Real-world clinical, psychosocial and economic burden of atopic dermatitis: Results from a multicountry study.

BACKGROUND: Atopic dermatitis (AD), a relapsing, inflammatory skin disease, is associated with pruritus that can negatively affect patients' quality of life. Understanding the burden of AD is critical for informing and tailoring treatment and disease management to improve patient outcomes. This study characterized global treatment patterns and the clinical, psychosocial and economic burden of moderate-to-severe AD. METHODS: MEASURE-AD was a cross-sectional 28-country study in patients with physician-confirmed moderate-to-severe AD who were either receiving or eligible for systemic therapy for AD. Patients ≥12 years were enrolled between December 2019 and December 2020 while attending routine office or clinic visit. Primary outcomes included Worst Pruritus Numeric Rating Scale (WP-NRS; range: 0-10) and Dermatology Life Quality Index (DLQI; range: 0-30) and Children's DLQI (CDLQI; range: 0-30). Secondary outcomes included physician- and patient-reported clinical, psychosocial and economic burden. RESULTS: Of the 1591 patients enrolled, 1558 (1434 adults and 124 adolescents) fulfilled all patient selection criteria and were included in this analysis. Almost all patients (98.4%) in the total population were using AD medications and more than half (56%) were receiving systemic medication (15% systemic monotherapy). The most used systemic therapies were dupilumab (56.3%), systemic glucocorticoids (18.1%) and methotrexate (16.2%). Mean WP-NRS was 5.3 in the total population, and most patients (≥55%) reported moderate-to-severe pruritus (WP-NRS ≥4). Mean DLQI was 10.8 and mean CDLQI was 9.6. Secondary endpoints demonstrated substantial clinical, psychosocial, and economic burden of disease. Subgroup analysis demonstrated that patients receiving systemic therapy had lower disease burden than those not taking systemic medications. CONCLUSIONS: While systemic therapy lowers overall disease burden, patients with moderate-to-severe AD continue to have substantial multidimensional disease burden and uncontrolled disease. Overall, there is a need for effective disease management, including effective treatments that improve patients' psychosocial outcomes and reduce the economic burden of AD.

Patients' Preferences for the Treatment of Metastatic Castrate-resistant Prostate Cancer: A Discrete Choice Experiment.

PURPOSE: Patient treatment preferences are increasingly being used to inform health care decision making. This discrete choice experiment assessed how men perceive the risks and benefits of hypothetical treatment options for metastatic castrate-resistant prostate cancer (mCRPC). METHODS: Treatment attributes for inclusion were identified through a review of the literature and product labels. Expert interviews confirmed clinical appropriateness and patient relevance of the attributes, which included effectiveness (delay in months before chemotherapy), steroid use, possible drug interactions (additional hospital visits for monitoring), fogginess (effects on cognition and memory), fatigue (extreme tiredness), food restrictions, and bone pain. Following a pilot, the final discrete choice experiment included 18 choice sets presenting treatments for mCRPC and was completed by men with mCRPC in France, Germany, and the United Kingdom. Data were analyzed using a conditional logit model, with odds ratios (ORs) used to indicate preference for attributes, and tradeoff measures (TOM) were estimated using the ratio of coefficients. FINDINGS: Within each attribute category and with all other factors being equal, participants (N = 285) indicated a strong preference for treatments that fully control bone pain (OR = 12.069 [95% CI, 10.555-13.800]) and for treatments that delay chemotherapy (OR, 1.727 [95% CI, 1.548-1.927]). They also preferred treatments that were associated with the lowest risk of fogginess (OR, 2.115 [95% CI, 1.849-2.420]), a lower risk of fatigue (OR, 1.365 [95% CI 1.219-1.528]), and fewer additional hospital visits (OR, 1.245 [95% CI 1.111-1.397]) than the respective reference categories. Participants preferred to use steroids under advice from a physician (OR, 1.275 [95% CI 1.132-1.437]). Food restrictions related to taking medication were not a significant concern for participants. TOM results indicated that large tradeoffs in effectiveness, fogginess, and fatigue are required for patients to prefer a treatment with uncontrolled bone pain that is very difficult to live with. IMPLICATIONS: Men with mCRPC consider a wide range of factors when making decisions regarding their treatment. They showed a strong preference for treatment associated with better control of bone pain. They also placed value on treatments that could delay the need for chemotherapy, and they preferred to avoid side effects such as cognition and memory loss, and extreme tiredness. TOMs highlighted the importance of symptom control, even compared with potential side effects. An understanding of the degree to which patients value the attributes associated with various treatment options will assist clinicians and health care professionals when making decisions regarding the management of men with mCRPC.

Systematic Review of Low-Density Lipoprotein Cholesterol Apheresis for the Treatment of Familial Hypercholesterolemia.

BACKGROUND: Apheresis is an important treatment for reducing low-density lipoprotein cholesterol (LDL-C) in patients with familial hypercholesterolemia (FH). We systematically reviewed the current literature surrounding LDL-C apheresis for FH. METHODS AND RESULTS: Electronic databases were searched for publications of LDL-C apheresis in patients with FH. Inclusion criteria include articles in English published in 2000-2013 that provide descriptions of practice patterns, efficacy/effectiveness, and costs related to LDL-C apheresis in patients with FH. Data were stratified by country and FH genotype where possible. Thirty-eight studies met the inclusion criteria: 8 open-label clinical trials, 11 observational studies, 17 reviews/guidelines, and 2 health technology assessments. The prevalence of FH was not well characterized by country, and underdiagnosis was a barrier to FH treatment. Treatment guidelines varied by country, with some guidelines recommending LDL-C apheresis as first-line treatment in patients with homozygous FH and after drug therapy failure in patients with heterozygous FH. Additionally, guidelines typically recommended weekly or biweekly LDL-C apheresis treatments conducted at apheresis centers that may last 2 to >3 hours per session. Studies reported a range for mean LDL-C reduction after apheresis: 57-75% for patients with homozygous FH and 58-63% for patients with heterozygous FH. Calculated annual costs (in US$2015) may reach US$66 374 to US$228 956 per patient for weekly treatment. CONCLUSIONS: LDL-C apheresis treatment may be necessary for patients with FH when drug therapy is inadequate in reducing LDL-C to target levels. While apheresis reduces LDL-C, high per-session costs and the frequency of guideline-recommended treatment result in substantial annual costs, which are barriers to the optimal treatment of FH.

Multivariate analysis of the factors involved in loss of renal differential function after laparoscopic partial nephrectomy: a role for warm ischemia time.

BACKGROUND: Partial nephrectomy (PN) is now the gold standard for the surgical treatment of small renal masses. We evaluated the effect of WIT and other factors on RDF assessed by preoperative and postoperative renal scintigraphy. METHODS: Between 2003 and 2008, 182 consecutive laparoscopic PN (LPN) were performed in an academic centre. Among those, 56 had mercaptoacetyl triglycine (MAG3) lasix renal scintigraphy preoperatively and postoperatively. RESULTS: Medians for age, preoperative estimated glomerular filtration rate and computed tomography scan tumour size were 62 years, 82 mL/min/1.73m(2) and 26 mm, respectively. Median WIT and preoperative RDF were 30 minutes and 50%, respectively. Median loss of RDF after surgery was 14%. Linear regression curves showed that loss in RDF rate was 0.2% per minute when WIT was <30 minutes and 0.7% per minute when WIT was ≥30 minutes. In multivariate analysis, length of WIT and endophytic tumour location were associated with a statistically significant loss of RDF (p < 0.05), but only in the group who experienced >30 minutes of WIT. INTERPRETATION: Our results suggest that the factors associated with loss of RDF are not the same before and after 30 minutes of WIT and that the rate of loss in RDF increases after 30 minutes. Since, the effect of WIT is small up to 30 minutes, we believe that surgery should focus on limiting the resection of normal parenchyma and to ensure negative margins and hemostasis, rather than on premature unclamping.

The relationship between the prevalence of nighttime gastroesophageal reflux disease and disease severity.

BACKGROUND: Nighttime gastrointestinal reflux disease (GERD) prevalence and severity estimates vary substantially across studies. METHODS: We assessed nighttime GERD (NTG) prevalence and symptom frequency and severity through a web survey of US adults, using the GERD Symptom and Medication Questionnaire (GERD-SMQ), a validated symptom questionnaire. NTG was based on episodes of nighttime heartburn per week and time of occurrence. Symptom severity and impact were assessed and compared for GERD cases with and without NTG. RESULTS: GERD prevalence among respondents (n = 2,603) was 27%. Forty-five percent of symptomatic GERD respondents had NTG. Among respondents with both daytime and nighttime symptoms, 51% reported that nighttime symptoms were more bothersome. NTG respondents reported greater disease severity compared with those without (P < 0.0001). CONCLUSIONS: NTG symptoms are very common among those identified with GERD. People with nighttime symptoms have greater disease severity than those with exclusively or primarily daytime symptoms.