全球急性髓系白血病造血干细胞移植应用情况分析
An Analysis of the Worldwide Utilization of Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia.
作者信息
Tokaz Molly C, Baldomero Helen, Cowan Andrew J, Saber Wael, Greinix Hildegard, Koh Mickey B C, Kröger Nicolaus, Mohty Mohamad, Galeano Sebastian, Okamoto Shinichiro, Chaudhri Naeem, Karduss Amado J, Ciceri Fabio, Colturato Vergílio Antonio R, Corbacioglu Selim, Elhaddad Alaa, Force Lisa M, Frutos Cristóbal, León Andrés Gómez-De, Hamad Nada, Hamerschlak Nelson, He Naya, Ho Aloysius, Huang Xiao-Jun, Jacobs Ben, Kim Hee-Je, Iida Minako, Lehmann Leslie, de Latour Regis Peffault, Percival Mary-Elizabeth M, Perdomo Martina, Rasheed Walid, Schultz Kirk R, Seber Adriana, Ko Bor-Sheng, Simione Anderson João, Srivastava Alok, Szer Jeff, Wood William A, Kodera Yoshihisa, Nagler Arnon, Snowden John A, Weisdorf Daniel, Passweg Jakob, Pasquini Marcelo C, Sureda Anna, Atsuta Yoshiko, Aljurf Mahmoud, Niederwieser Dietger
机构信息
Division of Medical Oncology, University of Washington, Seattle, Washington; Division of Hematology, University of Washington, Seattle, Washington; Clinical Research Division, Fred Hutchinson Cancer Center, Seattle, Washington.
University Hospital Basel, Center for International Blood and Marrow Transplant Research, Switzerland.
出版信息
Transplant Cell Ther. 2023 Apr;29(4):279.e1-279.e10. doi: 10.1016/j.jtct.2022.12.013. Epub 2022 Dec 23.
Acute myeloid leukemia (AML) has an aggressive course and a historically dismal prognosis. For many patients, hematopoietic stem cell transplantation (HSCT) represents the best option for cure, but access, utilization, and health inequities on a global scale remain poorly elucidated. We wanted to describe patterns of global HSCT use in AML for a better understanding of global access, practices, and unmet needs internationally. Estimates of AML incident cases in 2016 were obtained from the Global Burden of Disease 2019 study. HSCT activities were collected from 2009 to 2016 by the Worldwide Network for Blood and Marrow Transplantation through its member organizations. The primary endpoint was global and regional use (number of HSCT) and utilization of HSCT (number of HSCT/number of incident cases) for AML. Secondary outcomes included trends from 2009 to 2016 in donor type, stem cell source, and remission status at time of HSCT. Global AML incidence has steadily increased, from 102,000 (95% uncertainty interval: 90,200-108,000) in 2009 to 118,000 (104,000-126,000) in 2016 (16.2%). Over the same period, a 54.9% increase from 9659 to 14,965 HSCT/yr was observed globally, driven by an increase in allogeneic (64.9%) with a reduction in autologous (-34.9%) HSCT. Although the highest numbers of HSCT continue to be performed in high-resource regions, the largest increases were seen in resource-constrained regions (94.6% in Africa/East Mediterranean Region [AFR/EMR]; 34.7% in America-Nord Region [AMR-N]). HSCT utilization was skewed toward high-resource regions (in 2016: AMR-N 18.4%, Europe [EUR] 17.9%, South-East Asia/Western Pacific Region [SEAR/WPR] 11.7%, America-South Region [AMR-S] 4.5%, and AFR/EMR 2.8%). For patients <70 years of age, this difference in utilization was widened; AMR-N had the highest allogeneic utilization rate, increasing from 2009 to 2016 (30.6% to 39.9%) with continued low utilization observed in AFR/EMR (1.7% to 2.9%) and AMR-S (3.5% to 5.4%). Across all regions, total HSCT for AML in first complete remission (CR1) increased (from 44.1% to 59.0%). Patterns of donor stem cell source from related versus unrelated donors varied widely by geographic region. SEAR/WPR had a 130.2% increase in related donors from 2009 to 2016, and >95% HSCT donors in AFR/EMR were related; in comparison, AMR-N and EUR have a predilection for unrelated HSCT. Globally, the allogeneic HSCT stem cell source was predominantly peripheral blood (69.7% of total HSCT in 2009 increased to 78.6% in 2016). Autologous HSCT decreased in all regions from 2009 to 2016 except in SEAR/WPR (18.9%). HSCT remains a central curative treatment modality in AML. Allogeneic HSCT for AML is rising globally, but there are marked variations in regional utilization and practices, including types of graft source. Resource-constrained regions have the largest growth in HSCT use, but utilization rates remain low, with a predilection for familial-related donor sources and are typically offered in CR1. Further studies are necessary to elucidate the reasons, including economic factors, to understand and address these health inequalities and improve discrepancies in use of HSCT as a potentially curative treatment globally.
急性髓系白血病(AML)病程进展迅速,历来预后不佳。对许多患者而言,造血干细胞移植(HSCT)是实现治愈的最佳选择,但全球范围内的可及性、利用率以及健康不平等问题仍未得到充分阐明。我们希望描述全球AML患者HSCT的使用模式,以便更好地了解全球范围内的可及性、实践情况以及尚未满足的需求。2016年AML发病病例数的估计值来自《2019年全球疾病负担》研究。2009年至2016年期间,全球血液和骨髓移植网络通过其成员组织收集了HSCT活动数据。主要终点是AML的全球及区域使用情况(HSCT数量)和HSCT利用率(HSCT数量/发病病例数)。次要结果包括2009年至2016年期间供体类型、干细胞来源以及HSCT时的缓解状态趋势。全球AML发病率稳步上升,从2009年的102,000例(95%不确定区间:90,200 - 108,000例)增至2016年的118,000例(104,000 - 126,000例)(增长16.2%)。同期,全球HSCT数量从每年9659例增至14,965例,增长了54.9%,这是由异基因HSCT增加(64.9%)以及自体HSCT减少(-34.9%)推动的。尽管高资源地区进行的HSCT数量仍然最多,但资源受限地区的增长幅度最大(非洲/东地中海地区[AFR/EMR]增长94.6%;美洲 - 北部地区[AMR - N]增长34.7%)。HSCT利用率偏向高资源地区(2016年:AMR - N为18.4%,欧洲[EUR]为17.9%,东南亚/西太平洋地区[SEAR/WPR]为11.7%,美洲 - 南部地区[AMR - S]为4.5%,AFR/EMR为2.8%)。对于70岁以下患者,这种利用率差异更为明显;AMR - N的异基因利用率最高,从2009年到2016年有所增加(从30.6%增至39.9%),而AFR/EMR(从1.7%增至2.9%)和AMR - S(从3.5%增至5.4%)的利用率持续较低。在所有地区,首次完全缓解(CR1)时AML的HSCT总数均有所增加(从44.1%增至59.0%)。相关供体与无关供体的供体干细胞来源模式因地理区域而异。2009年至