Information about the efficacy and safety of the second-line therapy with dasatinib in patients in chronic phase chronic myeloid leukemia (CML-CP) at long-term follow-up is limited. Evaluation of benefits and risks of the treatment in a “real-world” study both from physician’s and patient’s perspective is worthwhile to better define treatment outcomes in this patients’ population. We aimed to study clinical and patient-reported outcomes as well as safety of the second-line therapy by dasatinib in CML-CP patients with imatinib resistance or intolerance treatment at long-term follow-up. 75 CML-CP patients resistant or -intolerant to imatinib were enrolled in the prospective, multicenter, non-interventional study (mean age 51.3 years old, SD 15.4; range 22–83 years; male/female – 37/38). All the patients received dasatinib as the second-line therapy (100 mg daily). Clinical and patient-reported outcomes were evaluated at base-line, 12, 18 and 24 months after treatment start. Twenty six patients were analyzed through all study time-points. For quality of life (QoL) and symptom assessment all the patients filled out the SF-36 and Comprehensive Symptom Profile in Chronic Myeloid Leukemia Patients (CSP Leuk-CML), respectively. Overall and progression-free survival rates as well as cumulative probability of achieving a complete cytogenetic response (CCgR) were calculated using Kaplan-Meier methods. To compare frequencies of CCgR χ2 criterion was applied. Comparison of QoL and symptom scores was conducted using t-test. QoL scores were analyzed using t-test, adjusting for sociodemographic and disease status. At 24 months of dasatinib treatment 94% patients achieved or maintained complete hematologic response and 69% – CCgR. The twenty four-month progression free survival rate was 79% (95% CI; 63.3–88%), overall survival rate – 93% (95% CI; 84–97%). One patient was resistant to dasatinib after 16 months of treatment. During the second year of dasatinib therapy one сase of pleural effusion (grade 3) was registered (at 18 months of treatment); other severe adverse effects (grade 4) were as follows: one patient – neutropenia (at 18 months), one patient – arthralgia/myalgia (at 18 months), one patient – memory loss (at 24 months), one patient – headache and hyperglycemia at 18 months and palpitations, alopecia, hyperglycemia at 24 months of treatment. At 24 months of dasatinib treatment improvement of QoL as compared with base-line was registered: Integral QoL index was significantly higher than at base-line (p<0.02). At 24 months follow-up the proportion of patients with no QoL impairment was 56%; 18.7% patients exhibited severe/critical QoL impairment. It was shown that 56.4% patients with no/mild QoL impairment before dasatinib treatment (favorable group) achieved CCgR as compared with 28% patients with severe/critical QoL impairment (unfavorable group). Progression-free survival rate was 87% in the favorable group vs 60% in the unfavorable group. Cumulative probability of CCgR achievement was higher in the favorable group vs the unfavorable group – 75% vs 50% (log-rank test, p<0.05). Thus, long-term outcomes of second-line therapy in CML-CP patients in a “real world” setting confirm that dasatinib treatment is effective both in terms of clinical outcomes and patient-reported outcomes, as well as exhibits good tolerability. At 24 months of treatment definite QoL improvement was registered. Patients with high QoL before second-line treatment have had better treatment outcomes at long-term follow-up. Comprehensive evaluation of the outcomes of the second-line treatment of CML-CP at long-term follow-up allows to assess the benefits and risks of therapy both from physician’s and patient’s perspective. Disclosures Ionova: BMS: Research Funding. Nikitina:BMS: Research Funding. Fedorenko:BMS: Research Funding. Gritsenko:BMS: Research Funding. Ivanova:BMS: Research Funding. Kuchma:BMS: Research Funding. Shnaider:BMS: Research Funding. Sannikova:BMS: Research Funding. Usacheva:BMS: Research Funding. Kurbatova:BMS: Research Funding.
There is limited published data about the efficacy and safety of the second-line therapy with dasatinib in patients in chronic phase chronic myeloid leukemia (CML-CP) in a “real world” patients setting outside clinical trials. In addition, comprehensive evaluation of benefits and risks of the treatment is worthwhile to better define treatment outcomes in this patients’ population. We aimed to study clinical and patient-reported outcomes as well as safety of dasatinib treatment in a “real world” setting within the context of its approved indication through the analysis of prospectively collected data in patients with imatinib resistance or intolerance receiving dasatinib as the second-line therapy. 75 CML-CP patients resistant or -intolerant to imatinib were enrolled in the prospective, multicenter, non-interventional study (mean age 51.3 years old, SD 15.4; range 22–83 years; male/female – 37/38). The median of disease duration was 5.0 years (0.75–17 years). 63 patients had resistance to imatinib; 12 patients were intolerant to imatinib; the median duration of imatinib treatment 40 months (3–121 months). All the patients received dasatinib as the second-line therapy (100 mg daily). Median follow-up was 12 months. For quality of life (QoL) and symptom assessment patients filled out the SF-36 and Comprehensive Symptom Profile in Chronic Myeloid Leukemia Patients (CSP Leuk-CML), respectively, at base-line, in 1, 3, 6 months after treatment start and every 6 months thereafter. Comparison of QoL and symptom scores was conducted using t-test. QoL scores were analyzed using t-test, adjusting for sociodemographic and disease status. Mean symptom severity and percentage of patients with moderate-to-severe (ratings ³ 5) symptoms was evaluated. After 12 months of treatment 83% patients achieved or maintained complete hematologic response and 35 % – complete cytogenetic response. The twenty four-month progression free survival rate was 93% (95% CI; 84–97%). Four cases of pleural effusion events were registered: they were easily managed in 3 cases; one patient died at 1 month after treatment start due to accompanied infection complication. No severe hematological adverse effects were observed except two cases of grade III-IV neutropenia. Two patients were resistant to dasatinib. Two patients died of disease progression at 6 months of follow-up. At 12 months of dasatinib treatment QoL parameters were stable for 5 out of 8 scales; vitality, social functioning and mental health significantly improved as compared with base-line (p< 0.01). At 24 months of dasatinib treatment improvement of physical functioning, vitality, social functioning and mental health as compared with base-line was registered (p< 0.01); no worsening was observed for other QoL scales. Before treatment 75% of patients experienced at least one moderate-to-severe symptom; more than 40% had more than 7 moderate-to-severe symptoms. The majority of patients (96%) experienced fatigue; half of them suffered from moderate-to-severe fatigue. While treatment the number of patients with moderate-to-severe symptoms decreased. After 12 months of therapy only 25% of patients experienced moderate-to-severe fatigue. Before treatment 36% of patients exhibited critical or severe QoL impairment. Remarkably, in the subgroup of patients (44%) with critical or severe QoL impairment at base-line dramatic QoL improvement was observed: QoL index increased 3.4 fold (p<0.01). Thus, our study on “real world” patient data confirms that dasatinib as second-line therapy in CML-CP patients is effective both in terms of clinical outcomes and patient-reported outcomes, as well as exhibits good tolerability. Comprehensive evaluation of the outcomes of the second-line treatment of CML-CP allows to assess the benefits and risks of therapy both from physician’s and patient’s perspective. Disclosures: Ionova: BMS: Research Funding. Nikitina:BMS: Research Funding. Gritsenko:BMS: Research Funding. Ivanova:BMS: Research Funding. Kuchma:BMS: Research Funding. Shnaider:BMS: Research Funding. Sannikova:BMS: Research Funding. Fedorenko:BMS: Research Funding. Kurbatova:BMS: Research Funding.
4757 Increased importance should be placed on the comprehensive symptom assessment in patients with multiple myeloma to accurately document the broad range of physical and psychological disease manifestations given from the patient perspective. The new developed symptom assessment tool - Comprehensive Symptom Profile in Multiple Myeloma Patients (CSP-MM) aims to provide an in-depth view of patient's problems. It is currently undergoing content validation through a structured, iterative process that conforms to the FDA industry guidance on the use of patient-reported outcome measures. We aimed to evaluate the utility of the new instrument providing evidence of its content validity. Pilot sample of twenty two patients with different stages of multiple myeloma were included in this qualitative research study. Patients underwent either conventional chemotherapy or autologous stem cell transplantation. Mean age was 58 years old; male/female distribution –11/11. The CSP-MM is a self-reported tool which consists of 51numeric rating scales (where “0” - no symptom, “10” - most expressed symptom). Evidence of adequate tool content was collected from multiple sources: literature review, pilot CSP-MM completion, cognitive interviews with patients and clinicians, expert evaluation, and data quality control. Patients filled out the CSP-MM before and at different time-points of treatment. The utility of the CSP-MM was demonstrated. After the completion of the pilot, patients were asked about their overall assessment of the questionnaire. Open-ended patient interviews provided a full understanding of the patient's perspective and showed that saturation of the items has been reached. The patients acknowledged the comprehensiveness of the tool. All of the items were easy for the patients to read and understand. Total number of completed surveys was 79 with only 1.6% missing items throughout all forms. Completion of the CSP-MM in paper and pencil format took 7–9 min. The data produced by the tool were clear for interpretation by physicians. According to the clinicians’ interviews, changes in symptom severity captured by the tool at different time-points of treatment gave the physicians information about patient experience and were used by them in day-to-day decision making. Thus, the CSP-MM is an appropriate and practical tool to assess the symptom severity in myeloma patients. The utility of the questionnaire was shown. Pending further validation, the CSP-MM can be used to assess symptoms in MM patients and the patient benefit from the treatment. Disclosures: No relevant conflicts of interest to declare.
2561 Comprehensive symptom assessment before and during treatment in lymphoma patients is worthwhile. We aimed to develop a new symptom assessment tool — Comprehensive Symptom Profile in Lymphoma Patients (CSP-Lym) and test its applicability to this patient population. The CSP-Lym is a self-reported tool which allows the assessment of the severity of 45 symptoms in lymphoma patients. It consists of numerical rating scales, scored from “0” (no symptom) to “10” (most expressed symptom). Thirteen symptom clusters have been identified, which were clinically relevant and increased the practicability of the tool. Utility of the CSP-Lym was demonstrated by means of interviewing both patients and physicians. Applicability of the tool with preliminary analysis of psychometric properties was tested in a pilot study. 75 patients with different types of malignant lymphomas (Stage – I-IV) were included in the study: Non-Hodgkin's lymphoma – 26; Hodgkin's lymphoma – 49. Mean age was 33.5 years old; male/female distribution –28/47. Patients filled out the CSP-Lym before and at different time-points of treatment. The utility of the CSP-Lym was demonstrated. The patients acknowledged the comprehensiveness of the tool. All of the items were easy for the patients to read and understand. Completion of the CSP-Lym in paper and pencil format took 5–7 min. The data produced by the tool were clear for interpretation by physicians and were used by them in day-to-day decision making. The construct validity of the CSP-Lym was proven by factor analysis and “known-group” comparison. A six factor structure was found for the core symptom items, which explained 65% of the total variance. The analysis of symptom severity in the groups with and without B-symptoms revealed differences between the groups: all of the symptoms were more severe in patients with B-symptoms. Statistically significant differences between the groups (p<0.05) were found for the majority of symptoms. Reliability of the CSP-Lym was satisfactory: Chronbach's alpha coefficient varied from 0.63 to 0.92. Sensitivity to changes was demonstrated by the comparison of symptom severity before and after treatment. The majority of symptoms after treatment were less severe than at base-line. Thus, the CSP-Lym is an appropriate and practical tool to assess the symptom severity in lymphoma patients. The utility of the questionnaire was shown; preliminary psychometric properties appeared to be satisfactory. Further studies are needed before the wide-spread use of the CSP-Lym in clinical practice and clinical trials. Disclosures: No relevant conflicts of interest to declare.
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