Exciting advancements in cell therapy for autoimmune diseases are offering hope to children with severe monogenic inflammatory bowel disease (IBD). A recent study revealed that among a cohort of patients, some had macroscopic ileal disease, upper gastrointestinal involvement, and perianal disease. Prior to hematopoietic stem cell transplant (HSCT), many patients had undergone luminal IBD surgery or had permanent ileostomies. The majority had received immunosuppressive therapy, including steroids and biologics/small molecules/immunomodulators. Interestingly, some patients, such as those with chronic granulomatous disease (CGD), were managed with antibiotics instead of anti-tumor necrosis factor α. At the time of transplant, some patients still had moderate/severe disease, while others, particularly those with CGD, were in remission. None of the patients were in sustained medication-free remission prior to HSCT. This research highlights the potential of stem cell transplant as a promising treatment option for children battling monogenic IBD.
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