Publications by authors named "M Morado-Arias"
This work is a review of the scientific evidence on the oral treatment of adult patients with Gaucher disease type 1 (GD1) with a clinical guideline format according to the Agree II regulations. It describes the main differences between the two oral treatments currently available for treating this disease (miglustat and eliglustat). This review reminds us that the criteria for starting oral treatment in patients with GD1 must be assessed individually.
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- Pyruvate kinase deficiency is a rare genetic disorder leading to chronic hemolytic anemia, and the phase 3 trial assessed the impact of mitapivat, a new medication, on this condition.
- In the trial, adults not on regular blood transfusions were randomized to receive either mitapivat or a placebo for 24 weeks, with the main goal of seeing significant increases in hemoglobin levels.
- Results showed that 40% of patients on mitapivat achieved a notable hemoglobin response compared to none in the placebo group, with some common side effects including nausea and headaches, but overall, mitapivat demonstrated a better efficacy in treating this disorder.
Introduction: Autoimmune haemolytic anaemia (AIHA) is an infrequent and heterogeneous disease in its pathophysiology and clinical behaviour, therefore it is generally managed empirically.
Patients And Methods: We conducted an observational, retrospective and multicentre study of 93 patients diagnosed with AHAI in 9 Spanish hospitals between 1987 and 2017, with a median follow-up of 28 months.
Results: Median age of 67 years; 85% AHAI for hot antibodies and 64% primary AHAI.