Holistic assessment of cochlear implant outcomes using the international classification of functioning disability and health model: data analysis of a longitudinal prospective multicenter study.

PURPOSE: To study outcome after cochlear implantation using the Cochlear Implant (CI) outcome assessment protocol based on the International Classification of Functioning, Disability and Health (ICF) model (CI-ICF). METHODS: Raw data of a prospective, longitudinal, multicenter study was analyzed. Seventy-two CI candidates were assessed preoperatively and six months postoperatively using the CI-ICF protocol. Following tools were used: (1) Work Rehabilitation Questionnaire (WORQ), (2) Abbreviated Profile of Hearing Aid Benefit (APHAB), (3) Audio Processor Satisfaction Questionnaire (APSQ), (4) Speech, Spatial, and Qualities of Hearing Scale (SSQ12), (5) Hearing Implant Sound Quality Index (HISQUI19), (6) Nijmegen CI Questionnaire (NCIQ) (7) pure tone audiometry, (8) speech audiometry, (9) sound localization. RESULTS: There was a significant improvement of speech discrimination in quiet (p = 0.015; p < 0.001) and in noise (p = 0.041; p < 0.001), sound detection (p < 0.001), tinnitus (p = 0.026), listening (p < 0.001), communicating with-receiving-spoken messages (p < 0.001), conversation (p < 0.001), family relationships (p < 0.001), community life (p = 0.019), NCIQ total score and all subdomain scores (p < 0.001). Subjective sound localization significantly improved (p < 0.001), while psychometric sound localization did not. There was no significant subjective deterioration of vestibular functioning and no substantial change in sound aversiveness. CI users reported a high level of implant satisfaction postoperatively. CONCLUSION: This study highlights the positive impact of cochlear implantation on auditory performance, communication, and subjective well-being. The CI-ICF protocol provides a holistic and comprehensive view of the evolution of CI outcomes.

Determinants of low health-related quality of life in patients with myelodysplastic syndromes: EUMDS Registry study.

Patients with myelodysplastic syndromes (MDS) frequently experience a significant symptom burden, which reduces health-related quality of life (HRQoL). We aimed to identify determinants of low HRQoL in patients recently diagnosed with MDS, for guiding early intervention strategies. We evaluated longitudinal data in 2205 patients with MDS during their first year after diagnosis. Median values of EQ-5D 3-level (EQ-5D-3L) index (0.78) and visual analog scale (VAS) score (0.70) were used as thresholds for low HRQoL. In addition, the 5 dimensions of EQ-5D-3L were analyzed for impairments (any level vs "no problem" category). After multiple imputation of missing values, we used generalized estimating equations (GEE) to estimate odds ratios (OR) for univariable determinant screening (P < .15), and to subsequently derive multivariable models for low HRQoL with 95% confidence intervals (CI). Multivariable GEE analysis showed the following independent determinants (OR, 95% CI) for low EQ-5D index: increased age (60-75 years: 1.33, 1.01-1.75; >75: 1.84, 1.39-2.45), female sex (1.70, 1.43-2.03), high serum ferritin level (≥1000 vs ≤300 µg/L: 1.41, 1.06-1.87), comorbidity burden (per unit: 1.11, 1.02-1.20), and reduced Karnofsky performance status (KPS, per 10 units: 0.62, 0.58-0.67). For low VAS score, additional determinants were transfusion dependence (1.53, 1.03-2.29), low hemoglobin <10 g/dL (1.34, 1.12-1.61), and high body mass index (≥30 vs 23-29.9 kg/m2: 1.26, 1.02-1.57). Sex, KPS, comorbidity burden, hemoglobin count, and transfusion burden were determinants for all EQ-5D dimensions. Low HRQoL is determined by multiple factors, which should be considered in the management and shared decision making of patients with MDS. This trial was registered at www.clinicaltrials.gov as #NCT00600860.

Raising the standards of patient-centered outcomes research in myelodysplastic syndromes: Clinical utility and validation of the subscales of the QUALMS from the MDS-RIGHT project.

BACKGROUND: Clinical decision-making for patients with myelodysplastic syndromes (MDS) is challenging, and both disease and treatment effects heavily impact health-related quality of life (HRQoL) of these patients. Therefore, disease-specific HRQoL measures can be critical to harness the patient voice in MDS research. METHODS: We report a prospective international validation study of the Quality of Life in Myelodysplasia Scale (QUALMS) with a main focus on providing information on the psychometric characteristics of its three subscales: physical burden (QUALMS-P), emotional burden (QUALMS-E), and benefit finding (QUALMS-BF). The analysis is based on patients enrolled from three European countries and Israel, participating to the MDS-RIGHT Project. The scale structure and psychometric properties of the QUALMS were assessed. RESULTS: Overall, 270 patients with a median age of 74 years were analyzed and the majority of them (60.3%) had a low MDS-Comorbidity Index score. Results of the confirmatory factor analysis supported the underlying scale structure of the QUALMS, which, in addition to a total score, includes three subscales: QUALMS-P, QUALMS-E, and the QUALMS-BF. The QUALMS-P exhibited the highest Cronbach's alpha coefficients. Discriminant validity analysis indicated good results with the QUALMS-P and QUALMS-E distinguishing between patients with different performance status, comorbidity, anemia, and transfusion dependency status. No floor and ceiling effects were observed. Responsiveness to change analysis supported the validity of the measure. Patients with a hemoglobin (Hb) level of <11 g/dL at study entry, who subsequently showed an improvement in their Hb levels, also reported a mean score change of 9 and 8 points (scales ranging between 0 and 100) in the expected direction of the QUALMS-E and QUALMS-P, respectively. CONCLUSIONS: Our study provides additional validation data on the QUALMS from the international MDS-RIGHT Project. The use of this disease-specific HRQoL measure may contribute to raise quality standards of patient-centered outcomes research in MDS.

Core set of patient-reported outcomes for myelodysplastic syndromes: an EUMDS Delphi study involving patients and hematologists.

Patient-reported outcomes (PROs) are relevant and valuable end points in the care of patients with myelodysplastic syndromes (MDS). However, a consensus-based selection of PROs for MDS, derived by both patients and hematologists, is lacking. We aimed to develop a core set of PROs for patients with MDS as part of the prospective European LeukemiaNet MDS (EUMDS) Registry. According to international guidelines, candidate PROs were identified from a comprehensive literature search of MDS studies. Overall, 40 PROs were selected and evaluated in a two-round Delphi survey by 40 patients with MDS and 38 hematologists in the first round and 38 patients and 32 hematologists in the second round. Based on an agreement scale and predefined inclusion criteria, both patients and hematologists selected "general quality of life" as a core PRO. Hematologists also selected "transfusion-dependency burden" and "ability to work/activities of daily living" as core PROs. The second Delphi round increased PRO rating agreements. Statistically significant rating differences between patients and hematologists were observed for 28 PROs (Mann-Whitney U test; P < .05) in the first round and for 19 PROs in the second round, with "disease knowledge" and "confidence in health care services" rated notably higher by patients. The overall mean PRO ratings correlation between the 2 groups was moderate (Spearman's rank correlation coefficient = 0.5; P < .05). This first consensus on a core set of PROs jointly developed by patients and hematologists forms the basis for patient-centered care in daily practice and clinical research.

The anemia-independent impact of myelodysplastic syndromes on health-related quality of life.

Myelodysplastic syndromes (MDS) are in the majority of cases characterized by anemia. Both anemia and MDS per se may directly contribute to impairments in health-related quality of life (HRQoL). In this study, we aimed to investigate the anemia-independent impact of MDS on HRQoL. We evaluated participants (≥ 50 years) from the large population-based Lifelines cohort (N = 44,694, mean age 59.0 ± 7.4 years, 43.6% male) and the European MDS Registry (EUMDS) (N = 1538, mean age 73.4 ± 9.0 years, 63.0% male), which comprises a cohort of lower-risk MDS patients. To enable comparison concerning HRQoL, SF-36 scores measured in Lifelines were converted to EQ-5D-3L index (range 0-1) and dimension scores. Lower-risk MDS patients had significantly lower HRQoL than those from the Lifelines cohort, as illustrated in both the index score and in the five different dimensions. Multivariable linear regression analysis demonstrated that MDS had an adjusted total impact on the EQ-5D index score (B = - 0.12, p < 0.001) and an anemia-independent "direct" impact (B = - 0.10, p < 0.001). Multivariable logistic regression analysis revealed an anemia-independent impact of MDS in the dimension mobility, self-care, usual activities, and anxiety/depression (all except pain/discomfort). This study demonstrates that the major part of the negative impact of lower-risk MDS on HRQoL is not mediated via anemia. Thus, the therapeutic focus should include treatment strategies directed at underlying pathogenic mechanisms to improve HRQoL, rather than aiming predominantly at increasing hemoglobin levels.

The cancer patient's perspective of COVID-19-induced distress-A cross-sectional study and a longitudinal comparison of HRQOL assessed before and during the pandemic.

BACKGROUND: To permit timely mitigation of adverse effects on overall clinical outcome, it is essential to understand how the pandemic influences distress and health-related quality of life (HRQOL) in cancer patients during the coronavirus disease 2019 (COVID-19) pandemic. METHODS: In this cross-sectional study, adult cancer patients, without COVID-19 symptoms, completed a 13-item questionnaire about the pandemic's impacts on distress and everyday-life; associations with age, sex, or impaired HRQOL were then assessed by binary logistic regressions. In a subsample of patients with HRQOL assessment available from both before and during the pandemic, we evaluated the pandemic's impact on longitudinal changes in HRQOL reported within 6 months before versus during the COVID-19 lockdown using McNemar's test, and thresholds for clinical importance. RESULTS: We consecutively enrolled 240 patients with solid (50%) or hematological (50%) cancers. Median age was 67 years, 46% were females. The majority ranked heeding their health (80%) and keeping their appointment schedule in hospital (78%) as important. Being younger than 60, or aged 60-70 was independently associated with limitations in everyday life (OR = 3.57, p < 0.001; and 2.05, p = 0.038); female individuals and those with restricted emotional functioning were more distressed by the COVID-19 situation (OR = 2.47, p = 0.040; and 3.17, p = 0.019); the latter group was also significantly more concerned about being a patient at risk (OR = 2.21, p = 0.029). Interestingly, in a subsample of patients (n = 47), longitudinal comparisons pre- versus during the pandemic revealed that HRQOL was not substantially affected by the pandemic. CONCLUSION: Particularly younger and female cancer patients, and those with impaired emotional functioning are distressed by COVID-19. During the first COVID-19 lockdown, cancer patients remained predominantly resilient. This analysis highlights the need to mitigate distress situations in vulnerable patients and thereby enhance resilience during pandemics.

Development of a core outcome set for myelodysplastic syndromes - a Delphi study from the EUMDS Registry Group.

Treatment options for myelodysplastic syndromes (MDS) vary widely, depending on the natural disease course and patient-related factors. Comparison of treatment effectiveness is challenging as different endpoints have been included in clinical trials and outcome reporting. Our goal was to develop the first MDS core outcome set (MDS-COS) defining a minimum set of outcomes that should be reported in future clinical studies. We performed a comprehensive systematic literature review among MDS studies to extract patient- and/or clinically relevant outcomes. Clinical experts from the European LeukemiaNet MDS (EUMDS) identified 26 potential MDS core outcomes and participated in a three-round Delphi survey. After the first survey (56 experts), 15 outcomes met the inclusion criteria and one additional outcome was included. The second round (38 experts) resulted in six included outcomes. In the third round, a final check on plausibility and practicality of the six included outcomes and their definitions was performed. The final MDS-COS includes: health-related quality of life, treatment-related mortality, overall survival, performance status, safety, and haematological improvement. This newly developed MDS-COS represents the first minimum set of outcomes aiming to enhance comparability across future MDS studies and facilitate a better understanding of treatment effectiveness.

Comorbidities cluster with impaired functional capacities and depressive mood and predict adverse outcome in older patients with hematological malignancies.

This study evaluates prevalence of comorbidities and their association with impairments in older patients with hematological malignancies at initial diagnosis (n = 209). At least one comorbidity was present in 62.2%, 68.5% and 93.8% as defined by CCI (Charlson Comorbidity Index), Cumulative Illness Rating Scale-Geriatric (CIRS-G) and HCT-Comorbidity Index, respectively. Severe comorbidities (CIRS-G Grade 3/4) were present in 57.9%. The mean number of affected organ systems was 3.6 (CIRS-G categories), with diabetes (18.2%), congestive heart failure and prior solid tumors (each 17.7%) detected most frequently. Comorbidities were significantly correlated with reduced functional and objective physical capacities, impaired performance and depressive mood. Both CCI and CIRS-G were found to be prognostic factors for OS (p < 0.05). CCI scoring of comorbidities, diagnosis MDS/AML and a body mass index <23kg/m2 were independent adverse predictors for OS. This first prospective analysis reveals a prognostic significance of comorbidities. Clustering of comorbidities with impairments suggests common mechanisms.

Malnutrition in Older Patients With Hematological Malignancies at Initial Diagnosis - Association With Impairments in Health Status, Systemic Inflammation and Adverse Outcome.

Poor nutritional status is a common problem in cancer patients at advanced age, but the prevalence and impact of malnutrition in hematological malignancies remains underinvestigated. To evaluate nutritional status in older adults over age 70 with newly diagnosed hematological malignancies, we enrolled 147 patients and assessed weight loss, food intake, Mini Nutritional Assessment (MNA), and BMI. We compared nutritional status with demographic data, inflammation markers, and restrictions in multidimensional geriatric assessment. MNA classified 43% of patients being at risk of, and 15% having manifest malnutrition. A moderate/severe decrease in food intake was reported by 24% or 16%, a recent weight loss of 1 to 3 kg or >3 kg by 19% or 31%, and a BMI <23 kg/m2 by 29%. Lowered serum albumin (<3.5 g/dL) was prevalent in 14% of patients, and in 38% Glasgow Prognostic Score indicated hyperinflammation. Principal component analysis clustered malnutrition with inflammation markers and pronounced impairments, that is, fatigue, depression, comorbidities, reduced functional capacities. Severe decrease in food intake (HR: 3.3 (1.9-5.8), p < 0.001), >3 kg weight loss (HR: 2.3 (1.4-3.9), p = 0.001), impaired MNA (HR: 2.8 (1.3-6.2), p = 0.010), and low serum albumin (HR: 2.1 (1.1-4.0), p = 0.030) were significantly associated with shortened overall survival. Recent weight loss >3 kg (HR: 2.2 (1.1-4.3), p = 0.022), and low BMI (HR: 3.3 (1.8-6.0), p < 0.001) remained independent adverse parameters in multivariate Cox proportional hazard regression analyses. Malnourishment at initial diagnosis is frequent in older patients with hematological malignancies and represents an adverse prognosticator. Clustering of malnutrition with impairments and systemic inflammation suggests an underlying common pathway.

Health-related quality of life in lower-risk MDS patients compared with age- and sex-matched reference populations: a European LeukemiaNet study.

In myelodysplastic syndromes (MDS), health-related quality of life (HRQoL) represents a relevant patient-reported outcome, which is essential in individualized therapy planning. Prospective data on HRQoL in lower-risk MDS remain rare. We assessed HRQOL by EQ-5D questionnaire at initial diagnosis in 1690 consecutive IPSS-Low/Int-1 MDS patients from the European LeukemiaNet Registry. Impairments were compared with age- and sex-matched EuroQol Group norms. A significant proportion of MDS patients reported moderate/severe problems in the dimensions pain/discomfort (49.5%), mobility (41.0%), anxiety/depression (37.9%), and usual activities (36.1%). Limitations in mobility, self-care, usual activities, pain/discomfort, and EQ-VAS were significantly more frequent in the old, in females, and in those with high co-morbidity burden, low haemoglobin levels, or red blood cells transfusion need (p < 0.001). In comparison to age- and sex-matched peers, the proportion of problems in usual activities and anxiety/depression was significantly higher in MDS patients (p < 0.001). MDS-related restrictions in the dimension mobility were most prominent in males, and in older people (p < 0.001); in anxiety/depression in females and in younger people (p < 0.001); and in EQ-VAS in women and in persons older than 75 years (p < 0.05). Patients newly diagnosed with IPSS lower-risk MDS experience a pronounced reduction in HRQoL and a clustering of restrictions in distinct dimensions of HRQoL as compared with reference populations.