Search results for " Replacement"
showing 10 items of 539 documents
Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial
2015
Objective: To report and discuss the multi-domain impact of elosulfase alfa, with focus on tertiary and composite endpoints, in the 24-week, randomized, double-blind, placebo-controlled phase 3 trial in patients with Morquio A syndrome (mucopolysaccharidosis IVA). Methods: Patients with Morquio A syndrome >= 5 aged years were randomized 1:1:1 to elosulfase alfa 2.0 mg/kg/week (qw; N = 58), elosulfase alfa 2.0 mg/kg/every other week (qow; N = 59), or placebo (N = 59) for 24 weeks. Primary and secondary efficacy measures were 6-minute walk test (6MWT; primary), 3-minute stair climb test (3-MSCT) and urinary keratan sulfate (KS). Safety was also evaluated. Tertiary efficacy measures included r…
Effect of oestrogen/gestagen replacement therapy on liver enzymes in patients with Ullrich-Turner syndrome.
1995
The absence of breast development and the prevention of osteoporosis in Ullrich-Turner syndrome (UTS) require oestrogen/gestagen substitution therapy. In 8 out of 35 (23%) patients with UTS treated with conjugated equine oestrogens and cyclically with norethisterone acetate, the serum liver enzymes increased to conspicuous levels (AST 35; 20-73 U/l, ALT 92; 37-141 U/l, GGT 77; 25-227 U/l, [median; min-max]). These findings were compared with those in 41 tall girls who received a six-fold larger dose of conjugated equine oestrogens for the reduction of final height. None of these 41 girls showed abnormal serum liver enzyme levels. The conspicuous rise in serum liver enzyme levels occurred in…
The internal calcar septum and its contact with the virtual stem in THR: a computer tomographic evaluation.
2003
The internal calcar septum is a ridge of cortical bone protruding from the inner cortical wall of the proximal femur into the medullary canal. It extends from the lesser trochanter into the femoral neck and narrows the femoral cavity in its dorsal third. This region is essential for THR stability, but the degree of contact between the septum and standard THR implants has never been studied. We obtained CT scans of 50 arthrotic hip joints from patients requiring THR. Virtual stems (50 straight/wedge-shaped and 50 anatomic stems) were placed in CT images of the femora using a PC-based preoperative planning unit. The dimensions of the septum, degree and location of contact between the septum a…
Lipoprotein(a) and aortic valve stenosis: A casual or causal association?
2022
Abstract Aims This review aims to provide an update of available methods for imaging calcification activity and potential therapeutic options. Data Synthesis: Aortic valve calcification represents the most common heart valve condition requiring treatment among adults in Western societies. No medical therapies are proven to be effective in treating symptoms or reducing disease progression. Therefore, surgical or transcatheter aortic valve replacement remains the only available treatment option. Elevated circulating concentrations of lipoprotein(a) is strongly associated with degenerative aortic stenosis. This relationship was first observed in prospective observational studies, and the causa…
Enzyme replacement therapy in heterozygous females with Fabry disease: results of a phase IIIB study.
2003
Summary: Fabry disease is an X-linked glycosphingolipid storage disorder caused by a deficiency of α-galactosidase A. Affected patients experience debilitating neuropathic pain and have premature mortality due to renal failure, cardiovascular disease or cerebrovascular complications. The disease may be X-linked dominant, since most females heterozygous for Fabry disease are affected clinically. We evaluated the safety, efficacy and pharmacokinetics of agalsidase alfa (Replagal) administered intravenously to female patients with Fabry disease in an open-label, single-centre study. Fifteen severely affected patients received agalsidase alfa at 0.2 mg/kg every other week for up to 55 weeks. Ag…
Hypogonadism and Hormone Replacement Therapy on Bone Mass of Adult Women with Thalassemia Major
2002
We studied bone mass and metabolism in 30 adult women (age 28.5 +/- 1.3) with thalassemia major (TM) and evaluated whether prolonged hormone replacement therapy (HRT) was able to optimize bone accrual. TM patients had reduced bone mass, increased bone turnover and lower serum gonadotropin and estradiol levels compared with 10 normal women of similar age. A significant correlation was found between bone mass and sex hormone levels. Six TM patients with normal ovarian function had normal bone turnover markers and modestly low bone mass (lumbar spine -1.29 +/- 0.31; femoral neck -0.60+/-0.21; Z-score). The other 24 TM women were hypogonadic and had significantly lower bone mass for age (lumbar…
Analysis of serum and endometrial progesterone in determining endometrial receptivity.
2021
Abstract STUDY QUESTION Is there a relationship between serum and endometrial progesterone (P4) levels, including P4 and metabolites (oestrone, oestradiol and 17α-hydroxyprogesterone), and endometrial receptivity? SUMMARY ANSWER Serum P4 levels were not correlated with endometrial P4, nor associated with endometrial receptivity as determined by the ERA® test; however, endometrial P4 and 17α-hydroxyprogesterone levels were positively correlated and related to endometrial receptivity by ERA. WHAT IS KNOWN ALREADY Acquisition of endometrial receptivity is governed by P4, which induces secretory transformation. A close relationship between serum P4 and pregnancy outcome is reported for hormone …
Characteristics of urinary luteinizing hormone (LH) during the induction of LH surges of different magnitude in blood
1995
Urinary luteinizing hormone (LH) testing has been proposed as a reliable method for the prediction of ovulation but its accuracy has been challenged by some studies. To check how accurately the oscillations of urinary LH reflected the plasma changes, surges of LH of different magnitude and duration were artificially induced in plasma and the hormone was measured simultaneously in urine. Post-menopausal women (n = 16) were stimulated during 1 week with a combination of transdermal oestradiol (400 micrograms) and i.m. progesterone (25 mg on day 4, 50 mg on day 5) to obtain an LH discharge comparable with the pre-ovulatory LH peak. A short and moderate peak of LH was induced by the i.v. inject…
A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease
2009
Although Fabry disease is X linked and considered to affect primarily male hemizygotes, female heterozygotes may experience all the signs and symptoms of this metabolic disorder. This prospective, single-center, open-label, clinical trial was performed to evaluate the long-term response of female patients with Fabry disease to enzyme replacement therapy.Symptomatic women (average age = 47 years) enrolled in this 4-year study were treated with agalsidase alfa (Replagal, Shire HGT, Inc.) at a dose of 0.2 mg/kg, every other week for 4 years (N = 36). Clinical and biochemical assessments were conducted at 12-month intervals.The Mainz Severity Score Index, a measure of total disease burden, was …
The effect of hormonal status on the expression of estrogen and progesterone receptor in vaginal wall and periurethral tissue in urogynecological pat…
2009
Abstract Objective Our objective was to study the expression of estrogen receptor (ER) isoforms ER alpha (α) and ER beta (β) and of progesterone receptor (PR) in the vaginal wall and in periurethral tissue of women who underwent urogynecological surgical treatment with reference to estrogen status. Study design The study included 89 patients undergoing vaginal surgery for urogynecological conditions. Patients’ history and clinical data including estrogen status and body mass index (BMI) were evaluated. Biopsies from the vaginal wall and from periurethral tissue were obtained during surgery. The expression of ER α and β and of PR in vaginal wall and periurethral tissue was measured by RT-PCR…