A Post-Authorization Safety Surveillance Study to Report Clinical Experience with Purified Factor IX Concentrate in Pediatric Patients with Hemophilia B.

Introduction: Purified factor IX (FIX) concentrate (IMMUNINE®, Takeda Manufacturing Austria AG, Vienna, Austria) is indicated for the treatment and prophylaxis of bleeding episodes in patients with congenital hemophilia B. Data on the use of purified FIX concentrate in patients ≤6 years old with congenital hemophilia B are limited. Aim: Document real-world clinical experience with purified FIX concentrate in routine practice for pediatric patients with hemophilia B. Methods: This prospective post-authorization safety surveillance study enrolled patients ≤6 years old with moderate or severe hemophilia B (baseline FIX ≤5%) who were prescribed purified FIX concentrate, as determined by the treating physician. The planned observation period for each patient was either 12 months or ≥50 exposure days, whichever occurred first. The primary endpoints were the occurrence of treatment-related adverse events (AEs) and serious AEs (SAEs), and inhibitor development. Results: Thirteen male patients (mean ± standard deviation age, 3.80 ± 1.76 years) enrolled and received ≥1 treatment with purified FIX concentrate. Thirty-two AEs were reported in 6 patients; 4 were SAEs. No AEs were considered related to purified FIX concentrate. No patients developed inhibitory antibodies. Inhibitor testing was not conducted in 2 patients. Eighteen bleeding episodes were treated with purified FIX concentrate in 6 patients. Hemostatic efficacy was rated as either "excellent" or "good" in all patients with an available rating. Conclusion: No treatment-related AEs were reported, and purified FIX concentrate was shown to be effective in treating and preventing bleeding episodes in pediatric patients ≤6 years old with hemophilia B.

[Local estrogen therapy--clinical implications--2012 update]. / Lokalna terapia estrogenowa--implikacje kliniczne--2012 update.

With increasing longevity in Poland, women can now expect to live around 40% of their lives after menopause, and there is a growing desire for older women to preserve their vitality sexual function and quality of life. The most common urogenital symptoms associated with menopause are dryness, followed by irritation or itching, and discharge, with a substantial number of post-menopausal women also being affected by dysuria. These symptoms are the result of vaginal atrophy which is in turn caused by reduced transudation through the vaginal epithelium and reduced cervical gland secretions resulting from post-menopausal estrogen depletion. Vaginal atrophy generally occurs 4-5 years after the last menstrual period and progressively increases in prevalence in the subsequent years. Importantly vaginal atrophy is strongly associated with sexual dysfunction, and lower urinary tract symptoms, such as frequency urgency nocturia and dysuria, as well as incontinence and recurrent infection are reported more frequently in the presence of vaginal atrophy Those symptoms, apart from being bothersome for the patients also negatively impact their quality of life. Consequently before irreversible changes occur, early detection and treatment of vaginal atrophy should be implemented. Estrogen therapy is the most commonly prescribed treatment. Estrogens restore the cytology pH and vascularity of the vagina, resulting in symptom resolution for the majority of treated women. Because vaginal atrophy symptoms tend to occur later than vasomotor symptoms, many women do not necessarily require or wish to take systemic estrogen treatment if their symptoms are restricted to the urogenital tract. Vaginal estrogen products deliver estrogen locally to vaginal tissues with little or no systemic absorption and provide an effective alternative to systemic estrogen therapy for these women. Various vaginal estrogen preparations such as conjugated equine estrogens, estradiol and estriol vaginal creams, a sustained-release intra-vaginal estradiol ring and a low-dose estradiol and estriol tablets are useful therapeutic options in the treatment of this condition. Moreover; a low dose treatment with a minimised systemic absorption rate may be considered in women with a history of breast cancer and associated severe vulvovaginal atrophy. It should be mentioned that vaginal lubricants once applied on a regular basis may also be effective in alleviating the symptoms of vaginal atrophy and should be offered to women wishing to avoid the use of local vaginal estrogen preparations and in cases where local estrogen therapy is contraindicated. Vaginal dehydroepiandrosterone (DHEA), vaginal testosterone, and tissue selective estrogen complexes are new, emerging therapies; however more clinical studies are necessary to confirm their efficacy and safety in the treatment of postmenopausal vulvovaginal atrophy.

Women's voices in the menopause: results from an international survey on vaginal atrophy.

OBJECTIVES: To consider issues relating to vaginal atrophy via an international survey. METHODS: Using a structured questionnaire, interviews were performed on 4246 women aged 55-65 years living in Sweden, Finland, the United Kingdom, the United States and Canada. RESULTS: Overall, 98% of survey respondents were postmenopausal. Thirty-nine percent of the postmenopausal women had experienced vaginal atrophy, with the prevalence varying between countries, from 34% in Canada to 43% in Finland and the United States. Attitudes towards symptoms also varied between countries. Symptoms were described as moderate or severe by less than half of women from Finland and Sweden, compared with nearly two-thirds of women from the United States. However, vaginal atrophy was deemed to impact on quality of life by a higher proportion of women in Finland and Sweden (≥60%) than in the United Kingdom, the United States and Canada (≤50%). Overall, 77% of respondents believed women were uncomfortable discussing vaginal atrophy and 42% did not know that local treatment was available. The proportions of women unaware of the availability of local treatment were higher in the United States, the United Kingdom and Canada (51%, 50% and 48%, respectively), and very low in Finland (10%). Whilst 63% of women who had experienced vaginal atrophy had never been prescribed treatment for the condition, 67% of those who had been treated reported positive effects. CONCLUSION: The survey results illustrate differing needs of menopausal women in different countries. Country-specific approaches may be required to improve the uptake of treatment for vaginal atrophy.