DMARDs Explained: Methotrexate, Sulfasalazine, Hydroxychloroquine, Mycophenolate and Leflunomide

Black and white photograph of methotrexate and hydroxychloroquine medication boxes used as DMARD treatments in rheumatology

Disease-modifying anti-rheumatic drugs (DMARDs) are the foundation of treatment for many inflammatory and autoimmune rheumatic diseases. They work by suppressing the immune-driven inflammation that causes pain, swelling, stiffness and long-term joint or organ damage.

Although these medications are often described as “strong” or “immunosuppressive”, they have been used safely and effectively for decades and remain the cornerstone of modern rheumatology care.

What Are DMARDs?

DMARDs are medications that modify the underlying disease process, rather than simply treating symptoms.

Unlike painkillers or anti-inflammatory drugs, they:

  • suppress immune-mediated inflammation

  • reduce disease activity

  • prevent long-term joint and organ damage

  • improve long-term outcomes and quality of life

They are used in conditions such as:

  • rheumatoid arthritis

  • psoriatic arthritis

  • axial spondyloarthritis

  • lupus

  • inflammatory myositis

  • vasculitis

  • connective tissue diseases

Why Are DMARDs Started Early?

In most inflammatory rheumatic diseases there is a recognised “window of opportunity”, where early treatment leads to better long-term outcomes.

Starting DMARDs early:

  • improves symptom control

  • increases the chance of remission

  • reduces irreversible joint damage

  • improves long-term function

This is why modern rheumatology focuses on early diagnosis and prompt treatment, rather than waiting for disease progression.

Methotrexate

Methotrexate is the most commonly prescribed DMARD in rheumatology and is considered the anchor drug for rheumatoid arthritis.

How It Works

At the low doses used in rheumatology, methotrexate acts as an immune modulator rather than a chemotherapy drug. It suppresses key inflammatory pathways involved in autoimmune disease.

What Conditions Is It Used For?

  • rheumatoid arthritis

  • psoriatic arthritis

  • inflammatory arthritis

  • some connective tissue diseases

  • vasculitis (in selected cases)

How Is It Taken?

  • once weekly (never daily)

  • as tablets or subcutaneous injections

  • usually combined with folic acid taken on a different day

Why the Dose Is Increased Gradually

Methotrexate is usually started at a low dose and increased gradually over several weeks.

This approach is used to:

  • improve tolerability

  • reduce early side effects

  • allow the body to adjust

  • find the lowest effective dose

For example:

  • often started at 7.5–10 mg once weekly

  • gradually increased (e.g. to 15–25 mg weekly) depending on response and tolerance

Because methotrexate is titrated slowly, early side effects often improve with time, dose adjustment, folic acid supplementation, or switching from tablets to injections.

Sulfasalazine

Sulfasalazine is a long-established DMARD used particularly in inflammatory arthritis.

What Conditions Is It Used For?

  • rheumatoid arthritis

  • psoriatic arthritis

  • peripheral spondyloarthritis

Key Points

  • taken orally, usually twice daily

  • slow onset (6–12 weeks)

  • generally well tolerated

  • useful in combination therapy

Hydroxychloroquine

Hydroxychloroquine is one of the mildest DMARDs and is widely used in autoimmune diseases.

What Conditions Is It Used For?

  • rheumatoid arthritis

  • lupus

  • connective tissue diseases

  • inflammatory arthritis

Key Points

  • mild immune-modulating effect

  • slow onset (8–16 weeks)

  • generally very well tolerated

  • usually started at full therapeutic dose

  • does not require dose titration

Hydroxychloroquine and the Eyes

Hydroxychloroquine can very rarely affect the retina at the back of the eye when taken long-term.

Important points:

  • the risk is very low at standard doses

  • modern screening has made this complication extremely uncommon

  • baseline and periodic eye checks are recommended

  • the drug is usually stopped if any early changes are detected

When prescribed at appropriate doses and monitored correctly, hydroxychloroquine is considered a very safe long-term medication.

Leflunomide

Leflunomide is an effective alternative to methotrexate for inflammatory arthritis.

What Conditions Is It Used For?

  • rheumatoid arthritis

  • psoriatic arthritis

Key Points

  • taken once daily

  • similar effectiveness to methotrexate

  • slow onset (8–12 weeks)

  • usually started at a fixed dose

  • requires regular blood monitoring

Mycophenolate Mofetil

Mycophenolate is a more potent immunosuppressive DMARD used mainly in systemic autoimmune disease.

What Conditions Is It Used For?

  • lupus (especially kidney or lung involvement)

  • vasculitis

  • inflammatory myositis

  • connective tissue disease-related lung disease

Key Points

  • not usually used for routine inflammatory arthritis

  • particularly valuable in organ-threatening disease

  • requires careful blood monitoring

How Long Do DMARDs Take to Work?

DMARDs are slow-acting medications.

Most take:

  • 6–16 weeks to show benefit

  • several months for full effect

Because of this, short-term treatments such as corticosteroids are sometimes used as a temporary bridge while DMARDs are taking effect.

Side Effects and Monitoring

All DMARDs can cause side effects, but serious complications are uncommon when they are:

  • prescribed appropriately

  • monitored correctly

  • used at the lowest effective dose

Common Side Effects

Most side effects are mild and manageable, particularly in the early months of treatment.

Common examples include:

  • nausea or indigestion

  • fatigue

  • headache

  • mouth ulcers (especially with methotrexate)

  • mild hair thinning

  • increased susceptibility to minor infections

Many of these improve with:

  • dose adjustment

  • folic acid supplementation

  • switching between tablet and injection formulations

  • simple supportive treatments

Less Common but More Serious Side Effects

More serious side effects are uncommon, particularly with appropriate monitoring.

These can include:

  • liver inflammation

  • low white blood cell counts

  • lung inflammation (rare with methotrexate)

  • severe allergic reactions (very rare)

This is why structured blood-test monitoring is a routine and essential part of DMARD therapy.

When abnormalities are detected early, problems can almost always be reversed by:

  • dose reduction

  • temporary drug interruption

  • switching to an alternative DMARD

Blood-Test Monitoring: How Often Is It Done?

Regular blood tests are required to ensure DMARDs remain safe.

Although schedules vary slightly between drugs and individuals, a typical approach is:

  • every 2–4 weeks for the first 2–3 months

  • then every 4–8 weeks once stable

  • sometimes every 12 weeks in long-term stable patients

These blood tests monitor:

  • liver function

  • kidney function

  • blood cell counts

This structured monitoring is evidence-based and designed to detect problems early, before they become clinically significant.

DMARDs, Fertility and Pregnancy

Family planning is an important consideration when choosing DMARD therapy, particularly for women of child-bearing age and men wishing to father a child.

Different DMARDs have very different safety profiles in pregnancy.

Methotrexate

  • not safe in pregnancy

  • must be stopped at least 1-3 months before conception

  • avoided in women planning pregnancy

  • associated with miscarriage and birth defects

Leflunomide

  • not safe in pregnancy

  • can remain in the body for a prolonged period

  • requires a drug elimination procedure before conception

  • avoided in both women and men planning pregnancy

Hydroxychloroquine

  • safe in pregnancy

  • often continued throughout pregnancy in lupus and inflammatory arthritis

  • does not impair fertility

  • considered one of the safest DMARDs

Sulfasalazine

  • generally safe in pregnancy

  • often continued

  • men may experience reversible low sperm counts

  • folic acid supplementation is recommended

Mycophenolate Mofetil

  • not safe in pregnancy

  • strongly associated with birth defects

  • must be stopped well in advance of conception

  • requires careful specialist planning

Why Specialist Advice Matters

Because these drugs differ so significantly:

  • pregnancy plans should always be discussed in advance

  • treatment can usually be adjusted safely

  • disease control can often be maintained with pregnancy-compatible drugs

This avoids both disease flares and unnecessary risk to the baby.

Are DMARDs Safe Long-Term?

Yes — when monitored properly, DMARDs are very safe long-term.

They have:

  • been used for decades

  • extensive safety data

  • well-established monitoring protocols

For most patients, the risks of untreated inflammation and active disease far outweigh the risks of DMARD therapy.

What If DMARDs Don’t Work?

If disease remains active despite conventional DMARDs, additional therapies are available.

These include:

  • biologic drugs (such as anti-TNF agents)

  • targeted synthetic DMARDs (such as JAK inhibitors)

(Related reading: Anti-TNF Treatments in Rheumatology – What Patients Should Know)

Why Treatment Is Individualised

There is no single “best” DMARD for everyone.

Treatment decisions are based on:

  • diagnosis

  • disease severity

  • organ involvement

  • other medical conditions

  • pregnancy plans

  • patient preference

This personalised approach maximises benefit and minimises risk.

In Summary

  • DMARDs suppress immune-driven inflammation

  • They prevent long-term joint and organ damage

  • Methotrexate is the most commonly used first-line drug

  • Sulfasalazine and hydroxychloroquine are often used in combination

  • Leflunomide is an effective alternative

  • Mycophenolate is used for systemic autoimmune disease

  • Some DMARDs are started low and increased gradually

  • All DMARDs require regular blood monitoring

  • Different DMARDs have different pregnancy safety profiles

  • Modern rheumatology aims for remission or low disease activity

Trusted Patient Information Resources

Arthritis UK provide overview of disease-modifying anti-rheumatic drugs, how they work, and common side effects.

Methotrexate

https://www.arthritis-uk.org/information-and-support/understanding-arthritis/arthritis-treatments/drugs/methotrexate/

Sulfasalazine

https://www.arthritis-uk.org/information-and-support/understanding-arthritis/arthritis-treatments/drugs/sulfasalazine/

Hydroxychloroquine

https://www.arthritis-uk.org/information-and-support/understanding-arthritis/arthritis-treatments/drugs/hydroxychloroquine/

Leflunomide

https://www.arthritis-uk.org/information-and-support/understanding-arthritis/arthritis-treatments/drugs/leflunomide/

Mycophenolate

https://www.arthritis-uk.org/information-and-support/understanding-arthritis/arthritis-treatments/drugs/mycophenolate/

Dr Animesh Singh, Consultant Rheumatologist. GMC: 6130215


Please note, these posts are for general information only and do not constitute medical advice. Dr Singh would encourage you to speak to your healthcare professional to be assessed and managed for your specific symptoms.

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