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

doi:10.1046/j.1365-2141.2001.02883.x

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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41 Scopus citations

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 Twelve patients completed 1 year of therapy: 13 were withdrawn because of disease progression and three because of drug intolerance.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 language	English (US)
Pages (from-to)	111-113
Number of pages	3
Journal	British journal of haematology
Volume	114
Issue number	1
DOIs	https://doi.org/10.1046/j.1365-2141.2001.02883.x
State	Published - 2001

Keywords

Cytokines
Erythropoiesis
Myelofibrosis with myeloid metaplasia
Pirfenidone
Stem cells

ASJC Scopus subject areas

Hematology

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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

Mesa, RA, Tefferi, A, Elliott, MA, Clark Hoagland, H, Call, TG, Schroeder, GS, Yoon, SY, Li, CY, Gray, LA, 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, vol. 114, no. 1, pp. 111-113. https://doi.org/10.1046/j.1365-2141.2001.02883.x

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

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.",

keywords = "Cytokines, Erythropoiesis, Myelofibrosis with myeloid metaplasia, Pirfenidone, Stem cells",

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

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AB - 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.

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

Abstract

Keywords

ASJC Scopus subject areas

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