TY - JOUR T1 - Caring for the orphan's orphan: treatment of patients with portopulmonary hypertension JF - European Respiratory Journal JO - Eur Respir J SP - 1038 LP - 1040 DO - 10.1183/09031936.00113407 VL - 30 IS - 6 AU - S. M. Kawut Y1 - 2007/12/01 UR - http://erj.ersjournals.com/content/30/6/1038.abstract N2 - Patients with portopulmonary hypertension (PPHTN) are incredibly unfortunate. By definition, these patients have portal hypertension, leading to well-known life-threatening gastrointestinal, neurological, infectious, haematological and renal complications requiring liver transplantation for cure. If this were not enough, these patients also have pulmonary arterial hypertension (PAH). The coexistence of portal hypertension and PAH frequently makes patients with this rare (or “orphan”) condition medical “untouchables”. PPHTN patients with progressive hepatic dysfunction and other complications are often deemed to be too “high-risk” for placement of a transjugular intrahepatic portosystemic shunt or liver transplantation, making them destined for a poor outcome. Even those who may be acceptable candidates for liver transplantation commonly face a complicated perioperative course. The situation is not much better from the vantage of their lung disease, despite these patients having the third most common form of PAH in a recent population-based epidemiological study from France 1. While there is some evidence regarding medications which should be avoided in PPHTN 2, therapeutic approaches used to treat other forms of PAH have never been trialled in PPHTN, are potentially complicated by coexistent liver disease, and are used in PPHTN without the evidence base which now exists for other types of PAH. Many individuals with PPHTN have a history of (or are currently) using alcohol or illicit drugs, addictions commonly linked to the aetiology of their liver disease, and some have HIV infection, all accompanied by significant social stigma. These comorbidities often further limit access to specialised centres and available treatments. The sum of these burdens and the high toll of the disease have proved significant obstacles to successful advocacy on behalf of this patient group. Who can blame industry sponsors, scientific advisory committees and regulatory boards for excluding these patients from clinical trials of new therapies for PAH? It is against … ER -