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  • Küçük Resim
    Öğe
    Different kinetics and risk factors for ısolated extramedullary relapse after allogeneic hematopoietic stem cell transplantation in children with acute leukemia
    (Elsevier, 2021) Hazar, Volkan; Öztürk, Gülyüz; Yalçın, Koray; Uygun, Vedat; Aksoylar, Serap; Küpesiz, A.; Ok Bozkaya, İkbal; Karagün, Barbaros Şahin; İleri, Talia; Atay, Didem; Koçak, Ülker; Tezcan Karasu, Gülsün; Yeşilipek, Akif; Gökçe, Müge; Kansoy, Savaş; Tüysüz Kintrup, Gülen; Karakükçü, Musa; Visal Okur, Fatma; Ertem, Mehmet; Kaya, Zühre; Gürsel, Orhan; Yaman, Yöntem; Özbek, Namık; Antmen, Bülent; Tüfekçi, Özlem; Albayrak, Canan; Adaklı Aksoy, Başak; Sezgin, Gülay; Albayrak, Davut; Sezgin Evim, Melike; Zengin, Emine; Pekpak, Esra; Turkish Pediatric Bone Marrow Transplantation Study Group
    Relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the most frequent cause ofpost-transplantation mortality. Isolated extramedullary (EM) relapse (iEMR) after HSCT is relatively rare and notwell characterized, particularly in pediatric patients. We retrospectively analyzed 1527 consecutive pediatricpatients with acute leukemia after allo-HSCT to study the incidence, risk factors, and outcome of iEMR comparedwith systemic relapse. The 5-year cumulative incidence of systemic relapse (either bone marrow [BM] only or BMcombined with EMR) was 24.8%, and that of iEMR was 5.5%. The onset of relapse after allo-HSCT was significantlylonger 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 wasslightly better than that of patients who developed systemic relapse (3-year overall survival, 16.5% versus 15.3%;P= .089). Patients experiencing theirfirst systemic relapse continued to have further systemic relapse, but only aminority progressed to iEMR, whereas those experiencing their iEMR atfirst relapse developed further systemicrelapse and iEMR at approximately similar frequencies. A second iEMR was more common after afirst iEMR thanafter afirst systemic relapse (58.8% versus 13.0%;P= .001) and was associated with poor outcome. iEMR has apoor prognosis, particularly after a second relapse, and effective strategies are needed to improve outcomes.© 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.
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    Öğe
    Neurologic status of patients with purine nucleoside phosphorylase deficiency before and after hematopoetic stem cell transplantation
    (2023) Gemici Karaaslan, Betül; Turan, Işılay; Aydemir, Sezin; Akyüncü Meriç, Zeynep; Atay, Didem; Akçay, Arzu; Ayaz Sarı, Aysun; Hersfield, Michael; Çipe, Funda; Adaklı Aksoy, Başak; Zengin Ersoy, Gizem; Bozkurt, Ceyhun; Kendir Demirkol, Yasemin; Öztürk, Gülyüz; Aydoğmuş, Çiğdem; Kıykım, Ayça; Çokuğraş, Haluk
    Background Purine nucleoside phosphorylase (PNP) defciency is a rare autosomal recessive combined immunodefciency. The phenotype is profound T cell defciency with variable B and NK cell functions and results in recurrent and persistent infections that typically begin in the frst year of life. Neurologic fndings occur in approximately two-thirds of patients. The mechanism of neurologic abnormalities is unclear. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for PNP defciency. Methods We report here six patients from fve unrelated families with PNP defciency treated in two centers in Turkey. We evaluated the neurological status of patients and compared to post-transplantation period if available. Then, we performed PubMed, Google Scholar, and Researchgate searches using the terms “PNP” and “hematopoietic stem cell transplantation” to fnd all reported cases of PNP transplantation and compared to our cohort. Results Six patients were treated in two centers in Turkey. One patient died from post-transplant complications. The other four patients underwent successful HSCT with good immune reconstitution after transplantation (follow-up 21–48 months) and good neurological outcomes. The other patient with a new mutation is still waiting for a matching HLA donor. Discussion In PNP defciency, clinical manifestations are variable, and this disease should be considered in the presence of many diferent clinical fndings. Despite the comorbidities that occurred before transplantation, HSCT currently appears to be the only treatment option for this disease. HSCT not only cures immunologic disorders, but probably also improves or at least stabilizes the neurologic status of patients.
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    Öğe
    Thalassemia-free and graft-versus-host-free survival: Outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience
    (Bone Marrow Transplantation, 2022) Yeşilipek, M. Akif; Uygun, Vedat; Küpesiz, Alphan; Karasu, Gülsün; Öztürk, Gülyüz; Ertem, Mehmet; Şaşmaz, İlgen; Daloğlu, Hayriye; Güler, Elif; Hazar, Volkan; Fışgın, Tunç; Sezgin, Gülay; Kansoy, Savaş; Kuşkonmaz, Barış; Akıncı, Burcu; Özbek, Namık; Ünal-İnce, Elif; Öztürkmen, Seda; Küpesiz-Tayfun, Funda; Yalçın, Koray; Anak, Sema; Bozkurt, Ceyhun; Karakükçü, Musa; Küpeli, Serhan; Albayrak, Davut; Öniz, Haldun; Aksoylar, Serap; Visal-Okur, Fatma; Albayrak, Canan; Demir-Yenigürbüz, Fatma; Ok-Bozkaya, İkbal; İleri, Talia; Gürsel, Orhan; Karagün, Barbaros Şahin; Tüysüz-Kintrup, Gülen; Çelen, Suna; Elli, Murat; Adaklı-Aksoy, Başak; Yılmaz, Ebru; Tanyeli, Atila; Turan-Akyol, Şule; Önder-Siviş, Zuhal; Özek, Gülcihan; Uçkan, Duygu; Kartal, İbrahim; Atay, Didem; Arzu, Akyay; Arman-Bilir, Özlem; Çakmaklı, Hasan Fatih; Kürekçi, Emin; Malbora, Barış; Akbayram, Sinan; Demir, Hacı Ahmet; Kılıç, Suar Çakı; Güneş, Adalet Meral; Zengin, Emine; Özmen, Salih; Antmen, Ali Bülent
    We report the national data on the outcomes of hematopoietic stem cell transplantation (HSCT) for thalassemia major (TM) patients in Turkey on behalf of the Turkish Pediatric Stem Cell Transplantation Group. We retrospectively enrolled 1469 patients with TM who underwent their first HSCT between 1988 and 2020 in 25 pediatric centers in Turkey. The median follow-up duration and transplant ages were 62 months and 7 years, respectively; 113 patients had chronic graft versus host disease (cGVHD) and the cGVHD rate was 8.3% in surviving patients. Upon the last visit, 30 patients still had cGvHD (2.2%). The 5-year overall survival (OS), thalassemia-free survival (TFS) and thalassemia-GVHD-free survival (TGFS) rates were 92.3%, 82.1%, and 80.8%, respectively. cGVHD incidence was significantly lower in the mixed chimerism (MC) group compared to the complete chimerism (CC) group (p < 0.001). In survival analysis, OS, TFS, and TGFS rates were significantly higher for transplants after 2010. TFS and TGFS rates were better for patients under 7 years and at centers that had performed over 100 thalassemia transplants. Transplants from matched unrelated donors had significantly higher TFS rates. We recommend HSCT before 7 years old in thalassemia patients who have a matched donor for improved outcomes.
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    Öğe
    The diagnostic value of hepatic arterial velocity in venoocclusive disease after pediatric hematopoietic stem cell transplantation
    (Lippincott Williams & Wilkins, 2017) Kaya, Nusabe; Erbey, Fatih; Atay, Didem; Akçay, Arzu; Bozkurt, Ceyhun; ÖztÜrk, GÜlyÜz
    The aim of this study was to determine usefulness of measurements of maximal systolic velocity of the hepatic artery with Doppler ultrasonography in the diagnosis of venoocclusive disease (VOD) after hematopoietic stem cell transplantation. We prospectively obtained 5 sonograms per patient: pretransplantation, day + 1, + 7, + 14, and + 28 on 36 nonconsecutive children who underwent hematopoietic stem cell transplantation. We examined the hepatic artery, the portal, hepatic and splenic veins, the thickness of the gallbladder wall, the presence of ascites, and the liver and spleen size. The diagnosis of VOD was based on clinical and laboratory data. Patients were divided into 2 groups: those with VOD (n=18) and those without VOD (n=18). The variance of 2 groups was analyzed. V-max of the hepatic artery had a strong correlation with clinical VOD diagnosis (P<0.001). There was no statistically significant difference in the other Doppler parameters. The results of our study showed that the measurement of V-max of the hepatic artery can provide important support in the diagnosis of VOD and can be useful in the follow-up of treatment response.