Management of Refractory Celiac Disease

Karoly Horvath, MD, PhD, Alessio Fasano, MD, Division of Pediatric Gastroenterology and Nutrition, Department of Pediatrics, University of Maryland School of Medicine, Baltimore, Maryland.

Disclosures
In This Article

Therapeutic Options

Patients must adhere to a strict gluten-free diet. A number of additional therapeutic interventions have been proposed; however, there are no randomized, placebo-controlled, multicenter therapeutic trials published on RS.

Elemental Diet

Anecdotal reports have been published in which patients affected by refractory CD experienced remarkable clinical and histologic improvement on elemental diet. The beneficial effect of elemental diet has been demonstrated in other chronic intestinal inflammatory diseases, such as Crohn's disease.

Total Parenteral Nutrition

Total parenteral nutrition (TPN) should be initiated in patients with weight loss, unresponsive nutritional deficiencies, hypoproteinemia, and worsening clinical symptoms. In a study by Cellier and colleagues, 10 patients with RS received TPN for 1-84 months (average = 21 months). These patients had either steroid resistance or dependence. Although 7 of these individuals had clinical response, none had histologic improvement.

Steroids

If the diagnosis of collagenous sprue can be established, therapy with steroids should be attempted. However, there are some concerns that steroid therapy may in fact mask the signs of intestinal lymphoma.

A combination of gluten-free diet and oral steroids (0.5-1.0 mg/kg/day of prednisolone) was used in 15 patients; 11 showed clinical improvement. However, histologic recovery occurred in only 3 of the 14 patients who had repeat biopsies.

Steroid therapy appears to be successful in patients who do not have the abnormal clonal IEL phenotype (described previously). The 3 patients without aberrant clonal IEL had complete clinical and histologic recovery after 6-36 months of steroid treatment plus gluten-free diet.

Interferon-alpha

Two French patients received this therapy in doses similar to those used for treatment of hepatitis C (ie, 3 million units given 3 times per week for 3-6 months), but neither had favorable response.

Immunosuppressive Therapies

Azathioprine. Azathioprine is used as a steroid-sparing agent in inflammatory bowel disease. A similar application -- to allow tapering of corticosteroids to lower doses -- has been reported for patients with RS. However, the response rate to this therapeutic course has been found to vary.

Cyclosporine. Wahab and colleagues treated 13 RS patients with cyclosporine. Eight patients reported a clinical response, 6 of whom had concomitant histologic improvement. Overall then, 8 of the 13 patients responded histologically to cyclosporine treatment. Normalization of villi was demonstrated in 5 individuals; however, 3 needed prolonged treatment. No serious side effects were documented in those patients whose serum cyclosporine level was maintained in the 100-200 ng/mL range.

Chemotherapy

Patients who are suspected of having cryptic T-cell lymphoma should be followed closely and treated with chemotherapeutic regimens. There is no specific protocol published for patients with RS who have developed lymphoma; their treatment is similar to that for those patients who have non-CD-associated intestinal lymphoma.

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