title: Successful treatment of life-threatening generalized infantile myofibromatosis using low-dose chemotherapy.
abstract: 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.
authors.affiliation: Department of Haematology/Oncology, Great Ormond Street Hospital for Sick Children NHS Trust, London, United Kingdom.
authors.forename: Minal M
authors.initials: MM
authors.lastname: Gandhi
authors.forename: Paul C
authors.initials: PC
authors.lastname: Nathan
authors.forename: Sheila
authors.initials: S
authors.lastname: Weitzman
authors.forename: Gill A
authors.initials: GA
authors.lastname: Levitt
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D003131:Combined Modality Therapy; D003422:Critical Care; D003609:Dactinomycin / Q000008:administration & dosage; D005260:Female; D006801:Humans; D007231:Infant, Newborn; D008297:Male; D008727:Methotrexate / Q000008:administration & dosage; D019042:Muscle Neoplasms / Q000151:congenital / Q000188:drug therapy; D018224:Myofibromatosis / Q000151:congenital / Q000188:drug therapy*; D010014:Osteolysis; D011239:Prednisolone / Q000008:administration & dosage; D012074:Remission Induction; D012121:Respiration, Artificial; D012878:Skin Neoplasms / Q000151:congenital / Q000188:drug therapy; D012983:Soft Tissue Neoplasms / Q000151:congenital / Q000188:drug therapy*; D014747:Vinblastine / Q000008:administration & dosage; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D003609:Dactinomycin; D014750:Vincristine; D014747:Vinblastine; D011239:Prednisolone; D008727:Methotrexate