Treatment Options

AAD Summer 1999 James F. Nigro, MD New York, New York July 31, 1999

Diagnosis and Management of Hemangiomas and Vascular Malformations in Childhood

I. Nomenclature of hemangiomas and vascular malformations

a. The major obstacle to the understanding and management of vascular birthmarks

b. Mulliken and Glowacki demonstrated that there are only two major types of vascular birthmarks based on differences in the following categories:
-Clinical
-Histologic
-Hematological
-Radiological
-Skeletal

c. Histologic features are the most important differences
-Hemangiomas have plump endothelia, increased mast cells, and multilaminated basement membranes
-Malformations have flat endothelia, normal mast cell numbers, and a thin basement membrane

d. Modern Nomenclature
-Hemangiomas are superficial, deep, or combined and may be proliferating or involuting
-Vascular malformations may be capillary, venous, arterial, lymphatic, or a combination of these

II. Hemangiomas

a. Introduction
-True benign neoplasm's
- Comprised of capillaries and venules in superficial and/or deep dermis
-Present during first few weeks of life
-Rapid proliferation and slow involution
-Most resolve completely without major complications

b. Incidence
-Female: male 3:1
-More common in Caucasians than in African Americans
-May be present in 10-20% of premature infants
-Solitary in 80% of patients

c. Location
-Based on percentage of body surface area, they are more common on the face
-By strict numbers, about 30% occur on the face or scalp

d. Precursor Lesions
-Appear prior to the actual proliferation of the hemangioma
-Pale patches
-Telangiectasia
-Macular erythema
-Bluish discoloration
-May be confused with port wine stain or nevus anemicus

e. Proliferative Phase
-Superficial lesions: red, raised, firm, well-demarcated
-Deep lesions; bluish, soft, slowly enlarging
-Wide variation in size -Growth phase: 3-12 months

f. Involution Phase
-Color change from bright to dull red
-Central greying
-Gradual softening
-Eventual resolution *50% by 5 years, 70% by 7 years, 90% by 9 years

g. Alarming Hemangiomas
-Vital/Important structures: eye, larynx, distal extremities
-Cosmetically sensitive regions: nose, lip, ear -Very large trunal resolution

h. Minor Complications
-Bleeding rare in these low flow lesions
-Infection: rare
-Ulceration: rapidly growing lesions and in the diaper area
-Residua: Telangiectasia, atrophy, hypopigmentation

i. Diffuse Neonatal Hemangiomatosis
-Multiple, small, cutaneous lesions
-Dome shaped, uniform in size
-May be associated with visceral lesions
· Liver, GI, CNS
· May be asymptomatic
· High-output cardiac failure, hemorrhage, obstructive jaundice, coagulopathy
-Involution of cutaneous and visceral lesions by age 2 years
-Ultrasound or MRI studies are indicated
-Treat symptomatic patients

j. Kasabach-Merritt Syndrome -hemangioendotheliama or tufted angioma
-extremely is usually involved
-coagulopathy associated with platelet trapping within lesions
-high mortality rate in untreated cases
-treatment
· Surgical excision
· Interferon
· Systemic corticosteroids

k. Associated Syndromes
-less common than with vascular malformations
-PHACE(S) syndrome
· Posterior fossa CNS malformations (Dandy Walker)
· Hemangioma
· arterial anomalies
· cardiac anomalies
· eye anomalies and (sternal defects)
-lumbosacral lesions
· spinal anomalies
· genitourinary anomalies

I. Therapy
-observation
· photography
· regular follow-up visits
· reserve right to initiate therapy at a later date
-systemic corticosteriods
· 2-3 mg/kg/day for 4-6 week and then slowly taper
· younger infants may require a longer or second course
· immunizations: hold until off steroids for 1 month
· side effects: increased appetite, change in sleep patterns, fussiness -intralesional corticosteroids
· 3-5 mg/kg/dose
· systemic absorption is significant
· potential adverse side effects
· soft tissue atrophy
· eyelid necrosis
· perforation of the globe
· retinal artery occlusion
-topical corticosteroids
· high potency
· may be effective in small superficial hemangiomas
-interferon alpha
· antiangiogenic activity
· 3 million units/meter sq/day subcutaneous
· treatment is required for several months
· excellent results in severe or life threatening hemangiomas unresponsive to corticosteroids
· adverse effects
· fever
· neutropenia
· spastic diplegia
· motor delay
-laser
· tunable yellow dye (flash lamp pumped pulse dye)
· very thin or precursor hemangiomas
· ulcerations
· residual telangiectasia
· Nd: YAG
· Bulky facial lesions
· Increased risk of scarring
· Experimental
-surgical excision
· protuberant lesions
· consider surgical consultation when parents are very anxious
· avoid if hemangioma is diffuse -duoderm
· excellent pain control in ulcerated perineal lesions
-cryosurgery
· risk of scarring
· good results are possible with experienced hands

III. Capillary Malformations (Port Wine Stains)

a. Introduction
-vascular malformations limited to dermal blood vessels
-present at birth
-permanent
-associated with other vascular malformations and congenital syndromes

b. Incidence
-0.3% of neonates
-equal sex and racial predilection
-50% of facial PWS restricited to one trigeminal sensory region -remainder involve more than one, cross midline, or are bilateral

c. Appearance
-pink, well-circumscribed patches
-growth is commensurate with growth of the child
-darken and thicken with age

d. Sturge-Weber syndrome -facial port wine stain
· V1 trigeminal sensory region must be involved
-CNS
· Seizures
· Mental retardation
· Railroad track calcifications or cortex
-opthalmologic
· Ipsilateral choroidal angiomatosis
· Glaucoma (can be seen with V2 lesions involving eyelid)

e. Treatment
-tunable dye laser
· Treatment of choice
· Multiple treatments required (average 6.4)
· Very good to excellent results in most patients
· Few side effects
-Anesthesia
· General: infants and children with large lesions
· Topical: older patients with large or small lesions
· None: most adults with small lesions
-psychological evaluation
-neurologic and opthalmologic exam
-other lasers, tattooing, excision, radiation are not indicated

IV. Venous Malformations

a. clinical features
-bluish patch or mass with indistinct borders
-present at birth but may not be evident
-compressible
-phleboliths, thrombosis, hemorrage
-frequently confused with deep hemangiomas

b. treatment
-none
-surgical excision
· Image prior to surgery to determine extent of lesion -sclerotherapy
-elastic stockings

V. Arteriovenous Malformations

a. clinical features
-high flow-may involve bone, muscle, viscera
-often undiagnosed until adulthood
-discoloration or pulsatile mass may be noted

b. treatment -surgical excision -embolization

VI. Lymphatic Malformations

a. localized or diffuse

b. may slowly enlarge over time

c. may be confused with deep hemangiomas

d. superficial lesions may respond to laser therapy

e. incomplete surgical excision can lead to massive overgrowth

f. support garments

VI. Syndromes Associated with Vascular Malformations

a. Klippel-Trenaunay
-definition: soft tissue hypertrophy and bony overgrowth of extremity with PWS
-clinical features
· Usually single lower extremity
· Overgrowth not present at birth
· Significant limb length discrepancy
· Prominent hypertrophy of foot and toes
· No CNS or visceral anomalies
-treatment
· Premature epiphyseal closure of longer leg
· Surgical debulking is usually not feasible

b. Maffucci's syndrome venous malformations -enchondromes -distal extremities

c. Blue Rubber Bleb Nevus syndrome -venous malformations of skin and GI tract -compressible, painful lesions -GI hemorrage is common cause of death

d. Gorham's syndrome -venous and lymphatic malformations involving skin and skeleton -osteolytic bone disease

e. Proteus syndrome: PWS, partial gigantism, macrocephaly, epidermal nevi

f. Wyburn-Mason syndrome: retinal and CNS AVM, facial PWS

g. Riley-Smith syndrome: cutaneous venous malformation, macrocephaly

h. Cobb syndrome: venous malformations of spinal cord, truncal PWS

i. Bannayan-Zonana syndrome: subcutaneous/ visceral venous malformation, lipomas, macrocephaly