Leukemia researchPub Date : 2025-05-26DOI: 10.1016/j.leukres.2025.107721
Xingying Li , Yu Cai , Jun Yang , Chongmei Huang , Xiaowei Xu , Huiying Qiu , Jiahua Niu , Kun Zhou , Ying Zhang , Xinxin Xia , Yu Wei , Chang Shen , Yin Tong , Baoxia Dong , Liping Wan , Xianmin Song
{"title":"Parous female donor is not inferior to the young male donor under the low-dose ATG/PTCy combination-based regimen for GVHD prophylaxis in haploidentical peripheral blood stem cell transplantation","authors":"Xingying Li , Yu Cai , Jun Yang , Chongmei Huang , Xiaowei Xu , Huiying Qiu , Jiahua Niu , Kun Zhou , Ying Zhang , Xinxin Xia , Yu Wei , Chang Shen , Yin Tong , Baoxia Dong , Liping Wan , Xianmin Song","doi":"10.1016/j.leukres.2025.107721","DOIUrl":"10.1016/j.leukres.2025.107721","url":null,"abstract":"<div><h3>Background</h3><div>An optimal donor is critical for patient survival undergoing haploidentical hematopoietic stem cell transplantation (haplo-HSCT). The impacts of different donors on outcomes under the combination regimens of anti-thymocyte globulin (ATG) and post-transplant cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis had limited data. Methods: Therefore, a single-center retrospective study was performed for patients undergoing haploidentical peripheral blood stem cell transplantation (haplo-PBSCT) with ATG/PTCy combination-based regimen. Results: The donors were divided into young male (age <30 years, n = 74), non-parous female (age <30 years, n = 22), older male (age ≥30 years, n = 117), and parous female (age ≥30 years, n = 42) donor groups. The cumulative incidences (CIs) of acute and chronic GVHD, and survival outcomes were similar between the 4 groups (P > 0.050), while the parous female donor could significantly reduce the cumulative incidence of relapse (CIR). Conclusions: The results showed that the parous female donor could significantly reduce CIR and might improve survival under the ATG and PTCy combination-based GVHD prophylaxis regimen after haplo-PBSCT.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"154 ","pages":"Article 107721"},"PeriodicalIF":2.1,"publicationDate":"2025-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144168242","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Treatment-free remission in chronic myeloid leukemia with rare ABL1 gene fusions: Real-life study from the French CML group Fi-LMC","authors":"Hyacinthe Johnson-Ansah , Aude Charbonnier , Gabriel Etienne , Lydia Roy , Laurence Legros , Gabrielle Roth-Guépin , Corentin Orvain , Anne Bouvier , Emilie Cayssials , Valérie Coiteux , Philippe Rousselot , Dina Naguib , Gandhi Damaj , Delphine Lebon , François-Xavier Mahon , Pascale Flandrin-Gresta , Stéphanie Dulucq , Sandrine Hayette , Jean-Michel Cayuela , Franck-Emmanuel Nicolini , Delphine Rea","doi":"10.1016/j.leukres.2025.107716","DOIUrl":"10.1016/j.leukres.2025.107716","url":null,"abstract":"<div><div>Tyrosine kinase inhibitors of the BCR::ABL1 oncoprotein can be stopped without subsequent molecular relapse or major safety concerns in 40–80 % of adult patients with P210<sup>BCR::ABL1</sup> positive chronic myeloid leukemia with sustained deep molecular responses. In contrast, ending treatment in patients with rare rearrangements located outside the major <em>BCR</em> region or within the exon 3 of <em>ABL1</em> remains to be explored. Twenty-four patients with chronic phase disease and diverse uncommon <em>BCR::ABL1</em> transcripts who obtained sustained molecular residual disease negativity and stopped therapy in a real-life setting for various reasons were retrospectively evaluated for treatment-free remission determinants. Six patients relapsed after a median time of 6 months (range; 3–49), relapse being defined as a rise in molecular residual disease above the 3-log threshold. Treatment-free remission probabilities at 12 and 60 months were 83.3 % (95 % CI: 68.4–98.2 %) and 70.6 % (95 % CI: 49.5–91.6), respectively. The type of <em>BCR::ABL1</em> transcript was the only relevant baseline factor associated with durable treatment-free remission and patients with fusions lacking exon a2 sequences had the best outcome. To conclude, treatment-free remission is a reasonably achievable goal in patients with rare <em>ABL1</em> fusion transcripts. Our results pave the way for recommendations in clinical practice. Nevertheless, further research is needed to determine which patients have highest chances to reach deep molecular response levels and become free from therapy and to decipher the biological impact of the different molecular rearrangements of <em>BCR::ABL1</em> on treatment-free remission.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"154 ","pages":"Article 107716"},"PeriodicalIF":2.1,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144106831","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Leukemia researchPub Date : 2025-05-13DOI: 10.1016/j.leukres.2025.107718
Laurenz Steiner , Navkirandeep Kaur , Johann-Christoph Jann , Alice Fabarius , Georgia Metzgeroth , Manja Meggendorfer , Wolf-Karsten Hofmann , Mohamad Jawhar , Daniel Nowak , Alexander Streuer
{"title":"Inter-modality variance of blast quantification in patients with myelodysplastic neoplasms (MDS) and its impact on risk stratification and overall survival","authors":"Laurenz Steiner , Navkirandeep Kaur , Johann-Christoph Jann , Alice Fabarius , Georgia Metzgeroth , Manja Meggendorfer , Wolf-Karsten Hofmann , Mohamad Jawhar , Daniel Nowak , Alexander Streuer","doi":"10.1016/j.leukres.2025.107718","DOIUrl":"10.1016/j.leukres.2025.107718","url":null,"abstract":"<div><div>In the revised International Prognostic Scoring System (IPSS-R), blast enumeration by bone marrow cytology (BM-c) is crucial for risk stratification in myelodysplastic neoplasms (MDS), in the IPSS-Molecular (IPSS-M) however, incorporation of molecular data gained more prognostic impact. Blast count differences in the commonly used methods BM-c, flow cytometry (BM-f) and histology (BM-h) could possibly impact categorisation and overall survival (OS). In n = 145 investigated cases treated since 2012 in our institution, discordance in IPSS-R blast categories was found in 62/145 (43 %) of cases with evaluable blast counts in ≥ 2 methods. Discordant cases scored by BM-c showed either no change (24 %), a downgrade (48 %) or an upgrade (28 %) of their IPSS-R category when applying BM-f or BM-h. Discordant LR-MDS patients had significantly worse OS than concordant (72 vs 35 months, p = 0.031). In contrast, stratification by IPSS-M revealed no OS difference for discordant LR-MDS patients (p = 0.46). We could demonstrate that discordance occurred in almost half of patients, leading to re-stratification in a substantial amount of cases. Furthermore, OS was worse for discordant patients, but differences were ameliorated by inclusion of molecular data. This highlights the growing importance of molecular data over blast quantification in MDS.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"154 ","pages":"Article 107718"},"PeriodicalIF":2.1,"publicationDate":"2025-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144068045","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Leukemia researchPub Date : 2025-05-13DOI: 10.1016/j.leukres.2025.107715
Sabrina Mariani , Luca Maurillo , Monica Piedimonte, Chiara Sarlo, Luana Fianchi, Francesco Buccisano, Maria Teresa Voso, Susanna Fenu, Anna Lina Piccioni, Tommaso Caravita di Toritto, Ida Carmosino, Laura Cicconi, Alessandro Pulsoni, Michelina Santopietro, Roberto Latagliata, Ambra Di Veroli, Emiliano Fabiani, Martina Quattrone, Flavia Mallegni, Francesca Romana Iovene, Agostino Tafuri
{"title":"Luspatercept in lower-myelodysplastic syndromes (MDS): Real-world data from the Gruppo Romano-Laziale Mielodisplasie (GROM-L) and review of literature","authors":"Sabrina Mariani , Luca Maurillo , Monica Piedimonte, Chiara Sarlo, Luana Fianchi, Francesco Buccisano, Maria Teresa Voso, Susanna Fenu, Anna Lina Piccioni, Tommaso Caravita di Toritto, Ida Carmosino, Laura Cicconi, Alessandro Pulsoni, Michelina Santopietro, Roberto Latagliata, Ambra Di Veroli, Emiliano Fabiani, Martina Quattrone, Flavia Mallegni, Francesca Romana Iovene, Agostino Tafuri","doi":"10.1016/j.leukres.2025.107715","DOIUrl":"10.1016/j.leukres.2025.107715","url":null,"abstract":"","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"154 ","pages":"Article 107715"},"PeriodicalIF":2.1,"publicationDate":"2025-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144069757","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Leukemia researchPub Date : 2025-05-05DOI: 10.1016/j.leukres.2025.107713
Usama Qamar, Siddharth Agarwal, Mohamad Khawandanah, Manu Pandey, Taha AlJuhaishi, Zain Ul Abideen Asad
{"title":"Cardiovascular disease-related mortality trends among adults with multiple myeloma in the United States from 1999 to 2023","authors":"Usama Qamar, Siddharth Agarwal, Mohamad Khawandanah, Manu Pandey, Taha AlJuhaishi, Zain Ul Abideen Asad","doi":"10.1016/j.leukres.2025.107713","DOIUrl":"10.1016/j.leukres.2025.107713","url":null,"abstract":"","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"154 ","pages":"Article 107713"},"PeriodicalIF":2.1,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924762","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Pre-transplant blinatumomab and/or inotuzumab ozogamicin therapy for relapsed/refractory acute lymphoblastic and B/myeloid mixed phenotype acute leukemia in adults","authors":"Kaori Kondo , Yuho Najima , Daichi Sadato , Chizuko Hirama , Kana Kato , Yasutaka Sadaga , Chika Kato , Satoshi Sakai , Yasuhiro Kambara , Yoshimi Nabe , Koh Teshima , Kazuya Asano , Kazuya Kurihara , Masashi Shimabukuro , Atushi Jinguji , Fumihiko Ouchi , Kazuki Inai , Satoshi Koi , Naoki Shingai , Kyoko Haraguchi , Noriko Doki","doi":"10.1016/j.leukres.2025.107704","DOIUrl":"10.1016/j.leukres.2025.107704","url":null,"abstract":"<div><div>Real-world data on blinatumomab (BLI) and inotuzumab ozogamicin (INO) for relapsed or refractory B-acute lymphoblastic leukemia (RR-ALL) before hematopoietic stem cell transplantation (HCT) are limited. To compare the efficacy of salvage therapy with BLI and/or INO and conventional chemotherapy as a bridge to HCT, we retrospectively evaluated patients with RR-ALL who underwent first HCT at our institute between 2004 and 2023. Based on whether they had received salvage therapy with BLI and/or INO, 70 recipients were divided into a BLI/INO (n = 22) and a control group (n = 48). The complete remission (CR) rate before HCT was higher in the BLI/INO group than in the control group (77.3 % vs. 35.4 %, p = 0.002). Two years after the first HCT, the overall survival (OS) and disease-free survival (DFS) were significantly higher in the BLI/INO group than in the control group (OS, 63.0 % vs. 31.2 %, p = 0.022; DFS 49.6 % vs. 22.9 %, p = 0.049), with comparable cumulative incidence of relapse (CIR, 41.3 % vs. 47.9 %; p = 0.767) and lower tendency of non-relapse mortality (NRM, 9.1 % vs. 29.2 %; p = 0.057). Multivariate analysis revealed that non-CR status before HCT was the only factor associated with poor OS (hazard ratio [HR], 4.263; p < 0.001) and higher CIR (HR, 2.250; p = 0.048). In patients in CR at HCT, there was no difference in HCT outcomes at 2 years (OS, 82.4 % vs. 58.8 %; p = 0.324; DFS, 64.2 % vs. 47.1 %; p = 0.496; CIR, 24.1 % vs. 41.2 %; p = 0.375; NRM, 11.8 % vs. 11.8 %; p = 0.950). BLI and/or INO therapy for RR-ALL was associated with better survival after HCT, probably due to the higher CR rate.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"153 ","pages":"Article 107704"},"PeriodicalIF":2.1,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143886158","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Leukemia researchPub Date : 2025-04-22DOI: 10.1016/j.leukres.2025.107703
Gentiana Elena Trotta , Enrico Santinelli , Sabrina Mariani , Katia Paciaroni , Tommaso Caravita di Toritto , Natalia Cenfra , Susanna Fenu , Paola Ricci , Luca Maurillo , Federico Moretti , Emiliano Fabiani , Carmelo Gurnari , Maria Teresa Voso , on behalf of Gruppo Romano Laziale Mielodisplasie (GROM-L)
{"title":"The role of morphology in diagnosing MDS with Non-clonal cytopenias","authors":"Gentiana Elena Trotta , Enrico Santinelli , Sabrina Mariani , Katia Paciaroni , Tommaso Caravita di Toritto , Natalia Cenfra , Susanna Fenu , Paola Ricci , Luca Maurillo , Federico Moretti , Emiliano Fabiani , Carmelo Gurnari , Maria Teresa Voso , on behalf of Gruppo Romano Laziale Mielodisplasie (GROM-L)","doi":"10.1016/j.leukres.2025.107703","DOIUrl":"10.1016/j.leukres.2025.107703","url":null,"abstract":"<div><div>Myelodysplastic syndromes/neoplasms (MDS) are a heterogeneous group of blood disorders characterized by distinctive biological and clinical features. Diagnosis typically relies on bone marrow examination and cytogenetic/molecular analysis. However, a small subset of patients lacks detectable cytogenetic or genetic abnormalities, making diagnosis challenging and reliant solely on marrow dysplasia and cytopenia. Here, we conducted a multicenter retrospective study investigating the clinical and hematological features of patients with MDS lacking detectable cytogenetic or molecular alterations. From a cohort of 329 MDS patients who underwent genetic testing at our laboratory of “Diagnostica Avanzata Oncoematologica”, we selected 62 patients presenting with unexplained cytopenia, suspected MDS, and a non-informative cytogenomic profile. Of these, 26 were ultimately diagnosed with alternative conditions such as aplastic anemia, drug/radiation toxicity, multiple myeloma, and others. The patients with non-informative genetic profile had a median age of 60 years and a female predominance (67 %). Autoimmune diseases (AD) were the most frequently observed comorbidities (26 %), and neutropenia was the most common type of cytopenia (56 %), followed by thrombocytopenia (22 %), and anemia (19 %). Most patients had bi-lineage or tri-lineage dysplasia, and had low-risk disease according to IPSS-R and IPSS-M. Only one patient progressed to AML at a median follow-up of 12 months (IQR for the test cohort: 2–25). MDS lacking classical myeloid clonal alteration represent a unique clinical-biological subtype whereby marrow assessment along with a thorough clinical workup is crucial.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"153 ","pages":"Article 107703"},"PeriodicalIF":2.1,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143868221","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Leukemia researchPub Date : 2025-04-20DOI: 10.1016/j.leukres.2025.107702
Gianluca Cavallaro , Silvia Salmoiraghi , Roberta Cavagna , Chiara Pavoni , Clara Belotti , Martina Milani , Elena Oldani , Marco Frigeni , Tamara Intermesoli , Anna Grassi , Renato Bassan , Orietta Spinelli , Alessandro Rambaldi , Federico Lussana
{"title":"Clinical impact of tyrosine kinases related mutations in NPM1 mutated acute myeloid leukaemia: A report from the Northern Italy Leukaemia Group (NILG) multicentre randomized trial 02/06","authors":"Gianluca Cavallaro , Silvia Salmoiraghi , Roberta Cavagna , Chiara Pavoni , Clara Belotti , Martina Milani , Elena Oldani , Marco Frigeni , Tamara Intermesoli , Anna Grassi , Renato Bassan , Orietta Spinelli , Alessandro Rambaldi , Federico Lussana","doi":"10.1016/j.leukres.2025.107702","DOIUrl":"10.1016/j.leukres.2025.107702","url":null,"abstract":"<div><div>Co-mutational patterns play a role in the clinical outcome of nucleophosmin-1 (<em>NPM1</em>) mutated acute myeloid leukaemia (AML). Tyrosine kinase (TK) related gene mutations are included in these additional mutations, but their impact on prognosis of <em>NPM1</em>-mutated AML is unknown. We analyzed the outcomes of patients with <em>NPM1</em> AML with TK related mutations treated with intensive chemotherapy in the prospective randomized trial NILG AML 02/06, with regards to <em>NPM1-FLT3-ITD</em> and <em>NPM1</em>-isolated. Data show that additional TK related mutations do not impact the favorable prognosis on <em>NPM1-</em>mutated AML, unlike <em>NPM1-FLT3-ITD</em> mutated AML, for which consolidation with allogeneic stem cell transplantation should be considered for all young and fit patients in first complete remission.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"153 ","pages":"Article 107702"},"PeriodicalIF":2.1,"publicationDate":"2025-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143868329","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}