title: POEMS syndrome treated with melphalan high-dose therapy and autologous blood stem cell transplantation: a single-institution experience.
abstract: The acronym POEMS syndrome stands for a rare multi-system disorder, comprised of polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes. Here, we present a single-center report of a series of five POEMS patients treated with melphalan high-dose therapy (HDT) with subsequent autologous blood stem cell transplantation (ABSCT). After a median follow-up of 52 months from time of diagnosis (range, 15-192) and a median follow-up of 18 months after ABSCT (range, 11-120), all patients were alive. Overall, no severe transplantation-associated complications such as engraftment syndrome or peri- or post-transplant death were noted. In two cases, HDT followed by ABSCT resulted in a complete hematologic response; in the additional three cases, partial responses (PR) were achieved including one very good hematologic PR. Only one patient with initial PR developed progressive disease nearly 2.5 years after transplantation. Consequently, a second HDT with ABSCT was successfully applied resulting in clinical improvement and hematologic PR. In line with previous single-center reports, melphalan HDT followed by ABSCT proved to be a first-line treatment option with tolerable side effects in severely affected POEMS patients with progressing symptoms.
authors.affiliation: Department of Medicine, Hematology/Oncology, University of Muenster, Muenster, Germany. Gabriela.Thoennissen@ukmuenster.de
authors.forename: Gabriela B
authors.initials: GB
authors.lastname: Thoennissen
authors.forename: Nils H
authors.initials: NH
authors.lastname: Thoennissen
authors.forename: Fleur
authors.initials: F
authors.lastname: Fritz
authors.forename: Andreas
authors.initials: A
authors.lastname: Hilbig
authors.forename: Andrea
authors.initials: A
authors.lastname: Kerkhoff
authors.forename: Ruediger
authors.initials: R
authors.lastname: Liersch
authors.forename: Utz
authors.initials: U
authors.lastname: Krug
authors.forename: Steffen
authors.initials: S
authors.lastname: Koschmieder
authors.forename: Carsten
authors.initials: C
authors.lastname: Müller-Tidow
authors.forename: Rolf
authors.initials: R
authors.lastname: Mesters
authors.forename: Martin
authors.initials: M
authors.lastname: Kropff
authors.forename: Wolfgang E
authors.initials: WE
authors.lastname: Berdel
mesh_terms: D000328:Adult; D018943:Anthracyclines / Q000627:therapeutic use; D003131:Combined Modality Therapy; D003907:Dexamethasone / Q000627:therapeutic use; D005260:Female; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000627:therapeutic use*; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000008:administration & dosage / Q000627:therapeutic use*; D009460:Neurologic Examination; D016878:POEMS Syndrome / Q000097:blood / Q000188:drug therapy* / Q000601:surgery; D036102:Peripheral Blood Stem Cell Transplantation; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D042461:Vascular Endothelial Growth Factor A / Q000097:blood
chemical_list: D018943:Anthracyclines; D019653:Myeloablative Agonists; C467484:VEGFA protein, human; D042461:Vascular Endothelial Growth Factor A; D003907:Dexamethasone; D008558:Melphalan