Pub Date : 2026-01-01DOI: 10.1016/j.jcyt.2025.09.012
María Isabel Benítez-Carabante , Angela Menárguez López , María Luz Uría Oficialdegui , Cristina Beléndez Bieler , Melissa Panesso Romero , Mercedes Plaza Fornieles , Carolina Fuentes , Sara Vinagre Enríquez , Graciela Gómez Silva , Mónica López Duarte , José María Pérez Hurtado , Montserrat Torrent , Laura Alonso García , Cristina Diaz-de-Heredia
Introduction
Patients with transfusion-dependent b-thalassaemia (TDT) suffer from severe anemia, both primary and secondary iron overload and end-organ damage. Although new disease-modifying and curative therapies are emerging, hematopoietic stem cell transplantation (HSCT) remains the most widely available curative option for TDT patients. This study aims to compare HSCT outcomes according to the type of donor used, considering not only survival, but also graft-versus-host disease (GVHD) and other transplant-related complications.
Methods
A multicentre retrospective study was conducted, in which children who received a first HSCT for TDT were included.
Results
Fifty-eight patients were included, with a median follow-up of 7.4 years. The median age at HSCT was 5.6 years (range 0.76–16.64). Thirty-nine patients received a matched family donor (MFD) transplant, and 19 received an unrelated donor transplant of whom 16 received a fully matched unrelated donor (MUD) and 3 a mismatched unrelated donor cord blood (CB) transplant. Most patients received bone marrow alone or in combination with CB (n = 49); six patients received CB, and 3 patients’ peripheral blood. Thirty-six patients received busulfan, while the remaining patients received a treosulfan-based conditioning regimen. The incidence of grade II-IV acute GVHD was significantly higher in the MUD group than in the MFD group (56.25% vs. 25.71%, P = 0.0178). No significant differences were found in grade III-IV acute GVHD or chronic GVHD. Although the incidence of endothelial complications was higher in patients receiving an MUD transplant, it was not statistically significant. With 2-year overall survival (OS), thalassaemia free survival (TFS) and chronic GVHD thalassaemia-free survival of 98%, 92.2% and 82.7%, respectively, there were no differences between the MFD and MUD groups.
Conclusion
Fully matched unrelated donor transplantation may offer a real curative opportunity for pediatric patients with TDT who lack an MSD, with excellent OS, TFS and low incidence of grade III-IV acute and chronic GVHD. Future research into the prevention and treatment of GVHD will further improve these results.
输血依赖性b-地中海贫血（TDT）患者患有严重贫血，原发性和继发性铁超载和终末器官损伤。虽然新的疾病改善和治疗方法正在出现，但造血干细胞移植（HSCT）仍然是TDT患者最广泛可用的治疗选择。本研究旨在根据使用的供体类型比较HSCT的结果，不仅考虑存活，还考虑移植物抗宿主病（GVHD）和其他移植相关并发症。方法：进行了一项多中心回顾性研究，其中包括首次接受HSCT治疗的儿童。结果：纳入58例患者，中位随访时间为7.4年。HSCT的中位年龄为5.6岁（范围0.76-16.64）。39例患者接受了匹配的家庭供体（MFD）移植，19例接受了非亲属供体移植，其中16例接受了完全匹配的非亲属供体（MUD）移植，3例接受了不匹配的非亲属供体脐带血（CB）移植。大多数患者接受骨髓单独或联合CB (n = 49)；6例患者接受CB， 3例患者接受外周血。36例患者接受布硫芬治疗，其余患者接受以曲硫芬为基础的调理方案。MUD组II-IV级急性GVHD发生率明显高于MFD组（56.25% vs. 25.71%, P = 0.0178）。III-IV级急性GVHD与慢性GVHD无显著差异。虽然接受MUD移植的患者内皮并发症的发生率较高，但没有统计学意义。2年总生存率（OS）、无地中海贫血生存率（TFS）和慢性GVHD无地中海贫血生存率分别为98%、92.2%和82.7%，MFD组和MUD组之间无差异。结论：完全匹配的非亲属供体移植可能为缺乏MSD的儿童TDT患者提供真正的治疗机会，这些患者具有良好的OS和TFS， III-IV级急慢性GVHD发病率低。未来对GVHD预防和治疗的研究将进一步完善这些结果。
{"title":"Unrelated donor transplantation offers a real curative opportunity for patients with transfusion-dependent thalassaemia. A multicenter retrospective study on behalf of the Spanish group for hematopoietic transplantation and cellular therapy (GETH-TC)","authors":"María Isabel Benítez-Carabante , Angela Menárguez López , María Luz Uría Oficialdegui , Cristina Beléndez Bieler , Melissa Panesso Romero , Mercedes Plaza Fornieles , Carolina Fuentes , Sara Vinagre Enríquez , Graciela Gómez Silva , Mónica López Duarte , José María Pérez Hurtado , Montserrat Torrent , Laura Alonso García , Cristina Diaz-de-Heredia","doi":"10.1016/j.jcyt.2025.09.012","DOIUrl":"10.1016/j.jcyt.2025.09.012","url":null,"abstract":"<div><h3>Introduction</h3><div>Patients with transfusion-dependent b-thalassaemia (TDT) suffer from severe anemia, both primary and secondary iron overload and end-organ damage. Although new disease-modifying and curative therapies are emerging, hematopoietic stem cell transplantation (HSCT) remains the most widely available curative option for TDT patients. This study aims to compare HSCT outcomes according to the type of donor used, considering not only survival, but also graft-versus-host disease (GVHD) and other transplant-related complications.</div></div><div><h3>Methods</h3><div>A multicentre retrospective study was conducted, in which children who received a first HSCT for TDT were included.</div></div><div><h3>Results</h3><div>Fifty-eight patients were included, with a median follow-up of 7.4 years. The median age at HSCT was 5.6 years (range 0.76–16.64). Thirty-nine patients received a matched family donor (MFD) transplant, and 19 received an unrelated donor transplant of whom 16 received a fully matched unrelated donor (MUD) and 3 a mismatched unrelated donor cord blood (CB) transplant. Most patients received bone marrow alone or in combination with CB (n = 49); six patients received CB, and 3 patients’ peripheral blood. Thirty-six patients received busulfan, while the remaining patients received a treosulfan-based conditioning regimen. The incidence of grade II-IV acute GVHD was significantly higher in the MUD group than in the MFD group (56.25% vs. 25.71%, <em>P = 0.</em>0178). No significant differences were found in grade III-IV acute GVHD or chronic GVHD. Although the incidence of endothelial complications was higher in patients receiving an MUD transplant, it was not statistically significant. With 2-year overall survival (OS), thalassaemia free survival (TFS) and chronic GVHD thalassaemia-free survival of 98%, 92.2% and 82.7%, respectively, there were no differences between the MFD and MUD groups.</div></div><div><h3>Conclusion</h3><div>Fully matched unrelated donor transplantation may offer a real curative opportunity for pediatric patients with TDT who lack an MSD, with excellent OS, TFS and low incidence of grade III-IV acute and chronic GVHD. Future research into the prevention and treatment of GVHD will further improve these results.</div></div>","PeriodicalId":50597,"journal":{"name":"Cytotherapy","volume":"28 1","pages":"Article 101989"},"PeriodicalIF":3.2,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145452011","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}
Pub Date : 2026-01-01DOI: 10.1016/j.jcyt.2025.07.008
Kyndal Goss , Melanie L. Grant , Lokesh Guglani , Hannah Miller , Erik J. Woods , Cherish Caldwell , Anne M. Fitzpatrick , Edwin M. Horwitz
Background
Asthma is the most common chronic lung disorder among children and young adults worldwide. Despite effective new biologic therapies, ∼10% of patients remain poorly controlled. Monocytes contribute to the proinflammatory milieu in asthmatic lungs but also suppress inflammation and promote tissue healing.
Aims
Compare the transcriptome of blood monocytes in asthma patients and healthy controls and assess the impact of an infusion of interferon y-activated mesenchymal stromal cells (yMSCs) on the transcriptome of asthmatic monocytes.
Methods
scRNA-seq was conducted on blood monocytes isolated from an asthma patient before and after an infusion of γMSCs, on blood monocytes from otherwise healthy subjects with asthma, and from healthy controls.
Results
One infusion of γMSCs induced profound gene expression changes, including reduced expression of HLA Class I and interferon signaling genes. Monocytes from asthma patients revealed increased expression of interferon response genes compared to healthy controls. Several viral and interferon response pathways were significantly enriched in severe versus mild asthma.
Conclusions
Our data suggest that monocyte directed therapy may be beneficial in asthma, especially for patients who are poorly controlled on currently available therapy, and additional studies of γMSC therapy for patients with poorly controlled asthma are warranted.
{"title":"Single cell transcriptional profiling of monocytes from asthma patients show a predisposition for an inflammatory response","authors":"Kyndal Goss , Melanie L. Grant , Lokesh Guglani , Hannah Miller , Erik J. Woods , Cherish Caldwell , Anne M. Fitzpatrick , Edwin M. Horwitz","doi":"10.1016/j.jcyt.2025.07.008","DOIUrl":"10.1016/j.jcyt.2025.07.008","url":null,"abstract":"<div><h3>Background</h3><div>Asthma is the most common chronic lung disorder among children and young adults worldwide. Despite effective new biologic therapies, ∼10% of patients remain poorly controlled. Monocytes contribute to the proinflammatory milieu in asthmatic lungs but also suppress inflammation and promote tissue healing.</div></div><div><h3>Aims</h3><div>Compare the transcriptome of blood monocytes in asthma patients and healthy controls and assess the impact of an infusion of interferon y-activated mesenchymal stromal cells (yMSCs) on the transcriptome of asthmatic monocytes.</div></div><div><h3>Methods</h3><div>scRNA-seq was conducted on blood monocytes isolated from an asthma patient before and after an infusion of γMSCs, on blood monocytes from otherwise healthy subjects with asthma, and from healthy controls.</div></div><div><h3>Results</h3><div>One infusion of γMSCs induced profound gene expression changes, including reduced expression of HLA Class I and interferon signaling genes. Monocytes from asthma patients revealed increased expression of interferon response genes compared to healthy controls. Several viral and interferon response pathways were significantly enriched in severe versus mild asthma.</div></div><div><h3>Conclusions</h3><div>Our data suggest that monocyte directed therapy may be beneficial in asthma, especially for patients who are poorly controlled on currently available therapy, and additional studies of γMSC therapy for patients with poorly controlled asthma are warranted.</div></div>","PeriodicalId":50597,"journal":{"name":"Cytotherapy","volume":"28 1","pages":"Article 101967"},"PeriodicalIF":3.2,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145566230","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}
Pub Date : 2026-01-01DOI: 10.1016/j.jcyt.2025.08.008
Giorgia Adamo , Ioannis Amarantos , Iordanis Arzimanoglou , Paolo Bergese , Antonella Bongiovanni , Benedetta Bussolati , Valentina Cauda , Marcella Chiari , George A. Garinis , Bernd Giebel , Paolo Guazzi , Marcin Jurga , Christian Neri , Pelle Ohlsson , Eva Rohde , Goreti Sales , Irene Trapani , Pieter Vader , Seppo Vainio , Marca H.M. Wauben , Natasa Zarovni
The field of extracellular vesicles (EVs) is rapidly advancing, offering promising applications in diagnostics, therapeutics, and drug delivery. However, the translation of EV-based technologies to the clinic faces significant challenges related to heterogeneity, scalable biomanufacturing, and regulatory compliance. To address these issues, the European Innovation Council (EIC), in collaboration with the Horizon2020-funded BOW project, organized the “Extracellular Vesicle EIC Cluster Meeting,” bringing together researchers, startups, and regulatory stakeholders across Europe. Discussions focused on overcoming bottlenecks in EV production, standardization, and clinical readiness. Key outcomes included the need for application-specific benchmarks, robust manufacturing pipelines, and regulatory frameworks tailored to EVs. The event emphasized the importance of interdisciplinary collaboration, coopetition, and continued EU funding to drive innovation and strengthen Europe’s leadership in the EV field.
{"title":"Spotlight on innovation: key insights from the extracellular vesicle cluster meeting at the European Innovation Council","authors":"Giorgia Adamo , Ioannis Amarantos , Iordanis Arzimanoglou , Paolo Bergese , Antonella Bongiovanni , Benedetta Bussolati , Valentina Cauda , Marcella Chiari , George A. Garinis , Bernd Giebel , Paolo Guazzi , Marcin Jurga , Christian Neri , Pelle Ohlsson , Eva Rohde , Goreti Sales , Irene Trapani , Pieter Vader , Seppo Vainio , Marca H.M. Wauben , Natasa Zarovni","doi":"10.1016/j.jcyt.2025.08.008","DOIUrl":"10.1016/j.jcyt.2025.08.008","url":null,"abstract":"<div><div>The field of extracellular vesicles (EVs) is rapidly advancing, offering promising applications in diagnostics, therapeutics, and drug delivery. However, the translation of EV-based technologies to the clinic faces significant challenges related to heterogeneity, scalable biomanufacturing, and regulatory compliance. To address these issues, the European Innovation Council (EIC), in collaboration with the Horizon2020-funded BOW project, organized the “Extracellular Vesicle EIC Cluster Meeting,” bringing together researchers, startups, and regulatory stakeholders across Europe. Discussions focused on overcoming bottlenecks in EV production, standardization, and clinical readiness. Key outcomes included the need for application-specific benchmarks, robust manufacturing pipelines, and regulatory frameworks tailored to EVs. The event emphasized the importance of interdisciplinary collaboration, coopetition, and continued EU funding to drive innovation and strengthen Europe’s leadership in the EV field.</div></div>","PeriodicalId":50597,"journal":{"name":"Cytotherapy","volume":"28 1","pages":"Article 101976"},"PeriodicalIF":3.2,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145369010","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}
Pub Date : 2026-01-01DOI: 10.1016/j.jcyt.2025.02.008
Mohammed Amine Bekadja , Luuk Gras , Laurien Baaij , Linda Koster , Nada Hamad , Ben Carpenter , Emma Nicholson , Victoria Potter , Annoek E.C. Broers , Didier Blaise , Jenny Louise Byrne , Anne Huynh , Francesca Kinsella , Péter Reményi , Matthew P. Collin Sr. , John G. Gribben , Simon Bulley , Alessandra Tucci , Natalia De Las Heras , Joanna Drozd-Sokolowska , Dietger Niederwieser
<div><h3>Introduction</h3><div>Most transplant centres cryopreserve peripheral blood stem cells (PBSC) for use in a subsequent autologous hematopoietic cell transplant (AHCT) using Dimethyl sulfoxide (DMSO) as a cryoprotectant. Non-cryopreserved (NCP) PBSC are used in autologous transplants for multiple myeloma (MM) in some countries with limited resources. We compared AHCT outcomes between patients in a large tertiary referral transplant centre in Oran, Algeria, who received non-cryopreserved PBSC and patients from EBMT-affiliated centres in countries where cryopreservation is standard.</div></div><div><h3>Patients and Methods</h3><div>MM patients who underwent AHCT between 2009 and 2020 inclusive using NCP PBSC were each matched with up to four patients from 420 EBMT-affiliated centres who received cryopreserved (CP) PBSC over the same period. Following standard procedures, PBSC were stored in a refrigerator at 4°C immediately following collection. PBSCs were then infused 24 hours following Melphalan administration (at a dose of either 140 or 200 mg/m<sup>2</sup>). The primary endpoints were neutrophil and platelet engraftment as defined by standard EBMT criteria. Secondary endpoints included non-relapse mortality (NRM), relapse incidence (RI), overall survival (OS) and progression free survival (PFS).</div></div><div><h3>Results</h3><div>Using NCP PBSC, 407 MM patients were autografted. The median number of collection procedures was two (range (r), (1–3)). The mean PBSC viability was 95% (r, 93.8–98.5%). A total of 367 (90%) of these patients had at least one match with a patient in the EBMT registry who had received CP PBSC. In total, 1,412 CP PBSC patients were included in this analysis. In the NCP group, the median dose of PBSCs collected was 3.2 × 10<sup>6</sup> CD34+ cells/kg (interquartile range (IQR, 2.4–4.5); for the CP group (available in 12% of cases), it was 4.0 × 10<sup>6</sup> CD34+ cells/kg (IQR, 2.8–5.3) (<em>P = 0</em>.005). The median number of days to neutrophil engraftment for the NCP and CP cohorts were 12 (IQR, 11–14) days, and 12 (IQR, 11–13) days, respectively (HR comparing CP versus NCP 1.13, 95% CI 0.99–1.29, <em>P = 0</em>.08). The median time to platelet engraftment >20 × 10<sup>9</sup>/L was similar in the NCP and CP cohorts: 12 (IQR, 11–14) days and 12 (IQR, 11–14) days, respectively. Nonrelapse mortality rates at Day +100 were 0.3% (0.0–0.8%) and 0.7% (0.5–1.0%), respectively, in the NCP and CP groups (<em>P = 0</em>.38). The relapse incidence was lower in the NCP group (HR = 0.23, <em>P = 0</em>.004). The PFS up to 3 years post-transplant was significantly longer in the non-cryopreserved cohort (HR = 0.71, <em>P</em> < 0.001). The OS rates at 3 years were 81% and 82% in the NCP group and the CP group, respectively (<em>P = 0</em>.47).</div></div><div><h3>Conclusion</h3><div>This EBMT registry study comparing the use of fresh stem cells with frozen stem cells did not find significant differences in either en
{"title":"A comparison of cryopreserved and noncryopreserved peripheral blood hematopoietic stem cells for autologous transplantation in multiple myeloma: a study from the chronic malignancies working party of the EBMT","authors":"Mohammed Amine Bekadja , Luuk Gras , Laurien Baaij , Linda Koster , Nada Hamad , Ben Carpenter , Emma Nicholson , Victoria Potter , Annoek E.C. Broers , Didier Blaise , Jenny Louise Byrne , Anne Huynh , Francesca Kinsella , Péter Reményi , Matthew P. Collin Sr. , John G. Gribben , Simon Bulley , Alessandra Tucci , Natalia De Las Heras , Joanna Drozd-Sokolowska , Dietger Niederwieser","doi":"10.1016/j.jcyt.2025.02.008","DOIUrl":"10.1016/j.jcyt.2025.02.008","url":null,"abstract":"<div><h3>Introduction</h3><div>Most transplant centres cryopreserve peripheral blood stem cells (PBSC) for use in a subsequent autologous hematopoietic cell transplant (AHCT) using Dimethyl sulfoxide (DMSO) as a cryoprotectant. Non-cryopreserved (NCP) PBSC are used in autologous transplants for multiple myeloma (MM) in some countries with limited resources. We compared AHCT outcomes between patients in a large tertiary referral transplant centre in Oran, Algeria, who received non-cryopreserved PBSC and patients from EBMT-affiliated centres in countries where cryopreservation is standard.</div></div><div><h3>Patients and Methods</h3><div>MM patients who underwent AHCT between 2009 and 2020 inclusive using NCP PBSC were each matched with up to four patients from 420 EBMT-affiliated centres who received cryopreserved (CP) PBSC over the same period. Following standard procedures, PBSC were stored in a refrigerator at 4°C immediately following collection. PBSCs were then infused 24 hours following Melphalan administration (at a dose of either 140 or 200 mg/m<sup>2</sup>). The primary endpoints were neutrophil and platelet engraftment as defined by standard EBMT criteria. Secondary endpoints included non-relapse mortality (NRM), relapse incidence (RI), overall survival (OS) and progression free survival (PFS).</div></div><div><h3>Results</h3><div>Using NCP PBSC, 407 MM patients were autografted. The median number of collection procedures was two (range (r), (1–3)). The mean PBSC viability was 95% (r, 93.8–98.5%). A total of 367 (90%) of these patients had at least one match with a patient in the EBMT registry who had received CP PBSC. In total, 1,412 CP PBSC patients were included in this analysis. In the NCP group, the median dose of PBSCs collected was 3.2 × 10<sup>6</sup> CD34+ cells/kg (interquartile range (IQR, 2.4–4.5); for the CP group (available in 12% of cases), it was 4.0 × 10<sup>6</sup> CD34+ cells/kg (IQR, 2.8–5.3) (<em>P = 0</em>.005). The median number of days to neutrophil engraftment for the NCP and CP cohorts were 12 (IQR, 11–14) days, and 12 (IQR, 11–13) days, respectively (HR comparing CP versus NCP 1.13, 95% CI 0.99–1.29, <em>P = 0</em>.08). The median time to platelet engraftment >20 × 10<sup>9</sup>/L was similar in the NCP and CP cohorts: 12 (IQR, 11–14) days and 12 (IQR, 11–14) days, respectively. Nonrelapse mortality rates at Day +100 were 0.3% (0.0–0.8%) and 0.7% (0.5–1.0%), respectively, in the NCP and CP groups (<em>P = 0</em>.38). The relapse incidence was lower in the NCP group (HR = 0.23, <em>P = 0</em>.004). The PFS up to 3 years post-transplant was significantly longer in the non-cryopreserved cohort (HR = 0.71, <em>P</em> < 0.001). The OS rates at 3 years were 81% and 82% in the NCP group and the CP group, respectively (<em>P = 0</em>.47).</div></div><div><h3>Conclusion</h3><div>This EBMT registry study comparing the use of fresh stem cells with frozen stem cells did not find significant differences in either en","PeriodicalId":50597,"journal":{"name":"Cytotherapy","volume":"28 1","pages":"Article 101906"},"PeriodicalIF":3.2,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145349721","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}
Pub Date : 2026-01-01DOI: 10.1016/j.jcyt.2025.09.007
Delphine Ammar , Carmen Sanges , David Henderson , Inga Schapitz , Michael Hudecek , Maik Luu , Martina Schüssler-Lenz , Christian Buchholz , Paul Franz , Kristin Reiche , Ulrike Köhl , Norbert Ifrah , Bruno Quesnel , Maria Thermeli , Marie Jose Kersten , Franco Locatelli , Maria Luisa D'Amore , Chiara Bonini , Monica Casucci , Ulrike Philippar , Jacquelyn Awigena-Cook
With the proposal for major reform of the pharmaceutical legislation (Regulation (EU) 2023/0131, Directive (EU) 2023/0132) in April 2023, the foundations of Europe’s regulatory architecture are being reshaped. The revised legislation proposes the removal of specialized committee functions (e.g., Committee for Advanced Therapies—CAT) by integrating their remit into other regulatory bodies, such as the Pharmacovigilance Risk Assessment Committee (PRAC) and Committee for Medicinal Products for Human Use (CHMP) and expert working groups. This decision may further challenge innovation in Europe, particularly in the field of advanced therapy medicinal products (ATMPs), which has already experienced a notable decline in global clinical trial activity over the past decade. This article emphasizes the importance of involving multistakeholder consortia such as T2EVOLVE in shaping pharmaceutical legislation to prevent the loss of specialized knowledge and the need for robust mechanisms that preserve developers’ access to ATMP regulatory expertise.
{"title":"Call for preserving specialized knowledge and contributions of the CAT to advancing ATMPs in Europe","authors":"Delphine Ammar , Carmen Sanges , David Henderson , Inga Schapitz , Michael Hudecek , Maik Luu , Martina Schüssler-Lenz , Christian Buchholz , Paul Franz , Kristin Reiche , Ulrike Köhl , Norbert Ifrah , Bruno Quesnel , Maria Thermeli , Marie Jose Kersten , Franco Locatelli , Maria Luisa D'Amore , Chiara Bonini , Monica Casucci , Ulrike Philippar , Jacquelyn Awigena-Cook","doi":"10.1016/j.jcyt.2025.09.007","DOIUrl":"10.1016/j.jcyt.2025.09.007","url":null,"abstract":"<div><div>With the proposal for major reform of the pharmaceutical legislation (Regulation (EU) 2023/0131, Directive (EU) 2023/0132) in April 2023, the foundations of Europe’s regulatory architecture are being reshaped. The revised legislation proposes the removal of specialized committee functions (e.g., Committee for Advanced Therapies—CAT) by integrating their remit into other regulatory bodies, such as the Pharmacovigilance Risk Assessment Committee (PRAC) and Committee for Medicinal Products for Human Use (CHMP) and expert working groups. This decision may further challenge innovation in Europe, particularly in the field of advanced therapy medicinal products (ATMPs), which has already experienced a notable decline in global clinical trial activity over the past decade. This article emphasizes the importance of involving multistakeholder consortia such as T2EVOLVE in shaping pharmaceutical legislation to prevent the loss of specialized knowledge and the need for robust mechanisms that preserve developers’ access to ATMP regulatory expertise.</div></div>","PeriodicalId":50597,"journal":{"name":"Cytotherapy","volume":"28 1","pages":"Article 101984"},"PeriodicalIF":3.2,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145490916","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}
Pub Date : 2026-01-01DOI: 10.1016/j.jcyt.2025.09.010
Seungwon An , Youngmin Park , Celeste Mora , Karen Sweiss , Chukwuemeka Uzoka , Matias Sanchez , Elisa Zucchetti , Sally Campbell-Lee , Weiwei Ma , Zhengjia Chen , Damiano Rondelli , Nadim Mahmud
Background aims
This retrospective study investigated the impact of dosing CD34+ cells on the basis of body weight, body surface area (BSA) and body mass index (BMI) on neutrophil and platelet engraftment after autologous stem cell transplantation (SCT) in patients with multiple myeloma (MM).
Methods
We performed a retrospective chart review of 114 patients including 97 patients (48 male and 49 female) diagnosed with MM and 17 patients diagnosed with Non-Hodgkin lymphoma. The study categorized subjects on the basis of BMI (<25 and ≥25) and BSA (<1.9 m² and ≥1.9 m²).
Results
The findings revealed no significant difference in time to neutrophil engraftment after SCT for different BMI groups, which was on average 11.1 days. However, BSA played a significant role as patients with a BSA ≥1.9 m² exhibited delayed platelet engraftment in contrast to the low BSA group (18.55 days versus 16.42 days, P < 0.05). Furthermore, although CD34+ cell doses were comparable across groups when adjusted for body weight, a BSA-based analysis indicated a greater absolute number of CD34+ cells were administered to patients with BSA ≥1.9 m² (6.07 versus 4.78 cells × 10⁸/m², P < 0.05). By using a multivariate analysis, we showed that for neutrophil engraftment, the best predictive model included age, fat-free mass (FFM) and CD34+ cells (P < 0.1) along with BSA (P = 0.12). For platelet engraftment, the best predictive model included sex and FFM (P < 0.1), with BSA as a significant predictor (P = 0.02). Patients with greater BSA (≥1.9 m²) had a nonsignificantly shorter adjusted time to neutrophil engraftment (LS mean = 10.96) compared with those with lower BSA (<1.9) (LS mean = 11.23). However, greater BSA (≥1.9 m²) was significantly associated with a longer adjusted time to platelet engraftment (LS mean = 18.94) versus lower BSA (<1.9) (LS mean = 16.21).
Conclusion
Our results highlight that male patients with a high BSA experienced a 4-day delay in platelet engraftment after autologous SCT. This delay emphasizes the need for optimized dosing strategies to enhance post-transplant recovery, particularly in patients with elevated BSA. Shortening the time to hematopoietic engraftment after SCT may significantly reduce hospital stays and lower the risk of infections, bleeding, and transfusion-related complications.
{"title":"Beyond body weight: investigating the impact of body surface area on graft CD34+ cell dosing: a single-center experience","authors":"Seungwon An , Youngmin Park , Celeste Mora , Karen Sweiss , Chukwuemeka Uzoka , Matias Sanchez , Elisa Zucchetti , Sally Campbell-Lee , Weiwei Ma , Zhengjia Chen , Damiano Rondelli , Nadim Mahmud","doi":"10.1016/j.jcyt.2025.09.010","DOIUrl":"10.1016/j.jcyt.2025.09.010","url":null,"abstract":"<div><h3>Background aims</h3><div>This retrospective study investigated the impact of dosing CD34+ cells on the basis of body weight, body surface area (BSA) and body mass index (BMI) on neutrophil and platelet engraftment after autologous stem cell transplantation (SCT) in patients with multiple myeloma (MM).</div></div><div><h3>Methods</h3><div>We performed a retrospective chart review of 114 patients including 97 patients (48 male and 49 female) diagnosed with MM and 17 patients diagnosed with Non-Hodgkin lymphoma. The study categorized subjects on the basis of BMI (<25 and ≥25) and BSA (<1.9 m² and ≥1.9 m²).</div></div><div><h3>Results</h3><div>The findings revealed no significant difference in time to neutrophil engraftment after SCT for different BMI groups, which was on average 11.1 days. However, BSA played a significant role as patients with a BSA ≥1.9 m² exhibited delayed platelet engraftment in contrast to the low BSA group (18.55 days versus 16.42 days, <em>P</em> < 0.05). Furthermore, although CD34+ cell doses were comparable across groups when adjusted for body weight, a BSA-based analysis indicated a greater absolute number of CD34+ cells were administered to patients with BSA ≥1.9 m² (6.07 versus 4.78 cells × 10⁸/m², <em>P</em> < 0.05). By using a multivariate analysis, we showed that for neutrophil engraftment, the best predictive model included age, fat-free mass (FFM) and CD34+ cells (P < 0.1) along with BSA (<em>P</em> = 0.12). For platelet engraftment, the best predictive model included sex and FFM (<em>P</em> < 0.1), with BSA as a significant predictor (<em>P</em> = 0.02). Patients with greater BSA (≥1.9 m²) had a nonsignificantly shorter adjusted time to neutrophil engraftment (LS mean = 10.96) compared with those with lower BSA (<1.9) (LS mean = 11.23). However, greater BSA (≥1.9 m²) was significantly associated with a longer adjusted time to platelet engraftment (LS mean = 18.94) versus lower BSA (<1.9) (LS mean = 16.21).</div></div><div><h3>Conclusion</h3><div>Our results highlight that male patients with a high BSA experienced a 4-day delay in platelet engraftment after autologous SCT. This delay emphasizes the need for optimized dosing strategies to enhance post-transplant recovery, particularly in patients with elevated BSA. Shortening the time to hematopoietic engraftment after SCT may significantly reduce hospital stays and lower the risk of infections, bleeding, and transfusion-related complications.</div></div>","PeriodicalId":50597,"journal":{"name":"Cytotherapy","volume":"28 1","pages":"Article 101987"},"PeriodicalIF":3.2,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145379821","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}
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