Rare Coagulation Factor Deficiencies: Multicenter Experience With 188 Cases

Objective: Rare factor deficiencies are a group of autosomal recessive bleeding disorders (with the exception of dysfibrinogenemia), which are characterized by the deficiency or dysfunction of one or more coagulation factors (F)I, FII, FV, FV+FVIII, FVII, FX, FXI, FXII, and FXIII.Materials and Metho...

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Main Authors: Veysel Gök, Esra Pekpak Şahinoğlu, Hüseyin Tokgöz, Fatma Türkan Mutlu, Can Acıpayam, Kamuran Karaman, Defne Ay Tuncel, Ayşe Ceyda Ören, Ayşe Şimşek, Bilal Arslan, Hatice Beyza Ünal, Alper Özcan, Ebru Yılmaz, Sinan Akbayram, Musa Karakükcü, Ahmet Fayik Öner, Ümran Çalışkan, Türkan Patıroğlu, Ekrem Ünal
Format: Article
Language:English
Published: Istanbul University Press 2023-12-01
Series:Çocuk Dergisi
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Online Access:https://cdn.istanbul.edu.tr/file/JTA6CLJ8T5/23AA12B34C34470EA0696EE2FDDBD12D
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author Veysel Gök
Esra Pekpak Şahinoğlu
Hüseyin Tokgöz
Fatma Türkan Mutlu
Can Acıpayam
Kamuran Karaman
Defne Ay Tuncel
Ayşe Ceyda Ören
Ayşe Şimşek
Bilal Arslan
Hatice Beyza Ünal
Alper Özcan
Ebru Yılmaz
Sinan Akbayram
Musa Karakükcü
Ahmet Fayik Öner
Ümran Çalışkan
Türkan Patıroğlu
Ekrem Ünal
author_facet Veysel Gök
Esra Pekpak Şahinoğlu
Hüseyin Tokgöz
Fatma Türkan Mutlu
Can Acıpayam
Kamuran Karaman
Defne Ay Tuncel
Ayşe Ceyda Ören
Ayşe Şimşek
Bilal Arslan
Hatice Beyza Ünal
Alper Özcan
Ebru Yılmaz
Sinan Akbayram
Musa Karakükcü
Ahmet Fayik Öner
Ümran Çalışkan
Türkan Patıroğlu
Ekrem Ünal
author_sort Veysel Gök
collection DOAJ
description Objective: Rare factor deficiencies are a group of autosomal recessive bleeding disorders (with the exception of dysfibrinogenemia), which are characterized by the deficiency or dysfunction of one or more coagulation factors (F)I, FII, FV, FV+FVIII, FVII, FX, FXI, FXII, and FXIII.Materials and Methods: 188 patients with a rare factor deficiency from seven distinct pediatric hematology centers in Turkey were obtained for the study. Results: 60 (31.9%) patients had a family history of bleeding. Consanguinity was detected in 85 patients (45.2%). 128 patients (68.1%) were symptomatic; the most common bleeding symptom was epistaxis (34.6%) and followed by the bleeding of skin (19.1%), oral cavity (16.1%), soft tissue (8%), central nervous system (CNS) (6.2%), uterine (4.9%), joint (3.7%), gastrointestinal system (GIS) (3.7%), and urinary system (US) (3.7%). The first bleeding sites consist of nose (39%), CNS (10.9%), oral cavity (10.9%), skin (10.9%), umbilical cord (10.2%), GIS (5.5%), US (5.5%), heel (4.7%), and musculoskeletal system (2.3%). CNS hemorrhage was the most common in fibrinogen (n:4), FVII (n:6), and FX (n:2) deficiency, umbilical cord bleeding was the most common in fibrinogen (n:3) and FXIII (n:7) deficiency, heel bleeding was frequently seen in fibrinogen (n:6) deficiency. The life-threatening bleedings were CNS (n:27, 77.1%), GIS (n:7, 20%), and iliopsoas (n:1, 2.9%), respectively. The reasons leading to the diagnosis were bleeding (57.4%), preoperative screening (15.4%), incidental (15.4%), family history (6.4%), and postoperative bleeding (5.3%). 2/5 FXII deficiency patients had mild bleeding symptoms. Conclusion: As bleeding disorders are somehow a rare group of disorder, early diagnosis and treatment are critical to reduce the high morbidity and mortality
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series Çocuk Dergisi
spelling doaj-art-4eb278f9690c44f88b201a41cd52c4e72025-08-20T01:51:16ZengIstanbul University PressÇocuk Dergisi1308-84912023-12-0123434935510.26650/jchild.2023.1308877123456Rare Coagulation Factor Deficiencies: Multicenter Experience With 188 CasesVeysel Gök0https://orcid.org/0000-0002-7195-2688Esra Pekpak Şahinoğlu1https://orcid.org/0000-0003-2143-1435Hüseyin Tokgöz2https://orcid.org/0000-0002-3064-4646Fatma Türkan Mutlu3https://orcid.org/0000-0003-4593-8679Can Acıpayam4https://orcid.org/0000-0002-6379-224XKamuran Karaman5Defne Ay Tuncel6https://orcid.org/0000-0002-1262-8271Ayşe Ceyda Ören7https://orcid.org/0000-0002-7676-0095Ayşe Şimşek8https://orcid.org/0000-0001-9250-078XBilal Arslan9https://orcid.org/0009-0001-3907-0888Hatice Beyza Ünal10https://orcid.org/0000-0002-5684-5657Alper Özcan11https://orcid.org/0000-0002-6100-1205Ebru Yılmaz12https://orcid.org/0000-0003-4802-0986Sinan Akbayram13https://orcid.org/0000-0001-7410-4310Musa Karakükcü14https://orcid.org/0000-0003-2015-3541Ahmet Fayik Öner15https://orcid.org/0000-0003-3858-8105Ümran Çalışkan16https://orcid.org/0000-0003-4695-7046Türkan Patıroğlu17https://orcid.org/0000-0003-2471-764XEkrem Ünal18https://orcid.org/0000-0002-2691-4826Erciyes Üniversitesi, Kayseri, TurkiyeGaziantep Üniversitesi, Gaziantep, TurkiyeNecmettin Erbakan Üniversitesi, Konya, TurkiyeKayseri Sağlık Bilimleri Üniversitesi, Kayseri, TürkiyeKahramanmaraş Sütçü İmam Üniversitesi, Kahramanmaras, TurkiyeVan Yüzüncü Yıl Üniversitesi, Van, TurkiyeAdana Sağlık Bilimleri Üniversitesi, Adana, TürkiyeGaziantep Üniversitesi, Gaziantep, TurkiyeNecmettin Erbakan Üniversitesi, Konya, TurkiyeVan Yüzüncü Yıl Üniversitesi, Van, TurkiyeErciyes Üniversitesi, Kayseri, TurkiyeErciyes Üniversitesi, Kayseri, TurkiyeErciyes Üniversitesi, Kayseri, TurkiyeGaziantep Üniversitesi, Gaziantep, TurkiyeErciyes Üniversitesi, Kayseri, TurkiyeVan Yüzüncü Yıl Üniversitesi, Van, TurkiyeNecmettin Erbakan Üniversitesi, Konya, TurkiyeErciyes Üniversitesi, Kayseri, TurkiyeHasan Kalyoncu Üniversitesi, Gaziantep, TurkiyeObjective: Rare factor deficiencies are a group of autosomal recessive bleeding disorders (with the exception of dysfibrinogenemia), which are characterized by the deficiency or dysfunction of one or more coagulation factors (F)I, FII, FV, FV+FVIII, FVII, FX, FXI, FXII, and FXIII.Materials and Methods: 188 patients with a rare factor deficiency from seven distinct pediatric hematology centers in Turkey were obtained for the study. Results: 60 (31.9%) patients had a family history of bleeding. Consanguinity was detected in 85 patients (45.2%). 128 patients (68.1%) were symptomatic; the most common bleeding symptom was epistaxis (34.6%) and followed by the bleeding of skin (19.1%), oral cavity (16.1%), soft tissue (8%), central nervous system (CNS) (6.2%), uterine (4.9%), joint (3.7%), gastrointestinal system (GIS) (3.7%), and urinary system (US) (3.7%). The first bleeding sites consist of nose (39%), CNS (10.9%), oral cavity (10.9%), skin (10.9%), umbilical cord (10.2%), GIS (5.5%), US (5.5%), heel (4.7%), and musculoskeletal system (2.3%). CNS hemorrhage was the most common in fibrinogen (n:4), FVII (n:6), and FX (n:2) deficiency, umbilical cord bleeding was the most common in fibrinogen (n:3) and FXIII (n:7) deficiency, heel bleeding was frequently seen in fibrinogen (n:6) deficiency. The life-threatening bleedings were CNS (n:27, 77.1%), GIS (n:7, 20%), and iliopsoas (n:1, 2.9%), respectively. The reasons leading to the diagnosis were bleeding (57.4%), preoperative screening (15.4%), incidental (15.4%), family history (6.4%), and postoperative bleeding (5.3%). 2/5 FXII deficiency patients had mild bleeding symptoms. Conclusion: As bleeding disorders are somehow a rare group of disorder, early diagnosis and treatment are critical to reduce the high morbidity and mortalityhttps://cdn.istanbul.edu.tr/file/JTA6CLJ8T5/23AA12B34C34470EA0696EE2FDDBD12Dbleedingdeficiencyfactorrare
spellingShingle Veysel Gök
Esra Pekpak Şahinoğlu
Hüseyin Tokgöz
Fatma Türkan Mutlu
Can Acıpayam
Kamuran Karaman
Defne Ay Tuncel
Ayşe Ceyda Ören
Ayşe Şimşek
Bilal Arslan
Hatice Beyza Ünal
Alper Özcan
Ebru Yılmaz
Sinan Akbayram
Musa Karakükcü
Ahmet Fayik Öner
Ümran Çalışkan
Türkan Patıroğlu
Ekrem Ünal
Rare Coagulation Factor Deficiencies: Multicenter Experience With 188 Cases
Çocuk Dergisi
bleeding
deficiency
factor
rare
title Rare Coagulation Factor Deficiencies: Multicenter Experience With 188 Cases
title_full Rare Coagulation Factor Deficiencies: Multicenter Experience With 188 Cases
title_fullStr Rare Coagulation Factor Deficiencies: Multicenter Experience With 188 Cases
title_full_unstemmed Rare Coagulation Factor Deficiencies: Multicenter Experience With 188 Cases
title_short Rare Coagulation Factor Deficiencies: Multicenter Experience With 188 Cases
title_sort rare coagulation factor deficiencies multicenter experience with 188 cases
topic bleeding
deficiency
factor
rare
url https://cdn.istanbul.edu.tr/file/JTA6CLJ8T5/23AA12B34C34470EA0696EE2FDDBD12D
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