TY - JOUR
T1 - Thalassaemia—A global view
AU - Hokland, Peter
AU - Daar, Shahina
AU - Khair, Wael
AU - Sheth, Sujit
AU - Taher, Ali T.
AU - Torti, Lorenza
AU - Hantaweepant, Chattree
AU - Rund, Deborah
N1 - © 2023 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.
PY - 2023/2/17
Y1 - 2023/2/17
N2 - The thalassaemias are a group of genetic disorders of haemoglobin which are endemic in the tropics but are now found worldwide due to migration. Basic standard of care therapy includes regular transfusions to maintain a haemoglobin level of around 10 g/dL, together with iron chelation therapy to prevent iron overload. Novel therapies, bone marrow transplantation, and gene therapy are treatment options that are unavailable in many countries with stressed economies. This Wider Perspectives article presents the strategies for management of an adolescent refugee patient with beta thalassaemia, as it would be performed by expert haematologists in six countries: Italy, Lebanon, Oman, the Sudan, Thailand and the United States. The experienced clinicians in each country have adapted their practice according to the resources available, which vary greatly. Even in the current modern era, providing adequate transfusions and chelation is problematic in many countries. On the other hand, ensuring adherence to therapy, particularly during adolescence, is a similar challenge seen in all countries. The concluding section highlights the disparities in available therapies and puts the role of novel therapies into a societal context.
AB - The thalassaemias are a group of genetic disorders of haemoglobin which are endemic in the tropics but are now found worldwide due to migration. Basic standard of care therapy includes regular transfusions to maintain a haemoglobin level of around 10 g/dL, together with iron chelation therapy to prevent iron overload. Novel therapies, bone marrow transplantation, and gene therapy are treatment options that are unavailable in many countries with stressed economies. This Wider Perspectives article presents the strategies for management of an adolescent refugee patient with beta thalassaemia, as it would be performed by expert haematologists in six countries: Italy, Lebanon, Oman, the Sudan, Thailand and the United States. The experienced clinicians in each country have adapted their practice according to the resources available, which vary greatly. Even in the current modern era, providing adequate transfusions and chelation is problematic in many countries. On the other hand, ensuring adherence to therapy, particularly during adolescence, is a similar challenge seen in all countries. The concluding section highlights the disparities in available therapies and puts the role of novel therapies into a societal context.
KW - diagnosis
KW - global view
KW - refugee
KW - thalassaemia
KW - therapy
KW - Blood Transfusion
KW - Humans
KW - Thalassemia/epidemiology
KW - Adolescent
KW - Iron Overload/therapy
KW - Chelation Therapy
KW - beta-Thalassemia/epidemiology
UR - http://www.scopus.com/inward/record.url?scp=85148438158&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85148438158&partnerID=8YFLogxK
UR - https://www.mendeley.com/catalogue/32eba5ab-c4ec-304e-b0ee-164cfd9eec9a/
U2 - 10.1111/bjh.18671
DO - 10.1111/bjh.18671
M3 - Review article
C2 - 36799486
AN - SCOPUS:85148438158
SN - 0007-1048
VL - 201
SP - 199
EP - 214
JO - British Journal of Haematology
JF - British Journal of Haematology
IS - 2
ER -