Therapeutic Plasma Exchange in Hepatology: Indications, Techniques, and Practical Application.

It is sobering that many liver failure patients die in the absence of liver transplantation (LT), and reducing its morbidity and mortality urgently needs more non-transplant treatment options. Among the several artificial liver support devices available, therapeutic plasma exchange (TPE) is the only one that improves survival in acute liver failure (ALF) patients. In many other disorders, data on survival benefits and successful bridging to transplant is encouraging. TPE removes the entire plasma, including damage-associated-molecular patterns, and replaces it with healthy donor fresh frozen plasma. In contrast, other artificial liver support systems (ALSS) correct the blood composition through dialysis techniques. TPE has become increasingly popular due to advances in apheresis techniques and a better understanding of its applicability in treating liver failure's pathophysiology. It provides metabolicdetoxification, and synthetic functions and modulates early innate immunity, fulfilling the role of ALSS. TPE is readily available in intensive care units, dialysis units, or blood banks and has enormous potential to improve survival outcomes. Hepatologists must take advantage of this treatment option by thoroughly understanding its most frequent indications and its rationale and techniques. This primer on TPE for liver clinicians covers its current clinical, technical, and practical applications, addresses the knowledge gaps, and provides future directions.

Postpartum Guillain-Barré Syndrome Presenting as Acute Motor Axonal Neuropathy in a Young Female: A Report of a Rare Case.

This case report presents a rare instance of acute motor axonal neuropathy (AMAN), a subtype of Guillain-Barré syndrome (GBS), in a 25-year-old postpartum female. The patient experienced a seven-day history of progressive, ascending motor weakness in all four limbs, beginning shortly before delivery and rapidly worsening postpartum. Notably, she exhibited no sensory deficits, a hallmark feature of AMAN, which was confirmed by nerve conduction studies revealing significant motor axonal involvement with preserved sensory function. The diagnostic process was complicated by the overlap of AMAN symptoms with other neurological conditions and the unusual postpartum context. Treatment with high-dose corticosteroids and plasmapheresis resulted in significant clinical improvement, underscoring the effectiveness of these interventions in managing AMAN. This case highlights the importance of considering AMAN in postpartum patients presenting with acute motor deficits, even in the absence of typical GBS sensory symptoms. It also emphasizes the need for early diagnosis and a multidisciplinary approach to optimize patient outcomes. By contributing to the limited literature on AMAN in postpartum women, this report aims to enhance clinical awareness, improve diagnostic accuracy, and inform treatment strategies for similar cases in the future.

Safety and efficacy of plasmapheresis in treatment of acute fatty liver of pregnancy-a systematic review and meta-analysis.

Introduction: Acute fatty liver of pregnancy (AFLP) is a fatal disease occurring in 3rd trimester. The safety and efficacy of plasmapheresis/plasma exchange (PP/PE) as an adjunctive treatment in patients of AFLP has been studied. We performed systematic review and meta-analysis to estimate the clinical parameters that included mortality rates and improvement of the biochemical parameters including Liver and Renal function enzymes, coagulopathy factors of AFLP patients. Methods: We searched PubMed, Ovid MEDLINE, Cochrane, CINAHL and Scopus, ClinicalTrials.gov. RevMan statistical software was used for meta-analysis. Results: Pooled survival proportion for AFLP patients treated with PP/PE was 87.74% (95% CI: 82.84 to 91.65). Efficacy of PP/PE was studied by its effect on mortality. PE/PP was associated with the reduction in the mortality with pooled odds ratio of 0.51 (95% CI: 0.08 to 3.09) with I2 = 86%. Sensitivity analysis after excluding outlier study, yielded a pooled odds ratio of 0.19 (95% CI: 0.02 to 1.52) with reduced heterogeneity (I2 = 63%). Biochemical parameter analysis demonstrated significant improvement post-PP/PE treatment, including decreased bilirubin (MD: 8.30, 95% CI: 6.75 to 9.84), AST (MD: 107.25, 95% CI: 52.45 to 162.06), ALT (MD: 111.08, 95% CI: 27.18 to 194.97), creatinine (MD: 1.66, 95% CI: 1.39 to 1.93), and Prothrombin time (MD: 5.08, 95% CI: 2.93 to 7.22). Discussion: Despite some heterogeneity, PP/PE shows promise in improving biochemical parameters in AFLP patients. PE can serve as a therapeutic approach for AFLP particularly in severe or refractory cases. PE provides the time for organ to recover and helps in creating a homeostatic environment for liver. Large RCTs and propensity matched studies are needed to better understand the safety and efficacy of the treatment. Systematic review registration: https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42022315698.

Efficacy of Therapeutic Plasma Exchange or Cangrelor as an Adjunctive Strategy to Facilitate Cardiopulmonary Bypass in Patients with Heparin-Induced Thrombocytopenia: A Systematic Review and Meta-Analysis.

OBJECTIVE: Conduct a systematic review and meta-analysis of the efficacy of therapeutic plasma exchange (TPE) or intravenous cangrelor to prevent thromboembolism in patients with heparin-induced thrombocytopenia (HIT) who undergo cardiopulmonary bypass (CPB) with heparin. DESIGN: Systematic review and meta-analysis. SETTING: N/A. PARTICIPANTS: Adults having cardiac surgery with a history of HIT who received preoperative or intraoperative TPE or intravenous cangrelor as an adjunct to CPB with heparin. INTERVENTIONS: None MEASUREMENTS AND MAIN RESULTS: A systematic review was performed using MEDLINE, PubMed, and Google Scholar. The primary outcome was avoidance of thromboembolism (venous or arterial) during or after CPB. Proportional meta-analysis with a random effects model was used to calculate a weighted-pooled proportion/efficacy for the study's primary outcome. Fifty-seven patients in 17 reports received TPE as an adjunctive treatment to prevent HIT-related thrombosis related to heparinization during CPB and 3 (5.3%) experienced thrombosis. Proportional meta-analysis suggested a weighted-pooled freedom from perioperative thromboembolism rate of 91.0% (95% CI 82.6%-96.9%). Fifteen patients in 6 reports received intravenous cangrelor as an adjunctive treatment to prevent HIT-related thrombosis related to heparinization during CPB and 2 (13.3%) experienced thrombosis. Proportional meta-analysis suggested a weighted-pooled freedom from perioperative thromboembolism rate of 83.0% (95% CI 61.2%- 97.6%). CONCLUSIONS: TPE and cangrelor are feasible strategies to prevent thromboembolism in adults with HIT who require CPB with heparin. Given the relatively small number of cases in the published literature and a high likelihood for publication and detection biases, prudence remains warranted when using these strategies.

Clogged Arteries, Safety Net Holes: Treating an Underinsured Patient for Homozygous Familial Hypercholesterolemia With Plasmapheresis.

This case report explores the difficulties with rapid lipid-lowering therapies in a resource-limited setting. We present a case of an individual with previously diagnosed homozygous familial hypercholesterolemia presenting with anginal chest pain concerning for non-ST elevation myocardial infarction (NSTEMI), with a low-density lipoprotein (LDL) of 984 mg/dL (reference range: 100-129 mg/dL) and a reversible perfusion defect on his nuclear medicine stress test. In addition to the standard treatment for NSTEMI, including cardiac catheterization, the patient was initiated on a proprotein convertase subtilisin/kexin type 9 inhibitor and underwent two rounds of plasmapheresis, which effectively and rapidly lowered his LDL levels.

Ineffectiveness of therapeutic plasma exchange as a last resort in severe COVID-19 cases: Experience from a tertiary intensive care unit.

AIM: Several studies have suggested that cytokine release syndrome (CRS) can be controlled by therapeutic plasma exchange (TPE) treatment. In this study, it was aimed to evaluate the efficacy of TPE treatment in patients who developed life-threatening respiratory failure syndrome (SARS) due to COVID-19 infection. METHODS: In this retrospective, case-control study, patients, who developed SARS, were infected with the COVID-19 virus, and required intensive care unit (ICU) admission were included. Patients included in the study were divided into groups according to whether TPE experience or not and if so, how many sessions were applied. Mortality rates of patients in the ICU and 30-day mortality ratios were evaluated. RESULTS: A total of 110 patients, 71.8% of whom were male, with a mean age of 59.7 ± 13.3 years, were included in our study. It was observed that 70% of the patients died within a month and 80% of them died during the ICU follow-up period. The 30-day mortality rates of patients who underwent TPE at least once and those who never underwent TPE were 72.2% and 67.9%, respectively (p: 0.617). CRP, D-dimer, fibrinogen and platelet levels showed to have a decreasing trend after plasmapheresis and fluctuated thereafter. It was observed that procalcitonin and IL-6 levels were increased in the group that underwent plasmapheresis but decreased in those who did not receive plasmapheresis. CONCLUSION: Patients severely infected with SARS-CoV-2 showed fluctuations in inflammatory parameters despite TPE treatment; CRS was not suppressed by TPE; and this treatment did not confer survival benefit in this patient group.

Exploring the role of plasmapheresis prior to thyroidectomy in managing thyrotoxicosis: a comprehensive scoping review.

A thyroid storm is the most extreme and life-threatening presentation of thyrotoxicosis. Thyroidectomy can be used for definitive treatment. It should be performed after euthyroidism is accomplished. The use of therapeutic plasma exchange (TPE) is a last resort option in cases where standard pharmacological therapy proves to be ineffective. Due to its rare prevalence, there are limited data evaluating the usefulness and efficacy of TPE as a bridging therapy to thyroidectomy. The absence of relevant literature prompted us to conduct a scoping review. The following bibliographic databases were searched for articles dated 30 November 2023: Medline, EMBASE, Web of Science and Google Scholar. The search identified 1047 records, of which 42 articles were accepted with a total of 234 patients. The dominant indications for TPE were side effects due to conventional treatment. The mean fT4 level decreased 51.9% of baseline after TPE, while the mean fT3 level decreased 66.6% of baseline. The main side effects observed with FFP were allergic reactions, while the use of an albumin solution was associated with perioperative bleeding. Based on the limited data available in the literature, we recognize plasmapheresis as an effective treatment option for reducing thyroid hormone levels prior to thyroidectomy in patients with thyrotoxicosis. Available data suggest that it might be reasonable to limit the number of sessions in favor of an earlier surgical intervention. To reduce the risk of bleeding, FFP may be a better option as a replacement fluid, especially in the session prior to thyroidectomy.

Effectiveness of double-filtration plasmapheresis in reducing immunoglobulin and culprit antibody levels in neuroimmune disorders: A single-center retrospective analysis from China.

OBJECTIVE: This study aims to evaluate the effectiveness of double-filtration plasmapheresis (DFPP) in reducing immunoglobulins and culprit antibodies in neuroimmune disorders. METHODS: A retrospective analysis was conducted on 51 patients with neuroimmune diseases treated with DFPP, immunotherapy, and symptomatic treatment. Immunoglobulin and antibody levels were measured pre- and post-treatment, along with neurological function assessments using scales like the modified Rankin Scale (mRS), Expanded Disability Status Scale (EDSS), Clinical Assessment Scale for Autoimmune Encephalitis (CASE), and Myasthenia Gravis-specific scales. RESULTS: The cohort included patients with neuromyelitis optica spectrum disorder (NMOSD), autoimmune encephalitis (AIE), myasthenia gravis (MG), anti-myelin oligodendrocyte glycoprotein associated disease (MOGAD), and paraneoplastic neurological syndromes (PNS). DFPP significantly reduced immunoglobulin levels (IgG, IgA, IgM) by ∼70 %. Most patients showed decreased antibody titers and significant neurological improvement. The median mRS score improved from 2 (IQR 2-3) to 1 (IQR 1-2) post-treatment, with further improvement at 90 days. Notable improvements were observed across various scales specific to NMOSD, MOGAD, AIE, and MG. Minor adverse events were reported, with no serious adverse events. CONCLUSIONS: DFPP is effective in reducing immunoglobulin and antibody levels, leading to improved neurological function in neuroimmune disorders. Further large-scale studies are warranted to confirm these findings.

Necrotizing Pancreatitis Due to Very High Triglyceride Level: A Case Report.

In the United States, acute pancreatitis is one of the most common gastrointestinal conditions that results in hospital admission. Necrotizing pancreatitis is a form of acute pancreatitis that can lead to various local and systemic complications. It is also associated with a high risk of mortality and morbidity without prompt intervention. In this case report, we discuss the case of a 33-year-old female with a history of alcoholism hospitalized with necrotizing pancreatitis due to hypertriglyceridemia. Our goal was to promptly identify the case by evaluating the signs and symptoms and intervening to prevent the associated complications. Our other objective was to change the diet and lifestyle of the patient to prevent the recurrence of necrotizing pancreatitis and readmission for the same reason.

Clinical application of centrifugal-membrane hybrid plasmapheresis in the treatment of hyperlipidemia.

OBJECTIVE: This study aimed to clarify the clinical application of centrifugal-membrane hybrid plasmapheresis (CMHP) in the treatment of hyperlipidemia. METHODS: A retrospective study was conducted on 48 patients who were diagnosed with hyperlipidemia and had received CMHP treatment. Serum total cholesterol (TC), triglycerides (TG), high-density lipoprotein cholesterol (HDL-C), and low-density lipoprotein cholesterol (LDL-C) were monitored, and adverse reactions to the treatment were observed. RESULTS: Forty-eight patients with hyperlipidemia received CMHP over 59 sessions. The average age of the 48 patients with hyperlipidemia, including 32 males (66.67%) and 16 females (33.33%), was 44.23 ± 12.02 years. Twenty-nine outpatients (60.42%) and 19 inpatients (39.58%) were included. Hypertriglyceridemia was diagnosed in 16 cases (33.33%), mixed hyperlipidemia in 31 cases (64.58%), and hypercholesterolemia in one case (2.08%). The pretreatment blood lipid concentrations were significantly different after the 59 CMHP treatments (p < .001). The concentrations of TC, TG, HDL-C, and LDL-C decreased significantly after the treatment, and the median ratios of reduction were 67.06% (range: 58.97%-71.87%), 63.33% (range: 55.20%-74.86%), 45.87% (range: 35.86%-52.95%), and 66.09% (range: 44.37%-73.94%), respectively. Three adverse reactions (5.08%) were recorded. No differences were detected in therapeutic parameters, effects, or adverse reactions between the two blood cell separators, there was no difference in Lipoprotein apheresis efficacy. CONCLUSION: This preliminary study demonstrated the clinical application of CMHP in patients in the treatment of hyperlipidemia. However, further studies are needed applying CMHP with hyperlipidemia.

Management and long-term outcome of recurrent idiopathic FSGS in pediatric kidney transplant recipients.

Focal segmental glomerulosclerosis (FSGS) is a major cause of pediatric kidney failure. Most cases of FSGS in children are idiopathic and have a high risk of post-transplantation recurrence and graft loss. Common treatments for recurrent FSGS (rFSGS) post-transplantation include plasmapheresis, immunoadsorption, and/or immunomodulatory therapy. This study retrospectively evaluated the efficacy and safety of early plasmapheresis followed by rituximab for inducing and maintaining remission in rFSGS. Between 2014 and 2023, 8 of 65 pediatric kidney transplant recipients at our center were diagnosed with idiopathic FSGS. rFSGS was diagnosed based on nephrotic range proteinuria with no other cause and managed with plasmapheresis. Rituximab therapy was used for those who did not achieve complete remission with prolonged plasmapheresis or remained plasmapheresis dependent. 6 of 8 (75%) transplant recipients with idiopathic FSGS experienced rFSGS. All patients achieved partial or complete remission with plasmapheresis, with response times ranging from 8 to 379 days (median 13 days). Rituximab therapy was introduced for 5 plasmapheresis-dependent patients, leading to sustained remission and cessation of plasmapheresis in 3 patients, while 2 showed improved proteinuria and reduced plasmapheresis frequency. Adverse effects included rituximab-induced serum sickness in one patient and one mild allergic reaction. One patient experienced graft loss due to humoral rejection, but no grafts were lost to rFSGS, and all other grafts remained functional over an average follow-up of 50 months. Early plasmapheresis followed by rituximab therapy effectively induces remission in most post-transplantation rFSGS cases, is well tolerated, and prevents graft loss. Larger studies are needed to confirm these findings.

Neuromyelitis optica spectrum disorder after post appendectomy peritonitis: A case report.

Neuromyelitis optica spectrum disorder (NMOSD), also known as "Devic's syndrome", is an autoimmune demyelination disorder. It affects the optic nerve and spinal cord, causing optic neuritis and transverse myelitis. It is associated with anti-aquaporin 4 antibodies that target the aquaporin channel on astrocytes. An 18-year-old male with a history of appendectomy 6 months ago presented with neck pain, numbness in limbs up to the umbilicus, paresis, and spasticity in the left leg. The brain magnetic resonance imaging (MRI) was normal, while the spinal cord MRI showed hyperintense foci at the T2 level. Cerebrospinal fluid (CSF) analysis was in the normal range and negative for oligoclonal bands. The serological assay was positive for anti-aquaporin-4 antibodies (AQP4-IgG). The patient improved significantly after administering high doses of methylprednisolone and supplements. Due to the unavailability of eculizumab, he underwent plasmapheresis sessions to remove antibodies, which improved to a reasonable extent. NMOSD most commonly targets older females, but in our report, it appeared in a young male. The patient only presented with transverse myelitis with no ophthalmologic problem. He made significant improvement with combination treatment of steroids, supplements, and plasmapheresis.

New insights into the use of high dose corticosteroids and plasmapheresis in persons with MOGAD and NMOSD.

BACKGROUND: Anti-myelin oligodendrocyte glycoprotein associated disease (MOGAD) and neuromyelitis optica spectrum disease (NMOSD) are antibody mediated diseases characterized by neurological symptoms including recurrent relapses of optic neuritis and/or myelitis, as well as other less frequent syndromes. The current treatment for acute attacks of NMOSD/MOGAD are based on clinical studies for other demyelinating diseases(i.e. Multiple Sclerosis). In NMOSD, high dose corticosteroids (HDS) are considered the standard first line therapy, with emerging evidence supporting the use of plasmapheresis (PLEX) as an acute therapy. In MOGAD, being a relatively new clinical syndrome, the consensus on acute treatments is yet to be reached. The objective of our study was to assess the efficacy of treatment regimens (no treatment vs. HDS vs. HDS and PLEX) on disability outcomes in persons with NMOSD and MOGAD-optic neuritis and myelitis. METHODS: We retrospectively extracted data from the MuSicaL-NeMo database using a mixed Natural Language Processing followed by investigators verification. We assessed the change in Expanded Disability Status Scale (EDSS) and Visual Acuity (VA) following HDS and PLEX, in persons with MOGAD and NMOSD following myelitis and optic neuritis. We used the novel statistical measure Wilcoxon-Mann-Whitney Odd (WMW-Odd) to calculate the change through all the spectrum of each ordinal scale (VA and EDSS). RESULTS: Eleven myelitis and 12 optic neuritis in 22 persons with MOGAD and 30 myelitis and 12 optic neuritis in 20 persons with NMOSD were included(15 Aquaporin-4 seropositive). In persons with MOGAD-optic neuritis the group receiving HDS had a WMW-Odd of 15.33(p ≤ 0.001), however those not receiving treatment also tended to improve (WMW-Odd=3.17, p = 0.06). NMOSD-optic neuritis treated with HDS only improve 33.3 % of the times (p=NS). Persons with MOGAD-myelitis receiving HDS significantly improved (WMW-Odd=7.33, p = 0.002). Persons with NMOSD-myelitis treated with HDS had an WMW-Odd of 2.56 (p = 0.002) and those treated with PLEX plus HDS (PLEX+), had similar WMW-Odd of 2.51 (p = 0.03). When correcting for disease severity by restricting inclusion to persons with NMOSD with EDSS≥4, both treatments showed a higher WMW-Odd, however the group receiving HDS continued to show higher WMW-Odd than the PLEX+ group(WMW-Odd= 3.75, p = 0.002 vs. WMW-Odd =3.05, p = 0.02, respectvely) CONCLUSION: Our study suggests that persons with MOGAD-optic neuritis improve without acute treatments, however they have very marked improvement when using HDS, as previously suggested. Patient with MOGAD-myelitis are also very responsive to HDS, however, as compared to MOGAD-optic neuritis, they displayed less improvement, if not treated. In the NMOSD group the use of PLEX in addition to HDS did not demonstrate any significant difference in EDSS outcomes. Contrary to previous suggestions, when adjusting for group differences (by only including EDSS ≥4), the use of HDS and PLEX+ did not show better results than the group using HDS.

Effect of therapeutic plasma exchange on tissue factor and tissue factor pathway inhibitor in septic shock.

BACKGROUND: Coagulopathy is part of the pathological host response to infection in sepsis. Higher plasma concentrations of both tissue factor (TF) and tissue factor pathway inhibitor (TFPI) are associated with occurrence of disseminated intravascular coagulation (DIC), multi-organ dysfunction and increased mortality in patients with sepsis. Currently no treatment approaches specifically targeting this axis are available. We hypothesize that therapeutic plasma exchange (TPE) might limit this coagulopathy by restoring the balance of plasma proteins. METHODS: This was a pooled post-hoc biobank analysis including 51 patients with early (shock onset < 24 h) and severe (norepinephrine dose > 0.4 µg/kg/min) septic shock, who were either receiving standard of care treatment (SOC, n = 14) or SOC + one single TPE (n = 37). Plasma concentrations of TF and TFPI were measured both at- and 6 h after study inclusion. The effect of TPE on concentrations of TF and TFPI was investigated and compared to SOC patients. Further, baseline TF and TFPI concentrations were used to modulate and predict clinical response to adjunctive TPE, indicated by longitudinal reduction of lactate concentrations over the first 24 h following study inclusion. RESULTS: TPE led to a significant reduction in circulating concentrations of both TF and TFPI while no difference was observed in the SOC group. Relative change of TF within 6 h was + 14 (-0.8 to + 30.4) % (p = 0.089) in the SOC and -18.3 (-32.6 to -2.2) % (p < 0.001) in the TPE group (between group p < 0.001). Similarly, relative change of TFPI was + 14.4 (-2.3 to + 30.9) % (p = 0.076) in the SOC and -20 (-32.8 to -7.9) % (p < 0.001) in the TPE group (between group p = 0.022). The ratio of TF to TFPI remained unchanged in both SOC and TPE groups. SOC patients exhibited an increase in lactate over the initial 24 h when TF and TFPI concentrations were higher at baseline. In contrast, patients undergoing TPE experienced a sustained longitudinal reduction of lactate concentrations across all levels of baseline TF and TFPI elevations. In a multivariate mixed-effects model, higher baseline TF (p = 0.003) and TFPI (p = 0.053) levels led to greater longitudinal lactate concentration reduction effects in the TPE group. CONCLUSIONS: Adjunctive TPE in septic shock is associated with a significant removal of both TF and TFPI, which may contribute to the early hemodynamic improvement observed in septic shock patients receiving TPE. Higher baseline TF (and TFPI) plasma concentrations were identified as a putative predictor of treatment response that could be useful for predictive enrichment strategies in future clinical trials.

Obstetrical Management of Severe Hypertriglyceridemia in Pregnancy: A Case Report.

Background Pregnant people with baseline hypertriglyceridemia are at increased risk of severe hypertriglyceridemia and the associated complications, yet there are no formal recommendations to guide management of these patients during pregnancy. Case We report a case of a patient with presumed familial hypertriglyceridemia who was taken off triglyceride-lowering medications preconception and developed acute pancreatitis at 23 weeks of gestation. She was managed with a very-low-fat diet, exercise, fenofibrate, omega-3-fatty acids, pravastatin, insulin infusion, and plasmapheresis. She delivered at 33 weeks of gestation after presenting with a placental abruption and subcapsular liver hematoma associated with HELLP (hemolysis, elevated liver enzyme levels, and low platelet) syndrome. Conclusion While rare in pregnancy, severe hypertriglyceridemia is associated with serious maternal risks. Preconception and antepartum obstetric management should incorporate shared decision-making considering both the potential fetal risks of treatment and the objective maternal risks of untreated disease.

Viscous Vengeance: A Case Report of Hyperviscosity Syndrome in IgA Lambda Multiple Myeloma.

Multiple myeloma (MM) is a relatively uncommon neoplastic proliferation of monoclonal plasma cells. Common manifestations are related to infiltration of plasma cells into bones and other organs, causing an increased total serum protein concentration, hypercalcemia, kidney injury, and anemia. The most common type of MM is IgG Kappa, and the second most common type is the IgA subtype. Hyperviscosity (HVS) is a rare presentation of MM and management includes prompt plasmapheresis, resulting in significant reduction of serum viscosity and symptomatic relief. We present a case of IgA Lambda MM presenting with HVS.

Deciphering Complexity: Atypical Hemolytic Uremic Syndrome Unraveled in the Wake of Elective Hip Arthroplasty.

Atypical hemolytic uremic syndrome (aHUS) is a rare and complex disease characterized by microangiopathic hemolytic anemia, thrombocytopenia, and acute renal failure. This case report details the clinical presentation, diagnosis, and management of a 49-year-old female who developed aHUS following elective hip arthroplasty. The patient, with a history of cardiovascular events and no prior renal disease, presented with elevated LDH levels, thrombocytopenia, and acute renal failure on the first postoperative day. A diagnostic workup confirmed aHUS, and the patient was successfully treated with therapeutic plasma exchange (TPE) and hemodialysis. The case underscores the importance of early recognition and aggressive management of aHUS, especially in the perioperative setting, and highlights the need for a multidisciplinary approach to optimize patient outcomes. Through this case, we aim to raise awareness about the potential for surgical stress to trigger aHUS and emphasize the critical role of TPE and supportive care in the treatment of this rare condition.

Lipoprotein apheresis: an established therapeutic modality for homozygous familial hypercholesterolemia patients refractory to PCSK9 inhibitors: a case report and literature review.

Homozygous familial hypercholesterolemia (HoFH), is a rare genetic disorder characterized by dual mutations in the low-density lipoprotein receptor (LDLR) gene, leading to dysfunctional or absent LDLRs, often accompanied by severe premature Atherosclerotic Cardiovascular Disease (ASCVD) and exhibiting refractoriness to aggressive pharmacological interventions. Double filtration plasmapheresis (DFPP), a form of lipoprotein apheresis (LA), has been effectively utilized as an adjunctive treatment modality to reduce serum LDL-C levels in refractory cases of HoFH. Here, we report a case of a 36-year-old female with HoFH who developed xanthomas on her limbs and waist at age 7. Despite maximum-tolerated doses of statins from age 32, combined with ezetimibe and evolocumab, her LDL-C levels remained critically elevated at 12-14 mmol/L. Her genetic testing confirmed a homozygous LDLR mutation. At 35 years old, she experienced exertional chest pain, and percutaneous coronary intervention revealed severe calcific left main stenosis, necessitating stent implantation. Subsequently, she initiated once every 1-2 months DFPP. Pre-DFPP, her LDL-C and total cholesterol (TC) levels were 13.82 ± 3.28 and 15.45 ± 0.78 mmol/L, respectively. Post-DFPP, her LDL-C and TC levels significantly decreased to 2.43 ± 0.33 mmol/L (81.76 ± 4.11% reduction) and 3.59 ± 0.41 mmol/L (76.76 ± 2.75% reduction), respectively. Lipoprotein (a) and triglycerides also decreased by 89.10 ± 1.39% and 42.29 ± 15.68%,respectively. Two years later, there was no progression of coronary artery disease, and her symptoms and xanthomas regressed significantly. Collectively, DFPP effectively reduces LDL-C levels in refractory cases of HoFH and contributes to delaying ASCVD progression, representing an efficacious adjunctive therapeutic modality.

Outcome of rituximab treatment in children with non-dialysis-dependent anti-GBM disease.

BACKGROUND: Anti-GBM disease is a rare vasculitis mediated by pathogenic antibodies against collagen IV. Anti-GBM disease presents with rapid progressive glomerulonephritis and leads to kidney failure if untreated. KDIGO recommends plasma exchanges (PEX) for antibody elimination and steroids plus cyclophosphamide (CTX) to suppress antibody production. CTX is associated with severe side effects including gonadal toxicity. Rituximab (RTX) and mycophenolate mofetil (MMF) might be a less toxic but equally efficient alternative to CTX. Studies in pediatric anti-GBM disease patients receiving RTX and MMF instead of CTX are lacking. METHODS: A retrospective survey in 8 tertiary German centers was performed. The clinical data of patients diagnosed between 2014 and 2022 were collected and analyzed. RESULTS: Five adolescent patients treated with PEX and RTX and/or MMF due to anti-GBM disease were analyzed. All patients had anti-GBM antibodies, hematuria, glomerular proteinuria, and pulmonary hemorrhage. eGFR was 124 ml/min/1.73 m2 (range 47-162), and all patients were non-dialysis-dependent but with relevant histological kidney affection (mean crescents on kidney biopsy 77%). Antibody clearance was achieved after 13 PEX cycles (range 6-31). Four out of 5 patients received methylprednisolone pulses. All patients received oral prednisolone and MMF, and four patients received a median of 4 RTX doses (range 2-4). After a mean follow-up of 27 months, 4/5 patients had conserved or improved kidney function, while one patient (20%) developed kidney failure. CONCLUSIONS: In this small series of pediatric non-dialysis-dependent anti-GBM disease patients, first-line treatment with RTX and MMF showed a favorable kidney outcome in 4/5 cases and had an acceptable side effect profile.