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Novel Approach for Bone Marrow Transplantation Conditioning in Acute Myelogenous Leukemia not Responding to the Induction Therapy Using Etoposide Carried in Lipid Core Nanoparticles: A Pilot Clinical Study 脂质核纳米颗粒携带依托泊苷诱导治疗对急性髓性白血病无反应的骨髓移植调节新方法:一项试点临床研究
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.07.010
Sandra S. Rohr , Raul C. Maranhão , Thauany M. Tavoni , Aleksandra T. Morikawa , Kelsy Areco , Debora F. Deus , José S.R. Oliveira

Allogeneic hematopoietic cell transplantation (HCT) is the treatment of choice for acute myelogenous leukemia (AML) not responding to induction therapy. It is a therapeutic choice for the blast phase of chronic myelogenous leukemia (CML-BP) in patients failing to respond to tyrosine kinase inhibitors (TKIs). Lipid core nanoparticles (LDEs) concentrate severalfold more in blast cells than in corresponding normal cells. Incorporation of anticancer drugs to LDE formulations increases the pharmacologic action and decreases the toxicity. We tested a drug-targeting system, LDE-etoposide plus total body irradiation (TBI; 1200 cGy dose), in 13 patients with AML not responding to the induction therapy and in 2 patients with CML-BP refractory to second-generation TKIs. The mean patient age was 46.7 years (range, 22 to 66 years). The LDE-etoposide dose was escalated at 20, 30, 40, 50, and 60 mg/kg. No patients developed grade 4 or 5 toxicity; however, mucositis grade 3 occurred in 6 patients, 3 patients experienced diarrhea, and 1 patient had an elevated total bilirubin level. No deaths were related to conditioning. All patients were successfully engrafted. The median times to neutrophil and platelet engraftment were 20 ± 5 days and 16 ± 4 days, respectively. Five patients (33.4%) had acute graft-versus-host-disease (GVHD), including 4 grade I, and 1 with grade II, and 8 patients (57.1%) had moderate-to-severe chronic GVHD. This pilot study shows the potential of LDE-etoposide plus TBI as an HCT conditioning regimen in AML patients not responding to the induction and refractory therapies for CML-BP patient. These findings pave the way for subsequent larger clinical trials.

同种异体造血细胞移植(HCT)是治疗急性髓性白血病(AML)不响应诱导治疗的选择。对于对酪氨酸激酶抑制剂(TKIs)无效的慢性髓性白血病(CML-BP)患者,它是一种治疗选择。脂质核纳米颗粒(LDEs)在胚细胞中的浓度是正常细胞的数倍。将抗癌药物掺入LDE制剂可增加药理学作用并降低毒性。我们测试了一种药物靶向系统,lde -依托泊苷加全身照射(TBI;1200 cGy剂量),13例对诱导治疗无反应的AML患者和2例对第二代TKIs难治的CML-BP患者。患者平均年龄46.7岁(22 ~ 66岁)。lde -依托波苷剂量逐步增加至20,30,40,50和60mg /kg。没有患者出现4级或5级毒性;然而,6例患者发生3级黏膜炎,3例患者出现腹泻,1例患者总胆红素升高。没有死亡与条件反射有关。所有患者均成功移植。移植中性粒细胞和血小板的中位时间分别为20±5天和16±4天。5例(33.4%)患者有急性移植物抗宿主病(GVHD),其中4例为I级,1例为II级,8例(57.1%)患者有中重度慢性GVHD。这项初步研究表明,lde -依托泊苷加TBI治疗对CML-BP患者的诱导和难治性治疗无反应的AML患者的HCT调节方案具有潜力。这些发现为后续更大规模的临床试验铺平了道路。
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引用次数: 3
Comparison of Myeloablative (CyTBI, BuCy) versus Reduced-Intensity (FluBu2TBI400) Peripheral Blood Stem Cell Transplantation in Acute Myeloid Leukemia Patients with Pretransplant Low WT1 Expression 骨髓清除(CyTBI, BuCy)与降低强度(FluBu2TBI400)外周血干细胞移植在移植前低WT1表达急性髓系白血病患者中的比较
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.07.006
Silvia Park , Gi June Min , Sung Soo Park , Seung-Ah Yahng , Young-Woo Jeon , Seung-Hwan Shin , Jae-Ho Yoon , Sung-Eun Lee , Byung Sik Cho , Ki-Seong Eom , Yoo-Jin Kim , Seok Lee , Chang-Ki Min , Seok-Goo Cho , Dong-Wook Kim , Jong Wook Lee , Hee-Je Kim

Relapse is a major concern with reduced-intensity conditioning. We analyzed 257 patients with acute myeloid leukemia (AML) who received allogeneic stem cell transplantation (SCT) and fulfilled the following criteria: intermediate- or poor-risk disease by National Comprehensive Cancer Network guidelines (2017, version 3), in first complete remission (CR1) at SCT, received either myeloablative conditioning (MAC; busulfan plus cyclophosphamide or cyclophosphamide plus total body irradiation) or reduced-intensity conditioning (RIC; FluBu2TBI400) peripheral blood SCT from 8/8 matched sibling or unrelated donor, and having bone marrow Wilms tumor gene 1 (WT1) expression results before transplant. We and other groups serially published a predictive value for pretransplant WT1 expression in patients with AML to identify patients at higher risk of relapse. Among the total 257 patients, 191 (74.3%) and 66 (25.7%) patients received MAC and RIC transplants, respectively. WT1 ≥250 copies/104 ABL was defined as WT1high. WT1high before SCT was found to be an independent prognostic factor for inferior overall survival (OS), disease-free survival (DFS), and higher cumulative incidence of relapse (CIR). There were 201 patients with WT1 low expression based upon pretransplant analysis. There was no significant difference in OS, DFS, CIR, and nonrelapse mortality between MAC and RIC patients. To conclude, post-transplant survival or relapse was not different by conditioning intensity in AML CR1 patients whose WT1 level was below 250 copies per 104 ABL at transplantation.

复发是低强度条件反射的主要问题。我们分析了257例接受同种异体干细胞移植(SCT)的急性髓性白血病(AML)患者,他们符合以下标准:国家综合癌症网络指南(2017年,第3版)的中度或低风险疾病,在SCT时首次完全缓解(CR1),接受骨髓清除调节(MAC;磺胺加环磷酰胺或环磷酰胺加全身照射)或降低强度调理(RIC;来自8/8匹配的兄弟姐妹或非亲属供者的外周血SCT,并在移植前有骨髓Wilms肿瘤基因1 (WT1)表达结果。我们和其他研究小组连续发表了移植前WT1表达在AML患者中的预测价值,以识别复发风险较高的患者。在257例患者中,分别有191例(74.3%)和66例(25.7%)患者接受了MAC和RIC移植。WT1≥250拷贝/104 ABL定义为WT1高。发现SCT前wt1高是较差的总生存期(OS)、无病生存期(DFS)和较高的累积复发发生率(CIR)的独立预后因素。移植前分析WT1低表达201例。MAC和RIC患者的OS、DFS、CIR和非复发死亡率无显著差异。综上所述,对于移植时WT1水平低于250拷贝/ 104 ABL的AML CR1患者,移植后生存或复发与调节强度无关。
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引用次数: 9
Challenging and Practical Aspects of Nutrition in Chronic Graft-versus-Host Disease 慢性移植物抗宿主病营养的挑战和实践方面
IF 4.3 Q1 Medicine Pub Date : 2020-11-01 DOI: 10.1016/j.bbmt.2020.08.004
Andrea Z. Pereira , Sandra Elisa Adami Gonçalves , Morgani Rodrigues , Nelson Hamerschlak , Mary E. Flowers

There is a paucity of information about nutrition in chronic graft-versus-host disease (GVHD). The role of nutrition is important because malnutrition is strongly associated with severe chronic GVHD manifestations. There is a high prevalence of metabolic syndrome and osteoporosis in this setting. Here we review the literature, describe main aspects of nutrition and discuss macronutrients (ie, vitamins), micronutrients (ie, Mg, Zn, Ca, and K) and supplements (probiotics and omega 3 fatty acids). A search was carried out in March 2020 using PubMed. Databases were screened for searching terms in titles and abstracts referring to chronic GVHD, nutrition intervention, protein, and body composition. Data were extracted for the following outcomes: nutrition, nutrition intervention, chronic GVHD, nutrition deficiencies, diet, vitamin, dry eye, probiotic, protein, and body composition. In this report, we summarize interventional nutrition studies reported in oncology and metabolic syndrome settings and describe our nutritional clinical practice in hematopoietic cell transplantation and chronic GVHD. The impact of nutrition evaluation and intervention on muscle mass loss, dry eye, dysgeusia, metabolic syndrome, osteoporosis, and comorbidities associated with chronic GVHD need to be studied prospectively.

关于慢性移植物抗宿主病(GVHD)的营养信息缺乏。营养的作用很重要,因为营养不良与严重的慢性GVHD表现密切相关。在这种情况下,代谢综合征和骨质疏松症的患病率很高。在这里,我们回顾了文献,描述了营养的主要方面,并讨论了宏量营养素(即维生素),微量营养素(即镁,锌,钙和钾)和补充剂(益生菌和ω - 3脂肪酸)。2020年3月,在PubMed上进行了一次搜索。在数据库中筛选有关慢性GVHD、营养干预、蛋白质和身体成分的标题和摘要。提取了以下结果的数据:营养、营养干预、慢性GVHD、营养缺乏、饮食、维生素、干眼症、益生菌、蛋白质和身体成分。在这篇报告中,我们总结了肿瘤学和代谢综合征背景下的介入营养研究,并描述了我们在造血细胞移植和慢性GVHD中的营养临床实践。营养评估和干预对慢性GVHD相关肌肉质量下降、干眼、视力障碍、代谢综合征、骨质疏松症和合并症的影响需要进行前瞻性研究。
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引用次数: 3
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。
{"title":"Prognostic Factors for Postrelapse Survival after ex Vivo CD34+-Selected (T Cell-Depleted) Allogeneic Hematopoietic Cell Transplantation in Multiple Myeloma","authors":"Alexandra Gomez-Arteaga ,&nbsp;Gunjan L. Shah ,&nbsp;Raymond E. Baser ,&nbsp;Michael Scordo ,&nbsp;Josel D. Ruiz ,&nbsp;Adam Bryant ,&nbsp;Parastoo B. Dahi ,&nbsp;Arnab Ghosh ,&nbsp;Oscar B. Lahoud ,&nbsp;Heather J. Landau ,&nbsp;Ola Landgren ,&nbsp;Brian C. Shaffer ,&nbsp;Eric L. Smith ,&nbsp;Guenther Koehne ,&nbsp;Miguel-Angel Perales ,&nbsp;Sergio A. Giralt ,&nbsp;David J. Chung","doi":"10.1016/j.bbmt.2020.07.016","DOIUrl":"10.1016/j.bbmt.2020.07.016","url":null,"abstract":"<div><p>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 (&lt;6 months; 28%), early (6 to 24 months; 50%), or late (&gt;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 (<em>P</em> = .002). Older age, relapse with EMD, &lt;PR before alloHCT, &lt;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.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 11","pages":"Pages 2040-2046"},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.016","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38195358","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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
期刊
Biology of Blood and Marrow Transplantation
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