NUTRITION SUPPLEMENTATION STUDIES IN SICKLE CELL DISEASE

镰状细胞病的营养补充研究

• 批准号: 7527258
• 负责人: VIRGINIA A. STALLINGS
• 金额: $ 24.88万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2003
• 资助国家: 美国
• 起止时间: 2003-07-22 至 2008-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7527258
• 关键词: adolescence (12-20); clinical trials; cooperative study; developmental nutrition; dietary supplements; human subject; human therapy evaluation; middle childhood (6-11); nutrition related tag; patient oriented research; preschool child (1-5); retinoids; sickle cell anemia; vitamin B6; vitamin therapy

For severaldecadesit has been recognizedthat childrenwithsicklecell disease(SCD), especiallythosewithHbSS genotypeSCD (SCD-SS), have poorgrowthanddelayed maturation.In addition,childrenwith$CD experiencefrequent SCD-reiated painandfever episodesand alsoinfection. Increasednutrientrequirementsand/orpoornui/;iJonalstatus have been documentedin childrenwithSCD suggestingthat chronicundemutritionmaycontributeto poorhealthoutcome, growthfailureand delayeddevelopment.Low serumlevelsof vitaminAand vitaminBehavebeen documentedinchildren withSCD suggestingthatdietary intakeof thesemicronutribentsmay notbe adequ._ tomeet the increasednutrient needsof childrenwith SCD. The proposedstudyconsistsof twoprojectsto determinewhether supplementationof vitamin Aor vitaminB6,,can improvehealth outcomes,nutritionalstatus,growthand hematologicstatus in childrenwithSCD-SS. Our preliminarydata fromprepubertalchildrenwithSCD showthat66% havesuboptimalvitaminA status(serumretinol< 30 pg/dL),and that thosechildrenwithsuboptimalstatushavemore frequenthospitalizations, have reducedbodymass index,and poorerhematologicstatusthan those withnormalvitaminA status. Furthermore,preliminarystudiesinchildren andadolescentswithSCD showthat suboptimalvitamin Bestatus(serumpyridoxal5'-phosphate[PLP]< 20 nmol/L)is alsoprevalent(77%), andthat low serumBeconcenltationis associatedwithpoornutritionalstatus as indicatedbyreduced bodymassindex,weightand mid-armcircumferenceandalsowithpoorerhematologicstatus. The firststudyis a randomizedplacebo-controlclinicaltrialto determinethe effectof vitaminA supplementationat thecurrentRecommended DietaryAllowance(RDA) on numberof hospitalizations,the numberand lengthof both SCD-related and nonSCD-related diseaseevents, on growth,bodycomposition,hematologicstatus,rod cell integrity,and immunestatus in prepubedal children(ages 2.0 to 9.9 years) withSCD. Seventy-five childrenwithSCD will be screenedforvitaminA status and44 subjectswith serumretinollevels< 30 pg/dLat screeningwillberandomizedto receiveeitherthe RDA for vitaminA daily or placebofor 12 months. The secondstudywill explore the effectof vitaminB, supplementationat twodoseson growth and nutritionalstatus and hematologicalindicesofSCD diseaseseverityinchildrenand adolescents (ages 6.0 to 17.9 years) with SCD. A total of 100 childrenand adolescentswithSCD and 100 healthycontrols,similarinage, genderand ethnicbackground,will bescreenedto documentthe prevalenceof vitaminBedeficiencyinchildrenwithSCD. Forty childrenwithserumPLP levels< 20 nmol/1,will be identifiedandrandomizedto receive either5 mg/day or25 rag/day vitaminBefor 12 months. Forboth supplementationstudies,assessmentswill be madeat baseline,3, 6, 9 and 12 months. These two projectswillprovidesystematicinvestigationsof theroles of both vitaminA and vitaminBeinimprovinghealth outcomes,growthand nutritionalstatus inchildrenwithSCD andwill potentiallyresultin cost-effectivenutritional interventionsthat can bequicklyimplementedintothe standardof care for thesechildren.

几十年来，镰状细胞病（SCD）儿童，尤其是HbSS儿童， 基因型SCD（SCD-SS），生长发育不良，成熟延迟。此外，患有$CD的儿童经常 SCD相关的疼痛和发热发作以及感染。营养需求增加和/或营养不良; 在患有SCD的儿童中已经证实，慢性营养不良可能导致不良的健康结果， 生长障碍和发育迟缓。儿童血清维生素A和维生素B水平低 SCD认为这些微量营养素的膳食摄入量可能不足。以满足日益增长的营养需求 SCD儿童的需求。建议的研究包括两个项目，以确定是否维生素 A或维生素B6可改善SCD-SS患儿的健康结局、营养状况、生长和血液学状况。 我们对青春期前SCD儿童的研究数据显示，66%的儿童维生素A水平低于最佳水平（血清视黄醇< 30 pg/dL），而那些处于次优状态的儿童更频繁地出现癫痫， 指数，血液学状态比维生素A状态正常者差。此外，对儿童的研究 用SCD的分析表明，次优维生素Bestatus（血清吡哆醛5 '-磷酸[PLP]< 20 nmol/L） 也普遍存在（77%），低血清Beconcentration与营养不良有关， 体重指数、体重和上臂围也较差。第一项研究是 随机安慰剂对照临床试验，以确定目前维生素A补充的效果推荐 根据住院人数、SCD相关和非SCD相关的人数和时间长度提供饮食津贴（RDA） 疾病事件对耻骨前肌生长、身体组成、血液学状态、视杆细胞完整性和免疫状态的影响 SCD儿童（年龄2.0 - 9.9岁）。75名患有SCD的儿童将接受维生素A状态筛查， 筛选时血清视黄醇水平< 30 pg/dL的受试者将随机接受每日维生素A RDA 或安慰剂12个月。第二项研究将探讨两种剂量的维生素B对生长的影响 儿童和青少年（年龄6.0 - 17.9岁）SCD疾病严重性的营养状况和血液学指标 年），与SCD。共100名SCD患儿和100名健康对照者，年龄、性别和 种族背景，将被筛选以记录SCD儿童维生素B缺乏的患病率。四十 将确定血清PLP水平< 20 nmol/l的儿童，并随机接受5 mg/天或25 mg/天的治疗 维生素12个月前。两项研究均在基线、3、6、9和12个月进行评估。 这两个项目将为维生素A和维生素B在改善健康中的作用提供系统的研究 SCD儿童的结局、生长和营养状况，并可能导致成本效益高的营养 可以快速实施的干预措施，以达到这些儿童的护理标准。