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OBJECTIVE: To report our clinical experience with bevacizumab in a cohort of Hereditary Hemorrhagic Telangiectasia (HHT) patients with severe hepatic involvement and/or refractory anemia. METHODS: Observational, ambispective study of the Institutional Registry of HHT at Hospital Italiano de Buenos Aires. Patients were treated with bevacizumab due to iron deficiency refractory anemia secondary to nasal/gastrointestinal bleeding and/or high output cardiac failure. We describe basal clinical data, bevacizumab schedules, efficacy outcomes and adverse events. Wilcoxon signed ranks test and longitudinal analysis were conducted. RESULTS: Twenty adult patients were included from July 2013 to June 2019. Clinical indications were: 13 for anemia, 4 for heart failure and 3 for both. In the anemia group, median pretreatment hemoglobin was 8.1 g/dl [IQR: 7.2-8.4] and median transfusion requirement was 4 units [2-6]. In heart failure group, pretreatment median cardiac index was 4.5 L/min/m2 [4.1-5.6] and cardiac output was 8.3 L/min [7.5-9.2]. Bevacizumab 5 mg/kg/dose every 2 weeks for 6 applications was scheduled. By the end of induction, median hemoglobin at 3 months was 10.9 g/dl [9.5-12.8] (p = 0.01) and median transfusion requirement 0 units [0-1] (p<0.01), and this effect was more or less sustained during a year. Regarding heart failure group, two patients had complete hemodynamic response and achieved liver transplantation and two had partial response. No serious adverse events were registered. CONCLUSION: Bevacizumab is a promising line of treatment for HHT patients with refractory anemia. For patients with high output cardiac failure, bevacizumab may be useful as bridge therapy awaiting for liver transplantation.

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Transformation of myelodysplastic syndrome (MDS) into acute myelogenous leukemia occurs in approximately 30 % of cases, while progression into acute lymphoblastic leukemia (ALL) is rare. We report on a 67-year-old man with the diagnosis of MDS, subtype refractory anemia with ring sideroblasts (RARS), karyotype 20q- , JAK-2 negative and grade III fibrosis on the bone marrow biopsy, who evolved into ALL 33 months after the diagnosis of MDS. RARS is one of the subtypes of MDS with most indolent course. Deletion of the long arm of chromosome 20 (20q-) is considered as good prognosis by the International Prognostic Scoring System, an important scoring system for predicting survival and evolution of MDS. Primary MDS with bone marrow fibrosis may represent a distinct clinicopathological and is supposed to have an unfavorable prognosis. The combined analysis of these features makes this rare report still more challenging and illustrates that biology of MDS is yet to be discovered.

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PURPOSE: Myelofibrosis is a common, poor-prognosis feature of myelodysplastic syndromes (MDS). The aim of this work was to evaluate quantitatively the extent of the juxtatrabecular fibrosis in primary MDS and in secondary myelodysplasias, along with the presence of immature precursor cells in anomalous position, that is, the displacement of granulopoiesis from the paratrabecular area to central positions. PATIENTS AND METHODS: Twenty-seven bone marrow samples were examined: 9 from primary MDS, 9 from secondary myelodysplasias, and 9 normal. The percentage of myeloblasts and promyelocytes with nucleoli located in the central areas was estimated, in an attempt to correlate such feature with the degree of juxtatrabecular fibrosis. The analysis of data was performed with the Kruskal-Wallis test, values of p < 0.01 being significant. RESULTS: Sectorial juxtatrabecular fibrosis was present in all the myelodysplastic samples, ranging from 6% to 55% of the trabecular surface; the highest values found in the controls were about 3.6% (p < 0.01). Although the juxtatrabecular fibrosis figures are higher in secondary MDS, the difference is not significant with regard to the primary MDS in the number of patients studied here. The count of myeloblasts and promyelocytes with nucleoli present in the MDS was significantly greater than that of the control group. The number of promyelocytes with nucleoli was significantly higher in the primary MDS with respect to the secondary ones, whereas no significant difference was seen between the two types of MDS regarding the myeloblast count. CONCLUSIONS: The increased number of central immature precursor cells in MDS was not directly correlated with the extent of the juxtatrabecular fibrosis. Although the number of cases is small, the fact that the juxtatrabecular fibrosis was higher in the two deceased patients in the series (49% and 56%, respectively) suggests a poor-risk prognosis for such finding.

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Estudiamos retrospectivamente a 18 pacientes con sindrome mielodisplásico (SMD) en un periodo de 7 años (enero de 1984 a diciembre de 1990) en el Hospital Nacional Cayetano Heredia. Diez fueron mujeres y ocho varones, la edad promedio fue de 61.1 años. El 61 por ciento presento pancitopenia; todos presentaron anemia, 83 por ciento trombocitopenia y el 65 por ciento neutropenia. Las medulas oseas fueron hiper o normocelulares; 95 por ciento de los pacientes presento diseritropoyesis y el 89 por ciento presento dismegacariopoyesis micromegacariocitos. De acuerdo a la clasificación FAB 44 por ciento presento anemia refractaria con sideroblastos en anillo (ARSA), 16.7 por ciento anemia refractaria con exeso de blastos (AREB), 22.2 por ciento con anemia refractaria con exesos de blastos en transformación (AREBt) y un 5.5 por ciento fue diagnosticado con leucemia mielomonocítica crónica (LMMC). Cinco pacientes (27.8 por ciento) se transformaron a otra variedad; cuatro de estos (22.2 por ciento) se transformaron a LMA. Un paciente con localización de precursores anormal en la biopsia de hueso evoluciono a LMA. Las complicaciones mas frecuentes fueron las infecciones (45.8 por ciento), un 16.6 por ciento tuvieron hemorragia en el SNC. El tiempo medio de sobrevida desde el diagnóstico fue 9.2 meses

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BACKGROUND AND METHODS: We analyze myelodysplastic patients with the 5q- marker as sole abnormality, or with additional anomalies, and their relationship to clinical evolution. The study was performed on 12 patients (6 females and 6 males): 3 with refractory anemia (RA), 5 with RA with ring sideroblasts (RA-S), 3 RA with excess of blasts (RAEB) and 1 with RAEB in transformation (RAEB-T). The cytogenetic study was carried out on bone marrow cells at the time of diagnosis. The G- banding technique was used for chromosome identification. The follow-up was for 50 months. RESULTS AND CONCLUSIONS: Five patients (3 with RA and 2 with RA-S) showed a single 5q- marker. They had a long survival without evolution to leukemia. All cases with 5q- plus additional abnormalities: del(12p), del(7q), del(14q), i(11q) and i(17q), showed a neoplastic evolution in a short period of time. We can conclude that the presence of the 5q- marker with complex karyotypes or additional abnormalities is associated with a high risk of neoplastic evolution, indicating the prognostic value of cytogenetic study in myelodysplasia.

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T cryptantigen can be exposed on the red cell membrane as a result of removal of terminal glycosides, either by bacterial enzymes or by incomplete synthesis of the cell membrane due to somatic mutation, usually caused by a neoplasm. T-activated erythrocytes have been observed in different pathologies, but they have not been seen associated with other abnormalities of red blood cell proteins described in myelodysplastic syndromes or acute leukaemias. A patient with initial diagnosis of refractory anaemia that evolved into erythroleukaemia showed prolonged T-activation, a depressed A blood-group antigen and an increase of foetal haemoglobin, simultaneously. The evolutive pattern of T-activation suggests more an abnormal erythropoiesis than an enzymatic effect and a certain relationship with the haemolytic syndrome.

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