There is no known cure for pediatric MCTD. However, there is effective treatment which can reduce or eliminate symptoms, allowing children with MCTD to lead healthy, productive lives.
There is no specific treatment for MCTD. Treatment will be tailored to a child’s pattern of symptoms. Over time, some patients develop mild arthritis and need only symptom relief. Patients who develop lung disease will require steroids and other immune-suppressing medications.
Raynaud's phenomenon responds well to protection from the cold, such as wearing mittens. Some children with Raynaud's may need drugs, like calcium channel blockers.
The following are a list of medications used to treat children with MCTD.
NSAIDs (Nonsteroidal Anti-Inflammatory Drugs)
Drugs like ibuprofen, naproxen and nabumetone are used to control the mild arthritis commonly seen in MCTD. Up to one-third of children get adequate disease control with the use of NSAIDs alone. These drugs are very well tolerated in children and uncommonly cause side effects like upset stomach.
Prednisone is the most commonly used drug in the group of medications called steroids, corticosteroids or glucocorticoids. Other drugs in this group are methylprednisolone or prednisolone. Prednisone (or one of the other steroids) may be used to treat severe arthritis unresponsive to NSAIDs, or to treat pulmonary hypertension.
Prednisone works quickly to calm the immune system and control inflammation. This drug is similar to cortisone, a natural hormone produced by our bodies. At first, high doses of this drug may be given for quick reduction of inflammation in the joints or lungs. As your child improves, the steroid dose will be reduced gradually to prevent side effects common at higher doses; side effects depend on both the dose and duration of therapy.
Common side effects include weight gain, increased appetite, increased risk for infections and facial swelling. Over a long period of time, the drug may cause decreased bone calcium content, cataracts, high blood pressure and a slowed growth rate. The doctor will try to lower the dose as soon as possible to decrease the risk of side effects, while keeping the disease under control.
Hydroxychloroquine (brand name Plaquenil)
This antimalarial drug is used to treat lupus, dosed as a once daily pill. Although hydroxychloroquine is generally well-tolerated, some children may develop stomach upset.
Approximately 1 in every 3,000-5,000 people who take high doses of hydroxychloroquine will accumulate pigment in the retina of the eye. If this is allowed to progress, it could interfere with a person's vision. For this reason, an ophthalmologist (eye doctor) needs to check your child's eyes once a year while your child is taking hydroxychloroquine. If the eye doctor finds any pigment accumulation, hydroxychloroquine will be stopped before any visual problems develop.
For patients with more severe symptoms like lung, kidney or central nervous system disease, drugs called immunosuppressives may be used. Immunosuppressive drugs calm the immune system by preventing new autoimmune cells from being formed.
Cyclophosphamide (brand name Cytoxan) is a type of immunosuppressive drug that is frequently used in combination with prednisone to treat kidney inflammation, central nervous system disease or pulmonary hypertension. Children receiving cyclophosphamide need to have their blood counts monitored carefully.
Side effects may include nausea, vomiting, hair loss, blood in the urine, decreased fertility or an increased risk of cancer or infection.