Education Program




Disclosure:
VERTEX - Consultancy (Includes expert testimony)

Advances in
transfusion practices, iron overload monitoring and chelation therapy, and
multidisciplinary care have significantly improved survival in individuals with
transfusion-dependent thalassemia (TDT) and non-transfusion dependent
thalassemia (NTDT). This educational session will explore some of the
challenges faced by individuals with thalassemia as we continue to aim for
improved survival and quality of life. As more women with TDT are surviving
into adulthood and pursuing pregnancies, this session will explore the
challenges and strategies to ensure optimal pregnancy outcomes. Gene therapy
has been transformative for individuals with TDT, however current approaches
are limited by high costs and risk of infertility. This session will highlight
new approaches aimed at making gene therapy more affordable and less toxic.
Lastly, individuals with NTDT experience increasing complications as they age.
The session will discuss the challenges of NTDT and strategies for early
interventions to change the disease course.


Dr. Farzana
Sayani will discuss the risks associated with pregnancy in women with TDT and
NTDT. She will discuss the importance of a multidisciplinary team approach and
key monitoring and intervention strategies, from pre-conception counselling
through to post-partum care, required to optimize pregnancy outcomes in women
with thalassemia.


Dr. Yongshui Fu
will provide an update on the current status of gene therapy in TDT,
specifically outlining the current challenges including high costs and
conditioning-related risk of infertility.  He will discuss strategies to
improve costs and toxicities, highlighting KL003, a lentiviral vector-based
gene therapy used with the Nansha conditioning protocol as one such strategy.Dr.
Forni will use a case-based approach to illustrate the challenges of managing
beta-NTDT. He will discuss the importance of monitoring for early
complications, and address strategies for early interventions to prevent severe
complications, including indications for phenocoversion to TDT.