Bone Marrow Transplantation

骨髓移植
  • 文章类型: Journal Article
    γδ(γδ)T细胞占人类T细胞库的一小部分,但在异基因造血干细胞移植(allo-HSCT)的背景下,在介导抗感染和抗肿瘤作用中起重要作用。我们进行了一项前瞻性研究,以分析不同移植方式对γδT细胞和亚群免疫重建的影响。平行分析CD3、CD4和CD8T细胞。其次,我们研究了γδT细胞重建对包括急性移植物抗宿主病(aGvHD)和病毒感染在内的临床结局的影响.我们的队列包括49名儿科患者,他们接受了来自匹配的无关(MUD)或匹配的相关(MRD)供体的未经操作的骨髓移植。该队列包括患有恶性和非恶性疾病的患者。在移植后15、30、60、100、180和240天使用流式细胞术测量细胞计数。对细胞进行CD3、CD4、CD8、CD45、TCRαβ、TCRγδ,TCRVδ1、TCRVδ2、HLA-DR及其组合。在单变量分析中,MRD患者在时间点+30、+60、+100(分别为p<0.001)和+180(p<0.01)表现出明显高于MUD患者的Vδ2+T细胞。这些结果在多变量分析中仍然显著。具有高相对丰度的总γδT细胞和Vδ2T细胞恢复的患者移植后II-IV级aGvHD的累积发生率显着降低(分别为p=0.03和p=0.04)。高相对丰度的Vδ2+T细胞也与较低的EBV感染发生率相关(p=0.02)。另一方面,与没有EBV感染的患者相比,EBV感染的患者在移植后第100天和第180天显示出更高的绝对Vδ1T细胞计数(分别为p=0.046和0.038)。该结果在多变量时间平均分析(q<0.1)中保持显著。我们的结果表明,γδT细胞,尤其是Vδ2T细胞亚群对小儿HSCT后aGvHD和EBV感染的发展具有保护作用。Vδ1+T细胞可能参与EBV感染后的免疫反应。我们的结果鼓励进一步研究γδT细胞作为HSCT后的预后标志物和过继性T细胞转移策略的可能靶标。
    Gamma delta (γδ) T cells represent a minor fraction of human T cell repertoire but play an important role in mediating anti-infectious and anti-tumorous effects in the context of allogeneic hematopoietic stem cell transplantation (allo-HSCT). We performed a prospective study to analyze the effect of different transplant modalities on immune reconstitution of γδ T cells and subsets. CD3, CD4 and CD8 T cells were analyzed in parallel. Secondly, we examined the impact of γδ T cell reconstitution on clinical outcomes including acute Graft-versus-Host-Disease (aGvHD) and viral infections. Our cohort includes 49 pediatric patients who received unmanipulated bone marrow grafts from matched unrelated (MUD) or matched related (MRD) donors. The cohort includes patients with malignant as well as non-malignant diseases. Cell counts were measured using flow cytometry at 15, 30, 60, 100, 180 and 240 days after transplantation. Cells were stained for CD3, CD4, CD8, CD45, TCRαβ, TCRγδ, TCRVδ1, TCRVδ2, HLA-DR and combinations. Patients with a MRD showed significantly higher Vδ2+ T cells than those with MUD at timepoints +30, +60, +100 (p<0.001, respectively) and +180 (p<0.01) in univariate analysis. These results remained significant in multivariate analysis. Patients recovering with a high relative abundance of total γδ T cells and Vδ2+ T cells had a significantly lower cumulative incidence of grade II-IV aGvHD after transplantation (p=0.03 and p=0.04, respectively). A high relative abundance of Vδ2+ T cells was also associated with a lower incidence of EBV infection (p=0.02). Patients with EBV infection on the other hand showed higher absolute Vδ1+ T cell counts at days +100 and +180 after transplantation (p=0.046 and 0.038, respectively) than those without EBV infection. This result remained significant in a multivariate time-averaged analysis (q<0.1). Our results suggest a protective role of γδ T cells and especially Vδ2+ T cell subset against the development of aGvHD and EBV infection after pediatric HSCT. Vδ1+ T cells might be involved in the immune response after EBV infection. Our results encourage further research on γδ T cells as prognostic markers after HSCT and as possible targets of adoptive T cell transfer strategies.
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  • 文章类型: Case Reports
    骨髓移植(BMT)挽救了Omenn综合征的生命,严重的免疫缺陷疾病.及时的遗传诊断和匹配的供体BMT至关重要。强调新生儿筛查和多学科护理可改善患有遗传性疾病的婴儿的结局。及时的干预和全面的管理对于成功的移植结果至关重要。
    Omenn综合征是联合免疫缺陷的一种严重变异,其特征是免疫反应失调和对反复感染的易感性。我们介绍了一个3个月大的女婴,最初出现上呼吸道感染症状和弥漫性皮疹,对当地治疗无反应。在4个月大的时候,患者接受了同种异体骨髓移植(BMT),该移植利用了来自完全匹配的同胞供体的干细胞.移植前条件包括抗菌预防和支持治疗。BMT之后,患者接受免疫抑制药物治疗以预防移植物排斥反应和移植物抗宿主病.移植后的临床监测显示及时的中性粒细胞和血小板植入,随后的随访表明,临床参数稳定,巨细胞病毒状态为阴性。该案例强调了及时诊断和治疗在管理严重免疫缺陷疾病中的重要性,通过适当的及时干预,证明成功结果的潜力。定期监测和后续任命对于确保治疗成功至关重要。该案例还强调了多学科护理和基因检测在识别和管理罕见免疫缺陷疾病中的重要性。这种情况下的成功结果为将来改善治疗选择和更好的患者结果提供了希望。
    UNASSIGNED: Bone marrow transplantation (BMT) saves lives in Omenn syndrome, a severe immunodeficiency disorder. Timely genetic diagnosis and matched donor BMT are crucial. Emphasis on newborn screening and multidisciplinary care improves outcomes for infants with inherited disorders. Prompt intervention and comprehensive management are vital for a successful transplant outcome.
    UNASSIGNED: Omenn syndrome represents a severe variant of combined immunodeficiency characterized by disregulated immune responses and susceptibility to recurrent infections. We present the case of a 3-month-old female infant initially presenting with upper respiratory infection symptoms and a diffuse rash, unresponsive to local treatment. At 4 months of age, the patient underwent allogeneic bone marrow transplantation (BMT) utilizing stem cells from a fully matched sibling donor. Pre-transplant conditioning included antimicrobial prophylaxis and supportive therapies. Following BMT, the patient received immunosuppressive medications to prevent graft rejection and graft-versus-host disease. Clinical monitoring post-transplant showed timely neutrophil and platelet engraftment, with subsequent follow-up demonstrating stable clinical parameters and negative cytomegalovirus status. The case highlights the importance of timely diagnosis and treatment in managing severe immunodeficiency disorders, demonstrating the potential for successful outcomes with appropriate timely interventions. Regular monitoring and follow-up appointments were crucial in ensuring the success of the treatment. This case also emphasizes the significance of multidisciplinary care and genetic testing in identifying and managing rare immunodeficiency disorders. The successful outcome in this case provides hope for improved treatment options and better patient outcomes in the future.
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  • 文章类型: Journal Article
    目的:评估源自骨髓抽吸物的生物再生支架的功效,松质骨自体移植,富血小板血浆和自体纤维蛋白治疗股骨髁上骨不连。方法和材料:三名在多次手术失败后出现骨不连的患者接受了骨稳定和新型生物再生支架的应用。术前、术后6个月、12个月和24个月收集X线和主观量表。结果:所有骨不连均表现出愈合,并形成足够的愈伤组织,放射学证实。六个月后,所有患者恢复完全负重行走,无疼痛.统计学分析表明,与手术前相比,所有量表均有所改善。结论:该方法可作为治疗多次手术失败后股骨髁上骨不连的一种选择。
    这个总结是关于什么的?这个案例系列研究的目的是评估一种新的生物自体支架的有效性,由干细胞和血细胞以及血液衍生物组成,治疗股骨髁上不愈合的挑战性病例。结果如何?三名参与者接受了这种手术方法的应用,并接受了为期2年的监测。该疗法耐受良好并且被认为是安全的。值得注意的是,所有3例患者均经历了疼痛显著减轻和功能改善.几个月后,他们能够完全负重地行走而没有疼痛,到6个月时,明显的骨愈合迹象明显。结果是什么意思?这项研究表明,自体血的手术应用,松质骨和骨髓,遵循所描述的概念和方法,是一种有效的,股骨骨不连的安全持久治疗。它明显减轻疼痛,增强腿部功能,并在生活质量方面有统计学意义的显着改善。
    Aim: To assess the efficacy of a bioregenerative scaffold derived from bone marrow aspirate, cancellous bone autograft, platelet-rich plasma and autologous fibrin in treating supracondylar femur nonunions. Methods & materials: Three patients with nonunions following multiple surgical failures underwent bone stabilization and the application of a novel bioregenerative scaffold. x-rays and subjective scales were collected before surgery and at 6, 12 and 24 months post-surgery. Results: All nonunions exhibited healing with sufficient callus formation, as confirmed radiologically. After 6 months, all patients resumed full weight-bearing walking without pain. Statistical analysis showed improvements in all scales compared with pre-surgical values. Conclusion: This method presents itself as an option for treating supracondylar femur nonunions following multiple surgical failures.
    What is this summary about? The objective of this case series study was to evaluate the effectiveness of a new biological autologous scaffold, comprised of stem and blood cells along with blood derivatives, in treating challenging cases of supracondylar femur nonunions.What were the results? Three participants underwent the application of this surgical method and were monitored for a period of 2 years. The therapy was well tolerated and deemed safe. Notably, all three patients experienced significant reductions in pain and improvements in functionality. Within a few months, they were able to walk with full weightbearing without pain, and clear indications of progressing toward bone union were evident by the 6 months.What do the results mean? This study demonstrates that the surgical application of autologous blood, cancellous bone and bone marrow, following the described concept and method, is an effective, safe and enduring treatment for femur nonunions. It markedly diminishes pain, enhances leg function and yields statistically significant improvements in quality of life.
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  • 文章类型: Journal Article
    烧伤的愈合通常与许多并发症有关。每年都会开发出各种新的修复材料,并在实验上用于治疗烧伤伤口。患有糖尿病的人通常遭受慢性伤口愈合。血管,神经病,免疫功能,和生化异常都有助于改变组织修复。伴随所有糖尿病溃疡的一个潜在因素是血管流动不良,阻碍伤口正常愈合的情况。许多研究已经强调了组织修复中足够的血管充足性和血管增殖的重要性以及在糖尿病伤口愈合中缺乏血管的重要性。其他研究已经研究了受损的毛细血管重塑和血管成熟是否可能在受损的糖尿病伤口愈合中起作用。
    这项研究计划报道石榴皮(PP)粉末和自体骨髓(BM)的混合物对烧伤治疗的影响实验诱发的糖尿病兔。
    四氧嘧啶一水合物已用于在50只兔子中产生糖尿病。然后在每只兔子中,实验创造了两个深二度烧伤伤口。然后将动物随机分为5个治疗部分:非治疗对照(C1),用可用的商业伤口粉末处理(C2),用PP粉末处理,单独使用BM治疗,最后一组用带有骨髓的PP粉末(PPBM)处理。测量伤口闭合的速度和愈合过程中的组织病理学变化。在第0、4、8和12天测量兔血小板衍生生长因子AA(PDGF-AA)和兔蛋白酶激活受体1(PAR-1)的生物标志物水平。
    与对照非治疗组相比,所有治疗组的伤口愈合明显更快。有趣的是,与其他治疗组相比,在PPBM组中观察到了快速的伤口治愈。组织学评估澄清了PPBM组的成纤维细胞和胶原评分相对于其他切片的显著升高。此外,各组间血清生物标志物PDGF-AA和PAR-1水平显著升高.
    根据当前研究的结果,可以得出结论,PP粉末与BMPPBM均显着加速了实验性糖尿病兔烧伤创面的愈合过程。
    UNASSIGNED: Healing of bum wounds is commonly associated with many complications. Every year various new repair materials are developed and experimentally used for treating burn wounds. Humans with diabetes mellitus usually suffer from chronic wound healing. Vascular, neuropathic, immune function, and biochemical abnormalities each contribute to the altered tissue repair. One underlying factor that accompanies all diabetic ulcerations is poor vascular flow, a circumstance that impedes proper wound healing. Numerous studies have highlighted the importance of adequate vascular sufficiency and vessel proliferation in tissue repair and the lack thereof in diabetic wound healing. Other studies have looked at whether disarrayed capillary remodeling and maturation of vessels might play a role in impaired diabetic wound healing.
    UNASSIGNED: This investigation has been planned to report the influence of treatment with a mixture of both the powder of pomegranate peel (PP) accompanied with an autologous bone marrow (BM) on the cure of burn injuries in experimentally induced diabetic rabbits.
    UNASSIGNED: Alloxan monohydrate has been applied to create diabetes in 50 rabbits. Then in each rabbit, two deep second-degree burn wounds were experimentally created. The animals were then divided randomly into 5 treatment sections: non-treatment controls (C1), treated with an available commercial powder for wound (C2), treatment with powder of PP, treatment with alone BM, and the final group treated with PP powder with bone marrow (PPBM). The speed of wound closure and the histopathological changes during healing were measured. The levels of the biomarkers of rabbit platelet-derived growth factor AA (PDGF-AA) and rabbit protease-activated receptor 1 (PAR-1) were measured on days 0, 4, 8, and 12.
    UNASSIGNED: Wound healing was markedly more rapid in all the treatment groups versus the control non-treated group. Interestingly, a rapid wound cure was significantly observed in the PPBM group versus the other treatment ones. The histological assessment clarified a significant elevation in the fibroblast and collagen scores in the PPBM group versus the other sections. In addition, there were significant increases in the serum levels of the biomarkers PDGF-AA and PAR-1 among groups.
    UNASSIGNED: Dependent on the results of current research, it can be concluded that both PP powder with BM PPBM significantly accelerate the healing process of burn wounds in experimentally induced diabetic rabbits.
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  • 文章类型: Journal Article
    背景:这项前瞻性多中心研究评估了姑息治疗单位(PCU)干预的影响(实验组,EG),自体造血干细胞移植(AHSCT)对生活质量(QoL),与标准做法相比,症状控制和医疗保健资源使用(控制组,CG)。我们在第0天(干细胞输注)使用了经过验证的量表,+7(骨髓发育不全,急性症状)和+21(再生障碍恢复)。
    结果:在40例患者中(20例EG/20CG:45%/25%女性,中位年龄57.5/59),QoL在第+7天(EG:中位数0.50;CG:-63.00;p<0.001)和第+21天(EG:-2.00;CG:-129.00;p<0.001)显著不同。在第0天,平均FACT-BMT评分为CG/EG:131/89.35,反映了PCU在EG患者中的移植前干预。对于疼痛(EG中位数0.00,CG中位数2.50;p=0.01),45%的EG患者在第0天使用阿片类药物(平均38.5mg吗啡/天/患者)。减少疼痛控制影响营养支持(肠外营养45%CG,5%EG;p=0.08)。CG的住院时间更长(中位数18.5;EG中位数13.00;p<0.001)。尽管随访时间短,样本量小,PCU和HD合作可改善急性AHSCT期间的QoL和症状管理,通过疼痛控制很明显,镇痛管理,肠外营养需求减少和住院时间缩短。
    BACKGROUND: This prospective multicentre study evaluates the impact of Palliative Care Unit (PCU) intervention (Experimental Group, EG), during autologous hematopoietic stem cell transplantation (AHSCT) on quality of life (QoL), symptom control and healthcare resource use compared to standard practice (Control Group, CG). We used validated scales on Days 0 (stem cell infusion), + 7 (bone marrow aplasia, acute symptoms) and + 21 (aplasia recovery).
    RESULTS: In 40 patients (20 EG/ 20 CG: 45%/25% female, median age 57.5/59), QoL differed significantly at Day + 7 (EG: median 0.50; CG: -63.00; p < 0.001) and Day + 21 (EG: -2.00; CG: -129.00; p < 0.001). On Day 0, mean FACT-BMT scores were CG/EG: 131/ 89.35, reflecting the pre-transplant intervention of the PCU in EG patients. For pain (EG median 0.00, CG median 2.50; p = 0.01), 45% EG patients used opioids on day 0 (mean 38.5 mg morphine/day/patient). Reduced pain control impacted nutritional support (parenteral nutrition 45% CG, 5% EG; p = 0.08). Hospitalisation duration was longer in CG (median 18.5; EG median 13.00; p < 0.001). Despite the short follow-up and small sample size, PCU and HD collaboration improves QoL and symptom management during acute AHSCT, evident through pain control, analgesia management, reduced parenteral nutrition need and shorter hospital stays.
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  • 文章类型: Journal Article
    目的:MASCC/ISOO临床实践声明(CPS)旨在为临床医生提供简洁的工具,它集中了癌症患者口腔并发症管理所需的实用信息。该CPS的重点是慢性移植物抗宿主病(cGVHD)的口腔表现的管理。
    方法:本CPS是在对文献进行批判性评估的基础上开发的,随后是一组领先专家的结构化讨论。MASCC/ISOO口腔护理研究小组的成员。该信息以简洁的项目符号和表格的形式呈现,以生成有关最佳护理标准的简短手册。
    结果:口服cGVHD的治疗目标是缓解疼痛和口干症,改善口腔功能,防止继发感染,防止牙列恶化,并尽早发现恶性转化。口腔粘膜病变的预防和治疗措施,唾液腺功能减退,详细介绍了口腔和口周组织的硬皮病变化,以及这些干预措施可能的并发症和副作用。
    结论:异基因造血细胞移植后的患者,在口腔和口周组织中表现出cGVHD,应定期监测并根据需要由口腔护理从业者进行治疗。这种CPS为临床医生提供了实用的检查工具,预防,并治疗可能影响这些患者口腔的各种后遗症。
    OBJECTIVE: A MASCC/ISOO Clinical Practice Statement (CPS) is aimed at generating a concise tool for clinicians, which concentrates practical information needed for the management of oral complications of cancer patients. This CPS is focused on the management of oral manifestations of chronic graft-versus-host-disease (cGVHD).
    METHODS: This CPS was developed based on critical evaluation of the literature followed by a structured discussion of a group of leading experts, members of the Oral Care Study Group of MASCC/ISOO. The information is presented in the form of succinct bullets and table to generate a short manual about the best standard of care.
    RESULTS: The treatment goals in oral cGVHD are to relieve pain and xerostomia, improve oral function, prevent secondary infection, prevent deterioration of the dentition, and detect malignant transformation as early as possible. The prevention and treatment measures for oral mucosal lesions, hypofunction of the salivary glands, and sclerodermatous changes in the oral and perioral tissues are detailed, as well as the possible complications and side effects of these interventions.
    CONCLUSIONS: Patients post allogeneic hematopoietic cell transplantations, with cGVHD manifest in the oral and perioral tissues, should be regularly monitored and treated as needed by an oral care practitioner. This CPS provides the clinician with practical tools for examining, preventing, and treating the various sequalae that may affect the oral cavity in these patients.
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  • 文章类型: Journal Article
    造血干细胞移植(HSCT)是几种恶性和非恶性血液学疾病的潜在治愈疗法。然而,接受HSCT的患者发生严重心血管事件的风险增加.心血管风险是否因使用的移植策略类型而异,同种异体与自体HSCT,是未知的。利用全国住院患者样本(2016-2019年),我们通过HSCT模式(同种异体vs自体)评估早期心血管事件的发生率.主要结果是房颤(AF)的发生率。次要结果是任何主要不良心脏事件(MACE)的发生,定义为急性心力衰竭,心肌梗死(MI),有症状的心房或室性心律失常或心脏传导阻滞,心血管死亡。比较了接受同种异体和自体HSCT的患者的结果。多变量回归,调整心血管和癌症相关因素,用于定义HSCT前因素与MACE之间的关联。我们通过计算校正比值比(aOR)以及相应的95%置信区间(CI)和p值,进一步评估了急性心血管事件对住院患者死亡率的影响。总的来说,确定了64,705例HSCT加权住院治疗,其中22,655(35.0%)为同种异体HSCT,42,050(65.0%)为自体HSCT。房颤的患病率为9.1%,和12.1%的心律失常。在多变量回归中,同种异体HSCT与HSCT周围急性心力衰竭的校正几率较高相关(aOR2.64;1.86-3.76;p<0.0001),QT延长(aOR1.40;1.04-1.88;p=0.025),MI(aOR2.87;1.16-7.11;p=0.023),任何主要心血管并发症(aOR1.16;1.03-1.32;p=0.016),和住院死亡率(aOR4.87;3.60-6.58;p<0.0001)。脑血管事件后,AF是死亡率的最强预测因子。在接受HSCT的患者中,同种异体HSCT与院内心血管并发症的几率更高相关。
    Hematopoietic stem cell transplantation (HSCT) is a potentially curative therapy for several malignant and non-malignant hematologic conditions. However, patients undergoing HSCT are at increased risk of developing serious cardiovascular events. Whether cardiovascular risks differ by the type of transplantation strategy used, allogeneic versus autologous HSCT, is unknown. Leveraging the National Inpatient Sample (2016-2019), we assessed the incidence of early cardiovascular events by HSCT mode (allogeneic vs autologous). The primary outcome was the incidence of atrial fibrillation (AF). The secondary outcome was the occurrence of any major adverse cardiac events (MACE), defined as acute heart failure, myocardial infarction (MI), symptomatic atrial or ventricular arrhythmia or heart block, and cardiovascular death. Outcomes were compared between those undergoing allogeneic versus autologous HSCT. Multivariable regression, adjusting for cardiovascular and cancer-related factors, was used to define the association between pre-HSCT factors and MACE. We further assessed the effect of acute cardiovascular events on in-patient mortality by calculating adjusted odds ratio (aOR) with corresponding 95% confidence intervals (CI) and p-values. Overall, 64,705 weighted hospitalizations for HSCT were identified, of which 22,655 (35.0%) were allogeneic HSCT and 42,050 (65.0%) were autologous HSCT. The prevalence of AF was 9.1%, and 12.1% for any arrhythmia. In multivariable regression, allogeneic HSCT was associated with higher adjusted odds of peri-HSCT acute heart failure (aOR 2.64; 1.86-3.76; p < 0.0001), QT prolongation (aOR 1.40; 1.04-1.88; p = 0.025), MI (aOR 2.87; 1.16-7.11; p = 0.023), any major cardiovascular complication (aOR 1.16; 1.03-1.32; p = 0.016), and inpatient mortality (aOR 4.87; 3.60-6.58; p < 0.0001). Following cerebrovascular events, AF was the strongest predictor of mortality. Allogeneic HSCT was associated with higher odds of in-hospital cardiovascular complications among patients undergoing HSCT.
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  • 文章类型: Case Reports
    供体淋巴细胞输注(DLIs)通常是异基因造血干细胞移植后的推荐产品,以增加移植物抗白血病的效果。据报道,在移植后复发的慢性粒细胞白血病中,DLI的成功率更高。无论DLI的指示是什么,与DLI输注后相关的死亡率为5%-20%,超过三分之一的患者在DLI后将发展为急性和/或慢性移植物抗宿主病(GVHD)。我们报告了两例移植后使用DLIs治疗残留病的病例。两个DLI都平淡无奇。输注后患者均未出现GVHD征象。虽然两个病人都因不同的原因而过期,均与DLI输注无关。来自已发表文献的信息表明,DLI应在复发后早期施用,或作为接受T细胞耗尽移植物的患者的预防策略。侵袭性疾病患者在DLI之前可能会从疾病减少中获益。然而,需要进一步的证据来评估其疗效,尤其是复发或残留的血液恶性肿瘤。
    Donor lymphocyte infusions (DLIs) are often recommended products after allogeneic hematopoietic stem cell transplant to increase graft - versus - leukemia effect. More success rate of DLI has been reported in relapsed posttransplant chronic myeloid leukemia. Whatever the indication for DLI, mortality related to post-DLI infusion is 5%-20%, and more than one-third of patients will develop acute and/or chronic graft versus host disease (GVHD) after DLI. We report two cases where DLIs were used for residual disease after posttransplant. Both of DLI went uneventful. None of the patient\'s developed signs of GVHD postinfusion. Although both patients expired with different causes, none were related to DLI infusion. Information from published literature suggests that DLI should be administered early after relapse or as a prophylactic strategy in patients receiving T-cell-depleted grafts, and patients with aggressive diseases may benefit from disease reduction before DLI. However, further evidence is required to evaluate its efficacy, especially in relapsed or residual hematological malignancies.
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  • 文章类型: Journal Article
    慢性移植物抗宿主病(cGvHD)是异基因造血干细胞移植(allo-HSCT)的严重晚期并发症。
    此多中心分析确定了cGvHD和晚期急性GvHD(laGvHD)的累积发生率(CI)及其对移植相关死亡率(TRM)的影响,复发(R),317例患者的总生存率(OS)[296例成人,21名儿科(<12岁)]在2017年接受了首次allo-HSCT。
    成人laGvHD的TheCI为10.5%,儿科为4.8%,在研究结束时,所有成人移植患者的cGvHDCI为43.0%,在成人高危队列中为50.2%.在42.0%的患者中,cGvHD的发作是从头的,静止在52.1%,进步5.9%。在成年人中,预防性使用抗胸腺细胞球蛋白或移植后环磷酰胺与cGvHD的发生率显着降低(28.7%)。钙调磷酸酶抑制剂(30.6%)和甲氨蝶呤/霉酚酸酯(58.4%)的标准预防(均p<0.01)。aGvHD患者的TRM明显高于(31.8%)。cGvHD(12.6%)和无GvHD(6.3%)(所有p=0.0001)。cGvHD患者的成人高危队列中的OS明显高于(78.9%)。由于复发率显着降低(cGvHD:14.5%;无cGvHD:27.2%;p=0.00016,HR0.41),无(66.2%;p=0.0022;HR0.48)。轻度(80.0%)和中度(79.2%)cGvHD患者的OS也显著高于没有cGvHD(66.2%),不包括重度cGvHD(72.7%)(所有p=0.0214)。
    严重cGvHD对OS的负面影响表明,有必要重点预防严重形式,以提高生存率和生活质量。
    UNASSIGNED: Chronic graft-versus-host disease (cGvHD) is a serious late complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT).
    UNASSIGNED: This multicenter analysis determined the cumulative incidence (CI) of cGvHD and late acute GvHD (laGvHD) and its impact on transplantation-related mortality (TRM), relapse (R), and overall survival (OS) in 317 patients [296 adults, 21 pediatrics (<12 years of age)] who underwent their first allo-HSCT in 2017.
    UNASSIGNED: The CI of laGvHD was 10.5% in adults and 4.8% in pediatrics, and the CI of cGvHD was 43.0% in all adult transplant patients and 50.2% in the adult at-risk cohort at the study end. The onset of cGvHD was de novo in 42.0% of patients, quiescent in 52.1%, and progressive in 5.9%. In adults, prophylactic use of antithymocyte globulin or posttransplant cyclophosphamide was associated with a significantly lower incidence of cGvHD (28.7%) vs. standard prophylaxis with calcineurin inhibitors (30.6%) and methotrexate/mycophenolate mofetil (58.4%) (all p < 0.01). TRM was significantly higher in patients with aGvHD (31.8%) vs. cGvHD (12.6%) and no GvHD (6.3%) (all p = 0.0001). OS in the adult at-risk cohort was significantly higher in patients with cGvHD (78.9%) vs. without (66.2%; p = 0.0022; HR 0.48) due to a significantly lower relapse rate (cGvHD: 14.5%; without cGvHD: 27.2%; p = 0.00016, HR 0.41). OS was also significantly higher in patients with mild (80.0%) and moderate (79.2%) cGvHD vs. without cGvHD (66.2%), excluding severe cGvHD (72.7%) (all p = 0.0214).
    UNASSIGNED: The negative impact of severe cGvHD on OS suggests a focus on prevention of severe forms is warranted to improve survival and quality of life.
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  • 文章类型: Case Reports
    背景技术范可尼贫血(FA)是一种遗传性疾病,其损害骨髓功能并使个体易患再生障碍性贫血。这种情况是由负责DNA修复的基因突变引起的。患有FA的人由于DNA损伤而发生肿瘤的风险增加。头部扁平细胞癌,脖子,食道,和生殖器官经常在患有FA的个体中观察到。病例报告由于CEA标记物水平升高,一名31岁的Fanconi贫血并有骨髓移植史的男子被送入普外科。移植前,染色体异常,骨髓发育不全,肾脏发育不全,并注意到骨缺损。移植后,他出现了皮疹。他还被诊断出患有唇鳞状细胞癌和慢性病,包括胆汁淤积性肝损伤,高血压,和甲状腺功能减退。在诊断过程中,计算机断层扫描显示巴雷特食管的迹象,胃和肠中有许多息肉,右侧髂区结节形成4.5×5×5.5cm。腹腔镜检查显示阑尾肿瘤在腹壁和网膜上有许多转移。组织学分析证实粘液性阑尾癌。患者在肿瘤中心接受姑息治疗,最终诊断为阑尾癌,黏液型,G3级。该病例强调了对FA患者进行早期和全面癌症筛查的重要性,特别是那些有骨髓移植史的人。结论该临床病例强调了对具有这种遗传病理学的个体进行彻底和及时的癌症诊断的至关重要性。
    BACKGROUND Fanconi anemia (FA) is a genetic disorder that impairs the function of the bone marrow and predisposes individuals to aplastic anemia. The condition is caused by mutations in genes responsible for DNA repair. People with FA have an increased risk of developing tumors due to DNA damage. Flat-cell carcinomas of the head, neck, esophagus, and genital organs are often observed in individuals with FA. CASE REPORT A 31-year-old man with Fanconi anemia and a history of bone marrow transplantation was admitted to the General Surgery Department due to elevated levels of the CEA marker. Before the transplantation, chromosomal anomalies, bone marrow hypoplasia, kidney agenesis, and bone defects were noted. After the transplantation, he developed a skin rash. He was also diagnosed with squamous cell carcinoma of the lip and chronic conditions, including cholestatic liver damage, hypertension, and hypothyroidism. During the diagnostic process, computed tomography showed signs of Barrett\'s esophagus, numerous polyps in the stomach and intestines, and a nodular formation measuring 4.5×5×5.5 cm in the right iliac region. Laparoscopy revealed a neoplasm of the appendix with numerous metastases on the inner abdominal wall and omentum. Histological analysis confirmed mucinous appendiceal cancer. The patient was discharged for palliative treatment at the Oncology Center with a final diagnosis of appendiceal cancer, mucinous type, grade G3. This case underscores the importance of early and comprehensive cancer screening in individuals with FA, particularly those with a history of bone marrow transplantation. CONCLUSIONS This clinical case underscores the critical importance of thorough and timely cancer diagnosis in individuals with this genetic pathology.
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