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Öğe Comparison of tacrolimus vs. cyclosporine in pediatric hematopoietic stem cell transplantation for thalassemia(Wiley, 2024) Zhumatayev, Suleimen; Yalcin, Koray; Celen, Safiye Suna; Karaman, Irem; Daloglu, Hayriye; Ozturkmen, Seda; Uygun, VedatObjectives: Graft-versus-host disease (GvHD) is one of the leading causes of morbidity and mortality in patients undergoing allogeneic HSCT, and effective prevention of GvHD is critical for the success of the HSCT procedure. Calcineurin inhibitors (CNI) have been used for decades as the backbone of GvHD prophylaxis. In this study, the efficacy and safety of Cyclosporine A (CsA) and tacrolimus (TCR) were compared in pediatric HSCT for thalassemia. Materials and Methods: This is a retrospective analysis of 129 pediatric patients who underwent HSCT with the diagnosis of thalassemia at Medicalpark Goztepe and Antalya Hospitals between January 2017 and December 2020. Results: Despite the GvHD prophylaxis, grade II-IV acute GvHD developed in 29 patients. Of these patients, 12 had only gut, 10 had only skin, 6 had combined gut and skin, and one had only liver GvHD. Fifteen of these 29 patients were in the CsA group, and 14 of them were in the TCR group. There was no significant difference between the groups in terms of acute GvHD occurrence, GvHD stage, or involvement sites. In terms of CNI-related toxicity, neurotoxicity in 15 (CsA n = 9, TCR n = 6) and nephrotoxicity in 18 (CsA n = 4, TCR n = 14) patients were observed. While there was no difference between the two groups in terms of neurotoxicity, more nephrotoxicity developed in patients using TCR (p = .013). There was no significant difference between the groups in terms of engraftment syndrome, veno-occlusive disease, CMV reactivation, PRES, or graft rejection. Conclusion: Regarding GvHD, there was no difference in efficacy between TCR and CsA usage. Patients taking TCR experienced noticeably higher nephrotoxicity in terms of adverse effects. This difference should be considered according to the patient's clinical situation while choosing a CNI.Öğe Different kinetics and risk factors for isolated 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; Bozkurt, CeyhunRelapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the most frequent cause of posttransplant mortality. Isolated extramedullary 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 to systemic relapse. The 5-year cumulative incidence of systemic relapse ?either bone morrow (BM) only or combined with EMR? and iEMR was 24.8% and 5.5%, respectively. 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: 0.013). CR2+/active disease at transplantation (HR, 3.1, p<0001) and prior extramedullary disease (HR, 2.3; p: 0.007) were independent risk factors for iEMR. Chronic graft-versus-host disease (GVHD) reduced the risk of systemic relapse (HR, 0.5; p: 0.043) 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% vs 15.3%, p: 0.089). Patients experiencing their first systemic relapse continued to have further systemic relapse, but only a minority progressed to iEMR, while those experiencing their iEMR at first relapse developed further systemic and isolated extramedullary relapses with approximately similar frequencies. Second iEMR was more common after the first iEMR than after the first systemic relapse (58.8% vs 13.0%, p: 0.001) and was associated with a poor outcome. iEMR has a poor prognosis, particularly after the 2nd relapse, and effective strategies are needed to improve outcomes.Öğe Epstein?Barr virus?related lymphoproliferative disorders in T?cell repleted haploidentical transplantation with post?transplant cyclophosphamide(Springer Link, 2021) Uygun, Vedat; Özsan, Nazan; Daloğlu, Hayriye; Öztürkmen, Seda; Yalçın, Koray; Karasu, Gülsün; Yeşilipek, AkifEBV-associated lymphoproliferative disorders (LPDs) are common in hematopoietic stem cell transplantation (HSCT) with T-cell-depleted grafts, but are extremely rare in HSCT patients with T-cell-replete grafts with post-transplant cyclophosphamide (PTCy). Here we present the cases of two pediatric patients who developed EBV-related LPD after T-cell-replete haplo-HSCT with PTCy. One of these is the first reported case of EBV-positive mucocutaneous ulcer (EBVMCU) developing after PTCy. EBV-related diseases are rare in T-cell-replete haplo-HSCT patients with PTCy. However, in patients with risk factors, it is reasonable to screen for EBV viremia for LPD.Öğe EXPERIENCES OF PARENTS WHO CONCEIVED SAVIOR SIBLINGS FOR BEING HEMATOPOIETIC STEM CELL DONORS TO THEIR SICK CHILDREN AND SUCCESSFULLY COMPLETED THE PROCESS: A QUALITATIVE STUDY(Springernature, 2023) Eker, Ibrahim; Ozdemir, Hamide Nur Cevik; Yilmaz, Firat; Yesilipek, Akif; Kupesiz, Alphan; Uygun, Vedat; Karasu, Gulsun[Abstract Not Available]Öğe Hematopoetic stem cell transplantation in CD40 ligand deficiency(SPRINGERNATURE, 2020) Uygun, Vedat; Uygun, Dilara Fatma Kocacık; Daloğlu, Hayriye; Öztürkmen, Seda; Kılıç, Suar Çakı; Bingöl, Aysen; Yalçın, Koray; Çelen, Safiye Suna; Hazar, Volkan; Tezcan Karasu, Gülsun; Yeşilipek, Akif[No Abstract Available]Öğe Hematopoietic stem cell transplantation in CD40 ligand deficiency: a single-center experience(Wiley, 2020) Uygun, Dilara Fatma Kocacık; Uygun, Vedat; Tezcan Karasu, Gülsun; Daloğlu, Hayriye; Öztürkmen, Seda Irmak; Çelmeli, Fatih; Torun, Selda Hançerli; Özen, Ahmet; Barış, Safa; Aydıner, Elif Karakoç; Yalçın, Koray; Kılıç, Suar Çaki; Hazar, Volkan; Bingöl, Aysen; Yeşilipek, AkifDeficiency of the CD40L, expressed on the surface of T lymphocytes, is caused by mutations in the glycoproteinCD40L (CD154)gene. Resulting defective humoral and cellular responses cause a clinical presentation that includes recurrent sinopulmonary bacterial infections, opportunistic infections, sclerosing cholangitis, neutropenia, and autoimmune manifestations. HSCT represents the only curative treatment modality. However, the therapeutic decision to use HSCT proves challenging in many cases, mainly due to the lack of a phenotype-genotype correlation. We retrospectively reviewed patients with CD40L deficiency who were transplanted in Antalya and Goztepe MedicalPark Pediatric HSCT units from 2014 to 2019 and followed by Akdeniz University School of Medicine Department of Pediatric Immunology. The records of eight male cases, including one set of twins, were evaluated retrospectively. As two transplants each were performed on the twins, a total of ten transplants were evaluated. Conditioning regimens were predominantly based on myeloablative protocols, except for the twins, who received a non-myeloablative regimen for their first transplantation. Median neutrophil and platelet engraftment days were 13 (range 10-19) and 14 (range 10-42) days, respectively. In seven of ten transplants, a CMV reactivation was developed without morbidity. None of the patients developed GVHD, except for one mild case of acute GVHD. All patients survived, and the median follow-up was 852 days. Our data show that HSCT for patients with CD40 ligand deficiency is a potentially effective treatment for long-term disease control.Öğe Hematopoietic stem cell transplantation in serine/threonine kinase 4 (stk4) deficiency: report of two cases and literature review(WILEY, 2022) Uygun, Vedat; Keleş, Sevgi; Daloğlu, Hayriye; Öztürkmen, Seda; Yalçın, Koray; Tezcan Karasu, Gülsün; Yeşilipek, AkifBackground Serine/threonine kinase 4 (STK4) deficiency is a combined immunodeficiency (CID) characterized by early onset recurrent bacterial, viral, and fungal infections. Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative therapy for CID; however, little is known about the necessity and benefits of HSCT in patients with STK4 deficiency. Methods We report two siblings with STK4 deficiency transplanted from two unrelated donors with the same conditioning regimen. Results In the conditioning regimen, rituximab was given on Day -11 (375 mg/m(2)), and sirolimus was added on the same day. Busulfan was administered at a myeloablative dose (3.2 mg/kg; Days -7 to -4) with 150 mg/m(2) of fludarabine (Days -7 to -3). They were transplanted with peripheral blood stem cells, and graft-versus-host disease (GVHD) prophylaxis was administered with 10 mg/m(2) methotrexate on Days 1, 3, and 6. In addition, mycophenolate mofetil (MMF) was started on Day 1 with ongoing use of sirolimus. We did not encounter veno-occlusive disease (VOD), high-grade acute GVHD, or significant organ toxicity in either patient. Both patients were well at the end of the first year after HSCT with complete donor chimerism. Conclusions Serine/threonine kinase 4 deficiency is a disease with high mortality post-HSCT; therefore, the conditioning regimen and GVHD prophylaxis strategies are important considerations in these patients. In our opinion, the conditioning regimen, which includes rituximab and busulfan and fludarabine (BU-FLU), GVHD prophylaxis with sirolimus and MMF, and short-term methotrexate, offers favorable outcomes and is well tolerated in our STK4-deficient patients.Öğe ILC3 deficiency and generalized ILC abnormalities in DOCK8-deficient patients(Wiley, 2020) Eken, Ahmet; Cansever, Murat; Okuş, Fatma Zehra; Erdem, Şerife; Nain, Ercan; Azizoğlu, Zehra Büşra; Haliloğlu, Yeşim; Karakukcu, Musa; Özcan, Alper; Devecioğlu, Ömer; Aksu, Güzide; Arıkan Ayyıldız, Zeynep; Topal, Erdem; Karakoç Aydıner, Elif; Kıykım, Ayça; Metin, Ayşe; Erol Çipe, Funda; Kaya, Ayşenur; Artaç, Hasibe; Reisli, İsmail; Güner, Şükrü N.; Uygun, Vedat; Tezcan Karasu, Gülsün; Dönmez Altuntaş, Hamiyet; Canatan, Halit; Oukka, Mohamed; Özen, Ahmet; Chatila, Talal A.; Keleş, Sevgi; Barış, Safa; Ünal, Ekrem; Patıroğlu, TürkanBackgroundDedicator of cytokinesis 8 (DOCK8) deficiency is the main cause of the autosomal recessive hyper-IgE syndrome (HIES). We previously reported the selective loss of group 3 innate lymphoid cell (ILC) number and function in a Dock8-deficient mouse model. In this study, we sought to test whether DOCK8 is required for the function and maintenance of ILC subsets in humans. MethodsPeripheral blood ILC1-3 subsets of 16 DOCK8-deficient patients recruited at the pretransplant stage, and seven patients with autosomal dominant (AD) HIES due to STAT3 mutations, were compared with those of healthy controls or post-transplant DOCK8-deficient patients (n = 12) by flow cytometry and real-time qPCR. Sorted total ILCs from DOCK8- or STAT3-mutant patients and healthy controls were assayed for survival, apoptosis, proliferation, and activation by IL-7, IL-23, and IL-12 by cell culture, flow cytometry, and phospho-flow assays. ResultsDOCK8-deficient but not STAT3-mutant patients exhibited a profound depletion of ILC3s, and to a lesser extent ILC2s, in their peripheral blood. DOCK8-deficient ILC1-3 subsets had defective proliferation, expressed lower levels of IL-7R, responded less to IL-7, IL-12, or IL-23 cytokines, and were more prone to apoptosis compared with those of healthy controls. ConclusionDOCK8 regulates human ILC3 expansion and survival, and more globally ILC cytokine signaling and proliferation. DOCK8 deficiency leads to loss of ILC3 from peripheral blood. ILC3 deficiency may contribute to the susceptibility of DOCK8-deficient patients to infections.Öğe Impact of Replacing Low Dose Cyclophosphamide with Fludarabine in Children with Acute Myeloid Leukemia Undergoing Transplantation During First Complete Remission(Akad Doktorlar Yayinevi, 2023) Uygun, Vedat; Karasu, Gulsun; Yalcin, Koray; Ozturkmen, Seda; Daloglu, Hayriye; Celen, Safiye Suna; Hazar, VolkanBusulfan (BU)-based myeloablative conditioning is a standard conditioning regimen for children with AML; however, it is not clear yet which combination of cyclophosphamide (CY) and fludarabine (FLU) is most effective. We performed a study to compare the results of BUCY120 and BU-FLU in pediatric patients with AML in CR1 undergoing allo-HSCT from matched sibling donors. With the combination of BU, 15 patients were given 120 mg/kg of CY, and 12 patients were given 150 mg/m2 of FLU, respectively, in the condition regimen. Patients treated with BUFLU relapsed less than those treated with BUCY120 (p= 0.03). Moreover, these patients engrafted platelets earlier than the BUCY120 administered patients (p= 0.03). The frequency of complications in both groups was comparable. There was no significant difference in survival analysis between the groups. BUFLU has a low toxicity profile, making it a reasonable choice for children with AML in CR1 with low risk and a lower relapse frequency compared to BUCY120.Öğe Mutational landscape of severe combined immunodeficiency patients from Turkey(Wiley, 2020) Fırtına, Sinem; Ng, Yuk Yin; Ng, Özden Hatırnaz; Kıykım, Ayça; Aydıner, Elif; Nepesov, Serdar; Camcıoğlu, Yıldız; Sayar, Esra H.; Reisli, İsmail; Torun, Selda H.; Çöğürlü, Tuba; Uygun, Dilara; Şimşek, Işıl E.; Kaya, Ayşenur; Erol Cipe, Funda; Çağdaş, Deniz; Yücel, Esra; Çekiç, Şükrü; Uygun, Vedat; Barış, Safa; Özen, Ahmet; Özbek, Ugur; Sayitoglu, MugeSevere combined immunodeficiency (SCID) has a diverse genetic aetiology, where a clinical phenotype, caused by single and/or multiple gene variants, can give rise to multiple presentations. The advent of next-generation sequencing (NGS) has recently enabled rapid identification of the molecular aetiology of SCID, which is crucial for prognosis and treatment strategies. We sought to identify the genetic aetiology of various phenotypes of SCIDs and assessed both clinical and immunologic characteristics associated with gene variants. An amplicon-based targeted NGS panel, which contained 18 most common SCID-related genes, was contumely made to screen the patients (n = 38) with typical SCID, atypical SCID or OMENN syndrome. Allelic segregations were confirmed for the detected gene variants within the families. In total, 24 disease-causing variants (17 known and 7 novel) were identified in 23 patients in 9 different SCID genes: RAG1 (n = 5), RAG2 (n = 2), ADA (n = 3), DCLRE1C (n = 2), NHEJ1 (n = 2), CD3E (n = 2), IL2RG (n = 3), JAK3 (n = 4) and IL7R (n = 1). The overall success rate of our custom-made NGS panel was 60% (39.3% for NK+ SCID and 100% for NK- SCID). Incidence of autosomal-recessive inherited genes is more frequently found in our cohort than the previously reported populations probably due to the high consanguineous marriages in Turkey. In conclusion, the custom-made sequencing panel was able to identify and confirm the previously known and novel disease-causing variants with high accuracy.Öğe Newborn screening for sickle cell anemia in Antalya, Türkiye(Turkish J Pediatrics, 2023) Ozturk, Zeynep; Kupesiz, O. Alphan; Karasu, Gulsun; Uygun, Vedat; Oygur, Nihal; Yesilipek, M. AkifBackground. In a screening study conducted on adults, the prevalence of sickle cell traits in Antalya was found to be 0.24%. Since no screening studies have been conducted in the neonatal period in our region, the exact incidence has not been determined. In this study, we aim to report our experience of neonatal screening for sickle cell disease in Antalya, Turkiye.Methods. During a 14-month period, 2562 heel prick blood samples, taken on filter paper from Akdeniz University Hospital, Antalya Education and Research Hospital and Antalya Ataturk State Hospital and four other healthcare centers, were studied using the high pressure liquid chromatography method. Blood samples were studied using the 'Sickle Cell Short Program' test method on a Bio Rad Variant device.Results. In the study, no patients with sickle cell disease were identified. Four newborns who were sickle cell carriers (0.15%) and two newborns who were Hemoglobin D carriers (0.08 %), were found. Conclusion.Considering the efficiency and cost calculations made as a result of the data obtained from our study, it was concluded that sickle cell screening would not be effective in newborns. It seems more effective and economical to screen the children of parents, who are found to be at risk for Hemoglobin S carriage as a result of premarital tests.Öğe Novel immunodeficiency caused by homozygous mutation in solute carrier family 19 member 1, which encodes the reduced folate carrier(Amer Soc Hematology, 2023) Shiraishi, Akira; Uygun, Vedat; Sharfe, Nigel; Beldar, Serap; Sun, Mark G. F.; Dadi, Harjit; Vong, Linda[Abstract Not Available]Öğe Pearson syndrome in a child transplanted for diamond-blackfan anemia(Sociedad Argentina de Pediatria, 2021) Uygun, Vedat; Daloğlu, Hayriye; Öztürkmen, Seda; Karasu, Gülsün; Yeşilipek, AkifPearson syndrome (PS), shares a number of overlapping features with Diamond-Blackfan anemia (DBA), including early onset of severe anemia, making differential diagnosis important. Differential diagnosis of DBA and PS is critical, since those with DBA may respond to treatment with steroids, may undergo remission, or may benefit from hematopoietic stem cell transplantation (HSCT). However, patients with PS have a different prognosis, with a very high risk of developing acidosis, metabolic problems, and pancreatic dysfunction, and a shorter life expectancy than those with DBA. Here we present a patient who underwent HSCT for DBA but was subsequently diagnosed with PS after developing some complications.Öğe Pearson syndrome in a patient transplanted for diamond-blackfan anemia(SPRINGERNATURE, 2020) Tezcan Karasu, Gülsün; Uygun, Vedat; Daloğlu, Hayriye; Öztürkmen, Seda; Yeşilipek, Akif[No Abstract Available]Öğe PRE-TRANSPLANTATION VITAMIN D DEFICIENCY INCREASES ACUTE GRAFT-VERSUS-HOST DISEASE AFTER HEMATOPOIETIC STEM CELL TRANSPLANTATION IN THALASSEMIA MAJOR PATIENTS(Springernature, 2023) Uygun, Vedat; Daloglu, Hayriye; Ozturkmen, Seda; Yalcin, Koray; Karasu, Gulsun; Yesilipek, Akif[Abstract Not Available]Öğe Pre-transplantation vitamin D deficiency increases acute graft-versus-host disease after hematopoietic stem cell transplantation in thalassemia major patients(WILEY, 2022) Daloğlu, Hayriye; Uygun, Vedat; Öztürkmen, Seda; Yalçın, Koray; Karasu, Gülsü; Yeşilipek, AkifBackground: Although there are many studies on the role of vitamin D deficiency (VDD) in hematopoetic stem cell transplantation (HSCT), outcomes have often reported conflicting results because of the heterogeneity of the patients in the studies. Methods: We investigated the association between VDD prior to HSCT and outcomes after HSCT in a relatively homogenous group of patients with thalassemia major (TM) who received identical treatment for TM before transplantation, and the same conditioning regimen and GVHD prophylaxis during and after transplantation. All patients, including the patients with normal vitamin D3 levels received 400 to 800 IU per day of vitamin D for the first 6 months after HSCT. Results: Pre-HSCT VDD increased the frequency of aGVHD after transplantation, particularly in HSCTs performed with PBSC for the stem cell source. Pre-transplant low vitamin D3 levels had no association with transplant outcomes such as engraftment, viral infections, alloimmunization, chronic GvHD, total days of hospitalization, and success in terms of transfusion independence. Conclusions: Low vitamin D3 levels before HSCT carry a significant risk for aGVHD. All patients with TM should be screened for VDD before HSCT, and every effort should be made to supplement vitamin D before the transplant in VDD patientsÖğe Prognostic factors for survival in children who relapsed after allogeneic hematopoietic stem cell transplantation for acute leukemia(2020) Hazar, Volkan; Tezcan Karasu, Gülsün; Öztürk, Gülyüz; Küpesiz, Alphan; Aksoylar, Serap; Özbek, Namık; Uygun, Vedat; İleri, Talia; Okur, Fatma Visal; Koçak, Ülker; Çakı Kılıç, Suar; Akçay, Arzu; Güler, Elif; Kansoy, Savaş; Karakükcü, Musa; Bayram, İbrahim; Aksu , Tekin; Yeşilipek, Akif; Karagün, Barbaros Şahin; Yılmaz, Şebnem; Ertem, Mehmet; Uçkan, Duygu; Fışgın, Tunç; Gürsel, Orhan; Yaman, Yöntem; Bozkurt, Ceyhun; Gökçe, MügeBackground: Post-transplant relapse has a dismal prognosis in children with acute leukemia undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Data on risk factors, treatment options, and outcomes are limited. Procedure: In this retrospective multicenter study in which a questionnaire was sent to all pediatric transplant centers reporting relapse after allo-HSCT for a cohort of 938 children with acute leukemia, we analyzed 255 children with relapse of acute leukemia after their first allo-HSCT. Results: The median interval from transplantation to relapse was 180 days, and the median follow-up from relapse to the last follow-up was 1844 days. The 3-year overall survival (OS) rate was 12.0%. The main cause of death was disease progression or subsequent relapse (82.6%). The majority of children received salvage treatment with curative intent without a second HSCT (67.8%), 22.0% of children underwent a second allo-HSCT, and 10.2% received palliative therapy. Isolated extramedullary relapse (hazard ratio (HR): 0.607, P = .011) and relapse earlier than 365 days post-transplantation (HR: 2.101, P < .001 for 0-180 days; HR: 1.522, P = .041 for 181-365 days) were found in multivariate analysis to be significant prognostic factors for outcome. The type of salvage therapy in chemosensitive relapse was identified as a significant prognostic factor for OS. Conclusion: A salvage approach with curative intent may be considered for patients with post-transplant relapse, even if they relapse in the first year post-transplantation. For sustainable remission, a second allo-HSCT may be recommended for patients who achieve complete remission after reinduction treatment.Öğe Ruxolitinib salvage therapy is effective for steroid-refractory graft-versus-host disease in children: a single-center experience(Wiley, 2020) Uygun, Vedat; Tezcan Karasu, Gülsün; Daloğlu, Hayriye; Öztürkmen, Seda; Kılıç, Suar Çakı; Yalçın, Koray; Çelen, Safiye Suna; Hazar, Volkan; Yeşilipek, AkifBackground Despite the increasing performance of allogeneic hematopoietic cell transplantation over the last decades, graft-versus-host disease (GVHD) remains the main cause of morbidity and mortality. The efficacy of ruxolitinib against GVHD has been demonstrated in adult studies; however, very few studies have been conducted in children. Procedure This study aimed to evaluate the efficacy of ruxolitinib in 29 children with steroid-refractory acute or chronic GVHD. Twenty-five (87%) patients received at least three different immune modulator agents, including methylprednisolone, before initiating ruxolitinib. Results All grade 2 acute GVHD patients completely responded to ruxolitinib treatment; 82% of high-grade (3-4) acute GVHD patients and 80% of chronic GVHD (moderate-severe) patients had at least a partial response. Of seven patients with bronchiolitis obliterans, five had a partial response after ruxolitinib. Of 29 patients, 22 were administered steroids at any time in the first month of acute GVHD or the first three months of chronic GVHD during ruxolitinib usage, which was significantly tapered by the end of the observation period. Conclusion Steroid-refractory acute and chronic pediatric GVHD patients treated with ruxolitinib had a high overall response rate, with the additional benefit of steroid sparing.Öğe Talasemi Majör Hastalarında Hematopoetik Kök Hücre Nakli Uzun Dönem Sağkalım Sonuçları(2023) Daloğlu, Hayriye; Uygun, Vedat; Öztürkmen, Seda; Yalçın, Koray; Çelen, Suna; Karasu, Gülsün; Yeşilipek, AkifAmaç: Günümüzde talasemi majör (TM) hastalarında en önemli sorun transfüzyonların neden olduğu demir yükü ve bunun doğurduğu komplikasyonlardır. Bu durumun günümüzde kabul edilen tek küratif tedavi seçeneği hematopoetik kök hücre nakli (HKHN)’dir. Çalışmamızda, TM nedeniyle akraba ve akraba dışı donörden merkezimizde HKHN uygulanmış hastalarımızın uzun dönem sağkalım sonuçları ve kronik graft versus host hastalığı (kGVHH) değerlendirilmiştir. Hastalar ve Yöntem: TM tanısı ile HKHN uygulanmış 378 hastanın klinik verileri retrospektif olarak incelendi. Tüm hastalara busulfan içeren myeloablatif hazırlama rejimi ve ATG uygulandı. Nakil öncesi ve sonrası nakil ilişkili veriler değerlendirildi ve tüm sağkalım (OS), talasemisiz sağkalım (TS), talasemi ve kGVHH´siz sağkalım (TGS) belirlendi. Bulgular: Ortanca 79 ay izlenen hastaların ikisinde primer greft rejeksiyonu, 22´sinde sekonder greft rejeksiyonu gelişti. Son kontrol tarihlerine göre, hastaların %92.9´unun ya tam kimerik ya da transfüzyon ihtiyacı olmadan stabil miksed kimerizm ile izlendiği saptandı. HKHN sonrası 15 hasta ölürken, sağ kalan 363 hastanın 25’inde kGVHH bulguları gelişti ancak 17´si izlemde düzeldi. Yedi yıllık OS, TS ve TGS sırası ile %95.9, %89.5 ve %87.1 olarak saptandı. Sonuç: Talasemi majör hastalarında, tam uyumlu donör varlığında nakil sonrası yaşam kalitesi de göz önünde bulundurularak organ hasarı gelişmeden, mümkün olan en erken dönemde HKHN uygulanmalıdır.Öğe Thalassemia-free and graft-versus-host-free survival: outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience(2022) Yeşilipek, M. Akif; Uygun, Vedat; Küpesiz, Alphan; Tezcan Karasu, Gülsün; Öztürk, GülyüzWe 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.
