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Pentostatin therapy for steroid-refractory acute graft versus host disease: identifying those who may benefit

Bone Marrow Transplantation volume 53, pages 315–325 (2018)Cite this article

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Abstract

We report outcomes of 60 patients with steroid-refractory (SR)-aGVHD treated with pentostatin. Almost half (47%) of patients had grade 4 GVHD—22% had stage 3–4 liver GVHD and 51% had stage 3–4 lower gastrointestinal tract (LGI) GVHD. Patients received a median of 3 courses (range, 1–9) of pentostatin. Day 28 overall response rate (ORR) was 33% (n = 20) (complete response 18% (n = 11), partial response 15% (n = 9)). Non-relapse mortality was 72% (95% confidence interval (CI) 61–84%) and overall survival (OS) was 21% (95% CI 12–32%) at 18 months. On univariate analysis, age >60 years (HR 1.9, 95% CI 1.01–3.7, p = 0.045) and presence of liver GVHD (HR 1.9, 95% CI 1.9, 95% CI 1.5–3.3, p = 0.03) were significant predictors of poor OS while patients with LGI GVHD had superior OS than those without (HR 0.4, 95% CI 0.2–0.8, p = 0.01). On stratified analysis, patients <60 years with isolated LGI GVHD had the best outcomes with an ORR of 48% and OS of 42% at 18 months. Among older patients, OS was 14% in those with isolated LGI aGVHD and 0% in others. Pentostatin remains a viable treatment option for SR-aGVHD, especially in patients 60 years or younger with isolated LGI involvement.

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    Brittany Knick Ragon, Rohtesh S. Mehta, Alison M. Gulbis, Rima M. Saliba, Julianne Chen, Gabriela Rondon, Uday R. Popat, Yago Nieto, Betul Oran, Amanda L. Olson, Krina Patel, Chitra M. Hosing, Muzaffar H. Qazilbash, Nina Shah, Partow Kebriaei, Elizabeth J. Shpall, Richard E. Champlin & Amin M. Alousi

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Ragon, B.K., Mehta, R.S., Gulbis, A.M. et al. Pentostatin therapy for steroid-refractory acute graft versus host disease: identifying those who may benefit. Bone Marrow Transplant 53, 315–325 (2018). https://doi.org/10.1038/s41409-017-0034-z

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