Pilot study of pegylated interferon-alpha 2b in patients with essential thrombocythemia

Yesid Alvarado, Jorge Cortes, Srdan Verstovsek, Deborah Thomas, Stephan Faderl, Zeev Estrov, Hagop Kantarjian, Francis J. Giles

Research output: Contribution to journalArticlepeer-review

46 Scopus citations


Purpose: Interferon-alpha (IFN-alpha) has been shown to control symptoms, reduce platelet counts, and reduce the bone marrow megakaryocyte mass in patients with essential thrombocythemia (ET). A semi-synthetic protein-polymer conjugate of IFN-alpha 2b (PEG-IFN2b) increases the serum half-life of IFN-alpha 2b. We conducted a pilot study of Peg-IFN2b in patients with ET. Patients and methods: Patients with a history of persistent (greater than 2 months) platelet counts > 600× 109/l, with hyperplasia of bone marrow megakaryocytes in the absence of an alternate identifiable cause of thrombocytosis were eligible. Patients were required to have either thrombohemorrhagic signs and/or symptoms if previously untreated; persistence of thrombohemorrhagic signs and/or symptoms if receiving anagrelide, IFN-alpha, or hydroxyurea; or intolerance to anagrelide, IFN-alpha, or hydroxyurea. The initial PEG-IFN2b dose was from 1.5 to 4.5 μg/kg per week subcutaneously with subsequent dose adjustments as indicated by response and adverse events. Results: Eleven patients (nine female, median age 54 years, range 26-69 years) were treated. PEG-IFN2b rapidly controlled platelet counts and resolved symptoms in all patients. The median duration of PEG-IFN2b therapy on-study was 9 months (range 4-17 months). No patient had signs or symptoms of thrombosis or hemorrhage while on study. After 2 months of therapy, 10 patients (91%) were in complete remission, and 11 (100%) after 4 months. One patient discontinued therapy at 4 months because of persistent grade 3 fatigue and a second at 5 months because of anxiety and depression. Conclusion: PEG-IFN2b has significant activity in patients with ET. Long-term follow-up of a larger cohort of patients is needed to define its role in this disease.

Original languageEnglish (US)
Pages (from-to)81-86
Number of pages6
JournalCancer Chemotherapy and Pharmacology
Issue number1
StatePublished - 2003
Externally publishedYes


  • Efficacy
  • Essential thrombocythemia
  • Pegylated interferon

ASJC Scopus subject areas

  • Oncology
  • Toxicology
  • Pharmacology
  • Cancer Research
  • Pharmacology (medical)


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