AVIDA: A Longitudinal Registry of Clinical and Quality of Life Outcomes in Patients with Hematologic Disorders Receiving Azacitidine.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 4605-4605
Author(s):  
David Grinblatt ◽  
Mohit Narang ◽  
James Malone ◽  
David Sweet

Abstract Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid neoplasms characterized by ineffective hematopoiesis and peripheral cytopenias. Treatment decisions are often based on age, performance status (PS), cytopenias, International Prognostic Scoring System (IPSS) classification, and MDS subtype. Patient-reported results from a few clinical trials suggest that MDS can have a negative effect on a patient’s quality of life (QoL) with responses to treatment having a positive effect. Azacitidine (Vidaza) is a hypomethylating agent approved in the US for the treatment of MDS. In a phase III study, patients (pts) treated with azacitidine experienced significantly greater improvement in QoL compared with supportive care (Kornblith AB, et al. J Clin Oncol. 2002;20:2441). Evaluation of QoL in MDS pts treated in community-based hematology clinics is not well characterized. Azacitidine is approved for a dosing schedule of 75 mg/m2/day x 7 day q 28 days. However, the dose and schedule of azacitidine used in clinical practice varies. AVIDA is a unique, longitudinal, multicenter patient registry designed to prospectively collect data from community-based hematology clinics on the natural history and management of pts with MDS and other hematologic disorders, including acute myeloid leukemia, who are treated with azacitidine. It aims to further the understanding of current azacitidine treatment patterns in the community, identify common care procedures and concomitant treatments, explore correlation between duration and number of treatment cycles with ongoing clinical response, and to investigate the effect of azacitidine on patient satisfaction and QoL. Patient-reported QoL will be based on the EORTC QLQ-C30 questionnaire with QoL measures obtained at baseline and at quarterly intervals for 2 years. Scores on the EORTC QLQ-C30 range from 0 to 100. Higher scores on the global health and functioning scales indicate better QoL in each measure/domain. Lower scores on the symptom and single-item scales indicate less impairment due to that symptom/single item. To date, 47 pts (34 males, 13 females; mean age, 73.2 years) with predominantly low-risk MDS have been enrolled in the registry. The majority (90%) is white and has an ECOG PS of 0 or 1. Median time from first MDS diagnosis is 2.2 months (mean, 14 months); 43 have primary and 4 have secondary MDS. IPSS is known for 36 pts; low for 8 pts, intermediate-1 for 21 pts, intermediate-2 for 6 pts, and high for 1 patient. Baseline QoL data are currently available for 42 pts. At baseline, pts reported a lower level (mean score) of global health (52), physical functioning (66) and role functioning (61) compared with cognitive (81), emotional (76), and social (71) functioning. Among the symptom/single item scales, fatigue scored the worst with a mean score of 48. Other symptom/single item scales that indicated pts were experiencing a moderate level of the measure included dyspnea (37), insomnia (29), and pain (22). Constipation (17), appetite loss (15), financial difficulties (15), and nausea/vomiting (8) were reported at lower levels. Ongoing results from this patient registry will provide insight into the QoL of pts with MDS and other hematologic malignancies in the real-world setting, and will explore any change in QoL associated with treatment and/or disease progression.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 4097-4097
Author(s):  
Juan W. Valle ◽  
Antoine Hollebecque ◽  
Junji Furuse ◽  
Lipika Goyal ◽  
Funda Meric-Bernstam ◽  
...  

4097 Background: In FOENIX-CCA2 (NCT02052778), a pivotal phase 2 study among iCCA patients (pts) with FGFR2 fusions/rearrangements, the highly selective, irreversible FGFR1–4 inhibitor futibatinib demonstrated a confirmed objective response rate of 41.7%, with a 9.7-month median duration of response. Adverse events were manageable with dosing modifications that did not adversely impact on response. We report outcomes for the preplanned analysis of Patient-Reported Outcomes (PROs) during futibatinib treatment as a secondary objective of FOENIX-CCA2. Methods: Pts enrolled in FOENIX-CCA2 had locally advanced/metastatic unresectable iCCA with FGFR2 fusions/rearrangements, ≥1 prior line of therapy (including gemcitabine/cisplatin) and ECOG PS 0-1. Pts received oral futibatinib 20 mg continuous QD dosing per 21-day cycle. PRO measures included EORTC-QLQ-C30 (1 global health, 5 functional, 9 symptom scales), EQ-5D-3L, and EQ visual analogue scale (VAS). PROs were collected at screening, cycles 2 and 4, every 3 cycles thereafter, and end of treatment. PRO data were evaluated up to cycle 13, the last visit before data were missing for >50% of the PRO population (PRO primary assessment time point). Results: 92/103 (89.3%) pts enrolled had PRO completion data at baseline and a minimum of 1 follow-up assessment (median age 58 y, 56.5% female), with 48 pts having PRO data at cycle 13. At baseline, mean (SD) EORTC QLQ-C30 global health status score was 70.1 (19.4) and EQ VAS score 71.7 (20.3). Mean EORTC QLQ-C30 global health status scores were maintained from baseline to cycle 13, corresponding to 9.0 months on treatment, with no clinically meaningful (≥10-point) changes in individual functional measures (Table). EORTC QLQ-C30 scores across individual symptom measures were also stable from baseline through cycle 13; only constipation showed an average of 10.0-point worsening at only cycle 4. Mean EQ VAS scores were sustained from baseline to cycle 13 (mean change ranging -1.8 to +4.8 across cycles), with values maintained within the population norm range from across 20 countries. Conclusions: Quality of life data from the phase 2 FOENIX-CCA2 trial show that physical, cognitive and emotional functioning, and overall health status were maintained among pts with advanced iCCA receiving futibatinib. Clinical trial information: NCT02052778. [Table: see text]


2010 ◽  
Vol 97 (2) ◽  
pp. 270-275 ◽  
Author(s):  
Kean Fatt Ho ◽  
Damien J.J. Farnell ◽  
Jacqueline A. Routledge ◽  
Meriel P. Burns ◽  
Andrew J. Sykes ◽  
...  

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 5030-5030
Author(s):  
Amarnath Challapalli ◽  
Susan Masson ◽  
Paul White ◽  
Narges Dailami ◽  
Sylvia Pearson ◽  
...  

5030 Background: Neoadjuvant cisplatin-based combination chemotherapy (NAC) improves survival in muscle invasive bladder cancer (MIBC). However, response rates and survival remain suboptimal. We sought to evaluate the efficacy, safety and tolerability of cisplatin cabazitaxel combination in this patient group. Methods: A phase 2 single arm trial (Simon 2 stage), to recruit at least 26 evaluable patients was designed with 80% power to detect the primary endpoint, objective response rate (ORR) of > 35%. ORR was defined as pathological complete response (pCR) plus partial response (pathological downstaging), measured by pathologic staging (T2 or greater at diagnosis, to T1 or less at radical cystectomy). Treatment was with Cisplatin 70mg/m2 and Cabazitaxel 15mg/m2 on day 1 of a 21 day cycle, for 4 cycles prior to surgery. Toxicity was recorded using CTCAE v.4.03. Quality of Life (QoL) data were collected at baseline, prior to each cycle of chemotherapy and at 3-5 weeks after 4th cycle of chemotherapy using EQ-5D and EORTC QLQ-C30, BLM30 questionnaires. Results: Objective response was seen in 15 out of 26 evaluable patients, 57.7% and over a third of patients achieved pCR (9/26; 34.6%). 78% (21/27) of patients completed all cycles of treatment, with only 6.7% of the reported adverse events (AEs) being graded 3 or 4. There were 6 treatment related SAEs reported but no SUSARs. In patients who achieved objective response the median progression free (PFS) and overall survival (OS) were not reached (median follow up: 41.5m). In contrast, median PFS (7.2m) and OS (16.9m) were significantly worse (p = 0.001) in patients who did not respond. Response rates for EORTC QLQ-C30, BLM 30 and EQ5D questionnaires was 70.4, 70.4 & 63% respectively, at end of treatment. There was no significant difference in EORTC QLQ C30 summary, global health scores and EQ5D score with treatment. There was a significant decline in mean QLQ C30 domain scores after 1st cycle compared to baseline, but no further deterioration with subsequent cycles of chemotherapy. Conclusions: Cabazitaxel with cisplatin as NAC of MIBC can be considered a safe, well-tolerated and effective regimen with higher pCR rate of 34.6%. This compares favorably to that with Cisplatin/Gemcitabine (23-26%). Minimal changes in Global Health & EQ5D observed during NAC further demonstrates the excellent tolerability of this regimen and to our knowledge are the first data regarding QoL in NAC in MIBC. These results warrant further evaluation in a larger phase 3 study. Clinical trial information: 2011 004090 82 .


Author(s):  
André L. Mihaljevic ◽  

Abstract Background The patient-reported outcomes (PRO) version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) and the computerized adaptive testing (CAT) version of the EORTC quality-of-life questionnaire QLQ-C30 have been proposed as new PRO measures in oncology; however, their implementation in patients undergoing cancer surgery has not yet been evaluated. Methods Patients undergoing elective abdominal cancer surgery were enrolled in a prospective multicenter study, and postoperative complications were recorded according to the Dindo–Clavien classification. Patients reported PRO data using the CAT EORTC QLQ-C30 and the PRO-CTCAE to measure 12 core cancer symptoms. Patients were followed-up for 6 months postoperatively. The study was carried out by medical students of the CHIR-Net SIGMA study network. Results Data of 303 patients were obtained and analyzed across 15 sites. PRO-CTCAE symptoms ‘poor appetite’, ‘fatigue’, ‘exhaustion’ and ‘sleeping problems’ increased after surgery and climaxed 10–30 days postoperatively. At 3–6 months postoperatively, no PRO-CTCAE symptom differed significantly to baseline. Patients reported higher ‘social functioning’ (p = 0.021) and overall quality-of-life scores (p < 0.05) 6 months after cancer surgery compared with the baseline level. There was a lack of correlation between postoperative complications or death and any of the PRO items evaluated. Feasibility endpoints for student-led research were met. Conclusion The two novel PRO questionnaires were successfully applied in surgical oncology. Postoperative complications do not affect health-reported quality-of-life or common cancer symptoms following major cancer surgery. The feasibility of student-led multicenter clinical research was demonstrated, but might be enhanced by improved student training.


Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4111-4111
Author(s):  
Qiufei Ma ◽  
Abigail Bailey ◽  
Neil Milloy ◽  
Jake Butcher ◽  
Ruben G.W Quek ◽  
...  

Abstract Introduction: Diffuse large B-cell lymphoma (DLBCL) is the most common type of aggressive non-Hodgkin's lymphoma (NHL) constituting 30-58% of all NHL (Tilly et al, 2015; Thieblemont et al, 2020). Treatment can include intensive multiagent chemotherapy and other novel therapies which carries risk for toxicities. Despite this, we lack data comprehensively depicting the quality of life of real-world patients with DLBCL, particularly in the modern era with novel therapies. Therefore, we aimed to compare Quality of Life (QoL) to reference populations and assess real-world DLBCL patients across multiple countries and lines of therapy. Methods: Real-world data were drawn from the Adelphi DLBCL Disease Specific Programme™ (DSP), a point-in-time survey of hematologists, hemato-oncologists, oncologists and their patients with DLBCL conducted in France (FR), Germany (DE), Italy (IT), Spain (SP), the United Kingdom (UK) and the United States of America (US) between Jan-May 2021. Patients were asked to voluntarily complete a patient self-completion form (PSC) capturing demographics and QoL data through the use of patient-reported outcome instruments: the European Organization for Research and Treatment of Cancer quality of life questionnaire (EORTC QLQ-C30), EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L), EQ-5D-5L Visual Analogue Scale and Work Productivity and Activity Impairment questionnaire. Bivariate analysis was conducted to compare all cancer and NHL-specific reference values from the EORTC QLQ-C30 manual (Scott et al, 2008) to DLBCL DSP data, and to review QoL across lines of therapy, defined as first line and second line (1L+2L) and third line and above (3L+). Statistical significance level was set at p&lt;.05. If functional scores were lower, when compared to reference values, within the DSP, this was indicative of a worse QoL in patients with DLBCL. For symptomatic scores the opposite was true; should the DSP value be higher, this was indicative of a worse QoL in patients with DLBCL. Results: Data analysis was conducted on 441 patients with DLBCL who completed a PSC (FR: n=80, DE: n=150, IT: n=54, SP: n=43, UK: n=34, US: n=80); at data collection, mean (standard deviation) age was 64.6 (12.39) years, 36% of patients were female, 19% working full- or part-time and 80% were relapsed/refractory, 29% were 3L+. 8%, 24%, 28% and 40% were at Ann Arbor disease stage I, II, III and IV respectively at the time of data collection. When comparing DLBCL DSP values to EORTC QLQ-C30 all cancer reference values for functional scores (Table 1), global health status, physical functioning, role functioning, emotional functioning, cognitive functioning and social functioning were significantly worse than all cancer reference values. In terms of symptoms, DSP values for fatigue, nausea and vomiting, dyspnea, appetite loss and diarrhea were significantly worse than all cancer reference values (Table 2). Results were mixed when comparing with EORTC QLQ-C30 NHL-specific reference values (Table 1) for functional scores; global health status was significantly worse for the DLBCL DSP population, whilst role functioning, cognitive functioning and social functioning were significantly better than NHL reference values. Significantly worse symptom scores were observed in the DLBCL DSP population (Table 2) for nausea and vomiting, pain, dyspnea and diarrhea when compared with the reference values. Functioning scores were significantly worse in 3L+ patients vs 1L+2L for global health status, physical functioning, role functioning, cognitive functioning and social functioning (Table 3). Fatigue, dyspnea and diarrhea symptomatic scores were significantly worse in 3L+ vs 1L+2L patients. Symptom burden was high across all lines of therapy (Table 4). Conclusion: Real-world patients with DLBCL demonstrated significantly worse QoL when compared with a general cancer reference population with respect to all functional scores, as well as fatigue, nausea and vomiting, dyspnea, appetite loss and diarrhea, underscoring the high symptom burden experienced by patients with DLBCL. Patients with DLBCL on 3L+ had significantly worse QoL than those on earlier lines of therapy with respect to global health status, physical functioning, role functioning, cognitive functioning and social functioning, fatigue, dyspnea and diarrhea indicating an unmet need in novel treatment options to help improve QoL in later lines. Figure 1 Figure 1. Disclosures Ma: Regeneron Pharmaceuticals Inc.: Current Employment, Current holder of individual stocks in a privately-held company. Quek: Regeneron Pharmaceuticals Inc.: Current Employment, Current holder of individual stocks in a privately-held company.


2014 ◽  
Vol 32 (31_suppl) ◽  
pp. 214-214
Author(s):  
Harminder Singh ◽  
Raja Banipal ◽  
Ritu Bala

214 Background: Cancer prevalence in India is estimated around 2.0-2.5 million, 0.7- 0.8 million new cases identified every year, and cancer deaths reported per year is 0.4-0.5 Million. The objective of this study was to analyze and compare patient-reported QoL (quality of life), and their physical/psychosocial symptom and adverse drug reaction in cancer patients. Methods: Study done by questionnaire EORTC QLQ-C30. Comparison among 3 distinctive groups (G) (varied number of Chemotherapy cycles) was done. ADR measured appropriately Results: 131 patients were recruited and QoL scoring GHS (global health status) and 4 items of symptom scale i.e. insomnia, pain, appetite loss, constipation, and financial difficulties attained a significance difference. GHS significantly improved in G3 as compared to G1, indicating that the patient overall health improved as the chemotherapy sessions progressed. Female patients had more ADR (mean 3.2/person) and G3 had more ADR (mean 3.96). Conclusions: QoL score didn’t show significant improvement in all areas (except insomnia, pain, appetite loss, constipation & financial difficulties), a judicious diagnosis with an appropriate treatment including chemotherapy may lessen the negative perception of cancer. [Table: see text]


Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2111-2111 ◽  
Author(s):  
Monica Else ◽  
Alastair G. Smith ◽  
Kim Hawkins ◽  
Shirley Crofts ◽  
Margaret Course ◽  
...  

Abstract The UK CLL4 trial randomised 777 previously untreated patients between 1999–2004 to chlorambucil (Chl) or fludarabine +/− cyclophosphamide (F v. FC). Other trial endpoints are reported elsewhere in this meeting (Catovsky et al). The current study seeks to determine whether there is any difference in the quality of life (QoL) of patients according to treatment arm; and whether QoL correlates with baseline patient characteristics and/or with treatment outcomes. All patients were asked to complete the validated EORTC QLQ-C30 (version 2.0) at start of therapy (baseline), at 3, 6 and 12 months, and annually thereafter. Scores (0–100) within each of 15 different measures of QoL (5 functional scales, 3 symptom scales, 1 global health/QoL scale, and 6 single items) were calculated at each point in time (currently up to 1 year), and analysed according to the EORTC QLQ-C30 Scoring Manual. Statistical significance was determined by the use of ANOVA. Patient compliance was high: 599 questionnaires were completed at baseline, 557 at 3 months, 559 at 6 months and 470 at 12 months, respectively 77%, 74%, 77% and 78% of those who had so far reached the time-period and remained alive. Analysis of baseline and 12 month QoL assessment by treatment group and response is presented. QOL results were the same for each treatment group at baseline and at 12 months (Table 1). Quality of response to treatment correlated with QoL at 12 months (Table 2). With treatment approaches having potentially different risk and side effect profiles, and perhaps limited impact on overall survival, QOL forms an important component of treatment outcome assessment. Further analysis of QoL parameters (in particular their relationship to prognostic factors and further follow-up) will be forthcoming within the next year. Table 1: Mean QoL Scores by Treatment Group* Functioning Scales Physical Role Emotional Cognitive Social Global Health No. B 12M B 12M B 12M B 12M B 12M B 12M Abbreviations: B = baseline; 12M = 12 months. Chl 175 81 78 74 75 75 78 83 82 79 77 66 69 F 92 82 82 72 74 80 81 82 82 80 79 64 68 FC 94 80 77 77 73 78 81 85 82 82 76 66 68 361 Table 2: Mean QoL Scores by Response to Treatment* Functioning Scales Physical Role Emotional Cognitive Social Global Health No. B 12M B 12M B 12M B 12M B 12M B 12M Abbreviations: B = baseline; 12M = 12 months; CR/NPR = complete/nodular partial response; PR = partial response; NR/PD = no response/disease progression. P-values compare response groups with respect to the change in scores from baseline to 12 months. (* Only those patients are shown in the Tables for whom data is available at both time-points.) CR/NPR 159 84 84 76 80 78 84 86 85 82 83 67 73 PR 125 79 76 73 73 78 78 82 79 80 77 63 68 NR/PD 56 77 74 71 61 74 74 81 79 74 65 65 60 340 p=0.02 p=0.03 p=0.002


2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e20070-e20070
Author(s):  
Vincent Lin ◽  
Bhagyashree Oak ◽  
Julia Snider ◽  
Josh Epstein

e20070 Background: CAR T therapy is being investigated as 2nd line therapy in RR-DLBCL. To evaluate its impact on patients’ HRQOL, it is essential to understand the HRQOL of patients receiving current standard of care (SOC) therapy for DLBCL and NHL patients. Methods: A systematic literature review (SLR) of studies reporting HRQOL in RR-DLBCL patients was conducted in March 2019 (updated to include RR-NHL in May 2019) using EMBASE, MEDLINE, Cochrane (all from 2007), Northern Light and International Society for Quality of Life Research abstracts (both from 2017). A targeted literature review (TLR) for untreated or 1st line patients was conducted in July 2019 using PubMed. Search terms included diseases, lines of therapy and patient-reported outcome (PRO) measures. Abstracts and publications were screened for eligibility and data were extracted. Results: Of the 977 publications screened for the SLR, 26 met the inclusion criteria. Another 18 studies were included from the TLR. The most commonly used PRO measures were the SF-36 (10 studies), EORTC QLQ-C30 and FACT-Lym (8 studies each). The EORTC QLQ-C30 showed statistically significant or clinically meaningful changes in a greater number of domains (86%) than the FACT-Lym (75%) and SF-36 (62%). Additional results in table. Conclusions: While research is limited, RR-DLBCL patients receiving current SOC therapy report decreases in HRQOL and health utility. Further research is needed on how existing and future therapies may affect HRQOL among RR-DLBCL patients. [Table: see text]


2008 ◽  
Vol 94 (6) ◽  
pp. 813-821 ◽  
Author(s):  
Leszek Miszczyk ◽  
Andrzej Tukiendorf ◽  
Aleksandra Gaborek ◽  
Jerzy Wydmański

Aims Evaluation of analgesic uptake, pain intensity, and quality-of-life changes after half-body irradiation of patients with bone metastases. Material and Methods Ninety-five patients (97 irradiations) were treated with single half-body irradiation fraction (3–8 Gy). Thirty-three patients had upper-half-body irradiation, 55 lower-half-body irradiation and 9 middle-half-body irradiation. The patients were examined on the day of irradiation, 2 and 4 weeks later, and then once a month. The intake of analgesics, pain level (from 0 to 10), and the quality of life (EORTC QLQ-C30) were evaluated. The fluctuations of pain levels and the particular scaling values of QLQ-C30 during a one-year period were analyzed (Kendall t correlation). Results Over the course of 5 months, the incidence of patients using strong opioids decreased from 43.8% to 33.3%, and the incidence of patients who did not need to resort to analgesics increased from 6.7% to 25%. The mean pain level decreased from 6.1 points (half-body irradiation) to 3.1 points 2 weeks later. An inverse correlation between pain level readings and time was statistically significant. An increase was observed in the values of the five functional scales as reflected on the EORTC QLQ-C30 questionnaire (four of which correlated significantly with the observation time). A similar situation prevailed with respect to global health status. A decrease was observed in most of the values on the symptoms scales; 6 saw a significant decrease, in correlation with the follow-up. Correlations were also found between pain intensity and functionality, and between symptoms scales readings and global health status. Conclusions Half-body irradiation of cancer patients suffering from painful multiple bone dissemination is an effective and simple treatment modality that affords significant quality-of-life improvement and pain relief, thus allowing for a reduction in the use of strong analgesics.


Author(s):  
Selda Çakın Ünnü ◽  
Ilkay Tugba Unek ◽  
Ömercan Topaloğlu

Objective: The self-administered questionnaires by the patients are among the most important methods to evaluate the patient’s health related quality of life. The objective of the study was to evaluate the effect of chemotherapy on quality of life of the patients receiving palliative chemotherapy with the diagnosis of metastatic gastric and colorectal cancer by using EORTC QLQ-C30. Methods: This study included 100 patients who were treated with palliative chemotherapy for the diagnosis of metastatic gastric or colorectal cancer in İzmir Tepecik Education and Research Hospital Department of Medical Oncology between 2011-2012. The EORTC QLQ-C30 questionnaire was filled twice by the patients before chemoterapy started and after chemotherapy completed. Results: When the two questionnaires were compared, it was found that global health status and physical functioning did not change after the chemotherapy. Role functioning, cognitive functioning, and social functioning impaired but emotional functioning improved (p<0.05). After the chemoterapy, scores of fatigue and constipation decreased but financial difficulties increased (p<0.05). The symptom scores of nausea-vomitting, pain, dyspnea, insomnia, anorexia, diarrhea did not change. Conclusion: The results of this study suggested that a quality of life assessment with the EORTC QLQ-C30 questionnaire would be beneficial in patients with metastatic gastric and colorectal cancer. In this way, impairments in functional scores, global health status and symptom scores that may occur after chemotherapy can be detected, clinicians can be helped to decide on the switch to chemotherapy regimens that are similar in effectiveness but have different side effects profile, the patients’ quality of life can be improved as a result of the application of the necessary palliative treatments.


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