Oncology and Therapy最新文献

{"title":"The Immunomodulatory Effects of Dexamethasone on Neoadjuvant Chemotherapy for Triple-Negative Breast Cancer.","authors":"Kai Conrad Cecil Johnson, Daniel Goldstein, Jasmin Tharakan, Dionisia Quiroga, Mahmoud Kassem, Michael Grimm, Abdul Miah, Craig Vargo, Michael Berger, Preeti Sudheendra, Ashley Pariser, Margaret E Gatti-Mays, Nicole Williams, Daniel Stover, Sagar Sardesai, Robert Wesolowski, Bhuvaneswari Ramaswamy, Gary Tozbikian, Patrick M Schnell, Mathew A Cherian","doi":"10.1007/s40487-023-00235-6","DOIUrl":"https://doi.org/10.1007/s40487-023-00235-6","url":null,"abstract":"<p><strong>Introduction: </strong>The immunomodulatory impact of corticosteroids and concurrent chemotherapy is poorly understood within triple-negative breast cancer (TNBC). On a biochemical level, steroids have been linked to the signaling of chemotherapy-resistant pathways. However, on a clinical level, steroids play an essential role in chemotherapy tolerance through the prevention of chemotherapy-induced nausea and vomiting (CINV) and hypersensitivity reactions. Given these conflicting roles, we wanted to evaluate this interplay more rigorously in the context of early-stage TNBC.</p><p><strong>Methods: </strong>We performed a retrospective analysis of patients with operable TNBC who received neoadjuvant chemotherapy (NAC) between January 2012 and November 2018, with the primary goal of examining the dose-dependent relationship between pathological complete response (pCR) rates and corticosteroid use. Secondary endpoints included the impact of steroid dosing on overall survival (OS) and recurrence-free survival (RFS), along with a breakdown in pCR rates based on steroid doses provided during each chemotherapy phase. Further adjusted analyses were performed based on patient age, diabetic status, and anatomical stage. Finally, we explored the relationship between tumor-infiltrating lymphocytes (TILs) seen on tissue samples at baseline and dexamethasone doses in terms of pCR rates.</p><p><strong>Results: </strong>In total, of the 174 patients screened within this study period, 116 met full eligibility criteria. Of these eligible patients, all were female, with a median age of 51.5 years (27.0 to 74.0) and a mean body mass index (BMI) of 29.7 [standard deviation (SD) 7.04]. The majority were nondiabetic (80.2%). For cancer stage, 69.8% (n = 81) had stage 2 breast cancer. We found no statistically significant association between pCR rates and dexamethasone use, both in terms of the total dose (p = 0.55) and mean dose per NAC cycle (p = 0.74). Similarly, no difference was noted when adjusting for diabetic status, metformin use, or age at diagnosis, regardless of the total steroid dose provided (p = 0.72) or mean dose per cycle (p = 0.49). No meaningful changes to pCR rate were seen with higher mean or higher total steroid doses during the paclitaxel (T) phase (adjusted p = 0.16 and p = 0.76, respectively) or doxorubicin and cyclophosphamide (AC) phase (adjusted p = 0.83 and p = 0.77, respectively). Furthermore, we found no clinically significant association between dexamethasone dose and either RFS (p = 0.45) or OS (p = 0.89). Of the 56 patients who had available pre-treatment biopsy tissue samples, 27 achieved pCR, with higher TILs at baseline being associated with higher pCR rates, regardless of the mean dexamethasone dose used.</p><p><strong>Conclusion: </strong>Our findings demonstrate that dexamethasone has no clinically significant impact on pCR, RFS, or OS when given concurrently with NAC in patients with curative TNBC, regardless of diabeti","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":"11 3","pages":"361-374"},"PeriodicalIF":2.7,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/6e/86/40487_2023_Article_235.PMC10447758.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10126285","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Extrapolation of Survival Data Using a Bayesian Approach: A Case Study Leveraging External Data from Cilta-Cel Therapy in Multiple Myeloma.","authors":"Stephen Palmer, Yi Lin, Thomas G Martin, Sundar Jagannath, Andrzej Jakubowiak, Saad Z Usmani, Nasuh Buyukkaramikli, Hilary Phelps, Rafal Slowik, Feng Pan, Satish Valluri, Lida Pacaud, Graham Jackson","doi":"10.1007/s40487-023-00230-x","DOIUrl":"10.1007/s40487-023-00230-x","url":null,"abstract":"<p><strong>Introduction: </strong>Extrapolating long-term overall survival (OS) from shorter-term clinical trial data is key to health technology assessment in oncology. However, extrapolation using conventional methods is often subject to uncertainty. Using ciltacabtagene autoleucel (cilta-cel), a chimeric antigen receptor T-cell therapy for multiple myeloma, we used a flexible Bayesian approach to demonstrate use of external longer-term data to reduce the uncertainty in long-term extrapolation.</p><p><strong>Methods: </strong>The pivotal CARTITUDE-1 trial (NCT03548207) provided the primary efficacy data for cilta-cel, including a 12-month median follow-up snapshot of OS. Longer-term (48-month median follow-up) survival data from the phase I LEGEND-2 study (NCT03090659) were also available. Twelve-month CARTITUDE-1 OS data were extrapolated in two ways: (1) conventional survival models with standard parametric distributions (uninformed), and (2) Bayesian survival models whose shape prior was informed from 48-month LEGEND-2 data. For validation, extrapolations from 12-month CARTITUDE-1 data were compared with observed 28-month CARTITUDE-1 data.</p><p><strong>Results: </strong>Extrapolations of the 12-month CARTITUDE-1 data using conventional uninformed parametric models were highly variable. Using informative priors from the 48-month LEGEND-2 dataset, the ranges of projected OS at different timepoints were consistently narrower. Area differences between the extrapolation curves and the 28-month CARTITUDE-1 data were generally lower in informed Bayesian models, except for the uninformed log-normal model, which had the lowest difference.</p><p><strong>Conclusions: </strong>Informed Bayesian survival models reduced variation of long-term projections and provided similar projections as the uninformed log-normal model. Bayesian models generated a narrower and more plausible range of OS projections from 12-month data that aligned with observed 28-month data.</p><p><strong>Trial registration: </strong>CARTITUDE-1 ClinicalTrials.gov identifier, NCT03548207. LEGEND-2 ClinicalTrials.gov identifier, NCT03090659, registered retrospectively on 27 March 2017, and ChiCTR-ONH-17012285.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":"11 3","pages":"313-326"},"PeriodicalIF":2.7,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/bc/c7/40487_2023_Article_230.PMC10447673.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10425793","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient Perceptions of CAR-T Therapy in the USA: Findings from In-Depth Interviews.","authors":"Todd J Bixby,&nbsp;Christine J Brittle,&nbsp;Patricia A Mangan,&nbsp;Edward A Stadtmauer,&nbsp;Lisa R Kallenbach","doi":"10.1007/s40487-023-00232-9","DOIUrl":"https://doi.org/10.1007/s40487-023-00232-9","url":null,"abstract":"<p><strong>Introduction: </strong>Chimeric antigen receptor-T cell (CAR-T) therapy has revolutionized advanced blood cancer treatment. However, preparation, administration, and recovery from these therapies can be complex and burdensome to patients and their care partners. Utilization of an outpatient setting for CAR-T therapy administration could help improve convenience and quality of life.</p><p><strong>Methods: </strong>In-depth qualitative interviews were conducted with 18 patients in the USA with relapsed/refractory multiple myeloma or relapsed/refractory diffuse large B-cell lymphoma, 10 of whom had completed investigational or commercially approved CAR-T therapy and 8 of whom had discussed it with their physicians. We aimed to better understand inpatient experiences and patient expectations regarding CAR-T therapy and to ascertain patient perspectives on the possibility of outpatient care.</p><p><strong>Results: </strong>CAR-T offers unique treatment benefits, particularly high response rates with an extended treatment-free period. All study participants completing CAR-T were very positive about their inpatient recovery experience. Most reported mild-to-moderate side effects; two experienced severe side effects. All said that they would opt to undergo CAR-T therapy again. Participants felt that the primary advantage of inpatient recovery was immediate access to care and on-going monitoring. Perceived advantages of the outpatient setting were comfort and familiarity. Because immediate access to care was seen as crucial, patients recovering in an outpatient setting would seek either a direct contact person or phone line for assistance if needed.</p><p><strong>Conclusion: </strong>As institutions become more experienced with CAR-T therapies, outpatient care may help reduce financial strain. Patient input can help institutions improve the outpatient experience and ensure safety and effectiveness of CAR-T programs.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":"11 3","pages":"303-312"},"PeriodicalIF":2.7,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/98/98/40487_2023_Article_232.PMC10200031.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10055902","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Biomarker Test Utilization and Subsequent Treatment in Patients with Early-Stage Non-small Cell Lung Cancer in the United States, 2011-2021.","authors":"Jessie T Yan,&nbsp;Yue Jin,&nbsp;Ernest Lo,&nbsp;Yilin Chen,&nbsp;Amy E Hanlon Newell,&nbsp;Ying Kong,&nbsp;Landon J Inge","doi":"10.1007/s40487-023-00234-7","DOIUrl":"https://doi.org/10.1007/s40487-023-00234-7","url":null,"abstract":"<p><strong>Introduction: </strong>Biomarker testing is increasingly crucial for patients with early-stage non-small cell lung cancer (eNSCLC). We explored biomarker test utilization and subsequent treatment in eNSCLC patients in the real-world setting.</p><p><strong>Methods: </strong>Using COTA's oncology database, this retrospective observational study included adult patients ≥ 18 years old diagnosed with eNSCLC (disease stage 0-IIIA) between January 1, 2011 and December 31, 2021. Date of first eNSCLC diagnosis was the study index date. We reported testing rates by index year for patients who received any biomarker test within 6 months of eNSCLC diagnosis and by each molecular marker. We also evaluated treatments received among patients receiving the five most common biomarker tests.</p><p><strong>Results: </strong>Among the 1031 eNSCLC patients included in the analysis, 764 (74.1%) received ≥ 1 biomarker test within 6 months of eNSCLC diagnosis. Overall, epidermal growth factor receptor (EGFR; 64%), anaplastic lymphoma kinase (ALK; 60%), programmed death receptor ligand 1 (PD-L1; 48%), ROS proto-oncogene 1 (ROS1; 46%), B-Raf proto-oncogene (40%), mesenchymal epithelial transition factor receptor (35%), Kirsten rat sarcoma viral oncogene (29%), RET proto-oncogene (22%), human epidermal growth factor receptor 2 (21%), and phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (20%) were the 10 most frequently tested biomarkers. The proportion of patients undergoing biomarker testing rose from 55.3% in 2011 to 88.1% in 2021. The most common testing methods were Sanger sequencing for EGFR (244, 37%), FISH (fluorescence in situ hybridization) for ALK (464, 75%) and ROS1 (357, 76%), immunohistochemical assay for PD-L1 (450, 90%), and next-generation sequencing testing for other biomarkers. Almost all the 763 patients who received the five most common biomarker tests had a test before the initiation of a systemic treatment.</p><p><strong>Conclusion: </strong>This study suggests a high biomarker testing rate among patients with eNSCLC in the US, with testing rates for various biomarkers increasing over the past decade, indicating a continuous trend towards the personalization of treatment decisions.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":"11 3","pages":"343-360"},"PeriodicalIF":2.7,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/72/73/40487_2023_Article_234.PMC10447355.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10067860","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient and Healthcare Professional Perspectives from ESMO 2022 on Bladder and Kidney Cancer: A Podcast.","authors":"Alex Filicevas,&nbsp;Thomas Powles","doi":"10.1007/s40487-023-00231-w","DOIUrl":"https://doi.org/10.1007/s40487-023-00231-w","url":null,"abstract":"<p><p>In this plain language podcast, highlights from the European Society for Medical Oncology (ESMO) Congress are discussed for a second year in a row, from the perspective of both a patient advocate and a healthcare professional. The patient advocacy track at the congress included two patient-focused sessions each day on a variety of topics. Here, the authors discuss the importance of involving patients in the design of clinical trials, as well as strategies to improve dialogue and connections between clinicians, researchers and patients. Patient advocacy organisations provide essential services to patients with cancer and their caregivers, and patient advocates play a critical role in helping to inform patients and caregivers in making clinical decisions. Congresses such as ESMO provide an important platform for patient advocates to connect with each other and with physicians and researchers to ensure that patients are placed at the centre of the conversation and are up to date on the latest findings that affect them. The authors also discuss the latest research on genitourinary cancers, focusing on bladder and kidney cancer. Promising results are emerging for combination antibody-drug conjugates and immunotherapy for patients with hard-to-treat, locally advanced or metastatic bladder cancer who are ineligible for platinum-based chemotherapy. In the management of kidney cancer, we may be reaching an end for immune checkpoint inhibitors on their own; the path ahead will be to find new targets and combinations. Podcast Audio (MP4 169766 KB).</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":"11 3","pages":"277-289"},"PeriodicalIF":2.7,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/bd/dd/40487_2023_Article_231.PMC10447799.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10071935","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Neoadjuvant Doxorubicin-Paclitaxel Combined Chemotherapy in Patients with Inoperable Stage III Breast Cancer: A Retrospective Cohort Study with 10 Years of Follow-Up in Vietnam.","authors":"Duc Thanh Le,&nbsp;Lap Thanh Bui,&nbsp;Chu Van Nguyen,&nbsp;Kien Hung Do,&nbsp;Giang Le Tran,&nbsp;Tu Anh Do","doi":"10.1007/s40487-023-00233-8","DOIUrl":"https://doi.org/10.1007/s40487-023-00233-8","url":null,"abstract":"<p><strong>Introduction: </strong>The combination of doxorubicin and paclitaxel (AP) is widely used in our country for the neoadjuvant treatment of breast cancer as well as metastatic breast cancer. The AP regimen has shown promise as a neoadjuvant therapy for breast cancer that improves pathological complete response (pCR), increases the rate of conservative surgery, and improves the survival of patients. However, up to now, no research has evaluated the response of this regimen for the neoadjuvant treatment of advanced breast cancer, especially with a 10-year period of follow-up.</p><p><strong>Methods: </strong>This retrospective analysis reviewed 126 patients with inoperable stage III breast cancer who received neoadjuvant chemotherapy with doxorubicin 50 mg/m² plus paclitaxel 175 mg/m² every 3 weeks for a maximum of six courses followed by surgery. pCR was evaluated. Survival was analyzed for all breast cancer patients using Kaplan-Meier and log-rank models.</p><p><strong>Results: </strong>Of 126 women treated with neoadjuvant chemotherapy (NAC), the overall pCR rate was 25.4% and was significantly higher in patients with tumor stage cT1-T2, hormone receptor-negative (HR-negative), and human epidermal growth factor receptor 2 (HER2)-positive disease. Patients achieving pCR had significantly longer disease-free survival (DFS) and overall survival (OS). Ten-year DFS rates were 43.8% vs. 25.0% (p = 0.030) and 10-year OS rates were 59.4% vs. 28.9% (p = 0.003) for patients with pCR and non-pCR, respectively. The cumulative 10-year DFS was 19.6% for patients with HR-negative disease and 37.3% for those with HR-positive disease. Achieving pCR was associated with improved 10-year OS and DFS. Several clinicopathological features were closely associated with pCR in the inoperable stage III breast cancer patients who were treated by neoadjuvant chemotherapy.</p><p><strong>Conclusion: </strong>Achieving pCR was associated with improved 10-year OS and DFS. Patients with advanced breast cancer with HR-negative and HER2-positive status who benefited from the AP neoadjuvant therapy regimen were significantly more likely to achieve pCR.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":"11 3","pages":"327-341"},"PeriodicalIF":2.7,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/26/8b/40487_2023_Article_233.PMC10447719.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10060757","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Small-Cell Carcinoma of the Prostate - Challenges of Diagnosis and Treatment: A Next of Kin and Physician Perspective Piece.","authors":"Trish Abbott,&nbsp;Kenrick Ng,&nbsp;Jenny Nobes,&nbsp;Paula Muehlschlegel","doi":"10.1007/s40487-023-00238-3","DOIUrl":"https://doi.org/10.1007/s40487-023-00238-3","url":null,"abstract":"<p><p>This article was co-authored by a patient's relative describing their experiences of receiving a diagnosis and subsequent clinical management of a rare form of prostate cancer, neuroendocrine prostate cancer (NEPC). The difficulty of receiving this diagnosis, particularly as this was terminal with no options for systemic treatment, and experiences throughout this process are detailed. The relative's questions regarding the care of her partner, NEPC and clinical management are answered. The treating physician's perspective regarding clinical management is enclosed. Prostate cancer remains one of the most common cancer diagnoses, with small-cell carcinoma (SCC) of the prostate representing 0.5-2% of these. Prostatic SCC frequently develops in patients previously treated for prostate adenocarcinoma, more rarely arising de novo. Diagnosis and management present clinical challenges owing to its rarity, frequently aggressive disease course, lack of specific diagnostic and monitoring biomarkers, and treatment limitations. Current pathophysiological understanding of prostatic SCC, genomics and contemporary and evolving treatment options in addition to current guidelines are discussed. Written principally from the patient's relatives and physician experience with discussion of current evidence, diagnostic and treatment options, we hope this piece is informative for both patients and healthcare professionals alike.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":"11 3","pages":"291-301"},"PeriodicalIF":2.7,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/9d/b2/40487_2023_Article_238.PMC10447819.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10071945","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"RETRACTED ARTICLE: Tarsoconjunctival Granulation Tissue Formation Associated with EGFR Inhibitors.","authors":"Ferhan Guler,&nbsp;Nilay Yuksel,&nbsp;Seda Kahraman,&nbsp;Mehmet Ali Nahit Sendur","doi":"10.1007/s40487-022-00216-1","DOIUrl":"https://doi.org/10.1007/s40487-022-00216-1","url":null,"abstract":"","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":"11 3","pages":"391-396"},"PeriodicalIF":2.7,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/eb/14/40487_2022_Article_216.PMC10447638.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10071361","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"French Retrospective Database Analysis of Patient Characteristics and Treatment Patterns in Patients with R/R FLT3-Mutated AML: A Registry-Based Cohort Study.","authors":"Andy Garnham,&nbsp;Franck Bruon,&nbsp;Céline Berthon,&nbsp;Delphine Lebon,&nbsp;Mounika Parimi,&nbsp;Rosalind Polya,&nbsp;Kahina M Makhloufi,&nbsp;Marie-Hélène Dramard-Goasdoue","doi":"10.1007/s40487-023-00239-2","DOIUrl":"https://doi.org/10.1007/s40487-023-00239-2","url":null,"abstract":"<p><strong>Introduction: </strong>There is a dearth of evidence to document treatment of FMS-like tyrosine kinase 3 (FLT3)-mutated acute myeloid leukemia (AML) in real-world settings before the introduction of FLT3 inhibitors. A retrospective cohort study was conducted to understand treatment practices prior to the availability of FLT3 inhibitors in patients with FLT3-mutated AML from two registries in France.</p><p><strong>Methods: </strong>Patient data from January 1, 2009 to December 31, 2017 were collected from the Hauts-de-France and Midi-Pyrénées registries. Patients aged ≥ 18 years at diagnosis with FLT3-mutated AML were included. Demographic and disease characteristics of patients with FLT3-mutated AML and relapsed or refractory (R/R) FLT3-mutated AML were documented. Treatment regimens, overall survival (OS), and event-free survival were assessed in patients with R/R FLT3-mutated AML who did not participate in clinical trials.</p><p><strong>Results: </strong>Overall, 819 and 1244 adult patients with AML from the Midi-Pyrénées and Hauts-de-France cohorts, respectively, underwent FLT3 mutation testing; 172 (21.0%) and 263 (21.1%) patients, respectively, had a FLT3 mutation. Primary R/R status was identified in 41.3% (n = 71/172) of the Midi-Pyrénées and 34.6% (n = 91/263) of the Hauts-de-France cohorts. Before R/R AML diagnosis, 82.0% and 97.5% of patients in the Midi-Pyrénées and Hauts-de-France cohorts, respectively, achieved complete remission (CR) or CR with incomplete hematologic recovery (CRi) following induction chemotherapy; after diagnosis of R/R AML, CR/CRi rates with salvage therapy were 33.3% and 28.1%, respectively. Median OS (interquartile range) in patients receiving salvage therapy (n = 49, n = 78) was 5.2 (2.3-11.1) and 6.1 (2.5-35.2) months, in the Midi-Pyrénées and Hauts-de-France cohorts, respectively. Across both cohorts, patients with R/R FLT3-mutated AML had low rates of CR/CRi with salvage therapy and a median OS of approximately 6 months.</p><p><strong>Conclusion: </strong>Before FLT3 inhibitor availability, real-world treatment patterns and outcomes in French patients with R/R FLT3-mutated AML were consistent with clinical trial data, highlighting a poor prognosis and unmet need for effective treatment.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":"11 3","pages":"375-389"},"PeriodicalIF":2.7,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/95/08/40487_2023_Article_239.PMC10447689.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10071627","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development of a Conceptual Model of the Patient Experience in Small Cell Lung Cancer: A Qualitative Interview Study.","authors":"Danielle E Altman,&nbsp;Xinke Zhang,&nbsp;An-Chen Fu,&nbsp;Alissa R Rams,&nbsp;Jessica A Baldasaro,&nbsp;Samir Ali Ahmad,&nbsp;Michael Schlichting,&nbsp;Patrick Marquis,&nbsp;Elena Benincasa,&nbsp;Camilo Moulin,&nbsp;Vivek Pawar","doi":"10.1007/s40487-023-00223-w","DOIUrl":"https://doi.org/10.1007/s40487-023-00223-w","url":null,"abstract":"<p><strong>Introduction: </strong>Small cell lung cancer (SCLC) is a subtype of lung cancer, the second most common cancer diagnosis worldwide. Currently, there is little published qualitative research that provides insight into the disease-related symptoms and impacts that are relevant to patients living with SCLC as directly reported by patients themselves.</p><p><strong>Methods: </strong>This qualitative, cross-sectional, noninterventional, descriptive study included concept elicitation interviews with participants diagnosed with SCLC and the development of a conceptual model of clinical treatment benefit.</p><p><strong>Results: </strong>Concept elicitation interview data from 26 participants with SCLC were used to develop a conceptual model of clinical treatment benefit that organized 28 patient-reported concepts into two domains: disease-related symptoms (organ-specific and systemic) and impacts. Organ-specific symptoms included cough, chest pain, and difficulty breathing. Systemic symptoms included pain, fatigue, appetite loss, and dizziness. Impacts included physical functioning, role functioning, reduced movement, impact on sleep, and weight loss.</p><p><strong>Conclusion: </strong>As evidenced by this study, people with SCLC experience considerable and significant symptoms and impacts, including physical and role functioning challenges, that affect their quality of life. This conceptual model will inform the design of a patient-reported outcome (PRO) questionnaire for a future SCLC clinical trial, helping to establish the content validity of the items and questionnaires used in the trial and ensuring that the questionnaires and items selected are appropriately targeted to the population. This conceptual model could also be used to inform future SCLC clinical trials.</p>","PeriodicalId":44205,"journal":{"name":"Oncology and Therapy","volume":"11 2","pages":"231-244"},"PeriodicalIF":2.7,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/d3/83/40487_2023_Article_223.PMC10260713.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9634954","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}