Publications by authors named "Maria Soledad Noya-Pereira"
Background: The availability of multiple treatments for type 1 Gaucher disease increases the need for real-life studies to evaluate treatment efficacy and safety and provide clinicians with more information to choose the best personalized therapy for their patients.
Aims: To determine whether treatment with eliglustat produces, in adult GD1 patients, ans optimal response in daily clinical practice.
Methods: We designed a real-life study with 2 years of follow-up (TRAZELGA [GEE-ELI-2017-01]) to uniformly evaluate the response and adverse events to eliglustat treatment.
Article Synopsis
- The primary goal of therapy for essential thrombocythemia (ET) is to lower the risk of blood clots, with a retrospective analysis of 1,381 ET patients revealing a low rate of major bleeding (MB) events.
- Factors such as age and leukocyte count increase the risk of MB, whereas low hemoglobin levels appear to offer some protection; however, existing risk scoring systems for thrombosis do not predict hemorrhage effectively.
- Anticoagulation therapies, particularly vitamin K antagonists, significantly raise the risk of MB events in ET patients, highlighting the need for improved risk assessment and further research on treatment options in ET management.
Article Synopsis
- Thrombosis and haemorrhage are common issues for patients with essential thrombocythaemia (ET), and the revised r-IPSET-t score is more effective than the original in predicting these risks among patients.
- In a study involving 1,381 ET patients over about 88 months, thrombotic events occurred at a rate of 0.578 per person-year, with a remarkable 10-year thrombosis-free survival of 88% for low-risk and 99% for very-low-risk groups.
- The findings also highlight concerns about the use of cytoreductive therapy and aspirin, which may pose additional risks for certain patients, despite the European LeukemiaNET not recommending aspirin for very-low-risk individuals.
Objectives: In patients with essential thrombocythemia (ET), after the JAK2V617F driver mutation, mutations in CALR are common (classified as type 1, 52-bp deletion or type 2, 5-bp insertion). CALR mutations have generally been associated with a lower risk of thrombosis. This study aimed to confirm the impact of CALR mutation type on thrombotic risk.
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