Vascular Anomalies
Conditions
Keywords
Vascular anomaly, Targeted therapy, Pi3KCA inhibitor, mTOR inhibitor, MEK inhibitor, QoL
Brief summary
This study aims to describe real-world patient characteristics, treatment patterns, and adverse events associated with targeted therapies used in patients with complex vascular anomalies. The study will create an active registry for participating centers to enter data on patients with complex vascular anomalies being treated with sirolimus/everolimus (mTOR inhibitors), with/without trametinib (MEK inhibitor), or alpelisib (PIK3CA inhibitor). Tertiary care centers in the United States (US) that receive referrals for complex vascular anomaly cases and use Electronic Health Records (EHRs) will contribute patient medical chart reviews to this registry.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
OTHER
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
0 Years to 90 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosed with a spectrum of vascular anomalies including but not limited to congenital vascular and lymphatic anomalies, vascular tumors and lymphatic malformations, and acquired vascular malformations. * Treated with ≥1 of mammalian target of rapamycin (mTOR) inhibitors, mitogen-activated protein kinase/ERK kinase (MEK) inhibitors and phosphoinositide 3-kinase (PI3K) inhibitors continuously for 3 months.
Exclusion criteria
* Patients diagnosed with a vascular anomaly who have not been treated with mTOR inhibitors, MEK inhibitors and PI3K inhibitors for at least 3 months. * Patients with other complex medical conditions; i.e. rare genetic syndromes. * Patients receiving many other systemic therapies making data collection not feasible. * Recurrent use of immunosuppressive agents, i.e. systemic steroids or targeted medical therapies for oncologic disorders, etc. * Patients with significant gaps in data collection. * Patients with concurrent enrollment in interventional trials. Other protocol-defined inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients by Treatment Received in Each Line of Therapy | Up to 10 years | — |
| Baseline Demographics | Baseline | — |
| Number of Patients by Clinical Characteristics | Baseline | Characteristics include vascular anomaly diagnosis, family history of vascular anomalies, cancer diagnosis, disease severity and anatomic locations involved, associated complications, other medical and surgical interventions, and other medications used. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Patients With Adverse Events | Up to 10 years | — |
| Number of Clinical Response Events PPPY | Up to 10 years | Clinical response: improvement of function, reduction of symptoms and complications, i.e. pain, infection, bleeding, hospitalization, etc. |
| Total Number of Clinical Response Events | Up to 10 years | — |
| Percentage of Patients Who Experience a Clinical Response | Up to 10 years | — |
| Treatment Duration | Up to 10 years | — |
| Number of Patients by Reason for Treatment Discontinuation | Up to 10 years | — |
| Frequency of Labs and Imaging for Disease Monitoring | Up to 10 years | — |
| Frequency of Adverse Events in Organ Systems | Up to 10 years | — |
| Percentage of Patients With Clinical Parameters Relevant to Routine Care | Up to 10 years | Clinical parameters will include dosing and treatment duration and frequency of follow up. |
| Percentage of Patients With Disease/Quality of Life (QoL) Impact | Up to 10 years | Since this is not a clinical trial and validated instruments are not frequently used in routine clinical visits, disease/QoL impact will be defined by impact on daily activities (walking ambulation, hobbies), demand on multidisciplinary care and impact on emotions (mood, self-esteem). |
| Number of Adverse Events per Person per Year (PPPY) | Up to 10 years | — |
| Total Number of Adverse Events | Up to 10 years | — |
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed