ALBERT

Beschreibung: Background The Philadelphia chromosome−negative classical MPNs (myelofibrosis [MF], polycythemia vera [PV], and essential thrombocythemia [ET]) are associated with a pronounced symptom burden, including fatigue, night sweats, itching, and inactivity that may affect patients' HRQOL. There is a limited real-world evidence from many of the Asian countries, including Middle East, regarding the impact of MPNs on HRQOL, or the economic burden associated with these diseases. MERGE is a multinational, multicenter, nonintervention study conducted in adult patients with MPNs in Asia, including Middle East, Turkey, and Algeria. The present analysis evaluated the impact of MPNs on HRQOL of patients from MERGE and explored the association of HRQOL with the clinical outcomes and resource utilization. Method The MERGE registry included patients with MPN diagnosed as per the World Health Organization 2008 criteria. Patients were planned to be followed up for 2 years (5 visits over the course of study). MPN Symptom Assessment Form Total Symptom Score (MPNSAF TSS) was administered during the routine biannual follow-up visits to assess the HRQOL. MPNSAF TSS score was calculated only for patients who completed at least 6 of the 10 items on the MPNSAF TSS questionnaire. The level of inter-rater agreement between physicians and patients on the proportion of symptomatic patients was assessed through Cohen's Kappa coefficient. Further, an ANCOVA (Analysis of covariance) model was applied to estimate the influence of the baseline variables (age, sex, weight, height, family history of MPNs, comorbidities, time to initial MPNs diagnosis, type of MPN initial diagnosis, baseline hemoglobin and platelet count, Eastern Cooperative Oncology Group Performance Status [ECOG PS], and time since diagnosis to treatment initiation) on the TSS score. Variables with univariate P-value 〈 0.20 were included in the multivariate model. Furthermore, medical resource utilization was assessed by evaluating the inpatient and outpatient visits, hospice care, emergency room visits, and day care. Results Of the 884 eligible patients with MPNs, 169 had MF, 301 had PV, 373 had ET, and 41 had unclassified MPN. During the study, 64% of patients received hydroxyurea, 15% interferon, 15% JAK inhibitors, and 10% anagrelide either alone or in combination. At baseline, the proportion of patients presenting at least 1 of the symptoms was higher in the MF group (96.4%), compared to the total MPN patients (91.8%). In addition, the TSS (mean [SD]) at baseline was highest in the patients with MF (23.5 [17.47]) as compared to patients with ET (14.6 [14.26]) and PV (16.6 [14.84]). During the study duration and follow-up, the TSS remained approximately constant at subsequent visits. Physicians reported a lower proportion of symptomatic patients than the proportion self-reported by patients, although, there was better agreement between physicians and patients for pruritus and night sweats (Kappa, 0.61-0.80) (Table 1) than for other symptoms (Kappa, 0.41-0.60). Across MPN subtypes, inter-rater agreement was lowest for patients with MF compared to patients with ET or PV. In a multivariate adjusted mixed effect regression model, female gender, type of MPN (MF), presence of comorbidities, lower baseline hemoglobin were independently and positively associated with the symptom score values. Overall, 24.3% of the patients had at least 1 inpatient visit, with a mean duration of 11.1 days. Outpatient visits were the most commonly observed healthcare resource used (95.5%), without remarkable change during the follow-up period. Patients with ET had a lower mean number of inpatient visits (mean [SD]: 0.9 [0.77] days), and patients with MF had more outpatient visits (mean [SD]: 5.2 [3.17] visit) on an average, compared to the entire MPN group. Conclusion Despite being on treatment, patients with MPN in MERGE had substantial symptom burden, which did not change over the course of the study. A discordance between physician and patient perception of symptom assessment was seen in this study, indicating that in addition to spleen and hematological investigations, a more systematic assessment such as the use of MPNSAF TSS could help to better evaluate patients' symptoms and understand the disease and its treatment burden. Disclosures Taher: Celgene Corp.: Research Funding; Ionis Pharmaceuticals: Consultancy; Protagonist Therapeutics: Consultancy; La Jolla Pharmaceutical: Research Funding; Novartis: Consultancy, Honoraria, Research Funding. Yassin:Novartis: Research Funding. Rippin:Employee of IQVIA - doing consultancy for Novartis: Consultancy. Sadek:Novartis Pharmaceutical Corporation: Employment. Siddiqui:Novartis Pharma AG: Employment, Equity Ownership. Wong:Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau.

Beschreibung: Background The Philadelphia chromosome−negative classical myeloproliferative neoplasms ([MPNs], including essential thrombocythemia [ET], polycythemia vera [PV], and myelofibrosis [MF]), are generally associated with a substantial disease burden, often leading to a reduced quality of life (QOL) and shortened survival in many patients (pts). There has been a lack of estimates for the incidence/prevalence and treatment patterns of MPNs in many regions of the world, including countries from South Asia, Asia Pacific, Middle East, and Turkey. Hence, there is a need to establish databases like registries, which would provide information on the "real-world" data in these regions. The MERGE registry was initiated with an objective to collect data on the epidemiological indices of MPNs and existing treatment patterns in Asia, including Middle East, Turkey, and Algeria. This primary descriptive analysis from the MERGE registry was performed to estimate the incidence/prevalence, natural disease course, and treatment patterns of MPNs in these countries. Methods MERGE is a multinational, multicenter, nonintervention study that included adult pts with MPNs, who were diagnosed according to World Health Organization 2008 criteria. The data were collected retrospectively (since diagnosis) and prospectively after enrollment in the study. Disease assessments were scheduled every 6 months up to 5 visits, with a minimum of 2 years of follow-up. Pts who participated or were participating in a randomized clinical trial were allowed. Data were analyzed descriptively. Results In total, 884 MPN pts (ET=373, PV=301, MF=169, and unclassified=41) were included in the full analysis data set. The median age was 58 years (range, 47-66 years; younger compared to other regions) and 50% pts were males. Baseline pt characteristics by MPN subtype are summarized in Table 1. About 57% of pts were diagnosed by incidental finding of abnormal blood results followed by bone marrow evaluation. In these countries, the prevalence and incidence of MPNs are estimated to be 57-81 and 12-15 per 100.000 hospital-patients per year over the last 4 years, respectively. As assessed by MPN Symptom Assessment Form (MPNSAF), 92% of the pts reported at least 1 symptom. At baseline, fatigue was the most common symptom in all 3 MPN subtypes (71% MPN; 78% MF; 71% ET; and 68% PV). Inactivity (64%), early satiety (61%), abdominal discomfort (57%), bone pain (56%), weight loss (52%), night sweats (46%), and fever (29%) were more common in MF; whereas, itching in PV pts (50%). MF pts had the highest total symptom score at baseline (mean [SD], 23.5 [17.47]) as compared to ET (mean [SD], 14.6 [14.26]) and PV pts (mean [SD], 16.6 [14.84]). Overall, 64% of pts had ECOG performance status of 0 and 26% had ECOG 1. During study period, the most common nonpharmacological intervention was red cell transfusion in MF and ET pts, and phlebotomy in PV pts. Splenectomy (n=2) and stem cell transplantation (n=4) were rarely employed (6 MF pts). Hydroxyurea (HU) was the most common first-line therapy in all 3 MPN subtypes (overall, 54%; PV, 61%; ET, 54%; and MF, 39%), followed by aspirin. Other common first-line therapies were anagrelide (10%) and interferon (9%) in ET pts, antineoplastic (6%) and clopidogrel (6%) in PV, and JAK2 inhibitors in MF pts (15%). More than 75% of the induction therapies were monotherapies, with less than 3% of pts receiving 3 or more drug combinations as primary treatment. Patients with MF often received monotherapy (81%), than the other patients with MPN. Median duration of first-line therapy was about 6 months (95% CI, 1-21 months), and first-line therapy discontinuation rates of 35%, 36%, and 30% were noted in ET, PV and MF pts, respectively. Interferon was used in 8% of in the second-line setting. JAK2 inhibitors were more frequently (14%-17%) used in the second-and third-line settings. Conclusions The prevalence and incidence of MPNs in countries from Asia Pacific were derived using the number of pts visiting the corresponding hospitals. Thus, the resulting incidence and prevalence reported here are not directly comparable with the country-based prevalence/incidence and may overestimate the actual values. At baseline, pts with MPNs had significant disease burden. The most common first-line therapy was HU, though discontinuation rates of first-line therapies were high, and JAK2 inhibitors were mostly used in the second-line/third-line settings. Disclosures Yassin: Novartis: Research Funding. Taher:La Jolla Pharmaceutical: Research Funding; Ionis Pharmaceuticals: Consultancy; Celgene Corp.: Research Funding; Novartis: Consultancy, Honoraria, Research Funding; Protagonist Therapeutics: Consultancy. Kim:Novartis Korea: Honoraria. Rippin:Employee of IQVIA - doing consultancy for Novartis: Consultancy. Sadek:Novartis Pharmaceutical Corporation: Employment. Siddiqui:Novartis Pharma AG: Employment, Equity Ownership. Wong:Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau.