BACKGROUND: Treatment options for people with haemophilia are evolving at a rapid pace and a range of prophylactic treatment options using various technologies are currently available, each with their own distinct safety and efficacy profile.
UNASSIGNED: The access to replacement therapy and prophylaxis has driven a dramatic reduction in mortality and resultant increase in life expectancy. Beyond this, the abolition of bleeds and preservation of joint health represent the expected, but rarely attained, goals of haemophilia treatment and care. These outcomes also do not address the complexity of health-related quality of life impacted by haemophilia and its treatment.
CONCLUSIONS: Capitalizing on the major potential of therapeutic innovations, \'Normalization\' of haemostasis, as a concept, should include the aspiration of enabling individuals to live as normal a life as possible, free from haemophilia-imposed limitations. To achieve this-being supported by the data reviewed in this manuscript-the concept of haemostatic and life Normalization needs to be explored and debated within the wider multidisciplinary teams and haemophilia community.

BACKGROUND: Selecting the appropriate treatment for older patients with non-muscle invasive (NMIBC) or muscle-invasive bladder cancer (MIBC) is challenging due to smoking-related comorbidities, treatment toxicity, and an increased risk of adverse health outcomes. Considering patient preferences prior to treatment is therefore crucial. Here, we aimed to identify the health outcome priorities of older patients with high-risk NMIBC (HR-NMIBC) or MIBC.
METHODS: Patients aged 70 years or older or at risk for frailty, diagnosed with HR-NMIBC or MIBC without distant metastases, were referred for a comprehensive geriatric assessment (CGA). The CGA consisted of an interview, physical examination, and several tests to examine physical, cognitive, functional, and social status. Quality of life was assessed using EQ5D and EORTC QLQ-C30 questionnaires. Health outcome priorities were discussed using the Outcome Prioritization Tool (OPT) and associations between health outcome priorities and CGA-determinants and quality of life were studied.
RESULTS: Of 146 patients (14 HR-NMIBC, 132 MIBC), OPT data was available for 139. Life extension was most often prioritized (44%), closely followed by preserving independence (40%). Reducing pain (7%) and other symptoms (9%) were less often prioritized. Patients prioritizing life extension had fewer musculoskeletal problems than patients prioritizing reducing pain or other symptoms (p = 0.02). Patients at risk of or suffering from malnutrition more frequently selected reducing pain or other symptoms as their health outcome priority (p = 0.004). For all other CGA-determinants and quality of life, there were no significant differences between groups based on health outcome priorities.
CONCLUSIONS: In older patients with HR-NMIBC and MIBC, life extension and preserving independence are the most common health outcomes priorities. CGA-determinants and quality of life are generally not associated with the prioritization of health outcomes. As health outcome priorities cannot be predicted by CGA-determinants or quality of life, it is crucial to discuss health outcome priorities with patients to promote shared decision-making.

For more than sixty years, surgeons have used bioethical strategies to promote patient self-determination, many of these now collectively described as \"informed consent.\" Yet the core framework-understanding, risks, benefits, and alternatives-fails to support patients in deliberation about treatment. We find that surgeons translate this framework into an overly complicated technical explanation of disease and treatment and an overly simplified narrative that surgery will \"fix\" the problem. They omit critical information about the goals and downsides of surgery and present untenable options as a matter of patient choice. We propose a novel framework called \"better conversations.\" Herein, surgeons provide context about clinical norms, establish the goals of surgery, and comprehensively delineate the downsides of surgery to generate a deliberative space for patients to consider whether surgery is right for them. This paradigm shift meets the standards for informed consent, supports deliberation, and allows patients to anticipate and prepare for the experience of treatment.

BACKGROUND: Treatment goals have been established in Australia to facilitate the management of adults with moderate to severe psoriasis. The Australasian College of Dermatologists sought to determine if and how these adult treatment goals could be modified to accommodate the needs of paediatric and adolescent patients.
METHODS: A modified Delphi approach was used. Comprehensive literature review and guideline evaluation resulted in the development of statements and other questions to establish current clinical practices. Two rounds of anonymous voting were undertaken, with a collaborative meeting held in between to discuss areas of discordance. Overall, consensus was defined as achievement of ≥75% agreement in the range 7-9 on a 9-point scale (1 strongly disagree; 9 strongly agree).
RESULTS: Consensus was achieved on 23/29 statements in round 1 and 17/18 statements in round 2. There was a high level of concordance with treatment criteria in the adult setting. The limitations of applying assessment tools developed for use in adult patients to the paediatric setting were highlighted. Treatment targets in the paediatric setting should include objective metrics for disease severity and psychological impact on the patients and their family, and be based on validated, age-appropriate tools.
CONCLUSIONS: While the assessment, classification and management of moderate to severe psoriasis in paediatric patients aligns with metrics established for adults, it is vital that nuances in the transition from childhood to adolescence be taken into account. Future research should focus on psoriasis severity assessment scales specific to the paediatric setting.

BACKGROUND: Since the last guidance was published by the Canadian Thoracic Society, there have been several advances in the clinical management of severe asthma. To gain a better understanding of the current standards of care and treatment patterns of patients, the CASCADE practice reflective program was established to conduct a real-world analysis of severe asthma management among specialists in Canada with a goal of identifying areas of opportunity to enhance patient management and outcomes.
METHODS: The CASCADE program was a two-part practice reflective and assessment program delivered through an on-line portal for selected specialists (Respirologists and Allergists) in Canada. The program consisted of a one-time overview survey of physician practice to establish overall practice parameters, followed by a review of at least 5 severe asthma patients to establish the current landscape of severe asthma management.
RESULTS: The program collected practice overview surveys from 78 specialists (52 Respirologists, 24 Allergists, and 2 General practice physicians with an interest in respiratory disease) in 8 provinces. Practices included a variety of types in both large metropolitan centres and smaller regional settings. There were 503 patients reviewed and included in the program. Most (65%) patients were currently using a biologic treatment, 30% were biologic naive, and 5% had used a biologic treatment in the past. Most patients (53%) were reported to have mixed allergic and eosinophilic phenotypes, despite a perception that allergic, eosinophilic and mixed phenotypes were evenly balanced in the physician practice. Overall, patients currently treated with biologic agents had parameters suggesting higher control and were more satisfied with treatment. However, there was less than optimal treatment satisfaction for more than half of all patients, particularly for those patients not treated with a biologic agent.
CONCLUSIONS: Phenotyping is hampered by poor availability for several assessments, and the full range of treatments are not currently fully utilized, partly due to physician familiarity with the agents and partly due to prescribing restrictions. Even when treated with biologic agents, patient satisfaction can still be improved.

BACKGROUND: The National Psoriasis Foundation (NPF) recommends evaluating patient response to treatment at week 12, with a target response of ≤ 1% body surface area (BSA) affected by plaque psoriasis and an acceptable response of BSA ≤ 3% or ≥ 75% improvement. This post hoc analysis compared the achievement of NPF target and acceptable responses for ixekizumab (IXE) versus other biologics.
METHODS: Outcomes were evaluated at week 12 for patients with moderate-to-severe plaque psoriasis from four head-to-head randomized clinical trials (RCTs; UNCOVER-2, UNCOVER-3, IXORA-R, and IXORA-S) and one real-world prospective observational study (Psoriasis Study of Health Outcomes; PSoHO). RCT patients were treated with IXE or etanercept (ETN; UNCOVER-2/3), guselkumab (GUS; IXORA-R), or ustekinumab (UST; IXORA-S). PSoHO patients were treated with anti-interleukin (IL)-17A biologics (IXE, secukinumab, SEC) and other approved biologics for the treatment of plaque psoriasis. Patients with missing outcomes were imputed as non-responder imputation. For RCT data, statistical comparisons between treatment groups were performed using Fisher\'s exact test with no multiplicity adjustments. For real-world data, adjusted comparative analyses were performed using frequentist model averaging (FMA) and reported as odds ratio (OR).
RESULTS: Across the four head-to-head clinical trials analyzed, significantly higher proportions of patients achieved target and acceptable responses at week 12 with IXE versus ETN, GUS, or UST. Likewise, the proportion of PSoHO patients achieving target and acceptable response at week 12 was higher with IXE compared with other individual biologics. Adjusted comparative analyses showed that IXE had significantly greater odds of target and acceptable response at week 12 versus SEC, GUS, risankizumab (RIS), adalimumab (ADA), UST, and tildrakizumab (TILD) and numerically greater odds of target and acceptable response at week 12 versus brodalumab (BROD).
CONCLUSIONS: Across both clinical studies and real-world settings, more patients treated with IXE achieved NPF target and acceptable responses at week 12 compared with those treated with other biologics.
BACKGROUND: UNCOVER-2 (NCT01597245); UNCOVER-3 (NCT01646177); IXORA-R (NCT03573323); IXORA-S (NCT02561806); PSoHO (EUPAS24207).

Alopecia areata (AA), an autoimmune disease -affecting the hair of the scalp, face, and/or body, can entail substantial psychological and physical burden for patients. There is currently no international agreement on how to treat AA and the approach may vary across countries. This study investigated the management of AA in clinical practice. Data from a point-in-time survey conducted in France, Germany, Italy, Spain, and the United Kingdom, between October 2021-June 2022, were analysed for adults with mild, moderate, and severe AA, based on physician assessment. Dermatologists were surveyed about factors used to assess disease severity, physician-reported treatment goals, and treatment patterns for AA, including the use of wigs. In total, 239 dermatologists reported on 2,083 patients. Physicians\' severity assessment and treatment goals were predominantly driven by scalp hair loss. Topical and intralesional corticosteroids were the most prescribed treatments for mild and moderate AA. Conventional immunosuppressants, oral Janus kinase inhibitors, and topical immunotherapy use generally increased with AA severity and therapy line. Wig use was greatest for severe AA. The primary reasons for the last treatment change in the moderate and severe groups were worsening of condition, lack of initial efficacy, and loss of response, and for mild group were improvement in condition, lack of initial efficacy, and worsening of condition. Findings were generally similar across countries. This analysis provides insights into the management of AA in five European countries and confirms the need for more effective therapies, especially for patients with severe AA.

Therapist-client cognitive match upon key constructs such as treatment goals is purported to be an important component of culturally competent care. For adolescent clients, treatment may involve both youths and their parents, suggesting the need to consider both youth-therapist and parent-therapist perspectives. This longitudinal study examined broadband youth-therapist and parent-therapist treatment goal matching and mismatching in relationship to treatment engagement in a culturally diverse sample of 245 outpatient mental health service-using youth. Although goal matching/mismatching did not uniformly predict treatment engagement as measured by a total score, youth-therapist internalizing goal matching predicted better youth engagement, and parent-therapist externalizing goal mismatch marginally predicted worse parent engagement. When selected post hoc analyses examined relationships to four individual engagement dimensions, youth-therapist internalizing goal matches positively predicted youth Client-therapist interaction, Communication/Openness, Client\'s perceived usefulness of therapy, and Collaboration with treatment, while parent-therapist externalizing goal mismatch negatively predicted parent Collaboration with treatment. Findings support the importance of cognitive match on treatment goals as well as the consideration of both parent and youth perspectives, matched and mismatched goals, internalizing and externalizing goals, and examining individual dimensions in addition to total scores of engagement.

Tralokinumab is a human monoclonal antibody targeting interleukin-13 that is approved for the treatment of moderate-severe atopic dermatitis. Studies analyzing the efficacy and safety of tralokinumab in a real-world setting are scarce.
A European, multicentric, real-world, retrospective cohort study was defined to assess the effectiveness and safeness profile of tralokinumab, investigating the achievement of pre-specified treatment goals; and to detect potential differences in terms of effectiveness and safeness across some selected patient subcohorts.
A total of 194 adult patients were included in this study. A significant improvement in physician-assessed disease severity was detected at each follow-up visit as compared with baseline and similar trend was observed for patient-reported outcomes and quality of life. No meaningful difference in effectiveness was found when considering patient age (<65 versus ≥65 years), neither dissecting patient cohort in dupilumab-naive vs dupilumab-treated subjects. Among tralokinumab-treated patients, 88% achieved at least one currently identified real-world therapeutic goal at week 16.
This retrospective multicenter study confirmed the effectiveness and safeness of tralokinumab throughout 32 weeks of observation, showing the achievement of therapeutic goals identified in both trial and real-world settings in a large proportion of tralokinumab-treated patients.

UNASSIGNED: To better understand what is most important to people living with human immunodeficiency virus (PLWH) when choosing their treatment. We assessed how PLWH trade off the potential risks and benefits of oral and long acting injectable (LAI) treatments.
UNASSIGNED: Firstly, in-depth interviews were conducted with 11 PLWH to develop a holistic understanding of experiences and determine attributes that contribute to treatment decision-making. Secondly, a discrete choice experiment (DCE) was used to understand the treatment preferences for PLWH with n = 99 PLWH aged 18 years or over with a diagnosis of human immunodeficiency virus (HIV) and who were currently using anti-retroviral therapy (ART). Study participants were presented with 12 scenarios and asked to select their preferred treatment among two hypothetical injectable treatment alternatives, \"injection 1\" and \"injection 2\" and their current oral ART treatment. The DCE data were modelled using a latent class model (LCM).
UNASSIGNED: The model revealed significant heterogeneity in preferences for treatment attributes among study participants. Two segments/classes of PLWH were identified. The first segment expressed a strong preference for their current oral treatment; the second segment showed strong preference for the injection treatment and for it to be administered in a GP clinic. Overall, out-of-pocket cost was the most important attribute for participants. One-third of PLWH were willing to switch to an LAI.
UNASSIGNED: Not all PLWH valued the same treatment attributes equally. Overall, out-of-pocket costs for treatments were considered by respondents as the most determining factor in making treatment choices. Results have important implications for healthcare policy and will serve to better inform patients and stakeholders involved in the treatment decision-making process about the treatment preferences of PLWH. Clinicians are encouraged to consider shared decision-making to establish the treatment course that best aligns with PLWH\'s treatment goals.