bims-hemali Biomed News
on Hematologic malignancies
Issue of 2026–01–11
23 papers selected by
Alexandros Alexandropoulos, Γενικό Νοσοκομείο Αθηνών Λαϊκό



  1. Expert Rev Hematol. 2026 Jan 09. 1-5
       INTRODUCTION: Chronic myeloid leukemia (CML) has been transformed by ATP-competitive BCR:ABL1 tyrosine kinase inhibitors (TKIs); however, resistance, intolerance, and long-term toxicity remain clinically relevant challenges, particularly in patients requiring prolonged therapy or multiple treatment lines. Asciminib, the first-in-class Specifically Targeting the ABL Myristoyl Pocket (STAMP) inhibitor, represents a paradigm shift by restoring physiologic ABL1 autoinhibition through allosteric binding.
    AREAS COVERED: This Special Report reviews the mechanistic basis, preclinical development, and clinical evidence supporting asciminib in CML. We summarize key data from pivotal trials, including ASCEMBL and ASC4FIRST, as well as emerging real-world studies, focusing on molecular response depth, safety, resistance mechanisms, and patient selection. Particular attention is paid to how STAMP inhibition differs from ATP-competitive TKIs in terms of selectivity, toxicity profile, and resistance patterns, and how asciminib can be positioned relative to ponatinib in later-line settings.
    EXPERT OPINION: Asciminib has established itself as an effective and generally well-tolerated option for patients with TKI-resistant or -intolerant CML and is poised to expand into earlier lines of therapy. Its ability to induce rapid and deep molecular responses with reduced off-target toxicity may have important implications for long-term disease control and future treatment-free remission (TFR) strategies. Ongoing studies will clarify its optimal sequencing, combination potential, and role in facilitating durable TFR.
    Keywords:  Chronic myeloid leukemia; STAMP inhibitor; TKI resistance; asciminib; tyrosine kinase inhibitor
    DOI:  https://doi.org/10.1080/17474086.2026.2614391
  2. Leuk Lymphoma. 2026 Jan 07. 1-11
      This clinical trial evaluated assess the efficacy and safety of combination therapy with the BCL-2 antagonist venetoclax and the demethylase inhibitor azacitidine in 440 patients diagnosed with multiple myeloma. The primary endpoints included overall response rate (ORR), complete response (CR) rate, progression-free survival (PFS), and safety profile. The venetoclax-azacytidine combination demonstrated comparable efficacy, with enhanced depth of response in patients with high-risk cytogenetic abnormalities. Adverse events were generally manageable, with neutropenia observed in 25% and fatigue in 30% of patients. Minimal residual disease (MRD) negativity and specific cytogenetic markers, such as t(11;14), were identified as significant independent predictors of favorable treatment response. The therapy demonstrated clinical benefit with a manageable toxicity profile, suggesting potential utility as a treatment option in selected patient subgroups. However, the need for longer follow-up and expanded cohorts remains critical to validate these outcomes and optimize patient selection. These findings support the further exploration of venetoclax and azacitidine as a viable treatment option for patients with refractory or high-risk multiple myeloma, warranting additional prospective trials before consideration for standard-of-care implementation.
    Keywords:  5-Azacitidine; Venetoclax; cytogenetic abnormalities; minimal residual disease; multiple myeloma
    DOI:  https://doi.org/10.1080/10428194.2025.2606209
  3. Front Cell Dev Biol. 2025 ;13 1725857
       Introduction: Therapy of chronic myeloid leukemia (CML) with tyrosine kinase inhibitors (TKIs) targeting the BCR::ABL1 kinase has become a paradigm for precision oncology. Despite the tremendous success of this strategy, with an overall long-term survival rate of 83%, approximately 25% of CML patients experience therapy failure within 5 years of treatment. TKI resistance is multifaceted, involving mutations in BCR::ABL1, but also BCR::ABL1-independent mechanisms. Among them, deregulation of cell adhesion and motility of CML cells has been observed in TKI-resistance. The extracellular matrix protein fibronectin 1 (FN1) has been shown to be deregulated in solid tumors promoting proliferation and metastasis. However, the role of FN1 in hematopoietic neoplasms remains to be fully elucidated. The aim of our study was to gain deeper insights into the role of FN1.
    Methods: FN1 mRNA and protein levels were analyzed using qPCR and immunoblotting. Transfection was performed using nucleofection or stable transfection, followed by analyses of cell number, proliferation and viability. Cell adhesion was assessed using Matrigel-coated surfaces, and FN1 localization was analyzed using immunofluorescence.
    Results: FN1 levels were significantly downregulated in CML cell lines resistant against BCR::ABL1 inhibitors in vitro. SiRNA-mediated FN1 knockdown reduced the cell's susceptibility to all generations of TKIs employed in treatment of CML, including asciminib. In contrast, the restoration of FN1 expression in TKI-resistant cells re-sensitized the cells to TKI treatment. This effect was also observed in K-562 cells that intrinsically harbor the BCR::ABL1 mutation p. E255K (-35.2%, p < 0.001), as well as in K-562 and Ba/F3 cells after stable transfection of the BCR::ABL1 wild-type or the p. T315I gatekeeper mutation. Clinically, deregulation of FN1 was also observed in peripheral blood cells derived from CML patients.
    Conclusion: Our data indicate that FN1 may serve as a potential therapeutic target to address TKI resistance or as a suitable biomarker for the treatment.
    Keywords:  chronic myeloid leukemia; drug resistance; fibronectin 1; imatinib; tyrosine kinase inhibitor
    DOI:  https://doi.org/10.3389/fcell.2025.1725857
  4. Leukemia. 2026 Jan 07.
      B-cell maturation antigen (BCMA)-targeting therapies provide a new approach to treating multiple myeloma (MM). Alnuctamab (ALNUC) is a 2 + 1 immunoglobulin G1-based bispecific antibody binding BCMA and CD3ε receptors on myeloma and T cells, respectively. CC-93269-MM-001 is a first-in-human, phase 1 dose escalation/expansion study investigating ALNUC in relapsed/refractory MM. Patients had ≥3 prior regimens, disease progression ≤60 days of last regimen, and were BCMA-directed therapy-naïve. ALNUC was administered intravenously (IV) and subcutaneously (SC); however, SC was selected for further evaluation due to the more favorable safety profile. Ninety-five patients received ALNUC SC; at data cutoff, 44.2% remained on treatment and median follow-up was 8.0 months. The recommended phase 2 dose was 30 mg. The most common treatment emergent adverse events (any grade/grade 3/4) were CRS (57.9%/0%), and neutropenia (53.7%/43.2%). Infections were also frequent (64.2%/14.7%). ORR was 58.9% for all ALNUC SC-treated patients and 71.4% for the 30-mg cohort; 47/95 (49.5%) were measurable residual disease (MRD) negative. Overall, the safety and efficacy of ALNUC SC were comparable to other BCMA-targeted therapies. These results support improved safety of SC versus IV, and corroborate a step-up dosing strategy to mitigate CRS. Importantly, a schedule that de-intensifies over time provides favorable toxicity that may be applicable to other bispecific engagers.
    DOI:  https://doi.org/10.1038/s41375-025-02841-x
  5. Blood Cancer J. 2026 Jan 03.
      In newly diagnosed multiple myeloma (NDMM), current risk stratification relies on cytogenetics and disease burden, but emerging immune parameters may refine prognosis. While a higher non-clonal plasma cell fraction (NCPF) predicts favorable outcomes in smoldering myeloma, its relevance in NDMM remains unexplored. We retrospectively analyzed 798 patients with NDMM where baseline bone marrow aspirates were tested to determine the NCPF, defined as the proportion of non-clonal plasma cells among total plasma cells. An NCPF ≥ 5% defined the NCPF-Enriched group. NCPF-High was observed in 124 patients (15.5% of all cohort). Compared to NCPF-Low, NCPF-Enriched patients had lower overall bone marrow plasma cell burden (median 20% vs. 50%, p < 0.0001), higher rates of hyperdiploidy (71% vs. 59%, p = 0.025), and similar cytogenetic risk. Six-year overall survival (OS) was significantly higher in NCPF-Enriched (70.3% vs. 56.5%, p = 0.0096), with independent prognostic value in multivariable analysis (HR 0.64, p = 0.03). Progression-free survival was also superior (HR 0.69, p = 0.006) in the NCPF-Enriched cohort in the setting of comparable treatment approaches. A higher non-clonal plasma cell fraction at diagnosis is independently associated with improved outcomes in NDMM, highlighting its potential as a novel immune prognostic biomarker warranting further investigation.
    DOI:  https://doi.org/10.1038/s41408-025-01449-9
  6. J Clin Med. 2025 Dec 24. pii: 128. [Epub ahead of print]15(1):
      Background: Ropeginterferon alfa-2b (Ropeg-IFNa) is increasingly used in myeloproliferative neoplasms (MPN), particularly polycythemia vera, but real-world data across subtypes are limited. We evaluated clinical and molecular responses to Ropeg-IFNa in routine practice. Methods: We retrospectively analyzed 20 JAK2V617F-positive MPN patients treated at a tertiary center. Baseline features, dosing, treatment line, hematologic responses, adverse events, and serial JAK2V617F variant allele frequency (VAF) were extracted from records. Results: Median age at initiation was 53 years; 55% were ELN high-risk. Ropeg-IFNa was started first-line or after peginterferon alfa-2a, hydroxyurea, or a tapered JAK2 inhibitor. Mean treatment duration was 14 ± 11 months at 195 ± 143 µg Q2W. Hematologic control increased from 45% at the start to 60% at the last follow-up. Among patients with serial molecular monitoring (n = 11), median JAK2V617F VAF declined from 21.2 to 12.7%. Ropeg-IFNa was generally well tolerated; adverse effects were mostly manageable, although 3/20 (15%) discontinued due to side effects, including mood disturbances, while others continued with supportive care and dose adjustments. Conclusions: In this single-center cohort, Ropeg-IFNa was tolerable and associated with improved hematologic control and modest VAF reductions, supporting its use in multi-subtype MPN cohorts. These findings underscore the value of longitudinal driver-mutation monitoring during therapy.
    Keywords:  JAK2 V617F; MPN-U; essential thrombocytopenia; pegylated interferon alpha; polycythemia vera; primary myelofibrosis
    DOI:  https://doi.org/10.3390/jcm15010128
  7. Cancer. 2026 Jan 01. 132(1): e70237
       BACKGROUND: Teclistamab is the first approved bispecific antibody targeting B-cell maturation antigen. It has demonstrated rapid, deep, durable responses with manageable safety in patients with triple-class exposed relapsed/refractory multiple myeloma (TCE RRMM).
    METHODS: The authors report results from three cohorts: Global Trial cohort consisting of 217 patients from three registrational studies (pivotal MajesTEC-1 study [n = 165], China cohort of MajesTEC-1 [n = 26], and the Japan MMY1002 study [n = 26]); the subset of 52 of 217 patients formed the Asian Trial cohort, and 42 patients treated outside of trials in the pre-approval access (PAA) program formed the Asian PAA cohort.
    RESULTS: In the Global Trial cohort, median age was 65 years, weight was 69 kg, and prior lines-of-therapy was five; 29.0% had high-risk cytogenetics and 18.9% had extramedullary disease. With 29.5 months median follow-up, overall response rate (ORR)/≥complete response (CR) was 66.4%/50.2%, median duration of response (DOR) was not reached; progression-free survival (PFS) and overall survival (OS) were 15.6, and 29.1 months, respectively. In the subset of Asian Trial cohort, baseline features were similar except for lower weight (median, 58 kg); median follow-up was 26.3 months. ORR/≥CR was 76.9%/63.5%, 24-month DOR, PFS, and OS rates were 67.5%, 59.5%, 71.4%, respectively, with medians not yet reached. Efficacy was consistent in the Asian PAA cohort with ORR/≥CR of 66.7%/40.5%. Most common adverse events were cytopenias, cytokine release syndrome, and infections. Infection management improved over time, supported by increased immunoglobulin use in later-enrolling Asian studies, aligned with guideline adoption.
    CONCLUSION: Teclistamab demonstrated clinically meaningful benefits across diverse patients, encompassing various weight categories and geographies, reinforcing its potential as a standard of care for TCE RRMM.
    TRIAL REGISTRATION: ClinicalTrials.gov MajesTEC-1 (NCT03145181 and NCT04557098) and Japan MMY1002 study (NCT04696809).
    Keywords:  Asian; bispecific antibody; outside trial setting; relapsed/refractory multiple myeloma; teclistamab; triple‐class exposed
    DOI:  https://doi.org/10.1002/cncr.70237
  8. bioRxiv. 2025 Dec 26. pii: 2025.12.24.696284. [Epub ahead of print]
      Acute myeloid leukemia (AML), the most common hematologic malignancy, generally has a poor prognosis. Despite initial favorable responses to the BCL2 inhibitor venetoclax (VEN), remission is transient, and AML is eventually fatal. Resistance to VEN is primarily due to the overexpression of anti-apoptotic proteins, including MCL-1, BCL2L1 (BCL-XL), and BCL2A1. Casein kinase II (CK2) is a serine-threonine kinase and a known suppressor of apoptosis. We and others have reported that protein kinase CK2 activity is high in leukemic stem cells (LSCs) and associated with resistance to chemotherapy. We have shown that the selective CK2 inhibitor, CX-4945, suppresses BCL-XL and has a significant anti-tumor effect in AML preclinical models. CK2 expression and activity are high in venetoclax-resistant AML (VR-AML) cell lines. Genetic and pharmacological inhibition of CK2 significantly altered VR-AML gene signature, decreased MCL-1 protein level, increased BH3 priming and sensitized VR-AML cells to apoptosis. More importantly, CX-4945 selectively targeted LSCs (CD34+CD38-) and chemoresistant (CD123+CD47+) subpopulation in VR-AML. CX-4945 combined with VEN decreased leukemia burden and prolonged the survival of VR-AML cell line-derived and patient-derived xenografts compared to either drug alone. The combinatorial treatment was well tolerated in mice without additional myelosuppression or organ toxicity. CX-4945 (silmitasertib) is being tested in several early-phase clinical trials against adult and pediatric cancers. These preclinical results support the use of CX-4945 in combination with VEN to overcome resistance to apoptosis and re-sensitize VR-AML to chemotherapy.
    DOI:  https://doi.org/10.64898/2025.12.24.696284
  9. Blood Neoplasia. 2026 Feb;3(1): 100171
      This phase 1 study investigated the addition of gemtuzumab ozogamicin (GO) to intensive chemotherapy with cytarabine, daunorubicin, and midostaurin in 21 patients with newly diagnosed (ND) FMS-like tyrosine kinase 3 (FLT3)-mutated acute myeloid leukemia (AML). Four dose levels of GO were evaluated. The use of GO was tolerable, with all dose-limiting toxicities similar to those seen in standard-of-care treatment. After induction, the median time to platelet recovery was 26 days, and the median time to absolute neutrophil count (ANC) recovery was 27 days. The maximum tolerated dose was cytarabine 100 mg/m2 on days 1 to 7, midostaurin 50 mg twice daily on days 8 to 21, daunorubicin 60 mg/m2 on days 1 to 3, and GO 3 mg/m2 on days 1 and 4. For the 18 patients who were evaluable for response after induction therapy, 16 patients (76%) achieved a composite complete response (complete remission [CR] + CR with incomplete hematologic recovery), and 2 (10%) had stable disease. Of the 14 patients who proceeded to consolidation, 5 discontinued the study for transplant, 1 for disease progression, and 1 for physician discretion. Seven patients completed consolidation therapy, all of whom achieved a CR. In total, 13 of the 21 patients (62%) received a hematopoietic stem cell transplant. Our results show that GO can safely be combined with intensive chemotherapy with midostaurin in ND, FLT3-mutated AML. This trial was registered at www.clinicaltrials.gov as #NCT03900949.
    DOI:  https://doi.org/10.1016/j.bneo.2025.100171
  10. Sci Rep. 2026 Jan 05.
      Chronic myeloid leukemia (CML) is an oncogenic hematologic disorder defined by the BCR-ABL1 fusion gene, which initiates pathological proliferation of myeloid lineage cells. While tyrosine kinase inhibitors (TKIs) have substantially improved clinical outcomes, therapeutic resistance associated with the T315I mutation continues to present a significant obstacle. This research evaluates the efficacy of caffeic acid phenethyl ester (CAPE), a bioactive natural product, in countering TKI resistance mediated by this specific genetic alteration. The investigation employed a comprehensive methodology including MTT assay, apoptotic analysis through flow cytometry, proteomic profiling, and bioinformatic interrogation to characterize CAPE's effects on both wild-type and T315I-mutant CML cellular models. MTT assay indicated that CAPE exhibited potent anti-metabolic activity against CML cells, promoting apoptosis in a dose-dependent manner. Proteomic analysis identified a marked effect of CAPE on the oxidative phosphorylation(OXPHOS) pathway, particularly through the inhibition of mitochondrial complex I(MCI) activity. This inhibition may disrupt cellular energy metabolism, potentially reducing ATP production and heightening susceptibility to cell death. Our findings indicate that CAPE could serve as an adjunctive therapy for CML against drug resistance caused by the T315I mutation through a mechanism that does not directly inhibit BCR-ABL1. This study underscores the promise of targeting mitochondrial metabolism as a novel approach for overcoming therapeutic resistance in CML.
    Keywords:  BCR-ABL1^T315I^ mutation; Caffeic acid phenethyl ester (CAPE); Mitochondrial complex i (MCI); Oxidative phosphorylation (OXPHOS)
    DOI:  https://doi.org/10.1038/s41598-025-34553-8
  11. Blood. 2025 Dec 23. pii: blood.2025031476. [Epub ahead of print]
      Circadian rhythms orchestrate immune activation and effector function, yet whether within-day timing influences chimeric antigen receptor (CAR) T-cell therapy outcomes remains unknown. We conducted an international, multicenter retrospective study of 1,052 adults with relapsed or refractory large B-cell lymphoma treated with CD19-directed CAR T-cell therapy across seven centers (2017-2025). The median infusion time was 11:48 AM (interquartile range, 11:06 AM-12:45 PM). Each hour later in infusion time was associated with an increased risk of progression, relapse, or death (hazard ratio 1.11; 95% CI, 1.03-1.20; P = 0.004) after adjustment for center, product, and key clinical variables. One-year progression-free survival (PFS) was 51.4% for early (<12:00 PM) versus 35.2% for late (≥12:00 PM) infusion, while overall survival was similar between groups. The PFS benefit was driven by lower relapse and higher complete response rates in the early infusion group. Although no differences were observed in immune toxicities, late infusion correlated with higher peak inflammatory markers and reduced day 7 CAR T-cell expansion. Together, these multicenter data provide the first clinical evidence that the timing of CAR T-cell infusion may influence therapeutic efficacy and support prospective evaluation of circadian-informed delivery strategies.
    DOI:  https://doi.org/10.1182/blood.2025031476
  12. Blood. 2026 Jan 05. pii: blood.2025031455. [Epub ahead of print]
      Secondary central nervous system (CNS) large B-cell lymphoma (SCNSL) occurs in the de novo setting, as a CNS-isolated relapse, or synchronous (concomitant CNS and systemic) relapse. SCNSL is a devastating event without therapeutic consensus. Thus, we aimed to evaluate treatment outcomes in an international cohort. Progression-free survival (PFS), overall survival (OS) and cumulative incidence of relapse (CIR, estimated using competing-risk models) were reported. Prognostic factors were identified in a 6-month landmark multivariate analysis. Outcomes following thiotepa autologous stem cell transplant (ASCT) and chimeric antigen receptor T-cell therapy (CAR-T) delivered at relapse were compared following propensity score matching (PSM). A total of 1139 patients were included in the analysis (de novo: 537; relapsed SCNSL: 602). 2-year PFS estimates were 40.4%, 43.9% and 16.2% for de novo SCNSL, CNS-isolated relapse, and synchronous relapse respectively. Patients with CNS-isolated relapse demonstrated low rates of systemic recurrence (24-month CIR 6%). Thiotepa-ASCT correlated with longer survival in de novo SCNSL (PFS: HR=0.57; P=0.005; and OS: HR=0.62; P=0.023) and CNS-isolated relapses (PFS: HR=0.55; P=0.002; and OS: HR=0.39; P<.0001) in 6-month multivariable landmark analysis. ASCT (thiotepa or non-thiotepa) also associated with improved survival in synchronous relapses (PFS: HR=0.57; P=0.023; and OS: HR=0.48; P=0.019). Higher survival with thiotepa-ASCT compared to CAR-T was observed in survival analyses following PSM (PFS: HR=0.45; P=0.005 and OS: HR=0.41; P=0.014). These data support thiotepa-ASCT in eligible patients, particularly de novo disease and CNS-isolated relapses. CNS-isolated relapse was infrequently associated with systemic recurrence, supporting treatment regimens adopted from primary CNS lymphoma.
    DOI:  https://doi.org/10.1182/blood.2025031455
  13. Cancers (Basel). 2026 Jan 02. pii: 159. [Epub ahead of print]18(1):
       BACKGROUND: Higher-risk myelodysplastic syndromes (HR-MDS) carry a high risk of progression to acute myeloid leukemia and poor overall survival. Hypomethylating agents (HMAs), such as azacitidine, remain the standard of care but have limited efficacy. A 15-day venetoclax-azacitidine regimen has shown promising objective response rates (ORR) and potential as a bridge to allogeneic hematopoietic stem cell transplantation (HSCT) in relapsed/refractory HR-MDS. We conducted a prospective multicenter trial to evaluate its efficacy and safety in previously untreated patients.
    METHODS: This multicenter prospective study enrolled treatment-naïve HR-MDS patients (IPSS-R > 3.5). Venetoclax was administered on days 1-15 (escalated from 100 to 400 mg), combined with azacitidine (75 mg/m2) on days 1-7 of each 28-day cycle. The primary endpoint was ORR (2006 IWG criteria); secondary endpoints included complete remission (CR), overall survival (OS), and AML progression.
    RESULTS: Twenty-eight patients (median age: 63 years) were enrolled, with a median follow-up of 8.5 months. ORR was 85.7% per 2006 IWG (CR: 35.7%, marrow CR: 50.0%), and 78.6% per 2023 IWG (CR: 35.7%). Responses were consistent across molecular and IPSS-R subgroups. Median OS was not reached. High neutrophil count and high cytogenetic risk were favorable factors; TP53 mutation/deletion was an adverse prognostic marker. Grade 3-4 hematologic toxicities included neutropenia (96.4%), anemia (71.4%), and thrombocytopenia (64.3%). Serious adverse events (35.7%) were mainly infections. No dose-limiting or unexpected toxicities were observed.
    CONCLUSIONS: The 15-day venetoclax plus azacitidine regimen demonstrated high efficacy and manageable toxicity in treatment-naïve HR-MDS. It may be particularly beneficial for patients with high neutrophil counts, adverse cytogenetics, or those eligible for HSCT, supporting further investigation in larger trials.
    Keywords:  azacitidine; hematopoietic stem cell transplantation; higher-risk myelodysplastic syndromes; venetoclax
    DOI:  https://doi.org/10.3390/cancers18010159
  14. Blood. 2026 Jan 09. pii: blood.2025028645. [Epub ahead of print]
      JAK inhibitors have changed the treatment landscape of myeloproliferative neoplasms, graft-versus host and several autoimmune conditions. While approved JAK inhibitors generally target the JAK2 kinase domain, and several also the JAK1 kinase domain in active form (type I inhibition), new inhibitors have progressed to clinical trials that either exhibit a type II mechanism of inhibition of the kinase domain in an inactive state or that target the pseudokinase domain with potential preference or specificity for the JAK2 V617F mutant. This is the most prevalent mutant in myeloproliferative neoplasms. An ideal inhibitor would target persistently activated JAK2 in MPNs, eradicate the clone or induce deep molecular remission in addition to clinical and hematological remission and spare wild type JAK2 that is critical for hematopoiesis and immune response. We discuss perspectives of these and other modes of JAK inhibition and primary as well as secondary/exploratory study endpoints in clinical trials design, along with potential biomarker correlates to evaluate potential efficacy of the next generation versus conventional JAK inhibitors.
    DOI:  https://doi.org/10.1182/blood.2025028645
  15. Oncol Res. 2025 ;34(1): 10
       Background: Breakpoint Cluster Region-Abelson (BCR::ABL1) fusion protein is essential in the pathogenesis of chronic myeloid leukemia (CML); however, the chronic-to-blast phase transformation remains elusive. We identified novel kinesin light chain 2 (KLC2) mutations in CML-myeloid blast phase patients. We aimed to examine the functional role of KLC2 mutations in leukemogenesis.
    Methods: To evaluate the biological role of KLC2 mutants (MT) in CML cells, we expressed KLC2-MT in different human CML cell lines harboring BCR::ABL1 and performed immunoblot, immunofluorescence, cell proliferation, differentiation, and apoptosis; Tyrosine kinase inhibitor (TKI)-drug activities; and clonogenic assays for in vitro functional analyses. We co-expressed KLC2-MT and BCR::ABL1 in mouse bone marrow cells (BMCs) to evaluate their clonogenic and self-renewal abilities ex vivo. Furthermore, we examined tumorigenic activity and drug efficacy in the K562 xenograft model.
    Results: KLC2-MT overexpression in BCR::ABL1-positive K562 and KU812 CML cells promoted cell proliferation and clonogenic potential, decreased imatinib sensitivity, and reduced apoptosis. Serial colony replating assays revealed that KLC2-MT and BCR::ABL1 co-expression enhanced the self-renewal ability of mouse BMCs with immature morphology. In the K562 xenograft model, KLC2-MT enhanced tumorigenic potential and diminished imatinib efficacy. Further studies reported that KLC2-MT augmented signal transducer and activator of transcription 3 (STAT3) activation and nuclear accumulation in imatinib-treated CML cells. KLC2-WT and KLC2-MT interacted with mothers against decapentaplegic homolog 2 (SMAD2); however, the latter impaired transforming growth factor-beta (TGF-β)-mediated SMAD2/3 activation while enhancing STAT3 phosphorylation.
    Conclusions: This study demonstrates the biological and functional importance of KLC2 mutation in CML cells, potentially enabling the development of better treatment strategies for CML patients carrying KLC2 mutations and providing enhanced understanding of the disease progression.
    Keywords:  Chronic myeloid leukemia (CML); drug resistance; kinesin light chain 2 (KLC2); myeloid blast transformation; signal transducer and activator of transcription 3 (STAT3)
    DOI:  https://doi.org/10.32604/or.2025.070259
  16. Blood Cancer J. 2026 Jan 08.
      In the decade since FDA approval of the first-generation Bruton's tyrosine kinase (BTK) inhibitor ibrutinib, the treatment landscape for chronic lymphocytic leukemia (CLL) has transformed. Targeted agents, as monotherapy or in combination regimens, have decisively replaced chemoimmunotherapy as the standard of care. In national CLL guidelines updated from 2023 through 2025 - including those from France, Germany, and the US - chemoimmunotherapy is considered a treatment option in only exceptional cases, reflecting broad consensus among experts that targeted therapies should be used universally in the management of previously untreated CLL. The primary first-line treatment options for CLL comprise continuous therapies based on the BTK inhibitors ibrutinib, zanubrutinib, or acalabrutinib, or fixed-duration regimens combining the BCL2 inhibitor venetoclax with obinutuzumab or the BTK inhibitors ibrutinib or acalabrutinib (with or without obinutuzumab). Selecting the appropriate targeted therapy requires careful evaluation of a multitude of factors, including molecular disease features (especially del[17p]/TP53 and IGHV mutational status), comorbidities, comedications, and the patient's preferences. Focusing on primary treatment options, we review data from the key controlled trials that support the first-line use of targeted therapies for patients with CLL, consider ongoing trials that may support clinical decision-making in the future, and assess the potential to personalize treatment regimens in CLL based on minimal residual disease status.
    DOI:  https://doi.org/10.1038/s41408-025-01434-2
  17. Haematologica. 2026 Jan 08.
      The 5-point Deauville score (DS) assesses end-of-treatment (EOT) response on PET/CT in diffuse large B-cell lymphoma patients, categorizing scans as 'positive' or 'negative' for complete metabolic response. However, the positive predictive value (PPV) is suboptimal at 60%. We evaluated whether quantitative PET parameters combined with clinical data could improve prediction of treatment failure in EOT PET-positive patients. Baseline and EOT PET/CT scans of 138 DS4-5 patients were analyzed. Lesions were segmented using a semi-automated adaptive method (SUV4.0 or MV3). PET parameters, including total metabolic tumor volume (TMTV), number of lesions (NOL), tumorSUV/liverSUV-ratio (TLR), the maximum distance between the largest and any other lesion (DmaxBulk), and changes over time, were obtained. Two Cox regression models predicted 2-year progression-free survival. Clinical data were combined with EOT PET in model 1, and baseline, EOT and delta values in model 2. After internal bootstrapping, models were evaluated for classification using different risk-of-progression cutoffs. Sensitivity, specificity, PPV and negative predictive values (NPV) were determined. Using forward selection, model 1 comprised two variables: the NOL and the tumorSUVpeak/liverSUVmean (TLRpeakmean) at EOT (AIC=690.072, c-index=0.747). Model 2 incorporated NOL, TLRpeakmean (EOT) and baseline SUVmean (AIC=687.064, c-index=0.762). The PPV improved to over 85% without compromising the NPV. False positives dropped from 54 (39%, by DS) to 9 (7%) and 6 (4%) for models 1 and 2, respectively. Adding baseline features did not notably impact the models' performance. Our models could support more accurate response-adapted treatment decisions, reducing unnecessary subsequent false positive-directed treatments to just 7%.
    DOI:  https://doi.org/10.3324/haematol.2025.288821
  18. Leukemia. 2026 Jan 07.
      Aggressive NK-cell leukemia (ANKL) is a rare NK-cell leukemia characterized by, an aggressive clinical course, and frequent Epstein-Barr virus association. We retrospectively collected 126 ANKL patients diagnosed between 2000 and 2021 from 71 institutes; 108 were evaluable for analysis to characterize the recent advances. The median age was 49 years (range: 17-90), and 63 patients were male. The median percentage of leukemic cells in the bone marrow and peripheral blood was 19.7% and 10.0%, respectively. The first-line regimens included SMILE (n = 38, 42%), CHOP(-like) (n = 26, 29%), DeVIC (n = 15, 17%), and other L-asparaginase-containing regimens (n = 8, 9%), with the overall response rates of 66%, 31%, 33%, and 50%, respectively. Median overall survival (OS) was 5.5 months, with a 2-year OS of 18.7%. Patients treated with SMILE had significantly better OS than others (2-year OS 30.1% vs. 7.0%; P < 0.001). Prognosis further improved with allogeneic hematopoietic stem cell transplantation (HSCT) (2-year OS 37.2% vs 3.5%; P < 0.001). Multivariate analysis confirmed treatment with SMILE [hazard ratio (HR) 0.53, 95% confidential interval (CI) 0.31-0.88] and allogeneic HSCT (HR 0.33, 95% CI 0.18-0.61) as independent favorable factors. In conclusion, SMILE chemotherapy followed by allogeneic HSCT may improve outcomes in ANKL.
    DOI:  https://doi.org/10.1038/s41375-025-02854-6
  19. Cytotechnology. 2026 Feb;78(1): 26
      Multiple myeloma (MM) is a clonal plasma cell disorder and is the second most common hematologic malignancy worldwide. In recent years, ferroptosis has emerged as an important target for cancer therapy, including MM. Cordycepin (COR) is a nucleoside antibiotic that was first isolated from fungi, with potential anti-tumor properties. The current research elucidated the therapeutic mechanism against MM of COR by investigating its effects on ferroptosis. U266 or NCI-H929 cells were incubated with COR (1, 3, and 10 µM) for 24 h, respectively. In both U266 and NCI-H929 cells, dramatically declined cell viability, reduced migrated cell counts, increased ROS productions and MDA levels, and repressed SOD activities were observed following COR incubation. Furthermore, in COR-treated U266 or NCI-H929 cells, markedly increased Fe2+ levels, upregulated ACSL4, and downregulated GPX4 were induced by COR, accompanied by an inhibition of HIF-1α/SLC7A11 axis and an upregulation of CLEC2. To confirm the role of ferroptosis and CLEC2 in COR's anti-tumor function, U266 cells were treated by COR for 24 h, followed by incubation with Fer-1 or transfected with si-CLEC2. The decreased cell viability, reduced migrated cells, increased ACSL4 levels, downregulated GPX4, and inhibited HIF-1α/SLC7A11 axis in COR-treated U266 cells were remarkably reversed by Fer-1 or silencing CLEC2. In addition, inhibitory effects of COR on the growth of U266 xenograft model, as well as facilitating effects of COR on ferroptosis in U266 xenograft tumor tissues, were reversed by Fer-1 or silencing CLEC2. Collectively, COR inhibited MM progression by inducing ferroptosis through upregulating CLEC2.
    Supplementary Information: The online version contains supplementary material available at 10.1007/s10616-025-00886-5.
    Keywords:  CLEC2.; Cordycepin; Ferroptosis; Multiple myeloma
    DOI:  https://doi.org/10.1007/s10616-025-00886-5
  20. Blood Adv. 2026 Jan 07. pii: bloodadvances.2025018757. [Epub ahead of print]
      Bruton tyrosine kinase inhibitors (BTKis) have dramatically changed the therapeutic landscape of chronic lymphocytic leukemia (CLL), with ibrutinib, first-in-class, demonstrating durable efficacy even in high-risk patients. However, off-target adverse events (AEs) have raised concerns, prompting the development of more selective second-generation BTKi, as zanubrutinib, designed to improve tolerability while maintaining efficacy. Despite encouraging results from clinical trials, real-world data comparing zanubrutinib with ibrutinib remain limited. In this multicenter, retrospective study, we analyzed 934 CLL patients treated outside clinical trials, including 393 receiving zanubrutinib and 541 receiving ibrutinib. We evaluated time to treatment discontinuation (TTD) and time to next treatment or death (TTNTD) in both the overall cohort and a propensity score-matched population. Zanubrutinib-treated patients experienced lower 12-month discontinuation rates (overall:12.6% versus 21.4%; matched:12.4% versus 20.2%) and higher 12-month TTNTD rates (overall:91.9% versus 83.0%; matched:93.2% versus 83.4%). Multivariable analyses confirmed zanubrutinib as an independent predictor of longer TTD and TTNTD, while high-risk features, including age, relapsed/refractory disease, Binet stage C, TP53 disruption, ECOG 2-3, and congestive heart failure, were consistently associated with poorer outcomes. AEs leading to discontinuation, particularly atrial fibrillation, bleeding, and infections, were less frequent with zanubrutinib, reflecting its favorable safety profile. These findings provide real-world evidence that zanubrutinib offers more durable disease control and improved persistence compared with ibrutinib, reinforcing its clinical value as a preferred second-generation BTKi. Nevertheless, the relatively short follow-up for zanubrutinib warrants cautious interpretation of long-term outcomes and underscores the need for ongoing observation to fully characterize its durability and safety.
    DOI:  https://doi.org/10.1182/bloodadvances.2025018757