Department of Radiology, University of Minnesota, Minneapolis, Minnesota.
Biostatistics Core, Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota.
Muscle Nerve. 2022 Jul;66(1):76-79. doi: 10.1002/mus.27564. Epub 2022 May 11.
INTRODUCTION/AIMS: Patients undergoing nusinersen treatment for spinal muscular atrophy are subject to measurements of platelet count and urine protein before each injection due to concern for platelet depletion and renal dysfunction according to the prescribing information. These tests may be uncomfortable or inconvenient and may cause delays in treatment. However, it is still unclear whether these values have been significantly affected by nusinersen treatment. Our aim in this study was to determine whether these measurements ever reached critical values that necessitated withholding treatment at our center.
Records from 57 patients treated with nusinersen at our institution between 2017 and 2020 were retrospectively analyzed. Laboratory values for platelet count, random urine protein, and total urine protein:creatinine ratio were collected from all patients before each procedure.
Mean patient age was 28.9 years (range, 2-76 years). Mean platelet count was 307 × 10 /L (range, 96-755 × 10 /L; normal lab limits, 150-450 × 10 /L), mean random urine protein was 0.164 g/L (range, <0.05-0.73 g/L), and mean total urine protein:creatinine ratio was 0.885 g per gram creatinine (range, 0.12-9.71 g per gram creatinine). No laboratory values precluded continuing treatment for any patient.
Although further study on a larger cohort is warranted for more definitive conclusions, it may not be necessary to measure platelet count and urine protein before each nusinersen treatment, particularly in the maintenance phase.
简介/目的：根据说明书，接受诺西那生钠治疗的脊髓性肌萎缩症患者，每次注射前都需要测量血小板计数和尿蛋白，以防血小板减少和肾功能障碍。这些测试可能会带来不适或不便，并可能导致治疗延迟。然而，目前尚不清楚诺西那生钠治疗是否会显著影响这些数值。我们的研究目的是确定这些测量值是否曾达到需要在我们中心停止治疗的临界值。
回顾性分析了 2017 年至 2020 年期间在我院接受诺西那生钠治疗的 57 例患者的记录。从所有患者中收集了每次治疗前血小板计数、随机尿蛋白和总尿蛋白与肌酐比值的实验室值。
患者平均年龄为 28.9 岁（范围，2-76 岁）。平均血小板计数为 307×10/L（范围，96-755×10/L；正常实验室限值，150-450×10/L），平均随机尿蛋白为 0.164g/L（范围，<0.05-0.73 g/L），平均总尿蛋白与肌酐比值为 0.885g/克肌酐（范围，0.12-9.71 g/克肌酐）。没有任何实验室值阻止任何患者继续治疗。
尽管需要进一步的大样本队列研究来得出更明确的结论，但在维持阶段，可能没有必要在每次接受诺西那生钠治疗前测量血小板计数和尿蛋白。
