A phase II trial of pirfenidone (5-methyl-1-phenyl-2-[1H]pyridone), a novel anti-fibrosing agent, in myelofibrosis with myeloid metaplasia

Ruben A. Mesa, Ayalew Tefferi, Michelle A. Elliott, H. Clark Hoagland, Timothy G. Call, Georgene S. Schroeder, Soo Young Yoon, Chin Yang Li, Leigh A. Gray, S. Margolin, C. Christopher Hook

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Abstract

The anti-fibrotic and cytokine modulatory properties of pirfenidone suggest its usefulness in the treatment of myelofibrosis with myeloid metaplasia (MMM). In a prospective study, 28 patients with MMM were treated with oral pirfenidone. Twelve patients completed 1 year of therapy: 13 were withdrawn because of disease progression and three because of drug intolerance. Only one patient experienced a clinically relevant benefit with respect to anaemia and splenomegaly. The overall lack of clinical benefit correlated with no significant improvement in the bone marrow morphological features of the disease. We conclude that pirfenidone has no significant clinical or biological activity in MMM.

Original languageEnglish (US)
Pages (from-to)111-113
Number of pages3
JournalBritish journal of haematology
Volume114
Issue number1
DOIs
StatePublished - Aug 14 2001

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Keywords

  • Cytokines
  • Erythropoiesis
  • Myelofibrosis with myeloid metaplasia
  • Pirfenidone
  • Stem cells

ASJC Scopus subject areas

  • Hematology

Cite this

Mesa, R. A., Tefferi, A., Elliott, M. A., Clark Hoagland, H., Call, T. G., Schroeder, G. S., Yoon, S. Y., Li, C. Y., Gray, L. A., Margolin, S., & Christopher Hook, C. (2001). A phase II trial of pirfenidone (5-methyl-1-phenyl-2-[1H]pyridone), a novel anti-fibrosing agent, in myelofibrosis with myeloid metaplasia. British journal of haematology, 114(1), 111-113. https://doi.org/10.1046/j.1365-2141.2001.02883.x