Transplantation and Cellular Therapy最新文献

{"title":"Pediatric Transplant and Cellular Therapy Consortium RESILIENT Conference on Pediatric Chronic Graft-versus-Host Disease Survivorship after Hematopoietic Cell Transplantation: Part II. Organ Dysfunction and Immune Reconstitution Considerations for Children with Chronic Graft-versus-Host Disease after Hematopoietic Cell Transplantation","authors":"Blachy J Dávila Saldaña ,&nbsp;Kirk R Schultz ,&nbsp;Archana Ramgopal ,&nbsp;Julie R. Boiko ,&nbsp;Kristen Beebe ,&nbsp;Paul A. Carpenter ,&nbsp;Sherwin S Chan ,&nbsp;Sophie Paczesny ,&nbsp;Paibel Aguayo-Hiraldo ,&nbsp;Geoffrey D.E. Cuvelier ,&nbsp;Seth J. Rotz ,&nbsp;Christine N. Duncan ,&nbsp;Kirsten M Williams","doi":"10.1016/j.jtct.2025.01.885","DOIUrl":"10.1016/j.jtct.2025.01.885","url":null,"abstract":"<div><div>While highly morbid forms of chronic graft versus host disease (cGVHD) and severe late effects of allogeneic hematopoietic cell transplantation (HCT) can impact children and adults alike, unique considerations arise in pediatric cases regarding diagnosis, monitoring, treatment, and likelihood of resolution. As children can present with atypical features of cGVHD and with more significant disease due to inability to communicate symptoms, they may be at increased risk for highly morbid forms of cGVHD and incur greater subsequent late effects, which may be more pronounced in those with underlying chromosomal breakage syndromes, with higher prevalence in pediatric HCT recipients. The long-term effects of cGVHD and its therapies include impaired immune reconstitution, leading to increased risks of infection and secondary malignant neoplasms. However, children also have the greatest potential for full immune reconstitution, due to thymus recovery that could impact the timing of vaccination with respect to tolerance and restoration of optimal immunity. Developing strategies to mitigate the late effects incurred with, and as a result of, cGVHD is of critical importance. The working group recommends surveillance strategies for late effects in patients with cGVHD, increased utilization of emerging diagnostic tools, integration of monitoring for cGVHD treatment response, and development of new treatments and specifies aims of future research endeavors.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 347.e1-347.e17"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143042252","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":"Best Practices in Gene Therapy for Sickle Cell Disease and Transfusion-dependent β-Thalassemia","authors":"Haydar Frangoul,&nbsp;Amanda Stults,&nbsp;Katie Bruce,&nbsp;Jennifer Domm,&nbsp;Clinton Carroll,&nbsp;Shelby Aide,&nbsp;Morgan Duckworth,&nbsp;Misty Evans,&nbsp;Meghann McManus","doi":"10.1016/j.jtct.2025.02.025","DOIUrl":"10.1016/j.jtct.2025.02.025","url":null,"abstract":"<div><div>Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are inherited blood disorders caused by pathogenic variants of the β-globin gene. Historically, allogeneic hematopoietic stem cell transplantation (HSCT) from human leukocyte antigen (HLA)–matched donors has been the only curative option. However, as most patients with SCD or TDT lack HLA-matched donors, autologous or patient-derived HSCT can provide an alternative, transformative option. Gene therapy–based autologous HSCT for the treatment of SCD and TDT entails a complex patient journey and requires the careful implementation of numerous policies and procedures. As gene therapies for these diseases are now commercially available, there is great value in institutions with developed and implemented approaches sharing their best practices. Here, we describe standardized approaches and best practices for the optimized implementation of gene therapies based on our experience in administering this novel class of medicines.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 352.e1-352.e10"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586978","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":"Officers and Directors of ASTCT","authors":"","doi":"10.1016/S2666-6367(25)01179-0","DOIUrl":"10.1016/S2666-6367(25)01179-0","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Page A4"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144212715","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":"Improvement in Skin Fibrosis and Lung Function with Autologous Hematopoietic Stem Cell Transplantation in Systemic Sclerosis","authors":"Nancy Maltez ,&nbsp;Mianbo Wang ,&nbsp;Georges A Wells ,&nbsp;Peter Tugwell ,&nbsp;Murray Baron ,&nbsp;Zora Marjanovic ,&nbsp;Pauline Lansiaux ,&nbsp;Dominique Farge ,&nbsp;Marie Hudson","doi":"10.1016/j.jtct.2025.02.005","DOIUrl":"10.1016/j.jtct.2025.02.005","url":null,"abstract":"<div><div>Systemic sclerosis (SSc) is a severe, progressive disease with limited treatment options. Autologous hematopoietic stem cell transplantation (AHSCT) has been shown to be an effective treatment for rapidly progressive SSc. The objective of this study was to evaluate the effectiveness of AHSCT for SSc compared to real-world clinical care. SSc patients from France who underwent AHSCT were compared to patients from Canada who met criteria for AHSCT (as defined in the ASTIS trial) but received conventional care. The primary outcome was overall survival. Secondary outcomes included modified Rodnan skin score (mRSS) and forced vital capacity (FVC). Overall survival was estimated by Kaplan–Meier survival curves. Measures of mRSS and FVC were compared using linear regression models. Analyses were adjusted for baseline scores and incorporated stabilized inverse probability of treatment weights to account for confounding by indication. Propensity scores were estimated using logistic regression. Forty-one AHSCT patients and 85 conventional care patients were compared. AHSCT was associated with a suggestive, though not statistically significant trend toward improvement in overall survival (log-rank <em>P</em> = .115). In follow-up, the mRSS was lower with AHSCT compared to conventional care: between group difference of 8.81; <em>P</em> ≤ .0001 at 12 months and 11.28; <em>P</em> = .011 at 60 months. There was no significant difference in FVC between groups at 12 months but at 24 months, AHSCT was associated with a higher FVC (between group difference of 10.53 (<em>P</em> = .05)). This study demonstrates with real-world long-term data that compared with conventional care, treatment with AHSCT may offer superior outcomes for SSc patients.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 6","pages":"Pages 359.e1-359.e10"},"PeriodicalIF":3.6,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143411101","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":"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 ,&nbsp;Zahra Mahmoudjafari ,&nbsp;Partow Kebriaei ,&nbsp;Rebecca A. Gardner ,&nbsp;Matthew J. Frigault ,&nbsp;Noelle V. Frey ,&nbsp;Krishna V. Komanduri ,&nbsp;Miguel-Angel Perales ,&nbsp;Sarah Nikiforow","doi":"10.1016/j.jtct.2025.02.009","DOIUrl":"10.1016/j.jtct.2025.02.009","url":null,"abstract":"&lt;div&gt;&lt;div&gt;The first 6 chimeric antigen receptor T cell (CAR-T) therapies approved in the United States have &lt;strong&gt;Risk Evaluation Mitigation Strategies&lt;/strong&gt; (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}