LeukemiaPub Date : 2025-02-24DOI: 10.1038/s41375-025-02536-3
Valentina Giudice, Carmine Selleri
{"title":"How fast does leukemia progress?","authors":"Valentina Giudice, Carmine Selleri","doi":"10.1038/s41375-025-02536-3","DOIUrl":"10.1038/s41375-025-02536-3","url":null,"abstract":"","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 4","pages":"805-809"},"PeriodicalIF":12.8,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143477505","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
LeukemiaPub Date : 2025-02-24DOI: 10.1038/s41375-025-02534-5
Rahul Banerjee, Amber R. Fritz, Othman S. Akhtar, Ciara L. Freeman, Andrew J. Cowan, Nina Shah, Heather J. Landau, Shaji K. Kumar, Dan T. Vogl, Yvonne A. Efebera, Philip L. McCarthy, David H. Vesole, Adam Mendizabal, Amrita Y. Krishnan, George Somlo, Edward A. Stadtmauer, Marcelo C. Pasquini
{"title":"Urine-free response criteria predict progression-free survival in multiple myeloma: a post hoc analysis of BMT CTN 0702","authors":"Rahul Banerjee, Amber R. Fritz, Othman S. Akhtar, Ciara L. Freeman, Andrew J. Cowan, Nina Shah, Heather J. Landau, Shaji K. Kumar, Dan T. Vogl, Yvonne A. Efebera, Philip L. McCarthy, David H. Vesole, Adam Mendizabal, Amrita Y. Krishnan, George Somlo, Edward A. Stadtmauer, Marcelo C. Pasquini","doi":"10.1038/s41375-025-02534-5","DOIUrl":"10.1038/s41375-025-02534-5","url":null,"abstract":"","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 4","pages":"1001-1004"},"PeriodicalIF":12.8,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143477562","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
LeukemiaPub Date : 2025-02-22DOI: 10.1038/s41375-025-02542-5
Jingru Huang, Jiaying Xie, Yin Wang, Mengyao Sheng, Yue Sun, Pingyue Chen, Shaoqin Rong, Dongrui Yin, Yuanxian Wang, Ping Zhu, Stefan K. Bohlander, Guo-Liang Xu, Hai Gao, Dan Zhou, Yuheng Shi
{"title":"STING mediates increased self-renewal and lineage skewing in DNMT3A-mutated hematopoietic stem/progenitor cells","authors":"Jingru Huang, Jiaying Xie, Yin Wang, Mengyao Sheng, Yue Sun, Pingyue Chen, Shaoqin Rong, Dongrui Yin, Yuanxian Wang, Ping Zhu, Stefan K. Bohlander, Guo-Liang Xu, Hai Gao, Dan Zhou, Yuheng Shi","doi":"10.1038/s41375-025-02542-5","DOIUrl":"10.1038/s41375-025-02542-5","url":null,"abstract":"Somatic mutations in DNA methyltransferase 3 A (DNMT3A) are frequently observed in patients with hematological malignancies. Hematopoietic stem/progenitor cells (HSPCs) with mutated DNMT3A demonstrate increased self-renewal activity and skewed lineage differentiation. However, the molecular mechanisms underlying these changes remain largely unexplored. In this study, we show that Dnmt3a loss leads to the upregulation of endogenous retroviruses (ERVs) in HSPCs, subsequently activating the cGAS-STING pathway and triggering inflammatory responses in these cells. Both genetic and pharmacological inhibition of STING effectively corrects the increased self-renewal activity and differentiation skewing induced by Dnmt3a deficiency in mice. Notably, targeting STING showed inhibited acute myeloid leukemia (AML) development in a Dnmt3a-KO; Flt3-ITD AML model, comparable to AC220, an FDA-approved FLT3-ITD inhibitor. A patient-derived xenograft (PDX) model further demonstrated that targeting STING effectively alleviates the leukemic burden of DNMT3A-mutant AML. Collectively, our findings highlight a critical role for STING in hematopoietic disorders induced by DNMT3A mutations and propose STING as a potential therapeutic target for preventing the progression of DNMT3A mutation-associated leukemia.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 4","pages":"929-941"},"PeriodicalIF":12.8,"publicationDate":"2025-02-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41375-025-02542-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143470701","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Distinct leukemogenic mechanism of acute promyelocytic leukemia based on genomic structure of PML::RARα","authors":"Mariko Minami, Teppei Sakoda, Gentaro Kawano, Yu Kochi, Kensuke Sasaki, Takeshi Sugio, Fumiaki Jinnouchi, Kohta Miyawaki, Yuya Kunisaki, Koji Kato, Toshihiro Miyamoto, Koichi Akashi, Yoshikane Kikushige","doi":"10.1038/s41375-025-02530-9","DOIUrl":"10.1038/s41375-025-02530-9","url":null,"abstract":"Leukemic stem cells (LSCs) of acute myeloid leukemia (AML) can be enriched in the CD34+CD38- fraction and reconstitute human AML in vivo. However, in acute promyelocytic leukemia (APL), which constitutes 10% of all AML cases and is driven by promyelocytic leukemia-retinoic acid receptor alpha (PML::RARα) fusion genes, the presence of LSCs has long been unidentified because of the difficulty in efficient reconstitution of human APL in vivo. Herein, we show that LSCs of the short-type isoform APL, a subtype of APL defined by different breakpoints of the PML gene, concentrate in the CD34+CD38− fraction and express T cell immunoglobulin mucin-3 (TIM-3). Short-type APL cells exhibited distinct gene expression signatures, including LSC-related genes, compared to the other types of APL. Moreover, CD34+CD38−TIM-3+ short-type APL cells efficiently reconstituted human APL in xenograft models with high penetration, whereas CD34− differentiated APL cells did not. Furthermore, CD34+CD38−TIM-3+ short-type APL cells reconstituted leukemia cells after serial transplantation. Thus, short-type APL was hierarchically organized by self-renewing APL-LSCs. The identification of LSCs in a subset of APL and establishment of an efficient patient-derived xenograft model may contribute to further understanding the APL leukemogenesis and devise individual treatments for the eradication of APL LSCs.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 4","pages":"844-853"},"PeriodicalIF":12.8,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143451472","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
LeukemiaPub Date : 2025-02-19DOI: 10.1038/s41375-025-02538-1
Fumiya Wada, Makoto Iwasaki, Masahiro Hirayama, Koji Kawamura, Katsuji Kaida, Noriko Doki, Hirohisa Nakamae, Yuta Hasegawa, Takahiro Fukuda, Tetsuya Eto, Nobuhiro Hiramoto, Yumiko Maruyama, Koji Nagafuji, Shuichi Ota, Jun Ishikawa, Toshihiko Ando, Tatsuo Ichinohe, Yoshiko Atsuta, Hideki Nakasone, Junya Kanda
{"title":"Donor selection in T-cell-replete haploidentical donor peripheral blood stem cell transplantation","authors":"Fumiya Wada, Makoto Iwasaki, Masahiro Hirayama, Koji Kawamura, Katsuji Kaida, Noriko Doki, Hirohisa Nakamae, Yuta Hasegawa, Takahiro Fukuda, Tetsuya Eto, Nobuhiro Hiramoto, Yumiko Maruyama, Koji Nagafuji, Shuichi Ota, Jun Ishikawa, Toshihiko Ando, Tatsuo Ichinohe, Yoshiko Atsuta, Hideki Nakasone, Junya Kanda","doi":"10.1038/s41375-025-02538-1","DOIUrl":"10.1038/s41375-025-02538-1","url":null,"abstract":"The effects of donor characteristics on outcomes after T-cell-replete (TCR) haploidentical donor peripheral blood stem cell transplantation (PBSCT) with post-transplant cyclophosphamide (PTCy) or low-dose antithymocyte globulin (ATG) remain unclear. We evaluated the impact in 1,677 patients who received a PTCy protocol (PTCy-haplo; n = 1,107) or low-dose ATG protocol (ATG-haplo; n = 570). A low CD34+ cell dose (<4 ×106/kg) was the only donor characteristic associated with worse overall survival (OS) after PTCy-haplo (adjusted hazard ratios [aHR] = 1.49, P = 0.008), whereas increasing donor age by decade (aHR = 1.12, P = 0.008) and human leukocyte antigen 2-3 antigen mismatches (aHR = 1.46, P = 0.010), compared to HLA 0-1 antigen mismatches, were associated with worse OS after ATG-haplo. Increasing donor age was associated with a high risk of grade III–IV acute GVHD both after PTCy-haplo (HR: 1.32, P = 0.009) and ATG-haplo (HR: 1.22, P = 0.006). Offspring donors had better relapse-free survival and GVHD-free relapse-free survival than sibling donors after ATG-haplo. Our data highlights the donor characteristics associated with improved transplant outcomes after TCR haploidentical donor PBSCT with PTCy or low-dose ATG.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 4","pages":"951-961"},"PeriodicalIF":12.8,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143443167","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
LeukemiaPub Date : 2025-02-17DOI: 10.1038/s41375-025-02529-2
Femke M. Hormann, Sean G. Rudd
{"title":"Nelarabine in T-cell acute lymphoblastic leukemia: intracellular metabolism and molecular mode-of-action","authors":"Femke M. Hormann, Sean G. Rudd","doi":"10.1038/s41375-025-02529-2","DOIUrl":"10.1038/s41375-025-02529-2","url":null,"abstract":"T-cell acute lymphoblastic leukemia (T-ALL) patients often have a poor 5-year event-free survival. The only T-ALL specific drug in clinical practice is nelarabine. A prodrug of the deoxyguanosine analog ara-G, nelarabine is a rationally designed agent selective for the treatment of T-cell malignancies. Originally approved for relapsed/refractory T-ALL, it is increasingly used in T-ALL therapy and is currently being evaluated in upfront treatment. Whilst the clinical use of nelarabine has been the topic of multiple review articles, a thorough overview of the preclinical data detailing the molecular underpinnings of its anti-leukemic activity is lacking, which is critical to inform mechanism-based use. Thus, in the present article we conducted a semi-systematic review of the literature and critically evaluated the preclinical knowledge on the molecular pharmacology of nelarabine. Whilst early studies identified ara-G triphosphate to be the principal active metabolite and nuclear DNA synthesis to be a key target, many fundamental questions remain that could inform upon future use of this therapy. These include the nature of nelarabine-induced DNA lesions and their repair, together with additional cellular targets of ara-G metabolites and their role in efficacy and toxicity. A critical avenue of research in need of development is investigation of nelarabine combination therapies, both in the context of current T-ALL chemotherapy regimens and with emerging anti-leukemic agents, and we highlight some areas to pursue. Altogether, we discuss what we can learn from the preclinical literature as a whole and present our view for future research regarding nelarabine treatment in T-ALL.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 3","pages":"531-542"},"PeriodicalIF":12.8,"publicationDate":"2025-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41375-025-02529-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Improved prognosis of advanced-stage extranodal NK/T-cell lymphoma: results of the NKEA-Next study","authors":"Ayumi Fujimoto, Kana Miyazaki, Kimikazu Yakushijin, Takahiro Fujino, Wataru Munakata, Yasuo Ejima, Dai Maruyama, Nobuko Kubota, Takeshi Maeda, Jun Takizawa, Nobuhiro Hiramoto, Masahiro Takeuchi, Rika Sakai, Noriko Fukuhara, Senzo Taguchi, Naoko Asano, Motoko Yamaguchi, Ritsuro Suzuki","doi":"10.1038/s41375-025-02527-4","DOIUrl":"10.1038/s41375-025-02527-4","url":null,"abstract":"A retrospective study of extranodal natural killer/T-cell lymphoma (ENKL) patients diagnosed between 2014 and 2021 in Japan was conducted. Among 351 patients with sufficient data, 116 (33%) were in the advanced stage (5 in stage III and 111 in stage IV) at diagnosis, and were further analyzed. The median age was 60 years (range: 19–90), and 68 (59%) were male. Ninety-four (85%) of stage IV patients had two or more extranodal involvements. The most common first-line regimen was SMILE (steroid, methotrexate, ifosfamide, L-asparaginase, and etoposide; 52%). The 2-year overall survival (OS) for all patients was 38.5%, which was significantly improved after 2017 (25.2% for 2014–2017 vs. 50.7% for 2018–2021; P = 0.008). Patients treated with SMILE showed better OS than those treated with DeVIC or CHOP (2y-OS: 57.1%, 35.8%, and 0%, respectively; P < 0.001). The prognosis was significantly better in patients who received hematopoietic stem cell transplantation (HSCT) than in those who did not (2-year OS: 68.3% vs. 17.6%, P < 0.001). Multivariate analysis showed SMILE and HSCT were significant factors for OS. In conclusion, the prognosis of advanced-stage ENKL has improved in recent years. The L-asparaginase-containing chemotherapy and subsequent HSCT is considered the recommended strategy.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 4","pages":"909-916"},"PeriodicalIF":12.8,"publicationDate":"2025-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41375-025-02527-4.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434913","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
LeukemiaPub Date : 2025-02-15DOI: 10.1038/s41375-025-02528-3
Sameem M. Abedin, Guru Subramanian Guru Murthy, Mehdi Hamadani, Laura C. Michaelis, Karen-Sue Carlson, Lyndsey Runaas, Katelyn Gauger, Avinash G. Desai, Mary M. Chen, Kate L. Li, Mojisola Rotibi, Umar Syed, Madhuri Vusirikala, Alexandra Harrington, Ehab L. Atallah
{"title":"Phase 1 study of lintuzumab-Ac225 combined with CLAG-M salvage therapy in relapsed/refractory acute myeloid leukemia","authors":"Sameem M. Abedin, Guru Subramanian Guru Murthy, Mehdi Hamadani, Laura C. Michaelis, Karen-Sue Carlson, Lyndsey Runaas, Katelyn Gauger, Avinash G. Desai, Mary M. Chen, Kate L. Li, Mojisola Rotibi, Umar Syed, Madhuri Vusirikala, Alexandra Harrington, Ehab L. Atallah","doi":"10.1038/s41375-025-02528-3","DOIUrl":"10.1038/s41375-025-02528-3","url":null,"abstract":"Lintuzumab-Ac255 is a humanized anti-CD33 antibody linked to Actinium-225 delivering high-energy alpha-particles to leukemia cells, inciting double-strand DNA breaks and cell death. This phase 1 study assessed the safety and efficacy of lintuzumab-Ac225 after CLAG-M salvage therapy in patients with relapsed/refractory acute myeloid leukemia (R/R AML). Primary objectives were determination of maximum tolerated dose (MTD), recommended phase 2 dose (RP2D), and safety. Using a 3 + 3 dose-escalation design, 21 patients were enrolled sequentially into 4 cohorts to receive a lintuzumab-Ac225 infusion (0.25–1.0 µCi/kg) 7 ( + 2) days after CLAG-M (days 1–6); 5 additional patients received the RP2D. Of evaluable patients, 86.7% had high-risk disease. The MTD and RP2D was 0.75 µCi/kg. Common grade 3/4 adverse events were febrile neutropenia (65.4%) and decreased white blood cells (50%). The composite complete remission (CRc) rates (CR/CRi) were 56.6% overall, 50% in patients with mutated TP53, and 38.5% in prior venetoclax-treated patients. Measurable residual disease (MRD)-negativity was achieved in 8 of 12 responders. Among all patients (n = 26), estimated 2-year OS was 23.1% (95% CI, 9.4–40.3) and estimated 1-year PFS was 30.8% (95% CI, 14.6–48.5). Lintuzumab-Ac225 plus CLAG-M was well tolerated with expected, manageable toxicities, while yielding deep and meaningful responses in high-risk R/R AML patients.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 4","pages":"837-843"},"PeriodicalIF":12.8,"publicationDate":"2025-02-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143417438","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
LeukemiaPub Date : 2025-02-12DOI: 10.1038/s41375-025-02520-x
Chunhua Shi, Ze Tian, Jun Yan, Mao Zhang, Pariya Sukhumalchandra, Edward Chang, Guojun Yang, Junping You, Meng Cui, Qing Shi, Celine Kerros, Anne Philips, Na Qiao, Hiroki Torikai, Sathvik Patchametla, Anna Sergeeva, Lisa St. John, Helen He, Dmitri Wiederschain, Benjamin H. Lee, Geraldine L. C. Paulus, Dongxing Zha, Jeffrey Molldrem, Gheath Alatrash
{"title":"Immunotherapy targeting a leader sequence cathepsin G-derived peptide","authors":"Chunhua Shi, Ze Tian, Jun Yan, Mao Zhang, Pariya Sukhumalchandra, Edward Chang, Guojun Yang, Junping You, Meng Cui, Qing Shi, Celine Kerros, Anne Philips, Na Qiao, Hiroki Torikai, Sathvik Patchametla, Anna Sergeeva, Lisa St. John, Helen He, Dmitri Wiederschain, Benjamin H. Lee, Geraldine L. C. Paulus, Dongxing Zha, Jeffrey Molldrem, Gheath Alatrash","doi":"10.1038/s41375-025-02520-x","DOIUrl":"10.1038/s41375-025-02520-x","url":null,"abstract":"Myeloid azurophil granules provide a rich source of intracellular leukemia antigens. Cathepsin G (CG) is a serine protease that has higher expression in acute myeloid leukemia (AML) blasts in comparison to normal myeloid progenitors. Based on the unique biology of HLA-A*0201 (HLA-A2), in which presentation of leader sequence (LS)-derived peptides is favored, we focused on the LS-CG-derived peptide CG1 (FLLPTGAEA). We previously detected CG1/HLA-A2 complexes on the surface of primary HLA-A2+ AML blasts and cell lines, and immunity targeting CG1/HLA-A2 in leukemia patients. T cell receptor (TCR)-mimic (m) antibodies are immunotherapeutic antibodies that target peptide-HLA (pHLA) complexes. Here we report on the engineering, preclinical efficacy, and safety evaluation of a novel CG1/HLA-A2-targeting, T cell-engager, bispecific antibody (CG1/A2xCD3). CG1/A2xCD3 showed high binding affinity to CG1/HLA-A2 monomers, CD3-Fc fusion protein, and to AML and T cells, with potent killing of HLA-A2+ primary AML and cell lines in vitro and in vivo. This correlated with both tumor- and CG1/A2xCD3-dependent T cell activation and cytokine secretion. Lastly, CG1/A2xCD3 had no activity against normal bone marrow. Together, these results support the targeting of LS-derived peptides and the continued clinical development of CG1/A2xCD3 in the setting of AML.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 4","pages":"888-898"},"PeriodicalIF":12.8,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41375-025-02520-x.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143393112","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
LeukemiaPub Date : 2025-02-11DOI: 10.1038/s41375-025-02526-5
Aldo A. Acosta-Medina, Jithma P. Abeykoon, Saurabh Zanwar, Gordon J. Ruan, Karen L. Rech, Aishwarya Ravindran, N. Nora Bennani, Caroline J. Davidge-Pitts, Matthew J. Koster, Jay H. Ryu, Mithun V. Shah, W. Oliver Tobin, Robert Vassallo, Jason R. Young, Ronald S. Go, Gaurav Goyal, on behalf of the Mayo Clinic - University of Alabama at Birmingham Histiocytosis Working Group
{"title":"Efficacy of MEK inhibitors in Erdheim-Chester disease: impact of MAPK pathway pathogenic variants","authors":"Aldo A. Acosta-Medina, Jithma P. Abeykoon, Saurabh Zanwar, Gordon J. Ruan, Karen L. Rech, Aishwarya Ravindran, N. Nora Bennani, Caroline J. Davidge-Pitts, Matthew J. Koster, Jay H. Ryu, Mithun V. Shah, W. Oliver Tobin, Robert Vassallo, Jason R. Young, Ronald S. Go, Gaurav Goyal, on behalf of the Mayo Clinic - University of Alabama at Birmingham Histiocytosis Working Group","doi":"10.1038/s41375-025-02526-5","DOIUrl":"10.1038/s41375-025-02526-5","url":null,"abstract":"","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 4","pages":"991-994"},"PeriodicalIF":12.8,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41375-025-02526-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143385120","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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