Quality of life of adults and children with TRK fusion cancer treated with larotrectinib compared to the general population.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 3614-3614
Author(s):  
Shivaani Kummar ◽  
Cornelis M. Van Tilburg ◽  
Catherine M. Albert ◽  
Jordan Berlin ◽  
Anna F. Farago ◽  
...  

3614 Background: NTRK gene fusions occur in diverse tumor types in adults and children. The selective TRK inhibitor, larotrectinib, has shown high response rates, durable disease control, and a favorable safety profile in patients (pts) with TRK fusion cancer. We report an expanded quality of life (QoL) analysis for pts treated with larotrectinib. Methods: QoL data were collected in two trials of larotrectinib in pts with TRK fusion cancer using EORTC QLQ-C30 (adults) and PedsQL (children) questionnaires, and were analyzed descriptively and longitudinally. EORTC QLQ-C30 global health scores (GHS) and PedsQL total scores range from 0 to 100, with higher scores indicating better QoL. We calculated the proportion of pts with normal/above and below normal QoL scores compared to values in the literature for the US general population. Results: By July 2019, 126 pts with TRK fusion cancer (74 adults, 24 children ≥2 yrs, and 28 infants <2 yrs) had received larotrectinib and completed baseline (BL) and ≥1 post-BL questionnaire. Most pts had clinically meaningful QoL improvements that reached or exceeded the minimally important difference (Table); a positive change from BL was also seen in infants: mean best change of 12.0 (SD 13.8). Of 52 adults with BL EORTC QLQ-C30 GHS at or above the population norm, 51 remained in this category on treatment and 1 moved into the below normal category. Of 22 adults with BL scores below the population norm, 20 moved into the normal/above normal category. All 9 children aged ≥2 yrs with BL PedsQL scores at or above the population norm remained in this category on treatment. Of 15 children with BL scores below the population norm, 10 moved into the normal/above normal category. Sustained QoL improvements (change from BL ≥0) occurred by 2 months of treatment in 69% of adults and 75% of children. Median duration of sustained improvement in EORTC QLQ-C30 GHS and PedsQL total score was 12.0 months (range 1.7–20.3) and not estimable (range 1.1–23.0), respectively. Conclusions: Adults and children with TRK fusion cancer treated with larotrectinib had rapid, clinically meaningful, and sustained improvements in QoL. Clinical trial information: NCT02576431, NCT02637687 . [Table: see text]

2019 ◽  
Vol 53 (3) ◽  
pp. 337-347 ◽  
Author(s):  
Sanja Ledinski Ficko ◽  
Vlatko Pejsa ◽  
Vesna Zadnik

Abstract Background The impact of disease and treatment on the patient’s overall well-being and functioning is a topic of growing interest in clinical research and practice. The aim of this study is to obtain reference data on quality of life of Croatian general population. Further, we aim to assess the impact of the disease and its primary systemic treatment on their health related quality of life (HrQoL) in multiple myeloma (MM) patients. Patients and methods Participants for the first part of the study were randomly selected from adult Croatian population. In the clinical part of the study MM patients were included as prospectively diagnosed within two years in two major Croatian haematological centres. The EORTC QLQ-C30 in both trials and QLQ-MY20 in MM patients only were applied for HrQoL assessment. Results Gender, age and place of residence have great impact on quality of life scores in Croatian population. The MM patients at the time of diagnosis have lower QLQ-C30 scores for global quality of life, functional and symptom scale scores, as well as single items. The type of disease followed by the choice of therapy options are important HrQoL determinants. Conclusions The norm values available now for Croatian population will help to interpret HrQoL for clinicians and aid in planning cancer care interventions. This study identified treatment effect consistent with those from other observational studies and provided new data on HrQoL across two different treatment choices for MM patients.


Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 1023-1023
Author(s):  
Valeria Santini ◽  
Alessandro Sanna ◽  
Alberto Bosi ◽  
Giuliana Alimena ◽  
Giuseppina Loglisci ◽  
...  

Abstract Abstract 1023 Background: The annual incidence rate of myelodysplastic syndromes (MDS) in the general population is estimated at around 3–5/100,000. Because of a lack of specific therapies, until recently small attention has been devoted to costs of treatment of MDS. A specific Diagnosis-Related Group is still missing. In the US the mean annual cost for MDS patients ranges from $19,811 to $51,066. Aims: To assess the annual cost of illness (COI) and quality of life (QoL) of patients with MDS in Italy. Methods: The Costo Sociale delle Sindromi Mielodisplastiche e Qualità della vita in Italia (CoSMIQ) is an observational, cross-sectional, retrospective, prevalence-based, multicenter study based on an International Prognostic Scoring System (IPSS)-stratified sample of patients with MDS ≥18 years of age seen in standard clinical practice at 7 hematologic institutions across Italy (3 in the North; 2 in the Center; and 2 in the South). Demographics, clinical history, health care and non-healthcare resource consumption, and patients' and caregivers' productivity losses were collected by physicians using clinical records and information provided by patients. COI (in Euro (€) 2010) was determined utilizing a societal perspective. QoL was assessed via EORTC QLQ-C30 and QOL-E v.2 questionnaires, the latter being a new specific tool for the assessment of health-related QoL in patients with MDS. Kendall's tau rank correlation, chi-squared test, multivariate analysis of variance (ANOVA; MANOVA) and multiple linear regressions were performed when appropriate. Results: In all, 225 of 234 patients who met the inclusion criteria were analyzed (IPSS low risk: 124 patients; intermediate-1 (Int-1) risk: 75 patients; Int-2 and high risk: 26 patients) (Table). The total COI reached €27,980.4 ± 28,322.2 (mean ± standard deviation) (Figure). Cost-drivers of COI were antianemics (lower-risk) and antineoplastics (higher-risk). The Italian National Health Service (INHS) funded the greatest share of COI (from 97.4% to 99.5%). In general, CoSMIQ patients perform worse than the general population in all EORTC QLQ-C30 domains; the total global health status (QL) domain reached 65.1 ± 22.2 (low risk: 64.2 ± 22.3; Int-1 risk: 67.4 ± 22.1; higher-risk: 62.5 ± 22.3). QL was negatively correlated with COI (p=0.049) and patients' age (p=0.089), and positively correlated with disease duration (p=0.203). Keeping the other predictors constant, RBC transfusion dependence predicts increased COI (p=0.006) and lower QL (p=0.009). Regarding the QOL-E questionnaire, the QOL-E MDS-specific domain (MDSS) was positively correlated with total COI (p=0.0002), and patients' age (p=0.348) and negatively correlated with disease duration (p=0.013). The total QOL MDSS was 27.5 ± 19.8 (low risk: 25.3 ± 19.4; Int-1 risk: 29.6 ± 19.3; higher-risk: 31.6 ± 22.2). The total treatment-outcome index domain (QOL TOI) was 49.4 ± 14.4 (low risk: 49.1 ± 14.7; Int-1 risk: 48.3 ± 14.7; higher-risk: 53.9 ± 11.7). QOL TOI was negatively correlated with total COI (p=0.379), and patients' age (p=0.0003) and positively correlated with disease duration (p=0.162). Differences by IPSS group were not statistically significant for COI (p=0.191) or for any domain of the EORTC QLQ-C30 or QOL-E questionnaires (p=0.124; p=0.467). Conclusion: MDS dramatically increases INHS budgets while negatively impacting patients' QoL. The results of the CoSMIQ study highlight the strong correlation between RBC transfusion dependence and both COI and QL. Disclosures: Lazzaro: Celgene Srl: Research Funding. Martelli:Celgene Srl: Employment, Equity Ownership.


Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 2567-2567
Author(s):  
Elena Liew ◽  
Eshetu G Atenafu ◽  
Shabbir M.H. Alibhai ◽  
Joseph M. Brandwein

Abstract Abstract 2567 Background: The use of pediatric-based protocols in the management of adult acute lymphoblastic leukemia (ALL) appears to result in improved survival, particularly in young adults. However, significant treatment-related toxicities have been identified with these regimens, including osteonecrosis and peripheral neuropathy. These are potentially disabling and can adversely affect quality of life (QoL); however, the long-term impact of such treatment regimens on health-related QoL in this adult population has not previously been reported. We therefore aimed to assess various dimensions of QoL in long-term survivors of adult ALL following treatment with a modified Dana Farber Cancer Institute 91-01 pediatric protocol, which is used as standard frontline therapy in our institution. Methods: QoL was self-reported using 5 psychometrically validated instruments: the EORTC QLQ-C30, the Functional Assessment of Cancer Therapy fatigue subscale (FACT-F), the Brief Pain Inventory, the Subjective Peripheral Neuropathy Screen (SPSS), and the 9-item Patient-Health Questionnaire (PHQ-9). Standard analyses of each instrument were conducted. Where available, results were compared to published population normative data. Results: 29 patients (median age 41 years, range 21–64, 90% male), in continuous complete remission at a median of 28 months following completion of the two-year treatment protocol, were enrolled between March 2010 - April 2012. In comparison to reference data from a general population, the mean global health score on the EORTC QLQ-C30 was similar (p=0.68), but leukemia survivors had lower cognitive (p<0.001) and social (p<0.001) function scores, as well as more marked financial difficulty (p<0.001). The most prevalent and severe of the symptom items assessed by the EORTC QLQ-C30 were fatigue and pain, both of which showed significant inverse correlation with global health status and all functional scales (physical, role, emotional, cognitive, and social). Some degree of fatigue was reported by 83% of patients. Evaluation by FACT-F revealed worse fatigue scores for leukemia survivors compared with the general population (p=0.03). Mean pain intensity was higher in those more than 24 months from completion of treatment, vs. < 24 months (p=0.04). In the 9 patients (31%) experiencing moderate-severe pain, the most common sites were joints and neck/back. Of the 10 patients reporting regular use of analgesics, 4 were using opioids. The SPSS identified 12 patients (43%) with moderate or severe symptoms associated with peripheral neuropathy (burning, paraesthesias and/or numbness), mostly affecting the lower extremities. Neuropathy was more severe in patients over age 40 (p<0.01). The PHQ-9 identified 4 patients (14%) with significant depressive symptoms. Conclusions: Long-term adult ALL survivors who were treated with a pediatric-based regimen generally do well in terms of global QoL. However, fatigue, joint-related pain and neuropathy symptoms are common and can negatively affect QoL. Impairments in the domains of cognitive function and social function, as well as the prominence of financial hardship in this population, are findings that warrant further exploration. Disclosures: No relevant conflicts of interest to declare.


BMC Cancer ◽  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Yacir El Alami ◽  
Hajar Essangri ◽  
Mohammed Anass Majbar ◽  
Saber Boutayeb ◽  
Said Benamr ◽  
...  

Abstract Background Health-related quality of life is mainly impacted by colorectal cancer which justified the major importance addressed to the development and validation of assessment questionnaires. We aimed to assess the validity and reliability of the Moroccan Arabic Dialectal version of the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Core Questionnaire (QLQ-C30) in patients with colorectal cancer. Methods We conducted a cross-sectional study using the Moroccan version of the EORTC QLQ-C30 on colorectal cancer patients from the National Oncology Institute of Rabat, in the period from February 2015 to June 2017. The QLQ-C30 was administered to 120 patients. Statistical analysis included reliability, convergent, and discriminant validity as well as known-groups comparisons. Results In total, 120 patients with colorectal cancer were included in the study with 38 (32%) patients diagnosed with colon cancers. Eighty-two patients (68%) had rectal cancer, among which 29 (24%) patients with a stoma. The mean age of diagnosis was 54 years (+/− 13.3). The reliability and validity of the Arabic dialectal Moroccan version of the EORTC QLQ-C30 were satisfactory. [Cronbach’s alpha (α =0.74)]. All items accomplished the criteria for convergent and discriminant validity except for question number 5, which did not complete the minimum required correlation with its own scale (physical functioning). Patients with rectal cancer presented with bad Global health status and quality of life (GHS/QOL), emotional functioning as well as higher fatigue symptoms compared to patients with colon cancer. The difference between patients with and without stoma was significant for diarrhea and financial difficulty. Conclusions The Moroccan Arabic Dialectal version of the QLQ-C30 is a valid and reliable measure of health-related quality of life (HRQOL) in patients with colorectal cancer.


Author(s):  
Albert Tuca Rodríguez ◽  
Miguel Núñez Viejo ◽  
Pablo Maradey ◽  
Jaume Canal-Sotelo ◽  
Plácido Guardia Mancilla ◽  
...  

Abstract Purpose The main aim of the study was to assess the impact of individualized management of breakthrough cancer pain (BTcP) on quality of life (QoL) of patients with advanced cancer in clinical practice. Methods A prospective, observational, multicenter study was conducted in patients with advanced cancer that were assisted by palliative care units. QoL was assessed with the EORTC QLQ-C30 questionnaire at baseline (V0) and after 28 days (V28) of individualized BTcP therapy. Data on background pain, BTcP, comorbidities, and frailty were also recorded. Results Ninety-three patients completed the study. Intensity, duration, and number of BTcP episodes were reduced (p < 0.001) at V28 with individualized therapy. Transmucosal fentanyl was used in 93.8% of patients, mainly by sublingual route. Fentanyl titration was initiated at low doses (78.3% of patients received doses of 67 μg, 100 μg, or 133 μg) according to physician evaluation. At V28, mean perception of global health status had increased from 31.1 to 53.1 (p < 0.001). All scales of EORTC QLQ-C30 significantly improved (p < 0.001) except physical functioning, diarrhea, and financial difficulties. Pain scale improved from 73.6 ± 22.6 to 35.7 ± 22.3 (p < 0.001). Moreover, 85.9% of patients reported pain improvement. Probability of no ≥ 25% improvement in QoL was significantly higher in patients ≥ 65 years old (OR 1.39; 95% CI 1.001–1.079) and patients hospitalized at baseline (OR 4.126; 95% CI 1.227–13.873). Conclusion Individualized BTcP therapy improved QoL of patients with advanced cancer. Transmucosal fentanyl at low doses was the most used drug. Trial registration This study was registered at ClinicalTrials.gov database (NCT02840500) on July 19, 2016.


2021 ◽  
pp. 0272989X2110035
Author(s):  
Dennis A. Revicki ◽  
Madeleine T. King ◽  
Rosalie Viney ◽  
A. Simon Pickard ◽  
Rebecca Mercieca-Bebber ◽  
...  

Background The EORTC QLU-C10D is a multiattribute utility measure derived from the cancer-specific quality-of-life questionnaire, the EORTC QLQ-C30. The QLU-C10D contains 10 dimensions (physical, role, social and emotional functioning, pain, fatigue, sleep, appetite, nausea, bowel problems). The objective of this study was to develop a United States value set for the QLU-C10D. Methods A US online panel was quota recruited to achieve a representative sample for sex, age (≥18 y), race, and ethnicity. Respondents undertook a discrete choice experiment, each completing 16 choice-pairs, randomly assigned from a total of 960 choice-pairs. Each pair included 2 QLU-C10D health states and duration. Data were analyzed using conditional logistic regression, parameterized to fit the quality-adjusted life-year framework. Utility weights were calculated as the ratio of each dimension-level coefficient to the coefficient for life expectancy. Results A total of 2480 panel members opted in, 2333 (94%) completed at least 1 choice-pair, and 2273 (92%) completed all choice-pairs. Within dimensions, weights were generally monotonic. Physical functioning, role functioning, and pain were associated with the largest utility weights. Cancer-specific dimensions, such as nausea and bowel problems, were associated with moderate utility decrements, as were general issues such as problems with emotional functioning and social functioning. Sleep problems and fatigue were associated with smaller utility decrements. The value of the worst health state was 0.032, which was slightly greater than 0 (equivalent to being dead). Conclusions This study provides the US-specific value set for the QLU-C10D. These estimated health state scores, based on responses to the EORTC QLQ-C30 questionnaire, can be used to evaluate the cost-utility of oncology treatments.


2004 ◽  
Vol 22 (2) ◽  
pp. 354-360 ◽  
Author(s):  
Philippe Rauch ◽  
Joelle Miny ◽  
Thierry Conroy ◽  
Lionel Neyton ◽  
Francis Guillemin

Purpose To identify factors affecting the quality of life (QoL) of disease-free survivors of rectal cancer. Patients and Methods One hundred twenty-one patients in complete remission more than 2 years after diagnosis were asked to complete three QoL questionnaires: the European Organization for Research and Treatment of Cancer (EORTC) QLQ-C30; its colorectal module, QLQ-CR38; and the Duke generic instrument. Results Patients reported less pain (P = .002) than did controls drawn from the general population. EORTC QLQ-C30 physical scores were also higher among rectal cancer survivors than in the general Norwegian or German population (P = .0005 and P = .002, respectively). Unexpectedly, stoma patients reported better social functioning than did nonstoma patients (P = .005), with less anxiety (P = .008) and higher self-esteem (P = .0002). In the present authors' experience, the QLQ-CR38 does not discriminate between these groups. Residual abdominal or pelvic pain and constipation had the most negative influence on QoL. Conclusion QoL is high among rectal cancer survivors, including stoma patients. Simultaneous use of several QoL questionnaires appears to have value in follow-up and in monitoring the effects of therapy. The impact of residual pain and constipation on long-term QoL should be considered when establishing a treatment regimen.


2013 ◽  
Vol 14 (1) ◽  
pp. 139-146 ◽  
Author(s):  
Gemma Cramarossa ◽  
Liang Zeng ◽  
Liying Zhang ◽  
Ling-Ming Tseng ◽  
Ming-Feng Hou ◽  
...  

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