Real-world insights and outcomes related to ciclosporin A 0.1% cationic emulsion for the long-term treatment of dry eye disease in Germany: Country-level sub-analysis of the PERSPECTIVE study.

PURPOSE: The PERSPECTIVE study was a real-world European, non-interventional, multicenter, observational study that evaluated the effectiveness, tolerability, and safety of ciclosporin A (CsA) 0.1% cationic emulsion (CE) in routine clinical practice as a treatment for adults with severe keratitis and dry eye disease (DED) that remained insufficiently controlled with artificial tears. This sub-analysis examined data from ophthalmology clinics in Germany to provide more precise insights into treatment patterns, outcomes, and clinical decision-making related to CsA 0.1% CE. METHODS: Study data were collected from adults starting CsA 0.1% CE (one drop in both eyes at bedtime) and followed up at Week 4, 12, and 24, and Month 12. The primary endpoint was mean change from baseline in corneal fluorescein staining (CFS) score (Oxford Grade Scale) at Month 12. Secondary endpoints examined the severity of ocular signs and symptoms, and adverse events (AEs). RESULTS: A total of 236 patients from 20 ophthalmology clinics in Germany participated in the PERSPECTIVE study (69.9% female; mean age 60.8 years). Following treatment with CsA 0.1% CE, patients experienced significant reductions in CFS score from Week 4, which were maintained through to Month 12 (P < 0.0001). From baseline, 81.6% of patients experienced an improvement in CFS score at Month 12. CsA 0.1% CE provided significant reductions in the severity of eyelid and conjunctival erythema at Month 12 compared with baseline (P < 0.001), as well as significant reductions in the severity of subjective ocular symptoms (all P ≤ 0.015). Safety data were consistent with the known safety profile of CsA 0.1% CE. Tolerability was rated as "satisfactory," "good," or "very good" by 97.2% of physicians and 95.7% of patients. CONCLUSION: Outcomes in Germany were similar to those reported for the overall European study population and are indicative of the treatment results that ophthalmologists may expect to see with CsA 0.1% CE treatment in real-life clinical practice. Treatment with CsA 0.1% CE provided long-term improvements over 12 months and was generally well tolerated.

Clinical and Ophthalmological Characteristics of Ocular Syphilis in a Retrospective Tertiary Hospital Cohort.

BACKGROUND: Data on ocular syphilis (OS) and its clinical presentation are currently insufficient. This study aimed to investigate the characteristics of a cohort with a high OS incidence at a university hospital in Germany, focusing on the clinical presentation of OS. METHODS: This single-center cohort study retrospectively analyzed data on 90 patients with 109 episodes of syphilis between 2008 and 2018. Cases of OS were identified and additionally reevaluated through a study-specific secondary assessment by an ophthalmologist specializing in uveitis. RESULTS: Twenty-three patients (26%) were diagnosed with OS, 16 (70%) of whom were with binocular involvement. Uveitis, especially that of the posterior segment, showed a high prevalence. Lumbar puncture was performed in 20 OS patients (87%), of whom 17 (85% of those with lumbar puncture/74% in total) met the 2018 Centers for Disease Control and Prevention criteria for likely neurosyphilis. Five (22%) of 23 patients had HIV infection, of whom 2 did not receive antiretroviral therapy. The preferred syphilis treatment regimens were benzylpenicillin and ceftriaxone, which yielded favorable serological, clinical, and ophthalmological outcomes. CONCLUSIONS: A high incidence of OS was identified, and physicians should be aware of uveitis as a manifestation of syphilis. Most patients presented with uveitis and syphilis in an early or late latent stage and showed central nervous system involvement.

Systematic review: non-adherence and non-persistence in intravitreal treatment.

PURPOSE: Intravitreal injection of VEGF inhibitors has become the standard of care for different macular diseases within the last years resulting in improved visual outcomes. Under real-life conditions, however, the necessity for frequent retreatments and reexaminations poses a burden for patients and treatment centers. Non-adherence and non-persistence to intravitreal treatment may lead to inferior clinical outcomes, and knowledge of contributing factors is crucial to improve adherence. This systematic review analyzes current literature for potential factors involved in non-adherence and non-persistence. METHODS: A systematic search was conducted in PubMed and Embase including three different aspects of intravitreal injection therapy: (1) diseases with intravitreal injections as treatment, (2) intravitreal injection, and (3) aspects of therapy adherence or therapy persistence. Data from identified quantitative studies were further extracted and grouped according to WHO criteria (condition, socio-economy, therapy, patient, and health system). The methodological quality of identified studies was graded. Identified qualitative studies (i.e., interviews) were descriptively analyzed and their findings narratively reported. RESULTS: Twenty-four publications were included. In 16 of those publications, a quantitative data analysis was conducted, analyzing factors associated with non-adherence. Worse visual acuity at baseline and unfavorable development of visual acuity, higher age, and greater distance to the treatment center were associated with non-adherence, while there was inconsistent evidence for an association of comorbidity. In qualitative studies, high follow-up/treatment burden, fear and anxiety, disappointed patient expectations, and lack of motivation to continue treatment were reported as reasons for non-persistence. CONCLUSIONS: Knowledge of potential barriers in IVT treatment may improve adherence and potentially clinical results. Improvements can be achieved particularly in the healthcare complex (organizational improvements) and the "patient" complex by establishing realistic expectations. Recurrent education of the patient may be necessary.

[Vascular Biomarkers in Retinal Vessel Analysis]. / Vaskuläre Biomarker der retinalen Gefäßanalyse.

Modern non-invasive imaging procedures - including fundus photography and optical filming - can be used to investigate objective aspects of changes in the function and structure of retinal vessels. In the pre-, post- and capillary areas, the status and behaviour of retinal vessels passively and actively influence blood flow and flow-related metabolism through changes in vascular diameter. Retinal vessels have the same structure and function as cerebral vessels and reflect the status of the microcirculation. In dynamic retinal vessel analysis, the function and structure of the longitudinal section of retinal vessels are subjected to a non-invasive functional and structural examination over a period before, during and after a specific vascular stimulation. The exact methodology of the evaluation and the designation of the parameters depend on the investigation. Retinal vessel analysis has been employed in several cross-sectional and interventional clinical studies in ophthalmology and other specialities, including cardiology, neurology, neurosurgery, nephrology, gynaecology, sports medicine, diabetology, hypertensiology and others. Static retinal vessel analysis is an inexpensive, reproducible, non-invasive technique, which can be used to make a prognostic statement on the vascular health of an individual patient. Dynamic retinal vessel analysis possesses a broader spectrum of diagnostic applications than the static procedure, as it examines changes in vessel diameter continuously over time. The use of several different methodological modalities for retinal vessel analysis together with their relevant quantitative biomarkers represents a promising approach for the evaluation of vascular diseases and cerebro- or cardiovascular morbidity and mortality. Interdisciplinary clinical application of these vascular biomarkers is becoming increasingly important in ophthalmology and other specialities.

Using TNF-alpha antagonist adalimumab for treatment for multisystem sarcoidosis: a case study.

We report the usage of the TNF-alpha antagonist adalimumab in patients with progressive multisystem sarcoidosis. Three patients with multisystem sarcoidosis (MSS) were treated with adalimumab for 12 months. All three patients were quickly responded to adalimumab and experienced a nearly complete regression of the symptoms that lead to an intensive immunosuppression. However, some accompanying symptoms of sarcoidosis, such as splenomegalia, did not respond. One patient suffered 18 months later a new unspecified abdominal lymphadenopathy. TNF-alpha antagonists can be helpful agents in the treatment for MSS. However, the experience with TNF-alpha antagonists in patients with sarcoidosis is still limited. Multicenter trials and a comparison of the different agents are needed to validate the safety and efficacy in these patients. Optimal dosage, duration of therapy and long-term toxicity of anti-TNF therapy in patients with refractory sarcoidosis are yet to be determined in prospective trials.

Real-World Effectiveness, Tolerability and Safety of Cyclosporine A 0.1% Cationic Emulsion in Severe Keratitis and Dry Eye Treatment.

INTRODUCTION: The PERSPECTIVE study evaluated, in routine clinical practice, the effectiveness, tolerability and safety of cyclosporine A (CsA) 0.1% cationic emulsion (CE) in controlling severe keratitis in adults with dry eye who remained insufficiently controlled despite artificial tear (AT) use. METHODS: A prospective, multicenter, observational study was conducted at 44 ophthalmology clinics across Finland, Germany, Norway, Sweden and the UK. Adults treated with ATs for severe keratitis and dry eye received CsA 0.1% CE therapy (1 drop in both eyes at bedtime) and were followed up at weeks 4, 12 and 24 and at month 12. Primary endpoint was mean [standard deviation (SD)] change from baseline in corneal fluorescein staining (CFS; Oxford Grade Scale) at month 12 following CsA 0.1% CE initiation. Secondary endpoints examined ocular sign and symptom severity and adverse events (AEs). RESULTS: The full analysis set included 472 adults (75.9% female). Mean (SD) age was 61.9 (15.41) years. Mean (SD) CFS score was significantly reduced from baseline [2.56 (1.10)] at month 12 [1.10 (SD 1.13); P < 0.0001]. CFS score reductions were statistically significant from week 4, with further incremental decreases reported at study visits through month 12 (P < 0.0001). Severity of eyelid and conjunctival erythema was significantly reduced from baseline at week 4 and maintained through month 12 (P < 0.001). Tear film breakup time increased significantly from baseline at all study visits through month 12 (P < 0.001). Ocular symptom severity was significantly reduced from baseline at all study visits through month 12 (P < 0.001). Overall, 101 treatment-related AEs were reported. Most were mild/moderate (83.6%) and resolved by month 12 (73.3%). CONCLUSIONS: In routine clinical practice, CsA 0.1% CE provided statistically significant reductions in dry eye signs and symptoms. Improvements were seen at week 4 and maintained over 12 months. Treatment tolerability was good and consistent with previous CsA 0.1% CE clinical studies. TRIAL REGISTRATION: EU PAS register number: EUPAS 22376.

Dynamic retinal vessel response to flicker in obesity: A methodological approach.

Obesity and related metabolic disorders affect vascular endothelial function. The use of the Dynamic Vessel Analyzer (DVA) represents a modern methodological approach to analyze vascular function in the retinal microcirculation. Whether the dynamic reaction to flicker stimulation in retinal vessels is altered in obese subjects is investigated. Retinal vessel reactions to flicker stimulation were examined by DVA in 46 obese individuals (49.6±10.0years) and 46 age- and gender-matched healthy controls. The clinical examination included anthropometry, blood pressure measurements and blood sampling. Mean maximal arteriolar dilation in response to flicker was reduced in the obese group (3.2±1.8%) compared to controls (4.1±2.0%, p<0.05) and the time to maximal arteriolar dilation was prolonged (18.0±9.4s vs. 14.6±3.8s, p=0.03). In addition, mean maximal venular dilation was reduced in obese subjects (3.9±1.7% vs. 4.7±1.8%, p<0.05). Among the microvascular parameters, the most significant correlation with waist circumference was found for the "area under the reaction curve 50-80s after stimulation" in arterioles (r=-0.40; p<0.001). Functional retinal arteriolar reactivity to flicker stimulation differs between obese and healthy lean subjects. Time course analysis of retinal vessel response and its quantitative parameters can comprehensively characterize alterations of retinal vessel reactivity in metabolic disease.

Non-penetrating intracanalicular partial trabeculectomy via the ostia of Schlemm's canal.

BACKGROUND: In order to distinguish a more pronounced and sustained hypotensive effect of non-penetrating glaucoma surgery, a modified surgical procedure is proposed, which provides enlargement of the filtration membrane area with maximal maintenance of natural aqueous outflow pathways. METHODS: In 21 eyes of 17 patients with open-angle glaucoma (intraocular pressure: 32.4 ± 4.7 mmHg; age: 69.2 ± 4.4 years) the following non-penetrating surgery was performed. After the excision of the outer wall of Schlemm's canal, outer layers of the trabecular meshwork were removed with a trabecular spatula (Geuder AG, G-16240) at the site of the open area of Schlemm's canal. A cannula-harpoon (Geuder AG, G-S02199) was introduced through both Schlemm's canal ostia, between the less permeable and well-permeable trabecular layers and separating them. Due to the harpoon configuration of the cannula, the superficial less permeable trabecular layers were removed within Schlemm's canal adjacent to its ostia during the retracting movement of the cannula. Thus, the filtration zone became extended using the outflow pathways into Schlemm's canal. RESULTS: A postoperative intraocular pressure of 13.4 ± 2.3 mmHg after 2 years of follow-up was measured. In all 21 eyes the hypotensive effect was absolute (without medications). In seven cases (33%) a hyphema had occurred during surgery, which dissolved in all cases within 2-3 days postoperatively. CONCLUSIONS: The results of the study demonstrate a pronounced and sustained effect of the modified surgery technique, and show that this surgery can be applied successfully in patients with therapy-resistant open-angle glaucoma. Further randomized prospective studies with larger case numbers are needed for generalized conclusions for a large population.

Simulation and modeling of metamorphopsia with a deformable amsler grid.

A method to simulate and model metamorphopsia by means of a deformable Amsler grid is proposed. The interactively deformable grid is based on cubic B-splines to obtain a locally controlled deformation. By simulating metamorphopsia on normal sight volunteers, acquisition of a correction percentage is possible as a result of analyzing the magnitude of the simulated distortion and the applied correction model. The correction percentage obtained is 75.78% (7.36% standard deviation). This can express the feasible correction rate with the guidance of the patient qualitative feedback. The present work is motivated by the idea of obtaining a correction model of a patient with metamorphopsia and to implement this model into a head-mounted display to compensate the patient's deformation in the near future.

[Adherence to anti-VEGF treatment-Considerations and practical recommendations]. / Adhärenz bei der Anti-VEGF-Therapie ­ Überlegungen und praktische Empfehlungen.

BACKGROUND: Numerous studies have identified a lack of treatment adherence as an important factor that often conflicts with the necessary number of anti-VEGF injections and therefore a better functional result. OBJECTIVE: This article discusses approaches and possible measures to reduce the risk of late and infrequent intravitreal injections leading to the major issue of undertreatment. MATERIAL AND METHOD: In the course of an expert dialogue, relevant parameters of treatment adherence and variables were identified. Meaningful processes were structured and assigned to organizational areas. RESULTS: The compilation of meaningful measures enables practitioners to optimize their own implementation in different areas. Regular monitoring measures can identify the extent of treatment interruption and discontinuation. For specific indicators (treatments per time interval, longest pause interval, minimum coverage per unit time, delay) an effect on the development of visual function was demonstrated. Organizational measures, training of teams and referring physicians, redundant and iterative information transfer to patients have been proven in the experience of experts. The firm integration of these processes into the structures is facilitated by working with checklists. CONCLUSION: An optimization of the processes is possible to improve the adherence and the functional results; however, interventional studies showing how adherence and persistence can be increased in the German treatment setting are still lacking.

Myeloid-related protein 8/14 complex describes microcirculatory alterations in patients with type 2 diabetes and nephropathy.

BACKGROUND: Inflammation contributes to cardiovascular complications in type 2 diabetes, which are often characterized by microvascular alterations. We investigated whether myeloid-related protein 8/14 complex (MRP8/14) secreted by transmigrating monocytes and granulocytes may represent a biomarker for microvascular alterations in patients with type 2 diabetes and nephropathy. METHODS: MRP8/14 was measured in 43 patients with type 2 diabetes and nephropathy. Additionally, the inflammatory markers Interleukin-6 (IL-6), Tumor necrosis factor-alpha (TNF-alpha) and C-reactive protein (CRP) were quantified. To detect microvascular alterations proteinuria and retinal vessel caliber were used as classical and novel marker, respectively. Proteinuria was quantified by protein-creatinine ratio (PCR); retinal vessel caliber was quantified after retina photography on digitalized retina pictures. RESULTS: MRP8/14 was positively associated with inflammation (r = 0.57), proteinuria (r = 0.40) and retinal arterial caliber (r = 0.48). Type 2 diabetic patients with MRP8/14 values above the median of 5.8 microg/ml demonstrated higher proteinuria and larger retinal artery caliber than patients with MRP8/14 values below the median (logPCR: -0.51 +/- 0.52 versus -0.96 +/- 0.46, P < 0.01; retinal artery lumen (microm): 178.3 +/- 14.1 versus 162.7 +/- 14.9 P < 0.01). Both groups did not differ with regard to metabolic factors and blood pressure. MRP8/14 was an independent predictor of retinal artery caliber in multivariate stepwise regression analysis (beta = 0.607) and was positively associated with IL-6 (r = 0.57, P < 0.001) and TNF-alpha (r = 0.36, P < 0.05). CONCLUSION: MRP8/14--a marker for transendothelial migration--describes not only the state of inflammation in diabetic nephropathy, but additionally the degree of microvascular alterations in the glomerular and retinal bed. Therefore, MRP8/14 may be a potentially selective novel biomarker for microcirculatory defects in diabetic nephropathy.

Non-diabetic chronic kidney disease influences retinal microvasculature.

BACKGROUND: Chronic kidney disease (CKD) is characterized by increased cerebrovascular risk. Retinal vessel analysis (RVA) is an accepted measure of the retinal microvasculature, mirrors hypertension and cardiovascular morbidity. Epidemiological studies demonstrate narrower retinal arterioles with declining renal function. The effect of CKD on the retinal microcirculation remains uncertain. METHODS: RVA was performed in 34 non-diabetic CKD patients and 33 age-matched volunteers with normal renal function. Retinal photographs were digitized, vascular lumen diameters measured and the ratio of retinal arteriolar and venular lumen diameters (AVR) calculated. Office blood pressure (BP) was measured and cardiovascular risk factors assessed. RESULTS: AVR was lower in CKD patients as compared to age-matched volunteers (0.77 +/- 0.05 vs. 0.86 +/- 0.06; p < 0.05). In particular, retinal arterioles were narrower in CKD patients as compared to volunteers (169.6 +/- 20.4 vs. 189.5 +/- 14.2 microm; p < 0.01). In CKD, estimated glomerular filtration rate, BP and renin-angiotensin system blocker independently predicted AVR. Moreover, retinal arteriolar diameter independently predicted renal function (beta = 0.33; p < 0.05). CONCLUSION: CKD narrowed retinal arterioles suggesting an extended effect of CKD on the cerebral microvasculature. This study shows that in CKD patients, renal function, BP status and renin-angiotensin system blockade independently predict AVR as a marker for microvascular damage and that retinal microvasculature can predict renal function.

Functional retinal changes in Gaucher disease.

In Gaucher disease, sphingolipid glucosylceramide is accumulated in cells of the reticulo-endothelial system. This leads to the formation of "Gaucher cells," which are enlarged macrophages. In the eye, circumscript preretinal deposits are a pathognomonic feature. We describe the case of a 23-year-old woman with no subjective complaints but a reduced ERG response, leading to the suspicion that in addition to circumscript deposition diffuse accumulation of metabolic endproducts might be present in the retina in Gaucher patients.

Postoperative aqueous outflow in the human eye after glaucoma filtration surgery: biofluidmechanical considerations.

To evaluate the possible postoperative outflow from the anterior chamber of the eye after filtration surgery, a mathematical model based on fluid mechanical principles and clinical data is proposed. Two ophthalmic surgical procedures, non-penetrating deep sclerectomy and trabeculectomy, were analyzed. Based on mathematical modeling, the amount of postoperative outflow through the fistula (after trabeculectomy) or membrane (after non-penetrating surgery) as well as the outflow through residual natural drainage pathways were calculated and compared. From our model, the following results were obtained: 1) if trabeculectomy is carried out in an eye with preoperative intraocular pressure (IOP) of 30 mm Hg and postoperative IOP=10 mm Hg, only 10% of aqueous utilizes the natural outflow pathway via the trabecular meshwork, whereas if non-penetrating surgery is carried out in the same eye with postoperative IOP=16 mm Hg, the outflow through the trabecular meshwork amounts to 35%. Thus, non-penetrating surgery provides more aqueous outflow along the natural outflow pathways than trabeculectomy. 2) Generally, the higher the postoperative IOP and/or the lower the preoperative IOP, the higher the amount of aqueous, which will utilize the natural outflow pathways postoperatively. 3) The reestablishment of aqueous production postoperatively in addition to other factors, such as wound healing, may be a reason for IOP increase during the postoperative period.

Systemic exposure to intracameral vs topical mydriatic agents: in cataract surgery.

OBJECTIVE: The objective of this study was to compare systemic exposure to tropicamide/phenylephrine following intracameral or topical administration before cataract surgery. PATIENTS AND METHODS: Mydriatics exposure was calculated in patients randomized to intracameral fixed combination of mydriatics and anesthetic ([ICMA]: tropicamide 0.02%, phenylephrine 0.31%, and lidocaine 1%, N=271) or mydriatic eye drops ([EDs]: tropicamide 0.5% and phenylephrine 10%, N=283). Additional doses were permitted if required. Mydriatic plasma levels were determined by mass spectrometric HPLC in 15 patients per group before and after administration. RESULTS: Most ICMA patients (73.6%) received a single dose (200 µL) representing an exposure to tropicamide of 0.04 mg and phenylephrine of 0.62 mg. None of these patients received additional mydriatics. In the control group (three administrations), the exposure was 0.45 (11.3-fold higher than ICMA) and 10.2 (16.5-fold higher) mg. When additional ED was used in this group (9.2% of patients), it was 37.5-fold higher for tropicamide (10 drops, 1.5 mg) and 54.8-fold higher for phenylephrine (10 drops, 34 mg) than the recommended ICMA dose. Tropicamide plasma levels were not detectable at any time point in ICMA patients while it was detectable in all ED patients at 12 and 30 minutes. Phenylephrine was detectable in 14.3% of ICMA patients compared to all ED patients at least at one time point. More ED patients experienced a meaningful increase in blood pressure and/or heart rate (11.2% vs 6.0% of ICMA patients; P=0.03). CONCLUSION: Systemic exposure to tropicamide/phenylephrine was lower and cardiovascular (CV) effects were less frequent with ICMA. This could be of particular significance in patients at CV risk.

Effect of aging on retinal artery blood column diameter measured along the vessel axis.

PURPOSE: To determine whether retinal branch arteries of healthy persons in different age groups show different longitudinal vessel profiles at baseline and during dynamic reaction to flicker stimulation. METHODS: Thirty-three healthy subjects (age groups: 21-27 years, 40-59 years, and 60-85 years) were examined with the use of a retinal vessel analyzer. A monochromatic flicker of 12.5 Hz was applied for 60 seconds. Arterial diameters were measured in vessel segments of 1 mm in length to obtain the longitudinal vessel profile. Differences in amplitude and frequency of arterial width changes were characterized by the parameter spectral edge frequency (SEF). RESULTS: SEF was significantly different between the young group and the senior group in each phase of the arterial reaction to flicker (baseline, dilation, constriction, relaxation; P < 0.05; Mann-Whitney U test). No significant difference within any age group was found in any phase of the arterial reaction. No significant difference between the middle-aged and either young or elderly subjects was found at baseline. However, after stimulation, the middle-aged group displayed a significant difference compared with the young group, with values resembling those of the elderly group. CONCLUSIONS: In healthy elderly subjects, retinal arteries assumed a significantly less regular longitudinal vessel profile than those of young subjects. Middle-aged subjects assumed a more irregular profile only in the stimulated states of dilation, constriction, and relaxation. Early age-related changes in vessel profile are noted only after metabolic demand. These changes might be a cause for impaired blood flow and blood-vessel wall interaction.

Enteric-coated mycophenolate sodium in the treatment of non-infectious intermediate uveitis: results of a prospective, controlled, randomised, open-label, early terminated multicentre trial.

BACKGROUND/AIMS: To evaluate the efficacy, safety and tolerability of enteric-coated mycophenolate sodium (EC-MPS) in combination with low-dose corticosteroids compared with a monotherapy with low-dose corticosteroids in subjects with non-infectious intermediate uveitis (IU). METHODS: Open-label, prospective, controlled, randomised multicentre trial. Patients were randomised in a 1:1 ratio to either the treatment group (prednisolone plus EC-MPS) or control group (prednisolone monotherapy). Patients in the control group who relapsed within 6 months changed to the crossover group (prednisolone plus EC-MPS). Maximum treatment duration was 15 months. The primary endpoint was the time to first relapse in the treatment group and control group. RESULTS: Forty-one patients at eight sites were analysed. Twenty-two patients were allocated to the treatment group, with 19 patients in the control group. A first relapse occurred in 9 patients (40.9%) in the treatment group and 15 patients (78.9%) in the control group (p=0.03). The median time to the first relapse was >15 months for the treatment group and 2.8 months for the control group (p=0.07). The probability of relapse-free survival at month 15 was estimated to be 52.9% in the treatment group and 19.7% in the control group (p=0.01). 15 patients changed to the crossover group. Of these, only four patients developed a second relapse. No safety concerns arose during the trial. Only one patient had to discontinue EC-MPS due to increased liver enzymes. CONCLUSION: EC-MPS can be considered an effective and well-tolerated immunosuppressive drug to prevent relapses in patients with chronic IU. TRIAL REGISTRATION NUMBER: EUDRACT number: 2009-009998-10, Results.

Fabry disease: case report with emphasis on enzyme replacement therapy and possible future therapeutic options.

A 38-year-old male Caucasian with Fabry disease presented with angiokeratomas and tortuous conjunctival and retinal vessels. Additionally, the patient showed characteristic skin lesions of psoriasis and seborrheic dermatitis. His past medical history revealed anhidrosis, acral paresthesias, myocardial infarction, phlebothrombosis, hypertension, antithrombin III deficiency, factor V Leiden disease, chronic obstructive lung disease, tinnitus, diarrhea, recurrent abdominal pain, headache, and depressive mood. He was treated with intravenous substitution of the deficient enzyme alpha-galactosidase A. Possible future options in treatment of Fabry disease are discussed.