Long-term outcome of pomalidomide therapy in myelofibrosis

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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.

Original languageEnglish (US)
Pages (from-to)66-68
Number of pages3
JournalAmerican Journal of Hematology
Volume87
Issue number1
DOIs
StatePublished - Jan 2012

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Primary Myelofibrosis
Splenomegaly
Therapeutics
Anemia
Prednisone
pomalidomide
Clinical Trials
Mutation
Survival
Pharmaceutical Preparations

ASJC Scopus subject areas

  • Hematology

Cite this

Long-term outcome of pomalidomide therapy in myelofibrosis. / Begna, Kebede; Pardanani, Animesh D; Mesa, Ruben; Litzow, Mark R; Hogan, William; Hanson, Curtis A.; Tefferi, Ayalew.

In: American Journal of Hematology, Vol. 87, No. 1, 01.2012, p. 66-68.

Research output: Contribution to journalArticle

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