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Refractory Celiac Disease Treatment

Options Include Drugs, Nutritional Support

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Updated January 22, 2013

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Refractory Celiac Disease Treatment
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Refractory celiac disease can be a frightening diagnosis: by definition, it means the standard treatment for celiac disease — the gluten-free diet — has failed to work, and you must now seek alternatives.

The good news, however, is that refractory celiac is extremely rare. In most cases, your problem isn't refractory celiac disease — instead, microscopic amounts of gluten in your diet or another medical condition likely are causing your continuing symptoms.

However, if your doctor rules out other causes and you ultimately are diagnosed with refractory celiac disease, you should know that physicians are able to treat the majority of cases successfully; treatment will depend on what type of refractory celiac you suffer from, along with other factors in your medical history and condition.

Learn more about diagnosis: Refractory Celiac Disease.

Type of Refractory Celiac Disease Determines Treatment Options, Course

There are two types of refractory celiac disease: Type I and Type II. When you're diagnosed, your doctor will tell you which type you have. Generally, Type I is more common and more easily treated, but recent studies have reported promising results for treating Type II.

Under most circumstances, you will be treated for refractory celiac disease at a celiac center with experience handling and monitoring the condition; because refractory celiac is so rare, many gastroenterologists haven't handled cases before.

Treatment May Start with Nutritional Support

Refractory celiac disease can lead to significant weight loss and malnutrition, even if you've been eating a wholesome, balanced gluten-free diet. That's because the damage to your small intestine, known as villous atrophy, hasn't healed — despite your careful diet.

So your physician may start your treatment by checking your body's level of vitamins, minerals and other nutrients, and prescribing nutritional support to help reverse your malnutrition. For up to 60% of patients, this nutritional support includes what's called Total Parenteral Nutrition, which is a nutrient solution delivered directly into a vein, bypassing your gastrointestinal tract.

Your physician may also decide to try what's called a strict elemental diet — a liquid diet that provides nutrients in a hypo-allergenic form already broken down into their basic amino acid building blocks. One small study found that eight of 10 patients with Type I refractory disease showed improvement to their intestinal villi on such a diet; six of those also experienced improvement in their symptoms.

In a few cases involving Type I disease, nutritional support and a very strict gluten-free diet may be all you need to begin healing. But most people also receive drug treatment.

Drug Therapy for Type I Usually Includes Steroids

To date, drug treatment of refractory celiac disease has focused on therapies designed to suppress your immune system in an effort to give your intestines a break from the relentless autoimmune attack. (Remember, it's not gluten itself that causes intestinal damage in celiac disease — it's your immune system's reaction to gluten. In refractory celiac disease, your immune system continues to attack your intestines, even though there's no more gluten in your diet.)

In both Type I and Type II refractory celiac disease, the first-line drug treatment is typically a form of steroid medication known as glucocorticoids. Glucocorticoids are frequently used in the treatment of other autoimmune conditions such as rheumatoid arthritis and inflammatory bowel disease.

Another drug that may be used in your treatment is azathioprine, which also works by suppressing your immune system. People with severe rheumatoid arthritis and those living with a transplanted organ use azathioprine.

Research shows that most people with Type I refractory celiac disease will go into remission — in other words, see their symptoms resolve and their intestines begin to heal — through the use of steroids, possibly combined with azathioprine.

Type II Refractory Celiac Disease Much Harder to Treat

Patients with Type II refractory disease often see some relief from their symptoms from this type of drug regimen, but unfortunately their intestinal lining doesn't heal from it, and it doesn't seem to protect them against the deadly form of non-Hodgkin lymphoma linked with celiac disease.

Other clinical trials have tested a drug called Cladribine — a powerful intravenous chemotherapy drug used for leukemia — in Type II refractory patients. One trial, conducted in the Netherlands, found that Cladribine did calm the disease enough to put it into remission in 18 out of 32 patients. But despite the fact that the drug puts some patients into remission, there's some fear that it may not prevent enteropathy-associated T-cell lymphoma (EATL): that rare, deadly form of lymphoma. EATL afflicts Type II patients disproportionately.

Nonetheless, some clinicians have begun to use Cladribine as their first choice for treating Type II refractory celiac disease, and find that they're able to put about half of their patients into remission with the drug. In that same trial, five-year survival in Type II patients who responded to Cladribine treatment was 83 percent, compared to 22 percent in those who didn't see positive results with treatment. Most significantly, there didn't seem to be an increase in lymphoma rates. However, those treatment results haven't yet been duplicated.

Finally, for those with Type II refractory celiac disease who fail to respond to all other treatments, including Cladribine, at least one celiac center — the same one that published the Cladribine trial results — has tested autologous stem cell transplant, a procedure in which stem cells from your bone marrow are harvested, grown in the lab and then transplanted back into you following high-dose chemotherapy. This is a risky procedure with a high rate of complications, including death.

In the one medical trial involving Type II refractory celiac disease and an autologous stem cell transplant, 11 of 13 patients' symptoms improved significantly within a year of the stem cell procedure. One person died from the transplant procedure itself, and two-thirds of the patients were still alive four years later. One patient developed EATL.

Your Refractory Celiac Treatment May Continue Long-Term

To prevent a relapse of your refractory celiac disease, you may need to take steroids indefinitely. But long-term use of steroids poses its own risks (including high blood sugar, thinning bones and an increased risk of infections). In addition, using steroids for a long time increases your risk of lymphoma — and of course, refractory celiac patients are already at a much higher than normal risk for this form of cancer.

Researchers are therefore exploring other potential drug treatments for refractory celiac disease, including a medication called mesalazine, an anti-inflammatory drug currently used to treat inflammatory bowel disease that's been tested in Type I patients. More research on this rare form of celiac disease will likely be released in the near future.

In the meantime, regardless of the long-term treatment that's recommended for you, you'll need to keep in close contact with your physician and be on the lookout for any signs (such as renewed weight loss and diarrhea) that your refractory celiac disease is going out of remission.

Sources:

Malamut G. et al. Presentation and long-term follow-up of refractory celiac disease: comparison of type I with type II. Gastroenterology. 2009 Jan;136(1):81-90. doi: 10.1053/j.gastro.2008.09.069. Epub 2008 Oct 7.

Malamut G. et al. Refractory celiac disease. Gastrointestinal Endoscopy Clinics of North America. 2012 Oct;22(4):759-72. doi: 10.1016/j.giec.2012.07.007. Epub 2012 Aug 24.

Mauriño E. et al. Azathioprine in refractory sprue: results from a prospective, open-label study. American Journal of Gastroenterology. 2002 Oct;97(10):2595-602.

Mooney P.D. et al. Treatment failure in coeliac disease: a practical guide to investigation and treatment of non-responsive and refractory coeliac disease. Journal of Gastrointestinal and Liver Diseases. 2012 Jun;21(2):197-203.

Roshan B. et al. The incidence and clinical spectrum of refractory celiac disease in a north american referral center. American Journal of Gastroenterology. 2011 May;106(5):923-8. doi: 10.1038/ajg.2011.104. Epub 2011 Apr 5.

Tack G.J. et al. Evaluation of Cladribine treatment in refractory celiac disease type II. World Journal of Gastroenterology. 2011 Jan 28;17(4):506-13. doi: 10.3748/wjg.v17.i4.506.

Tack G.J. et al. Auto-SCT in refractory celiac disease type II patients unresponsive to cladribine therapy. Bone Marrow Transplantation. 2011 Jun;46(6):840-6. doi: 10.1038/bmt.2010.199. Epub 2010 Sep 6.

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