Refractory Celiac Disease Responds to Stem Cell Transplant

Celiac.com 04/10/2007 - Patients suffering from refractory celiac disease with aberrant T cells seem to benefit from high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation. Refractory celiac disease with aberrant T cells has generally proven resistant to known celiac therapies, and patients are at high risk for developing enteropathy associated T-cell lymphoma. The small pilot trial was conducted by Dr. Abdulbaqi Al-toma and colleagues from VU University Medical Center, Amsterdam.

The study followed seven patients whose mean average age was 52.5 years old at the time of the procedure, and followed them for an average of 15.5 months (the lowest follow-up time was 7 months, the longest was 30 months). According to the study, there was no transplantation-related mortality, and only mild cases of transplantation-related toxicity. A one-month post-procedure follow-up showed remarkable clinical improvement all patients, including disappearance of abdominal pain, normalization of stool frequency, and improvement of biochemical markers.

The research team also noted that post-transplant histology of the small intestine revealed marked regeneration coupled with a disappearance of erosions and ulcerations. Furthermore, at 3 to 4 months, post-transplantation tests showed a decline in aberrant T cells from a mean of 63% at baseline to 38%. Additionally, at 2 years, tests for the first hematopoietic stem cell transplant patient showed continuing declines in aberrant T cells (to 3%).

It should be noted that one subject of the study showed no declines in aberrant T cell percentages, histology examination or CD8+ cells, and that the patient died 8 months after the stem-cell transplant.

The research team concluded that the promising short-term results enjoyed by this small test group warrants a longer-term follow-up to properly assess the significance of the findings.

Blood 2007;109:2243-2249.