Browsing by Author "Pekpak, Esra"

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Clinical Spectrum of Primary Hemophagocytic Lymphohistiocytosis: Experience of Reference Centers in Central and Southeast Anatolia
(Springer, 2024) Akyol, Sefika; Yilmaz, Ebru; Tokgoz, Huseyin; Karaman, Kamuran; Pekpak, Esra; Ozcan, Alper; Unal, Ekrem
Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening disease, with a high mortality if left untreated. In addition, the disease has unique diagnostic challenges. Therefore, despite the existing guidelines on management, current clinical practice data is informative on the course and outcome. Herein, a retrospective chart review study was conducted through the collaboration of six centers, located in central and southeastern Turkiye. The demographical data, laboratory results, and treatment outcomes were evaluated. Eighty-three patients were enrolled in the study. The mean age was 2 years, whereas the median age was 8 months with a range of a minimum of 1 week and a maximum of 12.6 years. Consanguineous marriage, history of sibling death, and familial history of similar disease were determined in 72.2% (n:60), 34.9% (n:29), and 39.8% (n:33) of the patients, respectively. The most common presentation was fever, followed by hepatosplenomegaly on admission. Disease-causing familial HLH variants were identified in 60.2% (n:50) of the patients. Hematopoietic stem cell transplantation (HSCT) was performed in 39.7% (n:33) of the cohort. The 2-year overall survival (OS) rate was 62.4% for the whole group. Comparing the patients who received HSCT and those who did not; the HSCT group had a 2-year OS of 84.7%, which was significantly better than patients who did not receive HSCT had a 2-year OS of 47.1% (p:0.001). Despite the improvement in HLH diagnostics and treatment options over the last decade, early death remains a leading problem for the survival of these patients. Therefore, appropriate assessment of the patients in experienced centers and HSCT are pivotal for better outcomes.
Different Kinetics and Risk Factors for Isolated Extramedullary Relapse After Allogeneic Hematopoietic Stem Cell Transplantation in Children With Acute Leukemia
(Elsevier Science inc, 2021) Hazar, Volkan; Ozturk, Gulyuz; Yalcin, Koray; Uygun, Vedat; Aksoylar, Serap; Kupesiz, A.; Pekpak, Esra
Relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the most frequent cause of post-transplantation mortality. Isolated extramedullary (EM) relapse (iEMR) after HSCT is relatively rare and not well characterized, particularly in pediatric patients. We retrospectively analyzed 1527 consecutive pediatric patients with acute leukemia after allo-HSCT to study the incidence, risk factors, and outcome of iEMR compared with systemic relapse. The 5 -year cumulative incidence of systemic relapse (either bone marrow [BM] only or BM combined with EMR) was 24.8%, and that of iEMR was 5.5%. The onset of relapse after allo-HSCT was significantly longer in EM sites than in BM sites (7.19 and 5.58 months, respectively; P =.013). Complete response (CR) 2 +/active disease at transplantation (hazard ratio [HR], 3.1; P <.001) and prior EM disease (HR, 2.3; P =.007) were independent risk factors for iEMR. Chronic graft-versus-host disease reduced the risk of systemic relapse (HR, 0.5; P=.043) but did not protect against iEMR. The prognosis of patients who developed iEMR remained poor but was slightly better than that of patients who developed systemic relapse (3 -year overall survival, 16.5% versus 15.3%; P =.089). Patients experiencing their first systemic relapse continued to have further systemic relapse, but only a minority progressed to iEMR, whereas those experiencing their iEMR at first relapse developed further systemic relapse and iEMR at approximately similar frequencies. A second iEMR was more common after a first iEMR than after a first systemic relapse (58.8% versus 13.0%; P =.001) and was associated with poor outcome. iEMR has a poor prognosis, particularly after a second relapse, and effective strategies are needed to improve outcomes. (C) 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.
Results of Multicenter Registry for Patients With Inherited Factor Vii Deficiency in Turkey
(Taylor & Francis Ltd, 2022) Akdeniz, Aydan; Unuvar, Aysegul; Ar, Muhlis Cem; Pekpak, Esra; Akyay, Arzu; Mehtap, Ozgur; Demir, Ahmet Muzaffer
Introduction: Inherited factor VII (FVII) deficiency (FVIID) is the most common of inherited rare bleeding disorders. Other determinants of clinical severity apart from FVII level (FVIIL) include genetic and environmental factors. We aimed to identify the cut-off FVIILs for general and severe bleedings in patients with FVIID by using an online national registry system including clinical, laboratory, and demographic characteristics of patients. Methods: Demographic, clinical, and laboratory data of patients with FVIID extracted from the national database, constituted by the Turkish Society of Hematology, were examined. Bleeding phenotypes, general characteristics, and laboratory features were assessed in terms of FVIILs. Bleeding rates and prophylaxis during special procedures/interventions were also recorded. Results: Data from 197 patients showed that 46.2% of patients had FVIIL< 10%. Most bleeds were of mucosal origin (67.7%), and severe bleeds tended to occur in younger patients (median age: 15 (IQR:6-29)). Cut-off FVIILs for all and severe bleeds were 16.5% and 7.5%, respectively. The major reason for long-term prophylaxis was observed as central nervous system bleeding (80%). Conclusion: Our data are consistent with most of the published literature in terms of cut-off FVIIL for bleeding, as well as reasons for prophylaxis, showing both an increased severity of bleeding and younger age at diagnosis with decreasing FVIIL. However, in order to offer a classification similar to that in Hemophilia A or B, data of a larger cohort with information about environmental and genetic factors are required.