Fostamatinib effectiveness and safety for immune thrombocytopenia in clinical practice.

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.

Treatment with fostamatinib in patients with immune thrombocytopenia: Experience from the Andalusian region in Spain-The Fostasur Study.

Immune thrombocytopenia (ITP) is characterized by low platelet counts (PLTs) and an increased risk of bleeding. Fostamatinib, a spleen tyrosine kinase inhibitor, has been approved as a second-line treatment for ITP. Real-world data on fostamatinib are lacking. This observational, retrospective, multicentre study, conducted in the Andalusia region of Spain, evaluated 44 adult primary ITP patients (47.7% female; median age 58 years; newly diagnosed ITP 6.8%; persistent 13.6%; chronic 79.5%; median four prior treatments) after ≥ 4 weeks of fostamatinib therapy. The median PLT at the initiation of fostamatinib was 15 × 109/L. Common reasons for starting fostamatinib were refractoriness or intolerance to prior therapy, oral medication preference, history of thrombosis and cardiovascular risk. Dosing was individualized based on efficacy and tolerance. After 2 weeks, global response rate was 56.8% (response and complete response). Response rates were 70.5%, 62.5% and 64% at 4 weeks, 12 weeks and at the end of the study respectively. Adverse events were mild, and no patients discontinued as a result. This real-world study demonstrated a response rate similar to fostamatinib as seen in the pivotal clinical trials while including newly diagnosed patients and allowing for individualized dosing.

The influence of the age in the risk of the prothrombin G20210A mutation for spontaneous venous thrombosis.

BACKGROUND AND OBJECTIVE: Factor V Leiden mutation (FVL), and the prothrombin G20210A mutation (PT G20210A) are polymorphisms with a weak risk factor for venous thromboembolic disease. The probability of spontaneous venous thrombosis in carriers of thrombophilic mutations (FVL and PT G20210A) was analyzed. PATIENTS AND METHOD: We studied 735 individuals (407 were healthy controls and 328 patients with venous thrombosis) with respect to FVL and PT G20210A, determined by polimerase chain reaction in liquid phase, real time. chi2 test and logistic regression analysis were used to evaluate the results. The dates were analyzed with the statistical program SPSS v. 14.0. RESULTS: The carrier patients of PT G20210A mutation whose age was over 40 years had a risk factor for spontaneous venous thrombosis which was 9.28 (odds ratio [OR]) (95% confidence interval [CI], 3.01-28.60; p < 0.0005) times greater than carriers of the PT G20210A mutation who were 40 year-old or younger. In our patients, being male meant a weak risk factor for venous spontaneous thrombosis (p = 0.021; OR = 1.64; 95% CI, 1.08-2.51) . CONCLUSIONS: Our results demonstrate a potentiation of the thrombotic risk by an increase of age in the carriers of the PT G20210A mutation.

Edad y mutación de la protrombina G20210A en los pacientes con trombosis venosa espontánea / The influence of the age in the risk of the prothrombin G20210A mutation for spontaneous venous thrombosis

Fundamento y objetivo: El factor V Leiden (FVL) y la mutación de la protrombina G20210A (PT G20210A) son unos polimorfismos que suponen un débil factor de riesgo para presentar enfermedad tromboembólica venosa. Hemos analizado la probabilidad de tener una trombosis venosa espontánea en los portadores de las mutaciones trombofílicas (FVL y PT G20210A). Pacientes y método: Hemos estudiado a 735 personas (407 controles sanos y 328 pacientes con trombosis venosa) con relación al FVL y a la PT G20210A, determinados por reacción en cadena de la polimerasa en fase líquida, en tiempo real. Para evaluar los resultados utilizamos el test de la x2 y el análisis de regresión logística. Los datos se analizaron con el programa estadístico SPSS versión 14.0. Resultados: Los pacientes con la mutación PT G20210A que eran mayores de 40 años tuvieron un factor de riesgo para trombosis venosa espontánea con una odds ratio (OR) 9,28 (intervalo de confianza (IC) del 95%, 3,01-28,60; p < 0,0005) veces mayor que los portadores de la mutación PT G20210A que tenían 40 años o menos. En nuestra serie el sexo masculino representó un factor de riesgo débil para trombosis venosa espontánea (p = 0,021; OR = 1,64; IC del 95%, 1,08-2,51). Conclusiones: Nuestros resultados demuestran una potenciación del riesgo trombótico por el incremento de la edad en los individuos portadores de la mutación PT G20210A

Background and objective: Factor V Leiden mutation (FVL), and the prothrombin G20210A mutation (PT G20210A) are polymorphisms with a weak risk factor for venous thromboembolic disease. The probability of spontaneous venous thrombosis in carriers of thrombophilic mutations (FVL and PT G20210A) was analyzed. Patients and method: We studied 735 individuals (407 were healthy controls and 328 patients with venous thrombosis) with respect to FVL and PT G20210A, determined by polimerase chain reaction in liquid phase, real time. x2 test and logistic regression analysis were used to evaluate the results. The dates were analyzed with the statistical program SPSS v. 14.0. Results: The carrier patients of PT G20210A mutation whose age was over 40 years had a risk factor for spontaneous venous thrombosis which was 9.28 (odds ratio [OR]) (95% confidence interval [CI], 3.01-28.60; p < 0.0005) times greater than carriers of the PT G20210A mutation who were 40 year-old or younger. In our patients, being male meant a weak risk factor for venous spontaneous thrombosis (p = 0.021; OR = 1.64; 95% CI, 1.08-2.51) . Conclusions: Our results demonstrate a potentiation of the thrombotic risk by an increase of age in the carriers of the PT G20210A mutation

Aplasia pura de serie roja asociada a leucemia linfoide crónica con respuesta favorable a rituximab / Pure red cell aplasia associated with chronic lymphocytic leukaemic succesfuly treated with rituximab

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