Atipik Hemolitik Üremik Sendromda Ekulizumab Kullanımı: Tek Merkez Deneyimi

Amaç: Hemolitik üremik sendrom (HÜS); hemolitik anemi, trombositopeni ve akut böbrek yetmezliği ile seyreden nadir bir hastalıktır. aHÜS ise kompleman yolağındaki bozukluklara bağlı oluşur. Tekrarlayan ataklar ve kronik böbrek hasarı ile ilişkilidir. Plazma infüzyonları ve değişimi uygulamaları hematolojik tabloyu düzeltse de altta yatan kompleman aktivasyonu devam eder ve olguların %33-40'ında son dönem böbrek yetmezliği ve ölüm görülür. Son yıllarda ülkemizde de kullanıma giren ekulizumab tedavisi ile hastalığın prognozunda önemli iyileşmeler olmuştur. Bu çalışmada ekulizumab tedavisi uygulanan yedi aHÜS olgusunu derledik. Yöntemler: Ocak 2012- Aralık 2015 tarihleri arasında aHÜS tanısı alan ve ekulizumab tedavisi verilen yedi çocuk çalışmaya dâhil edildi. Yaş, cinsiyet, başvuru semptomları, ishal öyküsü, başvuru ve takipteki laboratuar bulguları, diyaliz ihtiyacı, plazma değişimi/infüzyona yanıtı, ekulizumab tedavisine yanıtı ve son renal durumu kaydedildi. Bulgular: İshal öyküsü dört hastada vardı ancak dışkıda Enterohemorajik Escherichia coli (EHEC) analizi tüm hastalarda negatif bulundu. Oligüri/anüri altı hastada vardı. Dört hasta plazma değişimi tedavisi aldı. Mikroanjiopatik trombositopeniye bağlı olarak beş çocuğun diyaliz ihtiyacı oldu. Aşılamaya rağmen bir çocukta ekulizumab sonrası menenjit ve sepsis gelişti. Ekulizumab sonrasında tüm hastalarda hematolojik tam iyileşme görüldü ve bir hasta dışında hiç transfüzyon ihtiyacı olmadı. Son takipte diyaliz programında olan hasta yoktu. Sonuç: Atipik HÜS'de ekulizumab tedavisi hayat kurtarıcı ve başarılı bir tedavidir. Ancak menenjit gibi komplikasyonları tedavi öncesinde akılda tutmak gerekir.

ÖZGÜN MAKALE/ORIGINAL ARTICLE

Objectives: Hemolytic uremic syndrome (HUS) is defined by the presence of hemolytic anemia, thrombocytopenia, and acute renal failure. Atypical HUS (aHUS) is characterized by a relapsing course, high risk of permanent renal damage, and dysregulation in the complement pathway. Plasma exchange/ infusions may temporarily correct hematologic variables, but as the underlying complement dysregulation persists, end stage renal disease or death occurs in 33-40% of patients. In recent years, eculizumab has been used in the treatment of aHUS with high success rates. In this study we report seven pediatric cases with aHUS successfully treated with eculizumab. Methods: From January 2012 to December 2015, we investigated seven pediatric patients who had been diagnosed as aHUS and treated with eculizumab. Age, gender, presenting symptoms, presence of diarrhea, laboratory data on admission and follow-up, need for dialysis, response to plasma exchange, response to eculizumab, and outcome were recorded. Results: Diarrhea was present in four cases but the investigation about Enterohemorrhagic Echerichia coli (EHEC) was negative in stool specimens. Oliguria/anuria was present in six cases. Four of the seven children received plasma exchange. Five children needed dialysis. Despite vaccination, one patient had severe sepsis and meningitis as a complication of eculizumab. All had full recovery of hematologic parameters. Except one children none had erythrocyte transfusion after treatment. On the last follow up, none of the patients were on dialysis. Conclusion: Our results showed that eculizumab is a life-saving and successful treatment for use in cases of aHUS. However severe complications such as meningitis should be kept in mind before treatment.

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