ALBERT

Beschreibung: Background: Philadelphia-negative Myeloproliferative Neoplasms (Ph-MPN) are chronic hematological disorders characterized by the overproduction of one or more mature myeloid blood cell lineages. Classical Ph-MPN are Polycythemia Vera (PV), Essential Thrombocytopenia (ET) and Myelofibrosis (MF). The diagnosis includes clinical, histological and molecular features. There are not data from Chile. The aim of this study is to determinate epidemiological, clinical, diagnostic and therapeutic characteristics of Ph-MPN in our country. Methods: Descriptive and retrospective study. We reviewed the database of the Molecular Biology Laboratory at the Hospital del Salvador, a national reference laboratory, from 2012 to 2017. All patients referred as Ph-MPN were included. We reviewed the clinical records to obtain clinical information. Results: Clinical data was obtained from 468 cases from 12 public hospitals in Chile. Median age at diagnosis was 70 years. Female to Male ratio= 1,15:1, without significant differences between Ph-MPNs. ET was the most frequently Ph-MNP found, accounting for 49,4% of all Ph-MPN, followed by PV (37%) and MF (10,4%). A 66,2% of ET was JAK2 V617F+. Bone marrow biopsy was performed in 35% of ET cases. Only 7,8% had cytogenetic study. Splenomegaly was found in 8%. Thrombosis was observed in 23,8%. The median platelet count was 842x109/L. All patients received hydrea +/- aspirin or oral anticoagulation. Of the total of PV, 86,6% was JAK2+. Bone marrow biopsy was performed in a quarter of the cases. Thrombosis frequency was 14,5%. A 29% had splenomegaly. Median hemoglobin level was 18 gr/dl. All patients were treated with aspirin +/- phlebotomy and about half of them required cytoreduction. Two patients were refractory to hydrea and used ruxolitinib as second line treatment. A 63,3% of the MF were JAK-2+. Bone marrow biopsy was performed in 59% and 20% had a cytogenetic study. Only one fifth of patients had LDH measurement at diagnosis. Splenomegaly was observed in 75,5% of cases. Thrombosis frequency was 13%. Anemia was the most frequent finding in complete blood count. The treatments were heterogeneous, including hydrea, EPO, thalidomide/prednisone, danazol and ruxolitinib. Discussion: TE was the most common Ph-MPN. The epidemiological and blood count findings were similar to the data reported in the literature. It is important to note that with the 2016 WHO classification new criteria, some of patients diagnosed with ET, now will be in PV cathegory (21 patients in our serie). The distribution of JAK2V617F+ in Ph-MPN was similar to the published data, except for PV, in which we found a lower percentage of JAK2+. Thrombosis were lower than the data reported for PV. It is worrisome that bone marrow biopsy and cytogenetic study were performed only in a low percentage of the patients. The treatment strategies were heterogeneous and not standardized among the participating centers. These findings reveal a lack in the use of the diagnostic tools for Ph-MPN. It is important to improve clinical and molecular characterization of these patients in order to guide available therapeutic alternatives in our country. Disclosures No relevant conflicts of interest to declare.

Beschreibung: Background Multiple myeloma (MM) is a frequent hematologic malignancy. The current gold standard frontline strategy includes a proteasome inhibitor (PI)-based induction, followed by autologous stem cell transplant (ASCT). Access to novel drugs in Latin America (LA) is limited. ASCT is available in most countries, but real access to it is highly heterogeneous. Data regarding patients´ outcomes in candidates to ASCT in the region is scarce. The aim of this study was to describe clinical characteristics and outcomes of MM transplant eligible patients in LA countries. Material and Methods Retrospective international multicenter cohort study. Consecutive MM transplant- eligible patients diagnosed between 2010 and 2018 from participating centers in Chile, Argentina, Ecuador, Mexico, Colombia, and Uruguay were included. Data were collected from clinical records in a standardized report form. We analyzed clinical characteristics at diagnosis and frontline therapy outcomes, including ASCT. Transplant-eligible patients were defined as fit patients younger than 66 years old. Active MM and response to treatment were defined according to current IMWG criteria. Inclusion criteria: 1.- Patients with newly diagnosed active MM between 2010 and 2018. 2.- Older than 18 years, and younger than 66 years. 3- Candidates for ASCT according to the evaluation of the attending physician Exclusion criteria: 1- Lack of minimum data in the clinical history 2- Plasma cell leukemia, AL amyloidosis or solitary plasmacytoma. 3- HIV infection 4-No consent and/or Ethics Committee approvals. Statistical analysis A descriptive statistic has been done. Comparisons of characteristics between groups was made usingT-student, Chi2 or ANOVA, as appropriate. Survival analysis was performed using Kaplan-Meier curves. Comparisons of survival between groups were made by the logarithmic recording method and the calculations of the risk relationships by Cox regression. Statistical analysis was performed by using STATA 13. Results We included 1293 patients in the study, 363 from Chile, 395 from Argentina, 209 from Colombia, 45 from Ecuador, 151 from Mexico, and 130 from Uruguay. The main characteristics at diagnosis and therapeutic strategies are shown in Table 1. Optimal response (sCR, CR and VGPR) was achieved in 38% of the patients in the cyclophosphamide, bortezomib, and dexamethasone (CyBorD) group, in 46% in the bortezomib, thalidomide, and dexamethasone (VTD) group, and in 36% in the cyclophosphamide, thalidomide, and dexamethasone (CTD) group, the 3 main induction regimens used. Only 53% of patients finally received ASCT. Significant differences were found between both groups, private and public institutions, regarding burden of symptoms, ISS staging, access to PI based induction, ASCT completion and adequate maintenance, with patients from the latter being more symptomatic, and receiving suboptimal therapy. FISH analysis was performed in less than 50% of patients, both in the public and private setting. With a median follow up of 34 months (range 1-113), median overall survival (OS) was 86 months. The 5-year progression free survival (PFS) was 38% and 5- year overall survival (OS) was 64%. When comparing public vs private settings, 5 year OS was 45% vs 80%, with a median OS of 56 months vs not reached, respectively (P

Beschreibung: Background Multiple myeloma (MM) is a heterogeneous disease that is most frequently diagnosed in the elderly. Therefore, data on clinical characteristics and outcomes in the young population are scarce and it is recognized that it remains incurable even in this group of patients. We present here the outcomes of patients under 40 years old cohort in Latin-American countries. On behalf of GELAMM (Grupo de Estudio Latino-Americano de Mieloma Múltiple). Methods Retrospective international multicenter cohort study. We analyzed MM patients under 40 years old who received treatment in 6 Latin-American countries, between 2010 and 2018. Demographics and disease features were analyzed using descriptive statics. We examined treatment characteristics and response rates. The overall survival (OS) of the entire cohort was analyzed using Kaplan-Meier curves. Results Eighty-six patients of 6 countries were analyzed (Table1). The mean age was 35.4 years old, and 60% were male. The most frequent monoclonal component type was IgG followed by light chain MM. Risk determined by ISS was distributed in almost equal percentages. The most frequent cytogenetic alteration was the t (4;14) that was found in four patients out of 25 evaluated. The missing data were greater than 70%. Skeleton-related events were the most frequent clinical feature, followed by anemia and renal failure. Plasmacytomas and fractures were present in more than 20 percent of cases. With regard to treatment, VCD / CyBorD was the most used regimen, followed by VTD. The overall response rate (ORR) was 63%. Fifty-three patients received high dose therapy and autologous stem cell transplantation (62%). Only 8% received post-transplant consolidation, and 45% received maintenance therapy. The median OS of the entire cohort was 45 months, and a plateau in the survival curve was not observed, suggesting that patients continue relapsing over the time. Conclusion In this Latin American multicenter study, we found that the young population with MM has similar presentation characteristics to those of elderly patients. A significant amount of information is lost regarding the risk characterization, especially in regard with cytogenetics. With respect to treatment, less than half of the patients achieve very good partial response or better. It is striking that more than a third of this young patients did not access to high doses of chemotherapy and bone marrow transplantation. Maintenance therapy is offered to less than half patients. The median OS is lower than in other series of patients younger than 40 years, even than in the elderly cohorts. Prospective multicentric studies are required to elucidate the behavior of the disease in this group of patients. Disclosures Peña: Pfizer: Membership on an entity's Board of Directors or advisory committees; Janssen: Other: Congress inscription and flights; Biotoscana: Other: Congress inscription and flights; Novartis: Other: Congress inscription and flights; Tecnofarma: Other: Congress inscription and flights; Roche: Other: Congress inscription and flights. Rojas:Novartis: Membership on an entity's Board of Directors or advisory committees; Pfeizer: Membership on an entity's Board of Directors or advisory committees; Abbvie: Membership on an entity's Board of Directors or advisory committees; Roche: Membership on an entity's Board of Directors or advisory committees. Abello:Takeda: Other: Participation in advisory board meeting. Gomez-Almaguer:Takeda: Consultancy, Speakers Bureau; Celgene: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau; Janssen: Consultancy, Speakers Bureau; Teva: Consultancy, Speakers Bureau.