Bachleitner-Hofmann T, Gisslinger H
University of Vienna, Department of Internal Medicine I, Austria.
Ann Hematol. 1999 Dec;78(12):533-8. doi: 10.1007/s002770050554.
Idiopathic myelofibrosis (IMF) is a chronic myeloproliferative disorder characterized by fibrosis of the bone marrow, varying degrees of extramedullary hematopoiesis, splenomegaly, anemia, and a leukoerythroblastic peripheral blood smear. Bone marrow fibrosis develops as a secondary phenomenon and is caused by increased intramedullary activity of mitogens such as platelet-derived growth factor (PDGF), transforming growth factor-beta (TGF-beta), basic fibroblast growth factor (bFGF), epidermal growth factor (EGF), and calmodulin. Because of the variable clinical course of IMF, attempts have been made to define prognostic parameters that can be helpful in detecting patients with a shortened life expectancy. The most important adverse prognostic parameters that have been reported are hemoglobin concentration, age, leukocyte count, number of thrombocytes, and cytogenetic abnormalities. However, no standardized prognostic score for IMF has yet been established. Therapeutic strategies in IMF remain predominantly supportive. The most common are blood transfusions, androgens, and cytoreductive agents such as hydroxyurea. Bone marrow transplantation is increasingly being taken into consideration, but it still has to be regarded as an experimental approach. Interferon-alpha (IFN-alpha) has shown promising results in early hyperproliferative stages of IMF but has no or only very little effect in more advanced stages of the disease. Whether IFN-alpha is able to postpone marrow fibrosis if administered in early disease stages remains to be determined in future clinical trials.
原发性骨髓纤维化（IMF）是一种慢性骨髓增殖性疾病，其特征为骨髓纤维化、不同程度的髓外造血、脾肿大、贫血以及外周血涂片出现幼稚粒-幼红细胞。骨髓纤维化作为一种继发现象出现，是由骨髓内促有丝分裂原活性增加所致，如血小板衍生生长因子（PDGF）、转化生长因子-β（TGF-β）、碱性成纤维细胞生长因子（bFGF）、表皮生长因子（EGF）和钙调蛋白。由于IMF的临床病程多变，人们一直试图确定有助于检测预期寿命缩短患者的预后参数。已报道的最重要的不良预后参数是血红蛋白浓度、年龄、白细胞计数、血小板数量和细胞遗传学异常。然而，尚未建立针对IMF的标准化预后评分。IMF的治疗策略主要仍为支持性治疗。最常见的是输血、雄激素以及细胞减灭剂如羟基脲。骨髓移植越来越多地被考虑，但仍必须将其视为一种实验性方法。α干扰素（IFN-α）在IMF的早期增殖阶段已显示出有前景的结果，但在疾病的更晚期则没有效果或只有非常小的效果。在疾病早期给予IFN-α是否能够延缓骨髓纤维化仍有待未来临床试验确定。
