pazopanib / Generic mfg. - LARVOL DELTA

Real world evidence of treatment patterns in hereditary hemorrhagic telangiectasia (ASH 2025) - "Treatment examinedincluded bevacizumab, pazopanib, pomalidomide, aminocaproate, tranexamic acid (TXA), octreotide,lanreotide, thalidomide, tacrolimus, sirolimus, tamoxifen and raloxifene...The majority of HHT patients were treated withferrous sulfate (n=910), followed by iron sucrose, ferrous gluconate, ferric carboxymaltose, iron dextran,and ferric derisomaltose...While 8% of patients treated with TXA had VTE, the data does not support a definitiveconclusion of increased VTE in patients treated with anti-fibrinolytics. To further elucidate treatmentpractices in HHT, a multi-center approach incorporating review of medical records would providevaluable insights into real-world practices."

Clinical • HEOR • Real-world • Real-world evidence • Anemia • Aplastic Anemia • Cardiovascular • Cerebral Hemorrhage • CNS Disorders • Gastrointestinal Disorder • Hematological Disorders • Hypertension • Renal Disease • Venous Thromboembolism

fss25-67: Advances in Targeting Angiogenesis and Managing Severe Anemia in Vascular Bleeding Disorders:Hereditary Hemorrhagic Telangiectasia, Von Willebrand Disease, and Beyond (ASH 2025) - "This symposium will discuss the role of angiogenesis in these bleeding disorders, how to optimally manage this bleeding with anti-angiogenic agents such as bevacizumab, pomalidomide, and pazopanib, and how to properly manage the complex and difficult chronic, severe iron deficiency anemia that complicates the course of these patients. Current and future potential antiangiogenic and coagulation-based treatments will be discussed by international leaders in the field."

Anemia • Hematological Disorders • Hemophilia

Functional Precision Medicine In Glioblastoma: Advancing Drug Screening With Patient-Derived 3D Cultures And Advanced Imaging Analyses (SNO 2025) - "Resistance to the trimodal standard—resection, radiotherapy, and temozolomide—demands novel, patient-tailored strategies...(3) Drug efficacy proved patient-specific: in 3-D, imatinib was most potent in Patients 1, 3, 5, 6; crizotinib in Patients 2; osimertinib in Patients 3 and 5; and pazopanib in Patients 4 and 7... Integrating mechanistic assays with patient-matched organoids unmasks actionable kinase dependencies and eliminates misleading 2-D signals. This scalable workflow refines preclinical drug ranking, supports adaptive trial enrollment, and could anchor statewide precision-oncology programs aimed at extending survival for patients with GBM and related gliomas."

Metastases • Brain Cancer • Glioblastoma • Glioma • High Grade Glioma • Solid Tumor • ANXA5

EXCEPTIONAL RESPONSE TO COMBINATION IMMUNOTHERAPY WITH YH001 AND ENVAFOLIMAB IN METASTATIC SARCOMA. (CTOS 2025) - "Data from a group of patients at a single institution enrolled in a prematurely discontinued clinical trial are presented, focusing on one patient who showed an exceptional response. This discontinued phase 1/2 open-label study was designed to investigate the combination of YH001 (recombinant humanized anti-CTLA-4 IgG1 monoclonal antibody) and envafolimab (KN035, an anti-PD-L1 agent approved in China) in advanced sarcomas. A sustained disease response was identified in a patient with metastatic fibrosarcoma refractory to chemotherapy and TKI, after PD-L1/CTLA-4 treatment, underscoring potential for combined immunotherapeutics in sarcoma and the need for predictive biomarker identification beyond TMB and PD-L1."

IO biomarker • Metastases • Tumor mutational burden • Fibrosarcoma • Leiomyosarcoma • Liposarcoma • Oncology • Sarcoma • Solid Tumor • Synovial Sarcoma • TMB

E2F transcription factors as multimodal biomarkers for pan-cancer management. (PubMed, Sci Rep) - "There existed a positive correlation between E2F2 expression level and Dasatinib sensitivity, negatively related to drug sensitivity of Nelarabine, XK-469, Cyclophosphamide, etc. Pazopanib, Doxorubicin, and Paclitaxel sensitivity was all positively associated with E2F5 expression. According to these analysis and validation results, E2F genes are relevant to the occurrence and progression of various cancers, which may be biomarkers for tumor diagnostics and prognosis. The discovery of new therapeutic targets can lead to reshaping TME to promote tumor-suppressive metastasis rather than tumor-friendly metastasis."

Biomarker • IO biomarker • Journal • Pan tumor • Tumor mutational burden • Bladder Cancer • Genito-urinary Cancer • Microsatellite Instability • Oncology • Solid Tumor • E2F2 • E2F5 • E2F7 • MSI • TMB

Efficacy and safety comparison of small molecule anti-angiogenic drugs in the treatment of bone and soft tissue sarcomas : a network meta-analysis. (PubMed, BMC Cancer) - "Short-term efficacy of small-molecule anti-angiogenic TKIs varied across bone and soft tissue sarcomas, with trends favoring apatinib plus chemotherapy and anlotinib. However, substantial heterogeneity across studies, including sarcoma subtypes and prior therapies, limits definitive conclusions regarding comparative efficacy. A multidisciplinary team is needed to better manage the side effects."

Journal • Retrospective data • Review • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

MatchMel: Molecular Profiling and Matched Targeted Therapy for Patients With Unresectable Advanced or Metastatic Melanoma (clinicaltrials.gov) - P2 | N=200 | Completed | Sponsor: Melanoma Institute Australia | Active, not recruiting ➔ Completed | N=1000 ➔ 200 | Trial completion date: Dec 2028 ➔ Dec 2025

Enrollment change • Trial completion • Trial completion date • Melanoma • Oncology • Solid Tumor

Pulmonary Leiomyosarcoma: A Diagnostic Challenge. (PubMed, Cureus) - "She was treated with combination chemotherapy followed by oral pazopanib, showing good clinical and radiological improvement. This case highlights how rare and difficult to diagnose this tumor can be and the importance of tissue diagnosis in guiding effective treatment."

Journal • Cough • Leiomyosarcoma • Lung Cancer • Oncology • Respiratory Diseases • Sarcoma • Solid Tumor • Spindle Cell Sarcoma

Efficacy of Targeted Agents and Immune Checkpoint Inhibitors in Patients with Malignant Histiocytosis (ASH 2024) - "TAs included BRAF inhibitor (vemurafenib [1]), MEK inhibitors (binimetinib [1], cobimetinib [2], trametinib [2]) and others (dasatinib [1], pazopanib [1], pexidartinib [1]), while ICIs were exclusively pembrolizumab (5)...TAs included BRAF inhibitor only (vemurafenib [3], dabrafenib [3]), MEK inhibitor only (trametinib [5]), BRAF + MEK inhibitors (2), and others (apatinib, anlotimib, bevacizumab, daratumumab, dasatinib, imatinib, pazopanib, sorafenib, or combinations [9]), while ICIs included pembrolizumab (4), nivolumab (4), tislelizumab (1), and sintilimab (1)...Conclusion TAs and ICIs can be considered in the management of MH. The responses to ICI therapy may be associated with the degree of PDL1 expression"

Checkpoint inhibition • Clinical • IO biomarker • Hematological Malignancies • Oncology • Sarcoma • Solid Tumor • DOCK8 • MAP2K1 • PD-L1 • PTPN11

MAP3K3 Contributes to Myocardial Ischemia/Reperfusion Injury by Promoting Myeloid Cell Diapedesis through TAL1/JAM-A Pathway. (PubMed, Theranostics) - " MAP3K3 orchestrates myeloid cell diapedesis via a TAL1/JAM-A dependent program during MI/RI. Targeting MAP3K3, exemplified by pazopanib, may offer a therapeutic strategy for MI/RI and related inflammatory conditions."

Journal • Cardiovascular • Inflammation • Myocardial Ischemia • Reperfusion Injury • Targeted Protein Degradation • F11R

High-Throughput Screening Using the Self-Controlled Tree-Based Scan Statistic to Identify Medications Associated With Hospitalization for Severe Acute Liver Injury. (PubMed, Pharmacoepidemiol Drug Saf) - "High-throughput screening using tree-based scan statistics detected potentially hepatotoxic drugs for investigation in future pharmacoepidemiology studies."

Journal • CNS Disorders • Gastroenterology • Hepatology • Liver Failure • Pain

Efficacy of Pazopanib for patients for patients with aggressive fibromatosis [WITHDRAWN] (ESMO Asia 2025) - No abstract available

Clinical • Desmoid Tumors • Oncology • Sarcoma • Solid Tumor

Pazopanib as maintenance therapy in pediatric ewing sarcoma: a case series of six patients. (PubMed, Mol Cell Pediatr) - No abstract available

Journal • Ewing Sarcoma • Oncology • Pediatrics • Sarcoma • Solid Tumor

Encountering the unicorn - abscopal effect after fractionated stereotactic radiosurgery for brain metastasis in renal cell carcinoma: a case report and review of the literature. (PubMed, Ecancermedicalscience) - "Following an initial course of Pazopanib and subsequent Everolimus, the disease progressed. Notably, just 3 months after receiving fractionated stereotactic radiosurgery (FSR) for a cerebellar metastasis, spontaneous regression was observed at a distant skeletal site. This case highlights the significance of considering FSR as a therapeutic option for selected patients with ccRCC, as well as the potential role of an abscopal effect to impact the course of this aggressive malignancy."

Journal • Clear Cell Renal Cell Carcinoma • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor

OFF-LABEL TREATMENT AND ULTRARARE SARCOMAS - SINGLE INSTITUTION REPORT (CTOS 2025) - "Off-label therapies included: atezolizumab for alveolar ASPS (n=11); ivosidenib and pazopanib for chondrosarcoma (n=11; 2 and 9); imatinib and pembrolizumab for chordoma (n=14; 5 and 9); sirolimus for epithelioid hemangioendothelioma EHE (n=8); cabozantinib for Ewing sarcoma ES (n=11); crizotinib and alectinib for inflammatory myofibroblastic tumor IMT (n=1 and 2); sorafenib for osteosarcoma (n=7); pembrolizumab for sclerosing epithelioid sarcoma SEF (n=2)... Off-label treatments can improve outcomes in ultrarare sarcoma subtypes: ASPS (atezolizumab), EHE (sirolimus), SEF (pembrolizumab). 3-year survival rates and median EFS surpass historical data. Younger patients show more aggressive disease with shorter EFS."

Clinical • Chordoma • Ewing Sarcoma • Oncology • Osteosarcoma • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

Small Desmoplastic Round Cell Tumor of the Kidney following Hodgkin Lymphoma: Case Report and Literature Review. (PubMed, Case Rep Oncol) - "The literature on renal DSRCT was reviewed, and we describe a patient with thoracic relapse of DSRCT following a primary renal lesion, initially treated with radical nephrectomy and 14 cycles of chemotherapy (doxorubicin 225 mg/m2, cyclophosphamide 11.4 g/m2, ifosfamide 63 g/m2, etoposide 3.5 g/m2, and vincristine 12 mg/m2). The patient also had a distant history of nodular lymphocyte predominant Hodgkin lymphoma, in remission for 5 years following three cycles of doxorubicin 150 mg/m2, vincristine 8.4 mg/m2, prednisone 840 mg/m2, and cyclophosphamide 2.4 g/m2. Treatment with pazopanib for DSRCT was attempted but discontinued due to significant toxicity. DSRCT remains a highly aggressive malignancy with limited treatment options and poor prognosis. Our case highlights the rarity of primary renal DSRCT and its potential for thoracic relapse, further emphasizing the challenges in disease management."

Journal • Hematological Malignancies • Hodgkin Lymphoma • Kidney Cancer • Lymphoma • Nodular Lymphocyte Predominant Hodgkin Lymphoma • Oncology • Pediatrics • Renal Cell Carcinoma • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

Primary Monophasic Synovial Sarcoma of the Plantar Foot with Pulmonary Metastases in a Young Adult: A Rare Case Report. (PubMed, Case Rep Oncol) - "He was started on systemic chemotherapy (ifosfamide, gemcitabine, and taxane), followed by targeted therapy (pazopanib). This case illustrates a rare and aggressive presentation of synovial sarcoma arising in the plantar foot, emphasizing the importance of including synovial sarcoma in the differential diagnosis of soft tissue masses in distal extremities. Early imaging, accurate histopathological assessment, and a multidisciplinary treatment approach are essential for optimizing outcomes in such high-grade, metastatic presentations."

Journal • Oncology • Pain • Sarcoma • Soft Tissue Sarcoma • Solid Tumor • Synovial Sarcoma

Pazopanib therapy for children, adolescents, and young adults with relapsed and refractory sarcomas. (PubMed, Jpn J Clin Oncol) - "In a small cohort of children and adolescent and young adult patients with heavily pretreated relapsed or refractory sarcoma, pazopanib may be a feasible option. Further research on optimal therapeutic timing and the target population for pazopanib's indication is required."

Journal • Alveolar Soft Tissue Sarcoma • Leiomyosarcoma • Oncology • Osteosarcoma • Pediatrics • Respiratory Diseases • Rhabdomyosarcoma • Sarcoma • Solid Tumor

Chemotherapy Strategies and Their Efficacy for Mesenchymal Chondrosarcoma. (PubMed, Curr Oncol) - "Trabectedin demonstrates low disease control rate in translocation-related sarcomas, including few MCS cases. Anti-angiogenic tyrosine kinase inhibitors, such as apatinib and pazopanib, demonstrate activity in chondrosarcoma, but MCS-specific data are lacking. IDH1 inhibition benefits conventional subtypes rather than MCS. Early immunotherapy experience is limited, but pathway-directed strategies targeting BCL2 and PI3K-mTOR warrant evaluation."

Journal • Review • Oncology • Sarcoma • Solid Tumor • BCL2 • HEY1 • IDH1 • NCOA2

Pathological complete response to pembrolizumab in recurrent retroperitoneal dedifferentiated liposarcoma with high tumor mutational burden: a case report. (PubMed, World J Surg Oncol) - "This is the first reported case of recurrent retroperitoneal DDLPS with high TMB achieving pCR to pembrolizumab. High TMB and high TAM density in the tumor microenvironment may be predictive biomarkers for the response to ICIs in DDLPS."

Biomarker • IO biomarker • Journal • Tumor mutational burden • Genito-urinary Cancer • Immunology • Inflammatory Arthritis • Liposarcoma • Oncology • Peritoneal Cancer • Prostate Cancer • Retroperitoneal Sarcoma • Rheumatology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor • PD-L1 • TMB

BRCA2-MUTATED CONVENTIONAL CHONDROSARCOMA RESPONDING TO PARP INHIBITION: A CASE REPORT (CTOS 2025) - "She received multiple lines of systemic therapy, including anthracycline-based chemotherapy and pazopanib...Based on this finding, treatment with the PARP inhibitor olaparib was initiated as fourth-line therapy in July 2024. Despite prior disease progression on three lines of systemic therapy, olaparib led to a partial radiological response per RECIST criteria, with up to a 50% reduction in pulmonary lesions at the first disease assessment, 2 months after treatment initiation... This case highlights the potential sensitivity of BRCA-mutated conventional chondrosarcomas to PARP inhibition. It reinforces the rationale for tumor-agnostic approaches in molecularly selected sarcoma patients and supports further investigation of homologous recombination deficiency as a therapeutic target in bone sarcomas. Evaluation of the patient's germline BRCA2 status is ongoing to better characterize the biological context of this response."

Case report • Clinical • Oncology • Osteosarcoma • Sarcoma • Solid Tumor • BRCA • BRCA2 • CDKN2A • COL2A1 • HRD • IDH1 • IDH2

A novel disulfidptosis-related lncRNA signature to predict prognosis and immune response of cervical cancer. (PubMed, Medicine (Baltimore)) - "The low-risk group exhibited improved survival outcomes and increased sensitivity to immunotherapy, whereas the high-risk group showed heightened sensitivity to to bexarotene, bicalutamide, embelin, FH535, and pazopanib. Quantitative PCR results indicated that ILF3-DT and PPP1R14B-AS1 were downregulated in CC tissues, whereas RUSC1-AS1 was upregulated. In conclusion, we developed a novel prognostic risk signature based on 9 disulfidptosis-related lncRNAs, which may serve as an independent predictor of immunotherapy response and chemotherapy sensitivity in CC."

Biomarker • IO biomarker • Journal • Cervical Cancer • Oncology • Solid Tumor • PPP1R14B • RUSC1-AS1

TREATMENT OF ADVANCED EPITHELIOID SARCOMA WITH COMBINATION TAZEMETOSTAT AND PEMBROLIZUMAB (CTOS 2025) - "Here we present data from two patients at a single institution treated with tazemetostat in combination with immune checkpoint inhibitor therapy. We conducted a retrospective review of two patients with advanced ES treated with combination tazemetostat and pembrolizumab (PD-1 inhibitor) 200 mg every three weeks at our institution. Patient A, a 38-year-old man, presented with ES of the right hemipelvis, treated with neoadjuvant adriamycin/cisplatin, surgery, and adjuvant radiation (RT). Four years later found to have metastatic disease, treated with gemcitabine/paclitaxel, pazopanib, and targeted RT...Two years later found to have metastatic disease, treated with vincristine/cyclophosphamide/ifosfamide/etoposide, gemcitabine/docetaxel, pazopanib, and targeted RT... In both cases, pembrolizumab was added to tazemetostat at progression, yielding sustained and durable response for treatment of ES. This demonstrates the potential for combination epigenetic regulation and..."

Metastases • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

COMBINATION CHEMOTHERAPY WITH GEMCITABINE AND PACLITAXEL: A HIGHLY EFFICACIOUS AND BETTER TOLERATED OPTION FOR ANGIOSARCOMA (CTOS 2025) - "Current guidelines recommend single-agent Paclitaxel or Gemcitabine-based regimens, but Gemcitabine/Docetaxel causes significant myelosuppression and QoL-impacting toxicities...Pt has had clinical response and has continued on this regimen for residual disease for the last 6 months with minimal toxicities after progressing on prior treatments with Pazopanib and Pembrolizumab.Pt B (49F, metastatic breast AS): Diagnosed with AS 3 years ago and has lung and bone mets and has had clinical response and continuing on treatments for residual disease for 7 months with minimal toxicities after progressing on Doxorubicin and Pembrolizumab.Pt C (46F, forearm epithelioid AS): Developed extensive AS in background of right arm lymphedema... In all cases, Gemcitabine/Paclitaxel led to significant clinical responses and decreased toxicities compared to standard treatments (single-agent Paclitaxel, Anthracycline, other Gemcitabine regimens). Pts A and B have stable disease, and Pts C and..."

Angiosarcoma • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

Neoadjuvant tislelizumab plus pazopanib in renal cell carcinoma with venous tumour thrombus: A retrospective study. (PubMed, BJUI Compass) - "No grade 4-5 TRAEs or deaths were observed. The neoadjuvant combination of tislelizumab and pazopanib effectively reduced tumour size and thrombus length, narrowing the surgical scope and potentially leading to better postoperative outcomes."

Journal • Retrospective data • Dermatology • Fatigue • Genito-urinary Cancer • Hepatology • Oncology • Pruritus • Renal Cell Carcinoma • Solid Tumor • Thrombosis