Patient Related Outcome Measures最新文献

{"title":"Usage and Health Outcomes of Home Hemodialysis vs Center Hemodialysis in Racial/Ethnic Minority Groups in the United States a Quantitative Research in 2016–2019 USRDS Using Aday-Anderson Framework and Multiple Regression Models","authors":"Ying Zhu","doi":"10.2147/prom.s416279","DOIUrl":"https://doi.org/10.2147/prom.s416279","url":null,"abstract":"Introduction Home hemodialysis (HHD) offers patients with end-stage kidney disease (ESKD) greater flexibility and advantages of health outcomes over center hemodialysis (CHD). This study aims to investigate the differences between home and center hemodialysis with a focus on racial/ethnic minorities. Methods The US Renal Disease System (USRDS) 2019 patient core data containing mortality and hospitalization which are cumulative since 2010 were merged with 2016–2019 Medicare clinical claims. To assess demographic and medical factors adjusted utilization and mortality of HHD vs CHD within every racial/ethnic cohort, logistic regression was used, and negative binomial regression was conducted to analyze the number of hospitalizations. Results Evaluating 548,453 (97.48%) CHD patients and 14,202 (2.52%) HHD patients with Whites 47%, Blacks 32%, Hispanics 15%, Asians 4%, and other minorities 2%, the outcomes from adjusted regressions showed that: 1) minorities were significantly less likely to use HHD than Whites (Blacks: OR, 0.568, 95% CI, 0.546–0.592; Hispanics: OR, 0.510, 95% CI, 0.477–0.544; Asians: OR, 0.689, 95% CI, 0.619–0.766; Others: OR, 0.453, 95% CI, 0.390–0.525; p < 0.001); 2) most minority patients were younger and had fewer comorbidities than Whites, and all minority groups displayed significantly lower mortality and hospitalization incidences than the White group with adjustment on multiple covariates; 3) in the overall and main racial/ethnic cohorts, HHD showed a significantly lower risk of death than CHD after confounding for major risk factors (overall cohort: OR, 0.686, 95% CI, 0.641–0.734; White: OR, 0.670, 95% CI, 0.612–0.734; Blacks: OR, 0.717, 95% CI, 0.644–0.799; Hispanics: OR, 0.715, 95% CI, 0.575–0.889; Others: OR, 0.473, 95% CI, 0.265–0.844). Conclusion There are substantial racial/ethnic variations in home hemodialysis use and health outcomes in the United States.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"120 32","pages":"1 - 16"},"PeriodicalIF":2.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139453924","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exploring Effective Communication Strategies Employed by Physicians in Delivering Bad News in Ethiopian State Hospitals.","authors":"Alebel Guangul Gessesse, Jemal Mohammed Haile, Amanuel Gebru Woldearegay","doi":"10.2147/PROM.S390164","DOIUrl":"https://doi.org/10.2147/PROM.S390164","url":null,"abstract":"<p><strong>Introduction: </strong>Delivering Bad News (DBN) presents a highly challenging situation in physician-patient communication. This study aims to gain insight into the various communication strategies employed by physicians when DBN.</p><p><strong>Methods: </strong>This qualitative study conducted thematic analysis of in-depth interviews. Physicians from two comprehensive hospitals with large patient populations were selected purposively based on their engagement in delivering bad news to patients. Thematic analysis was made.</p><p><strong>Results: </strong>Thematic analysis of the data revealed several communication strategies physicians use when delivering bad news. These communication strategies include. Jointly Initiated Physician-Patient Communication Strategies: ((i) Discussing with patient family/caregivers, (ii) Collaborating with other physicians and specialists), Patient-Engaged/Led Communication Strategies: ((iii) Investigating with adolescents alone or without the family, (iv) Helping patients predict what the news is, (v) Identifying patients' emotions related to bad news, (vi) Assessing patients' level of understanding, (vii) Minimizing patient anxiety), Physician-Related Communication Strategies: ((viii) Making sure diagnostic results are accurate, (xi) Identifying causes for rejection, (x) deliveringbad news using clear and simple communication).</p><p><strong>Conclusion: </strong>Delivering bad news to patients can be challenging for physicians. It is important to be clear and accurate, and to prepare patients for the news. Patients may feel more comfortable and open when they are unaccompanied and with their healthcare provider. The study concluded that physicians need to be prepared to deliver bad news in a sensitive and effective manner.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"409-425"},"PeriodicalIF":2.1,"publicationDate":"2023-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10749095/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139032508","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lived Experiences of Patients on Hemodialysis Treatment at Kiruddu National Referral Hospital: A Phenomenological Study.","authors":"Alobo Jennifer Ogwang, Eric Baluku Murungi, Niyonzima Vallence, Beebwa Esther","doi":"10.2147/PROM.S431746","DOIUrl":"https://doi.org/10.2147/PROM.S431746","url":null,"abstract":"<p><strong>Background: </strong>There is increasing number of patients undergoing hemodialysis globally. Patients on hemodialysis experience physical and emotional stress due to the changes brought by chronic kidney disease.</p><p><strong>Aim: </strong>The study aimed at exploring the lived experiences of patients on hemodialysis treatment in Kiruddu National Referral Hospital.</p><p><strong>Methods: </strong>The study employed a phenomenological design. Data was collected using audio tape recording of the interview from 9 participants selected through purposive sampling at Kiruddu National Referral Hospital. The thematic aspects of the lived experience were uncovered using Van Manen data analysis which included three approaches: the detailed or line-by-line approach, selective or highlighting approach and holistic approach.</p><p><strong>Results: </strong>Six themes emerged during the analysis as hemodialysis prolongs survival; hemodialysis is indispensable, family financial support, physical limitations, emotional distress and adaptation.</p><p><strong>Conclusion: </strong>It was concluded from the findings that the patients undergoing haemodialysis are facing a wide range of problems such as physical and emotional problems during the course of their treatment and think that haemodialysis is the only way of survival, and these problems need to be addressed. Understanding gained in this study can help nurses to utilize this information in improving the quality of nursing care and guide patients to provide positive reinforcement for their future living.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"393-408"},"PeriodicalIF":2.1,"publicationDate":"2023-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10725628/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138808374","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Pattern of Admission, Clinical Characteristics, and Outcomes Among Patients Admitted to the Intensive Care Unit of a Tertiary Hospital in Tanzania: A 5-Year Retrospective Review","authors":"Nadeem Kassam, Philip Adebayo, Iris Matei, Eric L. Aghan, Samina S Somji, Samwel Kadelya, Yasson Abha, Frank Swai, Mangaro Mabusi, Kamran Hameed, Hanifa Mbithe, Alyyah Thawer, Mandela Makakala, Fatma Bakshi, Harrison Chuwa, M. Ng'wanasayi, C. Wambura, R. Mvungi, James Orwa, Munish Sharma, G. Udeani, S. Surani","doi":"10.2147/PROM.S441293","DOIUrl":"https://doi.org/10.2147/PROM.S441293","url":null,"abstract":"Introduction Despite the implementation of complex interventions, ICU mortality remains high and more so in developing countries. The demand for critical care in Sub-Saharan Africa is more than ever before as the region experiences a double burden of rising rates of non-communicable diseases (NCD) in the background battle of combating infectious diseases. Limited studies in Tanzania have reported varying factors associated with markedly high rates of ICU mortality. Investigating the burden of ICU care remains crucial in providing insights into the effectiveness and challenges of critical care delivery. Material and Methods A single-center retrospective study that reviewed records of all medically admitted patients admitted to the ICU of the Aga Khan Hospital, Dar-es-Salaam, from 1st October 2018 to 30th April 2023. To define the population in the study, we used descriptive statistics. Patients’ outcomes were categorized based on ICU survival. Binary logistic regression was run (at 95% CI and p-value < 0.05) to identify the determinants for ICU mortality. Results Medical records of 717 patients were reviewed. The cohort was male (n=472,65.8%) and African predominant (n=471,65.7%) with a median age of 58 years (IQR 45.0–71.0). 17.9% of patients did not survive. The highest mortality was noted amongst patients with septic shock (29.3%). The lowest survival was noted amongst patients requiring three organ support (n=12,2.1%). Advanced age (OR 1.02,CI 1.00–1.04), having more than three underlying comorbidities (OR 2.50,CI 1.96–6.60), use of inotropic support (OR 3.58,CI 1.89–6.80) and mechanical ventilation (OR 9.11,CI 4.72–18.11) showed association with increased risk for mortality in ICU. Conclusion The study indicated a much lower ICU mortality rate compared to similar studies conducted in other parts of Sub-Saharan Africa. Advanced age, underlying multiple comorbidities and organ support were associated with ICU mortality. Large multi-center studies are needed to highlight the true burden of critical care illness in Tanzania.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":" 6","pages":"383 - 392"},"PeriodicalIF":2.1,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138612769","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychometric Analysis of the Patient-Reported Hypoparathyroidism Symptom Diary Symptom Subscale Using Data from Two Clinical Trials.","authors":"Lauren Nelson, Steven W Ing, Mishaela R Rubin, Jia Ma, Susan Martin, Rohini Sen, Olulade Ayodele","doi":"10.2147/PROM.S414794","DOIUrl":"10.2147/PROM.S414794","url":null,"abstract":"Purpose The hypoparathyroidism symptom diary (HypoPT-SD) is a disease-specific patient-reported outcome (PRO) tool comprising a 7-item symptom subscale, a 4-item impact subscale and 1-item anxiety, and sadness or depression components. This analysis assessed the psychometric properties of the HypoPT-SD symptom subscale scores using data from two open-label, single arm, Phase 4 studies (Study 402 and Study 404). Patients and Methods Eligible patients were aged 18 years or older with a confirmed diagnosis of hypoparathyroidism. All patients received recombinant human parathyroid hormone (1–84) during the analysis period. Scores were recorded at baseline, and at months 6, 30 and 36 (end of treatment [EOT]) in Study 402, and at baseline and week 52 (EOT) in Study 404. The structure of the HypoPT-SD Symptom subscale was analyzed by measuring correlations between pairs of item scores; internal consistency and reliability were evaluated using Cronbach’s coefficient α; test-retest reliability was assessed using intraclass correlation; and construct validity was determined by performing correlational analyses between scores recorded using the HypoPT-SD and those for other conceptually similar PRO tools. Results A total of 60 patients were included in the analysis. Inter-item pairwise correlations were strong for all but 5 of the item pairs analysed. Cronbach’s α values for the HypoPT-SD Symptom subscale were 0.88 using data from Study 402 and 0.92 using data from Study 404. In general, the HypoPT-SD Symptom subscale scores had moderate or strong correlations with scores recorded using PRO tools. Intraclass correlation coefficients exceeded 0.70 using test–retest data from all patients in Study 402 and from a subgroup of patients with stable disease from Study 404. Conclusion This analysis demonstrated the test-retest reliability, internal consistency and construct validity of the HypoPT-SD using data from longitudinal prospective studies and supports the use of the HypoPT-SD in future clinical studies.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"355-367"},"PeriodicalIF":2.1,"publicationDate":"2023-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10693201/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138478324","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Burden of Sickle Cell Disease on Children and Their Caregivers: Caregiver Reports of Children's Health-Related Quality of Life and School Experiences, Caregiver Burden, and Their Association with Frequency of Vaso-Occlusive Crises.","authors":"Andrew Campbell, Avery A Rizio, Kristen L McCausland, Serban Iorga, Glorian P Yen, Jincy Paulose, Soyon Lee","doi":"10.2147/PROM.S419607","DOIUrl":"10.2147/PROM.S419607","url":null,"abstract":"<p><strong>Background: </strong>Children with sickle cell disease (SCD) experience a multiplex of disease-related symptoms and complications, including vaso-occlusive crises (VOCs), episodes characterized by extreme pain.</p><p><strong>Methods: </strong>A cross-sectional observational survey examined the health-related quality of life (HRQoL) and school experiences of children with SCD 2 months-11 years, burden experienced by their caregivers, and associations between these outcomes and VOC frequency. Caregivers (N=167) of children with SCD in the US completed the Infant-Toddler Quality of Life-Short Form 47 (ITQoL-SF47) for children 2 months-4 years, the Child Health Questionnaire-Parent Form 50 (CHQ-PF50) and PROMIS Pain Interference and Sleep Disturbance Parent Proxy short forms for children 5-11 years, and a study-specific survey of school experiences.</p><p><strong>Results: </strong>Children with SCD 2 months-4 years had lower ITQoL-SF47 scores (ie, worse HRQoL, p<0.001) than a normative sample of children; across domains, differences ranged from 18.73-45.03 points and exceeded minimal important difference (MID) thresholds. Except for the behavior domain, children with SCD 5-11 years had lower scores on all CHQ-PF50 domains than the normative sample (p<0.001); differences ranged from 6.78-36.37 points and exceeded MID thresholds. Children with more frequent VOCs had lower HRQoL and worse school experiences than children with less frequent VOCs (p<0.05, except for behavior domains). The largest differences based on VOC frequency were observed for overall health and bodily pain/discomfort among children 2 months-4 years (differences=40.88 and 32.50 points, respectively), and bodily pain and role/social limitations due to physical health among children 5-11 years (differences=38.99 and 37.80, respectively). Caregivers of children with more frequent VOCs experienced greater burden than caregivers of children with less frequent VOCs, though specific areas of impact (eg, caregiver emotions, time) differed across child age groups.</p><p><strong>Conclusion: </strong>VOC frequency is negatively associated with HRQoL, highlighting the burden experienced by children with SCD and their caregivers.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"369-381"},"PeriodicalIF":2.1,"publicationDate":"2023-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10693247/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138478325","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Qualitative Exploration in Exit Interviews of Changes Observed in Clinical Trials for Individuals with Autism Spectrum Disorder Without Intellectual Disability.","authors":"Michael Chladek, Claire Burbridge, Elizabeth Gibbons, Tom Willgoss, Janice Smith, Susanne Clinch","doi":"10.2147/PROM.S385682","DOIUrl":"https://doi.org/10.2147/PROM.S385682","url":null,"abstract":"<p><strong>Purpose: </strong>To explore, from the perspective of Study Partners (SPs; eg, caregivers) of clinical trial participants with autism spectrum disorder (ASD), any changes experienced in socialization and communication over the clinical trial, how these changes manifested, and the impact these changes had on the autistic individual, the SP, and family. This helps interpret whether changes in trial outcomes were meaningful.</p><p><strong>Patients and methods: </strong>Interviews were conducted with the SPs of individuals with ASD, without intellectual disability, from 2 clinical trials: 86 children (aged 5-12 years) or adolescents (aged 13-17 years) who took part in the aV1ation trial (83.7% male), and 41 adults (aged 18+ years) who took part in the V1aduct trial (80.5% male). The primary endpoint for both trials was change from baseline in the Vineland^TM-II two-domain composite, consisting of the mean of the Socialization and Communication domains. In these interviews the participants verbally indicated level of change for each of these key domains on 7-point change scales.</p><p><strong>Results: </strong>Improvements in the Socialization domain enabled greater awareness of the feelings of others and allowed for stronger empathy and kindness. Improvements in the Communication domain allowed for the autistic individual to be better at listening and better at self-expression. Together, changes in these two domains, which were considered most important, allowed for richer, deeper relationships. Study Partners noted that improvements in these domains allowed for better integration within the family unit, decreased stress, and increased optimism about the autistic individual's future.</p><p><strong>Conclusions: </strong>The impacts of changes in either domain were synergistic, combining together to create positive experiences which in turn led to further positive impacts in other skills. These qualitative insights provide context to the changes that were observed during the clinical trial and captured using the Vineland^TM-II, illustrating the meaning of these changes to the individuals with ASD without intellectual disability and their families, and the impact that they have on people's everyday lives and overall health-related quality of life.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"313-335"},"PeriodicalIF":2.1,"publicationDate":"2023-11-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10676099/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138461409","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Quantitative and Qualitative Exploration of Meaningful Change on the Vineland Adaptive Behavior Scales (Vineland™-II) in Children and Adolescents with Autism Without Intellectual Disability Following Participation in a Clinical Trial.","authors":"Susanne Clinch, Stacie Hudgens, Elizabeth Gibbons, Tom Willgoss, Janice Smith, Ela Polek, Claire Burbridge","doi":"10.2147/PROM.S385542","DOIUrl":"https://doi.org/10.2147/PROM.S385542","url":null,"abstract":"<p><strong>Purpose: </strong>The Vineland^TM Adaptive Behavior Scale is often used in autism spectrum disorder (ASD) trials. The Adaptive Behavior Composite Score (VABS-ABC) is the standardized overall score (the average of the Socialization, Communication and Daily Living skills domains), and the standardized 2-Domain Composite Score (VABS-2DC) is a novel outcome measure (average of the Socialization and Communication domains). A within-person meaningful change threshold (MCT) has not been established for the VABS-2DC. This paper presents a quantitative and qualitative interpretation of what constitutes a meaningful change in these scores to individuals with ASD without Intellectual Disability (ID; IQ≥70) and their families, as reported by their study partners (SPs).</p><p><strong>Participants and methods: </strong>Data were obtained from the aV1ation clinical trial in children and adolescents with ASD and associated exit interviews. The intent-to-treat (ITT) clinical trial population included 308 individuals with autism (85.4% male; average age: 12.4 years [standard deviation (SD)=2.97]); 124 in the child cohort (aged 5 to 12 years; average age: 9.4 years [SD=1.86]), and 184 in the adolescent cohort (aged 13 to 17 years; average age: 14.5 years [SD=1.39]). Study partners of 86 trial participants were included in the Exit Interview Population (EIP): participants represented were 83.7% male, average age: 12.3 years [SD=2.98]). Anchor and distribution-based methods were used to estimate within-person change to support a responder definition, to aid interpretation of the clinical trial data; qualitative data were used to contextualize the meaning of changes observed.</p><p><strong>Results: </strong>A within-person MCT range of 4 to 8 points was proposed for both VABS-ABC and VABS-2DC, which was associated with at least a 1-point improvement on 4 different anchors. Evidence for this within-person MCT was further supported by qualitative data, which suggested any change was considered meaningful to the individual with ASD, as reported by their SP, no matter what the magnitude.</p><p><strong>Conclusion: </strong>A change in standardized score of 4 to 8 points constitutes a within-person MCT on both VABS-ABC and novel VABS-2DC in those with ASD and no ID. A change of this, or more, was reported by the SPs in this trial to be meaningful and highly impactful upon the individuals with ASD and their family.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"337-354"},"PeriodicalIF":2.1,"publicationDate":"2023-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10675867/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138461410","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Ravulizumab on Patient Outcomes and Quality of Life in Generalized Myasthenia Gravis.","authors":"Carlo Antozzi, Renato Mantegazza","doi":"10.2147/PROM.S408175","DOIUrl":"10.2147/PROM.S408175","url":null,"abstract":"<p><p>Myasthenia gravis (MG) is an autoimmune ion channel disorder in which antibodies to different end-plate antigens impair neuromuscular transmission, ultimately leading to muscle weakness and fatigability. In about 85% of patients with MG, autoantibodies against the acetylcholine receptor (AChR) activate the complement cascade, causing damage to the neuromuscular junction. MG is a chronic disorder for which standard therapies with corticosteroids, immunosuppressive drugs, and immunomodulation with plasma exchange or intravenous immunoglobulins modify the course of the disease, but the residual burden of physical, psychological, and social disability highlights several unmet needs, among these the need for specific, targeted, and well tolerated therapies able to improve the patients' quality of life. Complement inhibition paved the way to precision medicine in MG since, for the first time, a specific therapy targeting a crucial pathogenetic step has been designed, tested, and proven to be effective in a controlled fashion. Ravulizumab represents the first long-acting complement inhibitor approved for treatment of patients with generalized MG, able to provide rapid, complete, and sustained complement inhibition. Ravulizumab improved the MG Activity of Daily Living scale and other clinical parameters up to 26 weeks as shown by the CHAMPION MG trial, and by its open label extension, with the added value of being administered every 8 weeks. The schedule of administration is likely to improve patients' adherence and hence their quality of life. The introduction of complement inhibition will considerably change the traditional therapeutic strategy for MG.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"305-312"},"PeriodicalIF":1.8,"publicationDate":"2023-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/db/f9/prom-14-305.PMC10590807.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"50158496","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Association Between Hematologic Response and Change in Health-Related Quality of Life Among Patients with Light-Chain (AL) Amyloidosis.","authors":"Avery A Rizio,&nbsp;Kristen L McCausland,&nbsp;Michelle K Carty,&nbsp;Ansgar Conrad,&nbsp;Tiffany P Quock","doi":"10.2147/PROM.S421211","DOIUrl":"10.2147/PROM.S421211","url":null,"abstract":"<p><strong>Purpose: </strong>The purpose of this secondary data analysis was to examine the association between hematologic response and health-related quality of life (HRQoL) among patients with light-chain (AL) amyloidosis.</p><p><strong>Patients and methods: </strong>Data for this secondary analysis were collected through a non-interventional, longitudinal, online self-report survey of patients with AL amyloidosis. Patients completed an initial online survey, with follow-up surveys administered 1, 6, 12, 18, and 24 months after completion of the initial survey. The online survey included an assessment of patients' most recent self-reported hematologic response status. Eight domains and 2 summary components of HRQoL were evaluated with the SF-36v2^® Health Survey. A series of logistic regression models were used to examine the association between self-reported hematologic response at 24 months (dichotomized as new or maintained complete hematologic response; less than a complete response) and change in HRQoL from baseline to 24 months (dichotomized as meaningful worsening; improvement or preservation).</p><p><strong>Results: </strong>For all measured domains of HRQoL except physical functioning, there was no statistically significant relationship between meaningful worsening in HRQoL and hematologic response status at 24 months. Patients without a complete hematologic response had an odds of experiencing meaningful worsening of HRQoL that was similar to that of patients with a complete hematologic response.</p><p><strong>Conclusion: </strong>Among patients with AL amyloidosis, change in HRQoL was generally not associated with hematologic response. Achieving a complete hematologic response does not necessarily mean that a patient will experience increased or stable HRQoL. When defining treatment success, it is important to recognize that clinical markers such as hematologic response may not fully encapsulate the patient experience.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"297-304"},"PeriodicalIF":2.1,"publicationDate":"2023-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/f9/4a/prom-14-297.PMC10576564.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41237433","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}