Assessment Of Chronic Illness Therapy Research Articles

Hematopoietic Stem Cell Transplantation Impact on Patients' Perceived Quality of Life: A Longitudinal Study.

The aim of this paper was to evaluate the quality of life of adult patients with onco-hematological disease treated with hematopoietic stem cell transplantation up to two years post-transplantation. A quantitative, observational, longitudinal, and analytical study was conducted with 121 participants diagnosed with onco-hematological cancer who underwent hematopoietic stem cell transplantation between October 2017 and September 2019, with a 2-year post-transplantation follow-up, of whom only 39 completed the study. The Functional Assessment of Cancer Therapy-Bone Marrow Transplantation (FACT-BMT) questionnaire and its subscales, Functional Assessment of Cancer Therapy-General (FACT-BMT) and Functional Assessment of Cancer Therapy Trial Outcome Index (FACT-TOI), developed by the Functional Assessment of Chronic Illness Therapy (FACIT) and validated for Spain, were used to assess quality of life. The average age for hematopoietic stem cell transplantation was 54 years, with a majority of male participants. The evaluation of quality of life showed a decrease at the time of hospital discharge, followed by a progressive improvement up to one year after the transplantation. There was a significant difference in the quality of life questionnaire scores between both sexes during all stages of the research, with higher scores in male participants. The length of hospital stay significantly affected patients' physical and functional well-being, and marital status was related to differences in the perception of quality of life. Despite the initial decrease in quality of life for patients undergoing hematopoietic stem cell transplantation, levels of quality of life similar to baseline are regained one year after the transplantation. Sociodemographic variables are related to how these patients perceive their quality of life. However, further studies with a larger sample size are needed for more precise results.

Translation and Cultural Adaptation into Arabic of Patient-Reported Outcome Measurement Information System® Item Banks: Cognitive Function Abilities and Physical Function for Samples with Mobility Aid Users.

This study aimed to provide Arabic-speaking individuals with tools to assess their cognitive abilities and physical function and to contribute to a better understanding of these capabilities in this population. Thus, the specific objective was to translate into Arabic and culturally adapt two Patient-Reported Outcome Measurement Information System (PROMIS) item banks: the Adult Cognitive Function Abilities and the Physical Function for Samples with Mobility Aid Users item banks. This study employed the Functional Assessment of Chronic Illness Therapy (FACIT) multilingual translation methodology to ensure cultural and linguistic relevance. The translation process included forward and back translations, expert reviews, and finalisation by a language coordinator. Cognitive debriefing interviews were conducted with 30 native healthy Arabic speakers to assess the clarity and comprehension of translated items. Most items were well understood, but two items related to cognitive ability and four related to physical functions required revision to address participant confusion. The translations were refined based on the participants' feedback and expert recommendations. This study followed a rigorous translation process and included cognitive debriefing interviews to ensure linguistic and cultural equivalence. The availability of these tools in Arabic enhances cross-cultural research and practice in healthcare and contributes to a global understanding of cognitive and physical functions.

Health-related quality of life and experience measures, to assess patients’ experiences of peripheral intravenous catheters: a secondary data analysis

BackgroundPeripheral intravenous catheters (PIVCs) are essential for successful administration of intravenous treatments. However, insertion failure and PIVC complications are common and negatively impact patients’ health-outcomes and experiences. We aimed to assess whether generic (not condition-specific) quality of life and experience measures were suitable for assessing outcomes and experiences of patients with PIVCs.MethodsWe undertook a secondary analysis of data collected on three existing instruments within a large randomised controlled trial, conducted at two adult tertiary hospitals in Queensland, Australia. Instruments included the EuroQol Five Dimension - Five Level (EQ5D-5L), the Functional Assessment of Chronic Illness Therapy – Treatment Satisfaction – General measure (FACIT-TS-G, eight items), and the Australian Hospital Patient Experience Question Set (AHPEQS, 12 items). Responses were compared against two clinical PIVC outcomes of interest: all-cause failure and multiple insertion attempts. Classic descriptives were reported for ceiling and floor effects. Regression analyses examined validity (discrimination). Standardised response mean and effect size (ES) assessed responsiveness (EQ5D-5L, only).ResultsIn total, 685 participants completed the EQ5D-5L at insertion and 526 at removal. The FACIT-TS-G was completed by 264 and the AHPEQS by 262 participants. Two FACIT-TS-G items and one AHPEQS item demonstrated ceiling effect. Instruments overall demonstrated poor discrimination, however, all-cause PIVC failure was significantly associated with several individual items in the instruments (e.g., AHPEQS, ‘unexpected physical and emotional harm’). EQ5D-5L demonstrated trivial (ES < 0.20) responsiveness.ConclusionsInitial investigation of an existing health-related quality of life measure (EQ5D-5L) and two patient-reported experience measures (FACIT-TS-G; AHPEQS) suggest they are inadequate (as a summary measure) to assess outcomes and experiences for patients with PIVCs. Reliable instruments are urgently needed to inform quality improvement and benchmark standards of care.

Translation and Validation of a Vietnamese Version of the Colorectal Cancer Subscale for the Functional Assessment of Cancer Therapy - Colorectal Questionnaire.

There is a need for a validated Vietnamese translation of the colorectal cancer subscale (CCS) of the functional assessment of cancer therapy-colorectal (FACT-C) questionnaire to assess colorectal cancer-specific concerns of Vietnamese persons with colorectal cancer post-surgery. This study aims to translate and validate the CCS of FACT-C questionnaire in Vietnamese persons with colorectal cancer post-surgery. The nine-item CCS was translated following the functional assessment of chronic illness therapy (FACIT) translation methodology guidelines. Psychometric properties of a Vietnamese version of the CCS were evaluated with a sample of 135 participants who were randomly selected from three hospitals in Vietnam, utilizing a multistage sampling method. Construct validity was examined through confirmatory factor analysis (CFA), and reliability was assessed using Cronbach's α coefficients. These measures aimed to validate the psychometric properties of the Vietnamese nine-item CCS version. Descriptive statistics were used to analyze participant demographics with SPSS. The translated version demonstrated equivalence to the original English version. CFA results for the CCS Vietnamese version indicated that all 9 items were consistent with a unidimensional questionnaire (χ2 = 69.669, p > .05, df = 27, χ2/df = 2.58, RMSEA = .074, CFI = .917, TLI = .901, SRMR = .057). The Cronbach's α coefficient was .86, indicating high reliability. The Correlated Item-Total Correlation for the 9 items ranged from .39 to .76. The nine-item CCS Vietnamese version demonstrated appropriate translation, establishing its validity and reliability in measuring colorectal cancer-related concerns within the health-related quality of life among Vietnamese persons post-surgery.

Validation of functional assessment of chronic illness therapy - spiritual well-being scale (FACIT-Sp-12) in Serbian oncology patients

Validation of functional assessment of chronic illness therapy - spiritual well-being scale (FACIT-Sp-12) in Serbian oncology patients

Fatigue Assessment Using FACIT-F Scale in Spondyloarthropathy Patients and its Correlation with BASDAI and BASFI Scores.

To measure fatigue in axial spondyloarthropathy patients and find its correlation with the disease activity measures. Cross-sectional, descriptive study. Place and Duration of the Study: Rheumatology Unit, Federal Government Polyclinic Hospital, from November 2021 to May 2022. This study included 45 patients fulfilling the ASAS criteria for spondyloarthropathy. Bathankylosing spondylitis disease activity (BASDAI), Bath ankylosing spondylitis functional index (BASFI), and functional assessment of chronic illness therapy- fatigue (FACIT-F) scores were measured for each patient. In this study, there were 9 (20%) female patients and 36 (80%) male patients. There were 39 (86.7%) patients who had ankylosing spondylitis, 4 (8.9%) had axial spondyloarthropathy with peripheral arthritis and 2 (4.4%) had enthesitis-related juvenile idiopathic arthritis. The mean duration of the disease was 5.45 ± 4.19 years. Active disease with a BASDAI score of ≥4 was found in 16 (35.6%) patients while 29 (64.4%) had a BASDAI score <4. Severe fatigue with a FACIT-F score of <30 was found in 31 (68.9%) of the patients while less fatigue with FACIT-F score >30 was found in 14 (31.1%). The mean BASFI score of the cohort was 3.23 ± 2.01. Spearman's rho correlation analysis showed a significant strong correlation between the FACIT-F score, BASDAI and BASFI scores (p<0.001). Patients with active disease and higher BASFI scores had a lower FACIT-F score suggesting more fatigue, thus correlating with the disease activity. Bath ankylosing spondylitis disease activity (BASDAI), Functional assessment of chronic illness therapy-fatigue (FACIT-F), Ankylosing spondylitis (AS), Bath ankylosing spondylitis functional index (BASFI), Assessment in ankylosing spondylitis (ASAS).

Quality of Life Assessment and Its Relationship with Fatigue Symptom, Demographic and Clinical Factors in Rheumatoid Arthritis Polish Patients

The variety of clinical symptoms occurring during rheumatoid arthritis may reduce patients’ quality of life. Hence, the study aimed to assess RA patients’ quality of life and determine its relationship with fatigue, demographic and clinical factors. The study group consisted of 128 RA patients diagnosed according to ACR/EULAR criteria and treated in rheumatology departments. The Arthritis Impact Measurement Scales-2 (AIMS-2) scale was used to assess the quality of life. The Functional Assessment of Chronic Illness Therapy - Fatigue Scale was used to assess the symptom of fatigue. The analyzed variables were sex, age, pharmacological treatment, arthritis pain, morning stiffness, hemoglobin, CRP, rheumatoid factor, Ritchie Articular Index, and DAS28 disease activity. In the AIMS-2 scale, the patients had the lowest quality of life in the following subscales: arthritis pain (average 6.25±2.52 points), walking and bending (average 5.63±2.71 points), and emotional tension (average 4.99±1.92 points); while patients presented the highest quality of life for the following subscales: support from family and friends (average 1.39±2.21 points) and self-care (average 2.11±2.45 points). There was a correlation between the level of quality of life and age (r=0.24), morning stiffness (r=0.45), CRP concentration (r=0.29), joint tenderness (r=0.34), and disease activity (r=0.39). Increasing the values of parameters such as arthritis pain, disease activity, morning stiffness, and CRP level may cause a decrease in the level of quality of life. Assessment of the quality of life should be a permanent element of assessing patients with RA.

Financial Toxicity and Quality of Life in Patients Undergoing Stem-Cell Transplant Evaluation: A Single-Center Analysis.

We investigated the prevalence of financial toxicity in a population undergoing hematopoietic cell transplantation (HCT) evaluation and measured its impact on post-transplant clinical and health-related quality-of-life outcomes. This was a prospective study in patients undergoing evaluation for allogeneic HCT between January 1, 2018, and September 23, 2020, at a large academic medical center. Financial health was measured via a baseline survey and the comprehensive score for financial toxicity-functional assessment of chronic illness therapy (COST-FACIT) survey. The cohort was divided into three groups: none (grade 0), mild (grade 1), and moderate-high financial toxicity (grades 2-3). Health-related quality of life outcomes were measured at multiple time points. Multivariate logistic regression analysis evaluated factors associated with financial toxicity. Kaplan-Meier curves and log-rank tests was used to evaluate overall survival (OS) and nonrelapse survival. Of 245 patients evaluated for transplant, 176 (71.8%) completed both questionnaires (median age was 57 years, 63.1% were male, 72.2% were White, and 39.2% had myelodysplastic syndrome, 38.1% leukemia, and 13.6% lymphoma). At initial evaluation, 83 (47.2%) patients reported no financial toxicity, 51 (29.0%) with mild, and 42 (23.9%) with moderate-high financial toxicity. Patients with financial toxicity reported significant cost-cutting behaviors, including reduced spending on food or clothing, using their savings, or not filling a prescription because of costs (P < .0001). Quality of life was lower in patients with moderate-high financial toxicity at 6 months (P = .0007) and 1 year (P = .0075) after transplant. Older age (>62; odds ratio [OR], 0.33 [95% CI, 0.13 to 0.79]; P = .04) and income ≥$60,000 in US dollars (USD) (OR, 0.17 [95% CI, 0.08 to 0.38]; P < .0001) were associated with lower odds of financial toxicity. No association was noted between financial toxicity and selection for transplant, OS, or nonrelapse mortality. Financial toxicity was highly correlated with patient-reported changes in compensatory behavior, with notable impact on patient quality of life after transplant.

Abstract B144: Associations between experiences of discrimination and quality of life in a cohort of Black/African American breast cancer survivors

Abstract Introduction: Racism and discrimination have been associated with poorer mental and physical health, and decreased health-related quality of life (QoL) in the general population, however; there are limited studies examining the impact of discrimination in cancer survivors. We examined the relationship between experiences of discrimination and QoL in Black women diagnosed with breast cancer. Methods: Data were obtained from women enrolled in the Women’s Circle of Health Follow-up Study, a population-based cohort study of Black breast cancer survivors in New Jersey, enrolled within 10 months of diagnosis. Women included in these analyses were diagnosed between 2015-2019. Self-reported experiences of discrimination along with information on potential covariates and effect modifiers were collected during baseline in-person interviews. QoL was assessed at follow-up visits approximately 24 months post-diagnosis using a validated self-reported QoL of measure, the Functional Assessment of Cancer Therapy – Breast (FACT-B), which consists of 5 individual domains (emotional, functional, social/family, and physical wellbeing, as well as a disease specific domain for breast cancer). We examined the association of experiences of discrimination (none, low (&amp;lt;median 43%), high (≥ median 43%)) with continuous total QoL score using linear regression, adjusted for age at diagnosis, tumor stage, educational attainment, household income, smoking status, insurance, and body mass index. We also examined statistical interaction by neighborhood socioeconomic index (nSES), coping strategy (assessed using the Brief Resilient Coping Scale), and spirituality (assessed using the Functional Assessment of Chronic Illness Therapy - Spiritual Well-Being (FACIT-Sp-12) scale) using cross-product terms with experiences of discrimination. Results: Of 220 study participants, 73% of participants reported experiencing any discrimination. In age adjusted models, women who reported the highest experiences of discrimination had lower QoL for overall FACT-B scores and the subscale scores (no discrimination, mean overall FACT-B:117.7, 95% confidence interval (CI): 112.4, 122.9; high discrimination, mean overall FACT-B: 106.8, 95% CI: 102.1, 111.5). Similar associations were observed in fully adjusted models, where women who reported the highest experiences of discrimination had lower QoL. For example, for women who reported no discrimination the mean overall FACT-B score was 114.7 (95% CI: 107.9, 121.4) and 100.8 (95% CI: 94.2, 107.5) for women who reported the highest level of discrimination. No evidence of statistically significant interaction was observed for nSES, spirituality, or coping strategy. Conclusions: In a population-based study of Black breast cancer survivors, experiences of discrimination were associated with poorer QoL, as measured by FACT-B. Citation Format: Nur Zeinomar, Marley M. Perlstein, Bo Qin, Hari S. Iyer, Jesse J. Plascak, Christine B. Ambrosone, Kitaw Demissie, Chi-Chen Hong, Elisa V. Bandera. Associations between experiences of discrimination and quality of life in a cohort of Black/African American breast cancer survivors [abstract]. In: Proceedings of the 16th AACR Conference on the Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved; 2023 Sep 29-Oct 2;Orlando, FL. Philadelphia (PA): AACR; Cancer Epidemiol Biomarkers Prev 2023;32(12 Suppl):Abstract nr B144.

Abstract A093: Implementation of a financial hardship screening among Native American patients with cancer

Abstract Introduction: Financial hardship and financial hardship screening are emerging concerns in oncology. Native American patients may be at increased risk of financial hardship due to factors including poverty, medical comorbidities, and lack of private health insurance coverage. Financial hardship for Native American patients with cancer has only rarely been studied, and implementation of screening for financial hardship for these patients has never been reported. Methods: Guided by input from a stakeholder advisory board consisting of patient, provider, and staff members at a single cancer center in Oklahoma, we implemented a financial hardship screening tool, the COmprehensive Score for financial Toxicity (COST) – Functional Assessment of Chronic Illness Therapy (FACIT) among 42 Native American patients with cancer. We conducted key informant interviews with ten of these patients and four clinical staff involved in the implementation process. Patient interviews included questions about current financial hardship, experiences in discussing financial hardship with the cancer care and primary care team, and acceptability of the COST-FACIT tool. Clinician interviews focused on their experience with the project, including implementation of the COST-FACIT tool, barriers to implementation, and sustainability. Recorded interviews were transcribed and thematically analyzed using MAXQDA® software. COST-FACIT tool data were analyzed using SAS v. 9.4 Results: Three-quarters (76%, n=32) of participants who completed the COST-FACIT tool reported moderate or severe financial hardship (Score &amp;lt;25 out of max 44, with lower scores indicating more hardship). A higher percentage of patients experiencing financial hardship were younger, covered by Medicaid, unemployed, not married/cohabiting, had lower income, and lower education compared to those with no/mild hardship (Scores ≥25). Among patients and clinicians, we identified themes of 1) financial hardship screening perception and intervention experiences, 2) screening efficacy and opportunities for improvement, and 3) systematic and patient related themes of Native American health systems and culture. Conclusions: Financial hardship was reported by the majority of Native American patients with cancer included in our study. Patients expressed a positive experience with the screening tool, including identification of financial challenges though timing of the tool in regards to their cancer diagnosis varied across patients. Clinicians experienced challenges in implementation of the COST-FACIT tool related to logistics, the COVID-19 pandemic, and concerns for sustainability. However, clinicians reported a positive experience with the tool and interactions with patients and referrals that resulted from the screening. Citation Format: Amanda E. Janitz, Amber S. Anderson-Buettner, Stefani D. Madison, Mark P. Doescher, Dorothy A. Rhoades. Implementation of a financial hardship screening among Native American patients with cancer [abstract]. In: Proceedings of the 16th AACR Conference on the Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved; 2023 Sep 29-Oct 2;Orlando, FL. Philadelphia (PA): AACR; Cancer Epidemiol Biomarkers Prev 2023;32(12 Suppl):Abstract nr A093.

Dutch–Flemish translation and validation of the gastrointestinal symptom scales from the patient‑reported outcomes measurement information system (PROMIS)®

PurposeTo translate the eight PROMIS® GastrointestinaI Symptom Scales into Dutch–Flemish and to evaluate their psychometric properties.MethodsThis study consisted of two parts: (1) translation according to the Functional Assessment of Chronic Illness Therapy (FACIT) translation methodology and (2) evaluation of psychometric properties: structural validity, using confirmatory factor analysis; and construct validity using hypothesis testing.ResultsIn the first part of the study, in 19 out of the 77 items (24.7%) translation was challenging. After discussion between the translators, consensus could be achieved. In the cognitive debriefing interview phase, ten minor changes in the wording of items were made. A universal Dutch–Flemish translation for all 77 items was obtained. In de second part of the study a good fit was found for three DF-PROMIS GI Scales: Bowel Incontinence, Gas and Bloating, and Belly Pain. Four scales (Reflux, Disrupted Swallowing, Diarrhea, and Constipation) did not show sufficient fit and fit for the Nausea and Vomiting scale could not be assessed because of skewed responses. Construct validity was considered sufficient for six out of eight DF-PROMIS GI Scales. Less than 75% of hypothesis for de Constipation and Disrupted Swallowing scales could be confirmed.ConclusionThe PROMIS GI Symptom Scales were successfully translated into DutchFlemish. The findings suggest a sufficient structural validity for the PROMIS GI Scales. Bowel Incontinence, Gas and Bloating and Belly Pain. Construct validity was sufficient for the Scales Gas and Bloating, Incontinence, Nausea and Vomiting, Reflux, Belly Pain, and Diarrhea.

Improvement of Patient-Reported Outcomes Among Heavily Pretreated Patients with Lower-Risk Myelodysplastic Syndromes and High Transfusion Burden Treated with Imetelstat on the IMerge Phase 3 Trial

CONCLUSIONS Transfusion-dependent anemia and related fatigue are commonly associated with lower-risk myelodysplastic syndromes (LR-MDS) and frequently not improved with therapy. Patients with symptoms associated with anemia have an impaired quality of life and inferior overall survival. In IMerge (NCT02598661), the phase 3, randomized, global trial of patients with LR-MDS and a red blood cell transfusion burden of ≥4 U, imetelstat-treated patients had a significantly higher transfusion independence rate than those treated with placebo and showed sustained improvement in patient-reported fatigue via the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue (Sekeres et al. EHA 2023. P732). Here, we assess the impact of imetelstat on several patient-reported outcomes (PROs) relevant to LR-MDS. Functional Assessment of Cancer Therapy-Anemia (FACT-An; 55 items) and Quality of Life in Myelodysplasia Scale (QUALMS; 38 items) were the main questionnaires used. Analyses included symptom-specific derived scores for the following parameters: dyspnea, measured by 1 FACT-An and 1 QUALMS item; physical function and systemic symptoms, by 3 FACT-An items each; pain, by 2 FACT-An items; and bleeding, by 1 QUALMS item. Higher scores indicated improvement. QUALMS total score and physical burden score were also analyzed. Psychometric analyses were conducted using blinded interim data to document the measurement properties of these item sets and define the scores that would be used to specify PRO end points in IMerge; all analyses were exploratory and were conducted in the intention-to-treat (ITT) population. The overall effect of imetelstat on each score was evaluated from the repeated measurement mixed model (RMMM) comparing the least-squares means (LSMs) of change in score estimated in the 2 treatment groups using all available data while on treatment up to cycle 30. A positive mean change in the dyspnea score was observed throughout the treatment, up to cycle 24, in the imetelstat group but not in the placebo group (Figure). The RMMM analysis showed an overall change in dyspnea score from baseline of 0.53 (by LSM with 95% CI, 0.241-0.825) with imetelstat versus −0.40 (by LSM with 95% CI, −0.794 to 0.003) with placebo, with a significant difference between the treatment groups (LSM difference, 0.93; 95% CI, 0.446-1.411; P &amp;lt; .001; Table). The model-based mean change estimates for each score showed that, despite having high transfusion burden at baseline, patients in the imetelstat group did not experience more deterioration in any of the other outcomes than those in the placebo group (Table). In addition, the QUALMS composite scores suggested significantly improved health outcomes with imetelstat versus placebo based on RMMM. LSM difference was 4.66 (95%CI, 0.862-8.461; P = .016) and 6.34 (95% CI, 1.771-10.913; P = .007) for QUALMS total and QUALMS physical burden, respectively (Table). Findings from multiple validated PRO questionnaires consistently show that patients with red blood cell transfusion-dependent LR-MDS enrolled into the IMerge randomized trial who were treated with imetelstat reported improved fatigue, dyspnea, and QUALMS composite scores (total and physical burden) compared with those in the placebo group. These data indicate that, in addition to improving transfusion burden in patients with LR-MDS, imetelstat targets multiple core symptoms of LR-MDS simultaneously, also improving PROs.

Quality of Life Analysis in Patients with AA Using the QLQ-AA/PNH-54 at Initial Diagnosis of AA - a Registry Based Analysis

Objectives: Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are ultra-rare, acquired bone marrow failure syndromes (BMFS) with need for long-term care and high morbidity despite recent therapeutic advances. There is a lack of disease-specific assessment tools of quality of life (QoL) in AA/PNH patients. So far, QoL has mostly been estimated according to surrogate parameters (e.g. blood counts, transfusion dependence or toxicity of treatment in AA patients) or has been assessed with tools specifically designed for cancer patients (e.g. EORTC Quality of Life Questionnaire QLQ-C30) or with tools focusing on fatigue (e.g. Functional Assessment of Chronic Illness Therapy, FACIT-Fatigue, PNH patients). Therefore, the QLQ-AA/PNH-54 has been developed to allow a patient tailored QoL assessment in patients with AA or PNH. Here, we present the first descriptive data of the QLQ-AA/PNH-54 of 74 AA patients at the timepoint of initial diagnosis collected between 2018 and 2020 (prior to the SARS-CoV-2 pandemic). Methods: The QLQ-AA/PNH-54 was designed, psychometrically validated, and is currently finally validated for PNH within the IPIG (international PNH interest group)-registry. Parallel to this process, the tool was implemented within the Aachen AA/BMF registry as an instrument for real-life QoL evaluation in AA patients. In total, 54 QoL items regarding physical functioning (21 items), psychological aspects (26 items) and health care related issues (7 items) are comprised. Patients rank the occurrence of each item within the last 14 days on a scale between 0 and 3 (0 = did not occur, 1 = low, 2 = moderate, 3 = severe). Results: 74 AA patients (37 female/ 37 male, mean/median/range age 38.42/ 35.47/ 11-73 years) were included. Patients were diagnosed with nsAA (n=11), sAA (n=23), vsAA (n=18), AA NOS (n=20) and AA/PNH-Overlap (n=2), respectively. Broken down into different functional scales, the most frequently and highest rated scales concerned psychosocial aspects (&amp;gt; 60 % of the patients scored 1-3; &amp;gt; 30% of the patients scored 3). Patients were especially burdened by worries and uncertainties with regards to their future, activity planning security and need for cautiousness in everyday life. Tiredness was the major symptom reported in relation to physical functioning. Patients often feared deterioration of their blood results as the most important health care-related issue. In comparison, low occurrence of pain, orthostatic dysregulation, susceptibility for infections, dyspnea, sleep disturbance and tendency to bleed was reported regarding physical functioning (scored 0 by over 40 to over 50 % of the patients). Similarly, low scores were reported for impairment of activities of daily life in terms of completion of household tasks and hobbies or loss of physical attraction. Conclusion: QoL of AA patients at initial diagnosis was mainly impaired by tiredness and a high psychological burden. Interestingly, complications frequently feared and highlighted by treating physicians had a minor impact on patients' QoL. These results emphasize the importance of further QoL analysis from patient view and recommend to especially focus on the psychological burden of AA patients in patient communication at initial diagnosis.

Efficacy and Safety Is Maintained in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan for up to 3 Years

Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease characterized by complement-mediated hemolysis and thrombosis. Pegcetacoplan, the first complement component 3 (C3) inhibitor for PNH, increased hemoglobin (Hb) levels in complement component 5 inhibitor (C5i)-experienced and -naive adult patients with PNH in 2 phase 3 clinical trials (PEGASUS [NCT03500549] and PRINCE [NCT04085601], respectively). This integrated analysis of data from the pivotal phase 3 trials and the subsequent open-label extension (study 307; NCT03531255) evaluated the long-term efficacy and safety of pegcetacoplan for PNH treatment. Methods: For this integrated analysis, baseline was defined as the initiation of pegcetacoplan, regardless of when in the trials this occurred. Patients initially received pegcetacoplan 1080 mg subcutaneously twice weekly; dose escalations to once every 3 days or 3 times weekly were permitted. Efficacy was evaluated from baseline up to Weeks 132 (2.5 years, PRINCE) and 156 (3 years, PEGASUS) by Hb, lactate dehydrogenase (LDH; upper limit of normal [ULN] 226 IU/L), absolute reticulocyte count (ARC), and indirect bilirubin values; Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores; and transfusion avoidance rates (percentages of patients who did not require a transfusion during treatment). Safety was assessed by incidence of adverse events (AEs) and serious AEs (SAEs) during pegcetacoplan monotherapy for up to 3 years. Results: Of the 133 patients in the phase 3 trials (PEGASUS, 80; PRINCE, 53), 114 enrolled in the extension study (PEGASUS, 64; PRINCE, 50). At least 75% had received a transfusion in the year before enrolling in PEGASUS or PRINCE. Prior to pegcetacoplan initiation, mean (SD) Hb levels were 8.95 (1.09) g/dL in PEGASUS, 9.27 (1.44) g/dL in PRINCE, and 9.08 (1.24) g/dL in the total population; median (interquartile range) LDH levels were 217.0 (184.8, 276.5) IU/L in PEGASUS, 1964.0 (1409.0, 2503.3) IU/L in PRINCE; and mean (SD) FACIT-Fatigue scores were 31.6 (11.7) in PEGASUS, 36.6 (10.0) in PRINCE, and 33.6 (11.3) in the total population. After pegcetacoplan initiation, mean Hb levels markedly improved from baseline and remained stable through Weeks 132 (2.5 years, PRINCE) and 156 (3 years, PEGASUS) ( Figure). Median LDH rapidly decreased and stabilized below the ULN ( Figure). Improvements in ARC and indirect bilirubin were similarly maintained. Average FACIT-Fatigue scores increased (indicating less fatigue) rapidly and were maintained near the general population norm of 43.6. Annual transfusion avoidance ranged from 71%-79% in PEGASUS and 80%-86% in PRINCE, with 52% of patients from PEGASUS avoiding transfusion for up to 3 years of pegcetacoplan treatment and 67% of patients from PRINCE avoiding transfusion for up to 2.5 years. Overall, &amp;gt;92% patients had compliance rates of at least 95%. Over 3 years, most patients experienced an AE; SAEs were reported in 50.0% of patients, with 4.5% experiencing a SAE deemed related to pegcetacoplan. Overall, 17 patients discontinued pegcetacoplan due to an AE; of those, 9 discontinued due to a hemolytic disorder, 7 of which discontinued within 1 year of starting pegcetacoplan. Over 3 years, 4 (3.0%) deaths occurred, none were deemed related to pegcetacoplan; 3 (2.3%) patients experienced a thrombotic event (in the context of multiple associated comorbidities or discontinuation of pegcetacoplan) and no cases of meningitis were reported. Overall, no new or unexpected safety findings were identified. Conclusions: Rapid improvements in Hb, LDH, ARC, indirect bilirubin, and FACIT-Fatigue values were observed and maintained up to 3 years in C5i-experienced patients and up to 2.5 years in C5i-naive patients. The transfusion burden was significantly reduced. No new safety findings were identified. This analysis demonstrates sustained efficacy and continued safety of long-term pegcetacoplan for patients with PNH, regardless of prior C5i treatment.

Cold Agglutinin Disease (CAD) Real-World Evidence (CADENCE) Registry: Update to the Study Design of the First International, Prospective CAD Registry

Cold agglutinin disease (CAD) is a rare chronic autoimmune hemolytic anemia (AIHA) characterized by classical complement pathway (CP)-mediated hemolysis. CAD is associated with a substantial disease burden negatively impacting quality of life (QoL), including severe fatigue, and increased risk of both thromboembolic events and early mortality. Due to the rarity of CAD, there is scarce information regarding its natural history, long-term impacts, and complications. Sutimlimab, a humanized monoclonal antibody that selectively inhibits the CP, is the first approved treatment for CAD. In the CARDINAL and CADENZA Phase 3 trials, sutimlimab reduced hemolysis, anemia, was generally well tolerated, and meaningfully improved QoL. The CADENCE Registry (NCT05791708) is a large international database of patients with CAD or cold agglutinin syndrome (CAS, a cold antibody-driven hemolytic anemia associated with an underlying condition). First launched in 2019, the first study patient was enrolled in 2022 following a COVID-19 pandemic-related hiatus. Presented here is an update on the current CADENCE protocol and study design. CADENCE is a multinational, multicenter, observational, prospective, longitudinal registry study enrolling patients ≥18 years of age diagnosed with CAD (diagnostic criteria: a monospecific direct antiglobulin test strongly positive for C3d and negative or weakly positive for immunoglobulin G and a cold agglutinin titer ≥1:64) or CAS (CAD diagnostic criteria, with causative infection, autoimmune disorder or overt malignancy). Exclusion criteria include patients with a diagnosis of warm or mixed AIHA and those actively participating in a CAD/CAS interventional trial. The main study objective is to better understand patient demographics, clinical characteristics, treatment patterns, healthcare resource utilization (HRU), natural history of disease, long-term clinical outcomes, and impact on patient QoL. A sutimlimab-treated cohort sub-study is ongoing to assess the real-world safety and effectiveness of sutimlimab in patients with CAD, fulfilling the regulatory commitment for a post-authorization safety study. The enrollment period is expected to last ~3 years and patients will be followed for 3 years after the last patient enrolled. Data collection will include both clinical assessments as per standard of care and patient-reported outcome (PRO) data collected at enrollment and at ~6-month interval follow-ups. Additional safety data will be collected for the sutimlimab cohort. The CADENCE Registry's first patient enrolled (after study restart) was in Q2 2022, with last patient's first visit expected in Q2 2025 and last patient's last visit expected in 2028 ( Figure). The registry will collect data on ~400 patients with CAD/CAS across ~90 sites in 8 countries: Austria, France, Germany, Italy, Japan, Spain, the UK, and the US. Among them, ≥30 patients with CAD treated with sutimlimab are expected to take part in the cohort study. Key data to be collected include clinical characteristics, disease complications, comorbidities, mortality, treatment regimens, HRU, and PROs. Biomarkers for hemolysis will be assessed, including lactate dehydrogenase, haptoglobin, total and indirect bilirubin. In the sutimlimab cohort, data will also be collected on adverse events (AEs), serious AEs, and AEs of special interest (serious infections, Raynaud's, arterial hypertension, autoimmune disorders, hypersensitivity/anaphylaxis, exposure in pregnancy, overdose). PRO measures include the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue, the 36-item Short Form Health Survey (SF-36), and the CAD symptoms and impact questionnaire (CAD-SIQ), a novel 11-item questionnaire specifically designed for CAD. Interim analyses will occur annually. Final analyses will be performed at study end both at regional and global levels. As of July 13, 2023, 47 of 82 targeted sites have been activated, and 113 of 405 targeted patients (CAD/CAS) have been enrolled, including 13 sutimlimab-treated patients. CADENCE is the first global registry for patients with CAD and will help advance understanding of the natural history and disease process of this rare condition. This registry will also assess the long-term safety and effectiveness of sutimlimab in patients with CAD in a real-world setting, providing clinicians with data that may help improve treatment practices.

Fatigue and health-related quality of life in patients with immune thrombocytopenia: a longitudinal assessment in China

ABSTRACT Background There is lacking studies of longitudinally assessment of fatigue and health-related quality of life (HRQoL) among Chinese immune thrombocytopenia (ITP) adults. We aimed to evaluate changes in fatigue and HRQoL and identify the associated factors. Methods Patients’ characteristics, Functional Assessment of Chronic Illness Therapy (FACIT-F) and the ITP-specific Patient Assessment Questionnaire (ITP-PAQ) scores at admission (T0), at discharge (T1), and three months after discharge (T2) were collected. Linear mixed effects models were used to examine changes over time. Results We included 175 patients. The mean score of FACIT-F at T0 was 37.2 and increased at T1 (39.0), while then decreased at T2 (34.7). Patients who were single, retired, had persistent ITP, splenomegaly had more severe fatigue, whereas those who had not received any prior treatment and had a bleeding score of 0 at admission had milder fatigue. The mean score of ITP-PAQ was 57.7 at T0, then gradually increased to 60.3 at T1 and 62.8 at T2. Patients with persistent ITP and those who have never received treatment for ITP have better HRQoL. Conclusion ITP adults’ fatigue and HRQoL were impaired. Patients’ fatigue improved at discharge but worsened at three months after discharge, while HRQoL gradually improved over time.

Real-World Treatment Outcomes of Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated with Pegcetacoplan and Complement Protein 5 Inhibitors (C5i)

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, potentially fatal disease characterized by complement-mediated hemolysis which clinically manifests with symptoms including fatigue, weakness and, in severe cases, thrombosis. Previous disease-modifying treatments solely comprised of complement protein 5 inhibitors (C5i) which effectively reduce intravascular hemolysis (IVH) in most patients with PNH. However, due to residual IVH and emerging extravascular hemolysis (EVH), complement protein 3 inhibitor (C3i), pegcetacoplan (PEG) has been recently approved to target the complement cascade further upstream, tackling both IVH and EVH. The aim of this study was to report real-world outcomes of PEG-treated and C5i-treated patients. Data were drawn from the Adelphi PNH Disease Specific Programme (DSP)™, a real-world, cross-sectional survey of physicians and their patients with PNH in France, Italy, Germany, Spain and the United States of America from January - November 2022. Patients were eligible for inclusion if they had received PEG for ≥1 month. Physicians recorded patient demographics, hemoglobin (Hb) levels, treatment administration and hospitalizations. The same patients were invited to self-report on symptomatology, fatigue via the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) questionnaire, and impact on work and productivity via the Work Productivity and Activity Impairment (WPAI) questionnaire. Descriptive statistics were reported for patients receiving PEG from the DSP and from previously published data describing patients receiving C5i treatment (Panse, J et al. Eur J Haematol. 2022 Oct;109(4):351-363 1; Sicre de Fontbrune F et al. Hematology. 2022 Dec;27(1):1140-1151 2). Fourteen physicians reported data for 61 patients receiving PEG. Mean (standard deviation; SD) age was 37.1 (11.3) years and 59.0% of patients were male. Mean (SD) time since diagnosis was 3.7 (3.3) years and mean (SD) time receiving PEG was 5.9 (4.0) months. All patients received 1080 mg dose of PEG, with 98.3% of patients receiving this dose twice weekly. For PEG-treated patients mean (SD) Hb from the most recent result was 11.5 (1.6) g/dL and 34.4% of patients had Hb &amp;gt;12 g/dL. Published literature based on a real-world survey reported C5i-treated patients had a mean (SD) Hb of 10.2 (2.0) g/dL and 14.3% had Hb &amp;gt;12 g/dL 1. C5i-treated patients had mean (SD), 0.4 (1.0) emergency room (ER) visits during the 12-months prior to data collection 2. In the DSP sample, no patients required ER visits during the time they were receiving PEG. Thirty PEG-treated patients provided self-reported data with less reporting to experience shortness of breath (23.3% vs 46.5%), difficulty swallowing (10.0% vs 19.7%) and abdominal pain (16.7% vs 19.7%) at the time of survey than C5i-treated patients 1. At the time of survey 83.3% of PEG-treated patients reported fatigue (63.4% C5i patients in the literature 1). PEG-treated patients reported a mean (SD) FACIT-F score of 37.4 (8.6). C5i-treated patients reported 35.0 (13.7) 1. 73.5% of PEG-treated patients were employed, 2.5% reported absenteeism and 22.4% reported overall activity impairment. For previous literature regarding C5i patients this was 57.7%, 10.2% and 37.5%, respectively 1. We assessed previously published literature of C5i-treated patients and demonstrated favorable outcomes for PEG-treated patients across the endpoints assessed in this analysis. Patients receiving PEG reported higher Hb and FACIT-Fatigue scores than C5i-treated patients. More PEG-treated patients remained in paid employment and reported less impact on their daily activities than C5i-treated patients. Further research is required to explore the real-world effectiveness of PEG compared to other therapeutic options and understand how newer therapies can improve outcomes for patients with PNH.

Evaluation of the effects of somatotype profiles on pain, proprioception, isokinetic muscle strength and kinesiophobia in patients with meniscopathy.

Somatotype causes differentiation of physical, physiological and biochemical metabolisms in the body. To what extent meniscopathy (M) is affected by somatotype profiles has been an issue of concern. The aim of the study was to investigate whether somatotype profiles have an effect on kinesiophobia, pain, proprioception and isokinetic muscle strength in patients with an M diagnosis. 172 (85 female, 87 male) M patients between the ages of 18 and 65 were included in the study. The Heath-Carter method was used to determine somatotype components. Biodex Isokinetic system at 120∘/sec angular speed was used for muscle strength measurements, a digital inclinometer with goniometer was used for proprioception measurement, the Tampa Kinesiophobia Scale (TKS) was used for the assessment of kinesiophobia, the Functional Assessment of Chronic Illness Therapy (FACIT) fatigue scale was used for the assessment of fatigue and pain, and the visual analogue scale (VAS) was used for pain assessment. Five somatotype profiles were found. When the right-left knee proprioception values were compared according to the somatotype profiles of patients, a significant difference was found in favor of balanced ectomorph at 15∘ and 30∘. No significant difference was found when TKS, FACIT, VAS values were compared in terms of somatotype profiles; while no significant difference was found in TKS, FACIT, VAS (REST-NIGHT) in terms of gender, a significant difference was found in VAS MOVE. Endomorph somatotype was dominant in the M patients. Individuals with M disease showed significant differences in terms of endomorph components. Obesity may also be one of the negative findings for M disease. Somatotype classification may represent a suitable tool for monitoring M.

Glucocorticoid Withdrawal Symptoms and Quality of Life in Patients with Systemic Lupus Erythematosus.

SLE patients whose prednisolone had been previously withdrawn or taken <5 mg/day were enrolled. Serum morning cortisol levels were collected after 72-hour GCS discontinuation, and low-dose ACTH stimulation test (LDST) was performed. Patient report outcomes (PROs) included SLE-specific quality of life questionnaire (SLEQoL), functional assessment of chronic illness therapy (FACIT), patient health questionnaire (PHQ-9), and Pittsburgh's sleep quality index (PSQI). Serum morning cortisol of 100 SLE patients was tested. Most patients were female (88%). Seventy-four patients showed remission. The mean ± SD of prednisolone was 0.73 ± 1.08 mg/day. Total SLEQoL and FACIT (mean ± SD) of all patients were 67.05 ± 26.15 and 13.7 ± 8.87, respectively. Eighteen percent of patients had moderate-severe depressive symptoms, and 49% were poor sleepers. Adrenal function was determined by LDST in only 39 patients; 5 patients (12.8%) were adrenal insufficiency (AI), and 34 patients were normal adrenal function. Compared to normal adrenal function patients, SLE patients with AI had higher proportion of moderate-severe depressive symptom (PHQ - 9 > 9), but not statistically significant (40% vs. 20.6%, p = 0.34). PROs were comparable between groups. Independent factors associated with SLEQoL were FACIT (adjusted β 1.31, 95% CI 0.76, 1.86, p < 0.001), PHQ-9 (adjusted β 5.21, 95% CI 4.32, 6.09, p < 0.001), and PSQI (adjusted β 4.23, 95% CI 3.01, 5.45, p < 0.001), but not with AI (adjusted β -5.2, 95% CI -33.26, 22.93, 0.71, p = 0.71). SLE patients with previous GCS exposure could experience AI and withdrawal symptoms such as sleep disturbance and depression during discontinuation of low-dose GCS. Fatigue, depression, and poor sleeper were significantly associated with poor SLEQoL.

Patient-Reported Outcomes (PROs) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Eculizumab: Results from the Phase III Randomized COMMODORE 2 and COMMODORE 1 Trials

Introduction COMMODORE 2 (NCT04434092) and COMMODORE 1 (NCT04432584) are global, randomized, open-label, multicenter Phase III trials evaluating crovalimab (crova) vs eculizumab (ecu) in patients (pts) with PNH. COMMODORE 2 tested the non-inferiority of crova vs ecu in C5i-naive pts and demonstrated that crova was non-inferior to ecu for hemolysis control, transfusion avoidance, breakthrough hemolysis, and hemoglobin stabilization (Röth EHA 2023; #S181). In both studies, crova was well tolerated for both C5i-naive and C5i-experienced pts (Röth EHA 2023; #S181, Scheinberg EHA 2023; #S183). To investigate the efficacy of crova vs ecu from a pt perspective, PRO tools were used to assess fatigue, PNH-related symptoms, functioning, and global health status/quality of life (GHS/QoL) among COMMODORE 2 and COMMODORE 1 pts. Methods In COMMODORE 2, C5i-naive pts were randomized 2:1 to receive crova or ecu during a 24-week primary treatment period. In COMMODORE 1, C5i-experienced pts receiving ecu at study start were randomized 1:1 to receive crova or ecu during a 24-week primary treatment period. Pts received crova as a weight-based tiered regimen (Liu ASH 2022; #293) that included loading doses followed by maintenance doses via subcutaneous injection every 4 weeks, or ecu via intravenous infusion every 2 weeks (900 mg per label). After completing 24 weeks of treatment, COMMODORE 1 and 2 pts randomized to crova continued crova, and those randomized to ecu switched to crova if continuing in the extension period. Paper PRO questionnaires were completed by adult pts at baseline (BL) and at Weeks 2, 5, 9, 17, and 25 during the primary treatment period (up to Week 25) and during the crova extension period (from Week 25 onwards). Fatigue was assessed with the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale (higher scores indicate lower fatigue severity). Additional PNH symptoms (dyspnea, dysphagia, headaches, abdominal pain, chest pain, and erectile dysfunction) were assessed using relevant questions selected from the European Organisation for Research and Treatment of Cancer (EORTC) Item Library (EORTC IL-40; higher scores indicate worse symptoms). Physical function, role function, and GHS/QoL were assessed with the EORTC Quality of Life Questionnaire Core 30 (EORTC QLQ-C30; higher scores indicate better functioning/quality of life). Results For the primary analysis, COMMODORE 1 randomized 45 pts to crova and 44 to ecu, and enrolled 38 pts in the descriptive arm; COMMODORE 2 randomized 135 pts to crova and 69 to ecu. In COMMODORE 2, C5i-naive pts in both arms showed rapid and sustained improvement across all PRO measures during the primary treatment period. Clinically meaningful improvement (≥5 points; Cella ASH 2021; #1952) in mean FACIT-Fatigue scores from BL occurred in both arms, with numerically higher improvement with crova (crova: 7.8, 95% CI: 6.5, 9.1; ecu: 5.2, 95% CI: 3.4, 6.9; Table 1). Mean fatigue levels of pts treated with crova improved by Week 2 and were similar to normative population values by Week 25. Analysis of the individual FACIT-Fatigue items was also conducted. Additionally, improvements were observed for both treatment arms in all EORTC IL-40 symptoms and in the functioning and GHS/QoL scales of the EORTC QLQ-C30, with numerically greater improvement with crova for most scales (Table 2). The improvements with crova treatment were maintained during the crova extension period. In COMMODORE 1, C5i-experienced pts randomized to receive crova or ecu maintained BL levels of fatigue, PNH symptoms, functioning, and GHS/QoL scores throughout the primary treatment period (Tables 1 and 2) and with crova treatment during the extension period. There was no meaningful change in the mean values of any PRO scores after pts switched from ecu to crova at Week 25. Conclusions C5i-naive pts inCOMMODORE 2 experienced rapid and sustained improvements from BL in fatigue, PNH symptoms, functioning, and GHS/QoL scores while on treatment with crova and ecu, with numerically greater improvement in fatigue and most GHS/QoL scales in the crova arm. C5i-experienced pts in COMMODORE 1 maintained BL levels during the first 24 weeks of treatment with crova or ecu. These trends were maintained after pts were switched from ecu to crova. These data provide supportive evidence for the treatment benefit of crovalimab for self-reported fatigue and quality of life.