Impact of pretransplant induction therapy on autologous stem cell transplantation for patients with newly diagnosed POEMS syndrome

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Abstract

POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes) is a rare plasma cell dyscrasia sometimes treated with a haematopoietic cell autotransplant. We analyzed data from 138 subjects with newly diagnosed POEMS syndrome receiving a autotransplant at our center. Thirty-two subjects with severe end-organ dysfunction ineligible for immediate autotransplant received pretransplant therapy, which made a subsequent autotransplant feasible. Pretransplant therapy resulted in vascular endothelial growth factor (VEGF) remissions in 15 (47%). Thirty-three transplant recipients (24%) had early posttransplant complications. Risk factors for these complications identified through multivariate analysis included age > 50 years (odds ratio (OR) 2.79, 95% confidence interval (CI) 1.09-7.14; P = 0.033), time from symptom onset to transplant > 5 years (OR 4.71, 95% CI 1.10-20.18; P = 0.037) and pleural effusion (OR 3.39, 95% CI 1.26-9.12; P = 0.016). Subjects receiving pretransplant therapy had fewer early complications than those who did not (OR 0.17, 95% CI 0.04-0.71; P = 0.015), especially in subjects with a VEGF remission (OR 0.05, 95% CI 0.01-0.49; P = 0.010). Autotransplants resulted in hematological remission in 60 (50%), VEGF remissions in 76 (72%) and improvements in other organ functions (65-90%). The 5-year progression-free survival (PFS) and overall survival were 76% (95% CI 64-84%) and 94% (95% CI 87-97%), respectively. Hematological (5-year PFS 83 vs 66%, P = 0.008), VEGF (5-year PFS 79 vs 57%, P = 0.021) remissions and especially both (5-year PFS 95 vs 61%, P = 0.004) were associated with better PFS.

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