INTRODUCTION: Targeting the DNA damage response (DDR) has emerged as a promising therapeutic strategy in oncology. This review highlights the growing clinical relevance of DDR inhibitors beyond PARP inhibitors, focusing...INTRODUCTION: Targeting the DNA damage response (DDR) has emerged as a promising therapeutic strategy in oncology. This review highlights the growing clinical relevance of DDR inhibitors beyond PARP inhibitors, focusing on novel targets, biomarker-driven patient selection, and rational combination strategies. AREAS COVERED: A comprehensive literature search was conducted using PubMed and ClinicalTrials.gov through May 2025, using terms such as 'DDR inhibitors,' 'synthetic lethality,' and the names of specific DDR targets. This review summarizes preclinical and clinical data on next-generation DDR inhibitors, including agents targeting , and . It discusses mechanistic rationale, clinical activity, and safety profiles, with emphasis on combination strategies involving chemotherapy, immunotherapy, radiotherapy, and other DDR agents. Key challenges such as resistance, cumulative toxicity, and the need for predictive biomarkers are also addressed. EXPERT OPINION: DDR-targeting agents hold significant promise, especially when guided by predictive biomarkers and combined with other therapies. As these agents move into later-phase trials, future development should emphasize biomarker-driven design, toxicity mitigation through dose optimization, and expansion into non-canonical tumor types to maximize clinical impact.
INTRODUCTION: Hepatocellular carcinoma (HCC), a common and aggressive liver cancer, frequently manifests at an advanced stage, restricting the effectiveness of standard treatments. Recent advancements in nanotechnology,...INTRODUCTION: Hepatocellular carcinoma (HCC), a common and aggressive liver cancer, frequently manifests at an advanced stage, restricting the effectiveness of standard treatments. Recent advancements in nanotechnology, particularly in liposome-based delivery systems, offer promising approaches to enhance treatment efficacy and immunological regulation in hepatocellular carcinoma (HCC). AREAS COVERED: This study emphasizes the diverse functions of liposomes in augmenting drug solubility, targeting neoplastic tissues, increasing immunological responses, and surmounting biological barriers. It investigates the synergy of liposomal platforms and immunotherapies, including checkpoint inhibitors, and their incorporation with locoregional treatments for advanced-stage hepatocellular carcinoma (HCC). EXPERT OPINION: Liposomes are a revolutionary approach in cancer immunotherapy. Innovations like PEGylation, surface ligand modification, and dual-drug encapsulation have significantly enhanced treatment outcomes in preclinical and early clinical contexts. Advancements, especially in patient-specific and stimuli-responsive systems, may transform the future of HCC care.
INTRODUCTION: Persistent human papillomavirus (HPV) infection is the causative factor for nearly all high-grade cervical lesions (CIN2/3) and invasive cervical cancers. HPV testing therefore plays a key role in the follo...INTRODUCTION: Persistent human papillomavirus (HPV) infection is the causative factor for nearly all high-grade cervical lesions (CIN2/3) and invasive cervical cancers. HPV testing therefore plays a key role in the follow-up of patients treated surgically or managed conservatively. AREAS COVERED: To optimize HPV testing in follow-up, we identified key clinical scenarios - conservative management of high-grade lesions, surveillance after conization or hysterectomy, post-surgical follow-up of cervical carcinoma, and monitoring after primary radiation therapy. We reviewed the evidence to propose a strategy for integrating HPV testing into follow-up. EXPERT OPINION: HPV testing shows high sensitivity and nearly 100% negative predictive value after conization for cervical high-grade lesions. Post-surgical HPV status is a more reliable predictor of recurrence than histopathological margin assessment. A negative result identifies patients with very low relapse risk. However, the lifetime risk of recurrent dysplasia remains increased and mainly depends on HPV status. Conversely, after fertility-sparing surgery for T1b cervical cancer, HPV testing alone is insufficient to rule out all recurrences. Its role is also unclear in patients after hysterectomy for invasive cancer and in those whose preinvasive lesions regressed spontaneously.
INTRODUCTION: Non-small cell lung cancer (NSCLC) accounts for the majority of lung cancer cases and remains the leading cause of cancer-related mortality worldwide. Accurate assessment of predictive biomarkers, such as A...INTRODUCTION: Non-small cell lung cancer (NSCLC) accounts for the majority of lung cancer cases and remains the leading cause of cancer-related mortality worldwide. Accurate assessment of predictive biomarkers, such as ALK and PD-L1, is essential for personalized therapy and to guide clinical decisions. Companion diagnostic (CDx) tests play a crucial role in this context. AREAS COVERED: PD-L1 and ALK are key predictive biomarkers for NSCLC treatment. PD-L1 expression on tumor cells (TCs) helps predict responses to anti - PD-1 and anti - PD-L1 treatments, with assays like 22C3 and SP263. ALK rearrangements, detected by immunohistochemistry (IHC) and fluorescence in situ hybridization (FISH), helps predict responses to ALK inhibitors like crizotinib. IHC is a practical and cost-effective screening method but combining IHC with molecular techniques such as next-generation sequencing (NGS) or FISH enhances accuracy. EXPERT OPINION: PD-L1 and ALK are crucial predictive biomarkers for patients with NSCLC. PD-L1 IHC expression predicts response to immune checkpoint inhibitors (ICIs), but its predictive value is not perfect, with some PD-L1-negative patients benefiting from specific therapy. ALK IHC, with confirmatory FISH or NGS, guides tailored treatment. In this context, an integrated predictive approach enables the best clinical and therapeutic management of patients with NSCLC.
INTRODUCTION: Human papilloma virus positive (HPV+) OPSCC has a favorable prognosis compared to its HPV- carcinomas but a significant proportion of patients will experience disease recurrence and death. Enhancement of su...INTRODUCTION: Human papilloma virus positive (HPV+) OPSCC has a favorable prognosis compared to its HPV- carcinomas but a significant proportion of patients will experience disease recurrence and death. Enhancement of surveillance strategies after treatment for primary HPV + OPSCC is of great importance. AREAS COVERED: The aim of this paper is to provide an updated synthesis of the current state of evidence on the surveillance of HPV+ OPSCC and provide guidance on future directions on this topic. Basic and clinical research efforts are directed on various aspects of surveillance, including but not limited to patterns of recurrence, radiology, circulating tumor HPV DNA, and follow-up strategies. EXPERT OPINION: The majority of OPSCC recurrences are detected by patients or during follow-up imaging. Rarely, recurrences are detected during the visits. Routine clinical surveillance can be scheduled once every 3 months in the first year, once every 6 months in the second year, and then annually up to year 5. Regarding imaging surveillance, positron emission tomography-computed tomography (PET-CT) of the head, neck, and chest is the most widely recommended modality. Current guidelines recommend one imaging at 3 months post-treatment, with subsequent imaging at the discretion of the clinician. Circulating HPV-DNA is an emerging technology that can be integrated soon into daily clinical practice.
INTRODUCTION: The incidence of intrahepatic cholangiocarcinoma has continued to increase, with dismal rates of overall survival. While upfront surgery remains the mainstay of resectable intrahepatic cholangiocarcinoma, s...INTRODUCTION: The incidence of intrahepatic cholangiocarcinoma has continued to increase, with dismal rates of overall survival. While upfront surgery remains the mainstay of resectable intrahepatic cholangiocarcinoma, systemic therapy has gained increasing acceptance worldwide, especially for advanced and metastatic cholangiocarcinoma. AREAS COVERED: In this article, we review the different genetic mutations associated with cholangiocarcinoma, as well as recent advances made in the management of intrahepatic cholangiocarcinoma using therapies that directly target actionable mutations. We also review the clinical trials that led to the approval of these drugs, and the specific indications for their use. EXPERT OPINION: While significant progress has been made in identifying actionable mutations and developing drugs that target these mutations, several challenges exist in the management of intrahepatic cholangiocarcinoma using these targeted therapies. These challenges include issues with drug resistance, efficacy and cost. Furthermore, enrollment in clinical trials for cholangiocarcinoma is very limited and completed trials often lack the diversity needed to generalize their findings.
INTRODUCTION: Oral squamous cell carcinoma (OSCC) is a heterogeneous malignancy with rapid progression, limited therapeutic options, and poor prognosis. Epigenetic alterations - including DNA methylation, histone modific...INTRODUCTION: Oral squamous cell carcinoma (OSCC) is a heterogeneous malignancy with rapid progression, limited therapeutic options, and poor prognosis. Epigenetic alterations - including DNA methylation, histone modifications, and noncoding RNA (ncRNA) regulation - are key drivers of oncogene activation and tumor suppressor silencing, offering novel therapeutic opportunities. AREAS COVERED: This review summarizes the major epigenetic mechanisms in OSCC and highlights recent progress in therapeutic agents and molecular targets. In particular, we emphasize ncRNA-based modulators, such as microRNAs (miRNAs) and long noncoding RNAs (lncRNAs), which play crucial roles in epigenetic regulation and represent promising therapeutic candidates. Innovative delivery strategies, including exosome-mediated transfer and nanoparticle formulations, are discussed for their potential to improve treatment specificity and overcome resistance. An updated summary of clinical trials targeting epigenetic regulators in OSCC is also presented, along with possible combination strategies to enhance clinical outcomes. EXPERT COMMENTARY: Epigenetic therapies hold significant promise for precision treatment of OSCC. Combining targeted agents with advanced delivery systems may accelerate clinical translation, while validated biomarkers could support early detection, risk stratification, and personalized therapy.A literature search was conducted in PubMed and ClinicalTrials.gov from January 2002 to July 2025 to identify high-quality studies on epigenetic therapies in OSCC.
BACKGROUND: Palbociclib is a cornerstone treatment for patients with hormone receptor-positive and human epidermal growth factor receptor 2-negative, metastatic breast cancer. RESEARCH DESIGN AND METHODS: This retrospect...BACKGROUND: Palbociclib is a cornerstone treatment for patients with hormone receptor-positive and human epidermal growth factor receptor 2-negative, metastatic breast cancer. RESEARCH DESIGN AND METHODS: This retrospective cohort study included 140 patients who were resistant to endocrine therapy and received palbociclib in combination with endocrine therapy. It is aimed to evaluate the efficacy and tolerability of palbociclib and to identify factors influencing progression-free survival and overall survival. RESULTS: A total of 140 were enrolled in the study; 57 and 83 patients were primary and secondary resistance to endocrine therapy, respectively. In the whole cohort, regarding progression-free survival; low Ki-67 and incidence of moderate-to-severe (grade 3/4) neutropenia were associated with longer median progression-free survival with -values of 0.004 (3.7 vs. 8.4 months) and 0.026 (4.7 vs. 6.5 months), respectively. Regarding overall survival, duration on palbociclib (≥12 months) had longer overall survival compared with patients who received palbociclib <12 months ( = 0.0012). The overall survival was longer in the secondary-resistant group than in the primary-resistant group (log-rank test = 0.0003). CONCLUSION: In a metastatic setting, low Ki-67 and moderate-to-severe neutropenia are associated with better survival outcomes in endocrine resistant patients treated with palbociclib (Clinical trial registration: www.clinicaltrials.gov identifier is NCT06338644).
INTRODUCTION: Patients with advanced/metastatic non-small cell lung cancer (NSCLC) and programmed death ligand-1 (PD-L1) tumor cell expression <1% are difficult to treat, and an optimal treatment strategy for these patie...INTRODUCTION: Patients with advanced/metastatic non-small cell lung cancer (NSCLC) and programmed death ligand-1 (PD-L1) tumor cell expression <1% are difficult to treat, and an optimal treatment strategy for these patients is yet to be defined. AREAS COVERED: There have been significant advances in immunotherapy for NSCLC in the past decade. This article aims to answer the question of the optimal first-line treatment for patients with advanced/metastatic NSCLC and PD-L1 expression <1%, based on efficacy and safety data from phase 3 studies (published up to 31 December 2024). EXPERT OPINION: Current evidence from subgroup and exploratory analyses of phase 3 studies and indirect comparisons suggest that PD-1/PD-L1 inhibitors (with or without chemotherapy) combined with a cytotoxic T lymphocyte-associated protein-4 inhibitor or antiangiogenic therapy may provide the highest progression-free survival (PFS) and overall survival (OS) benefits in patients with newly diagnosed advanced/metastatic NSCLC and PD-L1 tumor cell expression <1%. Of these regimens, dual immunotherapy with nivolumab and ipilimumab combined with chemotherapy appeared to offer some advantages in terms of OS, PFS, objective response rates, and duration of response, relative to other treatment approaches. Well-designed, comparative studies are warranted to more definitively determine the best first-line treatment for these patients.
Mustafa A, Khandker SS, Farwa U
… +14 more, Saeed MH, Sarfraz M, Manan A, Gillani HI, Akbar A, Ejaz KF, Atta A, Atta M, Pranto AH, Gomes B, Lubaba A, Mohaimeen T, Abir SN, Saleem Khan M
INTRODUCTION: Hematopoietic stem cell transplantation (HSCT) is a widely used transplant method for different cancerous and non-cancerous conditions, particularly when conventional treatments fail. Again, Epstein-Barr Vi...INTRODUCTION: Hematopoietic stem cell transplantation (HSCT) is a widely used transplant method for different cancerous and non-cancerous conditions, particularly when conventional treatments fail. Again, Epstein-Barr Virus (EBV), one of the major contributors to gastric carcinoma can cause post-transplantation lymphoproliferative disease (PTLD) after transplantations. However, the global prevalence of EBV infection and associated PTLD in HSCT recipients is yet to be determined. METHODS: This research examined 33 studies selected from 941 articles across three databases (i.e. ScienceDirect, PubMed, and Google Scholar) to investigate the global epidemiology of EBV infection and associated PTLD in HSCT patients. RESULTS: The overall prevalence was calculated primarily as 27.0%; 95% CI: 24.5-29.5, and 23.7%; 95% CI: 19.3-28.3 after removing outlier studies. The analyzed studies were of high quality. Among different continents, North America was determined to have the highest prevalence rate (33.3%; 95%CI: 12.5-54.1) followed by Europe (27.8%; 95%CI: 23.3-32.2), and Asia (20.7%; 95%CI: 17.4-23.9). CONCLUSION: This investigation highlights the need for early detection of EBV infection and associated PTLD, personalized treatment strategies, and continued research into the condition's underlying mechanisms. Addressing these aspects can enhance outcomes for HSCT patients and contribute to advancements in transplant medicine. REGISTRATION: PROSPERO (CRD420250583221).
INTRODUCTION: Deep learning (DL) is transforming cancer research by enabling data-driven drug discovery. However, its clinical translation, particularly in endometrial cancer (EC), faces significant challenges. AREAS COV...INTRODUCTION: Deep learning (DL) is transforming cancer research by enabling data-driven drug discovery. However, its clinical translation, particularly in endometrial cancer (EC), faces significant challenges. AREAS COVERED: This review discusses recent DL applications across drug discovery stages in EC, including target identification, virtual screening, and de novo drug design. We highlight key obstacles that hinder clinical translation, such as data scarcity, limited model explainability, biological validation gaps, and regulatory uncertainty, and propose practical solutions. Literature was sourced from PubMed, Web of Science, and relevant AI repositories, with an emphasis on peer-reviewed studies from the past five years. EXPERT OPINION: Despite early success, DL must overcome multiple translational bottlenecks to impact EC therapeutics meaningfully. A multidisciplinary approach that incorporates data quality improvements, functional validation, regulatory engagement, and clinician-focused decision support is essential to fully realize the clinical promise of DL-driven drug discovery in EC.
Expert Rev Anticancer Ther
· 2025 Dec · PMID 40908721
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INTRODUCTION: Radiation-related cognitive decline (RRCD), characterized by a decline in cognitive functioning within domains, such as memory and executive function, is a known potential consequence of cranial radiation....INTRODUCTION: Radiation-related cognitive decline (RRCD), characterized by a decline in cognitive functioning within domains, such as memory and executive function, is a known potential consequence of cranial radiation. Older adults are disproportionately vulnerable to cognitive side effects of radiation (RT), and there can be significant impacts on quality of life and independence. Various mechanisms underlying the development of RRCD have been proposed but have not been specifically evaluated in older adults. AREAS COVERED: In this article, we review the studies that have evaluated cognitive effects of cranial radiation in older adults and discuss the mechanisms and factors that may lead to increased vulnerability of RRCD development in older adults. EXPERT OPINION: The review of the literature is limited by the variety of cognitive outcome measurements used as well as different ages evaluated. However, most studies support increased vulnerability to RRCD in older adults. No studies include geriatric assessment or other measures of biological age. Potential interventions include redefining whether different dose constraints are warranted in the older adult population, evaluating new medication interventions and utilizing radiation techniques that treat smaller volumes. Further research is needed to determine whether there is a corresponding reduction in RRCD.
BACKGROUND: Research on urothelial carcinoma of the bladder (UCUB) in non-Hispanic Asian American (NHAA) populations typically amalgamate all subgroups, masking significant intra-ethnic difference. This study aimed to ex...BACKGROUND: Research on urothelial carcinoma of the bladder (UCUB) in non-Hispanic Asian American (NHAA) populations typically amalgamate all subgroups, masking significant intra-ethnic difference. This study aimed to examine variations in UCUB characteristics and outcomes within NHAA populations. RESEARCH DESIGN AND METHODS: Patients with UCUB were identified from the Surveillance, Epidemiology, and End Results database. The NHAA cohort disaggregated into Chinese, Filipino, South Asian, Japanese, Korean, Vietnamese, and other Asian subgroups. Kaplan-Meier and Cox proportional hazards models were used to estimate unadjusted and adjusted overall survival (OS) and cancer-specific survival (CSS). RESULTS: NHAA patients ( = 2,686) exhibited the longest median OS (88 months) compared to other cohorts ( < 0.001). Among NHAA subgroups, five-year OS rates ranged from 50% in Filipino patients to 64% in other Asian groups. In adjusted analyses, Chinese (HR 0.84, 95% CI 0.74-0.94), Korean (HR 0.74, 95% CI 0.61-0.91), and other Asian (HR 0.68, 95% CI 0.56-0.82) patients exhibited significantly reduced mortality risk relative to Non-Hispanic White patients. CONCLUSIONS: Filipino Americans faced comparatively poorer survival, while Chinese and Korean Americans showed more favorable prognoses. These findings highlight the need for targeted, culturally tailored interventions and refined risk stratification to enhance equity in the management of UCUB.
Xu Y, Li R, Wang N
… +19 more, Guo Y, Wang C, Ren Q, Lu S, Bi S, Tian H, Guo X, Zou Y, Yuan L, She W, Sun H, Dong Y, Zhang C, Ma Y, Shang Z, Jiang Y, Lv W, Lv H, Zhang M
OBJECTIVES: Prostate cancer (PCa) is one of the most common malignancies in males worldwide. Therefore, conducting the latest and comprehensive assessment of PCa is important. RESEARCH DESIGN AND METHODS: Average annual...OBJECTIVES: Prostate cancer (PCa) is one of the most common malignancies in males worldwide. Therefore, conducting the latest and comprehensive assessment of PCa is important. RESEARCH DESIGN AND METHODS: Average annual percent change for age-standardized rate trend was calculated by Joinpoint. Pearson correlation analyzed the relationship between PCa and Sociodemographic Index (SDI). Causal associations of risk factors were examined by two-sample mendelian randomization. Bayesian age-period-cohort model was used to predict incidence trends to 2050. RESULTS: In 2021, the global incidence of PCa was 1,324,383, an increase of 161.53% since 1990. It was mainly concentrated in the age group of 65-79. From 1990 to 2021, the global incidence and prevalence increased slightly. PCa was more common in regions with a high SDI. Mortality was the highest in regions with a low SDI. Smoking and telomere length were found to be risk factors for PCa, and diet low in calcium and milk was also related to PCa. The prediction results revealed that the incidence of PCa will slightly decrease in the future. CONCLUSIONS: The disease burden of PCa gradually increased from 1990 to 2021. There were significant differences across countries, SDI regions, and age groups. By 2050, the incidence of this disease is expected to decrease.
BACKGROUND: Isocitrate dehydrogenase (IDH) wild-type (wt) glioblastoma is a biologically aggressive adult-type diffuse glioma with poor prognosis. Gustave Roussy Immune Score (GRIm-Score), reflecting systemic inflammatio...BACKGROUND: Isocitrate dehydrogenase (IDH) wild-type (wt) glioblastoma is a biologically aggressive adult-type diffuse glioma with poor prognosis. Gustave Roussy Immune Score (GRIm-Score), reflecting systemic inflammation and nutritional status, has shown prognostic relevance in several cancers. Its prognostic value in IDH-wt glioblastoma remains undefined. METHODS: This retrospective single-center study included 186 patients with histologically confirmed IDH-wt glioblastoma. GRIm-Score was calculated using pretreatment NLR, albumin, and lactate dehydrogenase (LDH) levels. Patients were grouped into low- and high-risk categories. Associations with overall survival (OS) and progression-free survival (PFS) were assessed using Kaplan-Meier and Cox regression analyses. Correlations with other inflammation-based indices, such as PILE, CAR, and PIV, were also examined. RESULTS: A high GRIm-Score was significantly associated with reduced OS and PFS. Patients with higher scores more frequently exhibited poor prognostic features, including advanced age, worse ECOG performance, limited treatment response, and subtotal resection. Multivariate analysis identified GRIm-Score as an independent prognostic factor, particularly when analyzed alongside CAR and PIV. Strong correlations were observed between GRIm-Score and other immuno-nutritional markers. CONCLUSIONS: GRIm-Score is a simple and reliable prognostic indicator in IDH-wt glioblastoma. Its routine use may improve risk stratification and inform therapeutic decisions. Further prospective multicenter validation is warranted.
BACKGROUND: We aimed to investigate the effect of RAD51 expression on pathological response and survival in gastric cancer patients receiving fluorouracil plus leucovorin, cisplatin and docataxel (FLOT) as neoadjuvant ch...BACKGROUND: We aimed to investigate the effect of RAD51 expression on pathological response and survival in gastric cancer patients receiving fluorouracil plus leucovorin, cisplatin and docataxel (FLOT) as neoadjuvant chemotherapy (NACT). RESEARCH DESIGN AND METHODS: RAD51 immunohistochemistry staining was performed in the endoscopy biopsies of the patients, and the pathological responses in the surgery of the patients after NACT were evaluated using the Ryan tumor regression score (RTRS). RESULTS: 89 patients participated in this study. The factors affecting the pathological responses of the patients after NACT were examined, patients with high RAD51 nuclear expression percentage (NEP) responded better to NACT ( = 0.020). RAD51 nuclear expression density (NED) ( = 0.127), age ( = 0.999), sex ( = 0.098), clinical stage ( = 0.540), tumor pathology ( = 0.999) did not affect the pathological response after NACT. Age, gender, clinical stage, tumor pathology, RAD51 NEP or RAD51 NED did not affect DFS or OS. Patients with low RTRS had better DFS ( = 0.001) and OS ( = 0.009) results. CONCLUSIONS: As a result of our study, it was observed that patients with high RAD51 NEP had better pathological responses after NACT.
BACKGROUND: Lung adenocarcinoma (LUAD) is the most common subtype of lung cancer, with a low survival rate. TFB2M, a mitochondrial transcription factor, maintains normal mitochondrial function. Its role in LUAD is unclea...BACKGROUND: Lung adenocarcinoma (LUAD) is the most common subtype of lung cancer, with a low survival rate. TFB2M, a mitochondrial transcription factor, maintains normal mitochondrial function. Its role in LUAD is unclear. METHODS: We analyzed TFB2M expression in LUAD and normal tissues based on TCGA database. GSEA analyzed pathway enrichment. TFB2M-knockdown LUAD and control groups were constructed. Western blot detected levels of mitophagy- and ferroptosis-related proteins with/without mitophagy inhibitor (Mdivi-1, 10 μM). Malondialdehyde, glutathione, 4-hydroxynonenal, reactive oxygen species, and Fe levels were measured to evaluate ferroptosis. CCK-8, EdU experiments, and flow cytometry evaluated cell survival. Immunofluorescence detected co-localization of glutathione peroxidase 4 and mitochondrial outer membrane transferase 20. Mitochondrial-specific fluorescent probes evaluated mitochondrial changes. A LUAD xenograft mouse model was constructed, with tumor volume and weight (with/without mitophagy inhibitors, 50 mg/kg) measured. IHC detected TFB2M and ki67 expression. RESULTS: TFB2M was upregulated ( < 0.05), and enriched in ferroptosis and mitophagy-related pathways. Mitophagy inhibitors reversed the promotion of mitophagy and ferroptosis and the inhibition of cell proliferation conferred by TFB2M knockdown. In animal experiments, they weakened the inhibition of mitophagy and the alleviation of LUAD progression induced by TFB2M knockdown. CONCLUSION: TFB2M contributes to ferroptosis resistance in LUAD by suppressing mitophagy.
BACKGROUND: Desmoid tumors are rare, locally aggressive tumors that are often life-affecting with a significant burden on afflicted patients. While systemic therapy for progressive disease is often preferred, there is a...BACKGROUND: Desmoid tumors are rare, locally aggressive tumors that are often life-affecting with a significant burden on afflicted patients. While systemic therapy for progressive disease is often preferred, there is a paucity of studies comparing disease control rates of agents based on tumor location or other disease-related factors. METHODS: Dual-center retrospective analysis of disease control in patients with intra-abdominal (IA) and extra-abdominal (EA) desmoid tumors who received liposomal doxorubicin (LD). Disease control rate (DCR) was defined as stable disease (if evidence of progression prior to LD therapy) or partial response. RESULTS: 54 patients were included, 38.9% (21/54) with IA desmoid tumors and 35.2% (19/54) with Familial Adenomatous Polyposis (FAP)-associated desmoid tumors. DCRs for patients with IA and EA desmoid tumors were 76.2% and 45.5%, respectively (odds ratio 3.80; = 0.03). The DCR for patients with FAP-associated desmoid tumors was 73.7%, compared to 48.6% for those without FAP (odds ratio 2.96, = 0.09). Mean times to next treatment were 80.5 weeks and 36.3 weeks for patients with and without FAP. CONCLUSION: LD is effective and well-tolerated with a high potential for tumor control and prolonged duration of time until next treatment, especially in patients with FAP and IA desmoid tumors.