Assessment Of Chronic Illness Therapy Research Articles

Qualitative development of the PROMIS Profile v1.0-Familial Chylomicronemia Syndrome (FCS) 28

PurposeFamilial chylomicronemia syndrome (FCS) is a rare genetic disorder characterized by high triglyceride levels, significant disease burden, and negative impacts on health-related quality of life. This project aimed to create a PROMIS-based patient-reported outcome measure that represents valid and important concerns for patients with FCS.MethodsWe reviewed the literature and data from a previous qualitative study of FCS to identify key FCS symptoms and impacts, which were mapped to PROMIS domains to create a pool of eligible items. Candidate items were reduced per expert feedback and patients with FCS completed cognitive interviews to confirm content validity and measure content.ResultsLiterature and qualitative data review identified ten key symptoms and 12 key impacts of FCS, including abdominal pain, fatigue, difficulty thinking, and worry about pancreatitis attacks. We identified 96 items primarily from PROMIS, supplemented with items from the Quality of Life in Neurological Disorders™ (Neuro-QoL™) and the Functional Assessment of Chronic Illness Therapy (FACIT) measurement systems. This pool was reduced to 32 candidate items, which were assessed via cognitive interviews with eight participants with FCS. Cognitive interview results and additional expert feedback led to the removal of four items and finalization of the PROMIS Profile v1.0—familial chylomicronemia syndrome (FCS) 28.ConclusionsThe PROMIS Profile v1.0—familial chylomicronemia syndrome (FCS) 28 provides strong content validity for assessing quality of life among patients with FCS. The benefits of PROMIS, including norm-referenced mean values for each measure, will facilitate comparison of patients with FCS to other clinical populations.

HALT-D: a randomized open-label phase II study of crofelemer for the prevention of chemotherapy-induced diarrhea in patients with HER2-positive breast cancer receiving trastuzumab, pertuzumab, and a taxane

PurposeTo assess whether crofelemer would prevent chemotherapy-induced diarrhea (CID) diarrhea in patients with HER2-positive, any-stage breast cancer receiving trastuzumab (H), pertuzumab (P), and a taxane (T; docetaxel or paclitaxel), with/without carboplatin (C; always combined with docetaxel rather than paclitaxel).MethodsPatients scheduled to receive ≥ 3 consecutive TCHP/THP cycles were randomized to crofelemer 125 mg orally twice daily during chemotherapy cycles 1 and 2 or no scheduled prophylactic medication (control). All received standard breakthrough antidiarrheal medication (BTAD) as needed. The primary endpoint was incidence of any-grade CID for ≥ 2 consecutive days. Secondary endpoints were incidence of all-grade and grade 3/4 CID by cycle/stratum; time to onset and duration of CID; stool consistency; use of BTAD; and quality of life (Functional Assessment of Chronic Illness Therapy for Patients With Diarrhea [FACIT-D] score).ResultsFifty-one patients were randomized to crofelemer (n = 26) or control (n = 25). There was no statistically significant difference between arms for the primary endpoint; however, incidence of grade ≥ 2 CID was reduced with crofelemer vs control (19.2% vs 24.0% in cycle 1; 8.0% vs 39.1%, in cycle 2). Patients receiving crofelemer were 1.8 times more likely to see their diarrhea resolved and had less frequent watery diarrhea.ConclusionDespite the choice of primary endpoint being insensitive, crofelemer reduced the incidence and severity of CID in patients with HER2-positive breast cancer receiving P-based therapy. These data are supportive of further testing of crofelemer in CID.Trial registrationClinicaltrials.gov, NCT02910219, prospectively registered September 21, 2016.

Relationship between Self-Efficacy, Fatigue and Health-Related Quality of Life among Patients with Chronic Illness: A Cross-Sectional Study

Introduction: Chronic illnesses such as coronary heart disease, hypertension and diabetes mellitus are the main causes of death worldwide. Additionally, previous research has discovered that fatigue commonly associated with chronic illness impairs this population’s health-related quality of life (HRQoL), and self-efficacy is critical for self-management. Thus, this study aimed to investigate the association between fatigue and self-efficacy with HRQoL in patients with chronic illness. Methods: We conducted quantitative cross-sectional research using a sample of 160 patients from a public hospital in Klang Valley, Malaysia. Self-efficacy, fatigue, and HRQoL were assessed using a self-administered questionnaire that included the Self-Efficacy Questionnaire and the Functional Assessment of Chronic Illness Therapy – Fatigue Scale (FACIT-F), respectively. Results: The mean overall score for HRQoL is 79.91 (SD=13.67), fatigue is 35.28 (SD=7.44), whereas the mean self-efficacy score is 37.93 (SD=9.17). There is a positive correlation between HRQoL with fatigue (r = 0.810, p<0.001) and self-efficacy (r = 0.66, p<0.001). Multiple regression analysis found that sociodemographic characteristics (age, gender, income levels and living area), fatigue and self-efficacy explained 69.5% of variances in HRQoL. Meanwhile, income levels, fatigue and self-efficacy were significant predictors for HRQoL. Conclusion: Fatigue and self-efficacy are among the factors that influence HRQoL among patients with chronic illness. The findings indicated that health practitioners should develop a systematic intervention to prevent fatigue, promote patients’ self-efficacy and HRQoL.

Effect of respiratory muscle training on ventilatory function in women post mastectomy

Mastectomy is one of the widely spread surgeries to remove breast cancer, however, this type of intervention disturbs the ventilatory function of the patient after surgery. The role of physical therapy is crucial in such cases as to improve the function of the respiratory system, and chest expansion, decrease the severity of dyspnea, and provide better health after mastectomy. Purpose: to study the effect of respiratory muscle training by using (The Breather) on ventilatory functions in women after mastectomy. Objectives: 40 women with unilateral post-mastectomy were randomly selected from Baheya Center with an age range from (40-50) years old. (Received radiotherapy /chemotherapy and physical therapy sessions), divided into 2 equal groups (each group=20). Methodology: Group A received respiratory muscle training with the breather device with physical therapy rehabilitation and Group B received physical therapy rehabilitation only. Both groups received sessions twice weekly for 12 weeks. Equipment used for evaluation: ventilatory function measuring device (Spirometer) used to measure ventilatory function (FEV1, FVC, FEV1/FVC, MVV), Tape measurement used to measure chest expansion, and Functional Assessment of Chronic Illness Therapy - Dyspnea-10 item (FACIT-Dyspnea) Questionnaire.

Health-related quality of life and treatment satisfaction in Chronic Lymphocytic Leukemia (CLL) patients on ibrutinib compared to other CLL treatments in a real-world US cross sectional study

The objective of this study was to describe real-world health-related quality of life (HRQoL) and treatment satisfaction of ibrutinib-treated patients with CLL compared to a reference group. This study was completed in two parts. The first portion (Norming Study) was a US online survey conducted to serve as a reference population. The Norming Study included a total of 139 patients with CLL, excluding those treated with ibrutinib: 64 were treatment naive (Tx naive), 36 were 1st line (1L), and 38 were in or had completed ≥2 lines (2L+) patients with CLL. The second portion (CLL Ibrutinib Study) included 1L and 2L+ ibrutinib patients with CLL treated for ≥6 months in which 118 patients (1L n = 88 and 2L+ n = 30) completed the study. Respondents completed demographic and clinical information and the following HRQoL surveys: (Short Form-12v2® Health Survey [SF-12v2], Functional Assessment of Cancer Therapy-General [FACT-G], FACT-Leukemia [FACT-Leu] Functional Assessment of Chronic Illness Therapy [FACIT]-Fatigue, and Cancer Therapy Satisfaction Questionnaire [CTSQ]). Higher scores indicate better HRQoL/treatment satisfaction. Differences in effect sizes between the two samples at the group level were calculated using Hedges' g. Medium to large positive effects were seen in the CLL Ibrutinib group on several measures compared to the Reference Study groups. The FACT-G total score was 89.2±11.1 for CLL Ibrutinib Study patients compared to 75.8±22.6 CLL Norming Tx naïve patients, 61.3±21.8 in 1L, and 61.7±20.7 in 2L+. Similar trends were seen with FACT-Leu total score and FACIT-Fatigue. CLL Ibrutinib Study patients scored higher on all CTSQ domain scores compared to the CLL Norming patients treated with other CLL therapies. We found that Ibrutinib-treatment had better HRQoL and treatment satisfaction compared to patients receiving other therapies, irrespective of line of therapy.

MDS-111 Hemoglobin, Lactate Dehydrogenase, and FACIT-Fatigue Normalization in Pegcetacoplan-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria from Two Phase 3 Clinical Trials

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease involving complement-mediated hemolysis. Pegcetacoplan is the first FDA/EMA-approved C3 complement-inhibitor for adults with PNH. We report hemoglobin, lactate dehydrogenase (LDH), and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue normalization rates in the phase 3 PEGASUS (NCT03500549) and PRINCE (NCT04085601) studies. The PEGASUS study (2018-2020) randomized patients with hemoglobin levels <10.5 g/dL at screening despite stable eculizumab treatment (≥3-months) 1:1 to pegcetacoplan (n=41) or eculizumab (n=39) for the 16-week randomized controlled period (RCP). Eculizumab-treated patients switched to pegcetacoplan during the open-label period through Week 48. The 26-week PRINCE study (2019-2021) randomized complement-inhibitor naive patients 2:1 to pegcetacoplan (n=35) or control treatment ([n=18] without complement-inhibitors: eculizumab/ravulizumab). Normalization was defined separately as hemoglobin ≥gender lower limit of normal, LDH ≤upper limit of normal, and FACIT-Fatigue scores ≥population norm (43.6). Transfusion recipients (hemoglobin/LDH endpoints only), patients withdrawn or lost to follow-up, or control-escape patients (PRINCE) were considered not normalized. Safety endpoints included incidences of adverse events (AEs). Pegcetacoplan demonstrated rapid treatment effects in both studies; Week 2 hemoglobin normalization rates were 43.9% (PEGASUS, baseline 0%) and 22.9% (PRINCE, baseline 2.9%) and Week 2 LDH normalization rates were 87.8% (PEGASUS, baseline 41.5%) and 51.4% (PRINCE, baseline 0%). At Week 16 (PEGASUS) and Week 26 (PRINCE), higher normalization rates were achieved with pegcetacoplan versus respective comparator treatments in hemoglobin (PEGASUS: 34.1% versus 0%; PRINCE: 45.7% versus 0%), LDH (PEGASUS: 70.7% versus 15.4%; PRINCE: 65.7% versus 0%), and FACIT-Fatigue (PEGASUS: 48.8% versus 10.3%; PRINCE: 60.0% versus 11.1%). Numerically similar results were observed at Week 48 in PEGASUS patients randomized to pegcetacoplan or switched from eculizumab to pegcetacoplan. Common AEs in pegcetacoplan-treated patients were injection site reactions (ISRs), diarrhea, abdominal pain, nasopharyngitis, upper respiratory tract infection, hemolysis, cough, and headache for PEGASUS, and ISRs, hypokalemia, and dizziness for PRINCE. Pegcetacoplan rapidly enables higher rates of hemoglobin and LDH normalization, and improvements in FACIT-Fatigue scores compared to eculizumab and control treatment. This further supports the rapid treatment effect of pegcetacoplan in improving clinical parameters and health-related quality of life with a favorable safety profile.

MDS-112 Normalization of Hematologic and Health-Related Quality of Life Markers in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Pegcetacoplan and Baseline Hemoglobin at or Above 10 g/dL

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease characterized by chronic complement-mediated hemolysis. PNH-associated symptoms include anemia and fatigue, with patients requiring frequent blood transfusions. Pegcetacoplan is an FDA/EMA-approved C3 complement inhibitor for adults with PNH. Evaluate normalization rates of hematologic markers, fatigue, and safety after 16 weeks of pegcetacoplan monotherapy in a subgroup of patients with PNH and baseline hemoglobin levels ≥10.0 g/dL from the PEGASUS (NCT03500549), PADDOCK (NCT02588833), and PRINCE (NCT04085601) studies. PEGASUS (2018-2020) enrolled patients with hemoglobin levels <10.5 g/dL at screening despite stable eculizumab treatment (≥3 months) and randomized patients 1:1 to eculizumab or pegcetacoplan for the 16-week randomized controlled period (RCP). Eculizumab-treated patients in the RCP switched to pegcetacoplan through week 48 for the open-label period. PADDOCK (2015-2019) and PRINCE (2019-2021) evaluated pegcetacoplan in complement inhibitor-naïve patients. PRINCE compared pegcetacoplan to control (excluding eculizumab/ravulizumab) in patients with hemoglobin levels below gender-specific lower limits of normal. This post-hoc analysis included adults with PNH, baseline hemoglobin levels ≥10.0 g/dL, and no transfusions within 14 days of baseline. Hemoglobin, lactate dehydrogenase (LDH), absolute reticulocyte count (ARC), and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue normalization were evaluated after pegcetacoplan monotherapy for 16 weeks. Patients who withdrew or were lost to follow-up without efficacy data at specified timepoints were considered not normalized. Safety endpoints included incidences of adverse events (AEs). This analysis included 22 patients: 9 PEGASUS, 5 PADDOCK, and 8 PRINCE. Sixteen weeks of pegcetacoplan monotherapy led to increases in hemoglobin (PEGASUS: 66.7%; PADDOCK: 40.0%; PRINCE: 62.5%) and LDH (PEGASUS: 77.8%; PADDOCK: 60.0%; PRINCE: 100.0%) normalization rates, with similar results in ARC and FACIT-Fatigue. No serious AEs were reported in PADDOCK or PRINCE subgroups. One PEGASUS patient had a serious AE (breakthrough hemolysis; severe, unrelated to pegcetacoplan, resolved, no discontinuation) following upper respiratory infection. Injection site reactions and infections/infestations occurred at similar rates among subgroups. No thrombotic events occurred in this subgroup. Results suggest pegcetacoplan can be efficacious in patients with less severe anemia regardless of prior complement inhibitor treatment, further improving clinical markers of hemolysis and fatigue. The safety profile of pegcetacoplan was consistent with previous studies.

Differences in patient and caregiving partner responses to individual items on the FACIT-COST questionnaire.

183 Background: The financial impacts of cancer diagnosis and treatment extend beyond patients to affect intimate partners. Understanding how both members of the dyad experience financial toxicity (FT) can help inform dyadic interventions to address FT. We compare patient and partner responses to items on the Functional Assessment of Chronic Illness Therapy (FACIT)-COST, a patient-reported outcome measure of cancer-related financial distress. Methods: Adult patients with stage II-IV breast, lung, or colorectal cancer and their spouse/ cohabiting partner independently completed the 11-item FACIT-COST. Correlations between responses by both members were calculated using Pearson’s correlation coefficient. Results: There were 336 dyads (median patient age 53, 45% stage IV, 84% white) included in the analysis. Both members of the dyad had similar FT experiences in 74% of cases (26% and 48% reported FT and no FT respectively) while 26% diverged in their experience of FT. Responses to items 3, 6, 7, 10, and 11 (Table) were highly correlated (r &gt; 0.6) while responses to questions 4 (I feel I have no choice about the amount of money we spend on care) and 5 (I am frustrated that I cannot work or contribute as much as I usually do) were weakly correlated (0.3 &gt; r &gt; 0), with patients generally reporting worse scores than partners on these items. Conclusions: Within-couple correlation between FACIT-COST items varies, with multiple responses weakly correlated. Future research should assess the impact of this variation on psychosocial functioning and explore approaches to facilitate dyadic communication around this important issue.[Table: see text]

Verification of the Mandarin Chinese version of Advance Care Planning Engagement Survey Scale in community-dwelling older people.

Advance care planning (ACP) has become increasingly critical for older people. The Advance Care Planning Engagement Survey (ACPES) develops targeted interventions for older people by measuring ACP-related behaviours, while previous studies focus only on advance directives. Moreover, while ACPES has English and Dutch versions, it does not yet have a Mandarin Chinese version (ACPES-MC) that can be used for Chinese populations. To translate the ACPES into Mandarin Chinese and test its reliability and validity in community-dwelling older people with chronic diseases. The English version of the ACPES was translated into Mandarin Chinese using Functional Assessment of Chronic Illness Therapy. Four communities were recruited in central China (N=450) by convenience sampling. Internal consistency and construct validity were used to evaluate the reliability and validity of the ACPES-MC. The ACPES-MC consists of 34 items across five domains, with good internal consistency (0.817), with each dimension ranging from 0.606 to 0.881; exploratory factor analysis was distributed to four different factors and the total variance explained was 63.537%; and confirmatory factor analysis results showed that χ2 =3791.131 (p < .001), χ2 /df=1.106, CFI=0.980, IFI=0.980, NFI=0.827 and RMR=0.027, indicating a good model fit to previous factor structures. The ACPES-MC is an effective and reliable tool that can measure the ACP-related behaviour stage of community-dwelling older people and evaluate the effect of ACP intervention. The ACPES-MC can be used in healthcare to identify potential ACP-related behaviours in community-dwelling older people with chronic diseases, including native and ethnic Chinese, evaluate their behaviour change stages and promote the application of ACP.

Dynamics of patient reported outcomes during the use various biological disease-modifying antirheumatic drugs for rheumatoid arthritis

One of the main tasks of modern complex therapy of rheumatoid arthritis (RA) is to improve the quality of life of patients. To do this, it is necessary not only to achieve remission or low activity, but also to successfully control the main, most painful, manifestations of the disease. Therefore, when evaluating the results of RA treatment, the dynamics of not only standard indices (DAS28 (Disease Activity Score 28), CDAI (Clinical Disease Activity Index), SDAI (Simplified Disease Activity Index)), but also the so-called “patient reported outcomes” (PRO) – a patient’s global assessment of disease activity (PGA), pain, functional disorders and fatigue.This review examines the effect of one of the main classes of anti–rheumatic drugs - biological disease-modifying antirheumatic drugs (bDMARDs) on the PROs. The results of a series of randomized controlled trials are presented, in which changes in PROs were studied using various tumor necrosis factor α (TNF-α) inhibitors, abatacept T-lymphocyte co-stimulation inhibitor, rituximab CD20 inhibitor and interleukin (IL) 6 inhibitors.The use of bDMARDs in combination with methotrexate (MTX) provides a reduction in PGA and pain by 50-60%, functional disorders according to HAQ (Health Assessment Questionnaire) and fatigue according to FACIT-F (Functional Assessment of Chronic Illness Therapy – Fatigue) – by 15-30%. B DMARDs monotherapy (with the exception of the effect of tocilizumab on HAQ) does not exceed MTX monotherapy in its effect on PROs. Monotherapy with tocilizumab provides more favorable dynamics of PGA and pain than monotherapy with TNF-α inhibitors. An important advantage of IL-6 inhibitors is the rapid achievement of a clinical effect, which is noted already in the first 2 weeks after the first administration of the drug.

Measurement and Validation of the Comprehensive Score for Financial Toxicity (COST) in a Population With Diabetes.

The Comprehensive Score for Financial Toxicity-Functional Assessment of Chronic Illness Therapy (COST-FACIT) is a validated instrument measuring financial distress among people with cancer. The reliability and construct validity of the 11-item COST-FACIT were examined in adults with diabetes and high A1C. We examined the factor structure (exploratory factor analysis), internal consistency reliability (Cronbach α), floor/ceiling effects, known-groups validity, and predictive validity among a sample of 600 adults with diabetes and high A1C. COST-FACIT demonstrated a two-factor structure with high internal consistency: general financial situation (7-items, α = 0.86) and impact of illness on financial situation (4-items, α = 0.73). The measure demonstrated a ceiling effect for 2% of participants and floor effects for 7%. Worse financial toxicity scores were observed among adults who were women, were below the poverty line, had government-sponsored health insurance, were middle-aged, were not in the workforce, and had less educational attainment (P < 0.01). Worse financial toxicity was observed for those engaging in cost coping behaviors, such as taking less or skipping medicines, delaying care, borrowing money, "maxing out" the limit on credit cards, and not paying bills (P < 0.01). In regression models for the full measure and its two factors, worse financial toxicity was correlated with higher A1C (P < 0.01), higher levels of diabetes distress (P < 0.01), more chronic conditions (P < 0.01), and more depressive symptoms (P < 0.01). Findings support both the reliability and validity of the COST-FACIT tool among adults with diabetes and high A1C levels. More research is needed to support the use of the COST-FACIT tool as a clinically relevant patient-centered instrument for diabetes care.

Impact Of Covid-19 Infection On Activity Levels, Perceived Fatigue, And Mental Health

Physical activity patterns have changed dramatically during the COVID-19 pandemic. Whether perceived fatigue and mental health, which are known to be altered after surviving COVID-19 infection, impacts physical activity is not understood. PURPOSE: To determine associations between indices of mental health, perceptions of fatigue, and physical activity in COVID-19 survivors. METHODS: 24 COVID-19 survivor-control pairs (n = 48, males 14, females 34) matched for age, sex, and BMI and 2-13 months post infection (8.04 ± 0.71 months), aged 19-69 yr (38.6 ± 17.8 yr, 46 White, 2 Asian) completed 1) Past Medical History, 2) Depression, Anxiety, and Stress Scale (DASS), 3) Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F), and 4) wore an accelerometer to track activity for up to 7 days. RESULTS: There were no differences between controls (CON) and COVID-19 survivors (COV) for sedentary time/day (p = 0.14), sedentary bouts/day (p = 0.29), and steps/day (p = 0.31). COV scored higher than CON on the depression subsection of the DASS (6.17 ± 6.54 vs. 2.88 ± 3.99, p = 0.021). COV also scored higher than CON on the anxiety sub-section of the DASS (5.17 ± 4.57 vs. 2.83 ± 3.58, p = 0.028). However, COV were similar to CON on the stress sub-section of the DASS (6.83 ± 4.97 vs. 8.92 ± 7.25, p = 0.13). Additionally, COV reported greater feelings of fatigue, scoring lower than CON on the FACIT-F by 6.08 points (p = 0.004). There were no correlations between anxiety and depression with steps/day (R2 = 0.005-0.09), nor between time since infection with perceived fatigue, anxiety, and depression (R2 = 0.0007-0.2). CONCLUSION: Despite being 8 months post COVID infection on average, COVID survivors reported greater anxiety, depression, and feelings of fatigue than healthy matched controls during the pandemic. Physical activity (steps/day), thought to accelerate recovery from COVID-19, did not appear to mediate an individual’s perceived fatigue and mental health in these mild-to-moderate cases of COVID-19 infection. Funded by Institute for Women’s Leadership (Marquette University)

Effects of an outpatient exercise programme for Australians with cancer-related fatigue: an unmet need.

Cancer-related fatigue (CRF) is a common debilitating condition. International evidence supports an exercise prescription for CRF. The majority of Australians with cancer do not meet recommended exercise targets. To analyse the effects of a guideline-based supervised exercise programme on CRF among a representative private hospital cancer patient sample (n = 268). We collected data from 268 patients recruited from haematology and oncology over a 5-year period. Participants underwent a 3-month CRF exercise programme based on internationally recognised exercise guidelines. The programme, conducted by a multidisciplinary team, operated twice weekly sessions of 2 h duration comprising aerobic, resistance and balance exercises; hydrotherapy and condition counselling; fatigue management; and dietetic, speech pathology and swallowing education (head and neck cancers). The effect of the programme was measured in relation to the following outcomes: Functional Assessment of Chronic Illness Therapy, Fatigue (self-reported fatigue); Functional Assessment of Cancer Therapy, general quality of life (health-related quality of life in cancer); six-minute walk test; and Lawton's Instrumental Activities of Daily Living Scale. Multivariate outcomes showed statistically significant improvements in all four major outcome measures, plus a programme effect of greater than 0.7 for each outcome variable. The programme treatment outcomes were consistent over the 5 years of the programme. The outcomes of this programme contribute to exercise guidelines in Australia. Currently only position statements exist on the subject, but there are no programme guidelines. An exercise prescription is critical to cancer outcomes. This programme is likely to benefit cancer survivors experiencing CRF across private and public hospitals in Australia.

Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial

In the PEGASUS trial, the complement C3 inhibitor, pegcetacoplan, showed superiority to eculizumab in improving haematological outcomes in adult patients with paroxysmal nocturnal haemoglobinuria and suboptimal response to eculizumab at 16 weeks. The aim of the open-label period was to evaluate the long-term efficacy and safety of pegcetacoplan through to 48 weeks. PEGASUS was a phase 3, randomised, open-label, active-comparator controlled trial conducted in 44 centres in Australia, Belgium, Canada, France, Germany, Japan, Russia, South Korea, Spain, the UK, and the USA. Eligible participants were aged 18 years or older, had paroxysmal nocturnal haemoglobinuria, and had a haemoglobin concentration of less than 10·50 g/dL after 3 months or longer of stable eculizumab treatment. After a 4-week run-in with eculizumab plus pegcetacoplan, patients were randomly assigned (1:1) by interactive response technology to pegcetacoplan (1080 mg subcutaneously twice weekly) or eculizumab (according to their regimen at enrolment) for 16 weeks and could continue to the open-label period (32 weeks of pegcetacoplan monotherapy [pegcetacoplan-to-pegcetacoplan] or 28 weeks of pegcetacoplan monotherapy [eculizumab-to-pegcetacoplan]). Randomisation was stratified by platelet count and number of previous blood transfusions. The primary endpoint was change from baseline in haemoglobin at week 16, which has previously been reported. The outcomes of the open-label period (week 16 to week 48) are reported here. At 48 weeks, efficacy (including mean haemoglobin concentration and quality of life measured on the Functional Assessment of Chronic Illness Therapy [FACIT]-Fatigue scale) was assessed in the intention-to-treat population and safety was assessed per protocol. This trial was registered with ClinicalTrials.gov, NCT03500549, and has been completed. Between June 14, 2018, and Nov 14, 2019, 80 patients were randomly assigned to receive treatment with pegcetacoplan (41 patients) or eculizumab (39 patients). Most participants were women (49 [61%]) and 31 (39%) were men; 12 (15%) were Asian, two (3%) were Black, 49 (61%) were White, and 17 (21%) were another race or did not report their race. The open-label period had 77 participants (38 pegcetacoplan-to-pegcetacoplan, 39 eculizumab-to-pegcetacoplan). Patients in the pegcetacoplan-to-pegcetacoplan group maintained high mean haemoglobin concentrations between 16 weeks (11·54 g/dL [SD 1·96]) and 48 weeks (11·30 g/dL [1·77]; p=0·14). Patients in the eculizumab-to-pegcetacoplan group had significantly greater mean haemoglobin concentrations at 48 weeks (11·57 g/dL [2·21]) versus 16 weeks (8·58 g/dL [0·96]; p<0·0001). Clinically meaningful improvements in FACIT-Fatigue scores were observed at 48 weeks, with a mean change from baseline for all patients receiving pegcetacoplan monotherapy of 9·89 points (SD 9·63), for patients in the pegcetacoplan-to-pegcetacoplan group mean 10·14 points (9·06), and for patients in the eculizumab-to-pegcetacoplan group mean 9·62 points (10·34). During the entire study period, 13 (16%) of 80 patients discontinued treatment (three [7%] of 41 through to week 16 due to breakthrough haemolysis, and ten [13%] of 77 due to severe treatment-emergent adverse events) and 18 patients (eight pegcetacoplan-to-pegcetacoplan, ten eculizumab-to-pegcetacoplan) had at least one serious treatment-emergent adverse event during the open-label period, four were considered to be related to pegcetacoplan treatment. The most common treatment-emergent adverse events (in ≥10% patients) among both pegcetacoplan-treated groups during the open-label period were injection site reactions (in 20 [26%] of 77 patients), haemolysis (15 [19%]), nasopharyngitis (12 [16%]), and diarrhoea (ten [13%]). No treatment-related deaths occurred throughout the duration of the study. The durability of improved haematological outcomes and favourable safety profile over 48 weeks of treatment suggests that pegcetacoplan has the potential to improve treatment benefit and alter treatment goals in patients with paroxysmal nocturnal haemoglobinuria. Apellis Pharmaceuticals.

Impact of Rehabilitation on Fatigue in Post-COVID-19 Patients: A Systematic Review and Meta-Analysis

The post-COVID-19 syndrome may affect patients after the COVID-19 post-acute phase. In particular, the 69% of patients reported persistent fatigue at the discharge. To date, no clear data are available regarding the most effective rehabilitative approaches for the treatment of this condition. Thus, this systematic review aimed to evaluate the rehabilitation treatment’s efficacy on fatigue in post-COVID-19 patients. We systematically searched PubMed, Scopus, and Web of Science databases to find longitudinal study designs presenting: post-COVID-19 patients as participants; a rehabilitative approach aimed to reduce post-COVID-19 syndrome as intervention; and fatigue intensity assessed through an evaluation tool that quantified the perceived exertion (i.e., fatigue severity scale, FSS; Borg Scale (BS); Borg Category Ratio 10, CR10; Checklist Individual Strength (CIS) fatigue scale; FACIT (Functional Assessment of Chronic Illness Therapy) fatigue scale). The present systematic review protocol was registered on PROSPERO (registration number CRD42021284058). Out of 704 articles, 6 studies were included. Nearly all patients showed COVID-19-related fatigue, and after the rehabilitation treatment, only 17% of subjects reported the persistency of symptoms. The overall effect size reported a −1.40 decrease in Borg Category Ratio 10 with a SE of 0.05 and a 95% CI between −1.50 and −1.30 (p &lt; 0.001). The present systematic review and meta-analysis underlines the rehabilitation role in the fatigue reduction in patients affected by post-COVID-19 syndrome.

Sutimlimab improves quality of life in patients with cold agglutinin disease: results of patient-reported outcomes from the CARDINAL study

Patients with cold agglutinin disease (CAD) experience fatigue and poor quality of life. However, previous CAD-related studies have not explored patient-reported outcomes such as the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue. Sutimlimab, a C1s complement inhibitor, has been shown to halt haemolysis in CAD. Here, we present 26-weeks’ patient-reported data from CARDINAL Part A (ClinicalTrials.gov, NCT03347396), which assessed efficacy and safety of sutimlimab in patients with CAD and recent history of transfusion. Aside from measuring changes in haemolytic markers, FACIT-Fatigue was measured at the treatment assessment timepoint (TAT; average of weeks 23, 25, and 26). Exploratory endpoints included the change in EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L) and the 12-Item Short Form Health Survey (SF-12) at TAT, and Patient Global Impression of Change (PGIC), and Patient Global Impression of (fatigue) Severity (PGIS) at week 26. Mean (range) FACIT-Fatigue scores increased from 32.5 (14.0–47.0) at baseline (a score indicative of severe fatigue) to 44.3 (28.0–51.0) at TAT. Considerable improvements were reported for EQ-5D-5L at TAT, SF-12 scores at TAT, and PGIC and PGIS scores at week 26. Sutimlimab treatment resulted in sustained improvements in symptoms of fatigue and overall quality of life in patients with CAD. NCT03347396. Registered 20 November, 2017.

Oscillatory positive expiratory pressure therapy in COPD (O-COPD): a randomised controlled trial

BackgroundOscillatory positive expiratory pressure (OPEP) devices are intended to facilitate sputum clearance and reduce cough, but there is limited evidence for their effectiveness in COPD, or to guide patient selection....

Steel magnolias - shared telehealth for multidisciplinary gynecologic cancer survivorship: an innovative model for addressing geographic barriers to guideline adherent survivorship for rural patients (136)

Objectives: Geographic disparities are linked to suboptimal cancer care. We aimed to describe our experience with our Shared TElEheaLth for MultidisciplinAry GyNecOLogIc cAncer Survivorship (SM) program with regards to financial toxicity, patient satisfaction, and travel burden. We also performed initial comparisons to rural patients who traveled to our clinic. Methods: After IRB approval, women in remission and on surveillance from gynecologic cancer living in a rural region with no practicing gynecologic oncologist were approached for enrollment after a clinic visit. After consent, the Patient Satisfaction Questionnaire Short Form 18 (PSQ-18) and the Comprehensive Score for Financial Toxicity- Functional Assessment of Chronic Illness Therapy (COST FACIT) questionnaires were administered. Women in the SM program were seen in person by a gynecologist close to home with a concomitant hospital-supplied tablet-based virtual visit by a gynecologic oncologist to address cancer-related survivorship issues. Others surveyed traveled (T) for surveillance to visit our gynecologic oncologists in person. Fisher’s exact tests were used for categorical comparisons and Wilcoxon Rank-Sum tests for continuous comparisons. Conclusions: We describe our initial experience with an innovative hybrid-virtual surveillance visit concept. Collaboration with the patients’ local gynecologists allowed for an in-person gynecology visit with an in tandem tablet-based gynecologic oncology session during the same visit. In this diverse, rural patient population, we demonstrated that this novel survivorship care delivery model resulted in fewer miles and shorter time traveled for patients. Financial toxicity was high overall. There were no early signals that financial toxicity or patient satisfaction was compromised. A considerable portion of the women we served reported a lack of access to amenities necessary for travel or self-guided telemedicine visits. We were able to overcome many of these transportation and technology barriers with the STEEL MAGNOLIAS program, ensuring more equitable delivery of gynecologic oncology care in the survivorship period. Objectives: Geographic disparities are linked to suboptimal cancer care. We aimed to describe our experience with our Shared TElEheaLth for MultidisciplinAry GyNecOLogIc cAncer Survivorship (SM) program with regards to financial toxicity, patient satisfaction, and travel burden. We also performed initial comparisons to rural patients who traveled to our clinic. Methods: After IRB approval, women in remission and on surveillance from gynecologic cancer living in a rural region with no practicing gynecologic oncologist were approached for enrollment after a clinic visit. After consent, the Patient Satisfaction Questionnaire Short Form 18 (PSQ-18) and the Comprehensive Score for Financial Toxicity- Functional Assessment of Chronic Illness Therapy (COST FACIT) questionnaires were administered. Women in the SM program were seen in person by a gynecologist close to home with a concomitant hospital-supplied tablet-based virtual visit by a gynecologic oncologist to address cancer-related survivorship issues. Others surveyed traveled (T) for surveillance to visit our gynecologic oncologists in person. Fisher’s exact tests were used for categorical comparisons and Wilcoxon Rank-Sum tests for continuous comparisons. Conclusions: We describe our initial experience with an innovative hybrid-virtual surveillance visit concept. Collaboration with the patients’ local gynecologists allowed for an in-person gynecology visit with an in tandem tablet-based gynecologic oncology session during the same visit. In this diverse, rural patient population, we demonstrated that this novel survivorship care delivery model resulted in fewer miles and shorter time traveled for patients. Financial toxicity was high overall. There were no early signals that financial toxicity or patient satisfaction was compromised. A considerable portion of the women we served reported a lack of access to amenities necessary for travel or self-guided telemedicine visits. We were able to overcome many of these transportation and technology barriers with the STEEL MAGNOLIAS program, ensuring more equitable delivery of gynecologic oncology care in the survivorship period.

Burden and Disease Characteristics of Psoriatic Arthritis at a Tertiary Center.

Background: Psoriatic arthritis (PsA) has a detrimental influence on the quality of life (QoL). The goal of this study was to evaluate the QoL of patients with PsA and its determinants at the King Abdulaziz University Hospital in Jeddah, Saudi Arabia.Methods: A cross-sectional study was conducted on 60 PsA patients. A questionnaire was used to collect data about their demographics. Assessment of health-related quality of life (HRQOL) was done by the Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36). The Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and the FACIT-General (FACIT-G) scales were used to assess fatigue. The Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) was used to assess disease activity.Results: The mean age of the patients was 50.33 ± 11.15 years and 78.3% were females. The mean HRQOL, FACIT-G, FACIT, and BASDAI scores were 59.99 ± 34.67, 28.18 ± 5.95, 20.01 ± 9.68, and 4.05 ± 2.54, respectively. The HRQOL scores were found to have a highly significant negative correlation with both the FACIT and BASDAI scores, as well as the patients' age and BMI. The FACIT-G scores and the BASDAI scores, as well as the BASDAI scores and the FACIT scores, were found to have a strong positive correlation with age and BMI.Conclusion: PsA has a significant detrimental influence on QoL, with a link between QoL and disease activity and fatigue. A greater understanding of QoL issues will help improve the quality of care.

Association of Health Insurance Literacy With Financial Hardship in Patients With Cancer

Patient-reported financial hardship is an increasing challenge in cancer care delivery. Health insurance literacy and its association with financial hardship in patients with cancer, especially after controlling for financial literacy, have not been well examined. To examine the prevalence of and factors in the association between health insurance literacy and financial literacy as well as the overall and individual domains of financial hardship and their association with health insurance literacy, both independently and when adjusted for financial literacy, in patients with cancer. This cross-sectional survey study recruited and enrolled patients from 2 separate ambulatory infusion centers at Mayo Clinic Arizona in Phoenix, Arizona, and the University of Mississippi Medical Center in Jackson, Mississippi. Adult patients aged 18 years or older were enrolled from December 2019 to February 2020 and from August to October 2020 at Mayo Clinic Arizona (n = 299) and from September 2020 through January 2021 at the University of Mississippi Medical Center (n = 105). Survey respondents received a $5 gift card. Surveys included questions about sociodemographic characteristics, health insurance literacy and financial literacy, financial knowledge, and financial hardship and its domains (material hardship, psychological hardship, and behavioral hardship). Financial hardship was assessed using the COST-FACIT (Comprehensive Score for Financial Toxicity-Functional Assessment of Chronic Illness Therapy) measure and National Health Interview Survey questions to capture information about the domains of financial hardship. The Health Insurance Literacy Measure is a validated 21-item measure of a consumer's ability to select and use health insurance. Five questions from the National Financial Capability Study assessed financial literacy. A total of 404 participants were enrolled in the study. Median (IQR) age of the respondents was 63 (54-71) years, and 219 were women (54%), 307 were non-Hispanic White individuals (76%), 153 (38%) had private insurance, and 289 (72%) had solid tumors. Overall financial hardship (denoted by median COST-FACIT score <27 points) was reported by 49% (95% CI, 44%-53%) of the cohort. Prevalence of financial hardship was higher using the National Health Interview Survey questions, with 68% (95% CI, 63%-72%) of respondents reporting at least 1 hardship domain (n = 276). Sixty-six percent (95% CI, 60%-69%) of respondents (n = 265) had a high level of financial literacy. The mean (SD) Health Insurance Literacy Measure score was 64.9 (13.3) points. In multivariable analyses, each 10-point increase in the Health Insurance Literacy Measure score was associated with lower odds of financial hardship (odds ratio, 0.82; 95% CI, 0.68-0.99; P = .04). However, this association was no longer significant after adjusting for financial literacy. Results of this study showed that, despite a high level of health insurance literacy and financial literacy, the prevalence of financial hardship was high. Although there were lower odds of financial hardship with increased health insurance literacy, the association was no longer significant when financial literacy was added to the model, suggesting that a high level of financial literacy may help mitigate the adverse outcome of lower health insurance literacy levels in patients with cancer.