BACKGROUND: The current treatment of thalassaemia major (TM) is based
on a hypertransfusion regimen, with deferoxamine (DFO) chelation thera
py to minimize the consequences of iron overload. To evaluate the long
-term efficacy of chelation therapy, a group of 9 pacients treated for
a period of 9 years was studied. METHODS: The mean age of patients at
the beginning of chelation therapy was 7 years. The age range at the
moment of the study was 11 to 21 years. Pre-transfusion haemoglobin va
lues were maintained above 10 gr/dl. DFO was administered by 10-hour s
ub-cutaneous infusion, 5 or 6 days a week at a dose af 40 mg/kg. Diffe
rent iron overload parameters were considered, with special attention
to cardiac function, growth and endocrinologic development. Signs of D
FO toxicity were also studied. RESULTS: The final mean iron eliminatio
n rate was 72.66%. One patient died from cardiac haemosiderosis. Eight
of the 9 patients showed significant growth impairment and 7, who hav
e attained puberal or post-puberal age, suffer from one or more endocr
inologic disorders (6 hypogonadism, 2 diabetes mellitus, 2 hypothyroid
ism and 1 hypoparathyroidism). The only toxic effect observed was tran
sient crystalline opacity in 2 patients. CONCLUSIONS: Despite the earl
y initiation of chelation therapy, TM patients receiving hypertransfus
ion regimen showed iron overload, with myocardiopathy, growth retardat
ion and several endocrinologic disorders, mainly secondary hypogonadis
m, glucose metabolism disfunction and primary hypothyroidism.