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在艾曲泊帕时代之前30多年来小儿慢性免疫性血小板减少症的经验

Experience with Pediatric Chronic Immune Thrombocytopenia over 30 Years in the Era before Eltrombopag.

作者信息

Koc Begum S, Ozdemir Gul Nihal, Alakbarli Javid, Apak Hilmi, Celkan Tiraje

机构信息

Department of Pediatric Hematology and Oncology, Cerrahpasa Medical Faculty, Istanbul University, Istanbul 34696, Turkey.

Department of Pediatric Hematology and Oncology, Istinye University, Istanbul 34320, Turkey.

出版信息

Children (Basel). 2024 Aug 28;11(9):1051. doi: 10.3390/children11091051.

Abstract

BACKGROUND

There is limited information on the natural course of chronic ITP in children. We aimed to evaluate the clinical and demographic characteristics of children with chronic ITP in the era before the availability of eltrombopag.

METHODS

A total of 86 children with chronic ITP between 1978-2014 were included. Demographic findings, laboratory results, clinical signs, bleeding scores, response time and time of complete remission were recorded.

RESULTS

The male/female ratio was 1.09, and median follow-up time was 3 years (range: 1.5-17 years). The median age at diagnosis of chronic ITP was 7 years (range: 2-17), and the median initial platelet count was 10 × 10/L (range: 1-66 × 10/L). Petechiae/ecchymoses were the most common clinical sign (86%) and followed by mucosal bleeding (39.5%). Severe bleeding was seen in 5% of the patients. None of them had intracranial hemorrhage. Twenty patients underwent splenectomy, and the rate of complete remission was 70%. Spontaneous complete remission was seen in 29% of the patients, and the median time to spontaneous complete remission was 3 years.

CONCLUSIONS

Our study showed that almost one-third of patients with chronic ITP experienced spontaneous complete remission in an average of 3 years, and splenectomy provided satisfactory results in severe cases. This study demonstrates the natural history of chronic ITP in childhood before the era of eltrombopag.

摘要

背景

关于儿童慢性免疫性血小板减少症(ITP)的自然病程,相关信息有限。我们旨在评估在艾曲泊帕上市之前这一时期儿童慢性ITP的临床和人口统计学特征。

方法

纳入了1978年至2014年间共86例儿童慢性ITP患者。记录人口统计学结果、实验室检查结果、临床体征、出血评分、缓解时间和完全缓解时间。

结果

男女比例为1.09,中位随访时间为3年(范围:1.5至17年)。慢性ITP诊断时的中位年龄为7岁(范围:2至17岁),初始血小板计数中位数为10×10⁹/L(范围:1至66×10⁹/L)。瘀点/瘀斑是最常见的临床体征(86%),其次是黏膜出血(39.5%)。5%的患者出现严重出血。他们均无颅内出血。20例患者接受了脾切除术,完全缓解率为70%。29%的患者出现自发完全缓解,自发完全缓解的中位时间为3年。

结论

我们的研究表明,近三分之一的慢性ITP患者平均在3年内出现自发完全缓解,脾切除术在重症病例中取得了满意的效果。本研究展示了艾曲泊帕时代之前儿童慢性ITP的自然病程。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fe92/11430342/548ce19bca91/children-11-01051-g001.jpg

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