Grade 4 steroid refractory gut graft-versus-host disease (GVHD) is associated with significant mortality and morbidity. We successfully managed a patient with multiple anti-GVHD medications, ultimately achieving a positive response with combination of ruxolitinib, vedolizumab, and supportive care. Effective nursing care played a crucial role, providing the necessary time for these medications to take effect.
A 3-year-old girl with combined immunodeficiency presented with severe Pneumocystis jirovecii pneumonia, eczema, failure to thrive, recurrent respiratory viral infections, and CMV viremia and colitis at 12 months. Immune investigations revealed hypogammaglobulinemia. Definitive genetic is testing still ongoing. Due to the absence of a matched sibling donor, she underwent an unrelated (9/10) peripheral blood stem cell transplant. The conditioning regimen included treosulfan, fludarabine, and cyclophosphamide, with GVHD prophylaxis involving antithymocyte globulin (ATG), methotrexate, and tacrolimus. On Day +12, she experienced engraftment syndrome, presenting with fever, rash, and increased oxygen requirement, which was managed with three days of methylprednisolone. Successful engraftment occurred on Day +17. However, by Day +21, she developed diarrhea, escalating to grade 4 gut GVHD within 72 hours. Her stool output peaked at approximately 4 liters per day, including blood, necessitating three daily infusions of albumin (0.5g/kg) to maintain serum albumin levels above 20 g/L. Despite initiating standard treatment with methylprednisone at 2 mg/kg/day, there was no response by day five. Therefore, multiple anti-GVHD agents were administered, including infliximab, etanercept, basiliximab, alpha-1 antitrypsin, ATG, ruxolitinib, and vedolizumab. Throughout 8-10 weeks, she received comprehensive care, which included fluid and electrolyte management, transfusion support, opioids for pain management, total parenteral nutrition, and exemplary supportive and nursing care. Remarkably, she did not develop sepsis despite being on immunosuppression, nor did she have bed sores or perianal abscesses. During this period, she experienced reactivation of both EBV and CMV, requiring treatment with rituximab and ganciclovir. Ultimately, her condition began to improve, and her stool output decreased. By Day +180, she was discharged from the hospital. Immunosuppression was fully discontinued by Day +270. One year post-transplant, she demonstrated full donor CD3/CD33 chimerism and 50% CD19 donor chimerism. She is no longer on intravenous immunoglobulin (IVIG), has begun her immunizations, and shows no signs of chronic GVHD.
Despite the high mortality associated with grade 4 gut GVHD, dedicated perseverance, relentless persistence, and exceptional nursing care can lead to positive outcomes, providing sufficient time for immunosuppressive medications to take effect.
