Teduglutide in Enterocolitis Secondary to Graft-Versus-Host Disease Post-Bone Transplant: Case Report

Teduglutide may show promise in improving intestinal symptoms in a pediatric patient with refractory GI-GVHD, but further studies are needed to confirm its effectiveness.

Summary and findings

This case report describes the use of teduglutide in a 3-year-old patient with refractory gastrointestinal graft-versus-host disease (GVHD) following haploidentical hematopoietic stem cell transplantation. The patient received a dose of 0.9 mg subcutaneously once daily. Improvements in stool consistency, frequency, intestinal inflammation, and nutritional status were noted, but the patient ultimately died from unrelated pulmonary complications.

Abstract

<h4>Background: </h4> Gastrointestinal graft-versus-host disease (GVHD) is one of the most severe complications of hematopoietic stem cell transplantation (HSCT), particularly in pediatric patients. It is frequently associated with treatment refractoriness and prolonged dependence on parenteral nutrition. Teduglutide, a GLP-2 analog, has shown promise in intestinal mucosal regeneration, but its use in GVHD remains limited. This case report describes the clinical experience with teduglutide in a pediatric patient with refractory GVHD-associated enterocolitis. <h4>Methods:</h4> We report the case of a 3-year-old child with primary immunodeficiency (Hyper-IgM Syndrome – CD40L deficiency) who underwent haploidentical HSCT and subsequently developed grade III gastrointestinal GVHD. The patient presented with severe chronic diarrhea, rectal prolapse, extensive intestinal inflammation, and nutritional failure despite standard immunosuppressive therapy. Teduglutide was initiated on a compassionate basis at a dose of 0.9 mg subcutaneously once daily. <h4>Results:</h4> Following initiation of teduglutide, the patient showed sustained improvement in stool consistency and frequency, reduced signs of intestinal inflammation, nutritional stabilization, and resolution of rectal prolapse episodes. Clinical response was maintained until the patient's death due to pulmonary infectious complications unrelated to gastrointestinal involvement. <h4>Conclusions:</h4> The positive intestinal response observed in this case supports the potential role of teduglutide as an adjunctive therapy for mucosal recovery in refractory GI-GVHD. Although encouraging, its use in this setting warrants further investigation through controlled studies, particularly in pediatric populations.

Limitations

Not reported in abstract.

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