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New Therapeutic Approach in an Infant With Systemic Myofibromatosis and Intestinal Hemorrhage.
Journal of Pediatric Hematology/Oncology ( IF 1.2 ) Pub Date : 2021-09-06 , DOI: 10.1097/mph.0000000000002324
Christina Salvador 1 , Andreas Entenmann 2 , Roman Crazzolara 1 , Gabriele Kropshofer 1
Affiliation  

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.

中文翻译:

婴儿全身性肌纤维瘤病和肠出血的新治疗方法。

我们报告了一例影响胃和肠粘膜的多中心肌纤维瘤病婴儿,导致持续性肠道出血和缺铁。常规的长春碱和甲氨蝶呤联合治疗进行了 4 个月,但持续的肠道失血需要反复输血。由于肿瘤对治疗的反应不足,我们选择了雷帕霉素和达沙替尼的实验性组合。治疗开始六周后,血红蛋白水平在没有输血的情况下稳定下来,并且没有检测到粪便失血。此外,后续磁共振成像排除了肿瘤进展。
更新日期:2021-09-06
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