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تسجيل

Survival in Patients With Thalassemia Major

المؤلف:  Hoffman, R., Benz, E. J., Silberstein, L. E., Heslop, H., Weitz, J., & Salama, M. E.

المصدر:  Hematology : Basic Principles and Practice

الجزء والصفحة:  8th E , P574

2026-04-28

399

 Improved transfusion therapy and the consistent use of iron chelation therapy have extended the life span of patients with thalassemia major. In a multicenter study of 1079 patients in Italy, the probability of survival to age 20 years was 96% for patients born between 1975 and 1979, the time at which chelation therapy became a regular part of the overall management of thalassemia major (Table 1).  In contrast, the probabilities of survival at 20 years of age were only 61% and 69% for those born in the periods of 1960 through 1964 and 1965 through 1969, respectively. Other investigators have shown that survival or prevention of life-threatening com plications is strongly related to good chelation therapy, assessed either by compliance or by control of iron stores (Fig. 1). The importance of good compliance with chelation therapy is further demonstrated by data from the United Kingdom showing that the probability of survival for the 1975 through 1984 birth cohort is to date not substantially different than the probability of survival for the 1965 through 1974 birth cohort.  The researchers attribute this poorer than expected survival rate, despite the availability of deferoxamine, to a lack of adherence to the recommended schedule of treatment with this chelator.

Table1. Survival by Birth Cohort at Different Ages of Patients  With Transfusion-Dependent Thalassemia

Fig1. SURVIVAL WITHOUT CARDIAC DISEASE IN PATIENTS WITH THALASSEMIA MAJOR TREATED WITH DEFEROXAMINE ACCORDING TO THE PROPORTION OF SERUM FERRITIN MEASUREMENTS EXCEEDING 2500 ng/ml. The circles show cardiac disease-free survival among patients in whom less than 33% of ferritin measurements exceeded 2500 ng/mL; squares show survival among patients in whom 33% to 67% of ferritin measurements exceeded 2500 ng/mL; and triangles show survival among patients in whom more than 67% of ferritin measurements exceeded 2500 ng/mL. (Adapted from Olivieri NF, Nathan DG, MacMillan JH, et al. Survival in medically treated patients with homozygous beta-thalassemia. N Engl J Med. 1994;331:574.)

Consistent compliance with chelation to maintain a near-normal iron balance is undoubtedly the key to preventing complications in regularly transfused patients. Currently, with the advances in management of thalassemia, including a safer blood supply, lower rates of alloimmunization, oral chelators, improved methods of quantifying organ iron and tailoring chelation regimens, it may be fair to say that TDT individuals who are optimally managed with transfusions and chelation should anticipate near normal lifespans.