Quality of Life in Primary Open-Angle Glaucoma and Cataract: An Analysis of VFQ-25 and OSDI From the iStent inject® Pivotal Trial.

OBJECTIVE: To assess quality of life (QOL) as measured by patient-reported outcomes (PRO) within the iStent inject® pivotal trial. DESIGN: Randomized controlled trial analysis of secondary outcomes. METHODS: The Vision Function Questionnaire (VFQ-25) and Ocular Surface Disease Index (OSDI) questionnaire were administered at baseline and at months 1, 6, 12, and 24. PRO responders were defined as patients reaching improvement based on minimally important differences. RESULTS: A total of 505 patients were randomized (386 iStent inject® [Glaukos], 119 surgery alone). The iStent inject® group exhibited a greater percentage of PRO responders across all follow-up visits over 24 months, averaging 58.0% vs 45.8%; P < .05 for VFQ-25 composite scores and 56.7% vs 48.9%; P < .05 for OSDI composite scores. Odds of being a responder in the iStent inject® group was 60% (P < .05) higher for the VFQ-25 and 32% (P > .05) higher for the ODSI. Driving (49.0% vs 28.8%; P < .05), ocular pain (59.3% vs 47.2%; P < .05), and general vision (71.8% vs 60.0%; P < .05) were the VFQ-25 subscales responsible for differences between treatment groups. At month 24, 76.5% of VFQ-25 responders and 62.5% of nonresponders were medication free regardless of treatment group (P < .05). CONCLUSIONS: Exploratory analysis suggests that by reducing medication dependence, implantation with the micro-scale iStent inject® device with cataract surgery may improve QOL vs cataract surgery alone over 24 months, with improvements influenced by ocular symptoms and vision-related activities.

Real-world evidence: Patient views on asthma in respiratory specialist clinics in America.

BACKGROUND: Approximately 30% to 50% of patients with moderate/severe asthma have inadequately controlled disease despite adherence to inhaled corticosteroid (ICS)/long-acting ß2-agonist (LABA) therapy. Data on prevalence and burden of uncontrolled asthma in specialty settings are lacking. OBJECTIVE: To evaluate the prevalence and burden of uncontrolled asthma in respiratory specialist clinics in the United States. METHODS: Adults with physician-diagnosed asthma attending pulmonary and allergy clinics with self-reported ICS use in the previous 4 weeks completed an electronic questionnaire including the Asthma Control Test and St George's Respiratory Questionnaire. Additional information was collected using an electronic case report form. RESULTS: Of 774 patients attending 12 pulmonary and 12 allergy clinics, 53% were not well controlled (mean [SD] Asthma Control Test, 14.3 [3.6] vs 22.4 [1.6] in well-controlled patients). Among ICS/LABA users, 56% were not well controlled, which increased with increasing ICS dose (low-dose 45.7%; high-dose 59.7%). The not well-controlled group reported more respiratory illnesses, more comorbidities, and poorer health-related quality of life (mean [SD] St George's Respiratory Questionnaire, 46.1 [18.9] vs 19.8 [12.9] in the well-controlled group). These patients also had more asthma exacerbations (≥1 exacerbation, 68.9% vs 43.1%) and increased health care resource utilization (≥1 asthma-related hospitalization, 10.7% vs 2.7%); 27.3% were also receiving systemic corticosteroids. Approximately 40% of the population were eligible for step-up to ICS/LABA/long-acting muscarinic antagonist triple therapy, and 20% were eligible for biologic therapy. CONCLUSION: Substantial unmet needs exist among patients with inadequately controlled asthma managed in United States specialist settings, which may be addressed by improved patient and physician education, better guideline implementation, and improved adherence.

Teriparatide Did Not Increase Adult Osteosarcoma Incidence in a 15-Year US Postmarketing Surveillance Study.

The Osteosarcoma Surveillance Study was initiated in the United States in 2003 to monitor for a potential association between the osteoporosis treatment teriparatide and osteosarcoma. Osteosarcoma occurs at a background incidence rate of approximately 2.5 cases per million per year in US adults aged 40 years or older. For this study, incident cases of osteosarcoma diagnosed between January 1, 2003, and December 31, 2016, were identified through participating cancer registries in the United States. Information on prior exposure to medications and possible risk factors was obtained by self-report (or proxy report) in telephone interviews. Exposure information was verified through medical record abstraction for a sample of patients. A standardized incidence ratio was estimated to compare the observed and expected numbers of osteosarcoma patients with a prior history of teriparatide treatment. Interviews were completed for 24% (1173) of patients diagnosed with osteosarcoma between 2003 and 2016; three reports of teriparatide use before diagnosis were identified. Based on the background incidence rate, the expected number of osteosarcoma cases among patients treated with teriparatide was 4.17. Given the three observed cases, the standardized incidence ratio was 0.72 (90% confidence interval [CI], 0.20 to 1.86). Demographic characteristics were similar for interviewed and noninterviewed patients. Agreement was >90% between self-reported and chart-recorded exposure to osteoporosis medications. Mean age of interviewed patients was 61 years; 53% of patients were male, 84% were white, and 5% were Hispanic. The prevalence of suspected risk factors for development of osteosarcoma among the osteosarcoma cohort was 19% for history of radiation and 4% for history of Paget's disease of bone. These findings showed that the incidence of osteosarcoma associated with teriparatide use during the 15-year surveillance period was no different than would be expected based on the background incidence rate of osteosarcoma. © 2020 The Authors. Journal of Bone and Mineral Research published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research (ASBMR).

Treatment satisfaction in women receiving palbociclib combination therapies for advanced/metastatic breast cancer.

AIM: To understand treatment satisfaction in patients with advanced or metastatic breast cancer receiving palbociclib plus an aromatase inhibitor or palbociclib plus fulvestrant in a real-world setting. PATIENTS & METHODS: We performed an observational, cross-sectional, web-based survey of 604 patients with self-reported hormone receptor-positive (HR+)/HER2-negative (HER2-) ABC/mBC in six countries. RESULTS: Overall, more than 96% of patients reported the benefits of their palbociclib combination therapy met or exceeded their expectations. Patient expectations and satisfaction with therapy did not differ between patients on palbociclib plus letrozole and palbociclib plus fulvestrant, or between patients with visceral and nonvisceral metastases. CONCLUSION: The patients on palbociclib combination therapy reported high satisfaction scores across multiple countries.

Longitudinal Modeling Approaches to Assess the Association Between Changes in 2 Clinical Outcome Assessments.

BACKGROUND: Understanding how one clinical outcome assessment (COA) (eg, a patient-reported outcome [PRO]) relates to a second COA (eg, a clinician-reported outcome [ClinRO]) may provide insights into disease burden or treatment efficacy. We aimed to briefly review commonly used cross-sectional methods to evaluate the association between a PRO and a ClinRO and to demonstrate the advantages of longitudinal modeling approaches, particularly a joint mixed model for repeated measures (MMRM), to evaluate this association. METHODS: We generated an example longitudinal data set that included a PRO measured on an 11-point numeric rating scale and a binary ClinRO. The association between change in PRO score and ClinRO response at each time point was examined using 2 cross-sectional analyses: point biserial correlation and logistic regression. We conducted longitudinal analyses of the association between the 2 COAs across time points using MMRM and joint MMRM approaches. RESULTS: Point-biserial correlation and logistic regression analyses correctly captured the "built in" associations between the 2 COAs that strengthened over time, but each association was applicable only for a single time point. The MMRM approach provided correlations over time but only for a single outcome variable. The joint MMRM approach modeled the relationship between both outcome variables simultaneously, allowing for evaluation of the correlations both within and between the variables over time. CONCLUSION: Each analysis demonstrated the relationship between PRO score changes and ClinRO response. Longitudinal analysis methods, particularly the joint MMRM, allow for a more thorough examination of the correlations among the 2 outcomes than cross-sectional analysis methods.

Evaluation of Risk-Minimization Activities for Cyproterone Acetate 2 mg/Ethinylestradiol 35 µg: A Cross-Sectional Physician Survey.

BACKGROUND: Cyproterone acetate 2 mg/ethinylestradiol 35 µg, an estrogen/progestogen treatment with anti-androgenic properties, shares a thromboembolism risk with combined hormonal contraceptives. Educational materials (i.e., direct healthcare professional communication, patient information card, prescriber checklist) were distributed to physicians to increase risk awareness. OBJECTIVE: The objective of this study was to measure physician knowledge of thromboembolism risk of cyproterone acetate 2 mg/ethinylestradiol 35 µg and ascertain whether physicians received the educational materials. METHODS: A cross-sectional web-based physician survey of recent prescribers of cyproterone acetate 2 mg/ethinylestradiol 35 µg in Austria, the Czech Republic, France, the Netherlands, and Spain was conducted. Sampling targets for physician specialty were based on country-specific prescribing patterns. Frequency of correct responses was calculated for 14 knowledge questions. RESULTS: Among 759 physician respondents (37% of obstetricians/gynecologists, 42% of general practitioners, 20% of dermatologists), 51% received one or more of three educational materials. Knowledge was highest (≥80%) for symptoms of possible deep vein thrombosis, pulmonary embolism, and cerebrovascular accident; most important risk factors for thrombosis; use in smokers; indication for moderate-to-severe acne; and understanding that cyproterone acetate 2 mg/ethinylestradiol 35 µg should not be used for contraception alone. Knowledge varied for contraindications, myocardial infarction symptoms, other risk factors for thrombosis, instructions regarding anticipated prolonged immobilization, and selected concomitant medical conditions. Knowledge was lower regarding prescribing cyproterone acetate 2 mg/ethinylestradiol 35 µg for acne only after failure of topical therapy or systemic antibiotics. Generally, knowledge did not vary by physician specialty, receipt of educational materials, number of patients prescribed cyproterone acetate 2 mg/ethinylestradiol 35 µg in the previous 3 months, and years in practice. CONCLUSIONS: Knowledge was generally high for thromboembolism risk and varied for more complex or infrequent topics.

A dose-ranging, phase II study of the efficacy and safety of alosetron in men with diarrhea-predominant IBS.

BACKGROUND: A randomized, double blind, placebo-controlled dose-ranging study was conducted to assess the efficacy of alosetron in men with diarrhea-predominant irritable bowel syndrome (IBS). METHODS: Six hundred and sixty-two men were randomized to treatment with alosetron 0.5, 1.0, 2.0, 4.0 mg, or placebo twice daily for 12 wk, followed by a 4-wk posttreatment period. Adequate relief of IBS pain and discomfort during week 5-12 of the treatment phase was the primary endpoint; secondary endpoints included bowel urgency, stool frequency, and consistency, incomplete evacuation, bloating, and abdominal pain or discomfort. RESULTS: Subjects ranked urgency and abdominal pain as their most bothersome IBS symptoms. The average rate of adequate relief during week 5-12 was significantly higher in the alosetron 1.0 mg twice-daily group compared to placebo (53%vs 40%, p= 0.04), and all doses of alosetron significantly reduced stool consistency scores (p < 0.001) indicating firmer stools. No significant effects of alosetron were seen with regard to urgency, number of bowel movements, bloating, and incomplete evacuation. Constipation was the most common adverse event and occurred in a dose-related manner among subjects receiving alosetron, 9% (0.5 mg twice daily), 15% (1.0 mg twice daily), 11% (2.0 mg twice daily), and 21% (4.0 mg twice daily). No serious adverse events of constipation were reported. One subject in the 0.5 mg twice-daily group had an episode of rectal bleeding suggestive of a possible diagnosis of ischemic colitis. CONCLUSIONS: Alosetron 1 mg twice daily provided adequate relief of IBS pain and discomfort, and improved stool consistency in men with diarrhea-predominant IBS.