Why a successful transplant is only half the patient journey

People often think that the process of organ or stem cell transplantation ends once the patient receives the transplant. But this, in fact, is only half of the patient journey. 

Olivier Charmeil is the Executive Vice President of General Medicines, Sanofi.

We’ve all probably heard about organ donation, the need for matching a donor with a recipient, patient waiting lists, and the assessments needed to determine whether a patient is fit to receive a transplant. After all, these are the elements that regularly make the news and that form part of a patient’s journey leading up to the transplant. It is also the area where Europe has become a global leader, having significantly increased the number of donations and transplants over the past 15 years. 

However, despite the efforts of European healthcare systems around coordination and policies to increase transplants, limited attention is being paid to what happens after a transplant has been performed. Yet, it is this period of so-called post-transplant care, which can last multiple years, that poses many challenges to transplant patients – both for their survival and quality of life. These challenges need greater attention and awareness across Europe, not only for solid organ but also for stem cell transplants. 

The challenges of post-transplant care

Hematopoietic stem cell transplants, more commonly called bone marrow transplants, are a medical procedure for managing or curing life-threatening diseases such as lymphoma or leukaemia, as well as other debilitating diseases like multiple sclerosis. Leukaemia on its own accounts for a significant share of the 320,000 new blood cancer cases diagnosed every year in Europe, underlining just how large the patient population that may eventually benefit from stem cell transplants is.

As the number of stem cell transplants grow, there is an increasing need for post-transplant care, with many patients experiencing different complications. One of the most common of these complications is Graft-Versus-Host Disease (GVHD), where the recipient’s tissues and organs get inflamed or damaged because the donor’s immune system cells attack them. 

Up to 50% of patients who received a donated stem cell transplant develop chronic GVHD, causing them significant physical discomfort and pain. Joint stiffness, muscle weakness, fatigue, liver dysfunction, and difficulty breathing are only some of the symptoms of chronic GVHD that can impact people’s ability to go about their everyday lives. The disease also leaves a mark on people’s mental wellbeing, with almost one-in-three patients experiencing depression and anxiety, further impacting their quality of life. 

Beyond the individual impact, chronic GVHD places a significant long-term socioeconomic burden on healthcare systems, in particular specialised care resources, and on society through the loss of productivity due to sickness.

Low awareness of the diverse and evolving nature of GVHD symptoms among patients, relatives, and healthcare providers can lead to delays in diagnosis of chronic GVHD, making its treatment challenging and contributing to poor outcomes for transplant patients. Unfortunately, GVHD is one of the main causes of morbidity following stem cell transplants, with more than 10% of patients experiencing GVHD dying from this complication. 

This may seem like a discouraging number, but I firmly believe that it is within our power to make sure that more people can benefit from life-saving stem cell transplants without suffering from GVHD as a consequence. Among others, I see two key areas of action to address this challenge: greater awareness and more effective treatments.

Together for increased GVHD awareness 

It’s hard to effectively tackle a problem that only few people know about. Therefore, our first step in limiting the effects of GVHD and ensuring that patients receive optimal post-transplant care needs to focus on making patients, their relatives, and healthcare providers more aware of the signs of the disease. This will help them recognise symptoms sooner, paving the way for more timely, effective, and comprehensive care. 

Improving post-transplant care requires joint action by the whole GVHD community – and it’s promising to see that first steps in this direction are already being taken around the globe. The GVHD Alliance, which is celebrating its second anniversary this month, is one such joint initiative. Founded in the USA, the Alliance is making its way to Europe and works to help improve the lives of people with GVHD through educational, advocacy, and awareness raising activities including global GVHD Day. Taking place on 17 February every year, and for only the second time in 2024, this day unites people in their efforts to highlight GVHD patients’ needs and calls for change as well as better post-transplant care.

Chasing the miracles of science to fight GVHD

To ensure this better care for GVHD patients, effective treatments are needed. Stem cell transplant is a growing, but complex and often still underserved therapeutic area. Despite this, advances in scientific research over the past decades have already produced several medicines that help people fight GVHD and get back to living full lives post-transplant. 

However, we cannot rest on our laurels. The mainstay treatment for chronic GVHD can have various side effects and more than 50% of patients eventually stop responding to it, leaving those living with GVHD with a need for new treatment options which can help improve their symptoms and long-term outcomes. This is why innovation and R&D efforts to produce medicines for GVHD should not only continue but intensify. This is what we are committed to at Sanofi. For over 40 years, we have been working to bring transformative therapies to transplant patients. We continue to chase the miracles of science and leverage our research expertise to bring more solutions to patients in need of stem cell transplants.

A brighter future for transplant patients

Through common efforts, we can help foster Europe’s leadership across the entirety of the patient’s transplant journey. This requires that European policymakers, healthcare providers, academia, regulators, and pharmaceutical companies join forces and lead cross-border collaboration to address the unmet needs of post-transplant care and the GVHD patient community. 

Only by working together can we expand treatment options and ensure availability as well as timely patient access to life-saving solutions, so that no transplant patient is left behind.