Intrauterine closure of myelomeningocele: an update

2004 ◽  
Vol 16 (2) ◽  
pp. 1-4 ◽  
Author(s):  
Noel Tulipan
Keyword(s):  
Randomized Controlled Trial ◽  
Congenital Malformations ◽  
State Of The Art ◽  
Controlled Trial ◽  
Preliminary Evidence ◽  
Endoscopic Techniques ◽  
Randomized Controlled ◽  
Risks And Benefits ◽  
Myelomeningocele Repair ◽  
Intrauterine Closure

Preliminary evidence suggests that intrauterine myelomeningocele repair may benefit patients by reducing the both incidence of hydrocephalus and the severity of the Chiari malformation; however, this benefit remains unproved. Furthermore, the procedure entails substantial risks not associated with conventional therapy. A randomized controlled trial of intrauterine and conventional therapies is underway. This study should definitively establish the procedure-related risks and benefits. Regardless of the outcome, it is clear that the risks of intrauterine intervention need to be reduced before myelomeningocele, or other congenital malformations, can be effectively treated prior to birth. To that end, studies are being conducted to assess the potential advantages of applying state-of-the-art endoscopic techniques to intrauterine therapy. If benefit can be proven and risks reduced, intrauterine myelomeningocele repair has the potential to become the preferred therapy for patients suffering from this debilitating disease.

scholarly journals Fetal Myelomeningocele Closure: Technical Considerations

2014 ◽  
Vol 37 (3) ◽  
pp. 166-171 ◽  
Author(s):  
Gregory G. Heuer ◽  
N. Scott Adzick ◽  
Leslie N. Sutton
Keyword(s):  
Randomized Controlled Trial ◽  
Surgical Technique ◽  
General Surgery ◽  
Congenital Malformations ◽  
Controlled Trial ◽  
Spinal Fluid ◽  
Fetal Period ◽  
Randomized Controlled ◽  
Technical Considerations ◽  
Postnatal Closure

Myelomeningocele (MMC) is one of the most common serious congenital malformations. Typically this condition has been treated with closure of the MMC defect shortly after birth. In general, surgery for MMC aims to provide a multilayered closure to provide protection to the neural elements, prevent leakage of spinal fluid and reduce infection risks. A randomized controlled trial, the Management of Myelomeningocele Study (MOMS), has shown that closure during the fetal period can be performed relatively safely and can result in significant benefit to the child. Whereas the surgical technique of prenatal closure of an MMC defect is similar to a postnatal closure, there are some important technical differences. The goal of this paper is to describe the technique of fetal closure of MMC defects, highlight the unique steps that are needed for this surgery and delineate some potential pitfalls.

scholarly journals Disulfiram repurposing combined with nutritional copper supplement as add-on to chemotherapy in recurrent glioblastoma (DIRECT): Study protocol for a randomized controlled trial

F1000Research ◽  
2018 ◽  
Vol 7 ◽  
pp. 1797 ◽  
Author(s):  
Asgeir Store Jakola ◽  
Katja Werlenius ◽  
Munila Mudaisi ◽  
Sofia Hylin ◽  
Sara Kinhult ◽  
...  
Keyword(s):  
Randomized Controlled Trial ◽  
Anticancer Agent ◽  
Controlled Trial ◽  
Progression Free Survival ◽  
Preliminary Evidence ◽  
Recurrent Glioblastoma ◽  
Open Label ◽  
Randomized Controlled ◽  
Link Type ◽  
Methylguanine Methyltransferase

Background: Disulfiram (DSF) is a well-tolerated, inexpensive, generic drug that has been in use to treat alcoholism since the 1950s. There is now independent preclinical data that supports DSF as an anticancer agent, and experimental data suggest that copper may increase its anti-neoplastic properties. There is also some clinical evidence that DSF is a promising anticancer agent in extracranial cancers. In glioblastoma, DSF induced O6-methylguanine methyltransferase (MGMT) inhibition may increase response to alkylating chemotherapy. A recent phase I study demonstrated the safety of DSF in glioblastoma patients when DSF was administered at doses below 500 mg/day together with chemotherapy. We plan to assess the effects of DSF combined with nutritional copper supplement (DSF-Cu) as an adjuvant to alkylating chemotherapy in glioblastoma treatment. Methods: In an academic, industry independent, multicenter, open label randomized controlled phase II/III trial with parallel group design (1:1) we will assess the efficacy and safety of DSF-Cu in glioblastoma treatment. The study will include 142 patients at the time of first recurrence of glioblastoma where salvage therapy with alkylating chemotherapy is planned. Patients will be randomized to treatment with or without DSF-Cu. Primary end-point is survival at 6 months. Secondary end-points are overall survival, progression free survival, quality of life, contrast enhancing tumor volume and safety. Discussion: There is a need to improve the treatment of recurrent glioblastoma. Results from this randomized controlled trial with DSF-Cu in glioblastoma will serve as preliminary evidence of the future role of DSF-Cu in glioblastoma treatment and a basis for design and power estimations of future studies. In this publication we provide rationale for our choices and discuss methodological issues. Trial registration: The study underwent registration in EudraCT 2016-000167-16 (Date: 30.03.2016,) and Clinicaltrials.gov NCT02678975 (Date: 31.01.2016) before initiating the study.

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