Long-term outcomes from prophylactic or episodic treatment of haemophilia A: A systematic review.

INTRODUCTION: Evaluating treatment success in patients with haemophilia A (HA) remains a vigorous debate, especially concerning the interpretation of results from clinical and observational research. The benefits of short-term prophylaxis are well established, but long-term outcomes, particularly related to humanistic and economic burden, are not as well understood. AIM: We conducted a systematic literature review to evaluate the association of episodic or prophylactic bleed control with long-term clinical, humanistic and economic outcomes. METHODS: Studies published in English between 1 January 2006 and 15 December 2016 were included. Participants had HA (with or without inhibitors), received prophylactic or episodic treatment and had at least 4 years of treatment or follow-up. Results were analysed qualitatively with descriptive findings. RESULTS: A total of 2091 records were screened, resulting in 19 studies from 20 publications for inclusion. Most studies included children (84%), were limited to patients with severe disease (74%) and were conducted in Europe or North America (89%). Ten studies (53%) included patients with inhibitors. Median study follow-up ranged from 5 to 19 years. Long-term bleeding and haemarthrosis outcomes were consistently better for patients receiving prophylaxis, who also required fewer hospitalizations or surgeries. Health-related quality of life, functionality and productivity were generally more favourable in patients receiving prophylaxis. Quantitative comparisons were not feasible due to the lack of consistency in endpoint collection and reporting among studies. CONCLUSION: This systematic review confirmed that the benefits of prophylactic treatment on short-term outcomes translate to broader long-term clinical, humanistic and economic benefits. Better harmonization of data collection and outcome assessments across both registries and clinical studies is needed to allow for effective comparisons across studies and across data sources.

Patient satisfaction after the treatment of glabellar lines with Botulinum toxin type A (Speywood Unit): a multi-centre European observational study.

BACKGROUND: Whilst the efficacy and safety of glabella complex treatment with botulinum toxin type A (Speywood Unit) [BoNT-A (s.U)] has been comprehensively studied, there are very few trials on patient-reported outcomes and patient satisfaction associated with this treatment. OBJECTIVE: To assess the level of patient satisfaction 3 weeks and 4 months after the treatment of glabellar lines with BoNT-A (s.U). METHODS: This is a multi-centre, prospective, non-interventional observational study carried out in France, Germany, Spain and the United Kingdom. Subjects were eligible if the investigator had already decided to prescribe BoNT-A (s.U), according to the labelling. Subjects completed a questionnaire at both 3 weeks and 4 months after treatment. RESULTS: About 533 subjects completed at least one of the two questionnaires. About half of the subjects (47.9%) were naive to BoNT-A treatment of the glabella, while 50.6% had previously received another product. A high level of satisfaction was observed after the treatment, with 94.7% and 89.6% of subjects being satisfied or very satisfied with the aesthetic outcome at week 3 and month 4, respectively. Treatment was safe and well tolerated, as directly determined in the survey. Major reasons for satisfaction included the positive aesthetic outcome, a natural appearance, a rested look and comfort of injection. Most subjects felt the treatment brought them 'harmony', 'self-esteem/confidence' or 'youth'. Of the subjects who had previously been treated with another product, 51.2% considered the results obtained in the present study with BoNT-A (s.U) were better. CONCLUSION: Treatment of the glabellar lines with BoNT-A (s.U) led to a high level of patient satisfaction and a more positive self-perception up to 4 months after the treatment, regardless of whether the patients were naive or not to BoNT-A treatment.

Preventing melasma recurrence: prescribing a maintenance regimen with an effective triple combination cream based on long-standing clinical severity.

BACKGROUND: The relapsing nature of melasma emphasizes the need to maintain efficacy achieved after acute treatment. OBJECTIVE: To compare clinical efficacy and safety of two 6-month Triple Combination (TC; containing fluocinolone acetonide, hydroquinone and tretinoin) maintenance regimens in subjects with moderate to severe melasma, after daily treatment up to 8 weeks. METHODS: This randomized, investigator-blinded, controlled study had a maintenance phase of 6 months. Sixteen centres in Brazil and Mexico enrolled 242 subjects 18 years or older attaining no or mild melasma after 8 weeks of daily TC applications. Subjects were randomized to receive TC in a twice weekly or tapering regimen [3/week (1st month), 2/week (2nd month), 1/week (4th month)]. Efficacy and safety measurements included median time to relapse and relapse-free rate, Global Severity Score, Melasma Area and Severity Index score (MASI), subject's assessment, quality of life questionnaire (MelasQol), and adverse events. RESULTS: The majority (78.8%) had no or mild melasma (GSS ≤ 1) at week 8 and entered maintenance phase. After 6 months, 53% of patients remained relapse-free with improved quality of life, and time to relapse was similar between groups (about 190 days). Melasma severity at study entry, not maintenance baseline, influenced relapse rate. The twice weekly regimen tended to show better effectiveness in postponing relapse in severe melasma. Both regimens were safe. CONCLUSIONS: After resolution of melasma with TC, maintenance therapy over 6 months was successful in preventing relapse in over half of the patients who entered maintenance phase. Prescribing medicines should be adapted to patients based on melasma severity.

Combination therapy with adapalene-benzoyl peroxide and oral lymecycline in the treatment of moderate to severe acne vulgaris: a multicentre, randomized, double-blind controlled study.

BACKGROUND: Oral antibiotics in association with a topical retinoid with or without benzoyl peroxide (BPO) are the recommended first-line option in the treatment of moderate to severe acne vulgaris. OBJECTIVES: To evaluate the efficacy and safety of oral lymecycline 300 mg with adapalene 0·1%-BPO 2·5% (A/BPO) fixed-dose gel in comparison with oral lymecycline 300 mg with a vehicle gel in subjects with moderate to severe acne vulgaris. METHODS: A total of 378 subjects were randomized in a double-blind, controlled trial to receive once-daily lymecycline with either A/BPO or vehicle for 12 weeks. Evaluations included percentage changes from baseline in lesion counts, success rate (subjects 'clear' or 'almost clear'), skin tolerability, adverse events and patients' satisfaction. RESULTS: The median percentage reduction from baseline in total lesion counts at week 12 was significantly higher (P < 0·001) in the lymecycline with A/BPO group (-74·1%) than in the lymecycline with vehicle group (-56·8%). The success rate was significantly higher (47·6% vs. 33·7%, P = 0·002) in subjects treated with lymecycline and A/BPO. Both inflammatory and noninflammatory lesions were significantly reduced at week 12 (both P < 0·001) with a rapid onset of action from week 2 for noninflammatory lesions (P < 0·001) and week 4 for inflammatory lesions (P = 0·005). The A/BPO and lymecycline combination was well tolerated. The proportion of satisfied and very satisfied subjects was similar in both groups, but the number in the A/BPO group who were 'very satisfied' was significantly greater (P = 0·031). CONCLUSION: These results demonstrate the clinical benefit of combining A/BPO with lymecycline in the treatment of moderate to severe acne vulgaris.