Leukemia最新文献_第5页

Capillary leak phenotype as a major cause of death in patients with POEMS syndrome 毛细血管渗漏表型是POEMS综合征患者死亡的主要原因

IF 11.4 1区医学

Leukemia Pub Date : 2024-12-16 DOI: 10.1038/s41375-024-02489-z

Kenzie Lee, Taxiarchis Kourelis, Marcella Tschautscher, Rahma Warsame, Francis Buadi, Morie Gertz, Eli Muchtar, David Dingli, Suzanne Hayman, Ronald Go, Lisa Hwa, Amie Fonder, Wilson Gonsalves, Miriam Hobbs, Robert Kyle, Prashant Kapoor, Nelson Leung, Moritz Binder, Joselle Cook, Yi Lin, Michelle Rogers, S. Vincent Rajkumar, Shaji Kumar, Angela Dispenzieri

{"title":"Capillary leak phenotype as a major cause of death in patients with POEMS syndrome","authors":"Kenzie Lee, Taxiarchis Kourelis, Marcella Tschautscher, Rahma Warsame, Francis Buadi, Morie Gertz, Eli Muchtar, David Dingli, Suzanne Hayman, Ronald Go, Lisa Hwa, Amie Fonder, Wilson Gonsalves, Miriam Hobbs, Robert Kyle, Prashant Kapoor, Nelson Leung, Moritz Binder, Joselle Cook, Yi Lin, Michelle Rogers, S. Vincent Rajkumar, Shaji Kumar, Angela Dispenzieri","doi":"10.1038/s41375-024-02489-z","DOIUrl":"https://doi.org/10.1038/s41375-024-02489-z","url":null,"abstract":"Cause of death (COD) in POEMS (polyneuropathy, organomegaly, endocrinopathies, monoclonal protein and skin changes) syndrome is not well described. We investigated COD in patients with POEMS syndrome treated at Mayo Clinic between 2000 and 2022. Of the 89 deaths, 49 patients had known COD and were the subject of this study. Seventeen patients died of unrelated causes, while 32 patients (65%) died from causes related to POEMS syndrome including secondary malignancies like myelodysplastic syndrome and acute leukemia (n = 5) and complications from active therapy (n = 5). Notably, 19 patients died with a stereotypic syndrome we termed capillary leak phenotype (CLP), which was characterized by refractory ascites, effusions and/or anasarca that ultimately resulted in hypotension, renal failure and cardiopulmonary arrest. Alternate causes for these symptoms, such as cardiac and hepatic etiologies, were excluded. CLP as a COD was an earlier event with a median time from diagnosis to death of 2.5 years compared to 12.0 years for all other deceased patients (p = <0.0001). By definition, treatment of terminal CLP was unsuccessful with median survival of only 4 months after CLP onset. The driver of CLP is unknown, but recognition as an entity should allow for systematic study.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"18 1","pages":""},"PeriodicalIF":11.4,"publicationDate":"2024-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142825130","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}

引用次数: 0

Co-targeting of the thymic stromal lymphopoietin receptor to decrease immunotherapeutic resistance in CRLF2-rearranged Ph-like and Down syndrome acute lymphoblastic leukemia 共同靶向胸腺基质淋巴生成素受体，降低 CRLF2 重排的 Ph 型和唐氏综合征急性淋巴细胞白血病的免疫治疗抗药性

IF 11.4 1区医学

Leukemia Pub Date : 2024-12-16 DOI: 10.1038/s41375-024-02493-3

Tommaso Balestra, Lisa M Niswander, Asen Bagashev, Joseph P Loftus, Savannah L Ross, Robert K Chen, Samantha M McClellan, Jacob J Junco, Diego A Bárcenas López, Karen R. Rabin, Terry J Fry, Sarah K Tasian

{"title":"Co-targeting of the thymic stromal lymphopoietin receptor to decrease immunotherapeutic resistance in CRLF2-rearranged Ph-like and Down syndrome acute lymphoblastic leukemia","authors":"Tommaso Balestra, Lisa M Niswander, Asen Bagashev, Joseph P Loftus, Savannah L Ross, Robert K Chen, Samantha M McClellan, Jacob J Junco, Diego A Bárcenas López, Karen R. Rabin, Terry J Fry, Sarah K Tasian","doi":"10.1038/s41375-024-02493-3","DOIUrl":"https://doi.org/10.1038/s41375-024-02493-3","url":null,"abstract":"CRLF2 rearrangements occur in >50% of Ph-like and Down syndrome (DS)-associated B-acute lymphoblastic leukemia (ALL) and induce constitutive kinase signaling targetable by the JAK1/2 inhibitor ruxolitinib under current clinical investigation. While chimeric antigen receptor T cell (CART) immunotherapies have achieved remarkable remission rates in children with relapsed/refractory B-ALL, ~50% of CD19CART-treated patients relapse again, many with CD19 antigen loss. We previously reported preclinical activity of thymic stromal lymphopoietin receptor-targeted cellular immunotherapy (TSLPRCART) against CRLF2-overexpressing ALL as an alternative approach. In this study, we posited that combinatorial TSLPRCART and ruxolitinib would have superior activity and first validated potent TSLPRCART-induced inhibition of leukemia proliferation in vitro in CRLF2-rearranged ALL cell lines and in vivo in Ph-like and DS-ALL patient-derived xenograft (PDX) models. However, simultaneous TSLPRCART/ruxolitinib or CD19CART/ruxolitinib treatment during initial CART expansion diminished T cell proliferation, blunted cytokine production, and/or facilitated leukemia relapse, which was abrogated by time-sequenced/delayed ruxolitinib co-exposure. Importantly, ruxolitinib co-administration prevented fatal TSLPRCART cytokine-associated toxicity in ALL PDX mice. Upon ruxolitinib withdrawal, TSLPRCART functionality recovered in vivo with clearance of subsequent ALL rechallenge. These translational studies demonstrate an effective two-pronged therapeutic strategy that mitigates acute CART-induced hyperinflammation and provides potential anti-leukemia ‘maintenance’ relapse prevention for CRLF2-rearranged Ph-like and DS-ALL.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"29 1","pages":""},"PeriodicalIF":11.4,"publicationDate":"2024-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142825133","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}

引用次数: 0

Mpox in people with haematological cancers 血液癌症患者中的 Mpox。

IF 12.8 1区医学

Leukemia Pub Date : 2024-12-13 DOI: 10.1038/s41375-024-02465-7

RP Gale, A. Hochhaus

引用次数: 0

Relationship between donor source, pre-transplant measurable residual disease, and outcome after allografting for adults with acute myeloid leukemia 成人急性髓性白血病供体来源、移植前可测量的残留疾病与同种异体移植后预后的关系

IF 12.8 1区医学

Leukemia Pub Date : 2024-12-12 DOI: 10.1038/s41375-024-02497-z

Corentin Orvain, Filippo Milano, Eduardo Rodríguez-Arbolí, Megan Othus, Effie W. Petersdorf, Brenda M. Sandmaier, Frederick R. Appelbaum, Roland B. Walter

{"title":"Relationship between donor source, pre-transplant measurable residual disease, and outcome after allografting for adults with acute myeloid leukemia","authors":"Corentin Orvain, Filippo Milano, Eduardo Rodríguez-Arbolí, Megan Othus, Effie W. Petersdorf, Brenda M. Sandmaier, Frederick R. Appelbaum, Roland B. Walter","doi":"10.1038/s41375-024-02497-z","DOIUrl":"10.1038/s41375-024-02497-z","url":null,"abstract":"Lack of HLA-matched related/unrelated donor remains a barrier to allogeneic hematopoietic cell transplantation (HCT) for adult acute myeloid leukemia (AML), with ongoing uncertainty about optimal donor type if more than one alternative donor is available. To assess the relationship between donor type, pre-HCT measurable residual disease (MRD), and post-HCT outcomes, we retrospectively analyzed 1265 myelodysplastic neoplasm (MDS)/AML and AML patients allografted in first or second remission with an HLA-matched sibling (MSD) or unrelated donor (MUD), HLA-mismatched unrelated donor (MMD), an HLA-haploidentical donor, or umbilical cord blood (UCB) at a single institution. Relapse risk was non-significantly higher after HLA-haploidentical and lower after UCB HCT. Non-relapse mortality (NRM) was significantly higher in patients undergoing MMD HCT, HLA-haploidentical HCT, and UCB, translating into significantly lower relapse-free survival (RFS) and overall survival for MMD and HLA-haploidentical HCT. There was a significant interaction between conditioning intensity and post-HCT outcomes for UCB HCT with better RFS for UCB HCT after MAC but higher NRM after non-MAC. In patients with pre-HCT MRD receiving MAC, relapse risk was significantly lower and RFS higher in those who underwent UCB HCT in comparison to MSD/MUD. Together, UCB HCT is a valuable alternative for MAC HCT, particularly in patients with pre-HCT MRD.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 2","pages":"381-390"},"PeriodicalIF":12.8,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41375-024-02497-z.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142815781","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}

引用次数: 0

Growth retardation and adult height in pediatric patients with chronic-phase chronic myeloid leukemia treated with tyrosine kinase inhibitors 酪氨酸激酶抑制剂治疗慢性粒细胞白血病儿童患者的生长迟缓和成人身高

IF 12.8 1区医学

Leukemia Pub Date : 2024-12-12 DOI: 10.1038/s41375-024-02488-0

Haruko Shima, Chikako Tono, Akihiko Tanizawa, Masaki Ito, Akihiro Watanabe, Yuki Yuza, Kazuko Hamamoto, Hideki Muramatsu, Masahiko Okada, Shoji Saito, Hiroaki Goto, Masaru Imamura, Akiko M. Saito, Souichi Adachi, Eiichi Ishii, Hiroyuki Shimada

引用次数: 0

Acute myeloid leukemia associated RUNX1 variants induce aberrant expression of transcription factor TCF4 急性髓系白血病相关RUNX1变异诱导转录因子TCF4的异常表达

IF 12.8 1区医学

Leukemia Pub Date : 2024-12-12 DOI: 10.1038/s41375-024-02470-w

Mylène Gerritsen, Florentien E. M. in ’t Hout, Ruth Knops, Bas L. R. Mandos, Melanie Decker, Tim Ripperger, Bert A. van der Reijden, Joost H. A. Martens, Joop H. Jansen

引用次数: 0

Prognostic relevance of treatment deviations in children with relapsed acute lymphoblastic leukemia who were treated in the ALL-REZ BFM 2002 study 在ALL-REZ BFM 2002研究中接受治疗的复发性急性淋巴细胞白血病儿童治疗偏差的预后相关性

IF 12.8 1区医学

Leukemia Pub Date : 2024-12-11 DOI: 10.1038/s41375-024-02474-6

Eleni A. Argyriadi, Ingo G. Steffen, Christiane Chen-Santel, Andrej Lissat, Andishe Attarbaschi, Jean-Pierre Bourquin, Guenter Henze, Arend von Stackelberg

{"title":"Prognostic relevance of treatment deviations in children with relapsed acute lymphoblastic leukemia who were treated in the ALL-REZ BFM 2002 study","authors":"Eleni A. Argyriadi, Ingo G. Steffen, Christiane Chen-Santel, Andrej Lissat, Andishe Attarbaschi, Jean-Pierre Bourquin, Guenter Henze, Arend von Stackelberg","doi":"10.1038/s41375-024-02474-6","DOIUrl":"10.1038/s41375-024-02474-6","url":null,"abstract":"Relapsed Acute Lymphoblastic Leukemia (ALL) is among the most common causes of cancer-associated deaths in children. However, little is known about the implications of deviations from ALL treatment protocols on survival rates. The present study elucidates the various characteristics of treatment deviations in children with relapsed ALL included in the ALL-REZ BFM 2002 (i.e., Relapse Berlin-Frankfurt- Münster) trial and determines their prognostic relevance for relapse and death rates. Among 687 patients, 100 were identified with treatment deviations, further classified, and examined by occurrence time, cause and type. Protocol deviation was considered a time-dependent variable and its impact on Disease Free Survival (DFS) and Overall Survival (OS) was examined using the time-dependent model Mantel Byar. Five years after the relapse diagnosis, deviations were significantly related to both inferior DFS (38%) and OS (57%) rates compared to protocol conformed treatment (DFS = 61%; OS = 70%, P < 0.001). Based on multivariate analyses, protocol deviation proved to be an independent adverse prognostic factor of DFS. Moreover, deviations triggered by chemotherapy-induced toxicity were associated with a higher relapse rate compared to deviations due to insufficient response. Therefore, to avoid impairment of results by deviations, future clinical trials, and treatment strategies should focus on less toxic treatments and stricter protocol compliance.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"39 2","pages":"337-345"},"PeriodicalIF":12.8,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41375-024-02474-6.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142809726","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}

引用次数: 0

Surrogate endpoints in mature B-cell neoplasms – meaningful or misleading? 成熟b细胞肿瘤的替代终点——有意义还是误导？

IF 12.8 1区医学

Leukemia Pub Date : 2024-12-10 DOI: 10.1038/s41375-024-02483-5

Florian Simon, Othman Al-Sawaf, John F. Seymour, Barbara Eichhorst

引用次数: 0

Incidence of Richter transformation of chronic lymphocytic leukemia/small lymphocytic lymphoma in the targeted therapy era 靶向治疗时代慢性淋巴细胞白血病/小淋巴细胞淋巴瘤Richter转化的发生率

IF 12.8 1区医学

Leukemia Pub Date : 2024-12-10 DOI: 10.1038/s41375-024-02492-4

Paul J. Hampel, Kari G. Rabe, Yucai Wang, Steven R. Hwang, Saad S. Kenderian, Eli Muchtar, Jose F. Leis, Amber B. Koehler, Mazie Tsang, Talal Hilal, Ricardo Parrondo, Rachel J. Bailen, Susan M. Schwager, Curtis A. Hanson, Esteban Braggio, Susan L. Slager, Min Shi, Cinthya J. Zepeda-Mendoza, Daniel L. Van Dyke, Tait D. Shanafelt, Rebecca L. King, Timothy G. Call, Neil E. Kay, Wei Ding, Sameer A. Parikh

引用次数: 0

Allogeneic hematopoietic stem cell transplantation in adult acute myeloid leukemia with t(16;21)(p11;q22)/FUS::ERG 同种异体造血干细胞移植治疗成人急性髓性白血病伴t(16;21)(p11;q22)/FUS::ERG

IF 12.8 1区医学

Leukemia Pub Date : 2024-12-10 DOI: 10.1038/s41375-024-02495-1

Satoshi Mitsuyuki, Yoshimitsu Shimomura, Hiroki Mizumaki, Masamitsu Yanada, Shohei Mizuno, Naoyuki Uchida, Noriko Doki, Ayumu Ito, Masatsugu Tanaka, Tetsuya Nishida, Yuta Katayama, Satoshi Yoshihara, Tetsuya Eto, Satoru Takada, Shuichi Ota, Masako Toyosaki, Yuta Hasegawa, Hirohisa Nakamae, Koji Kawamura, Makoto Onizuka, Takahiro Fukuda, Marie Ohbiki, Yoshiko Atsuta, Takaaki Konuma

引用次数: 0