Quality of life, physical functioning, and psychosocial function among patients with achondroplasia: a targeted literature review.

PURPOSE: Achondroplasia (ACH) is the most common form of skeletal dysplasia, resulting in disproportionate short stature and medical complications. We review the literature on physical functioning, psychosocial function, and quality of life (QoL) in ACH individuals compared to average stature individuals or other short stature conditions. Studies that assess the association between these outcomes and height, limb length/lengthening surgery in ACH patients are also summarized. MATERIALS AND METHODS: PubMed/MEDLINE and Embase were searched through April 2021. Study inclusion criteria were: (1) quantitative design; (2) study population consisting solely/mainly of ACH patients; (3) reports of physical functioning, psychosocial functioning, and/or QoL. Included studies were summarized separately for pediatric and adult populations. RESULTS: Of 1664 records identified, 23 primary studies (sample size 8-437 participants) were included. Multiple tools were used across studies, including the generic PedsQL and SF-36 and height-specific QoLISSY. CONCLUSIONS: The literature demonstrates that ACH patients experience limitations in physical functioning and poorer QoL outcomes compared to average stature people across the life span. This appeared to be at least in part due to disproportionate short stature. Future research to better characterize QoL in ACH patients will assist clinicians to better evaluate the effectiveness of management programs including novel interventions.IMPLICATIONS FOR REHABILITATIONPatients with achondroplasia experience limitations in physical functioning and poorer quality of life throughout their life course when compared to average statured individuals.Psychosocial issues are also heightened in adults with achondroplasia compared to average statured peers but are observed less frequently in children and adolescents with achondroplasia.The overall impact that limb lengthening has on physical functioning and QoL remains unclear, although there is some evidence that greater height or upper limb length may lead to an improvement in these parameters.Rehabilitation professionals should regularly assess physical functioning, psychosocial wellbeing, and quality of life in individuals with achondroplasia using condition-specific tools.

High eosinophil counts predict decline in FEV1: results from the CanCOLD study.

INTRODUCTION: The aim of this study was to examine the association between blood eosinophil levels and the decline in lung function in individuals aged >40 years from the general population. METHODS: The study evaluated the eosinophil counts from thawed blood in 1120 participants (mean age 65 years) from the prospective population-based Canadian Cohort of Obstructive Lung Disease (CanCOLD) study. Participants answered interviewer-administered respiratory questionnaires and performed pre-/post-bronchodilator spirometric tests at 18-month intervals; computed tomography (CT) imaging was performed at baseline. Statistical analyses to describe the relationship between eosinophil levels and decline in forced expiratory volume in 1 s (FEV1) were performed using random mixed-effects regression models with adjustments for demographics, smoking, baseline FEV1, ever-asthma and history of exacerbations in the previous 12 months. CT measurements were compared between eosinophil subgroups using ANOVA. RESULTS: Participants who had a peripheral eosinophil count of ≥300 cells·µL-1 (n=273) had a greater decline in FEV1 compared with those with eosinophil counts of <150 cells·µL-1 (n=430; p=0.003) (reference group) and 150-<300 cells·µL-1 (n=417; p=0.003). The absolute change in FEV1 was -32.99 mL·year-1 for participants with eosinophil counts <150 cells·µL-1; -38.78 mL·year-1 for those with 150-<300 cells·µL-1 and -67.30 mL·year-1 for participants with ≥300 cells·µL-1. In COPD, higher eosinophil count was associated with quantitative CT measurements reflecting both small and large airway abnormalities. CONCLUSION: A blood eosinophil count of ≥300 cells·µL-1 is an independent risk factor for accelerated lung function decline in older adults and is related to undetected structural airway abnormalities.

Stability of Blood Eosinophil Count in Patients with COPD in the UK Clinical Practice Research Datalink.

Blood eosinophil counts may be predictive of corticosteroid response in chronic obstructive pulmonary disease (COPD) patients. However, little is known about measurement stability, which is important for understanding the utility of blood eosinophil counts as a potential biomarker. We evaluated the stability of blood eosinophil counts over 1 year in a population-based cohort of patients with COPD in primary care. Patients were aged ≥ 40 years with forced expiratory volume in 1 second/forced vital capacity < 0.7 and ≥ 1 blood eosinophil measurement taken during a period of stable disease within 6 months of a COPD diagnosis code recorded between January 1, 2010 and December 31, 2012. Generalized linear mixed models were fitted to log-transformed data to estimate the between-(s2between) and within-patient (s2within) variance in eosinophil count; an intra-class correlation coefficient Ri was calculated (s2between/[s2between + s2within]). A sensitivity analysis was performed from which patients who were prescribed systemic corticosteroids or antibiotics at any time during follow-up were excluded. All models were adjusted for age, gender, smoking status, and asthma history. Overall, 27,557 patients were included in the full cohort (51.5% male, mean age [standard deviation] 71.1 [10.6] years) and 54% of patients had ≥ 2 eosinophil measurements (median 2 [interquartile range 1]) during follow-up. For the full cohort, Ri = 0.64, and in the sensitivity analysis subgroup, Ri = 0.70, mainly due to a decrease in s2within. For patients with COPD in primary care, eosinophil measurements demonstrated reasonable repeatability over 1 year, which increased after exclusion of patients who were prescribed systemic corticosteroids or antibiotics.

Continuing to confront COPD International Surveys: comparison of patient and physician perceptions about COPD risk and management.

PURPOSE: Using data from the Continuing to Confront COPD International Physician and Patient Surveys, this paper describes physicians' attitudes and beliefs regarding chronic obstructive pulmonary disease (COPD) prognosis, and compares physician and patient perceptions with respect to COPD. METHODS: In 12 countries worldwide, 4,343 patients with COPD were identified through systematic screening of population samples, and 1,307 physicians who regularly saw patients with COPD were sampled from in-country professional databases. Both patients and physicians completed surveys about their COPD knowledge, beliefs, and perceptions; physicians answered further questions about diagnostic methods and treatment choices for COPD. RESULTS: Most physicians (79%) responded that the long-term health outlook for patients with COPD has improved over the past decade, largely attributed to the introduction of better medications. However, patient access to medication remains an issue in many countries, and some physicians (39%) and patients (46%) agreed/strongly agreed with the statement "there are no truly effective treatments for COPD". There was strong concordance between physicians and patients regarding COPD management practices, including the use of spirometry (86% of physicians and 76% of patients reporting they used/had undergone a spirometry test) and smoking cessation counseling (76% of physicians reported they counseled their smoking patients at every clinic visit, and 71% of smoking patients stated that they had received counseling in the past year). However, the groups differed in their perception about the role of smoking in COPD, with 78% of physicians versus 38% of patients strongly agreeing with the statement "smoking is the cause of most cases of COPD". CONCLUSION: The Continuing to Confront COPD International Surveys demonstrate that while physicians and patients largely agreed about COPD management practices and the need for more effective treatments for COPD, a gap exists about the causal role of smoking in COPD.

Disruptions in Liver Function among Cancer Patients and Patients Treated with Tyrosine Kinase Inhibiting Drugs: Comparisons of Two Population-Based Databases.

Liver toxicity is a recognized adverse event associated with small molecule tyrosine kinase inhibitors (TKIs). Electronic Medical Record (EMR) databases offer the most precise data to investigate the rate of liver function test (LFT) elevations; however, they can be limited in sample size and costly to access and analyze. Health insurance claims databases often contain larger samples sizes but may lack key health information. We evaluated the feasibility of utilizing a large claims database to calculate incidence rates (IRs) of LFT elevations among a general cohort of cancer patients and a cohort of patients treated with TKIs by comparing the results to a "gold standard" oncology-specific EMR database. IRs for the TKI cohorts were very similar between the two databases; however, IRs were higher in the EMR database for the cancer cohorts. Possible explanations for these differences include lack of specificity when defining a cancer case, poor capture of laboratory data, or inaccurate assessment of person-time in the insurance claims database. This study suggests that insurance claims data may provide reliable results when investigating liver toxicities associated with oncology drug exposure; however, there are limitations when assessing laboratory outcomes for cohorts defined solely by disease status.

Incidence of new-onset hypertension in cancer patients: a retrospective cohort study.

This retrospective cohort study was conducted to estimate incidence rates of new-onset hypertension in adult cancer patients identified from the Varian Medical Oncology outpatient database. Incidence rates of increasing levels of hypertension severity were calculated overall and for periods of chemotherapy exposure and nonexposure. Cox models sought predictors of new-onset hypertension severity among baseline and chemotherapy exposure variables. New-onset hypertension was observed in about one-third of 25,090 patients with various cancer types. The incidence rates (IR) of severe and crisis-level hypertension, respectively, were the highest in patients with gastric (18.5 cases per 100 person-years (PY), 5.6 per 100 PY) and ovarian cancer (20.2 per 100 PY, 4.8 per 100 PY). The highest IR of moderate hypertension was observed in patients with renal cancer (46.7 per 100 PY). Across all cancers, chemotherapy exposure was associated with a 2-3.5-fold increase in risk of any degree of hypertension compared to periods of no chemotherapy; higher hypertension levels showed greater variability in relative risks by type and line of therapy but indicated an overall increase associated with chemotherapy exposure. These results help to elucidate the factors influencing HTN among cancer patients and the incidence of HTN relative to chemotherapy exposure.

Prevalence and incidence of acute and chronic comorbidity in patients with squamous cell carcinoma of the head and neck.

BACKGROUND: Limited data exist on the burden of comorbidity among patients with squamous cell head and neck cancer (SCCHN) before and during cancer treatment. METHODS: The precancer prevalence and incidence rates of 8 comorbid conditions were estimated among a population-based cohort of 1499 patients with SCCHN in the Netherlands. Patients with cancer, treatments, and comorbidities were identified in the PHARMO Record Linkage System (RLS) using hospital admissions and/or dispensing codes. Prevalence proportions and incidence rates were also compared against a matched cancer-free population. RESULTS: Cardiovascular (41%) and respiratory diseases (12%) were the most prevalent comorbidities. Incidence rates of most comorbidities were highest the first 6 months after cancer diagnosis and decreased over time. Patients receiving chemotherapy-based treatment had significantly higher incidence rates of anemia and other malignant diseases. CONCLUSIONS: High rates of acute and chronic comorbidity were observed; knowledge of comorbidity burden aids in establishing a benefit-risk profile for investigational SCCHN therapies.

Head and neck cancer in South East England between 1995-1999 and 2000-2004: An estimation of incidence and distribution by site, stage and histological type.

Population-based data on head and neck cancer (HNC) stage and histological type are poorly described for England; these data are essential for clinical management and research. The aim of this study was to describe the distribution and incidence of all HNC and selected anatomical sites by sex, age, stage and histological type using a population-based cancer registry in South East England, and determine if the incidence changed between 1995-1999 and 2000-2004. We identified all HNC cancer cases registered by the Thames Cancer Registry for 1995-1999 and 2000-2004. Frequency distributions and age-standardised incidence rates were calculated by sex, age, stage and histological type and trends in incidence between the two time periods were described using incidence rate ratios and 95% confidence intervals. A total of 8700 HNC cases were reported in 2000-2004, representing an age-standardised incidence rate of 8.59 per 100000, which did not change significantly from 1995-1999. The three commonest HNC sites were intra-oral cavity, larynx and tonsil. Males were two to six times as likely as females to be diagnosed with HNC and there was a trend toward younger age at diagnosis over time. Significant increases in the incidence rate of intra-oral cavity cancer for both sexes and tonsillar cancer among males were observed. Conversely, laryngeal cancer incidence decreased over time. Staging data was only available for about 40% of HNC cases. Seventy six percent of HNC cases were squamous cell carcinomas. Trends in incidence varied between HNC sites, highlighting the importance of presenting data for individual HNC sites. The high proportion of unstaged cancers may result from incomplete recording in medical records; thus, the reporting of staging data should be made a priority.

Long-term trends in cancer mortality in the United States, 1930-1998.

BACKGROUND: Progress against cancer can be examined by analyzing long-term trends in cancer incidence and mortality. The recent directive from the U.S. Department of Health and Human Services to adopt the 2000 U.S. standard population for the age adjustment of death rates prompted the American Cancer Society to update historical cancer mortality statistics using the new standard. METHODS: Mortality data were abstracted by race, gender, year, and age at death for 1930 through 1959 from annual volumes of Vital Statistics of the United States. For 1960 through 1998, these data were obtained from data tapes provided by the National Center for Health Statistics. Two U.S. standard million populations (1970 and 2000) were used to calculate age-adjusted rates. Average annual percent change was estimated for each decade by site, gender, and age, and the statistical significance of the change was assessed at p < 0.05. RESULTS: After long-term increases or mostly level trends that date from the 1930s for some sites, death rates for cancers of the lung (in males), prostate, female breast, colon-rectum, pancreas, leukemia, and ovary were decreasing in the 1990s. Liver cancer death rates were increasing in the 1990s. Throughout the study period, death rates for female lung cancer increased, while death rates for stomach and uterine cancers declined. CONCLUSIONS: The trends of decreasing cancer death rates for the leading cancer sites in the 1990s are encouraging. However, surveillance researchers must continue to monitor these declines to assess whether the progress seen in this decade persists. Efforts also must be made to study the sites with increasing trends and identify potential underlying causes.