High-dose immunosuppressive therapy (HDIT) with autologous hematopoietic stem cell transplantation (AHSCT) is a promising approach to treatment of multiple sclerosis (MS) patients. In this paper, we present the long-term outcomes of a prospective single-center study with the analysis of the safety and efficacy of HDIT + AHSCT with reduced-intensity BEAM-like conditioning regimen in 99 MS patients: mean age-35 years old; male/female-39/60; median Expanded Disability Status Scale (EDSS) = 3.5; 43 relapsing/remitting MS, 56 progressive MS. No transplant-related deaths were observed. The mobilization and transplantation procedures were well tolerated. At 6 months post-transplant, neurological improvement or stabilization was observed in all the patients except one. Cumulative incidence of disease progression was 16.7 % at 8 years after HDIT + AHSCT. Estimated event-free survival at median follow-up of 48.9 months was 80 %: 83.3 % in relapsing/remitting MS vs 75.5 % in progressive MS. Sixty-four patients who did not progress during the first 3 years post-transplant and were monitored for more than 3 years were included in long-term outcome analysis. At the median long-term follow-up of 62 months, 47 % of patients improved by at least 0.5 points on the EDSS scale as compared to baseline and exhibited improvement during the entire period of follow-up; 45 % of patients were stable. No active, new, or enlarging lesions on magnetic resonance imaging were registered in patients without disease progression. AHSCT was accompanied by a significant improvement in patient's quality of life. Due to the fact that patient selection was quite different to the other studies and that the information about disease activity prior in the disease course and its treatment was inhomogeneous, comparison with the results in the literature should be done with caution. Thus, the risk/benefit ratio of HDIT + AHSCT with reduced-intensity BEAM-like conditioning regimen in our population of MS patients is very favorable. The consistency of our long-term clinical and quality of life results, together with the persistence of improvement, is in favor of the efficacy and safety of this treatment approach in MS patients.
A615Objectives: The approach of the diabetes register named DIAREG is to display the health care reality of patients with type 2 diabetes (T2D) in Germany. In comparison to other existing diabetes registries, DIAREG analyses patient-reported outcomes (PRO) in order to illustrate patient reported quality of life (QoL). MethOds: In July 2013 DIAREG was started including retrospective as well as prospective data from over 100 office based physicians (general practitioner and diabetologists). DIAREG analysis is based on 6 different PRO: generic quality of life questionnaire (SF-36); audit of diabetes-dependent quality of life (ADDQoL); diabetes treatment satisfaction questionnaire (DTSQ); impact of weight quality of life (IWQoL-Lite); Center of Epidemiological Studies Depression Scale (CES-D) and WHO five well being index (WHO-5). Results: By June 2015 more than 2,000 T2D patients were included in the register, involving an observation period of 22 months. Within this time frame PRO of more than 300 patients were completed. The mean age of T2D patients was 68.5 years, whereas 48% of the patient population was older than 70 years. According to a subgroup analysis of 1,646 patients, 16% of the patients were recruited by officebased diabetologists and the majority of 84% by primary care physicians. About 20% of these patients had their therapy changed within the last year due to inadequate HbA1C control. An adjustment of the current therapy was conducted at 81%, a change of the therapy at 46%. Main focus of the physicians for changing the therapy pattern was the decrease of the HbA1C. Based on this platform, further evaluation on QoL will be started. cOnclusiOns: In conclusion, DIAREG shows the treatment reality from the patient and physician perspective and will therefore describe the health care situation and QoL of diabetes patients in Germany.
The needs of the developmentally disabled individuals are somewhat different than the needs of individuals without disabilities. Policy recommendations should focus on increasing the number of outpatient centers as well as primary caregivers who can understand the disease management needs of the patient and accordingly collaborate with other specialized health care professionals to enhance the overall quality of care for the patient.
Dasatinib as a front-line is a promising treatment option for CP-CML pts. Assessment of benefits and risks of this treatment regimen both from physician's and patient's perspective sounds worthy. We aimed to study QoL and clinical outcomes of dasatinib as a front-line treatment in CP CML pts in a real world setting.
The total of 30 pts with CP CML (16 pts - TKI-naive, 14 pts - early switched to dasatinib after failure of imatinib treatment, median duration of imatinib treatment - 6 mths; switched pts) were involved in the multicenter observational prospective study. Median age - 47.6 y.o. (20-74), female - 33.3%; median disease duration - 5 mths; distribution of pts according to the Sokal score - 27.6% pts at low risk, 41.4% - at intermediate risk, 31% - at high risk. 27% pts had comorbidities: median Charlson Comorbidity Index was 3.0. All the pts filled out the QoL questionnaire SF-36 before dasatinib treatment, 3 and 12 mths after treatment start. Hematological, cytogenetic and molecular response rates (HR, CyR, MR) were registered after 3 and 12 mths of dasatinib treatment. Healthy controls (n=30) from QoL population normative database adjusted by age and gender to pts group were used for QoL comparison at baseline. Group comparisons were made using Mann-Whitney test, ANOVA and Wilcoxon matched pair test.
QoL in CP CML pts at baseline was lower than in healthy controls: Integral QoL Index - 0.358 in pts vs 0.497 in healthy controls (p<0.05). Physical functioning in CP CML pts was significantly lower than in healthy controls: 78.8 vs 90.8 (p<0.05). No QoL differences were found between TKI-naive and switched pts before dasatinib treatment (p>0.05). During 12 months of dasatinib treatment positive changes in QoL were observed: Integral Quality of Life Index increased from 0.358 at baseline to 0.493 after 12 mths (p=0.05) and became comparable to healthy controls (p>0.05). Significant QoL improvement was registered in 12 mths of dasatinib treatment for role emotional (48.1 vs 81.5), role physical (44.4 vs 56.5) and social functioning(62.5 vs 80.6); p<0.05. Clinical outcomes were similar to the data of randomized clinical trials. After 3 mths of dasatinib treatment the majority of pts (92%) achieved complete HR, others had partial HR. After 12 mths of treatment 81.6% pts maintained or achieved complete HR; 69.2% pts exhibited complete CyR; 53.8% pts - major/complete MR. Four pts were dropped out of the study due to resistance to dasatinib (n=2) or due to severe AE (n=2). No cases of death were observed during the study. AE were similar to the ones registered within the clinical trials: in the most cases they were non-severe/moderate; severe AE (lung edema, bleeding from stomach ulcer) were observed in 2 pts; no cases of pleural effusion were revealed.
In conclusion, patient-reported and clinical outcomes of dasatinib treatment in TKI-naïve pts and in pts early switched to dasatinib after failure of imatinib were obtained in a real world study. Significant QoL improvement in terms of role physical, role emotional and social functioning was observed. Clinical outcomes support the data obtained in clinical trials. Benefits and risks of front-line treatment with dasatinib in CML-CP both from physician's and patient's perspective were demonstrated in a real world setting.
Disclosures
Ionova: BMS: Research Funding; MSD: Speakers Bureau. Bulieva:BMS: Research Funding. Vinogradova:BMS: Research Funding. Kurbatova:BMS: Research Funding. Nikitina:BMS: Research Funding. Novitskaya:BMS: Research Funding. Rodionova:BMS: Research Funding. Usacheva:BMS: Research Funding. Chukavina:BMS: Research Funding. Shumkova:BMS: Research Funding.
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