Early use of erenumab in US real-world practice

Background: Erenumab, a monoclonal antibody (mAb) developed to block the calcitonin gene-related peptide (CGRP) receptor, is approved for the prevention of migraine in adults. This retrospective observational study sought to describe early real-world use of erenumab. Methods: This study used the Practice Fusion ambulatory Electronic Health Record database, which represents approximately 6% of ambulatory care among primary care and specialist practices in the United States. Among migraine patients initiating erenumab, demographics, migraine type, comorbidities, and prior treatments were assessed during the 12-month baseline period. Treatment patterns including changes in acute and preventive medications, switches to other CGRP mAbs (fremanezumab and galcanezumab), and for erenumab, changes in dose and adherence were examined among patients with 6 months of follow-up. Results: Of 3,336 patients identified (85.9% female; mean age = 49.1 years), approximately 40% had documentation of chronic migraine. Common comorbidities included non-migraine headache, anxiety, depression, and hypertension. Most patients (76.3%) initiated on the 70 mg dose of erenumab. Among 1,638 patients included in the treatment pattern analysis, 53.1% used acute medications and 55.7% used other non-specific preventive migraine medications during follow-up, reductions of 9.8% and 10.2%, respectively, over the same period of time before index. Switching to fremanezumab and galcanezumab were observed in 12.2% and 13.8% of patients, respectively. The mean proportion of days covered by erenumab at 6 months was 79%. Dosage of erenumab increased (from 70 mg to 140 mg) in 13.0% and decreased (from 140 mg to 70 mg) in 24.9% of patients. Conclusion: This early real-world study showed high adherence among erenumab users. This combined with observed reductions in previously used acute and preventive medications are suggestive of erenumab’s benefit.

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Abstract 1122‐000142: Safety and Efficacy of Surpass Streamline for Intracranial Aneurysms: A Multicenter US Real‐World Experience (SESSIA)

Stroke: Vascular and Interventional Neurology ◽

10.1161/svin.01.suppl_1.000142 ◽

2021 ◽

Vol 1 (S1) ◽

Author(s):

Juan Vivanco‐Suarez ◽

Alan Mendez‐Ruiz ◽

Farooqui Mudassir ◽

Cynthia B Zevallos ◽

Milagros Galecio‐Castillo ◽

...

Keyword(s):

Real World ◽

Intracranial Aneurysms ◽

The United States ◽

Chromium Alloy ◽

Cobalt Chromium ◽

Anterior Circulation ◽

Safety And Efficacy ◽

Post Marketing ◽

The Mean

Introduction : Flow diversion has established itself as standard treatment of wide complex intracranial aneurysms (IA). Its recognition has been validated with positive occlusion rates and favorable clinical outcomes. The Surpass Streamline (SS) flow diverter (FD) is a braided cobalt/chromium alloy implant with 72 or 96 wires approved by the FDA in 2018. The aim of this study is to determine the safety and efficacy of the SS in a post‐marketing large US cohort. Methods : We performed a multicenter, retrospective study for consecutive patients treated with the SS FD for IA between January 2018 and June 2021 in the United States. Inclusion criteria for participants were: 1. Adults (≥ 18 years) and 2. Treatment with SS FD for IA. Primary safety end point was a major ipsilateral stroke (increase in National Institutes of Health Stroke Scale Score of ≥ 4) or neurological death within 12 months. Primary efficacy was assessed using the 3‐point Raymond‐Roy (RR) occlusion scale on digital subtraction angiography (DSA) at 6‐12‐month follow‐up. Results : A total of 276 patients with 313 aneurysms were enrolled. The median age was 59 years and 199 (72%) were females. The most common comorbidities included hypertension in 156 (57%) subjects followed by hyperlipidemia in 76 (28%) patients. One hundred and twenty‐two (44%) patients were asymptomatic while subarachnoid hemorrhage was present in only 10 (4%) patients. A total of 143 (46%) aneurysms were left‐sided. Aneurysms were located as follows: 274 (88%) were in the anterior circulation with paraophthalmic being the most common in 120 (38%) followed by petrocavernous ICA in 81 (26%); 33 (11%) aneurysms were located in the posterior circulation with basilar trunk being the most common in 14 (5%). The mean maximum aneurysm dome width was 5.77 ± 4.7 mm, neck width 4.22 ± 3.8 mm and dome to neck ratio was 1.63 ± 1.3 mm. The mean number of SS FD implanted per aneurysm was 1.06 (range 1–3) with more than one SS FD implanted in 21 (7%) aneurysms. Modified Rankin Scale (mRS) of 0–2 was present in 206/213 (97%) patients at 6–12 month follow‐up. The complete aneurysm occlusion (RR 1) rate was 145/175 (83%) among subjects who had angiographic follow‐up at 6–12 months. Major stroke and death was encountered in 7 (2%) and 5 (1.8%) of the patients respectively. Conclusions : Our data represent the largest real‐world study using SS FD. These results corroborate its post‐marketing safety and efficacy for the treatment of intracranial aneurysms showing more favorable rates to the initial experience during SCENT trial.

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853. Real-World Persistency of Patients Receiving Tenofovir-Based Pre-Exposure Prophylaxis for the Prevention of HIV Infection in the US

Open Forum Infectious Diseases ◽

10.1093/ofid/ofab466.1048 ◽

2021 ◽

Vol 8 (Supplement_1) ◽

pp. S516-S517

Author(s):

Alan Oglesby ◽

Guillaume Germain ◽

Francois Laliberte ◽

Staci Bush ◽

Heidi Swygard ◽

...

Keyword(s):

Hiv Infection ◽

Real World ◽

Patient Characteristics ◽

Baseline Period ◽

Hiv Diagnosis ◽

Analysis Group ◽

Populations At Risk ◽

Proportion Of Days Covered ◽

Exposure Prophylaxis

Abstract Background Once-daily oral tenofovir-based combinations as pre-exposure prophylaxis (PrEP) have shown to be an effective biomedical HIV prevention strategy for populations at-risk of acquiring HIV-1. However, low adherence can lead to poor effectiveness. This study described the characteristics of commercially-insured US PrEP users. Methods This retrospective study used IQVIA™ PharMetrics Plus data (1/1/2015–3/31/2020) to identify adults newly initiated (index date) on emtricitabine/tenofovir disoproxil fumarate (FTC/TDF) or emtricitabine/tenofovir alafenamide (FTC/TAF) as daily PrEP. Users had ≥6 months of continuous enrollment pre-index (baseline); those diagnosed with HIV or with antiretroviral therapy (ART) use during baseline were excluded. User characteristics were described during the baseline period. For FTC/TDF users, proportion of days covered (PDC), persistence, treatment breaks, and switching were described during the follow-up period, which spanned from index to the earliest of disenrollment or end of data. Non-persistence was defined as a >90-day gap from last day of supply, with re-initiation after this gap indicating treatment break. For PDC and persistence, follow-up was censored at HIV infection, defined by both multi-class ART initiation and HIV diagnosis. Results In total 24,232 FTC/TDF and 1,187 FTC/TAF users were identified. Overall, mean age was 35.1 years and 94.5% were male (Table 1). Mean [median] length of follow-up was longer for FTC/TDF (504 [390] days) than FTC/TDF users (77 [70] days). On average, FTC/TDF users had 9.0 dispensings with 38.3 days of supply per dispensing over follow-up; 11.1% had ≥1 treatment break (mean length, 249 days). Among those initiated on FTC/TDF, 10.8% switched to FTC/TAF. The mean PDC for FTC/TDF users at 6 and 12 months was 0.74 and 0.67, respectively, corresponding to 63.7% and 57.9% of patients with PDC ≥0.70 (Figure 1). Persistence to FTC/TDF at 6 and 12 months was 70.2% and 57.4%, respectively (Figure 2). Table 1. Baseline Demographics and Clinical Characteristics of PrEP Users by Regimen Figure 1. Proportion of Days Covered of FTC/TDF Users Figure 2. Kaplan-Meier Persistence Rates of FTC/TDF Users Conclusion Patient characteristics of PrEP users are broadly similar between regimens, though switching from FTC/TDF to FTC/TAF is common. FTC/TDF users had lower real-world PDC and persistence than in recent clinical trials (DISCOVER and HPTN 083). Disclosures Alan Oglesby, MPH, GlaxoSmithKline (GSK) (Employee, Shareholder) Guillaume Germain, MSc, ViiV Healthcare (Other Financial or Material Support, I am an employee of Groupe d’analyse, Ltée, a consulting company that provided paid consulting services to ViiV Healthcare for the conduct of the present study.) Francois Laliberte, MA, Viiv (Research Grant or Support) Staci Bush, NP, GlaxoSmithKline (GSK) (Employee, Shareholder) Heidi Swygard, MD, ViiV Healthcare (Employee) Sean MacKnight, MScPH, Analysis Group (Employee) Annalise Hilts, BA, Analysis Group, Inc. (Employee) Mei Sheng Duh, MPH, ScD, ViiV Healthcare (Grant/Research Support)

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Characteristics and Treatment of Patients with Multiple Myeloma Who Received Daratumumab in a Large Claims Database in the United States

Blood ◽

10.1182/blood-2021-149001 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 4031-4031

Author(s):

Snow X Feng ◽

Alex Z. Fu ◽

Vipin Khare ◽

Augustina Ogbonnaya ◽

Shuchita Kaila

Keyword(s):

Multiple Myeloma ◽

Renal Impairment ◽

Real World ◽

Index Date ◽

The United States ◽

Baseline Period ◽

Cell Transplant ◽

Claims Database ◽

The Mean ◽

Diagnosis Date

Abstract Introduction: Daratumumab (DARA) is a human IgGk monoclonal antibody targeting CD38 with a direct on-tumor and immunomodulatory mechanism of action. Since its approval for relapsed or refractory multiple myeloma (MM) in 2015 and newly diagnosed MM (NDMM) in 2018, DARA has become the foundation of treatment for MM. However, there is limited real-world evidence describing patients (pts) with MM treated with DARA-containing regimens in the US, especially for NDMM pts. Thus, using a large US claims database, we evaluated pt and treatment characteristics among MM pts who received DARA-containing regimens as first-line (1L) or second-line (2L) therapy, and separately for the subset who received DARA plus lenalidomide/dexamethasone (D-Rd) given its more recent approval in NDMM. Methods: This retrospective, observational cohort study evaluated pts aged ≥18 years at the index date (first observed treatment date with ≥1 medical or pharmacy claim for DARA) from the Optum Clinformatics Data Mart database between January 1, 2013 and December 31, 2020, with ≥1 diagnosis code for MM before the index date, and who were continuously enrolled in a medical benefit plan and pharmacy plan from ≥6 months before the initial MM diagnosis date to the index date. Pts with a hematopoietic stem cell transplant from 6 months before the observed initial MM diagnosis date to the index date were excluded. Eligible pts were categorized into 4 cohorts: pts with (1) a DARA-containing regimen as 1L (1L DARA-based cohort), (2) a DARA-containing regimen as 2L (2L DARA-based cohort), (3) D-Rd as 1L (1L D-Rd cohort), and (4) D-Rd as 2L (2L D-Rd cohort) after the observed initial MM diagnosis. Demographics were assessed on the index date; clinical characteristics, all-cause healthcare resource utilization (HCRU), and total all-cause healthcare costs (medical + pharmacy costs) were measured during the 6-month baseline period before the index date. Results: Among the 1L DARA-based cohort (n=235), 48 (20.4%) pts received D-Rd (1L D-Rd cohort). Overall, the most common 1L DARA-based regimens (with or without dexamethasone or prednisone) were D-R (28.5%), DARA plus bortezomib (V)/R (D-VR; 23.8%), D-V (18.7%), and D (14.9%). Among the 2L DARA-based cohort (n=310), 44 (14.2%) pts received D-Rd (2L D-Rd cohort). In the 1L DARA-based and 1L D-Rd cohorts, the mean (SD) age on the index date was 72.4 (9.0) and 76.1 (7.9) years, respectively. In the 2L DARA-based and 2L D-Rd cohorts, the mean (SD) age on the index date was 72.7 (9.3) and 70.5 (11.5) years, respectively. Mean baseline Quan-Charlson Comorbidity index was higher in the 2L cohorts versus 1L cohorts (1L DARA-based: 3.1; 1L D-Rd: 2.8; 2L DARA-based: 3.5; 2L D-Rd: 3.6). Hypercalcemia, renal impairment, anemia, and/or bone lesions (CRAB symptoms) were identified in about 50% of pts in all cohorts. Renal impairment was identified in 32.3%, 22.9%, 32.9%, and 34.1% of pts in the 1L DARA-based, 1L D-Rd, 2L DARA-based, and 2L D-Rd cohorts, respectively. Cardiovascular conditions (myocardial infarction, angina, peripheral vascular disease, and/or congestive heart failure) were found in 26.4% of pts in the 1L DARA-based cohort, 29.2% in the 1L D-Rd cohort, 34.5% in the 2L DARA-based cohort, and 47.7% in the 2L D-Rd cohort. The most common (>5% of pts) 1L regimens for pts in the 2L D-Rd cohort were VRd (17 [38.6%] pts), Vd (6 [13.6%]), V/cyclophosphamide (C)/d (4 [9.1%]), Rd (4 [9.1%]), and VC (3 [6.8%]). In the 1L DARA-based, 1L D-Rd, 2L DARA-based, and 2L D-Rd cohorts, 47.7%, 41.7%, 47.7%, and 54.5% of pts, respectively, had ≥1 hospitalization, and mean numbers of outpatient encounters were 26.4, 25.1, 50.6, and 52.8 within the 6-month baseline period. Mean 6-month baseline total healthcare cost (in 2020 USD) was $53,874 (medical: $51,837; pharmacy: $2,038) in the 1L DARA-based cohort, $39,695 ($35,896; $3,799) in the 1L D-Rd cohort, $131,832 ($96,737; $35,095) in the 2L DARA-based cohort, and $136,218 ($97,380; $38,838) in the 2L D-Rd cohort. Conclusions: In this real-world assessment using claims data, pts with MM receiving DARA-based therapy in 1L or 2L had median age ≥70 years, with high clinical burden as observed by their comorbidities, HCRU, and costs. Understanding the characteristics of real-world pts with MM treated with DARA-based therapy can further aid in the understanding of effective use of DARA in the real world. Disclosures Feng: Janssen: Current Employment. Fu: Janssen: Current Employment. Khare: Janssen: Current Employment. Ogbonnaya: Janssen: Current Employment. Kaila: Janssen Scientific Affairs, LLC: Current Employment.

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Real-World Healthcare Resource Utilization in Patients with Classical Hodgkin Lymphoma Treated with Pembrolizumab and Nivolumab in the United States

Blood ◽

10.1182/blood-2019-128876 ◽

2019 ◽

Vol 134 (Supplement_1) ◽

pp. 4720-4720

Author(s):

François Laliberté ◽

Monika Raut ◽

Xiaoqin Yang ◽

Guillaume Germain ◽

Shuvayu S Sen ◽

...

Keyword(s):

Hodgkin Lymphoma ◽

Real World ◽

Research Funding ◽

Index Date ◽

The United States ◽

Healthcare Resource Utilization ◽

Clinical Activity ◽

Outpatient Visits ◽

The Mean

Background: Patients with classical Hodgkin lymphoma (cHL) relapsed or refractory (R/R) disease who relapse after or are ineligible for autologous stem cell transplantation have a poor prognosis. Recently, the anti-PD1 monoclonal antibodies nivolumab and pembrolizumab were approved by the FDA (May 2016 and March 2017, respectively) as treatment options for R/R cHL patients. In the absence of head-to-head clinical trials, observational data may provide hypothesis-generating insight into the real-world outcomes of patients receiving these PD-1 inhibitors. This study aims to evaluate healthcare resource utilization (HRU) among patients with cHL initiated on pembrolizumab compared to nivolumab in the United States. Methods: A retrospective database analysis was conducted using Symphony Health's Patient Integrated Dataverse® (07/2014-06/2018). The date of the first dispensing or administration of pembrolizumab or nivolumab was assigned as the index date. Patients who received both treatments and who initiated nivolumab prior to pembrolizumab approval, or in the first months after, were classified in the pembrolizumab cohort. Included patients were required to meet the following criteria: ≥12 months of continuous clinical activity prior to the index date, ≥1 inpatient or ≥2 outpatient visits with a cHL diagnosis prior to the index date, no diagnosis of nodular lymphocyte-predominant Hodgkin lymphoma, and ≥18 years of age at the index date. Baseline patient characteristics were assessed in the 12-month baseline prior to the index date. Inverse probability of treatment weighting (IPTW) based on the propensity score was used to adjust for observed differences in baseline covariates between cohorts. Rates per person-year (PPY) of all-cause and cHL-related (i.e., visits with a primary or secondary diagnosis of cHL) HRU were calculated for weighted cohorts during the follow-up period, which spanned from the index date to the end of clinical activity or data availability. Rates of HRU were compared using rate ratios (RRs) from Poisson regression models. Results: A total of 92 patients initiated on pembrolizumab and 218 patients initiated on nivolumab met the selection criteria and were included in the analysis. Of the 92 pembrolizumab patients, 6 patients received nivolumab during the baseline period. After weighting, the mean age was similar at 55 years in both cohorts, while the proportion of female was lower in the pembrolizumab cohort (35.3%) compared to the nivolumab cohort (44.1%; standardized difference [StD]=17.9%). The mean (median) follow-up period was 295 (264) days for pembrolizumab users and 274 (208) days for nivolumab users (StD=9.4%). Mean Quan-Charlson Comorbidity Index score was well balanced after weighting in the pembrolizumab and nivolumab cohorts (4.2 and 4.3, respectively; StD=2.2%); 13.8% and 15.0% had depressive disorders (StD=3.7%), and 8.5% and 10.2% had substance-related and addictive disorders (StD=5.8%), respectively. The mean number of baseline hospitalizations (pembrolizumab=1.6, nivolumab=1.5; StD=3.1%) was also well balanced after weighting. The total person-time of observation was 74.6 and 163.6 years for the weighted pembrolizumab and nivolumab cohorts, respectively. Over this period, patients in the pembrolizumab cohort had significantly lower rates of all-cause hospitalizations (0.46) PPY than those in the nivolumab cohort (1.39; RR [95% confidence interval, CI]=0.33 [0.09-0.80], P=0.014; Figure). Furthermore, the rate of cHL-related hospitalizations PPY was significantly lower in the pembrolizumab cohort (0.06) compared to the nivolumab cohort (0.42; RR [95% CI]=0.14 [0.02-0.37], P<0.001; Figure). A similar trend (i.e., ratio below 1) was observed for all-cause and cHL-related outpatient visits, but the difference was not statistically significant (all-cause RR [95% CI]=0.84 [0.56-1.11], P=0.200; cHL-related RR [95% CI]=0.90 [0.47-1.42], P=0.647). Conclusion: In this real-world study, adult cHL patients treated with pembrolizumab had significantly lower rates of all-cause and cHL-related hospitalizations than those treated with nivolumab. Fewer all-cause and cHL-related outpatient visits were also observed among pembrolizumab users. Additional research is warranted to further investigate these early trends in real-world HRU observed among patients with cHL receiving anti-PD1 therapies. Disclosures Laliberté: Janssen Scientific Affairs, LLC: Research Funding; Merck & Co., Inc.: Research Funding. Raut:Merck & Co., Inc.: Employment. Yang:Merck & Co.: Employment. Germain:Janssen Scientific Affairs, LLC: Research Funding; Merck & Co., Inc.: Research Funding. Sen:Merck & Co., Inc.: Employment. MacKnight:Merck & Co., Inc.: Research Funding; Janssen Scientific Affairs, LLC: Research Funding. Desai:Merck & Co., Inc.: Employment. Duh:Analysis Group, Inc., a consulting firm that has received research funding from Shire, a Takeda company, to conduct this study: Employment; Shire: Research Funding; Merck: Research Funding.

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Real-world effectiveness of app-based treatment for urinary incontinence: a cohort study

BMJ Open ◽

10.1136/bmjopen-2020-040819 ◽

2021 ◽

Vol 11 (1) ◽

pp. e040819

Author(s):

Pontus Rygh ◽

Ina Asklund ◽

Eva Samuelsson

Keyword(s):

Urinary Incontinence ◽

Cohort Study ◽

Real World ◽

Short Form ◽

Pelvic Floor Muscle ◽

Controlled Trial ◽

Healthcare Services ◽

International Consultation ◽

The Mean

ObjectivesThe efficacy of app-based treatment for stress urinary incontinence (SUI) has been demonstrated in a randomised controlled trial (RCT). In this study, we investigate the user characteristics and the effectiveness of the same app when freely available, and compare these results with the RCT.DesignProspective cohort study.ParticipantsDuring a 17-month period, 24 602 non-pregnant, non-postpartum women older than 18 years downloaded the app and responded anonymously to a questionnaire. Of these, 2672 (11%) responded to the 3-month follow-up.InterventionThree months’ use of the app Tät, containing information, a pelvic floor muscle training programme and lifestyle advice.Main outcome measuresChange in symptom severity (International Consultation on Incontinence Questionnaire-Urinary Incontinence Short Form (ICIQ-UI SF)) and subjective improvement (Patient Global Impression of Improvement (PGI-I)).ResultsOf the respondents, 88% lived in Sweden and 75% (18 384/24 602) were incontinent with a mean age of 45.5 (SD 14.1) years. The UI types, based on symptoms, were SUI (53%), urgency UI (12%), mixed UI (31%) and undefined (4%). The mean ICIQ-UI SF score was 8.2 (SD 4.0) at baseline. The mean ICIQ-UI SF score reduction at follow-up was 1.31 (95% CI: 1.19 to 1.44) with a larger reduction in those with more severe incontinence at baseline (severe/very severe 3.23 (95% CI: 2.85 to 3.61), moderate 1.41 (95% CI: 1.24 to 1.59) and slight 0.24 (95% CI 0.06 to 0.42). When the results were weighted to match the distribution of severity in the RCT, the ICIQ-UI SF score reduction was 2.2 compared with 3.9 in the RCT. Regarding PGI-I, 65% experienced improvement compared with 92% in the RCT.ConclusionsThe app Tät was effective for self-management of UI even in the real world. Although the reduction in incontinence symptoms was less than in the RCT, two-thirds of the users improved. App-based treatment reaches many women without requiring resources from ordinary healthcare services.

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Real-World Clinical Outcomes Associated with Canagliflozin in Patients with Type 2 Diabetes Mellitus in Spain: The Real-Wecan Study

Journal of Clinical Medicine ◽

10.3390/jcm9072275 ◽

2020 ◽

Vol 9 (7) ◽

pp. 2275

Author(s):

Juan J. Gorgojo-Martínez ◽

Manuel A. Gargallo-Fernández ◽

Alba Galdón Sanz-Pastor ◽

Teresa Antón-Bravo ◽

Miguel Brito-Sanfiel ◽

...

Keyword(s):

Diabetes Mellitus ◽

Real World ◽

Primary Outcome ◽

Baseline Hba1c ◽

Real World Setting ◽

Antihyperglycemic Therapy ◽

The Mean ◽

Hba1c Levels

The aims of this multicentric retrospective study were to assess in a real-world setting the effectiveness and safety of canagliflozin 100 mg/d (CANA100) as an add-on to the background antihyperglycemic therapy, and to evaluate the intensification of prior sodium–glucose co-transporter type 2 inhibitor (SGLT-2i) therapy by switching to canagliflozin 300 mg/d (CANA300) in patients with T2DM. One cohort of SGLT2i-naïve patients with T2DM who were initiated on CANA100 and a second cohort of patients with prior background SGLT-2i therapy who switched to CANA300 were included in the study. The primary outcome of the study was the mean change in HbA1c over the follow-up time. In total, 583 patients were included—279 in the cohort of CANA100 (HbA1c 8.05%, weight 94.9 kg) and 304 in the cohort of CANA300 (HbA1c 7.51%, weight 92.0 kg). Median follow-up periods in both cohorts were 9.1 and 15.4 months respectively. CANA100 was associated to significant reductions in HbA1c (−0.90%) and weight (−4.1 kg) at the end of the follow-up. In those patients with baseline HbA1c > 8% (mean 9.25%), CANA100 lowered HbA1c levels by 1.51%. In the second cohort, patients switching to CANA300 experienced a significant decrease in HbA1c (−0.35%) and weight (−2.1 kg). In those patients with baseline HbA1c > 8% (mean 8.94%), CANA300 lowered HbA1c levels by 1.12%. There were significant improvements in blood pressure in both cohorts. No unexpected adverse events were reported. In summary, CANA100 (as an add-on therapy) and CANA300 (switching from prior SGLT-2i therapy) significantly improved several cardiometabolic parameters in patients with T2DM.

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Connected health for growth hormone treatment research and clinical practice: learnings from different sources of real-world evidence (RWE)—large electronically collected datasets, surveillance studies and individual patients’ cases

BMC Medical Informatics and Decision Making ◽

10.1186/s12911-021-01491-0 ◽

2021 ◽

Vol 21 (1) ◽

Author(s):

Nea Boman ◽

Luis Fernandez-Luque ◽

Ekaterina Koledova ◽

Marketta Kause ◽

Risto Lapatto

Keyword(s):

Growth Hormone ◽

Observational Study ◽

Case Studies ◽

Real World ◽

Growth Disorders ◽

Injection Device ◽

Growth Outcomes ◽

Real World Evidence ◽

High Adherence

Abstract Background A range of factors can reduce the effectiveness of treatment prescribed for the long-term management of chronic health conditions, such as growth disorders. In particular, prescription medications may not achieve the positive outcomes expected because approximately half of patients adhere poorly to the prescribed treatment regimen. Methods Adherence to treatment has previously been assessed using relatively unreliable subjective methods, such as patient self-reporting during clinical follow-up, or counting prescriptions filled or vials returned by patients. Here, we report on a new approach, the use of electronically recorded objective evidence of date, time, and dose taken which was obtained through a comprehensive eHealth ecosystem, based around the easypod™ electromechanical auto-injection device and web-based connect software. The benefits of this eHealth approach are also illustrated here by two case studies, selected from the Finnish cohort of the easypod™ Connect Observational Study (ECOS), a 5-year, open-label, observational study that enrolled children from 24 countries who were being treated with growth hormone (GH) via the auto-injection device. Results Analyses of data from 9314 records from the easypod™ connect database showed that, at each time point studied, a significantly greater proportion of female patients had high adherence (≥ 85%) than male patients (2849/3867 [74%] vs 3879/5447 [71%]; P < 0.001). Furthermore, more of the younger patients (< 10 years for girls, < 12 years for boys) were in the high adherence range (P < 0.001). However, recursive partitioning of data from ECOS identified subgroups with lower adherence to GH treatment ‒ children who performed the majority of injections themselves at an early age (~ 8 years) and teenagers starting treatment aged ≥ 14 years. Conclusions The data and case studies presented herein illustrate the importance of adherence to GH therapy and how good growth outcomes can be achieved by following treatment as described. They also show how the device, software, and database ecosystem can complement normal clinical follow-up by providing HCPs with reliable information about patient adherence between visits and also providing researchers with real-world evidence of adherence and growth outcomes across a large population of patients with growth disorders treated with GH via the easypod™ device.

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A-136 The Transfer of Cognitive Training to Real-World Functioning: Results of our First Six Pilot Subjects at Six-Month Follow-Up

Archives of Clinical Neuropsychology ◽

10.1093/arclin/acab062.154 ◽

2021 ◽

Vol 36 (6) ◽

pp. 1187-1188

Author(s):

Jason A Blake ◽

Brandon Mitchell ◽

Staci McKay ◽

Gitendra Uswatte ◽

Edward Taub

Keyword(s):

Cognitive Training ◽

Real World ◽

Cognitive Task ◽

Case Series ◽

Performance Measure ◽

The Real ◽

Cognitive Activities ◽

The Mean ◽

Laboratory Measures

Abstract Objective Currently, the majority of cognitive training research measures treatment efficacy using in-laboratory measures, with minimal focus on real-world treatment changes. This case series demonstrates the feasibility of transferring cognitive improvements from the laboratory into the everyday life setting. Method This case series includes 6 chronic post-stroke participants; mild to moderate cognitive impairment. The intervention combines cognitive training with behavioral techniques, known as the Transfer Package (TP). The TP involves components that target functionality on IADLs in the real-world. Performance on cognitively-based IADLs in the real world are measured pre-treatment, post, and 6-month follow-up. Measures of real-world ability are the: Canadian Occupational Performance Measure (COPM), Cognitive Task Activity Log (CTAL) and Inventory of Improved and New Abilities (INCA). In-laboratory measures included the D-KEFS and Timed IADL assessments. Results The real-world outcome measures used in this study were the COPM and two measures developed for this study, the CTAL and INCA. The mean change from pre to post on the COPM Performance Scale was 2.18 (SD = 1.33) and the mean change on the COPM Satisfaction Scale was 2.70 (SD = 1.27). The mean change on the CTAL was 1.96 (SD = 0.93). On the INCA, the mean number of improved real-world cognitive activities was 11.8 (SD = 4.9) and the mean number of new cognitive activities was 7.6 (SD = 3.9). Follow-up reported near-perfect retention on CTAL and continued improvement on the INCA. There were minimal changes on in-laboratory measures. Conclusions This case series provides a framework for achieving the transfer of cognitive training treatment effects in the real-world life situation by overcoming behavioral barriers to functioning.

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Abstract P643: Incidence of Ischemic Stroke Among Migraineurs on Calcitonin Gene-Related Peptide Inhibitors

Stroke ◽

10.1161/str.52.suppl_1.p643 ◽

2021 ◽

Vol 52 (Suppl_1) ◽

Author(s):

Juliana Gomez ◽

Mark Burish ◽

Sean I Savitz ◽

Louise D McCullough ◽

Cecilia Ganduglia Cazaban

Keyword(s):

Ischemic Stroke ◽

Age Groups ◽

Vascular Risk Factor ◽

The United States ◽

Calcitonin Gene Related Peptide ◽

Related Peptide ◽

Administrative Dataset ◽

Calcitonin Gene

Introduction: Migraine is common and often disabling. Recent research has focused on a new class of treatments targeting the pain-signaling molecule calcitonin-gene-related peptide (CGRP). CGRP inhibitors are the first preventive therapies designed specifically for migraine. However, CGRP also induces vasodilation, and plays a role in cerebral blood flow autoregulation. The clinical cerebrovascular effects of long-term blockade of CGRP are not yet known. Methods: We analyzed a retrospective cohort of migraine patients from a large administrative dataset in the United States (OPTUM) that covers patients with commercial insurance and Medicare Advantage, during 2018 and 2019, using ICD-10 codes for all types of migraines. Migraine patients were divided into two groups based on their use of CGRP inhibitors (erenumab, fremanezumab, galcanezumab). We determined the incidence of stroke during 2018 and 2019 among both groups. For the group of migraine patients who used CGRP inhibitors, we focused on the incidence of stroke after these medications were initiated. Results: We identified 646,404 migraine patients; 18, 692 (2.9%) of them had used a CGRP inhibitor. Among the users of CGRP inhibitors, 86% were females, and 58% of patients were in the 41 to 65-year-old range. The average time of follow-up was 197 days after initiation of the medication. A total of 1.3 % of patients who used CGRP inhibitors had an ischemic stroke during the follow-up period versus 2.4% of patients who did not use CGRP inhibitors. The lower rate of stroke in the CGRP antibody group was found in both sexes and all age groups. Conclusions: These preliminary results suggest that CGRP inhibitors are not associated with a higher frequency of stroke in the initial months of use. This could be related to the avoidance of these medications in patients with known cardiovascular risk factors. Further analyses will be completed on vascular risk factor incidence in these populations as well as multiple regression. Prospective, long term studies are needed to confirm these results.

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OR30-01 Real-World Minimed™ 670G System Use and Glycemic Outcomes of Pediatric and Adult Individuals Living with Type 1 Diabetes (T1D) in the United States

Journal of the Endocrine Society ◽

10.1210/jendso/bvaa046.1708 ◽

2020 ◽

Vol 4 (Supplement_1) ◽

Author(s):

Robert Alan Vigersky ◽

Michael Stone ◽

Pratik Agrawal ◽

Alex Zhong ◽

Kevin Velado ◽

...

Keyword(s):

Glycemic Control ◽

Real World ◽

Adult Population ◽

Age Groups ◽

The United States ◽

System Use ◽

System Data ◽

The Mean ◽

The Impact ◽

The U.S

Abstract Introduction: The MiniMed™ 670G system was FDA-approved in 2016 for adults and adolescents ≥14yrs, and in 2018 for children ages 7-13yrs with T1D. Since then, use of the system has grown to over 180,000 people in the U.S. The glycemic control benefits of real-world MiniMed™ 670G system Auto Mode use in the U.S. were assessed. Methods: System data (aggregated five-minute instances of sensor glucose [SG]) uploaded from March 2017 to July 2019 by individuals (N=118,737) with T1D and ≥7yrs of age who enabled Auto Mode were analyzed to determine the mean % of overall time spent <54mg/dL/<70mg/dL (TBR); between 70-180mg/dL (TIR); and >180mg/dL/>250mg/dL (TAR). The impact of Auto Mode was further assessed in a sub-group of individuals (N=51,254) with, at least, 7 days of SG data for both Auto Mode turned ON and turned OFF. The % of TIR, TBR and TAR, and the associated glucose management indicator (GMI) were evaluated for the overall OFF (2,524,570 days) and ON (6,308,806 days) periods, and across different age groups. Results: System data TIR was 71.3%; TBR was 0.4% and 1.9%, respectively; and TAR was 26.8% and 6.2%, respectively. User-wise data of Auto Mode OFF versus ON showed a mean of 70.3% of the time spent in Auto Mode, that TIR increased from 60.9% to 69.9%; and that both TBR and TAR decreased. For those 7-13yrs (N=1,417), TIR increased from 48.7% to 61.5%; TBR increased from 0.5% to 0.6% and from 2.0% to 2.2%, respectively; and TAR decreased from 49.3% to 36.3% and from 20.5% to 13.0%, respectively. For those 14-21yrs (N=4,194), TIR increased from 51.0% to 61.5%; TBR decreased from 0.7% to 0.6% and from 2.3% to 2.0%, respectively; and TAR decreased from 46.7% to 36.5% and from 18.5% to 12.5%, respectively. For those ≥22yrs (N=45,643), TIR increased from 62.2% to 70.9%; TBR decreased from 0.7% to 0.5% and from 2.6% to 1.9%, respectively; and TAR decreased from 35.2% to 27.3% and from 9.9% to 6.3%, respectively. The mean GMI decreased by 0.23% (overall), 0.48% (7-13yrs), 0.35% (14-21yrs), and 0.22% (≥22yrs), respectively, with Auto Mode ON versus OFF. Discussion: In over 6 million days of real-world MiniMed™ 670G system Auto Mode use in the U.S., TIR of a large pediatric and adult population with T1D improved by 9% compared to when Auto Mode was OFF, which was comparable to or exceeded the TIR observed in the smaller pivotal trials. These results further support outcomes of the pivotal trials and increased glycemic control with system use.

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