The use of the immunosuppressive agent vedolizumab in treatment of gastrointestinal (GI) acute graft-versus-host disease (aGVHD) in 3 pediatric patients was described in a recent case report published in the International Journal of Hematology. This treatment appeared to be safe in these patients and was reportedly an effective treatment for 2 of the children.

Vedolizumab is a monoclonal antibody used to treat inflammatory bowel disease through inhibition of T-lymphocyte migration to the gut. The researchers explained that this agent has also shown efficacy in treating GI-aGVHD in adults. At their institution in Tokyo, Japan, they administered vedolizumab to 3 patients aged 1.5 to 4.4 years for GI-aGVHD.

All 3 patients underwent bone marrow transplantations (BMT); one case involved an 8/8 human leukocyte antigen (HLA)-matched unrelated donor and the other 2 involved an HLA-mismatched unrelated donor. Each patient had received tacrolimus and methotrexate as GVHD disease prophylaxis, and conditioning regimens varied among the patients.

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Patient One developed grade 2 aGVHD (GI stage 1, skin stage 2) at day 26 following transplantation. On day 32, methylprednisolone was administered, but by day 40 the aGVHD worsened, progressing to grade 3. The methylprednisolone dosage was increased, and the patient was given human mesenchymal stem calls. The child’s skin condition improved but the GI-aGVHD did not. At day 100, the patient was begun on a regimen of intravenous vedolizumab (177 mg/m2/dose), with stool improvement reported on day 113. The patient received multiple treatments with vedolizumab, eventually at 8-week intervals, and experienced no adverse events related to this agent.

Patient Two presented on day 132 with diarrhea, and GI-aGVHD was diagnosed. Prednisolone, methotrexate, and enteric-coated budesonide were given but the diarrhea did not completely resolve, and the patient experienced a recurrence when the prednisolone dosage was reduced. By day 183, the patient had developed stage 4 GI-aGVHD with bloody stools. On day 205, a first dose of intravenous vedolizumab (177 mg/m2/dose) was administered. The child developed elevated serum C-reactive protein, and abdominal radiography suggested enteritis. The second vedolizumab dose had been postponed, but the C-reactive protein level normalized on its own. On day 231 vedolizumab was continued but did not satisfactorily treat the GI-aGVHD, and other therapies were required.

Patient Three presented with diarrhea on day 188, and GI-aGVHD was diagnosed. Treatments involving several agents were attempted, including prednisolone at 2 dosages, methotrexate, budesonide, methylprednisolone, mycophenolate mofetil, cyclosporine, and infliximab, but the GI-aGVHD was refractory. On day 274, a vedolizumab regimen (177 mg/m2/dose) was initiated, eventually administered at 8-week intervals, and the patient experienced immediate improvement in diarrhea. By the second and seventh doses of vedolizumab, improvements were visible by colonoscopy. The patient reportedly experienced no adverse events associated with vedolizumab.

“Our experience suggests a potential efficacy and feasibility of vedolizumab for refractory GI-aGVHD in children,” the researchers concluded. However, they suggest more information is needed to confirm these results.


Isshiki K, Kamiya T, Endo A, et al. Vedolizumab therapy for pediatric steroid‑refractory gastrointestinal acute graft‑versus‑host disease. Int J Hematol. Published online November 1, 2021. doi:10.1007/s12185-021-03245-0