midostaurin / Generic mfg. - LARVOL DELTA

Mast Cell Leukemia: Comprehensive Review of Literature With Current Insights and Updates on Management. (PubMed, J Hematol) - "The two targeted therapies approved for MCL are midostaurin, a multikinase inhibitor, and avapritinib, a selective KIT D816V mutation-targeted tyrosine kinase inhibitor. In this article, we conducted a comprehensive yet simplified review of MCL, focusing primarily on its clinical manifestations and recent updates on management. We also identified the areas that require further research and emphasized the aggressive nature and poor prognosis associated with this disease."

Journal • Review • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Mast Cell Leukemia • Oncology • Transplantation

Real-world characteristics of systemic mastocytosis in Romania: insights from a reference-center-based descriptive study. (PubMed, J Med Life) - "First-line symptomatic therapy (H1/H2 antihistamines ± montelukast) was administered to 77% of patients, while four patients with advanced disease received midostaurin treatment. Immunophenotyping confirmed aberrant expression of CD2 and CD25 in all eight analyzed cases. This first national series from Romania underscores the predominance of indolent SM and the clinical burden of organ involvement, reinforcing the need for early diagnosis and personalized, risk-adapted therapeutic approaches."

Journal • Observational data • Real-world evidence • Retrospective data • Aggressive Systemic Mastocytosis • Hematological Disorders • Hematological Malignancies • Hepatology • Leukemia • Mast Cell Leukemia • Myeloproliferative Neoplasm • Oncology • Osteoporosis • Rare Diseases • Rheumatology • CD2 • IL2RA • ISG20 • KIT

Identification of biomarkers based on ubiquitin-correlated genes for predicting immune profile and drug sensitivity in lung adenocarcinoma. (PubMed, Front Pharmacol) - "The medications TAE684, Cisplatin, and Midostaurin exhibited the largest negative correlation with Riskscore, according to drug sensitivity study. Lastly, we demonstrated through in vitro tests that HEATR1 knockdown markedly reduced LUAD cell survival, migration, and invasion. This study is the first to systematically integrate the ubiquitin pathway with multi-omics data, constructing a robust risk model for LUAD prognosis and immune characteristics, providing a theoretical reference for future exploration of potential biomarkers for LUAD patients' diagnosis."

Biomarker • IO biomarker • Journal • Lung Adenocarcinoma • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • Targeted Protein Degradation • DNAJB4 • FAT1 • MTMR4

Impact of Molecular Profiling in Choosing First Line of Management in Acute Leukemia (SOHO 2025) - "Interventions: Post-diagnostic confirmation and genetic profiling, midostaurin or gilteritinib in cases with FLT3 mutations, dasatinib in cases with BCR-ABL mutation were added to standard chemotherapy...For ALL, 43.8% of patients had the BCR-ABL mutation, and those treated with dasatinib had a 1-year OS of 46.4%; CD20-positive ALL treated with rituximab showed a 1-year OS of 48.5%, while patients without targetable mutations had a 1-year OS of 85.7%... Flow cytometry plays a crucial role in the diagnosis of acute leukemia. Its risk stratification, treatment decisions, and prognosis should be integrated into diagnosis and treatment planning to optimize patient outcomes. Modern targetable drugs play a crucial role in extending PFS and OS of patients of acute leukemia."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • BCR • CD20 • FLT3

Systemic Mastocytosis in Armenia: A Rare Disease With Two Documented Cases (SOHO 2025) - "Treatment access is equally restricted —midostaurin, the standard therapy, is unaffordable for most, and alternatives like imatinib are ineffective in D816V-positive disease. Diagnosing SM is challenging due to its variable presentation and reliance on specialized tools. In Armenia, limited access to KIT D816V mutation testing and immunopheno-typing delays diagnosis, as seen for patient 1. Patient 2 sought care abroad due to local limitations."

Clinical • Hematological Malignancies • Oncology • IL2RA • ISG20 • KIT

Evolving Paradigms in AML: How Transplantation Fits in the Modern Therapeutic Landscape (ICBMT 2025) - "These include FLT3 inhibitors (midostaurin, gilteritinib, quizarti nib), IDH1/2 inhibitors (ivosidenib, enasidenib, olutasidenib), the BCL -2 inhibitor venetoclax, and oral hypomethylating agents, liposomal cytarabine , and targeted antibodies...In parallel, the CLIA -VEN regimen (cladribine, idarubicin, cytarabine, and venetoclax) has demonstrated high efficacy in younger AML patients, with CRc rates exceeding 90%, MRD negativity in 89%, and a 5 -year OS of 73% —even among high -risk subgroups such a s those with ELN adverse risk and KMT2A mutations (Bouligny et al...In a recent phase I/II study, the triplet combination of decitabine, venetoclax, and quizartinib in older patients with FLT3 -ITD AML resulted in 92% CRc, with 67 –71% The International Congress of BMT 202 5 September 11(Thu) – 13(Sat), 2025; BEXCO, Busan 2 / 2 achieving flow or molecular negativity (Yilmaz et al...Maximizing MRD negativity before transplant, integrating novel agents upfront, and o..."

Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Transplantation • FLT3 • IDH1 • IDH2 • KMT2A • NPM1

Tyrosine Kinase Inhibitors for Gastrointestinal Stromal Tumor After Imatinib Resistance. (PubMed, Pharmaceutics) - "Sunitinib, regorafenib, and ripretinib are currently approved as standard second-, third-, and fourth-line therapies, each demonstrating efficacy against distinct mutational profiles. Avapritinib, notably effective against PDGFRA D842V mutations, represents a milestone for previously untreatable subgroups. Several alternative agents-such as nilotinib, masitinib, sorafenib, dovitinib, pazopanib, and ponatinib-have shown varying degrees of success in refractory cases or specific genotypes. Investigational compounds, including crenolanib, bezuclastinib, famitinib, motesanib, midostaurin, IDRX-42, and olverembatinib, are under development to address resistant or wild-type GISTs...Future strategies include precision medicine approaches such as ctDNA-guided therapy, rational drug combinations, and novel drug delivery systems to optimize bioavailability and reduce toxicity. Ongoing research will be crucial for refining treatment sequencing and expanding therapeutic options,..."

Journal • Review • Gastrointestinal Cancer • Gastrointestinal Disorder • Gastrointestinal Stromal Tumor • Oncology • Sarcoma • KIT • PDGFRA

Identifying Drug Combination Strategies for ZMYM2: FGFR1 Fusion Positive Leukemia. (PubMed, Precis Oncogenom) - "After initially responding to ponatinib, the patient was switched to pemigatinib which eventually transitioned them to a successful transplant. Leukemia cells isolated from the patient exhibited ex vivo sensitivity to ponatinib, bortezomib and axitinib. ZMYM2:FGFR1-transformed Ba/F3 cells were exquisitely sensitive to next generation FGFR inhibitors, and combinations of FGFRi plus trametinib or midostaurin were found to be synergistic, suggesting novel therapeutic options for FGFR1-fusion positive patients."

Journal • Eosinophilia • Hematological Malignancies • Leukemia • Lymphoma • Oncology • Transplantation • FGFR1 • ZMYM2

Efficacy and Safety of FLT3 Inhibitors in Combination With Intensive Chemotherapy in Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia: A Systematic Review (SOHO 2025) - "Rates of CR were between 37% and 90%, with the greatest contributions observed in gilteritinib-containing regimens, followed by sorafenib (78%), and midostaurin (37%-58.9%). FLT3 inhibitors when added to intensive chemotherapy improved response and outcomes for newly diagnosis FLT3-mutated AML (in addition to some range of toxicities). This study provides strong support for adding FLT3 inhibitors, especially midostaurin, in combination with standard chemotherapy in this subset of AML, as these inhibitors have shown improved therapeutic benefits over standard chemotherapy alone. Further prospective investigations should aim to assess overall survival and optimal sequencing with other novel agents (eg, maintenance therapy or transplant)."

Clinical • Combination therapy • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3

FLT3+, CD33+, RUNX1+, DNMT3A+, NPM1+ AML Relapse Presenting with Subarachnoid Hemorrhage and Hyperleukocytosis: A Real-World Sequential Salvage Strategy (SOHO 2025) - "Case Presentation: A 68-year-old male with FLT3-ITD+, CD33+, RUNX1+, DNMT3A+, and NPM1+ AML (FLT3-ITD allelic ratio of 0.54) was treated with 7+3 induction chemotherapy and gemtuzumab ozogamicin (Mylotarg) followed by midostaurin (Rydapt)...Gilteritinib was well tolerated but failed to induce hematologic remission. The patient was then transitioned to azacitidine and venetoclax for further salvage... Relapsed FLT3+ AML with comutations and CNS hemorrhage presents a unique therapeutic dilemma. This case illustrates that leukapheresis and FLT3 inhibition may be feasible in the presence of thrombocytopenia and bleeding and that hypomethylating agents remain a vital salvage option in refractory disease."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD33 • DNMT3A • FLT3 • NPM1 • RUNX1

Characterization of an Oral Oncology Drug Repository Program for Hematology and Hematopoietic Cell Transplant Patients at a Comprehensive Cancer Center (SOHO 2025) - "The most common medications dispensed were venetoclax (34%), BCR-ABL tyrosine kinase inhibitors (25%), ruxolitinib (14%), midostaurin (11%), and infection prophylaxis (7%). The OODDR is a major resource for patients and care teams to improve access to oral oncology medications and supportive care agents. Increased efforts to improve medication access and awareness of medication assistance platforms are vital."

Clinical • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • Oral Cancer • ABL1 • BCR

Preliminary Results of FLAG-Venetoclax Induction in Newly Diagnosed Acute Myeloid Leukemia: A Single-Center Study (SOHO 2025) - "FLAG (fludarabine, cytarabine, and GCSF), along with venetoclax, has been an impressive anthracycline-free alternative backbone...Midostaurin was added for 2 weeks during induction for patients with FLT3 mutation... FLAG-venetoclax induction is a promising anthracycline-free regimen showing impressive induction outcomes in treatment-naïve AML patients in all three risk groups. The study warrants further evaluation in a large prospective trial setting."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • TP53

How to Best Incorporate FLT3 Inhibitors in the Management of FLT3 Mutated AML (SOHO 2025) - "Studies randomizing FLT3 inhibitors plus venetoclax and azacitidine vs venetoclax and azacitidine alone are also ongoing...Although no randomized quizartinib vs midostaurin studies are available or expected, results from randomized studies comparing gilteritinib to midostaurin added to cytarabine/daunorubicin-based intensive chemotherapy recently were presented in abstract form...Preclinical data have already suggested a substantial synergy of this approach. Data from ongoing trials combining will emerge soon and are eagerly awaited."

Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Oncology • ABL1 • BCR • DNMT3A • FLT3 • NPM1

RXRA Ligand and FLT3 Inhibitor Combination Shows Synergistic Effect in FLT3-ITD-Mutated Acute Myeloid Leukemia (SOHO 2025) - "Because we observed a synergistic effect of NHR ligand in combination with tyrosine kinase inhibitor in chronic myeloid leukemia (CML), we explored the effect of combining FLT3 inhibitors (midostaurin and gilteritinib) with retinoid X receptor alpha (RXRA) ligand (bexarotene) in FLT3-internal tandem duplication (ITD)-mutated AML. Our findings support the rationale for clinical evaluation of RXRA agonist/FLT3 inhibitor combination as a therapeutic strategy in FLT3-ITD-mutated AML."

Acute Myelogenous Leukemia • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology • CASP3 • FLT3

Mixed-Phenotype Acute Leukemia With T/Myeloid Lineage: A Case Presentation (SOHO 2025) - "The patient received induction therapy with 7+3 protocol (cytarabine and daunorubicin) plus midostaurin and dexamethasone. This condition presents a therapeutic challenge due to both the lack of specific management guidelines and limited access, particularly in our setting, to high complexity studies such as next-generation sequencing (NGS), which are highly valuable for prognostic assessment and therapeutic guidance. We present a rare case of MPAL with a driver mutation (FLT3-ITD), which showed a favorable response to induction therapy."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ANPEP • CD33 • CD34 • CD5 • CD7 • CD8 • CEBPA • FLT3 • ITGB3 • KIT • KMT2A • NPM1 • TNIP2

A Phase 1 Study of Gemtuzumab Ozogamicin Added to Cytarabine, Daunorubicin, and Midostaurin Induction in Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia (SOHO 2025) - P1 | "DL2 with two doses of GO was identified as the MTD. Response rates in this dose-finding study appear promising and warrant further evaluation in a larger cohort."

P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3

AI-driven pilot study of FLT3 inhibition: Evaluating the chemical space of midostaurin and staurosporine (ACS-Fall 2025) - "Compounds S3 and M5 displayed high affinity for FLT3 (SMINA Affinity > 70). This pilot study identified novel analogues with potential therapeutic benefit in AML and demonstrated the utility of AI methodologies to advance drug design."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • FLT3

Updates on Therapy Options in Fit and Unfit Patients with Newly Diagnosed AML. (PubMed, Curr Treat Options Oncol) - "In our practice, we continue to use 7 + 3 induction for fit patients, adding midostaurin or quizartinib for FLT3-mutated AML, or gemtuzumab ozogamicin for core binding factor (CBF) AML expressing CD33...In the presence of IDH1 mutations, we consider azacitidine combined with ivosidenib. If venetoclax is contraindicated or not tolerated, targeted therapies such as gilteritinib, ivosidenib, or enasidenib may be appropriate based on mutation profile...When feasible, clinical trial enrollment should be considered for newly diagnosed patients with these alterations. As the therapeutic landscape for AML continues to evolve, timely molecular characterization is more essential than ever to optimize outcomes and select the most appropriate frontline strategy."

Journal • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • CD33 • FLT3 • IDH1 • IDH2 • KMT2A • NPM1

Functional combination of resveratrol and midostaurin induces cytotoxicity to overcome acquired midostaurin resistance in FLT3-ITD expressing acute myeloid leukemia cells. (PubMed, Naunyn Schmiedebergs Arch Pharmacol) - "In conclusion, the FLT3/STAT5A axis and SK-1 might play an important role in the reversal of midostaurin resistance by resveratrol. Therefore, the concurrent administration of resveratrol plus midostaurin could potentially serve as a therapeutic approach to address midostaurin resistance and enhance the overall therapy efficacy for FLT3-ITD AML patients after being validated with future in vivo and ex vivo studies."

IO biomarker • Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • BAX • BCL2 • CASP3 • FLT3 • SPHK1 • STAT5

Signature of leukemia stem cell death pattern predicts prognosis and therapeutic response of acute myeloid leukemia patients. (PubMed, Sci Rep) - "Additionally, predictions regarding FDA-approved drugs indicated that the high LSCD score group is less sensitive to Venetoclax but more sensitive to Crenolanib, Tandutinib, or Midostaurin. In summary, we developed an LSCD model that shows the predictive potential of clinical prognosis and drug sensitivity. This model provides meaningful insights for personalized treatment of AML patients."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • CDK6 • HOXA9 • IL2RA • S100A4 • XIRP2

Neonatal Aggressive Systemic Mastocytosis Treated With Midostaurin and Systematic Review of the Literature. (PubMed, Pediatr Dermatol) - "Treatment with midostaurin, a multikinase inhibitor, led to significant clinical improvement, including resolution of skin lesions and reduction in hepatosplenomegaly. This case highlights the importance of early recognition of cutaneous signs in neonatal ASM and supports the potential role of targeted therapy with midostaurin."

Journal • Aggressive Systemic Mastocytosis • Genetic Disorders

A Rare Case of Acute Aleukemic Mast Cell Leukemia With Osteoblastic Lesions in the Appendicular Skeleton (SOHO 2025) - "We described here a very rare case of aleukemic MCL with diffuse osteoblastic metastatic bone lesions involving axial and appendicular skeleton. With this case, we would like to report that MCL is a malignancy that can present with both osteolytic and osteoblastic bone lesions and stress the importance of bone biopsy in suspected cases of MCL. The mainstay of management of MCL includes targeted chemotherapies, midostaurin or avapritinib, and supportive measures."

Clinical • Hematological Malignancies • Leukemia • Mast Cell Leukemia • Oncology • ANPEP • CD33 • CD4 • KIT

Innovative Therapeutic Approaches in Systemic Mastocytosis: an Updated Review. (PubMed, Maedica (Bucur)) - "The approval of tyrosine kinase inhibitors (TKIs), particularly midostaurin and avapritinib, has provided new therapeutic options for patients with advanced SM, demonstrating significant improvements in overall survival (OS) and symptom control. The present review provides an updated overview of the evolving therapeutic landscape of SM, emphasizing recent clinical trial data, novel targeted therapies and emerging treatment paradigms. We discuss the implications of this progress on patient outcomes and future directions for personalized medicine in SM."

Journal • Transplantation

Midostaurin in daily clinical practice of patients with advanced systemic mastocytosis. (PubMed, Br J Haematol) - "Following midostaurin discontinuation, 7/41 (17%) patients experienced a discontinuation syndrome, which was effectively prevented in subsequent patients through dose tapering and corticosteroid bridging. In conclusion, midostaurin demonstrated a favourable safety profile and yielded durable responses in AdvSM patients across dosing regimens."

Journal • Hematological Disorders • Myeloproliferative Neoplasm • Neutropenia • Oncology

Rational combination of homoharringtonine to selectively target FLT3-ITD acute myeloid leukemia through synthetic lethality. (PubMed, Phytomedicine) - "FLT3-ITD AML cells represent a responsive subgroup to HHT treatment in vitro and in vivo. The combination of HHT and quizartinib demonstrates optimal efficacy and selectivity against FLT3-ITD AML cells in xenografts, with no observed toxicity."

IO biomarker • Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • BAX • FLT3 • MCL1