Advanced POEMS syndrome treated with high-dose melphalan followed by autologous blood stem cell transplantation: a single-center experience

Abstract

BackgroundPOEMS syndrome is a rare paraneoplastic syndrome associated with plasma cell dyscrasia. High-dose chemotherapy followed by autologous stem cell transplantation has shown encouraging efficacy in the treatment of patients with POEMS syndrome. However, there are minimal reports on clinical outcomes after autologous stem cell transplantation for patients with advanced disease and very poor performance status.MethodsWe retrospectively evaluated 9 advanced POEMS syndrome patients, who had an Eastern Cooperative Oncology Group performance status score of 3 or 4, and were treated with high-dose melphalan therapy followed by autologous stem cell transplantation from 2004 to 2011.ResultsEight patients achieved initial hematologic response, 4 of whom had complete responses. At a median follow-up of 44 months (range, 8-94 months), 7 patients were alive, with 3-year overall survival rate of 77.8%. There were no hematologic relapses in the survivors. One patient died of disease progression; the other died of pneumonia despite a hematologic response 3 months after autologous stem cell transplantation. All survivors achieved improvement in general performance status and in clinical response.ConclusionHigh-dose melphalan followed by autologous stem cell transplantation can be considered a valid treatment option even for patients with advanced POEMS syndrome.

Update on the POEMS syndrome

“…At the time of transplantation, performance status has been identified as a risk factor for disease progression [ 34 ]. A Korean study in which ASCT was performed for the POEMS patients with advanced disease and poor performance status showed a 3-year overall survival rate of 77.8% [ 35 ]. ASCT, melphalan plus dexamethasone, lenalidomide, and dexamethasone were compared, and there was no survival difference.…”

Update on the Diagnosis and Treatment of POEMS (Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal Gammopathy, and Skin Changes) Syndrome

“…High-dose melphalan (140-200 mg/m 2 ) followed by autologous hematopoietic cell transplant (HCT) has been shown to be 1 of the most effective available therapies for eligible patients (Table). In a large series of 59 patients with POEMS syndrome who underwent HCT, most of whom received melphalan at a dose of 200 mg/m 2 ,more than 90% responded, with overall survival of 94% and 5-year progression-free survival of 25% . After HCT, VEGF levels and fluid overload improved more quickly (within 3 months) than neurologic response, which took up to 3 years to improve.…”

Autologous peripheral blood stem cell transplantation and the role of lenalidomide in patients affected by poems syndrome

“…Several published series reported successful mobilization and collection through chemo-mobilization, as well as with the use of the granulocyte colony stimulating factor (G-CSF) alone, in patients with POEMS syndrome (Table 2). 7,10,11,26,28,29,31,32 Patients mobilized with chemotherapy were usually treated with cyclophosphamide at doses ranging between 2 and 4 g/m 2 followed by G-CSF at doses of 5 or 10 μg/kg per day.…”

“…A study published in 2014 by Jang et al retrospectively evaluated 9 patients with advanced POEMS syndrome treated with HD melphalan followed by ASCT from 2004 to 2011. At a median follow‐up of 44 months (range 8–94 months), 7 patients were alive, with 3‐year OS rate of 77.8%.…”