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Company<\/strong><\/p>\n GEMMAC (Catalan Group for the Study of Multiple Myeloma and Amyloidosis)<\/p>\n Event Type<\/strong><\/p>\n Peer-Reviewed Publication (Multicenter Retrospective Cohort)<\/p>\n Platform<\/strong><\/p>\n Autologous Stem-Cell Transplantation (High-Dose Melphalan)<\/p>\n Indication<\/strong><\/p>\n POEMS Syndrome<\/p>\n A same-day peer-reviewed multicenter study followed 40 patients with POEMS syndrome treated at nine Catalan referral hospitals; 25 received first-line autologous stem-cell transplantation. After median follow-up of 94.2 months, five-year progression-free and overall survival were 69.0% and 87.6%. Among transplanted patients, 92% achieved a hematologic response and 95.2% of evaluable patients improved clinically. The study reinforces transplantation as a central strategy in eligible systemic disease but also reports 6.7% transplant-related mortality and second primary neoplasms in 17.5% of the total cohort, supporting prolonged surveillance.<\/p>\n The retrospective GEMMAC study included all 40 consecutive POEMS cases diagnosed from 1995 through June 2024 across nine referral hospitals in Catalonia, Spain. Median age was 65 years.<\/p>\n Twenty-five patients received first-line autologous transplantation, including ten who proceeded directly to transplant and 15 who received induction first. Hematologic response after transplant was 92%, including 52% complete response.<\/p>\n Clinical response occurred in 20 of 21 evaluable transplanted patients. Neuropathy improved in 85.7%, and median VEGF fell from 885 pg\/mL at diagnosis to 68 pg\/mL after transplant among patients with serial measurements.<\/p>\n Across the full cohort, median progression-free survival was 102.6 months and median overall survival was 146.9 months. Five-year rates were 69.0% and 87.6%, respectively.<\/p>\n Two of 30 transplant procedures were associated with treatment-related death. Seven of 40 patients developed second primary neoplasms, including MDS\/AML, AML, lymphoma, and solid tumors, at a median of 84 months.<\/p>\n The depth of hematologic and clinical response supports high-dose melphalan and autologous rescue as an effective disease-control strategy for eligible patients with systemic POEMS. Neurological improvement is particularly meaningful because disability, rather than plasma-cell burden alone, drives morbidity.<\/p>\n Complete response after transplantation correlated with longer progression-free survival, reinforcing clonal depth as a useful objective. The association is exploratory and cannot determine whether deeper response causes better outcome or identifies patients with more favorable disease.<\/p>\n POEMS is a plasma-cell-driven cytokine and angiogenic syndrome in which vascular endothelial growth factor contributes to vascular permeability, edema, pulmonary hypertension, and other manifestations. The pronounced post-transplant VEGF decline supports serial VEGF as a disease-activity biomarker.<\/p>\n VEGF measurements were available only in subsets and were not standardized across three decades. Biomarker kinetics should therefore be integrated with hematologic, neurological, imaging, and clinical response rather than used alone.<\/p>\n Engraftment syndrome occurred in 26.6% of transplant procedures, bacterial infection in 16.6%, and transplant-related mortality in 6.7%. These rates emphasize meticulous volume management, infection prophylaxis, and early recognition of engraftment inflammation in a population with baseline cardiopulmonary vulnerability.<\/p>\n Second primary neoplasms in 17.5% over long follow-up are clinically important, although causality cannot be assigned to melphalan because age, prior alkylators, repeated transplantation, disease biology, and surveillance intensity all contribute.<\/p>\n No significant outcome difference was detected between upfront and post-induction transplantation, but the comparison involved only ten and 15 patients and is underpowered. It does not establish equivalence or eliminate the value of cytoreduction before transplant.<\/p>\n Five patients underwent a second transplantation after long first remissions, with four remaining relapse-free at a median 71 months after the second procedure. This supports feasibility in selected patients but is too small to define a general salvage standard.<\/p>\n POEMS syndrome is a rare plasma-cell disorder defined by polyneuropathy, organomegaly, endocrinopathy, monoclonal plasma-cell disease, and skin changes. Elevated VEGF and cytokine dysregulation produce multisystem manifestations despite often low marrow tumor burden.<\/p>\n The Catalan Group for the Study of Multiple Myeloma and Amyloidosis coordinated the study. Autologous transplantation uses high-dose melphalan to suppress the pathogenic plasma-cell clone followed by reinfusion of the patient’s hematopoietic stem cells.<\/p>\n The study reinforces autologous transplantation as the most durable established strategy for eligible systemic POEMS while providing unusually long real-world follow-up in a rare disease.<\/p>\n The value lies as much in the safety boundary as in response rates. POEMS programs and transplant centers should pair depth-of-response goals with proactive engraftment management and surveillance extending well beyond initial remission.<\/p>\n Signal strength:<\/strong> 3\/5 \u2014 Medium-High. Importance:<\/strong> Medium-High \u2014 the study supplies long-term multicenter evidence in a rare plasma-cell disorder and identifies both durable benefit and clinically relevant late risk. Confidence:<\/strong> High for the descriptive outcomes within the cohort; medium for comparative treatment claims and causal risk factors because the sample is small, retrospective, and strongly selected.<\/p>\n","protected":false},"excerpt":{"rendered":" A same-day peer-reviewed multicenter study followed 40 patients with POEMS syndrome treated at nine Catalan referral hospitals; 25 received first-line autologous stem-cell transplantation. After median follow-up of 94.2 months, five-year progression-free and overall survival…<\/p>\n","protected":false},"author":1,"featured_media":2172,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1,4],"tags":[185,186],"class_list":["post-2155","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-all-categories","category-technology-modalities","tag-gemmac","tag-poems-syndrome"],"blocksy_meta":[],"_links":{"self":[{"href":"https:\/\/www.insilens.com\/index.php?rest_route=\/wp\/v2\/posts\/2155","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.insilens.com\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.insilens.com\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.insilens.com\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.insilens.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=2155"}],"version-history":[{"count":2,"href":"https:\/\/www.insilens.com\/index.php?rest_route=\/wp\/v2\/posts\/2155\/revisions"}],"predecessor-version":[{"id":2183,"href":"https:\/\/www.insilens.com\/index.php?rest_route=\/wp\/v2\/posts\/2155\/revisions\/2183"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.insilens.com\/index.php?rest_route=\/wp\/v2\/media\/2172"}],"wp:attachment":[{"href":"https:\/\/www.insilens.com\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=2155"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.insilens.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=2155"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.insilens.com\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=2155"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}Summary<\/h4>\n
Clinical Interpretation<\/h4>\n
Disease Biology and Biomarkers<\/h4>\n
Safety and Long-Term Surveillance<\/h4>\n
Treatment Strategy<\/h4>\n
Company and Product Background<\/h4>\n
Signal Extraction<\/h4>\n
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InSilens Take<\/h4>\n
Signal Assessment<\/h4>\n