scholarly journals Efficacy and safety of onabotulinumtoxinA with standardized occupational therapy for treatment of pediatric upper limb spasticity: Phase III placebo-controlled randomized trial

2021 ◽  
pp. 1-11
Author(s):  
Rozalina Dimitrova ◽  
Emily McCusker ◽  
Mark Gormley ◽  
Darcy Fehlings ◽  
Katharine E. Alter ◽  
...  
Keyword(s):  
Occupational Therapy ◽  
Upper Limb ◽  
Goal Attainment ◽  
Phase Iii ◽  
Efficacy And Safety ◽  
Secondary Endpoints ◽  
Limb Spasticity ◽  
Goal Attainment Scale ◽  
Global Impression Of Change ◽  
Post Hoc

BACKGROUND: This is the first large study of onabotulinumtoxinA as treatment for pediatric upper limb spasticity. OBJECTIVE: Evaluate efficacy and safety of a single treatment with onabotulinumtoxinA plus occupational therapy (OT). METHODS: In this registrational phase 3, multinational study (NCT01603602), participants were randomized 1:1:1 to onabotulinumtoxinA 3 U/kg/OT, 6 U/kg/OT, or placebo/OT. Primary endpoint was average change from baseline at weeks 4 and 6 in Modified Ashworth Scale-Bohannon (MAS) score. Secondary endpoints included Modified Tardieu Scale (MTS), Clinical Global Impression of Change (CGI) and functional Goal Attainment Scale (GAS). RESULTS: 235 participants were randomized. At weeks 4 and 6, onabotulinumtoxinA groups had greater mean reductions in MAS (both –1.9; p <  0.001) versus placebo (–1.2). OnabotulinumtoxinA doses improved dynamic tone per MTS. Mean CGI at weeks 4 and 6 was unchanged in the overall population, but improved in a post hoc analysis of patients with a single affected UL muscle group (elbow or wrist). GAS score for passive goals was significantly higher for 6 U/kg versus placebo at week 12. Most AEs were mild/moderate in severity; overall incidence was similar between groups. CONCLUSIONS: OnabotulinumtoxinA (3 and 6 U/kg) was safe and effective in reducing upper limb spasticity in pediatric participants.

485 Efficacy and Safety of Once- and Twice-Nightly Dosing of Lower-Sodium Oxybate in Adults With Idiopathic Hypersomnia

SLEEP ◽  
2021 ◽  
Vol 44 (Supplement_2) ◽  
pp. A191-A192
Author(s):  
Isabelle Arnulf ◽  
Anne Marie Morse ◽  
Patricia Chandler ◽  
Rupa Parvataneni ◽  
Dan Chen ◽  
...  
Keyword(s):  
Sodium Oxybate ◽  
Mean Difference ◽  
Controlled Study ◽  
Efficacy And Safety ◽  
Open Label ◽  
Double Blind ◽  
Patient Global Impression ◽  
Idiopathic Hypersomnia ◽  
Secondary Endpoints ◽  
Global Impression Of Change

Abstract Introduction Idiopathic hypersomnia (IH) is a rare central hypersomnolence disorder. In a randomized, controlled study of lower-sodium oxybate (LXB; Xywav™) in adults with IH (NCT03533114), significant differences for LXB compared with placebo were observed in Epworth Sleepiness Scale (ESS; primary efficacy endpoint), self-reported Patient Global Impression of Change (PGIc), and IH Severity Scale (IHSS; key secondary endpoints). In this clinical study, investigators were permitted to initiate LXB dosing on a once-nightly or twice-nightly regimen. Methods Eligible participants aged 18–75 years began LXB treatment, administered once or twice nightly during an open-label treatment/titration and optimization period (OLTTOP; 10–14 weeks); dose amount/regimen could be adjusted during this period. Participants next entered a 2-week, open-label, stable-dose period (SDP), then were randomized to placebo or to continue LXB treatment during a 2-week, double-blind, randomized withdrawal period (DBRWP). P values are nominal for this exploratory analysis. Results Of 154 enrolled participants, 40 (26%) initiated LXB treatment on a once-nightly regimen. In the efficacy population (n=115), 27 participants were on a once-nightly regimen during SDP (48.1% of whom initiated treatment once nightly during OLTTOP) and 88 participants were on a twice-nightly regimen during SDP (86.4% of whom initiated treatment twice nightly during OLTTOP). During SDP, median (min, max) LXB total dose was 4.5 (2.5, 6) g/night (once-nightly group) and 7.5 (4.5, 9) g/night (twice-nightly group). ESS scores worsened in participants randomized to placebo vs those continuing LXB in the once-nightly group (n=11 and n=15, respectively; LS mean difference [95% CI]: −4.93 [−7.41, −2.46]; P=0.0004) and twice-nightly group (n=47 and n=41, respectively; LS mean difference [95% CI]: −7.44 [−9.15, −5.72]; P&lt;0.0001). Worsening was also observed in PGIc (once-nightly: 81.8% [placebo] vs 26.7% [LXB]; P=0.0077; twice-nightly: 89.4% [placebo] vs 19.5% [LXB]; P&lt;0.0001) and IHSS score (estimated median difference [95% CI], once-nightly: −9.00 [−16.0, −3.0]; P=0.0028; twice-nightly: −12.00 [−15.0, −8.0]; P&lt;0.0001). Common adverse events included nausea (21.4%), headache (16.2%), anxiety (14.9%), dizziness (11.7%), insomnia (11.7%), and vomiting (10.4%). Conclusion The efficacy and safety of LXB in IH were demonstrated for both once-nightly and twice-nightly regimens. The majority of participants initiated and remained on a twice-nightly regimen. Support (if any) Jazz Pharmaceuticals

scholarly journals Duration of treatment effect using incobotulinumtoxinA for upper-limb spasticity: A post hoc analysis

Toxicon ◽  
2021 ◽  
Vol 190 ◽  
pp. S37
Author(s):  
Petr Kaňovský ◽  
Elie P. Elovic ◽  
Angelika Hanschmann ◽  
Irena Pulte ◽  
Michael Althaus ◽  
...  
Keyword(s):  
Upper Limb ◽  
Treatment Effect ◽  
Duration Of Treatment ◽  
Post Hoc Analysis ◽  
Limb Spasticity ◽  
Post Hoc

scholarly journals Efficacy and Safety of Teriflunomide in Multiple Sclerosis across Age Groups: Analysis from Pooled Pivotal and Real-world Studies

2021 ◽  
Vol 13 ◽  
pp. 117957352110287
Author(s):  
Jiwon Oh ◽  
Sandra Vukusic ◽  
Klaus Tiel-Wilck ◽  
Jihad Said Inshasi ◽  
David Rog ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Real World ◽  
Age Groups ◽  
Phase Iii ◽  
Efficacy And Safety ◽  
Additional Group ◽  
Disability Status ◽  
Scale Scores ◽  
Post Hoc ◽  
Phase Ii And Iii

Background: Evidence suggests that efficacy and safety of disease-modifying treatments for multiple sclerosis may differ with age. We evaluate efficacy and safety of teriflunomide across age subgroups of patients from pooled clinical trials and real-world studies. Methods: Post hoc analyses of patients who received teriflunomide 14 mg in the pooled phase II and III TEMSO, TOWER, TENERE, and TOPIC core and extension studies (n = 1978), and the real-world Teri-PRO (n = 928) and TAURUS-MS I (n = 1126) studies were conducted. Data were stratified by age at study entry: ⩽25, >25 to ⩽35, >35 to ⩽45, and >45 years. In Teri-PRO and TAURUS-MS I, an additional group, >55 years, was assessed. Results: In the pooled core studies, teriflunomide reduced annualized relapse rate (ARR) versus placebo across all ages. Unadjusted ARRs remained low across age groups in pooled extensions (0.18-0.30), Teri-PRO (0.10-0.35), and TAURUS-MS I (0.14-0.35). Baseline Expanded Disability Status Scale scores were higher with age, but stable through core and extension studies (mean increases over 7 years: ⩽25 years, +0.59; >25 to ⩽35 years, +0.46; >35 to ⩽45 years, +0.35; >45 years, +0.81). Across age groups, adverse event (AE) incidences were 78.4% to 90.7% in pooled core and extension studies and Teri-PRO, and 29.2% to 37.7% in TAURUS-MS I; serious AE incidences were ⩽21.3% in all studies. In pooled phase III and Teri-PRO studies, lymphocyte count decreases over 1 year after initiating teriflunomide, and proportions of patients developing lymphopenia, were small across age groups. Conclusions: Teriflunomide efficacy was demonstrated regardless of age. Safety was generally consistent across age groups.

Exploring the Role of Occupational Therapy With Mothers Who Breastfeed

2021 ◽  
Vol 75 (5) ◽  
Author(s):  
Lauren Sponseller ◽  
Fern Silverman ◽  
Pamela Roberts
Keyword(s):  
Occupational Therapy ◽  
Goal Attainment ◽  
Intervention Group ◽  
New Mothers ◽  
Lactation Consultant ◽  
Personal Wellness ◽  
Occupational Profile ◽  
Before And After ◽  
Goal Attainment Scale ◽  
Occupational Therapy Practitioners

Importance: Occupational therapy practitioners can play a pivotal role in supporting breastfeeding mothers as they transition to and form new routines for this occupation. Objective: To explore whether occupational therapy programming can assist breastfeeding mothers in reaching their personal occupation-based wellness goals. Design: Mixed-methods design that involved development of an occupational profile and a goal attainment scale (GAS). After the intervention, participants rescored their GAS goals and completed a semistructured exit interview. Setting: Nonprofit lactation center located in the suburbs of a large mid-Atlantic U.S. city. Participants: Women recruited through convenience sampling who had been breastfeeding an infant for &lt;6 mo, who were not currently weaning, and who had met with a lactation consultant at least once since giving birth were eligible (N = 17). Intervention: Group occupational therapy that consisted of 10 weekly 1-hr sessions. Topics were based on occupational profiles, GAS scores, and lactation consultant input. Outcomes and Measures: Each participant created and scored three goals using the GAS before and after the intervention. Results: Data from 14 of the 17 participants were analyzed. The average postintervention GAS score was 56.50 (M = 50), indicating that most personal wellness goals were reached. Thematic analysis revealed that occupational therapy programming helped mothers persevere with breastfeeding, feel more confident as new parents, and value both themselves and their baby. Conclusions and Relevance: There is an increasing role for occupational therapy practitioners in helping new mothers reach their personal wellness goals in ways that support their ability to continue breastfeeding. What This Article Adds: Maternal wellness and breastfeeding represent an emerging area of practice in which occupational therapy practitioners can provide new mothers with physical, social, and psychological supports that help them maintain self-efficacy related to breastfeeding and other meaningful occupations. This study provides foundational evidence in support of this collaboration.

Effect of adding upper limb rehabilitation to botulinum toxin-A on upper limb activity after stroke: Protocol for the InTENSE trial

2018 ◽  
Vol 13 (6) ◽  
pp. 648-653 ◽  
Author(s):  
Natasha A Lannin ◽  
Louise Ada ◽  
Coralie English ◽  
Julie Ratcliffe ◽  
Maria Crotty
Keyword(s):  
Botulinum Toxin ◽  
Upper Limb ◽  
Goal Attainment ◽  
Botulinum Toxin A ◽  
Phase Iii ◽  
Evidence Based ◽  
Toxin A ◽  
Movement Training ◽  
Severe Spasticity ◽  
To Receive

Rationale Although clinical practice guidelines recommend that management of moderate to severe spasticity include the use of botulinum toxin-A in conjunction with therapy, there is currently no evidence to support the addition of therapy. Aims To determine the effect and cost-benefit of adding evidence-based movement training to botulinum toxin-A. Sample size estimate A total of 136 participants will be recruited in order to be able to detect a between-group difference of seven points on the Goal Attainment Scale T-score with 80% power at a two-tailed significance level of 0.05. Methods and design The InTENSE trial is a national, multicenter, Phase III randomized trial with concealed allocation, blinded assessment and intention-to-treat analysis. Stroke survivors who are scheduled to receive botulinum toxin-A in any muscle(s) that cross the wrist because of moderate to severe spasticity after a stroke greater than three months ago, who have completed formal rehabilitation and have no significant cognitive impairment will be randomly allocated to receive botulinum toxin-A plus evidence-based movement training or botulinum toxin-A alone. Study outcomes The primary outcomes are goal attainment (Goal Attainment Scaling) and upper limb activity (Box and Block Test) at three months (end of intervention) and at 12 months (beyond the intervention). Secondary outcomes are spasticity, range of motion, strength, pain, burden of care and health-related quality of life. Direct costs, personal costs and health system costs will be collected at 12 months. Discussion The results of the InTENSE trial are anticipated to directly influence intervention for moderate to severe spasticity after stroke. Trial Registration ANZCTR12615000616572.

Poster 373-C Integrated Upper Limb Spasticity Management Including Botulinum Toxin A (BoNT-A) on Patient-Centered Goal Attainment: Methodology for ULIS-III and Initial Goal-Setting Data

PM&R ◽  
2016 ◽  
Vol 8 (9) ◽  
pp. S331
Author(s):  
Lynne Turner-Stokes ◽  
Jovita Balcaitiene ◽  
Stephen Ashford ◽  
Jorge Jacinto ◽  
Pascal Maisonobe ◽  
...  
Keyword(s):  
Botulinum Toxin ◽  
Goal Setting ◽  
Upper Limb ◽  
Goal Attainment ◽  
Botulinum Toxin A ◽  
Patient Centered ◽  
Toxin A ◽  
Limb Spasticity
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