一项关于艾曲泊帕对慢性免疫性血小板减少症患者骨髓影响的为期2年的纵向前瞻性研究。

A 2-Year, Longitudinal, Prospective Study of the Effects of Eltrombopag on Bone Marrow in Patients with Chronic Immune Thrombocytopenia.

作者信息

Brynes Russell K, Wong Raymond S M, Thein Maung M, Bakshi Kalpana K, Burgess Paul, Theodore Dickens, Orazi Attilio

机构信息

Department of Pathology, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.

出版信息

Acta Haematol. 2017;137(2):66-72. doi: 10.1159/000452992. Epub 2016 Dec 23.

DOI:10.1159/000452992

PMID:28006767

Abstract

BACKGROUND

The long-term effects of eltrombopag on bone marrow (BM) reticulin and/or collagen deposition in previously treated adults with chronic immune thrombocytopenia (ITP) were assessed.

METHODS

Three BM biopsies were collected at baseline and after 1 and 2 years of eltrombopag treatment. Specimens were centrally processed, stained for reticulin and collagen, independently reviewed by 2 hematopathologists, and rated according to the European Consensus 0-3 scale of marrow fibrosis (MF).

RESULTS

Of 162 patients enrolled, 93 completed all 3 protocol-specified BM biopsies. All patients with a baseline assessment were negative for collagen. Of 159 patients assessed at baseline, 150 (94%) had normal reticulin (MF-0) and 9 (6%) had minimally increased reticulin (MF-1). After 2 years, 83/93 patients (89%) with BM biopsies had MF-0, 10 (11%) had MF-1, and none had MF-2 or MF-3. Five out of 127 patients (4%) at 1 year and 1 out of 93 (1%) at 2 years had collagen deposition. None of the patients had clinical symptoms typical of BM dysfunction or abnormalities of clinical concern based on white blood cell count or peripheral blood smear.

CONCLUSION

For most patients with chronic ITP, eltrombopag is not associated with clinically relevant increases in BM reticulin or collagen formation.

摘要

背景

评估了艾曲泊帕对既往接受治疗的慢性免疫性血小板减少症（ITP）成人患者骨髓（BM）网硬蛋白和/或胶原蛋白沉积的长期影响。

方法

在基线以及艾曲泊帕治疗1年和2年后采集三次骨髓活检样本。样本进行集中处理，进行网硬蛋白和胶原蛋白染色，由2名血液病理学家独立评估，并根据欧洲骨髓纤维化（MF）共识0 - 3级进行评分。

结果

162名入组患者中，93名完成了所有3次方案规定的骨髓活检。所有进行基线评估的患者胶原蛋白均为阴性。在159名进行基线评估的患者中，150名（94%）网硬蛋白正常（MF - 0），9名（6%）网硬蛋白轻度增加（MF - 1）。2年后，93名进行骨髓活检的患者中，83名（89%）为MF - 0，10名（11%）为MF - 1，无患者为MF - 2或MF - 3。127名患者中有5名（4%）在1年时出现胶原蛋白沉积，93名患者中有1名（1%）在2年时出现胶原蛋白沉积。所有患者均无典型的骨髓功能障碍临床症状，也无基于白细胞计数或外周血涂片的临床相关异常。