ABSTRACT
PURPOSE: To evaluate the initial treatment response to low doses of prednisolone, compared with moderate doses, in ocular myasthenia gravis (OMG). PATIENTS AND METHODS: A retrospective chart review of patients with adult-onset (age ≥15 years old) OMG, who were treated with prednisolone, was conducted. Subjects were divided into two groups according to their prednisolone dosing regimen. The low-dose group was defined as those with an average 12-week cumulative dose of prednisolone <0.435 mg/kg/day and the moderate-dose group averaged 0.435-1.000 mg/kg/day. The primary outcome of interest was the comparison of clinical response to prednisolone at 12 weeks between the low-dose and moderate-dose groups. The secondary outcome was the difference in adverse events between treatment groups. RESULTS: Of 34 subjects, 16 subjects (47.1%) were male. The mean age at onset was 44.0±18.1 years. The most common presenting ocular feature was ptosis with ophthalmoplegia (22 subjects, 64.7%), followed by isolated ptosis (nine subjects, 26.5%) and isolated ophthalmoplegia (three subjects, 8.8%). Half of the subjects were treated with low-dose prednisolone and the other half were treated with moderate-dose prednisolone. There were no substantial differences in baseline characteristics between treatment groups. After 12 weeks of treatment, nine of 17 subjects (52.9%) and 13 of 17 subjects (76.5%) in the low- and moderate-dose groups, respectively, were regarded as responsive to the prednisolone treatment (P=0.28). Adverse events were exclusively observed in the moderate-dose group. CONCLUSION: Treatment of OMG with an average 12-week cumulative dose of prednisolone <0.435 mg/kg/day (low dose) shows a comparable responsive outcome to 0.435-1.000 mg/kg/day of prednisolone (moderate dose). Treating OMG with low-dose prednisolone can minimize prednisolone-related adverse events. However, a prospective randomized controlled trial with a larger study population is warranted in order to gain more insight into the proper dosage of prednisolone for OMG.
ABSTRACT
BACKGROUND: POEMS syndrome is a plasma cell disorder, which clinically manifests from paraneoplastic syndrome: polyneuropathy, organomegaly, endocrinopathy, monoclonal plasma cell disorder, and skin changes. The most common ocular manifestation is optic disc swelling, whereas other ocular manifestations; cystoid macular edema, serous macular detachment, venous sinus thrombosis, infiltrative orbitopathy, uveitis, neovascularization of the disc, peripapillary choroidal neovascularization and optic disc drusen, had also been reported. CASE PRESENTATION: A 52-year-old Thai man presented with 5-day sudden painless visual loss in the left eye. Ocular examination revealed visual acuity of 20/20 and no light perception in the right and left eye, respectively. Right fundoscopic examination was significant for hyperemic generalized optic disc swelling. Left fundoscopic examination revealed opaque and edematous entire retina giving the appearance of central retinal artery occlusion (CRAO) along with pallid "chalky white" optic disc swelling. Fluorescein angiography showed profound leakage of bilateral optic nerve heads and arteriolar filling defect in macular area along with leakage of small retinal arterioles in the left eye. Indocyanine green angiography demonstrated choroidal filling defect in the left eye only. Neuroimaging showed enhancement and luminal narrowing of left internal carotid artery, early subacute watershed infarctions in the left cerebral hemisphere and pachymeningeal enhancement. Cerebrospinal fluid analysis revealed high protein level with normal opening pressure. Intravenous methylprednisolone was initially started without any benefit. After extensive investigations, diagnosis of "POEMS syndrome" was made based on polyneuropathy, elevated lambda light chain level, elevated plasma vascular endothelial growth factor (VEGF), hepatomegaly, spinal sclerotic bone lesions, and thrombocytosis. Furthermore, sural nerve biopsy demonstrated neuropathy and positive VEGF staining. He was treated with eight cycles of bortezomib, cyclophosphamide and dexamethasone (BorCyDex). Polyneuropathy and thrombocytosis had remarkably improved after 2nd cycle, whereas, visual impairment had shown no recovery. Hepatomegaly was significantly reduced after the completion of BorCyDex. Our case eventually received autologous hematopoietic stem cell transplantation with high dose melphalan. CONCLUSIONS: To our knowledge, we illustrated the first patient given CRAO as the first presentation and ocular finding ever reported in POEMS syndrome. Both cerebral and ocular infarctions were presumably the result of VEGF-induced cranial vasculopathy as evidenced by neuroimaging.
