory authority for that product.

Attention! Always consult to a doctor or pharmacist before using pills or medicines.

Equivalent of Megestrol found in:

A-Z Index: