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EBMT 2020 Annual Meeting - Patient, Family and Donor Day

Patients & Donors
Patient Advocacy Committee

Sunday 30 August, 10:30-12:40H, Auditorium 6

The perspective of the patient in this session will be given by Agnès Huet-Vicens, 57 and based in Paris, France who will tell delegates about her battle with myelofibrosis, a serious bone marrow disorder that is an uncommon type of chronic leukaemia. She was diagnosed with the condition during childhood.

“While I knew there would be worse ahead, I got on with life - playing tennis, studying pharmacy and business at university and joining the pharmaceutical company Sanofi, almost 30 years ago,” says Agnès.

In her 40s, she started to suffer anaemia and splenomegaly, an abnormally enlarged spleen. In early 2014, she had a splenectomy followed by successful bone marrow transplantation with her older sister as the donor. After that, she needed a month in a sterile environment before returning home to recover. 

“Today, I am well – the transplant worked, though I do have chronic graft versus host disease and require treatment with corticoids and needed an oral immunosuppressant.  I returned to work in 2015. I always try to stay positive – when bad news comes, I say ‘It’s not about me!’ I want to pay tribute to my husband who is a physician and my haematologist. In my talk, I will give many details about the impact of bone marrow transplantation on my life.”

As she remains in the at-risk category for COVID-19 infection, Agnès continues to be extra cautious in her day-to-day life. “I am working at home since the beginning of the pandemic in mid-March. I am very prudent even with my son (who is 18 years old), maintaining social distancing and not having physical contact with him or others. Like most others, I am wearing a mask when needed and washing my hands very often.”

Also in this session, psychological wellbeing will be addressed by Dr Anna Barata of the Moffitt Cancer Center (Tampa, FL, USA). She will discuss how advances in hematopoietic cell transplant methodology and supportive care are increasing the population of survivors worldwide.

She says: “However, haematopoietic cell transplants keep entailing significant morbidity that impairs survivors’ mental health and recovery. In today’s presentation, I will review the psychological morbidity over the course of haematopoietic cell transplants, along with its impact on transplant outcomes.”

Her talk will include a discussion of risk factors such as graft-versus-host disease and long-term complications that place patients at risk of worse psychological wellbeing. She will also review guidelines, recommendations and the potential of patient-reported outcome measures to screen and address unmet needs for care.

Dr Barata concludes: “Finally, I will summarise randomised control trials showing efficacy in improving survivors’ psychological well-being and present projects in EBMT where patients can be actively involved, such as the Patient Engagement Task Force and the Patient Advocacy Committee. Challenges and opportunities involved in improving survivors’ psychological care will also be reviewed.”

Professor Jürgen Kuball of University Medical Center Utrecht will also provide delegates with an update on CAR T-cell therapy in this extended session.

He explains: “With the approval of the first chimeric antigen receptor (CAR) T-cell products on the market, the European Medicines Agency (EMA) required market authorisation holders to monitor the long-term efficacy and safety of CAR T-cells for 15 years after administration.”

In 2019, the cellular therapy module of the EBMT registry received a positive qualification opinion from the EMA, indicating that the registry fulfils the essential needs to capture such data. Dr Kuball concludes: “In my talk, I will explain how we investigated this novel treatment modality for all stakeholders with focus from a patient perspective.”