But most are reluctant to eat gluten or have a biopsy
By Amy Ratner, Medical and Science News Analyst
People who have celiac disease are willing to participate in celiac disease research, but less so if a study involves eating gluten or getting a biopsy, a new survey by Beyond Celiac has found.
The survey of about 4,500 adults with celiac disease was done in advance of the recent Beyond Celiac Research Summit and results were shared there. The summit was held to identify the sticking points that have prevented clinical trials from resulting in new treatments for patients. Nearly 500 parents of children with celiac disease also completed a separate survey.
Only about 9 percent of both adults and parents said they would not participate or allow participation in any kind of celiac disease research.
Overall, nearly 64 percent of adults surveyed said they would be willing to participate in a clinical trial for a drug to treat celiac disease. But the number dropped in almost every case when people were asked about specific clinical trial requirements.
|Clinical trial: A study in which participants receive specific drugs or other treatments according to a research plan created by the investigators. Study participants who receive the treatment are often compare to study participants who get a placebo, which mimics the pill or injection but does not contain the actual drug.|
About 13 percent said they would be willing to consume some amount of gluten for four to six weeks. Double that, about 26 percent, said they would be willing to do a shorter gluten challenge of one to three doses. Nearly 40 percent said they would be willing to have an endoscopy and biopsy.
Those willing to take a medication, keep a symptom diary, share their past medical records or take a stool test hovered around 50 percent.
The largest number, 86 percent, were willing to complete surveys, followed by 69 percent who would take a blood test and nearly 62 percent who would take a urine test.
When the survey was broken down by gender, with about 3,700 women and 700 men, men were more willing to complete components of clinical trials that overall were less appealing to celiac disease patients — consuming a gluten challenge and taking a medication. They were less willing to keep a symptom diary, complete surveys or take a blood test.
“We are grateful that so many people responded to the survey, both adults and parents because the patient voice in research needs to be heard,” said Alice Bast, Beyond Celiac chief executive officer. “The information shared can help us focus on what patients need to know about different aspects of clinical trials.”
It’s also important to meet patients where they are and let them know there are a number of different ways to contribute to the study of celiac disease, Bast noted. For researchers, the survey reveals how recruitment of men might have an impact on participation in clinical trials, Bast said.
Responses from parents of children with celiac disease parallel those from adults. Parents would be reluctant to let their children have a long gluten challenge, with only 8 percent saying they would allow it. Nearly 20 percent would allow a shorter challenge. Additionally, 20 percent would agree to a biopsy for their children as part of a study.
Parents would also hesitate to have children take a medication, with about 25 being willing. They would be most willing to fill out symptom diaries, share past medical records, and have their children have blood, urine and stool tests.
Involving children in clinical trials for celiac disease, which is often neglected, was one of the summit topics. Pediatric celiac disease experts argued that children can and should be included in clinical trials.
Several observational studies of children with celiac disease are underway, including the Celiac Disease Genomic Environmental Microbiome and Metabolic Study (CDGEMM) being done by the Center for Celiac Research at Massachusetts General Hospital, MassGeneral Hospital for Children, and the Celiac Program at Harvard Medical School. Researchers are following at-risk children from birth, collecting stool and blood samples to try to determine how celiac disease develops. Originally designed to follow the babies for 5 years, the study was recently expanded to track the children for a decade.
Clinical trials investigating new treatments for celiac disease often require study participants to keep a symptom diary and have biopsies, consume a gluten challenge and take a medication.
Other types of studies allow a patient to continue on their normal gluten-free diet without a challenge. For example, in the so-called Doggie Bag study being done by researchers from Boston Children’s Hospital and colleagues, patients provided samples of food they ate, which were tested for gluten content. Study participants then gave stool and urine samples to measure whether they were exposed to gluten.
Some research is based on surveys and symptom reporting alone. In most studies, more women than men participate. Overall, more women have been diagnosed with celiac disease.
Scientists investigating celiac disease have often said their success depends patients on who are willing to get involved in research.
At the summit, several people with celiac disease who had completed clinical trials shared their stories. In the survey, about 5 percent said they had been part of a study.
The online survey was sent out in October via email and social media by Beyond Celiac and the Canadian Celiac Association. In addition to asking about willingness to participate in clinical trials, the survey included questions about demographics, diagnosis, gluten exposure and the difficulties associated with having celiac disease.
More than half of adults surveyed said celiac disease has caused them to skip an activity or experience in the previous month and had at some point led them to sacrifice things like a new job, vacation or an opportunity to move.
About 58 percent of parents said celiac disease had dictated decisions for their family, including skipping a vacation or which school their child should attend. About 42 percent of children had missed an activity in the previous month.
Unintentionally consuming gluten was also frequently reported, with about a quarter of adults and 16 percent of parents of children saying accidental gluten exposure happened at least twice a month. Another 20 percent of both groups said they were not sure how often gluten exposure had occurred.
The fact that it is nearly impossible to completely avoid gluten from cross-contact even on a strict gluten-free diet and the impact that has on health and quality of life for those who have celiac disease is one of the reasons alternative treatments are needed are being pursued by celiac disease researchers.