Rome – "Establishment of a National Registry for Graft-Versus-Host Disease and Recognition of GvHD as a Rare and Disabling Disease."
These are just two of the ten requests contained in the policy document 'Chronic GvHD: from knowledge to patient management', presented in Rome during a press conference organized at the initiative of Senator Elisa Pirro, president of the Parliamentary Intergroup for the Donation and Transplantation of Organs, Tissues and Cells, which took place at the Montecitorio Meeting Center (MoMec).
Created as part of the GRACE (GvHD Response and Awareness for Care and Engagement) project, promoted by Sanofi with institutions, scientific societies, and patient associations, the document aims to raise awareness among institutions and stakeholders about chronic GvHD, a systemic and potentially fatal immune-mediated disease. The document promotes greater understanding of the disease and its management.
"Chronic GvHD is a condition that is still little understood, but it profoundly impacts the quality of life of those who deal with it," Senator Pirro said. "With the 'Grace' project, we want to give a voice to patients and caregivers, promote awareness, and build a shared path that improves access to care and support throughout the treatment process."
Other points included in the document include the establishment of a National Chronic GvHD Day and a National Hematopoietic Stem Cell Transplant Day; the promotion of awareness campaigns; and the creation of Diagnostic-Therapeutic Care Pathways (PDTA) to define care standards.
Furthermore, ensuring the training of multidisciplinary teams in transplant centers (nurses, hematologists, dermatologists, gastroenterologists, ophthalmologists, and pulmonologists) and ensuring equitable and timely access to new therapeutic solutions; ensuring collaboration between patient associations to develop shared educational programs for patients and caregivers; developing psychological and social support programs for patients and caregivers; and promoting clinical research.
“The goal of the project,” they announced during the event, “is to give patients a voice, understand the impact of this disease on their quality of life, overcome fears and resistance to bone marrow transplantation, identify and promote concrete and specific interventions that allow them to approach this process with awareness, with the ultimate goal of improving access to innovative therapies and forms of continuous support throughout the entire therapeutic process, raising the standards of care and the quality of life of patients.”
The document's recommendations are based "on both clinical evidence and authentic patient testimonials, emerging from discussions within the GRACE Working Group." They aim to promote a multidisciplinary and integrated approach to the management of GvHD, improving the early identification and treatment of disease complications, with the ultimate goal of raising standards of care and patients' quality of life.
“Sanofi continues and strengthens its commitment to transplantation,” commented Gilda Stivali, Medical Head of Sanofi General Medicines, Italy. “Our mission goes beyond research into innovative therapies: we work daily to support people undergoing hematopoietic stem cell transplants at every stage of their journey to ensure an improvement in their quality of life.
We are committed to identifying unmet clinical needs in the clinical management of GVHD and actively collaborate with the hematology community to advance scientific knowledge. We also support the GVHD Alliance initiative to create an informational and support network that connects people with GVHD, caregivers, and clinicians.
WHAT IS GVHD?
Graft-versus-Host Disease (GvHD) is a systemic immune-mediated condition that can affect between 20% and 50% of patients undergoing allogeneic bone marrow transplantation (or more correctly, hematopoietic stem cell transplantation), meaning transplants performed with cells from a donor.
GvHD develops when the donor's immune cells recognize the recipient's tissues as foreign, triggering an immune response that can damage them. There are two main forms of GvHD, each with distinct clinical, pathophysiological, and therapeutic characteristics: acute GvHD (aGvHD), which usually occurs within the first 100 days after transplantation and primarily affects the skin, liver, and gastrointestinal tract; and chronic GvHD (cGvHD), which generally occurs after 100 days after transplantation and up to two years after allogeneic transplantation, but can also appear earlier.
In addition to the organs affected in the acute form, it can also involve the skin, eyes, oral and esophageal mucosa, liver, intestine and musculoskeletal system.
THE REGULATORY FRAMEWORK OF REFERENCE
Currently, the regulatory framework for bone marrow transplants in Italy is based on national laws and European regulations that guarantee safety, quality, and transparency throughout the entire process, from donation to transplant.
Among the main regulations are Law 91/1999, which established the National Transplant Center and introduced informed consent, and Law 52/2001, which recognized the National Donor Registry. Various legislative decrees (from 2007 to 2012) regulate technical aspects, implementing European directives on quality and safety. State-Regional Agreements define the standards for transplant centers.
At the European level, the new European Regulation on Substances of Human Origin will come into force in 2027, harmonizing regulations on cells, blood, and tissues, strengthening traceability and safety. In January 2023, bill AS 499, co-authored by Senator Elisa Pirro, was introduced with the aim of establishing a National Day specifically dedicated to bone marrow donation, to "highlight the importance of this gesture of solidarity and raise public awareness of this specific issue."
This article is very interesting and touching, but I believe there is much room for improvement in the management of GvHD. Awareness is crucial, but more resources are also needed to support patients and their caregivers. It's important that institutions do more.