Patient: Female, 12-year-old Final Diagnosis: Ulcerative Symptoms: Bloody diarrhea Medication:— Clinical Procedure: — Specialty: Gastroenterology and Hepatology Objective: Rare disease Background: Ulcerative colitis (UC) is a chronic autoimmune inflammatory disease of the colon that infrequently affects children. The disease requires immunosuppressive therapy to achieve remission and keep the disease in remission. Currently, many therapies are approved for use in pediatric patients with UC, including steroid, 5-aminosalicylic acid (5-ASA), azathioprine, and biologic therapy with anti-tumor necrosis factor (TNF) inhibitors. Despite their efficacy, many patients have refractory severe disease that fails therapy and may require surgical interventions. Recently, the small molecule Janus Kinase (JAK) inhibitor tofacitinib has been approved for moderate to severe UC that fails biologic therapy in adults. However, the safety and efficacy of this drug has not been tested in pediatric UC patients. Case Report: We describe a case of a 13-year-old girl with 2-year history of severe UC who had secondary loss response to both infliximab and adalimumab over 2 years, despite adequate trough serum drug levels and the concomitant use of azathioprine. She was also dependent on steroid to control her disease. Infectious work-ups were always negative for infectious organisms. She was then successfully treated with tofacitinib 5 mg orally twice daily. She went into complete clinical, endoscopic, and steroid-free remission. Conclusions: This case report highlights the safety and efficacy of tofacitinib in pediatric patients with severe refractory UC, potentially avoiding proctocolectomy in this young patient population. Future research should study the role of tofacitinib in patients with moderate to severe UC in children.
Introduction Renal echinococcosis is of rare occurrence, and although often asymptomatic, it can present with various mild to drastic presentations, of which hydatiduria is pathognomonic. Diagnosis can be preliminarily established by imaging, and treatment is primarily surgical. We present a patient with renal echinococcosis treated successfully with exclusive antiparasitic pharmacotherapy after refusing surgery despite extensive renal involvement. We hope through this report to help establish future solid guidelines regarding this uncommon therapeutic approach. Case presentation This is a case of a 49-year-old Syrian shepherd presenting with flank pain and passage of grape-skin-like structures in urine. A diagnosis of renal echinococcosis with hydatiduria and significant parenchymal destruction was established based on exposure history, positive serology, imaging findings, and renal scintigraphy. After proper counseling, the patient refused nephrectomy and was therefore started on dual pharmacotherapy (albendazole and praziquantel) and is having an uneventful follow-up and a satisfactory response to treatment. Conclusion This case embodies the daily challenges physicians navigate as they uphold the ethical principles of their practice and support their patients’ autonomy while delivering the best standards of care and consulting the scientific evidence. Although surgery is the cornerstone of renal echinococcosis treatment, treating physicians should be prepared to tackle situations where surgery cannot be done and offer the best next available option for patients who refuse surgery. As data on exclusive pharmacotherapy are limited, future research should thoroughly investigate the efficacy of this uncommon approach and outline reliable recommendations, facilitating future clinical decision-making in this avenue.
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