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再生障碍性贫血:哥斯达黎加的人口统计学和临床特征

Aplastic Anemia: Demographic and Clinical Characteristics in Costa Rica.

作者信息

Rodríguez-Sevilla María, Valverde-Muñoz Kathia, García-Hernández Claudia, Sanabria-Castro Alfredo, Echeverri-McCandless Ann, Rojas-Chaves Sebastián

机构信息

Hematology Department, Hospital San Juan de Dios, Costa Rican Social Security Health Fund (CCSS), San José, CRI.

Hematology Department, Hospital Nacional de Niños, Costa Rican Social Security Health Fund (CCSS), San José, CRI.

出版信息

Cureus. 2024 Nov 10;16(11):e73403. doi: 10.7759/cureus.73403. eCollection 2024 Nov.

DOI:10.7759/cureus.73403
PMID:39664132
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11633852/
Abstract

Background Aplastic anemia (AA) is a rare and heterogeneous hematological disorder defined as pancytopenia with hypocellular bone marrow in the absence of abnormal infiltration or medullary fibrosis. Various causes of AA have been identified, such as autoimmune factors, bone marrow injuries, viral infections, and genetic disorders. The symptoms of AA are directly linked to pancytopenia and the most common are fatigue, recurrent infections, and bleeding problems. The treatment of AA varies according to the severity of the disease and includes immunosuppressive therapies and bone marrow transplantation. This study aims to identify the most relevant social, clinical, and demographic characteristics of patients with AA in Costa Rica. Methodology A retrospective, observational study was conducted in Costa Rica by reviewing the medical records of patients diagnosed with AA in the main hospitals of the Costa Rican Social Security Health Fund (CCSS, by its acronym in Spanish). A total of 109 patients who were evaluated between 2016 and 2018 were identified. Sociodemographic, clinical, and treatment information was collected for these patients in a database that was analyzed using statistical programs such as SPSS Statistics (version 24) and GraphPad Prism (version 8). Results Most patients were male (56%) with an average age of 32 years. Patients were classified according to the severity of the disease, and a higher mortality at 60 months was observed in those with very severe AA and in patients over 65 years old. The most commonly used first-line treatment was the combination of rabbit antithymocyte globulin (ATG) and cyclosporine (42.9%). Patients who required a greater number of blood transfusions had a more severe disease. Further, 46 patients requiring a second line of treatment were identified, and the most common treatment in this group was the combination of ATG with eltrombopag in 19.6% of the patients. The study results present the sociodemographic and clinical characteristics of patients with AA in Costa Rica. The lack of identification of a common external factor that may influence the development of the disease is highlighted. Treatment with rabbit ATG and cyclosporine demonstrated a good response in patients. The availability and cost of treatments are important considerations, especially in developing countries. Conclusions The study highlights significant progress in the understanding and treatment of AA in the Costa Rican context. The results support the efficacy of the combination of antibodies and cyclosporine as a therapeutic option. The importance of adapting treatments to the characteristics of the local population is emphasized, along with the need for further research to improve long-term outcomes.

摘要

背景

再生障碍性贫血(AA)是一种罕见的异质性血液系统疾病,定义为全血细胞减少且骨髓细胞减少,同时不存在异常浸润或骨髓纤维化。已确定AA的多种病因,如自身免疫因素、骨髓损伤、病毒感染和遗传疾病。AA的症状与全血细胞减少直接相关,最常见的症状是疲劳、反复感染和出血问题。AA的治疗根据疾病严重程度而异,包括免疫抑制治疗和骨髓移植。本研究旨在确定哥斯达黎加AA患者最相关的社会、临床和人口统计学特征。

方法

在哥斯达黎加进行了一项回顾性观察研究,通过查阅哥斯达黎加社会保障健康基金(CCSS,西班牙语首字母缩写)主要医院中被诊断为AA的患者的病历。共确定了2016年至2018年期间接受评估的109例患者。在一个数据库中收集了这些患者的社会人口统计学、临床和治疗信息,并使用SPSS Statistics(版本24)和GraphPad Prism(版本8)等统计程序进行分析。

结果

大多数患者为男性(56%),平均年龄为32岁。根据疾病严重程度对患者进行分类,发现极重度AA患者和65岁以上患者在60个月时死亡率较高。最常用的一线治疗是兔抗胸腺细胞球蛋白(ATG)和环孢素联合使用(42.9%)。需要更多输血的患者病情更严重。此外,确定了46例需要二线治疗的患者,该组中最常见的治疗方法是19.6%的患者使用ATG与艾曲泊帕联合治疗。研究结果呈现了哥斯达黎加AA患者的社会人口统计学和临床特征。强调了未发现可能影响疾病发展的共同外部因素。兔ATG和环孢素治疗对患者显示出良好反应。治疗的可及性和成本是重要考虑因素,尤其是在发展中国家。

结论

该研究突出了在哥斯达黎加背景下对AA的理解和治疗方面取得的重大进展。结果支持抗体与环孢素联合作为一种治疗选择的疗效。强调了使治疗适应当地人群特征的重要性,以及进一步研究以改善长期结局的必要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb5b/11633852/7a92247b056e/cureus-0016-00000073403-i03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb5b/11633852/d14d28b4c35c/cureus-0016-00000073403-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb5b/11633852/98a4397ed2f8/cureus-0016-00000073403-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb5b/11633852/7a92247b056e/cureus-0016-00000073403-i03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb5b/11633852/d14d28b4c35c/cureus-0016-00000073403-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb5b/11633852/98a4397ed2f8/cureus-0016-00000073403-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb5b/11633852/7a92247b056e/cureus-0016-00000073403-i03.jpg

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