Longitudinal Treatment Patterns Research Articles

Longitudinal Treatment Patterns of Chorea in North American Patients with Huntington's Disease: Data from Enroll-HD.

Chorea is the primary manifestation of Huntington's disease. Different clinicians pursue varied approaches to chorea management, and real-world evidence describing them is needed. The objective of this study was to assess the presence and severity of chorea, chorea pharmacotherapy, and treatment practice, and patterns in a large natural-history cohort with Huntington's disease. The Enroll-HD research platform Periodic Dataset 5.0 was used to select subjects. Outcomes included demographics, disease-related baseline characteristics (Primary Analysis Set), and treatment patterns (Treatment Analysis Set). A total of 2590 manifest participants comprised the Primary Analysis Set with 1040 in the Treatment Analysis Set; 96.8% of participants had chorea. Mean Unified Huntington's Disease Rating Scale scores for Total Maximal Chorea, Total Motor Score, and Total Functional Capacity were 9.6, 39.5, and 7.8, respectively. During the observation period from June 2012 to October 2020, 906 (36.1%) participants received treatment for chorea. Among these, the most common first-line therapies were monotherapy VMAT2 inhibitors (49.9%) and antipsychotics (27.7%), while 7.8% of participants discontinued first-line therapy. Of those receiving VMAT2 inhibitors or antipsychotics as first line, 92% and 84%, respectively, remained on VMAT2 inhibitors or antipsychotics alone or in combination for the duration of the study. The most common second-line treatment was combination therapy. Only 36.1% of participants with chorea were taking a medication indicated for chorea, and, while 49.9% of treated participants received VMAT2 inhibitors first-line, approximately half were prescribed off-label alternatives. It is unclear why patients with indications for treatment were untreated or why off-label alternatives were prescribed. Future research should elaborate on these observations.

Characterizing the journey of Rett syndrome among females in the United States: a real-world evidence study using the Rett syndrome natural history study database

BackgroundWith the advent of the first targeted therapy for Rett Syndrome (RTT), a comprehensive assessment of the journey of RTT is needed to elucidate on present unmet needs in this population. This study characterized females with RTT in the United States and their disease journey with respect to longitudinal treatment patterns, RTT-related outcomes, and changes in disease severity.MethodsThis retrospective cohort study used registry data of females with RTT from the 5211 RTT Natural History Study (RNHS) (November 2015–July 2021). Pharmacological and supportive therapy use, RTT-related outcomes, and RTT severity, as measured by the Clinical Severity Scale and Motor Behavioral Assessment scale, were evaluated following the first RNHS visit. Analyses were conducted overall and in subgroups by RTT type (classic and atypical RTT) and age at first visit (pediatric and adult).ResultsA total of 455 females with RTT were included in the study, of whom 90.5% had classic RTT and 79.8% were pediatric individuals. Over a median follow-up of 4 years, use of pharmacological therapies, including prokinetic agents (42.7% vs. 28.3%), and supportive therapies, including physical therapy (87.3% vs. 40.2%) and speech-language therapy (86.8% vs. 23.9%), were more common in pediatric than adult individuals (all p < 0.05). Nearly half (44.6%) of all individuals had a hospital or emergency room visit, with a higher proportion of visits in individuals with classic RTT than atypical RTT and pediatric than adult individuals (both p = 0.001). An increasing trend in clinical severity was observed in pediatric individuals (mean change per year: 0.24; 95% confidence interval [CI]: 0.03, 0.44), while an increasing trend in motor-behavioral dysfunction was observed in pediatric individuals (mean change per year: 1.12; 95% CI: 0.63, 1.60) and those with classic RTT (mean change per year: 0.97; 95% CI: 0.53, 1.41).ConclusionsFindings from this study highlight the considerable burden of RTT across disease subtype and age. Despite reliance on supportive therapies and healthcare encounters, individuals with RTT experience increasing disease severity and motor-behavioral dysfunction in childhood and adolescence, underscoring the unmet needs of this population and the value of early intervention to manage RTT in the long-term.

Anemia Treatment, Hemoglobin Variability, and Clinical Events in Patients With Nondialysis-Dependent CKD in Japan.

Anemia management in patients with non-dialysis-dependent chronic kidney disease has attracted attention with the introduction of novel therapeutic agents; however, few studies have provided comprehensive epidemiologic information. A retrospective cohort study was conducted in adult patients with stage ≥3a non-dialysis-dependent chronic kidney disease and hemoglobin <11 g/dL (January 2013-November 2021; N=26,626) to assess longitudinal treatment patterns, hemoglobin, and iron parameters (ferritin and transferrin saturation) for anemia management. Time-dependent Cox proportional hazard models were applied to assess the risk of clinical events, including death, cardiovascular events, dialysis introduction, and red-blood-cell transfusion, associated with temporal fluctuation patterns of hemoglobin levels. The cumulative incidence of anemia treatment initiation within 12 months was 37.1%, including erythropoiesis-stimulating agents 26.5%, iron oral 16.8%, iron intravenous 5.1%, and hypoxia-inducible factor prolyl hydroxylase inhibitor 0.2%. The mean (±standard deviation) hemoglobin levels were improved from 9.9±1.2 g/dL to 10.9±1.6 g/dL at 12 months. Despite erythropoiesis-stimulating agents or hypoxia-inducible factor prolyl hydroxylase inhibitor therapy, 30.1% of patients remained hemoglobin <10 g/dL. The risks of premature death, cardiovascular events, dialysis introduction, and red-blood-cell transfusion were significantly higher in groups with consistently low hemoglobin or low-amplitude hemoglobin fluctuation around the lower limit of target hemoglobin range than in patients with target hemoglobin range (p <0.05). Likewise, significantly higher risks for dialysis introduction and red-blood-cell transfusion were associated with high-amplitude hemoglobin fluctuation across target hemoglobin range were observed. The findings underscore the importance of stable hemoglobin control within the target range to reduce the mortality and morbidity risks in patients with non-dialysis-dependent chronic kidney disease while highlighting the suboptimal and heterogeneous treatment of anemia in clinical practice.

Longitudinal treatment patterns in patients recently diagnosed with type 2 diabetes mellitus in Catalonia

AimTo investigate initial and subsequent treatments prescribed to newly diagnosed type 2 diabetes mellitus (T2DM) patients. MethodsData from SIDIAP (Information System for Research in Primary Care) including all recorded incident T2DM patients in primary care between 2015 and 2020. We used descriptive statistics and different graphical techniques to describe the most frequent longitudinal patterns. ResultsA total of 86,854 patients were included. 78.3 % of the patients began treatment with a single metformin medication and 21.7 % began with a combination therapy (CT). Metformin was the most frequent treatment as first and third-line therapy, while the CT of metformin with DPP4i or sulfonylurea was more prevalent as second-line. Most common first to third-line pattern was initial metformin for 15 months, adding a second antidiabetic in the second line, staying in CT for 6 months, and switching back to single metformin. Treatment patterns varied depending on HbA1c levels, with higher levels (>8 %) being associated with changes to CT and lower levels with switches to monotherapy or temporary discontinuation. ConclusionThe study described in detail the different treatment patterns in incident T2DM patients in Catalonia, its adherence to the guidelines, and how the changes are associated to the HbA1c dynamics.

Concordance of the treatment patterns for major depressive disorders between the Canadian Network for Mood and Anxiety Treatments (CANMAT) algorithm and real-world practice in China.

Background: Antidepressant (AD) algorithm is an important tool to support treatment decision-making and improve management of major depressive disorder (MDD). However, little is known about its concordance with real-world practice. This study aimed to assess the concordance between the longitudinal treatment patterns and AD algorithm recommended by a clinical practice guideline in China. Methods: Data were obtained from the electronic medical records of Shanghai Mental Health Center (SMHC), one of the largest mental health institutions in China. We examined the concordance between clinical practice and the Canadian Network for Mood and Anxiety Treatments (CANMAT) algorithm among a cohort composed of 19,955 MDD patients. The longitudinal characteristics of treatment regimen and duration were described to identify the specific inconsistencies. Demographics and health utilizations of the algorithm-concordant and -discordant subgroups with optimized treatment were measured separately. Results: The overall proportion of algorithm-concordant treatment significantly increased from 84.45% to 86.03% during the year of 2015–2017. Among the patients who received recommended first-line drugs with subsequent optimized treatment (n = 2977), the concordance proportion was 27.24%. Mirtazapine and trazodone were the most used drugs for adjunctive strategy. Inadequate or extended duration before optimized treatment are common inconsistency. The median length of follow-up for algorithm-concordant (n = 811) and algorithm-discordant patients (n = 2166) were 153 days (Q1-Q3 = 79–328) and 368 days (Q1-Q3 = 181–577) respectively, and the average number of clinical visits per person-year was 13.07 and 13.08 respectively. Conclusion: Gap existed between clinical practice and AD algorithm. Improved access to evidence-based treatment is required, especially for optimized strategies during outpatient follow-up.

Longitudinal Treatment Patterns for Chorea in Patients with Huntington Disease: Data from Enroll-HD (S36.009)

Longitudinal Treatment Patterns for Chorea in Patients with Huntington Disease: Data from Enroll-HD (S36.009)

Abstract OT2-19-04: European treatment patterns and outcomes associated with first-line CDK4/6 inhibition and hormonal therapies assessed in a real-world non-interventional study (EUCHARIS)

Abstract Background: Palbociclib is currently the most commonly used cyclin-dependent kinase 4/6 inhibitor (CDK4/6i) for patients with hormone receptor-positive (HR+)/human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer (ABC) in Europe since its approval in 2017. However, large-scale assessments of palbociclib utilization and subsequent clinical outcomes in real-world settings are limited. Documentation of longitudinal treatment patterns and effectiveness following initiation of first-line (1L) palbociclib + aromatase inhibitor (AI) in more heterogeneous, real-world patient populations are needed to complement clinical trial findings. Trial Design: This is an observational cohort study using retrospective data abstracted from the medical records of patients with HR+/HER2- ABC who initiated 1L treatment (index date) with palbociclib + AI (September 2016-July 2020) or AI monotherapy (January 2010 to July 2020) as 1L therapy for ABC. Patients' study index dates will be at least 12 months before the data abstraction date to allow a minimum potential follow-up of at least 12 months. Patients who died within &amp;lt; 12 months after the index date will remain eligible. Eligibility Criteria: Adult patients who received a de novo or a recurrent diagnosis of locally advanced BC (i.e., not amenable to curative therapy) or metastatic BC will be included. Patients who participated in a clinical trial for ABC, had evidence of prior other malignant neoplasm, received any CDK4/6i for early stage disease, and received other CDK4/6i for ABC will be excluded. Study Aims: This study aims to describe patient characteristics, treatment patterns, and clinical outcomes among patients with HR+/HER2- ABC in Europe who received 1L CDK4/6i treatment. Key outcomes of interest include real-world tumor response, real-world progression-free survival, and overall survival. An exploratory objective will be to compare clinical outcomes between patients treated with 1L palbociclib + AI versus AI monotherapy. Statistical Methods: All study measures will be summarized descriptively. Time-to-event outcomes will be estimated using the Kaplan-Meier method. Direct covariate matching at the site-level and inverse probability weighting using propensity scores is planned to account for differences in baseline patient and clinical characteristics between treatment groups in the exploratory comparative analysis. Target Accrual: Data abstraction is anticipated to begin in September 2021. Data will be abstracted for up to 2,400 eligible patients. Approximately 60 total sites from across Germany, Spain, and the United Kingdom and, if feasible, from other European countries will be recruited. Citation Format: Ernest H. Law, Elena Galve-Calvo, Achim Wöckel, Rohan Parikh, Samantha K. Kurosky, Valerie Derrien Ansquer, Gaelle Frugier, Keith L. Davis, Elizabeth J. Esterberg, Olga Oikonomidou. European treatment patterns and outcomes associated with first-line CDK4/6 inhibition and hormonal therapies assessed in a real-world non-interventional study (EUCHARIS) [abstract]. In: Proceedings of the 2021 San Antonio Breast Cancer Symposium; 2021 Dec 7-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2022;82(4 Suppl):Abstract nr OT2-19-04.

PCN239 Development of the NOBLE (NOvartis Braf+ meLanoma patients obsErvational) Dataset: a Novel Approach to Creating a Longitudinal Melanoma Data Source by Harmonizing Multiple Electronic Health Record Databases

Although clinical trials have shown that novel immuno-oncology (IO) and targeted therapies (TT) improve clinical outcomes, monitoring longitudinal treatment patterns and outcomes among patients in real-world settings is crucial. Here, we describe the development of NOBLE (Novartis Braf+ meLanoma patients ObsErvational) dataset, to inform critical questions about risk factors, treatment patterns, real-world outcomes, and complement clinical trial data.

Treatment patterns in pediatric patients with multiple sclerosis in Germany-a nationwide claim-based analysis.

Background:The manifestation of multiple sclerosis (MS) in childhood and adolescence occurs in 3%−5% of all MS cases. However, the immunomodulatory and symptomatic treatment options in this population group are still limited.Objective:We aimed to elucidate the prescription frequency of medications used in pediatric patients with multiple sclerosis (PwMS) compared with the general population, considering the entire spectrum of medications prescribed.Methods:Based on nationwide outpatient drug prescription data and statutory health insurance (SHI) physicians’ claims data from 2018, we conducted a population-based cross-sectional study in Germany. Children and adolescents aged ⩽17 years (n = 11,381,939) diagnosed with MS (n = 613), and a matched (age, sex, and health insurance sector) control group (n = 6130) were included. The prescription prevalence was measured as the proportion of MS patients with ⩾1 prescription.Results:Of the 613 pediatric PwMS with a median age of 16 years, 403 (65.7%) were female. For 15 out of the 18 different active agents analyzed, PwMS had a significantly higher prescription prevalence than the control group (Fisher’s exact test: p ⩽ 0.037). The most frequently prescribed drugs in PwMS were ibuprofen (28.4%; anti-inflammatory drug), cholecalciferol (23.0%; vitamin D3), and interferon beta-1a (21.5%; disease-modifying drug, DMD). The proportions of DMD prescriptions and antibiotic prescriptions were higher among PwMS aged 15–17 years than among those ⩽14 years (DMD: 43.4% vs 34.2%, p = 0.05; antibiotic: 34.1% vs 24.8%, p = 0.031). In contrast, younger PwMS were more likely to receive a prescription for anti-inflammatory/anti-rheumatic drugs (36.6% vs 26.5%, p = 0.02).Conclusion:Our study analyzing real-world medication data showed that interferon beta, anti-inflammatory drugs, and vitamins play an essential role in the treatment of pediatric PwMS. Future research should evaluate longitudinal treatment patterns of pediatric PwMS, paying particular attention to the time of diagnosis, time of first DMD initiation, and therapy switches.

Epidemiology, longitudinal treatment patterns and outcomes of chronic lymphocytic leukemia in Israel

This study aims to describe chronic lymphocytic leukemia (CLL) epidemiology, treatment patterns and outcomes in a 2.3-million-member healthcare provider database (Maccabi Healthcare Services, Israel). Newly-diagnosed CLL patients (1999–2017) were followed through 31/3/2018. A total of 1857 newly-diagnosed CLL patients were included. Annual incidence was 5.82 per 100,000 population. Median overall survival (OS) was 12.7 (95%CI: 11.8–13.5) years since diagnosis. Approximately 1/3 initiated treatment within 5 y. A statistical trend (p = 0.066) for improved OS over time was observed among younger patients (age <70 y) treated in 2009–2017 vs. 1999–2008). Among patients treated since 2009 (n = 411; median age = 68y), fludarabine–cyclophosphamide–rituximab (FCR), bendamustine–rituximab and obinutuzumab ± chlorambucil accounted for 19.5%, 12.2% and 11.4% of first line, respectively. Median (95%CI) time to next treatment and OS were 3.1(2.6–3.6) and 7.0(6.3–7.7) years, respectively. CLL incidence in Israel is comparable to developed countries. Real-world data suggest a trend of improved survival over the last decade among patients treated before age 70.

Disease and Treatment Patterns Among Patients With Pouch-related Conditions in a Cohort of Large Tertiary Care Inflammatory Bowel Disease Centers in the United States.

BackgroundGaps exist in our understanding of the clinical course of pouch-related disorders.MethodsWe evaluated baseline disease activity and longitudinal treatment patterns among patients with inflammatory conditions of the pouch.ResultsAmong 468 patients with an ileal pouch-anal anastomosis (IPAA), 94 (20%) had acute pouchitis, 96 (21%) had chronic pouchitis, and 192 (41%) had Crohn disease of the pouch. Following an IPAA, 38% of patients were treated with a biologic and 11% underwent inflammatory bowel disease- or bowel-related surgery.ConclusionsTreatment patterns after IPAA indicate that pouch-related disorders have a significant impact on individual patients and the healthcare system.

Longitudinal treatment patterns and adverse events (AEs) in younger patients (Pts) with metastatic triple-negative breast cancer (mTNBC): A real-world landscape analysis.

e12562 Background: While no uniform standard of care exists for mTNBC, conventional chemotherapy remains the treatment mainstay. This retrospective analysis of U.S. commercial claims data was conducted to characterize real-world longitudinal chemotherapy treatment patterns and predictors of AEs in mTNBC. Methods: We assessed all pts with mTNBC aged 18-60 years starting first-line (1L) chemotherapy from 01/01/2011 to 12/31/2015 in the IMS LifeLink database, which does not report on investigational therapies. Longitudinal treatment patterns, treatment duration, and AEs were characterized by line-of-therapy (LOT). The primary and secondary endpoints were any AE and treatment duration. Multivariable logistic and Cox regression analyses were used to identify clinical risk factors for AEs and predictors of longer treatment duration, respectively. Results: 1,447 mTNBC pts receiving ≥1 LOT were identified (median [range] age, 51 [24-60] years), of which 54% received ≥2 and 8% ≥3 LOT. Combination therapy (combi-Tx) was used as 1L for 73%, second-line (2L) for 22%, and third-line (3L) for 32% of pts. The most common combi-Tx was cyclophosphamide/doxorubicin (1L: 40%; 2L: 5%), and carboplatin/gemcitabine in 3L (17%). The most common 1L and 2L monotherapies (mono-Tx) were taxanes (10% and 66%), and 3L capecitabine (13%). Median treatment duration for 1L to 3L was 56-58 days, and for combi- vs mono-Tx in 1L, 50 vs 71 days. The proportion of pts experiencing any AE was 34% during 1L, 38% during 2L, and 56% during 3L therapy. After adjusting for key confounders including Charlson comorbidity index, independent risk factors for AEs were later LOT, 3L vs 1L (OR = 3.20, 95%CI: 2.13-4.79), and younger age groups vs age 55-60 (OR = 1.94, 95%CI: 1.48-2.55). Additional analysis will assess independent predictors of treatment duration. Conclusions: This real-world study reveals considerable treatment heterogeneity and short treatment durations even with conventional combination chemotherapies. Among non-Medicare mTNBC pts, younger age is a risk factor for AEs possibly due to more aggressive therapies. More treatment options are needed for pts with mTNBC.

Economic burden and treatment patterns for patients with diffuse large B-cell lymphoma and follicular lymphoma in the USA.

Diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL) are common types of non-Hodgkin's lymphoma, and real-world evidence continues to be lacking for healthcare costs and utilization among DLBCL and FL patients. Our study aims to describe medical and pharmacy costs and health resource utilization and to characterize longitudinal treatment patterns among these patients. A retrospective observational study was performed among adult patients with DLBCL or FL using the US MarketScan (Truven) administrative claims data from 1 January2007 to 31 December2015. Diagnoses of DLBCL and FL were based upon ICD-9 codes. Identifications of treatment lines involved 30 lymphoma-specific anticancer systemic agents. Direct healthcare costs and utilizations were computed in the 1-year postdiagnosis period. Generalized linear models with a gamma link were used to compare healthcare costs between therapies with and without rituximab. A total of 2767 DLBCL and 5989 FL patients received frontline therapy. The majority received treatment within 3 months after initial diagnosis (DLBCL 79.9% and FL 62.4%) and were treated with rituximab or bendamustine either alone or in combination (DLBCL 67.4% and FL 84.7%). The total healthcare costs were US $15,555 and $10,192 per patient per month within 1 year following their initial diagnosis for DLBCL and FL, respectively. The medical costs were nearly twice as much as the drug costs for DLBCL patients. Both DLBCL and FL patients receiving rituximab had higher pharmacy costs but lower medical costs (p<0.001). During the first year following initial diagnosis, the resource utilization (per patient per month) of DLBCL patients included 0.21 inpatient admissions, 0.26 radiation therapy, 2.63 outpatient or office visits, 0.18 emergency room visits, 0.06 intensive care unit admissions and 0.10 stem cell transplantation. FL patients occupied less health resources than DLBCL patients. The healthcare costs and health resources utilized were considerable in non-Hodgkin's lymphoma, especially DLBCL patients.

Treatment Patterns in Type 2 Diabetes Patients with Chronic Kidney Disease-A Cohort Study

Introduction: The prevalence of type 2 diabetes mellitus (T2DM) is increasing in the U.S. and 40% of affected people develop chronic kidney disease (CKD). This study evaluated patients’ treatment patterns, disease burden and progression. Methods: This retrospective study used the U.S. claims database PharMetrics Plus (January 2006-December 2015) and the Experian consumer data asset (January 2007-December 2015) to evaluate longitudinal treatment patterns in patients with T2DM and CKD using ICD codes. In addition, CKD progression, all-cause mortality rate, clinical events and health care costs were described. Results: Of 250,742 patients identified, the median age was 61 years, 58% were male, 73% were Caucasian and 87% had hypertension. Proportions of patients using angiotensin II receptor blockers (ARB) or angiotensin-converting-enzyme inhibitors (ACEi) was lower in CKD stages 4 and 5 (54 and 52%, respectively) than in stages 1-3 (66-70%). The use of beta blockers (32-52%), calcium channel blockers (27-41%) and diuretics (37-54%) tended to increase from CKD stages 1 to 4 and decreased in stage 5. Antidiabetic drugs were prescribed to 65.7% of patients and their use decreased with increasing CKD stage. Of the 21% of patients who progressed within a year to a more severe CKD stage, the highest proportion advanced from stage 4 to 5 (42%). The most common clinical events were diabetic retinopathy, heart failure and acute kidney injury reported in 27%, 22% and 22% of patients, respectively. Overall, 4.6% died with a median time from index to death of 12.3 months. The annual health care costs of $46,028 per person included $9,103 and $36,925 for pharmacy and medical costs, respectively. Conclusions: The treatment of T2DM patients with CKD was mostly in line with current guideline recommendations. However, only up to 70% of patients used ARB and ACEi, the first line treatments. In addition, 21% of patients progressed to a more severe CKD stage. These findings suggest a need for new treatment options in this population. Disclosure M. Blankenburg: None. C.P. Kovesdy: Consultant; Self; Abbott. Advisory Panel; Self; AbbVie Inc.. Consultant; Self; Bayer AG. Advisory Panel; Self; AstraZeneca. Consultant; Self; Sanofi Genzyme. Advisory Panel; Self; Amgen Inc.. Consultant; Self; Keryx. S. Eisenring: None. A. Fett: Consultant; Self; IQVIA. E.W. Schokker: None. J. Korn: None. A. Gay: Employee; Self; Bayer AG.

Comorbidities, treatment patterns and cost-of-illness of acromegaly in Sweden: a register-linkage population-based study.

Acromegaly is a complex endocrine disease with multiple comorbidities. Treatment to obtain biochemical remission includes surgery, medical therapy and radiation. We aimed to describe comorbidities, treatment patterns and cost-of-illness in patients with acromegaly in Sweden. A nationwide population-based study. Patients with acromegaly were identified and followed in national registers in Sweden. Longitudinal treatment patterns were assessed in patients diagnosed between July 2005 and December 2013. The cost-of-illness during 2013 was estimated from a societal perspective among patients diagnosed between 1987 and 2013. Among 358 patients with acromegaly (48% men, mean age at diagnosis 50.0 (s.d. 15.3) years) at least one comorbidity was reported in 81% (n = 290). The most common comorbidities were hypertension (40%, n = 142), neoplasms outside the pituitary (30%, n = 109), hypopituitarism (22%, n = 80) and diabetes mellitus (17%, n = 61). Acromegaly treatment was initiated on average 3.7 (s.d. 6.9) months after diagnosis. Among the 301 treated patients, the most common first-line treatments were surgery (60%, n = 180), somatostatin analogues (21%, n = 64) and dopamine agonists (14%, n = 41). After primary surgery, 24% (n = 44) received somatostatin analogues. The annual per-patient cost was €12 000; this was €8700 and €16 000 if diagnosed before or after July 2005, respectively. The cost-of-illness for acromegaly and its comorbidities was 77% from direct costs and 23% from production loss. The prevalence of comorbidity is high in patients with acromegaly. The most common first-line treatment in acromegalic patients was surgery followed by somatostatin analogues. The annual per-patient cost of acromegaly and its comorbidities was €12 000.

Longitudinal treatment patterns with ADP receptor inhibitors after myocardial infarction: Insights from the Canadian Observational AntiPlatelet sTudy

BackgroundAfter myocardial infarction (MI) treated with percutaneous coronary intervention (PCI), guidelines recommend dual antiplatelet therapy (DAPT) with aspirin and an ADP receptor inhibitor (ADPri) for at least 1year. However, whether real-world Canadian practice patterns reflect this recommendation is unknown. MethodsWe studied 2175 MI patients treated with PCI and discharged from 26 Canadian hospitals between 12/2011 and 05/2013 in the Canadian Observational Antiplatelet sTudy (COAPT). Hierarchical Cox proportional hazard regression modeling was used to determine baseline demographic and clinical factors associated with duration of ADPri therapy post-discharge. ResultsAt index-hospitalization discharge, 1597 (73%) patients were treated with clopidogrel, 220 (10%) with prasugrel, and 358 (17%) with ticagrelor. ADPri was discontinued prior to 1year in 474 (21.8%) patients; discontinuation rates were lowest for patients discharged on prasugrel (17.7%), compared with clopidogrel (22.5%) or ticagrelor (21.0%), (log rank test, p=0.03). In addition to regional variability, factors associated with shorter ADPri duration included older age, low body weight, Killip III/IV heart failure, atrial fibrillation, ticagrelor on discharge, and bare metal stent use, while longer ADPri duration was associated with history of prior MI. ConclusionsOne in five PCI-treated MI patients did not complete Canadian guideline-recommended 1-year course of ADPri treatment. Premature ADPri discontinuation was most strongly associated with factors that increase the risk of bleeding. Further study is required to assess the clinical implications of premature ADPri discontinuation on patient outcomes.

Longitudinal treatment patterns among US patients with atherosclerotic cardiovascular disease or familial hypercholesterolemia initiating lipid-lowering pharmacotherapy

The most recent American College of Cardiology-American Heart Association guidelines recommend high-dose statin therapy for most patients with confirmed atherosclerotic cardiovascular disease (ASCVD) and patients with high cardiovascular risk. There is limited information regarding long-term treatment patterns among these patients. To examine longitudinal treatment modifications in patients with ASCVD or familial hypercholesterolemia (FH). This retrospective analysis of administrative claims data identified patients initiating statin or ezetimibe therapy between January 1, 2007, and December 31, 2012, who had evidence of ASCVD or FH. Patients were followed for up to 3years and up to 4 treatment episodes. After initial treatment, subsequent treatment episodes began on the date of a treatment modification, which included discontinuation, statin dose change, switching, and augmentation. A total of 92,621 patients (mean age 64.7years, 57.3% male) were identified; 91,740 had ASCVD, 937 had FH (56 had both). Most ASCVD (89.6%) and FH (85.8%) patients initiated on statin monotherapy. The most common treatment modification in the first treatment episode was discontinuation (ASCVD: 42.0%; FH: 58.4%); among patients who discontinued, most reinitiated therapy (70.5% of ASCVD, 76.8% of FH). Most ASCVD (68.2%) and FH (71.1%) patients initiated on moderate-dose statins; statin dose increase occurred in 10.3% of ASCVD and 18.5% of FH patients in the first episode. Among patients with high cardiovascular risk, most initiated on moderate-dose statins with infrequent uptitration. In light of the recent American College of Cardiology-American Heart Association guidelines, statin initiation practices will need to change to ensure appropriate therapy for high-risk patients.

Longitudinal Treatment Patterns and Associated Outcomes in Patients With Newly Diagnosed Systemic Lupus Erythematosus

PurposeThe treatment of systemic lupus erythematosus (SLE) is complex, with a wide range of drugs commonly prescribed. The aims of this study were to identify longitudinal treatment patterns in patients with incident SLE and to estimate the associations of treatment patterns with clinical and economic outcomes. MethodsThis retrospective, observational cohort study used a US managed care claims database to identify patients with newly diagnosed SLE and 4-year treatment follow-up. Patients were aged ≥18 years, with continuous medical and pharmacy benefits for 12 months before and 48 months after the index date (first medical claim with a diagnosis of SLE). Longitudinal treatment patterns were grouped using a k-means cluster analysis. Therapies were included in the cluster analysis if the mean number of prescriptions in each year was ≥0.05. Clinical and economic outcomes were compared across clusters using multivariate regression analyses. FindingsData from 1611 patients with incident SLE were analyzed (91.4% women; mean [SD] age, 44.5 [9.5] years; 56.2% managed primarily by a specialist). Hydroxychloroquine and corticosteroids were the most commonly prescribed therapies; methotrexate, azathioprine, and mycophenolate mofetil also met the criteria for inclusion in the cluster analysis. Ten treatment clusters were identified; the most common was minimally treated patients (42.8%). Hydroxychloroquine monotherapy, corticosteroid monotherapy, and corticosteroid/hydroxychloroquine combination therapy were received by 34.0%, 11.2%, and 7.8% of patients, respectively. Methotrexate or azathioprine with a corticosteroid/hydroxychloroquine were received by 4.2% of patients. Changes in therapy, except discontinuations, were rare. Compared with the minimally treated cluster, those that received corticosteroid monotherapy (mean dose, >12.0 mg/d) had poorer clinical and economic outcomes; the hydroxychloroquine-monotherapy cluster had similar or better outcomes; and patients who received a corticosteroid/hydroxychloroquine with or without methotrexate or azathioprine demonstrated outcomes that were poorer but that appeared better than those with corticosteroid monotherapy. SLE-related visits with a nonspecialist were common (~45%) and remained unchanged over time despite better clinical and economic outcomes associated with specialist visits. ImplicationsThis study utilized cluster analysis, an unsupervised machine-learning method, to systematically discern treatment patterns over 4 years and to estimate outcomes associated with the identified treatment patterns. The results suggest that minimal treatment is the most common approach in patients with newly diagnosed SLE. Clinical and economic outcomes are poorest with corticosteroid monotherapy but may improve with the addition of hydroxychloroquine and/or an immunosuppressive agent. A large proportion of SLE care is provided by nonspecialists despite the potential benefits of involving a specialist.

Cluster analysis of longitudinal treatment patterns in patients newly diagnosed with systemic lupus erythematosus in the United States

Cluster analysis of longitudinal treatment patterns in patients newly diagnosed with systemic lupus erythematosus in the United States

Treating late‐life depression with interpersonal psychotherapy in the primary care sector

Interpersonal psychotherapy (IPT) is an empirically-validated intervention for treating late-life depression. To determine the manner in which IPT is utilized by primary care physicians in relation to antidepressant medications. The authors reviewed treatment logs prepared by care managers during the first 12 months of a patient's participation in the PROSPECT clinical trial to determine initial and longitudinal treatment patterns utilized by physicians, and clinical outcomes associated with initial treatment assignment. Primary care physicians in practices randomized to PROSPECT's intervention arm initially prescribed an antidepressant medication for 58% of eligible patients and referred only 11% of them to IPT. Over time, however, 27% of patients participated in IPT as monotherapy or augmentation therapy. Initial treatment assignment was not associated with depressive status at 4 and 12 months nor with suicidal ideation at 4, 8, and 12 months. IPT is an effective treatment for late-life depression whose greater use by primary care physicians should be encouraged.