Long-term Impact of Lanadelumab on Patients with Hereditary Angioedema (HAE) Type 1/2: Patient Reported Outcome (PRO) Findings from the HELP Open-label Extension Study (OLE)

2021 ◽  
Vol 147 (2) ◽  
pp. AB24
Author(s):  
Maureen Watt ◽  
Marcus Maurer ◽  
Giovanna Devercelli ◽  
Kim Paes ◽  
Antoine Regnault ◽  
...  
Keyword(s):  
Hereditary Angioedema ◽  
Patient Reported Outcome ◽  
Extension Study ◽  
Open Label ◽  
Patient Reported ◽  
Open Label Extension ◽  
Long Term Impact

Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria

Blood ◽  
2005 ◽  
Vol 106 (7) ◽  
pp. 2559-2565 ◽  
Author(s):  
Anita Hill ◽  
Peter Hillmen ◽  
Stephen J. Richards ◽  
Dupe Elebute ◽  
Judith C. Marsh ◽  
...  
Keyword(s):  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Extension Study ◽  
Transfusion Rate ◽  
Complement Protein ◽  
Open Label ◽  
Hematologic Disorder ◽  
Humanized Monoclonal Antibody ◽  
Close Relationship ◽  
Open Label Extension

AbstractParoxysmal nocturnal hemoglobinuria (PNH) is a hematologic disorder characterized by clonal expansion of red blood cells (RBCs) lacking the ability to inhibit complement-mediated hemolysis. Eculizumab, a humanized monoclonal antibody that binds the C5 complement protein, blocks serum hemolytic activity. This study evaluated the long-term safety and efficacy of eculizumab in 11 patients with PNH during an open-label extension trial. After completion of an initial 12-week study, all patients chose to participate in the 52-week extension study. Eculizumab, administered at 900 mg every 12 to 14 days, was sufficient to completely and consistently block complement activity in all patients. A dramatic reduction in hemolysis was maintained throughout the study, with a decrease in lactate dehydrogenase (LDH) levels from 3110.7 IU/L before treatment to 622.4 IU/L (P = .002). The proportion of PNH type III RBCs increased from 36.7% at baseline to 58.4% (P = .005). The paroxysm rate of days with gross evidence of hemoglobinuria per patient each month decreased from 3.0 during screening to 0.2 (P < .001) during treatment. The median transfusion rate decreased from 1.8 U per patient each month before eculizumab treatment to 0.3 U per patient each month (P = .001) during treatment. Statistically significant improvements in quality-of-life measures were also maintained during the extension study. Eculizumab continued to be safe and well tolerated, and all patients completed the study. The close relationship between sustained terminal complement inhibition, hemolysis, and symptoms was demonstrated. (Blood. 2005; 106:2559-2565)

scholarly journals Long-term efficacy and safety of once-monthly Risperidone ISM® in the treatment of schizophrenia: Results from a 12-month open-label extension study

2022 ◽  
Vol 239 ◽  
pp. 83-91
Author(s):  
Yuriy Filts ◽  
Robert E. Litman ◽  
Javier Martínez ◽  
Lourdes Anta ◽  
Dieter Naber ◽  
...  
Keyword(s):  
Extension Study ◽  
Efficacy And Safety ◽  
Open Label ◽  
Term Efficacy ◽  
Open Label Extension

Long-Term Effectiveness and Safety of Vedolizumab in Patients with Ulcerative Colitis: 5-Year Cumulative Exposure of Gemini 1 Completers Rolling into the Gemini Open-Label Extension Study

2017 ◽  
Vol 152 (5) ◽  
pp. S602 ◽  
Author(s):  
Edward V. Loftus ◽  
Jean Frederic Colombel ◽  
Brian G. Feagan ◽  
Severine Vermeire ◽  
William J. Sandborn ◽  
...  
Keyword(s):  
Ulcerative Colitis ◽  
Cumulative Exposure ◽  
Extension Study ◽  
Open Label ◽  
Open Label Extension
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