>>Am J Respir Crit Care Med 1999 Apr;159(4 Pt 1):1061-9
Treatment of idiopathic pulmonary fibrosis with a new antifibrotic agent, pirfenidone: results of a prospective, open-label Phase II study.
Raghu G, Johnson WC, Lockhart D, Mageto Y.
University of Washington, and Statistics and Epidemiology Research Corporation, Seattle, Washington, USA.
Idiopathic pulmonary fibrosis (IPF) is a progressive clinical syndrome of unknown etiology and fatal outcome. Currently available therapies are ineffective and associated with significant adverse effects. Pirfenidone, a new, investigational antifibrotic agent, was evaluated for its tolerability and usefulness in terminally ill patients with advanced IPF. Consecutive patients with IPF and deterioration despite conventional therapy or who were unable to tolerate or unwilling to try conventional therapy were treated with oral pirfenidone. Treatment was administered on a compassionate basis (open-label). Fifty-four patients were followed for mortality, change in lung function, and adverse effects. Their mean age was 62, mean duration of symptoms 4.6 yr, and time since lung biopsy was 3.2 yr. Conventional therapy was discontinued in 38 of 46 patients; the other eight were able to decrease their prednisone dosage and eight had no previous conventional treatment. One- and 2-yr survival was 78% (95% CI 66%, 89%) and 63% (95% CI 50%, 76%), respectively. Patients whose lung functions had deteriorated prior to enrollment appeared to stabilize after beginning treatment. Adverse effects were relatively minor. The results of this study are encouraging. Pirfenidone is a promising new treatment for IPF that is well tolerated.<<
Full article is a freebie:
ncbi.nlm.nih.gov
A phase II for myelofibrosis with myeloid metaplasia didn't work out so well, and the drug was not well tolerated by some. So it sounds as though dose limits have been explored, though what was used here isn't shown.
>>Br J Haematol 2001 Jul;114(1):111-3
A phase II trial of pirfenidone (5-methyl-1-phenyl-2-[1H]-pyridone), a novel anti-fibrosing agent, in myelofibrosis with myeloid metaplasia.
Mesa RA, Tefferi A, Elliott MA, Hoagland HC, Call TG, Schroeder GS, Yoon SY, Li CY, Gray LA, Margolin S, Hook CC.
Division of Hematology and Internal Medicine, Mayo Clinic, Rochester, MN 55905, USA.
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.<<
But the full text article for IPF should allow comparison with Actimmune.
Cheers, Tuck |
