New Therapeutic Approach in an Infant With Systemic Myofibromatosis and Intestinal Hemorrhage

• Published in: Journal of pediatric hematology/oncology Vol. 44; no. 3; pp. 109 - 112
• Main Authors: Salvador, Christina, Entenmann, Andreas, Crazzolara, Roman, Kropshofer, Gabriele
• Format: Journal Article
• Language: English
• Published: United States Lippincott Williams & Wilkins 01-04-2022
• Subjects: Child; Dasatinib - therapeutic use; Humans; Infant; Methotrexate - therapeutic use; Myofibromatosis - drug therapy; Myofibromatosis - pathology; Sirolimus - therapeutic use; Vinblastine - therapeutic use
• ISSN: 1077-4114; 1536-3678
• DOI: 10.1097/MPH.0000000000002324

We report the case of an infant with multicentric myofibromatosis affecting the gastric and intestinal mucosa, leading to continuous intestinal hemorrhage and iron deficiency. Conventional vinblastine and methotrexate combination treatment was administered for 4 months, but persistent intestinal blood loss required repeated blood transfusions. Because of insufficient tumor response to treatment, we opted for the experimental combination of rapamycin and dasatinib. Six weeks after the start of this therapy, hemoglobin levels stabilized without transfusions, and no fecal blood loss was detected. In addition, a follow-up magnetic resonance imaging excluded tumor progression. We here show the effectiveness of an experimental therapy with rapamycin and dasatinib in a child with multicentric myofibromatosis after the failure of conventional therapy with vinblastine and methotrexate.