Reduction of Optic Disc Oedema by Bortezomib and Dexamethasone Followed by Autologous Peripheral Blood Stem Cell Transplantation in Patient with POEMS Syndrome

et al. 2020

Orphanet J Rare Dis

Background Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes (POEMS) syndrome is a rare paraneoplastic syndrome involving multisystem. Optic disc edema (ODE) is the most common ocular manifestation in patients with POEMS syndrome and serves as an independent prognostic factor. However, parameters previously used to estimate its severity were inconvenient and costly. This study was designed to bring forward a novel and practical parameter, optic disc edema area, to evaluate ODE in patients with this disease and applied it to assess effectiveness of lenalidomide combined with dexamethasone in respect of ODE. Results Forty-one treatment-naive patients with POEMS syndrome were enrolled in this single-center prospective study and treated with lenalidomide combined with dexamethasone. They received ocular examination to determine optic disc edema (ODE) area and other optic manifestations. Meanwhile, serum VEGF was measured before and after treatment. Among 41 enrolled patients, 38 received complete ocular examinations, and 25 of which had ODE at initial visit. Binocular mean ODE area of patients with ODE was significantly related to ODE grade (r = 0.620, p = 0.003) and peripapillary retinal thickness (r = 0.760, p < 0.001) before treatment. Serum VEGF was significantly higher in patients with ODE than their counterparts (p = 0.025) and positively correlated with binocular mean ODE area (r = 0.460, p = 0.036). After treatment, ODE area, along with serum VEGF, decreased markedly (p < 0.001). Conclusion ODE area was a reliable index to evaluate ODE severity and could precisely reflect ODE improvement through systemic treatment. Additionally, it was related to serum VEGF, a key factor in disease pathogenesis, suggesting its potential as an indicator of the overall severity of this disease. Trial registration Clinicaltrials, NCT01816620. Registered March 222,013.

Section: Discussionmentioning

confidence: 99%

Reliability of optic disc edema area in estimating the severity of papilledema in patients with POEMS syndrome

et al. 2020

Orphanet J Rare Dis

“…Circulating VEGF in choroidal vessels is more likely to leak out , binding to abluminal VEGFR2 to improve permeability of surrounding vessels. [6,21,22] Besides, there is an area with a defective blood-retinal barrier surrounding the optic disc, called the tissue of Elschnig border, where substances in choroidal vessels could leak out even more easily. Thus, ODE in POEMS syndrome is probably the result of choroidal edema caused by high serum VEGF level.…”

Section: Ode Area and Peripapillary Retinal Thickness (Prt)mentioning

confidence: 99%

Reliability of Optic Disc Edema Area in Estimating the Severity of Papilledema in Patients with POEMS Syndrome

et al. 2020

Preprint

Background: Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes (POEMS) syndrome is a rare paraneoplastic syndrome involving multisystem. Optic disc edema (ODE) is the most common ocular manifestation in patients with POEMS syndrome and serves as an independent prognostic factor. However, parameters previously used to estimate its severity were inconvenient and costly. This study was designed to bring forward a novel and practical parameter, optic disc edema area, to evaluate ODE in patients with this disease and applied it to assess effectiveness of lenalidomide combined with dexamethasone in respect of ODE.Results: Forty-one treatment-naive patients with POEMS syndrome were enrolled in this single-center prospective study and treated with lenalidomide combined with dexamethasone. They received ocular examination to determine optic disc edema (ODE) area and other optic manifestations. Meanwhile, serum VEGF was measured before and after treatment. Among 41 enrolled patients, 38 received complete ocular examinations, and 25 of which had ODE at initial visit. Binocular mean ODE area of patients with ODE was significantly related to ODE grade (r = 0.620, p = 0.003) and peripapillary retinal thickness (r = 0.760, p < 0.001) before treatment. Serum VEGF was significantly higher in patients with ODE than their counterparts (p = 0.025) and positively correlated with binocular mean ODE area (r = 0.460, p = 0.036). After treatment, ODE area, along with serum VEGF, decreased markedly (p < 0.001).Conclusion: ODE area was a reliable index to evaluate ODE severity and could precisely reflect ODE improvement through systemic treatment. Additionally, it was related to serum VEGF, a key factor in disease pathogenesis, suggesting its potential as an indicator of the overall severity of this disease.Trial registration: Clinicaltrials, NCT01816620. Registered March 22 2013, https://clinicaltrials.gov/ct2/show/NCT01816620.

“…[20] Due to the caliber and flow volume differences between choroidal and retinal vessels, circulating VEGFA can only leak out from choroidal vessels and bind to abluminal VEGFR2 to improve permeability of surrounding vessels. [6,[20][21][22] Also, previous study reported that subfoveal choroidal thickness was positively related to serum VEGF level in POEMS syndrome. [4] These findings together suggested that circulating VEGF triggered ODE in patients with POEMS syndrome through choroid, which could only be relieved through systemic treatment.…”

Section: Ode Area and Peripheral Retinal Thicknessmentioning

confidence: 99%

Reliability of Optic Disc Edema Area in Estimating the Severity of Papilledema in Patients with POEMS Syndrome

et al. 2019

Preprint

Background Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes (POEMS) syndrome is a rare paraneoplastic syndrome involving multisystem. Optic disc edema (ODE) is the most common ocular manifestation in patients with POEMS syndrome and serves as an independent prognostic factor. However, parameters previously used to estimate its severity were inconvenient and costly. This study was designed to bring forward a novel and practical parameter, optic disc edema area, to evaluate ODE in patients with this disease and applied it to assess effectiveness of lenalidomide and dexamethasone in respect of ODE.Results Forty-one treatment-naive patients with POEMS syndrome were enrolled in this single-center prospective study and treated with lenalidomide and dexamethasone. They received ocular examination to determine optic disc edema (ODE) area and other optic manifestations. Meanwhile, serum VEGF was measured before and after treatment. Among 41 enrolled patients, 38 received complete ocular examinations, and 25 of which had ODE at initial visit. Binocular mean ODE area of patients with ODE was significantly related to ODE grade (r=0.620, p =0.003) and peripheral retinal thickness (r=0.760, p =0.000) before treatment. Serum VEGF was significantly higher in patients with ODE than their counterparts ( p =0.025) and positively correlated with binocular mean ODE area (r=0.460, p =0.036). After treatment, ODE area, along with serum VEGF, decreased markedly ( p =0.000).Conclusion ODE area was a reliable index to evaluate ODE severity and could precisely reflect ODE improvement through systemic treatment. Additionally, it was related to serum VEGF, a key factor in disease pathogenesis, which suggests its potential capability of indicating the overall severity of this disease. Trial registration: Clinicaltrials, NCT01816620. Registered March 22 2013, https://clinicaltrials.gov/ct2/show/NCT01816620.