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Cytogenetic abnormalities (CA) such as t(4;14), t(14;16), and del(17p), are associated with a poor prognosis in Multiple Myeloma (MM) patients. However, there is scarce information regarding the Latin-American population. This study aims to analyze the impact of t(4;14), t(14;16), and del(17p) on the progression-free survival (PFS) and overall survival (OS) of transplant-eligible newly-diagnosed MM (NDMM) patients in Latin America. Retrospective survival analysis based on the Grupo de Estudio Latinoamericano de MM (GELAMM) registry, including all adult patients with NDMM harboring CA t(4;14), t(14;16), and/or del(17p). Fifty-nine patients were included; the median age was 57 years, 55.9% males, 22% ISS-I, 25.4% ISS-II, and 47.5% ISS-III. The majority (89.8%) had 1 alteration, whereas 10.2% had del(17p) and t(4;14). The frequencies of CA were del(17p) in 61.0%, t(4;14) in 25.4%, and t(14;16) in 3,4%. Autologous stem cell transplantation (ASCT) was performed in 61.0% of cases. Five-year OS for the entire cohort was 60.8% and 5-year PFS was 28.1%. Bortezomib-based induction regimen (BBR) (P = 0.029), consolidation with ASCT (P0.001), and maintenance therapy (P = 0.004) were associated with an improved 5-year OS. In the multivariate analysis, ASCT was the only variable with a positive impact on OS (HR 0.11, 95% CI 0.033 to 0.34, P0.001). The median PFS presented a non-statistically significant benefit in BBR, ASCT, and maintenance therapy groups. BBR induction, ASCT, and maintenance therapy were associated with improved OS in high-risk NDMM patients.

The aim of this study was to describe clinical and survival characteristics of transplant-eligible multiple myeloma (MM) patients in Latin America (LA), with a special focus on differences between public and private healthcare facilities. We included 1293 patients diagnosed between 2010 and 2018. A great disparity in outcomes and survival between both groups was observed. Late diagnosis and low access to adequate frontline therapy and ASCT in public institutions probably explain these differences. Patients treated with novel drug induction protocols, followed by autologous stem cell transplantation (ASCT) and maintenance, have similar overall survival compared to that published internationally.

Introducción: La enfermedad hepática crónica tiene repercusiones en diferentes niveles. Uno de ellos es la hipertensión portal, la cual produce alteraciones en las células sanguíneas, siendo la trombocitopenia la más frecuente. En el presente artículo buscamos describir las características del perfil hematológico de los pacientes cirróticos en un centro de trasplantes en Ecuador. Materiales y métodos: Se realizó un estudio retrospectivo con pacientes con diagnóstico de cirrosis [CIE10: K-74.60] que estuvieron hospitalizados entre enero 2016 y diciembre del 2016, que tuvieran al menos una biometría hemática y pruebas de coagulación. Resultados: Se incluyeron 111 pacientes, de los cuales 90 (81,08 %) presentaron anemia, 87 (78,37 %) trombocitopenia y 21 (18,92 %) leucopenia; 55 presentaron esplenomegalia, de los cuales 52 presentaron algún tipo de citopenia. Asimismo, 14 pacientes murieron, 12 de ellos presentaron anemia, 9 trombocitopenia, siendo choque hemorrágico la causa más común de mortalidad. Discusión: Anemia fue la esplenomegalia con hiperesplenismo fue la principal causa de citopenias. Sin embargo, gran cantidad de pacientes sin esplenomegalia presentaron bajos conteos celulares y signos de hipertensión portal. Por consiguiente, esto nos lleva a buscar en próximos trabajos las causas de citopenias y alteraciones del perfil hematológico en pacientes con enfermedad hepática crónica sin esplenomegalia.

The sickle cell trait is considered a benign entity that generally does not show clinical manifestations. However, some complications have been described under certain conditions, such as a decrease in oxygen level, dehydration, and strenuous physical efforts. Among them, splenic infarct is a rare complication that presents as left upper abdominal pain in a situation of stress such as high altitude exposure. We present two cases of splenic infarcts in patients with undiagnosed sickle cell trait who showed to our institution with severe abdominal pain after coming from high altitude cities.

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.

Abstract 5274 Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic and life-threatening hematopoietic stem cell disorder characterized by deficiency of the GPI-anchored complement inhibitory proteins CD55 and CD59 on blood cells. The resulting uncontrolled complement activation is responsible for chronic hemolysis and can lead to serious clinical morbidities including thromboembolism (TE) and chronic kidney disease (CKD), which have been shown to increase risk of mortality. Patients may also experience debilitating quality-of-life (QoL) issues, including fatigue, shortness of breath, erectile dysfunction, and abdominal pain, attributed to chronic hemolysis and resultant nitric oxide scavenging by free hemoglobin. Although hematopoietic stem cell transplantation (HSCT) remains the only potentially curative option for PNH, the risk for substantial morbidities and mortality still exist. In patients with PNH undergoing HSCT, up to 45% die or develop acute or chronic graft-versus-host disease. Eculizumab, a first-in-class terminal complement inhibitor, is the only approved treatment for patients with PNH. By inhibiting terminal complement activity and chronic hemolysis, eculizumab reduces the incidence of TE, CKD, and transfusion requirements, improves anemia and QoL, and normalizes survival in patients with PNH. Aim: Report the benefits of eculizumab in a patient with PNH who relapsed after HSCT. Case Report: A 27-year-old woman presented in December 1993 with fever, diarrhea, hemoglobinuria, and acute renal failure requiring temporary hemodialysis (Table). She was subsequently diagnosed with PNH. In February 1995, allogeneic HSCT from an HLA-identical sibling donor was performed. In 2003, 8 years after successful engraftment, the patient relapsed and presented with hemoglobinuria, abdominal pain, corticosteroid dependence requiring 20 to 40 mg methylprednisone, and high transfusional requirements. In November 2007, she had a granulocyte clone size of 37.2% as determined by flow cytometry. In June 2009, she started eculizumab therapy which was associated with a rapid reduction in lactate dehydrogenase (LDH) from 4964U/L to 456U/L. She subsequently achieved resolution of asthenia, disabling fatigue, and abdominal pain, as well as transfusion independence and improvement in hemoglobin. In May 2010, she had a granulocyte clone size of 86.1% as determined by both CD55- and CD59-negative cells, and a granulocyte clone size of 98.7% as determined by CD16b-negative cells. Conclusions: The only potential cure for PNH—bone marrow transplantation—is associated with high risks of morbidity and mortality; therefore, for most patients the associated risks preclude this option. In this case study, we show that HSCT may not be curative in all patients and the PNH associated symptoms can arise after BMT. The potential benefits of eculizumab in this patient population should be considered in light of recent data that demonstrated normalized survival in PNH patients receiving long-term eculizumab treatment. Disclosures: Brodsky: Alexion Pharmaceuticals, Inc.: Consultancy, Speakers Bureau. Velez:Alexion Pharmaceuticals, Inc.: Consultancy.