Abstract
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a lifesaving procedure in malignant and nonmalignant diseases. However, it is associated with a considerable risk of graft-versus-host disease (GvHD). Steroids are a first-line therapy for acute GvHD (aGvHD), but there is no standard treatment for steroid-resistant (SR) gastrointestinal (GI) aGvHD, which has a poor prognosis. The anti-integrin antibody, vedolizumab, could help in controlling SR GI aGvHD symptoms by blocking lymphocyte extravasation and infiltration of the intestinal wall. To report the outcomes of 3 children with SR GI aGvHD after allo-HSCT, treated with vedolizumab as the last chance drug. The study included 3 patients aged from 8 to 10 years who underwent HSCT in Department of Pediatric Bone Marrow Transplantation, Oncology and Hematology at Wroclaw Medical University, Poland, and who developed severe SR GI aGvHD. All patients had grade IV SR aGvHD with GI stage 4 manifestation. Vedolizumab was given as salvage therapy after an ineffective treatment with etanercept, basiliximab, ruxolitinib, extracorporeal photopheresis, and mesenchymal stem cell infusions. Vedolizumab was administered intravenously at a dose of 300 mg. Only 1 patient achieved GvHD remission and was alive and well 9 months after the discontinuation of the therapy. One child developed a relapse of malignant disease and eventually died, and the third child died of severe aGvHD. Vedolizumab can be safely used in children with SR GI aGvHD, offering an additional chance for heavily pretreated patients. Prospective pediatric studies on both prophylactic and therapeutic use of the drug are warranted, according to the preliminary results.
Highlights
Allogeneic hematopoietic stem cell transplantation is a lifesaving procedure in malignant and nonmalignant diseases
One child developed a relapse of malignant disease and eventually died, and the third child died of severe acute GvHD (aGvHD)
Vedolizumab can be safely used in children with SR GI aGvHD, offering an additional chance for heavily pretreated patients
Summary
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a lifesaving procedure in malignant and nonmalignant diseases. It is associated with a considerable risk of graft-versus-host disease (GvHD). The GvHD first-line treatment consists of steroids and calcineurin inhibitors (CNIs),[11] but up to 50% of patients with aGvHD do not respond to this therapy.[12] In steroid-resistant (SR) aGvHD, multiple therapies have been studied, ranging from high-dose steroids to mono- and polyclonal antibodies (basiliximab, daclizumab, etanercept, anti-thymocyte globulin (ATG), infliximab), extracorporeal photopheresis (ECP), or cellular therapies with mesenchymal stem cells (MSCs).[10,13] Steroid-refractory patients have a poor prognosis, and mortality in such patients reaches 80%.14,15. In steroid-resistant (SR) aGvHD, multiple therapies have been studied, ranging from high-dose steroids to mono- and polyclonal antibodies (basiliximab, daclizumab, etanercept, anti-thymocyte globulin (ATG), infliximab), extracorporeal photopheresis (ECP), or cellular therapies with mesenchymal stem cells (MSCs).[10,13] Steroid-refractory patients have a poor prognosis, and mortality in such patients reaches 80%.14,15 In this paper, we report the outcomes of 3 children with severe SR gastrointestinal (GI) aGvHD, who were treated with vedolizumab
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