scholarly journals Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Eva Revilla-López ◽  
◽  
Cristina Berastegui ◽  
Alejandra Méndez ◽  
Berta Sáez-Giménez ◽  
...  
Keyword(s):  
Term Treatment ◽  
Term Effect ◽  
Long Term Results ◽  
Published Data ◽  
Referral Centre ◽  
Partial Responder ◽  
Long Term Effect ◽  
Long Term Treatment ◽  
Predicted Values

AbstractThere are few published data on long-term treatment with sirolimus in lymphangioleiomyomatosis (LAM). The objective of this study was to describe the long-term effect of sirolimus in a series of LAM patients followed up in a referral centre, focusing on pulmonary function. We retrospectively reviewed a series of 48 patients with LAM diagnosed, followed up and treated with sirolimus in a single centre. Response to sirolimus was evaluated at 1 and 5 years. A negative sirolimus response was defined as an FEV1 decline greater than − 75 ml/year. A mixed-effects model was used to estimate the longitudinal changes in FEV1 (average slope), both as absolute (ml/year) and as predicted values (%predicted/year). From a total of 48 patients, 9 patients underwent lung transplantation and 4 died during the study. Mean (95% CI) FEV1 slope over 5 years was − 0.14 (− 26.13 to 25.85) ml/year in the whole LAM group, 42.55 (14.87 to 70.22) ml/year in the responder group, − 54.00 (− 71.60 to − 36.39) ml/year in the partial responder group and − 84.19 (− 113.5 to − 54.0) ml/year in the non-responder group. After 5 years of sirolimus treatment 59% had a positive response, 30% had a partial response and 11% had a negative response. Our study found that sirolimus treatment had a positive long-term effect on most LAM patients.

Relationship Among Serum and Urine Neopterin, Fibrinogen, and Other Serum Analytes in Atherosclerotic Patients and the Long-term Effect of H.E.L.P. Therapy

Pteridines ◽  
1999 ◽  
Vol 10 (4) ◽  
pp. 190-196
Author(s):  
Karoline Vrecko ◽  
Manfred Walz ◽  
Erwin Tafeit ◽  
G. Reibnegger
Keyword(s):  
Term Treatment ◽  
Term Effect ◽  
Long Term Effect ◽  
Long Term Treatment ◽  
Cellular Immune System ◽  
Before And After ◽  
Immune Activation Marker ◽  
Cellular Immune ◽  
Serum And Urine

Summary In 30 atherosclerotic patients different serum analytes and neopterin concentrations in serum and urine were determined before and after Heparin-Induced Extracorporeal LDL and Fibrinogen Precipitation therapy. Neopterin concentrations in serum of all 30 patients were increased (mean=9.18 nmol/l, SD=3.23) compared to age matched healthy people (mean=5.34 nmol/l, SD=2.70) and were significantly lowered after H.E.L.P therapy (mean=S.22 nmol/l; SD=3.22, p=0.002, t-test) . These results suggest the involvement of the cellular immune system in the occurrence of atherosclerosis. Further neopterin, the macrophagederived immune activation marker, is significantly correlated with fibrinogen (linear correlation coefficient r=0.36, p=0.05). Long-term neopterin concentrations in serum of 4 patients (three with coronary heart disease and one with Papilla edema), from the first up to the 9th, respectively 12th therapy decreased, in contrast to fibrinogen which stayed stable or even increased during long-term treatment.

scholarly journals The Long-Term Effect of Medically Enhancing Melanin Intrinsic Bioenergetics Capacity in Prematurity

2020 ◽  
Vol 21 (7) ◽  
pp. 525-530
Author(s):  
Arturo S. Herrera ◽  
Paola E. Solís Arias ◽  
María del C.A. Esparza ◽  
Luis F.T. Bernal ◽  
Andrey D. Bondarev ◽  
...  
Keyword(s):  
Molecular Hydrogen ◽  
Term Treatment ◽  
Term Effect ◽  
Metabolic Energy ◽  
Potential Implication ◽  
Carbon Chains ◽  
Long Term Effect ◽  
Long Term Treatment ◽  
Promising Application

Background: The ability of the human body to produce metabolic energy from light modifies fundamental concepts of biochemistry. Objective: This review discusses the relationships between the long-accepted concept is that glucose has a unique dual role as an energy source and as the main source of carbon chains that are precursors of all organic matter. The capability of melanin to produce energy challenges this premise. Methods: The prevalent biochemical concept, therefore, needs to be adjusted to incorporate a newly discovered state of Nature based on melanin’s ability to dissociate water to produce energy and to reform water from molecular hydrogen and oxygen. Results and Discussion: Our findings regarding the potential implication of QIAPI-1 as a melanin precursor that has bioenergetics capabilities. Conclusion: Specifically, we reported its promising application as a means for treating retinopathy of prematurity (ROP). The instant report focuses on the long-term treatment medical effects of melanin in treating ROP.

The Long-Term Effect of Medically Enhancing Melanin Intrinsic Bioenergetics Capacity in Prematurity

2019 ◽  
Vol 19 ◽  
Author(s):  
Arturo Solís Herrera ◽  
Paola E. Solís Arias ◽  
María del Carmen Arias Esparza ◽  
Luis Fernando Torres Bernal ◽  
Andrey D. Bondarev ◽  
...  
Keyword(s):  
Term Treatment ◽  
Term Effect ◽  
Metabolic Energy ◽  
Water Molecules ◽  
Potential Implication ◽  
Carbon Chains ◽  
Long Term Effect ◽  
Long Term Treatment ◽  
Promising Application

We have demonstrated the intrinsic capability of melanin to transform light into chemical energy. Melanin achieves this by dissociating water molecules, similar to the action of chlorophyll. This unforeseen ability of the human body to produce metabolic energy from light modifies fundamental concepts of biochemistry. The long-accepted concept is that glucose has a unique dual role as an energy source and as the main source of carbon chains that are precursors of all organic matter. The capability of melanin to produce energy challenges this premise. The prevalent biochemical concept, therefore, needs to be adjusted to incorporate a newly discovered state of Nature based on melanin’s ability to dissociate water to produce energy and to re-form water from molecular hydrogen and oxygen. Recently we reported our findings regarding the potential implication of QIAPI-1 as a melanin precursor that has bioenergetics capabilities. Specifically, we reported its promising application as a means for treating retinopathy of prematurity (ROP). This review report focuses on the long-term treatment medical effects of melanin in treating ROP.

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]

scholarly journals Systemic and presystemic conversion of carbamazepine to carbamazepine-10,11-epoxide during long term treatment

2006 ◽  
Vol 12 (1) ◽  
pp. 13-16 ◽  
Author(s):  
Pietro Fagiolino ◽  
Marta Vázquez ◽  
Ivette Olano ◽  
Aurora Delfino
Keyword(s):  
Plasma Concentrations ◽  
Chronic Administration ◽  
Hplc Method ◽  
Term Treatment ◽  
Published Data ◽  
Long Term Treatment ◽  
Important Pathway ◽  
Kinetics Of ◽  
Dose Dependent

INTRODUCTION: Carbamazepine (CBZ) undergoes biotransformation, being CYP3A4 the major cytocrome P450 (CYP) enzyme catalyzing the carbamazepine-10,11-epoxide (EPOX) formation, which is quantitatively the most important pathway in CBZ metabolism. There is evidence of dose-dependent elimination of this drug due to its autoinduction capacity. Moreover, published data showed an incomplete bioavailability of CBZ since its absorption increases when grapefruit juice was administered. Both CYP3A4 and MRP2 (located in the enterocyte) are autoinduced during long term use of CBZ. As the other enzymes involved in CBZ metabolism are negligible in the gut, presystemic biotransformation through CYP3A4 could be responsible for the bioavailability of the drug as well as EPOX formation. OBJECTIVE: The purpose of our study was to assess the importance of presystemic formation of EPOX during the autoinduction of CBZ versus the daily administered dose. PATIENTS AND METHODS: 40 adults (average age: 28 years) and 29 children (average age: 9 years) receiving CBZ as monotherapy were included in the study. CBZ and EPOX plasma concentrations were analyzed by a previous validated HPLC method. RESULTS AND CONCLUSION: The results obtained confirmed the metabolic induction after chronic administration and provided new elements to suggest a strong contribution of dose-dependent bioavailability in the non linear kinetics of CBZ.

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.

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 treatment with subcutaneous immunoglobulin in multifocal motor neuropathy

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Luca Gentile ◽  
Massimo Russo ◽  
Carmelo Rodolico ◽  
Ilenia Arimatea ◽  
Giuseppe Vita ◽  
...  
Keyword(s):  
Life Quality ◽  
Ivig Therapy ◽  
Multifocal Motor Neuropathy ◽  
Term Treatment ◽  
Published Data ◽  
Motor Neuropathy ◽  
Subcutaneous Immunoglobulin ◽  
Long Term Treatment

AbstractMultifocal motor neuropathy (MMN) is a rare disease with a prevalence of less than 1 per 100,000 people. Intravenous immunoglobulin (IVIG) therapy, performed for a long-term period, has been demonstrated able to improve the clinical picture of MMN patients, ameliorating motor symptoms and/or preventing disease progression. Treatment with subcutaneous immunoglobulin (SCIg) has been shown to be as effective as IVIG. However, previously published data showed that follow-up of MMN patients in treatment with SCIg lasted no more than 56 months. We report herein the results of a long-term SCIg treatment follow up (up to 96 months) in a group of 8 MMN patients (6 M; 2F), previously stabilized with IVIG therapy. Clinical follow-up included the administration of Medical Research Council (MRC) sum-score, the Overall Neuropathy Limitation Scale (ONLS) and the Life Quality Index questionnaire (LQI) at baseline and then every 6 months. Once converted to SCIg, patients’ responsiveness was quite good. Strength and motor functions remained stable or even improved during this long-term follow-up with benefits on walking capability, resistance to physical efforts and ability in hand fine movements.

scholarly journals Long-Term Effect of Aromatase Inhibition in Aromatase Excess Syndrome

2021 ◽  
Vol 5 (Supplement_1) ◽  
pp. A679-A679
Author(s):  
Gerhard Binder ◽  
Akie Nakamura ◽  
Roland Schweizer ◽  
Tsutomu Ogata ◽  
Maki Fukami ◽  
...  
Keyword(s):  
Tandem Duplication ◽  
Low Dose ◽  
Adult Height ◽  
Bone Age ◽  
Sporadic Case ◽  
Term Treatment ◽  
Sibling Pairs ◽  
Long Term Effect ◽  
Long Term Treatment

Abstract Aromatase excess syndrome (AEXS) is a very rare disorder characterized by prepubertal gynecomastia, bone age acceleration and early growth arrest. Heterozygote submicroscopic rearrangements within the promotor of CYP19A1 result in overexpression of aromatase and enhanced aromatization of androgens. Long-term treatment effects of aromatase inhibitors are unknown. Retrospectively we collected data from file records of 7 boys (three sibling pairs and one sporadic case) with AEXS. Genetic analysis revealed upstream of CYP19A1 a 165,901 bp deletion in 4 German cousins, a 198,662 bp deletion in 2 Japanese brothers and a 387,622 bp tandem duplication in a Japanese boy. All boys developed prepubertal gynecomastia, at 9.0 yr of age (median; range: 7.0 - 11.0). Height was +1.20 SDS (-0.24 - +1.98); predicted adult height was -1.29 (-3.29 - +1.09 SDS). Four boys were treated with anastrozole 1.0 mg daily, while three reached adult height untreated. Treatment with anastrozole was stopped after 5.6 yr (4.0 - 6.8). Three treated boys exceeded height prognosis by 2.4, 6.9 and 8.1 cm; while one untreated fell below prognosis by 8.6 cm. One treated with a low dose and two untreated reached their prognosis. Adult heights were -0.91 SDS with anastrozole (-2.86 - -0.29) and -0.15 SDS without (-2.31 - -0.03). Distance to target height was -0.22 SDS with anastrozole (-1.72 - +0.52) and +0.54 SDS without treatment (+0.23 - +1.30). Spontaneous growth in AEXS varied, even in the same family. Our data suggest that early started, long-term inhibition by aromatase inhibitor anastrozole (1 mg daily) promotes adult height in boys with AEXS.

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