Association of Rapidly Progressive Moyamoya Syndrome With Bevacizumab Treatment for Glioblastoma in a Child With Neurofibromatosis Type 1

Department of Neurology, Children's Hospital Boston, Boston, MA 02446, USA.
Journal of child neurology (Impact Factor: 1.67). 02/2011; 26(2):228-30. DOI: 10.1177/0883073810379639
Source: PubMed

ABSTRACT Neurofibromatosis type 1 is a common multisystemic disorder that can result in tumors of the central and peripheral nervous system. Individuals with neurofibromatosis type 1 are also at increased risk to develop moyamoya syndrome, which is a cerebrovascular condition that predisposes affected individuals to develop strokes as a result of progressive narrowing of the intracranial internal carotid arteries and failure of adequate blood supply through collateral vessels. We report a case of a young boy with neurofibromatosis type 1 with glioblastoma who developed rapidly progressive moyamoya vasculopathy after treatment with the angiogenesis inhibitor bevacizumab.

  • [Show abstract] [Hide abstract]
    ABSTRACT: Astrocytic tumors, especially optic pathway pilocytic astrocytomas, are common in pediatric NF1 patients. High-grade gliomas (HGGs) appear to be rare in adult and pediatric NF1 patients. This is a series of five consecutive, adult NF1 patients with recurrent HGGs treated at The University of Texas MD Anderson Cancer Center. Four patients met consensus clinical criteria for NF1 and one patient had presumed segmental NF1. Three patients had glioblastomas, one gliosarcoma, and one progressive, enhancing optic pathway glioma which was not biopsied. Two tumors had molecular testing performed; both were IDH wild type and activating oncogene mutations (1 BRAFV600E and 1 PIK3CA mutation) were found in these tumors. All five patients received bevacizumab-containing regimens at tumor recurrence. The median number of 4-week cycles of bevacizumab was 20. All five patients experienced prolonged post-recurrence survival following bevacizumab treatment ranging from ten to 72 months. The median overall survival from HGG diagnosis was 72.6 months with three patients alive and progression free at last follow-up. Three out of five patients developed vascular complications leading to bevacizumab discontinuation. In this case series, adult NF1 patients with recurrent HGGs had prolonged, post-recurrence survival after treatment with bevacizumab-containing regimens. Based on these results, further study of antiangiogenic therapy in NF1 patients with HGGs and bevacizumab-response in sporadic HGG patients with NF1-mutated tumors is warranted.
    Journal of Neurology 05/2014; 261(8). DOI:10.1007/s00415-014-7292-0 · 3.84 Impact Factor
  • Source
    [Show abstract] [Hide abstract]
    ABSTRACT: Neurofibromatosis type 1 (NF1) is the most prevalent autosomal dominant genetic disorder among humans. NF1 vasculopathy is a significant but underrecognized complication of the disease, affecting both arterial and venous blood vessels of all sizes. Moyamoya syndrome is a cerebral vasculopathy that is only rarely observed in association with NF1, particularly in the pediatric age range. Herein, we report of a 5-year-old female with NF1 and moyamoya syndrome and we briefly review the existing literature.
    Italian Journal of Pediatrics 06/2014; 40(1):59. DOI:10.1186/1824-7288-40-59
  • [Show abstract] [Hide abstract]
    ABSTRACT: OBJECTIVE: To determine whether vascular and other complications are more common in pregnant women with neurofibromatosis type 1 (NF1). STUDY DESIGN: We performed a population-based retrospective cohort study using the US Nationwide Inpatient Sample (NIS), 1988-2009, defining a cohort of pregnancy-related hospitalizations with an associated diagnosis of NF1 and comparing it to the control group not associated with NF1. Multivariable logistic regression was used to adjust for suspected confounders. RESULTS: Among 19 million pregnancy-related admissions between 1988 and 2009, we identified 1,553 associated with NF1 (prevalence 0.008%). A diagnosis of NF1 in delivering mothers was associated with gestational hypertension (adjusted odds ratio [AOR]: 1.6, 95% confidence interval [CI]: 1.2-2.0), pre-eclampsia (AOR: 2.8, CI: 2.3-3.4), IUGR (AOR: 4.6, CI: 3.7-5.6), cerebrovascular disease (OR: 8.1, CI: 2.6-25.4), preterm labor (AOR: 1.6, CI: 1.4-1.9), and cesarean delivery (AOR: 2.0, CI: 1.8-2.3). Women with NF1 were not significantly more likely to have DVT/PE, acute cardiac events, stillbirth, or to die during their hospitalizations compared to the general obstetric population. CONCLUSION: NF1 was associated with increased maternal morbidity in pregnancy (including hypertensive and cerebrovascular complications), but not increased maternal mortality. Obstetricians should be aware of the potential for increased antenatal and peripartum complications among women with NF1.
    American journal of obstetrics and gynecology 03/2013; 209(1). DOI:10.1016/j.ajog.2013.03.029 · 3.97 Impact Factor