Measurement of the major ignored burden of multiple myeloma, pernicious anaemia and of other haematological conditions on partners and family members: A cross-sectional study.

BACKGROUND: Having a haematological condition can adversely affect the quality of life (QoL) of family members/partners of patients. It is important to measure this often ignored burden in order to implement appropriate supportive interventions. OBJECTIVE: To measure current impact of haematological conditions on the QoL of family members/partners of patients, using the Family Reported Outcome Measure-16 (FROM-16). METHODS: A cross-sectional study, recruited online through patient support groups, involved UK family members/partners of people with haematological conditions completing the FROM-16. RESULTS: 183 family members/partners (mean age = 60.5 years, SD = 13.2; females = 62.8%) of patients (mean age = 64.1, SD = 12.8; females = 46.4%) with 12 haematological conditions completed the FROM-16. The FROM-16 mean total score was 14.0 (SD = 7.2), meaning 'a moderate effect on QoL'. The mean FROM-16 scores of family members of people with multiple myeloma (mean = 15.8, SD = 6.3, n = 99) and other haematological malignancies (mean = 13.9, SD = 7.8, n = 29) were higher than of people with pernicious anaemia (mean = 10.7, SD = 7.5, n = 47) and other non-malignant conditions (mean = 11, SD = 7.4, n = 56, p < .01). Over one third (36.1%, n = 183) of family members experienced a 'very large effect' (FROM-16 score>16) on their quality of life. CONCLUSIONS: Haematological conditions, in particular those of malignant type, impact the QoL of family members/partners of patients. Healthcare professionals can now, using FROM-16, identify those most affected and should consider how to provide appropriate holistic support within routine practice.

Symptom Burden, Perceived Control, and Quality of Life Among Patients Living With Multiple Myeloma.

BACKGROUND: New therapies for multiple myeloma (MM) have improved survival rates but often expose patients to heightened toxicities and prolonged treatment, leading to increasing complications and side effects. We evaluated the association between symptom burden, perceived control over illness, and quality of life (QoL) among a national sample of patients with MM. METHODS: For this observational, cross-sectional study, we used data from the Cancer Experience Registry research initiative to examine symptom- and functioning-related concerns among 289 patients with MM across the illness trajectory. We applied hierarchical multiple linear regression analyses to explore associations between symptom burden and perceived control over illness with QoL indicators: depression, anxiety, and social satisfaction. RESULTS: In our sample, 73% of participants with MM reported currently receiving treatment; 39% experienced relapse; 56% received 1 to 2 autologous transplants, 10% received ≥3 autologous transplants, and 4% received allogeneic and autologous transplants; 30% had not received a stem cell transplant. Average time since diagnosis was 4.4 years. The most highly endorsed concerns included eating and nutrition (61%), physical activity (59%), moving around (56%), fatigue (55%), pain (52%), and sleep (46%). Only 27% believed they had control over their disease, whereas 48% perceived having control over the physical side effects of MM. Approximately one-third of the variance in anxiety and depression and nearly two-thirds of variance in social satisfaction were explained by sociodemographic, clinical, and symptom burden variables. Perceived control over illness significantly predicted depression and anxiety, but not social satisfaction. Our results highlight substantial concern among patients with MM about physical symptoms and function. Additionally, greater symptom burden significantly accounted for poorer QoL, and lower perceived control over illness was linked to depression and anxiety. CONCLUSIONS: Patients with MM and survivors experience substantive long-term QoL issues. Together, these findings point to the critical need for comprehensive symptom management, integrated palliative care, and enhancement of social and emotional support for individuals with MM.

Challenges in the cultural adaptation of the German Myeloma Patient Outcome Scale (MyPOS): an outcome measure to support routine symptom assessment in myeloma care.

BACKGROUND: Patients with multiple myeloma report more problems with quality of life (QoL) than other haematological malignancies over the course of their incurable illness. The patient-centred Myeloma Patient Outcome Scale (MyPOS) was developed to assess and monitor symptoms and supportive care factors in routine care. Our aim was to translate and culturally adapt the outcome measure to the German context, and to explore its face and content validity. METHODS: Translation and cultural adaptation following established guidelines used an exploratory, sequential mixed method study design. Steps included: (1) forward translation to German; (2) backward translation to English; (3) expert review; (4) focus groups with the target population (patients, family members, healthcare professionals) to achieve conceptual equivalence; (5) cognitive interviews using Tourangeau's model with think-aloud technique to evaluate comprehension and acceptability; (6) final review. Results were analysed using thematic analysis. RESULTS: Cultural and linguistic differences were noted between the German and English original version. The focus groups (n = 11) and cognitive interviews (n = 9) both highlighted the need for adapting individual items and their answer options to the German healthcare context. Greater individuality regarding need for information with the right to not be informed was elaborated by patients. While the comprehensive nature of the tool was appreciated, item wording regarding satisfaction with healthcare was deemed not appropriate in the German context. Before implementation into routine care, patients' concerns about keeping their MyPOS data confidential need to be addressed as a barrier, whereas the MyPOS itself was perceived as a facilitator/prompt for a patient-centred discussion of QoL issues. CONCLUSION: With adaptations to answer options and certain items, the German version of the MyPOS can help monitor symptoms and problems afflicting myeloma patients over the course of the disease trajectory. It can help promote a model of comprehensive supportive and patient-centred care for these patients.

Factors influencing supportive care needs of multiple myeloma patients treated with chemotherapy.

PURPOSE: The present study is a descriptive survey conducted to identify factors influencing supportive care needs of patients with multiple myeloma treated with chemotherapy. METHODS: A structured questionnaire was provided to 141 patients with multiple myeloma who were treated as inpatients or outpatients at Seoul St. Mary's Hospital of the Catholic University of Korea. Data were analyzed with t test, ANOVA, Scheffe's test, correlational analysis, and linear regression analysis using SPSS version 21.0. RESULTS: The mean score of supportive care needs of patients with multiple myeloma was 1.51 out of 4 points. Of supportive care needs, information on future disease outcome scored the highest, with a mean score of 2.12, followed by easy and candid explanation by health care staff (2.11), and information on foods that are healthy for cancer patients (2.02). The mean score of disease-related knowledge was 0.52 of 1 point, and diagnostic test results scored the lowest among the subdomains of disease-related knowledge. The mean score of emotional state was 1.13 out of 3 points, mean anxiety score was 1.01 out of 3 points, and mean depression score was 1.26 out of 3 points. Anxiety and depression were identified as factors influencing supportive care needs of patients with multiple myeloma. CONCLUSIONS: The present study demonstrated that the supportive care needs of patients with multiple myeloma are associated with their emotional state. The supportive care needs of patients with multiple myeloma should be identified at an individual level, and should be addressed to reduce anxiety and depression.

Complementing clinical cancer registry data with patient reported outcomes: A feasibility study on routine electronic patient-reported outcome assessment for the Austrian Myelome Registry.

OBJECTIVES: Routinely assessed patient-reported outcomes (PROs), such as quality of life (QOL), are important to supplement clinical cancer data but requires rigorous implementation. This study aims at depicting the implementation procedure and evaluating the feasibility of routine electronic PRO monitoring (ePRO) for collecting data supplementing the Austrian Myeloma Registry (AMR). METHODS: Integration of ePRO monitoring into clinical routine was planned according to the Replicating Effective Programs framework. QOL data were assessed regularly during treatment and aftercare at the hematooncological outpatient unit at the Medical University of Innsbruck with the EORTC QLQ-C30/ +MY20 and the EQ-5D-5L. Feasibility and usability testing were performed via a multimethod approach. RESULTS: Within the first year, 94.4% of the MM patients (N = 142, mean age 65.4, SD 11.8, 60% male) provided 748 PRO assessment time points overall. Patients and clinicians were satisfied with ePRO monitoring and indicated no to little disruption in clinical routine. Patient preference on assessment time points and completion frequency became evident. CONCLUSIONS: Complementing the AMR with ePRO data proved to be feasible. Our findings provide useful insights for healthcare providers considering introducing ePRO monitoring to their units for informing clinical registries as well as individualised feedback to patients alike.

Is Early Palliative Care Feasible in Patients With Multiple Myeloma?

CONTEXT: Evidence for the benefits of early palliative care (EPC) in patients with solid tumors is strong, but EPC has received scant attention in hematologic malignancies. OBJECTIVE: To assess the benefits of outpatient-based EPC for symptom control in patients with multiple myeloma. METHODS: Retrospective study of patients attending the Multiple Myeloma Palliative Care Clinic at our hospital in the year 2013 (February 1-December 31). The following symptoms were assessed at baseline and at three follow-up consultations using a Numerical Visual Scale (0 = no symptoms; 10 = worst possible): pain, anorexia, constipation, insomnia, nausea/vomiting, dyspnea, anxiety, and sadness. Physical and emotional symptom burden scores were calculated. Pain interference with general activity, sleep, and mood was also evaluated. RESULTS: About 67 patients were included. The proportion of patients reporting moderate-to-severe pain (Numerical Visual Scale ≥5) decreased significantly from baseline to the final follow-up: worst pain decreased from 57% to 18% (P < 0.0001), whereas average pain fell from 24% to 2% (P < 0.0001). The percentage of patients reporting no pain interference increased significantly from baseline: general activity (52% vs. 82%; P = 0.0001), sleep (73% vs. 91%; P = 0.01), and mood (52% vs. 87.5%; P = 0.0001). Physical and emotional symptom burden also improved, with significantly fewer patients reporting depression (13% vs. 5%; P = 0.001). Most patients (86.6%) were alive and still attending the Multiple Myeloma Palliative Care Clinic at study end. CONCLUSIONS: These findings indicate that EPC is feasible in patients with multiple myeloma. Pain and other symptoms were well controlled.

The course of anxiety, depression and unmet needs in survivors of diffuse large B cell lymphoma and multiple myeloma in the early survivorship period.

PURPOSE: The purpose of the study is to examine the course of anxiety, depression and unmet needs in diffuse large B cell lymphoma (DLBCL) and multiple myeloma (MM) survivors in the first 2 years post diagnosis. METHODS: DLBCL and MM survivors, recruited through the Victorian Cancer Registry, completed two interviews approximately 7 and 15 months post diagnosis. Hospital Anxiety and Depression Scale (HADS) and Supportive Care Needs Survey (SCNS-SF34) were completed at both interviews. Primary outcomes were prevalence of anxiety, depression and unmet needs (any or moderate-high). Generalized estimating equation examined whether course of anxiety, depression and unmet needs differed between the two cancers. RESULTS: Overall, 236 DLBCL and 178 MM survivors completed both telephone interviews. Course of anxiety differed (p < 0.01) with rate increasing in DLBCL (14 to 22%) while remaining stable for MM (15 to 12%). Course of depression also differed (p < 0.01), decreasing for MM (22 to 12%) and remaining stable for DLBCL (15 to 16%) survivors. Change in unmet needs was generally similar for the two cancer groups, except for moderate to high psychological needs (p < 0.05). CONCLUSIONS: Patterns of change in anxiety and depression in first 2 years post diagnosis differ for DLBCL and MM survivors. IMPLICATIONS FOR CANCER SURVIVORS: Studying psychological outcomes in mixed haematological cancer samples may be inappropriate, at least in the early survivorship phase. Separate studies of the experiences of people with the different haematological cancer subtypes are needed to ensure psychosocial and supportive care interventions are appropriate to the needs of individuals with different haematological cancers.

Geriatric Assessment to Predict Survival and Risk of Serious Adverse Events in Elderly Newly Diagnosed Multiple Myeloma Patients: A Multicenter Study in China.

BACKGROUND: Elderly multiple myeloma (MM) patients often tend to suffer a variety of diseases, so the treatment of choice is very difficult for the elderly myeloma patients. The overall survival (OS) time and side effects with elderly patients are unclear in China. The study tried to find out the role of geriatric assessment in the Chinese elderly MM. METHODS: We retrospectively analyzed the data of 628 newly diagnosed patients from six hospitals from June 2011 to June 2013. A geriatric assessment had been performed to assess comorbidities, cognitive, and physical status for these patients. The primary endpoint was to evaluate different physical states of elderly patients with OS time and treatment-related side effects. RESULTS: An additive scoring system (range: 0-5), based on age, Katz's Activity of Daily Living (ADL) and Lawton's Instrumental Activity of Daily Living (IADL) ≤5 and Charlson Comorbidity Index (CCI) was developed to identify three groups: fit (score = 0); intermediate-fitness (score = 1); and frail (score ≥2). The 3-year OS was 63% in fit patients, 63% in intermediate-fitness patients, and 49% in frail patients ≥3 hematologic adverse events (AEs) were documented in 45 (35.4%) fit, 34 (34%) intermediate-fitness, and 121 (30.2%) frail patients. The risk of a grade ≥3 hematologic AEs was not significantly increase in intermediate-fitness (hazard ratios [HR]: 0.99, 95% confidence interval [CI]: 0.54-1.47, P = 1.000) and in frail patients (HR: 1.16, 95% CI: 0.70-1.93, P = 0.558) compared with fit ones. CONCLUSIONS: MM occurs earlier in life and being advanced when the diagnosis is made in the mainland of China. The overall survival in frailty with International Staging System (ISS) II/III was the worst in all patients.

Does cognitive decline decrease health utility value in older adult patients with cancer?

AIM: Cognitive decline is common among older adults with cancer. The present study aimed to investigate the impact of cognitive decline on health utility value in older adults suffering from cancer. METHODS: Consecutive patients aged 65 years or older with a primary diagnosis of malignant lymphoma or multiple myeloma were recruited. Patients were asked to complete the EuroQoL-5 (EQ-5D) scale to measure health utility and the Mini-Mental State Examination to assess cognitive decline. The potential impact of cognitive decline was investigated with univariate analysis. A multivariate regression analysis was conducted to control for potential confounding factors. RESULTS: Complete data were obtained from 87 patients, 29% of whom had cognitive decline. The mean ± SE EQ-5D score for patients with cognitive decline was significantly lower than that for those without cognitive decline (0.67 ± 0.04 vs 0.79 ± 0.03, t = 2.38, P = 0.02). However, multiple regression analysis showed that cognitive decline was not significantly associated with EQ-5D scores. Female sex and lower performance scores (worse physical condition) were significantly associated with EQ-5D scores. CONCLUSIONS: Cognitive decline may be involved in decreased health utility value in older adult patients with cancer. However, this effect does not seem to be independent, and the patient's physical condition may be a relevant confounding factor.

When "No" Is Not an Acceptable Answer for Treatment.

The focus of health care has moved toward prevention, and insurance companies are supporting preventive practices that enable their members to remain healthy. Many insurance companies have recorded healthy tips delivered by phone, employed case managers to assist patients in keeping physician appointments, created dedicated hot lines staffed by nurses, and developed resource centers. However, specific instances arise when insurance companies do not allow patients to have certain procedures because of contract language that was previously negotiated between the employer or individual and the insurance company. This was the case for my patient.
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Percutaneous Vertebroplasty for Pathological Vertebral Compression Fractures Secondary to Multiple Myeloma--Medium-Term and Long-Term Assessment of Pain Relief and Quality of Life.

BACKGROUND: In patients with multiple myeloma (MM) there is a high risk of compression fractures of the spine. In the majority of cases, the method of treatment is percutaneous vertebroplasty (PV) or kyphoplasty (PK). The number of studies verifying their efficacy in MM is still relatively small. OBJECTIVES: The aim of this study has been to assess medium- and long-term pain relief as well as improvement in the quality of life (QL) after PV in MM cases. MATERIAL AND METHODS: There was a prospective group of 34 MM cases in which a total of 131 vertebral bodies were augmented by means of PV. It was possible to follow up 22 patients who agreed to take part in the assessment. Their level of daily activity and the level of pain were assessed using the Oswestry Back Pain scale and a visual analogue scale (VAS) before PV and at a later date (medium-term follow up was a mean of 10 months after the last operation). Five out of eight cases in which 4.5-5 years had elapsed since the first PV were tested again (long-term follow-up). RESULTS: Relief of pain and improvement of QL, assessed a mean of 10 months after PV, proved to be statistically significant. On the average, pain decreased by 4.7 points as measured on the VAS scale and the average improvement in the QL measured on the Oswestry scale was 27.7%. There were no neurological or general complications. After 4.5-5 years, there has not been any significant change in the level of pain relief or the improvement in the QL in the 5 cases in which long-term assessment was possible. CONCLUSIONS: In MM cases, PV is a simple, effective and safe method for the treatment of vertebral infiltration and compression fractures, giving permanent long-term pain relief and concomitant improvement in the QL.

Improving the assessment of quality of life in the clinical care of myeloma patients: the development and validation of the Myeloma Patient Outcome Scale (MyPOS).

BACKGROUND: Multiple myeloma is an incurable cancer with a rising incidence globally. Less toxic treatments are increasingly available, so patients are living longer and treatment decisions are increasingly guided by QOL concerns. There is no QOL assessment tool designed specifically for use in the clinical care of people with myeloma. This study aimed to develop and test the psychometric properties of a new myeloma-specific QOL questionnaire designed specifically for use in the clinical setting - the MyPOS. METHODS: The MyPOS was developed using findings from a previously reported literature review and qualitative study. The prototype MyPOS was pretested using cognitive interviews in a purposive sample of myeloma patients and refined prior to field testing. The psychometric properties of the MyPOS were evaluated in a multi-centre, cross sectional survey of myeloma patients recruited from 14 hospital trusts across England. RESULTS: The prototype MyPOS contained 33 structured and open questions. These were refined using cognitive interviews with 12 patients, and the final MyPOS contained 30 items taken forward for field-testing. The cross-sectional survey recruited 380 patients for the MyPOS validation. Mean time to complete was 7 minutes 19 seconds with 0.58% missing MyPOS items overall. Internal consistency was high (α = 0.89). Factor analysis confirmed three subscales: Symptoms & Function; Emotional Response and Healthcare Support. MyPOS total scores were higher (worse QOL) in those with active disease compared to those in the stable or plateau phase (F = 11.89, p < 0.001) and were worse in those currently receiving chemotherapy (t = 3.42, p = 0.001). Scores in the Symptoms & Function subscale were higher (worse QOL) in those with worse ECOG performance status (F = 31.33, p < 0.001). Good convergent and discriminant validity were demonstrated. CONCLUSIONS: The MyPOS is the first myeloma-specific QOL questionnaire designed specifically for use in the clinical setting. The MyPOS is based on qualitative enquiry and the issues most important to patients. It is a brief, comprehensive and acceptable tool that is reliable and valid on psychometric testing. The MyPOS can now be used to support clinical decision making in the routine care of myeloma patients.

Use of Bayesian Markov chain Monte Carlo methods to estimate EQ-5D utility scores from EORTC QLQ data in myeloma for use in cost-effectiveness analysis.

BACKGROUND: Patient-reported outcome measures are an important component of the evidence for health technology appraisal. Their incorporation into cost-effectiveness analyses (CEAs) requires conversion of descriptive information into utilities. This can be done by using bespoke utility algorithms. Otherwise, investigators will often estimate indirect utility models for the patient-reported outcome measures using off-the-shelf utility data such as the EQ-5D or SF-6D. Numerous modeling strategies are reported; however, to date, there has been limited utilization of Bayesian methods in this context. In this article, we examine the relative advantage of the Bayesian methods in relation to dealing with missing data, relaxing the assumption of equal variances and characterizing the uncertainty in the model predictions. METHODS: Data from a large myeloma trial were used to examine the relationship between scores in each of the 19 domains of the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30/QLQ-MY20 and the EQ-5D utility. Data from 1839 patients were divided 75%/25% between derivation and validation sets. A conventional ordinary least squares model assuming equal variance and a Bayesian model allowing unequal variance were estimated on complete cases. Two further models were estimated using conventional and Bayesian multiple imputation, respectively, using the full data set. Models were compared in terms of data fit, accuracy in model prediction, and characterization of uncertainty in model predictions. CONCLUSIONS: Mean EQ-5D utility weights can be estimated from the EORTC QLQ-C30/QLQ-MY20 for use in CEAs. Frequentist and Bayesian methods produced effectively identical models. However, the Bayesian models provide distributions describing the uncertainty surrounding the estimated utility values and are thus more suited informing analyses for probabilistic CEAs.

Elderly multiple myeloma patients experience less deterioration in health-related quality of life than younger patients compared to a normative population: a study from the population-based PROFILES registry.

The objectives of this study were to compare health-related quality of life (HRQOL) between multiple myeloma (MM) patients aged ≤65 and >65 years and to compare this with a normative population. Factors associated with HRQOL were identified. The population-based Eindhoven Cancer Registry was used to select MM patients diagnosed from 1999 to 2010. Patients (n = 289) were invited to complete the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) and Quality of Life Questionnaire Multiple Myeloma Module 20 (QLQ-MY20), and 212 patients responded (73 %). Data from the normative population (n = 568) were used for comparison. MM patients >65 years scored better on emotional functioning (p < 0.05) and financial problems (p < 0.01) compared to patients ≤65 years. Patients ≤65 years reported better body image and future perspective (p < 0.01). Compared to the normative population, patients ≤65 years scored worse on all EORTC QLQ-C30 functioning scales and on global health/QOL, fatigue, pain, dyspnea, appetite loss, and financial problems (p < 0.01). Patients >65 years scored worse on social, physical, and role functioning and on global health/QOL, fatigue, pain, and dyspnea (p < 0.01). Younger patients had worse HRQOL compared to the normative population than elderly patients. Patients with comorbidities reported lower QOL. The longer the time since diagnosis, the better the physical functioning. No major differences in HRQOL were found between younger and older MM patients. Compared to that of the normative population, HRQOL in younger patients was worse than that in older patients. The number of comorbidities and time since diagnosis were associated with HRQOL. MM patients reported that a high symptom burden and therapy should, besides prolonging survival, be aimed at improving HRQOL.

Holistic needs assessment in advanced, intensively treated multiple myeloma patients.

PURPOSE: It is recommended that patients with multiple myeloma should be assessed for unmet holistic needs at key times in their disease trajectory. The aim of this exploratory study was to characterise the holistic needs of advanced, intensively treated multiple myeloma using a structured assessment tool. METHODS: Patients with multiple myeloma who had undergone a haematopoietic stem cell transplantation and subsequent treatment for at least one episode of progressive disease but were in stable plateau phase were included in the study. Patients' holistic needs were assessed using the self-reporting tool, Sheffield Profile for Assessment and Referral for Care (SPARC). RESULTS: Thirty-two patients with a median age of 60 years at assessment and a median of 5.5 years from diagnosis were recruited. Using the SPARC, half of the patients reported tiredness as 'quite a bit/very much,' while one third complained that daytime somnolence and insomnia were 'quite a bit/very much.' Forty-four percent of patients reported pain. One third of patients were bothered and distressed by the side effects from their treatment and were worried about long-term effects of their treatment. Thirty-one percent of patients felt that the effect of their condition had an impact on their sexual life, and 40 % were worried about the effect that their illness was having on their family or other people. CONCLUSION: This is the first study to use a self-reported holistic needs assessment tool in multiple myeloma. A multidimensional structured questionnaire like the SPARC could provide a useful first step in the effective delivery of supportive and palliative care for patients with multiple myeloma.

Psychosocial support in patients with multiple myeloma at time of diagnosis: who wants what?

OBJECTIVE: The aim of this study was to examine interest in psychosocial interventions among patients with multiple myeloma at time of diagnosis as well as associated factors. METHODS: Patients with newly diagnosed multiple myeloma were recruited from our outpatient myeloma unit at the Heidelberg University Hospital. Patients completed questionnaires that included a checklist on desired psychosocial interventions and the Patient Health Questionnaire 9-item (PHQ-9) depression and Generalized Anxiety Disorder 7-item scale (GAD-7) anxiety scales. Medical data were extracted from the patients' electronic records. RESULTS: The survey was completed by 114 out of the 130 myeloma patients. Half of the patients (51%) desired psychosocial interventions. The most common preferences were relaxation techniques (21%), psychological counseling (20%), and peer support groups (18%). Approximately 24% of the patients reported symptoms of depression, and 8% reported symptoms of anxiety. Patients with symptoms of depression had twice as many preferences for psychosocial interventions as nondepressed patients. They more frequently desired individual psychotherapy (p = 0.035) and peer support groups (p = 0.015). In general, lower age was associated with greater interest in psychosocial interventions. Medical status was not strongly associated with interest in psychosocial interventions. CONCLUSIONS: A high percentage (51%) of patients with multiple myeloma has psychosocial intervention desires at the time of diagnosis. The greatest interest was found in depressed and younger patients. However, depressed patients do not prefer a single intervention form but rather show a broad variability of wishes, so 'one size does not fit all'. Therefore, to reach all patients in need, a choice of psychosocial interventions should be offered.

Comparison of generic, condition-specific, and mapped health state utility values for multiple myeloma cancer.

OBJECTIVE: Resource allocation informed by cost-utility analysis requires that the benefits be comparable across patient groups and interventions. One option is to recommend the use of one generic utility measure, but this raises the issue of comparability when the preferred measure is inappropriate or unavailable. Many cancer trials do not include generic measures such as the EuroQol five-dimensional (EQ-5D) questionnaire and instead include condition-specific measures and use these to generate utility estimates. We analyze the comparability of generic, condition-specific, and mapped utility values for a multiple myeloma cancer patient data set. METHODS: Generic EQ-5D, condition-specific EORTC-8D, and EQ-5D utility values mapped from the EORTC QLQ-C30 were compared by using psychometric and statistical analysis to determine discrimination across severity groups, responsiveness, and agreement. RESULTS: Generic, condition-specific, and mapped utility estimates were responsive over time and show discriminative validity. The EQ-5D had higher responsiveness and detected a greater change across severity groups and treatment periods than did the EORTC-8D but has a higher proportion of responses at full health (12.8%). Differences in the EQ-5D and the EORTC-8D were due at least in part to differences in the classification system. Mapped EQ-5D estimates had a smaller SD and do not reflect the severe range of health states reported by using the EQ-5D. CONCLUSIONS: Our findings suggest that condition-specific EORTC-8D or mapped EQ-5D utility estimates are broadly comparable to directly obtained EQ-5D utilities for a multiple myeloma patient data set. However, EORTC-8D estimates captured changes in quality of life for patients in mild health states that were not captured by the EQ-5D, but estimated lower utility gains than did the use of the EQ-5D directly.

Deriving a preference-based measure for cancer using the EORTC QLQ-C30.

OBJECTIVE: The European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) is one of the most commonly used measures in cancer care but in its current form cannot be used in economic evaluation because it does not incorporate preferences. We address this gap by estimating a preference-based measure for cancer from the EORTC QLQ-C30. METHODS: Factor analysis, Rasch analysis, and other psychometric analyses were undertaken on a clinical trial dataset of 655 patients with multiple myeloma to derive a health state classification system amenable to valuation. Second a valuation study was conducted of 350 members of the UK general population using time trade-off. Mean and individual-level multivariate regression models were fitted to derive preference weights for the classification system. RESULTS: The health state classification system has eight dimensions (physical functioning, role functioning, social functioning, emotional functioning, pain, fatigue and sleep disturbance, nausea, constipation, and diarrhea) with four or five levels each. Regression models have few inconsistencies (0 to 2) in estimated preference weights and small mean absolute error ranges (0.046 to 0.054). CONCLUSIONS: It is feasible to derive a preference-based measure from the EORTC QLQ-C30 for use in economic evaluation. Future research will extend this to other countries and replicate across other patient groups.

Unmet supportive care needs, psychological well-being and quality of life in patients living with multiple myeloma and their partners.

PURPOSE: The aim of this project was to identify the nature and range of needs, as well as levels of quality of life (QOL), of both patients living with myeloma and their partners. METHODS: A cross-sectional survey was used, recruiting patients and their partners from 4 hospitals in the United Kingdom at a mean time post-diagnosis of 5 years. Patients completed a scale exploring their Supportive Care Needs, the Hospital Anxiety and Depression Scale (HADS) and the EORTC QOL scale with its Myeloma module. The partners completed the partners' version of the Supportive Care Needs scale and HADS. RESULTS: A total of 132 patients and 93 of their partners participated. One-quarter of the patients and one-third of the partners reported unmet supportive care needs. About 27.4% of patients reported signs of anxiety and 25.2% reported signs of depression. Almost half the partners (48.8%) reported signs of anxiety and 13.6% signs of depression. Anxious/depressed patients had more than double unmet needs than non-anxious/depressed patients (P<0.05). QOL was moderate, with key areas of impairment being physical, emotional, social and cognitive functioning, and patients complained of several symptoms, including tiredness (40.7%), pain (35.9%), insomnia (32.3%), peripheral neuropathies (28.3%) and memory problems (22.3%). About 40.8% were worried about their health in the future. CONCLUSION: Long-term supportive care services should provide support to both patients and their partners in relation to their unmet needs, screening them for psychological disorders, referring them appropriately and timely, and optimising symptom management in order to improve the patients' QOL.

Minimal important differences and response shift in health-related quality of life; a longitudinal study in patients with multiple myeloma.

BACKGROUND: We previously reported that changes of 6-17 percent in the EORTC QLQ-C30 scores are regarded important by patients with multiple myeloma and thus may be considered as Minimal Important Differences (MIDs). However, patients' internal standard of measurement may have changed over time (response shift, RS). In the present work, we evaluated whether myeloma patients experience RS and if this could affect the MID-estimates. METHODS: Between 2006 and 2008, 239 patients with multiple myeloma completed the EORTC QLQ-C30 at baseline (T1) and after three months (T2). At T2, patients were asked if they had noticed any change in the domains pain, fatigue, physical function and global quality of life. They were also asked to give a retrospective judgment of their baseline values on all the four domains. RESULTS: We found clear evidence of RS in myeloma patients. However, there were differences in both magnitude and direction between patients who stated that they improved and those who deteriorated. Deteriorating patients retrospectively reported better health-related quality of life at T1 for the domains pain, fatigue and physical function. In these patients, MIDs adjusted for RS were observed to increase up to 12 percentage points. In contrast, for patients stating that they improved, we only found evidence of statistically significant RS in the domain global quality of life. CONCLUSIONS: MIDs estimated from pre-test/post-test data appeared to be robust against RS in patients reporting improvement over 3-months. This could indicate that RS has a minimal impact on the results in patients who respond to treatment, and that RS may not have an important impact on interpretation of changes reported in clinical trials where an improvement occurs.Although the effect sizes of the RSs were small, RS in deteriorating patients may have an important impact on the interpretation of changes reported in clinical trials.