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P567. Allogeneic haematopoietic stem cell transplant (HSCT) for paediatric Crohn's disease: A case report

S. Chadokufa1, B. Huggett1, M. Mamoun Elawad2, J. Kammermeier1, F. Kiparissi2, N. Shah2, 1Great Ormond Street Hospital, Paediatric Gastroemterology, London, United Kingdom, 2Great Ormond Street Hospital, Gastro, London, United Kingdom

Background

Remission of several autoimmune diseases has been described after allogeneic HSCT. There are few reports in literature of curing IBD following HSCT for coexisting conditions. HSCT is an established treatment for IL-10 and IL-10R defects leading to intractable ulcerating colitis. We report a case of a male patient with severe early-onset Crohn's disease (CD) with no known genetic condition who underwent a successful matched unrelated HSCT.

Methods

An 8 year old boy born to non-consanguineous parents, developed CD ileo-colitis at aged 4. Initially treated with steroids, azathioprine and aminosalicylates. This Primary therapy was administered over 2 years with partial clinical response, but no mucosal healing evident. He remained steroid dependent. Aged 6 he underwent an emergency subtotal hemi-colectomy and ileostomy. Symptoms persisted, ileostomy prolapsed and ulcerated causing severe pain. Treatment was then escalated with Infliximab. There was some response, but anaphylaxis led to switch to Adalimumab. There was some response to Adalimumab but symptoms persisted, & he became steroid dependent and developed hypertension. His quality of life was poor and was unable to attend school.

Despite intensified treatment he had developed various extra-intestinal manifestations such as arthritis and debilitating pyoderma gangrenosum. He was assessed and enrolled on the genetic diagnostic study. Exclusion of immunological deficiency and other known genes for early onset IBD was made. Following assessment by the immunology, gastroenterology and BMT teams a decision was made to proceed for HSCT. The patient underwent reduced intensity conditioning using Treosulfan, Fludarabine and Alemtuzumab. GVHD prophylaxis treatment was Ciclosporin & MMF. Pre-transplant endoscopic showed severe active oesophagitis and chronic pan colitis.

Results

At 3 months, pyoderma gangrenosum healed at 3 and endoscopic assessment revealed only focal gastritis. Now 9 months post-transplant he is on a weaning dose of Ciclosporin for skin GVHD. He is clinically well with good energy level and is eating normally. He is planned for closure of his stoma once he is off Ciclosporin.

Conclusion

This is the first case report on successful allogeneic HSCT for severe refractory CD. Our patient had no major complications of HSCT and is on minimal immunosuppression 9 months post. Therefore we suggest that HSCT may be curative to patients with refractory CD with poor quality of life, developed complications related to immunosuppressive treatment and/or have exhausted medical therapy. Careful selection of patients and discussion in multi-professional teams are crucial to the success of this intensive treatment. A longer follow is needed to ensure that current remission is sustained.