Clinical Characteristics and Outcomes of Young Patients with Thrombotic Microangiopathy at Ain Shams University Children’s Hospital

Abstract

Abstract Introduction Thrombotic microangiopathies (TMA) is a life-threatening condition that requires urgent management. Survival of patients has improved greatly over the past two decades with improved supportive care and other therapeutic options. Objectives: to describe the clinical features and outcomes of patients with TMA at Ain Shams University Children’s Hospital. Methods In a retro-prospective study, we included patients diagnosed with TMA in the Pediatric Hematology and Pediatric Nephrology Units at Ain Shams University Children’s Hospital from 2009 through 2023. Results Thirty-three patients were enrolled, they were 15 females and 18 males with age ranges from 0.31 to 13.9 years and median (IQR) of 4.3 (1.4 – 9.0) years. Almost half of our patients had infection related hemolytic uremic syndrome (HUS) (51.5%), 30.3% had atypical HUS (aHUS), 9.1% had congenital thrombotic thrombocytopenic purpura (cTTP), while 9.1% were diagnosed with immune-mediated TTP (iTTP). Renal manifestations were the most common presenting symptoms (90.9%), hematological manifestations were present in (66.7%), while neurological manifestation were evident in (36.4%) of patients. Conservative management was the most commonly used approach (87.9%),while (75.8%) received plasma infusion and (69.7%) needed renal replacement therapy. On studying the outcome of our TMA cases, 33.3% had residual hematological manifestations, meanwhile 60.6% left with chronic kidney disease. Mortality was reported in 18.2% during the whole follow-up period. Conclusion infection-related HUS was the most common type of TMA in our cohort, and most patients were managed conservatively with disease resolution at 3 and 6 months follow up.