American Journal of Hematology最新文献

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Budesonide, Added to PTCy-Based Regimen, for Prevention of Acute GI GVHD After Allogeneic Stem Cell Transplantation
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-01-08 DOI: 10.1002/ajh.27581
Piyanuch Kongtim, Piyatida Chumnumsiriwath, Pongthep Vittayawacharin, Deepa Jeyakumar, Benjamin J. Lee, Jean Doh, Shawn P. Griffin, Richard A. Van Etten, Stefan Ciurea
{"title":"Budesonide, Added to PTCy-Based Regimen, for Prevention of Acute GI GVHD After Allogeneic Stem Cell Transplantation","authors":"Piyanuch Kongtim, Piyatida Chumnumsiriwath, Pongthep Vittayawacharin, Deepa Jeyakumar, Benjamin J. Lee, Jean Doh, Shawn P. Griffin, Richard A. Van Etten, Stefan Ciurea","doi":"10.1002/ajh.27581","DOIUrl":"https://doi.org/10.1002/ajh.27581","url":null,"abstract":"Oral budesonide exerts local effects with negligible systemic glucocorticoid activity, due to rapid first-pass metabolism, therefore, could potentially be efficacious in preventing gastrointestinal (GI) acute GVHD (aGVHD). We explored the use of budesonide, added to posttransplant cyclophosphamide (PTCy), tacrolimus, and mycophenolate mofetil, for prevention of GI aGVHD after allogeneic hematopoietic stem cell transplantation (AHSCT) in a prospective observational study and treated 80 patients with a median age of 53 years (range 19–74). Results were compared with a publicly available CIBMTR dataset of 646 patients who received PTCy-based GVHD prophylaxis (CIBMTR Study # GV17-02) (control). Cumulative incidence (CI) of 3-month grade 2–4 and grade 3–4 aGVHD in the budesonide and control groups were 3.8% vs. 34.4% (<i>p</i> &lt; 0.001) and 1.3% vs. 9.8% (<i>p</i> = 0.029), respectively. One-year GRFS (70.5% vs. 31.5%, <i>p</i> &lt; 0.001), PFS (73.4% vs. 52.8%, <i>p</i> = 0.003), and OS (80.1% vs. 64.2%, <i>p</i> = 0.038) were significantly higher in the budesonide group compared with control group. Propensity score-adjusted analyses showed that the addition of budesonide significantly decreased risk of aGVHD grade 2–4 (HR 0.29, <i>p</i> &lt; 0.001), grade 3–4 (HR 0.12, <i>p</i> = 0.045), and cGVHD (HR 0.22, <i>p</i> &lt; 0.001), which resulted in better GRFS (HR 0.38, <i>p</i> &lt; 0.001), PFS (HR 0.58, <i>p</i> = 0.012), and OS (HR 0.72, <i>p</i> = 0.044). Similar results were found when using propensity score-matched analysis restricted to recipients of haploidentical transplantation. In conclusion, addition of budesonide to PTCy-based GVHD prophylaxis is safe and effective in preventing severe acute GI GVHD with significantly improved GRFS. These results could facilitate transition to peripheral blood grafts for all allogeneic transplant recipients.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"13 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142935807","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
Breaking the Bone Marrow Barrier: Peripheral Blood as a Gateway to Measurable Residual Disease Detection in Acute Myelogenous Leukemia
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-01-07 DOI: 10.1002/ajh.27586
John T. Butler, William M. Yashar, Ronan Swords
{"title":"Breaking the Bone Marrow Barrier: Peripheral Blood as a Gateway to Measurable Residual Disease Detection in Acute Myelogenous Leukemia","authors":"John T. Butler, William M. Yashar, Ronan Swords","doi":"10.1002/ajh.27586","DOIUrl":"https://doi.org/10.1002/ajh.27586","url":null,"abstract":"Acute myeloid leukemia (AML) is a genetically heterogeneous disease with high rates of relapse after initial treatment. Identifying measurable residual disease (MRD) following initial therapy is essential to assess response, predict patient outcomes, and identify those in need of additional intervention. Currently, MRD analysis relies on invasive, serial bone marrow (BM) biopsies, which complicate sample availability and processing time and negatively impact patient experience. Additionally, finding a positive result can generate more questions than answers, causing anxiety for both the patient and the provider. Peripheral blood (PB) evaluation has shown promise in detecting MRD and is now recommended by the European Leukemia Net for AML for certain genetic abnormalities. PB-based sampling allows for more frequent testing intervals and better temporal resolution of malignant expansion while sparing patients additional invasive procedures. In this review, we will discuss the current state of PB testing for MRD evaluation with a focus on next-generation sequencing methodologies that are capable of MRD detection across AML subtypes.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"20 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142935108","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
Limited Benefit of Routine Clinical Follow-Up for Relapse Detection in Diffuse Large B-Cell Lymphoma Patients in Complete Remission Following First-Line Treatment
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-01-05 DOI: 10.1002/ajh.27577
Therese Lassen, Torsten H. Nielsen, Annika von Heymann, Lene K. Nielsen, Morten K. Larsen, Anne O. Gang, Christoffer Johansen, Lars M. Pedersen
{"title":"Limited Benefit of Routine Clinical Follow-Up for Relapse Detection in Diffuse Large B-Cell Lymphoma Patients in Complete Remission Following First-Line Treatment","authors":"Therese Lassen, Torsten H. Nielsen, Annika von Heymann, Lene K. Nielsen, Morten K. Larsen, Anne O. Gang, Christoffer Johansen, Lars M. Pedersen","doi":"10.1002/ajh.27577","DOIUrl":"https://doi.org/10.1002/ajh.27577","url":null,"abstract":"Despite advances in treatment, approximately 15% of patients with diffuse large B-cell lymphoma (DLBCL) who achieve complete remission (CR) after first-line therapy will experience a relapse. However, there is no consensus on the optimal follow-up strategies for detecting relapse after achieving CR. This population-based study, based on the Danish Lymphoma Registry (LYFO), identified a total of 1634 patients diagnosed with DLBCL between 2010 and 2017, including 105 patients who achieved CR following first-line R-CHOP-like therapy and subsequently relapsed. The median follow-up time was 6 years (range 3–8 years). Most cases of relapse were symptomatic (83%), with B symptoms and peripheral lymphadenopathy being the most common. Asymptomatic relapses were identified through physical examination (1%), blood tests (3%), or imaging findings (13%). The proportion of relapses identified outside routine visits was 70%. Only 5% of scheduled routine visits led to a relapse diagnosis, whereas 74% of unscheduled visits initiated by the patient outside routine follow-up resulted in relapse detection. Our findings highlight that systematic, scheduled monitoring of patients in remission after first-line treatment contributes only modestly to the early detection of disease recurrence. Future studies should explore alternative methods of relapse surveillance rather than relying solely on pre-scheduled clinical follow-up.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"73 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142929478","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
Hyper-CVAD and Sequential Blinatumomab Without and With Inotuzumab in Young Adults With Newly Diagnosed Philadelphia Chromosome-Negative B-Cell Acute Lymphoblastic Leukemia
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-01-05 DOI: 10.1002/ajh.27576
Hagop Kantarjian, Nicholas J. Short, Nitin Jain, Fadi G. Haddad, Tapan Kadia, Musa Yilmaz, Alessandra Ferrajoli, Koji Sasaki, Yesid Alvarado, Naveen Pemmaraju, Jayastu Senapati, Rebecca Garris, Farhad Ravandi, Elias Jabbour
{"title":"Hyper-CVAD and Sequential Blinatumomab Without and With Inotuzumab in Young Adults With Newly Diagnosed Philadelphia Chromosome-Negative B-Cell Acute Lymphoblastic Leukemia","authors":"Hagop Kantarjian, Nicholas J. Short, Nitin Jain, Fadi G. Haddad, Tapan Kadia, Musa Yilmaz, Alessandra Ferrajoli, Koji Sasaki, Yesid Alvarado, Naveen Pemmaraju, Jayastu Senapati, Rebecca Garris, Farhad Ravandi, Elias Jabbour","doi":"10.1002/ajh.27576","DOIUrl":"https://doi.org/10.1002/ajh.27576","url":null,"abstract":"Adding inotuzumab ozogamicin (InO) to hyper-CVAD and blinatumomab may improve outcomes in newly diagnosed Philadelphia chromosome (Ph)-negative B-cell acute lymphoblastic leukemia (B-ALL). Patients with newly diagnosed B-ALL received up to four cycles of hyper-CVAD followed by four cycles of blinatumomab. Beginning with patient #39, InO 0.3 mg/m<sup>2</sup> was added on Days 1 and 8 to two cycles of high-dose methotrexate and cytarabine, and two cycles of blinatumomab. The primary endpoint was the relapse-free survival (RFS) rate. Seventy-five patients were treated (median age of 33 years; range, 18–59), of whom 37 (49%) received hyper-CVAD with blinatumomab and InO (cohort 2). Measurable residual disease (MRD) negativity by next-generation sequencing (sensitivity: 1 × 10<sup>−6</sup>) was achieved in 79% of patients in cohort 2. The median follow-up was 44 months (range, 13–90) overall, and 26 months (range, 8–39) in cohort 2. For the entire cohort, the estimated 3-year RFS rate was 82% and the 3-year overall survival rate was 90%. These rates were 90% versus 74% (<i>p</i> = 0.06) and 100% versus 82% (<i>p</i> = 0.01) in patients who did or did not receive InO, respectively. No sinusoidal obstruction syndrome was observed. In summary, hyper-CVAD with blinatumomab and InO improved the outcomes of patients with newly diagnosed B-ALL.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"75 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142929324","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
The Interplay Between Ophthalmic and Systemic Outcomes in Patients With Sickle Cell Disease and Concurrent Retinopathy—A Population‐Based Study
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-01-04 DOI: 10.1002/ajh.27552
Purna Nangia, Karen M. Wai, Adrienne W. Scott, Ehsan Rahimy, Prithvi Mruthyunjaya
{"title":"The Interplay Between Ophthalmic and Systemic Outcomes in Patients With Sickle Cell Disease and Concurrent Retinopathy—A Population‐Based Study","authors":"Purna Nangia, Karen M. Wai, Adrienne W. Scott, Ehsan Rahimy, Prithvi Mruthyunjaya","doi":"10.1002/ajh.27552","DOIUrl":"https://doi.org/10.1002/ajh.27552","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"20 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-01-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142925093","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
Clinical Characteristics and Prognosis of Pediatric Idiopathic Multicentric Castleman Disease
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-01-03 DOI: 10.1002/ajh.27574
Yu-han Gao, Jia-feng Yao, Si-yuan Li, Yue Dang, Hao-yi Xu, Tong Zou, Jian Li, Lu Zhang, Rui Zhang
{"title":"Clinical Characteristics and Prognosis of Pediatric Idiopathic Multicentric Castleman Disease","authors":"Yu-han Gao, Jia-feng Yao, Si-yuan Li, Yue Dang, Hao-yi Xu, Tong Zou, Jian Li, Lu Zhang, Rui Zhang","doi":"10.1002/ajh.27574","DOIUrl":"https://doi.org/10.1002/ajh.27574","url":null,"abstract":"&lt;p&gt;Idiopathic multicentric Castleman disease (iMCD) is a rare lymphoproliferative disorder characterized by marked heterogeneity among patients, with severity ranging from mild to life-threatening [&lt;span&gt;1&lt;/span&gt;]. Although cases have been documented across all age groups, the diagnosis of iMCD typically occurs in the fifth decade of life and is relatively uncommon in children [&lt;span&gt;1&lt;/span&gt;].&lt;/p&gt;\u0000&lt;p&gt;A previous analysis of the ACCELERATE dataset revealed that 95% of patients with iMCD diagnosed before the age of 30 (&lt;i&gt;n&lt;/i&gt; = 35) had severe disease at onset [&lt;span&gt;2&lt;/span&gt;]. A subsequent subgroup analysis focusing on the pediatric cohort (&lt;i&gt;n&lt;/i&gt; = 19) highlighted a higher prevalence of the thrombocytopenia, anasarca, fever, renal dysfunction, and organomegaly (TAFRO) subtype [&lt;span&gt;3&lt;/span&gt;]. These findings raise important questions about whether the severity and dominance of the TAFRO subtype in children suggest a poorer prognosis compared to that of adults. However, existing studies lack comprehensive data on pediatric iMCD. In this study, we aim to characterize the clinical features, treatment strategies, and outcomes of individuals diagnosed with iMCD at or under the age of 18, thereby contributing to a comprehensive understanding of the disease in this patient group.&lt;/p&gt;\u0000&lt;p&gt;This retrospective cohort study was conducted at two tertiary hospitals: Peking Union Medical College Hospital and Beijing Children's Hospital. We enrolled patients aged ≤ 18 years who were diagnosed with iMCD based on the Castleman Disease Collaborative Network (CDCN) diagnostic consensus [&lt;span&gt;4&lt;/span&gt;] between January 2012 and January 2024. Patients were further classified into three clinical phenotypes: TAFRO, not otherwise specified (NOS), and idiopathic plasmacytic lymphadenopathy (IPL) [&lt;span&gt;5&lt;/span&gt;]. Severe iMCD was evaluated according to the CDCN criteria at diagnosis [&lt;span&gt;6&lt;/span&gt;].&lt;/p&gt;\u0000&lt;p&gt;The primary outcome was to describe the baseline characteristics. Other study outcomes included treatment regimens and responses, as well as prognosis. Treatments were further categorized into two main strategies: continual suppressive therapies, encompassing interleukin (IL)-6 targeting regimens, myeloma-like regimens, and mTOR-targeting regimens; and pulse therapy strategies, which include lymphoma-like regimens. The evaluation of treatment response adhered to the CDCN response criteria. Follow-up data, extending up to September 1st, 2024, were sourced from medical record systems and through telephone communication. Overall survival and time-to-next treatment (TTNT) were determined. Additional details on study methods are available in the Data S1 section.&lt;/p&gt;\u0000&lt;p&gt;A total of 23 patients (16 males and seven females [ratio, 2.3:1]), with a median age of 12 years at diagnosis (range: 2–18 years), were included in this analysis (Table S1). While there was no statistically significant difference in the age distribution between sexes, all children diagnosed at or before the","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"13 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142917419","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
Treatment of Relapsed/Refractory CLL Patients With PI3Kδ Inhibitor and Anti-CD20 Antibody Rapidly Decreases Tumor Burden but Could Induce Resistance
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-01-02 DOI: 10.1002/ajh.27569
Jennifer E. Bruno, Christine A. Herne, Andrea M. Baran, Karl R. VanDerMeid, Paul M. Barr, Alyssa R. Williams, Sally A. Quataert, Tim R. Mosmann, Clive S. Zent, Charles C. Chu
{"title":"Treatment of Relapsed/Refractory CLL Patients With PI3Kδ Inhibitor and Anti-CD20 Antibody Rapidly Decreases Tumor Burden but Could Induce Resistance","authors":"Jennifer E. Bruno, Christine A. Herne, Andrea M. Baran, Karl R. VanDerMeid, Paul M. Barr, Alyssa R. Williams, Sally A. Quataert, Tim R. Mosmann, Clive S. Zent, Charles C. Chu","doi":"10.1002/ajh.27569","DOIUrl":"https://doi.org/10.1002/ajh.27569","url":null,"abstract":"&lt;p&gt;Therapeutic unconjugated anti-CD20 monoclonal antibodies (mAb) and small molecule inhibitors of the B cell receptor (BCR) signaling pathway and B cell lymphoma-2 (BCL2) have greatly improved therapy for patients with progressive chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL). However, these therapies are not curative, and their efficacy can be compromised by side effects and acquired drug resistance, especially with indefinite duration regimens [&lt;span&gt;1&lt;/span&gt;]. To address these problems, we tested a limited–duration triple combination targeted therapy for relapsed/refractory CLL patients (U2-VEN, ClinicalTrials.gov NCT03379051) that involved three drugs with different mechanisms of action, which may increase effectiveness and reduce the likelihood of resistance (Figure 1A [&lt;span&gt;2&lt;/span&gt;]). We chose: (1) ublituximab (UBL), a glycoengineered chimeric monoclonal antibody (mAb) targeting a unique epitope on CD20 antigen (Ag) on surface of CLL cells, which destroys CLL cells primarily by antibody-dependent cellular phagocytosis (ADCP); (2) umbralisib (UMB), a novel small molecule B cell receptor (BCR) signaling inhibitor that is highly selective for phosphoinositide 3-kinase delta (PI3Kδ, shown) and casein kinase 1 epsilon, which causes CLL cell death by preventing cell survival signals; and (3) venetoclax (VEN), a small molecule anti-apoptosis inhibitor that blocks cell survival promoted by BCL2, which stops mitochondria from initiating apoptosis. An initial 12 week treatment with UBL and UMB was utilized to allow the BCR signal inhibiting drug to mobilize CLL cells into the circulation where they are more susceptible to mAb cytotoxicity and thus decrease the CLL tumor burden and risk of VEN-induced tumor lysis syndrome when it is added in the 13th week of therapy (Figure 1B).&lt;/p&gt;\u0000&lt;figure&gt;&lt;picture&gt;\u0000&lt;source media=\"(min-width: 1650px)\" srcset=\"/cms/asset/42baaa16-06ed-4c73-a745-641b06f16907/ajh27569-fig-0001-m.jpg\"/&gt;&lt;img alt=\"Details are in the caption following the image\" data-lg-src=\"/cms/asset/42baaa16-06ed-4c73-a745-641b06f16907/ajh27569-fig-0001-m.jpg\" loading=\"lazy\" src=\"/cms/asset/75c81c34-f5b8-452b-9e04-cb8948d70b52/ajh27569-fig-0001-m.png\" title=\"Details are in the caption following the image\"/&gt;&lt;/picture&gt;&lt;figcaption&gt;\u0000&lt;div&gt;&lt;strong&gt;FIGURE 1&lt;span style=\"font-weight:normal\"&gt;&lt;/span&gt;&lt;/strong&gt;&lt;div&gt;Open in figure viewer&lt;i aria-hidden=\"true\"&gt;&lt;/i&gt;&lt;span&gt;PowerPoint&lt;/span&gt;&lt;/div&gt;\u0000&lt;/div&gt;\u0000&lt;div&gt;UBL and UMB treatment in U2-VEN clinical trial results in rapid loss of CLL cells and CD20 levels. (A) Multi-drug combination targeting three different molecules to overcome single-agent resistance in relapsed/refractory CLL patients. (B) Diagram of U2-VEN clinical study design (ClinicalTrials.gov NCT03379051) [&lt;span&gt;2&lt;/span&gt;]. Treatment consists of initially 12 cycles of 28 days. Ublituximab (purple) was administered intravenously on indicated days of first six cycles. Umbralisib (green) was orally administered daily for 12 cycles. Venetoclax was ad","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"340 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142911722","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
Outcome of Splenectomy in JAK2 Inhibitor Treated Patients With Myelofibrosis: A Mayo Clinic Experience in 34 Consecutive Cases
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2024-12-26 DOI: 10.1002/ajh.27570
Patricia Carey, Animesh Pardanani, Patrick Starlinger, Ayalew Tefferi, Naseema Gangat
{"title":"Outcome of Splenectomy in JAK2 Inhibitor Treated Patients With Myelofibrosis: A Mayo Clinic Experience in 34 Consecutive Cases","authors":"Patricia Carey, Animesh Pardanani, Patrick Starlinger, Ayalew Tefferi, Naseema Gangat","doi":"10.1002/ajh.27570","DOIUrl":"https://doi.org/10.1002/ajh.27570","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"32 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142886952","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
Outcomes of Patients With Treated Secondary Acute Myeloid Leukemia: A High-Risk Subtype That Warrants an Independent Prognostic Designation
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2024-12-24 DOI: 10.1002/ajh.27561
Jayastu Senapati, Hagop M. Kantarjian, Fadi G. Haddad, Nicholas J. Short, Gautam Borthakur, Rashmi Kanagal-Shamanna, Guilin Tang, Elias Jabbour, Courtney D. DiNardo, Naval Daver, Guillermo Montalban-Bravo, Vishrut Shah, Amin Alousi, Elizabeth Shpall, Uday Popat, Guillermo Garcia-Manero, Farhad Ravandi, Tapan M. Kadia
{"title":"Outcomes of Patients With Treated Secondary Acute Myeloid Leukemia: A High-Risk Subtype That Warrants an Independent Prognostic Designation","authors":"Jayastu Senapati, Hagop M. Kantarjian, Fadi G. Haddad, Nicholas J. Short, Gautam Borthakur, Rashmi Kanagal-Shamanna, Guilin Tang, Elias Jabbour, Courtney D. DiNardo, Naval Daver, Guillermo Montalban-Bravo, Vishrut Shah, Amin Alousi, Elizabeth Shpall, Uday Popat, Guillermo Garcia-Manero, Farhad Ravandi, Tapan M. Kadia","doi":"10.1002/ajh.27561","DOIUrl":"https://doi.org/10.1002/ajh.27561","url":null,"abstract":"Patients who develop acute myeloid leukemia (AML) after having received treatment for myelodysplastic syndrome (MDS) or related conditions have particularly poor outcomes. This study analyzed adult patients with newly diagnosed AML who previously had MDS, chronic myelomonocytic leukemia (CMML), or MDS/myeloproliferative neoplasm (MPN) overlap syndrome, and who had received hypomethylating agents, chemotherapy, and/or allogeneic stem cell transplantation (HSCT) for these antecedent disorders. From January 2012 to August 2023, we included 673 patients with a median age of 70 years (range, 19–94); 536 (80%) had transformed from MDS, and the remainder from CMML or MDS-MPN. Additionally, 149 patients (22%) had prior therapy for nonmyeloid malignancies. Among 497 evaluable patients, 289 (58%) had adverse-risk (AR) cytogenetics, 34% had <i>TP53</i> mutation/s, and 71% were classified as AR by the ELN 2017 criteria. Most patients (67%) received low-intensity therapy (LIT) for AML, and 27% were treated with venetoclax. The overall response rate was 37%, and venetoclax improved the odds of response (OR = 2.5, 95% CI 1.6–3.7) in LIT–treated patients. At a median follow-up of 43 months, the median relapse-free survival (RFS) and overall survival (OS) were 4.6 and 4.8 months, respectively. Multivariate analysis showed that prior therapy for nonmyeloid disorders (HR = 1.30), ≥ 2 lines of therapy for antecedent myeloid disorders (HR = 1.23), and ELN AR risk (HR = 1.47) increased the hazards of death, while HSCT (HR = 0.50) was beneficial and validated on gradient-boosted regression. TS-AML is associated with poor outcomes irrespective of AML genomics and treatment, highlighting the need for its inclusion as an independent AR category for accurate prognostication and clinical trial reporting.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"13 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142880235","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
Unlocking Sanctuary Sites: The Efficacy of Brexucabtagene Autoleucel in Ocular B-ALL Relapse
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2024-12-24 DOI: 10.1002/ajh.27572
Ludovica Calabretta, Patrizia Zappasodi, Carlo Di Biase, Cristina Picone, Elisa Roncoroni, Marianna Rossi, Antonio Bianchessi, Irene Defrancesco, Caterina Zerbi, Beatrice Ferrari, Erica Consensi, Gianluca Martini, Claudia Patricia Tobar Cabrera, Claudia Battista, Alessandro Mazzacane, Francesco Romano, Giulia Losi, Alessia Taurino, Luca Arcaini, Nicola Polverelli
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