cytarabine / Generic mfg. - LARVOL DELTA

Phase 1/2, multi-center, open-label clinical study to evaluate the safety, pharmacokinetics, and efficacy of Zefamenib combined with chemotherapy or targeted Agents in patients with Acute Myeloid Leukemia (ASH 2025) - P1/2 | "Menin inhibitor revumenib combined with venetoclax and azacitidine have shown complete remission (CR) rate of 88.4% in newly diagnosed AML patients harboring these aberrations...Cohort A and D will enroll untreated AML patients, who will receive zefamenib combined with standard 7+3 chemotherapy for induction and zefamenib combined with high dose cytarabine for consolidation...This study will be the first to clarify the feasibility of zefamenib with intensive chemotherapy or targeted agents in the frontline treatment of AML patients or the efficacy and safety of zefamenib with targeted agents in R/R AML patients harboring NPM1 mutation or KMT2A/NUP98 rearrangements. Results of this study will provide ideas and evidence to improve the prognosis of Chinese AML patients with these genetic aberrations."

Clinical • P1/2 data • PK/PD data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • KMT2A • MEIS1 • NPM1 • NUP98

Trial in progress: Phase I trial to evaluate safety of oral azacytidine in combination with pomalidomide as maintenance therapy in AML (ASH 2025) - "Oral azacitidine has demonstrated improved relapse-free and overall survival in older, transplant-ineligible patients, establishing a role in AML maintenance (Hunault-Berger M Blood Cancer J. 2017). In resource-limited settings, delivery of high-dose cytarabine or allogeneic transplant is frequently compromised due to multidrug-resistant infections, poor performance status (PS), and lack of donors...Participants must have adequate hematologic recovery, ECOG PS 0–2, with no prior exposure to azacytidine or venetoclax...Participants are enrolled sequentially into each cohort to ensure safety. Dose escalation is ongoing as per protocol."

Clinical • Combination therapy • P1 data • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Heart Failure • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Liver Failure • Nephrology • Renal Disease • CRBN • IKZF1 • IKZF3

A phase 2 study of prophylactic intrathecal chemotherapy to prevent high-grade immune effector cell–associated neurotoxicity syndrome (ASH 2025) - "While prophylactic corticosteroids and cytokine-directed therapies like the IL-1 receptor antagonist anakinra have been explored, they carry potential risks and inconsistent efficacy...Study Design and This is a single-center, open-label, phase 2 trial evaluating the prophylactic use of IT methotrexate (12 mg), cytarabine (50 mg), and hydrocortisone (50 mg) in patients receiving standard-of-care axi-cel or brexu-cel for relapsed/refractory large cell or mantle cell lymphoma respectively...If effective, this approach could reduce the need for systemic corticosteroids, which are associated with significant morbidity. Further, prophylactic intrathecal therapy may be generalizable across existing and emerging CAR T-cell platforms, expanding eligibility to patients who might otherwise be excluded due to a high risk of neurologic toxicity."

P2 data • Acute Lymphocytic Leukemia • Brain Cancer • CNS Disorders • Epilepsy • Hematological Malignancies • Leukemia • Lymphoma • Mantle Cell Lymphoma

Venetoclax-based salvage therapy achieves high remission rates after 7+3 or low-dose cytarabine (ASH 2025) - "Introduction: In the Brazilian public health system (SUS), salvage chemotherapy for fit patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) after standard 7+3 induction (7 days of cytarabine plus 3 days of an anthracycline) is usually FLAG-IDA (fludarabine, cytarabine, granulocyte colony-stimulating factor [G-CSF], and idarubicin) or MEC (mitoxantrone, etoposide, and cytarabine)...Although the venetoclax–cytarabine (VEN-ARAC) or venetoclax–azacitidine (VEN-AZA) combination is approved as first-line therapy for unfit patients, it is generally not available in SUS... In this real-world study, VEN-based salvage therapy induced rapid and high CR rates after 7+3 or LDAC, with a favorable safety profile, and may serve as a less-toxic bridge to allo-HSCT."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia

Azole-adjusted venetoclax-based regimens in unfit AML patients: A real-world brazilian experience from a resource-limited setting (ASH 2025) - "After 2020, unfit patients were treated with Venetoclax-based regimens (n=54), either combined with Azacitidine 75 mg/m²/day SC for 7 days (AZA-VEN) or with low-dose Cytarabine 20 mg/m²/day SC for 10 days (LoDAC-VEN). All patients received, after ramp-up, azole-adjusted dosing of Venetoclax: 100 mg when combined with Voriconazole or 200 mg with Fluconazole... In this real-world study, Venetoclax-based regimens with azole-adjusted dosing showed better outcomes in unfit AML patients compared to LoDAC or BSC, reinforcing the applicability of such regimens in resource-limited settings. While overall survival in our cohort was lower than reported in pivotal trials, likely due to infectious complications and limited access to supportive care, response rates were comparable. These results support broader incorporation of this approach across diverse healthcare systems."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia

Imatinib in chronic myeloid leukemia: A comparative assessment of patients in pivotal clinical trials and real-world settings (ASH 2025) - " We included the imatinib arm of 5 pivotal CTs: the IRIS, which established the superiority of imatinib over interferon alfa plus cytarabine, and the DASISION, ENESTnd, BFORE, and ASC4FIRST trials, which supported the approvals of dasatinib, nilotinib, bosutinib, and asciminib, respectively. Patients in the RWD cohort exhibited higher clinical risk profiles, greater racial and ethnic diversity, and longer intervals between diagnosis and treatment initiation. These factors may contribute to the lower rates of achieving optimal treatment responses, including cytogenetic and molecular milestones. Despite these differences, long-term outcomes were comparable, aligning with current trends showing that survival in CML patients now approaches that of the general population."

Clinical • Real-world • Real-world evidence • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • ABL1

Utilization patterns of venetoclax-based regimens for AML in a community based setting & representative populational analyses (ASH 2025) - "In first line setting, 22 patients (52.3%) received Venetoclax with azacitidine, 17 patients 40.1% received decitabine, 2 patients (4.8%) with cladribine and cytarabine, and 2 patients (4.8%) received Venetoclax alone...6 patients (40%) received azacitidine with venetcloax, 6 patients (40%) received decitabine, 1 patient (6.7%) received cytarabine, 1 patient (6.7%) received enasidenib, and one patient (6.7%) received nelarabine, cyclophosphamide, and cytarabine... The median age at diagnosis was 64 years (range 21-86). 47% patients were female gender. 24.5% of patients were Black or African-American, 71.4% were White, 1% were Asian, and 3.1% were more than one race."

Clinical • Acute Myelogenous Leukemia • Febrile Neutropenia • Hepatology • Liver Failure • Neutropenia • Renal Disease • CEBPA • DDX41 • NPM1 • TP53

Safety and efficacy of ibrutinib as a first line agent for Mantle Cell Lymphoma : A systematic review and meta-analysis (ASH 2025) - "While standard first-line chemotherapy regimens include rituximab and bendamustine or cytarabine containing regimens, Bruton tyrosine kinase inhibitors (BTKis), such as ibrutinib and zanubrutinib, have demonstrated efficacy in relapsed or refractory MCL and are now being explored as frontline options. Notably, acalabrutinib in combination with bedamustine and rituximab has received FDA approval for treatment-naïve MCL patients who are ineligible for autologous hematopoietic stem cell transplantation (HSCT)...Conclusions Ibrutinib-based regimens demonstrate a high objective response rate and an acceptable safety profile when used as a first-line treatment for MCL. However, the substantial heterogeneity and potential publication bias is identified."

Retrospective data • Review • Atrial Fibrillation • Bone Marrow Transplantation • Cardiovascular • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Non-Hodgkin’s Lymphoma

De novo secondary central nervous system lymphoma: A real-world cohort study over two decades (ASH 2025) - "CNS-targeting treatment included intrathecal methotrexate (IT MTX) 31.8% (n=21), intravenous methotrexate (IV MTX) 25.8% (n=17) combination IT MTX and IT cytarabine (ARA-C) 18.2% (n=12), and both IV and IT MTX 13.6% (n=9). De novo SCNSL demonstrated favourable outcomes with median OS of more than 20 years. Further studies on optimal CNS-targeting regimens are warranted."

Clinical • Real-world • Real-world evidence • B Cell Lymphoma • Burkitt Lymphoma • CNS Disorders • CNS Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Natural Killer/T-cell Lymphoma • Non-Hodgkin’s Lymphoma • Peripheral T-cell Lymphoma • Secondary Central Nervous System Lymphoma • T Cell Non-Hodgkin Lymphoma

Treatment patterns and outcomes of primary central nervous system lymphoma treated with high-dose methotrexate with or without autologous stem cell transplantation or whole brain radiation in the rea-world setting (ASH 2025) - "During induction, 59% received HDMTX+rituximab (R), 39% received HDMTX+R+additional (A) chemotherapy such as temozolomide (MTR) (15%) and cytarabine/thiotepa (MATRix) (15%)...Thiotepa (TT)/BCNU was used for conditioning in 8 patients, and TT/Busulfan/Cyclophosphamide in 1... HDMTX-based induction chemotherapy is effective in patients with PCNSL, even in those with delayed diagnosis or initiation of therapy. Although a minority of patients received consolidation with ASCT, it was associated with 100% progression-free survival."

B Cell Lymphoma • CNS Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Primary Central Nervous System Lymphoma • Pulmonary Disease • Respiratory Diseases • Transplantation

Real-world treatment utilization, sequencing, and outcomes in Mantle Cell Lymphoma: Emerging treatment patterns in the United States (ASH 2025) - "Treatment regimens were categorized into 7 mutually exclusive groups: bendamustine (B)-based chemotherapy, R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone, with or without cytarabine), Bruton tyrosine kinase inhibitors (BTKi; covalent: zanubrutinib, acalabrutinib, ibrutinib, and non-covalent: pirtobrutinib), bortezomib (bort)-based, venetoclax (ven)-based, intensive chemotherapy (high-dose cytarabine, HyperCVAD (hyperfractionated cyclophosphamide, vincristine, adriamycin, and dexamethasone, etc.), and other regimens (CAR-T or others). However, chemotherapy and/or immunotherapy were associated with the highest HCRU while BTKi were associated with the lowest HCRU. Notably, more than half of patients previously treated with BTKi and anti-CD20 therapies were subsequently treated with another covalent BTKi or a non-covalent BTKi, while approximately one-third received chemotherapy and/or immunotherapy, further emphasizing the need for novel..."

Clinical • HEOR • Real-world • Real-world evidence • B Cell Non-Hodgkin Lymphoma • Chronic Lymphocytic Leukemia • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Non-Hodgkin’s Lymphoma

Impact of primary CNS prophylaxis in high CNS-IPI DLBCL: Analysis from the czech national lymphoma registry (ASH 2025) - P=N/A | "As conventional chemotherapeutics used in 1st line treatment do not effectively cross the blood-brain barrier, prophylactic methotrexate (MTX) or cytarabine (AraC) can be added intravenously or directly as intrathecal injection to selected patients (pts) based on risk factors...In total, 1196 pts with DLBCL diagnosed between 2010 and 2021 and treated initially with R-CHOP, either at the Charles University General Hospital in Prague or the University Hospital Brno, were included, of which 338 had low (0-1), 528 intermediate (2-3), and 330 high (>3) CNS-IPI, respectively...These data are consistent with a growing body of evidence suggesting that IV CNS prophylaxis may be an ineffective practice in 1st line DLBCL treatment, but prospective studies are needed to confirm this hypothesis. Supported by grants: NU23-03-00127 and NU21-03-00411"

B Cell Lymphoma • CNS Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma

Interim results of a quality improvement study evaluating subjective and objective sleep quality in hospitalized adults with acute leukemia (ASH 2025) - " In this observational study, we included adults with acute leukemia or high-grade myeloid neoplasms (≥10% blasts in blood/marrow) if they were planned for or had received recent treatment with high-dose cytarabine-based chemotherapy, targeting 60 patients across three cohorts: A) active chemotherapy (n=20), B) febrile neutropenia (FN; n=20) and C) low-risk hospitalized (n=20)... Our results suggest that detailed assessment of sleep quality is feasible in a larger study. There was also poor subjective sleep quality in hospitalized patients with AL, notably light sleep, increased awakenings, and poor sleep quality. Final results will provide baseline subjective and objective sleep quality to inform future interventional QI studies aimed at improving inpatient sleep quality and associated outcomes."

Clinical • CNS Disorders • Febrile Neutropenia • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Sleep Disorder

FLT3 testing and guideline concordance in Acute Myeloid Leukemia across an Indiana health system (ASH 2025) - "Current guidelines recommend FLT3 mutation testing at diagnosis to inform risk stratification and guide the use of FLT3 inhibitors such as midostaurin, quizartinib, and gilteritinib...The most frequently used induction therapies included venetoclax in combination with a hypomethylating agent (30%), 7+3 (15.7%), and a combination regimen consisting of fludarabine, cytarabine, idarubicin, and granulocyte colony-stimulating factor (FLAG IDA; 13.8%)... FLT3 testing was commonly performed in this cohort, yet notable variability in guideline adherence was observed. Guideline-concordant induction regimen selection was marginally higher for patients with FLT3 mutations. These findings underscore the need for institutional quality improvement initiatives aimed at enhancing the documentation of FLT3 status and optimizing the integration of guideline-directed therapies in AML management."

Discordant • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • FLT3

Enhanced antileukemic activity of momelotinib in combination with venetoclax and azacitidine compared to gilteritinib-based combination (ASH 2025) - "Despite extensive genomic insights, conventional chemotherapies, specifically anthracyclines and cytarabine , have remained the cornerstone of treatment for the past four decades...In contrast, selective inhibitors of JAK-STAT (ruxolitinib) or FLT3 (gilteritinib or quizartinib) alone failed to demonstrate comparable synergy, highlighting the distinct polypharmacological profile of momelotinib in mediating the cytotoxic response...Notably, while the efficacy of gilteritinib-based regimens was restricted to FLT3-mutant AML, the momelotinib combination demonstrated activity across both FLT3 mutant and FLT3 wild-type contexts. Altogether these preclinical data support clinical evaluation of momelotinib in combination with venetoclax and azacitidine as a potential effective treatment in AML."

Combination therapy • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • ACVR1 • FLT3 • IRAK1

Impact of pre-transplant cytoreductive therapy on post-transplant outcomes in patients with myelodysplastic syndromes with excess of blasts: A retrospective analysis (ASH 2025) - "The cytoreductive therapy included hypomethylating agents (90.6%), conventional induction chemotherapy (10.2%), and low-dose cytarabine (3.5%)...When comparing GVHD prophylaxis regimens, the use of post-transplant cyclophosphamide was associated with better OS than anti-thymocyte globulin... Pre-transplant cytoreductive therapy in MDS with EB did not improve post-transplant outcomes, and lack of response to cytoreductive therapy and delays in allo-HSCT adversely affected post-transplant outcomes. Donor type and GVHD prophylaxis also significantly impacts post-transplant outcomes. These findings suggest that pre-transplant therapy may help prevent disease progression, but it should be carefully considered and individualized."

Post-transplantation • Pre-transplantation • Retrospective data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation

Single-center retrospective analysis of venetoclax-based triple-drug combination therapy for relapsed Acute Myeloid Leukemia post allogeneic hematopoietic stem cell transplantation. (ASH 2025) - "Objective: To evaluate the efficacy and safety of venetoclax (VEN), cytarabine, and azacitidine (AZA) combination therapy in relapsed acute myeloid leukemia (AML) patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The VEN-cytarabine-AZA triple-drug regimen is an effective option for relapsed AML after allo-HSCT. However, prolonged neutropenia and thrombocytopenia necessitate vigilant monitoring for severe infections."

Combination therapy • Retrospective data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Pneumonia • Respiratory Diseases • Thrombocytopenia • Transplantation • CBFB • KMT2A • NUP98

FluMel100 with post-transplant cyclophosphamide: A safe and effective regimen for patients aged ≥75 years with MDS and AML (ASH 2025) - "Many published studies in older adults have often utilized non-myeloablative and reduced intensity conditioning regimens, including fludarabine/total body irradiation, fludarabine/melphalan (FluMel) and fludarabine/busulfan combined with varied graft versus host disease (GVHD) prophylaxis approaches...They underwent re-induction therapy with cladribine, low dose cytarabine and venetoclax, achieving a complete remission, followed by donor leukocyte infusion (DLI)... Eight patients were included: seven with AML (n=7) and one had MDS (n=1), Kanofsky Performance Status range 80-90%, HCT-CI (Comorbidity Index) range 0-7. All patients received peripheral blood stem cell (PBSC) grafts from human leukocyte antigen (HLA) matched unrelated donors (MUD). PT-Cy dose was 80 mg/kg (n= 7) and 50mg/kg (n= 1)."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Geriatric Disorders • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation • CD33 • HLA-DPB1

Diluting high yield hematopoietic stem cell apheresis for autologous transplantation results in high viability of fresh grafts beyond 72h (ASH 2025) - "At the Instituto de Prevision Social all lymphoma patients are transplanted with fresh grafts using a shortened BEAM protocol with time to collect to infusion of 96 hours (BCNU d-4; Ara-C d-4, d-3, d-2; Etoposide d-4, d-3, d-2; Melphalan d-2) and minimum CD34+ cells of 2,5 x 10*6...All patients were mobilized with GCSF +/- Plerixafor... While this is a very limited study due to number of participants, its retrospective nature and single center experience the data suggests diluting high yield apheresis products result in high viability of HPSC beyond 72 hours. Any HCT Program should aim to have cryopreservation facilities available as there are some patients that cannot be transplanted safely without said capacities. However, this may not be the case for Lymphoma patients."

Hematological Malignancies • Immunology • Lymphoma • Multiple Myeloma • Solid Tumor • Transplantation • CD34

Etoposide +cytarabine plus G-CSF mitigates daratumumab-associated impairment on stem cell mobilization yields in multiple myeloma (ASH 2025) - "Objective: Quadruplet induction therapy comprising daratumumab(dara) and lenalidomide(lena), followed by high-dose chemotherapy and autologous stem cell transplantation (ASCT) has become the standard frontline treatment for patient with multiple myeloma(MM). The median total CD34+ cells collection was 19.33 × 10⁶/kg in the dara group, significantly lower than that in lena group (25.59 × 10⁶/kg, p=0.005). Notably, patients receiving more than eight doses of dara showed a reduced CD34+ cell collection (median 14.16 × 10⁶/kg vs. 21.45 × 10⁶/kg; p = 0.039)."

Hematological Disorders • Hematological Malignancies • Lymphoma • Multiple Myeloma • Neutropenia • CD34

The clinical trials landscape in chronic lymphocytic leukemia: A systematic review of control arm adequacy (ASH 2025) - "The Alliance A041202 and CLL14 trials demonstrated substantial progression-free survival (PFS) benefits of BTKi monotherapy and fixed-duration venetoclax plus obinutuzumab (Ven/Obi), respectively, establishing them as standard first-line treatments...Six trials (42.8%) employed substandard control arms, all utilizing a combination of chemoimmunotherapy: fludarabine/cytarabine/rituximab or bendamustine/rituximab (n=4) or chlorambucil and rituximab (n=2)...Notably, 2 of the BTKi trials utilized ibrutinib rather than next-generation BTKis, such as acalabrutinib or zanubrutinib... Of all modern phase III trials for treatment-naive CLL, nearly half did not utilize control arms that align with the current SOC in the US or EU, risking inflated estimates of the efficacy of their experimental therapies and compromising the external validity of their results. However, although the small sample size limited statistical analysis, we found that the majority of these trials were..."

Clinical • Review • Chronic Lymphocytic Leukemia • Hematological Malignancies • Leukemia

Erythroblastic sarcoma transformation from chronic myeloid leukaemia: A case report (ASH 2025) - "Dasatinib was held due to concerns of drug-related pleural effusion and disease progression...She received three cycles of high-dose cytarabine and ponatinib...She continues to receive ponatinib with plans to add Azacitidine... Myeloid Sarcoma is a rare form of myeloid malignancies, which is usually composed of myeloblasts, Monoblasts or megakaryoblasts. Erythroblastic sarcoma is a rare form of MS which is associated with a poor prognosis. ES Transformation from CML is extremely rare, with only a few cases reported in the literature."

Case report • Clinical • IO biomarker • Acute Myelogenous Leukemia • Cardiovascular • Chronic Myeloid Leukemia • Hematological Malignancies • Hypertension • Leukemia • Lymphoma • Musculoskeletal Pain • Respiratory Diseases • Sarcoma • Solid Tumor • ABL1 • BCR • CD123 • CD33 • CD36 • CD38 • CD7 • IL3RA • KIT • SCARB1 • TFRC

Chronic myeloid leukemia and ASXL1 mutations: A distinct entity requiring special attention (ASH 2025) - "Initial treatment: nilotinib 800 mg/day, reduced to 200 mg BID due to hematologic toxicity...Started 7+3 (cytarabine + daunorubicin) + ponatinib on 05/20/2025...NGS:WT1 p.C29_L30insCRHPGPEPRSVC* VAF 1.39%, ASXL1 p.G645Vfs*58 VAF 1.55%, ASXL1 p.E929G VAF 1.46% ,ASXL1 p.T936= VAF 1.41%(Tier 1) Started R-HyperCVAD + dasatinib...In the current era of CML targeted therapy with TKIs or myristoyl inhibitors, the presence of associated genetic alterations (AGAs) becomes relevant, as they may predict aggressive behavior and open a window to evaluate transplantation in these patients for better therapeutic responses. Imatinib is not benefical in this patients."

Chronic Myeloid Leukemia • CNS Disorders • Hematological Disorders • Hematological Malignancies • Hepatology • Herpes Zoster • Infectious Disease • Leukemia • Liver Failure • Musculoskeletal Pain • Myeloproliferative Neoplasm • Respiratory Diseases • Varicella Zoster • ABL1 • ASXL1 • CD20 • CEBPA • GATA2 • WT1

Rapid transformation from JAK2-positive PMF to acute leukemia: Therapeutic challenges and rare DIC in an aggressive disease course (ASH 2025) - "Emergency leukapheresis and hydroxyurea were started for cytoreduction...Treatment options considered included combination chemotherapy with low-dose cytarabine (Ara-C), cladribine, and venetoclax, versus a palliative approach due to the patient's declining functional status and aggressive disease biology... This case underscores the clinical significance and poor prognosis associated with leukemic transformation of JAK2-positive PMF, particularly in the presence of high-risk mutations such as RUNX1 and TP53. These mutations not only predict an aggressive clinical course but also portend resistance to conventional hypomethylating agents. While decitabine provided initial stabilization, the early relapse highlights the limitations of current therapies in such molecularly adverse settings."

Acute Myelogenous Leukemia • Back Pain • Fibrosis • Hematological Malignancies • Immunology • Leukemia • Metabolic Disorders • Musculoskeletal Pain • Myelofibrosis • Myeloproliferative Neoplasm • Nephrology • Renal Disease • Thrombocytopenia • ABL1 • BCR • JAK2 • RUNX1 • TP53

Remission instead of eradication? MEK inhibition in primary refractory childhood LCH (ASH 2025) - "Patient and Methods We report on a 9-month-old infant with multisystemic LCH (thymus and cervical lymph nodes) who progressed under vinblastin/prednisone and did not respond to second-line cytarabine/vincristine therapy...This class confers resistance to first-and second generation BRAF inhibitors (e.g., Vemurafenib, Dabrafenib), which preferentially target monomeric BRAFV600E but not dimer-dependent BRAF. Functional ex vivo drug sensitivity profiling demonstarted superior tumor cell cytotoxicity of cobimetinib compared to other tested agents, including BRAF inhibitors and alternative MEK1/2 inhibitors, such as trametinib and selumetinib...However, the risk of clonal persistence underscores the need for integrated strategies. Future studies should investigate rational combinations of MEK inhibitors with senolytics and/or mTOR blockade to target both MAPK signaling and senescent cell survival, thereby suppressing SASP-related inflammation, aiming for durable molecular..."

Clinical • Hematological Malignancies • Langerhans Cell Histiocytosis • ARAF