Tip 1 Von Wıllebrand Hastalığı tanılı olguların yeni sınıflandırma sonrası değerlendirilmesi
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Tarih
2019
Yazarlar
Dergi Başlığı
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Cilt Başlığı
Yayıncı
Ege Üniversitesi, Tıp Fakültesi
Erişim Hakkı
info:eu-repo/semantics/openAccess
Özet
Amaç: Von Willebrand hastalığı, plazma von Willebrand faktöründeki kantitatif / kalitatif eksikliğinden kaynaklanan en yaygın kalıtsal kanama bozukluğudur. Son uluslararası rehberler ≥3 ay arayla en az ≥ 2 kere 30-50 IU / dl aralığında plazma von Willebrand faktör seviyesi saptanan ve önemli kanama skoru olan hastalara Düşük von Willebrand faktörlü tanısı konmasını önermektedir. Çalışmamızın amacı Uluslararası rehberlerin bu olguların yönetimi konusundaki önerilerin Türkiye popülasyonunda kullanımının uygunluğunu değerlendirmek ve kanama değerlendirme ölçeğinin kullanımının kanama belirtileri ile başvuran olguların değerlendirilmesinde önemi açısından farkındalığı arttırmaktır. Ayrıca, Düşük von Willebrand faktör'lü olgularda klinik anlam taşıyan kanama saptanmaması halinde onları "hasta" grubundan çıkarıp böylece kendi ve ailelerinin psikolojisini "hasta" etiketinden kurtarmak, gereksiz yere ilaç kullanımını önlemesini sağlamak da amaçlarımızdan diğeridir. Gereç ve Yöntem: Çalışmamız kesitsel olarak tasarlandı. Çalışmaya 1997-2018 yılları arasında Ege Üniversitesi Tıp Fakültesi Çocuk Hematoloji Bilim Dalında izlenmekte olan yaşları 0 -35 yaş arasında değişen daha önce von Willebrand Hastalığı Tip 1 tanısı almış, von Willebrand faktör düzeyi 10-50 IU/dl arasında olan 60 olgu çalışmaya alındı. Olgular VWF: Ag düzeyine göre VWF: Ag 10-30 IU/dl ve VWF: Ag 31-50IU/dl, ve yaşa göre 0-18 ve 19-35 yaş olmak üzere gruplara bölündü. Tüm olgulara yüz-yüze hasta veya velisi ile görüşme yöntemiyle Pediatrik Kanama Ölçeği uygulanarak kanama skorları belirlendi. Pediatrik kanama skoru ≥2 pozitif kabul edildi. Olguların yaş, semptom başlama yaşı, tanı yaşı, pediatrik kanama skoru, VWF: Ag, VWF: Ricof, faktör VIII düzeyleri, kan grubu fenotipi değerlendirildi. Bulgular: Grupların klinik ve laboratuvar verileri karşılaştırıldı. Düşük von Willebrand faktörlü olgularda yaş arttıkça plazma VWF:Ag düzeyinde artış eğilimi saptandı. Pediatrik kanama skoru açısından bakıldığında von Willebrand hastalığı tip 1 olgularında pediatrik kanama skoru ortanca değer, Düşük von Willebrand faktör grubuna göre istatistiksel anlamlı olarak daha yüksek saptandı. 0-18 yaş aralığında VWH tip 1 grubunda X ortanca pediatrik kanama skoru 6,9 iken Düşük von Willebrand faktör grubunda 3,8 saptandı. Bu fark istatistiksel anlamlı saptandı. 19-35 yaş olgularında pediatrik kanama skoru ortanca değeri açısından gruplar arasında istatistiksel anlamlı fark saptanmadı. Von Willebrand hastalığı tip 1 grubunda F VIII / VWF: Ag oranı ortanca değeri Düşük von Willebrand faktör grubu ile karşılaştırıldığında istatistiksel anlamlı yüksek bulundu. Çalışmamıza katılan olguların O ve non-O olarak bakıldığında kan grubu dağılımı açısından 2 grup arasında istatistiksel anlamlı fark saptanmadı. Sonuç: 0-18 yaş aralığında VWH tip 1 grubunda ortanca pediatrik kanama skoru Düşük VWF grubundan anlamlı olarak daha yüksek saptandı. Bu veriler, özellikle pediatrik yaş grubunda, Düşük von Willebrand faktörlü olgularında kanama fenotipinin belirgin olarak daha hafif olduğunu göstermekte ve kanama değerlendirme ölçeğinin kullanımının iki grubun arasında ayırıcı tanı açısından önemli diagnostik basamak olduğunu göstermektedir.
Aim: Von Willebrand disease is the most common hereditary bleeding disorder caused by a quantitative / qualitative deficiency in the plasma von Willebrand factor. Recent international guidelines recommend the diagnosis of Low von Willebrand factor in patients with a significant bleeding score of at least ≥ 2 times at plasma von Willebrand factor level of 30-50 IU / dL. The aim of our study is to assess the suitability of the use of recommendations by international guidelines on the management of these cases in Turkey population and is to increase awareness about the importance of bleeding assessment tool in the evaluation of cases presenting with bleeding symptoms. In addition, in cases with low von Willebrand factor, if there is no clinically meaningful bleeding, we can remove them from the “patient“ label by removing them from the “patient“ group and to prevent unnecessary use of the medicaments. Material and Methods: Our study is designed as a cross-sectional study. The study included 60 patients with von Willebrand Disease Type 1 von Willebrand factor between 10-50 IU / dl who were followed-up at Ege University Department of Pediatric Hematology between 1997-2018. Patients was divided into groups VWF: Ag 10-30 IU / dl and VWF: Ag 31-50IU / dl according to VWF: Ag level, and divided into groups as 0-18 and 19-35 years of age. Bleeding scores were determined by applying Pediatric Blood Questionnaire to all patients. Pediatric bleeding score was considered as ≥2 positive. Age, symptom onset age, age at diagnosis, pediatric bleeding score, VWF: Ag, VWF: Rco, factor VIII levels and blood group phenotype of all patients were evaluated. Results: Clinical and laboratory data of the groups were compared. In patients with low von Willebrand factor, plasma VWF: Ag level increased as age increased. In terms of pediatric bleeding score, pediatric bleeding score was found to be significantly higher in von Willebrand disease type 1 compared to the Low von Willebrand factor group. The median pediatric bleeding score was found to be 6,9 in the VWH type 1 group in the 0-18 age group and 3,8 in the Low von Willebrand factor group. This difference was statistically significant. No statistically significant difference was found between the groups in terms of median value of pediatric bleeding score in the 19-35 age groups. The median value of FVIII / VWF: Ag ratio was significantly higher in Von Willebrand disease type 1 group compared to the Low von Willebrand factor group. There was no statistically significant difference between the O ve non-O phenotype groups in terms of blood group distribution. Discussion: The median pediatric bleeding score was significantly higher in the VWD type 1 group in the 0-18 age group than in the low VWF group. These data show that the bleeding phenotype is significantly lighter, especially in the pediatric age group, and Low von Willebrand factor. İn addition, these data indicate that the use of the bleeding assessment tools is an important diagnostic step in the differential diagnosis between the two groups.
Aim: Von Willebrand disease is the most common hereditary bleeding disorder caused by a quantitative / qualitative deficiency in the plasma von Willebrand factor. Recent international guidelines recommend the diagnosis of Low von Willebrand factor in patients with a significant bleeding score of at least ≥ 2 times at plasma von Willebrand factor level of 30-50 IU / dL. The aim of our study is to assess the suitability of the use of recommendations by international guidelines on the management of these cases in Turkey population and is to increase awareness about the importance of bleeding assessment tool in the evaluation of cases presenting with bleeding symptoms. In addition, in cases with low von Willebrand factor, if there is no clinically meaningful bleeding, we can remove them from the “patient“ label by removing them from the “patient“ group and to prevent unnecessary use of the medicaments. Material and Methods: Our study is designed as a cross-sectional study. The study included 60 patients with von Willebrand Disease Type 1 von Willebrand factor between 10-50 IU / dl who were followed-up at Ege University Department of Pediatric Hematology between 1997-2018. Patients was divided into groups VWF: Ag 10-30 IU / dl and VWF: Ag 31-50IU / dl according to VWF: Ag level, and divided into groups as 0-18 and 19-35 years of age. Bleeding scores were determined by applying Pediatric Blood Questionnaire to all patients. Pediatric bleeding score was considered as ≥2 positive. Age, symptom onset age, age at diagnosis, pediatric bleeding score, VWF: Ag, VWF: Rco, factor VIII levels and blood group phenotype of all patients were evaluated. Results: Clinical and laboratory data of the groups were compared. In patients with low von Willebrand factor, plasma VWF: Ag level increased as age increased. In terms of pediatric bleeding score, pediatric bleeding score was found to be significantly higher in von Willebrand disease type 1 compared to the Low von Willebrand factor group. The median pediatric bleeding score was found to be 6,9 in the VWH type 1 group in the 0-18 age group and 3,8 in the Low von Willebrand factor group. This difference was statistically significant. No statistically significant difference was found between the groups in terms of median value of pediatric bleeding score in the 19-35 age groups. The median value of FVIII / VWF: Ag ratio was significantly higher in Von Willebrand disease type 1 group compared to the Low von Willebrand factor group. There was no statistically significant difference between the O ve non-O phenotype groups in terms of blood group distribution. Discussion: The median pediatric bleeding score was significantly higher in the VWD type 1 group in the 0-18 age group than in the low VWF group. These data show that the bleeding phenotype is significantly lighter, especially in the pediatric age group, and Low von Willebrand factor. İn addition, these data indicate that the use of the bleeding assessment tools is an important diagnostic step in the differential diagnosis between the two groups.
Açıklama
Anahtar Kelimeler
Von Willebrand Hastalığı, Von Willebrand Faktörü, Kanama, Von Willebrand Diseases, Von Willebrand Factor, Hemorrhage.
