Data on the long-term management of neurogenic bladder (NGB) are scarce. We evaluated the current status of NGB management in Japanese children over 24-month follow-up using the JMDC database. In this descriptive, observational, retrospective cohort study, patients (≤ 17years) with NGB were included. Patient characteristics and their management status were investigated. A multivariate analysis evaluating the potential risk factors for the development of urinary tract infection (UTI) was performed. The diagnosis of spina bifida, demographics, baseline comorbidities, and early use of clean intermittent catheterization (CIC) and/or overactive bladder (OAB) drugs were used as independent variables. Of 883 eligible children, 39.3% had spina bifida. Over 12/24-month post-index periods, renal urinary tract ultrasound and urinalysis were performed at least once in > 35%/> 45% patients, respectively, while specific tests (urodynamics, cystourethrography, scintigraphy) were performed in substantially fewer (< 11%/< 13%) patients. Over 24months, 21.5% patients used OAB medications (mostly anticholinergics) and 10.8% underwent CIC, alone or with medications; 1.2% patients underwent surgery. Lower UTI (23.3%), urinary incontinence (9.7%), and hydronephrosis (7.0%) were the most common incident complications. Multivariate analysis evaluating risk factors for UTI showed significantly higher odds ratios with point estimates of ≥ 2 for CIC (5.70), presence of spina bifida (2.86), and constipation (2.07). Overall, urodynamic assessments were inadequately performed. Patients with use of CIC and/or having spina bifida and constipation had a higher risk of UTI, suggesting the need for careful follow-up. More guideline-compliant and diligent patient management is necessary in Japanese children with NGB.

Erosive esophagitis (EE) is a severe form of gastroesophageal reflux disease commonly treated with proton pump inhibitors (PPIs). The aim of this retrospective, observational cohort study was to describe the characteristics and healthcare burden of patients with EE. We identified adults in the USA with an EE diagnosis between January 1, 2016 and February28, 2019 in a linked dataset containing electronic health records (EHR) from the Veradigm Network EHR and claims data from Komodo Health. Patients were required to have 1year of baseline data and 3years of follow-up data. Patients were stratified by the number of PPI lines of therapy (LOT) during the 4-year study period. We descriptively captured patient characteristics and treatment patterns, along with all-cause and EE-related healthcare utilization and costs. Among the 158,347 qualifying adults with EE, 71,958 (45.4%) had 1PPI LOT, 14,985 (9.5%) had 2LOTs, 15,129 (9.6%) had 3+LOTs, and 56,275 (35.5%) did not fill a PPI prescription. Omeprazole and pantoprazole comprised more than 70% of any LOT, with patients commonly switching between the two. Mean (standard deviation) annualized all-cause and EE-related healthcare costs in the follow-up period were $16,853 ($70,507) and $523 ($3659), respectively. Both all-cause and EE-related healthcare costs increased with LOTs. Patients with EE are commonly treated with prescription PPIs; however, 19.0% of patients cycled through multiple PPIs. Higher PPI use was associated with a higher comorbidity burden and higher healthcare costs compared to 0PPI use.

Progression of fibrosis in interstitial lung diseases (ILD) has been associated with poor prognosis, lower quality of life for patients and caregivers, and higher healthcare costs. This study estimated the burden of disease and productivity loss of progressively fibrosing ILD, focusing on progressive pulmonary fibrosis other than idiopathic pulmonary fibrosis (non-IPF PPF) and systemic sclerosis-associated ILD (SSc-ILD) in the European Economic Area (EEA). An economic model was built to estimate the clinical burden of SSc-ILD and non-IPF PPF. The model was based on published data on disease prevalence and disease burden (in terms of comorbidities, exacerbations, and deaths) as well as on productivity loss (in terms of sick days, early retirement, permanent disability, and job loss). Aggregate income loss was obtained by multiplying productivity loss by the median daily income in each country/area of investigation. A sensitivity analysis was performed to test the impact of the variability of the model assumptions. In the whole EEA, a total of 86,794 and 13,221 individuals were estimated to be affected by non-IPF PPF and SSc-ILD, respectively. Estimated annual sick days associated with the diseases were 3,952,604 and 672,172, early retirements were 23,174 and 5341, permanently disabled patients were 41,748 and 4037, and job losses were 19,789 and 2617 for non-IPF PPF and SSc-ILD, respectively. Annual exacerbations were estimated to be 22,401-31,181 and 1259-1753, while deaths were 5791-6171 and 572-638 in non-IPF PPF and SSc-ILD, respectively. The estimated annual aggregate income loss in EEA, accounting for losses due to annual sick days, early retirements, and permanently disabled patients, was €1433 million and €220 million in non-IPF PPF and SSc-ILD, respectively. The productivity loss due to job losses was €194 million and €26 million in non-IPF PPF and SSc-ILD, respectively. The main driver of aggregate income loss variability was the prevalence. The impact of non-IPF PPF and SSc-ILD on society is definitely non-negligible. Actions to reduce the burden on our societies are highly needed.

Data describing real-world treatment patterns in patients with metastatic urothelial carcinoma (mUC) in Central-Eastern Europe are scarce, and data from Hungary have not been published. This retrospective, nationwide, real-world study investigated patient characteristics, treatment patterns, comorbidities, and clinical outcomes in patients with mUC in Hungary. Adults diagnosed with mUC from January 2016 through June2021 were identified using the National Health Insurance Fund Administration database. Overall survival (OS) was estimated using the Kaplan-Meier method. In total, 2523 patients with mUC were identified. Median follow-up was 7.1months. Overall, 50% of patients received an identified systemic anticancer treatment; within this subgroup, first-line treatment was platinum-based chemotherapy (PBC) in 86%, non-PBC in 8%, and immune checkpoint inhibitor (ICI) in 6%. The proportion of patients receiving treatment increased from 41% in 2016 to 59% in 2020, driven by increased use of first-line PBC or first-line ICI treatment. Comorbidities were more common in patients receiving first-line ICI treatment vs PBC or non-PBC and in patients receiving carboplatin + gemcitabine vs cisplatin + gemcitabine. Overall, only 24% received a second-line treatment. Unadjusted median OS from the start of first-line treatment in the PBC, non-PBC, and ICI subgroups was 12.8, 7.5, and 6.3months, respectively. Median OS from date of diagnosis in untreated patients was 7.8months. OS comparisons adjusted for differences in baseline characteristics between subgroups could not be performed. To our knowledge, this is the first study to assess treatment patterns in patients with mUC in clinical practice in Hungary, using the national health insurance database. Rates of first- and second-line treatment were consistent with those observed in other countries. Avelumab first-line maintenance treatment became available for reimbursement in Hungary in late 2022, after the study period. Given the evolving landscape of reimbursed treatments in Hungary, further analyses are warranted.

Psoriasis, an incurable chronic inflammatory disease, affects over 6 million people in China. Ixekizumab, a monoclonal antibody against interleukin-17A, has demonstrated efficacy and safety for the treatment of moderate-to-severe plaque psoriasis, although limited data are available regarding its use in routine clinical practice in China. We investigated the real-world application of ixekizumab in China. Adults (≥18 years) with moderate-to-severe plaque psoriasis prescribed ixekizumab in routine clinical practice were enrolled in this prospective, observational, single-arm, multicenter, post-marketing surveillance study. The primary endpoint was the safety of ixekizumab at week 12. The effectiveness of ixekizumab, based on the Psoriasis Area and Severity Index (PASI) and Dermatology Life Quality Index (DLQI), was assessed as a secondary endpoint. In total, 666 patients were enrolled; 663 were included in the safety analysis, and 612 in the effectiveness analysis. At least one adverse event (AE) was reported by 42.7% (283/663) of patients, most of which were mild (242/283, 85.5%), and 32.7% (217/663) of patients reported AEs related to study treatment. The most frequently reported AEs were injection site reactions. AEs led to discontinuation in five patients (0.8%). Only three patients had a serious AE. Mean±standard deviation (SD) change from baseline in PASI score was reduction in 10.79±9.55 at week 2 and 16.80±12.15 at week 12. At week 2, 63.7% of patients achieved PASI 50. At week 12, 93.2%, 77.4%, and 45.1% of patients achieved PASI 75, PASI 90, and PASI 100, respectively. Mean±SD change from baseline in DLQI was reduction in 5.91±6.27 at week 2 and 9.76±7.16 at week 12. DLQI 0/1 was achieved by 19.8% and 59.9% of patients at week 2 and 12, respectively. Ixekizumab was well tolerated and effective in real-world clinical practice in Chinese adults with moderate-to-severe plaque psoriasis.

Clofarabine monotherapy at a dose of 52mg/m2 per day was approved in the USA in 2004 for the treatment of relapsed or refractory acute lymphoblastic leukemia (R/R ALL) in patients aged 1-21years after at least two prior regimens. To address a post-marketing requirement for additional evidence of the clinical benefit of clofarabine in its approved indication, a meta-analysis of patient-level data was conducted. A systematic literature review was conducted, using the Dr.Evidence software platform, DOC Search, and Embase, to identify clinical trials with patients with R/R ALL who received clofarabine monotherapy at 52mg/m2. The primary endpoint was complete remission (CR). Secondary endpoints were overall remission (OR, defined by CR or CR with either incomplete platelet recovery or incomplete neutrophil and platelet recovery), duration of response, overall survival (OS), and safety. A total of 754 patients in 12 clinical studies were analyzed including 682 patients with R/R ALL treated with clofarabine monotherapy at 52mg/m2; of them, 374 were aged < 22years (pediatric population). Rates of CR and OR were 16% (95% confidence interval [CI] 7, 26) and 28% (95%CI 20, 37), respectively, in the pediatric population and 12% (95%CI 5, 21) and 21% (95%CI 13, 31) in the overall population. Median OS (evaluable in three studies in pediatric patients) was 3.7months (95%CI 0.1, 31.4), reaching 10.1months (95%CI 0.3, 68.9) for those achieving OR. Sensitivity analyses supported these findings. The most frequent grade3-4 adverse events were liver abnormalities, anemia, diarrhea, and febrile neutropenia. In this meta-analysis, CR duration and median OS in pediatric patients with R/R ALL appeared to be slightly longer than in the phaseII study. No new safety signals were identified. Results support the use of clofarabine monotherapy in its approved indication.

Focal segmental glomerulosclerosis (FSGS) is a leading cause of kidney disease and can progress to end stage kidney disease (ESKD). An overview of symptoms and impacts of the disease experienced will help inform the selection or development of fit-for-purpose clinical outcome assessments (COA) to be used in FSGS clinical trials. This study aimed to develop a conceptual model (CM) of the adult and pediatric patient experience of FSGS including disease signs/symptoms, treatment side-effects, and impact on functioning and wellbeing. This study comprised a systematic review and thematic analysis of qualitative studies with adults and pediatric patients diagnosed with FSGS. Data sources were identified through an electronic database search of journal articles (Medline, Embase, PsycINFO; June 2021) and hand-searching of conference proceedings, patient advocacy group websites, and gray literature. Non-English articles were excluded. Identified data (patient/caregiver quotes, author summaries, and interpretations of patient experiences) were extracted from the articles. Extracted data were qualitatively analyzed aided by ATLAS.ti v7. Codes were applied to data and concepts (symptoms/impacts) were identified, named, and refined. A CM was developed by grouping related concepts into domains. In total, 12 sources were identified for analysis: 6 journal articles and 6 series of patient testimonials. Salient sign/symptom/side-effect domains included swelling/puffiness (edema), pain/aches/discomfort, fatigue, weight changes, skin problems, respiratory problems, and sleep problems. Salient impact domains included emotional/psychological wellbeing, physical functioning/activities of daily living, social functioning, and work/school. Secondary analysis of published qualitative literature permitted development of a CM describing the adult and pediatric experience of FSGS. Concept elicitation interviews are recommended to refine the CM, confirm the salient/most bothersome concepts, and confirm the extent of impact on daily life. The refined CM will provide a useful tool to inform the selection, development, and/or amendment of COAs for use in future FSGS clinical trials.

Hepatorenal syndrome (HRS), a special form of acute kidney failure, is a rare, acute, life-threatening complication of cirrhosis and has a very poor prognosis. Terlipressin (TERLIVAZ®) is the first and only pharmacological treatment approved by Food and Drug Administration (September 2022) to improve kidney functionfor adults with HRS with rapid reduction in kidney function. We constructed a decision analytic economic model to estimate the cost per complete response/HRS reversal of terlipressin+albumin from a United States hospital perspective. A decision analytic model was developed to estimate the HRS treatment-related cost per response over an HRS hospitalization (assuming 14days). Patients can experience either HRS reversal (complete response) or no HRS reversal (partial/no response) upon receipt of treatment. The efficacy, safety, and treatment duration data were from published head-to-head randomized international trials. Total treatment cost comprised drug acquisition and treatment-related costs (intensive care unit [ICU], dialysis [intermittent or continuous], pulse oximetry monitoring for terlipressin, and adverse events) sourced from the published literature. Cost per response, defined as the total treatment cost per HRS reversal was estimated for each treatment. The number needed to treat (NNT), defined as the number of patients treated to achieve HRS reversal in 1 additional patient, was estimated. Cost per response of terlipressin+albumin was lower than midodrine and octreotide+albumin (M&O) (US$85,315 vs. $467,794) and norepinephrine+albumin ($81,614 vs. $139,324). NNT for HRS reversal was 2 patients with terlipressin+albumin vs. M&O+albumin and 4 patients with terlipressin+albumin vs. norepinephrine+albumin, respectively. The analysis shows that terlipressin is a cost-effective treatment due to its higher efficacy and administration in the non-ICU setting. Terlipressin is a value-based treatment option for appropriate adults with HRS with rapid reduction in kidney function.

This is a summary of a previously published paper: Joint Healthcare Professional and Patient Development of Communication Tools to Improve the Standard of MS Care. It describes a collaboration between people with multiple sclerosis (PwMS) and healthcare professionals (HCPs) to identify challenges in multiple sclerosis (MS) care and design tools to improve communication during consultations.

Given the growing interest and use of interleukin-17 inhibitors (anti-IL17) for the treatment of psoriatic arthritis (PsA), an observational study has been conducted to characterize the patient profile, treatment patterns, and persistence of ixekizumab or secukinumab in patients with PsA receiving them as first anti-IL17. This is a multicenter retrospective study, conducted at eight Spanish hospitals where data from adult patients with PsA were collected from electronic medical records. Three cohorts of patients, initiating treatment with an anti-IL17 [secukinumab 150mg (SECU150), secukinumab 300mg (SECU300), or ixekizumab (IXE)] between January 2019 and March 2021, were included. Demographic and clinical patient characteristics, treatment patterns, and persistence were analyzed descriptively. Continuous data were presented as mean [standard deviation (SD)] and categorical variables as frequencies with percentages. Persistence rates at 3, 6, and 12months were calculated. A total of 221 patients with PsA were included in the study [SECU150, 103 (46.6%); SECU300, 38 (17.2%); and IXE, 80 (36.2%)]. Treatment patterns differed by clinical characteristics: SECU150 was initiated more frequently in patients with moderate PsA and less peripheraljoint involvement, while patients on SECU300 included those with a higher rate of enthesitis and active skin psoriasis, and patients on IXE showed a longer time since PsA diagnosis, more frequent comorbidities, joint involvement, and diagnosed skin psoriasis. Conventional synthetic disease-modifying anti-rheumatic drugs (csDMARDs) were previously administered in 88.2% of patients and biologic or targeted synthetic disease-modifying anti-rheumatic drugs (b/tsDMARDs) were administered in 72.9%. The mean number of previous b/tsDMARDs was 2.4 (SD 1.5) in the IXE cohort, 1.7 (SD 0.9) in the SECU300 cohort, and 1.6 (SD 1.0) for those in the SECU150 cohort. The global persistence on all anti-IL17 was 97.2%, 88.4%, and 81.0% at 3, 6, and 12months, respectively. The most frequent reason for discontinuation across the three cohorts was lack of effectiveness (16.7%; 37/221). Most of the patients with PsA treated with anti-IL17 in Spain had moderate to severe disease activity, high peripheral joint and skin involvement, and had received previous b/tsDMARDs. More than 80% of patients with a 1-year follow-up persisted on anti-IL17, with the highest rate observed in the IXE cohort, followed by the SECU150 then SECU300 cohorts.