A447Objectives: Acromegaly is a rare, chronic, hormonal disorder caused by excessive growth hormone (GH) and insulin-like growth factor 1 (IGF-1) production resulting predominantly from pituitary adenoma. The objective was to test endocrinologist acceptability of the newly developed SAGIT tool in clinical practice. MethOds: SAGIT (Signs and symptoms -Associated comorbidities -GH concentration level -IGF-1 -Tumour) is a Clinician-Reported Outcomes (ClinROs) tool developed with international experts in acromegaly; it allows patient classification and description in a standardised manner. The tool was pre-tested for acceptability, understanding and ease of use with practicing endocrinologists in France, Germany, UK, Spain, Italy and Brazil (n= 2 per country) using the PRAgmatic Content and face validity Test (PRAC-Test). The endocrinologists completed the SAGIT tool prior to and following an intervention (therapeutics or surgery) for three patients each (n= 36). Once completed, a one-hour phone interview was conducted with each endocrinologist to collect their feedback on the tool. Results: The tool was well accepted and deemed concise (n= 11) and informative (n= 10) by the endocrinologists. Several points were raised that illustrate its usefulness in clinical practice, including the removal of the subjectivity when assessing the disease severity, the possibility of rapid evaluation of the control/progression of acromegaly or of a treatment response, and the possibility for standardisation across countries. Key recommendations for improvements were the need to include: 1) instructions to facilitate the understanding and the use of the tool; 2) definitions of rules and recommendations for patient management; and 3) addition of other signs and symptoms and further details about tumour size to better reflect their clinical cases. cOnclusiOns: SAGIT is a useful tool for endocrinologists to accurately stage and classify acromegaly patients in clinical practice. It is currently being piloted in a cross-sectional study. Validation of scoring rules will confirm the utility of the tool to improve patient management.
A447Objectives: Acromegaly is a rare, chronic, hormonal disorder caused by excessive growth hormone (GH) and insulin-like growth factor 1 (IGF-1) production resulting predominantly from pituitary adenoma. The objective was to test endocrinologist acceptability of the newly developed SAGIT tool in clinical practice. MethOds: SAGIT (Signs and symptoms -Associated comorbidities -GH concentration level -IGF-1 -Tumour) is a Clinician-Reported Outcomes (ClinROs) tool developed with international experts in acromegaly; it allows patient classification and description in a standardised manner. The tool was pre-tested for acceptability, understanding and ease of use with practicing endocrinologists in France, Germany, UK, Spain, Italy and Brazil (n= 2 per country) using the PRAgmatic Content and face validity Test (PRAC-Test). The endocrinologists completed the SAGIT tool prior to and following an intervention (therapeutics or surgery) for three patients each (n= 36). Once completed, a one-hour phone interview was conducted with each endocrinologist to collect their feedback on the tool. Results: The tool was well accepted and deemed concise (n= 11) and informative (n= 10) by the endocrinologists. Several points were raised that illustrate its usefulness in clinical practice, including the removal of the subjectivity when assessing the disease severity, the possibility of rapid evaluation of the control/progression of acromegaly or of a treatment response, and the possibility for standardisation across countries. Key recommendations for improvements were the need to include: 1) instructions to facilitate the understanding and the use of the tool; 2) definitions of rules and recommendations for patient management; and 3) addition of other signs and symptoms and further details about tumour size to better reflect their clinical cases. cOnclusiOns: SAGIT is a useful tool for endocrinologists to accurately stage and classify acromegaly patients in clinical practice. It is currently being piloted in a cross-sectional study. Validation of scoring rules will confirm the utility of the tool to improve patient management.
overall survival (OS) in this cohort and for the subgroup of patients who fail to respond to the first CT. Results: A total of 36 EBV + PTLD pts were identified with a median follow up time of 23.4 months (mos) from the date of PTLD diagnosis. Median age at PTLD diagnosis was 47.5 years (yrs) (range 18-75); median time to PTLD onset from transplant was 2.4 yrs (range 0.2-28). Of these, 6 (16.7%) were polymorphic, 22 were diffuse large B cell lymphoma (61.1%), 3 were Burkitt lymphoma (8.3%), and other types 5 (13.9%). Among the 36 pts, 24 (66.7%) died (12 from PTLD, 6 treatment-related, 2 organ failure, and 4 other causes), and with a median overall survival (mOS) of 24.
Background & Aims. The article presents results of two observational, prospective, multicenter studies “Quality of Life, Symptom Profile, and Adherence to Treatment in Adult Patients with Newly Diagnosed Chronic Phase Chronic Myeloid Leukemia Receiving Dasatinib” (20122015) and “Quality of Life and Symptom Profile in Imatinib-Resistant or Intolerant Patients with Chronic Myeloid Leukemia” (2011-2014). Methods. Data of 107 patients with chronic myeloid leukemia in chronic phase were involved in the real-world analysis - 32 newly diagnosed patients on first-line treatment with dasatinib or after yearly switch to dasatinib after ima-tinib treatment failure and 75 imatinib-resistant or intolerant patients on second-line treatment with dasatinib. Treatment effectiveness and safety of dasatinib were assessed during first- and second-line dasatinib treatment using clinical outcomes as well as quality of life and symptom profile assessment. Results. The real-world data obtained during observational study in limited population of CML patients conform the results of clinical trials devoted to evaluation of treatment efficacy and safety of dasatinib treatment in first- and second-line treatment and demonstrate the importance of patient-reported outcomes. Patient's quality of life improved within 12 months of the first-line dasatinib therapy according to the following scales: role physical functioning, pain, vitality, social functioning and role emotional functioning. The most pronounced and clinically significant improvement was observed for the role emotional functioning (51.1 vs. 68.9). During the second-line dasatinib treatment, stabilization of quality of life parameters was registered for the following scales: vitality, social functioning, mental health, and pain. Significant improvement of the Integral Quality of Life Index was observed (p < 0.05). Positive dynamics of relevant symptoms was registered. The symptom severity decreased during both the first- and second-line therapy. Conclusion. Quality of life and symptom assessment in CML patients contribute to a better disease control in accordance with the principles of risk-adaptive therapy.
Background: Lower-risk (LR) myelodysplastic syndromes (MDS) are a group of hematologic disorders impacting patient survival and quality of life. Existing published survival data focus on describing duration of survival; however, it is diffi cult to interpret the seriousness of the disease objectively, since survival data have not been compared with data from the general population. Aims: To quantify the real-world life-year loss due to LR-MDS in comparison with the general population in Germany, France, Italy, Spain, and the United Kingdom (EU5). Methods: A simulation model was developed in Microsoft Excel using parameters sourced from a recently published report of the European LeukaemiaNet MDS (EUMDS) registry. 1 The patient cohort comprised patients diagnosed with LR-MDS from December 2007 to December 2010, during which time recombinant human erythropoietin was available as treatment in most EU countries. Baseline demographics predictive of survival, including age, sex, and revised International Prognostic Scoring System (IPSS-R) risk score, were simulated using published patient characteristics. 1 Survival (in years) of patients with LR-MDS was simulated using the exponential survival function. The exponential distribution parameter, modeled as a function of age, sex, and IPSS-R risk score, was estimated by Cox modeling using published survival curves and hazard ratios. Patient characteristics and survival statistics from the simulation sample were verifi ed against those published by de Swart et al. 1 Real-world life-year loss was defi ned as the difference between simulated survival time and the population-level life expectancy specifi c to patient age, sex, and country. Results: A total of 5,000 patients were included in the simulation; 1,000 patients from each country. The simulated sample generated similar patient characteristics, median survival, and hazard ratios as found by de Swart et al. 1 As shown in Table 1, the mean life-year loss was estimated at 6.3 years (median 6.8 years; 5 and 95 percentile [−13.9, 23.4]), a 40% relative survival loss for patients with LR-MDS compared with the general population. Overall, 77% of patients were estimated to experience life-year loss. Patients experiencing more pronounced lifeyear losses than the general population were those <60 years (mean 13.4 years, 43% relative survival loss, 80% with life-year loss) and those with Intermediate IPSS-R risk score (mean 11.6 years, 76% relative survival loss, 96% with life-year loss). The results were consistent among the EU5 countries and when other similar European LR-MDS real-world studies were used as a data source.Summary/Conclusion: LR-MDS patients in 5 European countries experience signifi cant real-world life-year loss when compared with the general population.
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