Lymphatic Anomalies Registry for the Assessment of Outcome Data

Sponsor

Boston Children's Hospital (Other)

Overall Status

Recruiting

CT.gov ID

NCT02399527

Collaborator

Lymphatic Malformation Institute (Other)

1,000

Enrollment

Location

264

Anticipated Duration (Months)

3.8

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the relative benefit of medical therapies and procedures is limited.The goal of this project is to better understand these diseases and improve the care of theses rare patients. To do this, the investigators are conducting an observational study of patients with lymphatic anomalies, including an annual follow-up questionnaire to gather prospective data on mortality, morbidity, treatments, and functionality as well as quality of life.

Condition or Disease	Intervention/Treatment	Phase
Lymphatic Malformation Generalized Lymphatic Anomaly (GLA) Central Conducting Lymphatic Anomaly CLOVES Syndrome Gorham-Stout Disease ("Disappearing Bone Disease") Blue Rubber Bleb Nevus Syndrome Kaposiform Lymphangiomatosis Kaposiform Hemangioendothelioma/Tufted Angioma Klippel-Trenaunay Syndrome Lymphangiomatosis

Detailed Description

The purpose of the Lymphatic Anomalies Registry, created at Boston Children's Hospital, is to create a database to help current and future patients diagnosed with a lymphatic anomaly. The ultimate goal of the registry is to better understand and predict responses to therapies and risk factors for complications. Although the Lymphatic Anomalies Registry exists at Boston Children's Hospital, patients can be entered into the registry regardless of whether or not they visit Boston Children's Hospital, thus increasing the program's accessibility. The Lymphatic Anomalies Registry includes patients who have vascular anomalies with a lymphatic component across various diagnoses. From the patient's perspective, participation in the Lymphatic Anomalies Registry means taking part in a short, thirty minute interview, and providing the registry with access to medical records. The interview is conducted verbally with study staff of the Lymphatic Anomalies Registry, and can take place either at the hospital, or over the phone. During the interview, the registry will inquire about the patient's diagnosis, disease features, medical therapies, and procedures. Interested prospective patients will receive an introductory packet from the registry with information on how to proceed in the registry process. All obtained patient information is housed on a secure, HIPPA compliant, internal database, managed by Boston Children's Hospital staff. Patient information entered into the external database is de-identified. The research teams will also obtain a medical record release form to request the patient's medical record for review in our study.

Study Design

Study Type:

Observational [Patient Registry]

Anticipated Enrollment :

1000 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Lymphatic Anomalies Registry for the Assessment of Outcome Data

Actual Study Start Date :

Jun 1, 2013

Anticipated Primary Completion Date :

Jun 1, 2035

Anticipated Study Completion Date :

Jun 1, 2035

Outcome Measures

Primary Outcome Measures

To characterize the heterogeneity of lymphatic disorders, including demographics, presentation, and complications. [15 years]
To identify factors that are prognostic of the occurrence of complications, including effusions, coagulopathy, ectatic draining veins, prior infections, visceral involvement, bone involvement, and development of cardiopulmonary symptoms. [15 years]
To identify factors prognostic of poor outcome and use them to develop "staging" of lymphatic anomalies. [15 years]
To describe the natural history of lymphatic anomalies, including morbidity and mortality. [15 years]
To describe the therapies (medical and procedural), adverse events and responses to therapy in patients with lymphatic anomalies. [15 years]
To pilot quality of life, functional assessment and pain scoring tools in this patient population. [15 years]

Secondary Outcome Measures

To estimate the proportion of time that patients with lymphatic anomalies have affected offspring. [15 years]
To assess for correlations of pregnancy complications or medications taken during pregnancy with the development of lymphatic anomalies. [15 years]