Intestinal outcome of bone marrow transplantation for monoge
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Some monogenic inflammatory bowel diseases (IBDs) have been shown to be resistant to medication. While allogeneic hematopoietic stem cell transplantation (alloHSCT) has become a curative therapeutic choice for certain monogenic IBDs, its efficacy in terms of endoscopic improvements has yet to be investigated.

Patients with monogenic IBDs who received alloHSCT at the National Center for Child Health and Development had their clinical course and endoscopic results reviewed retrospectively. The weighted Pediatric Crohn's Disease Activity Index (wPCDAI) was used to assess clinical disease activity, and the Simple Endoscopic Score for Crohn's Disease (SESCD) was used to assess endoscopic findings. Endoscopic remission was defined as a SESCD of 2 or less, and clinical remission was defined as a wPCDAI of less than 10.

Results:
--4 patients with severe monogenic IBDs, including 3 with X-linked inhibitor of apoptosis protein (XIAP) deficiency and one with interleukin-10 signaling defect, were treated with allo-HSCT with reduced intensity conditioning.

--In 4 patients, the maximum scores of wPCDAI and SES-CD before allo-HSCT ranged from 67.5 to 120 and 20 to 34, respectively.

--After allo-HSCT, all 4 patients showed a significant improvement in intestinal inflammation and achieved both clinical and endoscopic remission.

--Although patients with XIAP deficiency presented with post-transplant hemophagocytic lymphohistiocytosis and a relatively late engraftment, all patients achieved prolonged clinical remission, and IBD medications were successfully discontinued in all patients.

Finally, AlloHSCT for monogenic IBD resulted in full clinical resolution and mucosal healing as verified by endoscopic examination.

Source: https://onlinelibrary.wiley.com/doi/10.1111/ped.14750?af=R
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