Successful treatment of life-threatening generalized infantile myofibromatosis using low-dose chemotherapy

Infantile myofibromatosis is the most common fibrous tumor of infancy. The generalized form of the disease is associated with a high rate of early mortality, especially if visceral structures are involved. Various therapeutic strategies have been used in these patients, including high-dose chemotherapy, with the risk of therapy-related toxicity. The authors present two cases of generalized infantile myofibromatosis, with life-threatening visceral and nonvisceral involvement, in which the patients were cured with a combination of low-dose chemotherapy and intensive care. The authors propose a prospective international trial using a safe low-dose chemotherapy protocol to test the efficacy of this treatment strategy.