Translating Innovation Into Practice: Emerging Treatments and Real-World Data in GVHD - Episode 3

Case 1: Multiorgan cGVHD With Pulmonary Involvement

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Panelists discuss how a 52-year-old male patient developed multiorgan chronic GVHD 9 months post transplant with skin tightness, joint pain, and pulmonary involvement that showed minimal response to initial prednisone therapy and only moderate improvement after adding ruxolitinib, highlighting the importance of early detection through patient education and regular monitoring including spirometry to prevent irreversible fibrosis.

Case 1: Multiorgan cGVHD With Pulmonary Involvement

This segment presents a case study of a 52-year-old man who underwent myeloablative allogeneic transplant for acute myeloid leukemia. The patient received modern graft-vs-host disease (GVHD) prophylaxis with posttransplant cyclophosphamide, tacrolimus, and mycophenolate mofetil, representing current standard of care. Nine months post transplant, he developed chronic GVHD with multiorgan involvement including mildly tight skin with sclerotic features, joint pain without significant range of motion restriction, and moderate pulmonary involvement with reduced FEV1 and dyspnea on exertion. Initial treatment with prednisone 1 mg/kg daily showed minimal improvement after two weeks, accompanied by typical steroid adverse effects, including weight gain and insomnia.

The discussion emphasizes critical challenges in chronic GVHD management related to patient geography and monitoring. Both specialists noted that patients from large catchment areas eventually return home after the initial 100-day posttransplant monitoring period, creating gaps in surveillance during the typical onset period for chronic GVHD. Patient education becomes paramount, including providing educational materials and connecting patients to reliable resources like Be the Match (NMDP) websites. The speakers stress that early detection and treatment are crucial before fibrosis develops, as advanced fibrotic changes become extremely difficult to reverse even when symptoms can be controlled.

Ruxolitinib 10 mg twice daily was added as second-line therapy when steroids proved inadequate, showing moderate improvement at four weeks but with persistent dyspnea and unchanged skin tightness. The case illustrates typical presentation timing during immunosuppression tapering and highlights pulmonary involvement as a particularly concerning manifestation. While less common than skin involvement, pulmonary chronic GVHD causes disproportionate morbidity and mortality, significantly impacting quality of life and requiring aggressive monitoring. The specialists recommend routine spirometry every 3 months during the first year post transplant per guidelines, as FEV1 decline greater than 10% may occur before patients report symptoms, enabling earlier intervention.