Utility of [F-18] fluoroDOPA for neonatal hyperinsulism: A Phase II

描述(由申请人提供)： 先天性高胰岛素血症(CHI)是一种罕见的疾病，在美国大约每500,000名婴儿中就有1名受到影响。据估计，总患病率不到20万人。这种疾病的治疗需要手术切除胰腺以控制高血糖。有局灶性病变的婴儿有可能通过局部切除治愈。目前还没有可用的方法来在手术前识别这些局灶性病变。 申请人所在机构正在进行的一项初步研究表明，18F标记的L-氟二羟基苯丙氨酸(F-DOPA)的正电子发射断层扫描(PET)对确定和定位局灶性病变是有用的。F-DOPA成像将使高胰岛素血症儿童选择手术入路成为可能。申请人的提案调查了F-DOPA PET作为一种非侵入性工具的实用性，以指导胰腺切除(全切除与病变切除)伴局灶性先天性高胰岛素血症的活检和性质。建议这项研究将有助于批准F-DOPA PET用于先天性高胰岛素血症儿童的影像引导活检和胰腺切除术，从而改善这种孤儿疾病的预后。

DESCRIPTION (provided by applicant): Congenital hyperinsulinism (CHI) is a rare condition that affects approximately 1 in 500,000 infants in the United States. The total prevalence has been estimated to be less than 200,000. Treatment of this disease necessitates surgical removal of the pancreas to control hyperglycemia. Infants with focal lesions are potentially curable by local resection. There are no currently available methods for identifying these focal lesions prior to surgery. A preliminary study being conducted at the applicant's institution suggests that positron emission tomography (PET) with 18F-labeled L-fluoro-dihydroxyphenylalanine (F-DOPA) is useful for identifying and localizing focal lesions. Imaging with F-DOPA will enable selection of surgical approach in children with hyperinsulinism. The applicant's proposal investigates the utility of F-DOPA PET as a noninvasive tool to guide biopsy and nature of pancreatectomy (total versus lesion resection) with focal forms of congenital hyperinsulinism. It is proposed that this study will contribute to the approval of F-DOPA PET for use in image-guided biopsy and pancreatectomy of children with congenital hyperinsulinism, thereby improving the outcome for children with this orphan disease.