bims-hemali Biomed News
on Hematologic malignancies
Issue of 2026–07–26
twenty-six papers selected by
Alexandros Alexandropoulos, Γενικό Νοσοκομείο Αθηνών Λαϊκό



  1. Blood Cancer J. 2026 Jul 18.
      The introduction of venetoclax (a BCL2 inhibitor) and targeted therapies, including inhibitors of CD33, FLT3, IDH1, IDH2, and menin, has expanded treatment options for newly diagnosed acute myeloid leukemia (AML). In younger, fit patients, the primary goal remains long-term survival, which in most cases is secured through allogeneic stem cell transplant. Transplant in first complete remission is recommended for FLT-ITD, TP53 mutated, KMT2A rearranged, AML with other adverse genetic abnormalities, and is considered in most intermediate-risk patients. It is also recommended in relapsed/refractory disease or persistent measurable residual disease (MRD). The role of intensive chemotherapy, such as cytarabine (7) plus anthracycline (3), is limited to patients with core-binding factor AML, NPM1 mutation, CEBPA bZIP mutation and those with intermediate-risk disease. Intensive regimens such as FLAG-IDA and CLIA plus venetoclax have shown impressive long-term outcomes, but their use is not widespread. In FLT3 mutated AML, 7 + 3 plus an FLT3 inhibitor (midostaurin or quizartinib) remains a standard, with venetoclax-hypomethylating agent-FLT3 inhibitor triplets emerging as an alternative. Similarly, in IDH1/2 mutated AML, venetoclax-hypomethylating agent with or without IDH1/2 inhibitor combinations challenge intensive chemotherapy approaches. Patients with TP53 mutations or other adverse-risk features, where intensive chemotherapy is known to be less effective, should be referred for clinical trials. There remains ongoing debate regarding optimal management of fit patients with newly diagnosed AML without targetable mutations, as emerging data suggest that venetoclax- hypomethylating agents may be comparable to intensive chemotherapy in selected patients proceeding to transplant. Accordingly, treatment decisions should be individualized to maximize remission while minimizing toxicity.
    DOI:  https://doi.org/10.1038/s41408-026-01578-9
  2. Blood Cancer J. 2026 Jul 18.
      Quadruplets incorporating anti-CD38 monoclonal antibody have led to improved outcomes, potentially allowing limited-duration therapy and reduction in steroid doses. We designed this trial to examine the efficacy of the quadruplet regimen containing daratumumab, ixazomib, lenalidomide, and dexamethasone (Dara-IRd) given for fixed duration and to evaluate early discontinuation of steroids. Patients with untreated MM were enrolled, irrespective of their transplant eligibility. The primary objective was to determine the ≥complete response (CR) rate. Treatment involved 28-day cycles of ixazomib 4 mg days 1, 8, 15; lenalidomide 25 mg days 1-21, dexamethasone 40 mg, weekly and daratumumab 16 mg/kg, weekly for two cycles, every other week during cycles 3-6 and every 4-weeks thereafter during induction (12-cycles) followed by daratumumab and ixazomib maintenance (24-cycles). Seventy-eight patients were enrolled into two sequential cohorts: cohort A (n = 38) and B (N = 40), with dexamethasone given only for two cycles in Cohort B. At the time of the data cut-off, all patients had completed the treatment. The overall response rate was 96%, including a ≥CR rate of 32%, similar in both cohorts. Responses deepened over time; 32% achieved a marrow MRD negative status and 29% MRDneg-CR. After a median follow-up of 37.9 months, the median PFS or OS has not been reached. A grade ≥3 adverse event, at least possibly attributed to the study drugs, was seen in 55% of patients. The most common toxicities included fatigue, neutropenia, lymphopenia, peripheral neuropathy, diarrhea, and nausea. Stem cells were collected in 55 patients; the median sCD34+ cell yield (range) was 7.8 (2.8-15.9) × 106/kg. Dara-IRd is an active regimen in newly diagnosed MM, with high overall response rate as well as deep responses. Nearly a third of the patients attained MRDneg status, which improved over time. The PFS with finite-duration therapy is comparable to other studies in NDMM. Early discontinuation of dexamethasone did not impact efficacy.
    DOI:  https://doi.org/10.1038/s41408-026-01567-y
  3. Br J Haematol. 2026 Jul 21.
      While minimal residual disease (MRD) is a well-recognized prognostic marker in multiple myeloma (MM), the clinical significance of dynamic MRD trajectories and MRD-driven therapies remains incompletely elucidated. This retrospective study analysed serial MRD results from 231 newly diagnosed multiple myeloma (NDMM) patients who achieved ≥ complete remission (CR). MRD negativity independently predicted superior progression-free survival (PFS) (hazard ratio [HR] = 0.222; 95% confidence interval [CI] 0.124-0.400, p < 0.001) and overall survival (OS) (HR = 0.280; 95% CI 0.097-0.809, p = 0.019). Sustaining MRD negativity for ≥6 months improved OS (p < 0.001), while negativity duration ≥12 months conferred dual prognostic benefits for both PFS and OS (both p < 0.001). Patients with late MRD conversion (after ≥12 months of negativity) had OS comparable to those with sustained negativity (p = 0.339). Elevated circulating plasma cell at diagnosis (HR = 3.761; 95% CI 1.225-11.550, p = 0.021), failure to achieve MRD negativity post-induction (HR = 2.886; 95% CI 1.065-7.819, p = 0.037), and a high abnormal PC/BMPC% at MRD conversion (HR = 4.091; 95% CI 1.429-11.701, p = 0.009) were independent risk factors for progression post-conversion. These findings demonstrate the prognostic significance of sustained MRD negativity and support serial MRD monitoring as a potential basis for individualized MRD-driven treatment strategies in NDMM.
    Keywords:  dynamic changes; minimal residual disease; multiple myeloma; prognosis
    DOI:  https://doi.org/10.1111/bjh.70704
  4. Leukemia. 2026 Jul 24.
      The primary analysis of the phase 2 OPTIC trial (NCT02467270) demonstrated optimal benefit:risk with response-based ponatinib dosing (45 mg once daily (QD) reduced to 15 mg QD) upon achieving ≤1% BCR::ABL1IS in patients with tyrosine kinase inhibitor-resistant or T315I-positive chronic-phase chronic myeloid leukemia (CP-CML). Here, we report 5-year long-term outcomes. Overall, 283 patients were randomized to 45-mg, 30-mg, or 15-mg QD starting doses (n = 94, 95, and 94, respectively), with dose reduction to 15 mg QD upon response in the 45-mg and 30-mg cohorts. At data cutoff, 61 patients remained on trial. Median follow-up time was 75-78 months. By 5 years, 60%, 41%, and 40% of patients in the 45-mg, 30-mg, and 15-mg cohorts, respectively, achieved ≤1% BCR::ABL1IS. Five-year progression-free survival rates were 63%, 57%, and 60%, respectively, by cohort; overall survival rates exceeded 80%. In patients with a T315I mutation, 5-year rates of ≤1% BCR::ABL1IS, PFS, and OS were highest in the 45-mg cohort. Exposure-adjusted rates of adjudicated arterial occlusive events were 4.1, 3.8, and 2.0 patients per 100 patient-years, respectively, by cohort; results were comparable in T315I-positive patients. These findings support long-term clinical benefit of response-based ponatinib dosing in third-line CP-CML, especially in patients with the T315I mutation.
    DOI:  https://doi.org/10.1038/s41375-026-03009-x
  5. Adv Ther. 2026 Jul 24.
       INTRODUCTION: Rocbrutinib is a fourth-generation Bruton's tyrosine kinase (BTK) inhibitor that binds covalently to the wild type BTK and non-covalently to the cysteine 481 mutant variant. This phase I study evaluated the pharmacokinetics (PK), safety, and efficacy of rocbrutinib in Chinese patients with relapsed or refractory (R/R) non-germinal center B cell-like (non-GCB) diffuse large B cell lymphoma (DLBCL).
    METHODS: Eligible participants were adults with non-GCB DLBCL and had received ≥ 2 prior lines of systemic therapy, including an anti-CD20 antibody-based regimen. Patients received rocbrutinib at 100, 150, 200 or 300 mg once daily (QD) as single agent till disease progression or unacceptable toxicity. Response was evaluated using computerized tomography (CT) and positron emission tomography (PET)-CT at the protocol-defined timepoints by the investigators. PK samples and adverse events were collected per protocol.
    RESULTS: A total of 45 patients were enrolled, of whom 44.4% had received ≥ 3 lines of therapy. The overall response rate (ORR) was 57.8% [95% confidence interval (CI): 42.2, 72.3], and the complete response (CR) rate was 33.3% (95% CI 20.0, 49.0). In the ≥ 200 mg QD cohort (n = 18), the ORR was 72.2% (95% CI 46.5, 90.3) and the CR rate was 44.4% (95% CI 21.5, 69.2). Treatment-emergent adverse events (TEAEs) were reported in 97.8% patients, ≥ grade 3 TEAEs in 51.1% patients. Due to TEAEs, 2 patients (4.4%) with dose reduction and 1 (2.2%) discontinued study treatment, respectively. No atrial fibrillation/flutter was reported. In the ≥ 200 mg QD cohort, the safety profile was consistent with the overall population.
    CONCLUSION: Rocbrutinib demonstrated promising efficacy and favorable tolerability profile in heavily pre-treated R/R non-GCB DLBCL. A 200 mg QD dose was selected as recommended phase 2 dose for monotherapy. A randomized phase II clinical trial in this population is currently enrolling.
    TRIAL REGISTRATION: ClinicalTrials.gov identifier, NCT04993690.
    Keywords:  BTK inhibitor; Diffuse large B cell lymphoma; Efficacy; Non-covalent and covalent; Pharmacokinetics; Phase I clinical trial; Rocbrutinib; Safety
    DOI:  https://doi.org/10.1007/s12325-026-03711-3
  6. Hematol Oncol. 2026 Jul;44(4): e70224
      The molecular subtype characterized by co-occurring MYD88 and CD79B alterations (MCD) represents a biologically distinct subset of diffuse large B-cell lymphoma (DLBCL) with chronic active B-cell receptor signaling and a high risk of central nervous system (CNS) involvement. The clinical impact of Bruton tyrosine kinase inhibitors (BTKi) in this subtype remains unclear. We retrospectively analyzed 155 patients with newly diagnosed DLBCL harboring genetic features consistent with the MCD subtype. At a median follow-up of 34.1 months, the estimated 3-year progression-free survival (PFS) rate was 76.1%. BTKi exposure (n = 56) was associated with significantly improved PFS compared with no BTKi exposure (3-year PFS: 93.8% vs. 66.6%, p < 0.001) and remained independently associated with improved PFS after adjustment for IPI risk (HR 0.16, p < 0.001). Overall survival did not differ significantly between groups. Notably, all 15 CNS relapse events occurred in patients who did not receive BTKi, whereas no CNS relapse was observed in the BTKi-treated group. BTKi exposure was independently associated with a markedly reduced risk of CNS relapse (HR 0.06, p = 0.002) after adjustment for CNS-IPI risk and CNS prophylaxis. These findings suggest that BTK inhibition may improve outcomes and mitigate CNS relapse in MCD DLBCL.
    Keywords:  BCR signaling; BTK inhibitor introduction; CNS relapse; diffuse large B‐cell lymphoma; survival
    DOI:  https://doi.org/10.1002/hon.70224
  7. Blood Adv. 2026 Jul 21. pii: bloodadvances.2025018580. [Epub ahead of print]
      Double-hit or triple-hit lymphoma (DH/THL) is an aggressive subtype with poor prognosis. This study evaluated the efficacy and safety of selinexor, a first-in-class oral inhibitor of exportin 1 (XPO1), combined with R-CHOP (S-RCHOP) as first-line therapy for newly diagnosed DH/THL. This single-arm, prospective phase Ⅱ trial (NCT05974085) enrolled 13 patients between May 2022 and August 2024. Patients received up to six 21-day cycles of S-RCHOP (selinexor 60 mg on days 1, 8, 15). The primary endpoint was overall response rate (ORR). Secondary endpoints included progression free survival (PFS), overall survival (OS), central nervous system (CNS) relapse rate within 2 years, and adverse events (AEs). Exploratory analyses included next-generation sequencing (NGS) and circulating cell-free DNA (cfDNA) monitoring. 13 patients (9 DHL, 4 THL) were enrolled. The ORR was 100% (CR: 76.9%, PR: 23.1%). At a median follow-up of 25.4 months, the 2-year PFS and OS were 67.7% and 67.1%, respectively. One patient developed CNS relapse. The most common all-grade AEs included leukopenia/neutropenia (55.1% each), febrile neutropenia and fatigue (43.5% each), and thrombocytopenia (33.7%). NGS and cfDNA revealed frequent BCL6 and IGLL5 mutations. Post-treatment cfDNA negativity was achieved in 72.7% of patients, all in CR by PET-CT. In this exploratory phase Ⅱ study, S-RCHOP achieved high response rates in newly diagnosed DH/THL but was associated with frequent hematologic AEs. cfDNA monitoring provided valuable insights into treatment response. These preliminary findings support further investigation of XPO-1 inhibition with R-CHOP, with priority given to dose optimization. NCT05974085.
    DOI:  https://doi.org/10.1182/bloodadvances.2025018580
  8. Blood Adv. 2026 Jul 24. pii: bloodadvances.2026020789. [Epub ahead of print]
      Venetoclax was first approved for continuous use in relapsed-refractory chronic lymphocytic leukemia (RR CLL), but the efficacy and consequences of very long term BCL2 inhibition are unknown. We describe the frequency, characteristics and outcomes of patients with RR CLL treated with >5 years of continuous venetoclax. Long-term responders (>5 years of continuous therapy without progressive disease [PD]), were identified from a cohort of 86 RR CLL patients treated with continuous venetoclax± rituximab. Landmark analyses at 2 and 5 years assessed association between undetectable measurable residual disease (uMRD; 10-4 peripheral blood flow cytometry) and progression free survival (PFS). Next generation sequencing (NGS) was performed at PD for BCL2 and TP53 mutations. Twenty-nine (33%) patients were long-term responders. Compared to those with PD within 5 years, they were more likely to have mutated IGHV(44% vs. 14%, p =0.029), non-complex-karyotype (89 vs. 50%, p=0.043) and uMRD (79 vs. 30%, p<0.001). At median follow-up of 11.1 years, 76% had ceased venetoclax, mostly due to PD. In a landmark analysis of patients continuing venetoclax beyond 2 years, 5-year PFS was 87% for those with uMRD vs. 45% without (HR1.30, 95% CI 1.12-1.52, p=0.001). BCL2 mutations were detected in 4/8 patients at PD. Grade ≥3 toxicities - including infection (51%), neutropenia (20%) and thrombocytopenia (10%)- occurred at consistent frequency throughout treatment. Long-term responses to continuous venetoclax occur in approximately one third of RR CLL patients. Sustained uMRD confers the most favorable outcome, though all patients show a continuous risk of relapse, infection and cytopenias.
    DOI:  https://doi.org/10.1182/bloodadvances.2026020789
  9. Blood Adv. 2026 Jul 20. pii: bloodadvances.2026020463. [Epub ahead of print]
      
    DOI:  https://doi.org/10.1182/bloodadvances.2026020463
  10. Ann Med. 2026 Dec;58(1): 2697132
       BACKGROUND: CD5-positive diffuse large B-cell lymphoma (DLBCL) is an aggressive subtype with poor outcomes. Following the approval of Pola-R-CHP in China, this study aimed to evaluate its efficacy as initial therapy for this high-risk population.
    METHODS: We conducted a multicenter, retrospective study of previously untreated CD5-positive DLBCL patients who received Pola-R-CHP as first-line therapy between April 2023 and February 2025. Treatment response was assessed by PET/CT after 3 cycles and at the end of treatment.
    RESULTS: Among 32 enrolled patients (median age 63.5 years), 78.1% had stage III-IV disease and 59.4% had an International Prognostic Index score ≥3. The overall response rate (ORR) was 100% after 3 cycles, with a complete response rate (CRR) of 71.9%. At end of treatment, the ORR and CRR were 93.8% and 75%, respectively. After a median follow-up of 17.9 months, 2-year progression-free survival (PFS) and overall survival rates (OS) were 70% and 82.7%. After adjusting covariates, failure to achieve a complete response (CR) after 3 cycles was a predictor of inferior survival [PFS HR: 18.1, p = 0.02; OS HR: 12.3, p = 0.033]. Grade 3-4 adverse events occurred in 31.3% of patients, with neutropenia being most common.
    CONCLUSIONS: CD5-positive DLBCL patients exhibited rapid and promising responses to first-line Pola-R-CHP with an acceptable safety profile. Interim response assessment is a potential prognostic factor. In instances where CR is not attained by the interim PET/CT, it is advisable to consider treatment modification, as this may indicate a potential failure to achieve CR.
    Keywords:  CD5-positive DLBCL; Pola-R-CHP; early therapeutic adjustment; interim partial response; progression-free survival
    DOI:  https://doi.org/10.1080/07853890.2026.2697132
  11. Acta Haematol. 2026 Jul 23. 1
       INTRODUCTION: Treatment-free remission (TFR) is an established goal for patients with chronic myeloid leukemia in chronic phase (CML-CP) who achieve sustained deep molecular response (DMR) on tyrosine kinase inhibitor (TKI) therapy. However, the relationship between pre-discontinuation dose intensity and TFR outcome remains poorly characterized.
    METHODS: We retrospectively analyzed 146 patients with CML-CP who attempted TFR at Samsung Medical Center between 2003 and 2025. Dose intensity (DI) was defined as the time-weighted average ratio of actual to standard TKI dose during the 2-year period preceding discontinuation. The association between DI and TFR failure was modeled using Cox regression with restricted cubic splines (RCS) in the DI subcohort (n=126), using a DI of 1.0 as the reference.
    RESULTS: In the full cohort, at median follow-up of 22.3 months, 50 patients (34.2%) experienced molecular relapse. TFR probability was 79.5% at 6 months and 61.9% at 48 months. In the DI subcohort, an unadjusted RCS model suggested a nonlinear, inverted U-shaped pattern, but the overall DI effect was not significant (likelihood ratio P = .101; P for nonlinearity = .045). In exploratory post-hoc groups, intermediate DI showed the highest TFR failure rate (49.0% vs. 25.0% and 28.6%; P = .035). The pattern did not reach significance after adjustment for TKI generation (P for nonlinearity = .074).
    CONCLUSION: These exploratory findings suggest that the biology underlying DMR maintenance, rather than the absolute pre-discontinuation dose, may influence TFR outcome. Given the modest sample size and post-hoc design, this hypothesis-generating observation requires confirmation in independent cohorts.
    DOI:  https://doi.org/10.1159/aha/abkag001
  12. J Clin Oncol. 2026 Jul 22. JCO2600410
      Solitary plasmacytomas (SPs) are rare localized tumors of clonal plasma cells, either in the bone (solitary bone plasmacytoma) or in soft tissue/extraosseous (extramedullary) with either no or with minimal bone marrow (BM) infiltration (<10% clonal plasma cells by immunohistochemistry) and no evidence of systemic involvement or myeloma defining events. Approximately 50% of SPs will progress to symptomatic myeloma within 5 years after initial definitive local radiotherapy. Increased availability of improved diagnostic and monitoring tools has increased the sensitivity of detection of additional lesions and marrow involvement and has implications for the follow-up strategy after treatment. Thus, the definitions and requirements for the diagnosis and follow-up of SPs are evolving. The diagnosis of SP requires the careful exclusion of multiple myeloma (MM) that would require systemic therapy, by using all the available methods to detect systemic disease (advanced imaging, sensitive BM assessment methods, blood and urine tests). Local radiotherapy remains the mainstay of therapy, and despite the availability of innovative drugs for MM, the clinical benefit of systemic therapy currently remains poorly defined. The International Myeloma Working Group provides here updated recommendations for the diagnosis, evaluation, treatment, and response assessment of patients with SPs, incorporating recent data and advances in diagnostic tools.
    DOI:  https://doi.org/10.1200/JCO-26-00410
  13. Cancer. 2026 Aug 01. 132(15): e70533
       BACKGROUND: The approval of the BCR::ABL tyrosine kinase inhibitors (TKIs) and blinatumomab have improved outcomes in Ph-positive B-acute lymphoblastic leukemia (ALL). However, patients still relapse, and their outcomes in the TKI era have yet to be defined.
    METHODS: Patients with relapsed/primary refractory Philadelphia chromosome (pH)-positive B-ALL ≥16 years old treated in first salvage from 1992 to 2025 were analyzed.
    RESULTS: A total of 165 patients (median age, 48; range, 17-78 years) were analyzed. The overall complete remission (CR) rate was 80%. By multivariate analysis, only the use of TKIs was associated with a significant benefit for achieving CR. The median overall survival (OS) was 15 months. The 3-year OS rate was 31%. The 3-year OS rate was 57% with third-generation TKI combinations, 38% with second-generation TKI combinations, 8% with imatinib combinations, and 0% without TKIs. By multivariate analysis, three variables were independently predictive of survival: TKI-based therapy (hazard ratio [HR], 0.10-0.49; p values all <.001 for each TKI vs. no TKI), blinatumomab-based therapy (HR, 0.36; p = .0058), and white blood cell ≥50 × 109/L (HR, 1.96; p = .0076).
    CONCLUSIONS: This study establishes a modern expectation of outcome of Ph-positive B-cell ALL treated in salvage 1. Third-generation TKI plus blinatumomab combinations should be given consideration in this setting.
    Keywords:  ALL salvage therapy; CART; blinatumomab; inotuzumab
    DOI:  https://doi.org/10.1002/cncr.70533
  14. Exp Hematol Oncol. 2026 Jul 20. pii: 63. [Epub ahead of print]15(1):
      Early frontline data presented at the 2025 ASH Annual Meeting suggest that T cell-redirecting strategies, including chimeric antigen receptor (CAR)-T cells and bispecific antibodies (BsAbs), can induce rapid, high rates of minimal residual disease (MRD)-negative responses in newly diagnosed multiple myeloma (NDMM), including transplant-ineligible (TI) and high-risk populations. However, short follow-up, heterogeneous MRD methodologies, and infection risk-particularly with continuous bispecific exposure-underscore that these approaches remain investigational and should be advanced through randomized, MRD-guided fixed-duration trials.
    Keywords:  Bispecific antibody; CAR-T cells; Frontline therapy; Multiple myeloma; T cell-redirecting therapies
    DOI:  https://doi.org/10.1186/s40164-026-00809-w
  15. J Hematol. 2026 Jun;15(3): 162-168
      Chronic eosinophilic leukemia (CEL) is a rare myeloproliferative neoplasm characterized by sustained elevation of eosinophil counts greater than > 1.5 × 109/L in blood or bone marrow. Approximately 25-30% of patients with persistent hypereosinophilia have somatic mutations associated with myeloid neoplasms, and next generation sequencing has led to the use of newer treatments for CEL, including tyrosine kinase inhibitors (TKIs) such as imatinib. Before the advent of imatinib, the disease had a poor prognosis with a 5-year mortality close to 50%. However, in patients with CEL without the characteristic mutations, known as chronic eosinophilic leukemia, not otherwise specified (CEL-NOS), treatment options and guidance are limited. We present a case series of two CEL-NOS patients treated at our academic health sciences center.
    Keywords:  Chronic eosinophilic leukemia; Hypereosinophilia; Myeloproliferative neoplasm
    DOI:  https://doi.org/10.14740/jh2114
  16. Cancer. 2026 Jul 15. 132(14): e70478
       BACKGROUND: Functional high-risk (FHR) multiple myeloma (MM) was historically defined as progression within 18 months of starting therapy, with expected subsequent overall survival (OS) <2 years. The optimal definition of FHR in the era of combined quadruplet therapy (QUAD) + autologous stem cell transplantation (ASCT) is unknown.
    METHODS: The authors analyzed 310 patients with newly diagnosed MM who received QUAD + ASCT and had with a median follow up of 41.8 months, with 66 progression events, to identify the optimal definition of FHR MM and to explore the factors associated with outcomes of subsequent therapy.
    RESULTS: The cumulative incidence of progression with FHR cutoff points of within 12 months (FHR12), 18 months (FHR18), 24 months (FHR24), and 36 months (FHR36) of treatment initiation were 2.6%, 6.2%, 10.1%, and 16.4%, respectively. The median second PFS (2PFS) and OS from the onset of second-line therapy were 3.0 and 8.1 months, respectively, for FHR12; 2.7 and 8.1 months, respectively, for FHR18; 3.3 and 15.7 months, respectively, for FHR24; and 5.8 and 23.8 months, respectively, for FHR36, pointing to 36 months as the optimal cutoff point to define FHR MM. The 12-month 2PFS rate was 80% versus 23% for patients treated with versus without T-cell-redirecting therapy, respectively. In multivariable analysis, T-cell-redirecting therapy was associated with a substantially improved 2PFS even when adjusted for FHR status.
    CONCLUSIONS: In the current era of QUAD + ASCT, the definition of FHR MM should encompass patients who have MM with disease progression in the first 36 months of therapy. This study provides benchmark data for clinical trials deploying agents with novel mechanisms of action in this difficult-to-treat population (ClinicalTrtials.gov identifier NCT03224507).
    Keywords:  CD38 antigen; antibodies; antineoplastics; bispecific; chimeric antigen receptor therapy; monoclonal antibody; multiple myeloma
    DOI:  https://doi.org/10.1002/cncr.70478
  17. Cancer Immunol Immunother. 2026 Jul 20.
      Multiple myeloma (MM), a hematological malignancy, remains an incurable disease due to the development of resistance to the treatment; thus, there is an urgent need for new and effective therapeutic strategies, particularly for patients who do not respond to standard therapies. High levels of Cluster of Differentiation 47 (CD47) expression have been reported in MM and are associated with disease progression. CD47 acts as a cancer immune escape mechanism by binding to SIRPα protein, resulting in inhibiting phagocytosis of macrophages and NK cell activity. Therefore, blocking the CD47 signaling pathway has emerged as a promising strategy for cancer immunotherapy. In this study, we confirmed that MM cells have high CD47 expression. We generated and characterized a tri-specific killer engager targeting CD47, namely TriKE-CD47, that targets both CD47 on MM cells and CD16 on NK cells. Additionally, it incorporates an IL-15 moiety to enhance NK cell proliferation. TriKE-CD47 treatment promoted a remarkable proliferation of NK cells overexpressing CD16 (N6 cells). Co-culturing MM cells with N6 cells, primary NK cells, and monocyte-derived macrophages in the presence of 200 ng of TriKE-CD47 significantly improved NK cytotoxicity and macrophage phagocyte activities against MM cells. Notably, the efficacy of TriKE-CD47 was directly correlated with CD47 expression levels on the target cells reflecting the specificity of TriKE-CD47 to target antigen. Furthermore, TriKE-CD47 effectively suppressed tumor growth in MM xenograft mice models. Taken together, these findings strongly supported that TriKE-CD47 could be a potential therapeutic for MM patients.
    Keywords:  Biologics; Cluster of Differentiation 47 (CD47); Immunotherapy; Multiple myeloma; Natural killer cell; Tri-specific killer engager (TriKE)
    DOI:  https://doi.org/10.1007/s00262-026-04431-x
  18. Oncology. 2026 Jul 21. 1-19
       BACKGROUND: Classical Philadelphia-negative myeloproliferative neoplasms (MPNs), including polycythaemia vera, essential thrombocythaemia, and myelofibrosis, frequently occur in patients with renal, hepatic, cardiovascular, or hematologic comorbidities. These vulnerabilities complicate treatment selection, dose adjustment, and toxicity monitoring. However, patients with advanced kidney disease, dialysis dependence, decompensated cirrhosis, severe cytopenias, or overlapping organ impairment are often underrepresented in prospective trials.
    SUMMARY: This narrative review presents a practical, organ-adapted framework for selecting MPN therapy in clinically complex patients. We define renal and hepatic impairment, compare commonly used cytoreductive and targeted therapies-including hydroxyurea, busulfan, anagrelide, ruxolitinib, fedratinib, momelotinib, pacritinib, ropeginterferon alfa-2b, and pegylated interferon alfa-2a-and discuss their use in dialysis, decompensated liver disease, cytopenic myelofibrosis, anemia-dominant myelofibrosis, and combined organ vulnerability. The proposed approach prioritizes label-based dose modification when available, cautious dose initiation when evidence is extrapolated, structured early monitoring, and multidisciplinary review for high-risk patients.
    KEY MESSAGES: Management of Philadelphia-negative myeloproliferative neoplasms (MPNs) in organ dysfunction should be individualized according to disease phenotype, treatment goal, organ reserve, hematologic tolerance, frailty, and label-specific safety restrictions. In renal impairment, hydroxyurea and ruxolitinib may remain feasible in selected patients when dose-adjusted and monitored closely, whereas dialysis and severe renal impairment require conservative initiation and multidisciplinary review because direct evidence remains limited. In myelofibrosis, JAK inhibitor choice should be phenotype-adapted: ruxolitinib remains a common standard when blood counts and organ function permit, momelotinib is attractive in anemia-dominant disease, pacritinib is particularly relevant in severe thrombocytopenia, and fedratinib is useful in selected settings but requires thiamine-focused safety management. In hepatic dysfunction or overlapping organ vulnerability, therapy should prioritize safety, reversible contributors, conservative starting strategies, explicit stopping rules, and early reassessment.
    DOI:  https://doi.org/10.1159/000553574
  19. Clin Cancer Res. 2026 Jul 22.
       PURPOSE: This report presents long-term outcomes of third-generation anti-CD30 CAR T-cell therapy in relapsed/refractory (r/r) CD30+ lymphoma patients.
    PATIENTS AND METHODS: In this single-arm, multicentre, phase 1/2 trial, patients received lymphodepletion regimen comprising fludarabine and cyclophosphamide, followed by infusion of anti-CD30 CAR-T cells. Primary endpoints included safety and overall response rate (ORR), while secondary endpoints were progression-free survival (PFS) and overall survival (OS).
    RESULTS: Forty-four patients were enrolled, including 33 cases of Hodgkin lymphoma. Of 44 patients, 23 achieved complete response (CR) to CAR-T, and 19 achieved PR, resulting in a CR rate of 52.3% and an ORR of 95.5%. The most frequent toxicities were hematologic AEs of grade 3 or higher (68.2% of neutropenia). Cytokine release syndrome occurred in 18 patients (40.9%), with two cases (4.5%) being ≥ grade 3. In the follow-up period, 24 patients underwent auto-HSCT after CAR-T within a median of 3 months. The best ORR was 95.5%, with 27 patients (61.4%) achieving CR. The best CR rate was higher in patients receiving CAR-T followed by auto-HSCT compared to those receiving CAR-T alone (75% vs. 45%). 3-year OS and PFS rates for all patients were 79.0% (95%CI, 66.1%-91.9%) and 74.2% (95%CI, 60.3% -88.1%). Patients receiving consolidated auto-HSCT following CAR-T exhibited significantly longer OS and PFS compared to those treated with CAR-T alone.
    CONCLUSION: Third-generation anti-CD30 CAR-T demonstrates high efficacy and a favorable safety profile in r/r CD30+ lymphoma patients. Addition of auto-HSCT following CAR T-cell therapy improves depth of remission and potentially enhances OS and PFS.
    DOI:  https://doi.org/10.1158/1078-0432.CCR-26-1292
  20. Eur J Haematol. 2026 Jul 22.
       BACKGROUND: The 2022 International Consensus Classification (ICC) introduced myelodysplastic syndrome/acute myeloid leukemia (MDS/AML) as a distinct entity for patients with 10%-19% marrow blasts lacking AML-defining genetic abnormalities. The comparative prognostic utility of the Molecular International Prognostic Scoring System (IPSS-M), derived from MDS, and the European LeukemiaNet 2022/2024 (ELN 2022/2024) classification, designed for AML, within this overlap syndrome remain undefined.
    METHODS: We retrospectively analyzed 110 adults with ICC-defined MDS/AML treated at West China Hospital (2019-2024). Baseline risk was assessed using IPSS-M and ELN 2022. Treatment-matched exploratory analyses compared IPSS-M with ELN 2022 in intensively treated patients and with the 2024 ELN Less-Intensive genetic-risk classification in patients receiving HMA monotherapy or HMA plus venetoclax. Prognostic discrimination was assessed using the time-dependent area under the curve (AUC) and category-based Harrell's concordance index.
    RESULTS: Most patients were at high risk according to the IPSS-M (high/very high, 90.0%) and ELN 2022 (adverse, 79.1%). During a median follow-up of 36 months, the median OS was 25 months. In the full cohort, IPSS-M showed better discrimination than ELN 2022 did (C-index, 0.578 vs. 0.518). In the primary HMA-based cohort (n = 62), ELN 2024 classified 42 patients as favorable, 7 as intermediate, and 13 as adverse; the median OS was 33, 18, and 12 months, respectively (log-rank p = 0.005). ELN 2024 showed greater discrimination than IPSS-M did (C-index, 0.605 vs. 0.562), but the difference was not significant. Among the 91 response-evaluable patients, the highest observed ORR (83.3%) and CR rate (54.2%) were obtained for HMA plus venetoclax.
    CONCLUSION: IPSS-M provided better overall discrimination than ELN 2022 did in this MDS/AML cohort, whereas the 2024 ELN Less-Intensive classification produced clinically relevant risk separation and numerically higher discrimination in HMA-treated patients. Model performance was treatment context dependent, and subgroup results require validation in larger prospective cohorts.
    Keywords:  ELN 2022; ELN 2024; IPSS‐M; MDS/AML; hematopoietic stem cell transplantation; prognosis
    DOI:  https://doi.org/10.1111/ejh.70273
  21. Neoplasma. 2026 Jul 22. pii: 260404N105. [Epub ahead of print]
      Until 2020, patients with acute myeloid leukemia (AML) who were not suitable for intensive treatment were mostly limited to symptomatic and palliative care, or to low-intensity regimens including low-dose cytosine-arabinoside (ARA-C) and azacitidine (AZA) monotherapy, which didn't bring much benefit. The situation changed with the arrival of venetoclax, a Bcl-2 inhibitor that causes leukemic cells to rapidly undergo apoptosis. The aim of our retrospective study was to summarize the treatment outcomes of all newly diagnosed patients with AML, unsuitable for intensive chemotherapy, treated with a combination of venetoclax and AZA at the Department of Hematology and Transfusion Medicine, University Hospital in Bratislava from January 1, 2021, to December 31, 2025. A total of 108 patients underwent treatment with a median follow-up of 35.9 months; median age was 69.5 years (47-84 years) and median number of cycles 3 (1-34). Induction mortality rate was 7.4%, and tumor lysis syndrome occurred in 4.5%. The overall response rate, which included the number of complete remissions, complete remissions with incomplete hematopoietic recovery, and morphologically leukemia-free status (CR/CRi/MLFS), was 64%, the median overall survival was 9 months, and disease-free survival was 8 months. As of December 31, 2025, 23 patients are alive and 85 have died. The main cause of death was the progression of the disease. The main contribution of our study is the finding that shortening the duration of venetoclax treatment maintains efficacy. There was no significant difference in remission rate achieved or in overall survival based on the number of days of venetoclax administration (< 14 vs. 14 vs. 21 vs. 28 days). The combination of AZA and venetoclax in AML has proven to be highly effective in inducing complete remission, usually immediately after the first cycle. Unfortunately, remission is not long-lasting, relapses are frequent, and the median overall survival in real-world data is 7.9 to 13.6 months.
    DOI:  https://doi.org/10.4149/neo_2026_260404N105
  22. Eur J Haematol. 2026 Jul 23.
       INTRODUCTION: Thrombosis and bleeding events are major causes of morbidity and mortality in patients with myeloproliferative neoplasms (MPN). The contribution of non-driver mutations, in particular mutations in DNMT3A, TET2, and ASXL1 (collectively DTA) to thrombo-hemorrhagic risk remains unclear.
    METHODS: We conducted a retrospective analysis of 180 patients with MPN undergoing next generation sequencing to evaluate the association between DTA mutations and major bleeding and thrombotic events occurring from 3 years prior to diagnosis through follow-up. Patients were stratified by disease subtype, and Cox proportional hazards models were used for univariate and multivariate analyses.
    RESULTS: DTA mutations were present in 40% of the patients. 52 (29%) patients suffered from arterial or venous thrombotic events either following diagnosis or as early as 3 years prior to diagnosis, and 20 (11%) experienced major bleeding events during the same period. In patients with PV and ET, DNMT3A and TET2 (DT) mutations were significantly associated with venous thrombosis (HR 5.6, 95% CI 1.6-19.1) and were also associated with major bleeding in univariate analyses (HR 6.7, 95% CI 1.7-26.9). The association with venous thrombosis remained significant after multivariable adjustment, whereas the association with major bleeding was attenuated after adjustment for age and smoking status (HR 4.1, 95% CI 0.9-19.2). No such associations were observed in patients with myelofibrosis.
    CONCLUSION: Our findings highlight an association between DT mutations and thrombotic and bleeding complications in patients with PV and ET and support further evaluation of molecular markers as contributors to thrombo-hemorrhagic risk in MPN.
    Keywords:  DNMT3A; TET2; bleeding; clonal hematopoiesis; myeloproliferative disorders; thrombosis
    DOI:  https://doi.org/10.1111/ejh.70271
  23. Hematol Rep. 2026 Jul 02. pii: 48. [Epub ahead of print]18(4):
      Anemia of chronic disease (ACD) is a condition linked to chronic immune activation secondary to a wide range of infectious, inflammatory, and autoimmune diseases. It is characterized by a state of iron-restricted erythropoiesis, in which prolonged activation of cytokines leads to retention of iron within the reticulo-endothelial system, driven primarily by hepcidin. Reduced iron availability contributes to a blunted response by erythropoietin and impaired erythropoiesis, in addition to a shortened red cell lifespan. In patients found to have anemia and evidence of chronic inflammation, parameters such as mean cell volume, iron studies, percentage of hypochromic red cells, reticulocyte hemoglobin content, and levels of ferritin, serum transferrin receptor, hepcidin, erythropoietin, and GDF15 are all used to build a picture of anemia of chronic disease. Following this, management normally utilizes erythropoietin-stimulating agents alongside parenteral iron supplementation when treatment of the underlying cause is not available. Newer therapies, such as hypoxia-inducible factor prolyl hydroxylase inhibitors and hepcidin inhibitors, also play a role, while cytokine targets, carbon dots, androgens, and other therapies are emerging as possible treatment routes. Despite its high prevalence, there remain few standardized methods of diagnosis or management in anemia of chronic disease. This narrative review explores long-standing and emerging practices in the diagnosis and management of this condition to ensure an up-to-date understanding.
    Keywords:  anemia of chronic disease; functional iron deficiency; inflammation
    DOI:  https://doi.org/10.3390/hematolrep18040048