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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
Author(s) -
Mesa Ruben A.,
Tefferi Ayalew,
Elliott Michelle A.,
Hoagland H. Clark,
Call Timothy G.,
Schroeder Georgene S.,
Yoon SooYoung,
Li ChinYang,
Gray Leigh A.,
Margolin S.,
Hook C. Christopher
Publication year - 2001
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1046/j.1365-2141.2001.02883.x
Subject(s) - myelofibrosis , medicine , pirfenidone , metaplasia , gastroenterology , clinical trial , myeloid , phases of clinical research , disease , constitutional symptoms , dermatology , surgery , bone marrow , idiopathic pulmonary fibrosis , lung
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 Twelve patients completed 1 year of therapy; 13 were withdrawn because of disease progression and three because of drug intolerance.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.