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Background: It is unknown if glucocorticoid malabsorption contributes to the approximate 50% treatment failure rate in dogs with protein-losing enteropathy (PLE)., Objective: To compare pharmacokinetics (PK) of orally administered prednisolone in dogs with PLE vs healthy controls., Animals: Fourteen dogs with well-characterized PLE and 7 control dogs., Methods: Prospective case-controlled study. Dogs were treated with 1 mg/kg prednisolone PO once daily for approximately 3 weeks. Venous blood samples were collected at set timepoints before and after prednisolone administration on the first (T1) and final (T2) study days. Total and non-protein bound serum prednisolone concentrations were determined using liquid chromatography tandem-mass spectrometry, and pharmacokinetics variables were derived from the drug concentration data. Pharmacokinetics variables were compared between PLE and control dogs and between PLE short-term responders and non-responders., Results: The PLE dogs had a shorter half-life of the terminal slope than control dogs (harmonic mean of 1.3 vs 1.8 hours; P = .05) whereas the percentage of serum prednisolone that was non-protein bound was higher in PLE dogs than in control dogs (median of 15.7% vs 6.7%; P = .02) at T1. Total prednisolone drug exposures and maximum total serum drug concentrations did not differ between PLE and control dogs at T1 or T2, nor did they differ between short-term responders and non-responders within the PLE population (P > .05 for all comparisons)., Conclusions and Clinical Importance: Overall drug exposures are similar between PLE dogs and healthy controls. Glucocorticoid malabsorption is unlikely to be a common cause of treatment failure in dogs with PLE., (© 2024 The Author(s). Journal of Veterinary Internal Medicine published by Wiley Periodicals LLC on behalf of American College of Veterinary Internal Medicine.)

Background: Atypical hemolytic uremic syndrome (aHUS) is a life-threatening thrombotic microangiopathy. Genetic defects in the alternative complement (AP) pathway have been identified in 60-70% of individuals. Eculizumab is recommended as a first-line therapy., Methods: We collected the clinical data of a pediatric patient with aHUS accompanied by protein-losing enteropathy (PLE). Genetic testing was performed. Related literature on aHUS combined with PLE was reviewed., Results: A 15-year-old Chinese girl was diagnosed with aHUS at 3.7 years of age and experienced five episodes; her symptoms completely resolved with plasma treatment. Severe gastrointestinal symptoms and hypoalbuminemia presented after the first episode, and PLE was diagnosed. A novel homozygous CD46 variant was identified, and FACS revealed significantly decreased CD46 expression. She presented at a recent relapse with persistent GI symptoms and headache and progressed to chronic kidney failure; peritoneal dialysis was initiated. Eculizumab was given 8 months after the last recurrence. Surprisingly, PLE was cured. Afterward, dialysis was discontinued, and eGFR recovered to 44.8 ml/min/1.73 m 2 . A review of the literature indicated that PLE with thrombosis was caused by CD55 variants via hyperactivation of the AP system. We report an aHUS patient with PLE caused by CD46 variants. Symptoms of both PLE and aHUS were significantly alleviated in our patient and patients with CD55 variants treated with eculizumab, indicating that PLE was a new symptom of aHUS in our patient with a CD46 variant., Conclusions: Our case expands the phenotype of aHUS caused by a CD46 mutation and provides evidence of the efficacy of eculizumab after a long phase of chronic kidney failure., (© 2024. The Author(s), under exclusive licence to International Pediatric Nephrology Association.)

Pozelimab, a monoclonal antibody directed against C5, is the first and only treatment for adult and pediatric patients (≥ 1 year) with CD55-deficient protein-losing enteropathy (CHAPLE) disease. A target-mediated drug disposition (TMDD) population pharmacokinetic (PopPK) model was developed using pooled data from four phase 1-3 studies to characterize the pharmacokinetics (PK) of total pozelimab and total C5, and to simulate free pozelimab and free C5 to support the dose regimen in patients with CHAPLE disease. A TMDD PopPK model was developed using total pozelimab and total C5 concentration-time data from 106 participants (82 healthy volunteers; 24 patients with paroxysmal nocturnal hemoglobinuria [PNH]). This model was refined and updated to include PK data from 10 patients with CHAPLE disease from a phase 2/3 study. Stochastic simulations predicted concentration-time profiles for total pozelimab, free pozelimab, and free C5, to obtain pozelimab exposure metrics for patients with CHAPLE disease. A two-compartment TMDD model with two binding sites based on the quasi-equilibrium approximation adequately described the concentration-time profiles of total pozelimab and total C5. Body weight was identified as the most important source of pozelimab PK variability; therefore, the dose was adjusted based on body weight for the predominantly pediatric patients with CHAPLE disease. A robust TMDD PopPK model was developed to describe the PK of total pozelimab and total C5 following pozelimab administration. Reliable predictions for individual exposures of total pozelimab and free C5 were possible and supported the 10 mg/kg weight-based dose regimen in patients with CHAPLE disease., Competing Interests: Declarations. Ethical approval: All studies were conducted in accordance with ethical principles of the Declaration of Helsinki, and were consistent with International Conference on Harmonisation, Good Clinical Practices, and applicable regulatory requirements. Consent to participate: All participants provided consent prior to enrolling in these studies. Competing interests: Kuan-Ju Lin, Jeanne Mendell, John D. Davis, and Lutz Harnisch are all employees of and stockholders in Regeneron Pharmaceuticals, Inc., (© 2024. The Author(s).)

Lupus protein-losing enteropathy (LUPLE) is a rare condition in patients with systemic lupus erythematosus (SLE). Since the causes and exact pathological mechanism have not been elucidated, appropriate treatment has not been determined. Here, we report the case of a 69-year-old woman with systemic lupus erythematosus who developed LUPLE which was successfully treated with belimumab without an increase in glucocorticoid dose. This case suggests that belimumab monotherapy may be a treatment option for LUPLE., (© Japan College of Rheumatology 2024. Published by Oxford University Press.)

Protein-losing enteropathy is a severe complication of Fontan surgery and is associated with anaemia. Few studies have reported on the efficacy of an intravenous iron infusion for treating protein-losing enteropathy and low albuminemia after Fontan surgery. Herein, we present two cases of female patients who suffered from protein-losing enteropathy and low albuminemia following Fontan surgery, both of whom improved after an intravenous iron infusion.

Objectives: Determine comparative tolerance of daily oral and weekly parenteral cobalamin supplementation, in hypocobalaminaemic dogs with chronic enteropathy. Determine whether oral is as effective as parenteral supplementation at achieving eucobalaminaemia, in hypocobalaminaemic dogs with protein-losing enteropathy, severe hypocobalaminaemia or high canine inflammatory bowel disease activity index at inclusion., Materials and Methods: Thirty-seven client-owned dogs with hypocobalaminaemia and clinical signs of chronic enteropathy were prospectively enrolled in three UK referral centres. Dogs were randomly allocated to daily oral for 12 weeks or weekly parenteral cobalamin supplementation for 6 weeks and one additional dose 4 weeks later. Serum cobalamin, body condition score, canine inflammatory bowel disease activity index and bodyweight were assessed at inclusion, weeks 7 and 13. Serum methylmalonic acid concentration was evaluated at inclusion and at week 13. Owners completed treatment adherence, palatability, tolerance and satisfaction questionnaires at week 13., Results: Nineteen dogs completed the study. All dogs orally supplemented achieved normal or increased cobalaminaemia at weeks 7 and 13. There was no statistical difference in cobalamin concentration at week 13 in dogs treated with oral or parenteral supplementation, regardless of presence of protein-losing enteropathy, severity of hypocobalaminaemia or canine inflammatory bowel disease activity index at inclusion. Serum methylmalonic acid concentration was not significantly different between oral and parenteral groups, neither were treatment adherence, satisfaction, and tolerance scores at week 13., Clinical Significance: Oral is as effective and as well-tolerated as parenteral cobalamin supplementation in hypocobalaminaemic dogs with chronic enteropathy and severe clinical or biochemical phenotypes, and should be considered as a suitable treatment option regardless of disease severity., (© 2024 The Authors. Journal of Small Animal Practice published by John Wiley & Sons Ltd on behalf of British Small Animal Veterinary Association.)

The complement is a crucial factor of the innate immune system. However, its activation can lead to various diseases, so it needs to be controlled. In mammals, surface-bound complement regulatory proteins safeguard cells from uncontrolled complement-mediated lysis. One of the human complement regulators is CD55, also known as the decay-accelerating factor (DAF), a single-chain, type I cell surface protein anchored to glycosylphosphatidylinositol (GPI). The genetic loss of the complement regulatory protein CD55 leads to a fatal illness known as CHAPLE disease. The complement and innate immunity become hyperactive in this disease, causing angiopathic thrombosis and protein-losing enteropathy. Patients with CHAPLE disease experience abdominal pain, nausea, vomiting, diarrhea, loss of appetite, weight loss, impaired growth, and swelling. This genetic condition has no known cure, and managing its symptoms can be challenging. Pozelimab, a human monoclonal immunoglobulin IgG4 antibody, is a drug that targets the terminal complement protein C5. The drug has a high affinity for both wild-type and variant human C5. Pozelimab has received designations such as fast track, orphan drug, and rare pediatric disease, making it a significant medical breakthrough. It is currently the only available treatment for this disease. In this review, we have summarized the preclinical and clinical data on pozelimab., (© 2024 Walter de Gruyter GmbH, Berlin/Boston.)

Background: CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) is an ultra-rare genetic disorder characterised by intestinal lymphatic damage, lymphangiectasia, and protein-losing enteropathy caused by overactivation of the complement system. We assessed the efficacy and safety of pozelimab, an antibody blocking complement component 5., Methods: This open-label, single-arm, historically controlled, multicentre phase 2 and 3 study evaluated ten patients with CHAPLE disease. This study was conducted at three hospitals in Thailand, Türkiye, and the USA. Patients aged 1 year or older with a clinical diagnosis of CHAPLE disease and a CD55 loss-of-function variant identified by genetic analysis and confirmed by flow cytometry or western blot of CD55 from peripheral blood cells were eligible for this study. Patients received a single intravenous loading dose of pozelimab 30 mg per kg of bodyweight, followed by a once-per-week subcutaneous dose over the treatment period based on bodyweight at a concentration of 200 mg/mL as either a single injection (<40 kg bodyweight) or two injections (≥40 kg bodyweight). The primary endpoint was proportion of patients with serum albumin normalisation with an improvement in active clinical outcomes and no worsening in inactive clinical outcomes (frequency of problematic abdominal pain, bowel movement frequency, facial oedema severity, and peripheral oedema severity) at week 24 compared with baseline, assessed in the full analysis set. This study is registered with ClinicalTrials.gov (NCT04209634) and is active but not recruiting., Findings: 11 patients were recruited between Jan 27, 2020, and May 12, 2021, ten of which were enrolled in the study and included in the analysis populations. The efficacy data corresponded to all patients completing the week 48 assessment and having at least 52 weeks of treatment exposure, and the safety data included an additional 90 days of follow-up and corresponded to all patients having at least 72 weeks of treatment. Patients were predominantly paediatric (with a median age of 8·5 years), and originated from Türkiye, Syria, Thailand, and Bolivia. Patients had markedly low weight-for-age and stature-for-age at baseline, and mean albumin at baseline was 2·2 g/dL, which was considerably less than the local laboratory reference range. After pozelimab treatment, all ten patients had serum albumin normalisation and improvement with no worsening in clinical outcomes. There was a complete inhibition of the total complement activity. Nine patients had adverse events; two were severe events, and one patient had an adverse event considered related to pozelimab., Interpretation: Pozelimab inhibits complement overactivation and resolves the clinical and laboratory manifestations of CHAPLE disease. Pozelimab is the only currently approved therapeutic drug for patients with this life-threatening, ultra-rare condition. In patients with protein-losing enteropathy where known causes have been excluded, testing for a CD55 deficiency should be contemplated. A diagnosis of CHAPLE disease should lead to early consideration of treatment with pozelimab., Funding: Regeneron Pharmaceuticals and the Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health., Competing Interests: Declaration of interests AO is a consultant and steering committee member for Regeneron Pharmaceuticals; received sample analysis support for a previous collaborative study (https://doi.org/10.1038/s41590-020-00830-z) from Regeneron Pharmaceuticals; and has a pending patent on component 5 inhibitor treatment in CHAPLE disease. VC, NS, and KS received support to conduct the study and received provision of the investigational product from Regeneron Pharmaceuticals. JJJ, TB, and LP are Regeneron Pharmaceuticals employees and stockholders. KAM is a Regeneron Pharmaceuticals employee and stockholder and has both pending and issued patents with Regeneron Pharmaceuticals. HBF is a consultant and advisory board member for Regeneron Pharmaceuticals. IJF is an associate on a cooperative research and development agreement between Merck Pharmaceuticals and the National Institutes for Health. OAH and GDY are Regeneron Pharmaceuticals employees and stockholders and have a pending patent on complement component 5 inhibitor treatment in CHAPLE disease. MJL received support for a federally approved cooperative research and development agreement to support the clinical trial and has a pending patent on complement component 5 inhibitor treatment in CHAPLE disease. All other authors declare no competing interests., (Copyright © 2024 Elsevier Ltd. All rights reserved.)

Background: More than 50% of dogs with protein-losing enteropathy (PLE) fail to respond to standard therapies. Octreotide, a somatostatin analogue, is used in cases of intestinal lymphangiectasia (IL) in humans with some success., Objectives: Describe the use of octreotide in dogs with PLE including reason for and details of prescription, adverse effects, and apparent response., Animals: Eighteen dogs with PLE, 13 with histopathology available. Ninety-two percent (12/13) had IL diagnosed on biopsy. All 13 dogs had intestinal inflammatory infiltrates noted., Methods: Multicenter, retrospective, descriptive study. Cases were volunteered for inclusion by individual attending veterinarians who reported the use of octreotide in cases of PLE., Results: In 16/18 (89%) cases octreotide was prescribed to PLE dogs with a clinical suspicion or confirmed diagnosis of IL that were refractory to standard therapies. Median serum albumin at the time of octreotide prescription was 1.7 g/dL (range, 1.0-3.1 g/dL). The median dose of octreotide prescribed was 20 μg/kg, SQ, daily with a range of 4-39 μg/kg, SQ, daily. Adverse effects were noted in 3/18 (17%, 95% CI [4%, 41%]) of dogs; discontinuation of the drug was necessary in 1 dog. Improvement in clinical signs was noted in 6/12 (50%, 95% CI [21%, 79%])., Conclusions and Clinical Importance: Octreotide was most commonly prescribed to dogs with PLE and suspected or confirmed IL that had failed to respond to standard therapies. Though a benefit to PLE dogs cannot be confirmed, octreotide was well tolerated by the majority of dogs at the doses prescribed in this study., (© 2023 The Authors. Journal of Veterinary Internal Medicine published by Wiley Periodicals LLC on behalf of American College of Veterinary Internal Medicine.)

For dogs with protein-losing enteropathy (PLE) and evidence of lymphangiectasia, the efficacy of low-fat diet as monotherapy or combined with prednisone remains poorly characterized. In this prospective, observational cohort study of 14 dogs with presumptive PLE and ultrasonographic evidence of lymphangiectasia, subjects were placed on various low-fat diets as monotherapy and prednisone was added if response was deemed inadequate. Dogs were assessed and scored at four recheck examinations across a 6 mo study period, including a final recheck ultrasound. Clinical and clinicopathologic variables were collected and dogs were divided into three outcome groups: clinical remission on dietary monotherapy (LOF); clinical remission on dietary therapy plus immunosuppressive prednisone (LOP); and treatment failure (TXF). Eleven of 14 dogs were in clinical remission at the study end date (6 mo after enrollment): 6 LOF dogs and 5 LOP dogs. LOF dogs achieved a significant reduction in Canine Chronic Enteropathy Clinical Activity Index score and a significant increase in serum albumin within 2 wk of beginning dietary monotherapy. Four of 11 dogs in remission also had ultrasonographic evidence of resolution of linear striations. Low-fat diet appears to be an effective monotherapy in some dogs with presumptive PLE and ultrasonographic evidence of lymphangiectasia., (© 2023 by American Animal Hospital Association.)

Background: Protein-losing enteropathy (PLE) is characterized by leakage of serum proteins into the intestinal lumen, indicating hypoproteinemia. Immunosuppressive agents are the mainstay of treatment, but in many cases, patients are forced to taper off early owing to the induction of liver damage., Case Description: An 8-year-old, non-spayed female Chihuahua presented with diarrhea and ascites effusion lasting 2 weeks. Based on the results of radiography and blood tests, a diagnosis of PLE was made. Prednisolone (3 mg/kg semel in die [SID]) and MitoMax (200 mg/day) were administered, but ascites accumulation and diarrhea did not improve. Thus, azathioprine (2 mg/kg/day) was added, but there was no improvement, and liver damage developed. The liver injury did not improve immediately, but diarrhea and ascites effusion improved after serum total protein and serum albumin levels increased after they had decreased. Subsequent tapering of prednisolone from 3 mg/kg SID to 1 mg/kg SID, combined with MitoMax (200 mg/day) and equine placenta extract (eqPE) (2 ml/day), resulted in no recurrence of ascites or diarrhea., Conclusion: In canine PLE with prolonged diarrhea and ascites effusion, supplementation with eqPE may be considered a reasonable additional therapeutic strategy., Competing Interests: Nobuhisa Kakehi, Tsuyuko Watanabe, Kentarou Tahara, and Eiichi Hirano are employees of the Japan Bio Products Co., Ltd. Naoki Fukushima has no competing interests.

Objectives: To expand the number of conditions and interventions explored for their associations with thrombosis in the veterinary literature and to provide the basis for prescribing recommendations., Design: A population exposure comparison outcome format was used to represent patient, exposure, comparison, and outcome. Population Exposure Comparison Outcome questions were distributed to worksheet authors who performed comprehensive searches, summarized the evidence, and created guideline recommendations that were reviewed by domain chairs. The revised guidelines then underwent the Delphi survey process to reach consensus on the final guidelines. Diseases evaluated in this iteration included heartworm disease (dogs and cats), immune-mediated hemolytic anemia (cats), protein-losing nephropathy (cats), protein-losing enteropathy (dogs and cats), sepsis (cats), hyperadrenocorticism (cats), liver disease (dogs), congenital portosystemic shunts (dogs and cats) and the following interventions: IV catheters (dogs and cats), arterial catheters (dogs and cats), vascular access ports (dogs and cats), extracorporeal circuits (dogs and cats) and transvenous pacemakers (dogs and cats)., Results: Of the diseases evaluated in this iteration, a high risk for thrombosis was defined as heartworm disease or protein-losing enteropathy. Low risk for thrombosis was defined as dogs with liver disease, cats with immune-mediated hemolytic anemia, protein-losing nephropathy, sepsis, or hyperadrenocorticism., Conclusions: Associations with thrombosis are outlined for various conditions and interventions and provide the basis for management recommendations. Numerous knowledge gaps were identified that represent opportunities for future studies., (© 2022 The Authors. Journal of Veterinary Emergency and Critical Care published by Wiley Periodicals LLC on behalf of Veterinary Emergency and Critical Care Society.)

Background: Protein-losing gastroenteropathy (PLGE) is a syndrome with a chief complaint of hypoalbuminemia, which occurs due to plasma protein leakage in the gastrointestinal tract, leading to general edema, ascites, and pleural effusions., Case Presentation: A 71-year-old woman visited another hospital for evaluation of hypoalbuminemia and systemic edema. She was hospitalized for a close inspection of hypoalbuminemia and was diagnosed with PLGE. Steroid and azathioprine therapy was prescribed; however, hypoalbuminemia did not improve, and the patient's condition worsened due to anasarca. As hospitalization was prolonged, the patient was transferred to our hospital. She was infected with Helicobacter pylori, and we performed H. pylori eradication. Following H. pylori eradication, her edema improved remarkably., Conclusion: We present the first case wherein H. pylori eradication successfully improved protein leakage in the lower gastrointestinal tract in a patient diagnosed with PLGE complicated with refractory to immunosuppressant treatment. H. pylori eradication should be considered in patients with PLGE complicated with H. pylori infection, without specific endoscopic finding or refractory to immunosuppressants., (© 2022. The Author(s).)

Protein-losing enteropathy is rarely associated with malignant lymphoma. This report describes the case of a 67-year-old man with diffuse large B-cell lymphoma (DLBCL) and concomitant protein-losing enteropathy who was admitted to our hospital for evaluation of watery diarrhea, edema, and abdominal fullness. On admission, the patient reported a history of weight gain. Subsequent examination showed ascites, hepatosplenomegaly, and hypoalbuminemia. Notably, 99mTc-labeled human serum albumin scintigraphy revealed protein loss from the intestine, and the patient was diagnosed with protein-losing enteropathy. Endoscopy revealed erosive and edematous hyperplasia of the gastric-colonic mucosa, and histopathological evaluation of a biopsy specimen showed proliferation of CD20 + and CD5 + tumor cells. Thus, the diagnosis of DLBCL was histopathologically confirmed. Lymphomatous infiltration of the bone marrow was observed; however, no lymphadenopathy was detected. Based on these findings, the patient was diagnosed with protein-losing enteropathy associated with gastrointestinal infiltration of CD5 + DLBCL. Hypoalbuminemia and diarrhea improved following the initiation of R-CHOP regimen. The DLBCL showed a favorable response to treatment, and gastrointestinal lesions and hepatosplenomegaly improved, along with the resolution of protein-losing enteropathy.

Background: Disseminated Mycobacterium avium complex infection is an important indicator of acquired immunodeficiency syndrome (AIDS) in patients with advanced human immunodeficiency virus (HIV) infection. Effective antiretroviral therapy has dramatically reduced the incidence of and mortality due to HIV infection, although drug resistance and poor medication adherence continue to increase the risk of disseminated M. avium complex infection. However, gastrointestinal lesions in cases of disseminated M. avium complex infection resulting in protein-losing enteropathy have been rarely discussed. Therefore, we present a case of protein-losing enteropathy caused by disseminated M. avium complex infection in a patient undergoing antiretroviral therapy., Case Presentation: A 29-year-old man was diagnosed with AIDS 4 years ago and was admitted for a 10-month history of refractory diarrhea and fever. Despite receiving antiretroviral therapy, the viral load remained elevated due to poor medication adherence. The patient was diagnosed with disseminated M. avium complex infection and started on antimycobacterial drugs 2 years before admission. However, the infection remained uncontrolled. The previous hospitalization 1 year before admission was due to hypoalbuminemia and refractory diarrhea. Upper gastrointestinal endoscopy revealed a diagnosis of protein-losing enteropathy caused by intestinal lymphangiectasia, and treatment with intravenous antimycobacterial drugs did not resolve his intestinal lymphangiectasia. The patient inevitably died of sepsis., Conclusions: Clinical remission is difficult to achieve in patients with AIDS and protein-losing enteropathy caused by disseminated M. avium complex infection due to limited options of parenteral antiretroviral drugs. This report highlights the importance of identifying alternative treatments (such as an injectable formulation) for patients who do not respond to antiretroviral therapy due to protein-losing enteropathy with disseminated M. avium complex infection., (© 2021. The Author(s).)

Objective: To describe the clinical effect of dietary alteration as a sole change to therapy in dogs with steroid-resistant protein-losing enteropathy., Materials and Methods: Prospective study. Eligible enrolled dogs received dietary alteration as sole change to their therapeutic plan. Canine Chronic Enteropathy Clinical Activity Index and serum albumin were monitored for the 3-month study period. Long-term follow-up data were also available for some of the study participants., Results: Fifteen dogs were eligible for enrollment over the study period. Twelve were enrolled, 10 remained in the study at 30 days, nine completed the 3-month study period. Following dietary alteration, eight of 10 dogs achieved complete remission, one dog achieved partial remission and one dog had no response. Seven of eight dogs achieving complete remission have remained in remission up to 4 years following study. In dogs with complete remission, median Canine Chronic Enteropathy Clinical Activity Index score was 11.5 and 4, and median serum albumin concentration was 15 g/L and 26 g/L at 0 and 14-28 days, respectively., Clinical Significance: Dogs with protein-losing enteropathy with previous lack of response to a combination of dietary therapies, glucocorticoids and immunosuppressive medications can achieve remission following a dietary change. Improvement is likely to be seen within 14 to 30 days. A change in dietary approach may be an alternative to further immunosuppression or anti-inflammatory strategies in some dogs with difficult to treat protein-losing enteropathy., (© 2021 British Small Animal Veterinary Association.)

Symptomatic cytomegalovirus (CMV) infection in immunocompetent hosts has traditionally been considered to have a benign, self-limited course, and those who need intensive therapy are rare. Moreover, there are few reports of CMV infection with protein-losing enteropathy (PLE). We present an immunocompetent 74-year-old woman with CMV ileitis with PLE, which was diagnosed due to severe hypoalbuminemia and edema of the lower extremities. The patient was not immunocompromised, because a human immunodeficiency virus (HIV) antibody test was negative and she had not been taking immunosuppressants. Imaging tests including colonoscopy revealed ileitis with shallow widespread ulcers. 99m Tc-human serum albumin (HAS-D) scintigraphy suggested a possibility of protein loss in the ileum based on selective accumulation of nuclides in the right abdomen. Histological findings of the biopsy showed ulcerative mucosa with abnormal cells, which had enlarged nuclei with intranuclear inclusion bodies, including typical Cowdry A type. In immunohistochemistry, these cells were positive for anti-CMV staining. She was successfully treated with medical treatments including intravenous injection of ganciclovir (GCV) (500 mg/day). We described an extremely rare case of CMV ileitis with PLE in an immunocompetent adult who was treated successfully with medical treatments, including GCV., (© 2021. Japanese Society of Gastroenterology.)

Gastrointestinal bleeding complicated with protein-losing enteropathy after the Fontan procedure has been often reported in recent years, but there is no established therapy for it.We report the case of an 18-year-old boy who suffered from abdominal pain, melena, and anaemia due to intractable haemorrhagic protein-losing enteropathy after the Fontan procedure. He was successfully treated with octreotide therapy.

Background: Although cyclophosphamide (CPA) is the key drug for the treatment of autoimmune diseases including vasculitides, it has some well-known adverse effects, such as myelosuppression, hemorrhagic cystitis, infertility, and infection. However, CPA-associated severe enteritis is a rare adverse effect, and only one case with a lethal clinical course has been reported. Therefore, the appropriate management of patients with CPA-associated severe enteritis is unclear., Case Summary: We present the case of a 61-year-old woman diagnosed with granulomatosis with polyangiitis based on the presence of symptoms in ear, lung, and, kidney with positive myeloperoxidase-antineutrophil cytoplasmic antibody. She received pulsed methylprednisolone followed by prednisolone 55 mg/d and intravenous CPA at a dose of 500 mg/mo. Ten days after the second course of intravenous CPA, she developed nausea, vomiting, and diarrhea, and was admitted to the hospital. Laboratory testing revealed hypoalbuminemia, suggesting protein-losing enteropathy. Computed tomography revealed wall thickening of the stomach, small intestine, and colon with contrast enhancement on the lumen side. Antibiotics and immunosuppressive therapy were not effective, and the patient's enteritis did not improve for > 4 mo. Because her condition became seriously exhausted, corticosteroids were tapered and supportive therapies including intravenous hyperalimentation, replenishment of albumin and gamma globulin, plasma exchange, and infection control were continued. These supportive therapies improved her condition, and her enteritis gradually regressed. She was finally discharged 7 mo later., Conclusion: Immediate discontinuation of CPA and intensive supportive therapy are crucial for the survival of patients with CPA-associated severe enteritis., Competing Interests: Conflict-of-interest statement: The authors declare that they have no conflict of interest., (©The Author(s) 2021. Published by Baishideng Publishing Group Inc. All rights reserved.)

Severe atopic dermatitis (AD) may lead to various complications such as hypoproteinaemia. We describe the case of a 7-month-old male infant with severe AD complicated with protein-losing enteropathy (PLE). He was diagnosed with AD at 2 months of age; however, because of familial steroid phobia, topical corticosteroids were not administered. At 7 months of age, he was admitted to our hospital for decreased feeding, diarrhoea, reduced urine volume and recurrent vomiting. Class 3 topical corticosteroid treatment was initiated. On day 3, eczema had almost resolved. However, serum protein levels had not improved; oliguria persisted and oedema worsened. Serum albumin scintigraphy revealed radioisotopes in the distal duodenum, leading to PLE diagnosis. Systemic prednisolone and albumin were administered, with no PLE relapse after discontinuation. To our knowledge, only two infant PLE cases associated with AD were reported to date. PLE should be considered in patients with severe AD and persistent hypoproteinaemia., Competing Interests: Competing interests: None declared., (© BMJ Publishing Group Limited 2021. No commercial re-use. See rights and permissions. Published by BMJ.)

Complement hyperactivation, angiopathic thrombosis and protein-losing enteropathy (CHAPLE disease) is a lethal disease caused by genetic loss of the complement regulatory protein CD55, leading to overactivation of complement and innate immunity together with immunodeficiency due to immunoglobulin wasting in the intestine. We report in vivo human data accumulated using the complement C5 inhibitor eculizumab for the medical treatment of patients with CHAPLE disease. We observed cessation of gastrointestinal pathology together with restoration of normal immunity and metabolism. We found that patients rapidly renormalized immunoglobulin concentrations and other serum proteins as revealed by aptamer profiling, re-established a healthy gut microbiome, discontinued immunoglobulin replacement and other treatments and exhibited catch-up growth. Thus, we show that blockade of C5 by eculizumab effectively re-establishes regulation of the innate immune complement system to substantially reduce the pathophysiological manifestations of CD55 deficiency in humans.

In recent years, monogenic causes of immune dysregulation syndromes, with variable phenotypes, have been documented. Mutations in the lipopolysaccharide-responsive beige-like anchor (LRBA) protein are associated with common variable immunodeficiency, autoimmunity, chronic enteropathy, and immune dysregulation disorders. The LRBA protein prevents degradation of cytotoxic T-lymphocyte antigen 4 (CTLA4) protein, thus inhibiting immune responses. Both LRBA and CTLA4 deficiencies usually present with immune dysregulation, mostly characterized by autoimmunity and lymphoproliferation. In this report, we describe a patient with an atypical clinical onset of LRBA deficiency and the patient's response to abatacept, a fusion protein-drug that mimics the action of CTLA4.

Protein-losing enteropathy (PLE) is a condition characterized by gut mucosal injury that typically manifests with edema and hypoalbuminemia due to protein loss in the GI tract. We present a rare case of lupus-associated PLE (LUPLE) manifested by profound edema, diarrhea, and thrombotic complications. Through our case report, we discuss the typical clinical presentation, diagnostic studies available, and treatment options for these patients. Our patient's clinical picture and laboratory markers improved with the initiation of corticosteroids and belimumab, which is a novel treatment regimen for LUPLE. Moreover, our patient was found to have a clinically significant hypercoagulable state that was ultimately attributed to PLE in the setting of systemic lupus erythematosus (SLE). We highlight the increased thrombotic risk in these patients and the subsequent management implications with regard to anticoagulation. Gastroenterologists are likely to be involved in the care of these patients, and may be the first to recognize the constellation of findings in PLE, leading to potentially very effective treatment.

Nonsteroidal anti-inflammatory drug (NSAID)-induced enteropathy is considered a serious and increasing clinical problem without available treatment. Glycomacropeptide (GMP) is a 64-amino acid peptide derived from milk κ-casein with numerous biological activities. The aim of this study was to investigate the protective effect of GMP on NSAID enteropathy in rats. Enteropathy was induced by seven days oral indomethacin administration. Rats were orally GMP treated from seven days previous and during the establishment of the enteropathy model. Changes in metabolism, hematological and biochemical blood alterations, intestinal inflammation and oxidative damage were analyzed. Integrity barrier markers, macroscopic intestinal damage and survival rate were also evaluated. GMP treatment prevented anorexia and weight loss in animals. Furthermore, prophylaxis with GMP ameliorated the decline in hemoglobin, hematocrit, albumin and total protein levels. The treatment had no therapeutic efficacy on the decrease of occludin and mucin (MUC)-2 expression in intestinal tissue. However, GMP markedly decreased neutrophil infiltration, and CXCL1, interleukin-1β and inducible nitric oxide synthase expression. Nitric oxide production and lipid hydroperoxide level in the small intestine were also diminished. These beneficial effects were mirrored by preventing ulcer development and increasing animal survival. These results suggest that GMP may protect against NSAID enteropathy through anti-inflammatory and antioxidant properties.

To evaluate for evidence of systemic glucocorticoid absorption in cases of Fontan-associated protein-losing enteropathy (PLE) treated with enteral budesonide, we reviewed the charts of 27 patients with Fontan-associated PLE followed at Children's Hospital Colorado from 2005 to 2018. Cases were excluded for lack of budesonide thserapy or a treatment duration of less than 6 months. Charts were examined by two endocrinologists for review of prior biochemical endocrine evaluations, alterations in linear growth, and physical exam findings consistent with steroid excess. Twelve patients met inclusion criteria. Eight had prior documented cortisol screening. Three patients were tested while on treatment with a median fasting AM cortisol of 0.9 mcg/dL; two of these had a concomitantly measured ACTH, both below the detectable limit. Five patients were tested while weaning or having discontinued budesonide, with a median fasting AM cortisol of 9.1 mcg/dL. Eleven patients had decreases in height velocity associated with starting budesonide. Six patients had documentation of cushingoid features by an endocrinologist. In this cohort of children treated with budesonide for PLE following Fontan, clinical signs of systemic glucocorticoid absorption were frequent. Cortisol secretion was suppressed while on therapy, with adrenal recovery noted once budesonide was discontinued. Growth failure and cushingoid features were common findings. While these findings should be confirmed in larger cohorts, we recommend that the evaluation for systemic absorption of exogenous steroids be considered in patients treated with long-term enteral budesonide given the potential risk for adrenal crisis in times of physiologic stressors.

Background: Recent studies have shown that oral budesonide can be used to improve albumin level in patients with protein-losing enteropathy (PLE) following Fontan procedure. However, there has never been a systematic review and meta-analysis to confirm this finding. We performed a systematic review and meta-analysis to explore the therapeutic effect of budesonide in patients with PLE post-Fontan procedure., Methods: We searched the databases of MEDLINE and EMBASE from inception to January 2019. Included studies were published studies that evaluate albumin level before and after budesonide therapy in patients with PLE following Fontan procedure. Data from each study were combined using the random-effects model., Results: Five studies with 36 post-Fontan operation patients with PLE were included. In random-effects model, there was a statistically significant difference in albumin level between before and after budesonide treatment (weighted mean difference = 1.28, 95% confidence interval: 0.76-1.79). No publication bias was observed on a funnel plot and Egger test with a P value of .676., Conclusions: The results of this systematic review and meta-analysis show that budesonide can be used to increase albumin level in patients with PLE following Fontan operation. Further studies may focus on the impact of outcome of budesonide in this population.

Protein-losing enteropathy (PLE) is one of the major complications after a Fontan operation. Some PLE patients suffer from concurrent gastrointestinal bleeding. An effective treatment regimen for such patients has not been established yet. Further, it remains unknown whether PLE and gastrointestinal bleeding coexist independently, or protein losing is associated with gastrointestinal bleeding. We report a 7-year-old steroid-refractory post-Fontan PLE case suggesting the latter pathogenesis together with a literature review.

Gastrointestinal (GI) tract involvement in Langerhans cell histiocytosis (LCH) is extremely rare. Langerhans cell histiocytosis with GI tract involvement (GI-LCH) is frequently associated with multi-system disease, and usually presents with severe systemic symptoms, such as protein-losing enteropathy (PLE). Although the GI tract is not included among the organs at risk, the prognosis of GI-LCH is poor, and no effective chemotherapeutic regimen has been identified. Here, we report an infant case of primary refractory GI-LCH with PLE that showed marked improvement in response to 2-chlorodeoxyadenosine (2-CdA) therapy with no severe adverse events, even under conditions of deteriorating general health. The present findings indicate that 2-CdA may be effective for refractory GI-LCH with PLE. Further studies are warranted to determine the optimal therapeutic strategies for GI-LCH with PLE.

An 85-year-old woman was admitted to our hospital because of progressive hypoproteinemia and generalised oedema. Technetium-99m human albumin scintigraphy revealed protein leakage in the gastrointestinal tract. Upper gastrointestinal endoscopy revealed small whitish nodules from the gastric body up to the duodenal bulb. The urease test for Helicobacter pylori infection was positive. We diagnosed her as having protein-losing gastroenteropathy (PLGE) caused by H. pylori infection. The patient's hypoproteinemia and clinical symptoms promptly resolved after H. pylori eradication. Our results suggest that a trial of H. pylori eradication is warranted in patients with PLGE, even if endoscopy reveals neither giant rugal folds, erosion of the mucosa, nor polyposis, which are previously reported characteristic endoscopic findings of PLGE., Competing Interests: Competing interests: None declared., (© BMJ Publishing Group Limited 2019. No commercial re-use. See rights and permissions. Published by BMJ.)

Background: Protein-losing enteropathy (PLE) because of chronic inflammatory enteropathy (CIE) in dogs is often treated with a combination of glucocorticoids and second-line immunosuppressant (SLI). This combined approach might not be necessary in all dogs., Hypothesis/objectives: To describe diagnostic features and outcomes of dogs with PLE treated with glucocorticoids alone (group P) or with glucocorticoids and SLI (group S)., Animals: Thirty-one dogs with PLE., Material and Methods: Retrospective analysis of signalment data from diagnostic procedures, treatment, and outcome of dogs with CIE/PLE (from 2015 to 2017), using the hospital's digital case database. Dogs with hypoalbuminemia and CIE were included. Because of a stepwise treatment algorithm, dogs were allocated to group P or S. Time to serum albumin concentrations ≥20 g/L and survival data were collected. Dogs were additionally categorized by their albumin and cobalamin serum concentrations. Multivariate and univariate analysis as well as Pearson's correlation and Kaplan-Maier survival analysis were performed., Results: Seventeen dogs were included in group P and 14 in group S. World Small Animal Veterinary Association score of the duodenum was different between groups (P = .05), but none of the other examined data. Median time until serum albumin reached >20 g/L was 13 days. Median survival time after start of treatment was 85 days (range, 13-463 days) in group P and 166 days (range, 8-390 days) in group S., Conclusion and Clinical Importance: No routine diagnostic test was predictive of clinical response, treatment group, or outcome. Glucocorticoid treatment alone can be appropriate in dogs with PLE., (© 2019 The Authors. Journal of Veterinary Internal Medicine published by Wiley Periodicals, Inc. on behalf of the American College of Veterinary Internal Medicine.)

Whipple's disease is a rare, multisystem infection caused by the Gram-positive Tropheryma whippelii organism. In addition to neurological and rheumatological manifestations, this disease can result in significant gastrointestinal symptoms such as malabsorption, diarrhea, and weight loss. Given the diagnostic challenge and rare occurrence, a high index of suspicion is critical to prevent morbidity and mortality from this otherwise highly infectious disease transmitted via the fecal-oral route. We present a very rare but near-fatal case of hypovolemic shock secondary to protein-losing enteropathy and gastrointestinal bleeding from small bowel T. whippelii infection. Furthermore, the epidemiology, clinical presentation, diagnosis, and management of Whipple's disease is reviewed.

Objectives: Loss of the complement inhibitor CD55 leads to a syndrome of early-onset protein-losing enteropathy (PLE), associated with intestinal lymphangiectasia and susceptibility to large-vein thrombosis. The in vitro and short-term treatment benefits of eculizumab (C5-inhibitor) therapy for CD55-deficiency have been previously demonstrated. Here we present the 18-months treatment outcomes for 3 CD55-deficiency patients with sustained therapeutic response., Methods: Three CD55-deficiency patients received off-label eculizumab treatment. Clinical and laboratory treatment outcomes included frequency and consistency of bowl movements, weight, patient/parent reports of overall well-being, and serum albumin and total protein levels. Membrane attack complex deposition on leukocytes was tested by flow cytometry, before and during eculizumab treatment., Results: Marked clinical improvement was noted in all 3 patients with resolution of PLE manifestations, that is, diarrhea, edema, malabsorption, overall well-being, growth, and quality of life. In correlation with the clinical observations, we observed progress in all laboratory outcome parameters, including increase in albumin and total protein levels, and up to 80% reduction in membrane attack complex deposition on leukocytes (P < 0.001). The progress persisted over 18 months of treatment without any severe adverse events., Conclusions: CD55-deficiency patients present with early-onset diarrhea, edema, severe hypoalbuminemia, abdominal pain, and malnutrition. Targeted therapy with the terminal complement inhibitor eculizumab has positive clinical and laboratory outcomes in PLE related to CD55 loss-of-function mutations, previously a life-threatening condition. Our results demonstrate the potential of genetic diagnosis to guide tailored treatment, and underscore the significant role of the complement system in the intestine.

Protein losing enteropathy (PLE), a very rare disease with hypoproteinemia and edema as its characteristics, is caused by various diseases resulting in protein depletion from the gut. The diagnosis is relatively difficult due to its complex pathogeneses. The present paper reported a case whose symptom started with acute diarrhea and hypoproteinemia. Gastrointestinal endoscopies showed digestive ulcers and colon polyp. The treatments contained albumin infusion, Chinese herbal decoction and other symptomatic therapies. The hypoproteinemia become even worse and edema occurred after 4 days' treatment. A larger dose of albumin infusion (40-60 g/d) and modified herbal decoctions were prescribed. A final diagnosis of eosinophilic gastroenteritis (EG) complicated with PLE was confirmed by histopathological examination of a repeated gastroscopy. After three weeks' treatment, the serum albumin level was raised and the edema subsided gradually. In conclusion, herbs may have an effect on PLE patients, but PLE resulting from EG is very complex and easy to misdiagnose, especially in atypical conditions. Further studies are required to find the exact mechanisms.

A 64-year-old woman with leg edema was diagnosed with protein-losing gastroenteropathy and Sjögren's syndrome. Central venous nutrition led to infection of her catheter, ascites, and deep vein thrombosis. Following successful treatment of these conditions with antibiotics and anticoagulants, she was treated unsuccessfully with prednisolone and steroid pulse therapy. Mizoribine add-on markedly reduced edema and normalized serum albumin. This is the first report of a steroid-resistant protein-losing gastroenteropathy patient with Sjögren's syndrome successfully treated with mizoribine.

Systemic lupus erythematosus (SLE) is a multisystem disorder which can affect the gastrointestinal (GI) system. Although GI symptoms can manifest in 50% of patients with SLE, these have barely been reviewed due to difficulty in identifying different causes. This study aims to clarify clinical characteristics, diagnosis and treatment of the four major SLE-related GI system complications: protein-losing enteropathy (PLE), intestinal pseudo-obstruction (IPO), hepatic involvement and pancreatitis. It is a systematic review using MEDLINE and EMBASE databases and the major search terms were SLE, PLE, IPO, hepatitis and pancreatitis. A total of 125 articles were chosen for our study. SLE-related PLE was characterized by edema and hypoalbuminemia, with Technetium 99m labeled human albumin scintigraphy ( 99m Tc HAS) and alpha-1-antitrypsin fecal clearance test commonly used as diagnostic test. The most common site of protein leakage was the small intestine and the least common site was the stomach. More than half of SLE-related IPO patients had ureterohydronephrosis, and sometimes they manifested as interstitial cystitis and hepatobiliary dilatation. Lupus hepatitis and SLE accompanied by autoimmune hepatitis (SLE-AIH overlap) shared similar clinical manifestations but had different autoantibodies and histopathological features, and positive anti-ribosome P antibody highly indicated the diagnosis of lupus hepatitis. Lupus pancreatitis was usually accompanied by high SLE activity with a relatively high mortality rate. Early diagnosis and timely intervention were crucial, and administration of corticosteroids and immunosuppressants was effective for most of the patients.

PLE is an important cause of morbidity and mortality in patients who have undergone Fontan palliation. While multiple PLE therapies have been reported, none has proved consistently effective. Patients who do not respond to "standard" PLE therapies face poor long-term outcomes. We report here a significant response to dopamine infusion in three patients with chronic, refractory PLE. We hypothesize that this response may be at least partially due to a dopamine effect on lymphatic receptors rather than to an augmentation of cardiac output., (© 2017 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.)

Objective: To describe the administration of 25% human serum albumin (HSA) to dogs diagnosed with idiopathic inflammatory bowel disease (IBD) and protein-losing enteropathy (PLE). The secondary objectives were to report any acute and delayed adverse events and the effect of corticosteroids on the development of these reactions., Design: Retrospective study (2003-2013)., Setting: Private referral hospital., Animals: Twenty-one client owned dogs diagnosed with PLE and idiopathic IBD that received ≥ 1 transfusion of 25% HSA. Dogs were included in the study if they had panhypoproteinemia, serum albumin concentration < 15.0 g/L [< 1.5 g/dL] or extravascular fluid accumulation, idiopathic IBD confirmed on histopathology, and complete medical records., Interventions: None., Main Results: Two of the 21 patients (9.5%) developed signs consistent with an acute reaction; 1 of these dogs was euthanized due to the severity of the reaction. Two patients (9.5%) showed signs consistent with a delayed reaction; 1 of these dogs was euthanized 5 days after the reaction, though it is unclear whether the reaction and the euthanasia were related. Corticosteroid administration did not appear to affect the occurrence of adverse reactions., Conclusions: This retrospective study demonstrated that the administration of 25% HSA to dogs with moderate to severe hypoalbuminemia from PLE was associated with occasional acute and delayed adverse events, some of which were severe or fatal., (© Veterinary Emergency and Critical Care Society 2016.)

Objective: Severe gastrointestinal involvement of Henoch-Schönlein purpura (HSP) is rare but potentially life-threatening. Management of severe gastrointestinal involvement in HSP is not codified. Symptomatic care and steroids are a first-line therapy. Nonsteroidal immunomodulatory therapies have been anecdotally used to treat steroid-refractory forms. The aim of this study was to describe the outcome of patients with severe gastrointestinal involvement of HSP who required nonsteroidal immunomodulatory therapy., Methods: A French retrospective case series study was conducted. Pediatric consultants at 31 French academic pediatric centers were contacted. Patients were identified from memory or via an informatics diagnosis-related code system. Clinical, paraclinical, and therapeutic data were collected., Results: Twenty-nine responding centers provided nine cases, one of which was excluded. Five boys and three girls, aged 3-15years (median: 5.5years) from seven centers were included. Severe gastrointestinal involvement of HSP mainly included intense pain, digestive bleeding, and protein-losing enteropathy. All children had been treated with steroids at first line and intravenous immunoglobulins (IVIg) at second line. Six out of eight showed a complete response to IVIg within 7days and two out of eight had a partial response. Two out of eight relapsed with less severe gastrointestinal involvement requiring a second dose of IVIg and they did not relapse thereafter. Tolerance was good, but two out of eight developed high proteinuria on the day following IVIg infusion., Conclusion: Although a possible link with a flare-up of proteinuria needs to be addressed, IVIg appears to be a good candidate for treatment of severe gastrointestinal involvement of HSP., (Copyright © 2016 Elsevier Masson SAS. All rights reserved.)

Primary intestinal lymphangiectasia (PIL), also known as Waldmann's disease, is an exudative enteropathy resulting from morphologic abnormalities in the intestinal lymphatics. In this article, we describe a 12-year-old boy with PIL that led to protein-losing enteropathy characterized by diarrhea, hypoalbuminemia associated with edema (serum albumin level: 1.0 g/dL), and hypogammaglobulinemia (serum IgG level: 144 mg/dL). Severe hypoalbuminemia, electrolyte abnormalities, and tetany persisted despite a low-fat diet and propranolol. Everolimus (1.6 mg/m(2)/day) was added to his treatment as an antiangiogenic agent. With everolimus treatment, the patient's diarrhea resolved and replacement therapy for hypoproteinemia was less frequent. Hematologic and scintigraphy findings also improved (serum albumin level: 2.5 g/dL). There were no adverse reactions during the 12-month follow-up. To the best of our knowledge, this is the first report of everolimus use in a patient with PIL., (Copyright © 2016 by the American Academy of Pediatrics.)

Protein-losing enteropathy (PLE) is a rare and life-threatening complication that occurs after the Fontan procedure. We herein report the case of an 11-year-old Japanese boy who developed PLE six times after undergoing the Fontan procedure. High-dose spironolactone therapy has been effective for 2 years. His high level of serum aldosterone decreased to a nearly normal range and spironolactone may have a diuretic and anti-inflammatory potential.

A case is presented of a protein-losing enteropathy (PLE) as the initial manifestation of systemic lupus erythematosus (SLE) in a 17 year-old female patient, who presented with ascites, edema and hypoalbuminemia. The diagnosis of SLE was based on the presence of: malar rash, oral ulcers, thrombocytopenia, antinuclear antibodies, IgM anticardiolipin antibody, and lupus anticoagulant. Renal and liver diseases were ruled out. The PLE diagnosis was confirmed with fecal alpha 1-antitrypsin clearance. The PLE was refractory to different lines of immunosuppressive agents like glucocorticoids, cyclophosphamide, azathioprine, and cyclosporine, showing a satisfactory and sustained response with rituximab, allowing steroid sparing and long term remission., (Copyright © 2014 Elsevier España, S.L.U. y Sociedad Española de Reumatología y Colegio Mexicano de Reumatología. All rights reserved.)

Protein-losing gastroenteropathy (PLGE), a rare manifestation of primary Sjögren's syndrome (SS), is characterized by profound edema and severe hypoalbuminemia secondary to excessive serum protein loss from the gastrointestinal tract and is clinically indistinguishable from nephrotic syndrome. We report a case of a 30-year-old Taiwanese woman with PLGE-associated SS. In addition to a positive Schirmer's test, she had eye-dryness, thirst, and high levels of anti-SSA antibodies, fulfilling SS criteria. PLGE diagnosis was highly appropriate given the clinical profile of hypoalbuminemia, hypercholesterolemia, pleural effusion, and ascites, with absent cardiac, hepatic, or renal disease. We were unable to perform technetium-99 m-labeled human serum albumin scintigraphy ((99m)Tc-HAS). However, the patient's edema and albumin level improved dramatically in response to a 3-month regime of oral prednisolone followed by oral hydroxychloroquine., (© The Author(s) 2015.)

Background/aims: Protein-losing enteropathy (PLE), characterized by severe hypoalbuminemia and peripheral edema, is a rare manifestation of systemic lupus erythematosus. This present study aimed to identify the distinctive features of lupus-related PLE and evaluate the factors related to the treatment response., Methods: From March 1998 to March 2014, the clinical data of 14 patients with lupus PLE and seven patients with idiopathic PLE from a tertiary center were reviewed. PLE was defined as a demonstration of protein leakage from the gastrointestinal tract by either technetium 99m-labelled human albumin scanning or fecal α1-antitrypsin clearance. A positive steroid response was defined as a return of serum albumin to ≥ 3.0 g/dL within 4 weeks after initial steroid monotherapy, and remission as maintenance of serum albumin ≥ 3.0 g/dL for at least 3 months. A high serum total cholesterol level was defined as a level of ≥ 240 mg/dL., Results: The mean age of the lupus-related PLE patients was 37.0 years, and the mean follow-up duration was 55.8 months. Significantly higher erythrocyte sedimentation rate and serum total cholesterol levels were found for lupus PLE than for idiopathic PLE. Among the 14 patients with lupus PLE, eight experienced a positive steroid response, and the serum total cholesterol level was significantly higher in the positive steroid response group. A positive steroid response was associated with an initial high serum total cholesterol level and achievement of remission within 6 months., Conclusions: In lupus-related PLE, a high serum total cholesterol level could be a predictive factor for the initial steroid response, indicating a good response to steroid therapy alone.

There are few reports on successful high-dose spironolactone treatment of refractory protein-losing enteropathy (PLE) caused by Fontan procedure. We report successful diuretics treatment with spironolactone and furosemide at standard dose, of refractory PLE in a patient with Noonan syndrome and repaired congenital heart disease. This is the first successful application of diuretics treatment in a patient with refractory PLE without Fontan procedure. This case illustrates that diuretics treatment can be the first-line treatment of PLE regardless of the causative physiology, and can be effective in refractory PLE with Noonan syndrome., (© 2015 Japan Pediatric Society.)