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Sirolimus vascular malformation

Juan Putra, Anita Gupta
Kaposiform haemangioendothelioma (KHE) is a rare, locally aggressive/borderline vascular tumour primarily seen in neonates and children. KHE is occasionally associated with Kasabach-Merritt phenomenon and tends to have a poor clinical prognosis. While the histological features of tufted angiomas and KHE overlap, some believe tufted angiomas are a milder, benign, more localised counterpart of KHE. The other histological differential diagnoses of KHE include infantile haemangioma, congenital haemangioma, spindle cell haemangioma, verrucous malformation/haemangioma, and Kaposi sarcoma...
June 2017: Pathology
Kevin Y Zhuo, Susan Russell, Orli Wargon, Susan Adams
Venous malformations are slow-flow congenital vascular malformations that enlarge as the child ages and may be associated with localised intravascular coagulation, a consumptive coagulopathy characterised by elevated D-dimer and decreased fibrinogen levels. The authors review the known correlations between localised intravascular coagulation and venous malformation number, size and planes involved, and call attention to the concept of the progression of localised intravascular coagulopathy as the child ages and their venous malformations enlarge...
February 7, 2017: Journal of Paediatrics and Child Health
Türkan Akbayrak, Ceren Orhan, Emine Baran, Serap Kaya, Gürsoy Coskun, Ali Varan
The aim of the present case report was to investigate the effects of a physiotherapy program combined with sirolimus in a child patient with upper extremity edema and joint limitation due to low-flow vascular malformation. This case report included an 11-year-old male patient (26 kg, 130 cm) diagnosed with congenital lymphovascular malformation on the left and right chest. The patient, who had edema on the upper left extremity and experienced joint limitations, was administered complete decongestive therapy (CDT) and manual therapy in combination with sirolimus...
2016: Turkish Journal of Pediatrics
Aysel Ünlüsoy Aksu, Sinan Sari, Ödül Eğritaş Gürkan, Buket Dalgiç
Recently, sirolimus was demonstrated to be effective in treating vascular lesions and lessening the frequency of bleeding and secondary iron deficiency anemia. We present a child with blue rubber bleb nevus syndrome who had prolonged history of iron deficiency anemia secondary to unrecognized gastrointestinal bleeding. Treatment with propranolol, omeprazole and iron had failed. After 2.5 months of sirolimus therapy (trough levels 1 to 5 ng/mL), his hemoglobin concentration improved into the normal range and remained stable...
March 2017: Journal of Pediatric Hematology/oncology
Dov C Goldenberg, Marcelo Carvas, Denise Adams, Marcelo Giannotti, Rolf Gemperli
Management of complex vascular malformation represents a challenge as it may include a wide variety of options such as embolization, laser therapy, sclerotherapy, and surgical resection but may lead to significant morbidity and is associated with high recurrence rates. In extreme and/or recurrent cases, successful use of sirolimus has been described. We report a case of large unresectable complex venous malformation treated with oral sirolimus for 24 months. Therapy was well tolerated. Patient had substantial improvement in symptoms and shrinkage of the lesion...
May 2017: Journal of Pediatric Hematology/oncology
Paloma Triana, Mariela Dore, Vanesa Nuñez Cerezo, Manuel Cervantes, Alejandra Vilanova Sánchez, Miriam Miguel Ferrero, Mercedes Díaz González, Juan Carlos Lopez-Gutierrez
Aim of the Study mTOR inhibitors are showing promising results in the management of vascular anomalies. Although current controlled trials remain to be completed, many individual experiences are being published. We present our series of children with complex vascular anomalies treated with sirolimus. Patients and Methods A retrospective review of 41 patients treated with sirolimus between January 2011 and December 2015 was performed: 15% (n = 6) had vascular tumors (4 kaposiform hemangioendotheliomas, 1 PTEN) and 85% (n = 35) had malformations (13 generalized lymphatic anomalies/Gorham-Stout diseases [GSD], 1 kaposiform lymphangiomatosis [KLA], 11 large lymphatic malformations (LMs) in critical areas, 2 lymphedemas, 4 venous malformations, and 4 aggressive arteriovenous malformations [AVM])...
February 2017: European Journal of Pediatric Surgery
Cameron C Trenor
We have entered an exciting era in the care of patients with vascular anomalies. These disorders require multidisciplinary care and coordination and dedicated centers have emerged to address this need. Vascular tumors have been treated with medical therapies for many years, while malformations have been historically treated with endovascular and operative procedures. The recent serendipitous discoveries of propranolol and sirolimus for vascular anomalies have revolutionized this field. In particular, sirolimus responses are challenging the dogma that vascular malformations are not biologically active...
September 2016: Seminars in Cutaneous Medicine and Surgery
Sule Yesil, Hikmet Gulsah Tanyildiz, Ceyhun Bozkurt, Emin Cakmakci, Gurses Sahin
Vascular malformations (VMs) are described as congenital malformations of the vasculature derived from capillaries, veins, lymphatic vessels, arteries, or a combination of these vessels. They can cause significant morbidity resulting from soft tissue hypertrophy-related disfiguration, bony abnormalities, and even organ compromise. They are usually treated with various interventional procedures to achieve local control; however, the chance of success decreases as the anatomical distribution of the malformation widens...
April 2016: Pediatric Hematology and Oncology
Juliana R Gildener-Leapman, Jamie B Rosenberg, Anne Barmettler
A 15-month-old boy with left congenital proptosis presented to the emergency department with melena. Upper GI endoscopy and magnetic resonance angiography revealed vascular lesions, consistent with gastrointestinal tract manifestations of blue rubber bleb nevus syndrome. MRI revealed vascular malformations in both orbits, with mass effect on the left side. The patient was started on a trial of the antiangiogenic agent sirolimus (also known as rapamycin), and after 6 months of treatment showed clinical improvement in proptosis supported by radiologic evidence of regression in the larger, left orbital mass, with stability of the smaller, right orbital mass...
May 2017: Ophthalmic Plastic and Reconstructive Surgery
Stuart L Cramer, Shi Wei, Arnold C Merrow, Joseph G Pressey
Gorham-Stout disease is a life-threatening disorder often manifested by lymphatic malformation and osteolysis. Unfortunately, available therapies are not uniformly effective and often carry substantial morbidity. We report an 18-year-old male with Gorham-Stout disease manifested by lytic rib lesions and an intractable pleural effusion that responded dramatically to the combination of the mammalian target of rapamycin (mTOR) inhibitor sirolimus and the aminobisphosphonate zoledronic acid after failing interferon therapy...
April 2016: Journal of Pediatric Hematology/oncology
Ann M Defnet, Naina Bagrodia, Sonia L Hernandez, Natalie Gwilliam, Jessica J Kandel
Multimodal treatment of lymphatic malformations continues to expand as new information about the biology and genetics of these lesions is discovered, along with knowledge gained from clinical practice. A patient-centered approach, ideally provided by a multidisciplinary medical and surgical team, should guide timing and modality of treatment. Current treatment options include observation, surgery, sclerotherapy, radiofrequency ablation, and laser therapy. New medical and surgical therapies are emerging, and include sildenafil, propranolol, sirolimus, and vascularized lymph node transfer...
May 2016: Pediatric Surgery International
Marion Nadal, Bruno Giraudeau, Elsa Tavernier, Annie-Pierre Jonville-Bera, Gerárd Lorette, Annabel Maruani
Mammalian target of rapamycin (mTOR) inhibitors are a promising new treatment in vascular anomalies, but no published randomized controlled trials are available. The aim of this systematic review of all reported cases was to assess the efficacy and safety of mTOR inhibitors in all vascular anomalies, except cancers, in children and adults. In November 2014 MEDLINE, CENTRAL, LILACS and EMBASE were searched for studies of mTOR inhibitors in any vascular condition, except for malignant lesions, in humans. Fourteen publications and 9 posters, with data on 25 and 59 patients, respectively, all < 18 years old were included...
May 2016: Acta Dermato-venereologica
Thomas D Griffin, James P Foshee, Robert Finney, Nazanin Saedi
BACKGROUND AND OBJECTIVE: A port wine stain (PWS) is a type of capillary vascular malformation composed of malformed, dilated blood vessels within the papillary and reticular dermis. Currently, pulsed dye laser (PDL) is considered the therapeutic gold standard, although greater than 90% of lesions may be refractory to treatment. Studies have shown that a delay in treatment results in a higher proportion of patients who develop hypertrophy and nodularity within lesions that become more resistant to therapy...
February 2016: Lasers in Surgery and Medicine
Hélder Cardoso, Jorge Amil Dias, Marco Silva, Filipe Vilas-Boas, Eunice Trindade, Marta Tavares, Guilherme Macedo
No abstract text is available yet for this article.
March 2016: Journal of Gastroenterology and Hepatology
Aleksandar M Vlahovic, Natasa S Vlahovic, Emir Q Haxhija
Management of patients with complex vascular anomalies (VAs) is often associated with significant morbidity and mortality because of the lack of effective treatment modalities that may lead to significant improvement of the disease and/or healing. Recently, reports of treatment of patients with complex VAs with sirolimus revealed encouraging results. Sirolimus inhibits the mammalian target of rapamycin, which acts as a master switch of numerous cellular processes. We report a successful use of sirolimus for the treatment of a patient with a complex CLVM of the trunk and the right lower extremity believed to be untreatable...
August 2015: Pediatrics
Giacomo Colletti, Pietro Dalmonte, Laura Moneghini, Daris Ferrari, Fabiana Allevi
Arteriovenous malformations (AVMs) are high-flow vascular malformations characterised by a complex vessel network directly connecting feeding arteries and draining veins, typically featured by a natural history of progression, while spontaneous regressions are purely anecdotal. AVMs are very aggressive entities that possess a locally infiltrative behaviour like neoplasms. Complete "radical" surgical excision presents the highest chance of cure, but nowadays there is still considerable controversy on how to treat large AVMs that are not amenable of "radical" excision...
September 2015: Medical Hypotheses
Herwig Lackner, Anna Karastaneva, Wolfgang Schwinger, Martin Benesch, Petra Sovinz, Markus Seidel, Daniela Sperl, Sofia Lanz, Emir Haxhija, Friedrich Reiterer, Erich Sorantin, Christian E Urban
UNLABELLED: Vascular anomalies include a heterogeneous group of disorders that are categorized as vascular tumors or vascular malformations. Treatment options include resection, embolization, laser therapy, and sclerotherapy or medical treatment such as propranolol, steroids, interferon, and cytostatic chemotherapy. Mammalian target of rapamycin seems to play a key role in the signal pathway of angiogenesis and subsequently in the development of vascular anomalies. Recently, the successful use of sirolimus has been reported in children with lymphatic malformations and kaposiform hemangioendotheliomas...
December 2015: European Journal of Pediatrics
Naina Bagrodia, Ann M Defnet, Jessica J Kandel
PURPOSE OF REVIEW: To review the literature on lymphatic malformations and to provide current opinion about the management of these lesions. RECENT FINDINGS: Current treatment options include nonoperative management, surgery, sclerotherapy, radiofrequency ablation, and laser therapy. New therapies are emerging, including sildenafil, propranolol, sirolimus, and vascularized lymph node transfer. The primary focus of management centers on the patient's quality of life...
June 2015: Current Opinion in Pediatrics
David Kim, Latanya Benjamin, Ashley Wysong, David Hovsepian, Joyce Teng
Venolymphatic malformations (VLMs) are vascular anomalies consisting of both veins and lymph vessels. A 2-week-old newborn presented with large VLMs on the left forehead, temple, preauricular area, and orbit. Patient was at imminent risk for permanent vision loss due to a localized mass effect. Surgical excision or debulking was contraindicated due to its complexity and proximity to the left eye, and the patient failed to respond to the sildenafil treatment and sclerotherapy. Patient was subsequently started on oral sirolimus 0...
July 2015: Dermatologic Therapy
Laura Marqués, Jorge M Núñez-Córdoba, Leyre Aguado, Maider Pretel, Pablo Boixeda, Eduardo Nagore, Eulalia Baselga, Pedro Redondo
BACKGROUND: Sturge-Weber syndrome (SWS) is characterized by port-wine stains (PWS) affecting the face, eyes, and central nervous system. Pulsed dye laser (PDL) is the standard treatment for PWS. Unfortunately, recurrence is frequent because of reformation and reperfusion of blood vessels. OBJECTIVE: We sought to assess the clinical efficacy of topical rapamycin combined with PDL in PWS of patients with SWS. METHODS: We conducted a phase II, randomized, double-blind, intraindividual placebo-controlled, clinical trial...
January 2015: Journal of the American Academy of Dermatology
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