This approval offers patients, including those with milder symptoms, a long-acting C5 inhibitor with early onset and reliable efficacy." 1 Earlier intervention can preserve function and quality of life. Howard, MD, distinguished professor of neuromuscular disease, University of North Carolina School of Medicine, said in a statement that, “despite recent advances, managing gMG is complex. At the end of the treatment period, investigators observed statistically significant improvements on the primary end point of Myasthenia Gravis Activities of Daily Living (MG-ADL) total score compared with placebo (–3.1 vs –1.4 P <.001). The approval was based on data from the phase 3 CHAMPION MG trial (NCT03920293), in which treatment with ravulizumab resulted in rapid and sustained improvement of symptoms in patients with gMG for up to 26 weeks. With the decision, ravulizumab becomes the first approved long-acting C5 complement inhibitor for this patient population. Estimates of PNH prognostic factors may serve as baseline data in the assessment of current and future treatments for this disease.The FDA has approved ravulizumab (Ultomiris Alexion), a terminal compliment C5 inhibitor, for the treatment of patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. This large number of cases permitted a detailed analysis of prognostic factors for the first time, in this rare disease. The risk factors for development of myelodysplastic syndrome or acute leukaemia were abdominal pain crisis at presentation (10.5, p = 0.004) and year of diagnosis after 1983 (8.45, p = 0.004). The risk factors for progression to pancytopenia were absence at diagnosis of anaemia (4.03, p = 0.03) and neutropenia (2.45, p = 0.03). Factors associated in multivariate analysis with a high risk of thrombosis during the disease course were thrombosis at diagnosis (5.1, p = 0.0002), age over 54 years (2.6, p = 0.0099). Poor survival was associated with the occurrence of thrombosis as a complication (relative risk 10.2, p < 0.0001), evolution to pancytopenia (5.5, p < 0.0001), myelodysplastic syndrome or acute leukaemia (19.1, p < 0.001), age over 55 years at diagnosis (4, p < 0.0001), need for additional treatment (2.1, p < 0.003), and thrombocytopenia at diagnosis (2.2, p < 0.02). In multivariate analysis, seven factors were significantly associated with survival in patients with PNH. Demographic data, presenting features, initial treatment, complications, and causes of death were similar to those previously reported. 8-year cumulative incidence rates of the main complications (pancytopenia, thrombosis, and myelodysplastic syndrome) were 15% (3), 28% (4), and 5% (2), respectively. The Kaplan-Meier survival estimate was 65% (SE 4) at 10 years and 48% (6) at 15 years after diagnosis. Diagnosis of the disease required, at least, an unequivocally positive Ham's test. We undertook such an investigation.ĭata were collected on 220 patients with PNH diagnosed over a 46-year period (1950-1995) from participating French centres. Although knowledge about the pathophysiology of the disease is increasing, no multivariate analysis of factors influencing survival has been undertaken, mainly because the disease is rare. Paroxysmal nocturnal haemoglobinuria (PNH) is a rare acquired disorder of haematopoietic stem cells.
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