scholarly journals Long-term safety and efficacy of subcutaneous immunoglobulin IgPro20 in CIDP

2019 ◽  
Vol 6 (5) ◽  
pp. e590 ◽  
Author(s):  
Ivo N. van Schaik ◽  
Orell Mielke ◽  
Vera Bril ◽  
Nan van Geloven ◽  
Hans-Peter Hartung ◽  
...  
Keyword(s):  
Dose Reduction ◽  
Chronic Inflammatory Demyelinating Polyneuropathy ◽  
Term Treatment ◽  
Extension Study ◽  
Open Label ◽  
Safety And Efficacy ◽  
Long Term Treatment ◽  
Inflammatory Neuropathy ◽  
Relapse Rates

ObjectiveTo investigate the long-term safety and efficacy of weekly subcutaneous IgPro20 (Hizentra, CSL Behring) in chronic inflammatory demyelinating polyneuropathy (CIDP).MethodsIn a 48-week open-label prospective extension study to the PATH study, patients were initially started on 0.2 g/kg or on 0.4 g/kg weekly and—if clinically stable—switched to 0.2 g/kg weekly after 24 weeks. Upon CIDP relapse on the 0.2 g/kg dose, 0.4 g/kg was (re)initiated. CIDP relapse was defined as a deterioration by at least 1 point in the total adjusted Inflammatory Neuropathy Cause and Treatment score.ResultsEighty-two patients were enrolled. Sixty-two patients initially received 0.4 g/kg, 20 patients 0.2 g/kg weekly. Seventy-two received both doses during the study. Sixty-six patients (81%) completed the 48-week study duration. Overall relapse rates were 10% in 0.4 g/kg–treated patients and 48% in 0.2 g/kg–treated patients. After dose reduction from 0.4 to 0.2 g/kg, 51% (27/53) of patients relapsed, of whom 92% (24 of 26) improved after reinitiation of the 0.4 g/kg dose. Two-thirds of patients (19/28) who completed the PATH study without relapse remained relapse-free on the 0.2 g/kg dose after dose reduction in the extension study. Sixty-two patients had adverse events (AEs) (76%), of which most were mild or moderate with no related serious AEs.ConclusionsSubcutaneous treatment with IgPro20 provided long-term benefit at both 0.4 and 0.2 g/kg weekly doses with lower relapse rates on the higher dose. Long-term dosing should be individualized to find the most appropriate dose in a given patient.Classification of evidenceThis study provides Class IV evidence that for patients with CIDP, long-term treatment with SCIG beyond 24 weeks is safe and efficacious.

scholarly journals 46 Confirmed Safety of Deutetrabenazine for Tardive Dyskinesia in a 2-Year Open-label Extension Study

CNS Spectrums ◽  
2019 ◽  
Vol 24 (1) ◽  
pp. 201-201 ◽  
Author(s):  
Hubert H. Fernandez ◽  
David Stamler ◽  
Mat D. Davis ◽  
Stewart A. Factor ◽  
Robert A. Hauser ◽  
...  
Keyword(s):  
Tardive Dyskinesia ◽  
Dose Reduction ◽  
Term Treatment ◽  
Extension Study ◽  
Short Term ◽  
Open Label ◽  
Short Term Treatment ◽  
Study Objective ◽  
Long Term Treatment

AbstractStudy ObjectiveTo evaluate the long-term safety and tolerability of deutetrabenazine in patients with tardive dyskinesia (TD) at 2years.BackgroundIn the 12-week ARM-TD and AIM-TD studies, deutetrabenazine showed clinically significant improvements in Abnormal Involuntary Movement Scale scores compared with placebo, and there were low rates of overall adverse events (AEs) and discontinuations associated with deutetrabenazine.MethodPatients who completed ARM-TD or AIM-TD were included in this open-label, single-arm extension study, in which all patients restarted/started deutetrabenazine 12mg/day, titrating up to a maximum total daily dose of 48mg/day based on dyskinesia control and tolerability. The study comprised a 6-week titration period and a long-term maintenance phase. Safety measures included incidence of AEs, serious AEs (SAEs), and AEs leading to withdrawal, dose reduction, or dose suspension. Exposure-adjusted incidence rates (EAIRs; incidence/patient-years) were used to compare AE frequencies for long-term treatment with those for short-term treatment (ARM-TD and AIM-TD). This analysis reports results up to 2 years (Week106).Results343 patients were enrolled (111 patients received placebo in the parent study and 232 received deutetrabenazine). There were 331.4 patient-years of exposure in this analysis. Through Week 106, EAIRs of AEs were comparable to or lower than those observed with short-term deutetrabenazine and placebo, including AEs of interest (akathisia/restlessness [long-term EAIR: 0.02; short-term EAIR range: 0–0.25], anxiety [0.09; 0.13–0.21], depression [0.09; 0.04–0.13], diarrhea [0.06; 0.06–0.34], parkinsonism [0.01; 0–0.08], somnolence/sedation [0.09; 0.06–0.81], and suicidality [0.02; 0–0.13]). The frequency of SAEs (EAIR 0.15) was similar to those observed with short-term placebo (0.33) and deutetrabenazine (range 0.06–0.33) treatment. AEs leading to withdrawal (0.08), dose reduction (0.17), and dose suspension (0.06) were uncommon.ConclusionsThese results confirm the safety outcomes seen in the ARM-TD and AIM-TD parent studies, demonstrating that deutetrabenazine is well tolerated for long-term use in TD patients.Presented at: American Academy of Neurology Annual Meeting; April 21–27, 2018, Los Angeles, California,USAFunding Acknowledgements: Funding: This study was supported by Teva Pharmaceuticals, Petach Tikva, Israel

scholarly journals Long-term treatment of Cushing’s disease with pasireotide: 5-year results from an open-label extension study of a Phase III trial

Endocrine ◽  
2017 ◽  
Vol 57 (1) ◽  
pp. 156-165 ◽  
Author(s):  
S. Petersenn ◽  
L. R. Salgado ◽  
J. Schopohl ◽  
L. Portocarrero-Ortiz ◽  
G. Arnaldi ◽  
...  
Keyword(s):  
Cushing’S Disease ◽  
Term Treatment ◽  
Phase Iii ◽  
Cushing's Disease ◽  
Extension Study ◽  
Open Label ◽  
Phase Iii Trial ◽  
Long Term Treatment ◽  
Open Label Extension

scholarly journals 158 Long-Term Safety of Deutetrabenazine for the Treatment of Tardive Dyskinesia: Results From an Open-Label, Long-Term Study

CNS Spectrums ◽  
2018 ◽  
Vol 23 (1) ◽  
pp. 97-97
Author(s):  
Karen E. Anderson ◽  
Mat D. Davis ◽  
Stewart A. Factor ◽  
Robert A. Hauser ◽  
L. Fredrik Jarskog ◽  
...  
Keyword(s):  
Dose Reduction ◽  
Involuntary Movement ◽  
Incidence Rates ◽  
Extension Study ◽  
Total Daily Dose ◽  
Open Label ◽  
Long Term Study ◽  
Long Term Treatment ◽  
Pharmaceutical Industries

AbstractIntroductionIn the 12-week ARM-TD and AIM-TD studies, deutetrabenazine showed clinically significant improvements in Abnormal Involuntary Movement Scale (AIMS) scores at Week 12 compared with placebo, and was generally well tolerated.ObjectiveTo evaluate the long-term safety/tolerability and efficacy of deutetrabenazine in patients with TD. Week 54 open-labelresults are reported in this interim analysis.MethodsPatients with TD who completed ARM-TD or AIM-TD were included in this open-label, single-arm extension study, in which all patients restarted/started deutetrabenazine 12 mg/day, titrating up to a maximum total daily dose of 48 mg/day based on dyskinesia control and tolerability. The study comprised a 6-week titration period and a long-term maintenance phase. Safetymeasures included incidence of adverse events (AEs), serious AEs (SAEs), drug-related AEs, and AEs leading to withdrawal, dose reduction, or dose suspension. This analysis reports results up to Week 54.Results304 patients enrolled in the extension study. There were 215 patient-years of exposure in this analysis, and exposure-adjusted incidence rates (EAIRs) of AEs (incidence/patient-years) were comparable to or lower than those observed with short-term deutetrabenazine treatment and placebo. The frequency of SAEs (EAIR 0.14) was similar to rates observed with short-termplacebo (EAIR 0.33) and deutetrabenazine (EAIR range 0.06–0.33) treatment. AEs leading to study discontinuation (EAIR 0.08), dose reduction (EAIR 0.17), and dose suspension (EAIR 0.09) were uncommon.ConclusionsLong-term treatment with deutetrabenazine was generally safe and well tolerated in patients with TD, and did not result in cumulative toxicity.Presented at: The American Psychiatric Association 2017 Annual Meeting; May 20–24, 2017; San Diego, California, USA.Funding AcknowledgementsThis study was funded by Teva Pharmaceutical Industries, Petach Tikva, Israel.

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