Evolution of Thalassaemia Management Four Decades in Thalassaemia Care – Our Achievements and Challenges

Joint efforts for better future

Patients with TDT are facing long-term medical and health issues all through their life. There is inevitably psychosocial impact to the patient and their parents always. In 1982 Thalassaemia Association was established with substantial support from guidance of the late Dr. Anita Li who was a paediatrician paying a lot of efforts in raising the standard of care for thalassaemia patients in Hong Kong. The Thalssaemia Association has all along aimed to foster support among thalassaemia patients and their families. In 1993, Children’s Thalassaemia Foundation (CTF) was set up under the guidance of Prof. C.Y. Yeung, head from Department of Paediatrics at the University of Hong Kong. With the support from many volunteers and benevolent donors, the CTF have raised funds to support individual patients, promote public awareness, and fund research projects related to thalassemia.

The clinical care of patients with TDT has advanced substantially over the recent quarter of century with much improved outcome over the past 3 – 4 decades. The mortality has continued to fall Fig. 19 Ref. 2 and iron overloaded complications are much reduced with tailored made chelation regimen. Patients with transfusion-dependent thalassaemia can now enjoy better quality of life with regular transfusion and appropriate iron chelation regimens which should be tailored made based on the degree of iron overload. that individualize and optimize to achieve normal or near normal iron overload status. Haematopoietic stem cell transplantation enables patients free from transfusion with decreased morbidities and mortality. More patients are now eligible to the procedure due to the use of alternative stem cell donors other than HLA matched siblings. Haemopoietic stem cell transplantation service is now centralized in Hong Kong Children’s Hospital. A plan to develop a Thalassaemia Registry in Hong Kong is also in progress.

Thalassemia is now becoming more an adult disease, and some are already in their middle age advancing to old age. Many of the patients can work as normal people, achieve in careers and parenthood with the current improvements in treatment.

Transfusion safety has been enhanced but a threat with emerginginfection. In the future newer iron chelators may come up in the future. New problems are expected to be encountered as patients get into old age together with health problems associated with senility. Bone marrow transplant can achieve transfusion dependency with improvement to reduce graft rejection and recuse complications. Gene therapy brings promising hope in the future.

Four decades is a long time! A summary of progress in thalassaemia care is put into a chart. Fig. 20 Patients with TDT have been facing diverse medical problems and psychosocial difficulties at their time. The disease, in the past was viewed as a “bad disease” can be appropriately managed with options of transfusion and chelation. HSCT can now offer transfusion dependency in a proportion of patients and perhaps in the future gene therapy can be applicable to them. Patients are enjoying quality life and much reduced morbidities. This will not be materialized without their own consistent resilience to face the illness, support from their families and the medical and health care professionals. With joint efforts from all parties, it is hopeful that the future is even better!