bluuebunny/crossref_metadata_2025_split huggingface dataset:
Retrospective study of BRAF inhibitor-based therapy in patients with BRAF mutated advanced solid tumors (RBAST study).
Authors: Xiao Wenxuan , Liu Ying , Hou Lei , Hou Yinyin , Wei Xin , An Gaili , Zhang Fan , Zhang Yuan , Lei Yu , Wang Haipeng , Liu Yi , Duan Baojun , Cao Fei , Sun Chao , Wang Xi , Wang Weijiang , Yang Yanping , Bai Jun 
Published: June 2024

e23265 Background: BRAF mutations are frequently found in melanoma, non-small cell lung cancer (NSCLC), thyroid cancer, to a lesser degree in other tumor types. These mutations constantly activate the kinase domain and result in MAPK pathway hyperactivation. Successful inhibition of this pathway with BRAF/MEK inhibitors (BRAFi/MEKi) results in a clinically meaningful benefit for some patients. The RBAST study, to assess the efficacy and survival outcomes of BRAF inhibitor-based therapy were investigated in patients with solid tumors harboring BRAF mutations. Methods: Data was collected on a total of 697 cases of solid tumor throughout China between Sep 2018 and Dec 2023. Next Generation Sequencing (NGS) is based on the sequencing of tissue samples. Among the samples, the primary endpoints of this study were objective response rate (ORR), disease control rate (DCR), progress-free survival (PFS) and overall survival (OS). The impact of co-mutations with other genes and BRAF variant allele frequency on treatment efficacy was also assessed in this study. Results: BRAF mutations were detected in 36 cases and 15 patients received BRAFi based therapy were identified. For patients who received BRAFi therapy, the best ORR was 26.7% with 4 patients recorded partial response (PR). There were 2 patients with progressive disease giving a DCR of 86.7%. Responses were observed in 4 types of tumors. PR was observed in patients with colorectal cancer (CRC) and melanoma. Median PFS (mPFS) and median OS (mOS) of these patients were 6.0 months (95% CI: 2.3-7.0) and 17.1 months (95% CI: 4.4-NA), respectively. The co-occurring mutation rates of TP53, PTEN, FAT2 and NF1 were 40% (6/15), 20% (3/15), 20% (3/15) and 13.3% (2/15), respectively. No significant difference (p>0.05) in PFS and OS was observed in any co-occurrence of mutations. BRAF mutation variant allele frequency is not an independent prognostic factor for survival with patients received BRAF inhibitor treatment (p>0.05). Conclusions: To the best of our knowledge, this is the first retrospective study exploring BRAFi treatment effectiveness to BRAF mutated solid tumors for Chinese patients. Treatment with BRAF inhibitors is effective for BRAF mutated advanced solid tumors with DCR of 86.7%, mPFS of 6.0 months and mOS of 17.1 months. Co-mutations with other genes and BRAF variant allele frequency did not impact survival outcomes.
Real-world safety and healthcare resource utilization (HCRU) of lurbinectedin (lurbi) in patients (pts) with small cell lung cancer (SCLC): Jazz EMERGE 402 updated analysis.
Authors: Badin Firas Benyamine , Lammers Philip Edward , Patel Mehul P. , Shunyakov Leonid , Slater Dennis , Labbé Catherine , Naveh Navit , Boccuti Anne , Hanvesakul Raj , Li Wenyan , Rengarajan Badri , Halmos Balazs 
Published: June 2024

e23266 Background: Lurbi monotherapy received approval in multiple countries, including accelerated US FDA approval and conditional Health Canada approval for adults with metastatic (US) or stage III/metastatic (Canada) SCLC with disease progression on or after platinum-based chemotherapy, based on a phase 2, open-label, single-arm trial (Trigo et al, Lancet Oncol 2020). Jazz EMERGE 402 (NCT04894591) is a phase 4 observational study assessing the effectiveness, safety profile, and HCRU of lurbi in pts with extensive-stage SCLC treated in real-world clinical settings. Here, we report updated safety data and HCRU. Methods: This study is enrolling pts with extensive-stage SCLC treated with lurbi according to the local approved label in the US and Canada. Results: Enrollment is ongoing with a target of 300 pts. Between 28JUN2021 and 27DEC2022, 105 pts were enrolled and followed for ≥6 months after the first dose of lurbi (data cutoff: 27JUN2023). The median (range) age was 67 (44-87) years; 72 (69%) pts had prior immunotherapy; 22 (21%) had an Eastern Cooperative Oncology Group performance status of ≥2; and 25 (24%) had central nervous system involvement. All pts received ≥1 cycle of lurbi; 56 (53%) as second-line (2L) and 37 (35%) as third-line (3L) therapy. The median (range) number of lurbi cycles was 4 (1-31) and the median (range) duration of exposure was 97 (21-694) days. Dose modifications occurred in 27 (26%) pts, with 17 (16%) requiring dose reductions. Granulocyte colony-stimulating factor was administered in 43 (41%) pts (34 [79%] as primary and 7 [16%] as secondary prophylaxis; 2 [5%] were unknown). At the time of data extraction, lurbi treatment was ongoing in 11 (10%) pts and 23 (22%) pts received subsequent anticancer therapy after lurbi. Treatment-related adverse events (TRAEs) were reported in 40 (38%) pts and treatment-related serious adverse events (SAEs) in 13 (12%) pts. The most common TRAEs were anemia (n = 13, 12%), neutropenia (n = 10, 10%), nausea (n = 8, 8%), and fatigue (n = 7, 7%). Thirty-six pts were hospitalized for a median (interquartile range [IQR]) duration of 10 (4, 18) days (Table). No pts were hospitalized on the first day of treatment; 4 pts were hospitalized during the first 21-day cycle. The most common reasons for hospitalization were pneumonia (n = 10, 28%) and sepsis (n = 6, 17%; one was an SAE). Conclusions: In this phase 4 study, lurbi was well tolerated, with low rates of HCRU and a safety profile generally consistent with that reported in the phase 2 trial. Clinical trial information: NCT04894591 . [Table: see text]
Surgical decision-making after neoadjuvant chemotherapy for breast cancer.
Authors: Reddy Haritha , Lopez May Martina , Berkalieva Asem , Moshier Erin , Tsang Ashley Tai 
Published: June 2024

e23282 Background: Following neoadjuvant chemotherapy (NAC), a significant subset of breast cancer patients become candidates for breast conserving surgery (BCS) after tumor downstaging. Studies have shown there is no survival advantage between BCS and mastectomy after NAC and that patient prognosis is largely predicted by the pathologic response to treatment. While the literature highlights many aspects of surgical decision-making in breast cancer, there is little analysis of patient choice after NAC. The goal of this study is to investigate clinical and demographic factors that may associate with patients’ surgical decision-making after receiving NAC as well as related outcomes. Methods: A retrospective chart review was conducted on breast cancer patients at our institution who became BCS candidates after receiving NAC from 1/1/2010 to 9/30/2020. Patient demographics, clinical characteristics, choice of mastectomy versus BCS, and outcomes were compared. Exclusion criteria included inflammatory, recurrent, and bilateral breast cancers, and patients with genetic predisposition. T-tests and chi-squared tests of association were used for continuous and categorical variables respectively. Survival outcomes were reported using the log-rank test. The p-value threshold for significance was p < 0.05. Statistical analysis was performed using R software version 4.2.3. Results: In this analysis, 244 patients met study criteria, of which 181 (74.2%) patients chose BCS and 63 (25.8%) patients elected mastectomy. Patients who chose mastectomy were younger at time of diagnosis than those who chose BCS: median age 48 years in mastectomy group versus 53 years in BCS group (p = 0.003). Regarding physical exam findings, while palpability of the primary breast tumor was not associated with choice of surgery (p = 0.271), presence of axillary lymphadenopathy was associated with a higher likelihood of choosing mastectomy (44.4% in mastectomy subgroup versus 29.3% in BCS subgroup, p = 0.041). Patient race, pre-treatment clinical stage, and doxorubicin-based chemotherapy were not associated with patients’ decisions for type of surgery. With respect to outcomes, follow-up time was similar between both groups with overall median of 5 years, and there was no significant difference in local or distant recurrence, and overall mortality (log-rank p = 0.3). Conclusions: The majority of breast cancer patients elect BCS after downstaging from NAC. Factors that are associated with choosing mastectomy include younger age at diagnosis and pre-treatment palpable axillary lymphadenopathy, which may be perceived as more advanced stage, despite no difference in stage between both groups. Regardless of surgical choice, both recurrence and mortality rates did not significantly differ between patients who chose BCS versus mastectomy, which is consistent with the literature.
Cancer burden across South Asia in 2020: A global cancer observatory analysis.
Authors: Jain Urvish , Rahim Faraan , Jain Bhav , Arkalgud Aditya , Sabat Cameron , Hammond Alessandro , Tshering Phub , Patel Tej A. , Sirohi Bhawna , Jain Pankaj , Zeb Khan Shah , Gunasekera Sanjeeva , Shilpakar Ramila , Stanikzai Zabihullah , Chowdhury Arman Reza , Swami Nishwant , Dee Edward Christopher Christopher , Gyawali Bishal 
Published: June 2024

e23292 Background: The cancer burden in South Asia is often associated with late diagnosis and poor outcomes because of barriers to accessing advanced treatment facilities and targeted therapies, financial sequelae of care, and cultural stigmas and misconceptions. We use recent estimates of the Global Cancer Observatory (GCO) to better understand the epidemiology of cancer in South Asia, which may inform national and regional cancer control. Methods: Using publicly available data from the GCO and the World Bank, we collected cancer statistics and population statistics for Afghanistan, Bangladesh, Bhutan, India, Maldives, Nepal, Pakistan, and Sri Lanka (collectively, SAARC) from 2015-2020. Results: The crude incidence of cancer in this region was 1,733,573 cases, representing 9.0% of the incidence worldwide. The crude incidence rate (CIR, per 100,000) of cancer in SAARC was 93.4 compared to the worldwide rate of 247.5. The 5-year prevalence (in total cases) of cancer in South Asia was 3,474,184, compared to 50,550,287 globally. The crude mortality rate (CMR, per 100,000) in South Asia was 60.6, compared to 127.8 globally. However, the mortality-to-incidence ratio (MIR) (per 100,000) was 0.65, compared to 0.52 globally. Age-adjusted estimates are reported in the table. Conclusions: Our research highlights South Asia's unique cancer landscape with CIR and MIR, but elevated MIR compared to global figures. These findings underscore the need for a united, contextually relevant approach to addressing the burden of cancer in South Asia. Investment in collaborative, tailored cancer care programs will build the SAARC region's capacity to address the growing global cancer challenge. [Table: see text]
Juxtacortical chondrosarcoma: An NCDB analysis of demographics and socioeconomic factors.
Authors: Peterson Michael , Tade Yanick , Lopez Braden M. , Tandon Nikhita , Silberstein Peter T. , Heise Craig , Hsia Beau 
Published: June 2024

e23505 Background: Juxtacortical chondrosarcoma (JCC) is a rare cartilaginous tumor—accounting for less than 2% of all chondrosarcomas—and typically appears on the external surfaces of long bones. The prognosis of JCC is relatively promising compared to other chondrosarcomas, with the current therapy of choice being en-bloc resection. However, a significant amount of information regarding JCC remains unknown. Therefore, exploring diagnostic trends may provide greater insight into this cancer’s epidemiology and potential forms of treatment. The National Cancer Database (NCDB) was assessed to uncover the demographic factors in patients diagnosed with JCC. Methods: A retrospective cohort analysis utilizing the 2004–2020 National Cancer Database (NCDB) included 131 subjects with a histologically-confirmed diagnosis of JCC (ICD-0-3 code 9221). Demographic factors (age, sex, race, Hispanic status, insurance status, facility type, distance from facility, and Charleson-Deyo comorbidity score) were analyzed by descriptive statistics and incidence trends were interpreted in regression analysis. Results: Between 2004 and 2020, the NCDB reported a total of 131 patients with a confirmed diagnosis of JCC. This data indicated a rising incidence of patients diagnosed per year (R² = 0.0855), with an average age of diagnosis of 45 years (SD = 18, range = 8-88). Patients with JCC were more likely to be White (82%), of non-Hispanic origin (86%), and male (63%). The tumor was most commonly found in the long bones of the lower limbs (35%). Surgical removal was the primary treatment for the majority of patients (95%), while others received radiation (7%) or chemotherapy (4%). Most patients (83%) had a Charlson-Deyo comorbidity score of 0. Patients were commonly treated at academic and research facilities (61%), and no patients received palliative care. More than half of individuals were privately insured (57%) and resided in a metropolitan area with a population size of at least 1 million (59%). The mean distance between patient residence and treatment site was 104.1 miles (SD = 221, range = 11-1194 miles). Conclusions: To the best of our knowledge, this is the first study on juxtacortical chondrosarcoma utilizing NCDB data to address the significant lack of research on this subject. The majority of JCC patients are non-Hispanic, White, and have a primary site of diagnosis at the long bones of the lower limbs. This paper is the first to describe the socioeconomic factors of patients diagnosed with JCC: patients tend to be privately insured, live in large metropolitan areas, and are mostly treated in academic research facilities. Additional research is necessary to comprehend how socioeconomic and demographic factors influence the manner in which JCC patients are diagnosed and treated.
Place of rank ligand inhibitor in giant cell tumour of bone.
Authors: Kerboua Esma 
Published: June 2024

e23511 Background: Giant cell tumour of bone (GCTB) is a locally aggressive and osteolytic tumour.The peak incidence is during the third and fourth decades of life. The majority of tumours occur in the metaepiphyseal (end) region of the long bones. Surgical resection is the preferred treatment option, if possible but it is a tumor which always poses progressive problems. Denosumab is indicated in recurrent or unresectable GCTB. Methods: We report a series of 70 patients treated for GCT at the medical oncology department of the CPMC (Algiers) in collaboration with the orthopedics department of Mustapha University Hospital from January 2020 to December 2023. Results: 70 patients ( 50 women and 20 men). The sex ratio is 0.54 The average age is 22 years (11-54 years). Reason for consultation : - Pathological fracture: n = 10 - Pain: n = 30 - Functional impotence: n = 20 - Tumor mass: n = 5 - Concept of trauma: n = 5 Stage of the disease: -Localized: n = 29 -Recurrence: n = 30, -Recurrence + pulmonary metastases: n = 10, -Sarcomatous transformation: n = 1 Type of treatment: -Radiotherapy, n = 1 -Exercised biopsy: n = 10, -Curettage + cementoplasty: n = 50 -Exeresis with osteosynthesis material: n = 8, -Amputation : n = 1 Medical treatment: Denosumab: n = 45 The number of courses with denosumab was between 6 and 55 cycles. Denosumab was administered neoadjuvantly in 10 patients, in 20 patients with reccurence disease, 15 patients in the metastatic phase - In terms of toxicity, we noted moderate hypocalcemia in 3 patients. -Objective responses: 95% ORR, 5 complete radiological responses and 10 partial responses, 20 stable patients and 9 out of 10 patients who received neoadjuvant denosumab were able to operate.Progression Free Survival was 28 months. Conclusions: GCTs are aggressive tumors with intermediate malignancy that should not be underestimated. The treatment is essentially surgical. Denosumab is indicated for both neoadjuvant and adjuvant use, it allows obtaining a clear clinical and radiological improvement.
Procollagen C-protein enhancer 1 (PCPE-1) as a potential serum biomarker for osteosarcoma.
Authors: Strauss Gal Zvi , Halperin Sharon , Appel Sarit , Sheiman Avivit , Weitzen Rony , Ben Gal Ofir , Liberman Boaz , Ramu Eyal , Ben Ami Eytan Aharon 
Published: June 2024

e23518 Background: Osteosarcoma (OS) is the most common bone malignancy in children and young adults. Treatment with perioperative systemic chemotherapy has improved patient outcome but a third of patients will eventually develop incurable metastatic disease. Currently, there are no biomarkers for monitoring patients’ response to neo-adjuvant (NA) chemotherapy or for patients under active surveillance following treatments. Procollagen C-Proteinase Enhancer-1 (PCPE-1) is an extra-cellular matrix glycoprotein which promotes the maturation of pro-collagen into collagen in various tissues. Elevated serum levels of PCPE-1 are found in bone diseases such as Paget’s disease and Osteoporosis and in patients with breast cancer with bone metastases. Serum PCPE-1 has not been described in patients with OS. We aimed to describe PCPE-1 serum levels in patients with OS compared with healthy volunteers, and across different time points in their treatment and disease course. Methods: We collected serum samples from the Israeli national biobank of 18 adult OS patients. Serum samples were also collected from 6 healthy individuals. Additionally, we obtained serial samples from 4 OS patients prior to and after initiating NA therapy. Quantification of serum PCPE-1 levels was performed using a commercially available enzyme-linked immunosorbent assay (ELISA) kit. Results: We successfully analyzed serum PCPE-1 levels from all patients and healthy volunteers. Mean PCPE-1 levels of 553ng/ml (range 479-614ng/ml) were observed in OS patients, compared with 313ng/ml (285-339ng/ml) in healthy volunteers. Of 4 patients undergoing treatment who had serial samples, a decrease in serum levels was observed 2 responding patients, while an increase in PCPE-1 levels were observed in one patient after NA treatment who had no response to treatment at the time of surgery. Of notice, serum levels nearly doubled in a patient during active surveillance who experienced local disease recurrence (274ng/ml and 423ng/ml, respectively). Conclusions: This is the first study to show elevated PCPE-1 serum levels in patients with OS. Our study also demonstrates potential changes in PCPE-1 levels in response to treatment. We are expanding our study to include a pediatric population to investigate the role of PCPE-1 as a potential tumor biomarker for monitoring treatment and surveillance of OS.
Overall survival in soft tissue sarcomas (STS) in Hispanic vs non-Hispanic White patients: Single center experience.
Authors: Zamora DIana , Ikram Waleed , Braat Jonathan , Dinh Han , Azevedo Chelsea , Patel Shyamal , Wiseman Jason T. , Gonzalez Velez Miguel 
Published: June 2024

e23526 Background: Racial, ethnic, and socioeconomic factors have been implicated in the variability of survival rates among soft tissue sarcoma (STS). These disparities are multifactorial and may reflect differences in disease biology, access to care, treatment responses, and others. In situations where different racial groups have equitable access to healthcare, healthcare disparities can decrease, and survival gaps can narrow. Differences in survival in STS in racial minorities have been poorly studied. Recognizing the potential impact of such disparities, our study thereby contributes to the ongoing discourse on healthcare equity in STS outcomes. Methods: We conducted a retrospective analysis of 30 STS patients, stratifying them into Hispanic (H) and non-Hispanic white (NHW) groups at Dignity Health Cancer Institute in Phoenix, Arizona. The Mann-Whitney U test was chosen to compare survival distributions, given the non-normal distribution of our sample. Survival analysis was performed using the Kaplan-Meier method, and the log-rank test was applied to determine statistical significance. All analyses were executed in GraphPad Prism software. Results: Our cohort comprised 13 (43%) NHW and 17 (57%) H patients. The mean ages were 57.2 years for NHW and 48.9 years for H, with a balanced gender distribution across groups. The most common subtypes of STS in NHW were liposarcoma (n = 3, 23.1%), and spindle cell carcinoma (n = 2,15.4%). Comparatively, the most common subtypes of STS in H were liposarcoma (n = 5, 29.4%) and leiomyosarcoma (n = 3, 17.6%). All patients were diagnosed with Stage 4 STS. Treatment modalities differed marginally, with NHW patients undergoing more surgical interventions, while H patients were more frequently administered chemotherapy. Median overall survival from the start of first-line therapy was 412 days for NHW and 500 days for H patients. Despite the longer median overall survival observed in Hispanics, the intergroup difference did not reach statistical significance (P = 0.68). Conclusions: The similar survival times between H and NHW STS patients at our institution may hint at an equitable provision of care across racial demographics in a relatively controlled single-center experience. However, the trend of extended survival in the Hispanic group, while not statistically confirmed, raises questions that merit further inquiry. This study serves as a preliminary exploration into racial disparities in STS, advocating for larger-scale research with more diverse patient populations. Further studies are essential to understand these findings and identify and optimize factors contributing to narrowing the survival gap in minorities, with the goal of enhancing STS prognosis for all racial groups.
Clinical efficacy and safety of mTOR inhibitor in patients with advanced unresectable epithelioid hemangioendothelioma (EHE).
Authors: Tan Huijing , Sun Yuting , Chi Yihebali 
Published: June 2024

e23540 Background: Epithelioidhemangioendothelioma is a rare vascular soft tissue sarcoma, 90% of which contains WWTR1-CAMTA1 fusion gene. Studies have shown that mTOR inhibitors may have a certain effect on EHE patients. Methods: A retrospective analysis of the efficacy and safety of mTOR inhibitor everolimus in the treatment of advanced unresectable epithelioidhemangioendothelioma in our hospital in the past one year. Patients with advanced epithelioidhemangioendothelioma (EHE) confirmed by consultation in the pathological center received this regimen, everolimus 10mg, once a day orally for 28 days, every 4 weeks as a treatment cycle until the disease progressed or showed intolerable toxicity. Results: There were 3 females and 5 males, and their average age was 41.8 years old. All of them were unresectable with liver, lung, and shoulder primary lesions, and lungs, liver, pleura and bone metastasis. Ki67 is from 2% to 10%. 3 patients who received molecular pathological examination were all identified the WWTR1-CAMTA1 fusion gene, and one of them also had EWSR1 chromosome translocation. Previous antineoplastic treatments included gemcitabine, cisplatin, albumin paclitaxel, epirubicin, dacarbazine chemotherapy, anlotinib targeting, and toripalimab immune therapy. 5 patients were treated with first-line drugs, 2 cases with second-line and 1 case with third-line. In terms of overall efficacy, 1 case was not evaluated, 2 cases lost follow-up due to poor tolerance and economic factors, 2 cases were included in the clinical trails after reaching tumor control for 3 months, and the other 3 cases achieved tumor remission with a median PFS of 11.0 months, and the best curative effect was SDa. Severe oral mucositis can greatly affect the medication experience of patients, which can be relieved after symptomatic medication, or when interstitial pneumonia and hemogram decrease significantly, we can consider the oral administration of everolimus 5mg. Conclusions: At present, there is no standard treatment for EHE, we can roughly see the efficacy of mTOR inhibitor everolimus in some EHE patients, mainly disease stability. Emphasis on side effect management, improve tolerance, and systematic treatment of EHE needs to be further explored.