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Healthcare resource utilization and associated costs in patients with chronic graft-versus-host disease post allogeneic hematopoietic stem cell transplantation in England: HCRU and associated costs in patients with cGvHD in England. 英国异基因造血干细胞移植后慢性移植物抗宿主疾病患者的医疗资源利用率和相关费用:英国异基因造血干细胞移植后慢性移植物抗宿主疾病患者的医疗资源利用率和相关费用:HCRU 和相关费用。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-10-08 DOI: 10.1016/j.jtct.2024.10.002
D Avenoso, J A Davidson, H Larvin, H R Brewer, C T Rice, K Ecsy, A Sil, L Skinner, R D A Hudson
<p><strong>Background: </strong>Limited evidence suggests chronic graft-versus-host disease (cGvHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT) increases healthcare resource utilization (HCRU) and costs. However, this burden has not been well characterized in England.</p><p><strong>Objective: </strong>This study assesses secondary care HCRU and costs for patients following allo HSCT in England with cGvHD and patients who did not develop graft versus-host disease (GvHD). Further stratification was performed among patients who did or did not subsequently receive high-cost therapies for the treatment of cGvHD.</p><p><strong>Study design: </strong>This descriptive, retrospective cohort study used Hospital Episode Statistics (HES) data from April 2017-March 2022. HES data captures information on reimbursed diagnoses and procedures from all National Health Service (NHS) secondary care admissions and attendances in England. High-cost drugs as defined by NHS England are recorded in HES, these drugs and other procedures including plasma exchange, were used to identify patients with cGvHD who were in receipt of high-cost therapies. HCRU and costs were described for patients with cGvHD following allo-HSCT (n=721) and were matched with patients with no evidence of GvHD following allo-HSCT (n=718). HCRU and costs were also described for the subset of patients with cGvHD (n=198) following receipt of high-cost therapies and patients with cGvHD prior to or without such therapies (n=523).</p><p><strong>Results: </strong>A higher proportion of patients with cGvHD had at least one inpatient or intensive care unit (ICU) admission or emergency care attendance than patients without GvHD (inpatient: 74.6% vs 66.6%; emergency care: 39.3% vs 30.5%; ICU: 7.4% vs 4.7%; respectively); whilst the proportion of patients with an outpatient attendance were similar for both groups (outpatient: 80.3% vs 84.1%; respectively). The cost across all secondary care settings was higher for patients with cGvHD than patients without GvHD, with a mean cost of inpatient admissions of £17,339 ppy for those with cGvHD vs £8,548 ppy in patients without GvHD. A higher proportion of patients who received high-cost therapies for the treatment of cGvHD had at least one secondary care admission or attendance, than patients who did not (inpatient: 85.4% vs 66.4%; ICU: 7.1% vs 5.4%; outpatient: 87.9% vs 76.7%; emergency care: 44.4% vs 36.5%; respectively). Patients who were treated with high-cost therapies for the treatment of cGvHD had a greater mean number (14.6 vs 8.2 ppy, respectively) for all-cause inpatient admissions after treatment, than patients who did not. In all secondary care settings, the total cost ppy was higher for patients who received high-cost therapies for the treatment of cGvHD, than for those who did not. Patients who were treated with high-cost therapies for the treatment of cGvHD had a greater mean cost (£21,137 vs £15,956 ppy, respectively) for a
背景:有限的证据表明,异基因造血干细胞移植(allo-HSCT)后的慢性移植物抗宿主疾病(cGvHD)会增加医疗资源利用率(HCRU)和成本。然而,在英格兰,这种负担还没有得到很好的描述:本研究评估了在英国接受异体造血干细胞移植后,发生移植物抗宿主疾病(GvHD)的患者和未发生移植物抗宿主疾病(GvHD)的患者的二级护理HCRU和费用。对随后接受或未接受高成本疗法治疗 cGvHD 的患者进行了进一步分层:这项描述性、回顾性队列研究使用了2017年4月至2022年3月的医院病例统计(Hospital Episode Statistics,HES)数据。HES 数据收集了英格兰所有国民健康服务(NHS)二级医疗机构入院和就诊的有偿诊断和手术信息。HES 中记录了英格兰国家医疗服务体系定义的高成本药物,这些药物和包括血浆置换在内的其他程序被用来识别接受高成本疗法的 cGvHD 患者。对接受异体造血干细胞移植(allo-HSCT)后出现 cGvHD 的患者(人数=721)的 HCRU 和费用进行了描述,并与接受异体造血干细胞移植后未出现 GvHD 的患者(人数=718)进行了比对。此外,还对接受高成本疗法后出现 cGvHD 的患者(n=198)和接受或未接受此类疗法前出现 cGvHD 的患者(n=523)的 HCRU 和费用进行了描述:与未接受高成本疗法的患者相比,接受过至少一次住院治疗或重症监护室(ICU)治疗或急诊治疗的cGvHD患者比例更高(住院患者:74.6%对66.6%;急诊患者:39.3%对30.5%):39.3%对30.5%;重症监护室:7.4%对4.7%);而两组患者的门诊就诊比例相似(门诊:80.3%对84.1%)。cGvHD 患者在所有二级医疗机构的费用均高于非 GvHD 患者,cGvHD 患者的平均住院费用为每年 17,339 英镑,而非 GvHD 患者的平均住院费用为每年 8,548 英镑。在接受高成本疗法治疗 cGvHD 的患者中,至少接受过一次二级护理或就诊的患者比例高于未接受二级护理或就诊的患者(住院患者:85.4% 对 66.4%;重症监护室:7.1% 对 5.4%;门诊患者:87.9% 对 76.7%;急诊患者:44.4% 对 36.5%):分别为 44.4% vs 36.5%)。接受高成本疗法治疗 cGvHD 的患者在治疗后因各种原因住院的平均人数(分别为 14.6 人对 8.2 人)高于未接受治疗的患者。在所有二级医疗机构中,接受高成本疗法治疗 cGvHD 的患者的总成本ppy均高于未接受治疗的患者。与未接受高成本疗法治疗的患者相比,接受高成本疗法治疗的 cGvHD 患者因各种原因住院的平均费用更高(分别为 21,137 英镑 vs 15,956 英镑/年):本研究表明,在英格兰的各种二级医疗机构中,cGvHD 和使用相关高成本疗法对医疗活动和成本的影响要大于未接受 GvHD 治疗的患者和未接受高成本疗法的 cGvHD 患者。
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引用次数: 0
Advanced Practice Providers in Cellular Therapy: survey results from the ASTCT APP Special Interest Group exploring clinical roles, compensation and job satisfaction. 细胞疗法高级从业人员:ASTCT APP 小组对临床角色、报酬和工作满意度的调查结果。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-10-08 DOI: 10.1016/j.jtct.2024.10.001
Cory Edgar, Nancy Shreve, Misty D Evans, Chelsea Honstain, Michelle Skinner, Elizabeth Zerante, Rita Jakubowski, Kadee Raser

Background: Advanced practice providers (APPs), which include physician assistants/associates and advanced practice nurses, are critical members of the transplant and cellular therapy (TCT) care team. Despite broad utilization in transplant centers, there is little published literature on the clinical roles and responsibilities, staffing models, compensation structure, and job satisfaction of TCT APPs. This study represents the results of a national survey administered by the APP Special Interest Group to better characterize the TCT APP workforce.

Objective: To characterize the TCT APP workforce by investigating clinical roles and responsibilities, compensation and institutional support, and job satisfaction.

Methods: A 25-item web-based survey addressing four domains (transplant center data, APP roles and responsibilities, compensation and institutional support, and job satisfaction). Surveys were sent to participants through a chain-referral sampling method. Data were analyzed using descriptive statistics and multinomial logistic regression.

Results: A total of 198 responses were analyzed, representing 64 transplant centers of varying size from 29 states. APPs report working in inpatient and outpatient settings and performing a broad array of TCT-associated procedures including bone marrow biopsy (78%), lumbar puncture (43.2%), intrathecal chemotherapy (47.0%), and cellular infusions (45.9%). Median salary of respondents was $110,000 - $119,000 and was significantly associated with geographic location of transplant center and years of experience. A minority of respondents reported no funding (4.2%) or time (9.8%) supporting continuing education. A majority of APPs (55.1%) do not feel they are appropriately paid. A majority (54.3%) did not feel that their center supported a good work-life balance. Nearly 35.4% of respondents did not feel valued in their role.

Conclusions: This survey represents the first to characterize the TCT APP workforce in the United States. APPs are highly integrated into the TCT care team and can serve as means to improve patient access to TCT therapies given a worsening physician shortage. However, the lack of satisfaction with compensation and work-life balance could represent barriers to recruitment and retention of TCT APPs and warrant future studies to better characterize.

背景:包括医生助理/助理医师和高级执业护士在内的高级执业医师(APP)是移植和细胞治疗(TCT)护理团队的重要成员。尽管在移植中心得到了广泛应用,但有关 TCT APP 的临床角色和职责、人员配备模式、薪酬结构和工作满意度的公开文献却很少。本研究是 APP 小组为更好地了解 TCT APP 工作队伍的特点而进行的一项全国性调查的结果:通过调查临床角色和职责、薪酬和机构支持以及工作满意度,了解 TCT APP 工作队伍的特点:方法:针对四个领域(移植中心数据、APP 角色与职责、薪酬与机构支持以及工作满意度)进行 25 个项目的网络调查。调查表通过连锁推荐抽样方法发送给参与者。采用描述性统计和多项式逻辑回归对数据进行了分析:共分析了 198 份回复,代表了来自 29 个州的 64 个不同规模的移植中心。据 APP 报告,他们在住院和门诊环境中工作,执行一系列与 TCT 相关的程序,包括骨髓活检(78%)、腰椎穿刺(43.2%)、鞘内化疗(47.0%)和细胞输注(45.9%)。受访者的工资中位数为 110,000 - 119,000 美元,与移植中心的地理位置和工作年限密切相关。少数受访者表示没有资金(4.2%)或时间(9.8%)支持继续教育。大多数 APP(55.1%)认为他们没有得到适当的报酬。大多数受访者(54.3%)认为他们所在的中心不支持工作与生活的良好平衡。近 35.4% 的受访者认为自己的工作没有价值:这项调查首次描述了美国 TCT APP 工作队伍的特点。APP高度融入TCT医疗团队,在医生短缺日益加剧的情况下,可以作为改善患者获得TCT疗法的手段。然而,对薪酬和工作与生活的平衡缺乏满意度可能会成为招聘和留住TCT APP的障碍,需要在今后的研究中更好地加以分析。
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引用次数: 0
Superior survival after unrelated allogeneic stem cell transplantation with low-dose ATG compared to low-dose TBI in myeloablative fludarabine/busulfan-based regimen for MDS on behalf of the adult MDS Working Group of the JSTCT. 在基于氟达拉滨/布磺凡的髓鞘消融治疗MDS方案中,与低剂量TBI相比,使用低剂量ATG进行非亲属异基因干细胞移植后的存活率更高。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-10-05 DOI: 10.1016/j.jtct.2024.09.026
Machiko Fujioka, Hidehiro Itonaga, Hideyuki Nakazawa, Tetsuya Nishida, Keisuke Kataoka, Takashi Ikeda, Shinichi Kako, Ken-Ichi Matsuoka, Koji Adachi, Shini-Chiro Fujiwara, Nobuyuki Aotsuka, Toshiro Kawakita, Emiko Sakaida, Yoshinobu Kanda, Tatsuo Ichinohe, Yoshiko Atsuta, Yasushi Miyazaki, Ken Ishiyama

The fludarabine/intravenous busulfan 12.8 mg/kg (FB4) regimen is an effective conditioning regimen in allogeneic hematopoietic stem cell transplantation for myelodysplastic syndrome (MDS); however, limited data is available on the prognostic impact of FB4 with low-dose anti-thymoglobulin (ATG ≤ 5 mg/kg) or low-dose total body irradiation (TBI ≤ 4 Gy). Therefore, we retrospectively evaluated the outcomes in 280 adults with de novo MDS who underwent their first transplantation from an unrelated donor between 2009 and 2018. Median age was 61 years (range, 16 to 70 years). In the FB4 alone (FB4), FB4 plus ATG (FB4-ATG), and FB4 plus TBI (FB4-TBI) groups, 3-year overall survival (OS) rates were 39.9, 64.8, and 43.7 %; 3-year non-relapse mortality (NRM) were 32.1, 22.1, and 27.1%; and 3-year relapse incidences were 34.7, 21.2, and 28.9%, respectively. The multivariate analyses showed that FB4-ATG group significantly correlated with better OS (hazard Ratio [HR], 0.51; 95% confidence interval [CI], 0.27-0.95; P=0.032) than FB4 group. FB4-ATG group tended to correlate with lower NRM (HR, 0.36;95% CI, 0.13-1.06; P=0.063) than FB4 group. In comparison with FB4-TBI group, FB4-ATG group showed better OS (HR 0.52, 95% CI 0.27-0.99, P=0.049) and NRM (HR 0.034, 95% CI 0.11-0.92, P=0.034). No significant differences were observed in OS and NRM between the FB4-TBI and FB4 groups. The present study demonstrated that the FB4 plus low-dose ATG regimen improved OS and NRM, but FB4 plus low-dose TBI regimen had no clear benefit over FB4 alone, in MDS patients who used unrelated donors.

在骨髓增生异常综合征(MDS)的异基因造血干细胞移植中,氟达拉滨/静脉注射12.8 mg/kg(FB4)方案是一种有效的调理方案;然而,关于FB4与低剂量抗胸腺球蛋白(ATG ≤ 5 mg/kg)或低剂量全身照射(TBI ≤ 4 Gy)对预后影响的数据却很有限。因此,我们回顾性评估了2009年至2018年期间首次接受非亲属供体移植的280例成人新发MDS患者的治疗效果。中位年龄为61岁(16至70岁)。在单用FB4组(FB4)、FB4加ATG组(FB4-ATG)和FB4加TBI组(FB4-TBI)中,3年总生存率(OS)分别为39.9%、64.8%和43.7%;3年非复发死亡率(NRM)分别为32.1%、22.1%和27.1%;3年复发率分别为34.7%、21.2%和28.9%。多变量分析显示,与FB4组相比,FB4-ATG组与更好的OS显著相关(危险比[HR],0.51;95%置信区间[CI],0.27-0.95;P=0.032)。与 FB4 组相比,FB4-ATG 组往往与较低的 NRM 相关(HR,0.36;95% CI,0.13-1.06;P=0.063)。与 FB4-TBI 组相比,FB4-ATG 组显示出更好的 OS(HR 0.52,95% CI 0.27-0.99,P=0.049)和 NRM(HR 0.034,95% CI 0.11-0.92,P=0.034)。FB4-TBI组和FB4组在OS和NRM方面未观察到明显差异。本研究表明,在使用非亲属供体的MDS患者中,FB4加小剂量ATG方案可改善OS和NRM,但FB4加小剂量TBI方案与单用FB4相比无明显益处。
{"title":"Superior survival after unrelated allogeneic stem cell transplantation with low-dose ATG compared to low-dose TBI in myeloablative fludarabine/busulfan-based regimen for MDS on behalf of the adult MDS Working Group of the JSTCT.","authors":"Machiko Fujioka, Hidehiro Itonaga, Hideyuki Nakazawa, Tetsuya Nishida, Keisuke Kataoka, Takashi Ikeda, Shinichi Kako, Ken-Ichi Matsuoka, Koji Adachi, Shini-Chiro Fujiwara, Nobuyuki Aotsuka, Toshiro Kawakita, Emiko Sakaida, Yoshinobu Kanda, Tatsuo Ichinohe, Yoshiko Atsuta, Yasushi Miyazaki, Ken Ishiyama","doi":"10.1016/j.jtct.2024.09.026","DOIUrl":"https://doi.org/10.1016/j.jtct.2024.09.026","url":null,"abstract":"<p><p>The fludarabine/intravenous busulfan 12.8 mg/kg (FB4) regimen is an effective conditioning regimen in allogeneic hematopoietic stem cell transplantation for myelodysplastic syndrome (MDS); however, limited data is available on the prognostic impact of FB4 with low-dose anti-thymoglobulin (ATG ≤ 5 mg/kg) or low-dose total body irradiation (TBI ≤ 4 Gy). Therefore, we retrospectively evaluated the outcomes in 280 adults with de novo MDS who underwent their first transplantation from an unrelated donor between 2009 and 2018. Median age was 61 years (range, 16 to 70 years). In the FB4 alone (FB4), FB4 plus ATG (FB4-ATG), and FB4 plus TBI (FB4-TBI) groups, 3-year overall survival (OS) rates were 39.9, 64.8, and 43.7 %; 3-year non-relapse mortality (NRM) were 32.1, 22.1, and 27.1%; and 3-year relapse incidences were 34.7, 21.2, and 28.9%, respectively. The multivariate analyses showed that FB4-ATG group significantly correlated with better OS (hazard Ratio [HR], 0.51; 95% confidence interval [CI], 0.27-0.95; P=0.032) than FB4 group. FB4-ATG group tended to correlate with lower NRM (HR, 0.36;95% CI, 0.13-1.06; P=0.063) than FB4 group. In comparison with FB4-TBI group, FB4-ATG group showed better OS (HR 0.52, 95% CI 0.27-0.99, P=0.049) and NRM (HR 0.034, 95% CI 0.11-0.92, P=0.034). No significant differences were observed in OS and NRM between the FB4-TBI and FB4 groups. The present study demonstrated that the FB4 plus low-dose ATG regimen improved OS and NRM, but FB4 plus low-dose TBI regimen had no clear benefit over FB4 alone, in MDS patients who used unrelated donors.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142393597","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
N-803, an IL-15 superagonist complex as maintenance therapy after allogeneic donor stem cell transplant for acute myeloid leukemia or myelodysplastic syndrome; a Phase 2 trial. N-803是一种IL-15超拮抗剂复合物,用于急性髓性白血病或骨髓增生异常综合征异体供体干细胞移植后的维持治疗;2期试验。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-10-01 DOI: 10.1016/j.jtct.2024.09.023
Aimee Merino, Claudio C Brunstein, Ryan Shanley, Faridullah Rashid, Rose Wangen, Veronika Bachanova, Mark Juckett, Joseph Maakaron, Martin Felices, Daniel Weisdorf, Jeffrey S Miller

Maintenance therapy may improve natural killer (NK) cell surveillance after allogeneic donor hematopoietic cell transplant (HCT) for myeloid malignancies and represents a potential approach to improve cure rates. Interleukin-15 (IL-15) enhances lymphocyte proliferation and anti-tumor activity. In a prior Phase 1 study of an IL-15 superagonist (N-803) in patients with AML who relapsed after HCT, we observed in vivo expansion of NK cells and anti-tumor responses. The primary objective of this Phase 2 trial was to determine if post-transplant N-803 could reduce relapse. We administered N-803 (n=20) (dosed 6 mcg/kg subcutaneously (SQ) at day 60 after HCT to patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) who were in complete remission (CR). N-803 treatment was planned weekly, bi-weekly or every 4 weeks in 2 sequential cohorts. The most common adverse events after administration were self-limited injection sites skin rashes (n=20). One week after an N-803 dose, we observed enhanced NK cell proliferation and improved anti-tumor cytotoxicity without inducing immune exhaustion. Five patients who developed acute graft versus host disease (aGVHD) after N-803 responded promptly to steroids and 4 patients developed chronic GVHD. Patients receiving >4 doses of N-803 had a 3-fold decrease in relapse at two years (p=0.06). These findings support the safety, immune activation, and potential efficacy of N-803 to prevent relapse of AML/MDS after HSCT.

维持疗法可改善异体捐献造血细胞移植(HCT)治疗髓系恶性肿瘤后自然杀伤(NK)细胞的监控,是提高治愈率的一种潜在方法。白细胞介素-15(IL-15)能增强淋巴细胞增殖和抗肿瘤活性。在之前对 HCT 后复发的急性髓细胞性白血病患者进行的 IL-15 超拮抗剂(N-803)1 期研究中,我们观察到 NK 细胞的体内扩增和抗肿瘤反应。这项 2 期试验的主要目的是确定移植后 N-803 能否减少复发。我们在骨髓增生异常综合征(MDS)或急性髓性白血病(AML)完全缓解(CR)患者接受造血干细胞移植后第60天给予N-803(n=20)(剂量为6微克/千克皮下注射(SQ))。N-803计划每周、每两周或每4周治疗一次,分2批依次进行。用药后最常见的不良反应是自限性注射部位皮疹(20例)。服用N-803一周后,我们观察到NK细胞增殖增强,抗肿瘤细胞毒性提高,但不会引起免疫衰竭。5名患者在服用N-803后出现急性移植物抗宿主疾病(aGVHD),但他们对类固醇治疗反应迅速,4名患者出现慢性GVHD。接受4次以上N-803治疗的患者两年后复发率降低了3倍(P=0.06)。这些研究结果表明,N-803对预防造血干细胞移植后AML/MDS复发具有安全性、免疫激活作用和潜在疗效。
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引用次数: 0
Biologically Randomized Comparison of Haploidentical Versus Human Leukocyte Antigen-Matched Related Donor Reduced-Intensity Conditioning Hematopoietic Cell Transplantation. 单倍体与 HLA 匹配的亲缘供体降低强度调理造血细胞移植的生物学随机比较。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-26 DOI: 10.1016/j.jtct.2024.09.021
Michael R Grunwald, Wei Sha, Jiaxian He, Srinivasa Sanikommu, Jonathan M Gerber, Jing Ai, Thomas G Knight, Omotayo Fasan, Victoria Boseman, Whitney Kaizen, Aleksander Chojecki, Brittany K Ragon, James Symanowski, Belinda Avalos, Edward Copelan, Nilanjan Ghosh
<p><p>Using haploidentical donors for allogeneic hematopoietic cell transplantation (HCT) broadens transplant accessibility to a growing number of patients with hematologic disorders. Moreover, haploidentical HCT with post-transplant cyclophosphamide (PTCy) has become widespread practice due to accumulating evidence demonstrating favorable rates of survival and graft-versus-host disease (GvHD). Most studies comparing outcomes by donor sources have been confounded by variability in conditioning regimens, graft type (peripheral blood [PB] or bone marrow), and post-transplant GvHD prophylaxis (PTCy or non-PTCy), making it difficult to define the effect of donor source on outcomes. Levine Cancer Institute started a transplant and cellular therapy program in 2014, with both haploidentical and matched related donor (MRD) transplants initially performed using a uniform reduced-intensity conditioning (RIC) regimen, PB grafts, and PTCy-based GvHD prophylaxis. This retrospective observational study was conducted to compare the clinical outcomes associated with RIC haploidentical HCT and MRD HCT in patients receiving identical conditioning regimens, graft types, and supportive care. Our transplant database was queried to evaluate demographic characteristics, clinical features, and outcomes of RIC HCT for consecutive patients with hematologic malignancies who received haploidentical or MRD grafts between March 2014 and December 2017. An MRD was the preferred donor source; when unavailable, a haploidentical donor was used. Sixty-seven patients underwent haploidentical HCT and 25 MRD HCT. Overall, characteristics of transplant recipients were similar for the haploidentical and MRD groups; however, haploidentical donors were younger than MRDs (median 36 yr versus 57 yr, P < .0001). Results of univariable analysis showed similar overall survival (OS) for haploidentical and MRD HCT (hazard ratio [HR], 1.15; 95% CI, 0.61 to 2.15; P = .669). One-year, 1-yr, and 5-yr OS were 80.2%, 54.7%, and 41.2% for haploidentical HCT and 76.0%, 55.7%, and 51.1% for MRD HCT, respectively. With a median follow-up of 81.90 months, results of multivariable analysis revealed that donor source (haploidentical versus MRD) was not significantly associated with OS (HR, 0.97; 95% CI, 0.51 to 1.87; P = .933), relapse-free survival (HR, 0.75; 95% CI, 0.42 to 1.35; P = .337), cumulative incidence of relapse (HR, 0.81; 95% CI, 0.39 to 1.70; P = .579), or non-relapse mortality (HR, 1.12; 95% CI, 0.40 to 3.14; P = .827). Cumulative incidences of acute GvHD (aGvHD) and chronic GvHD (cGvHD) were not significantly different for haploidentical and MRD HCT (grades II to IV aGvHD: HR, 1.78; 95% CI, 0.72 to 4.37; P = .210; grades III to IV aGvHD: HR, 2.84; 95% CI, 0.34 to 23.63; P = .335; cGvHD: HR, 1.00; 95% CI 0.36 to 2.76; P = .995). With care that was homogenous in terms of conditioning regimens, graft type, GvHD prophylaxis, and supportive care, 92 patients who were biologically randomized to eit
背景:使用单倍体供体进行异基因造血细胞移植(HCT)扩大了移植的可及性,使越来越多的血液病患者可以接受移植。此外,由于越来越多的证据表明单倍体造血干细胞移植后环磷酰胺(PTCy)的存活率和移植物抗宿主病(GvHD)的发生率较高,因此单倍体造血干细胞移植已成为一种普遍的治疗方法。大多数比较不同供体来源治疗效果的研究都受到调理方案、移植物类型(外周血或骨髓)和移植后抗宿主病预防措施(PTCy 或非 PTCy)差异的影响,因此很难界定供体来源对治疗效果的影响。莱文癌症研究所(Levine Cancer Institute)于2014年启动了一项移植和细胞治疗项目,最初采用统一的降低强度调理(RIC)方案、外周血移植和基于PTCy的GvHD预防措施进行单倍体和配对亲缘供体(MRD)移植:这项回顾性观察研究旨在比较RIC单倍体造血干细胞移植和MRD造血干细胞移植对接受相同调理方案、移植物类型和支持治疗的患者的临床效果:对我们的移植数据库进行了查询,以评估2014年3月至2017年12月期间接受单倍体或MRD移植物的连续血液恶性肿瘤患者的人口统计学特征、临床特征和RIC HCT的结果。MRD是首选的供体来源;如果无法获得,则使用单倍体供体:67名患者接受了单倍体造血干细胞移植,25名患者接受了MRD造血干细胞移植。总体而言,单倍体组和 MRD 组移植受者的特征相似;但单倍体供者比 MRD 供者年轻(中位年龄为 36 岁对 57 岁,P 结论:单倍体组和 MRD 组移植受者的特征相似,但单倍体供者比 MRD 供者年轻(中位年龄为 36 岁对 57 岁,P 结论):在调理方案、移植类型、GvHD预防和支持治疗等方面相同的情况下,92名在PTCy进行RIC后生物随机接受单倍体HCT或MRD HCT的患者的治疗效果相当。
{"title":"Biologically Randomized Comparison of Haploidentical Versus Human Leukocyte Antigen-Matched Related Donor Reduced-Intensity Conditioning Hematopoietic Cell Transplantation.","authors":"Michael R Grunwald, Wei Sha, Jiaxian He, Srinivasa Sanikommu, Jonathan M Gerber, Jing Ai, Thomas G Knight, Omotayo Fasan, Victoria Boseman, Whitney Kaizen, Aleksander Chojecki, Brittany K Ragon, James Symanowski, Belinda Avalos, Edward Copelan, Nilanjan Ghosh","doi":"10.1016/j.jtct.2024.09.021","DOIUrl":"10.1016/j.jtct.2024.09.021","url":null,"abstract":"&lt;p&gt;&lt;p&gt;Using haploidentical donors for allogeneic hematopoietic cell transplantation (HCT) broadens transplant accessibility to a growing number of patients with hematologic disorders. Moreover, haploidentical HCT with post-transplant cyclophosphamide (PTCy) has become widespread practice due to accumulating evidence demonstrating favorable rates of survival and graft-versus-host disease (GvHD). Most studies comparing outcomes by donor sources have been confounded by variability in conditioning regimens, graft type (peripheral blood [PB] or bone marrow), and post-transplant GvHD prophylaxis (PTCy or non-PTCy), making it difficult to define the effect of donor source on outcomes. Levine Cancer Institute started a transplant and cellular therapy program in 2014, with both haploidentical and matched related donor (MRD) transplants initially performed using a uniform reduced-intensity conditioning (RIC) regimen, PB grafts, and PTCy-based GvHD prophylaxis. This retrospective observational study was conducted to compare the clinical outcomes associated with RIC haploidentical HCT and MRD HCT in patients receiving identical conditioning regimens, graft types, and supportive care. Our transplant database was queried to evaluate demographic characteristics, clinical features, and outcomes of RIC HCT for consecutive patients with hematologic malignancies who received haploidentical or MRD grafts between March 2014 and December 2017. An MRD was the preferred donor source; when unavailable, a haploidentical donor was used. Sixty-seven patients underwent haploidentical HCT and 25 MRD HCT. Overall, characteristics of transplant recipients were similar for the haploidentical and MRD groups; however, haploidentical donors were younger than MRDs (median 36 yr versus 57 yr, P &lt; .0001). Results of univariable analysis showed similar overall survival (OS) for haploidentical and MRD HCT (hazard ratio [HR], 1.15; 95% CI, 0.61 to 2.15; P = .669). One-year, 1-yr, and 5-yr OS were 80.2%, 54.7%, and 41.2% for haploidentical HCT and 76.0%, 55.7%, and 51.1% for MRD HCT, respectively. With a median follow-up of 81.90 months, results of multivariable analysis revealed that donor source (haploidentical versus MRD) was not significantly associated with OS (HR, 0.97; 95% CI, 0.51 to 1.87; P = .933), relapse-free survival (HR, 0.75; 95% CI, 0.42 to 1.35; P = .337), cumulative incidence of relapse (HR, 0.81; 95% CI, 0.39 to 1.70; P = .579), or non-relapse mortality (HR, 1.12; 95% CI, 0.40 to 3.14; P = .827). Cumulative incidences of acute GvHD (aGvHD) and chronic GvHD (cGvHD) were not significantly different for haploidentical and MRD HCT (grades II to IV aGvHD: HR, 1.78; 95% CI, 0.72 to 4.37; P = .210; grades III to IV aGvHD: HR, 2.84; 95% CI, 0.34 to 23.63; P = .335; cGvHD: HR, 1.00; 95% CI 0.36 to 2.76; P = .995). With care that was homogenous in terms of conditioning regimens, graft type, GvHD prophylaxis, and supportive care, 92 patients who were biologically randomized to eit","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354544","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
A Multicenter Analysis of Allogeneic Transplant Outcomes in Adults with Philadelphia-like B-cell Acute Lymphoblastic Leukemia in First Complete Remission. 首次完全缓解的费城样 B 细胞急性淋巴细胞白血病成人异基因移植结果的多中心分析。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-25 DOI: 10.1016/j.jtct.2024.09.020
Zaid Abdel Rahman, Tamer Othman, Rima M Saliba, Yenny Alejandra Moreno Vanegas, Razan Mohty, Celina Ledesma, Gabriela Rondon, Nitin Jain, Elias Jabbour, Vinod Pullarkat, Hassan B Alkhateeb, Hagop M Kantarjian, Patricia T Greipp, Ryotaro Nakamura, Mohamed A Kharfan-Dabaja, Richard E Champlin, Stephen J Forman, Elizabeth J Shpall, Mark R Litzow, James M Foran, Ibrahim Aldoss, Paul B Koller, Partow Kebriaei

Background: Philadelphia-like acute lymphoblastic leukemia (Ph-like ALL) is a high-risk subset of B-cell ALL with a poor prognosis with conventional therapies. Diagnostic challenges and lack of standardized treatment protocols contribute to suboptimal outcomes. Additionally, while allogeneic hematopoietic cell transplantation (HCT) is frequently recommended in adults with Ph-like ALL given its high-risk nature, data supporting its role remains limited.

Objective: We conducted a multicenter retrospective study evaluating outcomes of adult patients undergoing HCT in first complete remission (CR1) for Ph-like ALL compared to Philadelphia chromosome positive ALL (Ph-pos) and other B-cell Philadelphia negative (Ph-neg) ALL.

Study design: Data was collected from from five academic centers across the US, focusing on HCT outcomes for patients with ALL. Patients undergoing HCT in CR1 between 2006 and 2021 were included.

Results: Among 673 patients, 83 (12.3%) had Ph-like ALL, while 271 (40.3%) had Ph-pos and 319 (47.4%) had Ph-neg ALL. Outcomes following HCT in CR1 for Ph-like ALL were comparable to Ph-neg ALL, with no significant differences in 3-year overall survival (66% vs 59%, p=0.1), progression-free survival (59% and 54%, p=0.1), or relapse rates (22% vs 20%, p=0.7). In contrast, Ph-pos ALL had superior outcomes; 3-year OS (75%, p<0.001), PFS (70%, p=0.001) and relapse (12%, p=0.003), this is likely attributed to tyrosine kinase inhibitor therapy.

Conclusion: Our study suggests that HCT, coupled with effective 2nd line therapies can possibly mitigate the poor prognosis associated with Ph-like ALL and offers promising outcomes for patients with Ph-like ALL.

背景:费城样急性淋巴细胞白血病(Ph-like ALL)是B细胞ALL的高危亚型,传统疗法预后较差。诊断难题和标准化治疗方案的缺乏导致疗效不理想。此外,虽然异基因造血细胞移植(HCT)经常被推荐用于高危的成人类Ph-like ALL患者,但支持其作用的数据仍然有限:我们开展了一项多中心回顾性研究,评估了首次完全缓解(CR1)的Ph样ALL成人患者接受HCT治疗的疗效,并与费城染色体阳性ALL(Ph-pos)和其他B细胞费城阴性ALL(Ph-neg)进行了比较:研究设计:从全美五个学术中心收集数据,重点关注ALL患者的HCT疗效。研究结果:在673名患者中,83人(12人)接受了HCT治疗:在673名患者中,83人(12.3%)为Ph-like ALL,271人(40.3%)为Ph-pos ALL,319人(47.4%)为Ph-neg ALL。Ph-样ALL在CR1期接受造血干细胞移植后的结果与Ph-neg ALL相当,3年总生存率(66% vs 59%,P=0.1)、无进展生存率(59%和54%,P=0.1)或复发率(22% vs 20%,P=0.7)均无显著差异。相比之下,Ph-pos ALL 的疗效更佳;3 年 OS(75%,p=0.1),或复发率(22% vs 20%,p=0.7):我们的研究表明,造血干细胞移植与有效的二线疗法相结合,有可能减轻与Ph-like ALL相关的不良预后,并为Ph-like ALL患者提供有希望的治疗结果。
{"title":"A Multicenter Analysis of Allogeneic Transplant Outcomes in Adults with Philadelphia-like B-cell Acute Lymphoblastic Leukemia in First Complete Remission.","authors":"Zaid Abdel Rahman, Tamer Othman, Rima M Saliba, Yenny Alejandra Moreno Vanegas, Razan Mohty, Celina Ledesma, Gabriela Rondon, Nitin Jain, Elias Jabbour, Vinod Pullarkat, Hassan B Alkhateeb, Hagop M Kantarjian, Patricia T Greipp, Ryotaro Nakamura, Mohamed A Kharfan-Dabaja, Richard E Champlin, Stephen J Forman, Elizabeth J Shpall, Mark R Litzow, James M Foran, Ibrahim Aldoss, Paul B Koller, Partow Kebriaei","doi":"10.1016/j.jtct.2024.09.020","DOIUrl":"https://doi.org/10.1016/j.jtct.2024.09.020","url":null,"abstract":"<p><strong>Background: </strong>Philadelphia-like acute lymphoblastic leukemia (Ph-like ALL) is a high-risk subset of B-cell ALL with a poor prognosis with conventional therapies. Diagnostic challenges and lack of standardized treatment protocols contribute to suboptimal outcomes. Additionally, while allogeneic hematopoietic cell transplantation (HCT) is frequently recommended in adults with Ph-like ALL given its high-risk nature, data supporting its role remains limited.</p><p><strong>Objective: </strong>We conducted a multicenter retrospective study evaluating outcomes of adult patients undergoing HCT in first complete remission (CR1) for Ph-like ALL compared to Philadelphia chromosome positive ALL (Ph-pos) and other B-cell Philadelphia negative (Ph-neg) ALL.</p><p><strong>Study design: </strong>Data was collected from from five academic centers across the US, focusing on HCT outcomes for patients with ALL. Patients undergoing HCT in CR1 between 2006 and 2021 were included.</p><p><strong>Results: </strong>Among 673 patients, 83 (12.3%) had Ph-like ALL, while 271 (40.3%) had Ph-pos and 319 (47.4%) had Ph-neg ALL. Outcomes following HCT in CR1 for Ph-like ALL were comparable to Ph-neg ALL, with no significant differences in 3-year overall survival (66% vs 59%, p=0.1), progression-free survival (59% and 54%, p=0.1), or relapse rates (22% vs 20%, p=0.7). In contrast, Ph-pos ALL had superior outcomes; 3-year OS (75%, p<0.001), PFS (70%, p=0.001) and relapse (12%, p=0.003), this is likely attributed to tyrosine kinase inhibitor therapy.</p><p><strong>Conclusion: </strong>Our study suggests that HCT, coupled with effective 2<sup>nd</sup> line therapies can possibly mitigate the poor prognosis associated with Ph-like ALL and offers promising outcomes for patients with Ph-like ALL.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354541","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
A Peer Support Intervention in Patients with Hematologic Malignancies Undergoing Hematopoietic Stem Cell Transplantation (HSCT): The STEPP Proof-of-Concept Trial. 对接受造血干细胞移植(HSCT)的血液恶性肿瘤患者进行同伴支持干预:STEPP 概念验证试验。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-25 DOI: 10.1016/j.jtct.2024.09.022
Hermioni L Amonoo, Michelle Guo, Emma P Keane, Annabella C Boardman, M Tim Song, Emma D Wolfe, Corey Cutler, Heather S Jim, Stephanie J Lee, Jeff C Huffman, Areej El-Jawahri

Background: Although peer support interventions are associated with improved patient-reported outcomes in diverse cancer populations, structured peer support programs tailored to the needs of patients undergoing hematopoietic stem cell transplantation (HSCT) are lacking.

Objective: This single-arm, proof-of-concept trial aimed to refine the Supporting Transplant Experiences with Peer Program (STEPP), a structured, five-session, manualized, phone-delivered peer support intervention for patients undergoing HSCT, informed by qualitative feedback from patients.

Study design: Adult patients with hematologic malignancies scheduled to undergo allogeneic or autologous HSCT were eligible to participate in the study approximately two weeks prior to their HSCT hospitalization. Participants received the STEPP intervention, which focused on providing informational, emotional, and practical support. To refine the intervention, we conducted semi-structured qualitative exit interviews to gather feedback on the content of STEPP and to identify facilitators and barriers to engagement. Transcribed interviews were analyzed using rapid analytic methods by two coders.

Results: Of the 37 eligible patients, 25 enrolled in the study, 20 completed all intervention sessions and 20 completed exit interviews. Participants highlighted that discussions with peer mentors/STEPP interventionists about the transplant journey and processing information provided by the clinical team were the most valuable aspects of STEPP. Positive experiences during the first intervention session facilitated patient engagement with the program. Potential barriers to engagement included logistical challenges in connecting with interventionists while experiencing physical symptoms during inpatient hospitalization and being paired with an interventionist who had a different cancer diagnosis and/or type of transplant.

Conclusions: Patients undergoing HSCT reported positive experiences with the structured five-session, phone-delivered peer support intervention administered before and during the HSCT hospitalization. Patients' descriptions of barriers and facilitators to engagement with the STEPP intervention underscore the importance of patient input and programmatic structure in peer support interventions for this population. Insights from this proof-of-concept trial will be incorporated into future trials of STEPP to improve outcomes in HSCT recipients.

背景:尽管同伴支持干预与不同癌症人群中患者报告结果的改善有关,但目前还缺乏针对造血干细胞移植(HSCT)患者需求的结构化同伴支持计划:这项单臂概念验证试验旨在完善 "同伴支持移植体验计划"(Supporting Transplant Experiences with Peer Program,STEPP),这是一项针对造血干细胞移植患者的结构化、五次课、手册化、电话传递的同伴支持干预措施,并参考了患者的定性反馈:研究设计:计划接受异体或自体造血干细胞移植的成年血液恶性肿瘤患者在造血干细胞移植住院前两周左右有资格参与研究。参与者接受 STEPP 干预,该干预侧重于提供信息、情感和实际支持。为了完善干预措施,我们进行了半结构化定性退出访谈,以收集对 STEPP 内容的反馈意见,并确定参与的促进因素和障碍。访谈记录由两名编码员采用快速分析方法进行分析:在 37 名符合条件的患者中,25 人参加了研究,20 人完成了所有干预课程,20 人完成了退出访谈。参与者强调,与同伴导师/STEPP 干预人员讨论移植历程和处理临床团队提供的信息是 STEPP 最有价值的方面。第一次干预过程中的积极体验促进了患者参与该计划。参与的潜在障碍包括:在住院期间出现身体症状时与干预人员联系的后勤挑战,以及与不同癌症诊断和/或移植类型的干预人员配对:接受造血干细胞移植的患者对在造血干细胞移植住院前和住院期间通过电话提供的五节同伴支持干预报告了积极的体验。患者对参与 STEPP 干预的障碍和促进因素的描述强调了患者意见和计划结构在针对该人群的同伴支持干预中的重要性。这项概念验证试验的启示将被纳入 STEPP 未来的试验中,以改善造血干细胞移植受者的治疗效果。
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引用次数: 0
Officers and Directors of ASTCT ASTCT 的官员和董事
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-24 DOI: 10.1016/S2666-6367(24)00633-X
{"title":"Officers and Directors of ASTCT","authors":"","doi":"10.1016/S2666-6367(24)00633-X","DOIUrl":"10.1016/S2666-6367(24)00633-X","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142314200","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
Toward a Cure for Autoimmune Diseases 治疗自身免疫性疾病
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-24 DOI: 10.1016/j.jtct.2024.09.001
{"title":"Toward a Cure for Autoimmune Diseases","authors":"","doi":"10.1016/j.jtct.2024.09.001","DOIUrl":"10.1016/j.jtct.2024.09.001","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142314202","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
Rarity & Risk of Secondary Hematologic Neoplasms after CAR T-cell Therapies CAR T 细胞疗法后继发性血液肿瘤的罕见性和风险
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-24 DOI: 10.1016/j.jtct.2024.09.002
{"title":"Rarity & Risk of Secondary Hematologic Neoplasms after CAR T-cell Therapies","authors":"","doi":"10.1016/j.jtct.2024.09.002","DOIUrl":"10.1016/j.jtct.2024.09.002","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142314203","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
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Transplantation and Cellular Therapy
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