Department of Pediatrics, University of Minnesota Medical School, Minneapolis, Minnesota.
Department of Pediatrics, University of Minnesota Medical School, Minneapolis, Minnesota.
Biol Blood Marrow Transplant. 2018 Jun;24(6):1289-1293. doi: 10.1016/j.bbmt.2018.01.028. Epub 2018 Feb 1.
Hematopoietic cell transplantation is a life-saving procedure, but one associated with increasing long-term cardiovascular risk requiring frequent long-term follow-up. This therapy has significantly lengthened survival in mucopolysaccharidosis type IH (Hurler syndrome), a disease with known coronary artery involvement. Metabolic syndrome-a constellation of central obesity, high blood pressure, low high-density lipoprotein cholesterol, elevated triglycerides, and fasting blood glucose-is associated with increased cardiovascular risk, and occurs when any 3 or more of these 5 components is present within a single individual. The incidence of metabolic syndrome and its components is poorly defined after transplantation for Hurler syndrome. Chart review of all long-term survivors of hematopoietic cell transplantation for Hurler syndrome ≥9 years of age for factors comprising the metabolic syndrome: obesity, high blood pressure, low high-density lipoprotein cholesterol, elevated triglycerides, and fasting blood glucose. Sixty-three patients were evaluated, 20 of whom had components of the metabolic syndrome available for review. There was no significant difference in age at transplantation, sex, number of transplants, pretransplant radiation, or percent engraftment between those with and without these data. Median follow-up after transplantation for the 20 patients with data was 14.3 years. Only 1 (5%) patient of this group fulfilled the criteria for metabolic syndrome. Fifty-three percent of the patients had 1 or more components of metabolic syndrome: the most common was high blood pressure occurring in 40%. Metabolic syndrome is uncommon in this cohort of long-term survivors of hematopoietic cell transplantation for Hurler syndrome but almost half of the patients had 1 or more components of the syndrome, with high blood pressure being the most common. Further studies are needed to develop guidelines in this diagnosis as well as other nonmalignant diseases of children.
造血细胞移植是一种救命的程序，但它也与不断增加的长期心血管风险相关，需要频繁的长期随访。这种治疗方法显著延长了黏多糖贮积症 IH 型（Hurler 综合征）患者的生存时间，该疾病已知存在冠状动脉受累。代谢综合征是一种以中心性肥胖、高血压、低高密度脂蛋白胆固醇、高甘油三酯和空腹血糖为特征的综合征，当个体存在其中任何 3 种或以上情况时，即可诊断为代谢综合征，其与心血管风险增加相关。在 Hurler 综合征患者接受造血细胞移植后，代谢综合征及其组成部分的发病率尚不清楚。对所有年龄≥9 岁且接受过造血细胞移植的 Hurler 综合征长期幸存者的代谢综合征相关因素（肥胖、高血压、低高密度脂蛋白胆固醇、高甘油三酯和空腹血糖）进行图表回顾。共评估了 63 例患者，其中 20 例患者有代谢综合征组成部分的数据可供审查。有这些数据和没有这些数据的患者在移植年龄、性别、移植次数、移植前放疗和嵌合度方面无显著差异。有数据的 20 例患者在移植后的中位随访时间为 14.3 年。该组仅 1 例（5%）患者符合代谢综合征的标准。53%的患者有 1 种或多种代谢综合征成分：最常见的是高血压，发生率为 40%。代谢综合征在造血细胞移植后长期生存的 Hurler 综合征患者中并不常见，但几乎一半的患者有 1 种或多种综合征成分，其中高血压最常见。需要进一步的研究来制定该诊断以及其他儿童非恶性疾病的指南。
