Patient-reported problematic symptoms in an ALS treatment trial.
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ABSTRACT:
SUBMITTER: Raheja D
PROVIDER: S-EPMC4979603 | biostudies-literature | 2016 Apr-May
REPOSITORIES: biostudies-literature
Publications
Patient-reported problematic symptoms in an ALS treatment trial.
Raheja Divisha D Stephens Helen E HE Lehman Erik E Walsh Susan S Yang Chengwu C Simmons Zachary Z
Amyotrophic lateral sclerosis & frontotemporal degeneration 20160129 3-4
This study was undertaken to determine which symptoms are perceived to be most problematic for patients with ALS and how their severity changes over time. A retrospective study was performed of data from a randomized, double-blind, placebo-controlled trial of ceftriaxone in ALS. Participants completed the ALS Specific Quality of Life Instrument (ALSSQoL) at baseline and at intervals up to 96 weeks. Ten ALSSQoL items ask participants to rate how problematic symptoms are (the subjective feeling of ...[more]
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Project description:BackgroundAs novel therapeutic interventions are being developed and tested in the amyotrophic lateral sclerosis (ALS) population, there is a need to better understand the symptoms and issues that have the greatest impact on the lives of individuals with ALS. We aimed to determine the frequency and relative importance of symptoms experienced by adults in a national ALS sample and to identify factors that are associated with the greatest disease burden in this population.MethodsWe conducted 15 qualitative interviews of individuals with varied ALS phenotypes and analyzed 732 quotes regarding the symptomatic disease burden of ALS between August 2018 and March 2019. We subsequently conducted a national, cross-sectional study of 497 participants with ALS and ALS variants through the Centers for Disease Control and Prevention's (CDC) National ALS Registry between July 2019 and December 2019. Participants reported on the prevalence and relative importance of 189 symptomatic questions representing 17 symptomatic themes that were previously identified through qualitative interviews. Analysis was performed to determine how age, sex, education, employment, time since onset of symptoms, location of symptom onset, feeding tube status, breathing status and speech status relate to symptom and symptomatic theme prevalence.FindingsSymptomatic themes with the highest prevalence in our sample were an inability to do activities (93.8%), fatigue (92.6%), problems with hands or fingers (87.7%), limitations with mobility or walking (86.7%), and a decreased performance in social situations (85.7%). Participants identified inability to do activities and limitations with mobility or walking as having the greatest overall effect on their lives.InterpretationIndividuals with ALS experience a variety of symptoms that affect their lives. The prevalence and importance of these symptoms differ among the ALS population. The most prevalent and important symptoms offer potential targets for improvements in future therapeutic interventions.FundingResearch funding was provided by ALS Association.
| S-EPMC9755057 | biostudies-literature
Project description:PurposeTenosynovial giant cell tumor (TGCT), a rare locally aggressive neoplasm of the synovium of joints and tendon sheaths, is associated with joint destruction, inflammation, pain, and swelling, in part due to colony-stimulating factor 1 receptor-bearing macrophages recruited to the tumor by genetic elevation of colony-stimulating factor 1 activity. The most common treatment is surgery, although promising pharmacologic treatments are in development. Patient-reported outcome (PRO) instruments are critical end points in demonstrating the clinical relevance of standard oncologic outcome measures and the overall impact of novel pharmacologic therapies in nonmalignant neoplastic conditions such as TGCT. The content validity of PROs relevant to patients with TGCT has not been formally investigated, and instruments to evaluate such outcomes do not exist for this condition.MethodsPRO instruments of potential relevance were evaluated by using a literature review and by clinical and PRO experts. Patients with TGCT were recruited through clinical sites and the Internet for participation in qualitative research interviews to identify predominant symptoms and to test the relevance and content validity of several PRO measures. Select PRO measures were included in a Phase I clinical trial, and preliminary results of the PRO end points are reported descriptively.FindingsOf the 22 subjects who participated in qualitative interviews, 73% were female, and their mean age was 42.5 years (range, 27-56 years). The TGCTs (19 diffuse and 3 localized) were located in the knee (n = 15), hip (n = 3), ankle (n = 2), elbow (n = 1), and forearm (n = 1). The most common symptoms cited were pain (82%), swelling (86%), stiffness (73%), reduced range of motion (64%), and joint instability (64%), which were consistent with clinical expert input and with the content of instruments chosen by PRO experts. The worst pain numeric rating scale, Patient Reported Outcomes Measurement Information System physical functioning items, and the Western Ontario and McMaster Universities Osteoarthritis Index, as well as a worst stiffness numeric rating scale developed for TGCT, were confirmed as meaningful measures of TGCT patient symptoms and were well understood in qualitative interviews. Results from the Phase I trial showed trends of improvement in both pain and stiffness over time.ImplicationsThis study is the first to gather information directly from patients with TGCT regarding their symptom experiences. Pain, stiffness, and physical functioning are important treatment outcomes in patients with TGCT. We have identified content-valid PRO measures of these concepts, which are included in an ongoing Phase III TGCT clinical trial with pexidartinib (PLX3397) (NCT02371369).
| S-EPMC5469507 | biostudies-literature
Project description:Background A 'Patient-Reported Outcome Measure, Inquiry into Side Effects' (PROMISE) instrument was developed for patients to report common symptoms in clinical medication reviews. Objective To determine changes in patient-reported drug-associated symptoms collected by PROMISE before and after community pharmacist-led clinical medication reviews compared with usual care. Setting Community pharmacies in the Netherlands. Methods Patients were randomised into an intervention group (IG) and a control group (CG). PROMISE was used to collect symptoms experienced during the previous month, and any suspected drug-associated symptoms from both groups at baseline and at follow-up after 3 months. IG patients received a one-time clinical medication review, while CG patients received usual care. Main outcome measure Mean number of drug-associated symptoms at follow-up. Results Measurements were completed by 78 IG and 67 CG patients from 15 community pharmacies. Mean numbers of drug-associated symptoms per patient at follow-up were 4.0 in the IG and 5.0 in the CG, with an incident rate ratio between the groups of 0.90 (95% CI 0.62-1.33). Mean numbers of drug-associated symptoms per patient reported at follow-up and persisting since baseline were 2.1 in the IG and 2.6 in the CG, with an incident rate ratio of 0.85 (95% CI 0.43-1.42). The lowest percentages of persisting drug-associated symptoms detected in the IG were 'palpitations' and 'stomach pain, dyspepsia' while in the CG they were 'stomach pain, dyspepsia' and 'trembling, shivering'. Conclusion PROMISE provided meaningful information on drug-associated symptoms in clinical medication reviews, however the number of drug-associated symptoms was not reduced by performing clinical medication reviews compared with usual care.
| S-EPMC5840243 | biostudies-literature
Project description:ObjectiveTo determine the frequency and relative importance of the most meaningful symptoms in facioscapulohumeral muscular dystrophy (FSHD) and to identify the demographic and clinical features that are associated with the greatest disease burden in this population.MethodsWe performed a cross-sectional study involving 328 participants with FSHD. Collectively, participants reported the prevalence and relative importance of 274 symptoms and 15 symptomatic themes. We assessed the association between symptomatic theme prevalence and participants' age, sex, disease duration, pain level, employment status, and education.ResultsParticipants answered >48,000 questions regarding their disease burden. The symptomatic themes with the highest prevalence in our sample were problems with shoulders or arms (96.9%), limitations with activities (94.7%), core weakness (93.8%), fatigue (93.8%), limitations with mobility and walking (93.6%), changed body image due to the disease (91.6%), and pain (87.7%). Problems with shoulders and arms and limitations with mobility and walking had the greatest effect on participants' lives. Employment status and the report of pain had the most extensive association with the prevalence of symptoms, with employment being associated with 8 of 15 of the symptomatic themes and pain being associated with 7 of 15 of the symptomatic themes. Men and women with FSHD experienced a similar prevalence of all symptomatic themes.ConclusionsAdults with FSHD experience a variety of symptoms that play an important role in their disease burden. These symptoms have a variable prevalence and importance in the FSHD population and are associated with disease duration, employment status, and pain level.
| S-EPMC6808532 | biostudies-literature
Project description:BackgroundAdrenoleukodystrophy (ALD) is a multifaceted, X-linked, neurodegenerative disorder that comprises several clinical phenotypes. ALD affects patients through a variety of physical, emotional, social, and other disease-specific factors that collectively contribute to disease burden. To facilitate clinical care and research, it is important to identify which symptoms are most common and relevant to individuals with any subtype of ALD.MethodsWe conducted semi-structured qualitative interviews and an international cross-sectional study to determine the most prevalent and important symptoms of ALD. Our study included adult participants with a diagnosis of ALD who were recruited from national and international patient registries. Responses were categorized by age, sex, disease phenotype, functional status, and other demographic and clinical features.ResultsSeventeen individuals with ALD participated in qualitative interviews, providing 1709 direct quotes regarding their symptomatic burden. One hundred and nine individuals participated in the cross-sectional survey study, which inquired about 182 unique symptoms representing 24 distinct symptomatic themes. The symptomatic themes with the highest prevalence in the overall ALD sample cohort were problems with balance (90.9%), limitations with mobility or walking (87.3%), fatigue (86.4%), and leg weakness (86.4%). The symptomatic themes with the highest impact scores (on a 0-4 scale with 4 being the most severe) were trouble getting around (2.35), leg weakness (2.25), and problems with balance (2.21). A higher prevalence of symptomatic themes was associated with functional disability, employment disruption, and speech impairment.ConclusionsThere are many patient-relevant symptoms and themes that contribute to disease burden in individuals with ALD. These symptoms, identified by those having ALD, present key targets for further research and therapeutic development.
| S-EPMC10953228 | biostudies-literature
Project description:PurposeThere is growing interest to enhance symptom monitoring during routine cancer care using patient-reported outcomes, but evidence of impact on clinical outcomes is limited.MethodsWe randomly assigned patients receiving routine outpatient chemotherapy for advanced solid tumors at Memorial Sloan Kettering Cancer Center to report 12 common symptoms via tablet computers or to receive usual care consisting of symptom monitoring at the discretion of clinicians. Those with home computers received weekly e-mail prompts to report between visits. Treating physicians received symptom printouts at visits, and nurses received e-mail alerts when participants reported severe or worsening symptoms. The primary outcome was change in health-related quality of life (HRQL) at 6 months compared with baseline, measured by the EuroQol EQ-5D Index. Secondary endpoints included emergency room (ER) visits, hospitalizations, and survival.ResultsAmong 766 patients allocated, HRQL improved among more participants in the intervention group than usual care (34% v 18%) and worsened among fewer (38% v 53%; P < .001). Overall, mean HRQL declined by less in the intervention group than usual care (1.4- v 7.1-point drop; P < .001). Patients receiving intervention were less frequently admitted to the ER (34% v 41%; P = .02) or hospitalized (45% v 49%; P = .08) and remained on chemotherapy longer (mean, 8.2 v 6.3 months; P = .002). Although 75% of the intervention group was alive at 1 year, 69% with usual care survived the year (P = .05), with differences also seen in quality-adjusted survival (mean of 8.7 v. 8.0 months; P = .004). Benefits were greater for participants lacking prior computer experience. Most patients receiving intervention (63%) reported severe symptoms during the study. Nurses frequently initiated clinical actions in response to e-mail alerts.ConclusionClinical benefits were associated with symptom self-reporting during cancer care.
| S-EPMC4872028 | biostudies-literature
Project description:BackgroundAromatase inhibitor (AI) therapy results in substantial survival benefits for patients with hormone receptor-positive breast cancer. The rates of poor adherence and discontinuation of AI therapy are high, primarily because of treatment-related toxicities like musculoskeletal pain. Although pain-related symptoms may worsen during AI therapy, the authors hypothesized that nonpersistence with AI therapy was associated with symptoms that were present before treatment initiation.MethodsPostmenopausal women initiating AI therapy who were enrolled in a prospective clinical trial completed questionnaires at baseline to assess sleep, fatigue, mood, and pain. Reasons for treatment discontinuation during the first year of treatment were recorded. Associations between baseline patient-reported symptoms and treatment discontinuation because of toxicity were identified using logistic regression.ResultsFour hundred forty-nine patients were evaluable. The odds of treatment discontinuation were higher in patients who reported a greater number of symptoms before AI initiation. Baseline poor sleep quality was associated with early treatment discontinuation, with an odds ratio (OR) of 1.91 (95% confidence interval [CI], 1.26-2.89; P = .002). Baseline presence of tired feeling and forgetfulness had similar ORs for discontinuation (tired feeling: OR, 1.76; 95% CI, 1.15-2.67; P = .009; forgetfulness: OR, 1.66; 95% CI, 1.11-2.48; P = .015). An increasing total number of baseline symptoms was associated with an increased likelihood of treatment discontinuation, with an OR of 1.89 (95% CI, 1.20-2.96; P = .006) for 3 to 5 symptoms versus 0 to 2 symptoms.ConclusionsSymptom clusters in breast cancer survivors that are present before the initiation of adjuvant AI therapy may have a negative impact on a patient's persistence with therapy. Interventions to manage these symptoms may improve breast cancer outcomes and quality of life.
| S-EPMC4126845 | biostudies-literature
Project description:BackgroundThe Global Multiple System Atrophy Registry (GLOMSAR) was established in 2013. It is an online patient-reported contact registry open and free that relies on self-reported diagnosis by the patient or caregiver.ObjectivesTo report the demographics of patients enrolled in GLOMSAR and the results of an ancillary online symptom questionnaire.MethodsPatients enrolled in GLOMSAR were invited to complete a custom-designed online questionnaire about disease onset and symptom prevalence.ResultsAt the time of writing, there were 1083 participants in GLOMSAR, of which 33% (365) completed the questionnaire. The onset and frequency of most symptoms was similar to those reported in the literature in physician-reported studies. Some were understudied or not typically associated with multiple system atrophy (MSA), including reduced female sexual sensation (55%), forgetfulness (60%), pseudobulbar affect (37%), olfactory changes (36%), and visual hallucinations (21%).ConclusionsPatient-reported studies and ancillary online questionnaires are valid, underused research tools useful to advance our knowledge on understudied MSA features and highlight the patients' voice.
| S-EPMC9547130 | biostudies-literature
Project description:BackgroundIdentification of sex- and age-related differences in the presentation of atypical symptoms at stroke onset may reduce prehospital delay and improve stroke treatment if acknowledged at first contact.AimTo explore sex- and age-related differences in patient-reported typical and atypical symptoms of a stroke.MethodsWe used data from a cross-sectional survey at two non-comprehensive stroke units in the Capital Region of Denmark. Patient-reported symptoms, stroke knowledge, and behavioral response were analyzed by the Chi-square test or a Fisher's exact test separated by sex. Multivariable logistic regression adjusted for covariates were used to explore sex- and age-related differences according to each patient-reported typical or atypical symptoms.ResultsIn total, 479 patients with acute stroke were included (median age 74 years [25th to 75th percentile: 64-80], and 40.1% were women). Female sex was associated with higher odds of presenting with atypical symptoms, such as loss of consciousness (OR 2.12 [95% CI 1.08-4.18]) and nausea/vomiting (OR 2.33 [95% CI 1.24-4.37]), and lower odds of presenting with lower extremity paresis (OR 0.59 [95% CI 0.39-0.89). With each year of age, the odds decreased of presenting with sensory changes (OR 0.95 [95% CI 0.94-0.97]) and upper extremity paresis (OR 0.98 [95% CI 0.96-0.99]), whereas odds of presenting with dysphagia (OR 1.06 [95% CI 1.02-1.11]) increased.ConclusionsPatients of female sex and younger age reported on admission more frequently atypical stroke symptoms. Attention should be drawn to this possible atypical first presentation to facilitate correct identification and early stroke revascularization treatment to improve the outcome for both sexes.
| S-EPMC8978710 | biostudies-literature
Project description:ImportanceThe variability in individual symptom and adverse event reporting between pediatric patient-reports and proxy-reports is widely reported. However, the question of whether symptom profiles based on reports from children with cancer and their caregivers are similar or disparate have not yet been studied.ObjectiveTo compare proxy symptom reports with patient self-reports to assess alignment.Design, setting, and participantsA multicenter cohort study was conducted from October 2016 to December 2018 from data collected at 9 pediatric cancer centers. Participants were a convenience sample of family caregivers or proxies of children aged 7 to 18 years who had received disease-directed oncology treatment in the form of chemotherapy for at least 1 month. Data were analyzed identifying clusters of individuals (ie, latent profiles) based on various responses (ie, indicators) in August 2021.ExposuresThe children of proxy participants received upfront chemotherapy. Children and proxies completed Patient-Reported Outcomes Measurement Information System (PROMIS) surveys at 2 time points: within 72 hours preceding treatment initiation and following the course of chemotherapy.Main outcomes and measuresThe latent profile analysis methods were applied to caregiver-proxy reports of PROMIS Pediatric symptom and function measures (anxiety, depressive symptoms, pain interference, fatigue, psychological stress, and physical function-mobility). The instrument categorized respondents as high symptom suffering, medium symptom suffering, and low symptom suffering (hereafter, high, medium, and low symptom groups, respectively).ResultsOf 580 approached proxies, 431 (368 [85.00%] were female) identified as legal guardians of children aged 7 to 18 years with a first cancer diagnosis (mean [SD] age, 13.03 [3.40] years; 235 [54.65%] were male). Proxy reports of children's experiences based on the 5 proxy PROMIS measures comprised 3 distinct symptom profiles. The most common proxy assessments of children's experiences were the moderate symptom groups (45.7% [197 of 431]) and the low symptom groups profiles (40.1% [173 of 431]). A high symptom groups profile emerged which represented 14.2% (61 of 431) of proxy assessments. The number of profiles and observed distribution of profile membership was similar between child and proxy reports. Proxy reports of individual symptoms generally recorded higher scores than child reports; however, no significant difference was observed between proxies and child profile model results for the PROMIS measures.Conclusions and relevanceResults of this cohort study suggest that, at the level of symptom severity profile, proxy caregiver reports may approximate the children's reports and may serve as a guide to care when the child is not able to self-report.
| S-EPMC8965635 | biostudies-literature