Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options

Background: Hemophilia B (HB), an X-linked recessive inherited bleeding disorder, exhibits a high prevalence among males. Objectives: To present the first national cohort of persons with HB to define the demographics, clinical characteristics, and treatment patterns in Turkey. Methods: This multicen...

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Main Authors: Bülent Zülfikar, Başak Koç, Fahri Şahin, Hatice İlgen Şaşmaz, Kaan Kavaklı, Can Balkan, Ali Bülent Antmen, Sinan Akbayram, Birol Güvenç, Vahap Okan, Emine Türkkan, Canan Albayrak, Davut Albayrak, Nazan Sarper, Tülin Tiraje Celkan, Orhan Ayyıldız, Salih Aksu, Türkan Patıroğlu, Zafer Şalcıoğlu, Adalet Meral Güneş, Yasemin Altuner Torun, Ümran Çalışkan, Hüseyin Tokgöz, Yılmaz Ay, Gül Nihal Özdemir, Mehmet Sönmez, Ekrem Ünal, Ahmet Fayik Öner, Nil Güler, Osman Alphan Küpesiz, Hale Ören, Serap Karaman, Ayşegül Ünüvar, Mehmet Dağlı, Ahmet Muzaffer Demir, Murat Söker, Bülent Alioğlu, Zühre Kaya, Aylin Canbolat Ayhan, Zafer Bıçakçı, Yusuf Ziya Aral, Muhlis Cem Ar
Format: Article
Language:English
Published: Elsevier 2024-10-01
Series:Research and Practice in Thrombosis and Haemostasis
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Online Access:http://www.sciencedirect.com/science/article/pii/S2475037924002838
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author Bülent Zülfikar
Başak Koç
Fahri Şahin
Hatice İlgen Şaşmaz
Kaan Kavaklı
Can Balkan
Ali Bülent Antmen
Sinan Akbayram
Birol Güvenç
Vahap Okan
Emine Türkkan
Canan Albayrak
Davut Albayrak
Nazan Sarper
Tülin Tiraje Celkan
Orhan Ayyıldız
Salih Aksu
Türkan Patıroğlu
Zafer Şalcıoğlu
Adalet Meral Güneş
Yasemin Altuner Torun
Ümran Çalışkan
Hüseyin Tokgöz
Yılmaz Ay
Gül Nihal Özdemir
Mehmet Sönmez
Ekrem Ünal
Ahmet Fayik Öner
Nil Güler
Osman Alphan Küpesiz
Hale Ören
Serap Karaman
Ayşegül Ünüvar
Mehmet Dağlı
Ahmet Muzaffer Demir
Murat Söker
Bülent Alioğlu
Zühre Kaya
Aylin Canbolat Ayhan
Zafer Bıçakçı
Yusuf Ziya Aral
Muhlis Cem Ar
author_facet Bülent Zülfikar
Başak Koç
Fahri Şahin
Hatice İlgen Şaşmaz
Kaan Kavaklı
Can Balkan
Ali Bülent Antmen
Sinan Akbayram
Birol Güvenç
Vahap Okan
Emine Türkkan
Canan Albayrak
Davut Albayrak
Nazan Sarper
Tülin Tiraje Celkan
Orhan Ayyıldız
Salih Aksu
Türkan Patıroğlu
Zafer Şalcıoğlu
Adalet Meral Güneş
Yasemin Altuner Torun
Ümran Çalışkan
Hüseyin Tokgöz
Yılmaz Ay
Gül Nihal Özdemir
Mehmet Sönmez
Ekrem Ünal
Ahmet Fayik Öner
Nil Güler
Osman Alphan Küpesiz
Hale Ören
Serap Karaman
Ayşegül Ünüvar
Mehmet Dağlı
Ahmet Muzaffer Demir
Murat Söker
Bülent Alioğlu
Zühre Kaya
Aylin Canbolat Ayhan
Zafer Bıçakçı
Yusuf Ziya Aral
Muhlis Cem Ar
author_sort Bülent Zülfikar
collection DOAJ
description Background: Hemophilia B (HB), an X-linked recessive inherited bleeding disorder, exhibits a high prevalence among males. Objectives: To present the first national cohort of persons with HB to define the demographics, clinical characteristics, and treatment patterns in Turkey. Methods: This multicenter, retrospective study included 433 alive persons with HB registered in 35 centers between 1961 and 2018. Analyses were performed by age subgroups (0-17 years, 18-64 years, and ≥65 years), disease severity by factor levels (severe, <1 IU/dL; moderate, 1-5 IU/dL; mild, >5 IU/dL). Additionally, patients were stratified based on the initiation year of follow-up at the relevant study center, creating 2 periods: 1993-2006 (referred to as period A) and 2007-2018 (referred to as period B). Results: Predominantly male (98.6%), the median age at data entry was 22.1 years (n = 429). The majority (49.0%) had moderate HB, followed by severe (30.0%) and mild (15.7%) disease. Of the 377 patients with complete treatment details, 209 (55.4%) were under prophylaxis from their diagnosis onwards, while 79 patients (21.0%) only received on-demand treatment. Additionally, 89 patients (23.6%) initially underwent on-demand treatment and later were switched to prophylaxis. Knees were the primary site of bleeding and the most frequently intervened joints. Most of the major (47.5%) and minor (53.3%) orthopedic procedures were carried out in persons with severe HB, while half of radioactive synovectomy procedures were performed on persons with moderate HB. Conclusion: This paper describes the demographics, clinical characteristics, and treatments patterns of a large cohort of alive persons with HB on a national scale.
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spelling doaj-art-a0df4776626d4dedb5af1ad41e8bc5f52025-08-20T02:50:14ZengElsevierResearch and Practice in Thrombosis and Haemostasis2475-03792024-10-018710258810.1016/j.rpth.2024.102588Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment optionsBülent Zülfikar0Başak Koç1Fahri Şahin2Hatice İlgen Şaşmaz3Kaan Kavaklı4Can Balkan5Ali Bülent Antmen6Sinan Akbayram7Birol Güvenç8Vahap Okan9Emine Türkkan10Canan Albayrak11Davut Albayrak12Nazan Sarper13Tülin Tiraje Celkan14Orhan Ayyıldız15Salih Aksu16Türkan Patıroğlu17Zafer Şalcıoğlu18Adalet Meral Güneş19Yasemin Altuner Torun20Ümran Çalışkan21Hüseyin Tokgöz22Yılmaz Ay23Gül Nihal Özdemir24Mehmet Sönmez25Ekrem Ünal26Ahmet Fayik Öner27Nil Güler28Osman Alphan Küpesiz29Hale Ören30Serap Karaman31Ayşegül Ünüvar32Mehmet Dağlı33Ahmet Muzaffer Demir34Murat Söker35Bülent Alioğlu36Zühre Kaya37Aylin Canbolat Ayhan38Zafer Bıçakçı39Yusuf Ziya Aral40Muhlis Cem Ar41Hereditary Bleeding Disorders Unit, Istanbul University Oncology Institute, Istanbul, Turkey; Correspondence Bülent Zülfikar, Hereditary Bleeding Disorders Unit, Istanbul University Oncology Institute, Millet Street, Capa/Fatih, 34093 Istanbul, Turkey.Hereditary Bleeding Disorders Unit, Istanbul University Oncology Institute, Istanbul, TurkeyDepartment of Internal Diseases, Division of Hematology, Ege University Medical Faculty Hospital, Izmir, TurkeyDepartment of Pediatric Hematology, Cukurova University Faculty of Medicine, Adana, TurkeyDepartment of Children’s Health and Diseases, Division of Pediatric Hematology, Ege University Medical Faculty Hospital, Izmir, TurkeyDepartment of Children’s Health and Diseases, Division of Pediatric Hematology, Ege University Medical Faculty Hospital, Izmir, TurkeyDepartment of Pediatric Hematology and Oncology, Acibadem Hospital, Adana, TurkeyDepartment of Pediatric Hematology and Oncology, Gaziantep University Medical Faculty, Gaziantep, TurkeyDepartment of Internal Diseases, Division of Hematology, Cukurova University Faculty of Medicine, Adana, TurkeyDepartment of Internal Diseases, Division of Hematology, Gaziantep University Medical Faculty, Gaziantep, TurkeyDepartment of Pediatric Hematology-Oncology, Ministry of Health Prof. Dr. Cemil Taşçıoğlu City Hospital, Istanbul, TurkeyDepartment of Pediatric Hematology and Oncology, Ondokuz Mayıs University Faculty of Medicine, Samsun, TurkeyDepartment of Pediatric Hematology-Oncology, Samsun Medical Park Hospital, Samsun, TurkeyDepartment of Pediatric Hematology, Kocaeli University School of Medicine, Kocaeli, TurkeyDepartment of Pediatric Hematology and Oncology, İstinye University, Istanbul, TurkeyDepartment of Internal Medicine, Division of Hematology, Dicle University Medical Faculty, Diyarbakır, TurkeyDepartment of Internal Medicine, Division of Hematology, Hacettepe University Faculty of Medicine, Ankara, TurkeyDivision of Pediatric Immunology and Oncology, Losante Children’s and Adult Hospital, Ankara, TurkeyDepartment of Pediatric Hematology, Kanuni Sultan Süleyman Training and Research Hospital, Istanbul, TurkeyDepartment of Pediatric Hematology and Oncology, Uludağ University Faculty of Medicine, Bursa, TurkeyDepartment of Pediatric Hematology and Oncology, İstinye University, Istanbul, TurkeyDepartment of Pediatric Hematology, Necmettin Erbakan University Meram Medical Faculty Hospital, Konya, TurkeyDepartment of Pediatric Hematology, Necmettin Erbakan University Meram Medical Faculty Hospital, Konya, TurkeyDepartment of Pediatric Hematology, Pamukkale University School of Medicine, Denizli, TurkeyDepartment of Pediatric Hematology and Oncology, İstinye University, Istanbul, TurkeyDepartment of Internal Medicine, Division of Hematology, Karadeniz Technical University Faculty of Medicine, Trabzon, TurkeyDepartment of Pediatrics, Division of Pediatric Hematology and Oncology, Erciyes University Faculty of Medicine, Kayseri, TurkeyDepartment of Pediatric Hematology and Oncology, Yuzuncu Yil University Faculty of Medicine, Van, TurkeyDepartment of Internal Medicine, Division of Hematology, Pamukkale University School of Medicine, Denizli, TurkeyDepartment of Pediatric Hematology, Akdeniz University Faculty of Medicine, Antalya, TurkeyDepartment of Pediatrics, Division of Pediatric Hematology, Dokuz Eylul University Faculty of Medicine, İzmir, TurkeyDepartment of Pediatrics, Division of Pediatric Hematology-Oncology, Istanbul University Istanbul Faculty of Medicine, Istanbul, TurkeyDepartment of Pediatrics, Division of Pediatric Hematology-Oncology, Istanbul University Istanbul Faculty of Medicine, Istanbul, TurkeyDepartment of Internal Medicine, Division of Hematology, Selcuk University Faculty of Medicine, Konya, TurkeyDepartment of Internal Medicine, Division of Hematology, Trakya University Faculty of Medicine, Edirne, TurkeyDepartment of Pediatric Hematology, Dicle University Medical Faculty, Diyarbakır, TurkeyDepartment of Pediatric Hematology and Oncology, Ankara Training and Research Hospital, Ankara, TurkeyDepartment of Pediatric Hematology, Gazi University Faculty of Medicine, Ankara, TurkeyDepartment of Pediatric Oncology and Hematology, Istanbul Medeniyet University Faculty of Medicine, Istanbul, TurkeyDepartment of Pediatric Hematology and Oncology, Atatürk University, Erzurum, TurkeyDepartment of Pediatric Hematology, Adnan Menderes University School of Medicine, Aydın, TurkeyDepartment of Internal Medicine, Division of Hematology, Istanbul University – Cerrahpasa, Cerrahpasa Faculty of Medicine, Istanbul, TurkeyBackground: Hemophilia B (HB), an X-linked recessive inherited bleeding disorder, exhibits a high prevalence among males. Objectives: To present the first national cohort of persons with HB to define the demographics, clinical characteristics, and treatment patterns in Turkey. Methods: This multicenter, retrospective study included 433 alive persons with HB registered in 35 centers between 1961 and 2018. Analyses were performed by age subgroups (0-17 years, 18-64 years, and ≥65 years), disease severity by factor levels (severe, <1 IU/dL; moderate, 1-5 IU/dL; mild, >5 IU/dL). Additionally, patients were stratified based on the initiation year of follow-up at the relevant study center, creating 2 periods: 1993-2006 (referred to as period A) and 2007-2018 (referred to as period B). Results: Predominantly male (98.6%), the median age at data entry was 22.1 years (n = 429). The majority (49.0%) had moderate HB, followed by severe (30.0%) and mild (15.7%) disease. Of the 377 patients with complete treatment details, 209 (55.4%) were under prophylaxis from their diagnosis onwards, while 79 patients (21.0%) only received on-demand treatment. Additionally, 89 patients (23.6%) initially underwent on-demand treatment and later were switched to prophylaxis. Knees were the primary site of bleeding and the most frequently intervened joints. Most of the major (47.5%) and minor (53.3%) orthopedic procedures were carried out in persons with severe HB, while half of radioactive synovectomy procedures were performed on persons with moderate HB. Conclusion: This paper describes the demographics, clinical characteristics, and treatments patterns of a large cohort of alive persons with HB on a national scale.http://www.sciencedirect.com/science/article/pii/S2475037924002838factor IXhemophilia Bjoint diseasesprophylaxisretrospective studiessurgery
spellingShingle Bülent Zülfikar
Başak Koç
Fahri Şahin
Hatice İlgen Şaşmaz
Kaan Kavaklı
Can Balkan
Ali Bülent Antmen
Sinan Akbayram
Birol Güvenç
Vahap Okan
Emine Türkkan
Canan Albayrak
Davut Albayrak
Nazan Sarper
Tülin Tiraje Celkan
Orhan Ayyıldız
Salih Aksu
Türkan Patıroğlu
Zafer Şalcıoğlu
Adalet Meral Güneş
Yasemin Altuner Torun
Ümran Çalışkan
Hüseyin Tokgöz
Yılmaz Ay
Gül Nihal Özdemir
Mehmet Sönmez
Ekrem Ünal
Ahmet Fayik Öner
Nil Güler
Osman Alphan Küpesiz
Hale Ören
Serap Karaman
Ayşegül Ünüvar
Mehmet Dağlı
Ahmet Muzaffer Demir
Murat Söker
Bülent Alioğlu
Zühre Kaya
Aylin Canbolat Ayhan
Zafer Bıçakçı
Yusuf Ziya Aral
Muhlis Cem Ar
Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options
Research and Practice in Thrombosis and Haemostasis
factor IX
hemophilia B
joint diseases
prophylaxis
retrospective studies
surgery
title Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options
title_full Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options
title_fullStr Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options
title_full_unstemmed Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options
title_short Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options
title_sort retrospective analysis of hemophilia b in turkey identifying main characteristics and treatment options
topic factor IX
hemophilia B
joint diseases
prophylaxis
retrospective studies
surgery
url http://www.sciencedirect.com/science/article/pii/S2475037924002838
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