Editorial: liver transplantation for primary biliary cholangitis-the need for timely and more effective treatments. Authors' reply
Alimentary Pharmacology and Therapeutics , Volume 49 - Issue 4 p. 473- 474
Abstract Linked Content
This article is linked to Harms et al and Gerussi et al papers. To view these articles, visit https://doi-org.eur.idm.oclc.org/10.1111/apt.15060 and https://doi-org.eur.idm.oclc.org/10.1111/apt.15095.
We would like to thank Gerussi et al1 for their interest in our study and for highlighting valid remarks and questions that our paper raises.2
Rightfully, the authors state that “non‐response” to first‐line therapy leaves patients prone to the need for liver transplantation (LT). Although—as previously proposed3—we believe the term “non‐response” should be avoided and should rather be replaced with “incomplete response” given that these patients do in fact still benefit from ursodeoxycholic acid (UDCA) treatment, we would like to stress that our in‐depth analyses in the Dutch population show that a minority of patients with primary biliary cholangitis (PBC) that are initially classified as “complete responders” after the first year of therapy, eventually also undergoes LT. This might be explained by the fact that some patients should actually be reclassified as “incomplete responders” when re‐evaluating the response status during follow‐up, as recently shown by Goet et al.4
Secondly, the authors suggest that, in addition to epidemiological changes and a reduced rate of biochemical response in males, diagnostic delay might impact the increasing proportion of males transplanted for PBC. However, it seems unlikely that diagnostic delay in males has worsened over the past decades, leaving this as an implausible explanation for our finding.
Another comment was that potential under‐reporting of autoimmune variant syndrome in PBC might impact our findings. Although research on the incidence and prevalence of such variant syndromes is limited, and studies on the impact of concomitant autoimmune hepatitis on long‐term outcome are conflicting,5, 6 exploring this further could indeed improve therapeutic strategies for this subgroup of patients with PBC, with a potential reduction in LTs as a result.
Overall, we agree with Gerussi et al that our study stresses the unmet need of effective second‐line therapies in PBC that reduce progression to liver failure, and the correct implementation of such drugs in clinical practice. As new potentially effective therapies including obeticholic acid and fibrates currently brighten the therapeutic horizon, we propose to renew this study after a decade or so, hopefully to find a renewed reduction in the absolute number of LTs for PBC.
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Harms, M.H, van Buuren, H.R, Hansen, B.E, & Metselaar, H.J. (2019). Editorial: liver transplantation for primary biliary cholangitis-the need for timely and more effective treatments. Authors' reply. Alimentary Pharmacology and Therapeutics, 49(4), 473–474. doi:10.1111/apt.15118