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低收入国家慢性髓性白血病患者的晚期就诊:预后意义及对治疗结果的影响。

Late presentation of chronic myeloid leukaemia patients in a low-income country: the prognostic implications and impact on treatment outcome.

机构信息

Department of Haematology and Blood Transfusion, Obafemi Awolowo University, Teaching Hospitals Complex, Ile-Ife, Nigeria.

Department of Haematology and Immunology, Obafemi Awolowo University, Ile-Ife, Nigeria.

出版信息

BMC Res Notes. 2024 Sep 3;17(1):245. doi: 10.1186/s13104-024-06910-9.

DOI:10.1186/s13104-024-06910-9
PMID:39227850
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11373193/
Abstract

BACKGROUND

In Nigeria, since 2002, Imatinib mesylate (glivec) has been available freely to chronic myeloid leukaemia (CML) patients but only at a tertiary health care centre in the southwestern part of the country. Despite this, it is not readily accessible to many patients due to the distance and other challenges including low socioeconomic status and political problems, preventing timely access to specialist care. This study evaluated the effect of the baseline characteristics on the prognostic implication and treatment outcome of CML patients in Nigeria.

METHOD

This study retrospectively evaluated the baseline characteristics, clinical presentations and treatment outcomes of 889 CML patients over 18 years (2002-2020). Of these, 576 (65%) patients had complete information with up-to-date BCR::ABL1 records. These 576 patients were categorized based on their responses to Imatinib therapy into three groups viz.; Optimal response (OR) defined as BCR::ABL1 ratio of < 0.1% or major molecular remission (≥ 3-log reduction of BCR::ABL1 mRNA or BCR::ABL1 ratio of < 0.1% on the International Scale), Suboptimal response (SR) with BCR::ABL ratio of 0.1-1%, and Treatment failure (TF) when MMR has not been achieved at 12 months. The variables were analyzed using descriptive and inferential statistics and a p-value < 0.05 was considered statistically significant.

RESULTS

The result revealed a median age of 37 years at diagnosis with a male-to-female ratio of 1.5:1. The majority (96.8%) of the patients presented with one or more symptoms at diagnosis with a mean symptom duration of 12 ± 10.6 months. The mean Sokal and EUTOS scores were 1.3 ± 0.8 and 73.90 ± 49.09 respectively. About half of the patients presented with high-risk Sokal (49%) and EUTOS (47%) scores. Interestingly, both the Sokal (r = 0.733, p = 0.011) and EUTOS (r = 0.102, p = 0.003) scores correlated positively and significantly with the duration of symptoms at presentation. Based on response categorization, 40.3% had OR while 27.1% and 32.6% had SR and TF respectively.

CONCLUSION

This study observed a low optimal response rate of 40.3% and treatment failure rate of 32.6% in our CML cohort while on first-line Imatinib therapy. This treatment response is strongly attributable to the long duration of symptoms of 12 months or more and high Sokal and EUTOS scores at presentation. We advocate prompt and improved access to specialist care with optimization of tyrosine kinase inhibitor therapy in Nigeria.

摘要

背景

自 2002 年以来,尼日利亚一直免费向慢性髓性白血病(CML)患者提供甲磺酸伊马替尼(格列卫),但仅在该国西南部的一个三级保健中心提供。尽管如此,由于距离和其他挑战,包括社会经济地位低和政治问题,许多患者仍然难以获得这种药物,从而无法及时获得专家护理。本研究评估了基线特征对尼日利亚 CML 患者预后意义和治疗结果的影响。

方法

本研究回顾性评估了 889 名 18 岁以上(2002-2020 年)CML 患者的基线特征、临床表现和治疗结果。其中,576 名(65%)患者具有完整的信息和最新的 BCR::ABL1 记录。这些 576 名患者根据他们对伊马替尼治疗的反应分为三组:最佳反应(OR)定义为 BCR::ABL1 比值<0.1%或主要分子缓解(BCR::ABL1 mRNA 减少≥3 对数或 BCR::ABL1 比值<0.1%,国际标准),亚最佳反应(SR)为 BCR::ABL 比值为 0.1-1%,治疗失败(TF)为 12 个月时未达到主要分子缓解。使用描述性和推断性统计方法分析变量,p 值<0.05 被认为具有统计学意义。

结果

结果显示,中位诊断年龄为 37 岁,男女比例为 1.5:1。大多数(96.8%)患者在诊断时出现一种或多种症状,平均症状持续时间为 12±10.6 个月。平均 Sokal 和 EUTOS 评分分别为 1.3±0.8 和 73.90±49.09。约一半的患者表现出高风险的 Sokal(49%)和 EUTOS(47%)评分。有趣的是,Sokal(r=0.733,p=0.011)和 EUTOS(r=0.102,p=0.003)评分均与就诊时症状持续时间呈正相关且具有统计学意义。根据反应分类,40.3%有 OR,27.1%和 32.6%有 SR 和 TF。

结论

本研究观察到,在我们的 CML 队列中,在接受一线伊马替尼治疗时,OR 率仅为 40.3%,治疗失败率为 32.6%。这种治疗反应主要归因于 12 个月或更长时间的症状持续时间和高 Sokal 和 EUTOS 评分。我们主张在尼日利亚迅速改善获得专家护理的机会,并优化酪氨酸激酶抑制剂治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/60df/11373193/56912238b20d/13104_2024_6910_Fig3_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/60df/11373193/206690614e77/13104_2024_6910_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/60df/11373193/b3d82819b3db/13104_2024_6910_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/60df/11373193/56912238b20d/13104_2024_6910_Fig3_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/60df/11373193/206690614e77/13104_2024_6910_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/60df/11373193/b3d82819b3db/13104_2024_6910_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/60df/11373193/56912238b20d/13104_2024_6910_Fig3_HTML.jpg

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