title: Thalidomide for previously untreated indolent or smoldering multiple myeloma.
abstract: We conducted a clinical trial of thalidomide as initial therapy for asymptomatic smoldering (SMM) or indolent multiple myeloma (IMM). Sixteen patients were studied. Thalidomide was given orally at a dose of 200 mg/day for 2 weeks, and then increased as tolerated by 200 mg/day every 2 weeks to a maximum dose of 800 mg/day. Bone marrow microvessel density (MVD) and angiogenesis grading were estimated using CD34 immunostaining. Six patients had a confirmed response to therapy with at least 50% or greater reduction in serum and urine monoclonal (M) protein. When minor responses (25-49%) decrease in M protein concentration) were included, 11 of 16 patients (69%) responded to therapy. Major grade 3-4 toxicities included two patients with somnolence, and one patient each with syncope and neutropenia. Pre-treatment MVD was not a significant predictor of response to therapy, median MVD 4 and 12 in responders and non-responders respectively, P = 0.09. We conclude that thalidomide has significant activity in the treatment of newly diagnosed SMM/IMM. However, we do not recommend treatment with thalidomide at this stage since some patients with SMM/IMM can be stable for several months or years without any therapy. Additional randomized trials are needed to determine if thalidomide will delay progression to active multiple myeloma.
authors.affiliation: Division of Hematology, Mayo Clinic and Mayo Foundation, Rochester, MN 55905, USA.
authors.forename: S V
authors.initials: SV
authors.lastname: Rajkumar
authors.forename: A
authors.initials: A
authors.lastname: Dispenzieri
authors.forename: R
authors.initials: R
authors.lastname: Fonseca
authors.forename: M Q
authors.initials: MQ
authors.lastname: Lacy
authors.forename: S
authors.initials: S
authors.lastname: Geyer
authors.forename: J A
authors.initials: JA
authors.lastname: Lust
authors.forename: R A
authors.initials: RA
authors.lastname: Kyle
authors.forename: P R
authors.initials: PR
authors.lastname: Greipp
authors.forename: M A
authors.initials: MA
authors.lastname: Gertz
authors.forename: T E
authors.initials: TE
authors.lastname: Witzig
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000368:Aged; D020533:Angiogenesis Inhibitors / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009101:Multiple Myeloma / Q000188:drug therapy* / Q000473:pathology; D013792:Thalidomide / Q000008:administration & dosage* / Q000009:adverse effects; D016896:Treatment Outcome
chemical_list: D020533:Angiogenesis Inhibitors; D013792:Thalidomide