Eliglustat tartrate (Genz-112638), a specific inhibitor of glucosylceramide synthase, is under development as an oral substrate reduction therapy for Gaucher disease type 1 (GD1
This multicenter, open-label study evaluated the efficacy and safety of decitabine in patients from Argentina and South Korea with myelodysplastic syndromes or chronic myelomonocytic leukemia. Of 106 patients who received decitabine 20 mg/m(2) intravenously over 1 h once daily for 5 days in 4-week cycles, 99 patients were evaluable after receiving at least two cycles. The overall improvement rate was 35% (19% complete response +4% marrow complete response +4% partial response +8% hematologic improvement). Overall survival at 2 years was 71%. Treatment-related adverse events included febrile neutropenia, thrombocytopenia and bleeding, asthenia, fatigue, and eosinophilia. After complete response (CR), three patients received an allogeneic stem cell transplant. Four patients who relapsed after CR responded to decitabine retreatment. Acute myelogenous leukemia developed during follow-up in 21% of patients. Decitabine in a 5-day outpatient administration schedule was effective and well tolerated in typical clinical practice settings in South America and Asia.
Few salvage treatments are successful for patients with relapsed acute myelogenous leukemia after a short first remission, multiple relapses, or for patients with disease refractory to initial induction chemotherapy. To improve the results of salvage therapy we studied the efficacy and toxicity of a combination of G-CSF (5 mu tg/kg IV q day) used as a priming agent followed by continued exposure to G-CSF and high-dose cyatarabine (2 gm/m(2) IV q 12 hours x 12 doses) in fifteen adult patients with relapsed or refractory acute myelogenous leukemia. Nine of fourteen (64%; 95% confidence interval 35 to 87%) achieved complete remission, four failed to enter remission and one died of multiorgan system failure after progressive leukemia cutis despite chemotherapy-induced bone marrow aplasia. Median disease-free survival is 148 days and median survival from study entry for responding patient is 174 days. Three patients who achieved complete remission subsequently relapsed with a median time to relapse of 147 days. Median time to granulocyte >0.5 x 10(9)/L was 22 days (19 to 34 days) and the median time to platelet recovery >20 x 10(9)/L was 30 days (23 to 214 days). Although gastrointestinal toxicity was common, no patient developed severe cardiac, hepatic, pulmonary, or neurologic complications. An elevation in the percent of bone marrow blasts in S-phase after 48 hours of treatment with G-CSF was identified in 7 of 12 evaluable patients. These results demonstrate that the combination of G-CSF and high-dose cytarabine may be used as an effective salvage treatment for patients with resistant acute myelogenous leukemia.
Introducción: La morbimortalidad por COVID-19 se asocia principalmente con el síndrome respiratorio agudo severo (SARS), relacionado con una respuesta inmunitaria aumentada del huésped con aumento de los niveles circulantes de citoquinas. Métodos: En este estudio prospectivo, multicéntrico, de un solo brazo (en comparación con un control histórico), en fase 2, se agregó ruxolitinib 5 mg dos veces al día al estándar de tratamiento en pacientes con COVID-19. El objetivo principal fue determinar la eficacia y seguridad de ruxolitinib en pacientes con SARS relacionado con COVID-19. Resultados: aunque no fue posible demostrar una reducción significativa de la proporción de pacientes con neumonía por COVID-19 que requerían ingreso en la unidad de cuidados intensivos y ventilación mecánica (criterio de valoración principal), se observó una tendencia a una menor tasa de mortalidad en los pacientes críticos que recibieron ruxolitinib. La dosis de ruxolitinib administrada tuvo que aumentarse de acuerdo con el protocolo en el 32% de los pacientes, sin toxicidad adicional. Conclusión: El perfil de efectos secundarios fue manejable y el fármaco en estudio no causó lesiones orgánicas directas. El ruxolitinib tuvo un efecto antiinflamatorio rápido y un tercio de los pacientes manifestó bienestar inmediatamente después de comenzar el tratamiento.
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