Pentostatin therapy for steroid-refractory acute graft versus host disease: identifying those who may benefit. (March 2018)

• Record Type: Journal Article
• Authors: Ragon, Brittany; Mehta, Rohtesh; Gulbis, Alison; Saliba, Rima; Chen, Julianne; Rondon, Gabriela; Popat, Uday; Nieto, Yago; Oran, Betul; Olson, Amanda; Patel, Krina; Hosing, Chitra; Qazilbash, Muzaffar; Shah, Nina; Kebriaei, Partow; Shpall, Elizabeth; Champlin, Richard; Alousi, Amin
• Is Part Of: Bone marrow transplantation. Volume 53:Number 3(2018)
• Page Start: 315
• Page End: 325
• Publication Date: 2018-03
• Subjects: Bone marrow -- Transplantation -- Periodicals; 617.4410592
• DOI: 10.1038/s41409-017-0034-z
• Languages: English
• ISSNs: 0268-3369

Abstract

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.