Current Treatment Goals Research Articles

Disease severity in moderate‐to‐severe plaque psoriasis: Results from the Belgian REDISCOVER study

AbstractBackgroundIn the Belgian DISCOVER study, conducted in 2011–2012 before the advent of the IL‐17 and IL‐23 Inhibitors, significant undertreatment of patients with plaque psoriasis was reported.ObjectivesThe present study aimed to re‐evaluate the real‐world clinical management and quality of care of moderate‐to‐severe plaque psoriasis patients treated with systemic treatment for at least 24 weeks in clinical practice in Belgium.MethodsThis cross‐sectional and retrospective chart review (REDISCOVER) was conducted in 2021–2022 in private practices and hospitals in Belgium. Patient data were collected during one single visit and included assessments of psoriasis severity (Psoriasis Area Severity Index [PASI], Body Surface Area [BSA], Pruritus VAS scale) and Health‐Related Quality of Life (Dermatology Life Quality Index [DLQI]) and other selected criteria of the Belgian treat‐to‐target (T2T) outcome set.ResultsOf the 306 patients included in the study, 81.4% received treatment with biologicals and 18.6% with traditional systemics, that is, methotrexate or apremilast. IL‐23 Inhibitors (33.7%) and methotrexate (10.8%) were the most prescribed biologicals and traditional systemic, respectively. Between the time of treatment initiation and enrolment, the average PASI and BSA scores decreased, indicating treatment benefits. PASI scores were lower in patients treated with biologicals versus patients treated with traditional systemics. Full achievement of the two Belgian T2T guidelines dimensions was observed in 51% of the patients. Nonetheless, patients were generally satisfied with their treatment (96.1%) and physicians evaluated the treatment as adequate for most patients (87.3%).ConclusionsCompared to 10 years ago, a demonstrable improvement in care of moderate‐to‐severe psoriasis patients was observed in Belgium. This was reflected by substantial achievement of the current treatment goals and high treatment satisfaction by both patients and physicians. While one out of five patients is not achieving targeted goals, and is not treated with a biological, it seems there is still room for improvement.

Efficacy of Risankizumab in Patients with Suboptimal Response or Failure to Ustekinumab: A Multicenter Retrospective Study in Spain

Background. Current psoriasis treatment goals emphasize achieving complete or near‐complete skin clearance while preserving the quality of life, necessitating treatment modification for a suboptimal or inadequate response. Despite risankizumab’s demonstrated efficacy, evidence remains limited for patients switching from ustekinumab, particularly those with suboptimal or inadequate response. Objective. This study assesses risankizumab’s real‐world effectiveness in patients with suboptimal response (PASI 2‐3), inadequate response (PASI 3–5), or failure (PASI >5) to ustekinumab, based on a multicenter retrospective cohort in Spain. Method. A multicenter retrospective study across 24 Spanish hospitals included 102 patients previously treated with ustekinumab and switched to risankizumab. Results. Out of 102 patients, 78 experienced ustekinumab treatment failure (PASI >5), while 24 had an inadequate response (PASI 3–5), including 6 with a suboptimal response (PASI 2‐3). Remarkably, after one year of treatment with risankizumab, all patients demonstrated improvement, achieving near‐complete or complete skin clearance (PASI 0‐1). Conclusion. Risankizumab displayed effectiveness in patients with suboptimal/inadequate response or treatment failure to ustekinumab, aligning with the current treatment goals of complete or near‐complete skin clearance. These real‐world results corroborate clinical trial data, emphasizing risankizumab’s potential as a powerful therapeutic alternative for this patient population. Further prospective studies are essential to validate these findings.

Current treatment goals are achieved by the majority of patients with atopic dermatitis treated with tralokinumab: results from a multicentric, multinational, retrospective, cohort study

ABSTRACT Background 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. Research design and methods 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. Results 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. Conclusions 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.

The Lack of Tolerable Treatments Options That Can Induce Durable Responses without Fear of Relapse after Discontinuation Represents a Significant Unmet Need for Patients (Pts) with Immune Thrombocytopenia (ITP): Results from the ITP World Impact Survey (I-WISh) 2.0

Background: ITP is an acquired autoimmune disease characterized by transient or persistent thrombocytopenia. ITP substantially impairs pts' quality of life (QoL), including daily activities, social interactions, work and emotional wellbeing, with fatigue being the most common symptom reported by pts. Corticosteroids are the standard first-line treatment for ITP, while thrombopoietin receptor agonists (TPO-RAs), rituximab and fostamatinib are recommended for later lines; however, these treatments have limited sustained efficacy and may be associated with short- and long-term side effects that impact QoL. Aim: To assess pt and physician (MD) perceptions of current ITP treatment and treatment goals using data from I-WISh 2.0. Methods: I-WISh 2.0 was developed using findings from I-WISh 1.0 and surveyed 1018 pts with ITP aged &amp;gt;18 years and 431 MDs who managed ≥3 pts with ITP. I-WISh 2.0 was conducted from February to July 2022 in 15 countries. The surveys, developed by a steering committee (supported by Novartis) comprising expert ITP MDs and pt advocates, included questionnaires about prescribed ITP treatments, their impact on QoL and treatment satisfaction and goals. Results: Of 1018 pts (median age: 46 years), 67% were female and 48% reported ≥1 comorbidity. 97% (956/987) of pts had received ≥1 treatment for ITP and 12% (126/1018) had a splenectomy; 80% (811/1018) were receiving ITP treatment at survey completion and median time on most recent treatment was 30 weeks (n=608). Corticosteroids and TPO-RAs were the most common first- and second-line treatments prescribed by MDs, respectively (Figure 1); MDs estimated that 15% of their pts have opted against receiving ITP treatment (n=431). 73% of MDs (313/431) considered their pts' perception of medications when making treatment decisions. Pts reported a significant burden due to their current ITP medication: 34% (202/597) experienced daily disruption, 37% (214/576) were bothered by needing to plan meals, and 22% (204/939) had difficulty keeping up with daily medications. 19% (172/897) of pts were dissatisfied with their treatment's platelet level control, and 18% (155/843) disagreed that their medication effectively controlled their symptoms. Pts worried about their treatment's short- (48%; 400/833) and long-term (62%; 527/844) side effects. 38% (162/431) of MDs were neutral toward current ITP treatment options and 9% (37/431) were dissatisfied. Of the 37 dissatisfied MDs, limited options (68%), inability to achieve deep/stable remission (54%), lack of efficacy (43%) and inability to reach treatment goals (43%) were the most common reasons. 32% (327/1016) and 13% (127/1016) of pts believed they were not or did not know if they were in sustained remission, respectively. A treatment offering a sustained remission or cure for ITP was an important goal for 82% (823/1007) of pts. Of treatment goals mentioned in their top 3, the most common were healthy blood counts (56%), improving QoL (44%) and increasing energy levels (43%) for pts (n=1018), and reducing spontaneous bleeds (79%), healthy blood counts (60%) and improving QoL (52%) for MDs (n=431). Limiting time on treatment would substantially affect the treatment preference of 66% (668/1009) of pts. 38% (294/775) of pts did not want to take their ITP treatment for the foreseeable future; however, 67% (680/1013) were concerned about relapse if they stopped. Immunosuppression due to ITP treatment worried 414/787 (53%) pts, with 679/1007 (67%) considering it important when discussing treatment decisions. MDs considered corticosteroids and other immunosuppressants to have the most negative impact on fatigue (Figure 2), yet only 12% (52/431) strongly agreed they aimed to limit immunosuppressive effects. Conclusions: Pts with ITP are dissatisfied with current treatment options because of a perceived lack of efficacy, the burden of short- and long-term side effects, and the need for daily, life-long administration. Additionally, some commonly prescribed treatments for ITP are associated with fatigue. Pts and MDs would like new ITP treatments to offer a sustained period of remission or cure, and pts wish to be able to discontinue them without fear of relapse. There is an unmet need for well-tolerated therapies with disease-modifying potential that can safely achieve these goals.

Abstract P6-09-01: Treatment goals and quality of life (QOL) in a survey of patients with ER+/HER2- metastatic breast cancer (mBC)

Abstract Introduction: In patients (pts) with ER+/HER2- mBC, insights into the foremost concerns regarding their mBC treatment goals and QOL are often assumed by providers but are vastly understudied. The objectives of this survey were to better comprehend treatment goals and QOL concerns in pts with ER+/HER2- mBC. Methods: The 42-question, online EQUALS (ESR1 QUAlity of Life Survey) survey was sent to US subjects in June 2022 from 1) the Cure Media Group (n=6,625) by email, 2) private Facebook groups of pts with mBC and 3) members of a BC clinic. Subjects were eligible if they had ER+/HER2- mBC. A $10 gift card was obtained at survey completion. Survey answers were summarized descriptively. Results: 213 pts completed the survey. Respondents had a mean age of 57 y (range, 31–83 y), and were mostly white (91%), living in an urban/suburban setting (75%), with a higher education degree (71%) and household income ≥$75k (53%). Mean year of mBC diagnosis was 2018 (range, 1995–2022). Most common first-line mBC treatments were aromatase inhibitor (AI) + CDK4/6 inhibitor (CDK4/6i; 44%), AI alone (18%), or fulvestrant + CDK4/6i (16%);54% had received chemotherapy in the metastatic setting. Pts most frequently received information about new mBC treatments from other people living with mBC (42%), followed by physicians (34%), social media (31%), or medical journals/conferences (28%). Two-thirds of pts (64%) reported good/very good QOL, with 12% reporting poor/very poor QOL. Common side effects mostly/moderately impacting QOL were: fatigue (74%), joint pain (64%), vaginal atrophy/dryness (56%), and vasomotor symptoms (47%). Most (84%) were comfortable/very comfortable discussing side effects with their medical team (MT). Worry about disease progression occurred often: everyday (38%), a few times a week (21%) or month (18%), or only before scans (15%). Upon progression, pts worried more about efficacy of new treatment (76%) and having additional options (70%) than they did about side effects (33%). Pts’ current treatment goals were: control cancer growth/spread (93%), prolong life (82%), maintain QOL (81%), tolerate side effects (61%), and relieve suffering/pain (57%); similar to their goals at diagnosis. Almost two-thirds of MTs addressed these goals at the beginning of treatment (63%) and continued annually (60%). Most pts (70%) were very concerned that their mBC diagnosis impacted their family although 81% felt supported at home. Since diagnosis, major/moderate life impacts were: side effects (82%), mental health/stress (78%), QOL (71%), inability to engage in activities (62%), and finances (61%). Most pts (64%) thought their mBC or treatment impacted their intimate/sexual relationship negatively and half (50%) worried about sexual intimacy. Only 44% of pts were comfortable discussing intimacy/sexual side effects with their MT. More pts were comfortable/very comfortable discussing sexual side effects with their MT if their oncologists were female (64%) vs male (51%), BC (73%) vs general (45%), or academic (70%) vs community hospital (52%) or office-based practice (49%). Most (92%) were concerned that their treatments may have a negative impact on their bones. Conclusion: In this survey of pts with ER+/HER2- mBC, &amp;gt;70% received information about new mBC treatments from other pts or social media vs physicians. Pts’ primary concerns were disease control and treatment options, although treatment side effects had the most impact on QOL. Mental health/stress, intimacy and relationships, and bone health were also impacted. Respondents to online surveys in mBC may portray non-representative populations lacking diversity and attempts to diversify future research are much needed, and further efforts are ongoing to address this knowledge gap. Citation Format: Sarah L. Sammons, Jane Meisel, Kelly Shanahan, Timothy Pluard, Monica Kozlowski, Dominic Carroll, Elizabeth Attias. Treatment goals and quality of life (QOL) in a survey of patients with ER+/HER2- metastatic breast cancer (mBC) [abstract]. In: Proceedings of the 2022 San Antonio Breast Cancer Symposium; 2022 Dec 6-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2023;83(5 Suppl):Abstract nr P6-09-01.

National Trends in the Achievement of Recommended Strategies for Stroke Prevention in U.S. Adults With Type 2 Diabetes, 2001-2018.

To assess the national prevalence of and trends in achieving current guideline-recommended treatment goals and pharmacotherapies for primary and secondary prevention of stroke among U.S. adults with type 2 diabetes (T2D). We performed serial cross-sectional analyses in 4,834 adults aged ≥45 years with T2D who participated in the 2001-2018 National Health and Nutrition Examination Survey. With stratification by stroke history, we estimated the proportion of adults with T2D who achieved current guideline-recommended strategies for stroke prevention. Preventive strategies for stroke were benchmarked against diabetes care and cardiovascular risk reduction guidelines. Overall in 2001-2018, among those without stroke history, the proportion who achieved primary stroke prevention strategies ranged from 8.4% (95% CI 6.8-10.1) for aspirin/clopidogrel treatment in those with a higher cardiovascular disease risk to 80.5% (78.8-82.2) for nonsmoking. Among those with stroke history, the proportion who achieved secondary stroke prevention strategies ranged from 11.8% (8.7-14.8) for weight control to 80.0% (74.9-84.9) for glycemic control. From 2001 to 2018, among those without stroke history, there was a significant increase in statin therapy (Ptrend < 0.0001), smoking abstinence (Ptrend = 0.01), and ACE inhibitor/angiotensin receptor blocker treatment for hypertension (Ptrend = 0.04) but a substantial decline in weight control (Ptrend < 0.001). Among those with stroke history, only statin therapy (Ptrend = 0.01) increased significantly over time. During 2001-2018, the achievement of some current guideline-recommended strategies for stroke prevention among U.S. adults with T2D improved but remains a challenge overall. Efforts are needed to improve implementation of strategies for stroke prevention in this population.

CO120 Patients With Chronic Migraine Improving to Fewer than 4 Monthly Headache Days Is an Effective Treatment Goal for Chronic Migraine

Current therapeutic goals for migraine treatment include ≥50% or ≥75% reduction in monthly migraine days (ie, responder rates). Monthly headache days (MHDs) may be a more straightforward assessment tool for the treatment of patients with CM. The objective of this analysis was to determine a threshold number of MHDs that clinicians can use as a therapeutic goal for patients with chronic migraine (CM).

Mucosal healing with methotrexate versus azathioprine treatment in pediatric Crohn's disease as reflected by fecal calprotectin.

The current treatment goal for inflammatory bowel disease (IBD) is achievement of mucosal healing (MH). While established with biologic or azathioprine (AZA) therapies, the data on MH with methotrexate (MTX( treatment is scarce. We aimed to compare MH rate as reflected by FC in children with Crohn's disease (CD) treated with either MTX or thiopurines monotherapy. A cross-sectional multicenter study including children with CD (<18 years), with documented mucosal ulcerations/erosions on their first endoscopy, who were in clinical and biochemical remission for at least 6 months on MTX or AZA/6-MP monotherapy and had fecal calprotectin (FC) measurements during remission. Clinical remission was defined as PCDAI<10 and normal C-reactive-protein (CRP) level. FC < 100 μg/gr was used as a marker of MH. Demographic, clinical and laboratory data were retrieved from the medical charts. 64 patients (41 males, age 16.6±4.2 years) were included; 36 with MTX, 26 with AZA and 2 with 6-MP treatment. The mean treatment dose was 14.0±1.8 mg/m2 for MTX, and 1.8±0.66 mg/kg for AZA, and mean therapy duration was 22 ±17.1 months. MH (FC < 100 μg/gr) was demonstrated in 14/36 (39%) and 18/28 (64%) of patients on MTX and AZA/6-MP therapy, respectively (p=0.04). Rates of FC < 300 μg/gr were comparable [27/36 (75%) MTX, 24/28 (86%) AZA/6-MP, p=0.29]. MH was associated with longer treatment duration (p=0.03). MH as reflected by FC < 100 μg/gr, was higher with AZA/6-MP compared to MTX treatment in pediatric CD.

P183 A systematic review on outcome measures of long-term efficacy in clinical trials concerning Crohn’s disease patients

Abstract Background Treatment goals for Crohn’s disease (CD) tend to shift from clinical towards endoscopic remission as a potential means to reduce intestinal damage. Additionally, sustained corticosteroid (CS) free remission is warranted to prevent drug related side effects. In clinical trials, long-term efficacy of treatment is assessed by a range of outcome measures at variable time points. Besides clinical activity, it is recommended by the EMA to measure biochemical and endoscopic activity, given the poor correlation between commonly applied clinical activity indices and mucosal inflammation. Finally, STRIDE advices to add a PROM given the frequent perception gap in disease control between patient and health care professionals. Moreover, the relapsing-remitting nature of CD challenges timing of efficacy evaluations. Use of cross-sectional outcomes only on predetermined moments disregards the health status in between these measurements. In this systematic review, we provide an overview of the outcomes employed to assess long-term efficacy of maintenance (drug) therapy or treatment strategies in randomized controlled trials and their open label extensions in CD patients. Methods A systematic search of Pubmed and Embase databases was performed. Giving the emerging treatment goals, the search was restricted to trials published since 2007. Two independent reviewers screened titles and abstracts and selected eligible trials. We assessed whether and when long-term (i.e. &amp;gt;48 weeks) clinical, biochemical and/or endoscopic efficacy outcomes were used. In addition, data on patient perspective and CS use were collected. Results Sixty-seven out of 1947 articles were included (figure 1). Clinical activity indices were used in 66 studies (99%) as long-term efficacy outcome, with 20 studies (30%) taking into account concomitant CS use. C-reactive protein concentration was (part of) an outcome in 29 studies (43%), and faecal calprotectin in 15 studies (22%). Endoscopic activity was evaluated in 30 studies (45%). PROMs were used in 29 studies (43%), with IBDQ being the most frequently used. Seven studies measure long-term clinical, biochemical, endoscopic activity and patient perspective, in three studies a sustained CS free status as part of the outcome was required (table 1). In most studies cross-sectional measures or multiple measurements over time, so-called panel data, were used. Conclusion We established a lack of objective outcome measures of inflammation and patient perspective in clinical trials in CD, consequently missing pivotal data to evaluate current treatment goals. Additionally, cross-sectional outcomes on fixed time points were mostly applied, resulting in loss of efficacy information in chronic relapsing-remitting diseases.

Aiming for Functional Cure With Established and Novel Therapies for Chronic Hepatitis B.

Chronic hepatitis B virus (HBV) infection remains difficult to cure due to the persistent, self‐replenishing nature of the viral genome and impaired host immune responses. Current treatment goals for chronic hepatitis B (CHB) are to prevent or significantly delay liver‐related adverse outcomes and death, and two types of treatments are available: nucleos(t)ide analogues (NAs) and interferons (IFNs). NAs effectively suppress HBV replication, and IFNs improve serological response rates, thereby decreasing the risk of adverse outcomes. However, their efficacy in attaining serological responses, especially functional cure (i.e., loss of serum hepatitis B surface antigen), is very limited. Various strategies such as stopping antiviral therapy or combining therapies have been investigated to enhance response, but efficacy is only modestly improved. Importantly, the development of novel direct‐acting antivirals and immunomodulators is underway to improve treatment efficacy and enhance rates of functional cure. The present review provides an overview of the treatment goals and indications, the possibility of expanding indications, and the safety and efficacy of different treatment strategies involving established and/or novel therapies as we continue our search for a cure.

Gastrointestinal Manifestations of CML: A Systematic Review

IntroductionChronic myeloid leukemia (CML) is a myeloproliferative neoplasm characterized by overproduction of mature granulocytes. Up to half of the patients are asymptomatic and diagnosed during routine blood investigations; others present with fatigue and non-specific symptoms. Many patients develop gastrointestinal manifestations such as abdominal pain, bloody diarrhea, and pancreatitis during the disease course. Some presentations are related to leukemia itself, while others may be related to CML treatment.MethodsWe searched the English literature (PubMed, SCOPUS, and Google Scholar) for studies, reviews, case series, and case reports of patients with CML who developed any gastrointestinal manifestations involving the gastrointestinal tract from the esophagus down to the rectum Inclusion criteria comprised of patients above 18 years of age, with CML and gastrointestinal features. Pregnant women and bone marrow transplant recipients were excluded. Search terms included chronic myeloid leukemia, chronic myelogenous leukemia, with esophagitis, pancreatitis, duodenitis, gastritis, Crohn's disease, ulcerative colitis, inflammatory bowel disease, hepatocellular carcinoma, cholangiocarcinoma, colon cancer, malignancy, hepatitis, primary biliary cholangitis, primary biliary cirrhosis, primary sclerosing cholangitis, and perforation.ResultsA total of 129 patients were included. Patient characteristics are shown in table 1. Among the gastrointestinal manifestations, the most common treatment-related complications were drug-related hepatitis followed by reactivation of viral hepatitis B, pancreatitis, and typhlitis. Hepatitis in CML was reported with different TKIs but more commonly with imatinib. Reactivation of viral hepatitis B was common, while hepatitis C reactivation was rarely reported. Pancreatitis was associated mostly with nilotinib. Colitis is seen mainly with dasatinib. Inflammatory bowel diseases, liver diseases such as primary biliary cholangitis (PBC), were variable; some occurred after CML diagnosis while others preceded the diagnosis. Malignancies like pancreatic adenocarcinoma and hepatocellular carcinoma occurred after CML.DiscussionGastrointestinal features in patients with chronic myeloid leukemia can be the first presenting featuring of leukemia itself, arising during the course of CML or as a complication of the treatment. Interestingly, most of these presentations have been reported in patients with CML. These include inflammatory conditions such as pancreatitis and esophagitis, reactivation of viral hepatitis to the neoplastic process, and malignancy. In patients with CML, malignant tumors in the gastrointestinal tract can be caused by leukemic infiltration. Moreover, like other myeloproliferative neoplasms, CML confers a risk of developing a second non-hematological malignancy, including colonic neoplasms.Gastrointestinal complications can pose drastic impacts throughout the disease course; they may result in a change in the treatment, affect the prognosis, and may also be fatal, as in severe enterocolitis or fulminant liver failure. The treatment goal in patients with CML has changed significantly over the last decades. The current treatment goal is to achieve normal survival and good quality of life without the need for lifelong treatment. The improvement in CML treatment and prognosis is largely attributed to the introduction of tyrosine kinase inhibitors. However, most gastrointestinal features associated with treatment are related to tyrosine kinase. The exact pathogenesis of TKI injury is unclear but likely attributed to immune-related mechanisms. Imatinib is the first-line therapy for CML and is the most widely used TKI; however, not all the gastrointestinal features are associated with imatinib as expected. Various gastrointestinal features are prominent with other TKIs as well. Appropriately identifying which TKI is the likely trigger will help in avoiding highly suspected gastrointestinal complications or guide in switching to a safer TKI, thereby achieving treatment goals.ConclusionPatients with chronic myeloid leukemia can have a different gastrointestinal presentation which can alter their disease course. Such complications must be managed appropriately in order to improve outcome and quality of life in this group of patients and maintain treatment goals. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.

Calcitriol and Levothyroxine Dosing for Patients With Pseudohypoparathyroidism.

Pseudohypoparathyroidism (PHP) is a rare hormone resistance syndrome caused by mutations in GNAS. This cross-sectional study investigated whether PHP patients with parathyroid hormone (PTH), thyrotropin (thyroid stimulating hormone; TSH), and free thyroxine (T4) levels at goal required higher doses of levothyroxine and calcitriol than recommended by current guidelines to overcome mineral ion abnormalities due to hormone resistance.Baseline demographic and clinical data of participants enrolled in PHP research studies between 2012-2021 were collected via retrospective chart review. Longitudinally, data were recorded at a maximum frequency of once a year starting at 1 year of age. The PTH at goal (PAG) group was defined as PTH < 150 pg/mL and calcium ≥ 8.4 mg/dL, and the TSH and free T4 at goal (TAG) group was defined as TSH < 5 mIU/L and free T4 ≥ 0.8 ng/dL.The PAG group (n = 74) was prescribed higher calcitriol doses than the PTH not at goal (PNAG) group (n = 50) (0.9 ± 1.1 vs 0.5 ± 0.9 mcg/day, P = 0.04) and 21% of individual patients were prescribed ≥ 1.5 mcg of calcitriol daily. This remained true after normalization for body weight (0.013 ± 0.015 vs 0.0067 ± 0.0095 mcg/kg/day, P = 0.008). There was no statistically significant difference in levothyroxine dosing between the TAG group (n = 122) and TSH and free T4 not at goal (TNAG) group (n = 45) when normalized for weight (2.0 ± 0.7 vs 1.8 ± 0.7 mcg/kg/day, P = 0.2).More than one-third of patients with PHP had PTH levels not at goal and some patients required calcitriol doses ≥ 1.5 mcg/day to meet current treatment goals.

Glucose-lowering therapy in patients undergoing percutaneous coronary intervention.

The number of individuals with diabetes and pre-diabetes is constantly increasing. These conditions are overrepresented in patients undergoing percutaneous coronary intervention and are associated with adverse prognosis. Optimal glycaemic control during an acute coronary syndrome is a relevant factor for the improvement of longer-term outcomes. In addition, the implementation of newer glucose-lowering drugs with proven cardiovascular benefits has a remarkable impact on recurrence of events, hospitalisations for heart failure and mortality. In this narrative review, we outline the current state-of-the art recommendations for glucose-lowering therapy in patients with diabetes undergoing coronary intervention. In addition, we discuss the most recent evidence-based indications for revascularisation in patients with diabetes as well as the targets for glycaemic control post revascularisation. Current treatment goals for concomitant risk factor control are also addressed. Lastly, we acknowledge the presence of knowledge gaps in need of future research.

Clinical practice guidance for childhood-onset systemic lupus erythematosus-secondary publication.

Childhood-onset systemic lupus erythematosus (cSLE) has been recognised as a more acute and severe autoimmune disease than adult-onset SLE. With the development of medications for the disease and supportive therapy, the mortality rate associated with cSLE has drastically improved; the 10-year survival rate among patients with cSLE between 1995 and 2006 in Japan was 98.3%. However, the 10-year survival rate without any permanent functional impairment remained low at 66.1%. Therefore, the current treatment goal for cSLE is to ensure that they can perform normal daily activities throughout their lives by preventing the occurrence and/or progression of organ damage. For this purpose, appropriate treatments and evaluations are required according to the severity and risk of organ damage; however, there are no established guidelines for cSLE. Therefore, the Pediatric Rheumatology Association of Japan and the Pediatric Rheumatology Subcommittee in the Japan College of Rheumatology developed a comprehensive guidance for clinical practice based on cSLE-related data collected from Japanese national surveys and relevant articles from both domestic and international sources. However, due to the lack of indications for defined and objective evidence quality levels, this guidance should be used on the basis of the judgement of the attending physicians for individual patients.

P271 Evolving Targets in Ulcerative colitis: Defining Disease Clearance in the VARSITY Study

Abstract Background In VARSITY, the first head-to-head randomized controlled trial of biologics, vedolizumab (VDZ) was superior vs adalimumab (ADA) in achieving clinical remission (CR) at Week 52 in patients (pts) with moderate to severe ulcerative colitis (UC).1 Current treatment goals in UC are based on clinical symptoms and endoscopy, with histologic improvement considered an aspirational goal.2-4 In pts with CR in VARSITY, greater treatment differences between VDZ and ADA at Week 52 were seen in endoscopic improvement and histologic response. Here, data from VARSITY were used to define disease clearance: a novel composite outcome in UC comprising clinical and endoscopic remission plus minimal histologic disease activity. Methods VARSITY was a phase 3b, randomized, double-blind, double-dummy, active-control trial of treatment with VDZ (300mg IV Q8W) vs ADA (40mg SC Q2W) maintenance after induction. Pts were randomized in a treat through design, and no dose escalation was allowed. For these analyses, based on pts who completed the study until Week 52, disease clearance was defined as a composite outcome based on 1) CR: partial Mayo score ≤2 and no individual subscore &amp;gt;1 (excluding sigmoidoscopy subscore); 2) endoscopic improvement: endoscopic subscore ≤1; and 3) absence of active histologic disease (minimum histological disease activity [MHDA]): Robarts Histology Index (RHI) &amp;lt;5. Treatment failure was defined as discontinuation from treatment or completion of treatment but failure to achieve any of CR, endoscopic improvement, or MHDA. The rates of disease clearance and failures were compared between treatment groups and stratified by presence of inflammatory burden at baseline: C-reactive protein ≥5 mg/L and fecal calprotectin &amp;gt;100 µg/g. Results More pts in the VDZ treatment group than the ADA group achieved disease clearance at Week 52 (VDZ: 112/383, 29.2%, 95% CI 24.7-34.1 vs ADA: 63/386, 16.3%, 95% CI 12.8-20.4). Compared with treatment failures (VDZ: 92/383, 24.0% vs ADA: 134/386, 34.7%, Table 1), disease clearers had lower C-reactive protein (CRP) baseline mean values (7.4mg/L and 6.4mg/L vs 13.6mg/L and 9.4mg/L for disease clearers vs treatment failures, for VDZ and ADA, respectively). Fewer pts with inflammatory burden at baseline (CRP ≥5 mg/L and fecal calprotectin &amp;gt;100 µg/g) achieved disease clearance (Fig 1). Baseline corticosteroid use was more frequent in ADA disease clearers (42.9%) compared with VDZ disease clearers (32.1%). Conclusion Disease clearance is a novel and achievable composite outcome that is achieved by almost one third of pts in the VDZ treatment group in VARSITY. Analyses are ongoing to further characterize the trajectory leading to disease clearance as well as the role of baseline characteristics.

Pediatric Pleomorphic Xanthoastrocytoma: A National Database Inquiry on Current Treatment Approaches in the United States.

BackgroundPleomorphic xanthoastrocytomas (PXAs) account for <1% of primary brain tumors, occurring predominantly in children and young adults. Surgical resection serves as the primary treatment for PXAs, while radiotherapy (RT) and chemotherapy protocols remain poorly defined.AimThis study aims to determine current care patterns utilized for pediatric patients (≤ 18 years) diagnosed with PXAs and their effect on overall survival.MethodsThe United States National Cancer Database (NCDB) was queried between 2004 and 2015 for pediatric patients (≤18 years) diagnosed with PXAs.ResultsFrom the 224 qualifying patients, most patients proceeded with surgery only (78.1%), while 11.6% of patients received both adjuvant RT and chemotherapy. In the 2010‐2015 cohort, patients with subtotal resection were associated with poorer prognosis than those with gross‐total resection (hazard ratio = 17.44, 95% confidence interval = 2.10‐144.90, p < .001). RT and chemotherapy recipients were similarly associated with poorer survival than those treated with surgery only, with p‐values of <.001 and respective hazard ratios of 3.82 (95% confidence interval = 1.85‐7.90) and 6.68 (95% confidence interval = 3.21‐13.89). The key factors impacting the probability of RT delivery involved WHO grade (p < .001) and chemotherapy administration (p < .001). However, WHO grade alone did not significantly impact survival (p‐value = .088).ConclusionMaximally safe resection is the current treatment goal for patients with PXAs. RT and chemotherapy are poorly utilized but had a greater role in managing more aggressive cases of PXAs. Additional research focusing on the impact of adjuvant therapies on tumor progression is needed to better guide treatment decisions.

CORR Insights®: People Prefer to Continue with Painful Activities Even if They Lead to Earlier Surgery.

CORR Insights®: People Prefer to Continue with Painful Activities Even if They Lead to Earlier Surgery.

The impact of dose on the real-world effectiveness of vortioxetine in outpatients with mdd in greece

IntroductionThe current treatment goal in Major Depressive Disorder (MDD) is functional recovery (Zimmerman M et al, 2012). However, finding the “right dose for the right patient” may be challenging and the dose-response relationship for antidepressant efficacy remains controversial (Hieronymus F et al, 2016). Efficacy evaluated by MADRS increases with higher vortioxetine doses, based on meta-analysis data (Thase ME et al, 2016).ObjectivesThe aim of this exploratory analysis was to assess the impact of different doses on vortioxetine effectiveness in clinical practice in Greece.MethodsIn this non-interventional study, open-label vortioxetine was administered at a flexible dosage (5-20 mg/d). Patients receiving 5/10 mg vortioxetine (group A), at the end of the study, were compared to patients receiving 15/20mg vortioxetine (group B). At baseline, 1 and 3 months, depressive symptoms and functioning were assessed by MADRS and SDS. Multiple regression was used for the statistical analyses.ResultsThe study included 336 MDD patients. At the end of the study, 64.3% (n=200) of patients were receiving 15/20 mg vortioxetine. Higher vortioxetine dose at month 3 was significantly correlated with higher MADRS total score at baseline (p&lt;0.001). SDS total score change from baseline to month 3 was significantly associated with vortioxetine dose (p&lt;0.001), with group A and group B showing improvements of -9.2±8.2 and -12.1±6.0, respectively- whereas such association was not observed for MADRS total score.ConclusionsIn conclusion, patients with more severe depressive symptoms were treated with higher antidepressant doses. However, beyond symptom improvement, vortioxetine effectiveness on patient functioning seems to increase with higher doses.Conflict of interestA. Galanopoulos and E. Papalexi are full-time employees in Lundbeck Hellas. A. Ettrup is a full-time employee in H. Lundbeck A/S.

Eosinophilic Esophagitis: Etiology and Therapy.

Eosinophilic esophagitis (EoE) is a relatively recently identified but now frequently encountered antigen/immune-mediated disease which places significant burden on patients and the healthcare system. With its growing prevalence and recognition by healthcare providers in multiple disciplines, substantial progress has been made regarding the diagnostic criteria, clinical evaluation, tools for disease assessment, and immune pathways related to pathogenesis. Current treatment goals focus on the amelioration of inflammation and prevention of remodeling consequences using proton pump inhibitors, swallowed topical steroids, elimination diets, and esophageal dilation. Ongoing research holds promise for more efficacious and targeted therapies as well as a personalized approach to the care of patients with EoE.

Amyloidosis Appointment Companion: A Virtual Healthcare Tool to Optimize Shared Decision Making and Improve Patient Experience and Provider Satisfaction for Telehealth and in-Person Appointments

Systemic amyloidoses are a group of progressive and life-threatening rare diseases. Patients often feel overwhelmed by the complexities of symptoms, treatment options, and management of their care team. The Amyloidosis Appointment Companion (AAC) was created to help patients identify their goals of care, changes in their condition, as well as the challenges they are facing and share these with their health care providers (HCPs). The AAC has been developed to create a synergy between patients and their clinicians as it relates to not only their health and wellbeing but their overall life goals, and to guide discussion and development of their treatment plan. Here we report on the findings of the first 70 patients to use the AAC at the Amyloidosis Center of Boston University Medical Center (BUMC). The Amyloidosis Research Consortium (ARC) in collaboration with Patient Discovery, a virtual healthcare platform, created the AAC, a digital tool to guide patients through a personalized, interactive survey in order to capture their current status, treatment goals and preferences, correct misperceptions, frame decisions, and promote adherence to treatment plans. The tool was beta tested at 5 amyloidosis centers to assess dissemination and clinical integration strategies, decision-making processes, and communication. The tool launched at BUMC in January 2020, after being integrated into the appointment process as part of both in person and virtual telehealth visits via the Amyloid Center Office and included in the appointment scheduling workflow. Data collected via the tool is securely shared with BUMC by patients and entered into their charts. Additional telehealth-specific features were added to the AAC when the BUMC clinic added virtual appointments in response to COVID-19. This is a report of the findings from the first 70 patients utilizing the tool over a five-month time period. There was a completion rate of 82%, well above the industry standard for patient surveys of 41%. The average user age was 65 years (range, 24 to 86), and 41% (n=29) of patients used the AAC on a mobile device. Patients reported the following subtypes of amyloidosis: Patients spent an average of 16.5 minutes using the AAC prior to their appointment and completed the following tasks: Completing Clinic Intake FormsPreparing for Telehealth AppointmentsReconciling MedicationsCapturing Treatment Satisfaction and PreferencesSharing Goals of CareReporting Symptoms and Side EffectsCharting LabsReviewing Educational VideosReporting on Quality of Life Measures Of these 70 patients, 60% (n=42) patients reported slight to moderate levels of anxiety or depression and 69% (n=48) pain and 59% (n=41) restricted mobility. Two main types of reported goals of care were identified: 1) short term, isolated goals (such as attending a specific life event) and 2) long term, lifestyle goals (such as regaining exercise tolerance or living independently). Individualized to their responses, patients prioritized a list of topics to discuss with their clinicians, the most common topics were: 1. Understanding treatment options, 2. Learning more about my disease, 3. Managing my symptoms and side effects, 4. Concern my disease is progressing. Three providers within the Amyloidosis Center at BUMC independently reported enhanced satisfaction with outcome of appointments as a result of patients having used the AAC. The data patients shared led to greater efficiency during telehealth appointments in particular with the time saved on medicine reconciliation and vital signs, allowing more time during the appointment to focus on delivering personalized care. The AAC demonstrated improved patient to physician communication particularly during a time of increased telehealth visits. The AAC is a patient and provider centered tool that facilitates patient communication and involvement in decision making, as well as helping providers understand their patients' preferences and goals of care. The tool improves appointment efficiency in particular, reducing significantly the time taken up in a telehealth visit for medication reconciliation, obtaining vitals and in some cases, technical support. The tool emphasizes patient engagement to improve patient-provider communication. Furthermore, insights from these data provide powerful information that may help improve development of assistance programs, clinical trials, and research. Figure Disclosures Shore: Patient Discovery Solutions, Inc.: Current Employment, Current equity holder in private company. Sodowick:Patient Discovery Solutions, Inc.: Current Employment, Current equity holder in private company. Mendelson:Patient Discovery Solutions: Consultancy. Sanchorawala:Proclara: Other: advisory board; Caleum: Other: advisory board; Regeneron: Other: advisory board; Oncopeptide: Research Funding; Caelum: Research Funding; Prothena: Research Funding; Celgene: Research Funding; Takeda: Research Funding; Janssen: Research Funding; Abbvie: Other: advisory board; UpToDate: Patents &amp; Royalties.