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临床试验/NCT02063763
NCT02063763已完成不适用

Use of TPO-mimetics to Prepare for Splenectomy in Adult Patients With Primary Immune Thrombocytopenia. Brooklyn Observational Retrospective Study.

Gruppo Italiano Malattie EMatologiche dell'Adulto14 个研究点 分布在 1 个国家目标入组 31 人开始时间: 2014年5月14日最近更新:
适应症

试验速览

阶段
不适用
状态
已完成
入组人数
31
试验地点
14
主要终点
Number of patients responding to TPO-mimetics

研究概览

简要总结

This study aims at analyzing the therapeutic activity of TPO-mimetics Eltrombopag and Romiplostim as bridge therapy for splenectomy in adult patients with primary immune thrombocytopenia.

详细描述

Adult immune primary thrombocytopenia is an autoimmune malignancy characterized by platelet destruction and inadequate platelet production. Its incidence is of three cases per 100,000 people a year, with a prevalence in women in young and older adults.

Splenectomy is, still today, the therapeutic approach that offers a bigger guarantee of a long term response (around 60+70%). Nevertheless, splenectomy may be accompanied by peri-operative complications in almost 10% of patients, which in rare cases may be fatal. These are normally hemorrhagic complications due to low platelet count. Thus, a previous therapy to increase platelets is advisable before any splenectomy.

In patients who are refractory to corticosteroids and immunoglobulins or when its use is not indicated, splenectomy has a potential risk of more complications. Since the last few years, we count with TPO-mimetic drugs, specific for c-MPL receptor, able to stimulate platelet production, such as romiplostim and eltrombopag.

Nowadays, TPO-mimetics are allowed in Italy when patients refuse splenectomy or when splenectomy is not contraindicated, but being ITP with a low platelet count (< 20-50.000/mmc) a potential contraindication to splenectomy due to hemorrhagic events, these drugs should be considered. Nevertheless, there are no data on therapeutic risks and safety of these agents when used for this indication. The aim of the present study is to verify, on an Italian national scale, the frequency of use, its impact and the safety profile.

研究设计

研究类型
Observational
观察模型
Case Only
时间视角
Retrospective

入排标准

年龄范围
18 Years 至 —(Adult, Older Adult)
性别
All
接受健康志愿者

入选标准

  • Persistent or chronic ITP in symtomatic phase.
  • 18 years of age or older.
  • Indication for splenectomy due to refractory response to a previous therapy.
  • Have used eltrombopag or romiplostim to increase platelet count before splenectomy.
  • Have undergone splenectomy.

排除标准

  • No informed consent.

结局指标

主要结局

Number of patients responding to TPO-mimetics

时间窗: Six months.

According to platelet count and presence or absence of hemorrhagic events.

次要结局

  • Frequency and ways of administration.(Six months)
  • Number of patients on TPO-mimetics eltrombopag and romiplostim as bridge therapy before splenectomy.(Six months.)
  • Number of hemorrhagic events.(At thirty days from splenectomy.)
  • Frequency of toxicity.(Six months)

研究者

申办方类型
Other
责任方
Sponsor

研究点 (14)

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