title: Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): a single-center experience.
abstract: The POEMS syndrome (polyradiculoneuropathy, organomegaly, multiple endocrinopathies, monoclonal protein, skin changes) is a rare disease associated with a plasma cell dyscrasia. Patients with disseminated POEMS can be treated with high-dose therapy and autologous stem cell transplantation (ASCT). While clinical improvement is nearly universal in these patients, the long-term outcomes after transplantation are unclear. We therefore assessed the long-term clinical outcomes of 59 POEMS patients treated with ASCT at our institution. With a median follow-up of 45 months, 14 patients have progressed with a progression-free survival of 98% and 75% at 1 and 5 years, respectively. Factors associated with progression have included an IgG-λ monoclonal component (hazard ratio [HR] 7.5; 95% confidence interval [CI], 2.3-28.3; P = .0008), fluorodeoxyglucose-avid lesions on baseline positron emission tomography (HR 6.4; 95% CI, 1.2-120; P = .03), lack of complete hematologic response (HR 5.4; 95% CI, 1.8-16.7; P = .003), and patients aged 50 years or younger at transplantation (HR 4.4; 95% CI, 1.3-20; P = .01). The most common progression events have been radiologic followed by rising VEGF. Symptomatic progression has been rare. Most patients could be salvaged with immunomodulatory drugs or radiation. The 5-year survival is 94%. Herein, we describe a system of monitoring response and progression among patients with POEMS after ASCT.
authors.affiliation: Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN 55905, USA.
authors.forename: Anita
authors.initials: A
authors.lastname: D'Souza
authors.forename: Martha
authors.initials: M
authors.lastname: Lacy
authors.forename: Morie
authors.initials: M
authors.lastname: Gertz
authors.forename: Shaji
authors.initials: S
authors.lastname: Kumar
authors.forename: Francis
authors.initials: F
authors.lastname: Buadi
authors.forename: Suzanne
authors.initials: S
authors.lastname: Hayman
authors.forename: David
authors.initials: D
authors.lastname: Dingli
authors.forename: Steven
authors.initials: S
authors.lastname: Zeldenrust
authors.forename: Robert
authors.initials: R
authors.lastname: Kyle
authors.forename: Stephen
authors.initials: S
authors.lastname: Ansell
authors.forename: David
authors.initials: D
authors.lastname: Inwards
authors.forename: Patrick
authors.initials: P
authors.lastname: Johnston
authors.forename: Ivana
authors.initials: I
authors.lastname: Micallef
authors.forename: Luis
authors.initials: L
authors.lastname: Porrata
authors.forename: Mark
authors.initials: M
authors.lastname: Litzow
authors.forename: Dennis
authors.initials: D
authors.lastname: Gastineau
authors.forename: William
authors.initials: W
authors.lastname: Hogan
authors.forename: Angela
authors.initials: A
authors.lastname: Dispenzieri
mesh_terms: D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use; D059248:Chemoradiotherapy / Q000379:methods*; D018450:Disease Progression; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D008875:Middle Aged; D016878:POEMS Syndrome / Q000401:mortality* / Q000628:therapy*; D055502:Secondary Prevention; D013997:Time Factors; D014182:Transplantation, Autologous; D055815:Young Adult
chemical_list: D000970:Antineoplastic Agents