Stability of serial platelet and urine protein measurements in patients receiving nusinersen for spinal muscular atrophy.

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. METHODS: 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. RESULTS: Mean patient age was 28.9 years (range, 2-76 years). Mean platelet count was 307 × 109 /L (range, 96-755 × 109 /L; normal lab limits, 150-450 × 109 /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. DISCUSSION: 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.

Clinical Characteristics of a Case Series of Ocular Syphilis With 3 Cases Requiring Multiple Vitrectomies After Late Diagnosis.

Purpose: This work describes the characteristics and unique features of ocular syphilis. Methods: Ten serologically proven cases of ocular syphilis were retrospectively analyzed. Results: Eighteen eyes of 10 patients were affected. Nine of 10 patients were male and the mean age was 58 years (range, 36-81 years). HIV antibody testing was positive in 3 patients (30%). Five cases were first diagnosed by an ophthalmologist. One patient presented with a syphilitic rash. The most common ocular findings were panuveitis (n = 6) and cystoid macular edema (n = 4). Ocular involvement was unilateral in 2 cases and bilateral in 8. Best-corrected visual acuity improved in 13 of 18 eyes (72%) after treatment. Three cases developed recurrent retinal detachments that required repair with silicone oil. Conclusions: Most cases were HIV negative. Syphilitic uveitis can be the initial presentation of syphilis without classic systemic manifestation. Ophthalmologists play an important role in the diagnosis and treatment of syphilis.