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发展中国家慢性髓性白血病的造血细胞移植:酪氨酸激酶抑制剂时代后拉丁美洲的观点

Hematopoietic cell transplantation for chronic myeloid leukemia in developing countries: perspectives from Latin America in the post-tyrosine kinase inhibitor era.

作者信息

Pasquini Marcelo C

机构信息

Center for International Blood and Marrow Transplantation (CIBMTR), Medical College of Wisconsin, 9200 W. Wisconsin Ave, CCC5500, Milwaukee, WI 53226, USA.

出版信息

Hematology. 2012 Apr;17 Suppl 1:S79-82. doi: 10.1179/102453312X13336169155853.

Abstract

Tyrosine kinase inhibitors (TKIs) are currently the first line treatment for chronic myelogenous leukemia (CML) in countries with high and intermediate-high gross national income. Hematopoietic cell transplantation (HCT) in these countries is considered salvage therapy for eligible patients who failed TKI or progress to advanced disease stages. In Latin America, treatment for CML also changed with availability of TKI in the region. However, many challenges remain, as the cost of this class of medication and recommended monitoring is high. CML treatment practices in Latin America demonstrate that the majority of patients are treated with TKI at some point after diagnosis, most commonly imatinib mesylate, but still TKI can only be used after interferon failure in some countries. Other treatment practices are different from established international guidelines, outlying the importance of continuing medical education. Allogeneic HCT is a treatment option for CML in this region and could be considered a cost-effective approach in a small subset of young patients with available donors, as the overall cost of long-term non-transplant treatment may surpass the cost of transplantation. However, there are many challenges with HCT in Latin America such as access to experienced transplant centers, donor availability, and cost of essential drugs used after transplant, which further impacts expansion of this treatment approach in patients in need. In conclusion, Latin American patients with CML have access to state of the art CML treatment. Yet, drug costs have a tremendous impact on developing health systems. Optimization of CML treatment in the region with appropriate monitoring, recognizing patients who would be transplant candidates, and expanding access to transplantation for eligible patients may curtail these costs and further improve patient care.

摘要

在国民总收入较高和中高收入的国家,酪氨酸激酶抑制剂(TKIs)目前是慢性粒细胞白血病(CML)的一线治疗方法。在这些国家,造血细胞移植(HCT)被认为是TKI治疗失败或进展到晚期疾病阶段的合格患者的挽救疗法。在拉丁美洲,随着该地区TKI药物的可及性,CML的治疗也发生了变化。然而,许多挑战依然存在,因为这类药物的成本以及推荐的监测费用很高。拉丁美洲的CML治疗实践表明,大多数患者在诊断后的某个时间点接受TKI治疗,最常用的是甲磺酸伊马替尼,但在一些国家,仍只有在干扰素治疗失败后才能使用TKI。其他治疗实践与既定的国际指南不同,这突出了继续医学教育的重要性。异基因HCT是该地区CML的一种治疗选择,对于一小部分有可用供体的年轻患者,可以认为是一种具有成本效益的方法,因为长期非移植治疗的总成本可能超过移植成本。然而,拉丁美洲的HCT存在许多挑战,如难以获得经验丰富的移植中心、供体的可及性以及移植后使用的基本药物成本,这进一步影响了这种治疗方法在有需要的患者中的推广。总之,拉丁美洲的CML患者能够获得最先进的CML治疗。然而,药物成本对发展中的卫生系统产生了巨大影响。通过适当的监测优化该地区的CML治疗,识别可能成为移植候选者的患者,并为符合条件的患者扩大移植机会,可能会降低这些成本并进一步改善患者护理。

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