The Role Of High Dose Chemotherapy and Autologous Stem Cell Transplantation (ASCT) In Patients With POEMS Syndrome: A Retrospective study Of The Plasma Cell Disorders Subcommittee Of The Chronic Malignancy Working Party Of The EBMT
Authors: Cook Gordon , Garderet Laurent , Iacobelli Simona , Morris Curly , Kroeger Nikolaus , van Biezen Anja , Rovira M , Byrne Jenny , Bordessoule Dominique , Leone Giuseppe , Nagler Arnon , Cook Mark , Legros Laurence , Ossenkoppele Gert J. , Sanz R Gacria , Hoek Jennifer , Castagna Luca 
Published: October 2019

Abstract

Introduction
Polyneuropathy, organomegaly, endocrinopathy, dermopathy associated with a paraproteinaemia (POEMS syndrome) is a rare paraneoplastic syndrome secondary to a plasma cell dyscrasia. Effective treatment of the underlying plasma cell dyscrasia, including ASCT, can control the disease and often dramatically control symptoms though limited data is available for ASCT in POEMS.
Specific Aim
The aim of this study was to describe the clinical outcome of ASCT for patients with POEMS syndrome. We wish to determine the impact of patient and disease-specific factors on prognosis and effectively measure the extent of systemic disease involvement and organ-specific responsiveness of ASCT.
Methodology
Patient-, disease-, and transplant-related variables were collected according to the data entries in the EBMT database, including tracking incomplete data entries from participating centers. Systemic involvement and organ-specific response to ASCT was detailed utilizing an organ involvement tool pre- and post-ASCT.
Results
127 patients underwent an ASCT between 1997-2010 and satisfied the entry criteria. The median age was 50 years (range 26-69) with 51.2% ≤50 years of age. The extent of systematic disease involvement was: peripheral neuropathy in 58.6%, volume overload in 66.2%, organomegaly in 48.3%, papilledema in 19.8%, dermopathy in 46.6% and 34.5% had sclerotic bone lesions at presentation. The median time from diagnosis to ASCT was 7.5 months (range 1-346) with 31.5% of patients receiving an ASCT >12 months from diagnosis. The graft source was PBSC in 100% of patients. Disease status at ASCT was: 47.5% CR/PR, 34% SD/MR/untreated and 18.4% in PD (missing information in 19% of patients). The conditioning regimen was Melphalan ≥200mg/m2 in 52.5%, Melphalan <200mg/m2 in 9.3% (38.1 of data on dose is missing) and TBI-containing in only 1 patient. The total rate of engraftment at 3 months was 96.8%, with 2 patients (1.6%) dying before engraftment. Engraftment syndrome was documented in 37% of ASCT recipients including peri-engraftment fever in 23.4% & pulmonary infiltrates in 4.8%. Haematological complete response (CHR) was characterized in 25.2% with 16.5% having progressive disease or died without attaining CHR. CHR was demonstrated in13.3% at 12 month, 20% at 36 months and 23% at 60 months (median time to CHR of 8.6 months) post-ASCT. Both organ-specific & overall systemic response data is currently under analysis, including time-to-maximum response. With a median follow-up of 47.7 months (95%CI 38.3, 58.6), the non-relapse mortality at 1, 2 & 5 years was 3.3% (95%CI 0.1-6.4%), 4.4% (95%CI 0.6-8.2%) & 7.7% (1.9-13.6%), respectively. The median progression-free survival (PFS) was 106 months (95% CI 87.8, NR) with a 5-year PFS of 73.5% (95%CI 63.2-83.7%). The 5-year overall survival (OS) was 88.6% (95% CI 81.5-95.8%). The data analyzed in this study, demonstrates that ASCT can be an effective & safe therapeutic modality for patients with POEMS syndrome. Due to the systemic inflammatory nature of the condition, due care should be taken to supporting these patients through this procedure, especially in relation to engraftment syndrome.

Disclosures:
Nagler: Teva: Honoraria, Travel grants, research grants Other.