Infantile myofibromatosis: Excellent prognosis but also rare fatal progressive disease. Treatment results of five Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry.

BACKGROUND: Infantile myofibromatosis (IM) is a rare benign soft tissue tumor and often a self-limiting disease but rarely includes life-threatening complications. Little is known about optimal treatment of primary localized (LD) and multifocal disease (MFD). METHODS: Treatment and outcome of 95 children with IM registered within five Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry (1981-2016) were evaluated. RESULTS: LD was diagnosed in 71 patients at a median age of 0.4 years (range 0.0-17.7). MFD was present in 24 patients. The mainstay of treatment was watch-and-wait strategy (w&w) after initial biopsy or resection. Low-dose chemotherapy (CHT) was administered to 16/71 (23%) patients with LD and eight of 24 (33%) patients with MFD, imatinib was added in two. A delayed resection was possible in eight of 71 (11%) and five of 24 (21%) patients with LD and MFD, respectively. Overall, patients were alive in complete remission (n = 77) and partial remission (n = 10) at a median follow-up time of 3.4 years after diagnosis (range 0.01-19.4); no data available (n = 5). Three patients died of progressive disease (PD) despite CHT. Gender, tumor size, and location correlated with a favorable event-free survival (EFS) in patients with LD. The 5-year EFS and overall survival of patients with LD were 73% (±12, confidence interval [CI] 95%) and 95% (±6, CI 95%), respectively; for MFD 51% (±22, CI 95%) and 95% (±10, CI 95%). CONCLUSION: Prognosis is excellent in patients with LD and MFD. Targeted treatment needs to be evaluated for rare fatal PD.

Encouraging early clinical experience with deliberately delayed temporary fetoscopic tracheal occlusion for the prenatal treatment of life-threatening right and left congenital diaphragmatic hernias.

OBJECTIVE: In order to assess the effect of deliberately delayed percutaneous fetoscopic tracheal occlusion on survival of fetuses with life-threatening congenital diaphragmatic hernia. METHODS: Eight fetuses with life-threatening congenital diaphragmatic hernia underwent fetoscopic tracheal balloon occlusion between 29 + 0 and 32 + 4 weeks of gestation. Delayed occlusion was chosen in order to minimize potentially negative pulmonary effects from premature delivery as a result of fetal surgery. In addition, we wanted to become able to provide all available postnatal intensive care treatment means in these patients. RESULTS: Six of the 8 fetuses survived to discharge from hospital. CONCLUSION: Delayed fetoscopic tracheal balloon occlusion may be rewarded with lung growth sufficient to allow survival of fetuses with life-threatening congenital diaphragmatic hernia.