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    A Multi-Center Study on the Efficacy of Eltrombopag in Management of Refractory Chronic Immune Thrombocytopenia: A Real-Life Experience
    (2019) Çekdemir, Demet; Güvenç, Serkan; Özdemirkıran, Füsun; Eser, Ali; Şahin Haydaroğlu, Handan; Turak Ermiş, Esra; Esen, Ramazan; Cömert, Melda; Sadri, Sevil; Aslaner, Müzeyyen; Uncu Ulu, Bahar; Karakuş, Abdullah; Bapur Selim, Derya; Alacacıoğlu, İnci; Aydın, Demet; Tekinalp, Atakan; Namdaroğlu, Sinem; Ceran, Funda; Tarkun, Pınar; Kiper, Demet; Çetiner, Mustafa; Yenerel, Mustafa; Demir, Muzaffer Ahmet; Yılmaz, Güven; Terzi, Hatice; Atilla, Erden; Malkan, Yavuz Ümit; Acar, Kadir; Öztürk, Erman; Tombak, Anıl; Sunu, Cenk; Salim, Ozan; Alayvaz, Nevin; Sayan, Özkan; Ozan, Ülkü; Ozan, Mesut; Gökgöz, Zafer; Andıç, Neslihan; Kızılkılıç, Ebru; Noyan, Figen; Özen, Mehmet; Tanrıkulu Pepedil, Funda; Alanoğlu, Güçhan; Özkan, Hasan Atilla; Aslan, Vahap; Çetin, Güven; Akyol Erikçi, Alev; Deveci, Burak; Ersoy Dursun, Fadime; Dermenci, Hasan; Aytan, Pelin; Gündüz, Mehmet; Karakuş, Volkan; Özlü, Can; Demircioğlu, Sinan; Akay Yanar, Olga Meltem; Özatlı, Düzgün
    Abstract: Objective: The aim of the present study was to evaluate the efficacyand safety of eltrombopag, an oral thrombopoietin receptor agonist,in patients with chronic immune thrombocytopenia (ITP).Materials and Methods: A total of 285 chronic ITP patients (187women, 65.6%; 98 men, 34.4%) followed in 55 centers were enrolledin this retrospective cohort. Response to treatment was assessedaccording to platelet count (/mm3) and defined as complete (plateletcount of >100,000/mm3), partial (30,000-100,000/mm3 or doublingof platelet count after treatment), or unresponsive (<30,000/mm3).Clinical findings, descriptive features, response to treatment, and sideeffects were recorded. Correlations between descriptive, clinical, andhematological parameters were analyzed.Results: The median age at diagnosis was 43.9±20.6 (range: 3-95)years and the duration of follow-up was 18.0±6.4 (range: 6-28.2)months. Overall response rate was 86.7% (n=247). Complete andpartial responses were observed in 182 (63.8%) and 65 (22.8%)patients, respectively. Thirty-eight patients (13.4%) did not respondto eltrombopag treatment. For patients above 60 years old (n=68),overall response rate was 89.7% (n=61), and for those above 80 years old (n=12), overall response rate was 83% (n=10). Consideringthrombocyte count before treatment, eltrombopag significantlyincreased platelet count at the 1st, 2nd, 3rd, 4th, and 8th weeks oftreatment. As the time required for partial or complete responseincreased, response to treatment was significantly reduced. The timeto reach the maximum platelet levels after treatment was quitevariable (1-202 weeks). Notably, the higher the maximum plateletcount after eltrombopag treatment, the more likely that side effectswould occur. The most common side effects were headache (21.6%),weakness (13.7%), hepatotoxicity (11.8%), and thrombosis (5.9%).Conclusion: Results of the current study imply that eltrombopag isan effective therapeutic option even in elderly patients with chronicITP. However, patients must be closely monitored for response andside effects during treatment. Since both response and side effectsmay be variable throughout the follow-up period, patients should beevaluated dynamically, especially in terms of thrombotic risk factors.
  • Küçük Resim Yok
    Öğe
    Study for the Diagnostic Screening of Paroxysmal Nocturnal Hemoglobinuria in Older Patients with Unexplained Anemia and/or Cytopenia
    (Clin Lab Publ, 2020) Ozdemir, Zehra N.; Ilhan, Osman; Ozet, Gulsum; Falay, Mesude; Yenerel, Mustafa; Tuglular, Tulin; Turgut, Mehmet
    Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell disease that may lead to weakness and death of patients, if unrecognized and untreated. Although consensus guidelines were reviewed recently for the diagnostic screening of PNH with multi-parameter flow cytometry (FCM), until now, no study has investigated the efficiency of such clinical indications in older patients. Methods: Overall, 20 centers participated in the study and a total of 1,689 patients were included, 313 of whom were at geriatric age and 1,376 were aged 18 - 64 years. We evaluated the efficiency of consensus clinical indications for PNH testing using FCM in peripheral blood samples and compared the results of older patients and patients aged 18 - 64 years. Results: PNH clones were detected positive in 7/313 (2.2%) of the older patients. Five (74.4%) of the patients with PNH clones had aplastic anemia, 1 had unexplained cytopenia, and 1 patient had myelodysplastic syndrome (MDS) with refractory anemia. PNH clones were not detected in any older patients who were screened for unexplained thrombosis, Coombs (-) hemolytic anemia, hemoglobinuria, and others (e.g., elevated lactate dehydrogenase (LDH), splenomegaly). We detected PNH clones in 55/1376 (4%) samples of the patients aged under 65 years. Forty-two (76.4%) patients with PNH clones had aplastic anemia, 2 patients had Coombs (-) hemolytic anemia, 3 patients had unexplained cytopenia, 1 patient had MDS with refractory anemia, 1 patient had hemoglobinuria, and 6 (10.9%) had others (e.g., elevated LDH, splenomegaly). PNH clones were not detected in any patients who were screened for unexplained thrombosis. There was no statistical difference between the geriatric population and patients aged 18 - 64 years in terms of clinical indications for PNH screening with FCM (p = 0.49). Conclusions: Our results showed that the current clinical indications for PNH screening with FCM were also efficient in older patients. We suggest that older patients with unexplained anemia, myelodysplastic syndrome with refractory anemia, and unexplained cytopenia should be screened for PNH with FCM to identify patients who would benefit from treatment.