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Cryopreservation for All Is No Option in Unrelated Stem Cell Transplantation. Comment on Dholaria B, et al. Securing the Graft During Pandemic: Are We Ready for Cryopreservation for All? Biol Blood Marrow Transplant. 2020;26:e145-e146. 冷冻保存在非亲属干细胞移植中是没有选择的。对Dholaria B等的评论。在大流行期间保护移植物:我们准备好为所有人冷冻保存了吗?中华血液学杂志,2020;26:845 - 846。
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.08.011
Alexander H. Schmidt , Deborah Buk , Alexander Platz , Marcel R.M. van den Brink
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引用次数: 12
Prognostic Factors for Postrelapse Survival after ex Vivo CD34+-Selected (T Cell-Depleted) Allogeneic Hematopoietic Cell Transplantation in Multiple Myeloma 多发性骨髓瘤患者体外CD34+选择(T细胞缺失)异体造血细胞移植术后复发后生存的预后因素
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.07.016
Alexandra Gomez-Arteaga , Gunjan L. Shah , Raymond E. Baser , Michael Scordo , Josel D. Ruiz , Adam Bryant , Parastoo B. Dahi , Arnab Ghosh , Oscar B. Lahoud , Heather J. Landau , Ola Landgren , Brian C. Shaffer , Eric L. Smith , Guenther Koehne , Miguel-Angel Perales , Sergio A. Giralt , David J. Chung

Allogeneic hematopoietic cell transplantation (alloHCT) for multiple myeloma (MM), with its underlying graft-versus-tumor capacity, is a potentially curative approach for high-risk patients. Relapse is the main cause of treatment failure, but predictors for postrelapse survival are not well characterized. We conducted a retrospective analysis to evaluate predictors for postrelapse overall survival (OS) in 60 MM patients who progressed after myeloablative T cell-depleted alloHCT. The median patient age was 56 years, and 82% had high-risk cytogenetics. Patients received a median of 4 lines of therapy pre-HCT, and 88% achieved at least a partial response (PR) before alloHCT. Of the 38% who received preemptive post-HCT therapy, 13 received donor lymphocyte infusions (DLIs) and 10 received other interventions. Relapse was defined as very early (<6 months; 28%), early (6 to 24 months; 50%), or late (>24 months; 22%). At relapse, 27% presented with extramedullary disease (EMD). The median postrelapse overall survival (OS) by time to relapse was 4 months for the very early relapse group, 17 months for the early relapse group, and 72 months for the late relapse group (P = .002). Older age, relapse with EMD, <PR before alloHCT, <PR by day +100, and no maintenance were prognostic for inferior postrelapse OS on univariate analysis. On multivariate analysis adjusted for age and sex, very early relapse (hazard ratio [HR], 4.37; 95% confidence interval [CI], 1.42 to 13.5), relapse with EMD (HR, 5.20; 95% CI, 2.10 to 12.9), and DLI for relapse prevention (HR, .11; 95% CI, 2.10 to 12.9) were significant predictors for postrelapse survival. Despite their shared inherent high-risk status, patients with MM have significantly disparate post-HCT relapse courses, with some demonstrating long-term survival despite relapse.

同种异体造血细胞移植(alloHCT)治疗多发性骨髓瘤(MM),具有潜在的移植物抗肿瘤能力,是一种潜在的治疗高危患者的方法。复发是治疗失败的主要原因,但复发后生存的预测因素并没有很好的特征。我们进行了一项回顾性分析,以评估60例骨髓清除T细胞耗尽同种异体hct后进展的MM患者复发后总生存率(OS)的预测因素。患者中位年龄为56岁,82%有高危细胞遗传学。患者在hct前接受了中位4线治疗,88%的患者在同种异体hct前至少获得了部分缓解(PR)。在接受hct后预防性治疗的38%患者中,13人接受供体淋巴细胞输注(DLIs), 10人接受其他干预。复发定义为非常早期(6个月;28%),早期(6至24个月;50%),或晚些时候(24个月;22%)。复发时,27%表现为髓外疾病(EMD)。早期复发组的中位复发后总生存期(OS)为4个月,早期复发组为17个月,晚期复发组为72个月(P = 0.002)。单因素分析显示,年龄较大、EMD复发、同种异体移植前的PR、第100天的PR、无维持是复发后OS较差的预后因素。在调整了年龄和性别的多变量分析中,非常早的复发(风险比[HR], 4.37;95%可信区间[CI], 1.42 ~ 13.5), EMD复发(HR, 5.20;95% CI, 2.10 - 12.9)和预防复发的DLI (HR, 0.11;95% CI(2.10 ~ 12.9)是复发后生存的重要预测因子。尽管具有共同的固有高风险状态,MM患者在hct后有明显不同的复发过程,其中一些患者在复发后表现出长期生存。
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引用次数: 1
FLAMSA-Based Reduced-Intensity Conditioning versus Myeloablative Conditioning in Younger Patients with Relapsed/Refractory Acute Myeloid Leukemia with Active Disease at the Time of Allogeneic Stem Cell Transplantation: An Analysis from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation 同种异体干细胞移植时复发/难治性急性髓系白血病活动性年轻患者基于flamsa的降低强度调节与清髓调节:来自欧洲血液和骨髓移植学会急性白血病工作组的分析
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.07.020
Eduardo Rodríguez-Arbolí , Myriam Labopin , Johanna Tischer , Arne Brecht , Arnold Ganser , Jürgen Finke , Igor Wolfgang Blau , Nicolaus Kröger , Peter Kalhs , Edouard Forcade , Donald Bunjes , Alexandros Spyridonidis , Bipin Savani , Arnon Nagler , Mohamad Mohty

The use of myeloablative conditioning (MAC) in the setting of active relapsed/refractory (R/R) acute myeloid leukemia (AML) has been hindered by high historical rates of nonrelapse mortality (NRM). FLAMSA (fludarabine, Ara-C, and amsacrine) chemotherapy (CT) followed by reduced-intensity conditioning (RIC) has been proposed as an effective and potentially safer alternative in this scenario. As improvements in supportive care have contributed to decreasing NRM rates after MAC, a comparative reassessment of these two strategies was performed. This was a registry-based analysis by the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Eligibility criteria included age 18 to 50 years, primary refractory, first or second relapsed active AML, first allogeneic stem cell transplantation from a matched sibling donor (MSD) or an unrelated donor (UD) performed between 2005 and 2018, MAC or FLAMSA-RIC. A total of 1018 patients were included. The median patient age was 39 years (range, 18 to 50). Two hundred and fifty-eight patients received busulfan (Bu)/cyclophosphamide (Cy), 314 received Cy/total body irradiation (TBI), 318 received FLAMSA-TBI, and 128 received FLAMSA-CT. The median duration of follow-up was 50 months. In univariate analysis, the 2-year relapse incidence (RI) (54%; 95% confidence interval (CI), 50%-57%), leukemia-free survival (LFS) (30%; 95% CI, 27%-33%), and refined graft-versus-host disease-free, relapse-free survival (GRFS) (21%; 95% CI, 18%-24%) were not significantly different between cohorts. Lower 2-year NRM was observed in the FLAMSA-CT group (7% versus 16% in Bu/Cy, 19% in Cy/TBI, and 18% in FLAMSA-TBI; P = .04), as well as increased 2-year overall survival (OS) (50% versus 33% in Bu/Cy, 34% in Cy/TBI, and 36% in FLAMSA-TBI; P = .03). These results were maintained in the multivariate analysis (hazard ratio [HR] for NRM: .40, P = .01; HR for OS: .65, P = .01; Bu/Cy as reference). These data suggest that FLAMSA-CT may be a preferred conditioning regimen in patients with active R/R AML due to lower NRM. Yet, the high relapse rates observed in our analyses emphasize the need for novel therapeutic strategies in this clinical setting.

在活动性复发/难治性(R/R)急性髓性白血病(AML)中使用清髓调节(MAC)一直受到历史上高非复发死亡率(NRM)的阻碍。在这种情况下,FLAMSA(氟达拉滨,Ara-C和amsacrine)化疗(CT)随后进行降低强度调节(RIC)被认为是一种有效且可能更安全的替代方案。由于支持治疗的改善有助于降低MAC后的NRM率,因此对这两种策略进行了比较重新评估。这是由欧洲血液和骨髓移植学会急性白血病工作组进行的一项基于登记的分析。入选标准包括年龄18 - 50岁、原发性难治、首次或第二次复发的活动性AML、2005年至2018年间首次从匹配的兄弟姐妹供体(MSD)或非亲属供体(UD)进行同种异体干细胞移植、MAC或FLAMSA-RIC。共纳入1018例患者。患者年龄中位数为39岁(18 - 50岁)。258例患者接受了布硫丹(Bu)/环磷酰胺(Cy)治疗,314例接受了Cy/全身照射(TBI), 318例接受了FLAMSA-TBI治疗,128例接受了FLAMSA-CT治疗。中位随访时间为50个月。在单因素分析中,2年复发率(RI) (54%;95%置信区间(CI), 50%-57%),无白血病生存率(LFS) (30%;95% CI, 27%-33%),精致移植物抗宿主病无复发生存率(GRFS) (21%;95% CI, 18%-24%)组间无显著差异。FLAMSA-CT组2年NRM较低(7%,Bu/Cy为16%,Cy/TBI为19%,FLAMSA-TBI为18%);P = .04),以及2年总生存率(OS)的提高(50% vs . Bu/Cy 33%, Cy/TBI 34%, FLAMSA-TBI 36%;p = .03)。这些结果在多变量分析中保持不变(NRM的风险比[HR]: 0.40, P = 0.01;OS HR: 0.65, P = 0.01;但/Cy作为参考)。这些数据表明,由于NRM较低,FLAMSA-CT可能是活动性R/R AML患者的首选调节方案。然而,在我们的分析中观察到的高复发率强调了在这种临床环境中需要新的治疗策略。
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引用次数: 11
Identification of Neurotoxicity after Chimeric Antigen Receptor (CAR) T Cell Infusion without Deterioration in the Immune Effector Cell-Associated Encephalopathy (ICE) Score 嵌合抗原受体(CAR) T细胞输注后无免疫效应细胞相关脑病(ICE)评分恶化的神经毒性鉴定
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.07.031
Megan M. Herr, George L. Chen, Maureen Ross, Hillary Jacobson, Renee McKenzie, Laura Markel, Sophia R. Balderman, Christine M. Ho, Theresa Hahn, Philip L. McCarthy

A consensus grading schema for cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) resulting from chimeric antigen receptor (CAR) T cell therapy was published in 2019. Although this consensus grading schema has been imperative in identifying and monitoring CRS and ICANS in our CAR T cell population, we observed patients exhibiting subtle neurotoxicity symptoms prior to a decrease in their immune effector cell-associated encephalopathy (ICE) score, which is one component of ICANS. Because we treat grade 1 ICANS at our institution, identification of early neurotoxicity symptoms is important. Additionally, we found changes in personality, occupational confusion, or inability to answer dichotomous questions were early signs of neurotoxicity. Therefore, we developed a 3-step command tool to supplement the ICE evaluation. We present 2 examples of patients who exhibited early neurotoxicity symptoms and led us to develop this tool and 1 in whom it was effective. We propose that CAR T cell patients are consistently followed by a clinical care provider who is familiar with the patient to recognize early changes in personality, behavior, and cognition. Additionally, we propose that the multistep command tool be used in conjunction with the ICE score to detect early symptoms of ICANS. Early intervention has the potential to prevent irreversible neurotoxicity.

嵌合抗原受体(CAR) T细胞治疗导致的细胞因子释放综合征(CRS)和免疫效应细胞相关神经毒性综合征(ICANS)的共识分级方案于2019年发表。尽管在我们的CAR - T细胞群中识别和监测CRS和ICANS时,这种共识分级方案是必不可少的,但我们观察到患者在其免疫效应细胞相关脑病(ICE)评分下降之前表现出微妙的神经毒性症状,ICE评分是ICANS的一个组成部分。因为我们在我们的机构治疗1级ICANS,早期神经毒性症状的识别是重要的。此外,我们发现性格变化、职业混乱或无法回答二分性问题是神经毒性的早期迹象。因此,我们开发了一个三步命令工具来补充ICE评估。我们介绍了2例表现出早期神经毒性症状的患者,并引导我们开发了这种工具,其中1例有效。我们建议由熟悉患者的临床护理人员对CAR - T细胞患者进行持续随访,以识别患者人格、行为和认知的早期变化。此外,我们建议将多步骤命令工具与ICE评分结合使用,以检测ICANS的早期症状。早期干预有可能预防不可逆的神经毒性。
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引用次数: 10
ASTCT Notes ASTCT笔记
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.10.012
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引用次数: 0
Combination of FLT3-ITD Allelic Ratio, NPM1 Mutation, and Immunophenotypic Markers to Modulate Outcome Prediction in Patients with Normal Karyotype Acute Myelogenous Leukemia Undergoing Hematopoietic Stem Cell Transplantation FLT3-ITD等位基因比例、NPM1突变和免疫表型标记联合调节正常核型急性髓性白血病患者接受造血干细胞移植的预后预测
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.07.017
Gina Jiang , Jose-Mario Capo-Chichi , Aijun Liu , Eshetu G. Atenafu , Rajat Kumar , Mark D. Minden , Hong Chang

NPM1 mutation status and the allelic ratio (AR) of FLT3-internal tandem duplication (FLT3-ITD) are routinely tested for disease risk stratification in patients with normal karyotype (NK) acute myelogenous leukemia (AML); however, the predictive impact of immunophenotypic markers on different NPM1/FLT3 genotypes remains unclear. We performed a retrospective analysis of 423 patients with NK-AML subclassified into groups based on NPM1/FLT3 genotype. Allogeneic hematopoietic stem cell transplantation (HSCT) was performed in 124 of 423 patients (29%) and was significantly associated with longer event-free survival (EFS) and overall survival (OS), except for patients with the favorable genotype, defined as mutated NPM1 (NPM1mut) combined with normal FLT3 status (FLT3-ITDneg) or FLT3-ITD AR <.5 (FLT3-ITDlow). A subset of AML patients bearing the favorable NPM1mut/FLT3-ITDneg/low genotype share similar outcomes with AML patients who have the intermediate FLT3/NPM1 genotype defined by normal NPM1 (NPM1wt) and FLT3-ITDneg/low. In these individuals, the lack of CD13 expression (CD13neg) was associated with shorter EFS (P = .041) and OS (P = .017). CD13neg was an independent predictor for shorter OS (hazard ratio, 1.985; P = .028).

在正常核型(NK)急性髓性白血病(AML)患者中,常规检测NPM1突变状态和flt3 -内串联重复(FLT3-ITD)等位基因比率(AR)用于疾病风险分层;然而,免疫表型标记物对不同NPM1/FLT3基因型的预测作用尚不清楚。我们根据NPM1/FLT3基因型对423例NK-AML患者进行了回顾性分析。423例患者中有124例(29%)接受了同种异体造血干细胞移植(HSCT),除了具有有利基因型的患者(定义为突变的NPM1 (NPM1mut)合并正常FLT3状态(FLT3- itdneg)或FLT3- itd AR)外,HSCT与更长的无事件生存期(EFS)和总生存期(OS)显著相关(FLT3-ITDlow)。具有有利的NPM1mut/FLT3- itdneg /low基因型的AML患者子集与具有由正常NPM1 (NPM1wt)和FLT3- itdneg /low定义的中间FLT3/NPM1基因型的AML患者具有相似的结果。在这些个体中,缺乏CD13表达(CD13neg)与较短的EFS (P = 0.041)和OS (P = 0.017)相关。CD13neg是较短OS的独立预测因子(风险比,1.985;p = .028)。
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引用次数: 2
Development and Evaluation of a Whiteboard Video Series to Support the Education and Recruitment of Committed Unrelated Donors for Hematopoietic Stem Cell Transplantation 开发和评估白板视频系列,以支持教育和招募承诺的非亲属造血干细胞移植供者
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.07.008
Edward W. Li , Anna Lee , Maryam Vaseghi-Shanjani , Alexander Anagnostopoulos , Gabriele Jagelaviciute , Elena Kum , Tanya Petraszko , Heidi Elmoazzen , David Allan , Warren Fingrut

Whiteboard videos are a popular video format, allowing viewers to see drawings of concepts alongside explanatory text and speech. We hypothesized that whiteboard videos could support the education and recruitment of unrelated stem cell donors in Canada. A series of 5 sharable whiteboard videos about stem cell donation was produced and posted online in September 2018, including 1 full-length video (https://youtu.be/V4fVBtxnWfM) and 4 shorter videos titled “What Is Stem Cell Transplantation?” “How Does the Matching Process Work?” “How Are Stem Cells Donated?” and “How Can I Register as a Stem Cell Donor?” In the videos, metaphorical interpretations of stem cells as factories and genetic markers as barcode labels are employed to communicate complex concepts. The particular need for young, male, and ethnically diverse donors is reflected in the characters portrayed. Surveys demonstrated the videos (1) were used and valued by stakeholders in donor recruitment and (2) significantly improved objective and self-reported knowledge about stem cell donation and reduced donation-related ambivalence among viewers from the most-needed donor demographics. Use of the whiteboard videos was also associated with improved donor recruitment outcomes in Canada. Our work is relevant to donor registries and recruitment organizations worldwide that seek to improve their recruitment efforts.

白板视频是一种流行的视频格式,观众可以看到概念图以及解释性文本和演讲。我们假设白板视频可以支持加拿大非亲属干细胞供体的教育和招募。2018年9月,我们制作并发布了5个关于干细胞捐赠的白板视频,包括1个完整视频(https://youtu.be/V4fVBtxnWfM)和4个题为“什么是干细胞移植?”“匹配过程是如何工作的?”“干细胞是如何捐献的?”和“如何登记成为干细胞捐献者?”在视频中,干细胞作为工厂和基因标记作为条形码标签的隐喻性解释被用来传达复杂的概念。对年轻、男性和不同种族的捐赠者的特别需求反映在所描绘的人物身上。调查显示,这些视频(1)在捐赠者招募中被利益相关者使用和重视,(2)显著提高了关于干细胞捐赠的客观和自我报告的知识,并减少了来自最需要的捐赠者人口统计数据的观众中与捐赠相关的矛盾心理。在加拿大,白板视频的使用也与捐助者招募结果的改善有关。我们的工作与世界各地寻求改进其招聘工作的捐助者登记和招聘组织有关。
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引用次数: 8
Outcomes and Challenges of Reproductive Health in Hematopoietic Stem Cell Transplantation Survivors 造血干细胞移植幸存者生殖健康的结局和挑战
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.07.007
Christianne Bourlon , Santiago Riviello-Goya , Aldo A Acosta-Medina , Rosa E. Caballero-Landinez , Angelica Manrique-Rubio , Kevin Teran-De-la-Sancha , Alfonso Gulias-Herrero , Maria T. Bourlon

Long-term therapy-related reproductive health side effects impact the quality of life of hematopoietic stem cell transplantation (HSCT) survivors. In this study, we evaluated the prevalence of gonadal dysfunction (GD) pre- and post-HSCT, analyzed factors associated with GD, and explored rates of fertility assessment (FA) and fertility preservation (FP) in a resource-limited setting. FA and outcomes of patients age ≤45 years undergoing HSCT between June 2000 and May 2018 were collected retrospectively. We included 213 patients with a median age of 26 years. Pre-HSCT FA was performed in 71.8%, with a GD rate of 17%. The rate of GD was not different between the sexes (females, 19.5% versus males, 16.1%; P = .616) and was only associated with increasing age. The rate of cryopreservation in the cohort was 3.3%. Almost one-half (47.7%) of post-HSCT patients completed FA and evidenced an increase in GD rate to 48.9%. Comparing pre-HSCT and post-HSCT GD rates, women had a significant increase (19.5% versus 81.4%; P < .001), whereas men did not (16.1% versus 20.4%; P = .76). These results were confirmed by a multiple imputation analysis accounting for missing data. Female sex, pre-HSCT cytotoxic therapy, myeloablative conditioning, and germ cell tumor (GCT) diagnosis were associated with post-HSCT GD. Reproductive health preservation can be positively impacted when FA and FP are prioritized at the initial diagnosis in HSCT candidates, particularly in women of older age and men with a diagnosis of GCT. The low FP success observed urges implementation of strategies that favor accessibility and improve quality of life of HSCT survivors in low- and middle-income countries.

长期治疗相关的生殖健康副作用影响造血干细胞移植(HSCT)幸存者的生活质量。在这项研究中,我们评估了hsct前后性腺功能障碍(GD)的患病率,分析了与GD相关的因素,并在资源有限的情况下探讨了生育能力评估(FA)和生育能力保存(FP)的比率。回顾性收集2000年6月至2018年5月期间年龄≤45岁接受HSCT的患者FA和结果。我们纳入213例患者,中位年龄为26岁。hsct前FA的发生率为71.8%,GD率为17%。性别间GD比率无差异(女性为19.5%,男性为16.1%;P = .616),且仅与年龄增长有关。该队列的冷冻保存率为3.3%。几乎一半(47.7%)的hsct后患者完成了FA, GD率增加到48.9%。比较hsct前和hsct后的GD率,女性显著增加(19.5% vs 81.4%;P & lt;.001),而男性没有(16.1%对20.4%;p = .76)。这些结果通过考虑缺失数据的多重输入分析得到证实。女性、hsct前的细胞毒性治疗、清骨髓调节和生殖细胞肿瘤(GCT)诊断与hsct后GD相关。当在HSCT候选者的初始诊断中优先考虑FA和FP时,特别是在老年女性和诊断为GCT的男性中,生殖健康保护可能会受到积极影响。观察到的低生育成功率促使实施有利于低收入和中等收入国家HSCT幸存者可及性和改善生活质量的战略。
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引用次数: 0
Guidelines for COVID-19 Management in Hematopoietic Cell Transplantation and Cellular Therapy Recipients 造血细胞移植和细胞治疗受者COVID-19管理指南
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.07.027
Alpana Waghmare , Maheen Z. Abidi , Michael Boeckh , Roy F. Chemaly , Sanjeet Dadwal , Zeinab El Boghdadly , Mini Kamboj , Genovefa A. Papanicolaou , Steven A. Pergam , Zainab Shahid

There are currently limited data on the epidemiology, clinical manifestations, and optimal management of Coronavirus Disease 2019 (COVID-19) in hematopoietic cell transplantation and cellular therapy recipients. Given the experience with other respiratory viruses, we anticipate that patients may develop severe clinical disease and thus provide the following general principles for cancer centers across the nation. These guidelines were developed by members of the American Society for Transplantation and Cellular Therapy Infectious Diseases Special Interest Group. Specific practices may vary depending on local epidemiology and testing capacity, and the guidance provided in this document may change as new information becomes available.

目前,关于造血细胞移植和细胞治疗受者2019冠状病毒病(COVID-19)的流行病学、临床表现和最佳管理的数据有限。鉴于其他呼吸道病毒的经验,我们预计患者可能会出现严重的临床疾病,因此为全国的癌症中心提供以下一般原则。这些指南是由美国移植和细胞治疗学会传染病特别兴趣小组的成员制定的。具体做法可能因当地流行病学和检测能力而异,本文件提供的指导可能会随着获得新信息而改变。
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引用次数: 62
Effect of Ruxolitinib on Lung Function after Allogeneic Stem Cell Transplantation 鲁索利替尼对同种异体干细胞移植后肺功能的影响
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.07.033
Louise Bondeelle , Sylvie Chevret , Charlotte Hurabielle , Laila Samy , Tiphaine Goletto , Adrien Costantini , Flore Sicre de Fontbrune , David Michonneau , Gérard Socié , Abdellatif Tazi , Jean-David Bouaziz , Anne Bergeron

Ruxolitinib, a selective Janus kinase (JAK)1/2 inhibitor, has recently been proposed for steroid-refractory chronic graft-versus host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation (HSCT), particularly in severe skin cGVHD. Lung function impairment is common in severe skin cGVHD through concomitant bronchiolitis obliterans syndrome (BOS) or restrictive lung disease (RLD) from skin sclerosis. To date, no treatment has shown a benefit on lung function in this context. We retrospectively assessed the effect of ruxolitinib on lung function in a cohort of 70 patients diagnosed with sclerotic-type skin cGVHD between March 2015 and April 2018. Among these patients, 36 received ruxolitinib. To handle confounding by indication bias, exposure groups were matched on the propensity score to receive ruxolitinib, incorporating age, myeloablative conditioning, total body irradiation, BOS, forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and tobacco use at the time of cohort entry, as well as the time from transplantation. The 1:1 matching used a greedy-matching algorithm with replacement, with a caliper of 0.10. FVC and FEV1 trajectories during follow-up were compared in the matched samples, using linear mixed-effects models. The median duration of follow-up of the 46 matched patients was 58 months (interquartile range, 32 to 84 months). Ten patients had an RLD (6 exposed, 4 unexposed), and 13 patients were diagnosed with BOS (8 exposed, 5 unexposed). FEV1 decreased significantly over time independent of exposure to ruxolitinib (P < .0001). The FEV1 trajectory was similar in the exposed patients and the unexposed patients (P = .11). In conclusion, ruxolitinib administration did not demonstrate any improvement in the course of respiratory function in allogeneic HSCT recipients with sclerotic-type skin cGVHD.

Ruxolitinib是一种选择性Janus激酶(JAK)1/2抑制剂,最近被提议用于异体造血干细胞移植(HSCT)后类固醇难治性慢性移植物抗宿主病(cGVHD),特别是严重的皮肤cGVHD。严重皮肤cGVHD伴发闭塞性细支气管炎综合征(BOS)或皮肤硬化症引起的限制性肺疾病(RLD),常见肺功能损害。迄今为止,在这种情况下,没有任何治疗显示对肺功能有益。我们回顾性评估了ruxolitinib对2015年3月至2018年4月期间诊断为硬化型皮肤cGVHD的70例患者肺功能的影响。其中36例患者接受鲁索利替尼治疗。为了处理指征偏倚带来的混淆,暴露组接受鲁索利替尼的倾向评分进行匹配,包括年龄、清骨髓调节、全身照射、BOS、1秒用力呼气量(FEV1)、用力肺活量(FVC)、队列进入时的烟草使用情况以及移植后的时间。1:1匹配使用带有替换的贪婪匹配算法,卡尺为0.10。使用线性混合效应模型比较匹配样本在随访期间的FVC和FEV1轨迹。46例匹配患者的中位随访时间为58个月(四分位数范围为32至84个月)。10例患者发生RLD(6例暴露,4例未暴露),13例患者诊断为BOS(8例暴露,5例未暴露)。FEV1随时间显著下降,与鲁索利替尼暴露无关(P <。)。暴露组与未暴露组的FEV1轨迹相似(P = .11)。总之,ruxolitinib给药并没有显示出对患有硬化型皮肤cGVHD的异体造血干细胞受体呼吸功能的任何改善。
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引用次数: 5
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Biology of Blood and Marrow Transplantation
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