Acta Haematologica最新文献

{"title":"Treatment Decision-making for Individuals with Relapsed/Refractory Chronic Lymphocytic Leukemia (CLL) in the United States.","authors":"Catherine C Coombs, Thomas W LeBlanc, Katherine B Winfree, Catherine E Muehlenbein, Urvi Desai, Noam Y Kirson, Ashley Holub, Alice Qu, Ellen Sears, Brian Koffman, Meghan C Thompson","doi":"10.1159/000552243","DOIUrl":"https://doi.org/10.1159/000552243","url":null,"abstract":"<p><strong>Background: </strong>As available treatment options for relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) increase, treatment decisions become increasingly complex. This cross-sectional study evaluated patient and physician decision-making when selecting treatment for R/R CLL in the United States.</p><p><strong>Methods: </strong>After an initial pilot, 100 individuals with R/R CLL and 100 physicians who treat R/R CLL completed web-based surveys on factors that influenced treatment selection and levels of involvement during treatment selection. Categorical measures were summarized using numbers/ proportions and continuous measures were summarized using means/standard deviations.</p><p><strong>Results: </strong>Individuals with R/R CLL and physicians prioritized treatment efficacy (individuals: N=47 of 78, 60.3%; physicians: N=79 of 100, 79.0%) and potential side effects (individuals: N=43 of 78, 55.1%; physicians: N=50 of 79, 63.3%) when selecting treatment. Differences in the relative importance of specific factors were observed. Financial and cost factors were important for individuals while medical history was important for physicians. Both cohorts reported a shared decision-making process.</p><p><strong>Conclusions: </strong>Building upon previous findings in the front-line CLL setting, this study confirms that individuals with R/R CLL and physicians commonly consider efficacy and safety of different treatment options when making their decision. Relative importance of specific factors within these and other categories differs across the samples.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-21"},"PeriodicalIF":1.1,"publicationDate":"2026-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147832214","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":"Diagnostic Pathways and Procedural Risk in Primary Central Nervous System Lymphoma: A Nationwide Analysis.","authors":"Kashif Qureshi, Sujai Arakali, Jose Anzueto, Dillon Huddleston, Joshua Hobbs, Sebastian Saenz, Rhea Thakkar, Rahul H Jayaram, Carlos Goulart, Khaled Alok, Mihir Gupta","doi":"10.1159/000552333","DOIUrl":"https://doi.org/10.1159/000552333","url":null,"abstract":"<p><strong>Introduction: </strong>Primary central nervous system lymphoma (PCNSL) is an aggressive malignancy in which timely diagnosis is critical. While neurosurgical biopsy remains the gold standard, cerebrospinal fluid (CSF)-based diagnostics are frequently deployed. Owing to the rarity of this condition, little is known about clinical characteristics, test utilization, social determinants of health, and complication rates in the context of securing a PCNSL diagnosis. We evaluated approaches for diagnosing PCNSL, post-procedural complications, and clinical and socioeconomic associations with adverse events.</p><p><strong>Methods: </strong>We queried the PearlDiver Mariner claims database to identify adults with newly diagnosed PCNSL between 2011 and 2023. CPT and ICD codes were used to identify lumbar puncture (LP) and/or neurosurgical biopsy performed before diagnosis. Patients were stratified by diagnostic pathway: LP-only, Biopsy-only, or both LP and biopsy. We used logistic regression to identify predictors of adverse events. Results Among 2,549 total patients, 1957 (76.8%), 398 (15.6%), and 194 (7.6%) comprised the LP-only, Biopsy-only, or both LP and biopsy pathways, respectively. LP-only patients were younger, more frequently male, Medicaid-insured, and economically disadvantaged (P< 0.001 for each). Rates of post-procedural thromboembolic complications were 5.4%, 10.1%, and 6.7% for LP-only, Biopsy-only, and both LP and biopsy, respectively (P<0.002). Biopsy-only was associated with increased odds of procedural complications (OR 3.21, 95% CI 1.79-5.69, P<0.001), thromboembolic events (OR 1.85, 95% CI 1.39-2.45, P<0.001), and medical complications (OR 1.87, 95% CI 1.49-2.33, P<0.001) compared to LP alone.</p><p><strong>Conclusions: </strong>The choice of diagnostic modalities utilized to secure a diagnosis of PCNSL may be influenced by patient-level clinical and socioeconomic factors. PCNSL patients may represent a uniquely high-risk subgroup for neurosurgical biopsy, emphasizing the importance of advancing CSF-based diagnostics.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-11"},"PeriodicalIF":1.1,"publicationDate":"2026-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147832229","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":"Prognostic factors and survival outcomes in ovarian lymphoma: a population-based analysis.","authors":"Pierre Loap, Youlia Kirova","doi":"10.1159/000552294","DOIUrl":"https://doi.org/10.1159/000552294","url":null,"abstract":"<p><strong>Background: </strong>Primary ovarian lymphoma represents a rare clinical entity accounting for 0.5% of non-Hodgkin lymphomas and 1.5% of ovarian neoplasms. Current literature is limited to small case series, with poorly defined prognostic factors and optimal treatment strategies.</p><p><strong>Methods: </strong>This retrospective cohort study utilized the SEER database (2000-2020) to identify patients with ovarian lymphoma. Patients were categorized as localized primary ovarian lymphoma (L-POL, stage I) or lymphoma involving the ovary (LIO, stages II-IV or unknown). Cox proportional hazards regression identified independent prognostic factors for overall survival (OS) and cancer-specific survival (CSS).</p><p><strong>Results: </strong>A total of 221 patients were identified, representing the largest ovarian lymphoma cohort ever reported. The cohort included 75 L-POL (33.9%) and 146 LIO (66.1%) patients. Median age was 49 years. Diffuse large B-cell lymphoma was the most common histology (62.0%). L-POL patients demonstrated higher proportions of Black patients (13.3% vs 4.1%, p=0.022), follicular lymphoma (22.7% vs 12.3%), and unilateral disease (89.3% vs 61.6%). At median follow-up of 80 months, 5-year and 10-year CSS rates were 77.7% and 75.3% overall, and 87.4% and 85.5% for L-POL patients. In multivariate analysis, age (HR 1.035, p<0.001), L-POL (grade I) classification (HR 0.435, p=0.023), and high-grade histology (HR 4.898, p=0.009) were independent prognostic factors for CSS. Surgery, radiotherapy, and chemotherapy showed no survival benefit in multivariate analysis. Surgery was performed in 86.9% of patients, with 9.9% undergoing inappropriate debulking procedures.</p><p><strong>Conclusions: </strong>L-POL represents a distinct clinical presentation with excellent long-term prognosis. Age, L-POL classification, and histological grade are independent prognostic factors. The high rate of inappropriate surgical procedures, ranging from hysterectomy and debulking to pelvic exenteration, underscores the critical need for improved preoperative recognition. Optimal management should emphasize limited surgical intervention for diagnosis and fertility preservation, followed by systemic chemotherapy tailored to lymphoma subtype and stage.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-15"},"PeriodicalIF":1.1,"publicationDate":"2026-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147809127","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":"Persistent Splenomegaly is associated with Morbidity in Tanzanian Children with Sickle Cell Anemia: Secondary Analysis of the SPHERE Trial.","authors":"Emmanuela E Ambrose, George Tomlinson, Patrick S Ngoya, Susan E Stuber, Teresa S Latham, Russell E Ware, Abel N Makubi, Luke R Smart","doi":"10.1159/000552026","DOIUrl":"10.1159/000552026","url":null,"abstract":"<p><strong>Introduction: </strong>African children with sickle cell anemia (SCA) often have splenomegaly with recurrent cytopenias that complicate management, rather than the splenic atrophy typically observed in SCA.</p><p><strong>Methods: </strong>We analyzed the presence, persistence, and impact of splenomegaly in the Stroke Prevention with Hydroxyurea Enabled through Research and Education (SPHERE) trial (NCT03948867), an open-label trial of dose-escalated hydroxyurea to prevent stroke in Tanzanian children with SCA. Palpable splenomegaly was recorded at each visit. Annual ultrasound measurements were compared to age- and height-related references. Longitudinally, palpable splenomegaly was categorized as infrequent (present in <5% visits), intermittent (10-50% visits), or persistent (>50% visits). Adverse events (AE) and hydroxyurea dose-limiting toxicities (DLT) were compared using incidence rate ratios (IRR) among the three categories.</p><p><strong>Results: </strong>At enrollment, splenomegaly was identified in 48/196 (25%) by palpation and 78 (40%) by ultrasound. Across 221 patient-years of treatment in 53 children, splenomegaly was intermittent in 19% and persistent in 21%. At 12 months, children with persistent splenomegaly were compared to those with infrequent splenomegaly and had achieved similar hydroxyurea dose (23.1 vs 27.6 mg/kg/day, p=0.136), hemoglobin (9.0 vs 9.1g/dL, p=0.877) and HbF (23.8 vs 24.3%, p=0.481), but experienced more vaso-occlusive AE (IRR=3.8, p-value 0.122), transfusions (IRR=7.1, p-value 0.014), and DLTs (IRR=3.2, p-value 0.073).</p><p><strong>Conclusion: </strong>Splenomegaly is common in Tanzanian children with SCA, often persistent, worsens clinical outcomes and complicates hydroxyurea. Its cause remains unclear and further investigation is urgently needed to clarify its etiology, improve its management, and ensure optimal hydroxyurea treatment.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-18"},"PeriodicalIF":1.1,"publicationDate":"2026-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147759518","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":"Acta Haematologica in the Era of Artificial Intelligence.","authors":"Pia Raanani","doi":"10.1159/000552225","DOIUrl":"https://doi.org/10.1159/000552225","url":null,"abstract":"","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-6"},"PeriodicalIF":1.1,"publicationDate":"2026-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147759491","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":"Venetoclax-Based Regimens in Chronic Myelomonocytic Leukemia: A Systematic Review and Meta-Analysis.","authors":"Mohammed Abdulgayoom, Mohammad S Afana, Leen Haj Saleh, Aadhila Abbas Manthiri, Noor Adel Aweer, Mohammad Bakheet, Abdulrahman F Al-Mashdali, Shehab F Mohamed","doi":"10.1159/000551885","DOIUrl":"https://doi.org/10.1159/000551885","url":null,"abstract":"<p><p>Chronic myelomonocytic leukemia (CMML) is a biologically heterogeneous myelodysplastic/myeloproliferative neoplasm with limited disease-modifying options beyond hypomethylating agents (HMAs) and allogeneic hematopoietic stem cell transplantation (allo-HSCT). Venetoclax (VEN), a selective BCL-2 inhibitor, is increasingly used off-label in CMML, yet CMML-specific efficacy and safety remain incompletely defined. We conducted a systematic review and meta-analysis in accordance with PRISMA 2020, including adult CMML cohorts receiving VEN-based therapy with extractable CMML-specific outcomes. Seventeen publications representing nine unique studies were included, comprising 145 VEN-treated CMML patients. Most regimens combined VEN with azacitidine, decitabine, or oral decitabine-cedazuridine, using heterogeneous dosing schedules with CYP3A-guided dose modifications. Responses occurred early, typically within one to two treatment cycles, but durability was modest. Random-effects meta-analysis yielded pooled complete remission (CR) rates of 19.1% (95% CI, 9.4-34.9; I²=55%), marrow CR (mCR) rates of 36.4% (95% CI, 24.7-50.0; I²=21%), and an overall response rate (ORR) of 71.9% (95% CI, 56.5-83.4; I²=56%). VEN-based therapy was associated with substantial myelosuppression, including frequent grade ≥3 neutropenia and thrombocytopenia, with clinically relevant infectious complications; early mortality was low. In summary, VEN-based regimens demonstrate measurable but limited activity in CMML, with high ORR, but low CR rates. Prospective CMML-specific trials are needed to define optimal use and patient selection.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-25"},"PeriodicalIF":1.1,"publicationDate":"2026-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147632176","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":"A Case Report of Tyrosine Kinase Inhibitor Interruption in a Patient with Multilineage Philadelphia Positive Acute Lymphoblastic Leukemia.","authors":"Hannah Goulart, Julie Braish, Nicholas J Short, Nitin Jain, Koji Sasaki, Hagop Kantarjian, Elias J Jabbour","doi":"10.1159/000551699","DOIUrl":"https://doi.org/10.1159/000551699","url":null,"abstract":"<p><strong>Introduction: </strong>Measurable residual disease (MRD) assessment in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) commonly relies on RT-PCR detection of BCR::ABL1 transcripts. However, discordance between BCR::ABL1 PCR and next-generation sequencing (NGS)-based MRD assays targeting immunoglobulin/T-cell receptor (IG/TR) rearrangements may occur, particularly in cases of multilineage Ph+ ALL, complicating treatment decisions such as tyrosine kinase inhibitor (TKI) discontinuation.</p><p><strong>Case presentation: </strong>We report a 54-year-old female with Ph+ ALL who achieved durable remission with sustained NGS MRD-negativity by IG/TR despite persistent low-level BCR::ABL1 transcript positivity. Following self-discontinuation of ponatinib, she experienced a rapid rise in BCR::ABL1 transcripts and loss of cytogenetic remission in myeloid cells, while remaining morphologically in remission and NGS MRD-negative. Reintroduction of ponatinib resulted in prompt transcript decline.</p><p><strong>Conclusion: </strong>This case highlights the clinical significance of multilineage Ph+ ALL and its distinct biological background, thereby underscoring caution with TKI discontinuation despite deep NGS MRD-negativity, and may support continued TKI therapy in this setting.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-8"},"PeriodicalIF":1.1,"publicationDate":"2026-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147589256","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":"A Complex Case of Anti-e Mimicking Autoantibody Development Leading to Warm Autoimmune Hemolytic Anemia: Case Report.","authors":"Ilsa Hale, Kristina L Kunes, Jean Sabile, Jacob Dougherty, Trisha Wong, Michael Heinrich","doi":"10.1159/000550486","DOIUrl":"10.1159/000550486","url":null,"abstract":"<p><strong>Introduction: </strong>Newly diagnosed autoimmune hemolytic anemia (AIHA) develops in 1-3 individuals per 100,000 people per year. Warm autoimmune hemolytic anemia (WAIHA) is the most common type of AIHA, accounting for approximately 70-80% of cases. This report outlines a complex presentation of DAT-negative, anti-e-mimicking autoantibody mediated WAIHA following a transient viral infection in an adult male patient with no prior transfusions.</p><p><strong>Case presentation: </strong>The 55-year-old male patient presented with several weeks of flu-like symptoms followed by jaundice, dyspnea, and progressive fatigue. Initial laboratory workup revealed hemolytic anemia, and subsequent serologic testing identified a warm autoantibody with anti-C specificity, but was ultimately consistent with an anti-e mediated WAIHA.</p><p><strong>Conclusion: </strong>This report highlights a complex case of a patient with an autoantibody mimicking anti-e associated WAIHA and the challenge of identifying suitable transfusion options.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-6"},"PeriodicalIF":1.1,"publicationDate":"2026-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147455089","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":"Chronic Myeloid Leukemia (CML): historical perspective, pathophysiology, and treatment advances.","authors":"Songphol Tungjitviboonkun, Lea Daran, Sorrawit Unsuwan","doi":"10.1159/000551493","DOIUrl":"https://doi.org/10.1159/000551493","url":null,"abstract":"<p><p>Chronic myeloid leukemia (CML) was the first leukemia to be described in medical literature and remains one of the most well-studied hematologic malignancies. This review traces the historical evolution of CML research, from its first clinical recognition in the mid-19th century to modern molecular diagnostics and targeted therapy. Key milestones include the discovery of the Philadelphia chromosome in 1960, identification of the BCR::ABL1 fusion gene in the 1980s, and the subsequent development of tyrosine kinase inhibitors (TKIs). The introduction of imatinib in the early 2000s revolutionized CML treatment, transforming a fatal disease into a chronic condition with near-normal life expectancy for most patients. Second- and third-generation TKIs have since been introduced to overcome drug resistance and target specific BCR::ABL1 mutations, such as T315I. Recently, research has focused on mechanisms of TKI resistance, novel signaling pathways, and strategies to achieve treatment-free remission (TFR). Emerging therapies such as vamotinib, KF1601, and combination regimens are being explored. Furthermore, new insights into non-kinase functions of BCR::ABL1 and the role of microRNAs in resistance open additional therapeutic avenues. This review provides a concise overview of CML from a historical and molecular perspective, highlighting diagnostic advances, evolving response criteria, and future directions in treatment.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-28"},"PeriodicalIF":1.1,"publicationDate":"2026-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147455106","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":"Differentiation Therapy in Acute Myeloid Leukemia: Advances in Phenotypic Screening and CRISPR-based Functional Genomics.","authors":"Shinichiro Takahashi","doi":"10.1159/000551445","DOIUrl":"https://doi.org/10.1159/000551445","url":null,"abstract":"<p><p>Acute myeloid leukemia (AML) is a clinically and genetically heterogeneous malignancy marked by a differentiation block in myeloid progenitors. Despite advances in molecular-targeted therapies, long-term outcomes remain poor, underscoring the need for novel treatment strategies. Differentiation therapy, exemplified by the success of all-trans retinoic acid in acute promyelocytic leukemia, offers a compelling alternative to cytotoxic approaches. This review highlights recent advances in phenotypic screening strategies-including high-throughput compound libraries, computational tools, and CRISPR-based functional genomics-which have revealed novel differentiation inducers, genes, and regulatory pathways. Notably, recent phenotypic screening studies identified Triciribine (TCN), an AKT inhibitor, as a differentiation inducer in AML cells. In addition, CRISPR loss- and gain-of-function screens have uncovered key regulators of differentiation, such as transcriptional (KAT6A), metabolic (NMNAT1, GLUT1), and post-transcriptional (ZFP36L2, YTHDC1) effectors. Emerging computational methods, such as the Lineage Maturation Index and single-cell data integration, further enhance target prioritization and translational relevance. However, differentiation therapy outside APL has shown variable and often incomplete clinical success, frequently limited by partial maturation and context-dependent responses. Together, these approaches reveal novel therapeutic vulnerabilities in AML and support the development of differentiation-based strategies for a broader range of patients.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-36"},"PeriodicalIF":1.1,"publicationDate":"2026-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147455118","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}