ABSTRACT
Fostamatinib, a recently approved syk inhibitor used in adult primary immune thrombocytopenia (ITP), has been shown to be safe and effective in this disorder. However, clinical trial results may not be similarly reproduced in clinical practice. Here 138 ITP patients (both primary and secondary) from 42 Spanish centers who had been treated with fostamatinib were evaluated prospectively and retrospectively. The median age of our cohort (55.8% women) was 66 years (interquartile range, IQR, 56-80 years). The median time since ITP diagnosis at fostamatinib initiation was 51 months (IQR, 10-166 months). The median number of therapies prior to fostamatinib initiation was 4 (IQR, 2-5), including eltrombopag (76.1%), romiplostim (57.2%) and intravenous immunoglobulins (IVIG) (44.2%). Fifty-eight patients (42.0%) had signs/symptoms of bleeding in the month prior to treatment initiation. 79.0% of patients responded to fostamatinib with 53.6% complete responses (platelet count > 100 x 109 /L). Eighty-three patients (60.1%) received fostamatinib monotherapy achieving a high response rate (85.4%). The proportion of time in response during the 27-month period examined was 83.3%. The median time to platelet response was 11 days (IQR, 7-21 days). Sixty-seven patients (48.5%) experienced adverse events, mainly grade 1-2, the commonest of which were diarrhea (n = 28) and hypertension (n = 21). One patient had deep venous thrombosis and one patient developed acute myocardial infarction. Fostamatinib was shown to be effective with good safety profile in patients with primary and secondary ITP across a wide age spectrum in this real-world study.
ABSTRACT
(1) Background: New therapeutic strategies have improved the prognosis of multiple myeloma (MM), changing the accepted view of this disease from being incurable to treatable. (2) Methods: We studied 1001 patients with MM between 1980 and 2020, grouping patients into ten-year periods by diagnosis 1980-1990, 1991-2000, 2001-2010 and 2011-2020. (3) Results: After 65.1 months of follow-up, the median OS of the cohort was 60.3 months, and OS increased significantly over time: 22.4 months in 1980-1990, 37.4 months in 1991-2000, 61.8 months in 2001-2010 and 103.6 months in 2011-2020 (p < 0.001). Using novel agents in the front-line setting for myeloma patients yielded a significantly better OS than in those treated with conventional therapies, especially when combinations of at least two novel agents were used. The median OS of patients treated with the combination of at least two novel agents in induction was significantly prolonged compared to those treated with a single novel agent or conventional therapy in induction: 143.3 vs. 61.0 vs. 42.2 months (p < 0.001). The improvement was apparent in all patients regardless of age at diagnosis. In addition, 132 (13.2%) patients were long-term survivors (median OS ≥ 10 years). Some independent clinical predictors of long-term survival were identified: ECOG < 1, age at diagnosis ≤ 65 years, non-IgA subtype, ISS-1 and standard-risk cytogenetic. Achieving CR and undergoing ASCT were positively associated with >10 years of survival. (4) Conclusions: The combination of novel agents appears to be the main factor for the improvement in survival in MM, which is becoming a chronic and even curable disease in a subtype of patients without high-risk features.
Subject(s)
Flow Cytometry , Immunophenotyping , Multiple Myeloma/diagnosis , Multiple Myeloma/metabolism , Neoplastic Cells, Circulating/metabolism , Plasma Cells/metabolism , Flow Cytometry/methods , Humans , Immunophenotyping/methods , Multiple Myeloma/mortality , Multiple Myeloma/therapy , Neoplastic Cells, Circulating/pathology , Plasma Cells/pathology , PrognosisABSTRACT
BACKGROUND: We aimed to assess the accuracy of a diagnostic strategy including broth clinical assessment and determination of D-dimer (DD) in patients with clinically suspicion of low pretest probability of deep venous thrombosis (DVT). METHOD: 149 outpatients (mean age 69; SD 16) with clinically suspected proximal DVT attending our Emergency Department and classified as low pretest probability were included in an observational prospective study. In patients with a DD (STA Liatest D-Di, Diagnostica Stago, Asnières sur Seine, France) concentration below the cut-off value (0.4 ng/ml) the diagnosis of DVT was readily ruled out, whereas those individuals with a positive DD result underwent compression Doppler venous ultrasound. A 3-month clinical follow-up was carried out in those patients in whom a diagnosis of DVT was initially excluded. RESULTS: Only 2 cases of DVT were confirmed (prevalence 1.3%; CI 95%, 0.2-5.3). In 47.6% of cases, a DD negativity ruled out the diagnosis of DVT. The rate of negative DD results was significantly lower in patients below 70 years of age as compared to older patients (73.6 versus 33.3%) (p < 0.001). Sensitivity, specificity, positive predictive value and negative predictive value of DD in our series were 100% (CI 95%,19.7-95.1), 48.3% (CI 95%, 40.0-56.7), 2.6% (CI 95%, 0.4-9.8) and 100% (CI 95%, 93.6-99.8) respectively, the latter being similar in the two age groups. No case of DVT was diagnosed during the follow-up period. CONCLUSIONS: In patients with a low pretest probability of DVT a negative DD result reliably and safely rules out such diagnosis. However, the diagnostic value of DD is lower in elderly patients (>= 70 years of age) due to a lower rate of negative results in these individuals.
Subject(s)
Fibrin Fibrinogen Degradation Products/analysis , Venous Thrombosis/diagnosis , Aged , Follow-Up Studies , Humans , Prospective Studies , Reproducibility of Results , Venous Thrombosis/bloodABSTRACT
FUNDAMENTO: Análisis de una estrategia diagnóstica que incluye la evaluación clínica y el dímero-D (DD) en pacientes con sospecha clínica de baja probabilidad de trombosis venosa profunda (TVP). MÉTODO: Se ha realizado un estudio observacional y prospectivo que incluía a 149 pacientes (edad media [DE] 69 [16] años) que acudieron al Servicio de Urgencias de nuestro hospital con una sospecha clínica de TVP proximal y que fueron clasificados como de baja probabilidad clínica. En aquellos sujetos con un valor de DD (STA Liatest® D-Di, Diagnostica Stago, Asnières sur Seine, Francia) negativo (< 0,4 ng/ml) se consideró como excluido el diagnóstico, mientras que en aquellos con positividad de dicho parámetro se indicó una ecografía Doppler de compresión venosa. Se ha llevado a cabo un seguimiento clínico de 3 meses de duración en aquellos pacientes en los que el diagnóstico de TVP fue inicialmente excluido. RESULTADOS: Se confirmaron dos casos de TVP (prevalencia, 1,3 por ciento; intervalo de confianza [IC] del 95 por ciento, 0,2-5,3). En un 47,6 por ciento de los casos el diagnóstico de TVP fue excluido debido a la negatividad del DD; la proporción de resultados negativos del DD fue significativamente inferior en el grupo de pacientes menores de 70 años (73,6 por ciento) que en los de dicha edad o mayores (33,3 por ciento) (p < 0,001). La sensibilidad, la especificidad, el valor predictivo positivo y el valor predictivo negativo del DD en nuestra serie fueron del 100 por ciento (IC del 95 por ciento, 19,7-95,1), el 48,3 por ciento (IC del 95 por ciento, 40,0-56,7), el 2,6 por ciento (IC del 95 por ciento, 0,4-9,8) y el 100 por ciento (IC del 95 por ciento, 93,6-99,8), respectivamente, siendo este último similar en los dos grupos de edad mencionados. No se diagnosticó ningún caso de TVP durante el período de seguimiento. CONCLUSIONES: En pacientes con baja probabilidad clínica de TVP proximal, la negatividad del DD excluye el diagnóstico de un modo seguro, si bien el valor de este parámetro es menor en pacientes ancianos ( 70 años) debido a la menor proporción de resultados negativos en este grupo de pacientes (AU)
Subject(s)
Aged , Humans , Reproducibility of Results , Prospective Studies , Venous Thrombosis , Follow-Up Studies , Fibrin Fibrinogen Degradation ProductsABSTRACT
La mayoría de los casos de urticaria aguda son de causa desconocida. La infección aguda por el virus de Epstein-Barr se puede manifestar, infrecuentemente, en forma de urticaria a frigore. Reconocer esta sintomatología puede permitir un diagnóstico precoz de la mononucleosis infecciosa y descartar otras causas de urticaria aguda como ocurrió en la paciente de 17 años que aquí se describe (AU)
