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  • Küçük Resim
    Öğe
    Juvenile Myelomonocytic Leukemia in Turkey: A Retrospective Analysis of Sixty-five Patients
    (2018) Tüfekçi, Özlem; Koçak, Ülker; Kaya, Zühre; Yenicesu, İdil; Albayrak, Canan; Albayrak, Davut; Tokgöz, Hüseyin; Çalışkan, Ümran; Yılmaz Bengoa, Şebnem; Patıroğlu, Türkan; Karakükçü, Musa; Ünal, Ekrem; Ünal İnce, Elif; İleri, Talia; Ertem, Mehmet; Celkan, Tiraje; Özdemir, Gül Nihal; Sarper, Nazan; Kaçar, Dilek; Yaralı, Neşe; Özbek, Namık Yaşar; Küpesiz, Alphan; Karapınar, Tuba; Vergin, Canan; Tokgöz, Hüseyin; Sezgin Evim, Melike; Baytan, Birol; Güneş, Adalet Meral; Yılmaz Karapınar, Deniz; Karaman, Serap; Uygun, Vedat; Karasu, Gülsun; Yeşilipek, Mehmet Akif; Koç, Ahmet; Erduran, Erol; Atabay, Berna; Öniz, Haldun; Ören, Hale
    Amaç: Türkiye'deki juvenil miyelomonositik lösemi (JMML) hastalarının durumunu, tanı zamanı, klinik özellikler, mutasyon çalışmaları, klinik gidiş ve tedavi stratejileri açısından ortaya koymaktır.Gereç ve Yöntemler: Ülkemizdeki pediatrik hematoloji ve onkoloji kliniklerinden veri istenerek, JMML tanısı ile takip ve tedavisi yapılan hastaların klinik ve laboratuvar bulguları geriye dönük olarak değerlendirildi.Bulgular: On sekiz merkezden, 2002-2016 tarihleri arasında JMML tanısı alan toplam 65 hasta çalışmaya dahil edildi. Ortanca tanı yaşı 17 ay idi (2-117 ay). Splenomegali tanıda %92 hastada vardı. Ortanca lökosit, monosit ve trombosit sayıları sırasıyla 32,9x109/L, 5,4x109/L ve 58,3x109/L idi. Monozomi 7, %18 hastada saptanmıştı. JMML mutasyonları 32 hastada (%49) çalışılmış olup, en sık rastlanan mutasyon PTPN11 idi. Hematopoetik kök hücre nakli (HKHN) 7 was present in 18% of patients. JMML mutational analysis was performed in 32 of 65 patients (49%) and PTPN11 was the most common mutation. Hematopoietic stem cell transplantation (HSCT) could only be performed in 28 patients (44%), the majority being after the year 2012. The most frequent reason for not performing HSCT was the inability to find a suitable donor. The median time from diagnosis to HSCT was 9 months (min-max: 2-63 months). The 5-year cumulative survival rate was 33% and median estimated survival time was 3017.4 months (95% CI: 0-64.1) for all patients. Survival time was significantly better in the HSCT group (log-rank p0.019). Older age at diagnosis (2 years), platelet count of less than 40x109/L, and PTPN11 mutation were the factors significantly associated with shorter survival time. Conclusion: Although there has recently been improvement in terms of definitive diagnosis and HSCT in JMML patients, the overall results.
  • Küçük Resim Yok
    Öğe
    Outcomes of Eltrombopag Treatment and Development of Iron Deficiency in Children with Immune Thrombocytopenia in Turkey
    (Galenos Yayincilik, 2020) Yozgat, Ayca Koca; Leblebisatan, Goksel; Akbayram, Sinan; Ozel, Simge Cinar; Karakas, Zeynep; Erduran, Erol; Yilmaz, Sebnem
    Objective: Immune thrombocytopenia (ITP) is a rare autoimmune disease and hematologic disorder characterized by reduced platelet counts that can result in significant symptoms, such as bleeding, bruising, epistaxis, or petechiae. The thrombopoietin receptor agonist eltrombopag (EPAG) is a second-line agent used to treat chronic ITP purpura in adults and children. Materials and Methods: The present retrospective study evaluated the efficacy, safety, and side effects of EPAG treatment in pediatric patients with acute refractory and chronic immune thrombocytopenia, particularly focusing on iron-deficiency anemia. Results: The diagnosis was chronic ITP in 89 patients and acute refractory ITP in 16 patients. The mean age of patients was 9.5 +/- 4.5 years (minimum-maximum: 1.2-18 years) at the beginning of EPAG treatment. The overall response rate was 74.3% (n=78). The mean time for platelet count of >= 50x109/L was 11.6 +/- 8 weeks (range: 1-34 weeks). The treatment was stopped for 27 patients (25.7%) at an average of 6.8 +/- 9 months (range: 1-38 months). The reason for discontinuation was lack of response in 18 patients, nonadherence in 4 patients, and hepatotoxicity in 2 patients. Response to treatment continued for an average of 4 months after cessation of EPAG in 3 patients. Conclusion: Results of the current study imply that EPAG is an effective therapeutic option in pediatric patients with acute refractory and chronic ITP. However, patients must be closely monitored for response and side effects during treatment, and especially for iron deficiency.