Leukemia
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Jul 20 – Jul 27, 2026
CD20 expression dynamics in adult B-cell acute lymphoblastic leukemia and impact on anti-CD20 treatment.
J HEMATOL ONCOL · Q1 JOURNAL - RANK #1/98TOP-TIER
This prospective GMALL 08/2013 trial evaluated the clinical impact of baseline CD20 expression levels on B-cell acute lymphoblastic leukemia (B-ALL) and explored rituximab’s effect on treatment outcomes. Patients received a cyclophosphamide/dexamethasone prephase, induction, and consolidation therapy with four rituximab doses in BCR::ABL1-negative cases, and CD20 expression was measured repeatedly in marrow and blood samples. Higher CD20 expression correlated with improved early MRD responses and higher molecular complete remission rates (30.5% vs 10.6% after Induction I; 72.6% vs 50.0% after Consolidation I). The authors conclude that reassessing CD20 expression post-prephase identifies additional patients for rituximab therapy, potentially improving outcomes.
10.1186/s13045-026-01832-4
Jul 13 – Jul 20, 2026
Toxicity during induction of pulsed versus continuous prednisolone in children with acute lymphoblastic leukaemia: a multi-centre, open label, randomised, phase 3 trial from India (2016-2022).
LANCET REG HLTH-SE A · Q1 JOURNAL - RANK #10/185
This open-label, multicentre, randomised phase 3 trial in India compared continuous versus pulsed prednisolone during induction therapy for children ≤10 years with newly diagnosed standard- or intermediate-risk B-cell precursor acute lymphoblastic leukaemia. 1,246 eligible patients were randomised 1:1 to continuous 60 mg/m²/day for 4 weeks with taper (R1A) or pulsed dosing on days 1–14 and 22–28 (R1B); primary endpoint was grade 3-5 toxicity, with CR, MRD, 3-year event-free survival (EFS) and overall survival (OS) as secondary endpoints. Induction deaths were significantly lower with pulsed dosing (1.3% vs 3.5%; absolute risk difference 2.3%, p=0.0149; HR 3.06), while grade 3-5 toxicities were comparable (45.4% vs 46.4%); CR rates (98.0-98.8%), MRD ≥0.01% (26-28%), 3-year EFS (72.2-72.7%) and OS (85-87%) did not differ. Authors conclude that pulsed prednisolone reduces treatment-related mortality without impairing remission or survival, and that anthracycline use independently elevates mortality and toxicity risks.
10.1016/j.lansea.2026.100788
Jul 06 – Jul 13, 2026
Venetoclax in combination with cytarabine with or without idarubicin or azacitidine in children, adolescents, and young adults with relapsed or refractory acute myeloid leukaemia (VENAML): a multicentre, phase 1 expansion study.
LANCET HAEMATOL · Q1 JOURNAL - RANK #3/98TOP-TIER
This multicenter, phase 1 study evaluated the efficacy and safety of venetoclax combined with cytarabine ± idarubicin or azacitidine in pediatric patients (ages 2–24) with relapsed/refractory acute myeloid leukemia. The expansion cohort phase results included 44 patients treated at the recommended phase 2 dose (RP2D). After one treatment cycle, 57% (95% CI 41-72) of patients achieved a complete response with or without hematologic recovery, with measurable residual disease negativity in 19 cases; however, grade 3/4 adverse events such as febrile neutropenia (52%) and severe infections (25%) were notable. Venetoclax in combination with cytarabine demonstrated activity and acceptable safety, supporting further investigation in pediatric phase 3 trials (e.g., NCT05183035).
10.1016/S2352-3026(26)00136-5
Orelabrutinib versus chemoimmunotherapy in treatment-naïve chronic lymphocytic leukemia/small lymphocytic lymphoma: a randomized, phase 3 trial.
SIGNAL TRANSDUCT TAR · Q1 JOURNAL - RANK #1/319TOP-TIER
This randomized, phase 3 trial evaluated the efficacy and safety of orelabrutinib versus chlorambucil plus rituximab in 192 treatment-naïve chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) patients. At a median follow-up of 21.4 months, orelabrutinib significantly improved progression-free survival (not reached vs. 19.4 months, HR 0.32, 95% CI: 0.18-0.58, p<0.0001), overall response rate (90.1% vs. 79.2%, p=0.041), and duration of response (HR 0.30, 95% CI: 0.15-0.60, p=0.0003) compared to chemoimmunotherapy. Treatment-related adverse events occurred with similar frequency in both groups, but fewer grade 3 or worse events were reported in the orelabrutinib group (35.2% vs. 60.2%). These findings support orelabrutinib as a safe and effective first-line treatment for CLL/SLL patients, with improved efficacy and manageable safety compared to chemoimmunotherapy.
10.1038/s41392-026-02818-x
Jun 29 – Jul 06, 2026
Photobiomodulation to prevent oral mucositis and physical function impairments after hematopoietic cell transplantation: POMFITT randomized trial.
SUPPORT CARE CANCER · Q1 JOURNAL - RANK #17/173
The study evaluated the effectiveness of photobiomodulation versus standard care in preventing oral mucositis and functional impairment in 30 adults undergoing hematopoietic cell transplantation (HCT) for hematologic malignancies, using a randomized controlled trial design. The intervention involved InGaIP diode laser treatment (660 nm and 808 nm wavelengths, 100 mW, administered three times weekly from conditioning day 1 to day +3), with outcomes measured via WHO oral mucositis scale, 2-min step test, Jamar dynamometer, Sit-to-Stand test, and FACT-BMT. Severe oral mucositis occurred in 26.6% of controls versus 0% in the intervention group (p=0.032), but no significant differences were observed in hospitalization days, handgrip strength, aerobic capacity, or lower-limb strength. The study concluded that photobiomodulation reduced severe oral mucositis incidence without impacting physical function or quality of life, with high patient acceptance (93.3% rated 10/10).
10.1007/s00520-026-10970-x
Inotuzumab ozogamicin in paediatric very high risk first B-cell acute lymphoblastic leukaemia relapse (ITCC-059): a multicentre, single-arm, phase 2 trial.
LANCET HAEMATOL · Q1 JOURNAL - RANK #3/98TOP-TIER
Objective: evaluate the activity and safety of single‑agent inotuzumab ozogamicin (IO) in children (1–<18 years) with CD22‑positive very high‑risk first relapsed B‑ALL in a multicentre, international, single‑arm phase 2 cohort (ITCC‑059, cohort 3). Methods: IO 1.8 mg/m2 in cycle 1 (0.8/0.5/0.5 mg/m2 days 1/8/15), then 1.5 mg/m2 per cycle for responders, up to 6 cycles; primary endpoint was overall response rate (CR+CRp+CRi) in the amended minimal sample. Results: among the first 31 treated, ORR was 71% (22/31; 80% CI 58–82); across all 37 treated, grade 3–4 hematologic abnormalities were universal (neutropenia 95%, thrombocytopenia 84%); grade 3–4 non‑hematologic AEs included febrile neutropenia 30%, infections 22% (one grade‑5 lung infection), AST 32%, ALT 27%; serious AEs in 46%; no treatment‑related deaths; post‑transplant SOS occurred in 17% (4/23), all resolved. Conclusion: IO showed high reinduction activity with manageable toxicity.
10.1016/S2352-3026(26)00104-3
Jun 22 – Jun 29, 2026
SAVE, Safe Accelerated Venetoclax Escalation: A phase Ib study of obinutuzumab plus venetoclax with daily ramp-up in CLL.
BLOOD ADV · Q1 JOURNAL - RANK #13/98
This Phase Ib prospective trial was designed to evaluate an accelerated daily venetoclax (VEN) ramp-up with obinutuzumab in patients with chronic lymphocytic leukemia (CLL). The methodology enrolled 40 patients, with 15 (37.5%) relapsed/refractory and 13 (32.5%) with elevated tumor lysis syndrome (TLS) risk. Treatment was safely administered with a median inpatient time of 7 days, only one (2.5%) laboratory TLS event, and an overall response rate of 78.0% at 3 months. Investigators concluded that the accelerated VEN regimen is feasible and warrants further study in broader clinical settings.
10.1182/bloodadvances.2026020392
Jun 15 – Jun 22, 2026
WT1 peptide vaccines of post-allogeneic HSCT maintenance immunotherapy for pediatric acute leukemias: a phase II study.
BLOOD ADV · Q1 JOURNAL - RANK #13/98
This phase II prospective clinical trial assessed two intradermal Wilms’ tumor-1 (WT1) peptide vaccines (MCI) as post-allogeneic hematopoietic stem-cell transplantation maintenance immunotherapy in 17 pediatric patients (2–18 years) with refractory acute leukemia. Patients received repeated vaccinations and were followed for clinical response, WT1-specific cytotoxic T-lymphocyte (CTL) induction, overall survival (OS) and safety. The 3-year OS was 70.6% (95% CI 43.1–86.6%), exceeding the 30% historical control; responders maintaining remission at 1 year showed a 3-year OS of 91.7%, and immune responders (≥1.455-fold CTL rise) had higher 3-year OS than immune nonresponders (90.9% vs 40.0%, P = 0.027). Vaccination significantly raised WT1-specific CTL frequency (0.25 ± 0.08% to 1.07 ± 0.24%, P < 0.001) without serious toxicity, suggesting effective, safe relapse prevention.
10.1182/bloodadvances.2025019352
Jun 08 – Jun 15, 2026
CD19/CD22 bivalent CAR T cells in children, adolescents and young adults with B-ALL: final phase 1 trial results.
J IMMUNOTHER CANCER · Q1 JOURNAL - RANK #12/183TOP-TIER
This phase 1 clinical trial evaluated a bivalent CD19.22.BBζ CAR T-cell therapy in children, adolescents, and young adults (n=30; 28 B-ALL, 2 Burkitt) treated at the recommended phase 2 dose. Safety outcomes showed CRS in 20/28 B-ALL patients (71.4%; grade 3 in 2/20 [10%]) and grade 3 ICANS in 3/28 (10.7%); one patient’s CRS resolved with first-line siltuximab. Efficacy included measurable residual disease–negative complete remission in 25/28 B-ALL patients (89.3%), with 23/28 (82.1%) proceeding directly to HSCT a median 51 days post-infusion (range 45–68). Median relapse-free survival among responders was not reached; median overall survival for all 28 patients was 34 months (95% CI 17–NE), supporting this construct as an effective bridge to HSCT and highlighting non‑CNS extramedullary disease as a barrier to response.
10.1136/jitc-2026-015296
Total marrow and lymphoid irradiation in combination with cyclophosphamide and etoposide before haematopoietic cell transplantation for relapsed or refractory acute leukaemia: a single-centre, open-label, phase 2 trial.
LANCET HAEMATOL · Q1 JOURNAL - RANK #3/98TOP-TIER
This single-centre, open-label, phase 2 clinical trial tested a conditioning regimen of total marrow and lymphoid irradiation (2000 cGy), high-dose cyclophosphamide, and etoposide prior to allogeneic haematopoietic cell transplantation in adults with relapsed or refractory acute leukaemia. 106 eligible patients received the regimen, with toxicity assessed in an initial safety lead-in, and 2-year progression-free survival as the phase 2 primary endpoint. The 2-year progression-free survival was 34% (95% CI 25-43%), with common grade 3-4 toxicities including cytopenias (91%) and metabolic disorders (78%). The trial concluded that the novel irradiation regimen was safe and offered encouraging survival outcomes.
10.1016/S2352-3026(26)00014-1
Bendamustine/Rituximab Induction Followed by Venetoclax/Rituximab Consolidation in Frontline Treatment of Chronic Lymphocytic Leukemia: A Phase 2 Multi-Center Study.
HEMATOL ONCOL · Q1 JOURNAL - RANK #23/98
This multicenter, single-arm Phase 2 trial (NCT03609593) tested a time-limited frontline regimen for untreated CLL/SLL: three 28-day cycles of bendamustine/rituximab (BR) induction followed by venetoclax ramp-up to 400 mg daily plus rituximab every 28 days for six cycles, with venetoclax continued to 12 cycles (total 15 months). The primary endpoint was ORR, with secondary endpoints including uMRD, PFS/OS, and CTCAE v4 safety; 42 patients enrolled and 93% completed therapy. ITT ORR was 86% (CR/CRi 64%, PR 21%); among efficacy-evaluable patients, ORR was 100% (CR/CRi 75%), and end-of-therapy uMRD was 83% in marrow and 84% in blood; 3-year PFS and OS were 87.5% and 92.7%. No TLS events occurred (risk downstaged post-BR); febrile neutropenia occurred in 12%, two on-study deaths were due to COVID-19, and no Richter transformation occurred, supporting durable, safe remissions.
10.1002/hon.70205
All-Oral Treatment of Newly Diagnosed Acute Myeloid Leukemia.
NEW ENGL J MED · Q1 JOURNAL - RANK #2/332TOP-TIER
This phase 1-2 open-label, multicenter, non-randomized clinical trial evaluated an all-oral regimen of decitabine-cedazuridine plus venetoclax in 189 newly diagnosed acute myeloid leukemia patients ≥75 years old or ineligible for intensive chemotherapy. Patients received oral decitabine-cedazuridine with venetoclax; phase 1–2a assessed pharmacokinetic interaction, and phase 2a-b measured efficacy, with schedule adjustments in phase 2b to limit myelosuppression. In the pivotal phase 2b (n=101), complete response (CR) was achieved in 47% (95% CI 36–57), CR or CRi in 63% (95% CI 53–73), median overall survival was 15.5 months (95% CI 7.6–NE), and grade ≥3 anemia, neutropenia, and febrile neutropenia occurred in 30%, 26%, and 25% of patients, respectively; 30- and 60-day mortality were 3% and 10%. The investigators conclude that the all-oral combination produced meaningful response rates without pharmacokinetic interaction, offering a potentially convenient alternative for frail AML patients despite notable myelosuppression.
10.1056/NEJMoa2510223
Jun 01 – Jun 08, 2026
Dynamic genetic and nongenetic RAS pathway activation drives resistance to FLT3 and BCL2 inhibitor therapy.
BLOOD · Q1 JOURNAL - RANK #2/98TOP-TIER
This Phase 1b trial of combined venetoclax (BCL2 inhibitor) and gilteritinib (FLT3 inhibitor) in patients with acute myeloid leukemia aimed to characterize mechanisms of resistance. They used multiomic single-cell DNA/protein and RNA/protein profiling to analyze malignant clones and transcriptional states. While venetoclax and gilteritinib therapy eliminated FLT3-mutant clones, diverse RAS-activating events emerged, including selection for RAS mutations, non-mutational upregulation of RAS pathways, and a phenotypic switch to monocytic AML. RAS pathway inhibition restored sensitivity to venetoclax/gilteritinib, suggesting a potent therapeutic strategy against acquired resistance.
10.1182/blood.2025032466
Dual epitope anti-LILRB4 synthetic T-cell receptor and antigen receptor (STAR)-T-cell therapy for relapsed/refractory acute myeloid leukemia.
SIGNAL TRANSDUCT TAR · Q1 JOURNAL - RANK #1/319TOP-TIER
This first-in-human, prospective clinical trial (NCT05548088) evaluated dual-epitope nanobody-based anti-LILRB4 synthetic T-cell receptor and antigen receptor (STAR) T-cell therapy in nine adults with relapsed/refractory LILRB4-positive acute myeloid leukemia. Patients received a single infusion of autologous STAR-T cells and were followed for a median of 10.7 months; six patients were evaluable for safety and efficacy. No grade ≥3 cytokine release syndrome or ICANS occurred, although three patients died from documented infections. The best overall response rate was 50 % (3/6) in the efficacy set and 33.3 % in the full cohort, with on-treatment expansion of LILRB4-targeted STAR-T cells and reduction of LILRB4-positive blasts; single-cell RNA-seq suggested monocyte-mediated T-cell suppression in nonresponders.
10.1038/s41392-026-02765-7