原发性免疫性血小板减少症中促血小板生成素受体激动剂：病例系列描述及反应预测因素分析
TPO-RAs in pITP: description of a case series and analysis of predictive factors for response.
作者信息
Mazzucconi Maria Gabriella, Santoro Cristina, Baldacci Erminia, De Angelis Federico, Chisini Marta, Ferrara Grazia, Volpicelli Paola, Foà Roberto
机构信息
Hematology, Department of Cellular Biotechnologies and Hematology, 'Sapienza' University of Rome, Rome, Italy.
出版信息
Eur J Haematol. 2017 Mar;98(3):242-249. doi: 10.1111/ejh.12822. Epub 2016 Nov 18.
OBJECTIVE
To report our experience concerning sustained response (SR) after TPO-RA discontinuation in adult pITP patients and to identify possible predictive factors for outcome.
METHODS
Thirty-nine pITP patients who received a TPO-RA were evaluated. Response (R) was defined as a platelet count ≥30 × 10 /L and at least a twofold increase in the baseline count and complete response (CR) as a platelet count ≥100 × 10 /L, in the absence of bleeding. Durable response (DR) was defined as a R/CR persisting ≥4 wk with a stable dose of TPO-RA, and SR as the first assessed platelet count ≥30 × 10 /L, available at more than 4 wk after discontinuation of TPO-RA, in the absence of other concomitant or rescue therapies.
RESULTS
Twenty-nine/39 (74%) were responders: 18 (46%) reached a CR and 11 (28%) a R. A DR was observed in 16/29 (55%) responders. Seven SR (18%) were reached: five of seven patients achieved a SR from a prior DR. CR was statistically associated with the achievement of a subsequent DR: 13/18 (72%) CR patients obtained a DR, while only three of 11 (27%) R ones did (P = 0.027).
CONCLUSIONS
CR was a significant prognostic factor for the achievement of a DR. Moreover, we observed a trend for DR patients to obtain a subsequent SR.
目的
报告我们关于成人慢性免疫性血小板减少症（pITP）患者停用促血小板生成素受体激动剂（TPO-RA）后持续缓解（SR）的经验，并确定可能的预后预测因素。
方法
对39例接受TPO-RA治疗的pITP患者进行评估。缓解（R）定义为血小板计数≥30×10⁹/L且至少为基线计数的两倍增加，完全缓解（CR）定义为血小板计数≥100×10⁹/L且无出血。持久缓解（DR）定义为在稳定剂量的TPO-RA治疗下R/CR持续≥4周，SR定义为在停用TPO-RA后4周以上首次评估的血小板计数≥30×10⁹/L，且无其他伴随或挽救治疗。
结果
29/39（74%）例患者有反应：18例（46%）达到CR，11例（28%）达到R。16/29（55%）例有反应者观察到DR。达到7例SR（18%）：7例患者中有5例从先前的DR达到SR。CR与随后达到DR在统计学上相关：18例CR患者中有13例（72%）获得DR，而11例R患者中只有3例（27%）获得DR（P = 0.027）。
结论
CR是实现DR的重要预后因素。此外，我们观察到DR患者有获得后续SR的趋势。
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