Mesa Ruben A
Division of Hematology, Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA.
Curr Hematol Rep. 2003 May;2(3):264-70.
Myelofibrosis with myeloid metaplasia (MMM) is a progressive, clonal, and lethal myeloproliferative disorder with a variable prognosis. The only potentially curative therapy for MMM is allogeneic stem cell transplantation, but this therapy is too toxic for older patients and inappropriate for young patients with good prognostic features. A nonmyeloablative or autologous stem cell transplant may broaden the applicability of high-dose therapy for MMM, but the therapy for MMM is palliative in intent and aimed at symptomatic relief. There is no approved therapy for MMM; however, certain agents have been useful for targeted palliation. Myelosuppressive drug therapy, targeted radiation, or splenectomy has decreased symptomatic myeloproliferation (i.e., splenomegaly). MMM-associated cytopenias may be improved by the selective use of growth factors, androgens, and thalidomide. Debilitating constitutional symptoms improve with the pharmacologic blockage of tumor necrosis factor-alpha. Further molecular understanding of MMM is necessary for more effective targeted therapies.
伴有髓外化生的骨髓纤维化（MMM）是一种进行性、克隆性且致命的骨髓增殖性疾病，预后各异。MMM唯一可能治愈的疗法是异基因干细胞移植，但该疗法对老年患者毒性过大，对预后良好的年轻患者也不适用。非清髓性或自体干细胞移植可能会扩大高剂量疗法对MMM的适用性，但MMM的治疗目的是姑息性的，旨在缓解症状。目前尚无获批用于MMM的疗法；然而，某些药物已被证明对靶向姑息治疗有效。骨髓抑制药物治疗、靶向放疗或脾切除术可减轻有症状的骨髓增殖（即脾肿大）。选择性使用生长因子、雄激素和沙利度胺可能改善与MMM相关的血细胞减少。通过肿瘤坏死因子-α的药物阻断可改善使人虚弱的全身症状。为了实现更有效的靶向治疗，有必要对MMM进行进一步的分子研究。
