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What disease does hydroxychloroquine treat? An Overview of Its Therapeutic Uses

4 min read

Over 60 years ago, hydroxychloroquine was developed for malaria, but was soon found to be effective for treating autoimmune diseases. This established the foundation for understanding what disease does hydroxychloroquine treat and its broader therapeutic applications as an immunomodulatory drug.

Quick Summary

Hydroxychloroquine is a medication used to treat several autoimmune conditions, including lupus and rheumatoid arthritis, and to prevent and treat malaria. Its anti-inflammatory effects help calm an overactive immune system, but it gained controversial attention as a debunked COVID-19 treatment.

Key Points

  • Autoimmune Treatment: Hydroxychloroquine (HCQ) is primarily prescribed as a disease-modifying anti-rheumatic drug (DMARD) for conditions like systemic lupus erythematosus (SLE) and rheumatoid arthritis (RA).

  • Malaria Prevention and Treatment: HCQ is also used to prevent and treat certain types of malaria, though its effectiveness is limited to regions without resistance to chloroquine.

  • Mechanism of Action: For autoimmune diseases, HCQ works by modulating the immune system and reducing inflammation by interfering with cellular pathways.

  • Long-Term Safety: The most serious potential side effect of long-term HCQ use is retinal damage (retinopathy), which necessitates regular eye exams for early detection and prevention.

  • COVID-19 Efficacy: HCQ has been extensively studied for COVID-19, and major clinical trials have shown that it is not an effective treatment and carries potential cardiac risks.

  • HCQ vs. Chloroquine: HCQ is generally preferred for autoimmune treatment over chloroquine due to its better safety profile and lower risk of eye toxicity.

In This Article

Hydroxychloroquine (HCQ), sold under the brand name Plaquenil, is a derivative of the antimalarial drug chloroquine. While its initial use was for combating malaria, doctors soon discovered its effectiveness in managing a range of autoimmune and inflammatory conditions. Today, it is best known for its role as a disease-modifying antirheumatic drug (DMARD), offering significant benefits to patients with specific chronic conditions by moderating the body's immune response.

Hydroxychloroquine for Autoimmune Diseases

HCQ's primary and most widespread use today is in the long-term management of autoimmune diseases. In these conditions, the body's immune system mistakenly attacks its own healthy tissues. HCQ helps to regulate this overactive immune response by interfering with cellular pathways, such as Toll-like receptor signaling, which reduces inflammation and tissue damage.

Systemic Lupus Erythematosus (SLE)

HCQ is a cornerstone therapy for systemic lupus erythematosus, a chronic autoimmune disease that can affect many parts of the body, including the joints, skin, and kidneys. Studies have shown that HCQ use significantly decreases lupus flares and can lower mortality rates in patients. By reducing the activity of the immune system, it helps manage the skin rashes, joint pain, and fatigue commonly associated with lupus.

Discoid Lupus Erythematosus (DLE)

For patients with DLE, a form of lupus that primarily affects the skin, HCQ is a standard treatment. It helps to clear up the characteristic painful, inflammatory, and scaly skin lesions.

Rheumatoid Arthritis (RA)

As a DMARD, HCQ is used to treat both acute and chronic rheumatoid arthritis, a condition causing painful swelling, stiffness, and joint damage. While it does not provide immediate pain relief like an NSAID, it can help reduce inflammation over time, slowing the progression of the disease and preventing further joint damage. It may be used alone for mild disease or in combination with other medications for more active arthritis.

Prevention and Treatment of Malaria

HCQ's original purpose was as an antimalarial drug. It works by killing the parasite that causes malaria within the red blood cells. For travelers visiting areas where malaria is common, HCQ can be used for prevention, starting one to two weeks before travel and continuing for several weeks after returning. However, it is only effective in regions where there is no known chloroquine resistance, which limits its use in many parts of the world. For active malaria infection, HCQ is prescribed in higher doses to treat the acute attack.

Side Effects and Safety Considerations

While generally well-tolerated, HCQ can cause a range of side effects. Most are mild and often resolve with time or by taking the medication with food.

Common side effects include:

  • Nausea, stomach pain, and vomiting
  • Diarrhea
  • Headache
  • Skin rashes and itching
  • Hair color changes or hair loss

Serious, but rare, side effects include:

  • Retinal damage (retinopathy): This is the most serious potential complication and is more likely with high doses over many years. Regular eye exams are crucial for long-term users.
  • Heart rhythm problems: Including prolonged QT interval, particularly when combined with other drugs.
  • Hypoglycemia (low blood sugar)
  • Muscle weakness
  • Neurological symptoms: Such as mood changes or seizures

Other Potential Uses and Controversies

Beyond its primary uses, HCQ is sometimes explored for other conditions. For instance, it has been used for porphyria cutanea tarda, a skin and nervous system disorder. Its use in Sjögren's syndrome, another autoimmune condition causing dry eyes and mouth, is more controversial, with some studies showing benefit for joint symptoms but not for the hallmark dryness.

HCQ gained significant public attention during the COVID-19 pandemic as a potential treatment. However, multiple rigorous clinical trials concluded that it offered no substantial benefit to hospitalized COVID-19 patients and was associated with increased cardiac risks. The World Health Organization and the FDA both recommended against its use for treating or preventing COVID-19. This episode highlights the difference between anecdotal reports and evidence-based medicine from controlled clinical trials.

Comparison of Hydroxychloroquine and Chloroquine

Feature Hydroxychloroquine (HCQ) Chloroquine (CQ) Notes
Autoimmune Use Common for SLE, RA, DLE Less common due to safety concerns HCQ is preferred for chronic autoimmune therapy.
Malaria Use Common, but limited by resistance Less common due to widespread resistance Both are antimalarials, but resistance patterns differ.
Side Effects Generally well-tolerated, lower risk of retinopathy Reputable for more serious side effects HCQ's better safety profile is a key reason for its widespread use in autoimmune care.
Retinal Damage Risk is rare, but possible with long-term, high doses Risk is considered higher than HCQ Regular eye exams are critical for both, especially for long-term use.
Dosing Typical dosing for autoimmune conditions is 200-400 mg daily Dosing can differ depending on the condition. Your doctor will determine the appropriate dose.
Mechanism Immunomodulatory and antimalarial Immunomodulatory and antimalarial Both are 4-aminoquinolines and work similarly.

Conclusion

Hydroxychloroquine is a versatile and long-standing medication, most notably used to manage chronic autoimmune diseases such as lupus and rheumatoid arthritis. It is also still employed for the prevention and treatment of malaria in specific regions. Its effectiveness in tempering the inflammatory response is central to its therapeutic benefits. While it is generally considered safe with a good risk-benefit profile, particularly when compared to its predecessor, chloroquine, careful monitoring for side effects, especially retinal damage, is essential for long-term use. The highly publicized, though unsubstantiated, claims surrounding its use for COVID-19 serve as a reminder of the importance of evidence-based medical practice over anecdotal recommendations. For individuals considering or using HCQ, ongoing consultation with a healthcare provider is vital to ensure its appropriate and safe application. For more authoritative information on HCQ and rheumatic diseases, you can consult the American College of Rheumatology guidelines.

Frequently Asked Questions

The main purpose of hydroxychloroquine is to treat autoimmune conditions like lupus (SLE and DLE) and rheumatoid arthritis. It is also used to prevent and treat malaria.

In autoimmune diseases, HCQ works by calming the overactive immune system. It acts as an anti-inflammatory and immunomodulatory agent, though its exact mechanism is not fully understood, by interfering with certain cellular functions to reduce inflammation.

Hydroxychloroquine does not work immediately. For autoimmune conditions like rheumatoid arthritis and lupus, it can take several weeks to months before you notice a significant improvement in your symptoms.

Common side effects include gastrointestinal issues like nausea, stomach pain, and diarrhea, as well as headaches. Taking the medication with food can often help reduce these symptoms.

Yes, although rare, long-term use of hydroxychloroquine can cause irreversible retinal damage, known as retinopathy. Because of this risk, individuals on long-term treatment need regular eye exams to monitor their vision.

No, extensive clinical trials have shown that hydroxychloroquine is not an effective treatment for COVID-19. Health authorities, including the FDA and WHO, have issued warnings against its use for this purpose due to a lack of benefit and potential cardiac risks.

Yes, hydroxychloroquine is generally considered safe for use during pregnancy and breastfeeding, particularly for women with autoimmune diseases. Continuing treatment is often recommended to prevent disease flares.

Both are antimalarials, but HCQ is a derivative of chloroquine with a better safety profile, especially regarding the risk of retinopathy. For this reason, HCQ is typically the preferred medication for treating autoimmune conditions.

References

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Medical Disclaimer

This content is for informational purposes only and should not replace professional medical advice.