bims-hemali Biomed News
on Hematologic malignancies
Issue of 2026–03–15
twenty-two papers selected by
Alexandros Alexandropoulos, Γενικό Νοσοκομείο Αθηνών Λαϊκό



  1. Int J Hematol. 2026 Mar 09.
      Ropeginterferon alfa-2b, a monopegylated interferon α-2b, is a cytoreductive treatment for polycythemia vera (PV). In Japan, the current regimen involves titration in 50-µg increments every 2 weeks until reaching the maximum dose (500 µg), which requires considerable time. This phase 3b, open-label, single-arm, multicenter study (October 2023-July 2024) assessed the efficacy and safety of a three-step dose escalation regimen (day 1: 250 µg, week 2: 350 µg, week 4: 500 µg) of ropeginterferon alfa-2b in Japanese patients with PV (NCT06002490). Twenty-one patients were included (mean age: 57 years); most (95.2%) received ropeginterferon alfa-2b for at least 24 weeks. The complete hematologic response rate (primary endpoint; hematocrit < 45%, no phlebotomy in the previous 12 weeks, platelet count ≤ 400 × 109/L, and white blood cell count ≤ 10 × 109/L) was 23.8% (95% confidence interval 8.2, 47.2) at week 12 and 57.1% (34.0, 78.2) at week 24. Most patients (90.5%) reached the maximum dose of 500 µg by week 4. All patients had treatment-emergent adverse events, but none led to death, treatment discontinuation, or study withdrawal. This three-step dose escalation regimen of ropeginterferon alfa-2b has the potential to provide faster therapeutic effects in patients with PV without additional safety concerns.
    Keywords:  Japan; Polycythemia vera; Ropeginterferon alfa-2b; Three-step dose escalation regimen
    DOI:  https://doi.org/10.1007/s12185-026-04163-9
  2. Acta Haematol. 2026 Mar 13. 1-28
      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.
    DOI:  https://doi.org/10.1159/000551493
  3. Haematologica. 2026 Mar 12.
      Targeted therapies have made near normal life span an attainable goal for many patients with chronic phase (CP) chronic myeloid leukemia (CML). Most patients require years of therapy and not everyone may be able to discontinue treatment permanently without CML recurrence. BCR::ABL1 targeted tyrosine kinase inhibitors (TKIs) including ATP binding site and allosteric inhibitors that bind to the myristoyl pocket are associated with treatment-emergent adverse events (TEAEs) that may compromise quality of life and well-being. Although alternative treatment options exist, side effects may persist, or new ones occur after a therapy switch. Using a case-based approach, this review examines the incidence of non-hematologic and hematologic TEAEs with specific therapies, provides guidance on AE management, and describes the impact of therapy dose reduction on efficacy and tolerability.
    DOI:  https://doi.org/10.3324/haematol.2025.288334
  4. Blood Adv. 2026 Mar 13. pii: bloodadvances.2025018482. [Epub ahead of print]
      Patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) typically have a poor prognosis. In preclinical studies, lenalidomide and a Bruton tyrosine kinase (BTK) inhibitor demonstrated synergistic antitumor effects. Zanubrutinib, a next-generation BTK inhibitor, has greater selectivity to minimize off-target binding. BGB-3111-110 was a phase 1 multicenter dose escalation/expansion study. Patients with R/R DLBCL received zanubrutinib 160 mg twice daily + lenalidomide (15, 20, or 25 mg once daily) until progression or unacceptable toxicity. Primary endpoints were safety, recommended phase 2 dose (RP2D), and overall response rate (ORR; Lugano 2014 criteria). Sixty-six patients were enrolled and treated. Patients had a median of 2 prior therapies, 83% had stage III/IV disease, and approximately 67% had non-geminal center B-cell-like or activated B-cell-like DLBCL. No dose-limiting toxicities occurred; the lenalidomide RP2D was 25 mg once daily when combined with zanubrutinib 160 mg twice daily. Grade ≥3 treatment-emergent AEs (TEAEs) occurred in 74%; the most common (>20%) grade ≥3 TEAEs were neutrophil count decreased (58%) and white blood cell count decreased (29%). TEAEs led to 7 treatment discontinuations (11%) and 2 deaths (3%). At the RP2D, ORR and complete response rate were 58% and 42%, respectively; median time to response was 2.8 months. Median duration of response was 14.9 months. Median progression-free survival was 5.5 months (95% CI, 2.9-11.1 months); the 12-month event-free rate was 34% (95% CI, 21%-48%). Zanubrutinib + lenalidomide demonstrated acceptable tolerability and antitumor activity in patients with R/R DLBCL. This trial was registered at ClinicalTrials.gov as #NCT04436107.
    DOI:  https://doi.org/10.1182/bloodadvances.2025018482
  5. Br J Haematol. 2026 Mar 08.
      The modern management of chronic myeloid leukaemia (CML) identifies a new therapeutic goal of treatment-free remission (TFR). Half of CML patients in durable deep molecular response (DMR) (MR4 or better) can remain off tyrosine kinase inhibitors (TKIs) without experiencing loss of major molecular response. Despite a large number of TFR studies to date, there are no consistent predictors of successful TFR. We conducted a single-centre cohort study on 197 patients discontinuing TKIs in DMR ≥1 year and TKI therapy ≥3 years. After TKI discontinuation, 98 patients (49.7%) lost MR4; of these, 90 (91.8%; and 45.7% of the whole cohort) lost major molecular response (MMR or MR3) after a median of 3.8 months (1-93.3). The 2-year probability of TFR (pTFR) was 57.7%. In multivariable analysis, male sex, age at diagnosis >40 years, faster achievement of MR4 and longer duration of DMR were the only variables significantly associated with higher pTFR. Based on the multivariable analysis results, we built a TFR prognostic score (TPS) able to distinguish three groups with different 2-year pTFR: good (89.9%), intermediate (61.2%) and poor (18.4%) TFR probability (p < 0.0001). We validated the TPS on an independent cohort of 91 patients. We propose that the TPS could become a useful guide for CML clinicians.
    Keywords:   DMR ; TFR ; TKI ; prognostic score
    DOI:  https://doi.org/10.1111/bjh.70409
  6. Clin Lymphoma Myeloma Leuk. 2026 Feb 16. pii: S2152-2650(26)00040-6. [Epub ahead of print]
       OBJECTIVES: To explore the efficacy and safety olverembatinib-based therapy in patients with transformed chronic myeloid leukemia in lymphoid blast phase (CML-LBP) or relapsed or refractory Philadelphia chromosome-positive acute lymphoblastic leukemia (R/R Ph+ ALL).
    METHODS: Data of patients with transformed CML-LBP or R/R Ph+ ALL receiving olverembatinib-based therapy from 24 academic centers in China were retrospectively interrogated. Cox regression model was utilized to identify covariates associated with survival.
    RESULTS: 75 patients with transformed CML-LBP (n = 26, 35%) and R/R Ph+ ALL (n = 49, 65%) were enrolled in this study. Median age was 51 years old (interquartile range [IQR], 40-57 years). With a median follow-up of 18 months (IQR, 11-27 months), excluding 1 patient with early death, 58 patients (78%) achieved complete remission/complete remission with incomplete hematologic recovery by 28 days. The 2-year probability of overall survival was 43% (95% Confidence Interval [CI], 29-56%) with median overall survival of 18 months (95% CI, 7-29 months). The therapy was well-tolerated. In multivariate analyses, higher hemoglobin concentration (Hazard ratio [HR] = 0.9 [0.7, 1.0], P = .049) and receiving a transplant (HR = 0.2 [0.1, 0.5], P = .001) were significantly-associated with better survival.
    CONCLUSIONS: Olverembatinib-based therapy is effective and tolerable in patients with transformed CML-LBP and R/R Ph+ ALL.
    Keywords:  Cardiovascular events; Efficacy; Safety; Tyrosine kinase inhibitor
    DOI:  https://doi.org/10.1016/j.clml.2026.02.005
  7. Br J Haematol. 2026 Mar 12.
      Imatinib has revolutionized the management of chronic-phase chronic myeloid leukaemia (CP-CML). This study aimed to evaluate the long-term efficacy, safety and prognostic determinants of imatinib in a large cohort of CP-CML patients in real-life clinical practice. We conducted a retrospective, monocentric observational study including all adult patients with CP-CML treated with imatinib between 2000 and 2025. Overall survival (OS), event-free survival (EFS) and progression-free survival (PFS) were estimated using Kaplan-Meier analysis. A total of 210 patients were included, of whom 81% received imatinib as first-line therapy. The median follow-up was 12.4 years. At 25 years, OS and PFS were 71% and 88% respectively. The EUTOS long-term survival (ELTS) score independently predicted OS, EFS and PFS (p < 0.001), while lower risk categories were associated with a faster achievement of major molecular response (MMR) and a higher probability of deep molecular response (DMR). Dose reductions were applied in 26.2% of patients, mainly older individuals with higher Charlson comorbidity index (CCI), without affecting molecular or survival outcomes. Higher CCI values correlated with inferior OS. The ELTS score remains a powerful prognostic tool for long-term outcomes. No unexpected safety concerns were observed in the long term. Over 25 years of real-world experience, imatinib has demonstrated sustained efficacy, safety and tolerability in CP-CML.
    Keywords:  efficacy; imatinib; long‐term follow‐up; outcomes; safety
    DOI:  https://doi.org/10.1111/bjh.70418
  8. Am J Hematol. 2026 Mar 07.
      Venetoclax and pirtobrutinib have emerged as two chemotherapy-free options for relapsed or refractory Waldenström macroglobulinemia (WM). However, evidence to guide treatment sequencing or identify molecular subsets most likely to benefit from each agent remains limited. We retrospectively evaluated consecutive WM patients treated with venetoclax or pirtobrutinib. Major response rate (MRR) and progression-free survival (PFS) were assessed for each agent, with predictors of response and PFS analyzed using logistic and Cox regression. Comparative efficacy was examined in unmatched analyses and in a 1:1 matched cohort. Among 91 treatment exposures (64 venetoclax and 27 pirtobrutinib) across 80 unique patients, treatment discontinuation due to adverse effects occurred in 12 of 64 patients (19%) treated with venetoclax and in none treated with pirtobrutinib. In the venetoclax cohort, TP53 alterations were associated with shorter PFS (10.0 vs. 35.6 months; p < 0.001). In the pirtobrutinib cohort, CXCR4 mutations predicted lower MRR (40% vs. 91%; p = 0.01) and shorter PFS (8.3 months vs. not reached; p = 0.02). When transitioning from a cBTKi, IgM rebound occurred in 62% (8/13) of patients initiating venetoclax without overlap, whereas no rebound was observed with cBTKi-venetoclax overlap (0/5) or with pirtobrutinib initiation (0/15). In the matched cohort (n = 42), venetoclax and pirtobrutinib demonstrated comparable outcomes for MRR (p = 0.91) and PFS (p = 0.83). Despite the retrospective design and limited sample size, these findings indicate comparable efficacy between venetoclax and pirtobrutinib with distinct molecular vulnerabilities and support consideration of pirtobrutinib sequencing when transitioning from a cBTKi, as well as further exploration of combination strategies that may exploit complementary vulnerabilities.
    Keywords:  CXCR4; IgM rebound; TP53; Waldenström macroglobulinemia; pirtobrutinib; venetoclax
    DOI:  https://doi.org/10.1002/ajh.70269
  9. Blood Cancer J. 2026 Mar 10.
      Multiple myeloma (MM), long viewed as incurable, is entering a transformative era marked by deeper remissions, prolonged survival, and the realistic prospect of cure. The greatest opportunity lies at diagnosis, before genomic heterogeneity and immune escape limit therapeutic impact. High-risk smoldering MM provides a preclinical window where early intervention with targeted antibodies or combinations can delay progression and, as in some trials, improve survival. Refining the threshold for treatment-entry requires integrated risk models that combine genomics, microenvironmental and immune profiling, and artificial intelligence. The therapeutic goal is sustained minimal residual disease (MRD) negativity at a sensitivity of 10⁻⁶, validated with functional imaging to exclude focal disease. Achieving two years of sustained MRD negativity in standard-risk and three years or longer in high-risk disease strongly predicts long-term progression-free survival; five years off therapy may approximate operational cure. Quadruplet induction, consolidation, and tailored maintenance regimens maximize depth and durability of response. To advance this vision, priorities include standardized MRD and imaging assays, MRD-adapted clinical trials, strategies balancing treatment toxicity with quality of life, and equitable global access. With decisive and risk-adapted strategies, achieving cure in MM within the next decade is no longer aspirational but increasingly within reach.
    DOI:  https://doi.org/10.1038/s41408-026-01461-7
  10. Blood. 2026 Mar 11. pii: blood.2025030972. [Epub ahead of print]
      Intensive chemotherapy is standard for AML but carries high risks of life-threatening complications, particularly in vulnerable patients. We aimed to compare the efficacy and safety of a low-dose chemotherapy (LDC) regimen for induction of AML. A randomized, multicenter, noninferiority trial was conducted in patients aged <18 years with AML. Patients received low-dose cytarabine, mitoxantrone or idarubicin, and G-CSF (LDC) or standard-dose induction chemotherapy (SDC) (cytarabine, daunomycin, and etoposide). All patients received post-remission consolidation with standard chemotherapy and/ or hematopoietic stem cell transplantation. The primary endpoint was to compare response rates between treatments. The secondary endpoints were to compare the outcomes, toxicity, and safety of the LDC and SDC regimens. The two treatment arms showed no significant differences in outcomes. Complete remission (CR/CRi) rates after induction were 95.1% and 95.3% in the LDC and SDC arms, respectively. Measurable residual disease < 0.1% after induction II was observed in 87.4% and 87.1% of patients in the LDC and SDC arms, respectively. Median time to neutrophil and platelet recovery was significantly shorter among patients receiving the LDC regimen. Patients in the LDC arm had a 4-year overall survival (OS) of 81.3% vs. 83.6% (P = .611) and a 4-year event-free survival (EFS) of 61.5% vs. 63.1% (P = .832). In conclusion, the LDC regimen was well tolerated and was associated with CR, EFS, and OS rates that were not inferior to those of patients treated with the SDC regimen. The trial was registered at Chinese Clinical Trial Registry (ChiCTR1800015883).
    DOI:  https://doi.org/10.1182/blood.2025030972
  11. Blood Cancer J. 2026 Mar 10.
      Diffuse large B-cell lymphoma (DLBCL) is an aggressive B-cell malignancy and is the most common subtype of lymphoma. Treatment is administered with curative intent and approximately two thirds of patients are expected to have durable long-term survival. To achieve this, anthracycline-based chemotherapy in combination with rituximab is typically administered as initial therapy. Management is optimized based on the disease stage, prognostic clinical features, and histological or molecular subclassification. In patients with the activated B-cell subtype of DLBCL, polatuzumab vedotin is commonly included in the combination. For those with Myc and BCL-2 rearrangements, a more treatment intense approach is used. Despite this risk-adapted approach, at least one third of patients relapse. Those who relapse within 1 year, or are resistant to initial therapy typically receive chimeric antigen receptor (CAR) T-cell therapy. For those relapsing more than a year post initial treatment, salvage chemotherapy followed by an autologous stem cell transplant is offered. In patients ineligible for cellular therapy, or those who progress after CAR T-cell treatment, management is palliative and includes administration of bispecific antibodies or antibody drug conjugate combinations. To further improve the outcome of DLBCL patients, incorporation of cellular and bispecific therapies into front-line treatment is currently being tested.
    DOI:  https://doi.org/10.1038/s41408-026-01458-2
  12. Am J Surg Pathol. 2026 Mar 10.
      Classic Hodgkin Lymphoma (CHL) remains difficult to treat in patients with relapsed and refractory disease. The utility of immunotherapies, many of which target B-cell markers, is still under investigation. Although the neoplastic Hodgkin and Reed-Sternberg (HRS) cells are thought to lose most B-cell markers, studies have shown retention of these markers, mainly CD20, in some cases. However, the CD19 staining profile of HRS cells has not been thoroughly assessed and warrants exploration in the era of CD19-directed immunotherapies. We assessed CD19 immunohistochemical staining in 41 cases of CHL and correlated with histologic subtype, other markers, and clinical parameters. 13/41 cases (31.7%) demonstrated CD19 staining in HRS cells, with variation in staining pattern and intensity. When compared with CD19-negative cases, CD19 positivity correlated significantly with mixed-cellularity subtype (53.8% vs. 7.1%, P=0.002), CD20 (46.2% vs. 14.3%, P=0.0485), CD79a (53.8% vs. 9.1%, P=0.006), Epstein-Barr Virus (EBV) positivity (53.8% vs. 7.1%, P=0.002), and older age (median age 52 vs. 28.5 y, P=0.0006). 7/9 (77.8%) EBV+ cases demonstrated CD19 expression on HRS cells. By Kaplan-Meier analysis, CD19+ cases demonstrated worse overall survival compared with CD19- cases (P=0.011), with EBV-, CD19- cases demonstrating the best overall survival. On the basis of these findings, CD19 expression on HRS cells should not preclude the pathologic diagnosis of CHL, particularly in the context of mixed-cellularity or EBV+ disease. In addition, older patients with mixed-cellularity and/or EBV+ disease may represent a subgroup of patients with CHL who could benefit from CD19-directed therapies.
    Keywords:  CD19; Classic Hodgkin lymphoma; immunohistochemistry; immunotherapy implications
    DOI:  https://doi.org/10.1097/PAS.0000000000002519
  13. Leuk Res Rep. 2026 ;25 100578
      Bruton tyrosine kinase (BTK) inhibitors are used to treat chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), providing survival benefits over traditional chemoimmunotherapy. Richter transformation is the transformation of CLL/SLL into an aggressive lymphoma, typically necessitating an immediate start of treatment. This case report presents a CLL/SLL patient developing pathology-proven Richter transformation after a short hold of the BTK inhibitor, without clinical evidence of disease progression. This case also demonstrates that the pathology-only Richter transformation after a pause of the BTK inhibitor can respond to resumption of the inhibitor, without the addition of intensive chemotherapy.
    Keywords:  Bruton tyrosine kinase inhibitor; Chronic lymphocytic leukemia; Ibrutinib; Richter transformation; Richter-like transformation; Small lymphocytic lymphoma
    DOI:  https://doi.org/10.1016/j.lrr.2026.100578
  14. Blood Cancer Discov. 2026 Mar 11.
      Bispecific antibodies have revolutionized the treatment of multiple myeloma (MM), however primary treatment failure occurs in 30-40% of patients. In this study, we analyzed correlates of response to teclistamab and strategies to overcome primary resistance. Across two independent cohorts (n=90), we developed Hi-MM, defined by extramedullary disease, plasma cell leukemia, bone marrow plasmacytosis ≥50%, or transfusion within 30 days, as a composite correlate of non-response. Patients without Hi-MM had ORR of 84-96% (vs 20-40% with Hi-MM), and significantly superior progression-free (p<0.001) and overall survival (p<0.001). Debulking chemotherapy was utilized in 19 patients; 79% then responded to teclistamab, including 100% who no longer had Hi-MM. All four patients who were primary refractory to a BCMA bispecific immediately prior to debulking then achieved deep responses to teclistamab. In conclusion, simple clinical parameters correlate with response to teclistamab, while debulking chemotherapy can overcome Hi-MM and successfully bridge patients to teclistamab or salvage non-responders.
    DOI:  https://doi.org/10.1158/2643-3230.BCD-25-0372
  15. ESMO Open. 2026 Mar 11. pii: S2059-7029(25)01924-6. [Epub ahead of print]11(3): 106054
      The treatment landscape of multiple myeloma (MM) has been revolutionized over the past two decades, leading to unprecedented deep and durable responses, prolonged survival, and improved quality of life. Nonetheless, MM is still an incurable disease, and many patients, especially those with high-risk cytogenetics, renal impairment, or early drug resistance, continue to face poor outcomes. Selinexor, the first-in-class, orally bioavailable selective inhibitor of exportin 1 (XPO1), has shown encouraging results in combination with other agents, and selinexor-based therapy has been approved for the treatment of relapsed/refractory MM, with selinexor-bortezomib-dexamethasone approved for patients with at least one prior line of therapy and selinexor-dexamethasone approved in the later-relapse setting. Notably, selinexor-based combinations have demonstrated consistent efficacy across different subgroups of patients, including those with triple-class refractory disease, renal dysfunction, high-risk cytogenetics, and prior anti-CD38 therapy. We herein provide an overview of selinexor, by describing its mechanism of action, potential interaction with other classes of drugs, novel combinations under investigation, and its role in treatment sequencing, by discussing latest results and potential strategies to mitigate toxicities, increase efficacy, and implement treatment adherence in MM. Overall, selinexor continues to represent a valuable option, especially for patients who are ineligible to receive T-cell-redirecting therapies, or difficult-to-treat patient subgroups, where alternative strategies remain limited. Meanwhile, further data on the use of selinexor-based combinations in different settings are eagerly awaited, to help clarify its role and address persistent unmet clinical needs.
    Keywords:  exportin 1 inhibition; multiple myeloma; relapsed/refractory multiple myeloma; selinexor; selinexor-based therapies
    DOI:  https://doi.org/10.1016/j.esmoop.2025.106054
  16. Ann Hematol. 2026 Mar 12. pii: 172. [Epub ahead of print]105(4):
      
    Keywords:  Light-chain amyloidosis; Plasma cell disorders; Relapse/refractory amyloidosis; T-cell engagers
    DOI:  https://doi.org/10.1007/s00277-026-06931-3
  17. Ann Med. 2026 Dec;58(1): 2624909
       BACKGROUND: Histologic transformation from Waldenström macroglobulinemia (WM) to diffuse large B-cell lymphoma (DLBCL) is a rare but clinically challenging event.
    METHODS: In this retrospective study, we analyzed 15 cases of histologic transformation among WM patients treated at the Department of Hematology, Jiangsu Province Hospital, between October 2015 and February 2025.
    RESULTS: The median age at transformation was 67 years, with a median time from initial WM diagnosis to transformation of 8 months (range: 0-177 months). Six patients (40%) received no WM-directed therapy before transformation. At transformation, 13 patients (86.7%) had stage IV disease. Extranodal involvement was frequent: 6 patients (40%) had ≥2 extranodal sites involved, with the most common sites being bone/bone marrow (each 33.3%), central nervous system (CNS, 20.0%), and nasopharynx/testis/gastrointestinal tract/peritoneum/skin (each 13.3%). Involvement of immune-privileged sites (CNS, testis) was observed in 5 patients (33.3%). Immunophenotyping revealed 13 cases (86.7%) as non-germinal center B-cell (non-GCB) DLBCL. Prognostic analysis showed a median overall survival (OS) of 26.0 months from transformation. Patients receiving Bruton's tyrosine kinase inhibitor (BTKi)-based regimens after transformation showed significantly prolonged OS (p = 0.007). Additionally, patients receiving BTKi-based therapy at any point showed a trend toward improved survival (p = 0.092).
    CONCLUSIONS: Although rare, histologic transformation from WM to DLBCL exhibits aggressive clinical behavior, frequent extranodal involvement, and poor prognosis. BTKi-based regimens may provide significant survival benefits in this patient population.
    Keywords:  BTK inhibitor; Waldenström macroglobulinemia; diffuse large B-cell lymphoma; histologic transformation
    DOI:  https://doi.org/10.1080/07853890.2026.2624909
  18. Toxicol Appl Pharmacol. 2026 Mar 11. pii: S0041-008X(26)00091-8. [Epub ahead of print] 117795
      Chronic myeloid leukemia (CML) cells exhibit a distinct reliance on oxidative phosphorylation (OXPHOS), presenting a metabolic vulnerability for therapeutic intervention. While Hyperoside, a natural flavonol, demonstrates anti-leukemic activity, its precise molecular targets and impact on mitochondrial bioenergetics remain elusive. In this study, we combined in silico modeling and biophysical validation to identify NADPH oxidase 4 (NOX4) as a direct molecular target of Hyperoside. Surface plasmon resonance and cellular thermal shift assays confirmed high-affinity binding between Hyperoside and NOX4. Hyperoside promotes the proteasomal degradation of NOX4 by enhancing its interaction with the E3 ubiquitin ligase STUB1. This Hyperoside-induced recruitment of STUB1 accelerates NOX4 ubiquitination and turnover in K562 and Meg-01 cells. Functionally, Hyperoside treatment resulted in a severe impairment of mitochondrial respiration, including reduced oxygen consumption rates, diminished ATP production, and inhibitory phosphorylation of the pyruvate dehydrogenase complex. Importantly, these metabolic defects were reversed by NOX4 overexpression or STUB1 knockdown. In summary, our findings characterize a novel STUB1/NOX4 signaling axis and establish Hyperoside as a targeted metabolic modulator that dismantles the bioenergetic machinery essential for CML cell survival.
    Keywords:  Chronic myeloid leukemia; Hyperoside; Mitochondrial respiration; NOX4; STUB1
    DOI:  https://doi.org/10.1016/j.taap.2026.117795
  19. Actas Dermosifiliogr. 2026 Mar 09. pii: S0001-7310(26)00016-5. [Epub ahead of print] 104606
      
    DOI:  https://doi.org/10.1016/j.ad.2026.104606