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Long-Term Cardiac Function Remains Stable to Improved Following Curative Allogeneic Hematopoietic Cell Transplantation for Pediatric Sickle Cell Disease: A STAR Analysis 儿童镰状细胞病同种异体造血细胞移植治疗后,长期心脏功能保持稳定并得到改善:一项STAR分析
IF 4.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-01 DOI: 10.1016/j.jtct.2025.12.091
Samaher Sukkar MD , Katie Liu MS , Scott Gillespie MS , Tami D. John MD , Allistair Abraham MD , Rikin K Shah MD , Deepak Chellapandian MD MBBS , Monica Bhatia MD , Christine Camacho-Bydume MD , Sonali Chaudhury MD, MBBS , Michael J Eckrich MD MPH , Gregory MT Guilcher MD , Dr. Jennifer J. Jaroscak MD , Kimberly A. Kasow DO , Alexander Ngwube MD , Hemalatha G Rangarajan MD , John Horan MD, MPH , Lakshmanan Krishnamurti MD , Shalini Shenoy MD , Niti Dham MD , Elizabeth O. Stenger MD MSc
Cardiac dysfunction causes morbidity and mortality in sickle cell disease (SCD). Allogeneic hematopoietic cell transplantation (HCT) is the most common curative option, yet its impact on long-term cardiac function is not well defined in multicenter cohorts. After our Sickle cell Transplant Advocacy and Research (STAR) retrospective registry analysis on long-term organ function, we sought to complete more detailed cardiac function analysis and determine predictors of dysfunction.
This retrospective cohort study of pediatric patients (pts) with SCD post-HCT with pre- and post-HCT echocardiograms (echo) in the STAR registry (Figure 1). Additional echo data was extracted from source documents and linked to existing data. Cardiac dysfunction was defined as ejection fraction (EF) <55% or shortening fraction (SF) <28%. Data were summarized as median (IQR) or N (%). Linear mixed effect models were used to compare pre- to post-HCT data, accounting for within-pt clustering. Associations between risk factors and cardiac dysfunction were assessed using Firth logistic regression.
Within 228 pts (Table), age at HCT was 8.8 years (yrs), and most pts received matched related donor (MRD; 75%) bone marrow (76%) after myeloablative conditioning (MAC; 76%). Engraftment was prompt, with most pts remaining alive and free from severe graft-versus-host disease (GVHD) or rejection. At 3.4 yrs (2.2, 6.3) post-HCT, while EF significantly decreased, SF was stable (Figure 2A). Left ventricular (LV) size and function significantly decreased (Figures 2B-C). Mitral and tricuspid valve (MV, TV) flow significantly decreased (Figure 2D), as did tricuspid regurgitant jet velocity (TRJV; Figure 2A). While most (93%) remained free of cardiac dysfunction, significantly more pts had low EF (1.6%→6.7%, p=0.008) or SF (0.9%→4.1%, p=0.022) post-HCT. Controlling for MAC and cytoxan (Cy) dose, male sex was significantly associated with post-HCT cardiac dysfunction and age <6 yrs was protective.
In pediatric SCD, HCT is associated with favorable reverse remodeling and reduced pulmonary pressures, but with a modest EF decrease. Early HCT appears protective and male sex increases risk. Longer follow-up is needed to clarify durability and clinical significance of these findings. Additional analyses of predictors for diastolic dysfunction are planned.
心功能障碍导致镰状细胞病(SCD)的发病率和死亡率。同种异体造血细胞移植(HCT)是最常见的治疗选择,但其对长期心功能的影响在多中心队列中尚未得到很好的定义。在我们的镰状细胞移植倡导和研究(STAR)对长期器官功能进行回顾性登记分析后,我们试图完成更详细的心功能分析并确定功能障碍的预测因素。这项回顾性队列研究在STAR注册表中对hct前后超声心动图(echo)的SCD患儿(pts)进行了研究(图1)。从源文档中提取额外的回波数据并链接到现有数据。心功能障碍定义为射血分数(EF) <;55%或缩短分数(SF) <28%。数据汇总为中位数(IQR)或N(%)。线性混合效应模型用于比较hct前后的数据,考虑到pt内聚类。使用Firth logistic回归评估危险因素与心功能障碍之间的关联。228例患者(表),HCT时的年龄为8.8岁,大多数患者在清髓调节(MAC, 76%)后接受了匹配的相关供体(MRD, 75%)骨髓(76%)。移植迅速,大多数患者存活,没有严重的移植物抗宿主病(GVHD)或排斥反应。在hct后3.4年(2.2、6.3),虽然EF显著下降,但SF稳定(图2A)。左室(LV)大小和功能明显降低(图2B-C)。二尖瓣和三尖瓣(MV, TV)流量明显减少(图2D),三尖瓣反流射流速度也明显减少(TRJV;图2A)。虽然大多数(93%)患者没有心功能障碍,但hct后出现低EF(1.6%→6.7%,p=0.008)或SF(0.9%→4.1%,p=0.022)的患者明显更多。在控制MAC和环磷酰胺(cytoxan)剂量的情况下,男性与hct后心功能障碍显著相关,6岁具有保护作用。在儿童SCD中,HCT与有利的反向重塑和肺压降低有关,但与EF适度降低有关。早期HCT似乎具有保护作用,而男性性行为会增加风险。需要更长时间的随访来阐明这些发现的持久性和临床意义。计划进一步分析舒张功能障碍的预测因素。
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引用次数: 0
Officers and Directors of ASTCT ASTCT的官员和董事
IF 4.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-01 DOI: 10.1016/S2666-6367(25)02660-0
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引用次数: 0
Mining Safety Signals of Letermovir Drug Combinations: Real-World Pharmacovigilance Analysis 挖掘利特莫韦药物组合的安全信号:现实世界的药物警戒分析。
IF 4.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-01 DOI: 10.1016/j.jtct.2025.09.043
Jingjing Lan , Yulan Qiu , Mengmeng Teng , Chao Meng , Dan Sun , Ziyuan Ma , Shiqi Cheng , Xiaoning Wang , Juan Ren , Siying Chen , Yalin Dong
<div><div>Letermovir (LMV) is vital for cytomegalovirus prevention in transplant recipients, but its safety and drug–drug interactions (DDI) related adverse event (AE) risks require further investigation. This study conducted a pharmacovigilance analysis to identify novel AE signals of LMV and assess DDI-related AEs with commonly co-administered drugs. AE reports from 29 quarters, from the launch of LMV to the fourth quarter of 2024, were collected from the Food and Drug Administration Adverse Event Reporting System for analysis. Two statistical models (reporting odds ratio [ROR] method and medicines and healthcare products regulatory agency) were used to identify AEs meeting the threshold, which were then compared with LMV drug labels and designated medical event (DME) risk signals. Four models were applied to detect potential AE signals related to LMV combination use, and DDI-related AEs and DMEs were analyzed in conjunction with single-drug mining results. Finally, 54 LMV AE signals were identified, of which 4 AE signals had appeared in the LMV drug labels, namely edema (ROR = 3.69), acute heart failure (ROR = 14.48), pericardial effusion (ROR = 4.77), and atrial fibrillation (ROR = 3.61). Among the remaining new AE signals, AEs with stronger ROR values included herpetic gastritis (ROR = 23,772.49), human herpesvirus 6 encephalitis (ROR = 214.17), and acute graft-versus-host disease (aGVHD) in skin (ROR = 190.2). Eight DME signals of LMV were determined, such as hepatic failure, drug-induced liver injury, renal failure, and acute kidney injury (AKI). In the DDI analysis, 279 DDI-related AE signals meeting all four model criteria were identified. Among 28 drugs commonly co-administered with LMV, immunomodulatory agents contributed the highest proportion of signals (34.4%, 96/279), with cyclosporine associated with the most AE types (10.4%, 29/279). The most frequent AEs included aplastic anemia (4.3%), esophagitis (3.9%), and renal failure (3.6%). There was a positive AE signal difference when LMV was combined with cyclosporine or tacrolimus. When used in combination with cyclosporine, in addition to cardiac events and peripheral edema listed in the LMV drug label, DME signals such as pancytopenia, febrile neutropenia, AKI, and hepatic failure may also occur; when used in combination with tacrolimus, one needs to be alert to the occurrence of aGVHD in skin/intestine. LMV use may induce cardiac AE signals such as acute heart failure, pericardial effusion, and atrial fibrillation, and may increase the risk of herpes virus reactivation. When LMV is used in combination with drugs such as cyclosporine or tacrolimus, it may exacerbate hepatic and renal toxicity. Acute heart failure and atrial fibrillation AE signals were detected when combined with cyclosporine. Future efforts should focus on the safety of LMV in clinical practice and real-world settings, with optimized medication strategies to minimize risks and enhance therapeutic outcomes.</div></
背景和目的:Letermovir (LMV)对移植受者预防巨细胞病毒至关重要,但其安全性和药物相互作用(DDI)相关的不良事件(AE)风险有待进一步研究。本研究进行了药物警戒分析,以识别LMV的新AE信号,并评估ddi与常用联合用药的AE。方法:从FDA不良事件报告系统中收集LMV上市至2024年第四季度29个季度的AE报告进行分析。采用报告优势比(ROR)法和药品和保健产品监管机构(MHRA)两种统计模型识别符合阈值的ae,并将其与LMV药品标签和指定医疗事件(DME)风险信号进行比较。应用4种模型检测与LMV联合用药相关的潜在声发射信号,并结合单药挖掘结果对ddi相关的声发射信号和声发射信号进行分析。结果:最终共识别出54个LMV AE信号,其中4个AE信号出现在LMV药物标签上,分别为水肿(ROR=3.69)、急性心力衰竭(ROR=14.48)、心包积液(ROR=4.77)和房颤(ROR=3.61)。在其余新的AE信号中,ROR值较高的AE包括疱疹性胃炎(ROR=23772.49)、人类疱疹病毒6型(HHV-6)脑炎(ROR=214.17)和皮肤急性移植物抗宿主病(aGVHD) (ROR=190.2)。测定LMV的肝衰竭、药物性肝损伤、肾功能衰竭和急性肾损伤(AKI)等8种DME信号。在DDI分析中,发现279个与DDI相关的AE信号满足所有四个模型标准。在与LMV共给药的28种药物中,免疫调节剂贡献的信号比例最高(34.4%,96/279),环孢素与AE类型相关最多(10.4%,29/279)。最常见的ae包括再生障碍性贫血(4.3%)、食管炎(3.9%)和肾功能衰竭(3.6%)。LMV与环孢素或他克莫司联用时AE信号有阳性差异。当与环孢素合用时,除了LMV药物标签中列出的心脏事件和外周水肿外,还可能出现全血细胞减少症、发热性中性粒细胞减少症、AKI和肝功能衰竭等DME信号;当与他克莫司合用时,需要警惕皮肤/肠道发生aGVHD。结论:使用LMV可诱发急性心力衰竭、心包积液、房颤等心脏AE信号,并可能增加疱疹病毒再激活的风险。当LMV与环孢素或他克莫司等药物合用时,可能会加重肝和肾毒性。联合环孢素可检测急性心力衰竭和心房颤动AE信号。未来的工作应侧重于LMV在临床实践和现实环境中的安全性,优化用药策略以最大限度地降低风险并提高治疗效果。
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引用次数: 0
National Landscape of Logistical and Nonmedical Requirements for Transplantation and Cellular Therapy 移植和细胞治疗的后勤和非医疗需求的国家景观。
IF 4.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-01 DOI: 10.1016/j.jtct.2025.10.031
Hannah R. Abrams , Helene Starks , Lindsey Bandini , Terry Harrington , Mary-Elizabeth M. Percival , Roland B. Walter , Kathryn Russell , Raya Mawad , Jacob Appelbaum , Mohamed L. Sorror , Anna B. Halpern

Background

New disease indications and supportive care advances have expanded the populations who may benefit from cellular therapies, including autologous or allogeneic hematopoietic cell transplant (HCT) and/or chimeric antigen receptor T-cell therapy (CAR T). However, nonmedical requirements for these therapies, such as caregiver and housing availability, may limit access. We sought a detailed national assessment of nonmedical requirements for HCT and CAR T-cell therapy to describe and quantify barriers.

Objective

Describe the national landscape and implementation of logistical and nonmedical requirements for HCT and CAR T-cell therapy.

Study Design

We developed a web-based survey of HCT/CAR T-cell therapy requirements and obtained pilot feedback from 3 centers that regularly perform HCT/CAR T-cell therapy. We distributed the survey via the NCCN Best Practices Committee, which is composed of physician, nursing, and administrative leaders from 34 member institutions. We requested survey completion by an institutional content area expert.

Results

The response rate was 91% (31/34). >80% of centers required a caregiver, local housing, and local transportation, but the number of days required, institutional supports, and acceptable distances varied widely. A subset of centers offered "all-outpatient" procedures and required more stringent logistical criteria, including autologous HCT by 13/19 centers (68%), allogeneic HCT by 4/8 centers (50%), and CAR T-cell therapy by 10/16 centers (63%). Many centers excluded patients with a history of medication nonadherence, substance use, or psychiatric comorbidity, but most did not employ formal adjudication or definition of patient eligibility. Institutions noted “soft” contraindications or case-by-case review for these patients and those experiencing homelessness, lack of insurance, or without citizenship. Overall, caregiving, housing, cost, and insurance coverage emerged as the top nonmedical barriers to HCT and CAR T-cell therapy.

Conclusions

Significant variability exists in nonmedical requirements for HCT and CAR T-cell therapy, with a lack of standard policies across institutions. We recommend that centers formally track reasons for noneligibility to identify nonmedical barriers to HCT and CAR T-cell therapy and develop targeted interventions to reduce the number of patients who are excluded for nonmedical reasons.
背景:新的疾病适应症和支持性护理的进展扩大了可能受益于细胞治疗的人群,包括自体或异体造血细胞移植(HCT)和/或嵌合抗原受体T细胞治疗(CAR - T)。然而,这些疗法的非医疗要求,如护理人员和住房供应,可能会限制获得治疗。我们寻求对HCT和CAR - t细胞治疗的非医疗需求进行详细的国家评估,以描述和量化障碍。目的:描述HCT和CAR - t细胞治疗的后勤和非医疗要求的国家概况和实施情况。研究设计:我们开发了一项基于网络的HCT/CAR - t细胞治疗需求调查,并从三个定期进行HCT/CAR - t细胞治疗的中心获得试点反馈。我们通过NCCN最佳实践委员会分发调查,该委员会由来自34个成员机构的医生、护理和行政领导组成。我们要求由机构内容领域专家完成调查。结果:有效率为91%(31/34)。>80%的中心需要一名护理人员、当地的住房和当地的交通工具,但所需的天数、机构支持和可接受的距离差异很大。一部分中心提供“全门诊”程序,需要更严格的后勤标准,包括13/19中心的自体HCT(68%), 4/8中心的同种异体HCT(50%), 10/16中心的CAR - t细胞治疗(63%)。许多中心排除了有药物依从史、药物使用史或精神合并症的患者,但大多数中心没有对患者资格进行正式的裁决或定义。机构注意到这些患者和那些无家可归、缺乏保险或没有公民身份的患者的“软”禁忌症或逐案审查。总的来说,护理、住房、费用和保险是HCT和CAR - t细胞治疗的主要非医疗障碍。结论:HCT和CAR - t细胞治疗的非医疗需求存在显著差异,各机构缺乏标准政策。我们建议各中心正式跟踪不符合资格的原因,以确定HCT和CAR - t细胞治疗的非医疗障碍,并制定有针对性的干预措施,以减少因非医疗原因而被排除在外的患者数量。
{"title":"National Landscape of Logistical and Nonmedical Requirements for Transplantation and Cellular Therapy","authors":"Hannah R. Abrams ,&nbsp;Helene Starks ,&nbsp;Lindsey Bandini ,&nbsp;Terry Harrington ,&nbsp;Mary-Elizabeth M. Percival ,&nbsp;Roland B. Walter ,&nbsp;Kathryn Russell ,&nbsp;Raya Mawad ,&nbsp;Jacob Appelbaum ,&nbsp;Mohamed L. Sorror ,&nbsp;Anna B. Halpern","doi":"10.1016/j.jtct.2025.10.031","DOIUrl":"10.1016/j.jtct.2025.10.031","url":null,"abstract":"<div><h3>Background</h3><div>New disease indications and supportive care advances have expanded the populations who may benefit from cellular therapies, including autologous or allogeneic hematopoietic cell transplant (HCT) and/or chimeric antigen receptor T-cell therapy (CAR T). However, nonmedical requirements for these therapies, such as caregiver and housing availability, may limit access. We sought a detailed national assessment of nonmedical requirements for HCT and CAR T-cell therapy to describe and quantify barriers.</div></div><div><h3>Objective</h3><div>Describe the national landscape and implementation of logistical and nonmedical requirements for HCT and CAR T-cell therapy.</div></div><div><h3>Study Design</h3><div>We developed a web-based survey of HCT/CAR T-cell therapy requirements and obtained pilot feedback from 3 centers that regularly perform HCT/CAR T-cell therapy. We distributed the survey via the NCCN Best Practices Committee, which is composed of physician, nursing, and administrative leaders from 34 member institutions. We requested survey completion by an institutional content area expert.</div></div><div><h3>Results</h3><div>The response rate was 91% (31/34). &gt;80% of centers required a caregiver, local housing, and local transportation, but the number of days required, institutional supports, and acceptable distances varied widely. A subset of centers offered \"all-outpatient\" procedures and required more stringent logistical criteria, including autologous HCT by 13/19 centers (68%), allogeneic HCT by 4/8 centers (50%), and CAR T-cell therapy by 10/16 centers (63%). Many centers excluded patients with a history of medication nonadherence, substance use, or psychiatric comorbidity, but most did not employ formal adjudication or definition of patient eligibility. Institutions noted “soft” contraindications or case-by-case review for these patients and those experiencing homelessness, lack of insurance, or without citizenship. Overall, caregiving, housing, cost, and insurance coverage emerged as the top nonmedical barriers to HCT and CAR T-cell therapy.</div></div><div><h3>Conclusions</h3><div>Significant variability exists in nonmedical requirements for HCT and CAR T-cell therapy, with a lack of standard policies across institutions. We recommend that centers formally track reasons for noneligibility to identify nonmedical barriers to HCT and CAR T-cell therapy and develop targeted interventions to reduce the number of patients who are excluded for nonmedical reasons.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"32 2","pages":"Pages 209.e1-209.e13"},"PeriodicalIF":4.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145453222","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
Effect of Inflammatory Bowel Disease on Clinical Outcomes of Hematopoietic Cell Transplantation for Inborn Errors of Immunity 炎性肠病对先天性免疫缺陷患者造血细胞移植临床疗效的影响。
IF 4.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-01 DOI: 10.1016/j.jtct.2025.11.006
Ryu Yanagisawa , Satoshi Miyamoto , Yoji Sasahara , Michiko Kajiwara , Masataka Ishimura , Hirotoshi Sakaguchi , Takehiko Doi , Yoshiyuki Takahashi , Yuko Cho , Maho Sato , Saori Katayama , Katsuyoshi Koh , Masakatsu Yanagimachi , Daiichiro Hasegawa , Keiko Okada , Junko Takita , Shoji Saito , Atsushi Sato , Moeko Hino , Kimikazu Matsumoto , Katsutsugu Umeda
Inflammatory bowel disease (IBD) is a phenotype of patients with inborn errors of immunity (IEI). Allogeneic hematopoietic cell transplantation (HCT) is an established curative treatment for IEI; however, the effect of IBD on HCT outcomes is unclear. We evaluated the impact of IBD on post-HCT prognosis in pediatric patients with IEI. We used the Japanese transplant registry database to retrospectively analyze the medical records of 625 patients with pediatric IEI who underwent allogeneic HCT between 2007 and 2022 to evaluate the clinical impact of IBD on these patients. Sixty-six (10.6%) patients had concurrent IBD before HCT. Compared with patients without IBD, those with IBD did not show inferior overall survival, transplant-related mortality, or neutrophil and platelet engraftment. Moreover, no significant differences were observed in the incidence of acute or chronic graft-versus-host disease (GVHD) between the two groups. However, the IBD group had a higher incidence of acute gastrointestinal GVHD. These findings remained consistent even after propensity score matching, accounting for the identified risk factors, including age at HCT, performance status, total HCT-specific comorbidity index score, HLA mismatch, era of HCT, and underlying disease classification. IBD has a limited effect on HCT outcomes in patients with pediatric IEI, despite the higher incidence of acute gastrointestinal GVHD. Nevertheless, in this study, we included patients with heterogeneous and complex disease backgrounds, indicating the need for further investigation to confirm the findings.
背景:炎症性肠病(IBD)是先天性免疫缺陷(IEI)患者的一种表型。同种异体造血细胞移植(HCT)是治疗IEI的有效方法;然而,IBD对HCT结果的影响尚不清楚。目的:我们评估IBD对IEI患儿hct后预后的影响。研究设计:我们使用日本移植登记数据库,回顾性分析2007年至2022年间625例接受同种异体HCT的儿童IEI患者的医疗记录,以评估IBD对这些患者的临床影响。结果:66例(10.6%)患者在HCT前并发IBD。与没有IBD的患者相比,IBD患者没有表现出较差的总生存率、移植相关死亡率(TRM)或中性粒细胞和血小板植入。此外,两组间急性或慢性移植物抗宿主病(GVHD)的发生率无显著差异。然而,IBD组急性胃肠道GVHD的发生率更高。即使在倾向评分匹配后,这些发现仍然一致,考虑到确定的危险因素,包括HCT年龄、运动状态、HCT特异性共病指数总分、HLA错配、HCT时代和潜在疾病分类。结论:尽管急性胃肠道GVHD的发生率较高,但IBD对儿童IEI患者HCT结果的影响有限。然而,在这项研究中,我们纳入了异质性和复杂疾病背景的患者,这表明需要进一步的调查来证实这些发现。
{"title":"Effect of Inflammatory Bowel Disease on Clinical Outcomes of Hematopoietic Cell Transplantation for Inborn Errors of Immunity","authors":"Ryu Yanagisawa ,&nbsp;Satoshi Miyamoto ,&nbsp;Yoji Sasahara ,&nbsp;Michiko Kajiwara ,&nbsp;Masataka Ishimura ,&nbsp;Hirotoshi Sakaguchi ,&nbsp;Takehiko Doi ,&nbsp;Yoshiyuki Takahashi ,&nbsp;Yuko Cho ,&nbsp;Maho Sato ,&nbsp;Saori Katayama ,&nbsp;Katsuyoshi Koh ,&nbsp;Masakatsu Yanagimachi ,&nbsp;Daiichiro Hasegawa ,&nbsp;Keiko Okada ,&nbsp;Junko Takita ,&nbsp;Shoji Saito ,&nbsp;Atsushi Sato ,&nbsp;Moeko Hino ,&nbsp;Kimikazu Matsumoto ,&nbsp;Katsutsugu Umeda","doi":"10.1016/j.jtct.2025.11.006","DOIUrl":"10.1016/j.jtct.2025.11.006","url":null,"abstract":"<div><div>Inflammatory bowel disease (IBD) is a phenotype of patients with inborn errors of immunity (IEI). Allogeneic hematopoietic cell transplantation (HCT) is an established curative treatment for IEI; however, the effect of IBD on HCT outcomes is unclear. We evaluated the impact of IBD on post-HCT prognosis in pediatric patients with IEI. We used the Japanese transplant registry database to retrospectively analyze the medical records of 625 patients with pediatric IEI who underwent allogeneic HCT between 2007 and 2022 to evaluate the clinical impact of IBD on these patients. Sixty-six (10.6%) patients had concurrent IBD before HCT. Compared with patients without IBD, those with IBD did not show inferior overall survival, transplant-related mortality, or neutrophil and platelet engraftment. Moreover, no significant differences were observed in the incidence of acute or chronic graft-versus-host disease (GVHD) between the two groups. However, the IBD group had a higher incidence of acute gastrointestinal GVHD. These findings remained consistent even after propensity score matching, accounting for the identified risk factors, including age at HCT, performance status, total HCT-specific comorbidity index score, HLA mismatch, era of HCT, and underlying disease classification. IBD has a limited effect on HCT outcomes in patients with pediatric IEI, despite the higher incidence of acute gastrointestinal GVHD. Nevertheless, in this study, we included patients with heterogeneous and complex disease backgrounds, indicating the need for further investigation to confirm the findings.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"32 2","pages":"Pages 203.e1-203.e11"},"PeriodicalIF":4.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145507474","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
Real-World Outcome of Defibrotide Treatment for Severe Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Cell Transplantation 去纤维肽治疗造血细胞移植后严重肝脏VOD/SOS的实际治疗效果。
IF 4.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-01 DOI: 10.1016/j.jtct.2025.10.003
Jae-Ho Yoon, Daehun Kwag, Gi June Min, Sung-Soo Park, Silvia Park, Sung-Eun Lee, Byung-Sik Cho, Ki-Seong Eom, Yoo-Jin Kim, Hee-Je Kim, Chang-Ki Min, Seok-Goo Cho
Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a serious complication following hematopoietic cell transplantation (HCT), with a markedly poor prognosis in patients with severe or very severe presentations. Defibrotide (DF) is the sole approved treatment for severe to very severe VOD/SOS, but real-world evidence remains limited, particularly in East Asian populations. We retrospectively analyzed 73 adult patients with severe or very severe VOD/SOS treated with DF between 2016 and 2023 at a single tertiary center in Korea. Diagnosis and grading were based on the revised European Society for Blood and Marrow Transplantation criteria. During the study period, national reimbursement guidelines for DF evolved from requiring ≥4 of 6 severity criteria to ≥2 of 5 severity criteria, allowing earlier initiation of treatment. The median time from HCT to diagnosis of VOD/SOS was 24 days (range, 1 to 843 days), and DF was administered within a median of 1 day after diagnosis. At DF initiation, 40 patients were classified as severe and 33 were classified as very severe; disease progression resulted in 53 patients reaching the very severe category. The overall response rate was 46.5%, and complete resolution was achieved in 39.7% of cases. The 100-day overall survival was 34.2%, significantly higher in the patients receiving DF within 2 days of diagnosis (36.0% versus 17.4%; P = .049). High total bilirubin levels at diagnosis and at the worst disease period were strongly associated with poor long-term survival regardless of severity criteria. These data provide real-world support for the early use of DF and suggest the prognostic utility of total bilirubin level in guiding treatment for VOD/SOS following HCT.
肝静脉闭塞性疾病/静脉窦阻塞综合征(VOD/SOS)是造血细胞移植(HCT)后的严重并发症,严重或极严重的患者预后明显差。去纤肽(DF)是唯一被批准的治疗严重至极严重VOD/SOS的方法,但现实证据仍然有限,特别是在东亚人群中。我们回顾性分析了2016年至2023年在韩国一个三级医疗中心接受DF治疗的73例严重或极严重VOD/SOS成年患者。诊断和分级基于修订后的EBMT标准。在研究期间,国家DF报销指南从要求6项严重性标准中的≥4项发展到要求5项严重性标准中的≥2项,从而允许更早开始治疗。从HCT到VOD/SOS诊断的中位时间为24天(范围从D-1到D+843), DF在诊断后的中位时间为1天。在DF开始时,40例患者被分类为严重,33例为非常严重;疾病进展导致53名患者达到非常严重的级别。总有效率为46.5%,完全缓解率为39.7%。100天总生存率(OS)为34.2%,在诊断2天内接受DF的患者中显着更高(36.0%比17.4%,p=0.049)。无论严重程度标准如何,诊断时和疾病最严重时期的高总胆红素水平与较差的长期生存率密切相关。这些数据为早期使用DF提供了现实支持,并提示胆红素水平在指导HCT后VOD/SOS治疗中的预后效用。
{"title":"Real-World Outcome of Defibrotide Treatment for Severe Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Cell Transplantation","authors":"Jae-Ho Yoon,&nbsp;Daehun Kwag,&nbsp;Gi June Min,&nbsp;Sung-Soo Park,&nbsp;Silvia Park,&nbsp;Sung-Eun Lee,&nbsp;Byung-Sik Cho,&nbsp;Ki-Seong Eom,&nbsp;Yoo-Jin Kim,&nbsp;Hee-Je Kim,&nbsp;Chang-Ki Min,&nbsp;Seok-Goo Cho","doi":"10.1016/j.jtct.2025.10.003","DOIUrl":"10.1016/j.jtct.2025.10.003","url":null,"abstract":"<div><div>Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a serious complication following hematopoietic cell transplantation (HCT), with a markedly poor prognosis in patients with severe or very severe presentations. Defibrotide (DF) is the sole approved treatment for severe to very severe VOD/SOS, but real-world evidence remains limited, particularly in East Asian populations. We retrospectively analyzed 73 adult patients with severe or very severe VOD/SOS treated with DF between 2016 and 2023 at a single tertiary center in Korea. Diagnosis and grading were based on the revised European Society for Blood and Marrow Transplantation criteria. During the study period, national reimbursement guidelines for DF evolved from requiring ≥4 of 6 severity criteria to ≥2 of 5 severity criteria, allowing earlier initiation of treatment. The median time from HCT to diagnosis of VOD/SOS was 24 days (range, 1 to 843 days), and DF was administered within a median of 1 day after diagnosis. At DF initiation, 40 patients were classified as severe and 33 were classified as very severe; disease progression resulted in 53 patients reaching the very severe category. The overall response rate was 46.5%, and complete resolution was achieved in 39.7% of cases. The 100-day overall survival was 34.2%, significantly higher in the patients receiving DF within 2 days of diagnosis (36.0% versus 17.4%; <em>P</em> = .049). High total bilirubin levels at diagnosis and at the worst disease period were strongly associated with poor long-term survival regardless of severity criteria. These data provide real-world support for the early use of DF and suggest the prognostic utility of total bilirubin level in guiding treatment for VOD/SOS following HCT.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"32 2","pages":"Pages 193.e1-193.e11"},"PeriodicalIF":4.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145293863","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
Herpes Zoster Frequency and Adjuvanted Recombinant Zoster Vaccination after Allogeneic Hematopoietic Cell Transplantation 异体造血细胞移植后的带状疱疹发病率和佐剂重组带状疱疹疫苗接种。
IF 4.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-01 DOI: 10.1016/j.jtct.2025.10.028
Victoria G. Hall , Caden Chiarello , Mats Remberger , Deepali Kumar , Ivan Pasic , Dennis Dong Hwan Kim , Rajat Kumar , Auro Viswabandya , Fotios V. Michelis , Arjun Law , Armin Gerbitz , Sapna Humar , Wilson Lam , Igor Novitzky-Basso , Jonas Mattsson
<div><div>The prevention of herpes zoster (HZ) is of prime importance in patients undergoing allogeneic hematopoietic cell transplantation (alloHCT). Shingrix, an inactivated, adjuvanted recombinant zoster vaccine (aRZV), is now available and recommended for preventing HZ in seropositive immunocompromised patients. The safety and efficacy of this vaccine have been established in autologous HCT (autoHCT) recipients and other immunocompromised patients; however, data on alloHCT recipients are limited. The primary outcome of this study was a comparison of HZ frequency in alloHCT recipients who received aRZV vaccination and those who did not. Other characteristics of interest include the clinical presentation of HZ, treatment, and outcomes related to the HZ episode. This single-center retrospective observational cohort study was conducted at Princess Margaret Hospital, Toronto, Canada between April 1, 2018, and May 1, 2024. Adult patients who underwent alloHCT between April 1 2018, and December 31, 2022, were included in a prospective registry. Follow-up was censored at time of death, at 90 days after the first episode of HZ, at loss to follow-up, at the end of the study period, or 36 months post-alloHCT. Data were collected systematically from the electronic medical record or registry. A confirmed HZ episode was defined by clinical and/or molecular criteria. Statistical analysis was performed to determine risk factors associated with HZ and the relative risk (RR) of HZ episodes occurring in those ≥12 months post-alloHCT considering aRZV status and the cumulative follow-up period. A total of 445 patients were included. The median age was 56 years (interquartile range [IQR], 38 to 64 years), with a slight male predominance (n = 241/445; 54.2%). From ≥12 months post-alloHCT, there were 43 HZ episodes, including 26 of 263 patients (9.9%) who had received at least 1 dose of aRZV, 19 of 263 (7.2%) who had received 2 doses, 7 of 70 (10.0%) who did not receive RZV, and 10 of 112 (8.9%) with unknown vaccination status. The median time to HZ after receipt of any dose of aRZV was 8.1 months (IQR, 3.9 to 12.3 months). Overall, the median time to first HZ episode post-alloHCT was 20.7 months (IQR, 16.9 to 27.9 months), and the majority of HZ episodes occurred after cessation of antiviral prophylaxis (n = 38 of 43; 88.4%). Most HZ episodes were localized (n = 38; 88.4%) and treated in the outpatient setting with oral antiviral therapy (n = 39; 90.7%) for a median of 10 days (IQR, 7 to 14 days). Disseminated HZ did not occur in any aRZV recipients. Considering aRZV status and follow-up period, the RR of HZ in those who had received 2 doses of aRZV compared to those who had not received any doses of aRZV was 0.90 (95% confidence interval, 0.75 to 1.07; <em>P</em> = .22). In a sensitivity analysis, the risk of HZ was reduced (lower RR) in those receiving aRZV ≥12 months post-alloHCT compared with those who did not receive aRZV. Overall, the receipt of aRZV did not a
背景:预防带状疱疹(HZ)对异体造血细胞移植(alloHCT)后的患者至关重要。Shingrix是一种灭活佐剂重组带状疱疹疫苗(aRZV),目前已上市,推荐用于预防血清阳性免疫功能低下患者的HZ。该疫苗在自体HCT和其他免疫功能低下患者中的安全性和有效性已经确定,然而,对于同种异体HCT接受者,数据有限。目的:主要结果是接受aRZV疫苗接种的患者与未接受aRZV疫苗接种的患者在同种异体hct后发生HZ的频率。其他感兴趣的特征包括HZ的临床表现、治疗和与HZ发作相关的结果。研究设计:2018年4月1日至2024年5月1日,在加拿大多伦多玛格丽特公主医院进行了一项单中心回顾性观察队列研究。在2018年4月1日至2022年12月31日期间接受同种异体hct治疗的成年患者纳入前瞻性登记。随访在死亡时、首次HZ发作后90天、随访失败、研究期结束或同种hct后36个月时停止。系统地从电子病历或登记中收集数据。确认的HZ发作由临床和/或分子标准定义。考虑aRZV状态和累计随访时间,进行统计学分析以确定与HZ相关的危险因素以及HCT后≥12个月发生HZ发作的相对风险(RR)。结果:共纳入445例患者。年龄中位数为56岁(四分位数范围,IQR, 38 ~ 64),男性占轻微优势(241/445,54.2%)。从同种异体hct后≥12个月开始,发生了43次HZ发作,其中至少接受过一剂aRZV的患者中有26/263(9.9%),接受过两次aRZV的患者中有19/263(7.2%),未接受aRZV的患者中有7/70(10.0%),接种状况未知的患者中有10/112(8.9%)。任何剂量aRZV后至HZ的中位时间为8.1个月(IQR为3.9 - 12.3)。总体而言,异位hct后到HZ发作的中位时间为20.7个月(IQR为16.9 - 27.9),大多数发生在停止抗病毒预防治疗后(38/43,88.4%)。大多数HZ发作是局部的(38/43,88.4%),在门诊接受口服抗病毒治疗(39/43,90.7%),中位时间为10天(IQR 7 -14)。aRZV感染者无播散性HZ。考虑到aRZV状态和随访期,接受过两剂aRZV的患者与未接受任何剂量aRZV的患者相比,HZ的RR为0.90(95%置信区间0.75-1.07,p = 0.22)。在一项敏感性分析中,与未接受aRZV的患者相比,在hct后接受aRZV≥12个月的患者发生HZ的风险降低(较低的RR)。结论:总体而言,接受aRZV似乎并不影响同种异体HCT后患者HZ的频率,然而,如果在HCT后接受超过12个月的aRZV治疗,可能会有疾病严重程度的改变和获益。我们提供的初步发现将受益于前瞻性随机试验的进一步评估。
{"title":"Herpes Zoster Frequency and Adjuvanted Recombinant Zoster Vaccination after Allogeneic Hematopoietic Cell Transplantation","authors":"Victoria G. Hall ,&nbsp;Caden Chiarello ,&nbsp;Mats Remberger ,&nbsp;Deepali Kumar ,&nbsp;Ivan Pasic ,&nbsp;Dennis Dong Hwan Kim ,&nbsp;Rajat Kumar ,&nbsp;Auro Viswabandya ,&nbsp;Fotios V. Michelis ,&nbsp;Arjun Law ,&nbsp;Armin Gerbitz ,&nbsp;Sapna Humar ,&nbsp;Wilson Lam ,&nbsp;Igor Novitzky-Basso ,&nbsp;Jonas Mattsson","doi":"10.1016/j.jtct.2025.10.028","DOIUrl":"10.1016/j.jtct.2025.10.028","url":null,"abstract":"&lt;div&gt;&lt;div&gt;The prevention of herpes zoster (HZ) is of prime importance in patients undergoing allogeneic hematopoietic cell transplantation (alloHCT). Shingrix, an inactivated, adjuvanted recombinant zoster vaccine (aRZV), is now available and recommended for preventing HZ in seropositive immunocompromised patients. The safety and efficacy of this vaccine have been established in autologous HCT (autoHCT) recipients and other immunocompromised patients; however, data on alloHCT recipients are limited. The primary outcome of this study was a comparison of HZ frequency in alloHCT recipients who received aRZV vaccination and those who did not. Other characteristics of interest include the clinical presentation of HZ, treatment, and outcomes related to the HZ episode. This single-center retrospective observational cohort study was conducted at Princess Margaret Hospital, Toronto, Canada between April 1, 2018, and May 1, 2024. Adult patients who underwent alloHCT between April 1 2018, and December 31, 2022, were included in a prospective registry. Follow-up was censored at time of death, at 90 days after the first episode of HZ, at loss to follow-up, at the end of the study period, or 36 months post-alloHCT. Data were collected systematically from the electronic medical record or registry. A confirmed HZ episode was defined by clinical and/or molecular criteria. Statistical analysis was performed to determine risk factors associated with HZ and the relative risk (RR) of HZ episodes occurring in those ≥12 months post-alloHCT considering aRZV status and the cumulative follow-up period. A total of 445 patients were included. The median age was 56 years (interquartile range [IQR], 38 to 64 years), with a slight male predominance (n = 241/445; 54.2%). From ≥12 months post-alloHCT, there were 43 HZ episodes, including 26 of 263 patients (9.9%) who had received at least 1 dose of aRZV, 19 of 263 (7.2%) who had received 2 doses, 7 of 70 (10.0%) who did not receive RZV, and 10 of 112 (8.9%) with unknown vaccination status. The median time to HZ after receipt of any dose of aRZV was 8.1 months (IQR, 3.9 to 12.3 months). Overall, the median time to first HZ episode post-alloHCT was 20.7 months (IQR, 16.9 to 27.9 months), and the majority of HZ episodes occurred after cessation of antiviral prophylaxis (n = 38 of 43; 88.4%). Most HZ episodes were localized (n = 38; 88.4%) and treated in the outpatient setting with oral antiviral therapy (n = 39; 90.7%) for a median of 10 days (IQR, 7 to 14 days). Disseminated HZ did not occur in any aRZV recipients. Considering aRZV status and follow-up period, the RR of HZ in those who had received 2 doses of aRZV compared to those who had not received any doses of aRZV was 0.90 (95% confidence interval, 0.75 to 1.07; &lt;em&gt;P&lt;/em&gt; = .22). In a sensitivity analysis, the risk of HZ was reduced (lower RR) in those receiving aRZV ≥12 months post-alloHCT compared with those who did not receive aRZV. Overall, the receipt of aRZV did not a","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"32 2","pages":"Pages 213.e1-213.e9"},"PeriodicalIF":4.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145410121","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
Excellent Outcome of 1-Day Nonmyeloablative Salvage Regimen for Pediatric Patients with Graft Failure following Haploidentical Hematopoietic Stem Cell Transplantation 单倍同型造血干细胞移植后移植失败的儿童患者的1天非清髓挽救方案的良好结果。
IF 4.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-01 DOI: 10.1016/j.jtct.2025.10.030
Wilson Y.K. Chan , Pamela P.W. Lee , Daniel K.L. Cheuk , Wing Leung
<div><div>Haploidentical (haplo-) hematopoietic stem cell transplantation (HSCT) has been increasingly used as an alternative transplantation strategy for patients lacking a suitable HLA-matched donor. T cell depletion reduces the risk of graft-versus-host disease (GVHD) but at a cost of increased risk of graft failure, necessitating immediate retransplantation if a cryopreserved autologous graft is not available for rescue. Traditional high-intensity reconditioning with myeloablative and immunoablative regimens of approximately 1 week’s duration aimed at further suppressing the recipient-derived immune system capable of rejecting the primary graft, is associated with high morbidity and mortality. A shortened and reduced-intensity conditioning regimen might reduce infection risk and mortality, but sustained engraftment would be a concern with lowered intensity. Here we report the excellent outcomes of 11 pediatric patients who received a 1-day reduced-intensity preparative regimen prior to retransplantation for graft failure following initial myeloablative haplo-HSCT for various malignant and nonmalignant disease conditions. This was a retrospective study conducted at the Hong Kong Children’s Hospital, the sole territory-wide pediatric HSCT center in Hong Kong. All pediatric patients who were age ≤18 years at the time of initial haplo-HSCT and subsequently underwent salvage haplo-HSCT with a 1-day nonmyeloablative salvage regimen owing to graft failure between June 1, 2021, and May 31, 2024, were included. The salvage regimen consisted of fludarabine (30 mg/m<sup>2</sup>), cyclophosphamide (2000 mg/m<sup>2</sup> or 60 mg/kg), and alemtuzumab (0.3 mg/kg), with or without total body irradiation (2 Gy), all administered 1 day before retransplantation. G-CSF-mobilized peripheral blood stem cells (PBSCs) were collected and transplanted fresh without ex vivo T cell depletion whenever possible, while cryopreserved PBSCs were used if fresh apheresis was not possible. GVHD prophylaxis consisted of cyclosporine, mycophenolate mofetil, tacrolimus, or sirolimus. A total of 11 patients were recruited, including 9 males and 2 females, with a median age of 8.8 years (range, 2.4 to 17.5 years). Underlying diseases included transfusion-dependent anemias (n = 7; beta-thalassemia major = 4, hemoglobin Hammersmith = 1, pyruvate kinase deficiency = 1, severe aplastic anemia = 1), chronic granulomatous disease (n = 1), acute lymphoblastic leukemia (n = 1), post-liver transplant lymphoproliferative disease (n = 1) and neuroblastoma (n = 1). The median interval between haplo-HSCT and administration of the 1-day regimen was 26 days (range, 18 to 50 days). Fresh PBSC grafts were used in 9 patients, and cryopreserved PBSC grafts were used in 2 patients. Median cell viability was 99.6%, with a median stem cell dose of 7.2 × 10<sup>6</sup> CD34<sup>+</sup> cells/kg (range, 4.81 to 20.6 × 10<sup>6</sup> cells/kg) and a median total nucleated cell (TNC) dose of 8.0 × 10<sup>8<
背景:单倍体造血干细胞移植(haploo - hsct)越来越多地被用于缺乏合适的人类白细胞抗原匹配供体的患者的替代移植策略。t细胞耗损降低了移植物抗宿主病(GVHD)的风险,但代价是移植物衰竭的风险增加,如果冷冻保存的自体移植物无法抢救,则需要立即再次移植。传统的高强度修复与清髓和免疫消融方案持续约1周,旨在进一步抑制能够排斥原发移植物的受体来源免疫系统,其发病率和死亡率较高。缩短和降低强度的调理方案可能降低感染风险和死亡率,但持续植入将是一个降低强度的问题。目的:在这里,我们报告了11名儿童患者的良好结果,他们在各种恶性和非恶性疾病的初始清髓单倍造血干细胞移植后,因移植物衰竭而在再次移植前接受了1天降低强度的准备方案。研究设计:这是一项在香港儿童医院进行的回顾性研究,香港儿童医院是香港唯一的区域性儿童造血干细胞移植中心。所有在2021年6月1日至2024年5月31日期间因移植物失败而接受首次单倍造血干细胞移植并随后接受为期一天的非清髓性单倍造血干细胞移植的18岁或以下儿童患者均被纳入研究。挽救方案包括氟达拉滨(30mg/m2),环磷酰胺(2000mg/m2或60mg/kg)和阿仑单抗(0.3mg/kg),有或没有全身照射(2Gy),均在再次移植前1天给予。收集gcsf动员的外周血干细胞(PBSCs),并尽可能在没有体外t细胞耗尽的情况下进行新鲜移植,而如果无法进行新鲜分离,则使用冷冻保存的PBSCs。GVHD预防包括环孢素、霉酚酸酯、他克莫司或西罗莫司。结果:共纳入11例患者,其中男性9例,女性2例,中位年龄8.8岁(2.4-17.5岁)。潜在的疾病包括transfusion-dependent贫血(n = 7;beta-thalassemia主要 = 4,血红蛋白哈默史密斯 = 1,丙酮酸激酶缺乏症 = 1,严重的再生障碍性贫血 = 1),慢性肉芽肿性疾病(n = 1),急性淋巴细胞白血病(n = 1), post-liver移植淋巴增殖性疾病(n = 1)和神经母细胞瘤(n = 1)。单倍造血干细胞移植到使用一日方案的中位间隔为26天(范围18-50天)。9例患者使用新鲜PBSC移植,2例患者使用冷冻保存的PBSC移植。细胞存活率中位数为99.6%,干细胞中位数剂量为7.2 × 106个CD34+细胞/kg(范围4.81-20.6),总有核细胞剂量为8.0 × 108个/kg(范围5.17-10)。9例移植新鲜PBSC的患者均表现出持续的移植和造血功能恢复。中位中性粒细胞和血小板(≥20 × 109/L)分别在+12天(范围10-19)和+16天(范围10-35)植入。没有人发展为III/IV级急性GVHD或严重慢性GVHD。对于2例使用冷冻保存的PBSC移植物的患者,均导致移植物失败,其中1例通过使用新鲜PBSC移植物的另一个为期一天的方案成功挽救。另一名患者进行了自体再生,并在完全清髓后再次成功地接受了来自同一供体的单倍造血干细胞移植。在中位随访35个月(18-52个月)时,所有11例患者的总生存率均为100%。结论:本地经验表明,改良的1天低强度方案联合新鲜而非冷冻保存的PBSC移植物是可行的,有望实现持续植入。这是一种安全,适当的方法抢救儿科患者的移植失败,需要立即再移植。
{"title":"Excellent Outcome of 1-Day Nonmyeloablative Salvage Regimen for Pediatric Patients with Graft Failure following Haploidentical Hematopoietic Stem Cell Transplantation","authors":"Wilson Y.K. Chan ,&nbsp;Pamela P.W. Lee ,&nbsp;Daniel K.L. Cheuk ,&nbsp;Wing Leung","doi":"10.1016/j.jtct.2025.10.030","DOIUrl":"10.1016/j.jtct.2025.10.030","url":null,"abstract":"&lt;div&gt;&lt;div&gt;Haploidentical (haplo-) hematopoietic stem cell transplantation (HSCT) has been increasingly used as an alternative transplantation strategy for patients lacking a suitable HLA-matched donor. T cell depletion reduces the risk of graft-versus-host disease (GVHD) but at a cost of increased risk of graft failure, necessitating immediate retransplantation if a cryopreserved autologous graft is not available for rescue. Traditional high-intensity reconditioning with myeloablative and immunoablative regimens of approximately 1 week’s duration aimed at further suppressing the recipient-derived immune system capable of rejecting the primary graft, is associated with high morbidity and mortality. A shortened and reduced-intensity conditioning regimen might reduce infection risk and mortality, but sustained engraftment would be a concern with lowered intensity. Here we report the excellent outcomes of 11 pediatric patients who received a 1-day reduced-intensity preparative regimen prior to retransplantation for graft failure following initial myeloablative haplo-HSCT for various malignant and nonmalignant disease conditions. This was a retrospective study conducted at the Hong Kong Children’s Hospital, the sole territory-wide pediatric HSCT center in Hong Kong. All pediatric patients who were age ≤18 years at the time of initial haplo-HSCT and subsequently underwent salvage haplo-HSCT with a 1-day nonmyeloablative salvage regimen owing to graft failure between June 1, 2021, and May 31, 2024, were included. The salvage regimen consisted of fludarabine (30 mg/m&lt;sup&gt;2&lt;/sup&gt;), cyclophosphamide (2000 mg/m&lt;sup&gt;2&lt;/sup&gt; or 60 mg/kg), and alemtuzumab (0.3 mg/kg), with or without total body irradiation (2 Gy), all administered 1 day before retransplantation. G-CSF-mobilized peripheral blood stem cells (PBSCs) were collected and transplanted fresh without ex vivo T cell depletion whenever possible, while cryopreserved PBSCs were used if fresh apheresis was not possible. GVHD prophylaxis consisted of cyclosporine, mycophenolate mofetil, tacrolimus, or sirolimus. A total of 11 patients were recruited, including 9 males and 2 females, with a median age of 8.8 years (range, 2.4 to 17.5 years). Underlying diseases included transfusion-dependent anemias (n = 7; beta-thalassemia major = 4, hemoglobin Hammersmith = 1, pyruvate kinase deficiency = 1, severe aplastic anemia = 1), chronic granulomatous disease (n = 1), acute lymphoblastic leukemia (n = 1), post-liver transplant lymphoproliferative disease (n = 1) and neuroblastoma (n = 1). The median interval between haplo-HSCT and administration of the 1-day regimen was 26 days (range, 18 to 50 days). Fresh PBSC grafts were used in 9 patients, and cryopreserved PBSC grafts were used in 2 patients. Median cell viability was 99.6%, with a median stem cell dose of 7.2 × 10&lt;sup&gt;6&lt;/sup&gt; CD34&lt;sup&gt;+&lt;/sup&gt; cells/kg (range, 4.81 to 20.6 × 10&lt;sup&gt;6&lt;/sup&gt; cells/kg) and a median total nucleated cell (TNC) dose of 8.0 × 10&lt;sup&gt;8&lt;","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"32 2","pages":"Pages 201.e1-201.e16"},"PeriodicalIF":4.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145426983","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
Applicability of Allogeneic Hematopoietic Cell Transplant for TP53-Mutated Myeloid Neoplasms: Are We There Yet? 同种异体造血细胞移植治疗tp53突变骨髓肿瘤的适用性:我们还在那里吗?
IF 4.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-01 DOI: 10.1016/j.jtct.2026.01.012
Mithun Vinod Shah
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引用次数: 0
Refined Elderly Comorbidity Index (RECI) Is Highly Predictive of Post-Transplant Survival in Older Adults with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) 精细化老年共病指数(RECI)可高度预测老年急性髓性白血病(AML)和骨髓增生异常综合征(MDS)患者移植后的生存
IF 4.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-01 DOI: 10.1016/j.jtct.2025.12.105
Fnu Amisha MD , Corbin Wright MD , Sean Haney MD , Pujan Patel MD , Biwei Cao PhD , Jongphil Kim PhD , Onyee Chan MD , Andrew T Kuykendall MD , Nancy Torres MD , Reshma Ramlal MD , Rawan Faramand MD , Abu-Sayeef Mirza MD, MPH , Fabiana Perna MD, PhD , Allison Walker MD , Asmita Mishra MD , Lia Elena Perez MD , Eric Padron MD , Aleksandr Lazaryan MD, MPH, PhD , Farhad Khimani MD , David A. Sallman MD , Nelli Bejanyan MD
<div><h3>Background</h3><div>Older age is a known risk factor for non-relapse mortality (NRM) after allogenic stem cell transplantation (HCT). Reduced-intensity conditioning (RIC) has made HCT- the only curative option- more accessible to elderly AML and MDS patients (pts). Age adjusted HCT comorbidity index (HCT-CI), though widely use, was developed from a heterogeneous cohort and its predictive accuracy may be limited by changes in clinical practice and patient characteristics over time. We present additional predictors of NRM and overall survival (OS) to introduce RECI for patients ≥60 years (yrs) receiving HCT for AML/MDS.</div></div><div><h3>Methods</h3><div>In this single-center retrospective study, we evaluated predictive markers and survival outcomes in 941 pts aged ≥60 yrs who underwent HCT (2005-2023) for AML or MDS (Fig 1). The primary endpoints were 2-year NRM and OS. Adjusted hazard ratios (HR) of significant variables from multivariate Cox model of NRM were converted to an integer score to develop the RECI. HR of 1.3-2, 2.1-3, and 3-4, were assigned weights of 1, 2, and 3, respectively. RECI, the sum of integer weights, was used to stratify patients according to 2-year NRM and OS.</div></div><div><h3>Results</h3><div>Baseline characteristics are summarized (Fig 1, 2). At 2-year post-HCT, NRM was 22% (95% CI: 20-25) and OS was 59% (95% CI: 56-62) for the entire cohort. In multivariate analysis, NRM and OS were similar in patients aged 60-69 and ≥70 years. NRM was higher with HCT-CI ≥3 (HR 1.61, 95% CI 1.19- 2.18, p=0.002), eGFR <60 (HR 1.83, 95% CI 1.26- 2.67, p=0.002), albumin <3.5 (HR 1.83, 95% CI 1.25- 2.65, p=0.002), and CMV R+ (HR 1.36, 95% CI 1.01- 1.83, p=0.04). OS was worse with HCT-CI ≥3 (HR 1.46, 95% CI 1.15- 1.86, p=0.0018), albumin <3.5 (HR 1.54, 95% CI 1.1- 2.16, p=0.01), ferritin ≥700 (HR 1.43, 95% CI 1.10- 1.87, p=0.007), and high risk AML/MDS (HR 1.58, 95% CI 1.25- 1.99, p= <0.0001).</div><div>Components of RECI are summarized (Fig 3). RECI ranged from 0 to 4 and stratified patients into 3 risk groups with score of 0 (33%), 1 (53%), and ≥2 (14%). RECI was strongly prognostic for 2-year NRM ranging from 15% (95% CI: 11-19) in RECI 0, 22% (95% CI: 19-26) in RECI 1 (HR=1.6, 95% CI 1.13-2.25, p=0.009) and 38% (95% CI: 29-46) in RECI ≥2 (HR=3.0, 95% CI 1.99-4.46, p<0.001) (Fig 4). The prognostic significance of RECI was also determined for OS ranging from 66% (95% CI: 61-72) in RICI 0, 58% (95% CI: 54-63) in RECI 1 (HR=1.3, 95% CI 1.04-1.68, p=0.02) and 44% (95% CI: 36-53) in RECI ≥2 (HR=2.2, 95% CI 1.60-2.91, p<0.0001) (Fig 4).</div></div><div><h3>Conclusions</h3><div>RECI applied at time of HCT strongly predicts NRM and OS in patients aged ≥60 with AML/MDS. No difference in survival outcomes was observed in patients aged 60-69 and ≥70 yrs. While elderly patients should be considered candidates for HCT, predictive factors that affect NRM and OS should be carefully assessed prior to HCT. If validated in l
背景:年龄是同种异体干细胞移植(HCT)术后非复发死亡率(NRM)的已知危险因素。低强度调节(RIC)使得HCT(唯一的治疗选择)更容易被老年AML和MDS患者接受。年龄调整的HCT共病指数(HCT- ci)虽然被广泛使用,但它是从异质队列中发展而来的,其预测准确性可能受到临床实践和患者特征随时间变化的限制。我们提出了NRM和总生存期(OS)的其他预测因素,以引入≥60岁(yrs)接受HCT治疗的AML/MDS患者的RECI。在这项单中心回顾性研究中,我们评估了941名年龄≥60岁的AML或MDS患者(2005-2023年)的预测标志物和生存结果(图1)。主要终点为2年NRM和OS。将NRM多变量Cox模型中显著变量的校正风险比(HR)转换为整数分值,形成RECI。HR分别为1.3-2、2.1-3和3-4,权重分别为1、2和3。采用整数权值和RECI,根据2年NRM和OS对患者进行分层。基线特征总结(图1、2)。在hct后2年,整个队列的NRM为22% (95% CI: 20-25), OS为59% (95% CI: 56-62)。在多变量分析中,60-69岁和≥70岁患者的NRM和OS相似。当HCT-CI≥3 (HR 1.61, 95% CI 1.19- 2.18, p=0.002)、eGFR <60 (HR 1.83, 95% CI 1.26- 2.67, p=0.002)、白蛋白<;3.5 (HR 1.83, 95% CI 1.25- 2.65, p=0.002)和CMV R+ (HR 1.36, 95% CI 1.01- 1.83, p=0.04)时,NRM较高。HCT-CI≥3 (HR 1.46, 95% CI 1.15- 1.86, p=0.0018)、白蛋白3.5 (HR 1.54, 95% CI 1.1- 2.16, p=0.01)、铁蛋白≥700 (HR 1.43, 95% CI 1.10- 1.87, p=0.007)和高风险AML/MDS (HR 1.58, 95% CI 1.25- 1.99, p= <0.0001)的OS较差。总结了RECI的组成部分(图3)。RECI评分范围从0到4,将患者分为3个危险组,分别为0分(33%)、1分(53%)和≥2分(14%)。RECI对2年NRM有很强的预后作用,RECI 0组为15% (95% CI: 11-19), RECI 1组为22% (95% CI: 19-26) (HR=1.6, 95% CI 1.13-2.25, p=0.009), RECI≥2组为38% (95% CI: 29-46) (HR=3.0, 95% CI 1.99-4.46, p<0.001)(图4)。RECI对OS的预后意义也被确定,范围从RICI 0的66% (95% CI: 61-72), RECI 1的58% (95% CI: 54-63) (HR=1.3, 95% CI 1.04-1.68, p=0.02)和RECI≥2的44% (95% CI: 36-53) (HR=2.2, 95% CI 1.60-2.91, p<0.0001)(图4)。结论在HCT时应用reci对≥60岁AML/MDS患者的NRM和OS有很强的预测作用。60-69岁和≥70岁患者的生存结局无差异。虽然老年患者应被视为HCT的候选者,但在HCT之前应仔细评估影响NRM和OS的预测因素。如果在大型独立HCT队列中得到验证,RECI作为一种新的预后工具可以帮助老年患者进行HCT的适当选择。
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Transplantation and Cellular Therapy
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