Complications of beta-thalassemia major in North America.
Treatment of patients with beta-thalassemia major has improved dramatically during the past 40 years; however, the current clinical status of these patients remains poorly characterized. We performed a cross-sectional study of 342 patients in the Registry of the National Institutes of Health-sponsored Thalassemia Clinical Research Network. Evidence of hepatitis C exposure was present in 35% of tested patients, was associated with age, and had a rate of spontaneous viral clearance of 33%. Ferritin levels ranged from 147 to 11 010 ng/mL (median, 1696 ng/mL). Median hepatic iron content was 7.8 mg/g dry weight and 23% of patients had values of 15 mg/g dry weight or higher. No patients 15 years or younger and 5% of patients aged 16 to 24 years had heart disease requiring medication. Ten percent had cirrhosis on biopsy. Endocrinologic complications were common among adults. Seventy-four (22%) patients had recent implantable central venous access devices (CVADs) placed. Among 80 episodes of bacteremia in 38 patients, 90% were attributable to the CVAD. Among 330 patients who had received deferoxamine chelation therapy, 224 (68%) reported no complications. We conclude that hepatitis C, iron-related organ dysfunction, and complications of iron chelation therapy are strongly age-dependent in North American patients with beta-thalassemia.
Children's Hospital, Dana Farber Cancer Institute and Harvard Medical School, Boston, MA 02115, USA.
Melody J
MJ
Cunningham
Eric A
EA
Macklin
Ellis J
EJ
Neufeld
Alan R
AR
Cohen
D000293:Adolescent; D000328:Adult; D000367:Age Factors; D002648:Child; D002675:Child, Preschool; D003430:Cross-Sectional Studies; D003676:Deferoxamine / Q000009:adverse effects; D004700:Endocrine System Diseases / Q000453:epidemiology / Q000209:etiology; D005293:Ferritins / Q000097:blood; D005838:Genotype; D006331:Heart Diseases / Q000453:epidemiology / Q000209:etiology; D006801:Humans; D007223:Infant; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use; D019190:Iron Overload / Q000453:epidemiology; D008099:Liver / Q000737:chemistry / Q000473:pathology; D008103:Liver Cirrhosis / Q000453:epidemiology / Q000209:etiology; D008875:Middle Aged; D012189:Retrospective Studies; D065227:Transfusion Reaction; D017086:beta-Thalassemia / Q000097:blood / Q000150:complications* / Q000453:epidemiology / Q000628:therapy
D007502:Iron Chelating Agents; D005293:Ferritins; D007501:Iron; D003676:Deferoxamine