Applying Survival Data Methodology to Analyze Longitudinal Quality of Life Data

Awad, Lucile; Zuber, Emmanuel; Mesbah, Mounir

doi:10.1007/978-1-4757-3625-0_18

Cited by 13 publications

(9 citation statements)

References 7 publications

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“…The patients were censored at the last follow-up time or at time of death. The Kaplan-Meier method and log-rank tests were applied [ 22 ]. In addition to the pre-defined QOL scales, a post-hoc “cardiorespiratory-related” composite event was defined as deterioration in any of the dyspnea, physical functioning, fatigue, and/or pain measures.…”

Section: Methodsmentioning

confidence: 99%

Cardiopulmonary-related patient-reported outcomes in a randomized clinical trial of radiation therapy for breast cancer

et al. 2021

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Background Long-term prospective patient-reported outcomes (PRO) after breast cancer adjuvant radiotherapy is scarce. TomoBreast compared conventional radiotherapy (CR) with tomotherapy (TT), on the hypothesis that TT might reduce lung-heart toxicity. Methods Among 123 women consenting to participate, 64 were randomized to CR, 59 to TT. CR delivered 50 Gy in 25 fractions/5 weeks to breast/chest wall and regional nodes if node-positive, with a sequential boost (16 Gy/8 fractions/1.6 weeks) after lumpectomy. TT delivered 42 Gy/15 fractions/3 weeks to breast/chest wall and regional nodes if node-positive, 51 Gy simultaneous-integrated-boost in patients with lumpectomy. PRO were assessed using the European Organization for Research and Treatment of Cancer questionnaire QLQ-C30. PRO scores were converted into a symptom-free scale, 100 indicating a fully symptom-free score, 0 indicating total loss of freedom from symptom. Changes of PRO over time were analyzed using the linear mixed-effect model. Survival analysis computed time to > 10% PRO-deterioration. A post-hoc cardiorespiratory outcome was defined as deterioration in any of dyspnea, fatigue, physical functioning, or pain. Results At 10.4 years median follow-up, patients returned on average 9 questionnaires/patient, providing a total of 1139 PRO records. Item completeness was 96.6%. Missingness did not differ between the randomization arms. The PRO at baseline were below the nominal 100% symptom-free score, notably the mean fatigue-free score was 64.8% vs. 69.6%, pain-free was 75.4% vs. 75.3%, and dyspnea-free was 84.8% vs. 88.5%, in the TT vs. CR arm, respectively, although the differences were not significant. By mixed-effect modeling on early ≤2 years assessment, all three scores deteriorated, significantly for fatigue, P ≤ 0.01, without effect of randomization arm. By modeling on late assessment beyond 2 years, TT versus CR was not significantly associated with changes of fatigue-free or pain-free scores but was associated with a significant 8.9% improvement of freedom from dyspnea, P = 0.035. By survival analysis of the time to PRO deterioration, TT improved 10-year survival free of cardiorespiratory deterioration from 66.9% with CR to 84.5% with TT, P = 0.029. Conclusion Modern radiation therapy can significantly improve long-term PRO. Trial registration Trial registration number ClinicalTrials.govNCT00459628, April 12, 2007 prospectively.

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Section: Methodsmentioning

confidence: 99%

Cardiopulmonary-related patient-reported outcomes in a randomized clinical trial of radiation therapy for breast cancer

et al. 2021

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“…A recent systemic review of patient reported outcomes in pancreatic cancer revealed that questionnaire completion rates fell below 10% of the original cohort within 12 months in patients with unresectable stage (compared to 75% in patients with resectable stage) 24 . For this reason and also because there is no gold standard for handling missing data in variance analysis, we chose the parameter of time to definitive deterioration of GHS/QOL by 5% as the primary end point of the QoL analysis following the methodology described by Awad et al 1 Because a definitive deterioration can often be captured before a patient's deterioration in physical status affects compliance, this methodology is less affected by missing data than the classical repeated measures analysis of variance. It also allows keeping patients in the analysis even if some of their questionnaires are missing if they have assessable questionnaires afterwards.…”

Section: Discussionmentioning

confidence: 99%

“…It is consensus that, besides efficacy and survival, quality of life (QoL) is an important objective in patients with incurable cancer. 1 In patients receiving chemotherapy, treatment-related toxicity and efficacy represent important determinants of patient's QoL. 2 Moreover, associations between poor survival and impairment of baseline QoL have been observed.…”

Section: Introductionmentioning

confidence: 99%

Quality of life and outcome of patients with metastatic pancreatic cancer receiving first‐line chemotherapy with nab‐paclitaxel and gemcitabine: Real‐life results from the prospective QOLIXANE trial of the Platform for Outcome, Quality of Life and Translational Research on Pancreatic Cancer registry

Al‐Batran

Hofheinz

Reichart

et al. 2020

Intl Journal of Cancer

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Few data exist on health‐related quality of life (QoL) in patients with metastatic pancreatic cancer (mPC) receiving first‐line chemotherapy (Awad L ZE, Mesbah M Boston, MA. Applying survival data methodology to analyze quality of life data, in Mesbah M, Cole BF, Ting Lee M‐L (eds): Statistical Methods for Quality of Life Studies: Design, Measurements and Analysis. Kluwer Academic Publishers 2002). The QOLIXANE study is a prospective, noninterventional, multicenter substudy of the Platform for Outcome, Quality of Life and Translational Research on Pancreatic Cancer (PARAGON) registry, which evaluated QoL in patients with mPC receiving first‐line gemcitabine and nab‐paclitaxel chemotherapy in real‐life setting. QoL was prospectively measured via EORTC QLQ‐C30 questionnaires at baseline and every month thereafter. Therapy and efficacy parameters were prospectively collected. Main objectives were the rate of patients without deterioration of Global Health Status/QoL (GHS/QoL) at 3 and 6 months. Six hundred patients were enrolled in 95 German study sites. Median progression‐free survival was 5.9 months (95% confidence interval [CI], 5.2‐6.3). Median overall survival (OS) was 8.9 months (95% CI, 7.9‐10.2), while median time to deterioration of GHS/QoL was 4.7 months (95% CI, 4.0‐5.6). With a baseline GHS/QoL score of 46 (SD, 22.8), baseline QoL of the patients was severely impaired, in most cases due to loss in role functioning and fatigue. In the Kaplan‐Meier analysis, 61% and 41% of patients had maintained GHS/QoL after 3 and 6 months, respectively. However, in the QoL response analysis, 35% and 19% of patients had maintained (improved or stable) GHS/QoL after 3 and 6 months, respectively, while 14% and 9% had deteriorated GHS/QoL with the remaining patients being nonevaluable. In the Cox regression analysis, GHS/QoL scores strongly predicted survival with a hazard ratio of 0.86 (P < .0001). Patients with mPC have poor QoL at baseline that deteriorates within a median of 4.7 months. Treatment with gemcitabine and nab‐paclitaxel is associated with maintained QoL in relevant proportions of patients. However, overall, results remain poor, reflecting the aggressive nature of the disease.

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“…Awad et al (2002), Dupuy and Mesbah (2002), and Mesbah et al (2004) describe one specific study conducted in metastasic colorectal cancer, and propose various methodologies for answering the scientific issue, which is to explain the effect of a specific treatment on quality of life and survival after adjustment on some pronostic variables Z.…”

Section: Quality Of Life In Oncologymentioning

confidence: 99%

Measurement and analysis of health related quality of life and environmental data

Mesbah

2004

Environmetrics

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SUMMARYThe definition (or construction) of variables and indicators, and the analysis of the evolution of their joint distribution between various populations, times and areas are generally two different, well separated steps of the work for a statistician in the field of environment and/or health related quality of life.The first step generally deals with calibration and metrology of variables. Key words are measurement or scoring, depending on the area of application. Most of the time, the statistical methods used are exploratory. The kinds of models specified are generally structural: classical factorial analysis models or modern item response theory models. The second step is certainly more known by most inferential statisticians. Linear, generalized linear, time series and survival models are very useful models in this step, where the variables constructed in the first step are incorporated and their joint distribution with the other analysis variables (treatment group, time, duration of life, etc.) is investigated.In this article, the simple strategy of separating the two steps is compared with the global strategy of defining and analysing a global model, including both the measurement and the analysis step.

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Applying Survival Data Methodology to Analyze Longitudinal Quality of Life Data

Cited by 13 publications

References 7 publications

Cardiopulmonary-related patient-reported outcomes in a randomized clinical trial of radiation therapy for breast cancer

Cardiopulmonary-related patient-reported outcomes in a randomized clinical trial of radiation therapy for breast cancer

Measurement and analysis of health related quality of life and environmental data

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