Miltefosine Side Effects

Medically reviewed by Drugs.com. Last updated on May 28, 2024.

Applies to miltefosine: oral capsule.

Precautions

It is very important that your doctor check your or your child's progress at regular visits. This will allow your to see if the medicine is working properly and to decide if you should continue to take it. Blood tests will be needed to check for unwanted effects.

Using this medicine while you are pregnant can harm your unborn baby. Use an effective form of birth control to keep from getting pregnant during treatment and for 5 months after the last dose of this medicine. If you think you have become pregnant while using this medicine, tell your doctor right away.

You must have a negative urine or blood pregnancy test before you will be allowed to take this medicine. If you miss a period while you are using this medicine, tell your doctor right away.

Vomiting or diarrhea may occur while taking this medicine. These side effects may prevent birth control pills from working properly. You may use other forms of birth control include condoms, diaphragms, or contraceptive foams or jellies.

Serious skin reactions can occur with this medicine. Check with your doctor right away if you or your child have blistering, peeling, or loose skin, red skin lesions, severe acne or skin rash, sores or ulcers on the skin, or fever or chills while you are using this medicine.

Common side effects of miltefosine

Some side effects of miltefosine may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects.

Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

Serious side effects of miltefosine

Along with its needed effects, miltefosine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur while taking miltefosine:

For healthcare professionals

Applies to miltefosine: oral capsule.

General adverse events

Serious side effects and side effects resulting in discontinuation were Stevens-Johnson syndrome, melena, thrombocytopenia, arthritis, skin rash, diarrhea (grade 4), and hyperbilirubinemia (grade 4).^[Ref]

Genitourinary

• Very common (10% or more): Abnormal sperm parameters/reductions in sperm parameters, decreased semen volume (up to 75%), reduced total sperm count (up to 54%), reduced sperm motility (up to 51%), reduced sperm morphology (up to 31%), reduced sperm concentration (up to 26%)
• Frequency not reported: Testicular pain, testicular swelling
• Postmarketing reports: Scrotal pain, decreased ejaculate volume, absent ejaculation, scrotal tenderness, epididymitis^[Ref]

This drug was associated with reductions in all sperm parameters at the end of therapy; all sperm parameter reductions (except sperm concentration) recovered on follow-up assessments at 3 and 6 months after therapy completion. For sperm concentration, small mean decreases persisted on follow-up assessments at 3 and 6 months after the last dose. Semen analyses were not conducted beyond 6 months in any patient.

At end of therapy, decreased semen volume (less than 1.5 mL: 75%), reduced sperm count (at least 50% reduction from baseline: 54%; less than 39 million: 33%), reduced sperm concentration (at least 50% reduction from baseline: 21%; less than 20 million sperm/mL: 12%), reduced sperm motility (at least 25% reduction from baseline: 51%; less than 40% with total motility: 33%), and reduced sperm morphology (at least 25% reduction from baseline: 31%) were reported. During the follow-up period at the last observation (either 3 or 6 months after therapy completion), decreased semen volume (less than 1.5 mL: 15%), reduced sperm count (at least 50% reduction from baseline: 19%; less than 39 million: 6%), reduced sperm concentration (at least 50% reduction from baseline: 26%; less than 20 million sperm/mL: 8%), reduced sperm motility (at least 25% reduction from baseline: 9%; less than 40% with total motility: 6%), and reduced sperm morphology (at least 25% reduction from baseline: 15%) were reported.

Among 33 young male patients treated with this drug at a single center, 21 reported decreased ejaculate volume, 2 reported temporary absent ejaculation, 4 reported scrotal tenderness, and 1 had epididymitis diagnosed.^[Ref]

Hematologic

• Very common (10% or more): Decreased platelet counts (up to 62%)
• Common (1% to 10%): Lymphangitis
• Frequency not reported: Anemia, lymphadenopathy
• Postmarketing reports: Thrombocytopenia, agranulocytosis^[Ref]

In 1 study, decreased platelet counts (less than 150,000: 62%; less than 50,000: 2.4%) were reported at the end of therapy.^[Ref]

Hepatic

• Very common (10% or more): Transaminase elevations (up to 50%)
• Common (1% to 10%): Elevated AST, elevated ALT
• Frequency not reported: Hyperbilirubinemia
• Postmarketing reports: Jaundice^[Ref]

In 1 study, transaminase elevations during therapy were reported in up to 50% of patients; elevations were mild (less than 3 times the upper limit of normal [3 x ULN]) or moderate (3 to 5 x ULN) in 94% and 6%, respectively, of patients who had an elevation. No patient discontinued therapy due to transaminase elevations.

About 5% of patients reported elevated AST and ALT above ULN at end of therapy.^[Ref]

Gastrointestinal

• Very common (10% or more): Nausea (up to 41.7%), vomiting (up to 37.8%), diarrhea (up to 20.4%), abdominal pain (up to 11.2%)
• Frequency not reported: Abdominal distension, constipation, dysphagia, flatulence
• Postmarketing reports: Melena^[Ref]

Nervous system

• Very common (10% or more): Motion sickness (up to 29.2%), headache (up to 28.1%), dizziness (up to 12.5%)
• Common (1% to 10%): Somnolence
• Frequency not reported: Paresthesia
• Postmarketing reports: Seizure^[Ref]

Renal

• Very common (10% or more): Creatinine elevations (up to 25%)^[Ref]

In 1 study, creatinine elevations (at least 1.5 times above baseline) were reported in about 10% of patients at the end of therapy and 10% of patients at 6 months follow-up. In the placebo-controlled trial, 13.4% of patients had creatinine elevations of 1.5 to 3 times above baseline at end of therapy. In the comparative trial, about 5% of patients had creatinine elevations above baseline at 3 and 6 months after therapy. In the 2 active controlled trials, about 25% of patients had creatinine elevations 1.5 to 3 times above baseline at end of therapy.^[Ref]

Metabolic

• Very common (10% or more): Decreased appetite (up to 23.1%)^[Ref]

Dermatologic

• Common (1% to 10%): Pruritus
• Frequency not reported: Stevens-Johnson syndrome, skin rash, cellulitis, ecthyma, pyoderma, rash, urticaria^[Ref]

Other

• Common (1% to 10%): Asthenia, malaise, pyrexia
• Frequency not reported: Fatigue, abscess
• Postmarketing reports: Generalized edema, peripheral edema^[Ref]

Musculoskeletal

• Frequency not reported: Arthritis^[Ref]

Respiratory

• Postmarketing reports: Epistaxis

See also:

Further information

Miltefosine side effects can vary depending on the individual. Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Note: Medication side effects may be underreported. If you are experiencing side effects that are not listed, submit a report to the FDA by following this guide.

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