RARE DISEASE
Coats disease
Coats disease
Coats disease
Synonyms: Congenital retinal telangiectasia, Leber miliary aneurysm
Synonyms: Congenital retinal telangiectasia, Leber miliary aneurysm
Synonyms: Congenital retinal telangiectasia, Leber miliary aneurysm
Drug discovery
2
drugs
With orphan designations
Overview
Coats Disease Overview
Coats disease is a rare, idiopathic retinal vascular disorder characterized by telangiectasia, aneurysmal dilations, and exudative retinal detachment, typically presenting unilaterally in young males. Onset is often before age 10, with symptoms including leukocoria, strabismus, and vision loss. Progressive exudation can lead to complications like glaucoma and cataracts. Management includes laser photocoagulation, cryotherapy, and surgery for advanced cases, with adjunctive anti-VEGF therapies under investigation [1][2][4][7].
Categories: rare developmental anomalies during embryogenesis, rare genetic diseases, rare neurological diseases, rare ophthalmic disorders
Research Papers
270 drug discovery papers about Coats disease, with 4 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
270 drug discovery papers about Coats disease, with 4 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
categories:
Small molecules
small molecules
2026-06-09 | Long-Term Outcomes of Two-port Non-vitrectomy Endolaser Therapy in Moderate-to-severe Stage 3A Coats Disease.
To report the long-term outcomes of two-port non-vitrectomy endolaser therapy (TPNVE) in moderate-to-severe stage 3A Coats disease. In this retrospective observational case series, patients with moderate-to-severe stage 3A Coats disease who were treated with TPNVE in a single tertiary referral vitreoretinal practice between March 2014 and May 2024 were included. The anatomic success rate, number of treatment sessions, and complications were analyzed. Of the 46 patients (47 eyes) included in the study, 33 (70.2%) eyes were stage 3A1, and the rest were stage 3A2. The mean (median, range) extent of ERD was 6.1 (5.0, 4.0-12.0) clock hours. The median (range) follow-up time was 67.7 (13.9-135.4) months. All 47 eyes achieved anatomic success with a median (range) treatment session of 2.0 (1-6) sessions. The median number of TPNVE treatments and conventional laser photocoagulation treatments was 1.2 (range, 1-2) and 0.0 (range, 0-4), respectively. Twenty (42.5%) eyes required only one session. There were 47 instances of recurrence in 27 eyes, of which over half (59.5%) took place in the first 6 months. Complications included cataracts in 11 (23.4%), vitreoretinal fibrosis in 11 (23.4%), subretinal fibrosis in 11 (23.4%), tractional retinal detachment in one (2.1%), and glaucoma in one (2.1%) of the eyes. TPNVE demonstrated efficacy in ablating abnormal vessels and reattaching the retina for moderate-to-severe stage 3A Coats disease. Approximately half of the eyes with an ERD extent of at least 4 clock hours achieved anatomical success after a single TPNVE surgery.
2026-06-09 | Management of a patient with late-onset Coats’ disease
Coats’ disease is an idiopathic condition characterized by the formation of retinal telangiectasias, as well as micro- and macroaneurysms, accompanied by intraretinal and subretinal exudation. Due to the inconspicuous progression of the early stages, diagnosis is often significantly delayed. This necessitates the use of multimodal imaging as a key tool for the timely detection of the pathology. Currently, there are no standardized guidelines for the treatment of Coats' disease, which may be attributed to its relatively low prevalence in the general population. We report a case involving a 49-year-old patient diagnosed with Stage 2 Coats’ disease (according to the classification by J. Shields et al., 2000). The management strategy selected was a multi-stage laser treatment based on a topographically oriented approach. This clinical case illustrates the potential of multimodal imaging in the early diagnosis of exudative-hemorrhagic retinitis, the determination of management tactics, the execution of topographically oriented treatment, and postoperative monitoring. Keywords: Coats' retinitis; laser coagulation; optical coherence tomography; multimodal imaging; fluorescence angiography; clinical case
2026-01-19 | A Case of Coats Disease Diagnosed during Amblyopia Treatment.
Coats disease is a rare, nonhereditary retinal disorder, and early detection and treatment are crucial for preserving visual function. This case report details a pediatric case where Coats disease was diagnosed during follow-up for amblyopia. An 8-year-old boy with a history of bilateral hyperopic amblyopia and right congenital ptosis underwent eyeglass and occlusion therapy starting at age 2. By age 4, he had achieved good visual acuity. At a routine follow-up 4 years later, fundus examination revealed exudative lesions in the left macula. Optical coherence tomography (OCT) showed macular hyperreflective lesions and the thickened choroid in his left eye, leading to a diagnosis of Coats disease. The patient was treated with retinal photocoagulation. Ten months after treatment, his visual acuity remained stable, and a follow-up OCT showed a reduction in the hyperreflective lesions in the outer macular layers. Routine dilated fundus examinations and OCT-based retro-choroidal morphology for anisometropic amblyopia facilitated early detection and timely intervention for Coats disease, which lacks overt symptoms in its early stages. This case highlights the importance of retinal monitoring in amblyopic eyes to preserve visual function.
2025-10-09 | Mapping Macrophage Diversity in Coats' Disease: A Lipid-Associated Subset in the Pathogenesis of Exudative Retinopathy.
Coats' disease is a retinal vasculopathy characterized by retinal telangiectasia, exudation, and retinal detachment. Lipid exudates serve as crucial biomarkers for the assessment and prediction of disease progression. However, the pathophysiological mechanisms driving exudation remain inadequately understood. This study aimed to investigate the cellular and molecular mechanisms underlying the pathogenesis of Coats' disease. Single-cell suspensions of epiretinal membrane and vitreous samples were collected from a patient with Coats' disease, and corresponding vitreous and epiretinal membrane samples from four patients with proliferative vitreoretinopathy were used as controls. The samples were analyzed by single-cell RNA sequencing using Seurat, Slingshot, hdWGCNA, and scFEA in R/Python software. In this study, we present the first single-cell atlas of the retinal microenvironment in Coats' disease, derived from clinical samples of epiretinal membrane and vitreous cells. Our analysis identified a predominance of macrophages, with a dominant cluster exhibiting a transcriptional profile characteristic of lipid-associated macrophages (LAMs). Single-cell trajectory and metabolic analysis revealed a gradual decrease in lipid oxidative catabolism in LAMs, with a progressive shift in biological function from phagocytosis to inflammation and extracellular matrix remodeling. Furthermore, we identified that the transition of LAMs was regulated by a gene module centered around ABCA1, a key regulator in the progression of atherosclerosis. Single-cell drug response analysis further infers that an antiatherosclerotic regimen may elicit a superior response in LAMs compared with conventional treatments. Collectively, our characterization of LAM phenotypes may provide novel insights into the pathogenesis of Coats' disease and identify potential targets for disease-specific interventions.
2025-09-25 | Coats Disease With Macular Nodule.
To describe a case series of Coats disease with macular nodule mimicking retinoblastoma. Retrospective case series. Of 339 patients with Coats disease, 7 (2%) with macular nodules were included. All had a referral diagnosis of retinoblastoma. All were males (mean age-6 y). The most common presenting complaint was decreased vision (n=4, 57%). Median best corrected visual acuity at presentation was counting fingers close to face, which was maintained at last follow-up (mean, 8 months). The nodule had a mean diameter of 5x4x3 mm, was grayish white, and bilobed (n=3, 43%), or dome shaped (n=4, 57%) with surface pigmentation (n=4, 57%), involving fovea (n=5, 71%), with surrounding exudates and second- or third-order retinal vessels. OCT (n=4) showed a well-defined hyperreflective subretinal nodule with posterior shadowing, cysts, and exudates. USG (n=6) showed an echodense intraocular nodule with moderate internal reflectivity with hyperechoic foci in 2 (33%) cases. FFA showed early- and mid-phase hypo-fluorescence with focal leakage with late-phase hyperfluorescence. Focal green laser photocoagulation of the telangiectatic vessels was performed in 5, one underwent subretinal fluid drainage and scleral buckling, and one was observed. Macular nodule in Coats disease represents a preferential accumulation of lipid in the macula. This rare variant of Coats disease can mimic retinoblastoma process. Be aware of this entity to ensure appropriate diagnosis and treatment.
antibodies
2026-04-28 | Adult-onset Coats disease: A systematic review and meta-analysis of imaging biomarkers and targeted therapies.
Coats disease diagnosed in adulthood is a rare idiopathic retinal telangiectatic vasculopathy that may differ in phenotype from childhood-diagnosed cases. This systematic review synthesizes current evidence to characterize its clinical spectrum, imaging features, treatment outcomes and underlying mechanisms, with a focus on immunovascular pathways. Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA 2020) guidelines, PubMed, Scopus and Web of Science were searched through October 2025 for studies including patients ≥18 years with confirmed Coats disease. Clinical and imaging findings, treatment strategies and outcomes were extracted and narratively synthesized. When possible, random-effects meta-analysis was performed. Thirty-five studies comprising 240 patients (250 eyes) were included. Disease presentation was predominantly unilateral and diagnosed at Shields Stage 2 or 3. Laser photocoagulation and anti-vascular endothelial growth factor (VEGF) agents were the most frequently described therapies, with anatomical stabilization or regression of exudation reported in approximately half of treated eyes across heterogeneous studies, and better outcomes in earlier stages. Visual acuity was stabilized or improved in 57.1% of treated eyes, while recurrence occurred in 19.3%. Multimodal imaging consistently identified peripheral telangiectasia, lipid exudation and localized capillary nonperfusion. Aqueous cytokine profiling in small cohorts has reported elevated inflammatory mediators (e.g. interleukin [IL]-6 and IL-1β) in some adults, suggesting that inflammatory signalling may contribute to blood-retina barrier dysfunction and macular oedema alongside VEGF-related permeability. Current evidence supports Coats disease diagnosed in adulthood as a chronic telangiectatic retinal vasculopathy with frequent macular involvement. Therapeutic outcomes appear to favour individualized, imaging-guided combination strategies and prospective studies with standardized endpoints are needed to refine management.
2026-04-18 | Anterior Chamber Cholesterolosis in a 1-Year-Old Patient with Coats' Disease. A Case Report.
To present a rare case of anterior chamber cholesterolosis in a patient with Coats' disease with emphasis on the differential diagnosis, to compare the clinical findings and therapeutic approach with previously reported cases; and to clarify the pathogenetic mechanisms and associated complications of anterior chamber cholesterolosis, based on an analogy with previously published cases. A 14-month-old boy was brought to our clinic with a painful, irritated right eye. Anterior chamber cholesterolosis was identified, associated with acute glaucoma, total retinal detachment, and vitreous hyperechogenicity with suspected calcifications on ultrasound (US). Coats' disease with anterior chamber cholesterolosis or retinoblastoma-associated masquerade syndrome were considered in the differential diagnosis. Magnetic resonance imaging (MRI) did not confirm retinoblastoma. The patient underwent anterior chamber lavage, followed by intracameral and intravitreal injection of an anti-vascular endothelial growth factor (anti-VEGF) agent. A cytological analysis of the anterior chamber aspirate showed no evidence of malignant cells. Although the treatment achieved the expected effect, the eye gradually progressed to phthisis bulbi. Nevertheless, the eye has remained painless, it has shown no signs of irritation and the intraocular condition has remained stable over the long term. Anterior chamber cholesterolosis is a rare complication of Coats' disease, reported in less than 3% of patients. Exclusion of retinoblastoma-associated masquerade syndrome is essential in the differential diagnosis. In comparison with previously published cases of anterior chamber cholesterolosis as a complicating factor in Coats' disease, we have observed a similar character and course of the disease in patients with onset in early childhood. However, a difference can be observed in the therapeutic approach. In all the previously reported cases, the painful eye was enucleated. In our case, preserving the blind eye through a more conservative surgical approach proved beneficial.
2026-01-09 | Coats' disease: current treatment trends. A literature review
In 1908, G. Coats first described a group of patients with eye damage, manifested by telangiectasia with massive intraand subretinal exudation, leading to local elevation of the retina and/or its complete detachment. Until now, a generally accepted pathogenesis model has not been developed and the causes of Coats disease (CD) have not been identified. It was assumed that the cause of the disease could be an infectious process, an inflammatory reaction, but there is currently no consensus on this issue. A study of the cytokine profile showed an increased concentration of VEGF in the intraocular, subretinal fluid and vitreous humor, an increase in the concentration of IL-6, IL-1b and MCP-1 levels was also noted. Complications of CD include vasoproliferative tumors, neovascular glaucoma, intraretinal and intraocular hemorrhages, vitreous fibrosis, traction retinal detachment. CD treatment includes laser coagulation, cryotherapy, intravitreal administration of glucocorticosteroids and angiogenesis inhibitors. In severe forms and advanced stages of CD, vitreoretinal surgery is resorted to. Enucleation may be necessary is some cases.
2025-12-31 | Adult-onset Coats’ disease. Case series and literature review
Adult-onset Coats’ disease is a rare idiopathic retinal vascular disorder. We present three cases of unilateral retinal telangiectasia and exudation diagnosed through clinical examination and fluorescein angiography. All patients were treated with laser photocoagulation and/or: two also underwent cryotherapy, one received transpupillary thermotherapy, and two received adjunctive anti-VEGF therapy. A literature review highlights male predominance, variable age of onset, and frequent macular involvement. Initial vision acuity appears to be the primary predictor of post-treatment outcomes. Early diagnosis and combined therapy can stabilise the disease and preserve vision.
2025-01-13 | Coats disease presenting with vitreous hemorrhage and neovascular glaucoma.
To describe a patient with Coats disease with an atypical presentation of neovascular glaucoma and vitreous hemorrhage. A 15-year-old male presented with five days of pain, redness, and swelling and was found to have neovascular glaucoma in his right eye. Further evaluation revealed Coats disease stage 3AI with a subtotal exudative retinal detachment inferiorly, telangiectatic vessels, and vitreous hemorrhage. Intravitreal anti-vascular endothelial growth factor, laser photocoagulation, pars plana vitrectomy, and valved tube-shunt placement was performed, eventually resulting in a favorable visual outcome. We report a patient with a unique presentation of advanced Coats disease. By highlighting the treatment approach and potential factors that led to the preservation of visual function, we hope to expand the literature on the diagnosis and management of Coats disease.
cell therapies
2025-09-20 | Successful Liver Transplantation for Coats Plus Syndrome With Hepatopulmonary Syndrome: A Case Report and Literature Review.
Coats plus syndrome (CPS) is a rare telomere biology disorder (TBD) linked to mutations in the CTC1-STN1-TEN1 complex and characterized by cerebroretinal microangiopathy, bone marrow failure, and liver disease, often progressing to hepatopulmonary syndrome (HPS). The case is a 16-year-old boy clinically diagnosed with CPS at age 7. He developed exertional dyspnea at 14 with 93% of resting oxygen saturation (SpO2), 70 mmHg of partial pressure of oxygen (PaO2), and 22.9 mmHg of alveolar-arterial oxygen difference (A-aDO2) under room air, prompting home oxygen therapy (HOT). Imaging and liver biopsy showed significant collateral circulation and portal vein fibrosis and dilation without cirrhosis or pulmonary fibrosis. HPS was diagnosed by positive microbubble contrast echocardiography and 15.7% of shunt fraction in pulmonary ventilation-perfusion scintigraphy. Due to the rapid progression of HPS, he underwent living-donor LT. The postoperative disease course was good, except for acute liver rejection, and the patient was discharged on postoperative day 40. One month after transplantation, echocardiography showed a negative bubble study, pulmonary scintigraphy revealed an improved shunt fraction of 10.2%, resting SpO2 under room air was 100%, and he is doing well without the recurrence of HPS. Historically, LT for HPS patients with TBDs was avoided because of uncertain prognosis and potential disease progression in other organs. However, early-stage LT in TBDs may be preferable and reduce the complexity of LT. Before irreversible changes in intrapulmonary blood vessels occur, early hypoxemia monitoring and regular imaging are essential to ensure timely LT for HPS.
2025-03-07 | [Analysis of 5-year efficacy of adjuvant external drainage of subretinal fluid therapy in Coats disease with severe exudative retinal detachment].
Objective: To study the 5-year long-term efficacy and complications of adjuvant external drainage of subretinal fluid therapy in patients with severe exudative retinal detachment (ERD) secondary to stage 3B juvenile Coats disease. Methods: This was a retrospective case series study. From January 2014 to December 2018, twenty-three eyes of 23 patients with severe ERD secondary to stage 3B juvenile Coats disease diagnosed at Beijing Tongren Hospital, Capital Medical University were included in this study. All patients underwent wide-field retinal imaging, fluorescein fundus angiography (FFA) and ocular color Doppler imaging. All cases received external drainage of subretinal fluid therapy combined with treatments including intravitreal injection of anti-vescular edothelial growth factor (VEGF) agents, retinal photocoagulation and/or cryotherapy. Clock-hour extent and occlusion of retinal vascular telangiectasia, changes of subretinal fluid, best corrected visual acuity (BCVA, logMAR) and incidences of complications including vitreous fibrosis, subretinal fibrosis, tractional retinal detachment (TRD), retinal cyst and complicated cataract were observed. Statistical analyses were performed using Fisher's exact test, corrected chi-square test and chi-square test, Mann-Whitney U test, paired-sample Wilcoxon signed-rank test and Logistic regression analysis. Results: Among 23 eyes of 23 patients, 22 patients (95.7%) were male and 1 patient (4.3%) was female. All patients had unilateral Coats disease. The median age of initial visit was 43.0 (38.0, 47.0) months (range, 26 to 129 months). The median clock-hour extent of retinal vascular telangiectasia was 9.0 (7.0, 10.0) (range, 5 to 12). The median time of external drainage of subretinal fluid therapy was 1.0 (1.0, 2.0). The median time of retinal photocoagulation was 2.0 (2.0, 3.0). The median time of intravitreal injection of anti-VEGF agents was 4.0 (3.0, 6.0). Retinal cryotherapy was performed in 14 eyes (60.9%). The median follow-up after initial treatment was 66.5 (64.0, 70.0) months (range, 60 to 89 months). At last follow-up, retinal vascular telangiectasia of all patients were completely or partially occluded, and subretinal fluid was completely absorbed or obviously reduced; of which 8 eyes (34.8%) were completely occluded, and 15 eyes (65.2%) were partially occluded. The incidences of vitreous fibrosis, TRD and complicated cataract in the completely occluded eyes were lower than those in the partially occluded eyes (all P<0.005). No global enucleation was performed in any patient because of disease progression. Among 15 eyes of 15 patients underwent BCVA examination, 3 eyes had an improved vision, 10 eyes had a stable vision and 2 eyes had a decreased vision. The difference between median BCVA after treatment and that before treatment was not statistically significant [2.6 (2.6, 2.9) vs. 2.9 (2.6, 3.2), Z=-0.14, P=0.891]. Seventeen eyes (73.9%) developed vitreous fibrosis; 21 eyes (91.3%) developed subretinal fibrosis; 12 eyes (52.2%) developed TRD, 5 eyes (21.7%) developed retinal cyst and 15 eyes (65.2%) developed complicated cataract. Patients with complicated cataract had a higher incidence of TRD than that in patients without complicated cataract (11/15 vs. 1/8; χ2=10.96, P=0.001). The occurrence of TRD was associated with the greater clock-hour extent of retinal vascular telangiectasia and the presence of complicated cataract (OR=2.04, 95%CI: 1.14-3.64, P=0.016; OR=73.18, 95%CI: 4.58-1 168.40, P=0.002). Conclusions: External drainage of subretinal fluid is a long-term effective adjuvant therapy in stage 3B juvenile Coats disease with severe ERD. However, there are possible complications such as TRD and complicated cataract after surgeries.
2025-03-03 | Active External Drainage of Subretinal Fluid without Vitrectomy to Treat Advanced Coats Disease with Exudative Retinal Detachment - A Case Report and Literature Review.
To illustrate the successful management of advanced coats disease with exudative retinal detachment with posterior lens touch in a three year old young boy using subretinal drainage technique using an extrusion cannula. Under general anesthesia, external drainage of subretinal fluid (SRF) was performed using an active extrusion cannula. Concurrent cryotherapy and endolaser treatment were administered, with cytological analysis of SRF to exclude retinoblastoma. The retina achieved near-complete reattachment on the first postoperative day and maintained stability during the six-month follow-up period. Active extrusion cannula-based sub retinal drainage technique might be helpful to achieve quicker reattachment of retina in advanced coats disease with massive exudative retinal detachment.
small molecules
2026-06-09 | Long-Term Outcomes of Two-port Non-vitrectomy Endolaser Therapy in Moderate-to-severe Stage 3A Coats Disease.
To report the long-term outcomes of two-port non-vitrectomy endolaser therapy (TPNVE) in moderate-to-severe stage 3A Coats disease. In this retrospective observational case series, patients with moderate-to-severe stage 3A Coats disease who were treated with TPNVE in a single tertiary referral vitreoretinal practice between March 2014 and May 2024 were included. The anatomic success rate, number of treatment sessions, and complications were analyzed. Of the 46 patients (47 eyes) included in the study, 33 (70.2%) eyes were stage 3A1, and the rest were stage 3A2. The mean (median, range) extent of ERD was 6.1 (5.0, 4.0-12.0) clock hours. The median (range) follow-up time was 67.7 (13.9-135.4) months. All 47 eyes achieved anatomic success with a median (range) treatment session of 2.0 (1-6) sessions. The median number of TPNVE treatments and conventional laser photocoagulation treatments was 1.2 (range, 1-2) and 0.0 (range, 0-4), respectively. Twenty (42.5%) eyes required only one session. There were 47 instances of recurrence in 27 eyes, of which over half (59.5%) took place in the first 6 months. Complications included cataracts in 11 (23.4%), vitreoretinal fibrosis in 11 (23.4%), subretinal fibrosis in 11 (23.4%), tractional retinal detachment in one (2.1%), and glaucoma in one (2.1%) of the eyes. TPNVE demonstrated efficacy in ablating abnormal vessels and reattaching the retina for moderate-to-severe stage 3A Coats disease. Approximately half of the eyes with an ERD extent of at least 4 clock hours achieved anatomical success after a single TPNVE surgery.
2026-06-09 | Management of a patient with late-onset Coats’ disease
Coats’ disease is an idiopathic condition characterized by the formation of retinal telangiectasias, as well as micro- and macroaneurysms, accompanied by intraretinal and subretinal exudation. Due to the inconspicuous progression of the early stages, diagnosis is often significantly delayed. This necessitates the use of multimodal imaging as a key tool for the timely detection of the pathology. Currently, there are no standardized guidelines for the treatment of Coats' disease, which may be attributed to its relatively low prevalence in the general population. We report a case involving a 49-year-old patient diagnosed with Stage 2 Coats’ disease (according to the classification by J. Shields et al., 2000). The management strategy selected was a multi-stage laser treatment based on a topographically oriented approach. This clinical case illustrates the potential of multimodal imaging in the early diagnosis of exudative-hemorrhagic retinitis, the determination of management tactics, the execution of topographically oriented treatment, and postoperative monitoring. Keywords: Coats' retinitis; laser coagulation; optical coherence tomography; multimodal imaging; fluorescence angiography; clinical case
2026-01-19 | A Case of Coats Disease Diagnosed during Amblyopia Treatment.
Coats disease is a rare, nonhereditary retinal disorder, and early detection and treatment are crucial for preserving visual function. This case report details a pediatric case where Coats disease was diagnosed during follow-up for amblyopia. An 8-year-old boy with a history of bilateral hyperopic amblyopia and right congenital ptosis underwent eyeglass and occlusion therapy starting at age 2. By age 4, he had achieved good visual acuity. At a routine follow-up 4 years later, fundus examination revealed exudative lesions in the left macula. Optical coherence tomography (OCT) showed macular hyperreflective lesions and the thickened choroid in his left eye, leading to a diagnosis of Coats disease. The patient was treated with retinal photocoagulation. Ten months after treatment, his visual acuity remained stable, and a follow-up OCT showed a reduction in the hyperreflective lesions in the outer macular layers. Routine dilated fundus examinations and OCT-based retro-choroidal morphology for anisometropic amblyopia facilitated early detection and timely intervention for Coats disease, which lacks overt symptoms in its early stages. This case highlights the importance of retinal monitoring in amblyopic eyes to preserve visual function.
2025-10-09 | Mapping Macrophage Diversity in Coats' Disease: A Lipid-Associated Subset in the Pathogenesis of Exudative Retinopathy.
Coats' disease is a retinal vasculopathy characterized by retinal telangiectasia, exudation, and retinal detachment. Lipid exudates serve as crucial biomarkers for the assessment and prediction of disease progression. However, the pathophysiological mechanisms driving exudation remain inadequately understood. This study aimed to investigate the cellular and molecular mechanisms underlying the pathogenesis of Coats' disease. Single-cell suspensions of epiretinal membrane and vitreous samples were collected from a patient with Coats' disease, and corresponding vitreous and epiretinal membrane samples from four patients with proliferative vitreoretinopathy were used as controls. The samples were analyzed by single-cell RNA sequencing using Seurat, Slingshot, hdWGCNA, and scFEA in R/Python software. In this study, we present the first single-cell atlas of the retinal microenvironment in Coats' disease, derived from clinical samples of epiretinal membrane and vitreous cells. Our analysis identified a predominance of macrophages, with a dominant cluster exhibiting a transcriptional profile characteristic of lipid-associated macrophages (LAMs). Single-cell trajectory and metabolic analysis revealed a gradual decrease in lipid oxidative catabolism in LAMs, with a progressive shift in biological function from phagocytosis to inflammation and extracellular matrix remodeling. Furthermore, we identified that the transition of LAMs was regulated by a gene module centered around ABCA1, a key regulator in the progression of atherosclerosis. Single-cell drug response analysis further infers that an antiatherosclerotic regimen may elicit a superior response in LAMs compared with conventional treatments. Collectively, our characterization of LAM phenotypes may provide novel insights into the pathogenesis of Coats' disease and identify potential targets for disease-specific interventions.
2025-09-25 | Coats Disease With Macular Nodule.
To describe a case series of Coats disease with macular nodule mimicking retinoblastoma. Retrospective case series. Of 339 patients with Coats disease, 7 (2%) with macular nodules were included. All had a referral diagnosis of retinoblastoma. All were males (mean age-6 y). The most common presenting complaint was decreased vision (n=4, 57%). Median best corrected visual acuity at presentation was counting fingers close to face, which was maintained at last follow-up (mean, 8 months). The nodule had a mean diameter of 5x4x3 mm, was grayish white, and bilobed (n=3, 43%), or dome shaped (n=4, 57%) with surface pigmentation (n=4, 57%), involving fovea (n=5, 71%), with surrounding exudates and second- or third-order retinal vessels. OCT (n=4) showed a well-defined hyperreflective subretinal nodule with posterior shadowing, cysts, and exudates. USG (n=6) showed an echodense intraocular nodule with moderate internal reflectivity with hyperechoic foci in 2 (33%) cases. FFA showed early- and mid-phase hypo-fluorescence with focal leakage with late-phase hyperfluorescence. Focal green laser photocoagulation of the telangiectatic vessels was performed in 5, one underwent subretinal fluid drainage and scleral buckling, and one was observed. Macular nodule in Coats disease represents a preferential accumulation of lipid in the macula. This rare variant of Coats disease can mimic retinoblastoma process. Be aware of this entity to ensure appropriate diagnosis and treatment.
antibodies
2026-04-28 | Adult-onset Coats disease: A systematic review and meta-analysis of imaging biomarkers and targeted therapies.
Coats disease diagnosed in adulthood is a rare idiopathic retinal telangiectatic vasculopathy that may differ in phenotype from childhood-diagnosed cases. This systematic review synthesizes current evidence to characterize its clinical spectrum, imaging features, treatment outcomes and underlying mechanisms, with a focus on immunovascular pathways. Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA 2020) guidelines, PubMed, Scopus and Web of Science were searched through October 2025 for studies including patients ≥18 years with confirmed Coats disease. Clinical and imaging findings, treatment strategies and outcomes were extracted and narratively synthesized. When possible, random-effects meta-analysis was performed. Thirty-five studies comprising 240 patients (250 eyes) were included. Disease presentation was predominantly unilateral and diagnosed at Shields Stage 2 or 3. Laser photocoagulation and anti-vascular endothelial growth factor (VEGF) agents were the most frequently described therapies, with anatomical stabilization or regression of exudation reported in approximately half of treated eyes across heterogeneous studies, and better outcomes in earlier stages. Visual acuity was stabilized or improved in 57.1% of treated eyes, while recurrence occurred in 19.3%. Multimodal imaging consistently identified peripheral telangiectasia, lipid exudation and localized capillary nonperfusion. Aqueous cytokine profiling in small cohorts has reported elevated inflammatory mediators (e.g. interleukin [IL]-6 and IL-1β) in some adults, suggesting that inflammatory signalling may contribute to blood-retina barrier dysfunction and macular oedema alongside VEGF-related permeability. Current evidence supports Coats disease diagnosed in adulthood as a chronic telangiectatic retinal vasculopathy with frequent macular involvement. Therapeutic outcomes appear to favour individualized, imaging-guided combination strategies and prospective studies with standardized endpoints are needed to refine management.
2026-04-18 | Anterior Chamber Cholesterolosis in a 1-Year-Old Patient with Coats' Disease. A Case Report.
To present a rare case of anterior chamber cholesterolosis in a patient with Coats' disease with emphasis on the differential diagnosis, to compare the clinical findings and therapeutic approach with previously reported cases; and to clarify the pathogenetic mechanisms and associated complications of anterior chamber cholesterolosis, based on an analogy with previously published cases. A 14-month-old boy was brought to our clinic with a painful, irritated right eye. Anterior chamber cholesterolosis was identified, associated with acute glaucoma, total retinal detachment, and vitreous hyperechogenicity with suspected calcifications on ultrasound (US). Coats' disease with anterior chamber cholesterolosis or retinoblastoma-associated masquerade syndrome were considered in the differential diagnosis. Magnetic resonance imaging (MRI) did not confirm retinoblastoma. The patient underwent anterior chamber lavage, followed by intracameral and intravitreal injection of an anti-vascular endothelial growth factor (anti-VEGF) agent. A cytological analysis of the anterior chamber aspirate showed no evidence of malignant cells. Although the treatment achieved the expected effect, the eye gradually progressed to phthisis bulbi. Nevertheless, the eye has remained painless, it has shown no signs of irritation and the intraocular condition has remained stable over the long term. Anterior chamber cholesterolosis is a rare complication of Coats' disease, reported in less than 3% of patients. Exclusion of retinoblastoma-associated masquerade syndrome is essential in the differential diagnosis. In comparison with previously published cases of anterior chamber cholesterolosis as a complicating factor in Coats' disease, we have observed a similar character and course of the disease in patients with onset in early childhood. However, a difference can be observed in the therapeutic approach. In all the previously reported cases, the painful eye was enucleated. In our case, preserving the blind eye through a more conservative surgical approach proved beneficial.
2026-01-09 | Coats' disease: current treatment trends. A literature review
In 1908, G. Coats first described a group of patients with eye damage, manifested by telangiectasia with massive intraand subretinal exudation, leading to local elevation of the retina and/or its complete detachment. Until now, a generally accepted pathogenesis model has not been developed and the causes of Coats disease (CD) have not been identified. It was assumed that the cause of the disease could be an infectious process, an inflammatory reaction, but there is currently no consensus on this issue. A study of the cytokine profile showed an increased concentration of VEGF in the intraocular, subretinal fluid and vitreous humor, an increase in the concentration of IL-6, IL-1b and MCP-1 levels was also noted. Complications of CD include vasoproliferative tumors, neovascular glaucoma, intraretinal and intraocular hemorrhages, vitreous fibrosis, traction retinal detachment. CD treatment includes laser coagulation, cryotherapy, intravitreal administration of glucocorticosteroids and angiogenesis inhibitors. In severe forms and advanced stages of CD, vitreoretinal surgery is resorted to. Enucleation may be necessary is some cases.
2025-12-31 | Adult-onset Coats’ disease. Case series and literature review
Adult-onset Coats’ disease is a rare idiopathic retinal vascular disorder. We present three cases of unilateral retinal telangiectasia and exudation diagnosed through clinical examination and fluorescein angiography. All patients were treated with laser photocoagulation and/or: two also underwent cryotherapy, one received transpupillary thermotherapy, and two received adjunctive anti-VEGF therapy. A literature review highlights male predominance, variable age of onset, and frequent macular involvement. Initial vision acuity appears to be the primary predictor of post-treatment outcomes. Early diagnosis and combined therapy can stabilise the disease and preserve vision.
2025-01-13 | Coats disease presenting with vitreous hemorrhage and neovascular glaucoma.
To describe a patient with Coats disease with an atypical presentation of neovascular glaucoma and vitreous hemorrhage. A 15-year-old male presented with five days of pain, redness, and swelling and was found to have neovascular glaucoma in his right eye. Further evaluation revealed Coats disease stage 3AI with a subtotal exudative retinal detachment inferiorly, telangiectatic vessels, and vitreous hemorrhage. Intravitreal anti-vascular endothelial growth factor, laser photocoagulation, pars plana vitrectomy, and valved tube-shunt placement was performed, eventually resulting in a favorable visual outcome. We report a patient with a unique presentation of advanced Coats disease. By highlighting the treatment approach and potential factors that led to the preservation of visual function, we hope to expand the literature on the diagnosis and management of Coats disease.
cell therapies
2025-09-20 | Successful Liver Transplantation for Coats Plus Syndrome With Hepatopulmonary Syndrome: A Case Report and Literature Review.
Coats plus syndrome (CPS) is a rare telomere biology disorder (TBD) linked to mutations in the CTC1-STN1-TEN1 complex and characterized by cerebroretinal microangiopathy, bone marrow failure, and liver disease, often progressing to hepatopulmonary syndrome (HPS). The case is a 16-year-old boy clinically diagnosed with CPS at age 7. He developed exertional dyspnea at 14 with 93% of resting oxygen saturation (SpO2), 70 mmHg of partial pressure of oxygen (PaO2), and 22.9 mmHg of alveolar-arterial oxygen difference (A-aDO2) under room air, prompting home oxygen therapy (HOT). Imaging and liver biopsy showed significant collateral circulation and portal vein fibrosis and dilation without cirrhosis or pulmonary fibrosis. HPS was diagnosed by positive microbubble contrast echocardiography and 15.7% of shunt fraction in pulmonary ventilation-perfusion scintigraphy. Due to the rapid progression of HPS, he underwent living-donor LT. The postoperative disease course was good, except for acute liver rejection, and the patient was discharged on postoperative day 40. One month after transplantation, echocardiography showed a negative bubble study, pulmonary scintigraphy revealed an improved shunt fraction of 10.2%, resting SpO2 under room air was 100%, and he is doing well without the recurrence of HPS. Historically, LT for HPS patients with TBDs was avoided because of uncertain prognosis and potential disease progression in other organs. However, early-stage LT in TBDs may be preferable and reduce the complexity of LT. Before irreversible changes in intrapulmonary blood vessels occur, early hypoxemia monitoring and regular imaging are essential to ensure timely LT for HPS.
2025-03-07 | [Analysis of 5-year efficacy of adjuvant external drainage of subretinal fluid therapy in Coats disease with severe exudative retinal detachment].
Objective: To study the 5-year long-term efficacy and complications of adjuvant external drainage of subretinal fluid therapy in patients with severe exudative retinal detachment (ERD) secondary to stage 3B juvenile Coats disease. Methods: This was a retrospective case series study. From January 2014 to December 2018, twenty-three eyes of 23 patients with severe ERD secondary to stage 3B juvenile Coats disease diagnosed at Beijing Tongren Hospital, Capital Medical University were included in this study. All patients underwent wide-field retinal imaging, fluorescein fundus angiography (FFA) and ocular color Doppler imaging. All cases received external drainage of subretinal fluid therapy combined with treatments including intravitreal injection of anti-vescular edothelial growth factor (VEGF) agents, retinal photocoagulation and/or cryotherapy. Clock-hour extent and occlusion of retinal vascular telangiectasia, changes of subretinal fluid, best corrected visual acuity (BCVA, logMAR) and incidences of complications including vitreous fibrosis, subretinal fibrosis, tractional retinal detachment (TRD), retinal cyst and complicated cataract were observed. Statistical analyses were performed using Fisher's exact test, corrected chi-square test and chi-square test, Mann-Whitney U test, paired-sample Wilcoxon signed-rank test and Logistic regression analysis. Results: Among 23 eyes of 23 patients, 22 patients (95.7%) were male and 1 patient (4.3%) was female. All patients had unilateral Coats disease. The median age of initial visit was 43.0 (38.0, 47.0) months (range, 26 to 129 months). The median clock-hour extent of retinal vascular telangiectasia was 9.0 (7.0, 10.0) (range, 5 to 12). The median time of external drainage of subretinal fluid therapy was 1.0 (1.0, 2.0). The median time of retinal photocoagulation was 2.0 (2.0, 3.0). The median time of intravitreal injection of anti-VEGF agents was 4.0 (3.0, 6.0). Retinal cryotherapy was performed in 14 eyes (60.9%). The median follow-up after initial treatment was 66.5 (64.0, 70.0) months (range, 60 to 89 months). At last follow-up, retinal vascular telangiectasia of all patients were completely or partially occluded, and subretinal fluid was completely absorbed or obviously reduced; of which 8 eyes (34.8%) were completely occluded, and 15 eyes (65.2%) were partially occluded. The incidences of vitreous fibrosis, TRD and complicated cataract in the completely occluded eyes were lower than those in the partially occluded eyes (all P<0.005). No global enucleation was performed in any patient because of disease progression. Among 15 eyes of 15 patients underwent BCVA examination, 3 eyes had an improved vision, 10 eyes had a stable vision and 2 eyes had a decreased vision. The difference between median BCVA after treatment and that before treatment was not statistically significant [2.6 (2.6, 2.9) vs. 2.9 (2.6, 3.2), Z=-0.14, P=0.891]. Seventeen eyes (73.9%) developed vitreous fibrosis; 21 eyes (91.3%) developed subretinal fibrosis; 12 eyes (52.2%) developed TRD, 5 eyes (21.7%) developed retinal cyst and 15 eyes (65.2%) developed complicated cataract. Patients with complicated cataract had a higher incidence of TRD than that in patients without complicated cataract (11/15 vs. 1/8; χ2=10.96, P=0.001). The occurrence of TRD was associated with the greater clock-hour extent of retinal vascular telangiectasia and the presence of complicated cataract (OR=2.04, 95%CI: 1.14-3.64, P=0.016; OR=73.18, 95%CI: 4.58-1 168.40, P=0.002). Conclusions: External drainage of subretinal fluid is a long-term effective adjuvant therapy in stage 3B juvenile Coats disease with severe ERD. However, there are possible complications such as TRD and complicated cataract after surgeries.
2025-03-03 | Active External Drainage of Subretinal Fluid without Vitrectomy to Treat Advanced Coats Disease with Exudative Retinal Detachment - A Case Report and Literature Review.
To illustrate the successful management of advanced coats disease with exudative retinal detachment with posterior lens touch in a three year old young boy using subretinal drainage technique using an extrusion cannula. Under general anesthesia, external drainage of subretinal fluid (SRF) was performed using an active extrusion cannula. Concurrent cryotherapy and endolaser treatment were administered, with cytological analysis of SRF to exclude retinoblastoma. The retina achieved near-complete reattachment on the first postoperative day and maintained stability during the six-month follow-up period. Active extrusion cannula-based sub retinal drainage technique might be helpful to achieve quicker reattachment of retina in advanced coats disease with massive exudative retinal detachment.
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Drug Discovery Landscape
2 orphan drug designations for Coats disease.
2 orphan drug designations for Coats disease.
Drug | Therapy type | Orphan designation | Clinical trial status | Sponsor |
|---|---|---|---|---|
faricimab | antibodies | FDA | — | Genentech, Inc. |
bevacizumab | antibodies | FDA | MicroSert, Ltd. |
“Terminated” – programs with discontinued clinical trials, or with no updates reported for 5 years after completion or 12 years after the last trial started.
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