BACKGROUND: Facial angiofibromas (FAs) are a major feature of tuberous sclerosis complex (TSC). Topical rapamycin can successfully treat FAs. A new stabilized cream formulation that protects rapamycin from oxidation has been developed in 0.5% and 1% concentrations. OBJECTIVES: To assess the efficacy and safety of a novel, stabilized topical rapamycin cream formulation. METHODS: This multicentre double-blind randomized placebo-controlled dose-response phase II/III study with a parallel design included participants aged 6-65 years with FAs of mild or moderate severity according to the Investigator's Global Assessment (IGA) scale. Participants were randomized to one of three treatment arms: topical rapamycin 0.5%, topical rapamycin 1% or placebo. Treatment was applied once daily for 26 weeks. Safety and efficacy measures were assessed at days 14, 56, 98, 140 and 182. The primary endpoint was the percentage of participants achieving IGA scores of 'clear' or 'almost clear' after 26 weeks of treatment. Secondary measures included Facial Angiofibroma Severity Index (FASI) and participant- and clinician-reported percentage-based improvement. Safety measures included the incidence of treatment-emergent adverse events and blood rapamycin concentration changes over time. RESULTS: Participants (n = 107) were randomized to receive either rapamycin 1% (n = 33), rapamycin 0.5% (n = 36) or placebo (n = 38). All treated participants were included in the final analysis. The percentage of participants with a two-grade IGA improvement was greater in the rapamycin 0.5% treatment group (11%) and rapamycin 1% group (9%) than in the placebo group (5%). However, this was not statistically significant [rapamycin 0.5%: odds ratio (OR) 1.71, 95% confidence interval (CI) 0.36-8.18 (P = 0.50); rapamycin 1%: OR 1.68, 95% CI 0.33-8.40 (P = 0.53)]. There was a statistically significant difference in the proportion of participants treated with rapamycin cream that achieved at least a one-grade improvement in IGA [rapamycin 0.5%: 56% (OR 4.73, 95% CI 1.59-14.10; P = 0.005); rapamycin 1%: 61% (OR 5.14, 95% CI 1.70-15.57; P = 0.004); placebo: 24%]. Skin adverse reactions were more common in patients following rapamycin application (64%) vs. placebo (29%). CONCLUSIONS: Both rapamycin cream formulations (0.5% and 1%) were well tolerated, and either strength could lead to clinical benefit in the treatment of FA.

• MeSH
• angiofibrom * komplikace   farmakoterapie   MeSH
• dvojitá slepá metoda MeSH
• emoliencia terapeutické užití   MeSH
• imunoglobulin A MeSH
• imunosupresiva škodlivé účinky   MeSH
• lidé MeSH
• sirolimus MeSH
• tuberózní skleróza * komplikace   farmakoterapie   MeSH
• výsledek terapie MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• klinické zkoušky, fáze II MeSH
• multicentrická studie MeSH
• randomizované kontrolované studie MeSH

Angiofibromas are rare vascular tumors which originate predominantly in the nasopharynx and occur typically in male adolescents. Extranasopharyngeal sites such as nasal cavity and paranasal sinuses are less frequent. This review article was undertaken to evaluate the incidence, clinical features and management of extranasopharyngeal angiofibromas originating exclusivelly from nasal cavity structures. Our focus of interest was to evaluate the significance of immunohistochemical analysis in diagnosis of such extremely rare neoplasms. In the PubMed and Google Search, we found only 39 cases of nasal angifibroma, 27 males and 12 females from 1980 to 2012. The most prevalent site of origin was nasal septum, followed by inferior and middle turbinate. The commonest symptoms were nasal obstruction and epistaxis. Nasal angiofibromas are clinically distinct from nasopharyneal angiofibromas and can therefore be misdiagnosed. The differential diagnosis includes other vascular lesions, such as lobular capillary hemangioma and sinonasal-type hemangiopericytoma. Although immunohistochemistry is not necessary for differentiation between angiofibroma and capillary hemangioma, that diagnostic procedure may be helpful in distinction from sinonasal hemangiopericytoma. As an ilustration for immunohistochemical analysis, we presented a case of an elderly woman with tumor arising from the middle turbinate, diagnosed as angiofibroma. The staining was positive for CD34, CD31, factor VIII, vimentin and smooth muscle alpha-actin, and negative for desmin.

• MeSH
• angiofibrom komplikace   diagnóza   metabolismus   MeSH
• biopsie MeSH
• diferenciální diagnóza MeSH
• imunohistochemie metody   MeSH
• lidé středního věku MeSH
• lidé MeSH
• nádorové biomarkery metabolismus   MeSH
• nádory nosu komplikace   diagnóza   metabolismus   MeSH
• nosní dutina patologie   MeSH
• nosní obstrukce diagnóza   etiologie   metabolismus   MeSH
• počítačová rentgenová tomografie MeSH
• Check Tag
• lidé středního věku MeSH
• lidé MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH
• kazuistiky MeSH
• přehledy MeSH

Článek shrnuje literární přehled o embolizační léčbě nádorů hlavy a krku pro klinickou praxi. Chirurgická léčba hypervaskularizovaných nádorů hlavy, krku a mozku může být během operace spojena s velkými krevními ztrátami. Předoperační embolizace vybraných hypervaskularizovaných nádorů je v literatuře uváděna jako metoda, která krevní ztráty během operace snižuje. Tato technika spočívá v superselektivní katetrizaci přívodných tepen a v infuzi embolizačních částic nebo tekutých embolizačních látek (akrylátové lepidlo nebo etanol) tak, aby bylo vyplněno řečiště nádoru ve snaze navodit devaskularizaci, eventuálně nekrózu nádoru. Předoperační emboloterapie u vaskularizovaných nádorů je rozšířenou metodou; je však kontraverzní s ohledem na možnost komplikací a celkový zisk pro úspěšnou léčbu.

This is a review article on endovascular embolization of head, neck and brain tumors with the aim of helping the clinical management of these lesions. These hypervascular tumours can be associated with significant blood loss during surgery. Pre-operative embolization of these lesions has been proposed as a method of reducing the intra-operative blood loss.This technique involves the supraselective catheterisation of the feeding arteries of the tumour bed, followed by infusion of embolic particles or liquid agents (acrylic glue or ethanol) to obliterate the tumour bed with the aim of inducing devascularization, and eventually necrosis. Pre-operative embolotherapy for vascular tumours is well-established method, although controversial in terms of its risk-benefit ratio.

• MeSH
• angiofibrom diagnóza   komplikace   terapie   MeSH
• angiografie metody   využití   MeSH
• hemangiopericytom diagnóza   komplikace   terapie   MeSH
• lidé MeSH
• magnetická rezonanční tomografie využití   MeSH
• meningeom diagnóza   komplikace   terapie   MeSH
• nádory hlavy a krku diagnóza   komplikace   terapie   MeSH
• paragangliom diagnóza   komplikace   terapie   MeSH
• počítačová rentgenová tomografie využití   MeSH
• terapeutická embolizace metody   trendy   využití   MeSH
• Check Tag
• lidé MeSH

V práci sa pojednáva o neobvyklej lokalizácii juvenilného angiofibrómu u 16 ročného chlapca. Problémy pacienta – sťažené dýchanie cez pravú polovicu nosovej dutiny a recidivujúca epistaxa boli dôvodom opakovaných otorinolaryngologických vyšetrení. Pri rinoskopickom vyšetrení zistený polyp v pravej polovici nosovej dutiny, následne bol indikovaný na exstirpáciu v lokálnej anestézii. Operácia bola vykonaná bez komplikácií, peroperačné krvácanie minimálne, bez nutnosti naloženia tamponády. Pri histologickom vyšetrení polypu bola potvrdená diagnóza juvenilného angiofibrómu. V odstupe 3 týždňov po operácii sa objavila recidivujúca epistaxa z pravej polovice nosovej dutiny. V odstupe 6 týždňov od prvej operácie pri rinoskopickom vyšetrení zistená recidíva polypu v pravej polovici nosovej dutiny s krvácajúcou sliznicou, polyp vyrastal z predného okraja strednej lastúry. Bolo vykonané CT vyšetrenie ako i angiografické vyšetrenie pacienta. Následne vykonaná exstirpácia polypu s parciálnou resekciou strednej lastúry – endoskopickým prístupom. Histologické vyšetrenie potvrdilo, že sa jedná o juvenilný angiofibróm. Pacient je v dispenzárnom sledovaní, za obdobie 5 rokov od druhej endoskopickej operácie nedošlo k recidíve angiofibrómu ani k recidivujúcej epistaxe.

The unusual localization of juvenile angiofibroma of the sixteen-years old boy is discussed in this work. The patient's problems – hindered breathing through the right nasal cavity and recurrent bleeding from the nose – were the reasons of this recurrent examination at ENT ambulance. Polyp in the right nasal cavity was diagnosed by the rhinoscopic examination and was indicated for the extirpation in the local anaesthesia. The operation itself was made without bleeding complication. Postoperative bleeding was minimal, without needing of the firm nasal tamponade. Juvenile angiofibroma was stated by the histological analysis. In the period of three weeks after the first operation the relapse of the bleeding from the right nasal cavity appeared. Six weeks after the operation a polyp was authenticated in the right nasal cavity with the bleeding mucos, outgrowing from the front edge of the middle nasal conch. There were completed CT and angiographic examination and them extirpation of the polyp with partial resection of middle nasal conch was indicated – by the endonasal microsurgery. The histological analysis confirmed juvenile angiofibroma. Since the second operation the patient has been under dispensarization for five years now, and there have been found neither relapse of angiofibroma nor the episodes of epistaxis .

• MeSH
• angiofibrom diagnóza   chirurgie   komplikace   MeSH
• lidé MeSH
• mladiství MeSH
• nosní dutina abnormality   chirurgie   patologie   MeSH
• nosní polypy chirurgie   komplikace   terapie   MeSH
• Check Tag
• lidé MeSH
• mladiství MeSH
• mužské pohlaví MeSH
• Publikační typ
• kazuistiky MeSH
• srovnávací studie MeSH