Patient population with multiple myeloma and transitions across different lines of therapy in the USA: an epidemiologic model.

PURPOSE: Multiple myeloma (MM) is a progressive, malignant neoplasia with a worldwide, age-standardized annual incidence of 1.5 per 100 000 individuals and 5-year prevalence around 230 000 patients. Main favorable prognostic factors are younger age, low/standard cytogenetic risk, and undergoing stem cell transplantation. Our aim was to estimate the size of the patient population with MM eligible to receive a new MM therapy at different lines of therapy in the USA. METHODS: We constructed a compartmental, differential equation model representing the flow of MM patients from diagnosis to death, via two possible treatment pathways and distinguished in four groups based on prognostic factors. Parameters were obtained from published references, available statistics, and assumptions. The model was used to estimate number of diagnosed MM patients and number of patient transitions from one line of therapy to the next over 1 year. Model output included 95% credible intervals from probabilistic sensitivity analyses. RESULTS: The base-case estimates were 80 219 patients living with MM, including 70 375 on treatment, 780 symptomatic untreated patients, and 9064 asymptomatic untreated patients. Over a 1-year period, the number of MM patients on treatment line 1 was estimated at 23 629 (credible intervals 22 236-25 029), and the number of transitions from treatment line 1 to treatment line 2 was estimated at 14 423. CONCLUSIONS: The size of the patient population with MM on different lines of therapy and in patient subgroups of interest estimated from this epidemiologic model can be used to assess the number of patients who could benefit from new MM therapies and their corresponding budgetary impact. Copyright © 2016 John Wiley & Sons, Ltd

Linaclotide utilization and potential for off-label use and misuse in three European countries

INTRODUCTION Linaclotide is approved for adults with moderate-to-severe irritable bowel syndrome (IBS) with constipation (IBS-C). Linaclotide is not indicated for weight loss or for patients with inflammatory bowel disease (IBD); it is contraindicated in patients with mechanical bowel obstruction (MBO). Some patients with obesity or eating disorders (ED) may use linaclotide off-label for weight loss or as a laxative. OBJECTIVES To describe the use of linaclotide in clinical practice, including patients with potential for off-label use or misuse. METHODS Post-authorization safety study conducted in three databases from the linaclotide launch date to 2017: the Clinical Practice Research Datalink in the United Kingdom (UK), the Information System for Research in Primary Care database in Spain and the linked Patient, Prescription and Causes of Death Registries in Sweden. Cohorts of patients were identified as having IBS using diagnostic and treatment codes; IBS subtypes were identified using symptoms and treatment codes; patients with obesity, ED, MBO, and IBD were identified using diagnostic codes or body mass index. RESULTS There were 1319, 1981, and 5081 linaclotide users from the United Kingdom, Spain, and Sweden with a median age of 45, 57, and 51 years, respectively; most were females. In the United Kingdom, Spain, and Sweden, respectively: 59.0%, 60.3%, and 31.3% of linaclotide users had an IBS diagnosis recorded, and among those, 68.8%, 61.3%, and 92.7% were classified as IBS-C. The proportions of linaclotide users considered at risk for potential off-label use for weight loss or as a laxative were 17.1%, 29.7%, and 1.7%, and the proportions of users considered at risk of misuse due to a history of MBO or IBD were 3.5%, 4.6%, and 5.7% in the United Kingdom, Spain, and Sweden, respectively. CONCLUSIONS Potential linaclotide off-label use and misuse appears limited, as evidenced by the small sizes of the patient subgroups at risk for off-label use and misuse

Global patterns of comprehensive cardiovascular risk factor control in patients with type 2 diabetes mellitus: Insights from the DISCOVER study.

AIM: To investigate global patterns of cardiovascular risk factor control in patients with type 2 diabetes mellitus (T2D). METHODS: DISCOVER is an international, observational cohort study of patients with T2D beginning second-line glucose-lowering therapy. Risk factor management was examined among eligible patients (ie, those with the risk factor) at study baseline. Inter-country variability was estimated using median odds ratios (MORs). RESULTS: Among 14 343 patients with T2D from 34 countries, the mean age was 57.4 ± 12.0 years and the median (interquartile range) duration of T2D was 4.2 (2.0-8.0) years; 11.8% had documented atherosclerotic cardiovascular disease (ASCVD). Among eligible patients, blood pressure was controlled in 67.5% (9284/13756), statins were prescribed in 43.7% (5775/13208), angiotensin-converting enzyme inhibitors/angiotensin II receptor blockers were prescribed in 55.6% (5292/9512), aspirin was prescribed in 53.3% of those with established ASCVD (876/1645), and 84.4% (12 102/14343) were non-smoking. Only 21.5% of patients (3088/14343) had optimal risk factor management (defined as control of all eligible measures), with wide inter-country variability (10%-44%), even after adjusting for patient and site differences (MOR 1.47, 95% confidence interval 1.24-1.66). CONCLUSION: Globally, comprehensive control of ASCVD risk factors is not being achieved in most patients, with wide variability among countries unaccounted for by patient and site differences. Better country-specific strategies are needed to implement comprehensive cardiovascular risk factor control consistently in patients with T2D to improve long-term outcomes

Metformin discontinuation in patients beginning second-line glucose-lowering therapy: results from the global observational DISCOVER study programme.

OBJECTIVES: To evaluate the extent to which patients with type 2 diabetes discontinue metformin therapy when initiating second-line treatment and factors associated with metformin discontinuation, using baseline data from the DISCOVER study programme. DESIGN: DISCOVER is a 3-year, prospective, observational study programme including data from 38 countries across 6 continents from 2014 to 2019. SETTING: Primary and secondary healthcare centres, hospitals and specialist diabetes centres in both urban and rural locations. PARTICIPANTS: A total of 15 992 patients with type 2 diabetes initiating second-line glucose-lowering therapy. PRIMARY AND SECONDARY OUTCOME MEASURES: The proportion of patients who discontinued metformin as a second-line therapy and the factors associated with this treatment change. RESULTS: Of the 14 668 patients (from 37 countries) with valid treatment data, 11 837 (80.7%) received metformin as first-line glucose-lowering therapy; 8488 (71.7%) received metformin monotherapy and 3349 (28.3%) received metformin as part of a combination therapy. Overall, treatment with metformin was discontinued in 15.1% (1782) of patients who received first-line metformin (14.1% (1194) and 17.6% (588) in those who received metformin as monotherapy and as part of a combination, respectively); this proportion varied across regions from 6.9% (54) in Africa to 20.6% (628) in South-East Asia. On metformin discontinuation, 73.6% (1311) of patients received a non-insulin monotherapy at second line. Factors associated with an increased odds of metformin discontinuation were older age (≥75 years) and having a history of chronic kidney disease. The probability of metformin monotherapy discontinuation was lower in patients from Africa than in those from Europe. CONCLUSIONS: A substantial number of patients discontinued taking metformin when beginning second-line therapy. Most of these patients subsequently received a non-insulin monotherapy at second line, in contradiction to international guidelines and potentially leaving them at an increased risk of hyperglycaemia and associated adverse outcomes. TRIAL REGISTRATION NUMBERS: NCT02322762 and NCT02226822

Valoración de la discapacidad física: el indice de Barthel

En Salud Pública existe una tendencia creciente a valorar el impacto que los problemas de salud tienen, tanto sobre la calidad de vida de las personas como sobre el uso de servicios sanitarios. En este sentido, la evaluación de la discapacidad está adquiriendo una relevancia cada vez mayor. El índice de Barthel es un instrumento ampliamente utilizado para este propósito y mide la capacidad de la persona para la realización de diez actividades básicas de la vida diaria, obteniéndose una estimación cuantitativa del grado de dependencia del sujeto. El Indice de Barthel se ha venido utilizando desde que fue propuesto en 1955 y ha dado lugar a múltiples versiones, además de servir como estándar para la comparación con otras escalas. Es una medida fácil de aplicar, con alto grado de fiabilidad y validez, capaz de detectar cambios, fácil de interpretar y cuya aplicación no causa molestias. Por otra parte, su adaptación a diferentes ámbitos culturales resulta casi inmediata. A pesar de tener algunas limitaciones, el Indice de Barthel puede recomendarse como un instrumento de elección para la medida de la discapacidad física, tanto en la práctica clínica como en la investigación epidemiológica y en Salud Pública

Valoración de la discapacidad física: el indice de Barthel

In Public Health there exists a growing tendency to evaluate the impact of health problems both on the quality of life of the persons involved as well as the use of health services. In this sense, the evaluation of incapacity is acquiring ever greater relevance. The Barthel Index is an instrument widely used to this end and measures the capacity of the person for the execution of ten basic activities in daily life, obtaining a quantitative estimation of the subject´s level of dependency. The Barthel Index has been used, since its introduction in 1955, resulting in numerous versions, as well as serving as a standard of comparison with other scales. It is an easily applicable method, with a high level of realibility and validity, capable of detecting changes, easy to interpret and the application of which is not problematic. On the other hand, its adaptation to different cultural environments is almost immediate. Although it has a few limitations, the Barthel Index may be recommended as a selection method for measuring physical incapacity, both in clinical practice as well as in epidemiological investigation and Public Health.En Salud Pública existe una tendencia creciente a valorar el impacto que los problemas de salud tienen, tanto sobre la calidad de vida de las personas como sobre el uso de servicios sanitarios. En este sentido, la evaluación de la discapacidad está adquiriendo una relevancia cada vez mayor. El índice de Barthel es un instrumento ampliamente utilizado para este propósito y mide la capacidad de la persona para la realización de diez actividades básicas de la vida diaria, obteniéndose una estimación cuantitativa del grado de dependencia del sujeto. El Indice de Barthel se ha venido utilizando desde que fue propuesto en 1955 y ha dado lugar a múltiples versiones, además de servir como estándar para la comparación con otras escalas. Es una medida fácil de aplicar, con alto grado de fiabilidad y validez, capaz de detectar cambios, fácil de interpretar y cuya aplicación no causa molestias. Por otra parte, su adaptación a diferentes ámbitos culturales resulta casi inmediata. A pesar de tener algunas limitaciones, el Indice de Barthel puede recomendarse como un instrumento de elección para la medida de la discapacidad física, tanto en la práctica clínica como en la investigación epidemiológica y en Salud Pública

Incidence and prevalence of idiopathic pulmonary fibrosis: review of the literature

Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive, fibrosing interstitial pneumonia of unknown aetiology. It is a rare disease, and its incidence and prevalence are not clear. Therefore, we sought to review the published evidence on the global epidemiology of IPF. A comprehensive review of English language literature was performed by searching Medline and EMBASE for studies on IPF epidemiology published between January 1990 and August 2011. Studies providing quantitative data on IPF incidence and/or prevalence were identified and key data collected. 15 studies reporting on the incidence and/or prevalence of IPF were identified and summarised. IPF prevalence estimates in the USA varied between 14 and 27.9 cases per 100,000 population using narrow case definitions, and 42.7 and 63 per 100,000 population using broad case definitions. In Europe, IPF prevalence ranged from 1.25 to 23.4 cases per 100,000 population. The annual incidence of IPF in the USA was estimated at 6.8–8.8 per 100,000 population using narrow case definitions and 16.3–17.4 per 100,000 population using broad case definitions. In Europe, the annual incidence ranged between 0.22 and 7.4 per 100,000 population. IPF prevalence and incidence increase with age, are higher among males and appear to be on the increase in recent years. IPF is an orphan disease that affects a potentially increasing number of people in Europe and the USA. The observed variability in IPF incidence and prevalence may be explained by the differences in diagnostic criteria used, case definition, study population and study design

An analysis of characteristics of post-authorisation studies registered on the ENCePP EU PAS Register [version 2; referees: 2 approved]

Background: The objective of this study was to investigate the study design characteristics of Post-Authorisation Studies (PAS) requested by the European Medicines Agency which were recorded on the European Union (EU) PAS Register held by the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP). Methods: We undertook a cross-sectional descriptive analysis of all studies registered on the EU PAS Register as of 18th October 2016. Results: We identified a total of 314 studies on the EU PAS Register, including 81 (26%) finalised, 160 (51%) ongoing and 73 (23%) planned. Of those studies identified, 205 (65%) included risk assessment in their scope, 133 (42%) included drug utilisation and 94 (30%) included effectiveness evaluation. Just over half of the studies (175; 56%) used primary data capture, 135 (43%) used secondary data and 4 (1%) used a hybrid design combining both approaches. Risk assessment and effectiveness studies were more likely to use primary data capture (60% and 85% respectively as compared to 39% and 14% respectively for secondary). The converse was true for drug utilisation studies where 59% were secondary vs. 39% for primary. For type 2 diabetes mellitus, database studies were more commonly used (80% vs 3% chart review, 3% hybrid and 13% primary data capture study designs) whereas for studies in oncology, primary data capture were more likely to be used (85% vs 4% chart review, and 11% database study designs). Conclusions: Results of this analysis show that PAS design varies according to study objectives and therapeutic area