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Corrigendum to: "Comparison of three strategies of GvHD prophylaxis after T-cell replete haploidentical hematopoietic transplantation: Tacrolimus vs calcineurin inhibitors-MMF vs sirolimus-MMF" Journal: Transplant Cell Ther. 2024 Oct;30(10):1025.e1-1025.e14. doi: 10.1016/j.jtct.2024.07.027. 《t细胞全单倍相同造血移植后预防GvHD的三种策略的比较:他克莫司与钙调神经磷酸酶抑制剂- mmf vs西罗莫司- mmf》杂志:移植细胞杂志,2024年10月;30(10):1025.01 - 1025.06。doi: 10.1016 / j.jtct.2024.07.027。
IF 3.6 3区 医学
Transplantation and Cellular Therapy Pub Date : 2025-06-12 DOI: 10.1016/j.jtct.2025.06.011
Albert Esquirol, Maria Jesus Pascual, Juan Montoro, José Luis Piñana, Christelle Ferrà, Beatriz Herruzo, Irene Garcia-Cadenas, Aitana Balaguer, Ariadna Perez, Maria Huguet, Sara Redondo, Marta Villalba, Juan Carlos Hernandez-Boluda, Pedro Chorao, Rafael Hernani, Jaime Sanz, Carlos Solano, Jorge Sierra, Rodrigo Martino
{"title":"Corrigendum to: \"Comparison of three strategies of GvHD prophylaxis after T-cell replete haploidentical hematopoietic transplantation: Tacrolimus vs calcineurin inhibitors-MMF vs sirolimus-MMF\" Journal: Transplant Cell Ther. 2024 Oct;30(10):1025.e1-1025.e14. doi: 10.1016/j.jtct.2024.07.027.","authors":"Albert Esquirol, Maria Jesus Pascual, Juan Montoro, José Luis Piñana, Christelle Ferrà, Beatriz Herruzo, Irene Garcia-Cadenas, Aitana Balaguer, Ariadna Perez, Maria Huguet, Sara Redondo, Marta Villalba, Juan Carlos Hernandez-Boluda, Pedro Chorao, Rafael Hernani, Jaime Sanz, Carlos Solano, Jorge Sierra, Rodrigo Martino","doi":"10.1016/j.jtct.2025.06.011","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.06.011","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295009","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
Evaluating the Role of CAR-T Cell Therapy in the Context of Current Therapy Options for Patients With Relapsed or Refractory Follicular Lymphoma. 评估CAR-T细胞治疗在复发或难治性滤泡性淋巴瘤患者当前治疗方案中的作用
IF 3.6 3区 医学
Transplantation and Cellular Therapy Pub Date : 2025-06-12 DOI: 10.1016/j.jtct.2025.06.013
Julio C Chavez, Marc S Hoffmann, Leslie L Popplewell
{"title":"Evaluating the Role of CAR-T Cell Therapy in the Context of Current Therapy Options for Patients With Relapsed or Refractory Follicular Lymphoma.","authors":"Julio C Chavez, Marc S Hoffmann, Leslie L Popplewell","doi":"10.1016/j.jtct.2025.06.013","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.06.013","url":null,"abstract":"<p><p>Follicular lymphoma (FL), the most common subtype of indolent non-Hodgkin lymphoma, exhibits significant clinical heterogeneity, with some patients enjoying durable periods of active surveillance and others having a more aggressive course characterized by frequent relapses and sometimes transformation to high-grade lymphoma. Consequently, treatment is highly individualized. Currently, there is no standard regimen established for patients with relapsed or refractory (r/r) FL. The only established curative-intent treatment for r/r FL is hematopoietic stem cell transplantation, but its application is limited by toxicity. Currently there is a need for effective therapies that could provide longer disease control without significant increase in toxicities. Agents in development include chimeric antigen receptor (CAR)-T cell therapy, monoclonal anti-cluster of differentiation (CD)20 antibodies, kinase inhibitors, enhancer of zeste homolog 2 inhibitors, cereblon E3 ligase modulatory drugs, and bispecific antibodies. Some of these therapies have already been approved for use in patients with r/r FL with ≥2 previous lines of therapy, but sequencing and standardization of treatment are still lacking. CAR-T cell therapy has been shown to have durable efficacy with manageable adverse events, such as cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome. Bispecific antibodies have been shown to demonstrate a good overall response rate but their long-term efficacy has not been established. Several trials on targeted therapies have also shown promising results. Clinical trials using a combination of these therapeutic agents are still limited, as are real-world studies in patients with r/r FL given cellular therapy. Despite this expansion of the treatment landscape among patients with third-line r/r FL, there still exists an unmet need for a standardized, stepwise approach in the treatment of this population. Herein we review the efficacy and safety of CAR-T cell therapy and non-CAR-T cell therapy in the management of r/r FL.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295010","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
An ASTCT, CIBMTR, EBMT, and APBMT Consensus Statement Defining Response Criteria for Hematopoietic Cell Transplantation Associated Thrombotic Microangiopathy (TA-TMA) Directed Therapy: Consensus Response Criteria for TA-TMA. 一项ASTCT、CIBMTR、EBMT和APBMT共识声明,定义了造血细胞移植相关血栓性微血管病(TA-TMA)指导治疗的反应标准:TA-TMA的一致反应标准。
IF 3.6 3区 医学
Transplantation and Cellular Therapy Pub Date : 2025-06-11 DOI: 10.1016/j.jtct.2025.05.028
Michelle L Schoettler, Eleni Gavriilaki, Enric Carreras, Bo-Kyoung Cho, Christopher E Dandoy, Vincent T Ho, Sonata Jodele, Ivan Moiseev, Isabella Sánchez-Ortega, Alok Srivastava, Yoshiko Atsuta, Paul A Carpenter, John Koreth, Nicolaus Kröger, Per Ljungman, Kristen Page, Uday Popat, Bronwen E Shaw, Ana Maria Sureda, Robert Soiffer, Sumithira Vasu
{"title":"An ASTCT, CIBMTR, EBMT, and APBMT Consensus Statement Defining Response Criteria for Hematopoietic Cell Transplantation Associated Thrombotic Microangiopathy (TA-TMA) Directed Therapy: Consensus Response Criteria for TA-TMA.","authors":"Michelle L Schoettler, Eleni Gavriilaki, Enric Carreras, Bo-Kyoung Cho, Christopher E Dandoy, Vincent T Ho, Sonata Jodele, Ivan Moiseev, Isabella Sánchez-Ortega, Alok Srivastava, Yoshiko Atsuta, Paul A Carpenter, John Koreth, Nicolaus Kröger, Per Ljungman, Kristen Page, Uday Popat, Bronwen E Shaw, Ana Maria Sureda, Robert Soiffer, Sumithira Vasu","doi":"10.1016/j.jtct.2025.05.028","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.028","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Transplant associated thrombotic microangiopathy (TA-TMA) confers significant morbidity and mortality in hematopoietic cell transplant recipients. The safety and efficacy of multiple TA-TMA directed therapeutic agents are being tested in ongoing clinical trials. In the absence of approved drugs, several treatments are used off-label. Response definitions to TA-TMA directed therapy from retrospective studies and ongoing interventional clinical trials vary widely, limiting cross study comparisons. An expert panel from multiple international blood and marrow transplant societies (ASTCT, CIBMTR, EBMT, APBMT) who initially convened to harmonize diagnostic and risk stratification criteria for TA-TMA extended its mandate to review response criteria.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;Our objective was to propose clinically meaningful response criteria for TA-TMA directed therapy to enhance consistent evaluation of therapeutic agents in clinical practice, interventional trials, and registry studies.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;After a relevant literature review, the Delphi method was used to achieve consensus on proposed response criteria.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The panel focused on the three key concepts. First, due to ongoing concurrent co-morbidities and severity of illness, the complete resolution of TA-TMA manifestations may be difficult to achieve immediately after initiating treatment, making the definition of clinically meaningful partial responses essential to assess early efficacy of TA-TMA-directed therapies. Second, because hematologic manifestations may resolve faster than organ damage, we suggest assessing hematologic/biochemical and organ manifestations independently, in addition to having an overall response assessment. Finally, using the previously established diagnostic criteria as a framework, we propose objective response definitions for each TA-TMA criterion and organ manifestation. While consensus was achieved on response definitions, due to the lack of evidence, there was no agreement on standardized time points for assessing response or when to consider alternative therapies for patients unresponsive to initial treatments. Hematologic and biochemical response assessments include anemia and thrombocytopenia, with criteria accounting for transfusion dependence or independence at the time of treatment, schistocytes, lactate dehydrogenase, and soluble C5b-9. Patients with other established etiologies of cytopenias (i.e. poor graft function or relapsed hematologic malignancy) should be considered unevaluable for hematologic response. Responses for involved organ manifestations were also proposed. In an overall assessment, the best overall response is limited by the lowest hematologic/biochemical or organ response. NR of either hematologic/biochemical or organ is considered an overall NR.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;The consensus response criteria proposed by this expert panel are a ste","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295004","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
Conditioning regimens for second allogeneic hematopoietic stem cell transplantation for patients with primary graft failure. 原发性移植失败患者第二次异基因造血干细胞移植的调理方案。
IF 3.6 3区 医学
Transplantation and Cellular Therapy Pub Date : 2025-06-11 DOI: 10.1016/j.jtct.2025.06.007
Nihar Desai, Sergio Rodriguez Rodriguez, Carol Chen, Eshetu G Atenafu, Tommy Alfaro-Moya, Arjun Datt Law, Eshrak Al-Shaibani, Igor Novitzky-Basso, Ivan Pasic, Fotios V Michelis, Auro Viswabandya, Dennis D Kim, Jonas Mattsson, Rajat Kumar
{"title":"Conditioning regimens for second allogeneic hematopoietic stem cell transplantation for patients with primary graft failure.","authors":"Nihar Desai, Sergio Rodriguez Rodriguez, Carol Chen, Eshetu G Atenafu, Tommy Alfaro-Moya, Arjun Datt Law, Eshrak Al-Shaibani, Igor Novitzky-Basso, Ivan Pasic, Fotios V Michelis, Auro Viswabandya, Dennis D Kim, Jonas Mattsson, Rajat Kumar","doi":"10.1016/j.jtct.2025.06.007","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.06.007","url":null,"abstract":"<p><strong>Background: </strong>Primary graft failure (PGF) is a life-threatening complication of allogeneic hematopoietic stem cell transplantation (HSCT). The optimal conditioning strategies for salvage HSCT in PGF remain undefined.</p><p><strong>Objectives and methods: </strong>We retrospectively analysed the outcomes of 19 patients with PGF who underwent a second HSCT between 2017 and 2024. Eleven patients (58%) received a novel one-day conditioning regimen comprising fludarabine, cyclophosphamide, alemtuzumab, and low-dose total body irradiation (Group I), while eight received a multi-day reduced intensity conditioning regimen (fludarabine-busulfan-2Gy total body irradiation) (Group II).</p><p><strong>Results: </strong>All patients in Group I engrafted neutrophils compared to 50% in Group II. The cumulative incidence of neutrophil engraftment at day +28 was 82% in Group I and 50% in Group II (p=0.22). Platelet engraftment by day +28 was observed in 70% of patients in Group I and 54% in Group II (p=0.61). The median follow-up of survivors after second HSCT was 16.5 months (95% CI: 5.9-39). The 12-month overall survival (OS) was 53.3% in Group I and 37.5% in Group II (p=0.29). The day +100 non-relapse mortality (NRM) was 30.3% in Group I and 62.5% in Group II (p=0.12). No patients developed grade III-IV acute graft-versus-host disease (GvHD) or chronic GvHD.</p><p><strong>Conclusion: </strong>A one-day alemtuzumab-based conditioning regimen for salvage HSCT appears to be well tolerated and may be associated with improved engraftment, NRM, and OS when compared to the fludarabine-busulfan-2Gy total body irradiation regimen.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295008","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
Association of Cyclosporine Exposure Post-Allogeneic Hematopoietic Cell Transplant with Graft Failure and Relapse Risk in Hematological Malignancies. 异基因造血细胞移植后环孢素暴露与恶性血液病移植失败和复发风险的关系
IF 3.6 3区 医学
Transplantation and Cellular Therapy Pub Date : 2025-06-11 DOI: 10.1016/j.jtct.2025.05.024
Ahmed Alnughmush, Mats Remberger, Vathany Kulasingam, Ivan Pasic, Igor Novitzky-Basso, Arjun D Law, Wilson Lam, Dennis D Kim, Armin Gerbitz, Rajat Kumar, Auro Viswabandya, Jonas Mattsson, Fotios V Michelis
{"title":"Association of Cyclosporine Exposure Post-Allogeneic Hematopoietic Cell Transplant with Graft Failure and Relapse Risk in Hematological Malignancies.","authors":"Ahmed Alnughmush, Mats Remberger, Vathany Kulasingam, Ivan Pasic, Igor Novitzky-Basso, Arjun D Law, Wilson Lam, Dennis D Kim, Armin Gerbitz, Rajat Kumar, Auro Viswabandya, Jonas Mattsson, Fotios V Michelis","doi":"10.1016/j.jtct.2025.05.024","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.024","url":null,"abstract":"<p><strong>Background: </strong>Allogeneic hematopoietic cell transplantation (HCT) is a cornerstone in the treatment of many high-risk hematological malignancies. Calcineurin inhibitors (CNIs), essential components of GVHD prophylaxis, require careful monitoring of levels to optimize outcomes. This study evaluated the association between cyclosporine (CsA) exposure during the first 90 days post-HCT and key transplant outcomes.</p><p><strong>Methods: </strong>A retrospective analysis was performed on 373 patients who underwent allogeneic HCT between January 2019 and July 2021 at the Princess Margaret Cancer Centre. CsA trough levels were routinely measured, and area under the curve for the first 90 days post-transplant (AUC90) was calculated. Associations between CsA AUC90 and graft failure (GF), relapse, acute and chronic GVHD (aGVHD, cGVHD), and infections were assessed.</p><p><strong>Results: </strong>In a cohort predominantly receiving contemporary GVHD prophylaxis with PTCy-based regimens, higher CsA AUC90 was significantly associated with a reduced risk of GF (p<0.001) and lower grades of aGVHD (p<0.001). Univariable analysis confirmed that higher CsA exposure was linked to a lower risk of GF (HR 0.22, p<0.001). CsA AUC90, however, was not associated with relapse risk (p=0.56) or cGVHD severity (p=0.38). Increased CsA exposure was associated with a higher risk of CMV reactivation (p=0.03), though this risk was mitigated in patients receiving letermovir prophylaxis (p=0.44).</p><p><strong>Conclusion: </strong>This study underscores the importance of CsA monitoring to reduce the risks of GF and aGVHD without increasing relapse in hematological malignancies. However, higher CsA exposure requires careful management due to its association with CMV reactivation. These findings contribute to optimizing immunosuppression strategies in the context of contemporary GVHD prophylaxis.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295005","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
Seizure prophylaxis and its impact on busulfan pharmacokinetics and dosing in a novel timed sequential protocol: MD Anderson experience. 癫痫预防及其对布苏凡药代动力学和剂量的影响在一个新的时间顺序协议:MD安德森经验。
IF 3.6 3区 医学
Transplantation and Cellular Therapy Pub Date : 2025-06-11 DOI: 10.1016/j.jtct.2025.05.029
Jitesh D Kawedia, Victoria W Handy, Alan L Myers, Alison M Gulbis, Rohtesh Mehta, Richard E Champlin, Elizabeth J Shpall, Yago Nieto, Borje S Andersson, Uday R Popat
{"title":"Seizure prophylaxis and its impact on busulfan pharmacokinetics and dosing in a novel timed sequential protocol: MD Anderson experience.","authors":"Jitesh D Kawedia, Victoria W Handy, Alan L Myers, Alison M Gulbis, Rohtesh Mehta, Richard E Champlin, Elizabeth J Shpall, Yago Nieto, Borje S Andersson, Uday R Popat","doi":"10.1016/j.jtct.2025.05.029","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.029","url":null,"abstract":"<p><p>High dose busulfan (Bu) is widely used in conditioning regimens for patients undergoing hematopoietic stem cell transplantation. Like all alkylating agents, it has significant inter-patient pharmacokinetic (PK) variability. The influence of Bu plasma exposure on treatment outcomes and toxicities have led to the continued use of therapeutic drug monitoring of Bu. We investigated Bu PK in a unique myeloablative fractionated Bu regimen, designed to lower non-relapse mortality in older patients and those with comorbidities. Intravenous (IV) Bu was administered once daily for 6 days, with a 7-day break after the first 2 Bu doses. Since seizures are a major risk of high-dose Bu, phenytoin was given as seizure prophylaxis. In an interim analysis, we noted a substantially increased (14.7%) Bu clearance on day -6 compared with day -13. When phenytoin was replaced by levetiracetam, Bu clearance increased by only 4.9% between days -13 and -6, significantly lower than that observed with phenytoin (p=0.00001). These results indicate the presence of a drug-drug interaction (DDI) between Bu and phenytoin. There was no difference in efficacy of seizure prophylaxis between phenytoin and levetiracetam, and there were no experienced adverse events related to levetiracetam. Our results indicate that levetiracetam is a safe and efficacious alternative to phenytoin for seizure prophylaxis in Bu-based conditioning regimens in stem cell transplantation, notably without the significant drug-drug interaction observed with phenytoin.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295011","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
Clinical practice recommendations on the role of allogeneic hematopoietic cell transplantation and chimeric antigen receptor T-cell therapy in patients with chronic lymphocytic leukemia on behalf of the American Society for Transplantation and Cellular Therapy. 代表美国移植和细胞治疗学会就异基因造血细胞移植和嵌合抗原受体t细胞治疗在慢性淋巴细胞白血病患者中的作用提出临床实践建议。
IF 3.6 3区 医学
Transplantation and Cellular Therapy Pub Date : 2025-06-11 DOI: 10.1016/j.jtct.2025.06.002
Mohamed A Kharfan-Dabaja, Ambuj Kumar, Javier Pinilla-Ibarz, Jennifer R Brown, Mazyar Shadman, Farrukh T Awan, Saad S Kenderian, Tanya Siddiqi, Jeremy S Abramson, Taha Al-Juhaishi, Danielle M Brander, Catherine C Coombs, Richard R Furman, Nitin Jain, Nadia Khan, Nakhle S Saba, Jennifer M Collins, Amer Beitinjaneh, Deborah M Stephens, Jennifer Woyach, Mehdi Hamadani
{"title":"Clinical practice recommendations on the role of allogeneic hematopoietic cell transplantation and chimeric antigen receptor T-cell therapy in patients with chronic lymphocytic leukemia on behalf of the American Society for Transplantation and Cellular Therapy.","authors":"Mohamed A Kharfan-Dabaja, Ambuj Kumar, Javier Pinilla-Ibarz, Jennifer R Brown, Mazyar Shadman, Farrukh T Awan, Saad S Kenderian, Tanya Siddiqi, Jeremy S Abramson, Taha Al-Juhaishi, Danielle M Brander, Catherine C Coombs, Richard R Furman, Nitin Jain, Nadia Khan, Nakhle S Saba, Jennifer M Collins, Amer Beitinjaneh, Deborah M Stephens, Jennifer Woyach, Mehdi Hamadani","doi":"10.1016/j.jtct.2025.06.002","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.06.002","url":null,"abstract":"<p><p>Chimeric antigen receptor T-cell therapy (CAR T-cell) is a new treatment option for relapsed and/or refractory (R/R) chronic lymphocytic leukemia (CLL). Novel therapies including Bruton's tyrosine kinase inhibitors (BTK), covalent or non-covalent, and an inhibitor of the B-cell leukemia/lymphoma 2 protein (BCL-2), venetoclax, have replaced chemoimmunotherapy (CIT) regimens in the front-line and the R/R setting, and have relegated allogeneic hematopoietic cell transplantation (allo-HCT) to later treatment stages. Updating the 2016 clinical practice recommendations on allo-HCT in CLL is necessary to help guide contemporary clinical practice. A panel of 18 physicians with diverse expertise across different CLL treatment modalities and one methodologist participated in this effort. Any recommendation receiving ≥ 70% votes was considered a consensus. CAR T-cell therapy is recommended for patients not responding or relapsing after at least 2 lines of therapy consisting of a covalent BTK inhibitor and a BCL-2 inhibitor. In addition, CAR T-cell therapy is recommended for patients who subsequently received a non-covalent BTK inhibitor in the third-line or later setting, regardless of response. CAR T-cell therapy is also recommended in CLL relapsing after an allo-HCT, assuming that patients are fit for the procedure. In those CLL patients who are candidates, allo-HCT is recommended if disease is R/R to CAR T-cell therapy provided that an objective response is demonstrated prior to the allograft. Allo-HCT is also recommended in patients with clonally-related Richter transformation (RT) after demonstrating an objective response to front-line CIT or other treatments. CAR T-cell therapy is recommended in R/R RT. We emphasize the importance of enrolling patients in clinical trials whenever available to continue to advance the field and improve prognosis of R/R CLL. We acknowledge that there are unique clinical scenarios not covered herein which may require a case-by-case approach.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295006","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
Upfront haploidentical hematopoietic cell transplantation using αβ+ T cell-depleted peripheral blood stem cells for pediatric patients with acquired severe aplastic anemia. αβ+ T细胞缺失外周血干细胞用于小儿获得性重度再生障碍性贫血的前期单倍体造血细胞移植
IF 3.6 3区 医学
Transplantation and Cellular Therapy Pub Date : 2025-06-10 DOI: 10.1016/j.jtct.2025.06.012
Sung Han Kang, Ho Joon Im, Eun Seok Choi, Soo-Hyun Yoon, Jin-Kyung Suh, Hyery Kim, Kyung-Nam Koh, Dae-Hyun Ko, Miyoung Kim, Sang-Hyun Hwang, Young-Uk Cho, Seongsoo Jang
{"title":"Upfront haploidentical hematopoietic cell transplantation using αβ<sup>+</sup> T cell-depleted peripheral blood stem cells for pediatric patients with acquired severe aplastic anemia.","authors":"Sung Han Kang, Ho Joon Im, Eun Seok Choi, Soo-Hyun Yoon, Jin-Kyung Suh, Hyery Kim, Kyung-Nam Koh, Dae-Hyun Ko, Miyoung Kim, Sang-Hyun Hwang, Young-Uk Cho, Seongsoo Jang","doi":"10.1016/j.jtct.2025.06.012","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.06.012","url":null,"abstract":"<p><strong>Background: </strong>Allogeneic hematopoietic cell transplantation (HCT) using a haploidentical family donor (HFD) is an accepted option for patients with severe aplastic anemia (SAA) without a matched related or unrelated donor. Although HFDs have been used as alternative donors for HCT in refractory SAA, upfront use requires further evidence.</p><p><strong>Objective(s): </strong>In this study, we evaluated the outcomes of ex vivo αβ<sup>+</sup> T cell-depleted haploidentical HCT (haplo-HCT) as a front-line therapy for pediatric patients with acquired SAA who were treatment-naïve.</p><p><strong>Study design: </strong>A total of 37 pediatric patients underwent haplo-HCT using ex vivo αβ<sup>+</sup>T cell-depleted peripheral blood stem cells between December 2015 and July 2024. The conditioning regimen consisted of fractionated total body irradiation (TBI) and rabbit ATG (rATG), along with fludarabine (180 mg/m<sup>2</sup>) and cyclophosphamide (100 mg/kg). Twelve patients were administered TBI at 400 cGy combined with rATG at 7.5 mg/kg, while the remaining 25 patients were administered TBI at 600 cGy with rATG at a dose of ≤ 5 mg/kg. Ex vivo depletion of αβ<sup>+</sup> T cells was the graft-versus-host disease (GVHD) prophylaxis approach in our haplo-HCT platform without immunosuppressants or with only mycophenolate mofetil (MMF) for 1month post-transplant. Donors comprised 10 mothers, 14 fathers, and 13 siblings.</p><p><strong>Results: </strong>36 patients achieved neutrophil engraftment at a median of 10 days (range, 9-12) after haplo-HCT. One patient experienced primary graft failure (GF). Another patient experienced late GF, and 1 patient had poor graft function. All of these patients were rescued with subsequent haplo-HCT. The cumulative incidence (CI) rates for ≥ grade 2 and ≥ grade 3 acute GVHD (aGVHD) were 37.1% and 11.5%, respectively. No patient developed grade 4 aGVHD. The CI for moderate-to-severe chronic GVHD (cGVHD) was 5.9%. All grade 3 aGVHD and cGVHD cases were observed among patients who received 600 cGy TBI. In contrast, CMV disease and EBV reactivation were significantly prevalent among patients who received 400 cGy compared with 600 cGy TBI (P=.016 and P≤.001). Overall, 2 patients died from transplant-related mortality, and 35 patients survived with complete donor chimerism and were free of transfusion. At a median follow-up of 60 months (range, 6-111), overall survival and failure-free survival rates were 94% ± 3.9% and 89% ± 5.3%, respectively. Survival rates were not significantly different according to the doses of TBI or rATG as part of the conditioning regimen.</p><p><strong>Conclusions: </strong>Our upfront haplo-HCT platform using αβ<sup>+</sup> T cell-depleted peripheral blood stem cellsmay be a realistic alternative for pediatric patients with acquired SAA who have no suitable related or unrelated donors.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144286588","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
Prolonged hospitalization for hematopoietic cell transplantation (HCT): Characteristics, risk factors and associations with patient-reported and clinical outcomes. 造血细胞移植(HCT)的长期住院:特征、危险因素及其与患者报告和临床结果的关联
IF 3.6 3区 医学
Transplantation and Cellular Therapy Pub Date : 2025-06-10 DOI: 10.1016/j.jtct.2025.05.026
Lucy Gao, Ashley Nelson, Anna Barata, Nora Horick, Braelyn Wekwerth, Ally Wood, Anushka Fernandes, Stephanie J Lee, Thomas W LeBlanc, Hermioni L Amonoo, Areej El-Jawahri, Richard Newcomb
{"title":"Prolonged hospitalization for hematopoietic cell transplantation (HCT): Characteristics, risk factors and associations with patient-reported and clinical outcomes.","authors":"Lucy Gao, Ashley Nelson, Anna Barata, Nora Horick, Braelyn Wekwerth, Ally Wood, Anushka Fernandes, Stephanie J Lee, Thomas W LeBlanc, Hermioni L Amonoo, Areej El-Jawahri, Richard Newcomb","doi":"10.1016/j.jtct.2025.05.026","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.026","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Patients hospitalized for hematopoietic cell transplantation (HCT) may experience prolonged length of stay (PLOS). However, the associations between PLOS and patient-reported outcomes (PROs) during and after HCT hospitalization is unknown.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;We aimed to evaluate the associations of pre-HCT demographic and disease characteristics and PROs with PLOS, as well as the associations between PLOS and trajectory of PROs and risk of rehospitalization in the first year post-HCT.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Study design: &lt;/strong&gt;We conducted a secondary analysis of data from adult patients with hematologic malignancies undergoing HCT who were enrolled in a prospective observational study or one of two randomized clinical trials evaluating integrated specialty palliative care during HCT hospitalization. PLOS was defined as ≥ 30 continuous days for allogeneic HCT and ≥ 21 continuous days for autologous HCT. Quality of life (QOL; FACT-BMT), symptom burden (ESAS), anxiety and depression symptoms (HADS and PHQ-9), and posttraumatic stress symptoms (PCL) were measured at time of admission (i.e., prior to HCT), 2 weeks, and 3- and 6- months post-HCT. Multivariate logistic regression was used to assess the association between pre-HCT PROs and PLOS adjusting for relevant covariates. Linear mixed effects models were used to characterize the trajectory of PROs by PLOS during and after HCT. Cox proportional hazards regression was used to evaluate differences between length of stay groups in time to readmission or death in the first year post-HCT.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;606 patients (mean age=55.7 years [18.3-78.0 years]; 56.6% male; 81.5% White; 53.1% allogeneic HCT) were included. Patients with PLOS were younger (mean 53.3 vs 56.6 years, p=0.004), in complete remission prior to HCT (52.8% vs 46.3%, p=0.02), diagnosed with acute leukemia (34.2% vs 26.1%, p&lt;0.001), and underwent allogeneic HCT (62.1% vs 49.9%, p&lt;0.0001). In multivariate analyses, worse pre-HCT QOL (OR 0.99, p=0.003), symptom burden (OR 1.02, p=0.01) and depressive symptoms (OR 1.07, p=0.01) were associated with higher risk of PLOS. Patients with PLOS reported worse QOL at two weeks (∆ = -12.3, p&lt;0.0001), three months (∆ = -6.9, p=0.002), and six months post-HCT (∆ =-4.8, p=0.02) compared to those without PLOS. Patients with PLOS reported greater symptom burden at two weeks (∆ =10.2, p&lt;0.0001) and three months (∆ = 3.9, p=0.04), but not six months post-HCT (∆ = 0.5, p=0.79). Patients with PLOS reported higher depression burden at two weeks (∆ = 2.5, p&lt;0.0001) and three months (∆ = 1.1, p=0.03), but not six months post-HCT (∆ = 0.6, p=0.19). Patients with PLOS experienced shorter time to death or re-admission in the first year post-HCT (median 221 days vs not reached, HR 1.7; CI 1.3-2.2, p&lt;0.001).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Pre-HCT PROs including QOL, symptom burden, and depressive symptoms were associated with PLOS. Moreover, patients w","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144286587","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
How to Improve the Sleep of Hospitalized Pediatric Patients: Family and Staff Focus Groups. 如何改善住院儿科患者的睡眠:家庭和工作人员焦点小组。
IF 3.6 3区 医学
Transplantation and Cellular Therapy Pub Date : 2025-06-07 DOI: 10.1016/j.jtct.2025.05.025
Andrea L Fidler, Gabby O'Connor, Dean W Beebe, YunZu Michele Wang, Lori E Crosby, Christopher E Dandoy
{"title":"How to Improve the Sleep of Hospitalized Pediatric Patients: Family and Staff Focus Groups.","authors":"Andrea L Fidler, Gabby O'Connor, Dean W Beebe, YunZu Michele Wang, Lori E Crosby, Christopher E Dandoy","doi":"10.1016/j.jtct.2025.05.025","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.025","url":null,"abstract":"<p><strong>Background: </strong>Pediatric patients undergoing bone marrow transplant (BMT) face lengthy hospital stays designed to support healing. However, the hospital environment, treatment side effects, and care practices can disrupt sleep, negatively impacting patients' quality of life and recovery. Prior qualitative studies focused on staff or family feedback, but none have incorporated the viewpoints of both groups. This project sought to uniquely identify barriers and solutions acceptable to multiple stakeholders.</p><p><strong>Objective: </strong>Identify barriers to good quality sleep among hospitalized BMT patients and their families, as well as actionable intervention targets.</p><p><strong>Study design: </strong>Eight semi-structured focus groups were conducted with 15 family members of hospitalized BMT patients and 20 staff at Cincinnati Children's Hospital Medical Center. Focus groups were transcribed, coded using content analysis, and reviewed in consensus meetings involving medical staff and family partners of recently hospitalized children to ensure the credibility of findings.</p><p><strong>Results: </strong>Participants described sleep as fragmented and of poor quality. Negative consequences of inadequate sleep included emotion dysregulation and reduced participation in rounds. Both family members and staff reported that medical interventions and environmental factors, such as noise and light, frequently disrupt rest. Suggested interventions included altering care schedules, improving staff-family communication, and environmental changes, like light dimming, noise reduction, and more comfortable beds. Family members were more likely to suggest interventions to support overall well-being, such as additional support for caregiver physical and mental health. Staff were more likely to suggest changing patient and family behaviors as a means to improve sleep, such as sleep schedules.</p><p><strong>Conclusion: </strong>Sleep quality in pediatric BMT patients and their families can be improved through environmental modifications and procedural adjustments, aligning with broader efforts to create sleep-friendly hospital environments. These strategies are adaptable to other care settings, improving overall patient and family well-being.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144258916","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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