NUTRITION SUPPLEMENTATION STUDIES IN SICKLE CELL DISEASE

几十年来，人们已经认识到儿童患有病态细胞病(SCD)，特别是那些患有HbSS的儿童 SCD-SS(SCD-SS)，生长发育不良，成熟延迟。此外，儿童常有$CD经历 与SCD相关的疼痛和发热发作，也有感染。Increasednutrientrequirementsand/orpoornui/；iJonalstatus 在患有SCD的儿童中有记录表明，长期缺乏营养可能会导致不良的健康结果， 生长衰退和发育迟缓。儿童血清中维生素A和维生素水平较低已有文献记载 SCD提示膳食中微量营养素的摄入量可能不太好，以适应增加的营养 患有SCD的儿童的需要。这项拟议的研究包括两个项目，以确定是否补充维生素 维生素B6可以改善SCD-SS儿童的健康状况、营养状况、生长发育和血液学状况。 我们对患有SCD的青春期前儿童的初步数据显示，66%的儿童处于亚最佳维生素A状态(血清视黄醇和维生素C； 30pg/dL)，且状态不佳的受试者具有更多的激发频率，从而减少了体质量 与维生素A状态正常的患者相比，患者的血液学状态更差。此外，儿童的初步研究 SCD检测结果表明，次优维生素水平(血清5‘-磷酸吡哆醇)为20nmol/L。 也很普遍(77%)，而且低血清浓度与营养状况差有关，这表明减少 体重指数、体重和mid-armcircumferenceandalsowithpoorerhematologicstatus.第一项研究是 随机安慰剂对照临床试验确定当前建议的维生素补充剂的效果 饮食津贴(RDA)：住院次数、与SCD相关和非与SCD相关的人数和时间 疾病事件，对生长、身体成分、血液学状态、视杆细胞完整性和免疫状态的影响 儿童(2.0岁至9.9岁)患有SCD。75名SCD儿童将接受维生素A状态筛查，44名儿童将接受筛查 接受血清视黄素水平和30pg/d筛查的受试者将被要求每天接受维生素的RDA 或安慰剂治疗12个月。第二项研究将探讨维生素B对生长发育的影响。 儿童和青少年(6.0~17.9岁)SCD疾病严重程度的营养状况和血液学指标 年)，患有SCD。共有100名患有SCD的儿童和青少年和100名健康对照，年龄、性别和 种族背景，将进行筛选，以记录患有SCD的儿童中维生素缺乏的流行情况。四十 PLP水平为20nmol/l的儿童将被识别并随机接受每天5毫克或25毫克/天的治疗 维他命12个月前。对于这两项补充研究，将在基线、3、6、9和12个月进行评估。 这两个项目对维生素和维生素改善健康的理论进行了系统的研究 SCD儿童的结局、生长发育和营养状况，并将潜在地导致成本效益营养 能够迅速落实到儿童护理标准的干预措施。

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.