Update on thalassemia treatment in Taiwan, including bone marrow transplantation, chelation therapy, and cardiomyopathy treatment effects.
Over the past few decades, Taiwan has seen striking improvements in the life expectancy of its 400 registered beta-thalassemia major (beta-TM) patients due mainly to adequate transfusion regimens and effective iron chelation therapy. Since 1995, Taiwanese citizens have enjoyed universal health care through National Health Insurance (NIH), receiving comprehensive treatment at minimal cost. In 1984, a national program for thalassemia prevention, control, and hematopoietic stem cell transplantation (HSCT) was initiated. Recent data show 1- and 2-year event-free survival rates of 85 and 78%, respectively. Chelation agents like deferoxamine (DFO), deferiprone (L1) and deferasirox (DFRA) are available in Taiwan, and therapy is tailored to individuals based on drug availability and tissue distribution of iron load. Intensive chelation regimens combining L1 and DFO are recommended in patients with cardiac complications, while DFRA has been found to be effective in reducing serum ferritin, with acceptable side effects. Here, we report advances in thalassemia treatment in Taiwan and suggest treatment guidelines.
Peng
Ching-Tien
CT
Department of Pediatrics, China Medical University Hospital, Taichung, Taiwan.
Chang
Jeng-Sheng
JS
Wang
Lin-Yen
LY
Chiou
Shyh-Shin
SS
Hsiao
Chih-Cheng
CC
Wang
Shih-Chung
SC
Hung
Giun-Yi
GY
Wu
Kang-Hsi
KH
D001565:Benzoates / Q000008:administration & dosage / Q000627:therapeutic use; D001803:Blood Transfusion; D016026:Bone Marrow Transplantation; D009202:Cardiomyopathies / Q000188:drug therapy*; D015913:Chelation Therapy; D002986:Clinical Trials as Topic; D003131:Combined Modality Therapy; D000077588:Deferasirox; D000077543:Deferiprone; D003676:Deferoxamine / Q000008:administration & dosage / Q000627:therapeutic use; D005293:Ferritins / Q000097:blood; D017408:Guidelines as Topic; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D011728:Pyridones / Q000008:administration & dosage / Q000627:therapeutic use; D017262:Siderophores / Q000008:administration & dosage / Q000627:therapeutic use; D012806:Siderosis / Q000188:drug therapy*; D013624:Taiwan; D016896:Treatment Outcome; D014230:Triazoles / Q000008:administration & dosage / Q000627:therapeutic use; D017086:beta-Thalassemia / Q000188:drug therapy / Q000601:surgery / Q000628:therapy*
D001565:Benzoates; D007502:Iron Chelating Agents; D011728:Pyridones; D017262:Siderophores; D014230:Triazoles; D000077543:Deferiprone; D005293:Ferritins; D003676:Deferoxamine; D000077588:Deferasirox