scholarly journals Cyclosporine A in Ullrich Congenital Muscular Dystrophy: Long-Term Results

2011 ◽  
Vol 2011 ◽  
pp. 1-10 ◽  
Author(s):  
Luciano Merlini ◽  
Patrizia Sabatelli ◽  
Annarita Armaroli ◽  
Saverio Gnudi ◽  
Alessia Angelin ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Cyclosporine A ◽  
Congenital Muscular Dystrophy ◽  
Respiratory Muscles ◽  
Primary Outcome Measure ◽  
Term Treatment ◽  
Long Term Results ◽  
Long Term Treatment ◽  
Ullrich Congenital Muscular Dystrophy

Six individuals with Ullrich congenital muscular dystrophy (UCMD) and mutations in the genes-encoding collagen VI, aging 5–9, received 3–5 mg/kg of cyclosporine A (CsA) daily for 1 to 3.2 years. The primary outcome measure was the muscle strength evaluated with a myometer and expressed as megalimbs. The megalimbs score showed significant improvement (P=0.01) in 5 of the 6 patients. Motor function did not change. Respiratory function deteriorated in all. CsA treatment corrected mitochondrial dysfunction, increased muscle regeneration, and decreased the number of apoptotic nuclei. Results from this study demonstrate that long-term treatment with CsA ameliorates performance in the limbs, but not in the respiratory muscles of UCMD patients, and that it is well tolerated. These results suggest considering a trial of CsA or nonimmunosuppressive cyclosporins, that retains the PTP-desensitizing properties of CsA, as early as possible in UCMD patients when diaphragm is less compromised.

Long-Term Results of Radiofrequency Volumetric Tissue Reduction of the Palate for Snoring

2003 ◽  
Vol 112 (3) ◽  
pp. 276-279 ◽  
Author(s):  
Bassem Said ◽  
Marshall Strome
Keyword(s):  
Patient Satisfaction ◽  
Medical Center ◽  
Academic Medical Center ◽  
Term Treatment ◽  
Response To Treatment ◽  
Long Term Results ◽  
Long Term Treatment ◽  
Radiofrequency Volumetric Tissue Reduction ◽  
Tissue Reduction

To assess the long-term efficacy and morbidity of radiofrequency volumetric tissue reduction (RFVTR) of the soft palate for snoring, we examined the medical records of 39 patients who received this treatment at an academic medical center. Telephone interviews were conducted with the patients to evaluate the long-term subjective efficacy and sequelae. The average follow-up was 14 months (range, 3 to 26 months). Twenty-eight patients (72%) responded to treatment, defined as a 4-point decrease on a 10-point scale. The self-reported snoring score decreased an average of 52% (8.8 ± 1.9 to 4.2 ± 2.9). Sixty-seven percent of the patients were satisfied. The response to treatment did not always correlate with patient satisfaction. The snoring relapse rate was 11% among responders. No significant differences were identified between responders and nonresponders. No significant complications or long-term sequelae were observed. We conclude that RFVTR of the palate is a relatively safe and effective long-term treatment for snoring. Defining realistic pretreatment expectations is important in maximizing patient satisfaction.

Atezolizumab (atezo) in patients with metastatic urothelial carcinoma (mUC): A 2-year clinical update from a phase Ia study.

2017 ◽  
Vol 35 (6_suppl) ◽  
pp. 290-290 ◽  
Author(s):  
Daniel Peter Petrylak ◽  
Thomas Powles ◽  
Joaquim Bellmunt ◽  
Fadi S. Braiteh ◽  
Yohann Loriot ◽  
...  
Keyword(s):  
Objective Response ◽  
The United States ◽  
Term Treatment ◽  
Long Term Results ◽  
Platinum Based Chemotherapy ◽  
Long Term Treatment ◽  
Previously Treated ◽  
Ecog Ps

290 Background: Atezo (anti–PD-L1) has demonstrated safety and efficacy in a broad range of cancers and is approved in the United States for mUC previously treated with platinum-based chemotherapy. Here we report long-term results in mUC from Phase Ia study NCT01375842 (PCD4989g). Methods: Previously treated mUC patients received atezo 15 mg/kg or 1200 mg IV q3w. Enrollment in this Phase Ia expansion cohort initially required PD-L1–selected status and later opened to patients regardless of PD-L1 expression on tumor-infiltrating immune cells. The primary endpoint was safety/tolerability. Secondary endpoints included investigator-assessed RECIST v1.1 ORR (confirmed), DOR and OS. Results: 95 patients were safety evaluable (Table). Median age was 66 years, 76% were male and 80% had primary bladder tumors. 61% had ECOG PS 1. 52% received ≥ 3 prior systemic therapies for mUC (70% platinum). Median treatment duration was 3 months (range: 0-32 months); 24% were treated for ≥ 1 year. Treatment-related AEs occurred in 66% (all Grade) and 8% (Grade 3-4) of patients. No treatment-related deaths were reported. In 94 objective response–evaluable patients (follow-up ≥ 12 weeks), the ORR was 27% (95% CI: 18, 37%), and the CR rate was 10%; the SD rate was 19%. mDOR was 22.1 months (95% CI: 12.1, NE months) in all patients; 56% of responses (7/9 CRs and 7/16 PRs) were ongoing at the December 15, 2015 data cutoff. With a 24-month median follow-up duration (range: 1+ to 32 months), the 1-year OS rate was 47% (95% CI: 36, 58%), and the 2-year rate was 29% (19, 40%); mOS is in the Table. Updated clinical data with further follow-up and analyses by PD-L1 status will be presented. Conclusions: Long-term treatment with atezo was well tolerated, without new safety signals in heavily pre-treated mUC patients. The durability of responses, including CRs, along with extended OS, confirm atezo as a new standard for previously treated mUC patients. Clinical trial information: NCT01375842. [Table: see text]

LONG TERM TREATMENT OF IgA NEPHROPATHY WITH CYCLOSPORINE A

Renal Failure ◽  
2000 ◽  
Vol 22 (1) ◽  
pp. 55-62 ◽  
Author(s):  
Vera Chábová ◽  
Vladimir Tesar ◽  
Jiri Zabka ◽  
Ivan Rychlik ◽  
Miroslav Merta ◽  
...  
Keyword(s):  
Iga Nephropathy ◽  
Cyclosporine A ◽  
Term Treatment ◽  
Long Term Treatment

scholarly journals Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls

2021 ◽  
pp. 1-11
Author(s):  
Jerry R. Mendell ◽  
Navid Khan ◽  
Nanshi Sha ◽  
Helen Eliopoulos ◽  
Craig M. McDonald ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Mutations ◽  
Exon Skipping ◽  
Term Treatment ◽  
Long Term Treatment ◽  
The Difference ◽  
Dmd Gene ◽  
6 Minute Walk Test

Background: Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal, degenerative neuromuscular disease caused by DMD gene mutations. A relationship between exon skipping and dystrophin production in exon 51-amenable patients treated with eteplirsen (EXONDYS 51 ®) is established. Once-weekly eteplirsen significantly increased dystrophin, with slower decline in ambulatory function compared to baseline. Long-term treatment with eteplirsen leads to accumulation of dystrophin over time and observed functional benefits in patients with DMD. Objective: Compare long-term ambulatory function in eteplirsen-treated patients versus controls. Methods: Study 201/202 included 12 eteplirsen-treated patients assessed twice/year for ambulatory function over 4 years. Ambulatory evaluations (6-minute walk test [6MWT], loss of ambulation, and North Star Ambulatory Assessment [NSAA]) were compared with matched controls from Italian Telethon and Leuven registries. Results: At Years 3 and 4, eteplirsen-treated patients demonstrated markedly greater mean 6MWT than controls (difference in change from baseline of 132 m [95%CI (29, 235), p = 0.015] at Year 3 and 159 m [95%CI (66, 253), p = 0.002] at Year 4). At Year 4, a significantly greater proportion of eteplirsen-treated patients were still ambulant versus controls (10/12 vs 3/11; p = 0.020). At Year 3, eteplirsen-treated patients had milder NSAA decline than controls (difference in change from baseline of 2.6, 95%CI [-6, 11]), however, the difference was not statistically significant; Year 4 control NSAA data were not available. Conclusion: In this retrospective matched control study, eteplirsen treatment resulted in attenuation of ambulatory decline over a 4-year observation period, supporting long-term benefit in patients with DMD.

scholarly journals Cyclosporine A treatment for Ullrich congenital muscular dystrophy: a cellular study of mitochondrial dysfunction and its rescue

Brain ◽  
2009 ◽  
Vol 132 (1) ◽  
pp. 147-155 ◽  
Author(s):  
D. Hicks ◽  
A. K. Lampe ◽  
S. H. Laval ◽  
V. Allamand ◽  
C. Jimenez-Mallebrera ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Mitochondrial Dysfunction ◽  
Cyclosporine A ◽  
Congenital Muscular Dystrophy ◽  
Ullrich Congenital Muscular Dystrophy

Peripheral Neurostimulation for Treatment of Intractable Occipital Neuralgia

Neurosurgery ◽  
2006 ◽  
Vol 58 (1) ◽  
pp. 112-119 ◽  
Author(s):  
Konstantin V. Slavin ◽  
Hrachya Nersesyan ◽  
Christian Wess
Keyword(s):  
Pain Syndrome ◽  
Good Alternative ◽  
Term Treatment ◽  
Long Term Results ◽  
Intractable Pain ◽  
Occipital Neuralgia ◽  
Beneficial Effects ◽  
Long Term Treatment ◽  
Patient Reported

Abstract OBJECTIVE: Medically intractable pain caused by occipital neuralgia (ON) can be very difficult to control with traditional pain management. Peripheral nerve stimulation (PNS) may serve as a good alternative to destructive surgical manipulations used currently for the treatment of severe ON. METHODS: We analyzed records of 14 consecutive patients (9 women and 5 men; mean age, 43.3 yr) with intractable ON treated with PNS during the period from April 2002 to November 2004. Five patients had unilateral and nine had bilateral PNS electrodes inserted for trial, which was considered successful if patient reported at least 50% decrease of pain on the visual analogue scale. Ten patients proceeded with system internalization, and their long-term results were analyzed. RESULTS: At the time of the last follow-up examination (5–32 mo, mean 22 mo), seven patients (70%) with implanted PNS systems continue to experience beneficial effects of stimulation, including adequate pain control, continuous employment, and decrease in oral pain medications intake. Two patients had their systems explanted because of loss of stimulation effect or significant improvement of pain, and one patient had part of his hardware removed because of infection. CONCLUSION: Overall, the beneficial effect from chronic stimulation in our series persisted in more than half of the patients for whom procedure was considered and in 80% of those who significantly improved during the trial and proceeded with internalization. Thus, chronic PNS may be a safe and relatively effective method for long-term treatment of chronic pain syndrome in patients with medically intractable ON.

scholarly journals LONG-TERM RESULTS OF SURGICAL TREATMENT OF DIVERTICULAR DISEASE OF COLON

2016 ◽  
Vol 175 (2) ◽  
pp. 101-104
Author(s):  
V. I. Pomazkin ◽  
V. V. Khodakov
Keyword(s):  
Diverticular Disease ◽  
Emotional Disorders ◽  
Elective Surgery ◽  
Term Treatment ◽  
Long Term Results ◽  
Number Of Patients ◽  
Sf 36 ◽  
Long Term Treatment

The work evaluated the long-term treatment results of 103 patients with complicated diverticular disease, who underwent the elective surgery in the volume of left hemicolectomy. The first group included 53 patients with recurrent diverticula. The second group consisted of 50 patients, who underwent the operation on diverticula of sigmoid colon with formation of sigmostoma on the first stage. The number of patients with functional gastro-intestinal and psyco-emotional disorders was more significant in the first group compared with the second group and it was associated with presence of these disorders before the operation. There was noted the reliable decrease of quality of life rates according to questionnaire scales MOS-SF 36 in the first group. The average indices of scales had some matching to the rates of health people in the patients of the second group. The authors came to conclusion, that indications for elective surgery should be determined very carefully in case of recurrent diverticula.

scholarly journals Real-World, Long-Term Quality of Life Following Therapeutic OnabotulinumtoxinA Treatment

2016 ◽  
Vol 43 (5) ◽  
pp. 687-696 ◽  
Author(s):  
Mandar Jog ◽  
Theodore Wein ◽  
Meetu Bhogal ◽  
Sonja Dhani ◽  
Robert Miller ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Short Form ◽  
Health Utility ◽  
Primary Outcome Measure ◽  
Term Treatment ◽  
Subsequent Treatment ◽  
Long Term Treatment ◽  
Over Time

AbstractBackground:OnabotulinumtoxinA is an efficacious treatment option for patients with various conditions. Although studies have reported on the efficacy of onabotulinumtoxinA, quality of life (QoL) data are limited. This study evaluated QoL in patients treated with onabotulinumtoxinA across various therapeutic indications.Methods:MDs on BOTOX Utility (MOBILITY) was a prospective, multicenter, observational Canadian study in patients initiating (naïve) or receiving ongoing (maintenance) onabotulinumtoxinA treatment. Health utility was the primary outcome measure and was obtained from the Short Form-12 Health Survey using the Short Form-6D at baseline, week 4 posttreatment, and up to five subsequent treatment visits. The safety cohort included patients who received ≥1 onabotulinumtoxinA treatment.Results:The efficacy cohort included 1062 patients; the majority were Caucasian, female, and on maintenance onabotulinumtoxinA treatment. Adult focal spasticity (n=398), blepharospasm (n=81), cerebral palsy (n=22), cervical dystonia (n=234), hemifacial spasm (n=116), and hyperhidrosis (n=211) patients were included. Baseline health utility was generally higher in maintenance versus naïve patients; however, naïve patients showed the greatest improvements over time. Health utility was generally maintained or trended toward improvement across all cohorts, including maintenance patients who had been treated for up to 22 years before study entry. Eighteen of 1222 patients (2%) in the safety cohort reported 28 treatment-related adverse events; eight were serious in four patients.Conclusion:MOBILITY is the largest prospective study to date to provide QoL data over a variety of therapeutic indications following treatment with onabotulinumtoxinA. Although the QoL burden varies by disease, data suggest that long-term treatment may help improve or maintain QoL over time.

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