Clinical trial protocol for TRANSFORM-UK: A therapeutic open-label study of tocilizumab in the treatment of pulmonary arterial hypertension

PVRI Member Authors: Paul Corris, Mark Toshner, Nicholas Morrell, Colin Church, Sean Gaine, Louise Harlow, Emily Knightbridge


Our aim is to assess the safety and potential efficacy of a novel treatment paradigm in pulmonary arterial hypertension (PAH), immunomodulation by blocking interleukin-6 (IL6) signaling with the IL6 receptor antagonist, tocilizumab. Inflammation and autoimmunity are established as important in PAH pathophysiology. One of the most robust observations across multiple cohorts in PAH has been an increase in IL6, both in the lung and systemically. Tocilizumab is an IL-6 receptor antagonist established as safe and effective, primarily in rheumatoid arthritis, and has shown promise in scleroderma. In case reports where the underlying cause of PAH is an inflammatory process such as systemic lupus erythematosus, mixed connective tissue disease (MCTD), and Castleman’s disease, there have been case reports of regression of PAH with tocilizumab. . . . .

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Pharmacology: Pharmacokinetics and Pharmacodynamics
Pulmonary Arterial Hypertension
Pulmonary Hypertension


Jules Hernández-Sánchez, Kate Bunclark, Dee Gor, Alun Bedding

Published in:

Pulmonary Circulation Vol 8: No 1 cover image

March 2018

Pulmonary Circulation Vol 8: No 1

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