Autologous stem cell transplantation in POEMS syndrome: Multicenter real-world treatment patterns and outcomes

POEMS syndrome is a rare multisystem plasma cell disorder for which evidence guiding management is largely derived from single-center retrospective series. We conducted a retrospective multicenter study including all consecutive patients diagnosed between January 1995 and June 2024 across nine referral hospitals in Catalonia, Spain, within the GEMMAC collaborative network. Clinical characteristics, treatment strategies, transplant outcomes, and long-term survival were analyzed. Forty patients were included, with a median age of 65 years; 25 underwent autologous stem cell transplantation (ASCT). Hematologic response to first-line therapy was achieved in 77.5% of patients. After a median follow-up of 94.2 months, median progression-free survival was 102.6 months and median overall survival was 146.9 months, with 5-year rates of 69.0% and 87.6%, respectively. ASCT was associated with deep and durable hematologic and clinical responses. Baseline ECOG performance status and depth of hematologic response correlated with outcomes. VEGF levels declined markedly after treatment, particularly following ASCT. Transplant-related toxicity was manageable, although late complications were observed. These findings support ASCT as a central therapeutic strategy in eligible patients and underscore the need for long-term follow-up.

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Publication Details

Journal
Bone Marrow Transplantation
Published
2026-07-25
DOI
https://doi.org/10.1038/s41409-026-02984-0
Primary Topic
Peripheral Neuropathies and Disorders
Type
article
Field-Weighted Citation Impact
0.00

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article

Autologous stem cell transplantation in POEMS syndrome: Multicenter real-world treatment patterns and outcomes

Laura Rosiñol, Lourdes Escoda, Mercedes Gironella, Luis Gerardo Rodríguez‐Lobato et al.
Bone Marrow Transplantation
Peripheral Neuropathies and Disorders
article

Autologous stem cell transplantation in POEMS syndrome: Multicenter real-world treatment patterns and outcomes

Laura Rosiñol, Lourdes Escoda, Mercedes Gironella, Luis Gerardo Rodríguez‐Lobato, Natalia Tovar, J Verge, Carlos Fernández de Larrea, Cristina Motlló, Albert Oriol, C. Baca, Anna de Daniel, Carlos Castillo, Anna Sureda, Eugènia Abella, Jordi López‐Pardo, Carles Tolosa, Itziar Carro Arostegui, M Teresa Cibeira, Iago Arribas Castro, Josep María Martí-Tutusaus
article en

Abstract

POEMS syndrome is a rare multisystem plasma cell disorder for which evidence guiding management is largely derived from single-center retrospective series. We conducted a retrospective multicenter study including all consecutive patients diagnosed between January 1995 and June 2024 across nine referral hospitals in Catalonia, Spain, within the GEMMAC collaborative network. Clinical characteristics, treatment strategies, transplant outcomes, and long-term survival were analyzed. Forty patients were included, with a median age of 65 years; 25 underwent autologous stem cell transplantation (ASCT). Hematologic response to first-line therapy was achieved in 77.5% of patients. After a median follow-up of 94.2 months, median progression-free survival was 102.6 months and median overall survival was 146.9 months, with 5-year rates of 69.0% and 87.6%, respectively. ASCT was associated with deep and durable hematologic and clinical responses. Baseline ECOG performance status and depth of hematologic response correlated with outcomes. VEGF levels declined markedly after treatment, particularly following ASCT. Transplant-related toxicity was manageable, although late complications were observed. These findings support ASCT as a central therapeutic strategy in eligible patients and underscore the need for long-term follow-up.

Bone Marrow Transplantation
Innsbruck Medical University (AT), Universität Innsbruck (AT), Hospital de Sant Pau (ES), Institut d'Investigació Biomédica de Bellvitge (ES), Josep Carreras Leukaemia Research Institute (ES), Institut Català d'Oncologia (ES), Hospital Clínic de Barcelona (ES), Hospital de Sabadell (ES), Vall d'Hebron Hospital Universitari (ES), Mútua Terrassa (ES), Hospital Del Mar (ES), Hospital Universitari Joan XXIII de Tarragona (ES), Universitat de Barcelona (ES)
Instituto de Salud Carlos III
Openalex Percentile: Top 8%
Peripheral Neuropathies and Disorders
DOIOpen Access PDFOpenAlex
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