Clinical trial NCT00394888: Hemangioma Associated With High Rates of Morbidity
Official title: Hemangioma Associated With High Rates of Morbidity:A Prospective Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Hemangioma
Keywords: Hemangioma
PHACE
Lumbosacral hemangioma
Occult Spinal Dysraphism
Lead sponsor: Medical College of Wisconsin
Sponsor class: OTHER
Collaborators: name: University of California, San Francisco
name: Columbia University
name: Baylor College of Medicine
name: St. Justine's Hospital
name: State University of New York - Downstate Medical Center
name: Children's Mercy Hospital Kansas City
name: Children's Hospital Medical Center, Cincinnati
name: Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
First posted: 2006-11-02
Enrollment: 433
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: SCREENING

Brief summary: We are conducting a study on the possible presence of PHACES in children with large facial hemangiomas and lumbosacral hemangiomas of infancy (hemangioma in the lower back) . With this study we hope to better understand the risk of this syndrome and to develop guidelines for its evaluation and management.

Detailed description: Large hemangiomas of the face can be associated with anomalies of the blood vessels of head and chest. The acronym PHACES indicates the association of Posterior fossa and other brain malformations, facial Hemangioma, Arterial anomalies, Coarctation of the aorta and other cardiac defects, Eye abnormalities and Sternal malformations. Study subjects will be recruited through the Pediatric Dermatology department in several cities. All patients age 0-1 year old of age, who present with large facial hemangioma (\>22 cm\^2) will be offered to participate in the study. Parents will be interviewed to obtain personal, medical, and family history.

Patients will undergo standard of care evaluation for facial hemangioma with risk of PHACE syndrome. This includes skin, eye and neurological examination, photograph, magnetic resonance imaging (MRI) of head/neck/chest, and lab tests requiring samples of blood, urine or stool. No other tests will be performed for participating in the study.

Lumbosacral hemangiomas of infancy (hemangioma in the lower back) can be associated with anomalies of the spine. "Tethered cord syndrome" indicates a condition caused by abnormally stretched spinal cord. Over time this condition can lead to neurological damage. Although often there are no symptoms until adulthood, it can become apparent during childhood. Common symptoms are: lower back pain, pain and weakness of the legs, walking problems, and bladder and bowel loss of control. "Occult spinal dysraphism" is the term used when the defect of the spine is hidden under normal skin. We noticed that infants with hemangioma in the lower back area are more inclined to present a hidden spine defect.

Study subjects will be recruited through the Pediatric Dermatology departments in several cities. All patients age 0-18 year old of age, who present with lumbosacral hemangioma (\> 2.5 cm of diameter overlying the spine) will be offered to participate in the study. Parents will be interviewed to obtain personal, medical, and family history.

Patients will undergo standard of care evaluation for lumbosacral hemangioma. This includes skin, neurological examination, photograph, magnetic resonance imaging (MRI) of the back, lab tests requiring samples of blood, urine or stool. No additional tests will be performed only for participating in the study.

Eligibility criteria: Segmental Facial Hemangioma

Inclusion criteria:

* Infants less than 1 year of age
* Hemangiomas of the head /facial area measuring 22cm2 or greater.

Exclusion criteria:

* Children greater than 1 year of age.
* Children with segmental hemangiomas present in locations other than the head.
* Children presenting with localized (focal) or indeterminate hemangiomas in any location.
* Children with other vascular tumors (such as tufted angioma, Kaposiform hemangioendothelioma, non-involuting congenital hemangioma or rapidly-involuting congenital hemangioma) or vascular malformations.

Lumbosacral Hemangioma

Inclusion criteria:

* Individuals less than 18 years of age.
* Hemangioma, hemangioma precursor, or definitive residual hemangioma larger then 2.5 cm in diameter, overlying the midline lumbar spine or sacral spine in which any portion of the hemangioma is located over the midline.

Exclusion Criteria:

* Excluding perirectal hemangiomas that do not extend onto the sacral spine.
* Excluding perineal hemangiomas that do extend to overlay the sacral spine.

Primary outcomes:
measure: MRI/MRA of Head/Neck/Chest.
timeFrame: 2 years
measure: Clinical Diagnosis of PHACE Syndrome
timeFrame: 2 years
measure: Spinal Abnormalities
timeFrame: 2 years
measure: Cerebrovascular and Structural Brain Abnormalities
timeFrame: 2 years
measure: Cardiac Abnormalities Detected Via Clinical Examination
timeFrame: 2 years
measure: Frequency of Hepatic Hemangiomas Detected Via Abdominal Ultrasound
timeFrame: 2 years
Secondary outcomes:
Other outcomes:
Locations:
city: Milwaukee
country: United States
facility: Beth Drolet, MD
state: Wisconsin
Study officials:
affiliation: Medical College of Wisconsin
name: Beth Drolet, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Columbia University
name: Maria Garzon, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Baylor College of Medicine
name: Denise Metry, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of California, San Francisco
name: Ilona Frieden, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hopital Sainte-Justine
name: Julie Powell, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital of Cincinnati
name: Anne Lucky, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: State University of New York - Downstate Medical Center
name: Sharon Glick, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
name: Eulalia Baselga, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Indiana University
name: Anita Haggstrom, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Northwestern University
name: Anthony Mancini, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Mercy Hospital Kansas City
name: Kimberly Horii, MD
role: PRINCIPAL_INVESTIGATOR
References: