Kotb Rami, Pinganaud Caroline, Trichet Catherine, Lambotte Olivier, Dreyfus Marie, Delfraissy Jean-François, Tchernia Gil, Goujard Cécile
Department of Internal Medicine, Hôpital de Bicêtre, AP-HP, Faculte de Medecine Paris-Sud, 78 rue du Général Leclerc, 94275 Le Kremlin-Bicêtre, France.
Eur J Haematol. 2005 Jul;75(1):60-4. doi: 10.1111/j.1600-0609.2005.00437.x.
Treatment of auto-immune cytopenia refractory to front line therapy with intravenous immunoglobulins and steroids is a matter of concern. We assessed the efficacy and safety of mycophenolate mofetil in a prospective open preliminary study.
Adult patients with steroid refractory auto-immune cytopenias were included. Mycophenolate mofetil (MMF) was added to treatment given at the time of inclusion, and efficacy was evaluated in term of improvement of platelet/haemoglobin levels and in term of reduction of previously given drugs, if any. All auto-immune thrombocytopenic purpura (AITP) patients had serologic assessment for associated auto-antibodies at the time of inclusion. Cytopenias associated with other auto-immune diseases, lymphoproliferative diseases or HIV infection were excluded.
From November 1999 through November 2003, 13 patients were included (nine AITP, three auto-immune haemolytic anaemia (AIHA), one Evans' syndrome; four males, nine females; age: 35-72 yr). For AITP patients, an overall response of 78% was observed. Retrospective analysis showed no significant difference between patients having a short disease duration (<1 yr) and longer disease duration; between patients who previously received more or less than three treatments; and between patients for whom MMF was started as monotherapy or in association with prednisone, However, all AITP patients presenting associated auto-antibodies responded to MMF, while only 50% of patients without associated antibodies were responders. All patients presenting AIHA and Evans' syndrome were responders. The drug was well tolerated, with no significant side effects reported. The cumulative data suggest a potential place for MMF in the treatment arsenal of refractory cytopenias.
对于一线治疗（静脉注射免疫球蛋白和类固醇）难治的自身免疫性血细胞减少症的治疗是一个令人关注的问题。我们在一项前瞻性开放性初步研究中评估了霉酚酸酯的疗效和安全性。
纳入对类固醇难治的自身免疫性血细胞减少症的成年患者。在纳入时将霉酚酸酯（MMF）添加到治疗中，并根据血小板/血红蛋白水平的改善情况以及先前使用药物（如有）的减少情况来评估疗效。所有自身免疫性血小板减少性紫癜（AITP）患者在纳入时均进行了相关自身抗体的血清学评估。排除与其他自身免疫性疾病、淋巴增生性疾病或HIV感染相关的血细胞减少症。
从1999年11月至2003年11月，纳入了13例患者（9例AITP、3例自身免疫性溶血性贫血（AIHA）、1例伊文氏综合征；4例男性，9例女性；年龄：35 - 72岁）。对于AITP患者，观察到总体缓解率为78%。回顾性分析显示，疾病持续时间短（<1年）和长的患者之间；先前接受治疗或多或少少于三次的患者之间；以及MMF作为单一疗法或与泼尼松联合开始治疗的患者之间，均无显著差异。然而，所有存在相关自身抗体的AITP患者对MMF有反应，而无相关抗体的患者只有50%有反应。所有表现为AIHA和伊文氏综合征的患者均有反应。该药物耐受性良好，未报告有显著副作用。累积数据表明MMF在难治性血细胞减少症的治疗手段中可能占有一席之地。
