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

J Pediatr Hematol Oncol. 2003 Sep;25(9):750-4. doi: 10.1097/00043426-200309000-00016.

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.

Publication types

  • Case Reports
  • Review

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Combined Modality Therapy
  • Critical Care
  • Dactinomycin / administration & dosage
  • Female
  • Humans
  • Infant, Newborn
  • Male
  • Methotrexate / administration & dosage
  • Muscle Neoplasms / congenital
  • Muscle Neoplasms / drug therapy
  • Myofibromatosis / congenital
  • Myofibromatosis / drug therapy*
  • Osteolysis
  • Prednisolone / administration & dosage
  • Remission Induction
  • Respiration, Artificial
  • Skin Neoplasms / congenital
  • Skin Neoplasms / drug therapy
  • Soft Tissue Neoplasms / congenital
  • Soft Tissue Neoplasms / drug therapy*
  • Vinblastine / administration & dosage
  • Vincristine / administration & dosage

Substances

  • Dactinomycin
  • Vincristine
  • Vinblastine
  • Prednisolone
  • Methotrexate