LONG TERM OUTCOME OF THALASSAEMIA
MAJOR IN HONG KONG
Li CK1, Luk CW2,
Ling SC3, Chik KW1, Yuen HL2, Li CK3,
Shing MMK1, Chang KO2
1Prince of Wales Hospital, The Chinese University of Hong
Kong; 2Queen Elizabeth Hospital; 3Princess Margaret
Hospital; Hong Kong, China
Objectives: To study the long term
outcome of transfusion dependent thalassaemia major (TM) patients treated
in 3 regional hospitals.
Method: A retrospective study of TM
patients diagnosed since 1970 or since the opening of these hospitals, and
analysis was done at Jan 2001 or at time of death. The patients were
treated with regular blood transfusion and desferrioxamine. Bone marrow
transplantation (BMT) was performed centrally at one hospital.
Result: A total of 232 patients
were diagnosed at the mean age of 14 + 18.8 months and analysed at
15.7 + 6.3 years (range 1.4-30.3). Age of starting desferrioxamine
was 5.2 + 3.1 years. Patients were divided into 3 cohorts: born
before 1980 (cohort 1, n=60), from 1980-1989 (cohort 2, n=116), from 1990
onward (cohort 3, n=56). Cardiomyopathy occurred in 13.6% at age of 16.2 +
4.3 years, and was of 32% and 10% in cohort 1 and 2. Diabetes mellitus
occurred in 7.9% at age of 16 + 2.9 years, and was of 19.6% and 6.1%
in cohort 1 and 2. Hypothyroidism occurred in 6.6%, hypoparathyroidism in
3.5%. None of the patients in cohort 3 developed the above complications.
BMT was performed in 47 patients with 2 years event free survival of 85%.
There were 24 deaths (10.3%) at age of 16.4 + 4.3 years, was 21.6%,
8.6% and 1.8% of cohort 1, 2 and 3 (p=0.03). The commonest cause of death
was cardiomyopathy (62%). Probability of survival to 15, 20 and 25 years in
cohort 1 was 93%, 86% and 80%. The probability of survival to 15 years in
cohort 2 was 95%.
Conclusion: There was significantly
less mortality in patients born after 1980. Cardiomyopathy and other
complications were reduced with regular use of desferrioxamine.