Martinón-Torres F, Iglesias Meleiro J María, Fernández Sanmartín M, Rodríguez Núñez A, Martinón Sánchez J María
Servicio de Críticos, Intermedios y Urgencias Pediátricas, Departamento de Pediatría, Hospital Clínico Universitario de Santiago de Compostela, Universidad de Santiago de Compostela, Santiago de Compostela, Spain.
An Pediatr (Barc). 2004 Sep;61(3):261-5. doi: 10.1016/s1695-4033(04)78806-2.
Meningococcal purpura fulminans (MPF) produces high mortality and morbidity, despite appropriate standard therapy. Administration of recombinant human activated protein C (rhAPC) has been successfully applied in adults with MPF and pediatric studies are under way. We report three pediatric patients with MPF treated with rhAPC as compassionate therapy. In two of these patients, positive clinical and laboratory effects were observed and both children achieved full recovery. The remaining patient died after 36 hours from refractory multiorgan failure. No rhAPC-related adverse effects were detected. The reported cases highlight the usefulness of rhAPC in children with MPF at least as a rescue compassionate treatment. Further clinical trials are needed to better delineate its efficacy and administration schedule in children.
尽管采用了适当的标准治疗方法,暴发性脑膜炎球菌性紫癜(MPF)的死亡率和发病率仍然很高。重组人活化蛋白C(rhAPC)已成功应用于成年MPF患者,儿科研究正在进行中。我们报告了3例接受rhAPC作为同情性治疗的MPF儿科患者。其中2例患者观察到了积极的临床和实验室效果,两名儿童均完全康复。其余1例患者在出现难治性多器官功能衰竭36小时后死亡。未检测到与rhAPC相关的不良反应。报告的病例突出了rhAPC在MPF儿童中至少作为一种挽救性同情治疗的有用性。需要进一步的临床试验来更好地描述其在儿童中的疗效和给药方案。