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

Abstract

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.

• Authors: Christina Salvador, Andreas Entenmann, Roman Crazzolara, Gabriele Kropshofer
• Journal: Journal of pediatric hematology/oncology (J Pediatr Hematol Oncol) Vol. 44 Issue 3 Pg. 109-112 (Apr 01 2022) ISSN: 1536-3678 [Electronic] United States
• PMID: 34486566 (Publication Type: Case Reports, Journal Article)
• Copyright: Copyright © 2021 Wolters Kluwer Health, Inc. All rights reserved.

Chemical References

• Vinblastine
• Dasatinib
• Sirolimus
• Methotrexate

Topics

• Child
• Dasatinib (therapeutic use)
• Humans
• Infant
• Methotrexate (therapeutic use)
• Myofibromatosis (drug therapy, pathology)
• Sirolimus (therapeutic use)
• Vinblastine (therapeutic use)