New Approaches to Vascular Anomalies On the Horizon

Change in lesion size was graded as complete (90%-100%), substantial (60%-89%), intermediate (20%-59%), or none (<20%). Ninety-four percent of patients with macrocystic tumors had a complete or substantial response, whereas 63% of those with mixed LMs responded.

Patients with microcystic LMs did not respond to the treatment. Microcysts don’t respond well to sclerotherapy because it is impossible to inject the material into such a small cavity, said Dr. Smith. We’re still trying to determine how best to treat children with microcystic lymphatic malformations, he said, adding that watchful waiting is often used in these patients.

The OK-432 Collaborative Study Group also found that fewer than 2% of patients experienced spontaneous resolution. A 9% recurrence rate was observed during a median follow-up of 2.9 years. Eleven patients experienced adverse effects, such as airway obstruction, edema, and infection related to therapy. When compared with available surgical data, OK-432 is more effective and has a lower morbidity for the treatment of LMs, they wrote.

Obtaining US Food and Drug Administration approval of OK-432 is the next big step in trying to more successfully treat children with LMs, said Dr. Smith. Obtaining FDA approval is expensive and difficult for drugs used to treat orphan diseases like LM, but we remain optimistic that it will occur, said Dr. Bauman.

Vascular Anomalies

Vascular Tumors

• Infantile hemangiomas
• Congenital hemangiomas
• Rapidly involuting congenital hemangiomas (RICH)
• Non-involuting congenital hemangiomas (NICH)
• Other rare vascular tumors
• Kaposiform hemangioendotheliomas
• Tufted angiomas
• Hemangiopericytomas
• Angiosarcomas

Vascular Malformations

• High-flow lesions with arterial component
• Low-flow lesions
• Venous malformations
• Lymphatic malformation
• Microcystic
• Macrocystic
• Mixed-flow lesions
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