Yetgin Sevgi, Ozsoylu Sinasi
Department of Pediatric Hematology, Faculty of Medicine, Hacettepe University, Ihsan Dogramaci Children's Hospital, Ankara, Turkey.
J Pediatr Hematol Oncol. 2007 Apr;29(4):253-9. doi: 10.1097/MPH.0b013e3180335be0.
Glucocorticoids (GCs) are known for their clinically useful effects in immunologic and inflammatory disorders. Although there is a huge volume of knowledge concerning the cellular and molecular effects of GCs, statements regarding their effects in multiple diseases at variable doses are not clear-cut owing to pharmacogenetic differences. The main actions of GCs in hematologic disorders have been related to their differentiation-inducing and apoptosis-inducing effects, but modification of several steps of the hematopoietic and/or immune pathway has also been reported. In our clinic, mega-dose methylprednisolone (MDMP) has been successfully used for treatment of different hematologic diseases, such as leukemias, bone marrow failure in aplastic anemia, hypoplastic anemia, myelodysplastic syndrome, neutropenia, autoimmune diseases, and in some congenital hereditary diseases. Both clinical and experimental studies in our department revealed that MDMP was more effective than conventional dose steroids. It is interesting that MDMP can be curative in some congenital hereditary diseases such as Diamond-Blackfan syndrome. However, more research is required to clarify their roles in biology, physiology, and molecular genetics.
糖皮质激素（GCs）因其在免疫和炎症性疾病中的临床有益作用而闻名。尽管关于GCs的细胞和分子效应已有大量知识，但由于药物遗传学差异，关于其在多种疾病中不同剂量效应的表述并不明确。GCs在血液系统疾病中的主要作用与其诱导分化和诱导凋亡的效应有关，但也有报道称其对造血和/或免疫途径的多个步骤有调节作用。在我们的临床实践中，大剂量甲泼尼龙（MDMP）已成功用于治疗不同的血液系统疾病，如白血病、再生障碍性贫血的骨髓衰竭、增生低下性贫血、骨髓增生异常综合征、中性粒细胞减少症、自身免疫性疾病以及一些先天性遗传性疾病。我们科室的临床和实验研究均表明，MDMP比传统剂量的类固醇更有效。有趣的是，MDMP对某些先天性遗传性疾病如先天性纯红细胞再生障碍性贫血可能具有治愈作用。然而，需要更多的研究来阐明它们在生物学、生理学和分子遗传学中的作用。
