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Uniform Graft-versus-Host Disease Prophylaxis using Post-Transplantation Cyclophosphamide, Methotrexate, and Cyclosporine following Peripheral Blood Hematopoietic Stem Cell Transplantation from Matched and Haploidentical Donors for Transfusion-Dependent Thalassemia: A Retrospective Report from the Bone Marrow Failure Working Group of Hunan Province, China. 输血依赖型地中海贫血患者从匹配供者和单倍体供者处移植外周血造血干细胞后,统一使用移植后环磷酰胺、甲氨蝶呤和环孢素预防移植物抗宿主病:来自中国湖南省BMF-WG的回顾性报告。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-03 DOI: 10.1016/j.jtct.2024.08.022
Susu Gong, Xin Tian, Rui Yang, Liangchun Yang, Zhiming Wang, Kaitai Yang, Keke Chen, Xianglin He, Wenjun Deng, Xiaoyang Yang, Meiqing Lei, Bin Fu
<p><p>Although the survival of patients with transfusion-dependent thalassemia (TD-TM) is reportedly inferior after haploidentical hematopoietic stem cell transplantation (HSCT), the heterogeneity of transplantation approaches in studies suggests the need to assess the effect of conditioning regimen on matched and haploidentical transplantation outcomes. A novel post-transplantation cyclophosphamide (PTCy)-based approach for patients with TD-TM undergoing haploidentical HSCT was reported in our prior study. Here we aimed to retrospectively evaluate the real-world efficacy and safety of graft-versus-host disease (GVHD) prophylaxis in patients with TD-TM after HSCT from matched donors and haploidentical donors (HIDs). In this retrospective multicenter study, among 238 patients with TD-TM who underwent HSCT, 160 underwent peripheral blood HSCT, using uniform GVHD prophylaxis with PTCy, methotrexate, and cyclosporine, at member centers of the Bone Marrow Failure Working Group of Hunan Province between 2019 and 2023. The median age of the cohort at transplantation was 6 years (95% confidence interval [CI], 6 to 7 years). The 160 donors included 99 (61.9%) haploidentical family members, 13 matched sibling donors, and 48 matched or mismatched unrelated donors. The engraftment rate was 98.8% (95% CI, 96.1% to 97.7%). HSCT from HIDs had a lower risk of mixed chimerism (HR, .078; P = .022). Within 100 days after transplantation, 31 patients (19.6%; 95% CI, 14.0% to 26.3%) had grade II-IV acute GVHD (aGVHD), 9 of whom had grade III-IV aGVHD (5.7%; 95% CI, 2.9% to 10.1%). HIDs were significantly associated with a higher risk of grade II-IV aGVHD (HR, 3.973; P = .009). Nineteen patients (11.9%; 95% CI, 7.6% to 17.6%) developed late aGVHD after a median of 516 days (95% CI, 407 to 709 days). Twenty-six patients (16.5%; 95% CI, 11.3% to 22.8%) exhibited any 1 of the diagnostic, distinctive, or atypical features of chronic GVHD (cGVHD) according to the 2014 National Institutes of Health (NIH) criteria after a median of 690 days (95% CI, 496 to 902 days). Among these 26 patients, 7 had NIH-defined cGVHD, 14 had only 1 distinctive sign with no histologic evidence, and 5 had only atypical cGVHD signs. Of the 26 patients, 5 were classified with overlap syndrome. Of 21 patients classified with NIH-defined and potential cGVHD, 3 had moderate cGVHD and 1 had severe cGVHD. Logistic regression analyses identified that grade II-IV aGVHD independently predicted subsequent cGVHD (HR, 3.920; P = .006). The rates of cGVHD were similar in the matched donor and HID groups. Thalassemia-free survival (TFS) and event-free survival (EFS) were 97.5% (95% CI, 94.2% to 99.2%) and 90.6% (95% CI, 85.4% to 94.4%), respectively, after a median of 690 days (95% CI, 496 to 902 days). TFS rates were similar in the matched donor and HID groups (P = .549). The EFS rate was significantly higher in the matched donor group compared to the HID group (P = .033). Our study suggests that when PTCy-ba
背景:据报道,输血依赖型地中海贫血(TD-TM)患者接受单倍体移植后的存活率较低,但研究中移植方法的异质性表明,有必要评估特定调理方案对配型和单倍体移植结果的影响:我们在之前的研究中报道了一种基于PTCy的新方法,用于接受单倍体造血干细胞移植的TD-TM患者。我们旨在回顾性评估TD-TM患者接受配型供者和单倍体供者(HID)造血干细胞移植后预防GvHD的实际疗效和安全性:这是一项回顾性多中心研究。在接受造血干细胞移植的238例TD-TM患者中,160例接受了外周血造血干细胞移植,并于2019年至2023年期间在湖南省骨髓衰竭工作组的成员中心使用PTCy、甲氨蝶呤和环孢素统一预防GvHD:队列的中位年龄为 6 岁(95% 置信区间 [CI],6-7 岁)。在160名供者中,99名(61.9%)为单倍体家庭成员,其他为配型供者(13名配型相合的兄弟姐妹,48名配型相合或不相合的非亲属供者)。移植率为 98.8%(95% CI:96.1%-97.7%)。HID造血干细胞移植出现混合嵌合体的风险较低(HR 0-078,P=0.022)。移植后100天内,31名患者(19.6%,95% CI:14.0%-26.3%)出现II-IV级急性GvHD,其中9人出现III-IV级急性GvHD(5.7%,95% CI:2.9%-10.1%)。HID与II-IV级急性GvHD的高风险明显相关(HR 3.973,P = 0.009)。19名患者(11.9%,95% CI:7.6%-17.6%)在中位516天(95% CI:407-709天)后出现晚期急性GvHD。26名患者(16.5%,95% CI:11.3%-22.8%)在中位数690天(95% CI:496-902天)后,根据2014年美国国立卫生研究院(NIH)标准表现出慢性GvHD的诊断性、显著性或非典型性特征。在这些患者中,7 人有 NIH 定义的慢性 GvHD,14 人仅有一个明显体征但无组织学证据,5 人仅有非典型慢性 GvHD 体征。在 26 名患者中,有 5 人被归类为重叠综合征。在21名被归类为NIH定义的潜在慢性风湿性关节炎患者中,3人患有中度慢性风湿性关节炎,1人患有重度慢性风湿性关节炎。逻辑回归分析发现,II-IV 级急性 GvHD 可独立预测随后的慢性 GvHD(HR 3.920,P=0.006)。匹配组和 HID 组的慢性 GvHD 发生率相似。在中位 690 天(95% CI:496-902 天)后,无地中海贫血生存期(TFS)和无事件生存期(EFS)分别为 97.5%(95% CI:94.2%-99.2%)和 90.6%(95% CI:85.4%-94.4%)。匹配组和 HID 组的 TFS 率相似(P = 0.549)。匹配组的 EFS 率明显高于 HID 组(p = 0.033):我们的研究表明,如果采用基于 PTCy 的统一 GVHD 预防措施,配型供体和 HID 的造血干细胞移植可降低严重 GVHD 的发生率和治疗相关死亡率,并获得令人满意的存活率。
{"title":"Uniform Graft-versus-Host Disease Prophylaxis using Post-Transplantation Cyclophosphamide, Methotrexate, and Cyclosporine following Peripheral Blood Hematopoietic Stem Cell Transplantation from Matched and Haploidentical Donors for Transfusion-Dependent Thalassemia: A Retrospective Report from the Bone Marrow Failure Working Group of Hunan Province, China.","authors":"Susu Gong, Xin Tian, Rui Yang, Liangchun Yang, Zhiming Wang, Kaitai Yang, Keke Chen, Xianglin He, Wenjun Deng, Xiaoyang Yang, Meiqing Lei, Bin Fu","doi":"10.1016/j.jtct.2024.08.022","DOIUrl":"10.1016/j.jtct.2024.08.022","url":null,"abstract":"&lt;p&gt;&lt;p&gt;Although the survival of patients with transfusion-dependent thalassemia (TD-TM) is reportedly inferior after haploidentical hematopoietic stem cell transplantation (HSCT), the heterogeneity of transplantation approaches in studies suggests the need to assess the effect of conditioning regimen on matched and haploidentical transplantation outcomes. A novel post-transplantation cyclophosphamide (PTCy)-based approach for patients with TD-TM undergoing haploidentical HSCT was reported in our prior study. Here we aimed to retrospectively evaluate the real-world efficacy and safety of graft-versus-host disease (GVHD) prophylaxis in patients with TD-TM after HSCT from matched donors and haploidentical donors (HIDs). In this retrospective multicenter study, among 238 patients with TD-TM who underwent HSCT, 160 underwent peripheral blood HSCT, using uniform GVHD prophylaxis with PTCy, methotrexate, and cyclosporine, at member centers of the Bone Marrow Failure Working Group of Hunan Province between 2019 and 2023. The median age of the cohort at transplantation was 6 years (95% confidence interval [CI], 6 to 7 years). The 160 donors included 99 (61.9%) haploidentical family members, 13 matched sibling donors, and 48 matched or mismatched unrelated donors. The engraftment rate was 98.8% (95% CI, 96.1% to 97.7%). HSCT from HIDs had a lower risk of mixed chimerism (HR, .078; P = .022). Within 100 days after transplantation, 31 patients (19.6%; 95% CI, 14.0% to 26.3%) had grade II-IV acute GVHD (aGVHD), 9 of whom had grade III-IV aGVHD (5.7%; 95% CI, 2.9% to 10.1%). HIDs were significantly associated with a higher risk of grade II-IV aGVHD (HR, 3.973; P = .009). Nineteen patients (11.9%; 95% CI, 7.6% to 17.6%) developed late aGVHD after a median of 516 days (95% CI, 407 to 709 days). Twenty-six patients (16.5%; 95% CI, 11.3% to 22.8%) exhibited any 1 of the diagnostic, distinctive, or atypical features of chronic GVHD (cGVHD) according to the 2014 National Institutes of Health (NIH) criteria after a median of 690 days (95% CI, 496 to 902 days). Among these 26 patients, 7 had NIH-defined cGVHD, 14 had only 1 distinctive sign with no histologic evidence, and 5 had only atypical cGVHD signs. Of the 26 patients, 5 were classified with overlap syndrome. Of 21 patients classified with NIH-defined and potential cGVHD, 3 had moderate cGVHD and 1 had severe cGVHD. Logistic regression analyses identified that grade II-IV aGVHD independently predicted subsequent cGVHD (HR, 3.920; P = .006). The rates of cGVHD were similar in the matched donor and HID groups. Thalassemia-free survival (TFS) and event-free survival (EFS) were 97.5% (95% CI, 94.2% to 99.2%) and 90.6% (95% CI, 85.4% to 94.4%), respectively, after a median of 690 days (95% CI, 496 to 902 days). TFS rates were similar in the matched donor and HID groups (P = .549). The EFS rate was significantly higher in the matched donor group compared to the HID group (P = .033). Our study suggests that when PTCy-ba","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142141205","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Effects of Consolidation Therapy With Autologous Hematopoietic Stem Cell Transplantation After BCMA-CAR T-Cell Therapy on the Survival of Patients With Relapsed or Refractory Multiple Myeloma. BCMA-CAR T细胞疗法后进行自体造血干细胞移植巩固治疗对复发或难治性多发性骨髓瘤患者生存期的影响。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-03 DOI: 10.1016/j.jtct.2024.08.024
Ziwei Zhou, Xuan Liu, Xuejun Zhang, Shupeng Wen, Huan Hua, Zheng Xu, Fuxu Wang

Despite the success of chimeric antigen receptor (CAR) T-cell therapy for relapsed or refractory multiple myeloma (RRMM), failure after CAR T-cell therapy remains an unmet medical need. An effective consolidation therapy after CAR T-cell therapy may improve the prognosis of RRMM. To investigate the effects of consolidation therapy with autologous hematopoietic stem cell transplantation (AHCT) after B-cell maturation antigen (BCMA)-targeted CAR T-cell therapy on the prognosis of RRMM patients. This retrospective study included 39 RRMM patients who received BCMA-targeted CAR T-cell therapy. Basic clinical, therapy, and outcome data were collected, and factors associated with survival were analyzed. Among the 39 RRMM patients included in the study, 15 had high-risk cytogenetics and 11 had extramedullary disease (EMD). All 39 patients reached peak CAR T-cell expansion within 28 days after infusion. Twenty-six patients developed cytokine release syndrome, including 12 grade 1 and 14 grade 2 cases. Survival analysis revealed that high-risk cytogenetics, high tumor load (International Staging System [ISS] stage III), and EMD were negatively associated with progression-free survival (PFS) and overall survival (OS). Thirteen patients received consolidation AHCT therapy 50-276 days after CAR T-cell therapy, with a median interval of 92 days. No serious complications occurred after consolidation AHCT. Survival analysis showed that consolidation AHCT effectively improved OS and PFS over maintenance chemotherapy. Moreover, Cox regression analysis identified low tumor load (ISS stage I/II) and consolidation AHCT as independent predictors of superior PFS and OS and high-risk cytogenetics as an independent risk factor for poor PFS. Consolidation AHCT after CAR T-cell therapy in RRMM patients can improve patient survival.

背景:尽管嵌合抗原受体(CAR)T细胞疗法在治疗复发或难治性多发性骨髓瘤(RRMM)方面取得了成功,但CAR T细胞疗法后的失败仍是一项尚未满足的医疗需求。在CAR T细胞治疗后进行有效的巩固治疗可改善RRMM的预后:目的:研究B细胞成熟抗原(BCMA)靶向CAR T细胞治疗后进行自体造血干细胞移植(AHCT)巩固治疗对RRMM患者预后的影响:这项回顾性研究纳入了39名接受BCMA靶向CAR T细胞治疗的RRMM患者。收集了基本的临床、治疗和预后数据,并分析了与生存相关的因素:在纳入研究的39例RRMM患者中,15例有高风险细胞遗传学,11例有髓外疾病(EMD)。所有39名患者均在输注后28天内达到CAR T细胞扩增峰值。26名患者出现了细胞因子释放综合征,包括12例1级病例和14例2级病例。生存分析显示,高危细胞遗传学、高肿瘤负荷(国际分期系统[ISS]III期)和EMD与无进展生存期(PFS)和总生存期(OS)呈负相关。13名患者在CAR T细胞治疗后50-276天接受了AHCT巩固治疗,中位间隔为92天。巩固AHCT治疗后未出现严重并发症。生存期分析表明,与维持化疗相比,巩固AHCT能有效改善OS和PFS。此外,Cox回归分析发现,低肿瘤负荷(ISS I/II期)和巩固性AHCT是优越PFS和OS的独立预测因素,而高危细胞遗传学是不良PFS的独立风险因素:结论:RRMM患者接受CAR T细胞治疗后进行AHCT巩固治疗可提高患者生存率。
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引用次数: 0
ASTCT and USCLC Clinical Practice Recommendations for Allogeneic Stem Cell Transplant in Mycosis Fungoides and Sézary Syndrome. ASTCT 和 USCLC 关于真菌病和塞扎里综合征异基因干细胞移植的临床实践建议。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-01 DOI: 10.1016/j.jtct.2024.08.020
Amrita Goyal, Daniel O'Leary, Bouthaina Dabaja, Wen-Kai Weng, Jasmine Zain, Corey Cutler, Joan Guitart, Youn H Kim, Larisa J Geskin, Richard T Hoppe, Lynn D Wilson, Anne W Beaven, Steve Horwitz, Pamela B Allen, Stefan K Barta, Kimberly Bohjanen, Jonathan E Brammer, Joi B Carter, Nneka Comfere, Jennifer A DeSimone, Kathryn Dusenbery, Madeleine Duvic, Auris Huen, Deepa Jagadeesh, Chris R Kelsey, Michael S Khodadoust, Mary Jo Lechowicz, Neha Mehta-Shah, Alison J Moskowitz, Elise A Olsen, Christina Poh, Barbara Pro, Christiane Querfeld, Craig Sauter, Lubomir Sokol, Olayemi Sokumbi, Ryan A Wilcox, John A Zic, Mehdi Hamadani, Francine Foss

Mycosis fungoides (MF) and Sézary syndrome (SS) are the most common subtypes of cutaneous T-cell lymphoma (CTCL). While MF generally follows an indolent course, a subset of patients will experience progressive and/or treatment-refractory disease; Sézary syndrome is an aggressive lymphoma associated with high morbidity and mortality. Although allogeneic hematopoietic cell transplant (allo-HCT) is the only currently available potentially curative treatment modality for MF/SS there is no published guidance on referral criteria, transplant timing orallo-HCT approach. To develop consensus clinical practice recommendations, we performed a Delphi survey of 32 specialists in dermatology (n = 9), transplant hematology/oncology (n = 10), non-transplant hematology/oncology (n = 8), and radiation oncology (n = 5) from across the United States. Consensus required agreement of ≥75% of participants. Sixteen consensus statements were generated on four topics: (1) criteria for referral for consideration for allo-HCT, (2) allo-HCT preparative regimens and procedures (3) disease status at the time of allo-HCT, and (4) multidisciplinary management in the pre- and post-transplant settings. These clinical practice guidelines provide a framework for decision-making regarding allo-HCT for MF/SS and highlight areas for future prospective investigation.

导言:真菌病(MF)和塞扎里综合征(SS)是皮肤T细胞淋巴瘤(CTCL)最常见的亚型。霉形体病的病程一般比较缓慢,但也有一部分患者会出现进展性和/或难治性疾病。塞扎里综合征是一种侵袭性 CTCL,由于免疫受损和机会性感染,发病率和死亡率都很高。尽管异基因造血细胞移植(allo-HCT)是目前唯一可治愈 MF/SS 的治疗方式,并已被纳入 NCCN 和 ASTCT 治疗指南,但目前还没有关于转诊标准、时机和 allo-HCT 方法的公开指南来帮助指导临床医生治疗这些患者:对全美 32 名皮肤科(9 人)、移植血液/肿瘤科(10 人)、非移植血液/肿瘤科(8 人)和放射肿瘤科(5 人)的专家进行德尔菲调查。达成共识需要≥75%的参与者同意:结果:就四个主题达成了 16 项共识声明:1) 考虑接受同种异体肝移植的转诊标准;2) 同种异体肝移植的准备方案和程序;3) 同种异体肝移植时的疾病状态;4) 移植前后的多学科管理:这些临床实践指南为MF/SS的allo-HCT决策提供了一个框架,并强调了未来前瞻性研究的领域。
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引用次数: 0
Cutaneous Chronic Graft-Versus-Host Disease: Clinical Manifestations, Diagnosis, Management, and Supportive Care. 皮肤慢性移植物抗宿主病:皮肤慢性移植物抗宿主病:临床表现、诊断、管理和支持性护理》(Clinical Manifestations, Diagnosis, Management, and Supportive Care)。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-01 DOI: 10.1016/j.jtct.2024.05.020
Connie R Shi, Alana L Ferreira, Manjit Kaur, David Xiang, Jean Caputo, Hannah K Choe, Nada Hamad, Edward W Cowen, Benjamin H Kaffenberger, Emily Baumrin

Cutaneous chronic graft-versus-host disease (cGVHD) is associated with morbidity, mortality, and impaired quality of life after hematopoietic stem cell transplantation. The clinical features of cutaneous cGVHD are heterogeneous but can be broadly classified into nonsclerotic or sclerotic presentations. This review provides an overview of clinical presentation, diagnosis and differential diagnosis, grading, and treatment of cutaneous cGVHD. Particular attention is given to cutaneous cGVHD in skin of color, which can have unique features and is generally underrepresented in the literature leading to delays in diagnosis. Finally, an overview of long-term skin care for patients with cutaneous cGVHD is provided in order to support patients from a dermatologic perspective as they recover from cGVHD. Multidisciplinary care with frequent communication between transplant specialists and dermatologists is critical to effectively managing cutaneous cGVHD.

皮肤慢性移植物抗宿主疾病(cGVHD)与造血干细胞移植后的发病率、死亡率和生活质量下降有关。皮肤 cGVHD 的临床特征各不相同,但大致可分为非硬化性和硬化性两种表现。本综述概述了皮肤cGVHD的临床表现、诊断和鉴别诊断、分级和治疗。本综述特别关注有色人种皮肤 cGVHD,因为这种疾病可能具有独特的特征,而且在文献中通常代表性不足,导致诊断延误。最后,概述了皮肤 cGVHD 患者的长期皮肤护理,以便在患者从 cGVHD 康复过程中从皮肤科角度为他们提供支持。移植专家和皮肤科医生之间经常沟通的多学科护理对于有效控制皮肤 cGVHD 至关重要。
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引用次数: 0
Improving Outcomes in Allogeneic Transplantation and Chronic Graft-versus-Host Disease Patients through Lifestyle Medicine: Current Landscape and Future Directions. 通过生活方式医学改善同种异体移植和慢性移植物抗宿主疾病患者的治疗效果:当前形势与未来方向》。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-01 DOI: 10.1016/j.jtct.2024.05.023
Aaron T Zhao, Noa G Holtzman, Mladen Golubic, Steven Z Pavletic

Although lifestyle interventions have shown promise in oncology and for cancer survivorship, their potential to improve outcomes in allogeneic hematopoietic cell transplantation (allo-HCT) and chronic graft-versus-host disease (cGVHD) patients remains to be fully explored. Given the high rates of cardiovascular disease, metabolic syndrome, and secondary malignancy in this patient population, lifestyle modifications can serve as a vital frontline defense against chronic diseases. Current research has illuminated the potential supportive role of lifestyle interventions in the solid cancer patient population, which is encouraging future lifestyle medicine research for patients with hematologic malignancies and allo-HCT recipients. Recent studies have indicated the pernicious effects of poor lifestyle choices on the course of cGVHD development and survival. The intersection between certain pillars of lifestyle medicine (ie, nutrition and exercise) and allo-HCT patient outcomes has been more well documented than that of other pillars (ie, social relationships and spirituality). Ongoing randomized trials studying the effects of exercise and nutrition on clinical outcomes in cGVHD and allo-HCT patients may provide important future evidence of the role of lifestyle medicine in this patient population. In this review, we describe the current landscape of lifestyle medicine in allo-HCT and cGVHD, its potential, and propose ways to further develop this evolving field of medicine.

尽管生活方式干预在肿瘤学和癌症生存方面已显示出前景,但其改善异基因造血细胞移植(allo-HCT)和慢性移植物抗宿主病(cGVHD)患者预后的潜力仍有待充分发掘。鉴于这类患者中心血管疾病、代谢综合征和继发性恶性肿瘤的发病率较高,改变生活方式可以成为预防慢性疾病的重要前线。目前的研究揭示了生活方式干预在实体癌患者群体中的潜在支持作用,这鼓励了未来针对血液系统恶性肿瘤患者和异体肝移植受者的生活方式医学研究。最近的研究表明,不良生活方式会对 cGVHD 的发展和生存产生有害影响。生活方式医学的某些支柱(即营养和运动)与异体肝移植患者预后之间的交叉关系比其他支柱(即社会关系和精神)得到了更多的记录。正在进行的随机试验研究了运动和营养对 cGVHD 和allo-HCT 患者临床疗效的影响,这可能会为生活方式医学在这一患者群体中的作用提供重要的证据。在这篇综述中,我们描述了生活方式医学在异体肝移植和 cGVHD 中的现状、潜力,并提出了进一步发展这一不断发展的医学领域的方法。
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引用次数: 0
Differences in Acute Graft-Versus-Host Disease (GVHD) Severity and Its Outcomes Between Black and White Patients. 黑人和白人患者的急性移植物抗宿主病(GVHD)严重程度及其结果的差异。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-01 DOI: 10.1016/j.jtct.2024.08.019
Carlos A Ortega Rios, Muna Qayed, Aaron M Etra, Ran Reshef, Richard Newcomb, Nicholas Yuhasz, Elizabeth O Hexner, Paibel Aguayo-Hiraldo, Pietro Merli, William J Hogan, Daniela Weber, Carrie L Kitko, Francis Ayuk, Matthias Eder, Stephan A Grupp, Sabrina Kraus, Karam Sandhu, Evelyn Ullrich, Ingrid Vasova, Matthias Wölfl, Janna Baez, Rahnuma Beheshti, Gilbert Eng, Sigrun Gleich, Nikolaos Katsivelos, Steven Kowalyk, Ioannis Evangelos Louloudis, George Morales, Nikolaos Spyrou, Rachel Young, Ryotaro Nakamura, John E Levine, James L M Ferrara, Yu Akahoshi

Acute graft-versus-host disease (GVHD) is a significant complication following hematopoietic stem cell transplantation (HCT). Although recent advancements in GVHD prophylaxis have resulted in successful HCT across HLA barriers and expanded access to HCT for racial minorities, less is known about how race affects the severity and outcomes of acute GVHD. This study examines differences in the clinical course of acute GVHD and the prognostic value of GVHD biomarkers for Black and White recipients. We conducted a retrospective analysis of patients in the Mount Sinai Acute GVHD International Consortium (MAGIC) database who underwent HCT between 2014 and 2021 to describe the difference in clinical course of acute GVHD and significance of GVHD biomarkers between Black and White recipients. We used propensity score matching to generate a 1:3 matched cohort of 234 Black patients and 702 White patients with similar baseline characteristics. In the first year after HCT Black patients experienced a higher cumulative incidence of grade III-IV acute GVHD (17% versus 12%, P = 0.050), higher nonrelapse mortality (NRM; 18% versus 12%, P = .009), and lower overall survival that trended toward statistical significance (73% versus 79%, P = .071) compared to White patients. The difference in NRM in the first year was even greater among Black patients who developed GVHD than White patients (24% versus 14%, P = .041). The distribution of low, intermediate, and high MAGIC biomarker scores at the time of treatment was similar across racial groups (P = .847), however, Black patients with high biomarker scores experienced significantly worse NRM than White patients (71% versus 32%, P = .010). Our data indicate that Black patients are at a higher risk of NRM following HCT, primarily from a higher incidence of severe GVHD. Serum biomarkers at treatment initiation can stratify patients for risk of NRM across races, however Black patients with high biomarker scores had a significantly greater NRM risk. These results suggest a need for strategies that mitigate the higher risk for poor GVHD outcomes among Black patients.

背景:急性移植物抗宿主疾病(GVHD急性移植物抗宿主疾病(GVHD)是造血干细胞移植(HCT)后的重要并发症。尽管最近在预防GVHD方面取得的进展已使HCT成功跨越了HLA障碍,并扩大了少数种族接受HCT的机会,但人们对种族如何影响急性GVHD的严重程度和结果知之甚少:本研究探讨了黑人和白人受者急性GVHD临床过程的差异以及GVHD生物标志物的预后价值:我们对西奈山急性GVHD国际联盟(MAGIC)数据库中2014年至2021年间接受HCT的患者进行了回顾性分析,以描述黑人和白人受者之间急性GVHD临床过程的差异以及GVHD生物标志物的意义。我们使用倾向得分匹配法生成了一个1:3的匹配队列,其中包括234名黑人患者和702名基线特征相似的白人患者:与白人患者相比,黑人患者在接受造血干细胞移植后第一年的 III-IV 级急性 GVHD 累积发生率更高(17% vs 12%,P = 0.050),非复发死亡率(NRM;18% vs 12%,P = 0.009)更高,总生存率更低,且有统计学意义(73% vs 79%,P = 0.071)。与白人患者相比,出现 GVHD 的黑人患者第一年的 NRM 差异更大(24% vs 14%,P = 0.041)。不同种族群体在接受治疗时MAGIC生物标志物得分的低、中、高分布相似(P = 0.847),但生物标志物得分高的黑人患者的NRM明显低于白人患者(71% vs 32%,P = 0.010):我们的数据表明,黑人患者接受 HCT 后出现 NRM 的风险较高,主要是因为严重 GVHD 的发生率较高。开始治疗时的血清生物标志物可对不同种族患者的 NRM 风险进行分层,但生物标志物评分高的黑人患者的 NRM 风险明显更高。这些结果表明,有必要采取一些策略来降低黑人患者出现不良 GVHD 后果的较高风险。
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引用次数: 0
Forward to Chronic GVHD Supplement in Transplantation and Cellular Therapy. 转发至《移植和细胞疗法中的慢性 GVHD 增补》。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-01 DOI: 10.1016/j.jtct.2024.08.017
Corey Cutler
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引用次数: 0
Patient and Physician Communication in the Allogeneic Transplantation Setting: Challenges and Potential Solutions. 同种异体移植中的患者与医生沟通:挑战与潜在解决方案。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-01 DOI: 10.1016/j.jtct.2024.04.020
Anna Barata, Guy Tavori, Daniel Wolff, Anne Herrmann

Allogeneic hematopoietic cell transplantation is a challenging treatment characterized by multiple morbidities, the need for long-term care, and a significant mortality risk. Consequently, close patient and physician communication throughout treatment is crucial. We aimed to review the literature examining patient and physician communication around critical aspects experienced by allogeneic survivors over the transplantation trajectory, such as the informed consent process, transplantation-related morbidity (eg, psychosocial distress, cognitive dysfunction, sexuality), adherence to treatment, and the use of complementary and alternative medicine, as well as interventions and strategies to improve patient and physician communication. We found a paucity of studies examining communication on these topics. Nevertheless, there is evidence of significant communication gaps around morbidities often experienced by allogeneic survivors, such as psychosocial distress, fatigue, and sexual functioning, due to both patient and physician barriers. Similarly, there is a concern that gaps also exist when addressing the informed consent process, cognitive dysfunction, adherence to treatment, and use of complementary and alternative medicine. The use and discussion of patient-reported outcome measures as part of clinical care is associated with patient and physician satisfaction with communication and better detection and management of symptoms. Although other strategies, such as decision aids, question prompt lists, and communication skills training, have improved communication in oncology, they have not been tested in the allogeneic setting. Future research is clearly needed to examine patient and physician communication in the allogeneic transplantation setting and test strategies to improve communication during this challenging treatment.

异基因造血细胞移植是一种具有挑战性的治疗方法,其特点是多种发病率、需要长期护理以及显著的死亡风险。因此,在整个治疗过程中,患者与医生的密切沟通至关重要。我们旨在查阅相关文献,围绕异体移植幸存者在移植过程中经历的关键环节,如知情同意程序、移植相关发病率(如社会心理困扰、认知功能障碍、性生活)、坚持治疗、使用补充和替代药物等,研究患者与医生的沟通情况,以及改善患者与医生沟通的干预措施和策略。我们发现就这些主题进行沟通的研究很少。尽管如此,有证据表明,由于患者和医生双方的障碍,异体移植幸存者在社会心理困扰、疲劳和性功能等常见疾病方面的沟通存在很大差距。同样,在处理知情同意程序、认知功能障碍、坚持治疗以及使用补充和替代药物方面也存在差距。作为临床护理的一部分,使用和讨论患者报告的结果测量与患者和医生对沟通的满意度以及更好地发现和管理症状有关。虽然决策辅助工具、问题提示清单和沟通技巧培训等其他策略改善了肿瘤学中的沟通,但这些策略尚未在异体病例中进行过测试。未来的研究显然需要对异体移植环境中患者和医生的沟通情况进行检查,并测试在这种具有挑战性的治疗过程中改善沟通的策略。
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引用次数: 0
Understanding Ocular Graft-versus-Host Disease to Facilitate an Integrated Multidisciplinary Approach. 了解眼部移植物抗宿主病,促进多学科综合方法。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-01 Epub Date: 2024-07-08 DOI: 10.1016/j.jtct.2024.06.031
Pier Luigi Surico, Zhonghui K Luo

Ocular graft-versus-host disease (oGVHD) remains a challenging and potentially devastating complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT). Although oGVHD significantly impacts the quality of life of affected survivors, it often goes unrecognized, particularly in the early stages. Targeting all providers in the HSCT community who see patients regularly and frequently for their post-allo-HSCT care, this review and opinion piece introduces the basic concepts of ocular surface pathophysiology, dissects the different stages of clinical presentation of oGVHD, explains why the current diagnostic criteria tend to capture the late disease stages, and highlights the warning signs of early disease development to facilitate prompt referral of oGVHD suspects for ocular specialist care. Along with introducing a comprehensive list of treatment options, this review emphasizes basic therapeutic strategy and options that can be safely and effectively initiated by any care provider. We believe in empowering patients as well as care providers beyond disciplinary boundaries to provide the most cohesive and integrated care in a multidisciplinary approach.

眼部移植物抗宿主疾病(oGVHD)仍然是异基因造血干细胞移植(allo-HSCT)后的一种具有挑战性和潜在破坏性的并发症。它严重影响受影响幸存者的生活质量,但往往未被充分认识,尤其是在早期阶段。这篇综述和观点文章针对造血干细胞移植社区中定期和经常为异体造血干细胞移植后患者提供护理的所有医疗人员,介绍了眼表病理生理学的基本概念,剖析了oGVHD临床表现的不同阶段,解释了为什么目前的诊断标准倾向于捕捉疾病的晚期阶段,强调了疾病早期发展的警示信号,希望能促进oGVHD疑似患者及时转诊接受眼科专科护理。除了介绍全面的治疗方案外,这篇综述还强调了基本的治疗策略和方案,这些方案安全有效,任何医疗服务提供者都可以采用。我们相信,为了以多学科方法为患者提供最具凝聚力的综合治疗,患者和医疗服务提供者都应超越学科界限。
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引用次数: 0
Late-Onset Noninfectious Pulmonary Complications after Hematopoietic Stem Cell Transplantation. 造血干细胞移植后晚期非感染性肺部并发症。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-01 DOI: 10.1016/j.jtct.2024.05.022
Andrew C Harris, Kimia Ganjaei, Camila Vilela, Alexander Geyer
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引用次数: 0
期刊
Transplantation and Cellular Therapy
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