sorafenib / Generic mfg. - LARVOL DELTA

Design, synthesis, and biological evaluation of quinazoline-benzohydrazide and quinazoline-benzothiazole hybrids uncovering a dual EGFR/VEGFR-2 inhibitor with pronounced cytotoxic activity against triple-negative breast Cancer. (PubMed, Bioorg Med Chem) - "Among the tested compounds, 7a demonstrated the most potent activity, with IC₅₀ values ranging from 3.98 to 9.67 μM, comparable with doxorubicin...It also markedly inhibited EGFR and VEGFR-2 with IC₅₀ values of 1.67 and 6.72 μM, compared to Erlotinib and Sorafenib, respectively...Molecular docking studies confirmed the strong binding affinity of 7a within the active sites of both EGFR and VEGFR-2, supporting its proposed mechanism of action. These findings position compound 7a as a promising lead for further development as a dual EGFR/VEGFR-2-targeting anticancer agent, particularly for aggressive cancers such as TNBC."

IO biomarker • Journal • Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • BAX • BCL2 • CASP3 • CASP9 • EGFR

Maintenance therapy in AML: A meta-analysis and a single center retrospective study (ASH 2025) - "Therapeutic interventions were analyzed, including CC-486, sorafenib, gilteritinib, FLT3 inhibitors, azacitidine, and decitabine. The meta-analysis suggests that maintenance therapy in AML patientsis associated with improved OS, DFS, and RFS, with particularly pronounced benefits observed in MRD+ and intermediate-risk subgroups. Although maintenance therapy increases hematologic and dermatologic toxicities, its overall safety is acceptable. The retrospective analysis indicated that the VA regimen showed certain efficacy and safety in unfit or elderly AML patients, but the study is limited in number and needs more cases and a prospective cohort study to further verify."

Retrospective data • Acute Myelogenous Leukemia • Hematological Disorders • Infectious Disease • Neutropenia • Thrombocytopenia

The molecular landscape of patients with malignant histiocytosis (ASH 2025) - "Responders received a range of agents matched to their mutations: trametinib ( BRAFV600E, MAP2K1F53I, KRASQ16H/PTEND24G/EPHB1R327C, MAP2K1F53I/BCL2/CDKN1B/KIT, and CALR), dabrafenib + trametinib( BRAFG596R/KRASQ61H) , imatinib ( MAP2K1C121S/METG28881T/DUSP2G137D/HIST1H3BC97Y/GRIN2AS1216C and PTPN11/STK11/GNA11/JAK2) , chidamide + sintilimab( IDH2G515A/RHOAG50T/TET23344delC), sirolimus( PTEN/FGFR3/SETD2) and sorafenib ( BRAFD594G/KRASK117N/TP53). Despite this complexity, durable clinical responses to targeted agents are achievable, even in heavily mutated cases. These findings support the use of comprehensive genomic profiling in MH to identify therapeutic targets and guide precision treatment strategies."

Clinical • IO biomarker • Sarcoma • Solid Tumor • ADGRG6 • BCL2 • BCL6 • BRAF • CALR • CDK1 • CDKN1A • CDKN1B • CDKN2A • CDKN2B • CREBBP • CYTOR • DNMT3A • EGFR • FGFR3 • GNA11 • JAK2 • KRAS • MAP2K1 • NF1 • NRAS • NRF1 • PTEN • PTPN11 • SETD2 • STK11 • TET2 • TP53

Comparative safety and efficacy of first- and second-generation FLT3 inhibitors in newly diagnosed and relapsed AML: A meta-analysis stratified by molecular biomarkers (ASH 2025) - "First-generation (sorafenib and midostaurin) and second-generation (gilteritinib, quizartinib, and crenolanib) FLT inhibitors demonstrated efficacy and safety across disease stages and are now used as a standard of treatment for FLT3-mutated AML. Conclusions Second-generation FLT inhibitors exhibit enhanced efficacy in relapsed-refractory AML scenarios and comparable outcomes to first-generation inhibitors in newly diagnosed AML.Stratification using ITD allelic ratio and NPM1 co-mutation is emerging as a critical factor. In light of these findings, it's imperative to integrate second-generation inhibitors in salvage settings and Biomarker-guided treatment decisions in front-line FLT3 mutant-AML"

Biomarker • Retrospective data • Acute Myelogenous Leukemia • Cardiovascular • Hematological Disorders • Hypertension • Neutropenia • FLT3 • NPM1

CHG combined with venetoclax and azacytidine as induction chemotherapy in acute myeloid leukemia (ASH 2025) - P2 | "Based on this premise, we endeavored to develop a low-dose, long-course CHG regimen (cytarabine, homoharringtonine, and granulocyte stimulating factor) in combination with the demethylating agent azacytidine and the Bcl-2 inhibitor venetoclax as an induction regimen(NCT06470841)...One patient achieved CRi after receiving daunorubicin plus cytarabine (DA) induction chemotherapy. However, the patient relapsed after completing 2 courses of consolidation chemotherapy (1 course of DA and 1 course of venetoclax + azacytidine + sorafenib)...In another patient, non-response (NR) was observed after 1 course of idarubicin plus cytarabine... In this trial, we utilized a low-dose, long-course regimen of CHG in combination with the demethylating drug azacytidine and the Bcl-2 inhibitor venetoclax as an induction regimen. Notably, all 5 patients achieved complete remission by a single course of VACHG induction chemotherapy, and the combined chemotherapy regimen demonstrated..."

Acute Kidney Injury • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Nephrology • Renal Disease

Hyperbaric oxygen targets RCN1 to modulate ER-mitochondria crosstalk and ameliorate sorafenib resistance in hepatocellular carcinoma. (PubMed, Drug Resist Updat) - "In xenograft and spontaneous models, combined HBO-TKIs treatment delays tumor progression and modulates the HNF4A/RCN1 axis. Taken together, our findings elucidate a hitherto uncharacterized role of HBO in regulating ER-mitochondria calcium homeostasis and support its clinical application as an adjunctive therapy in TKI-resistant HCC."

Journal • Hepatocellular Cancer • Metabolic Disorders • Oncology • Solid Tumor • HNF1A • HSPA9

Haematological toxicities with targeted drugs in tumors: A systematic review and network meta-analysis of randomized controlled trials (ASH 2025) - "1 patient treated with sorafenib plus chemotherapy was reported to have died as a result of pancytopenia and 1 patient treated with chemotherapy (gemcitabine plus cisplatin) died due to anemia. Our study confirmed that chemotherapy with or without a placebo, one targeted drug with chemotherapy was associated with more severe hematologic toxicities compared with the use of one targeted drug, tepotinib plus gefitinib, tivantinib plus erlotinib. In the targeted drug monotherapy category, for the primary outcome, we found that alectinib and gefitinib had a higher risk of all-grade (grade 1-5) and severe-grade (grade 3-5) anemia, respectively...Ganitumab plus chemotherapy had the highest risk of grade 1-5 anemia and thrombocytopenia. Afatinib plus chemotherapy had the highest risk of grade 3-5 anemia and thrombocytopenia. Ramucirumab plus chemotherapy had the highest risk of grade 1-5 and 3-5 neutropenia. Veliparib plus chemotherapy had the highest risk of grade 1-5 and 3-5..."

Retrospective data • Review • Febrile Neutropenia • Leukopenia • Lung Cancer • Neutropenia • Non Small Cell Lung Cancer • Oncology • Solid Tumor • Thrombocytopenia

Unveiling FLT3 in B-ALL: Molecular drivers and targeted treatment opportunities (ASH 2025) - "In vitro assays withincreasing concentrations of 6 FLT3i for 24, 48, 72h [Gilteritinib (Gil), Midostaurin, Crenolanib, Sorafenib,Quizartinib, Ponatinib (Pon)] and Venetoclax (Ven) were performed on primary patient samples (n=29; FLT3-mut n=5/29), 11 B-ALL wt cell lines, 2 B-ALL mut (NALM6-mut, KASUMI-10), and 4 AML cell lines (OCI-AML3 wt; MV-4-11, MOLM-13, MONO-MAC6 FLT3-mut). FLT3 alterations define a novel Ph-negative B-ALL subgroup withtherapeutic relevance. Given the responses in FLT3-wt in addition to the mutated models, FLT3i may alsobenefit patients without FLT3 muts. Thanks to: Ricerca Corrente by the Italian MoH L3P1946 andAILTreviso."

Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • T Acute Lymphoblastic Leukemia • FLT3 • IKZF1 • KMT2A • PBX1 • TCF3 • TP53 • ZNF384

FLT3 (CD135) expression in pediatric AML is associated with specific disease characteristics and worse outcome in the setting of sorafenib theraphy: A report from the Children's Oncology Group AAML1031 protocol. (ASH 2025) - "We hypothesized thathigh CD135 expression would be associated with differences in outcome in the context of exposure toFLT3 directed therapy. In the AAML1031 phase 3 trial pediatric patients with de novo AML were randomized to receivestandard chemotherapy (Arm A) or standard chemotherapy with bortezomib (Arm B); patients with highallelic ratio FLT3-ITD were eligible for standard chemotherapy plus sorafenib (Arm C) if they signedsecond consent and the treatment arm was open. These data demonstrate that high levels of FLT3 (CD135) expression is associated withmonocytic immunophenotypes, KMT2A fusions, and NPM1 mutations. Patients in Arm C (those whoreceived chemotherapy and sorafenib) with the highest CD135 expression had inferior outcomes, bothcompared to all other patients in Arm C and high expressing patients who did not receive sorafenib. Thissuggests that elevated CD135 expression may mechanistically contribute to resistance or reducedefficacy of sorafenib-based..."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Pediatrics • CD14 • CD34 • CD36 • FLT3 • ITGAM • KMT2A • NPM1 • PTPRC • SCARB1

Multiarm Phase I study of the cyclin dependent kinase (CDK4/6) inhibitor palbociclib in combination for patients with relapsed and refractory acute leukemias (ASH 2025) - "Four combination arms were studied (in a 28-day cycle): (A)sorafenib 400mg PO BID on D1-28; (B) decitabine 20 mg/m2 IV on D8-17; (C) dexamethasone 40mg on D1-4 & 11-14, and (D) Venetoclax 400 mg on D1-21. While no complete remissions were observed, clinical benefit was noted in severalpts, particularly in combination with HMA. Further evaluation of palbo, especially in earlier lines oftherapy and in combinations for KMT2a-r or FLT3mutAML may be warranted."

Clinical • P1 data • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Dermatology • Febrile Neutropenia • Infectious Disease • Neutropenia • Respiratory Diseases • T Acute Lymphoblastic Leukemia • ASXL1 • FLT3 • KMT2A • RUNX1 • TP53 • WT1

Functional analysis of STAT3 activating mutations in LGL leukemia reveals epigenetic reprograming and therapeutic vulnerabilities. (ASH 2025) - "One of those drugsis the kinase inhibitor Sorafenib which decrease cell viability and proliferation in STAT3Mut lines. Thesefindings provide mechanistic insight into how STAT3 activating mutations alter transcriptional activity,epigenetic regulation, and tumor behavior in LGLL, while identifying potential therapeutic vulnerabilitiesthat could guide precision treatment strategies."

Hematological Malignancies • Leukemia • CD5 • CD8 • CDX2 • GZMB • IL15 • NLRC5 • PDGFB • STAT3 • YAP1

Post-transplant FLT3 inhibitor maintenance is associated with improved survival in FLT3-ITD positive acute myelogenous leukemia irrespective of pre-transplant MRD status (ASH 2025) - "Fourteen (44%) received gilteritinib, 13 (41%) received sorafenib,and 5 (16%) received midostaurin as their first TKI post-HCT. Despite limitations, including limited sample size, we observed an improvement in OS in patients whoreceived post-HCT TKIm, irrespective of pre-HCT MRD status and conditioning regimen intensity.Consistent TKIm post-HCT is challenging due to the high discontinuation rate from toxicity."

Clinical • Post-transplantation • Pre-transplantation • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Transplantation • FLT3

A prospective study reveals feasibility and merits of drug profiling-guided precision therapy for children with high-risk leukemias (ASH 2025) - P | "Frequently useddrug combinations included chemotherapy plus venetoclax, homoharringtonine, or bortezomib.According to the best response per patient, 4 patients achieved complete remission (CR) and 3 achievedpartial remission (PR) while 5 patients had stable disease (SD), and 2 patients had progressive disease(PD)...Four patients (28.6%) hadactionable targets, including JAK2 in 2 patients, KIT in 1 patient, and FLT3-ITD in 1 patient, and only thepatient with FLT3-ITD received sorafenib as a matched therapy...In addition, the median TAT for returning genomic profiling results to the tumorboard was 30 days, which was significantly longer than that of drug profiling.Conclusions This study represents the largest case series with a pure cohort of pediatric acute leukemias to evaluatethe feasibility and merits of FPM, and demonstrated that FPM-guided therapy could achieve favorableresponses in pediatric patients with very high-risk leukemias, with a short TAT and high..."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • T Acute Lymphoblastic Leukemia • FLT3 • JAK2

Ex vivo drug sensitivity testing in Korean AML patients: Integration of functional and genomic profiles for predicting clinical response and survival (ASH 2025) - "Drug responses were highly variable across patients,with dasatinib, venetoclax, gilteritinib, and mitoxantrone showing the greatest inter-patient variability.Genomic stratification revealed established associations: TP53 mutations correlated with multi-agentresistance, while NPM1 and IDH1/2 mutations were associated with increased sensitivity to venetoclax-based combinations...Among ND patients treated withvenetoclax + hypomethylating agents (HMA), ex vivo sensitivity to venetoclax, azacitidine, and decitabineshowed strong correlation with overall survival (OS), with ROC-AUCs of 0.87, 0.67, and 0.87, respectively.Kaplan-Meier survival analysis based on DST-derived risk groups revealed significant differences:Venetoclax (hazard ratio, HR of 9.1, P10.0, P10, P<0.05)...Integration with genomicdata confirmed known drug–mutation relationships and revealed novel sensitivity patterns, includingIDH2–sorafenib and RUNX1–midostaurin. These findings support the incorporation of..."

Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • ASXL1 • DNMT3A • FLT3 • IDH1 • IDH2 • NPM1 • RUNX1 • TP53

Venetoclax combined with three-day multi-frequency decitabine (DEC3-VEN) VS venetoclax combined with azacitidine in elderly patients with Acute Myeloid Leukemia:a phase III, prospective, multicenter, randomized controlled trial (ASH 2025) - P3 | "This trial has been registered on ClinicalTrials.gov (NCT06073730) and is currently continuing torecruit patients.Induction regimen: Experimental Group(DEC3-VEN):VEN: 100 mg on Day 1, 200 mg on Day 2, 400 mg on Days 3-14; Decitabine: 20 mg/m² every 8 hours on Days 4-6 (infusion time >2 hours); Sorafenib: 600 mg/day on Days8-14 (for FLT3/ITD positive patients)Control Group(VEN+AZA) : VEN: 100 mg on Day 1, 200 mg on Day 2, 400 mg on Days 3-28; Azacitidine(AZA): 75 mg/m²/day on Days 3-9Main outcomes and measures: The primary endpoint is whether DEC3-VEN improves event-free survival (EFS) compared to VEN+AZA inelderly or unfit adult AML patients.As of June 30, 2025, 120 patients were enrolled. DEC3+VEN shows good efficacy and tolerability as an induction therapy for de novo or unfit AML patients,compared with VEN+AZA."

Clinical • P3 data • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • FLT3

Treatment intensity may alter the prognostic impact of baseline co-mutations in newly diagnosed FLT3-ITD AML treated with FLT3 inhibitor-based therapy (ASH 2025) - " A total of 213 pts were included; 83 received IC + FLT3i [sorafenib in 56 (68%), midostaurin in 1(1%), gilteritinib in 23 (28%), and quizartinib in 3 (4%)] while 130 received LIT + FLT3i [including sorafenibin 32 (25%), midostaurin in 1 (1%), gilteritinib in 48 (37%), and quizartinib in 49 (38%)]. The impact of baseline co-mut on frontline FLT3i-based therapy in ND FLT3-ITD mut AMLmay vary by the nature of the induction regimen. The presence of NPM1 mut +/- epigenetic regulator mutwas associated with improved outcomes with IC + FLT3i, consistent with findings from QuANTUM-1, butimportantly, we noted that co-mut in RAS pathway and MR genes portended a poorer prognosis. Incontrast, co-mut in NPM1 and MR genes did not influence outcomes with LIT + FLT3i regimens (with orwithout VEN)."

Acute Myelogenous Leukemia • Myelodysplastic Syndrome • ASXL1 • DNMT3A • FLT3 • IDH1 • IDH2 • KRAS • NPM1 • NRAS • PTPN11 • RUNX1 • TET2 • WT1

Risk factor analysis of relapse risk during FLT3-inhibitor post-transplant maintenance therapy following allogeneic hematopoietic stem cell transplantation in Acute Myeloid Leukemia with FLT3-ITD (ASH 2025) - "At HCT, 195 pts (83.7%) were in CR1, 15 (6.4%) in CR2, and 23 (9.9%) beyond CR2(including active disease up to 10% blasts).The most used FLT3i PTM was sorafenib (n=194, 83.3%), followed by gilteritinib (n=50, 21.5%); 1 ptreceived midostaurin. The current study strongly suggests a significantly increased risk of relapse in the group of FLT3-ITD AMLpatients, who do not achieve at least CR2 pre-transplant, have a high DRI or do not develop cGVHD,despite receiving FLT3i PTM. This suggests that those pts should continue FLT3i PTM indefinitely andother methods should be incorporated such as prophylactic DLI within the first 6 months post-transplantto reduce the risk of relapse"

Clinical • Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Transplantation • ASXL1 • DNMT3A • FLT3 • IDH2 • NPM1 • RUNX1 • TET2 • WT1

Real world outcomes of FLT3 inhibitors and hypomethylating agents as post transplant prophylaxis in Acute Myeloid Leukemia patients transplanted in first complete remission: From the EBMT acute leukemia working party (ASH 2025) - "Several studiesreported promising efficacy and an acceptable safety of post-transplant prophylaxis to prevent suchrelapses, mainly using FLT3 inhibitors (FLT3i), whereas the use of hypomethylating agents (HMA) such asazacitidine (AZA) or decitabine (DEC) remains controversial...Sorafenib was used in 58%, midostaurin in 30%, gilteritinib in 11% while 3 patients receivedsorafenib and midostaurin... This large study further strengthens the role of sorafenib and other FLT3i as standard postHSCT maintenance with 2-year LFS exceeding 80%, not affected by pretransplant use of FLT3i or MRDstatus. Encouraging results were also observed with HMA maintenance, with 2-year LFS exceeding 70%,particularly for patients who had GVHD prior to initiation of maintenance."

Clinical • Post-transplantation • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Transplantation • FLT3 • NPM1

Sphingosine-1-phosphate receptor modulators overcome FLT3 inhibitor resistance in Acute Myeloid Leukemia with FLT3-ITD and NRAS mutations through sphingosine kinase 1/AKT pathway downregulation. (ASH 2025) - "SphK1 is linked toFLT3 inhibitor resistance, as prolonged sorafenib exposure was shown to activate the Sphk1/S1P axis.Here we studied the efficacy of targeting Sphk1 with sphingosine-1-phosphate receptor (S1PR)modulators in conjunction with FLT3 inhibitors to overcome FLT3 inhibitor resistance mediated by NRASmutations in AML cells with FLT3-ITD. MethodsMOLM-14 and MV4-11 human FLT3-ITD AML cell lines with NRAS mutations including G12D, G12S, G12C,Q61K and Q61H and FLT3-ITD AML patient blasts with G13V and G13D mutations were cultured with theFLT3 inhibitors gilteritinib (10 nM) or quizartinib (1 nM) and/or the S1PR modulators fingolimod (FTY720; 2.5 μM) or mocravimod (KRP203; 5 μM)...ConclusionsThe S1PR agonists fingolimod (FTY720) and mocravimod (KRP203) resensitize FLT3-ITD AML cellsharboring G12D, G12S, Q61K, and Q61H, but not G12C, NRAS mutations to FLT3 inhibitors. The datasupport potential clinical efficacy of combination regimens with these clinically applicable..."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • ANXA5 • BAD • FLT3 • NRAS • SPHK1 • STAT5

Identification of therapeutic vulnerabilities in FLT3-mutated AML through customized CRISPR screens (ASH 2025) - "FLT3mutations occur in ~30% of AML cases and are targeted by tyrosine kinase inhibitors (TKIs) such asmidostaurin, sorafenib, and gilteritinib across treatment stages. CRISPR/Cas9 screen identified effective genetic modulators of FLT3 inhibitor response inacute myeloid leukemia (AML). These findings reveal critical mechanisms underlying resistance, laying thegroundwork for rational combination therapies to enhance treatment outcomes in FLT3-mutated AML.Future studies will validate these targets using gene knockdown in resistant and sensitive models toevaluate their impact on FLT3 inhibitor response.."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • CDK6 • FASN • FLT3 • NF1 • PTEN • STAT5

Determining sensitivity to FLT3 inhibitors prior to therapy in FLT3 mutant acute myelogenous leukemia (ASH 2025) - "For ex-vivo sensitivity assays, AML cells were treated with several dilutions of a FLT3i(Sorafenib, Midostaurin, Crenolanib, Quizartinib, Gilteritinib, Tuspetinib and MAX-40279 (a dualFLT3/FGFR inhibitor)) and cell viability was assessed with CellTiter-Glo® (Promega). Using our MS-based proteomics measurements and sensitivity results with our proprietary MLmodel, we processed both AML patient samples (N=57) and patient-derived cell lines (N=59), and wewere able to identify 7 distinct clusters. Previously we demonstrated that RPPA proteomics could discriminate FLT3i sensitive andresistant cases and here we present orthogonal confirmation by MS proteomics and ex-vivo sensitivityassays. Moreover, we show that the expression of only three proteins forms a robust biomarker topredict FLT3i sensitivity of FLT3-MUT AML patients prior to therapy. We also identified AML cell lines thatmimic both FLT3i resistance and sensitivity, and combined with deep proteomics data, these..."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • CD34 • FLT3 • PXN • SMARCA2

PTP4A3: A potential therapeutic target in Acute Myeloid Leukemia (ASH 2025) - "Sorafenib is identified as a potential PTP4A3-targeting inhibitor that suppresses AMLmalignancy via this mechanism. These findings highlight PTP4A3 as a novel therapeutic target in AML andsuggest sorafenib's potential utility, offering new insights for targeted therapy development."

Acute Myelogenous Leukemia • Colorectal Cancer • Hematological Malignancies • Leukemia • Solid Tumor • ANXA5 • PTP4A3

Azacitidine monotherapy versus combination regimens as post-allogeneic transplantation maintenance in high-risk myeloid malignancies: A retrospective cohort Study (ASH 2025) - "This study addresses acritical knowledge gap by conducting the first direct evaluation of AZA monotherapy versus AZAcombined with immunotherapy (interferon-α, IFN-α) or molecularly targeted agents(sorafenib/venetoclax) within a minimal residual disease (MRD)-negative cohort post allogeneichematopoietic stem cell transplantation (allo-HSCT). We conducted a retrospective cohort analysis of 59 consecutive high-risk AML/MDS patients(AML=56, MDS=3) undergoing allo-HSCT at our institution between 2019-2023, all receiving protocolizedAZA-based maintenance therapy initiated at a median of 120 days post-transplant. This study established that AZA-based maintenance therapy achieves exceptional 3-yearsurvival in rigorously selected MRD-negative high-risk myeloid malignancy patients post-allo-HSCT. AZAmonotherapy demonstrates non-inferior efficacy to combination regimens with comparable toxicity,supporting its role as a foundational strategy. While limited by retrospective design..."

IO biomarker • Monotherapy • Retrospective data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD34 • FLT3 • IFNA1 • TP53

Post-transplant maintenance with sorafenib is safe and effective in pediatric patients with har FLT3/ITD AML: A report from the Children's oncology group protocol AAML1031 (ASH 2025) - "Patients who received sorafenib also have substantially improved OS but this may bein part due to a favorable clinical status among patients who restart sorafenib. Further work is needed tounderstand whether its benefit is limited to specific disease subsets and/or those with pre HCT MRD andto clarify the optimal duration of dosing."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hypertension • Immunology • Leukemia • Neutropenia • Pediatrics • Transplantation • FLT3

Sorafenib enhances anti-leukemia effect via promoting macrophage-NK cell immune crosstalk in FLT3-ITD+ AML patients receiving allo-HSCT (ASH 2025) - "The PKM2 activator,DASA-58, diminished the effect of sorafenib on NK cells and macrophages. These data indicate thatsorafenib enhances IL-18 secretion and transcription by promoting PKM2-RelA binding.Taken together, our work reveals that post-transplantation sorafenib maintenance increases glycolysis inmacrophages, promoting PKM2-RelA binding, resulting in increased transcription and secretion of IL-18,eventually promoting the expansion of the IFN-γ+ NK cell subset."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • FLT3 • GZMB • GZMH • IFNG • IL18 • LAMP1