Transplantation and Cellular Therapy最新文献

{"title":"Masthead (Purpose and Scope)","authors":"","doi":"10.1016/S2666-6367(25)01177-7","DOIUrl":"10.1016/S2666-6367(25)01177-7","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages A1-A2"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144212713","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}

{"title":"Awakening from REMS: ASTCT 80/20 Ongoing Recommendations for Safe Use of Chimeric Antigen Receptor T Cells","authors":"Frederick L. Locke , Zahra Mahmoudjafari , Partow Kebriaei , Rebecca A. Gardner , Matthew J. Frigault , Noelle V. Frey , Krishna V. Komanduri , Miguel-Angel Perales , Sarah Nikiforow","doi":"10.1016/j.jtct.2025.02.009","DOIUrl":"10.1016/j.jtct.2025.02.009","url":null,"abstract":"<div><div>The first 6 chimeric antigen receptor T cell (CAR-T) therapies approved in the United States have <strong>Risk Evaluation Mitigation Strategies</strong> (REMS) programs mandated by the US Food and Drug Administration (FDA). REMS programs aim to ensure the safe use of CAR-T therapy through timely recognition and management of unique severe risks and toxicities that cannot be mitigated by labeling alone, such as cytokine release syndrome and neurotoxicity syndromes. At the launch of each of the first 6 products, CAR-T REMS programs mandated product-specific education and training for clinical staff, patients, and caregivers; adequate access to medications to treat expected toxicities; and reporting of toxicities either to the product manufacturer or to the FDA. Each manufacturer ensures that treatment centers comply with the REMS program for their individual product in different ways, involving time-consuming and often redundant training, testing, and audits. The American Society for Transplantation and Cellular Therapy (ASTCT) 80/20 Subcommittee convened its second workshop in June 2023, inviting approximately 70 cellular therapy stakeholders to discuss whether safety and quality workflows embedded in existing resources within the cellular therapy field could replace FDA-mandated and company-monitored REMS programs. Attendees were clinicians at large academic medical centers experienced in cellular therapy, regulators, members of accrediting bodies and professional societies, and manufacturers of immune effector cell (IEC) therapies at multiple stages of development. Discussion centered on (1) educational requirements for safe delivery and management, (2) goals and mechanisms for data reporting and to whom, and (3) what entities should oversee these quality safeguards around CAR-T administration and management. Broad support was voiced for (1) conducting training programs administered by treatment centers and/or professional societies to replace manufacturers’ product training; (2) reporting standardized data points into a central, accessible repository for tracking of safety trends and identification of new signals; and (3) enabling accrediting bodies to attest to programs’ quality and ongoing compliance with field safety expectations, thereby replacing intensive manufacturer initial evaluation and ongoing REMS audits. The strong consensus of the second multidisciplinary ASTCT 80/20 Workshop was that such measures would allow elimination, or at least significant reduction and simplification, of current CAR-T REMS programs. Development of educational resources and funding for data reporting outside of a mandated REMS structure were identified as critical, particularly to support treatment centers new to cellular therapy, as were ongoing collaborations with FDA and manufacturers. These consensus recommendations were shared with the FDA at the Cell Therapy Liaison Meeting and in multiple professional society meetings and other public forum","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 349.e1-349.e12"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426356","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}

{"title":"Integrating Social Work Throughout the Hematopoietic Cell Transplantation Trajectory to Improve Patient and Caregiver Outcomes","authors":"Jill Randall ,&nbsp;Ana Gordon ,&nbsp;Clair Boyle ,&nbsp;Darah W. Curran ,&nbsp;Hailey Hassel ,&nbsp;Jessie Russell ,&nbsp;Ben Tweeten ,&nbsp;Kristina Walker ,&nbsp;Kate Zoll","doi":"10.1016/j.jtct.2025.03.013","DOIUrl":"10.1016/j.jtct.2025.03.013","url":null,"abstract":"<div><div>Clinical social workers possess a dual skillset of social care and mental health care and are the largest group of psychosocial care providers in oncology. Psychosocial care is an integral component of quality healthcare. The prevailing model of psychosocial care in oncology is a brief consultation for patients who screen positive for distress at a particular timepoint. This model is insufficient for hematopoietic cell transplantation (HCT). Patients and caregivers have evolving needs throughout the HCT process, and psychosocial care models should meet these needs. This white paper, a collaboration between the Association of Oncology Social Work's Blood Cancer/HCT Special Interest Group and the American Society for Transplantation and Cellular Therapy's Social Work Special Interest Group, presents a gold standard model for the integration of social work in HCT. The model structures social work visits in every phase of HCT and integrates social workers within the interdisciplinary team. In this model, social workers conduct assessments with all patients (autologous and allogeneic) at the initial HCT consultation and again during work-up. They subsequently follow all patients and caregivers as they progress through transplant. This ongoing management reduces the burden on other team members to identify and address psychosocial needs. It also creates many organic opportunities to implement interventions to improve outcomes. There is a need to build institutional capacity for psychosocial care. Strategies that centers can use to build capacity are presented. As a complex clinical intervention, the gold standard model is well-suited for implementation research within a quality improvement framework.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 353.e1-353.e12"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143693402","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}

{"title":"Clinical Outcome of UCBT for Children With CAEBV: A Retrospective Analysis of a Single Center","authors":"Zhiyu Fu ,&nbsp;Biyun Li ,&nbsp;Yujie Chai ,&nbsp;Xifeng Guo ,&nbsp;Xinghua Chen ,&nbsp;Lei Zhang ,&nbsp;Jiao Chen ,&nbsp;Dao Wang","doi":"10.1016/j.jtct.2025.02.026","DOIUrl":"10.1016/j.jtct.2025.02.026","url":null,"abstract":"<div><div>Chronic active Epstein–Barr virus (CAEBV) infection is a severe, life-threatening condition characterized by persistent Epstein–Barr virus (EBV) infection and the clonal expansion of infected T or NK cells, leading to systemic inflammation, organ damage, and complications such as hemophagocytic lymphohistiocytosis and lymphoma. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only effective treatment for eradicating EBV-infected cells; however, donor availability is limited. Umbilical cord blood stem cell transplantation (UCBT) is a promising alternative owing to its rapid availability and lower complication risk. However, there are fewer existing reports on UCBT in pediatric patients with CAEBV. This study aimed to assess the feasibility and clinical efficacy of UCBT as a potential treatment for pediatric patients with CAEBV. We investigated children with CAEBV who did not have matched donors and underwent UCBT in the First Affiliated Hospital of Zhengzhou University and Zhengzhou People's Hospital, China, between 2016 and 2022. We retrospectively analyzed the clinical characteristics, pretreatment regimens, transplantation-related complications, and clinical outcomes of this group of cases to explore the efficacy of UCBT in CAEBV treatment in children. Eight patients, including four males and four females, with a diagnosis age of 4 (1 to 8) years and a transplantation age of 4 (2–8) years, were enrolled in this study. The mean time from diagnosis to transplantation was 5 (2 to 14) months. The mean follow-up period for surviving patients was 49.75±29.66 months, with a maximum follow-up of 101.0 months. All eight patients exhibited successful engraftment. Acute GVHD was observed in six patients, while chronic GVHD was observed in only one patient, with the case being relatively mild. 2 patients developed CMV reactivation. EBV reactivation and post-transplant lymphoproliferative disease (PTLD) were not observed. Case 4 experienced relapse 10 months post-UCBT and achieved survival following a subsequent haplo-identical HSCT from her father. Case 8 succumbed to thrombotic microangiopathy (TMA) on post-transplant day 50. By the end of the follow-up, the 3-year overall survival rate (OS) was estimated to be 87.5% (95% CI: 0.529 to 0.994). The 3-year EFS rate was estimated to be 75% (95% CI: 0.409 to 0.956). The estimated 3-year GRFS rate was also 75.0% (95% CI: 0.409–0.956). UCBT emerges as a safe and effective treatment for CAEBV in children, serving as a viable alternative for patients without matched donors or emergency transplantation.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 386.e1-386.e9"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586981","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}

{"title":"Evaluation of Immunosuppression Levels and Risk of Graft-Versus-Host Disease in Allogeneic Blood or Marrow Transplantation With Post-Transplantation Cyclophosphamide","authors":"John J. Lee ,&nbsp;Haval Norman ,&nbsp;Jamie E. Ziggas ,&nbsp;Javier Bolaños-Meade ,&nbsp;Timothy J. Porter","doi":"10.1016/j.jtct.2025.02.024","DOIUrl":"10.1016/j.jtct.2025.02.024","url":null,"abstract":"&lt;div&gt;&lt;div&gt;Post-transplantation cyclophosphamide (PTCy) is standard graft-versus-host disease (GVHD) prophylaxis for allogeneic blood or marrow transplantation (alloBMT), although optimal therapeutic levels of immunosuppression (IS) therapy combined with PTCy remain contested. Previously, with tacrolimus and methotrexate GVHD prophylaxis, week 1 tacrolimus levels &gt;12 ng/mL were associated with a decreased incidence of grade 2 to 4 acute GVHD (aGVHD). We evaluated if associations between aGVHD and early IS levels were observed amongst patients receiving PTCy. This retrospective single-center study consisted of 349 patients who received PTCy and mycophenolate mofetil, with either tacrolimus (n = 185) or sirolimus (n = 164) from September 1, 2017, to September 30, 2019. The median age of patients receiving tacrolimus and sirolimus were 58 and 54 years, respectively. The primary diagnosed diseases for both cohorts were acute myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome, and lymphoma. While most patients receiving tacrolimus were bone marrow graft sourced (78.4%), the majority of patients receiving sirolimus were peripheral blood sourced (80.5%). All patients were transplanted with FluCyTBI as the conditioning regimen. The primary outcome was grade 2 to 4 aGVHD incidence at 150 days post alloBMT between weekly IS levels &lt;10 ng/mL versus ≥10 ng/mL throughout the 4 weeks post-alloBMT. Secondary endpoints included moderate to severe chronic GVHD (cGVHD) incidence, median overall survival (OS), relapse-free survival (RFS), and GVHD-free relapse-free survival (GRFS) at 2 years and the correlation between weekly IS levels &lt;10 ng/mL versus ≥10 ng/mL throughout 4 weeks post-alloBMT. Patients receiving tacrolimus were compared to others in the tacrolimus cohort, and similarly for sirolimus. No correlation was found between IS levels at any individual week and cumulative aGVHD incidence for either tacrolimus or sirolimus. In the sirolimus cohort, no correlation for moderate to severe cGVHD was observed. However, at week 4, patients in the tacrolimus cohort with levels ≥10 ng/mL experienced significantly higher incidence of moderate to severe chronic GVHD than patients with weekly levels &lt;10 ng/mL (20% versus 8%, &lt;em&gt;P&lt;/em&gt; &lt; .001). When evaluating survival outcomes, post-alloBMT week 1 tacrolimus levels ≥10 ng/mL were associated with decreased OS (HR 3.84, 95% CI [1.16 to 12.67]; &lt;em&gt;P&lt;/em&gt; = .027), but no correlation was seen in RFS (HR 1.62, 95% CI [0.56 to 4.72]; &lt;em&gt;P =&lt;/em&gt; .377), or GRFS (HR 1.56, 95% CI [0.89 to 2.74]; &lt;em&gt;P =&lt;/em&gt; .124). Post-alloBMT week 1 sirolimus ≥10 ng/mL levels were associated with decreased OS (HR 2.74, 95% CI [1.37 to 5.48]; &lt;em&gt;P =&lt;/em&gt; .004) and GRFS (HR 1.93, 95% CI [1.19 to 3.12]; &lt;em&gt;P =&lt;/em&gt; .007), but not RFS (HR 1.60, 95% CI [0.78 to 3.30]; &lt;em&gt;P =&lt;/em&gt; .202). Overall, early IS levels in patients receiving PTCy-based GVHD prophylaxis did not correlate with aGVHD incidence, althou","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 363.e1-363.e11"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568325","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}

{"title":"Clinical effects of granulocyte colony-stimulating factor administration and the timing of its initiation on allogeneic hematopoietic cell transplantation outcomes for myelodysplastic syndrome","authors":"Takaaki Konuma ,&nbsp;Machiko Fujioka ,&nbsp;Kyoko Fuse ,&nbsp;Hiroki Hosoi ,&nbsp;Yosuke Masamoto ,&nbsp;Noriko Doki ,&nbsp;Naoyuki Uchida ,&nbsp;Masatsugu Tanaka ,&nbsp;Masashi Sawa ,&nbsp;Tetsuya Nishida ,&nbsp;Jun Ishikawa ,&nbsp;Noboru Asada ,&nbsp;Hirohisa Nakamae ,&nbsp;Yuta Hasegawa ,&nbsp;Makoto Onizuka ,&nbsp;Takeshi Maeda ,&nbsp;Takahiro Fukuda ,&nbsp;Koji Kawamura ,&nbsp;Yoshinobu Kanda ,&nbsp;Marie Ohbiki ,&nbsp;Hidehiro Itonaga","doi":"10.1016/j.jtct.2025.03.010","DOIUrl":"10.1016/j.jtct.2025.03.010","url":null,"abstract":"<div><div>Granulocyte colony-stimulating factor (G-CSF) accelerates neutrophil recovery after allogeneic hematopoietic cell transplantation (HCT). However, the optimal use of G-CSF and the timing of its initiation after allogeneic HCT for myelodysplastic syndrome (MDS) according to graft type have not been determined. This retrospective study aimed to investigate the effects of using G-CSF administration and the timing of its initiation on transplant outcomes in adult patients with MDS undergoing allogeneic HCT. Using Japanese registry data, we retrospectively investigated the effects of G-CSF administration and the timing of its initiation on transplant outcomes among 4140 adults with MDS after bone marrow transplantation (BMT), peripheral blood stem cell transplantation (PBSCT), or single-unit cord blood transplantation (CBT) between 2013 and 2022. Multivariate analysis showed that early (days 0 to 4) and late (days 5 to 10) G-CSF administration significantly accelerated neutrophil recovery compared with no G-CSF administration following BMT, PBSCT, and CBT, but there was no benefit of early G-CSF initiation for early neutrophilic recovery regardless of graft type. Late G-CSF initiation was significantly associated with a higher risk of overall chronic GVHD following PBSCT (hazard ratio [HR], 1.63; 95% confidence interval [CI], 1.18 to 2.24; <em>P =</em> .002) and CBT (HR, 2.09; 95% CI, 1.21 to 3.60; <em>P =</em> .007) compared with no G-CSF administration. Late G-CSF initiation significantly improved OS compared with no G-CSF administration only following PBSCT (HR, 0.74; 95% CI, 0.58 to 0.94; <em>P =</em> .015). However, G-CSF administration and the timing of its initiation did not affect acute GVHD, relapse, or non-relapse mortality, irrespective of graft type. These results suggest that G-CSF administration significantly accelerated neutrophil recovery after BMT, PBSCT, and CBT, but increased risk of overall chronic GVHD after PBSCT and CBT. However, the effect of early and late G-CSF initiation on transplant outcomes needs further study in adult patients with MDS.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 388.e1-388.e14"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664614","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}

{"title":"Allogeneic Stem Cell Transplantation in Participants With Hematologic Malignancies Following Pembrolizumab Therapy","authors":"John Kuruvilla ,&nbsp;Philippe Armand ,&nbsp;Alex F. Herrera ,&nbsp;Vincent Ribrag ,&nbsp;Catherine Thieblemont ,&nbsp;Bastian von Tresckow ,&nbsp;Guoqing Wang ,&nbsp;Patricia Marinello ,&nbsp;Samhita Chakraborty ,&nbsp;Robert Orlowski ,&nbsp;Pier Luigi Zinzani","doi":"10.1016/j.jtct.2025.02.022","DOIUrl":"10.1016/j.jtct.2025.02.022","url":null,"abstract":"<div><div>The safety and efficacy of allogeneic stem cell transplantation (allo-SCT) following antiprogrammed cell death protein 1 (PD-1) therapy in participants with hematologic malignances is of high clinical interest. The objective of the study is to present outcomes in participants enrolled in 4 clinical trials who underwent allo-SCT within 2 years of their last dose of pembrolizumab therapy. This analysis included participants from the phase 1b KEYNOTE-013 study (n = 20), the phase 2 KEYNOTE-087 study (n = 31), the phase 2 KEYNOTE-170 study (n = 5), and the phase 3 KEYNOTE-204 study (n = 14). Outcomes of interest included acute and chronic graft-versus-host disease (GVHD), progression-free survival (PFS) and overall survival (OS), transplant-related mortality (TRM), and relapse. Of 70 participants included in the analysis, 57 had classical Hodgkin lymphoma (cHL) and the remainder had B-cell non-Hodgkin lymphoma, multiple myeloma, and myelodysplastic syndrome. Overall, 31 participants (44%) were in remission at first allo-SCT. The median duration of treatment with pembrolizumab was 5.3 months (range, 0.7 to 29.6), and the median time from last dose of pembrolizumab to allo-SCT was 4.6 months (range, 1 to 20). The estimated 6-month cumulative incidence of grade II-IV acute GVHD was 41% (95% confidence interval [CI], 30% to 53%); the estimated 6-month cumulative incidence of grade III-IV acute GVHD was 20% (95% CI, 12% to 30%). The estimated 1-year incidence of chronic GVHD was 19% (95% CI, 11% to 29%). After a median follow-up of 40.1 months, both the estimated median PFS and median OS after allo-SCT were not reached; the 40-month PFS rate was 56.8% and the 40-month OS rate was 76.5%. The estimated 40-month cumulative incidence of TRM and relapse was 17% (95% CI, 9% to 27%) and 27% (95% CI, 16% to 38%), respectively. Among participants with cHL (median follow-up, 39.5 months), both the estimated median PFS and median OS after allo-SCT were not reached; the 40-month PFS rate was 59.7% and the 40-month OS rate was 83.4%. The estimated 40-month cumulative incidence of TRM and relapse in participants with cHL was 12% (95% CI, 5% to 23%) and 23% (95% CI, 12% to 36%), respectively. Overall, the incidences of acute and chronic GVHD in this cohort were within the expected ranges. PFS and OS outcomes were favorable, and the rates of TRM and relapse were low. These results support allo-SCT as a useful and feasible salvage option after anti–PD-1 therapy.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 357.e1-357.e11"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568322","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}

{"title":"Fludarabine Plus Myeloablative Dose of Busulfan Regimen Was Associated with High Nonrelapse Mortality in Allogeneic Hematopoietic Stem Cell Transplantation for Malignant Lymphoma: A Propensity Score-Matched Comparison Study with Fludarabine Plus High-Dose Melphalan","authors":"Akihito Shinohara ,&nbsp;Michiho Shindo ,&nbsp;Satoshi Yamasaki ,&nbsp;Koji Kato ,&nbsp;Satoshi Yoshihara ,&nbsp;Go Yamamoto ,&nbsp;Keisuke Kataoka ,&nbsp;Takashi Ikeda ,&nbsp;Hikaru Kobayashi ,&nbsp;Kentaro Serizawa ,&nbsp;Yasuo Mori ,&nbsp;Nobuyuki Takayama ,&nbsp;Hideyuki Nakazawa ,&nbsp;Ayumu Ito ,&nbsp;Yuta Katayama ,&nbsp;Yoshinobu Kanda ,&nbsp;Makoto Yoshimitsu ,&nbsp;Takahiro Fukuda ,&nbsp;Yoshiko Atsuta ,&nbsp;Eisei Kondo","doi":"10.1016/j.jtct.2025.03.008","DOIUrl":"10.1016/j.jtct.2025.03.008","url":null,"abstract":"<div><div>In recent years, there have been notable advancements in the treatment of malignant lymphoma. However, a certain percentage of patients are unlikely to achieve a cure through chemotherapy alone. Therefore, allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains a crucial curative treatment for malignant lymphoma. FluBu4, comprising fludarabine (Flu) combined with a myeloablative dose of intravenous busulfan (Bu; 12.8 mg/kg in total), is a widely used conditioning regimen for allo-HSCT, but its usefulness in malignant lymphoma (ML) has not been fully investigated. The objective of this study was to evaluate the efficacy and safety of FluBu4 in allo-HSCT for lymphoma by comparing the outcomes of two conditioning regimens: FluBu4 and FluMel140. We used a Japanese national database from the Transplant Registry Unified Management Program to retrospectively analyze the first allo-HSCT for ML in patients aged ≥16 years. Allo-HSCT cases treated with posttransplant cyclophosphamide were excluded. Two groups, namely FluBu4 and FluMel140 were selected by propensity score matching (PSM) with a case ratio of 1:2. From 921 cases, 113 were selected by PSM for the FluBu4 group and 226 for the FluMel140 group. The median age was 54 (19 to 68) years, the median observation period of survivors was 33.8 months, and 145 (42.7%) had a history of autologous HSCT. There were no significant differences in patients’ backgrounds between the two groups after PSM. Three-year overall survival (OS) was significantly worse for FluBu4 than for FluMel140 (28.0% versus 48.6%; <em>P</em> &lt; .01). The 3-year cumulative relapse rate was comparable for FluBu4 and FluMel140 (40.1% versus 38.5%; <em>P</em> = .65). However, 3-year nonrelapse mortality was significantly higher for FluBu4 than for FluMel140 (35.3% versus 22.5%; <em>P</em> = .02). There was no significant difference between the two treatment groups in the cumulative incidence of acute graft-versus-host disease (aGVHD) at day 100 after allo-HSCT and the 3-year cumulative incidence of chronic GVHD. While the common and major cause of death was the relapse of lymphoma, aGVHD, and noninfectious lung complications were observed more frequently with FluBu4 than with FluMel140. One-year cumulative incidence of interstitial pneumonia was significantly higher for FluBu4 than for FluMel140 (5.3% versus 0.4%; <em>P</em> = .03). FluBu4 use was associated with worse nonrelapse mortality (NRM) and OS in allo-HSCT for ML compared with FluBu4 and FluMel140 adjusted by PSM. Patients treated with FluBu4 had a higher incidence of noninfectious pulmonary complications and an increased number of associated deaths. A higher rate of NRM in the patients treated with FluBu4 was particularly evident in patients aged ≥60, and its use should be avoided in this patient population.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 382.e1-382.e17"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143650972","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}

{"title":"Infectious Enterocolitis in Hematopoietic Cell Transplant with Post-Transplant Cyclophosphamide","authors":"Pedro Chorão ,&nbsp;André Airosa Pardal ,&nbsp;Santiago de Cossio ,&nbsp;Aitana Balaguer-Roselló ,&nbsp;Juan Montoro ,&nbsp;Marta Villalba ,&nbsp;Eva María González ,&nbsp;María Dolores Gómez ,&nbsp;Inés Gómez ,&nbsp;Pilar Solves ,&nbsp;Marta Santiago ,&nbsp;Pedro Asensi ,&nbsp;Pablo Granados ,&nbsp;Alberto Louro ,&nbsp;Paula Rebollar ,&nbsp;Aurora Perla ,&nbsp;Miguel Salavert ,&nbsp;Javier de la Rubia ,&nbsp;Miguel A. Sanz ,&nbsp;Jaime Sanz","doi":"10.1016/j.jtct.2025.02.027","DOIUrl":"10.1016/j.jtct.2025.02.027","url":null,"abstract":"<div><div>Despite the high incidence of diarrhea in hematopoietic cell transplant (HCT) and the frequent involvement of infections, evidence concerning patients receiving post-transplant cyclophosphamide (PTCy) as graft-versus-host disease (GVHD) prophylaxis in the molecular diagnostic era is limited. This study aimed to evaluate the characteristics, incidence, risk factors, and outcomes impact of infectious enterocolitis in patients with hematologic malignancies undergoing HCT from matched sibling, matched unrelated, and haploidentical donors using PTCy as GVHD prophylaxis. Retrospective analysis of infectious enterocolitis episodes in 399 patients undergoing HCT at a single institution. Uniform GVHD prophylaxis with PTCy, sirolimus, and mycophenolate mofetil was given, irrespective of donor type or conditioning intensity. Levofloxacin was used prophylactically until myeloid engraftment. Infectious enterocolitis episodes were diagnosed by both molecular-based techniques and stool cultures. Infectious enterocolitis affected 21% of patients, with 19% having more than one episode. The median onset and duration was of 83 and 13 days, respectively, 20% were nosocomial and 58% were managed ambulatorily. The 1-year cumulative incidence was 19%, with 39% occurring beyond day 100, and was similar for <em>Clostridioides difficile</em> infection (CDI; 7%)<em>,</em> non-CDI bacterial (8%), and viral enterocolitis (6%), with no differences in clinical features. However, toxin-positive CDI lasted longer (22 days) than toxin-negative cases (10 days, <em>P</em> = .03) Bone marrow HCT significantly increased the risk of overall infectious enterocolitis, while moderate-severe chronic GVHD increased all-cause and viral enterocolitis incidence. Infectious enterocolitis did not significantly impact overall survival, GVHD disease-free relapse-free survival, and non-relapse mortality. Approximately one-fifth of PTCy-based HCT recipients develop infectious enterocolitis in the first year, typically resolving within 2 weeks, with higher incidence in bone marrow recipients and those with moderate-severe chronic GVHD. CDI, non-CDI bacterial, and viral infections had similar incidences and clinical features. While infectious enterocolitis does not significantly impact transplant outcomes, its diagnosis remains challenging.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 392.e1-392.e11"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606504","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}

{"title":"Efficacy of Pomalidomide on Motor Performance and Functional Abilities in Patients with Steroid Refractory Chronic Graft Versus Host Disease: A Randomized Clinical Study","authors":"Jessica J. Jorgensen ,&nbsp;Galen O. Joe ,&nbsp;Rafael Jiménez-Silva ,&nbsp;Pei-Shu Ho ,&nbsp;Tiara Dunigan ,&nbsp;Sandra A. Mitchell ,&nbsp;Steven Z. Pavletic ,&nbsp;Lauren M. Curtis ,&nbsp;Leora E. Comis","doi":"10.1016/j.jtct.2025.02.023","DOIUrl":"10.1016/j.jtct.2025.02.023","url":null,"abstract":"<div><div>Deficits in motor performance and functional abilities represent a severe complication for individuals with steroid refractory chronic graft versus host disease (cGVHD) and is associated with decreased survival and high morbidity. The objective of this study was to characterize the impact of pomalidomide on motor and functional outcomes in patients with cGVHD. Thirty-four adult patients with cGVHD were enrolled in a randomized and unblinded trial. Pomalidomide was administered orally at two dose levels: low (0.5 mg/d) or high (initial 0.5 mg/d, escalating 0.5 mg/d every 2 weeks to a maximum 2 mg/d). Efficacy was assessed primarily by the Activity Card Sort (ACS), 2 Minute Walk Test (2MWT), Medical Outcomes Study Short Form 36 (SF-36), Active Range of Motion (AROM), Disabilities of the Arm, Shoulder, and Hand (DASH), and Manual Abilities Measure 36 (MAM). Compared to baseline, the pooled sample of study participants at 6 months showed improvement in hand skills (MAM, <em>P</em> = .01), upper extremity (UE) function (DASH, <em>P</em> = .01), and health related quality of life (SF-36 Physical Component Summary score (PCS), <em>P</em> = .02). Though no statistically meaningful differences between the two dose groups were found, the low-dose group had greater improvements in AROM, walk distance, UE and hand function, and in the high-demand physical leisure and social subdomains of the ACS as compared to the high-dose group. Responders to pomalidomide performed better than nonresponders on most measures at the 6-month endpoint. The study suggests pomalidomide, at both dose levels, may improve several aspects of motor and functional abilities. However, further study is warranted to determine if the trends found in this study, are sustained over time in larger, and in more diverse cGVHD populations. The findings highlight the potential utility of administering functional and motor tests, such as the ACS, DASH, and MAM, to patients with cGVHD, to fully elucidate the efficacy of treatment options for persons with steroid refractory cGVHD.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 361.e1-361.e15"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143587010","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}