Kwong Yok-Lam, Ha Shau-Yin, Chan Vivian
Department of Medicine, Queen Mary Hospital, University of Hong Kong, Pokfulam Road, Hong Kong, China.
Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, Pokfulam Road, Hong Kong, China.
Hematol Oncol Clin North Am. 2016 Apr;30(2):445-56. doi: 10.1016/j.hoc.2015.11.010. Epub 2016 Feb 2.
Thalassemias and hemophilias are the most important inherited hematological diseases in Hong Kong and China. Prenatal diagnosis has significantly decreased the burden of these diseases. For thalassemia major, adequate transfusion and iron chelation therapy have dramatically improved patient outlook. Hematopoietic stem cell transplantation is curative for thalassemia major and is increasingly adopted. The efficacy of arsenic trioxide in acute promyelocytic leukemia (APL) was discovered in China. An oral formulation of arsenic trioxide was developed in Hong Kong for newly diagnosed and relapsed APL patients. With combination chemotherapy containing non-P-glycoprotein-dependent drugs and L-asparaginase, durable remission can be achieved in the most patients.
地中海贫血和血友病是中国香港和内地最重要的遗传性血液疾病。产前诊断显著减轻了这些疾病的负担。对于重型地中海贫血，充足的输血和铁螯合疗法显著改善了患者的预后。造血干细胞移植可治愈重型地中海贫血，且应用越来越广泛。三氧化二砷治疗急性早幼粒细胞白血病（APL）的疗效是在中国发现的。香港研发了一种三氧化二砷口服制剂，用于新诊断和复发的APL患者。联合使用不含P-糖蛋白依赖性药物和L-天冬酰胺酶的化疗方案，大多数患者可实现持久缓解。
