4.7 Article

Preliminary results of the European multicentric phase III trial regarding sirolimus in slow-flow vascular malformations

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Pharmacology & Pharmacy

Evaluating everolimus for the treatment of breast cancer

Camille Moreau-Bachelard et al.

Summary: Everolimus is an oral drug that inhibits mTOR with immunosuppressive and antiproliferative characteristics. It is commonly used in association with exemestane in hormone receptor (HR)-positive advanced breast cancer (ABC). This review summarizes the publications relating to everolimus in breast cancer and highlights its efficacy and tolerability, particularly in patients who have previously been treated with aromatase inhibitors (AI).

EXPERT OPINION ON PHARMACOTHERAPY (2023)

Article Surgery

A prospective multicenter study of sirolimus for complicated vascular anomalies

Yi Ji et al.

Summary: The study showed that sirolimus is effective for pediatric patients with various types of complicated VAs, with main outcomes including reduction in lesion volume and improvements in disease severity score and quality of life. Some types of VAs responded better to sirolimus than others, but close monitoring of possible adverse events is necessary.

JOURNAL OF VASCULAR SURGERY (2021)

Review Cardiac & Cardiovascular Systems

Genetic Basis and Therapies for Vascular Anomalies

Angela Queisser et al.

Summary: In recent years, research on genetic and somatic mutations in vascular and lymphatic malformations has led to the evaluation of preexisting cancer drugs that interfere with these signaling pathways, marking the beginning of personalized treatment for vascular anomalies.

CIRCULATION RESEARCH (2021)

Article Genetics & Heredity

Non-hotspot PIK3CA mutations are more frequent in CLOVES than in common or combined lymphatic malformations

Pascal Brouillard et al.

Summary: Theragnostic management focuses on precise treatment according to molecular targets, with most patients with common or combined lymphatic malformations harboring somatic PIK3CA mutations. However, a quarter of patients do not have such mutations, indicating the presence of other causes. Hotspot mutations were more frequently detected in common and combined LMs compared to syndromic cases, suggesting the need for broader diagnostic genotyping beyond PIK3CA hotspot mutations.

ORPHANET JOURNAL OF RARE DISEASES (2021)

Article Dermatology

Sirolimus (Rapamycin) for Slow-Flow Malformations in Children The Observational-Phase Randomized Clinical PERFORMUS Trial

Annabel Maruani et al.

Summary: This study aimed to evaluate the efficacy and safety of sirolimus in treating children with slow-flow vascular malformations. Results showed that sirolimus significantly reduced volume and pain for children with pure lymphatic malformations. Overall, sirolimus had positive effects on pain, bleeding, oozing, self-assessed efficacy, and quality of life.

JAMA DERMATOLOGY (2021)

Review Dermatology

Theranostic Advances in Vascular Malformations

Valerie Dekeuleneer et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2020)

Review Dermatology

New and Emerging Targeted Therapies for Vascular Malformations

An Van Damme et al.

AMERICAN JOURNAL OF CLINICAL DERMATOLOGY (2020)

Article Computer Science, Interdisciplinary Applications

The REDCap consortium: Building an international community of software platform partners

Paul A. Harris et al.

JOURNAL OF BIOMEDICAL INFORMATICS (2019)

Article Cardiac & Cardiovascular Systems

Incidence of Malignancies in Patients Treated With Sirolimus Following Heart Transplantation

Rabea Asleh et al.

JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY (2019)

Article Genetics & Heredity

Somatic Activating PIK3CA Mutations Cause Venous Malformation

Nisha Limaye et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2015)

Article Medicine, Research & Experimental

Rapamycin improves TIE2-mutated venous malformation in murine model and human subjects

Elisa Boscolo et al.

JOURNAL OF CLINICAL INVESTIGATION (2015)

Review Genetics & Heredity

Immunosuppressive drugs and fertility

Clara Leroy et al.

ORPHANET JOURNAL OF RARE DISEASES (2015)

Article Biochemistry & Molecular Biology

Venous malformation-causative TIE2 mutations mediate an AKT-dependent decrease in PDGFB

Melanie Uebelhoer et al.

HUMAN MOLECULAR GENETICS (2013)

Article Genetics & Heredity

Variable Somatic TIE2 Mutations in Half of Sporadic Venous Malformations

J. Soblet et al.

MOLECULAR SYNDROMOLOGY (2013)

Review Cardiac & Cardiovascular Systems

Pediatric Vascular Malformations: Pathophysiology, Diagnosis, and the Role of Interventional Radiology

Anne Marie Cahill et al.

CARDIOVASCULAR AND INTERVENTIONAL RADIOLOGY (2011)

Article Surgery

Pathogenesis of Vascular Anomalies

Laurence M. Boon et al.

CLINICS IN PLASTIC SURGERY (2011)

Article Pediatrics

Vascular anomalies in 5621 patients: guidelines for referral

Arin K. Greene et al.

JOURNAL OF PEDIATRIC SURGERY (2011)

Review Biochemistry & Molecular Biology

From germline towards somatic mutations in the pathophysiology of vascular anomalies

Nisha Limaye et al.

HUMAN MOLECULAR GENETICS (2009)