Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine.
OBJECTIVE
Seven Italian centers reported data on survival, causes of death and appearance of complications in patients with thalassemia major. The interactions between gender, birth cohort, complications, and ferritin on survival and complications were analyzed.


METHODS
Survival after the first decade was studied for 977 patients born since 1960 whereas survival since birth and complication appearance was studied for the 720 patients born after 1970. Better survival was demonstrated for patients born in more recent years (p<0.00005) and for females (p=0.0003); 68% of the patients are alive at the age of 35 years. In the entire population 67% of the deaths were due to heart disease.


RESULTS
There was a significant association between birth cohort and complication-free survival (p<0.0005). The prevalence of complications was: heart failure 6.8%, arrhythmia 5.7%, hypogonadism 54.7%, hypothyroidism 10.8%, diabetes 6.4%, HIV infection 1.7%, and thrombosis 1.1%. Lower ferritin levels were associated with a lower probability of heart failure (hazard ratio =3.35, p<0.005) and with prolonged survival (hazard ratio = 2.45, p<0.005), using a cut-off as low as 1,000 ng/mL.


CONCLUSIONS
Survival and complication-free survival of patients with thalassemia major continue to improve, especially for female patients born shortly before or after the availability of iron chelation.
Department of Pediatrics, University of Ferrara, Italy.bre@unife.it
Caterina
C
Borgna-Pignatti
Simone
S
Rugolotto
Piero
P
De Stefano
Huaqing
H
Zhao
Maria Domenica
MD
Cappellini
Giovanni Carlo
GC
Del Vecchio
Maria Antonietta
MA
Romeo
Gian Luca
GL
Forni
Maria Rita
MR
Gamberini
Roberta
R
Ghilardi
Antonio
A
Piga
Avital
A
Cnaan
D000328:Adult; D000367:Age Factors; D001145:Arrhythmias, Cardiac / Q000453:epidemiology / Q000209:etiology; D001803:Blood Transfusion; D015913:Chelation Therapy / Q000009:adverse effects; D015331:Cohort Studies; D003676:Deferoxamine / Q000009:adverse effects / Q000627:therapeutic use*; D003922:Diabetes Mellitus, Type 1 / Q000453:epidemiology / Q000209:etiology; D005260:Female; D005293:Ferritins / Q000032:analysis; D015658:HIV Infections / Q000453:epidemiology / Q000209:etiology; D006333:Heart Failure / Q000453:epidemiology / Q000209:etiology; D006801:Humans; D007006:Hypogonadism / Q000453:epidemiology / Q000209:etiology; D007037:Hypothyroidism / Q000453:epidemiology / Q000209:etiology; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007558:Italy / Q000453:epidemiology; D015993:Life Tables; D008297:Male; D012189:Retrospective Studies; D013156:Splenectomy; D016019:Survival Analysis; D013789:Thalassemia / Q000150:complications / Q000188:drug therapy / Q000401:mortality / Q000628:therapy*; D013927:Thrombosis / Q000453:epidemiology / Q000209:etiology; D065227:Transfusion Reaction
D007502:Iron Chelating Agents; D005293:Ferritins; D003676:Deferoxamine