Martine Aliana Rothblatt (born 1954) is an American lawyer, author, and entrepreneur. She founded United Therapeutics in 1996, to make medicines for her daughter who suffered from pulmonary arterial hypertension, and is now developing genetically modified pig organs for human transplant. On the latter topic she has written a book titled “Your life or mine […]
This review covers the full clinical picture of pulmonary arterial hypertension (PAH) — from its pathophysiology and diagnosis through to current and emerging treatments. Understanding the Disease Pulmonary arterial hypertension is driven by progressive structural remodelling of the pulmonary arteries, creating a vicious cycle of vasoconstriction, inflammation, and uncontrolled cell growth that ultimately leads to
Tacrolimus (FK506), discovered in 1984 from Japanese soil fungus, revolutionized organ transplantation. Dr. Thomas Starzl’s pioneering work at the University of Pittsburgh demonstrated dramatic improvements over cyclosporine: liver graft survival increased from 65.5% to 86.2%, with rejection rates dropping to 1-2%. The Food and Drug Administration (FDA) approval followed for liver (1994), kidney (1997), and
Pulmonary arterial hypertension patients face unique challenges in lung transplantation. Unlike patients with other lung diseases, they often have relatively normal lung function tests but are in critical condition due to heart failure. This means they receive low priority scores in the standard Lung Allocation Score (LAS) system, which was designed primarily for patients with
A modified Delphi consensus was conducted by the Pulmonary Vascular Research Institute (PVRI) Lung Transplantation Workstream, involving 29 international experts on pulmonary arterial hypertension (PAH) and lung transplantation. The process generated consensus ( mean scores >_2.5 on Likert scale ) across 141 out of 223 statements relating to when to refer pulmonary arterial hypertension patients
The Council of Europe’s EDQM (European Directorate for the Quality of Medicines and HealthCare) Newsletter Transplant provides regular updates on transplantation-related developments across Europe. It covers policy, regulation, quality standards, and best practices related to organ donation, allocation, and transplant safety, ensuring harmonisation across European Union member states. Additionally, the newsletter shares news on scientific
Tacrolimus therapeutic drug monitoring is challenging due to high inter- and intra-patient variability. A prospective randomized controlled trial was conducted by researchers at the University Hospital in Leuven, Belgium, on 293 de novo kidney transplant recipients during the first 14 days post-transplant. A fully automated Model-informed precision dosing (MIPD) application (1) was integrated into the
A newly published review in Transplantation Direct explores the growing significance ofextracorporeal photopheresis (ECP), a therapy that modulates immune responses usingpatients’ own treated blood cells. The article, authored by Panagiotis Parsonidis, MSc, and Prof. Thomas Wekerle of the Medical University of Vienna, Austria, highlights extracorporeal photopheresis’s established safety record while pointing to future opportunities for
What is pulmonary arterial hypertension and why does risk assessment matter? Pulmonary arterial hypertension is a rare, serious disease where the blood vessels in thelungs become too narrow. This makes the right side of the heart work harder, which overtime can lead to heart failure. Because it can progress quickly, doctors use risk assessment (sometimes
Terumo, a leading medical technology company based in Japan, has announced a definitive agreement to acquire all outstanding shares of OrganOx, for approximately $1.5 billion. OrganOx specializes in advanced organ preservation devices using Normothermic Machine Perfusion technology, which preserves organs longer by circulating oxygen and nutrient-rich fluids through organs at near-body temperature. This technology enables
The key findings from the Pulmonary Hypertension Global Patient Survey (PHGPS) adult cohort of 3,329 patients have been published in the ERJ Open Research 2025, highlighting the achievements, shortcomings & regional disparities of clinical care & research globally. The Pulmonary Hypertension Global Patient Survey represents the most extensive survey of its type in the pulmonary
A large US registry study of 1,671 patients with pulmonary arterial hypertension (PAH) over 4607 person-years of follow up found that lung transplant referrals are occurring far too infrequently and too late in the disease course. Despite professional society recommendations for early referral, only 12% of patients were referred for transplantation over the study period.
In their recent open-access article for the Zentralblatt für Chirurgie, Drs.: Szofia Kovacs, Alberto Benazzo, and Peter Jaksch review the latest International Society for Lung and Heart Transplantion (ISHLT) guidelines on lung transplantation, highlighting a major shift toward personalized, inclusive care. Beyond the underlying lung disease, transplant decisions now consider age, frailty, comorbidities, and psychosocial
Globally, over 4,500 lung transplants are performed annually. Pulmonary Arterial Hypertension represents a unique transplantation indication because: Given these specific challenges, the authors developed guidelines for managing Pulmonary Arterial Hypertension patients who are lung transplant candidates through a modified Delphi study. The expert panel reached unanimous consensus on several key aspects of lung transplantation for
A systematic review and meta-analysis to determine whether tacrolimus or cyclosporine is more effective at reducing chronic lung allograft dysfunction (CLAD) in lung transplant patients was publshed on the British Medical Journal in April 2025. The researchers analyzed 4 randomized trials involving 662 patients with a mean age of 45-55 years, mostly with Chronic Obstructive
Pulmonary arterial hypertension (PAH) represents a significant economic burden on society, as revealed by a comprehensive Swedish study published on Pulmonary Circulation on April 17, 2025, examining the societal costs associated with this condition. The research tracked patients across various pulmonary arterial hypertension subgroups—idiopathic/hereditary pulmonary arterial hypertension, pulmonary arterial hypertension associated with connective tissue disease,
Three heart failure patients with pulmonary hypertension (WHO Group II) became eligible for heart transplants after receiving the Aeson Total Artificial Heart, which effectively took place after 243, 155 and 109 days respectively. This innovative device, implanted with the help of the advanced Carmat system, replaces the heart’s ventricles and maintains continuous blood circulation, stabilizing
Transplant International has published a special issue of its journal on “Diversity, Equity, and Inclusion in Transplantation”. This issue presents a collection of studies that examine inequities in transplantation related to race, gender, socioeconomics, and geography, providing evidence and insights into these persistent disparities. Download the pdf of the special issue of Transplant International at
During the European Respiratory Society 2024 Congress in Vienna, which was held 7-11 September 2024, the European Lung Foundation and ERN-LUNG lung transplantation group presented a new booklet sharing the patient perspective on transplantation. The booklet features an introduction by the European Lung Foundatiion Chair Dimitris Kontopidis and testimonials from lung transplant patients Natalia Maeva
The Concept Paper issued by the Ministry of the Interior of the Hungarian government, holding the European Council Presidency from July to December 2024, outlines a comprehensive strategy that encompasses increased organ availability, enhanced efficiency and accessibility of transplant systems, and the improvement of quality and safety standards. It stands as a call to action
