scholarly journals Improvement of Renal Function by Long-Term Sustained Eculizumab Treatment in a Patient with Paroxysmal Nocturnal Hemoglobinuria

2015 ◽  
Vol 2015 ◽  
pp. 1-4
Author(s):  
Haruhiko Ninomiya ◽  
Naoshi Obara ◽  
Akiko Niiori-Onishi ◽  
Yasuhisa Yokoyama ◽  
Mamiko Sakata-Yanagimoto ◽  
...  
Keyword(s):  
Renal Function ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Estimated Glomerular Filtration Rate ◽  
Intravascular Hemolysis ◽  
Open Label ◽  
Open Label Study ◽  
Anaphylatoxin C5a ◽  
Advanced Stages ◽  
Interstitial Deposition

Chronic kidney disease (CKD) is one of the major manifestations of paroxysmal nocturnal hemoglobinuria (PNH). CKD in PNH is induced mainly by intravascular hemolysis of PNH-affected red blood cells (RBC) missing the glycosylphosphatidylinositol-anchored proteins with complement-regulatory activities, CD55 and CD59. CKD develops by heme absorption in the proximal tubules resulting in the interstitial deposition of iron in the kidneys. We administered eculizumab to a patient with PNH, who was one of 29 patients enrolled in the AEGIS clinical trial, an open-label study of eculizumab in Japan. The patient was complicated by stage 3 CKD with impaired estimated glomerular filtration rate (eGFR), at grade G3b, and had obvious proteinuria (2-3+, 1-2 g/day). In a two-year extension to the 12-week AEGIS study, eGFR improved significantly, and the eGFR has since been maintained at grade G2 without proteinuria by sustained eculizumab treatment (>6 years). Renal function improved and maintained by long-term sustained eculizumab treatment, presumably by clearance of iron from the kidney as well as inhibition of the production of anaphylatoxin C5a, even in advanced stages of CKD, is one of the benefits of eculizumab treatment in PNH.

Open-Label Study (ARIDO) Evaluating Long-Term Safety of Topical Glycopyrronium Tosylate (GT) in Patients with Axillary Hyperhidrosis

2017 ◽  
Vol 1 ◽  
pp. s95 ◽  
Author(s):  
Dee Anna Glaser ◽  
Adelaide A Hebert ◽  
Alexander Nast ◽  
William P Werschler ◽  
Stephen Shideler ◽  
...  
Keyword(s):  
Axillary Hyperhidrosis ◽  
Open Label ◽  
Open Label Study ◽  
Label Study

Abstract Not AvailableDisclosure: Study supported by Dermira.

Augmentation strategy with olanzapine in resistant obsessive compulsive disorder: an Italian long-term open-label study

2005 ◽  
Vol 19 (4) ◽  
pp. 392-394 ◽  
Author(s):  
Donatella Marazziti ◽  
Chiara Pfanner ◽  
Bernardo Dell’osso ◽  
Antonio Ciapparelli ◽  
Silvio Presta ◽  
...  
Keyword(s):  
Obsessive Compulsive Disorder ◽  
Obsessive Compulsive ◽  
Open Label ◽  
Open Label Study ◽  
Label Study ◽  
Compulsive Disorder ◽  
Augmentation Strategy

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)

Fluvoxamine in the Treatment of Trichotillomania: An 8-Week, Open-Label Study

CNS Spectrums ◽  
1998 ◽  
Vol 3 (9) ◽  
pp. 64-71 ◽  
Author(s):  
Gary A. Christenson ◽  
Scott J. Crow ◽  
James E. Mitchell ◽  
Thomas B. Mackenzie ◽  
Ross D. Crosby ◽  
...  
Keyword(s):  
Treatment Response ◽  
Term Treatment ◽  
Hair Pulling ◽  
Short Term ◽  
Open Label ◽  
Short Term Treatment ◽  
Open Label Study ◽  
Label Study ◽  
Long Term Treatment

AbstractThis short-term, open-label study investigates short- and long-term effects of the selective serotonin reuptake inhibitor (SSRI) fluvoxamine for the treatment of trichotillomania (TTM). Additionally, this study aimed to test the hypothesis that the presence of hair pulling compulsiveness is predictive of SSRI response. Nineteen subjects meeting the Diagnostic and Statistical Manual of Mental Disorders, Third Edition Revised, (DSM-III-R) criteria for TTM were treated with fluvoxamine at doses up to 300 mg/day. Random regression analysis of change across time for patients who completed the study (n=14) and those who dropped out (n=5) revealed statistically significant improvements in Physician Rating Scale, hair-pulling episodes, Trichotillomania Impairment Scale, and Trichotillomania Symptom Severity Scale, but not in estimated amount of hair pulled. In addition, the percentage of patients' focused or compulsive hair-pulling symptoms was predictive of treatment response. Unfortunately, all three subjects who entered long-term treatment displayed substantial movement back toward baseline by the end of 6 months. We concluded that fluvoxamine produces moderate reductions in symptoms during the short-term treatment of TTM and that the presence of focused or compulsive hair pulling may be predictive of treatment response. However, responses may be short lived when treatment is extended.

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