Patient-relevant Benefits Research Articles

Is artificial intelligence for medical professionals serving the patients?

BackgroundAlgorithmic decision-making (ADM) utilises algorithms to collect and process data and develop models to make or support decisions. Advances in artificial intelligence (AI) have led to the development of support systems that can be superior to medical professionals without AI support in certain tasks. However, whether patients can benefit from this remains unclear. The aim of this systematic review is to assess the current evidence on patient-relevant benefits and harms, such as improved survival rates and reduced treatment-related complications, when healthcare professionals use ADM systems (developed using or working with AI) compared to healthcare professionals without AI-related ADM (standard care)—regardless of the clinical issues.MethodsFollowing the PRISMA statement, MEDLINE and PubMed (via PubMed), Embase (via Elsevier) and IEEE Xplore will be searched using English free text terms in title/abstract, Medical Subject Headings (MeSH) terms and Embase Subject Headings (Emtree fields). Additional studies will be identified by contacting authors of included studies and through reference lists of included studies. Grey literature searches will be conducted in Google Scholar. Risk of bias will be assessed by using Cochrane’s RoB 2 for randomised trials and ROBINS-I for non-randomised trials. Transparent reporting of the included studies will be assessed using the CONSORT-AI extension statement. Two researchers will screen, assess and extract from the studies independently, with a third in case of conflicts that cannot be resolved by discussion.DiscussionIt is expected that there will be a substantial shortage of suitable studies that compare healthcare professionals with and without ADM systems concerning patient-relevant endpoints. This can be attributed to the prioritisation of technical quality criteria and, in some cases, clinical parameters over patient-relevant endpoints in the development of study designs. Furthermore, it is anticipated that a significant portion of the identified studies will exhibit relatively poor methodological quality and provide only limited generalisable results.Systematic review registrationThis study is registered within PROSPERO (CRD42023412156).

Pharmacological interventions for preventing upper gastrointestinal bleeding in people admitted to intensive care units: a network meta-analysis

ObjectivesTo assess the efficacy and safety of pharmacological interventions for preventing upper gastrointestinal (GI) bleeding in people admitted to intensive care units (ICUs).Design and settingSystematic review and frequentist network meta-analysis...

Rehabilitation for patients with chronic pruritus

For along time, chronic pruritus was considered difficult to treat. Modern therapy options and detailed guidelines have created new opportunities for patients to improve their quality of life. However, due to the complexity of the disease, the need for multimodal treatment remains. This article aims to investigate whether dermatological rehabilitation offers additional benefits to those affected and whether it should therefore be part of the treatment concept for chronic pruritus. After introduction of the pruritus program of arehabilitation clinic, aprospective study is presented that focuses on the patient-relevant benefits of therapy. It used standardized questionnaires to record pruritus intensity, the presence of depression, anxiety, itch-related limitations, and quality of life in patients with chronic pruritus before and after rehabilitation. Of the patients surveyed, 91.7% achieved apatient-relevant benefit through rehabilitation. Pruritus intensity, depression, anxiety, itch-related limitations, and restrictions on quality of life decreased significantly. Almost half of all participating patients had been initially diagnosed more than 10years ago. Dermatological rehabilitation with amultimodal concept for the treatment of chronic pruritus can help affected patients and should be part of the therapy concept. This should be done as early as possible before the disease progresses over along period of time and makes treatment more difficult.

Point: Center-Based Pulmonary Rehabilitation Is the Standard.

Currently, a major pulmonary rehabilitation focus is on expanding access. At-home rehabilitation is being explored as an in-center pulmonary rehabilitation alternative. It has been asserted that in-home pulmonary rehabilitation confers similar benefits to in-center pulmonary rehabilitation. An extensive database documents that in-center pulmonary rehabilitation confers a range of patient-relevant benefits. Recently, evidence has been presented that in-center pulmonary rehabilitation improves survival, perhaps the most important benefit of all. It can be argued that improvements in physical fitness, assessed as exercise capacity, are mechanistically related to survival improvements. Therefore, in-home rehabilitation must demonstrate exercise capacity improvements similar to those regularly seen in-center to be considered equivalent. A literature search identified 11 studies that compared in-home with in-center pulmonary rehabilitation for COPD that recorded exercise tolerance outcomes. Despite being described as in-home programs, almost all featured prefatory in-center evaluation; some featured in-home visits by rehabilitation professionals. In 6 of the 11 studies, only walking exercise was prescribed. Only 3 included 2-way audio/visual patient-therapist contact. With regard to exercise outcomes; in 3, there was greater in-center group improvement; in 4, outcomes were similar; and, in 4, the in-center group failed to demonstrate clinically important exercise outcome increases; decidedly mixed results. Importantly, in 8 of 11 studies, the 6-min walk test was an exercise outcome. It is argued that the 6-min walk test does not generally elicit physiologically maximum responses and cannot be used to assess exercise capacity improvements. Of the 4 studies that used other exercise outcomes, in 2, exercise endurance increase was similar between in-home and in-center groups; in the other 2, the in-center group had superior improvements. Mixed results indeed! In conclusion, there is insufficient evidence to conclude that in-home pulmonary rehabilitation yields improvements equivalent to center-based programs in physical function, the outcome likely driving long-term prognosis. Moreover, it needs to be established which of the wide variety of in-home program designs now being offered should be promoted.

Avoidable Care Transitions: A Consensus-Based Definition Using a Delphi Technique.

Older adults are at increased risk of frequent transitions between care settings, even though some care transitions are avoidable. The term "avoidable care transitions" is not clearly defined in the research literature. This study aimed to find a consensus-based definition for "avoidable care transitions." This study was conducted as part of the TRANS-SENIOR research network. A 4-round Delphi survey was based on a literature review that identified existing definitions of "avoidable care transitions." Articles in MEDLINE via PubMed and CINAHL were searched. In total 95 references were included, and 106 definitions were identified. Definitions were coded to find themes, resulting in 3 themes with 2 codes for each. In total, 99 experts from 9 countries were invited, and the response rates in Delphi Rounds 1, 2, 3, and 4 were 37.5%, 19.1%, 33.3%, and 23.3%, respectively. Upon reaching the predefined minimum of 90% agreement, the following definition was declared as final: "Avoidable care transitions (a) are without significant patient-relevant benefits or with a risk of harm outweighing patient-relevant benefits and/or (b) are when a comparable health outcome could be achieved in lower resource settings using the resources available in that place/health care system, and/or (c) violate a patient's/informal caregiver's preference or an agreed care plan." Consensus on a definition for "avoidable care transitions" was reached by a multidisciplinary and international panel of experts comprising researchers and providers. The resulting definition consists of 3 distinct dimensions relating to the balance of benefit and harm to a patient, resource consumption, and a patient's or informal caregiver's preferences. The new definition might enhance the common understanding of avoidable care transitions and is now ready for application in research and quality and safety management in health care.

Single-fraction stereotactic radiosurgery versus microsurgical resection for the treatment of vestibular schwannoma: a systematic review and meta-analysis

BackgroundVestibular schwannomas are benign tumours for which various treatments are available. We performed a systematic review of prospective controlled trials comparing the patient-relevant benefits and harms of single-fraction stereotactic radiosurgery (sfSRS) with microsurgical resection (MR) in patients with vestibular schwannoma.MethodsWe searched for randomized controlled trials (RCTs) and non-randomized prospective controlled trials in MEDLINE, Embase, the Cochrane Central Register of Controlled Trials, and study registries (last search: 09/2021) and also screened reference lists of relevant systematic reviews. Manufacturers were asked to provide unpublished data. Eligible studies investigated at least one patient-relevant outcome. We assessed the risk of bias (high or low) at the study and outcome level. If feasible, meta-analyses were performed. We graded the results into different categories (hint, indication, or proof of greater benefit or harm).ResultsWe identified three non-randomized prospective controlled trials of generally low quality with evaluable data on 339 patients with unilateral vestibular schwannoma. There was an indication of greater benefit of sfSRS compared with MR for facial palsy (OR 0.06, 95% CI 0.02–0.21, p < 0.001, 2 studies), hearing function (no pooled estimate available, 2 studies), and length of hospital stay (no pooled estimate available, 2 studies). We found no clinically relevant differences for mortality, vertigo, headaches, tinnitus, balance function, work disability, adverse events, and health-related quality of life.ConclusionsOur systematic review indicates that sfSRS has greater benefits than MR in patients with unilateral vestibular schwannoma. However, it is unclear whether this conclusion still holds after 2 years, as long-term studies are lacking. It is also unclear whether the effects of sfSRS are similar in patients with bilateral vestibular schwannomas. Long-term prospective studies including patients with this condition would therefore be useful.Systematic review registrationThe full (German language) protocol and report (Commission No. N20-03) are available on the institute’s website: www.iqwig.de/en/projects/n20-03.html

EE100 An Approach to Value-Based Pricing for Digital Health Applications in Germany

In December 2019, legislative changes established a fast-track market access and pricing process for digital health applications (DiGAs) in the German statutory health insurance (SHI). Accordingly, after one year of free pricing by manufacturers, DiGA prices must account for the applications’ positive healthcare effects (pVE) in terms of a medical benefit or improvements in care structure and processes. However, the regulations do not indicate how the patient-relevant benefits of a DiGA should be translated into a reasonable, value-based price. Therefore, this study aims to present and discuss an approach for the practical implementation of value-based DiGA pricing.

626-P: TheraKey Diabetes 3.0 App: Better Self-Management, Well-Being, Adherence, and Less Diabetes Distress of People with Diabetes

Aim: The TheraKey Diabetes 3.0 app supports people with diabetes (PwD) during diabetes diagnosis and in the further course of the disease by providing personalized content (e.g., information, graphics, educational videos, testimonials, worksheets, interactive elements) , knowledge and skills for better self-management and coping with the diabetes disease or therapy in everyday life. This study examined the effect in terms of patient-reported outcomes. Methods: Online survey of users of MyTherakey Diabetes 3.0 with a pre-post design at 4 (t1) and 12 weeks (t2) . 185 PwD (15,1% type 1 diabetes (T1D) , 79.5% type 2 diabetes (T2D) , 43,2% female, average age 53.5 years, diabetes duration 3,6 (± 7,2) years, BMI 32,3 (± 7,8) kg/m², 34,6% insulin therapy, HbA1c 8,3 ± 2,5%) . Response rate at t2: 55% (n=105) . Diabetes Self-Management: Diabetes Self-Management Questionnaire (DSMQ) , Problem Areas in Diabetes Scale (PAID-5) . Well-being: WHO-5. Active Patient Participation: Patient Activation Measure (PAM-13D) . Self-efficacy expectancy: General Self-efficacy Scale (GSE) . Health literacy: European Health Literacy Survey (HLS-EU-Q16) . Adherence: MARS D. Results: (t2) : DSMQ: 10,5 ± 10,8/13,3 ± 9,5, p&amp;lt;.0 (t1 p&amp;lt;.001) . WHO-5: 10,5 ± 4,4/11,7 ± 4,1, p=.0 (t1 p= .006) . PAID-5: 33,4 ± 22,5/29,6 ± 21,5, p=.015 (t1 p=.181) . MARS-D: 6,3 ± 1,7/6,8 ± 2,5, p=.022 (t1 p=.432) . PAM-13D: 42,7 ± 4,8/43,4 ± 5,1, p=.136 (t1 p=.426) . GSE: 21,4 ± 5,1/21,8 ± 5,0, p=.297 (t1 p=.167) . HLS-EU-Q16: 13,8 ± 7,4/12,4 ± 8,1, p=.036 (t1 p=.793) . Subgroup analyses show that PwD with short (&amp;lt; 1 year) and long duration of diabetes (&amp;lt; 5 years) and PwD-T2D benefit most. Conclusion: After 3 months of using the diabetes app, patient-relevant benefits can be observed in terms of improved self-management, better well-being, reduced diabetes-related burden, and better adherence. It remains unclear why health literacy is declining. The TheraKey 3.0 can positively support PwD in managing their diabetes. Disclosure B.Kulzer: Advisory Panel; Bayer AG, Bayer AG, Becton, Dickinson and Company, Becton, Dickinson and Company, Berlin-Chemie AG, Berlin-Chemie AG, Dexcom, Inc., Dexcom, Inc., Insulet Corporation, Novo Nordisk, Roche Diabetes Care, Sanofi, Sanofi-Aventis Deutschland GmbH, Research Support; Novo Nordisk, Roche Diabetes Care. D.Ehrmann: Advisory Panel; Dexcom, Inc., Consultant; mySugr, Roche Diabetes Care, Speaker's Bureau; Berlin-Chemie AG, Dexcom, Inc. T.Roos: None. N.Hermanns: Advisory Panel; Abbott Diabetes, Research Support; Becton, Dickinson and Company, Roche Diagnostics, Speaker's Bureau; Berlin-Chemie AG, Novo Nordisk, Sanofi.

Determinants of costs and benefits in atopic dermatitis routine care in Germany

The economic burden of atopic dermatitis (AD) is of particular interest. The present study aims to analyse the association of disease-related characteristics, annual costs and treatment benefits in AD. Between August 2017 and June 2019, a cross-sectional observational study in patients with AD was conducted in Germany. Cost-of-illness data were assessed from the societal perspective. Disease characteristics included severity, time since diagnosis and therapy, as well as atopic comorbidity and the implementation of prevention measures. Subgroup analyses of the total costs were conducted for these characteristics. A linear regression model was applied to analyse the impact of disease characteristics on the costs. Furthermore, associations of biologic treatment with outcome parameters were analysed. A total of 1291 patients from 111 centres were included in the analyses. The total costs amounted on average to € 3660 ± € 6428 per patient and year. Higher costs were shown in various patient groups, for example, in patients using biologics (€ 20 983 vs. € 2470). In a regression analysis, gender, education and the number of implemented prevention measures were identified as significant predictors of costs. Patients treated with biologics showed consistently better outcome parameters and were more often satisfied with their treatment. Gender, education and implemented prevention measures are significant cost determinants in AD. The results confirm that treatment with biologics is the main cost driver in AD. However, incremental patient-relevant benefits of high-priced therapy are reflected by the significantly better clinical outcomes in the group treated with biologics.

The role of inflammatory bowel disease nurses for patients on biologic therapy during the COVID-19 pandemic

Background: During the COVID-19 pandemic, many inflammatory bowel disease (IBD) centres had to reorganise their work, and multidisciplinary teams, including IBD nurses, have had to try to deliver services to patients as safely as possible. Aims: This survey aimed to assess the thoughts, opinions and feelings of IBD patients on biologics regarding the efforts of IBD nurses to organise care during the COVID-19 pandemic. Methods: An evaluation survey based on an anonymous questionnaire was distributed to IBD nurses in five Italian IBD centres, in parallel. Findings: Of 306 participants, 55.6% had Crohn's disease and 44.4% had ulcerative colitis. Most (79.1%) were aware of the specialist IBD nurse role, and 75.8% could distinguish it from generalist nurses. Of patients, 99.7% felt IBD nurses were competent to care for their condition and 91.2% felt that IBD nurses could provide valid scientific information. Meanwhile, 81.4% had a good or excellent relationship with their IBD nurse. Patients reported that most nurses wore personal protective equipment (94.4%), promoted self-care (91.8%), asked about presence of fever or cough (84.3%), asked about patient health status (83.7%) and covered pandemic safety and social distancing rules (69.3%), but fewer spoke about patient mood (44.4%) or relevant benefits, rules and exemptions (26.8%). Most participants (77.8%) faced pandemic-related issues for infusions. Nearly all (97.4%) knew immunosuppressed people were vulnerable to COVID-19, but only 42.8% spoke to their nurse about it. Some 61.8% of patients were concerned about COVID-19, but only 39.2% raised this with the IBD nurse. However, many more patients spoke to a nurse about concerns regarding biologics (41.8%) and worsening IBD symptoms (46.4%) in the pandemic than the actual number experiencing these issues (13.7% and 4.3%, respectively). Conclusion: The results show that IBD patients appreciate the role of the specialist nurse in their care.

Cerebral Oximetry in Preterm Infants-To Use or Not to Use, That Is the Question.

The Safeguarding the Brains of our smallest Children (SafeBoosC) project was initially established to test the patient-relevant benefits and harms of cerebral oximetry in extremely preterm infants in the setting of a randomized clinical trial. Extremely preterm infants constitute a small group of patients with a high risk of death or survival with brain injury and subsequent neurodevelopmental disability. Several cerebral oximeters are approved for clinical use, but the use of additional equipment may disturb and thereby possibly harm these vulnerable, immature patients. Thus, the mission statement of the consortium is “do not disturb—unless necessary.” There may also be more tangible risks such as skin breakdown, displacement of tubes and catheters due to more complicated nursing care, and mismanagement of cerebral oxygenation as a physiological variable. Other monitoring modalities have relevance for reducing the risk of hypoxic-ischemic brain injury occurring during acute illness and have found their place in routine clinical care without evidence from randomized clinical trials. In this manuscript, we discuss cerebral oximetry, pulse oximetry, non-invasive electric cardiometry, and invasive monitoring of blood pressure. We discuss the reliability of the measurements, the pathophysiological rationale behind the clinical use, the evidence of benefit and harms, and the costs. By examining similarities and differences, we aim to provide our perspective on the use or non-use of cerebral oximetry in newborn infants during intensive care.

Real-World Experience of Patient-Relevant Benefits and Treatment Satisfaction with Apremilast in Patients with Psoriasis: An Analysis of the APPRECIATE Study.

IntroductionIn the real-world APPRECIATE study (NCT02740218), most patients with psoriasis demonstrated notable improvements on disease severity measures and reported clinically meaningful treatment benefits with apremilast.ObjectiveWe aim to further describe patient-relevant needs and benefits and patient satisfaction with apremilast, including subgroup analyses based on patient characteristics.MethodsAPPRECIATE, a multinational, retrospective, cross-sectional study, enrolled patients with chronic plaque psoriasis who started apremilast according to the European label. Patient Benefit Index (PBI; range 0 (no patient-relevant benefit) to 4 (maximum patient-relevant benefit), global PBI score ≥ 1 indicating minimum patient-relevant benefit and ≥ 3 indicating high benefit) and nine-item Treatment Satisfaction Questionnaire for Medication (TSQM-9; range 0–100) were assessed 6 (± 1) months after apremilast initiation and summarized descriptively. Relationships between global PBI and TSQM-9 assessments were analyzed by Pearson correlations.ResultsOf 480 enrolled patients, 347 (72.3%) had remained on apremilast at 6 (± 1) months; 90.9% (300/330) achieved global PBI score ≥ 1. Mean (standard deviation) global PBI score was 2.8 (1.2). Higher achievement of global PBI score ≥ 3 was observed in patients with no prior treatments (61.1% (22/36)) or prior phototherapy (64.6% (42/65)) versus prior conventional systemic (54.4% (100/184)) or biologic (38.6% (17/44)) treatment. Strong correlations were observed between the global PBI score and the TSQM-9 global satisfaction and effectiveness subscale scores.ConclusionPatients continuing apremilast for 6 (± 1) months in APPRECIATE reported patient-relevant treatment benefits. Findings suggest that receiving apremilast earlier versus later in treatment management is consistent with greater improvements in patient-relevant treatment outcomes.Supplementary InformationThe online version contains supplementary material available at 10.1007/s13555-021-00628-3.

Which PASI Outcome Is Most Relevant to the Patients in Real-World Care?

In psoriasis treatment, there is a high need to define meaningful endpoints and differences from the patient perspective to analyze patient-relevant differences of frequently used outcome methods for psoriasis under real-world conditions. A sample of 3116 patients from the German Psoriasis-Registry PsoBest was analyzed for clinical as well as patient-reported outcomes (PRO) after 3- and 6-month treatment. The parameters PASI, DLQI, and PBI were intercorrelated and related to two anchoring variables: (1) patient satisfaction with treatment and (2) perceived complete clearance. Baseline data were as follows: PASI 10.5 ± 9.1, DLQI 12.4 ± 3.4, and PBI 2.7 ± 0.3. There was an almost linear relationship between “complete patient satisfaction” and the relative differences in PASI in the range from PASI 25 to PASI 90. However, there was no additional benefit between PASI 90 and PASI 100. The same finding resulted from the anchoring variable “perception of complete healing”. When related to DLQI outcomes, relative PASI changes as well as absolute changes and PASI at 3 and 6 months showed relevant differences between the PASI classes 25 to 90 but not between PASI 90 and PASI 100. Under real-world conditions, changes in PASI and DLQI reflect patient-relevant benefits.

Oral corticosteroids for post-infectious cough in adults: study protocol for a double-blind randomized placebo-controlled trial in Swiss family practices (OSPIC trial)

BackgroundCough is a common reason for patients to visit general practices. So-called post-infectious cough is defined as lasting 3 to 8 weeks after an upper respiratory tract infection. It can be disabling in daily activities, with substantial impact on physical and psychosocial health, leading to impaired quality of life and increased health care costs. Recommendations for the management of post-infectious cough in primary care are scarce and incoherent. A systematic review and meta-analysis of randomized clinical trials (RCT) assessing patient-relevant benefits and potential harms of available treatments identified six eligible RCTs assessing different treatment regimens (i.e. inhaled fluticasone propionate, inhaled budesonide, salbutamol plus ipratropium-bromide, montelukast, nociception-opioid-1-receptor agonist, codeine, gelatine). No RCT found clear patient-relevant benefits and most had an unclear or high risk of bias.Post-infectious cough is thought to be mediated by inflammatory processes that are also present in exacerbations of asthma or chronic obstructive pulmonary diseases for which there is strong evidence that oral corticosteroids provide patient-relevant benefit without relevant harm. We therefore plan to conduct the first RCT evaluating the effectiveness of oral corticosteroids for post-infectious cough.MethodsWe are conducting a triple-blinded randomized-controlled and multicentred superiority trial in primary health care practices in Switzerland. We will include 204 adult patients who consult their general practitioner (GP) for a cough lasting 3 to 8 weeks following an upper respiratory tract infection. Participants will be randomly allocated to either the 5-day treatment with oral corticosteroids or placebo. The primary outcome is cough-related quality of life assessed by the Leicester Cough Questionnaire score 14 days after randomization. Secondary outcomes include cough-related quality of life at several time points, overall cessation of cough and adverse events.DiscussionThis RCT will provide evidence on whether oral corticosteroids are beneficial and safe in patients with post-infectious cough. Results can have a substantial impact on the well-being and management of these patients in Switzerland and beyond. An evidence-based treatment for this condition may reduce re-consultations with GPs and spending for antitussive drugs, thus possibly having an impact on health care spending.Trial registrationClinicalTrials.gov NCT04232449. Prospectively registered on 18 January 2020.

Patient-Focused Benefits of at-Home Versus in-Clinic Administration of Cancer Therapy: New Considerations for the COVID-19 Era

Introduction: Cancer treatment includes conventional clinic-based infusions and various options for home administration of medication. Clinic-based treatment can be disruptive and costly to patients and caregivers, requiring transportation, time away from work/family responsibilities, and stressful clinical settings. The COVID-19 pandemic has increased concerns that patients may be exposed-or experience anxiety about exposure-to viruses and other pathogens. Potential benefits to home-based chemotherapy include cost/healthcare resource utilization savings and decreased infection exposure by avoiding clinical settings. In the COVID-19 era, ASH, ASCO, ESMO, and other groups have recommended home administration of chemotherapy, including infusions at home and self-administered oral and subcutaneous treatments, for certain cancer types where feasible. This literature review evaluated patient- and caregiver-relevant advantages of home-administered cancer therapy.Methods: A strategic literature review was conducted using the pearl growing/snowball method, wherein core publications were identified using an initial PubMed search strategy with the MeSH heading “Antineoplastic Agents/Administration & Dosage” and the search terms “Home Care Services” and “Patient Satisfaction.” Results were limited to English-language publications dated January 2000 to July 2020, ≥10 study subjects, adult subjects only, with no limits for geography or cancer type. A total of 21 initial results were retrieved in PubMed. Six core publications were used to establish keywords and for bibliographic and prospective citation searches to identify additional relevant publications relating to patient preference and patient/caregiver-reported outcomes regarding cancer treatment administration settings. In addition to PubMed, the following congresses were searched: ASH (2004-2019), ASCO (2011-2020), EHA (2016-2019) and ESMO (2010-2019).Results: Thirty-one studies involving both hematologic cancers and solid tumors were identified from North and South America, Europe, Asia, and Australia. Cancer therapy was administered by patients/caregivers (ie, oral or subcutaneous) or a visiting nurse. All the studies reviewed reported benefits for home- vs clinic-based treatment. Patient expressed preference/satisfaction and willingness to continue with home-based regimens; while methodologies varied among studies, 70% to 100% of patients preferred home administration. Across studies, over half of patients receiving home treatment cited improvement in outcomes including well-being, activities of daily living, and family/social life, with benefits including convenience, comfort, reduced travel/financial burden, limited waiting time, and greater ability to maintain daily family/social activities. The impact on patient health-related quality of life (HRQoL) could not be compared quantitatively among studies as <25% of the studies used validated tools, e.g., EORTC-QOL-C30. Among studies using validated tools, HRQoL outcomes were generally similar for patients treated at home or in-clinic. There were very few reports of patients needing or choosing to return to clinic-based care after initiating home treatment. Studies that captured safety outcomes did not report increased adverse effects or emergency room visits among patients treated at home. Of the 3 studies reporting caregiver outcomes, most caregivers expressed satisfaction with and preference for home treatment.Conclusions: The prioritization of therapies that can be administered at home has been proposed as a strategy for infection control in the COVID-19 era, but it is not currently a standard approach in the US. This targeted literature review consistently found patient-relevant benefits with home-administered chemotherapy. The identified studies provided minimal information on caregiver-reported outcomes, which is a limitation given that home-based cancer treatment impacts caregivers as well as patients. Home-based treatment may enhance quality of survival time and reduce healthcare resource utilization while maintaining clinical benefits of treatment and reducing contact with people in a busy clinical setting. While treatment decisions should consider patient preference for home-based treatment, some patients' treatment pathways will require care at an outpatient or inpatient facility.DisclosuresSandman:Pfizer Inc.: Consultancy. Bell:Pfizer: Current Employment, Current equity holder in publicly-traded company.

Negative pressure wound therapy in patients with wounds healing by secondary intention: a systematic review and meta-analysis of randomised controlled trials

BackgroundNegative pressure wound therapy (NPWT) is a widely used method of wound treatment. We performed a systematic review of randomised controlled trials (RCTs) comparing the patient-relevant benefits and harms of NPWT with standard wound therapy (SWT) in patients with wounds healing by secondary intention.MethodsWe searched for RCTs in MEDLINE, Embase, the Cochrane Central Register of Controlled Trials, and study registries (last search: July 2018) and screened reference lists of relevant systematic reviews and health technology assessments. Manufacturers and investigators were asked to provide unpublished data. Eligible studies investigated at least one patient-relevant outcome (e.g. wound closure). We assessed publication bias and, if feasible, performed meta-analyses, grading the results into different categories (hint, indication or proof of a greater benefit or harm).ResultsWe identified 48 eligible studies of generally low quality with evaluable data for 4315 patients and 30 eligible studies with missing data for at least 1386 patients. Due to potential publication bias (proportion of inaccessible data, 24%), we downgraded our conclusions. A meta-analysis of all wound healing data showed a significant effect in favour of NPWT (OR 1.56, 95% CI 1.15 to 2.13, p = 0.008). As further analyses of different definitions of wound closure did not contradict that analysis, we inferred an indication of a greater benefit of NPWT. A meta-analysis of hospital stay (in days) showed a significant difference in favour of NPWT (MD − 4.78, 95% CI − 7.79 to − 1.76, p = 0.005). As further analyses of different definitions of hospital stay/readmission did not contradict that analysis, we inferred an indication of a greater benefit of NPWT. There was neither proof (nor indication nor hint) of greater benefit or harm of NPWT for other patient-relevant outcomes such as mortality and adverse events.ConclusionsIn summary, low-quality data indicate a greater benefit of NPWT versus SWT for wound closure in patients with wounds healing by secondary intention. The length of hospital stay is also shortened. The data show no advantages or disadvantages of NPWT for other patient-relevant outcomes. Publication bias is an important problem in studies on NPWT, underlining that all clinical studies need to be fully reported.

Benefit evaluation in multiple sclerosis relapse treatment from the patients’ perspective – Development and validation of a new questionnaire

BackgroundLittle is known on how to measure patient-relevant benefit of relapse treatment in relapsing-remitting multiple sclerosis (MS). The objective of this study was to develop and validate a new method for monitoring recovery from MS relapses and patient-relevant treatment benefits. MethodsA 27-item questionnaire was developed using a multi-step approach comprising open item collection, multidisciplinary expert panel and cognitive debriefing. It was evaluated regarding psychometric properties and feasibility in a longitudinal validation study with 100 patients with MS undergoing relapse treatment. Construct validity was tested by correlations with patient and physician global impressions of change as well as disease-specific and generic health-related quality of life (HRQoL) measures. ResultsResults of the feasibility survey indicated high patient acceptance. Reliability was high (Cronbach's α = 0.90). While the Expanded Disability Status Scale (EDSS) was not sensitive to change, Patient Benefit Index for Multiple Sclerosis (PBI-MS) showed a high correlation cross-sectionally with patient global impression of change (PaGIC) (r = 0.60, p < 0.001). Significant moderate to high correlations were found with change in generic HRQoL (r = 0.55–0.61, p < 0.001) and lower correlations with change in disease-specific HRQoL (r = −0.36, p < 0.01). ConclusionThe PBI-MS is a reliable and valid instrument for ascertaining patient-relevant benefits of acute relapse treatment; it appears suited for use in routine care and in clinical or health care studies.

Treatments for subacute cough in primary care: systematic review and meta-analyses of randomised clinical trials.

Subacute cough following a non-specific viral infection lasting 3-8 weeks is common. However, despite many treatment options there are no systematic reviews evaluating these. To provide a systematic overview of treatment options and outcomes evaluated in randomised clinical trials (RCTs). Systematic review and meta-analyses assessing the overall effects of any treatment for subacute cough. The authors systematically searched PubMed/MEDLINE and the Cochrane Central Register of Controlled Trials (last search March 2017) for RCTs in adult patients with subacute cough. The authors considered trials evaluating any outcome of any drug or non-drug treatments, apart from traditional Chinese and Asian medicines. They combined treatment effects on cough-related outcomes in random effects meta-analyses. Six eligible RCTs including 724 patients were identified. These assessed montelukast, salbutamol plus ipratropium bromide, gelatine, fluticasone propionate, budesonide, and nociception opioid 1 receptor agonist and codeine. Five studies reported effects on various cough severity scores at various timepoints. No treatment option was associated with a clear benefit on cough recovery or other patient-relevant outcomes in any of the studies or in meta-analyses for cough outcomes at 14 days and 28 days. Reported adverse events were rather mild and reported for 14% of patients across all treatments. Evidence on treatment options for subacute cough is weak. There is no treatment showing clear patient-relevant benefits in clinical trials.

PCN101 - Key Decision Drivers of German Health Technology Assessment in Non-Small Cell Lung Cancer

Categorization and analysis of positive and negative decision drivers for Health Technology Assessment (HTA) by Gemeinsame Bundesausschuss (G-BA) to understand evidence requirements in Germany. Non-Small Cell Lung Cancer (NSCLC) was chosen for this research because of dramatic changes in the treatment algorithms over the past 5 years. All appraisal results of medicines in NSCLC published by G-BA between Jan 2013 and Feb 2018 were analyzed using primary data from the G-BA website. Additional information was obtained from the global HTA database Evalumade. We found 20 assessments for 12 medicines and 46 subgroups. Fourteen subgroups were given a positive rating (major, considerable or minor added benefit) because of one or more supportive pieces of evidence in the four major domains (mortality, morbidity, QoL and safety). Morbidity benefit contributed to the positive rating in many cases (12 out of 14 subgroups). Thirty-one subgroups received no added benefit and one subgroup less benefit. About one third of them were due to an inappropriate comparator. Other negative drivers were 1) small number of patients in a trial, 2) no relevant data generated, 3) potential bias of the study and/or endpoint, 4) unaccepted historical or indirect comparison and 5) transferability of the data to the German population. We found that generating convincing access evidence in NSCLC for Germany has involved: 1) including sufficient number of patients in a trial to prepare for extensive subgroup analyses or keeping a trial population homogeneous, 2) designing a study to show patient relevant benefits in at least one of the four domains (particularly morbidity and mortality) and 3) designing a trial that includes a patient population that is generalizable to the German population. It remains the responsibility of pharmaceutical companies whether to take into account German access evidence requirements as well as other country requirements in drug development decisions.

Interventions for preventing upper gastrointestinal bleeding in people admitted to intensive care units.

Interventions for preventing upper gastrointestinal bleeding in people admitted to intensive care units.