Aim: Atypical haemolytic uraemic syndrome (aHUS) is a debilitating condition that can cause significant morbidity and mortality in children if not adequately and promptly treated. This report shares real-world data on the use of eculizumab in children with aHUS. Methods: We report our experience with the use of eculizumab in 14 children with aHUS. Results: The median age at aHUS diagnosis was 12 months (range: 2-108 months), with six (42.9%) patients presenting in infancy and six (42.9%) being males. Eculizumab therapy was associated with haematological and thrombotic microangiopathy responses in 14 (100%) and 13 (92.9%) patients after a median of 9 days (range: 7-12 days) and 9.5 days (range: 7-14 days), respectively. None of the six patients who were previously treated with plasma therapy required any further infusions. Of the six patients who previously required dialysis, only one patient continued to do so and eventually received a renal transplant. The median time to ≥25% decrease in serum creatinine level in the remaining patients was 16 days (range: 14-21 days), and estimated glomerular filtration rate increased from a median of 17-101 mL/min/1.73 m 2 . The safety profile was similar to that reported in the literature, and 10 patients continue to receive therapy, with 3 being on the drug for 4 or more years. Conclusion: Our study adds to the growing body of evidence highlighting the efficacy and safety of eculizumab for the management of children with aHUS.
Renal transplantation is the treatment of choice for ESRD in children. It is associated with better quality of life, growth of children, and improved long‐term survival. The aim of the study was to evaluate the outcomes of pediatric renal transplantation at a tertiary care center in UAE. A retrospective chart review was undertaken for all the pediatric renal transplants performed at Sheikh Khalifa Medical City, Abu Dhabi, UAE, over the past 9 years. The study evaluated the demographic data, outcomes, and complications of pediatric renal transplantation. The post‐transplantation outcomes including surgical complications, documented infections, graft rejection, graft and patient survival, effect on growth, and eGFR were reviewed. Between 2010 and 2018, 30 pediatric patients underwent renal transplantation. The follow‐up period ranged from 1 to 9 years with a mean of 3.3 years. The mean age of the patients at the time of transplant was 9.8 years, and 56.7% were males. Prior to the transplantation, the majority of the recipients were on peritoneal dialysis (70.0%). Main source of renal donation at our center was from LRD, chiefly from parents. Patient survival at 1 and 5 years was 100% and 96.7%, respectively. Graft survival at 1 and 5 years was 96.7% and 83.3%, respectively. During the 9‐year follow‐up period, 5 (16.7%) recipients experienced rejection episode. This study demonstrates that during 5‐year period, pediatric kidney transplantation program has achieved optimal patient (96.7%) and graft (83.3%) survival rates and is comparable to well‐established centers.
Mitochondrial encephalomyelitis, lactic acidosis and stroke-like episodes (MELAS) syndrome is the most common of the rare mitochondrial disorders. It frequently is secondary to a point mutation in mitochondrial DNA m.3243 A to G. The main features of the disease include myopathy, encephalopathy, lactic acidosis, stroke-like episodes, seizures and migraine however the clinical presentations can vary widely. Age of onset of the disease has a wide range but patients are typically under 40 years of age, nonetheless cases have been reported in the elderly population. A number of drugs have been used for the management of patients with MELAS including CoEnzyme Q10, Vitamin K-3, Vitamin K-1, Ascorbate and Riboflavin. It is also important to note that seizures in MELAS can be difficult to manage often requiring a combination of antiepileptic drugs. We report the case of a 76-year-old man (the second oldest diagnosed case to the authors' knowledge) whose case exemplifies the complexity of diagnosing and managing these patients including the complications of the disease.
Spontaneous iliopsoas haematoma is an uncommon complication of anticoagulation. Such bleeding can be life threatening and therefore early recognition is important. In the published literature the universal presenting symptom is pain. We report on the unusual case of an elderly patient who was anticoagulated with Warfarin for Atrial fibrillation, and who presented with painless weakness of the right lower limb. Anticoagulation was reversed and her condition was managed conservatively. Her case is presented in the context of the published literature.
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