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利妥昔单抗治疗自身免疫性溶血性贫血和免疫性血小板减少性紫癜:一项比利时回顾性多中心研究。

Rituximab in auto-immune haemolytic anaemia and immune thrombocytopenic purpura: a Belgian retrospective multicentric study.

作者信息

Dierickx Daan, Verhoef G, Van Hoof A, Mineur P, Roest A, Triffet A, Kentos A, Pierre P, Boulet D, Bries G, Lê P-Q, Janssens A, Delannoy A

机构信息

Department of Haematology, University Hospital Gasthuisberg Leuven, 3000 Leuven, Belgium.

出版信息

J Intern Med. 2009 Nov;266(5):484-91. doi: 10.1111/j.1365-2796.2009.02126.x. Epub 2009 Apr 27.

Abstract

OBJECTIVES

For better characterizing the effect of anti-CD20 therapy, we analysed the use of rituximab in Belgian patients experiencing auto-immune haemolytic anaemia (AIHA) and immune thrombocytopenic purpura (ITP).

DESIGN

We performed a retrospective multicentric analysis of patients with AIHA and ITP treated with rituximab in Belgium.

SETTING

Haematological departments were invited to fill in a questionnaire about patient and disease characteristics.

SUBJECTS

All patients with AIHA and ITP, both primary and secondary to other diseases, who received one or more courses of rituximab during their disease course were included. Sixty-eight courses of rituximab in 53 patients with AIHA and 43 courses in 40 patients with ITP were analyzed.

INTERVENTION

Response rates, duration of response and factors predictive for response were assessed.

RESULTS

All patients were given rituximab after failing at least one previous line of treatment, including splenectomy in 19% and 72.5% of AIHA-patients and ITP-patients respectively. Overall response rates were 79.2% in AIHA and 70% in ITP, with a median follow-up since first rituximab administration of 15 months (range 0.5-62) in AIHA and 11 months (range 0-74) in ITP. Progression free survival at 1 and 2 years were 72% and 56% in AIHA and 70% and 44% in ITP. In this retrospective analysis we were not able to identify pretreatment characteristics predictive for response to rituximab. Nine patients with AIHA and three patients with ITP were given one or more additional courses of rituximab. Most of these patients, who had responded to a previous course, experienced a new response comparable to the previous one, both in terms of quality and of duration of response. Finally, the outcome of patients who failed to respond to rituximab therapy was poor both in terms of response to subsequent therapy and in terms of survival.

CONCLUSIONS

This study confirms that rituximab induces responses in a majority of previously treated patients with AIHA and ITP. Response duration generally exceeds 1 year. Retreatment with rituximab in responding patients is most often successful. The outcome of patients who fail on rituximab is poor. We were not able to identify pretreatment patient characteristics predicting for response.

摘要

目的

为了更好地描述抗CD20疗法的效果,我们分析了利妥昔单抗在比利时自身免疫性溶血性贫血(AIHA)和免疫性血小板减少性紫癜(ITP)患者中的应用情况。

设计

我们对比利时接受利妥昔单抗治疗的AIHA和ITP患者进行了一项回顾性多中心分析。

背景

血液科被邀请填写一份关于患者和疾病特征的调查问卷。

研究对象

纳入所有在病程中接受过一个或多个疗程利妥昔单抗治疗的AIHA和ITP患者,包括原发性和继发于其他疾病的患者。分析了53例AIHA患者的68个疗程利妥昔单抗以及40例ITP患者的43个疗程。

干预措施

评估缓解率、缓解持续时间及缓解的预测因素。

结果

所有患者在至少一线先前治疗失败后接受利妥昔单抗治疗,其中分别有19%的AIHA患者和72.5%的ITP患者接受过脾切除术。AIHA的总体缓解率为79.2%,ITP为70%,自首次使用利妥昔单抗给药后的中位随访时间,AIHA为15个月(范围0.5 - 62个月),ITP为11个月(范围0 - 74个月)。AIHA在1年和2年时的无进展生存率分别为72%和56%,ITP分别为70%和44%。在这项回顾性分析中,我们未能确定预测利妥昔单抗反应的预处理特征。9例AIHA患者和3例ITP患者接受了一个或多个额外疗程的利妥昔单抗。这些患者中的大多数之前对一个疗程有反应,在反应质量和持续时间方面都经历了与之前相当的新反应。最后,未对利妥昔单抗治疗产生反应的患者,在后续治疗反应和生存方面结果都很差。

结论

本研究证实利妥昔单抗能使大多数先前接受过治疗的AIHA和ITP患者产生反应。反应持续时间一般超过1年。对有反应的患者再次使用利妥昔单抗治疗大多成功。对利妥昔单抗治疗无效的患者预后较差。我们未能确定预测反应的预处理患者特征。

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