b-thalassemia in Germany - Results of the cooperative beta-thalassemia study

At present, about 300 patients with thalassemia major are living in Germany . Starting in 1991, a multicenter study in Germany has concentrated on iden tifying all patients suffering from thalassemia as well as on establishing a uniform therapy protocol including follow-up diagnostic procedures. After six years of study, the data of 198 patients suffering from thalassae mia major were analysed. The majority of these patients originate from ende mic regions around the Mediterranean Sea. The patient's median age is 13.8 years (range 1-37.5 yrs.). At present, about 20% of patients are older than 21 years. Regarding transfusion therapy, a shortening of the average transfusion inte rval to 3 weeks in most cases occurred. Throughout the entire period, media n baseline haemoglobin concentrations of 10.0 g/dl could be observed. The evaluation of serum ferritin levels revealed considerable differences d epending on patients age. 60% of patients in the first decade of life showe d good therapeutic results with serum ferritin levels below 1800 ng/ml. In contrast, 52% of patients older than ten years presented with ferritin leve ls above 2500 ng/ml. During the observation, a decreasing number of patient s with ferritin levels above 2500 ng/ml was observed in patients aged 15 to 21 years of age. The situation of patients aged 9 to 15 years proved to be more problematic. More than half of all treated patients presented with siderotic complicatio ns as cardiac disease in 13%, liver disease in 21%, impaired glucose metabo lism in 14%, hypothyroidism in 24% and hypogonadism in 59% of all patients. These values did not change considerably during the observation apart from an increase of cardiac disorders to 20%. Since the situation concerning si derosis and the lack of compliance proved to be particularly difficult in a dolescent patients, further efforts has to concentrate on this age group.