Retrospective Analysis Of Real-world Data Research Articles

Metabolic Syndrome and Its Components Have a Different Presentation and Impact as Cardiovascular Risk Factors in Psoriatic and Rheumatoid Arthritis.

Patients with chronic inflammatory arthritis have a higher cardiovascular (CV) risk than the general population. Traditional CV risk factors are clearly implicated, while the impact of metabolic syndrome (MetS) is less defined. The aim of this study was to compare MetS prevalence and impact on the CV risk in psoriatic arthritis (PsA) versus rheumatoid arthritis (RA). A retrospective analysis of real-world data of PsA and RA patients referred to a rheumatology clinic was conducted. The following data were extracted and compared: demographic data; clinical data; presence of traditional CV risk factors and MetS. Univariate and multivariate models were used to compare the impact of MetS and its components in patients with PsA versus RA. Overall, 170 patients were included (PsA: 78; RA; 92). The two groups differed significantly in mean age, disease duration, and presence of MetS, while other variables were comparable. Univariate and multivariate analysis identified distinct predictors of MetS in PsA (hypertension) and RA (dyslipidemia). The history of CV events was similar in the two groups. Predictors of CV events were MetS and most of its components in PsA, while dyslipidemia was the strongest predictor in RA. These associations were stronger in PsA than in RA. In conclusion, the impact of MetS and its components is different in PsA and RA. The association of these risk factors with CV events is stronger in PsA than in RA. This suggests the implication of different mechanisms, which may require distinct strategies for the prevention of CV events in PsA and RA.

Identifying factors associated with user retention and outcomes of a digital intervention for substance use disorder: a retrospective analysis of real-world data.

Successful delivery of digital health interventions is affected by multiple real-world factors. These factors may be identified in routinely collected, ecologically valid data from these interventions. We propose ideas for exploring these data, focusing on interventions targeting complex, comorbid conditions. This study retrospectively explores pre-post data collected between 2016 and 2019 from users of digital cognitive behavioral therapy (CBT)-containing psychoeducation and practical exercises-for substance use disorder (SUD) at UK addiction services. To identify factors associated with heterogenous user responses to the technology, we employed multivariable and multivariate regressions and random forest models of user-reported questionnaire data. The dataset contained information from 14 078 individuals of which 12 529 reported complete data at baseline and 2925 did so again after engagement with the CBT. Ninety-three percent screened positive for dependence on 1 of 43 substances at baseline, and 73% screened positive for anxiety or depression. Despite pre-post improvements independent of user sociodemographics, women reported more frequent and persistent symptoms of SUD, anxiety, and depression. Retention-minimum 2 use events recorded-was associated more with deployment environment than user characteristics. Prediction accuracy of post-engagement outcomes was acceptable (Area Under Curve [AUC]: 0.74-0.79), depending non-trivially on user characteristics. Traditionally, performance of digital health interventions is determined in controlled trials. Our analysis showcases multivariate models with which real-world data from these interventions can be explored and sources of user heterogeneity in retention and symptom reduction uncovered. Real-world data from digital health interventions contain information on natural user-technology interactions which could enrich results from controlled trials.

Identification and Management of Thyroid Dysfunction Using At-Home Sample Collection and Telehealth Services: Retrospective Analysis of Real-World Data.

Programs aimed at modernizing thyroid care by pairing at-home sample collection methods with telehealth options may serve an important and emerging role in thyroid care. The primary objective of this analysis was to evaluate telehealth use, demographics, and clinical characteristics of a cohort of consumer-initiated at-home laboratory thyroid test users who were also offered the option of follow-up telehealth consultations. This was a retrospective analysis of real-world data from a deidentified consumer database of home-collected, mail-in thyroid tests used from March to May 2021 (N=8152). The mean age was 38.6 (range 18-85) years, and 86.6% (n=7061) of individuals identified as female. In total, 7% (n=587) of test takers fell into a thyroid dysfunction category (overt hypothyroidism: n=75, 0.9%; subclinical hypothyroidism: n=236, 2.9%; overt hyperthyroidism: n=5, 0.1%; and subclinical hyperthyroidism: n=271, 3.3%). Overall, 12% (n=984) of the overall sample opted into a telehealth consultation, with 91.8% (n=903) receiving a nontreatment telehealth consultation and 8.2% (n=81) receiving a treatment telemedicine consultation. Furthermore, 16% (n=96) of individuals with overt or subclinical thyroid dysfunction engaged in telehealth consultations. The majority of treatment consultations (59.3%, n=48) were conducted with people reporting a history of thyroid issues, with 55.6% (n=45) of people indicating wanting to discuss their current thyroid medication and 48% (n=39) receiving a prescription medication. The combination of at-home sample collection and telehealth is an innovative model for screening thyroid disorders, monitoring thyroid function, and increasing access to care, which can be implemented at a large scale and across a wide range of age groups.

Changes in ambulatory glucose profile in people with type 1 diabetes using an automated insulin delivery system - two year follow-up retrospective analysis of real-world data

The number of available automated insulin delivery (AID) systems is increasing in Austria and people with diabetes (PwD) replace sensor-augmented pump (SAP) therapy more and more frequently. The present study is the two years follow-up of our prior monocentric, retrospective analysis conducted between 2019 and 2021, comparing SAP and open-source AID systems in people with type 1 diabetes. This second-year analysis included 25 PwD and investigated glycemic changes based on ambulatory glucose profiles (AGP). In comparison to the first year, a worsening of mean glucose (125.4 to 135.2 mg/dl, P = 0.038), time in range ((TIR), 84.2 to 77.0%, P = 0.012), time above range ((TAR), 11.6% to 18.5%, P = 0.017) and glycemia risk index ((GRI), 24.8 to 35.0%, P = 0.026) was observed. The reduction of mean glucose and glucose variability with AID in the first year was due to a significant decrease in time in hyperglycemia with resulting higher TIR and lower GRI. In this second-year follow up, TIR and GRI showed a significant deterioration, a familiar phenomenon in diabetology. However, open-source AID systems showed continuous safety, as there was no increase in time below range (TBR) even after two years. Despite the slight deterioration in the glycemic parameters, open-source AID systems were able to demonstrate sufficient glycemic control according to international consensus guidelines while offering the characteristic benefits of a reduced burden of diabetes management. A descriptive comparison of different AID algorithms indicated an improved glycemic control with more advanced features such as basal rate modification, auto bolus function and autotuning.

Retrospective Analysis of Real-World Data for the Treatment of Obstructive Sleep Apnea with Slow Maxillary Expansion Using a Unique Expansion Dental Appliance (DNA).

In addition to mandibular advancement devices, dental expansion appliances are an important clinical approach for achieving an increased intra-oral space that promotes airflow and lessens the frequency or severity of apneic events in patients diagnosed with obstructive sleep apnea (OSA). It has been thought that dental expansion in adults must be preceded by oral surgery; however, in this paper, we examine the results of a new technique for slow maxillary expansion without any surgical procedures. The palatal expansion device, DNA (Daytime-Nighttime Appliance), was reviewed in this retrospective study, particularly regarding its effects on measurements of transpalatal width, airway volume, and apnea-hypopnea indices (AHI) as well as its common modalities and complications. The DNA effectively reduced AHI by 46% (p = 0.00001) and significantly increased both airway volume and transpalatal width (p < 0.00001). After DNA treatment, 80% of patients showed some improvement in AHI scores with 28% of patients having their OSA symptoms completely resolved. Compared to the use of mandibular appliances, this approach is intended to create a sustained improvement in airway management that can reduce or eliminate dependence on continuous positive airway pressure (CPAP) or other OSA treatment devices.

Treatment patterns, indicators of receiving systemic treatment, and clinical outcomes in metastatic urothelial carcinoma: A retrospective analysis of real-world data in Germany.

464 Background: This study assessed real-world treatment (tx) and outcomes in patients with metastatic urothelial carcinoma (mUC) in Germany. Methods: Using 2 statutory health insurance (SHI) claims databases (SHI 1 and SHI 2; 2013-2020, ≈8 million insured), adults with an incident mUC diagnosis from 2015-2019 were identified. Those with other malignant tumors were excluded. Patients were observed for ≥12 mo after incident mUC diagnosis (index) or until death. Treated patients were delineated into 3 groups based on first-line (1L) tx received: platinum-based chemotherapy (PB CT), non–PB CT, and immunotherapy (IO). Patient characteristics were analyzed descriptively. Multivariable logistic regression was used to identify factors associated with receiving tx. Overall survival (OS) was calculated from 1L tx initiation by Kaplan-Meier estimation. Analyses were done separately for each database. Results: The study included 3,226 patients with mUC (male, 70.8%), with a mean (SD) follow-up of 13.8 mo (16.1). The mean (range) age was 73.8 y (23-99), and the mean (SD) Elixhauser Comorbidity score was 17.6 (11.4). Overall, 1,286 patients (39.9%) received tx in the first 12 mo post index; PB CT was the most common 1L tx (n=825, 64.2%), followed by non–PB CT (n=322, 25.0%) and IO (n=139, 10.8%). Over time, the number of patients receiving 1L tx increased (2015, 35.8%; 2019, 45.7%). Multiple factors were associated with a higher likelihood of receiving 1L tx: younger age (OR, 0.93), male sex (OR, 0.83), lower comorbidity score (OR, 0.97), previous UC-related interventions (OR, 1.65), and a more recent mUC diagnosis (OR, 1.11). In treated patients, the unadjusted median OS (interquartile range [IQR]) from index diagnosis was 13.7 mo (7-33) for SHI 1 and 13.8 mo (7-42) for SHI 2. In untreated patients, the median OS (IQR) from index diagnosis was 3.0 mo (1-11) for SHI 1 and 3.6 mo (1-18) for SHI 2. The median OS (IQR) after 1L tx initiation in PB CT–treated patients was 12.9 mo (6-33) for SHI 1 and 13.8 mo (7-49) for SHI 2; in non–PB CT–treated patients was 11.2 mo (4-36) for SHI 1 and 6.5 mo (3-15) for SHI 2; and in IO-treated patients was 4.11 mo (2-14) for SHI 1 and 8.19 mo (3 to not reached) for SHI 2. Conclusions: Our study describes real-world tx patterns/rates and clinical outcomes in patients with mUC in Germany, and highlights that the majority received no systemic tx within the first 12 mo, despite a positive trend in 1L tx rates over time. Treated patients were more likely to be younger, male, and have fewer comorbidities vs untreated patients. Systemic tx was associated with longer OS. Among treated patients, OS was longer in those receiving 1L PB CT vs other 1L tx. Future research should explore the unmet need in untreated patients to confirm alignment with updated tx guidelines and newer standards of care.

Longitudinal detection of circulating tumor DNA in patients with advanced renal cell carcinoma.

715 Background: Treatment response monitoring is key to effective disease management for patients with advanced (RCC) and can potentially improve clinical outcomes. Circulating tumor DNA (ctDNA) has shown promise as a biomarker in the early identification of treatment response in a variety of tumor types. Herein, we evaluated ctDNA-derived molecular residual disease (MRD) detection and dynamics in both clear cell (ccRCC) and non-clear cell (nccRCC) RCC patients treated with standard of care therapy and provide correlations with clinical outcomes. Methods: This was a multi-center retrospective analysis of real-world data obtained from commercial ctDNA testing (Signatera, Natera, Inc.), 41 patients (142 plasma samples) with high-risk resected or metastatic RCC were analyzed for ctDNA. Clinical data were collected on IMDC risk category, pathologic subtype, tumor stage and grade including the presence of sarcomatoid/rhabdoid features. Progression on radiological imaging was based on RECIST 1.1. criteria. Results: Of the 41 patients, 73% (30/41) had ccRCC and the remaining 27% (11/41) were nccRCC. Immunotherapy was administered as a single agent or in combination with other immune checkpoint inhibitor agents/targeted therapy in 78% (32/41) in either frontline or refractory settings. ctDNA detection at any time point was 70% (29/41) and patients were followed-up for a median of 26.4 weeks (range: 0.9-90.6) from the first ctDNA time point. Fourteen patients (34%) experienced disease progression during follow-up and all had ctDNA detected ahead of radiological progression (100% sensitivity) with a median lead time of 13.6 weeks (range: 1-39.7 weeks). Concordance between clinical outcome and ctDNA status (detected or not detected) was observed in 78% (32/41) of the patients, 12% (5/41) showed discordance, and imaging data is pending for 10% (4/41) at the time of analysis. Conclusions: Collectively, our study demonstrates high concordance between MRD status and subsequent clinical outcomes. MRD had 100% sensitivity for predicting subsequent radiographic progression with significant median lead time. MRD may serve as a valuable tool for monitoring patients on immunotherapy-based regimens for RCC.

Circulating tumor DNA (ctDNA) informs clinical practice in patients with recurrent/metastatic gastroesophageal cancers.

427 Background: Gastric and esophageal cancers (GECs) together account for a significant global burden in terms of new diagnoses and deaths. Over the last several years, the utility of ctDNA has ranged from estimating tumor burden and characterizing the genomic landscape of tumor biology and response to therapy in the metastatic setting to detection of minimal residual disease (MRD) and cancer surveillance in the locally advanced setting. We have previously studied the role of a commercial ctDNA assay for MRD detection and surveillance in locally advanced GECs. Herein, we present our experience with the same ctDNA assay in advanced stage settings for GECs. Methods: In this retrospective analysis of real-world data obtained from commercial circulating tumor DNA (ctDNA) testing, 53 patients with recurrent/ metastatic esophageal cancer were analyzed. A total of 216 plasma samples were collected between 10/29/2008 and 08/23/2022. The patients were divided into 3 cohorts: Cohort A (N=30) included patients with stage II/III disease who had confirmed clinical recurrence (R). Cohort B (N=25) included recurrent and Stage IV patients who achieved a state of no evidence of disease (NED) on imaging. Cohort C (N=5) included recurrent and Stage IV patients who transiently achieved NED on imaging. A personalized, tumor-informed multiplex PCR-based next-generation sequencing assay (Signatera) was used to quantify ctDNA either postoperatively, on adjuvant or palliative therapy, or during active surveillance. Results: Of 53 patients, 30 patients with recurrent stage II/III esophageal cancer had ctDNA status available postoperatively within 150 days of confirmed clinical recurrence. Cohort A: Among these cases, 25 patients were ctDNA-positive ahead of clinical recurrence (sensitivity 83.3%) with a median of 31 days (range: 1-147 days). Cohort B: Next, we explored the correlation between ctDNA status and imaging, wherein, 96% (24/25) of patients showed a significant correlation between ctDNA status (positive or negative) and disease status by imaging (Fisher exact test p=0.0001). Of the 23 patients, 17 patients were ctDNA-negative and showed NED on imaging (negative predictive value of 100%; 17/17). Cohort C: 100% of patients (N=6) who demonstrated recurrent disease on imaging after NED were ctDNA-positive prior to imaging (positive predictive value of 100%). Conclusions: Our data demonstrate the utility of ctDNA in accurately predicting disease progression and support the potential use of ctDNA to inform treatment decisions or prompt early radiographic imaging. ctDNA may ultimately supersede traditional radiographic surveillance with the advantage of being minimally invasive and cost-effective in monitoring patients during/post-treatment.

Glycemic Outcomes and Feature Set Engagement Among Real-Time Continuous Glucose Monitoring Users With Type 1 or Non-Insulin-Treated Type 2 Diabetes: Retrospective Analysis of Real-World Data.

The benefits of real-time continuous glucose monitoring (RT-CGM) are well established for patients with type 1 diabetes (T1D) and patients with insulin-treated type 2 diabetes (T2D). However, the usage and effectiveness of RT-CGM in the context of non-insulin-treated T2D has not been well studied. We aimed to assess glycemic metrics and rates of RT-CGM feature utilization in users with T1D and non-insulin-treated T2D. We retrospectively analyzed data from 33,685 US-based users of an RT-CGM system (Dexcom G6; Dexcom, Inc) who self-identified as having either T1D (n=26,706) or T2D and not using insulin (n=6979). Data included glucose concentrations, alarm settings, feature usage, and event logs. The T1D cohort had lower proportions of glucose values in the 70 mg/dl to 180 mg/dl range than the T2D cohort (52.1% vs 70.8%, respectively), with more values indicating hypoglycemia or hyperglycemia and higher glycemic variability. Discretionary alarms were enabled by a large majority in both cohorts. The data sharing feature was used by 38.7% (10,327/26,706) of those with T1D and 10.4% (727/6979) of those with T2D, and the mean number of followers was higher in the T1D cohort. Large proportions of patients with T1D or T2D enabled and customized their glucose alerts. Retrospective analysis features were used by the majority in both cohorts (T1D: 15,783/26,706, 59.1%; T2D: 3751/6979, 53.8%). Similar to patients with T1D, patients with non-insulin-treated T2D used RT-CGM system features, suggesting beneficial, routine engagement with data by patients and others involved in their care. Motivated patients with diabetes could benefit from RT-CGM coverage.

Analysis of Circulating Tumor DNA to Predict Risk of Recurrence in Patients With Esophageal and Gastric Cancers.

Circulating tumor DNA (ctDNA) analyses allow for postoperative risk stratification in patients with curatively treated colon and breast cancers. Use of ctDNA in esophagogastric cancers (EGC) is less characterized and could identify high-risk patients who have been treated with curative intent. In this retrospective analysis of real-world data, ctDNA levels were analyzed in the preoperative, postoperative, and surveillance settings in patients with EGC using a personalized multiplex polymerase chain reaction-based next-generation sequencing assay. Plasma samples (n = 943) from 295 patients at > 70 institutions were collected before surgery, postoperatively, and/or serially during routine clinical follow-up from September 19, 2019, to February 21, 2022. ctDNA detection was annotated to clinicopathologic features and recurrence-free survival. A total of 295 patients with EGC were analyzed, and 212 patients with stages I-III disease were further explored. Pretreatment ctDNA was detected in 96% (23/24) of patients with preoperative time points. Postoperative ctDNA was detected in 23.5% (16/68) of patients with stage I-III EGC within 16 weeks (molecular residual disease window) after surgery without receiving systemic therapy. ctDNA detection at any time point after surgery (hazard ratio [HR], 23.6; 95% CI, 10.2 to 66.0; P < .0001), within the molecular residual disease window (HR, 10.7; 95% CI, 4.3 to 29.3; P < .0001), and during the surveillance period (HR, 17.7; 95% CI, 7.3 to 50.7; P < .0001) was associated with shorter recurrence-free survival. In multivariable analysis, ctDNA status and clinical stage of disease were independently associated with outcomes. Using real-world data, we demonstrate that postoperative tumor-informed ctDNA detection in EGC is feasible and allows for enhanced patient risk stratification and prognostication during curative-intent therapy.

Prognostic factors for survival in extensive-stage small cell lung cancer: An Italian real-world retrospective analysis of 244 patients treated over the last decade.

Potential relationships with the prognosis of patients with extensive-stage non-small cell lung cancer (ES-SCLC) have been investigated without valid results. A retrospective analysis of real-world data of consecutive patients with ES-SCLC admitted to our Medical Thoracic Oncology Unit was carried out from 2010 to 2020, focusing on identification of prognostic factors. Kaplan-Meier analysis was used to represent progression-free survival (PFS) and overall survival (OS). Univariable and multivariable Cox models were used to investigate prognostic factors. The analysis included 244 patients. The median OS was 8months (95% confidence interval [CI]: 8-10) and the median PFS was 5months (95% CI: 5-6). The univariable analysis showed that factors associated with shorter OS were older age (p=0.047), TNM stage 4 versus 3 (p < 0.001), Eastern Cooperative Oncology Group (ECOG) performance status (PS) 1 and 2 versus 0 (p < 0.001), and >2 metastatic sites (p=0.004). Mediastinal radiotherapy (RT) (p < 0.001), >1 irradiated site (p=0.026), 3 and 4 chemotherapy (CT) lines versus 1 (p=0.044 and 0.001, respectively), prophylactic cranial irradiation (PCI) (p < 0.001), and surgery (p=0.001) correlated with longer OS. The multivariable analysis revealed statistically significant associations for TNM, ECOG PS 2 versus 0, number of CT lines, PCI, and surgery. A total of 23 patients (9.4%) survived ≥24 months, 39% of whom had received four CT lines and 48% had mediastinal RT. Our data suggest that tumor burden, PS, and mediastinal RT strongly correlate with outcome. With the addition of immunotherapy to CT, the identification of new biomarkers as predictive factors is urgently required.

PSAT095 Examination of Treatment Patterns in Patients with Classic Congenital Adrenal Hyperplasia (CAH) Compared to Treatment Guidelines

Abstract Introduction Classic congenital adrenal hyperplasia (CAH) is a rare autosomal recessive disorder, usually due to a deficiency in the 21-hydroxylase enzyme, that results in impaired cortisol synthesis and excess androgen production. Recommended guidelines for management of CAH include assessment of androgen control by routine clinical investigations and patient examination for treating both disease-related and drug-related symptoms. The objective of this study was to identify treatment patterns in patients with classic CAH with reference to guideline-directed care in the United States (US). Methods A retrospective analysis of real-world data on patients with classic CAH was carried out using claims data between Oct 2012 and July 2020 (8 years) from the EVERSANA open claims database of 290 million US citizens. The initial cohort included patients with two or more CAH-related ICD 9/ICD 10 codes at least 28 days apart, and two or more oral glucocorticoids (GCs) prescribed at least 28 days apart within one year of CAH diagnosis. The final CAH cohort combined unique patients identified from two sub-cohorts based on GC adherence (sensitivity 1: patients with 60% proportion of days covered and sensitivity 2: patients with five or more GC prescriptions from date of diagnosis to end of study period or available data for that patient). Information on demographic and clinical characteristics, comorbidities, prescribed medications (including corticosteroids), procedures, and referrals were analysed. Results The final cohort included 11,765 patients (overall mean age 28.7 years) of which 36.9% (n=4,338) were pediatric (0-17 years of age; mean age 8.4 years) and 63.1% were adults (n=7,427; mean age 40.5 years). The majority of CAH patients (62%; n=7,260) reported hydrocortisone use, with a higher proportion of pediatric patients on hydrocortisone (96%; n=4,158) compared to adults (42%; n=3,102). Other frequently prescribed corticosteroids were prednisone (30% pediatric; 68% adults), fludrocortisone (65% pediatric; 34% adults), dexamethasone (25% pediatric; 50% adults), and methylprednisolone (6% pediatric; 43% adults). Among diagnostic procedures and labs, total testosterone (47% pediatric; 46% adults), thyroid stimulating hormone (29% pediatric; 55% adults), 17-hydroxyprogesterone (62% pediatric; 33% adults), androstenedione (55% pediatric; 20% adults), glycated hemoglobin (18% pediatric; 41% adults) and free thyroxine (23% pediatric; 36% adults) were most common. Furthermore, bone age studies were reported in 19% of pediatric patients (51% of pediatric CAH patient population). Only 44% of adult CAH patients and 34% of pediatric CAH patients visited an endocrinologist or pediatric endocrinologist between Oct 2012 and July 2020. Conclusions In line with guidelines, most pediatric patients received short-acting hydrocortisone. However, a large proportion of patients with classic CAH were not treated by an endocrinologist and did not receive guideline-recommended therapy or laboratory testing or bone age assessments, putting them at risk for poor disease control and GC-related adverse events. Presentation: Saturday, June 11, 2022 1:00 p.m. - 3:00 p.m.

The Epidemiology, Treatment Patterns and Economic Burden of Different Phenotypes of Multiple Sclerosis in Italy: Relapsing-Remitting Multiple Sclerosis and Secondary Progressive Multiple Sclerosis

A retrospective analysis of real-world data was performed to assess the epidemiology and economic burden of multiple sclerosis (MS), relapsing-remitting MS (RRMS), and secondary-progressive MS (SPMS) in Italy. An observational study on administrative databases from a sample of Italian entities was carried-out. Between 01/2010-12/2017, patients with ≥1 MS diagnosis code (ICD-9-CM:340 and/or exemption code:046) and/or ≥1 disease-modifying therapies (DMTs) prescription, were included. Among MS-cohort, SPMS patients were identified by ≥2 hospitalizations or by ≥2 drug prescriptions related to MS progression. MS patients not fulfilling SPMS criteria were included as RRMS. Mean annual healthcare costs were reported during follow-up and stratified by DMT treatment/untreatment. Overall, 9543 MS patients were included; 8397 with RRMS and 1146 with SPMS. Estimated prevalence of MS was 141.6/100,000 inhabitants (RRMS 124.4/100,000 and SPMS 17.2/100,000). Mean annual cost for untreated and treated patient was respectively: €3638 and €11796 (MS-cohort), €3183 and €11486 (RRMS-cohort), €6317 and €15511 (SPMS-cohort). The first-line DMT treatment duration averaged 27.4 ± 22.8 months; the mean cost was 19004€ for the whole period. The second-line DMT treatment lasted on average 31.1 ± 24.5 months; the mean cost was 47293€ for the whole period. This study provided insights into the MS epidemiology in Italy and its economic burden. Healthcare costs associated with MS management were mainly driven by DMTs expenditure. A trend of higher healthcare-resource consumption was observed among SPMS-cohort.

Risk of Severe COVID-19 Outcomes Among Patients with Immune-Mediated Inflammatory Diseases or Malignancies: A Retrospective Analysis of Real-World Data in the United States.

IntroductionThere are concerns that patients in an immunocompromised state may be at risk for increased coronavirus disease 2019 (COVID-19) severity. The aim of this study was to describe the characteristics of patients with COVID-19 and immune-mediated inflammatory diseases (IMIDs) or malignancies and evaluate their risk of developing severe COVID-19.MethodsCases of COVID-19 (ICD-10 code U07.1 or U07.2, or positive polymerase chain reaction or antigen test) among patients with IMIDs or malignancies were identified in the US-based Optum® Electronic Health Records database between 1 February 2020 and 3 March 2021. Age- and sex-standardized risks of severe COVID-19 were calculated by condition of interest. The risks were further adjusted by multiple covariates, and 95% confidence intervals were estimated.ResultsA total of 499,772 patients with COVID-19 were identified (mean [SD] age, 46.9 [20.7] years; 57.0% female). Patients with hematologic cancers (adjusted risk ratio [aRR] 2.0, 1.8–2.1), solid tumors (aRR 1.1, 1.1–1.1), or rheumatoid arthritis (aRR 1.2, 1.1–1.3) had a significantly higher risk of severe COVID-19 compared to the general population of patients with COVID-19. Patients with systemic lupus erythematosus (aRR 1.1, 0.9–1.2), psoriasis (aRR 1.0, 0.7–1.2), ulcerative colitis (aRR 0.9, 0.8–1.1), Crohn’s disease (aRR 0.9, 0.7–1.0), or ankylosing spondylitis (aRR 0.8, 0.5–1.0) showed a comparable risk of severe COVID-19. Patients with atopic dermatitis (aRR 0.8, 0.7–0.9) or psoriatic arthritis (aRR 0.8, 0.6–1.0) showed a lower risk of severe COVID-19.ConclusionsThe risk of developing severe COVID-19 varied between the studied IMIDs and malignancies. Patients with hematologic cancers, solid tumors, or rheumatoid arthritis had significantly increased risk for severe COVID-19 compared to the general population. These findings highlight the need to protect and monitor immunocompromised patients such as those with IMIDs or malignancies as part of the strategy to control the pandemic worldwide.Supplementary InformationThe online version contains supplementary material available at 10.1007/s12325-022-02293-0.

Impact of COVID-19 Pandemic on Initiation of Immunosuppressive Treatment in Immune-Mediated Inflammatory Diseases in Austria: A Nationwide Retrospective Study.

Objective: Conventional immunosuppressive and advanced targeted therapies, including biological medications and small molecules, are a mainstay in the treatment of immune-mediated inflammatory diseases (IMID). However, the COVID-19 pandemic caused concerns over these drugs’ safety regarding the risk and severity of SARS-CoV-2 infection. Thus, we aimed to assess the impact of the COVID-19 pandemic on the initiation of these treatments in 2020. Study Design and Setting: We conducted a population-based retrospective analysis of real-world data of the Austrian health insurance funds on the initiation of conventional immunosuppressive and advanced targeted therapies. The primary objective was to compare the initiation of these medications in the year 2020 with the period 2017 to 2019. Initiation rates of medication were calculated by comparing a certain unit of time with an average of the previous ones. Results: 95,573 patients were included. During the first lockdown in Austria in April 2020, there was a significant decrease in the initiations of conventional immunosuppressives and advanced targeted therapies compared to previous years (p < 0.0001). From May 2020 onwards, numbers rapidly re-achieved pre-lockdown levels despite higher SARS-CoV-2 infection rates and subsequent lockdown periods at the end of 2020. Independent from the impact of the COVID-19 pandemic, a continuous increase of starts of advanced targeted therapies and a continuous decrease of conventional immunosuppressants during the observation period were observed. Conclusions: In IMID patients, the COVID-19 pandemic led to a significant decrease of newly started conventional immunosuppressive and advanced targeted therapies only during the first lockdown in Austria.

P-177: Outcomes in risk-stratified patients with smoldering multiple myeloma: a retrospective analysis of real-world data from the Czech Registry of Monoclonal Gammopathies

P-177: Outcomes in risk-stratified patients with smoldering multiple myeloma: a retrospective analysis of real-world data from the Czech Registry of Monoclonal Gammopathies

Clinical Significance of Color Doppler Ultrasound Applied in Patients with Peripherally Inserted Central Venous Catheters before Extubation: A Retrospective Analysis of Real-World Data

Aims and Objectives: By comparing peripherally inserted central venous catheter (PICC)-related complications in real-life patients, we aimed to analyze the significance of color Doppler ultrasound (CDU) applied routinely or once with symptoms of thrombosis before extubation. Methods: For this retrospective study, patient data from January to December 2020 were collected from an intelligent safe platform for infusion, which was embedded in the hospital information system (HIS). After screening by the inclusion and exclusion criteria, venous therapy liaisons were interviewed regarding the PICC procedures and complications of the enrolled patients. Then, the patient medical records were checked to verify the CDU usage and collect the CDU results. Difficulties in extubation and serious complications, including catheter fracture or pulmonary embolism during or after extubation, were also collected. This paper adheres to the RECORD checklist of EQUATOR guidelines. Results: Of the 1455 patients, 489 underwent CDU examination before extubation. Among these, thrombosis was detected in 37 (7.6%) patients and only 1 (0.2%) patient without thrombosis experienced difficulties in extubation. No serious complications were observed in the CDU group. Meanwhile, among the 966 patients in the non-CDU group, three (0.3%) faced difficulties and one patient developed symptoms of pulmonary embolism. Furthermore, among patients with thrombosis in the CDU group, the duration of catheter retention was significantly shorter than that in patients without thrombosis (76.65±42.55 vs. 121.04±45.99 days, P &lt;0.001). Conclusions: After evaluation of their clinical symptoms, patients without symptoms could be directly removed according to conventional procedures, while patients with symptoms should undergo CDU examination before extubation. Relevance to Clinical Practice: When removing the catheter for PICC carriers, it is assessed that the patients have no discomfort symptoms and can be directly extubated without B-ultrasound examination.

CtDNA in treatment response monitoring in patients with relapsed gynecologic malignancies.

e17501 Background: The role of immunotherapy in gynecologic malignancies continues to evolve. Use of immune checkpoint blockade (ICB) agents have consistently demonstrated less than a 15% response rate in ovarian, cervical, and subsets of endometrial cancer. Given the limited number of responders, a novel approach to treatment monitoring is needed to better sequence therapies. Circulating tumor DNA (ctDNA) surveillance in patients treated with ICB is one such approach that has been shown to predict clinical benefit. We sought to evaluate this approach in patients with recurrent gynecologic malignancies managed with ICB therapy. Methods: In this retrospective analysis of real-world data, plasma samples (n = 208) from 28 patients with recurrent/metastatic ovarian (n = 8), endometrial (n = 14), and cervical (n = 6) cancers were identified as having received ICB therapy. A personalized and tumor-informed multiplex PCR assay (Signatera™ bespoke mPCR NGS assay) was used for the detection of ctDNA in plasma samples. Serial time points were collected to monitor ctDNA levels in response to immunotherapy. Results: Response assessment results and longitudinal plasma samples were available for 19 patients. Pre-ICB plasma samples were available for 11 patients. Of these ctDNA was detected in 73% (8/11) of patients. All patients with progressive disease (10/10) had rising ctDNA levels that preceded radiological findings by a median of 2.8 months. One patient with rising ctDNA concentration presented with stable disease by imaging at the last follow-up. The remaining 8 patients demonstrated response to therapy that coincided with a declining or negative ctDNA level; 5 with partial response had a decline in ctDNA level prior to imaging (n = 3) or lack of ctDNA detection (n = 2), and 3 with complete response remained ctDNA negative, prior to imaging. Patients that exhibited a rise in ctDNA concentration (n = 11) (consecutive time points) while undergoing ICB therapy were associated with resistance to treatment (HR = 4.58 95%CI: 1.048-20.03, p = 0.04). TMB and MSI status (binary) were not predictive of response in univariate (p = 0.8, p = 0.9) analyses. Conclusions: ctDNA monitoring during the course of immunotherapy for relapsed gynecologic malignancies allows for accurate determination of therapeutic response and early prediction of disease progression. This enables timely assessment with imaging, and change in treatment plan. Our data suggest that patients with gynecologic malignancies may benefit from personalized, tumor-informed ctDNA testing to guide treatment decisions. Prospective studies are needed to establish the clinical utility of ctDNA monitoring in patients with gynecologic malignancies undergoing systemic therapies.

Features of Drug Therapy for Patients with Ankylosing Spondylitis in Moscow

Background. Systematic research work on the assessment of medicines consumption allows timely identification of effective and problematic aspects of the existing system of preferential medicines provision, allowing to regulate the optimality of its functioning. Aims to identify regional epidemiological features and key aspects of drug provision for patients with ankylosing spondylitis (AS) in Moscow in dynamics for the period 20172020. To identify the trend of changes and their specifics in order to optimize further preferential drug provision (DLO) for patients with AS in Moscow. Materials and methods. The sources of the study were the data of LLO of patients with AS within the outpatient care, registered in Moscow in 20172020. The researchers analyzed the number of patients with AS who are entitled to free and preferential drug provision, who received drugs as an outpatient link in the period 20172020. The age structure of beneficiaries with AS and their ratio by gender has been determined. A tendency was revealed in the distribution of patients according to the main drug therapy regimens, according to the drugs they received during the reporting period. The structure of LLO of patients with AS was revealed by international non-proprietary names of drugs, their dosage form, quantities, cost minus payment by patients. Results. A retrospective analysis of real-world data showed that the population with AS in Moscow increased by 1.5 times from 2017 to 2020, as well as the cost of expenses for LLO of these patients, excluding patient payments, also increased by 1.5 times. The average age of patients was 5014 years in 2017, and in 2018, 2019, and 2020. This indicator increased to 5111 years. The ratio of patients by sex was 2:1 male and female, respectively, in all periods studied. The maximum proportion of patients fell on the age group 4059 years, which was about 50% of the entire population of patients with AS during the entire study period. The second place was occupied by the age group 6079 years, which accounted for 28.10% of all patients with AS in 2017 and by 2020 decreased by 3.38%, already amounting to 24.72% of patients, the third place in the number of patients belongs to the age group 3039 years, which accounted for about 1718% of the entire population with AS during the entire study period. The analysis of the polypharmacy indicator indicates a low drug load of most patients since a small proportion of patients from 1.54% (20 people) to 2.71% (49 people) had more than 10 items for international nonproprietary names per year. The leading position was occupied by the therapeutic scheme of monotherapy NSAIDs in 2017, since 2018 the first place belongs to the scheme of therapy with one iTNF-. Conclusions. The increase in costs for the relief of adverse reactions and the treatment of concomitant diseases indicates a shift in the approach to the treatment of diseases of the musculoskeletal system, as pathologies accompanied by a wide variety of comorbid and polymorbid conditions. Despite the positive changes in the regional system of LLO of Moscow, it is possible to improve the indicators of the provision of patients with AS when evaluating its effectiveness, taking into account clinical indicators and the results of the assessment of the quality of life reported by patients.

Efficacy and Safety of Omidenepag Isopropyl 0.002% Ophthalmic Solution: A Retrospective Analysis of Real-World Data in Japan.

IntroductionThis study aimed to clarify the efficacy and safety of omidenepag isopropyl (OMDI) in a retrospective, real-world, multicenter setting.MethodsA retrospective medical chart review of patients with glaucoma and ocular hypertension receiving OMDI from November 2018 to November 2019 with at least 12 weeks of follow-up was conducted in 11 eye clinics in Japan. The participants were categorized into three therapy groups, designated the naïve monotherapy, switching monotherapy, and concomitant therapy groups. The main outcome measures were the change in intraocular pressure (IOP) at week 4 and week 12 after the initiation of OMDI treatment, and frequency of adverse drug reactions.ResultsData were collected from 827 patients. The baseline IOP in the naïve group was 16.6 ± 4.2 mmHg. The mean IOP reduction at week 4 and week 12 was − 2.9 ± 3.2 mmHg (P < 0.0001) and − 2.5 ± 2.9 mmHg (P < 0.0001), respectively. Eyes with baseline IOP less than 16 mmHg also showed a significant reduction of IOP of − 1.4 ± 2.0 mmHg at week 12. OMDI significantly reduced IOP not only in eyes with primary open-angle glaucoma but also in eyes with primary angle-closure glaucoma and secondary glaucoma. In the switching monotherapy group, IOP did not change significantly after switching from most classes of medications to OMDI, but further IOP reduction was observed in the case of switching from beta-blockers to OMDI. The frequency of adverse drug reactions was 14.1% in all participants, and the most common adverse reaction was ocular hyperemia (7.6%). No serious and severe side effects were observed in this study.ConclusionOMDI showed an IOP-lowering effect in eyes with various types of glaucoma and using various therapeutic regimens in real-world clinical practice. In addition, OMDI did not show any serious and severe side effects, suggesting the potential of OMDI as a first-line medicine for the treatment of glaucoma.Trial RegistrationUniversity Hospital Medical Information Network (UMIN): 000040040.Supplementary InformationThe online version contains supplementary material available at 10.1007/s12325-022-02069-6.