María Queralt Salas, Raquel Salinas-González, Laia Guardia, María Teresa Solano, Cristina Padilla, Cristina Moreno, Paola Charr, Noemi de Llobet, Carla Mestre, Enric Cascos, Joan Cid, Miquel Lozano, Cristina Gallego, Bernat Carreras, Vanessa Vilas, Montserrat Duch, Julia Martínez-Sanchez, Maribel Díaz-Ricart, Jordi Esteve, Enric Carreras, María Suárez-Lledó, Laura Rosiñol, Sara Laxe, Concepción Closa, Carmen Martínez, Francesc Fernández-Avilés, Montserrat Rovira
{"title":"Frailty assessment and outpatient pre-habilitation for adults undergoing allogeneic hematopoietic cell transplantation.","authors":"María Queralt Salas, Raquel Salinas-González, Laia Guardia, María Teresa Solano, Cristina Padilla, Cristina Moreno, Paola Charr, Noemi de Llobet, Carla Mestre, Enric Cascos, Joan Cid, Miquel Lozano, Cristina Gallego, Bernat Carreras, Vanessa Vilas, Montserrat Duch, Julia Martínez-Sanchez, Maribel Díaz-Ricart, Jordi Esteve, Enric Carreras, María Suárez-Lledó, Laura Rosiñol, Sara Laxe, Concepción Closa, Carmen Martínez, Francesc Fernández-Avilés, Montserrat Rovira","doi":"10.1038/s41409-025-02571-9","DOIUrl":"10.1038/s41409-025-02571-9","url":null,"abstract":"<p><p>This study reports the preliminary results of a Frailty program involving all allo-HCT candidates, initiated at our institution in April 2021. The program started with the assessment of patients' frailty at first consultation and at transplant admission using the HCT Frailty Scale. After observing the dynamics of the frailty syndrome, in June 2022 the program incorporated an individualized 4-8-weeks pre-habilitation program composed by personalized home-based exercise regimens to mitigate or prevent patients' frailty at transplantation. This study includes the 120 patients transplanted during the program, 58 of them participating in the pre-habilitation program. The results indicate that the distribution of frailty was the same in first consultation and at HCT admission in non-pre-habilitated patients, but it changed from the proportions of fit, pre-frail and frail patients of 22.8%, 66.7%, 10.5% at first consultation, to the proportions 45.6%, 50.9% and 3.5% (P = 0.041) at admission in the pre-habilitated ones. And that the likelihood of OS was lower in the frail than in the fit and the pre-frail groups (1-year OS: 45.7% vs. 77.2%, O = 0.021). Therefore, the study provides preliminary evidence that pre habilitation programs significantly reduces frailty among transplant candidates and, eventually, increase the likelihood of OS.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143810472","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Candice Laverne Hendricks, Juanita Mellet, Voula Stivaktas, Melvin Ambele, Michael Sean Pepper
{"title":"HIV-exposed uninfected umbilical cord blood haematopoietic stem/progenitor cells differ immunophenotypically from those from HIV unexposed umbilical cord blood but have similar expansion and colony-forming properties in vitro.","authors":"Candice Laverne Hendricks, Juanita Mellet, Voula Stivaktas, Melvin Ambele, Michael Sean Pepper","doi":"10.1038/s41409-025-02574-6","DOIUrl":"10.1038/s41409-025-02574-6","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143802464","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Protective factors, management and prognosis of mixed chimerism after allogeneic hematopoietic stem cell transplantation for severe aplastic anemia in children.","authors":"Jian Liu, Yumiao Mai, Zhaohe Jing, Linchao Zhao, Hongyun Niu, Simin Qiu, Pengpeng Dong","doi":"10.1038/s41409-025-02577-3","DOIUrl":"10.1038/s41409-025-02577-3","url":null,"abstract":"<p><p>Mixed chimerism (MC) frequently arises in children with severe aplastic anemia (SAA) after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Nonetheless, there is a paucity of research regarding potential predictors and effective interventions. This retrospective study, performed on 150 pediatric patients with SAA who underwent allo-HSCT between December 2015 and June 2022, explored the characteristics, risk factors, treatment, and prognosis of MC. A total of 29 patients (19.3%) developed MC following allo-HSCT, with two individuals experiencing MC twice. The CTX + ATG regimen was associated with the development of MC. Peripheral blood (PB) + bone marrow (BM) stem cell graft and a high number of CD34<sup>+</sup> cells were identified as independent protective factors for MC. The cumulative incidence of grade II-IV acute graft-versus-host disease was significantly elevated in donor chimerism (DC) relative to MC. Among MC patients with cytopenia, only two patients who received increased immunosuppression alone were effective. Complete DC was achieved in all four patients who received the second transplantation. In conclusion, we emphasize that prompt second transplantation is essential when cellular therapy and enhanced immunosuppression fail for MC patients with cytopenia.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143787701","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Anmol Baranwal, Rabee Kassis, Khalil Hassan, Jade Kutzke, Gabriel Bartoo, Robert Wolf, Aasiya Matin, Mehrdad Hefazi Torghabeh, Abhishek Mangaonkar, Mark R Litzow, Mithun V Shah, William J Hogan, David Dingli, Hassan B Alkhateeb
{"title":"Outcomes of attenuated dose fludarabine and melphalan based conditioning regimen in patients undergoing allogeneic stem cell transplant: a propensity score-matched analysis.","authors":"Anmol Baranwal, Rabee Kassis, Khalil Hassan, Jade Kutzke, Gabriel Bartoo, Robert Wolf, Aasiya Matin, Mehrdad Hefazi Torghabeh, Abhishek Mangaonkar, Mark R Litzow, Mithun V Shah, William J Hogan, David Dingli, Hassan B Alkhateeb","doi":"10.1038/s41409-025-02565-7","DOIUrl":"https://doi.org/10.1038/s41409-025-02565-7","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143787769","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Improved post-transplant outcomes for elderly acute myeloid leukemia patients conditioned with FLU/BU4 rather than conventional MAC regimens.","authors":"Takuya Shimizu, Yasuyuki Arai, Tadakazu Kondo, Shingo Yano, Yoshimitsu Shimomura, Noriko Doki, Takahiro Fukuda, Tetsuya Nishida, Satoshi Takahashi, Shuichi Ota, Yoshinobu Kanda, Takuro Kuriyama, Naoki Kurita, Toshiro Kawakita, Yuta Hasegawa, Nobuhiro Hiramoto, Makoto Onizuka, Yoshiko Atsuta, Masamitsu Yanada","doi":"10.1038/s41409-025-02573-7","DOIUrl":"https://doi.org/10.1038/s41409-025-02573-7","url":null,"abstract":"<p><p>It is uncertain whether FLU/BU4 regimens, classified as myeloablative conditioning (MAC), improve prognosis compared to conventional MAC regimens (conv-MAC) such as CY/TBI and BU/CY. We compared FLU/BU4 with conv-MAC among 6551 patients (FLU/BU4 905, conv-MAC 5646), including acute myeloid leukemia (AML) patients aged 16-59 who received a first allogeneic transplantation from the Japanese nationwide registry. The primary endpoint was overall survival (OS), while secondary endpoints were treatment-related mortality (TRM) and relapse at 3 years. Results indicated comparable OS for conv-MAC regimens among the entire cohort (3-year OS: FLU/BU4 50.4% vs. conv-MAC 55.4%, p < 0.001). Subgroup analysis focusing on elderly patients (aged 50-59) indicated that FLU/BU4 showed a statistically significant improvement in OS (47.0% vs. 42.8%, p = 0.036). Notably, for patients in this cohort transplanted at complete remission (CR), FLU/BU4 demonstrated a substantial advantage over conv-MAC with superior OS (HR 0.75, p = 0.046), lower TRM (HR 0.66, p = 0.035), and comparable relapse (HR 0.84, p = 0.390). These benefits were not observed in elderly patients transplanted at non-CR or in other age groups. In summary, our findings suggest that FLU/BU4 regimen, rather than conv-MAC, may be preferable in MAC-tolerant AML patients, aged 50-59 at CR status. This treatment improves survival by reducing TRM without increasing relapse risk.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143787763","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
M Peña, D F Martinez, A Paviglianiti, A Balaguer, J Sanz, M J Pascual, B Herruzo, C Solano, A Benzaquén, M Q Salas, M Rovira, A Nieto, I Español, M Huguet, L Bento, A J Saéz, A Mussetti
{"title":"Thiotepa, busulfan and fludarabine conditioning regimen for adult patients with lymphoid malignancies undergoing allogeneic stem cell transplantation: a study on behalf of GETH-TC.","authors":"M Peña, D F Martinez, A Paviglianiti, A Balaguer, J Sanz, M J Pascual, B Herruzo, C Solano, A Benzaquén, M Q Salas, M Rovira, A Nieto, I Español, M Huguet, L Bento, A J Saéz, A Mussetti","doi":"10.1038/s41409-025-02559-5","DOIUrl":"https://doi.org/10.1038/s41409-025-02559-5","url":null,"abstract":"<p><p>The efficacy of thiotepa, busulfan, and fludarabine (TBF) conditioning in lymphoproliferative disorders remains under investigation. We analyzed outcomes in 157 patients with lymphoid malignancies who underwent a first allogeneic hematopoietic stem cell transplantation (alloHCT) following TBF conditioning. Non-relapse mortality (NRM) at 3 years reached 32%, while the cumulative incidence of relapse (CIR) was 19%. At 3 years, progression-free survival (PFS), overall survival (OS), and graft-versus-host disease (GVHD)-free/relapse-free survival (GRFS) were 49.7%, 58.0%, and 44.2%, respectively. The cumulative incidences of grade III-IV acute GVHD at 100 days and moderate-to-severe chronic GVHD at 3 years were 8.3% and 12%, respectively. On multivariate analysis, patients receiving high-dose thiotepa (10 mg/kg) demonstrated a significantly lower CIR than those receiving low-dose thiotepa (5 mg/kg) (HR 2.95 [95% CI, 1.37-6.33], p = 0.006), with no significant effect on NRM. Female donor-to-male recipient transplants were associated with reduced OS (HR 2.0 [95% CI, 1.17-3.44], p = 0.011) and increased NRM (HR 2.43 [95% CI, 1.29-4.35], p = 0.005). TBF conditioning demonstrated a substantial anti-tumor effect, counterbalanced by elevated toxicity. Careful patient selection and effective toxicity mitigation strategies are essential to ensure individuals can tolerate TBF's toxicity while maximizing its benefits in disease control.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143771287","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
D P Nurse, H Li, D Cenin, S Patel, D Dima, J Edwards, S Farlow, A Bragg, A Mehdi, R Hanna, S J Rotz, D Jagadeesh, A T Gerds, R M Dean, B Pohlman, B K Hamilton, C Brunstein, M Kalaycio, C S Sauter, R M Sobecks
{"title":"Influence of pre-transplant estimated glomerular filtration rate (eGFR) on clinical outcomes after allogeneic hematopoietic cell transplantation.","authors":"D P Nurse, H Li, D Cenin, S Patel, D Dima, J Edwards, S Farlow, A Bragg, A Mehdi, R Hanna, S J Rotz, D Jagadeesh, A T Gerds, R M Dean, B Pohlman, B K Hamilton, C Brunstein, M Kalaycio, C S Sauter, R M Sobecks","doi":"10.1038/s41409-025-02556-8","DOIUrl":"https://doi.org/10.1038/s41409-025-02556-8","url":null,"abstract":"<p><p>Pretransplant renal dysfunction has historically been associated with increased non-relapse mortality (NRM) and inferior overall survival. Novel approaches in conditioning and GVHD prophylaxis have reduced the toxicity of transplant over time, however, the impact of pre-transplant eGFR in the contemporary era is unknown. The aim of this study was to identify a pre-transplant eGFR value associated with increased transplant-related mortality. This retrospective study was performed using data from 724 adult patients who underwent first allogeneic hematopoietic cell transplant (alloHCT) from January 2012 through December 2021. The optimal pre-transplant eGFR value for risk of NRM was identified using Cox-restricted cubic spline plot analysis. Those with an eGFR <70 ml/min had the highest risk for NRM (p < 0.0001). Multivariate analysis confirmed that the risk of NRM remained significantly higher for eGFR <70 ml/min compared to the other higher eGFR categories, while there were no significant differences between the higher eGFR categories. Pre-transplant renal dysfunction is associated with poor outcomes after alloHCT and remains an important criterion when considering patients for transplant. Efforts to preserve renal function prior to transplant by limiting nephrotoxic exposures may have implications for optimizing outcomes after transplant, particularly in patients with other comorbidities.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143771284","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
David Michonneau, Florent Malard, Sophie Le Grand, Leonardo Magro, Maud D'Aveni, Jean Jacques Tudesq, Alban Villate, Mathieu Meunier, Natacha Maillard, Cristina Castilla-Llorente, Ambroise Marçais, Quentin Cabrera, Anne Huynh, Anne-Lise Menard, Edouard Forcade, Hélène Labussière-Wallet, Nicole Raus, Michael Loschi
{"title":"Efficacy and safety of belumosudil for treatment of cGVHD: multicenter retrospective analysis of the French cohort of the compassionate use program, on behalf of the French Society of Bone Marrow Transplantation and Cellular Therapy.","authors":"David Michonneau, Florent Malard, Sophie Le Grand, Leonardo Magro, Maud D'Aveni, Jean Jacques Tudesq, Alban Villate, Mathieu Meunier, Natacha Maillard, Cristina Castilla-Llorente, Ambroise Marçais, Quentin Cabrera, Anne Huynh, Anne-Lise Menard, Edouard Forcade, Hélène Labussière-Wallet, Nicole Raus, Michael Loschi","doi":"10.1038/s41409-025-02554-w","DOIUrl":"https://doi.org/10.1038/s41409-025-02554-w","url":null,"abstract":"<p><p>Chronic graft versus host disease is a major cause of morbidity after allogeneic haematopoietic cell transplantation. Belumosudil has recently been approved for the treatment of cGVHD refractory after two lines of treatment. However, few data are available to evaluate its efficacy and safety in real life. 68 patients with cGVHD received belumosudil through a compassionate access program in France. The median follow-up was 337 days from belumosudil initiation. Eighty-two percent of patients had severe cGVHD with a median of three organs involved. Patients had received a median of three prior treatment lines. Median treatment duration was 251 days. The best overall response rate (ORR) was 57.3%, including 14.7% complete remission (CR) and 42.6% partial response (PR). The ORR at three and six months was 47% and 45.6%, respectively. Liver and mouth involvement showed the highest response rates (72.7% and 70.4%), while lung involvement had the lowest (17.2%). Median failure-free survival (FFS) was not reached, with 6- and 12-month FFS rates of 89.1% and 80.4%, respectively. Nine patients died, mainly from GVHD (n = 5). Ten adverse events were reported, leading to treatment discontinuation in three cases. These results support the efficacy and safety of belumosudil in refractory cGVHD.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143762990","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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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