利妥昔单抗治疗免疫性血小板减少症：性别、年龄及反应作为长期缓解的预测因素

Rituximab in immune thrombocytopenia: gender, age, and response as predictors of long-term response.

作者信息

Marangon Miriam, Vianelli Nicola, Palandri Francesca, Mazzucconi Maria Gabriella, Santoro Cristina, Barcellini Wilma, Fattizzo Bruno, Volpetti Stefano, Lucchini Elisa, Polverelli Nicola, Carpenedo Monica, Isola Miriam, Fanin Renato, Zaja Francesco

机构信息

Institute of Hematology L.A. Seragnoli, University of Bologna, Bologna, Italy.

Ematologia, Dipartimento di Biotecnologie Cellulari ed Ematologia, Università Sapienza, Roma, Italy.

出版信息

Eur J Haematol. 2017 Apr;98(4):371-377. doi: 10.1111/ejh.12839. Epub 2017 Jan 20.

DOI:10.1111/ejh.12839

PMID:27981682

Abstract

OBJECTIVES

To evaluate the efficacy of a salvage treatment with rituximab (RTX) in adults with primary immune thrombocytopenia (ITP), in terms of short-term response and long-term response (LTR, i.e., probability to achieve and maintain response) and to identify biological and clinical predictors of response.

METHODS

We retrospectively evaluated the outcome of patients with primary ITP treated with standard dosage RTX (375 mg/m × 4) as salvage therapy in five Italian centers. One hundred and three patients, median age of 46 yr, were included. The median period of observation was 59 months.

RESULTS

Response (R) and complete response (CR) were documented in 57 (55%) and 37 (36%) patients, respectively. Patients younger than 40 yr had a higher probability to achieve CR (P = 0.025). Younger women (age < 40 yr) had a significantly higher probability to achieve R and CR (P = 0.039 and P = 0.009, respectively). The estimated LTR rate was 36% and 31% after 48 and 72 months, respectively; female sex (P = 0.033) and younger age (P = 0.021) were associated with better LTR. Younger women had the highest LTR rate (P = 0.006). Response duration was associated with the obtainment of CR after RTX (CR vs. partial response, P = 0.002).

CONCLUSIONS

The effect of RTX salvage treatment appears higher in younger women, with LTR rate possibly approaching that of splenectomy.

摘要

目的

评估利妥昔单抗（RTX）挽救治疗对成人原发性免疫性血小板减少症（ITP）的疗效，包括短期反应和长期反应（LTR，即实现并维持反应的概率），并确定反应的生物学和临床预测因素。

方法

我们回顾性评估了意大利五个中心采用标准剂量RTX（375mg/m×4）作为挽救治疗的原发性ITP患者的结局。纳入了103例患者，中位年龄为46岁。中位观察期为59个月。

结果

分别有57例（55%）和37例（36%）患者记录到反应（R）和完全反应（CR）。年龄小于40岁的患者实现CR的概率更高（P = 0.025）。年轻女性（年龄<40岁）实现R和CR的概率显著更高（分别为P = 0.039和P = 0.009）。48个月和72个月后的估计LTR率分别为36%和31%；女性（P = 0.033）和年轻年龄（P = 0.021）与更好的LTR相关。年轻女性的LTR率最高（P = 0.006）。反应持续时间与RTX治疗后获得CR相关（CR与部分反应，P = 0.002）。