Nusinersen treatment in patients with spinal muscular atrophy: A safety analysis of laboratory parameters / 西安交通大学学报(医学版)

ABSTRACT

【Objective】 To explore the changes of laboratory parameters and safety of nusinersen in the treatment of spinal muscular atrophy (SMA). 【Methods】 Retrospective analysis was made on the six SMA patients treated with nusinersen in the Department of Neurology at The First Affiliated Hospital of Xi’an Jiaotong University from December 2021 to December 2022. We summarized the patients’ clinical data, including genetic diagnosis results, disease classification, and clinical manifestations. Intrathecal injection of 5 mL/12 mg of nusinersen administered on the 1st, 14th, 28th, and 63rd days, followed by maintenance treatment every 4 months. After each administration, we compared and evaluated the patients’ cerebrospinal fluid, blood routine, liver function, kidney function, and coagulation function with baseline values. We regularly followed up the patients and recorded adverse reactions after administration to evaluate medication safety. 【Results】 A total of six patients were diagnosed with SMA, including four cases of SMA3 type and two cases of SMA4 type. The deletion of exon 7 of the survival motor neuron gene 1 (SMN1) in patients led to changes in motor neuron, most of which were caused by limb weakness; in severe cases, the patients were unable to stand. The baseline abnormal test indicators of patients included the increase of cerebrospinal fluid lactate dehydrogenase (CSF-LDH), the increase of creatine kinase (CK), and the decrease of creatinine (Cr). After four times of treatment, the patients’ CSF-WBC, CSF-Glu, CSF-Pro, CSF-LDH, WBC, PLT, RBC, ALT, AST, GGT, BUN, uric acid (UA), INR, aPTT, and D-dimer had no significant difference from the baseline (P>0 05). The patients had no other significant adverse reactions except headaches, dizziness, and back pain after puncture. 【Conclusion】 Nusinersen has good safety on SMA patients.