Current Treatment Options in Oncology最新文献

{"title":"Dermatofibrosarcoma Protuberans (DFSP): Current Treatments and Clinical Trials.","authors":"Piotr Remiszewski, Agata Pisklak, Kinga Filipek, Mateusz J Spałek, Anna Szumera-Ciećkiewicz, Bartłomiej Szostakowski, Maria Krotewicz, Anna M Czarnecka","doi":"10.1007/s11864-025-01348-y","DOIUrl":"https://doi.org/10.1007/s11864-025-01348-y","url":null,"abstract":"<p><strong>Opinion statement: </strong>Dermatofibrosarcoma protuberans (DFSP) is a rare soft tissue sarcoma with an incidence of 0.008 to 0.045 cases per 100,000 per year, accounting for less than 1% of all soft tissue sarcomas. It mainly affects adults aged 30-50 years, usually on the trunk and proximal extremities. DFSP is Locally aggressive, with metastases occurring in up to 5% of cases, typically < 1%. Diagnosis is based on histology, including CD34 antigen expression and COL1A1-PDGFB fusion detection by FISH or RT-PCR. Mohs micrographic surgery is the mainstay of treatment, ensuring clear margins to minimise recurrence. Radiotherapy is used as adjuvant or preoperative therapy to improve Local control. Imatinib, a tyrosine kinase inhibitor, is highly effective in unresectable or metastatic DFSP, especially in cases with PDGFB mutations, achieving disease control in over 70% of patients and partial responses in 36%. The 10-year overall survival rate is 90.7%, although the fibrosarcomatous variant (DFSP-FS) has worse outcomes, with a 5-year Local progression-free survival rate of 33%. Rare metastases to the lungs, lymph nodes or brain are treated surgically; chemotherapy remains ineffective. We comprehensively reviewed the clinical data regarding DFSP, highlighting subtype differences (myxoid, pigmented, myoid, granular cell, sclerosing, atrophic, and fibrosarcomatous), as well as reconstruction methods.</p>","PeriodicalId":50600,"journal":{"name":"Current Treatment Options in Oncology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145213839","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Update in the Management of ACTH-Secreting Gastroenteropancreatic and Thoracic Neuroendocrine Neoplasms.","authors":"Marina Tsoli, Anat Bel-Ange, Karine Atlan, Simona Ben-Haim, Gregory Kaltsas, Simona Grozinsky-Glasberg","doi":"10.1007/s11864-025-01354-0","DOIUrl":"https://doi.org/10.1007/s11864-025-01354-0","url":null,"abstract":"<p><strong>Opinion statement: </strong>The adrenocorticotropic hormone (ACTH)-secreting neuroendocrine neoplasms (NENs) represent a rare but frequently severe disease that is associated with poor prognosis, if not adequately managed. Treatment strategies vary according to the characteristics of the tumor and the severity of hypercortisolism. The optimal treatment is surgical resection of the NEN with a curative intent. In unresectable or metastatic tumors, medical control of hypercortisolism with concomitant treatment of NENs based on current guidelines is suggested. Steroidogenesis inhibitors are the principal choice to control hypercortisolism while bilateral adrenalectomy should be considered in cases of failure to control severe life-threatening hypercortisolism. Additionally, amongst the various anti-tumor systemic treatment options, peptide receptor radionuclide therapy (PRRT) seems to also be an effective option for the control of hypercortisolism. Preventive and curative treatment of cortisol-induced complications and comorbidities is also important to reduce morbidity and mortality. Overall, the management of ACTH secreting NENs may be very complex and requires an individualized approach in a multidisciplinary context to achieve the best and timely outcome for the patient.</p>","PeriodicalId":50600,"journal":{"name":"Current Treatment Options in Oncology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145213820","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Dermatofibrosarcoma Protuberans (DFSP): Diagnostics and Molecular Pathology.","authors":"Piotr Remiszewski, Joanna Taczała, Marcin Rosiński, Anna Szumera-Ciećkiewcz, Bartłomiej Szostakowski, Anna M Czarnecka","doi":"10.1007/s11864-025-01350-4","DOIUrl":"https://doi.org/10.1007/s11864-025-01350-4","url":null,"abstract":"<p><strong>Opinion statement: </strong>Dermatofibrosarcoma protuberans (DFSP) is a rare, low-grade mesenchymal neoplasm that arises in the dermis and subcutaneous tissue. Clinically, DFSP presents as a slow-growing cutaneous plaque or nodular mass that is often initially mistaken for a benign dermatological condition, such as a keloid, a dermatofibroma or morphea. Due to its local aggressiveness and proclivity for subclinical spread and recurrence, accurate diagnosis and complete surgical excision are critical. Although DFSP rarely metastasises, its morbidity primarily stems from locally invasive growth and potential disfigurement due to extensive resections. The pathognomonic COL1A1-PDGFB fusion, which can be detected using fluorescence in situ hybridisation (FISH) or reverse transcription polymerase chain reaction (RT-PCR), sustains autocrine PDGFRβ activation and can be used as a diagnostic marker and a target for the drug imatinib. Emerging genomic studies have revealed additional fusions, such as COL1A2-PDGFB and FBN1-CSAD, as well as mutations, such as CDKN2A/B deletions, that correlate with more aggressive, fibrosarcomatous (FS) variant. We reviewed the diagnostics of DFSP, including histopathology, immunohistochemistry (e.g. CD34, factor XIIIa, S100 and PRAME) as well as clinical presentation and recommended imaging modalities (e.g. ultrasound, MRI and PET/CT). To provide a better understanding of DFSP we discussed the molecular basis of DFSP, including the main genetic drivers and downstream signalling pathways, such as Ras-MAPK, PI3K-Akt and FGFR. Moreover, as there is no definitive staging system for DFSP, we proposed a novel one, integrating the presence of FS differentiation.</p>","PeriodicalId":50600,"journal":{"name":"Current Treatment Options in Oncology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145202049","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Advancing Bladder Cancer Management: The Role of Neoadjuvant and Adjuvant Therapies and Biomarkers in Muscle Invasive Bladder Cancer.","authors":"Lingbin Meng, Adam Khorasanchi, Rohit Jain","doi":"10.1007/s11864-025-01355-z","DOIUrl":"https://doi.org/10.1007/s11864-025-01355-z","url":null,"abstract":"<p><strong>Opinion statement: </strong>The management of muscle-invasive bladder cancer (MIBC) is evolving rapidly, with the integration of neoadjuvant and adjuvant therapies and biomarker-driven patient selection now essential to refining treatment decisions. While cisplatin-based neoadjuvant chemotherapy has been the standard of care, its underutilization due to toxicity and patient ineligibility underscores the need for alternative strategies. Immune checkpoint inhibitors and targeted therapies, particularly fibroblast growth factor receptor (FGFR) inhibitors and antibody-drug conjugates (ADCs) like enfortumab vedotin (Nectin-4-directed) and disitamab vedotin (human epidermal growth factor receptor 2 [HER2]-directed), have transformed the management of metastatic urothelial carcinoma and are now being investigated in MIBC. The next challenge is to deploy these agents rationally by using robust biomarkers. FGFR3 alterations are predictive of response to FGFR inhibitors, whereas HER2 over-expression is chiefly prognostic but may also predict benefit from HER2-targeted ADCs. Circulating tumor DNA (ctDNA) is both prognostic and predictive, guiding dynamic therapy escalation when positive and potential de-escalation when negative in ongoing perioperative trials. In the adjuvant setting, immune-checkpoint blockade has begun to change practice: nivolumab in CheckMate 274 and pembrolizumab in the Alliance AMBASSADOR study each produced a statistically significant improvement in disease-free survival for high-risk patients after radical cystectomy, with overall survival (OS) data still Maturing. More recently, the phase 3 NIAGARA trial showed that adding perioperative durvalumab to neoadjuvant gemcitabine/cisplatin, followed by surgery and adjuvant durvalumab, conferred significant gains in both event-free survival and OS compared with chemotherapy alone. ctDNA's role as a marker of minimal residual disease is especially compelling. Trials, such as IMvigor010, have laid the foundation for ctDNA's utility as an MRD Marker, while the IMvigor 011 and VOLGA trials are utilizing ctDNA-driven treatment escalation or de-escalation to enable appropriate patient selection. Moving forward, the integration of multi-omics technologies, liquid biopsies, and adaptive trial designs will be crucial in optimizing treatment strategies. Challenges remain in standardizing biomarker assays, validating their predictive value, and translating findings into routine clinical practice. Collaborative efforts and large-scale prospective studies are necessary to bridge existing gaps and advance precision medicine tailored for MIBC.</p>","PeriodicalId":50600,"journal":{"name":"Current Treatment Options in Oncology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145151636","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"PAI-1 in Skin Malignancies: a Central Regulator of Tumor Progression and Therapeutic Resistance.","authors":"Taku Fujimura, Yoshihide Asano","doi":"10.1007/s11864-025-01357-x","DOIUrl":"https://doi.org/10.1007/s11864-025-01357-x","url":null,"abstract":"<p><strong>Opinion statement: </strong>Plasminogen activator inhibitor-1 (PAI-1) plays a multifaceted and central role in the tumor biology of various skin malignancies. Beyond its classical function in fibrinolysis, PAI-1 contributes to tumor progression by promoting immunosuppression, angiogenesis, cellular senescence, and tissue remodeling. Its expression is particularly elevated in aggressive disease stages across cutaneous melanoma, cutaneous squamous cell carcinoma (cSCC), cutaneous angiosarcoma (CAS), and mycosis fungoides (MF), and is associated with poor clinical outcomes. The ability of PAI-1 to induce senescence-associated secretory phenotype (SASP), modulate PD-L1 expression, and recruit tumor-associated macrophages (TAMs) and cancer-associated fibroblasts (CAFs) suggests a key role in shaping the immunosuppressive tumor microenvironment (TME). This positions PAI-1 as both a potential biomarker for disease progression and a therapeutic target for restoring immune responsiveness, especially in tumors resistant to immune checkpoint inhibitors (ICIs). The PAI-1 inhibitor TM5614 has demonstrated promising activity in early clinical studies, particularly in anti-PD-1-refractory melanoma, and is currently under evaluation in multiple Phase II and III trials. Future strategies should focus on patient stratification using biomarkers such as SASP factors and PAI-1 levels, as well as rational combination therapies targeting interconnected pathways like IL-17/IL-23, AhR, and senescence signaling. Overall, PAI-1 inhibition offers a novel and mechanistically grounded approach to improve outcomes in skin cancers characterized by therapy resistance and an immunosuppressive microenvironment.</p>","PeriodicalId":50600,"journal":{"name":"Current Treatment Options in Oncology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145151692","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Care of Older Adults with Acute Myeloid Leukemia: The Role of Geriatric Assessment.","authors":"Ojbindra Kc, Moataz Ellithi, Vijaya Raj Bhatt","doi":"10.1007/s11864-025-01341-5","DOIUrl":"https://doi.org/10.1007/s11864-025-01341-5","url":null,"abstract":"<p><strong>Opinion statement: </strong>Older adults with acute myeloid leukemia (AML) face disproportionately poor outcomes, driven by a combination of high-risk disease biology and functional vulnerabilities that are often overlooked by conventional oncology assessments. Geriatric assessment (GA) offers a multidimensional approach to uncover these impairments-spanning physical function, cognitive status, comorbidity burden, and emotional well-being-and provides critical information that independently predicts survival and treatment tolerance. Early studies demonstrate that GA can personalize treatment intensity decisions, inform supportive care interventions, and improve quality of life, yet its incorporation into routine AML care remains limited. Barriers to broader implementation include the need for streamlined workflows, clinician training, and large-scale validation. Future efforts should focus on integrating GA with biological markers of aging and disease to refine risk stratification, and leveraging digital tools and artificial intelligence to enhance feasibility and scalability. Ultimately, optimizing the management of older adults with AML will require a personalized, goal-concordant approach that combines GA-driven risk assessment with emerging therapeutic and supportive care strategies, ensuring that treatment intensity is matched to both disease biology and individual patient resilience.</p>","PeriodicalId":50600,"journal":{"name":"Current Treatment Options in Oncology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145132420","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Perioperative Immune Checkpoint Inhibitors for the Treatment of Locally Advanced Gastric and Gastroesophageal Junction Cancer: Current Status and Future Prospects.","authors":"Siyuan Cui, Kui Zhao, Na Wang, Yuan Meng, Cheng Zhao, Honggen Liu, Wen Xin, Fanming Kong","doi":"10.1007/s11864-025-01353-1","DOIUrl":"https://doi.org/10.1007/s11864-025-01353-1","url":null,"abstract":"<p><strong>Opinion statement: </strong>Novel strategies utilizing immune checkpoint inhibitors (ICIs) have emerged over the past several years and substantially changed the treatment landscape of advanced gastric cancer (GC). Based on the encouraging results achieved, numerous clinical studies have been conducted to identify the feasibility and efficacy of integrating ICIs into the perioperative treatment of locally advanced gastric and gastroesophageal junction cancer (GC/GEJC). Initial clinical trials have indicated that the addition of immunotherapy to chemotherapy, concurrent chemoradiotherapy, or chemotherapy combined with anti-angiogenic therapy in the perioperative setting significantly improves the pathological remission of patients, thereby promising to improve the prognosis. However, in the absence of strong evidence from long-term follow-up data, these approaches have yet to be entirely translated into clinical practice. Therefore, it is recommended that the aforementioned patients be prioritized for participation in clinical trials. In addition, based on the negative results of ATTRACTION-05, current evidence does not support the use of adjuvant immunotherapy in patients without neoadjuvant immunotherapy. For patients with dMMR/MSI-H disease, neoadjuvant immunotherapy has demonstrated excellent pCR rates, and the 2024 NCCN guidelines have recommended neoadjuvant or perioperative ICIs for these patients. In HER2-positive locally advanced GC/GEJC, neoadjuvant dual PD-1 and HER2 inhibition combined with chemotherapy has also shown promising prospects. However, the development of perioperative ICIs has also posed formidable therapeutic challenges. Given the high heterogeneity of GC, there is an urgent need to select patients likely to benefit from ICIs with precision through microenvironment analysis, molecular biomarker screening, and clinical subgroup analysis. In parallel, the benefits of ICIs in locally advanced GC/GEJC should be carefully weighed against their associated adverse effects and significant financial toxicity.</p>","PeriodicalId":50600,"journal":{"name":"Current Treatment Options in Oncology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145071047","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"From Fallopian Tube to Ovarian Cancer: Understanding the Evaluation and Management of Serous Tubal Intraepithelial Carcinoma Lesions.","authors":"Vinita Popat, Ernest Han","doi":"10.1007/s11864-025-01346-0","DOIUrl":"https://doi.org/10.1007/s11864-025-01346-0","url":null,"abstract":"<p><strong>Opinion statement: </strong>Ovarian cancer, particularly high-grade serous carcinoma (HGSC), remains a leading cause of mortality in gynecologic oncology. Emerging research identifies serous tubal intraepithelial carcinoma (STIC) as a precursor lesion in many HGSC cases, highlighting its role in ovarian cancer pathogenesis and prevention. Management of STIC is challenging, as there is only limited data available to guide clinical decision-making. For average-risk women, opportunistic salpingectomy is increasingly being adopted during routine procedures such as hysterectomy or cesarean section. This intervention has demonstrated significant potential in reducing ovarian cancer incidence while maintaining safety and feasibility. For high-risk individuals, particularly BRCA mutation carriers, risk-reducing salpingo-oophorectomy (RRSO) remains the gold standard. RRSO significantly lowers ovarian cancer risk, though alternative approaches like salpingectomy alone or radical fimbriectomy are under investigation to preserve ovarian function in younger patients. To improve STIC detection, SEE-FIM pathology protocol is recommended when patients are undergoing risk-reducing surgery to prevent ovarian cancer, but challenges such as diagnostic variability and limited data persist. When STIC is detected incidentally, management varies based on risk factors and lesion characteristics. Genetic counseling and testing are essential when STIC is identified, as hereditary predisposition may guide further management. Surgical management is advised for cases of STIC with microinvasive carcinoma, but routine use of surgical management for STIC is not clearly defined in the literature. Bilateral oophorectomy is generally recommended when STIC is identified, and adnexal structures have not yet been removed. Chemotherapy is not recommended for treatment of STIC. Surveillance is suggested when STIC has been diagnosed, but there are no set guidelines as to the frequency and type of monitoring. Future directions include refining molecular profiling to predict progression and conducting randomized studies to establish evidence-based guidelines. Multidisciplinary collaboration is essential to optimize prevention and treatment, ultimately reducing HGSC incidence and improving patient outcomes.</p>","PeriodicalId":50600,"journal":{"name":"Current Treatment Options in Oncology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145001798","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Revolutionizing Breast Cancer Therapeutics: Intersecting Frontiers of Precision Medicine, Nanotechnology, and Drug Delivery Innovations.","authors":"Anandini Chattopadhyay, Falak Goyal, Abhishek Sehrawat, Inderpal Singh Sidhu, Vikramdeep Monga, Gurjit Kaur Bhatti, Jasvinder Singh Bhatti","doi":"10.1007/s11864-025-01343-3","DOIUrl":"10.1007/s11864-025-01343-3","url":null,"abstract":"<p><strong>Opinion statement: </strong>The ongoing challenge of addressing breast cancer, one of the most prevalent cancers and a principal cause of mortality among women globally, has reached a critical juncture with the advent of precision medicine and the promise of nanotechnology. As the scientific community's understanding of breast cancer's genomic landscape has deepened, it has become evident that a one-size-fits-all approach to treatment is obsolete. The evolution from rudimentary immunohistochemical classifications to intricate molecular profiling has ushered in an era where therapy is increasingly tailored to the individual's genetic makeup, environmental factors, and lifestyle choices. This shift towards personalized, biomarker-driven treatments not only aims to enhance prognosis but also to minimize adverse effects by meticulously matching therapy to the unique molecular characteristics of each tumor. The integration of nanotechnology, particularly through the deployment of nanoparticles for targeted drug delivery and nano-theranostics, represents a groundbreaking stride in oncological treatment. This convergence of precision medicine and nanotechnology in breast cancer care suggests a future where combination therapies and multifunctional approaches could potentially outsmart drug resistance and augment treatment efficacy. However, the path forward is fraught with challenges such as overcoming inherent tumor heterogeneity and improving the accessibility of cutting-edge treatments. The exploration of these innovative strategies in breast cancer therapeutics underscores the critical need for a multifaceted approach to cancer care, emphasizing the potential of these advances to revolutionize treatment paradigms and offer new hope to patients.</p>","PeriodicalId":50600,"journal":{"name":"Current Treatment Options in Oncology","volume":" ","pages":"775-796"},"PeriodicalIF":4.7,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144876631","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immunotherapy in the Treatment of Undifferentiated Pleomorphic Sarcoma and Myxofibrosarcoma.","authors":"Jerry T Wu, Elizabeth Nowak, Jarrell Imamura, Jessica Leng, Dale Shepard, Shauna R Campbell, Jacob Scott, Lukas Nystrom, Nathan Mesko, Gary K Schwartz, Zachary D C Burke","doi":"10.1007/s11864-025-01349-x","DOIUrl":"https://doi.org/10.1007/s11864-025-01349-x","url":null,"abstract":"<p><strong>Opinion statement: </strong>Undifferentiated pleomorphic sarcoma (UPS) and myxofibrosarcoma (MFS) are among the most common adult soft tissue sarcoma (STS) subtypes. Due to their high genetic complexity, heterogeneity, and lack of specific genetic alterations, no consistent molecular targets for targeted therapy have been identified for UPS and MFS. Recently, immune checkpoint inhibition (ICI) has emerged as a promising treatment modality for UPS and MFS. However, the efficacy of ICI in UPS and MFS remains far lower than in other cancers such as melanoma. Strategies to increase the efficacy of ICI, including selecting patients based on putative biomarkers and combining ICI with chemotherapy, targeted therapies, and/or radiation therapy, are currently in clinical development. In this review, we first summarize the clinical characteristics of UPS and MFS, examining the tumor microenvironment (TME) and its effect on the efficacy of ICI. We then review putative biomarkers of ICI response and highlight clinical trials testing ICI in patients with UPS and MFS. Finally, we discuss other forms of immunotherapy for UPS and MFS currently under preclinical investigation. The combination of ICI plus radiation therapy appears to have benefit for patients with localized UPS and MFS. ICI should be considered for patients with advanced or unresectable UPS and MFS, especially those with potential biomarkers of response such as tertiary lymphoid structures (TLS). However, singular biomarkers such as TLS may prove inadequate to predict ICI response; more accurate prediction will likely require a panel of biomarkers including TLS, immune cell infiltration, PD-L1 expression, and other TME components.</p>","PeriodicalId":50600,"journal":{"name":"Current Treatment Options in Oncology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144976773","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}