Comparison of oral and subcutaneous iron chelation therapies in the prevention of major endocrinopathies in beta-thalassemia major patients.
While hypertransfusion and subcutaneous iron chelation therapy have increased longevity of patients with beta-thalassemia (thal) major, endocrinopathies have become more common and impair the quality of their lives. Additionally, subcutaneous iron chelation therapy is an uncomfortable experience and can prevent patients from regular compliance with iron chelation therapy. We compared the efficacy of oral deferiprone (L1) to subcutaneous desferrioxamine (DFO) chelation therapy for the prevention of major endocrinopathies (growth hormone insufficiency, diabetes mellitus and gonadal dysfunction) among patients with beta-thal major to see if we could offer these patients an easier and more painless way to reduce their body iron load and related endocrine complications.
Wang
Chung-Hsing
CH
Department of Pediatrics, China Medical University Hospital, Taichung, Taiwan.
Wu
Kang-Hsi
KH
Tsai
Fuu-Jen
FJ
Peng
Ching-Tien
CT
Tsai
Chang-Hai
CH
D000284:Administration, Oral; D000293:Adolescent; D000328:Adult; D015913:Chelation Therapy / Q000379:methods*; D002648:Child; D003131:Combined Modality Therapy; D000077543:Deferiprone; D003676:Deferoxamine / Q000008:administration & dosage / Q000627:therapeutic use*; D003920:Diabetes Mellitus / Q000209:etiology / Q000517:prevention & control; D004393:Dwarfism, Pituitary / Q000209:etiology / Q000517:prevention & control; D004700:Endocrine System Diseases / Q000209:etiology / Q000517:prevention & control*; D005260:Female; D019382:Human Growth Hormone / Q000172:deficiency; D006801:Humans; D007006:Hypogonadism / Q000209:etiology / Q000517:prevention & control; D007263:Infusions, Parenteral; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology; D008297:Male; D010349:Patient Compliance; D011628:Puberty, Delayed / Q000209:etiology / Q000517:prevention & control; D011728:Pyridones / Q000008:administration & dosage / Q000627:therapeutic use*; D040521:Subcutaneous Tissue; D065227:Transfusion Reaction; D017086:beta-Thalassemia / Q000150:complications / Q000188:drug therapy* / Q000628:therapy
D007502:Iron Chelating Agents; D011728:Pyridones; D019382:Human Growth Hormone; D000077543:Deferiprone; D003676:Deferoxamine