7 results on '"O. Alphan Kupesiz"'
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2. Current Use of Androgens in Bone Marrow Failure Disorders: A Report from the Severe Aplastic Anemia Working Party (SAAWP) of the European Society of Blood and Marrow Transplantation (EBMT)
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Simona Pagliuca, Austin Kulasekararaj, Dirk-Jan Eikema, Brian Piepenbroek, Raheel Iftikhar, Tariq Mahmood Satti, Morag Griffin, Marica Laurino, O. Alphan Kupesiz, Yives Bertrand, Bruno Fattizzo, Ibrahim Yakoub-Agha, Mahmoud Aljurf, Paola Corti, Erika Massaccesi, Bruno Lioure, Marisa Calabuig, Matthias Klammer, Emel Unal, Depei Wu, Patrice Chevallier, Edouard Forcade, John A. Snowden, Hakan Ozdogu, Antonio Risitano, and Régis Peffault de Latour
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Immunology ,Cell Biology ,Hematology ,Biochemistry - Published
- 2022
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3. Busulfan/Fludarabine- or Treosulfan/Fludarabine-Based Conditioning Regimen in Patients with Wiskott-Aldrich Syndrome Given Allogeneic Hematopoietic Cell Transplantation — an EBMT Inborn Errors Working Party and Scetide Retrospective Analysis
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Junfeng Wang, Christoph Klein, Karl-Walter Sykora, Marco Zecca, Tatjana A Bykova, Krzysztof Kałwak, Nizar Mahlaoui, Paul Veys, Maria Ester Bernardo, Ekrem Unal, Mary Slatter, Michael H. Albert, Ivana Bodova, Andrew R. Gennery, Despina Moshous, Fulvio Porta, Henric-Jan Blok, Ansgar Schulz, Alain Fischer, Robert Chiesa, Benedicte Neven, Svetlana Kozlovskaya, Jacek Winiarski, Virginie Courteille, Tayfun Guengoer, Renata Formankova, O. Alphan Kupesiz, Bénédicte Bruno, Arjan C. Lankester, and Franco Locatelli
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Oncology ,medicine.medical_specialty ,Cyclophosphamide ,Wiskott–Aldrich syndrome ,business.industry ,medicine.medical_treatment ,Immunology ,Cell Biology ,Hematology ,Hematopoietic stem cell transplantation ,ThioTEPA ,Treosulfan ,medicine.disease ,Biochemistry ,Fludarabine ,Transplantation ,03 medical and health sciences ,0302 clinical medicine ,030220 oncology & carcinogenesis ,Internal medicine ,medicine ,business ,Busulfan ,030215 immunology ,medicine.drug - Abstract
PV and AL contributed equally Multiple studies from the EBMT registry and others have shown excellent survival rates after allogeneic haematopoietic stem cell transplantation (HSCT)for Wiskott-Aldrich syndrome (WAS) patients (Ozsahin et al, Blood 2008). The importance of myeloid engraftment for full disease correction has also been demonstrated (Moratto et al, Blood 2011). However, the vast majority of HSCTs in these studies were performed with (oral) busulfan/cyclophosphamide-based conditioning and in the early 2000 years or before. In 2005, the inborn errors working party (IEWP) of EBMT and ESID first recommended busulfan/fludarabine (BuFlu) or treosulfan/fludarabine (TreoFlu) based conditioning for primary immunodeficiencies such as WAS, with some centers deciding to add thiotepa (TT) to the conditioning. We performed a retrospective analysis via the EBMT and SCETIDE registries of WAS patients transplanted between 01/01/20006 and 12/31/2016 with these two regimens. The primary objective was to compare the overall (OS) and event-free survival (EFS) after HSCT with either BuFlu±TT or TreoFlu±TT conditioning. Secondary objectives included the influence of either conditioning regimen on acute and chronic GVHD, the degree of donor chimerism, incidence of secondary procedures after HSCT (2nd HSCT, stem cell boost, DLI, gene therapy or splenectomy) and rates of disease-specific complications after HSCT. At the time of this interim analysis, 174 patients were included, 92 (53%) with BuFlu±TT and 82 (47%) with TreoFlu±TT conditioning with a median age of 1.57 years (range 0.21-29.96) at HSCT and a median follow-up of 32.9 months (range 1.5-128.9). The donor was an HLA-matched sibling (MSD) in 30, a matched related donor (MRD) in 5, a matched unrelated donor (MUD, 9/10 or 10/10) in 105, a mismatched unrelated donor (MMUD, 5 years had a worse OS as compared to those 5 years or younger at HSCT (74.9% vs. 90.8%; log-rank test p=0.005). The type of donor had no influence on OS: 96.4% for MSD/MFD, 86.8% for MUD/MMUD and 87.7% for MMFD (log-rank test p=0.4). Whole blood chimerism was complete (>90% donor) in 60/75 evaluable patients (80%) at last follow-up or before secondary procedure (if a patient had one), 39/40 (98%) in the BuFlu±TT group and 21/35 (60%) in the TreoFlu±TT group. Twenty-six patients required a secondary procedure: stem cell boost in 4 patients, donor lymphocyte infusion in 9, 2nd HSCT in 15 and splenectomy in 1. Twenty-two of these 26 (84.6%) are alive and 14 of 16 with available chimerism data have a complete donor chimerism (>90%donor) at last follow-up. The 2-year cumulative incidence (CI) of secondary procedures was higher at 33.9% in the TreoFlu±TT versus 12.8% in the BuFlu±TT group (Gray's test p=0.017), and 2-year EFS (secondary procedure or death as event) was 61.4% in the TreoFlu±TT and 75.0% in the BuFlu±TT group (log-rank test p=0.2). Grade II-IV acute GVHD had the same incidence in both groups (100 day CI 24.4% vs. 26.3%; Gray's test p=0.849) and chronic GVHD of any grade was borderline more frequent in the TreoFlu±TT group (2 year CI 17.2% vs 6.7%; Gray's test p=0.054). The cumulative incidence of disease-specific complications occurring more than 6 months post HSCT (severe infections, bleeding, autoimmunity) was not different between the two groups (6.5% vs. 6.4%; Gray's test p=0.92). There was no malignancy reported after HSCT except for one EBV-post-transplant lymphoproliferative disorder (PTLD) 2.7 months after HSCT. In summary, HSCT with either BuFlu±TT or TreoFlu±TT conditioning reliably cures almost 90% of patients with WAS regardless of donor type. WAS-related complications are very rare events more than 6 months post HSCT. More patients required secondary procedures after treosulfan-based than busulfan-based conditioning. These data confirm the feasibility and efficacy of the regimens currently recommended by the IEWP. Disclosures Slatter: Medac: Other: Travel assistance. Chiesa:Gilead: Consultancy; Bluebird Bio: Consultancy. Kalwak:Sanofi: Other: travel grants; medac: Other: travel grants. Locatelli:Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees; bluebird bio: Consultancy; Bellicum: Consultancy, Membership on an entity's Board of Directors or advisory committees; Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees; Miltenyi: Honoraria. Sykora:Aventis-Behring: Research Funding; medac: Research Funding. Zecca:Chimerix: Honoraria. Veys:Pfizer: Honoraria; Servier: Research Funding; Novartis: Honoraria.
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- 2018
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4. Hematopoietic Cell Transplantation in Thalassemia and Sickle Cell Disease: Report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry: 2000-2017
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Donatella Baronciani, Ardeshir Ghavamzadeh, Gérard Socié, Peter Bader, Gian Luca Forni, O. Alphan Kupesiz, Arjan C. Lankester, Abdelghani Tbakhi, Josu de la Fuente, Marco Zecca, Amal Al-Seraihy, Javid Gaziev, Ariane Boumendil, Emanuele Angelucci, Vassiliki Kitra-Roussou, and Arnaud Dalissier
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Bone marrow transplantation ,Hematopoietic cell ,business.industry ,Thalassemia ,Immunology ,Cell ,Cell Biology ,Hematology ,Disease ,medicine.disease ,Biochemistry ,Transplantation ,03 medical and health sciences ,0302 clinical medicine ,medicine.anatomical_structure ,Hemoglobinopathy ,030220 oncology & carcinogenesis ,medicine ,business ,030215 immunology - Abstract
INTRODUCTION: Allogeneic hematopoietic cell transplantation (HCT) is a diffuse curative option for transfusion dependent thalassemia (TDT) and sickle cell disease (SCD). To verify transplant activity, distribution, demography, policies and outcomes the Hemoglobinopathy Registry was established inside the European Group for Blood and Marrow Transplantation (EBMT). After a previous analysis limited to TDT for the 2000-2010 period data (BMT 2016; 51:536-41), we performed an updated report considering TDT and SCD patients transplanted in the last eighteen years (years 2000-2017). METHODS: Data on pediatric patients transplanted between Jan 1st, 2000 through Dec 31st, 2017 were extracted by the EBMT promise Hemoglobinopathy registry database. Only first transplants were considered. Data are expressed as median with range unless specifically indicated. Survival probabilities were calculated with the method of Kaplan and Meier and expressed as means and 95% confidence intervals (95%CI). Differences between survival probabilities were tested by means of the log-rank test. RESULTS: In the above-specified period 3856 consecutive pediatric patients affected by TDT (2936, 76%) or SCD (920, 24%) were transplanted in 166 HCT centers distributed in 36 countries in Europe, Asia and Africa. Median age at transplant was 7.2 years (range 0.48-17.9). 3342 (87%) transplants were performed on patients Figure 1 reports pediatric transplant activity inside the EBMT showing an increased numbers of patients transplanted after year 2010. After a median follow up of 24 months, the 2 years overall survival (OS) and event-free survival (EFS) were 91% (95%CI 90-92) and 86% (95%CI 85-87) for the entire population. In TDT, OS and EFS were 90% (95%CI 89-92) and 84% (95%CI 82-85), respectively. In SCD, OS and EFS were 94% (95%CI 92-96) and 92% (95%CI 90-94), respectively. In both diseases no outcome difference was recorded on the basis of year of transplant (data not shown). Source of hematopoietic cells was bone marrow in 70% of TDT transplants and 81% of SCD transplants. In both diseases better results were recorded with the use of bone marrow versus peripheral blood [OS 91% versus 85% (P< 0.001); 95% versus 84% (P= 0.004) for TDT and SCD, respectively]. Similar results were recorded when EFS was analyzed. 176 patients received hematopoietic cells from single cord blood donors. Of them only 21 were from an unrelated donor. 119 patients received cord blood + bone marrow. Results of related HLA identical cord blood were similar to that of HLA identical related bone marrow (data not shown). Transplantation from an HLA identical sibling offered the best results in OS and EFS compared to other donor options (P< 0.001), both for TDT and SCD. Transplant results by donor type are reported in table 1. The threshold age for optimal transplant outcomes is confirmed 14 years, with OS 92% (95%CI 91-93), EFS 87% (95%CI 85-88) versus OS 85% (95%CI 82-89), EFS 81% (95%CI 77-85) ( P The two years incidence of cumulative extensive chronic graft versus host disease was 4.1% in TDT and 4.4 % in SCD (P=ns). CONCLUSIONS: Allogeneic HCT for TDT and SCD is a widely available curative approach; the procedure has been increasing and internationally performed during the years with excellent results. The emerging gene therapy approach will have to be compared to these well-established results. Disclosures Zecca: Chimerix: Honoraria. Forni:Novartis: Other: travel expenses, Research Funding; Celgene: Research Funding; Roche: Consultancy; Shire: Research Funding; Apopharma: Other: DSM Board. Bader:Medac: Patents & Royalties, Research Funding; Neovii: Research Funding; Novartis: Consultancy, Speakers Bureau; Riemser: Research Funding; Cellgene: Consultancy. Angelucci:Vertex Pharmaceuticals Incorporated (MA) and CRISPR CAS9 Therapeutics AG (CH): Other: Chair DMC; Novartis: Honoraria, Other: Chair Steering Comiittee TELESTO Protocol; Roche Italy: Other: Local (national) advisory board; Jazz Pharmaceuticals Italy: Other: Local ( national) advisory board; Celgene: Honoraria, Other: Chair DMC.
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- 2018
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5. Fibrinolytic parameters in children with noncatheter thrombosis: a pilot study
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Ozgun Tosun, Jeanne M. Lusher, Meera Chitlur, Ronald Thomas, Madhvi Rajpurkar, Wendy Hollon, Indira Warrier, and O. Alphan Kupesiz
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Adult ,Male ,medicine.medical_specialty ,Adolescent ,medicine.medical_treatment ,Gastroenterology ,Tissue plasminogen activator ,Fibrin ,Pathogenesis ,Cohort Studies ,Tissue factor ,Young Adult ,Sex Factors ,Internal medicine ,Fibrinolysis ,medicine ,Humans ,Child ,Venous Thrombosis ,medicine.diagnostic_test ,biology ,business.industry ,Racial Groups ,Age Factors ,Hematology ,General Medicine ,medicine.disease ,Thrombosis ,Thromboelastography ,Thrombelastography ,Venous thrombosis ,Child, Preschool ,Tissue Plasminogen Activator ,biology.protein ,Female ,business ,medicine.drug - Abstract
Although the incidence of pediatric thrombosis has increased over the last decade, noncatheter-related deep venous thrombosis (nCDVT) is rare in children. Congenital and acquired hypercoagulable states may play an important role in the pathogenesis of nCDVT. In this study, we evaluated fibrinolytic parameters by measuring individual concentrations of fibrinolytic proteins and by tissue factor initiated whole blood thromboelastography (TEG), in which a fibrin clot was lyzed by exogenously added tissue plasminogen activator (tPA). Children with nCDVT were compared with age and sex-matched controls. TAFI concentrations were significantly higher in the patient group but there was no difference in the PAI-1, tPA and lipoprotein (a) concentrations. Significantly decreased fibrinolysis was found on TEG in the patient group suggesting that hypofibrinolysis may play an important role in the pathogenesis of nCDVT in children. To our knowledge, this is the first pediatric study that has systematically evaluated the role of fibrinolysis in the pathogenesis of DVT. Given our results, the role of fibrinolysis in the pathogenesis of nCDVT in children should be further evaluated in larger studies.
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- 2010
6. Castleman’s Disease: Four Extraordinary Presantations and Review of Literature
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O. Alphan Kupesiz, Gokben Yildirim Kupesiz, Bahar Akkaya, and Gulten Karpuzoglu
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medicine.medical_specialty ,Pathology ,business.industry ,Immunology ,Hepatosplenomegaly ,Hypergammaglobulinemia ,Lymphoproliferative disorders ,Mediastinum ,Cell Biology ,Hematology ,Disease ,medicine.disease ,Biochemistry ,Dermatology ,medicine.anatomical_structure ,Localized disease ,medicine ,Hypoalbuminemia ,medicine.symptom ,business ,POEMS syndrome - Abstract
Castleman’s disease is a rare disorder of the lymphoid tissue with three possible histological variants--the hyalin-vascular type, with a good prognosis, the plasma-cellular type and the mixed type; the latter two are both more aggressive than the hyalin-vascular type. Two clinical types of this disease have already been described: the localized or unifocal type and the multicentric or multifocal type. Localized and multicentric Castleman’s disease may be different clinical disorders with overlapping histologic features. Localized disease generally presented with a single enlarged lymph node or widening of the mediastinum, whereas multicentric disease is a systemic lymphoproliferative disorder characterized by lymphadenopathy, hepatosplenomegaly, constitutional symptoms, anemia, hypoalbuminemia, and hypergammaglobulinemia. Unlike the localized type, for which surgical excision is curative regardless of the histologic type, multicentric disease often necessitates aggressive systemic therapy and portends a poorer outcome. The aetiology of Castleman’s disease remains unclear. Here, we report four cases of Castleman’s disease, with extraordinary presentations. Tumors from retroperitoneum, mesenterium, bladder vicinity and nasopharenx locations were included. All of patients were taken to the surgery with different pre operative diagnosis. There were 1 female and 3 males; ages ranged from 16–62 years old; sizes ranged from 4– 9 cm (mean 5.5 cm). All patients presented with localized disease. One of the patients had previous NHL and lung cancer history. None of the patients demonstrated systemic involvement or signs of the POEMS syndrome. Hystopathologically 3 of the cases were classified as hyalin-vascular type and 1 of the cases were classified as plasma-cellular type. None of the patients had recurrences. Castleman ‘s disease is an uncommon cause of lymphadenopathy, and must be considered after all other causes have been eliminated. We also add the overview of the literature of the cases with extraordinary presentations.
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- 2007
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7. Peripheral Blood Stem Cell Transplantation in Fanconi Aplastic Anemia: Report of Ten Cases
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Volkan Hazar, M. Akif Yesilipek, Gulsun Tezcan, and O. Alphan Kupesiz
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medicine.medical_specialty ,Platelet Engraftment ,business.industry ,medicine.medical_treatment ,Immunology ,Cell Biology ,Hematology ,Hematopoietic stem cell transplantation ,Total body irradiation ,medicine.disease ,Biochemistry ,Gastroenterology ,Surgery ,Fludarabine ,Transplantation ,Regimen ,Cyclosporin a ,Internal medicine ,medicine ,Aplastic anemia ,business ,medicine.drug - Abstract
Allogeneic hematopoietic stem cell transplantation (SCT) from healthy donors is the only treatment modality for the correction of hematological abnormalities in Fanconi aplastic anemia (FAA) patients. We have performed SCT by using two different non total body irradiation conditioning regimens. While anti-thymocyte globulin (ATG, 10–30mg/kg/day, 3 days), cyclophosphamide (5mg/kg/day, 4 days) and thoraco-abdominal radiation (total 5Gy) were used for six patients (regimen A), fludarabine (120–150mg/m2 totally), cyclophosphamide (10mg/kg/day, 4 days), ATG (30mg/kg/day, 3 days) were given to four patients (regimen B). Six of the patients received regimen A and 4 regimen B. Donors were HLA-matched sibling in 5, HLA-matched parent in 2, partly HLA-matched parent in 2 and HLA-matched unrelated donor in one. All patients and donors were screened by diepoxybutane (DEB) test. Seven of the patients were DEB positive. All donors were DEB negative. Median age of the patients was 10 years. All patients received antimicrobial, antifungal prophylaxis and intravenous immunoglobulin (IVIG, 500 mg/kg weekly) from day −7 to day +180. Cyclosporin A (CsA) was used for graft-versus-host disease (GVHD) prophylaxis in eight patients and CsA plus mycophenolate mofetil in one matched unrelated patient. Neutrophil and platelet engraftment occurred in all patients on day 10 (median) and day 21 (median), respectively. Grade II-IV acute GVHD occurred in two patients who received regimen A. Conditioning-related toxicity was milder in regimen B than that in regimen A. Three patients succumbed from complications of grade IV acute GVHD, post-transplant acute myeloid leukemia and fungal pulmonary infection in regimen A group. All of the regimen B group were alive with normal hematological parameters. Totally, seven patients are alive with sustained engraftment and transfusion independent with median follow-up 19 months (range 2–50). We conclude that fludarabine based conditioning regimen is well tolerated and ideally suited to reduce regimen-related toxicities while achieving sustained engraftment in FAA patients SCT using sibling, related and unrelated donor.
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- 2005
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