title: Long-term outcome of pomalidomide therapy in myelofibrosis.
abstract: Ninety-four Mayo Clinic patients with myelofibrosis (MF) participated in two consecutive clinical trials of pomalidomide (0.5-3.5 mg/day), with or without prednisone. Overall anemia response was 27% and increased to 53% in JAK2V617F-positive patients with <10 cm palpable splenomegaly and <5% circulating blasts; response rate was 0% in mutation-negative patients with either ≥10 cm splenomegaly or ≥5% circulating blasts (P = 0.0001). Median duration of anemia response was 16 months. Treatment effect on splenomegaly was negligible. To date, pomalidomide therapy has been discontinued in 86 (91%) patients at a rate of 68% at 1 year and 89% at 2 years. Grade 1 sensory neuropathy developed in 4 (13%) of 30 patients treated for ≥1 year. Risk-adjusted survival in pomalidomide-treated primary MF patients (n = 72) was similar to their counterparts not exposed to the drug (n = 471; P = 0.19). Long-term follow-up of pomalidomide treatment in MF reveals palliative value for a select group of patients and treatment-emergent sensory neuropathy.
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota, USA.
authors.forename: Kebede H
authors.initials: KH
authors.lastname: Begna
authors.forename: Animesh
authors.initials: A
authors.lastname: Pardanani
authors.forename: Ruben
authors.initials: R
authors.lastname: Mesa
authors.forename: Mark R
authors.initials: MR
authors.lastname: Litzow
authors.forename: William J
authors.initials: WJ
authors.lastname: Hogan
authors.forename: Curtis A
authors.initials: CA
authors.lastname: Hanson
authors.forename: Ayalew
authors.initials: A
authors.lastname: Tefferi
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D055728:Primary Myelofibrosis / Q000188:drug therapy* / Q000401:mortality; D011379:Prognosis; D016019:Survival Analysis; D013792:Thalidomide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D013792:Thalidomide; C467566:pomalidomide