Merup M, Kutti J, Birgergård G, Mauritzson N, Björkholm M, Markevärn B, Maim C, Westin J, Palmblad J
Department of Hematology, Huddinge University Hospital, Stockholm, Sweden.
Med Oncol. 2002;19(2):79-86. doi: 10.1385/mo:19:2:79.
We conducted a nonrandomized prospective phase II study of thalidomide in anemic patients with myelofibrosis with myeloid metaplasia (MMM), with or without preceding polycythemia vera or essential thrombocythemia, with a primary aim to improve anemia. Thalidomide was given in escalating doses with a target dose of 800 mg daily, but the median dose of thalidomide that was actually tolerated was 400 mg daily. Fifteen patients were entered into the study and 14 were evaluable for response. Five of 14 (36%) patients discontinued thalidomide before 3 mo because of side effects, and none of these five patients had a response at the time when thalidomide was stopped. When evaluated after 3 mo of therapy, none of the remaining nine patients exhibited a discernible clinical response. Three patients showed progressive disease defined as > 50% increase in the need for red cell transfusions. Treatment was poorly tolerated, with all patients reporting side effects of thalidomide, the most prominent being fatigue documented in 80% of patients. Two patients died while on study, one from acute myelogenous leukemia and one from pneumonia. We conclude that thalidomide given in doses employed in the treatment of multiple myeloma gives no clinically relevant hematological effects in advanced MMM and is hampered by a very high incidence of side effects.
我们开展了一项非随机前瞻性II期研究，对象为患有骨髓纤维化伴髓样化生（MMM）的贫血患者，这些患者既往有或无真性红细胞增多症或原发性血小板增多症，主要目的是改善贫血状况。沙利度胺以递增剂量给药，目标剂量为每日800毫克，但实际耐受的沙利度胺中位剂量为每日400毫克。15名患者进入研究，14名患者可评估疗效。14名患者中有5名（36%）因副作用在3个月前停用沙利度胺，这5名患者在停用沙利度胺时均无反应。在治疗3个月后评估时，其余9名患者均未表现出明显的临床反应。3名患者病情进展，定义为红细胞输注需求增加>50%。治疗耐受性差，所有患者均报告有沙利度胺的副作用，最突出的是80%的患者出现疲劳。2名患者在研究期间死亡，1名死于急性髓性白血病，1名死于肺炎。我们得出结论，用于治疗多发性骨髓瘤的剂量的沙利度胺在晚期MMM中未产生临床相关的血液学效应，且因副作用发生率极高而受到阻碍。
