Leo Pharma (France)

IntroductionThe effectiveness of psoriasis treatment is assessed by standardized tools such as the Dermatology life Quality Index (DLQI) and Psoriasis Area Severity Index (PASI). However, discrepancies between patients and physicians in terms of treatment success and goals, along with the growing importance of shared decision-making in healthcare, highlight the need for tools specifically designed for psoriasis. Such tools can enhance communication between patients and physicians, encouraging shared decision-making and improving the assessment of patient treatment expectations.MethodsComparison of the new PSO-TARGET grid, which consists of 12 therapeutic goals evenly distributed across 4 major components commonly used in quality of life (QoL) studies for chronic diseases, with DLQI as a standard tool, was utilized.ResultsA total of 143 adult patients with moderate-to-severe psoriasis and treated with brodalumab were included. On the basis of a blind assessment, dermatologists were not able to identify the patients’ chosen PSO-TARGET goal in more than 50% of cases. The comparison after 12/16 weeks of treatment revealed some discrepancies between the two QoL tools. Compared with the rest of the population, the patients who achieved their PSO-TARGET goal, but still reported a DLQI > 1, had higher baseline PASI scores (18.6 versus 14.8; p = 0.067), higher DLQI scores (14.1 versus 10.1; p = 0.004), and a higher number of hard-to-treat locations (median of 2 versus 1; p = 0.004). In addition, patients who had not reached their PSO-TARGET goal but reported a DLQI ≤ 1, all had psoriasis on the scalp at the baseline and were generally younger (median of 31 versus 52 years, p = 0.001).ConclusionsThis study highlights the importance of considering patient characteristics of those with psoriasis and perspectives when evaluating treatment outcomes. Using shared decision-making tools such as the PSO-TARGET grid can improve communication and understanding between dermatologists and patients.Trial RegistrationClinicalTrials.gov identifier, NCT04765332.Supplementary InformationThe online version contains supplementary material available at 10.1007/s13555-025-01356-8.

A workshop held at the 18th Annual Conference of the Pharmaceutical Contract Management Group in Krakow on 9 September 2022 asked over 200 delegates what the clinical trial landscape would look like in 2050. Issues considered included who will be running the pharmaceutical industry in 2050; how 'health chips', wearables and diagnostics will impact on finding the right patients to study; how will artificial intelligence be designing and controlling clinical trials; and what will the role of the Clinical Research Associate, the critical observer, documenter and conductor of a clinical trial need to look like by 2050. The consensus was that, by 2050, if you are working in clinical trials, you will be a data scientist. We can expect to see an increasing role of new technologies and a new three-phase registration model for novel therapies. The first phase will involve an aspect of quality evaluation and biological proof-of-concept probably involving more preclinical modelling and engineered human cell lines and fewer animal studies than currently used. Once registered, new products will enter a period of adaptive clinical development (delivered as a single study) intended to establish safety. This phase will most likely take around 1-2 years and explore tailored options for administration. Investigations will most likely be conducted in patients, possibly in a 'patient-in-a-box' setting (hospital or healthcare centre, virtual or microsite). On completion of safety licencing, drugs will begin an assessment of efficacy in partnership with those responsible for reimbursement - testing will be performed in patients, possibly where individual patient involvement in safety testing will offer some reimbursement deal for future treatment. Change is coming, though its precise form will likely depend on the creativity and vision of sponsors, regulators and payers.

Updated clinical practice guidelines recommend the long-term use of low-molecular-weight heparins or direct oral anticoagulants as the preferred option for the treatment of cancer-associated thrombosis (CAT), using a personalized approach matching the right drug to the right patient. In most cases, the benefit of anticoagulant therapy outweighs the risk. However, the long-term use of anticoagulants is associated with a non-negligible risk of bleeding, which constitutes a rare but serious adverse effect. Bleeding complications have been reported to be overall 2 to 3 times more frequent in cancer patients with CAT receiving anticoagulation than in non-cancer patients, with a reported incidence of major bleeding ranging from 2.4 to 16.0% in randomized controlled trials (RCT). In the absence of validated risk assessment model to predict the risk of bleeding in these patients, a careful evaluation of each individual profile, with adequate selection of the most appropriate anticoagulant for each individual patient, is warranted for overcoming management challenges, taking in account the numerous factors which may potentiate the overall bleeding risk in these complex patients, such as advanced or metastatic disease, older age, anemia, thrombocytopenia, renal impairment, liver dysfunction, and concomitant anticancer therapies. The purpose of this review is to call for awareness on bleeding complications as a major safety issue of CAT treatment and to summarize data from recent RCT and real-world studies on the incidence and risk factors for bleeding in this unique and challenging population to further help clinicians in decision-making.

Recurrent venous thromboembolism (VTE) despite curative anticoagulation is frequent in patients with cancer. Identifying patients with a high risk of recurrence could have therapeutic implications. A prospective study was designed to validate the Ottawa risk score of recurrent VTE in cancer patients. In a prospective multicenter observational cohort, adult cancer patients with a recent diagnosis of symptomatic or incidental lower limb deep vein thrombosis or pulmonary embolism (PE) were treated with tinzaparin for 6 months. The primary endpoint was the recurrence of symptomatic or asymptomatic VTE within the first 6 months of treatment. All clinical events were centrally reviewed and adjudicated. Time-to-event outcomes were estimated by the Kalbfleisch and Prentice method to take into account the competing risk of death. A C-statistic value of > 0.70 was needed to validate the Ottawa score. A total of 409 patients were included and analyzed on an intention-to-treat basis. Median age was 68 years, 60.4% of patients had PE, and VTE was symptomatic in 271 patients (66.3%). The main primary sites were lung (31.3%), lower digestive tract (14.4%), and breast (13.9%) cancers. The Ottawa score was high (≥ 1) in 58% of patients. The 6-month cumulative incidence of recurrent VTE was 7.3% (95% confidence interval [CI]: 4.9-11.1) overall, and 5.0% (95% CI: 2.3-10.8) versus 9.1% (95%CI: 6.1-13.6) in the Ottawa low versus high risk groups, respectively. The C-statistic value was 0.60 (95% CI: 0.55-0.65). In this prospective cohort of patients with cancer receiving tinzaparin for VTE, the Ottawa score failed to accurately predict recurrent VTE.

Topical treatments are first-line therapies, prescribed to most patients with chronic plaque psoriasis. This non-interventional, longitudinal study examined data regarding the treatment pathways of French patients with psoriasis vulgaris using a pharmacy database. From this database, patients with an initial prescription of a topical treatment of interest (ie, calcipotriol alone and/or calcipotriol/betamethasone) between March and October 2013 were included in the study. The primary objective was to capture the switch from a topical treatment, from treatment initiation to receipt of a systemic therapy over a period of 3 years. A total of 26 605 patients were included in the study. The mean age was 58.5 years. The majority of patients (94.7%) maintained topical treatment during the 3 years, receiving a mean of 1.1 different therapies. Of 1400 patients who switched to a systemic therapy, 93.1% switched to a non-biological (mean time to switching >400 days), maintaining this for the remainder of the follow-up period. The most commonly prescribed first non-biological systemic therapy was methotrexate (37%). Less than 1% of patients switched to a biological therapy during follow up. Cohort analyses suggest that patients progressing to use of a systemic therapy within 12 months were those with more severe disease. There was a low rate of transition from topical to systemic therapies in patients with chronic plaque psoriasis during the first 3 years of treatment, suggesting stability of disease severity over time with topical therapy alone, potentially due to good patient adherence.