BACKGROUND: Understanding the patient journey of hepatocellular carcinoma (HCC) may inform future clinical decision-making and enhance the patient experience. The objectives of this study were to explore the patient experience of HCC in relation to treatment options, treatment decision-making and treatment goals throughout the disease journey. This study also aimed to determine the symptoms and impacts of HCC across early, intermediate and advanced HCC.
METHODS: Semi-structured 60-min interviews were conducted with n = 50 patients with HCC and n = 12 healthcare professionals (HCPs) with experience of treating patients with HCC. Interview data were analyzed using directed content analysis techniques with a hybrid inductive and deductive approach. An assessment of conceptual saturation was conducted for patients\' symptom experience.
RESULTS: Patients described treatment decisions as mostly HCP-led. In this study, surgery/resection was the most frequently offered treatment option across the HCC journey, and most patients were satisfied with the treatment options presented to them. Overall, patients described extending their overall survival (OS) and preserving quality of life (QoL) as their most important treatment goals, with patients diagnosed with advanced/unresectable HCC prioritizing QoL. HCPs also prioritized OS and progression-free survival (PFS) though reported that QoL became more important as HCC progressed. Patients experienced various symptoms across the HCC journey including fatigue, nausea, appetite loss, diarrhea and pain.
CONCLUSIONS: Overall, HCPs and patients collaborate throughout the treatment journey regarding treatment decisions and shared treatment goals. OS is critically important to patients and HCPs, though treatment goals may change depending on various clinical factors.
Liver cancer is the third leading cause of cancer death worldwide. Very few studies have directly explored how patients experience liver cancer and its treatment. This research was performed to understand the patient experience of liver cancer including treatment decisions and goals, communication dynamics between patients and their treating doctors, the types of treatment offered to patients and symptoms that patients experience while living with liver cancer. Patients and doctors were interviewed to understand their experience of living with and treating liver cancer. Overall, patients and doctors work together to decide on treatment. Patients generally trusted their doctor’s knowledge and expertise when selecting the best treatment(s) for them. Treatment decisions are mostly based on how far along the cancer has progressed. Patients described living longer and maintaining a good quality of life as their most important treatment goals, as well as avoiding liver cancer returning or worsening. Doctors identified that patients living longer, time without worsening of liver cancer and managing side effects as important treatment goals.

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 examine the treatment and long-term outcome preferences for people living with schizophrenia.
UNASSIGNED: Sixty-six Australian adults, living with schizophrenia completed a novel online survey with six sections: Demographic characteristics; Disease history; Quality-of-life; Patient support programmes; Discrete Choice Experiment, and Best-Worst Scaling exercise.
UNASSIGNED: Participants indicated that they preferred to be involved in treatment decision with their doctor. A minority of participants reported having been previously involved in a patient support programme (28.8%) and only one in six participants had a National Disability Insurance Scheme (NDIS) package (16.7%) with over a third of participants indicating that they were ineligible (37.9%). Participants\' average quality-of-life score was 60%.
UNASSIGNED: Recent hospitalisation influenced the relative importance of treatment attributes, with effectiveness on hearing voices being the most important treatment attribute. The most important long-term goals were having a stable place to live, being independent, and physical health. People with schizophrenia care about their long-term functional recovery outcomes, rating symptom control and independence as their highest priority. They want to be part of the treatment conversation with their doctors. Therefore, psychiatrists are encouraged to use shared decision-making to establish the treatment course that best aligns with individuals\' long-term goals.

To obtain additional information on the incremental differences between using a sensor-augmented pump (SAP) without automated insulin delivery (AID), using it with predictive low-glucose management (PLGM) or as hybrid closed loop (HCL), in preschool and school children.
We conducted a monocentric, randomized, controlled, two-phase crossover study in 38 children aged 2-6 and 7-14 years. The primary endpoint was the percentage of time in range (TIR) of 70-180 mg/dl. Other continuous glucose sensor metrics, HbA1c, patient-related outcomes (DISABKIDS questionnaire, Fear of Hypoglycaemia Survey) and safety events were also assessed. Results from 2 weeks of SAP, 8 weeks of PLGM and 8 weeks of HCL were compared using a paired t-test or Wilcoxon signed-rank test.
Overall, we found a high rate of TIR target (>70%) achievement with HCL in preschool (88%) and school children (50%), with average times in Auto Mode of 93% and 87%, respectively. Preschool children achieved a mean TIR of 73% ± 6% (+8% vs. SAP, +6% vs. PLGM) and school children 69% ± 8% (+15% vs. SAP and + 14% vs. PLGM). Overall, HbA1c improved from 7.4% ± 0.9% to 6.9% ± 0.5% (P = .0002). Diabetes burden and worries and fear of hypoglycaemia remained at low levels, without significant changes versus PLGM. No events of severe hypoglycaemia or diabetic ketoacidosis occurred.
Preschool children profit from AID at least as much as those aged 7 years and older. To ensure safe use and prescribing modalities, regulatory approval is also required for young children.

Aim: Clinical efficacy of sublingual immunotherapy for grass pollen-induced allergic rhinitis (AR) needs to translate into patient benefit. Patients & methods: Patients received Oralair (Stallergenes, Antony, France) in real-life medical practice. Patient-relevant treatment benefits were measured with the AR-specific Patient Benefit Index. Subgroups were analyzed regarding distinct patient characteristics. Results: Data of 883 patients (children, adolescents, and adults) were analyzed. The highest-ranked patient needs referred to having less AR symptoms, being able to go outdoors, and being free in the choice of leisure activities. Most patients (89.2-94.6%) attained at least minimally relevant benefit. All subgroups reported relevant benefits, with significantly higher scores in some subgroups. Conclusion: Treatment with Oralair was associated with considerable patient-relevant benefit in all age groups.

Previous studies have investigated patients\' treatment goals before starting a treatment for metastatic lung cancer. Data on the evaluation of treatment goals are lacking.
To determine if patients with metastatic lung cancer and their oncologists perceive the treatment goals they defined at the start of systemic treatment as achieved after treatment and if in hindsight they believe it was the right decision to start systemic therapy.
A prospective multicenter study in 6 hospitals across the Netherlands between 2016 and 2018. Following systemic treatment, 146 patients with metastatic lung cancer and 23 oncologists completed a questionnaire on the achievement of their treatment goals and whether they made the right treatment decision. Additional interviews with 15 patients and 5 oncologists were conducted.
According to patients and oncologists, treatment goals were achieved in 30% and 37% for \'quality of life,\' 49% and 41% for \'life prolongation,\' 26% and 44% for \'decrease in tumor size,\' and 44% for \'cure\', respectively. Most patients and oncologists, in hindsight, felt they had made the right decision to start treatment even if they had not achieved their goals (72% and 93%). This was related to the feeling that they had to do \'something.\'
Before deciding on treatment, the treatment options, including their benefits and side effects, and the goals patients have should be discussed. It is key that these discussions include not only systemic treatment but also palliative care as effective options for doing \'something.\'

OBJECTIVE: Misconceptions regarding activity and toxicity of therapeutic interventions are common among cancer patients. There is little knowledge about the factors that contribute to a more realistic perception by patients.
METHODS: This pilot study was designed as a prospective questionnaire survey and included 101 therapy-naïve patients treated at the Division of Oncology, Medical University of Vienna. After obtaining written informed consent, patients\' expectations about treatment aims, side effects and the satisfaction with their oncologic consultation were interrogated before the first treatment cycle by questionnaires.
RESULTS: Of 101 patients, 53 (53%) were female and 67/101 (66%) were treated with curative attempt in an adjuvant or neo-adjuvant setting. The most common diagnoses were lung cancer (31%) and breast cancer (30%). Although 92% of patients were satisfied with the information given by their oncologist, palliative patients were more likely to declare that not everything was explained in an intelligible manner (p = 0.01). Patients with a first language other than German stated more often that their physician did not listen carefully enough (p = 0.02). Of 30 patients, 26 (87%) receiving chemotherapy with palliative intent believed that their disease was curable. Concerning adverse events, female patients anticipated more frequently hair loss (p = 0.003) and changes in taste (p = 0.001) compared to men. Patients under curative treatment were more likely to expect weight loss (p = 0.02) and lack of appetite (p = 0.01) compared to patients with palliative treatment intent.
CONCLUSIONS: In conclusion, cancer patients were satisfied with the patient-doctor communication. This prospective study aggregated patients\' concerns on side effects and the perception of therapeutic goals in therapy-naïve patients. Of note, the majority of patients treated in the palliative setting expected their treatment to cure the disease.

BACKGROUND: Lung cancer has a high impact on both patients and relatives due to the high disease burden and short life expectancy. Previous studies looked into treatment goals patients have before starting a systemic treatment. However, studies on relatives\' perceptions of treatment at the end of life are scarce. Therefore, we studied the perspectives of relatives in hindsight on the achievement of treatment goals and the choice to start treatment for metastatic lung cancer of their loved one.
METHODS: We conducted a structured telephone interview study in six hospitals across the Netherlands, one academic and five non-academic hospitals, between February 2017 and November 2019. We included 118 relatives of deceased patients diagnosed with metastatic lung cancer who started a systemic treatment as part of usual care (chemotherapy, immunotherapy or targeted therapy with tyrosine kinase inhibitors (TKIs) and who completed a questionnaire on their treatment goals before the start of treatment and when treatment was finished. We asked the relatives about the achievement of patients\' treatment goals and relatives\' satisfaction with the choice to start treatment. This study is part of a larger study in which 266 patients with metastatic lung cancer participated who started a systemic treatment and reported their treatment goals before start of the treatment and the achievement of these goals after the treatment.
RESULTS: Relatives reported the goals \'quality of life\', \'decrease tumour size\' and \'life prolongation\' as achieved in 21, 37 and 41% respectively. The majority of the relatives (78%) were satisfied with the choice to start a treatment and even when none of the goals were achieved, 70% of the relatives were satisfied. About 50% of relatives who were satisfied with the patients\' choice mentioned negative aspects of the treatment choice, such as the treatment did not work, there were side effects or it would not have been the relatives\' choice. Whereas, 80% of relatives who were not satisfied mentioned negative aspects of the treatment choice. The most mentioned positive aspects were that they tried everything and that it was the patient\'s choice.
CONCLUSIONS: The majority of relatives reported patients\' treatment goals as not achieved. However, relatives were predominantly satisfied about the treatment choice. Satisfaction does not provide a full picture of the experience with the treatment decision considering that the majority of relatives mentioned (also) negative aspects of this decision. At the time of making the treatment decision it is important to manage expectations about the chance of success and the possible side effects of the treatment.

Sublingual immunotherapy (SLIT) with birch pollen extract has been shown to be an efficacious treatment of allergic rhinitis (AR). An as-yet unanswered question is whether and how clinical benefit translates into patient benefit, i.e. what benefit patients derive from this treatment.
This 1-year, open, prospective, multicenter, non-interventional study conducted in 75 German centers measured patient-relevant benefit of birch pollen SLIT (Staloral® Birch) using the questionnaire \"Patient Benefit Index for Allergic Rhinitis (PBI-AR)\". At treatment onset, patients rated the importance of 25 treatment needs; after the first birch pollen season on treatment, goal achievement was evaluated. A preference-weighted benefit index was calculated and its association with gender, asthma, allergy status, and severity of AR symptoms was determined.
Mean age of the 291 adult patients was 38.8 years; 58.4% were female. The most important treatment goals were to \"be able to stay outdoors without symptoms\" (87.3% quite or very important), \"no longer have a runny or stuffed-up nose\" (86.9%), and \"be able to breathe through your nose more freely\" (86.9%). The treatment goals with the highest benefit ratings (referring to those patients to whom the respective goal applied) were to \"have confidence in the therapy\" (60.5% has helped \"quite\" or \"very much\"), \"have an easily applicable treatment\" (55.6%), and \"be able to breathe through my nose more freely\" (51.7%). The average PBI-AR global score was 2.19 (SD 1.04) (0-4; with 4 indicating maximum benefit). No significant differences in PBI-AR global score or subscales were found between men and women, poly- and monoallergic patients, or patients with severe versus mild rhinoconjunctivitis. Patients with asthma reported relevant but lower benefit than patients without asthma.
After 1 year of birch pollen SLIT treatment, patients reported considerable benefit, mainly due to a reduction of physical symptoms and treatment burden.

To estimate the proportion of patients with a recent myocardial infarction (MI) who would be eligible for additional lipid-lowering therapy according to the 2019 European Society of Cardiology (ESC)/European Atherosclerosis Society (EAS) guidelines for the management of dyslipidaemias, and to simulate the effects of expanded lipid-lowering therapy on attainment of the low-density lipoprotein cholesterol (LDL-C) target as recommended by the guidelines.
Using the nationwide SWEDEHEART register, we included 25 466 patients who had attended a follow-up visit 6-10 weeks after an MI event, 2013-17. While most patients (86.6%) were receiving high-intensity statins, 82.9% of the patients would be eligible for expanded lipid-lowering therapy, as they had not attained the target of an LDL-C level of <1.4 mmol and a ≥50% LDL-C level reduction. When maximized use of high-intensity statins followed by add-on therapy with ezetimibe was simulated using a Monte Carlo model, the LDL-C target was reached in 19.9% using high-intensity statin monotherapy and in another 28.5% with high-intensity statins and ezetimibe, while 50.7% would still be eligible for proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors. When use of alirocumab or evolocumab was simulated in those who were eligible for PCSK9 inhibitors, around 90% of all patients attained the LDL-C target.
Our study suggests that, even with maximized use of high-intensity statins and ezetimibe, around half of patients with MI would be eligible for treatment with PCSK9 inhibitors according to the 2019 ESC/EAS guidelines. Considering the current cost of PCSK9 inhibitors, the financial implications of the new guidelines may be substantial.