Hematology, Transfusion and Cell Therapy最新文献

{"title":"Mesenchymal stem cell supported hematopoietic stem cell transplantation from a mismatched unrelated donor to children with Fanconi anaemia: A successful technique","authors":"Zeynep Canan Özdemir","doi":"10.1016/j.htct.2024.04.037","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.037","url":null,"abstract":"<div><h3>Case report</h3><p>Over the past 20 years, hematopoietic stem cell transplantation (HSCT) outcomes in patients with Fanconi Anaemia (FA) have improved dramatically. It is well established that the addition of mesenchymal stem cells (MSCs) to HSCT regimens in aplastic anaemias has positive effects on transplantation results. Considering these results, we present the transplantation procedure successfully performed on a patient with FA, supported by the MSC infusion from a 9/10 HLA-matched unrelated donor.</p></div><div><h3>Case</h3><p>An 11-year-old girl was admitted with multiple congenital anomalies and pancytopenia. DEB test was positive, compound heterozygous FANCA mutation was detected. A diagnosis of Fanconi anemia (FA) was made. We continued administering oral prednisolone and danazol without transfusion for 3 years. The need for platelet transfusion guided us to schedule HSCT. Given the absence of any matched family donor in her case, a 9/10 HLA matched donor was found from the national stem cell bank. Reduced-intensity conditioning regimen (Fludarabine, 30 mg/m²/day, days -7 to -3; cyclophosphamide; CY, 10 mg/kg/day, days -6 to -3) and serotherapy (ATG, 10 mg/kg/day, days -4 to -2) were performed. Mesenchymal stem cells (MSC) infusion (1 × 10⁶/kg) was administered on days -1 and +7, along with a dose of 6.2 × 10⁶/kg peripheral stem cells. Tacrolimus, methotrexate, and prednisolone (1 mg/kg/day, 28 days) were administered as graft versus host disease (GVHD) prophylaxis. Neutrophil engraftment (2020/mm³) occurred on the 9th day, platelet engraftment (135000/mm³) occurred on the 12th day. She had CMV reactivation in the 3rd month of HSCT. Antiviral treatment for CMV infection was carried out for 3 weeks. On day +100, a steroid was added due to grade II skin acute GVHD (aGVHD). Following its tapering off after 15 days, steroid administration was stopped. The patient achieved complete chimerism, allowing the discontinuation of immunosuppressive treatments in the first year itself.</p></div><div><h3>Discussion</h3><p>MRD and MUD transplants yield the highest success rate in patients with FA. However, the results of HSCT from an alternative donor are still unsatisfactory. MSCs are responsible for immune regulation, tissue repair and regeneration, homing, and support of the hematopoietic system. It has been reported that infusion of MSCs can reduce the development of aGVHD by 3-fold and improve the OS of patients after allogeneic HSCT in comparison to standard prophylaxis. The addition of MSC to the conditioning regimens for MMUD transplants in patients with FA has been proven advantageous due to its graft-supporting, immunosuppressive, and immunomodulatory properties. However, large-scale randomised controlled trials are yet required to back these benefits.</p><p><strong><em>Keywords:</em></strong> <em>Fanconi anemia, HSCT, mesenchymal stem cell</em></p></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2531137924001196/pdfft?md5=f0b7ad71a4d47df38b22423713d5ca85&pid=1-s2.0-S2531137924001196-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140879026","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"ISOLATED CENTRAL NERVOUS SYTEM BURKITT'S LYMPHOMA IN ADVANCED AGE: A CASE STUDY","authors":"ORHAN AYYILDIZ ,&nbsp;SONGUL BESKISIZ ,&nbsp;ABDULLAH KARAKUŞ","doi":"10.1016/j.htct.2024.04.032","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.032","url":null,"abstract":"<div><h3>Objective</h3><p>Burkitt lymphoma is an aggressive type of B-cell lymphoma that is usually seen in the pediatric and young adult population and characteised with myc gene translocation. This disease manifests itself with rapidly growing abdominal mass and systemic sign and symptoms. However, atypical involvements, such as isolated cranial involvements, face both diagnostic and therapeutic challenges, especially in older age groups. Although isolated cranial involvement of Burkitt's lymphoma is rare in older patients, this case report emphasizes the challenges in clinical practice.</p></div><div><h3>Case report</h3><p>A 67-year-old female patient was taken with complaints of headache and vomiting in June 2022. An MRI scan revealed a mass measuring 3.3 × 2.8 × 1.5 cm in the left temporal region. Upon this finding, the patient was referred to the neurosurgery department and the mass was surgically removed. As a result of the pathological examination resulting from the operation, CD10, CD20 were diffusely positive; BCL2 negative; BCL6 positive; C-MYC 70% positive; Kİ67 is 100% positive and confirms Burkitt's Lymphoma. In the PET-CT scan performed for the staging of the patient, reticular dense growths and irregular growth FDG uptakes in ground glass density areas were observed in the medial posterobasal segment of the lower lobe of two lungs and in the anterior segment of the upper lobe of the left lung. In the mediastinal area, increased degrees of FDG uptake were detected in bilateral lower paratracheal and subcarinal lymph nodes. These findings were evaluated as a potential infectious event. While there were no findings in hemogram and biochemical pathological tests, HbsAg positivity was detected but no active disease was found.Prophylactic intrathecal(IT) treatmentwas also recommended for the disease, which started to systemic chemotherapy, but IT chemotherapy was rejected.. In subsequent MRI examinations, the defect formed after craniotomy in the left temporofrontoparietal region and fluid collection in the calvarium were observed, while no residue or recurrence was observed in the operation area.However, a lesion measuring 2 × 3 cm in size was detected in the left parietal at the vertex level, which was primarily considered a fibroma and showed marked hypointenses and heterogeneous contrast enhancement in all sections.</p><p>In the evaluation PET-CT performed after four cycles of the R-HYPERCVAD regimen, a mild increase in metabolic activity was observed in the mediastinal lymph nodes, but this was consistent with inflammatory processes, and no signs of recurrence or metastasis were found in other parts of the body. Despite these findings, which were accepted as a response to treatment, the planned OKIT treatment was not accepted by the patient and their relatives.</p><p>After completing the seventh course of treatment, the patient presented to the emergency room with altered consciousness and recurrent headaches. Antieodema treatment ","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2531137924001147/pdfft?md5=67c088ae42f5d3d9c799ce8fe9d674ef&pid=1-s2.0-S2531137924001147-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140878832","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy Assessment of Cytomegalovirus-Specific Immunoglobulins for the Management of CMV Reactivation in High-Risk Hematopoietic Stem Cell Transplant Recipients","authors":"Ignas Gaidamavičius ,&nbsp;Domas Vaitiekus ,&nbsp;Rolandas Gerbutavičius ,&nbsp;Milda Rudžianskienė ,&nbsp;Rūta Dambrauskienė ,&nbsp;Miglė Kulbokė ,&nbsp;Martyna Beitnerienė","doi":"10.1016/j.htct.2024.04.048","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.048","url":null,"abstract":"<div><h3>Background</h3><p>Cytomegalovirus (CMV) infection remains a prevalent and an important challenge encountered post haematopoietic stem cell transplantation (HSCT). If left unaddressed, CMV infection can escalate to CMV disease with adverse outcomes. Additionally, CMV infection alone can indirectly contribute to reduced overall survival (OS) and increased non-relapse mortality (NRM). Prevention serves as the cornerstone for managing CMV infection, while early pre-emptive strategies are employed to mitigate the risk of end-organ disease. Risk factors for CMV infection or disease include CMV seropositive recipients, mismatched and unrelated transplants, the use of T cell depletion agents, steroid therapy, graft-versus-host disease (GvHD), administration of CMV-positive blood products in seronegative recipients, among the other factors. Valganciclovir or ganciclovir remain the mainstream of CMV management in HSCT; however, due to adverse effects such as leukopenia and nephrotoxicity, some patients may exhibit intolerance to these medications or necessitate early discontinuation. Recent survey from the European Society for Blood and Marrow Transplantation (EBMT) highlight the inclusion of CMV immunoglobulin (CMVIG) as a therapeutic option for prophylaxis or pre-emptive interventions.</p></div><div><h3>Objectives</h3><p>To assess the efficacy of CMVIG in managing early CMV reactivation among high-risk HSCT recipients.</p></div><div><h3>Methods</h3><p>Between December 2022 and February 2024, 13 high-risk patients’ post-prophylaxis with early detection of CMV reactivation were treated with CMVIG. All patients were managed with CMVIG as monotherapy. Clinical parameters such as viral load (IU/ml), medication side effects, and patient tolerance were systemically evaluated.</p></div><div><h3>Results</h3><p>All patients experiencing CMV reactivation exhibited at least one-risk factor predisposing them to CMV reactivation, including D+/R+ serostatus, exposure to anti-thymocyte globulin (ATG), or received a matched unrelated donor (MUD) or haploidentical donor. All the patients were receiving Valacyclovir 500 mg b.i.d. for prophylaxis. The median age at transplantation was 53.8 years (range: 24-69). The median viremia level detected during treatment was 3175 IU/ml (n=6). A favourable response defined as achieving undetectable CMV viral load was achieved in all patients. None of the patients required additional antiviral therapy following early detection of CMV viral load. The median duration to achieve viral response was 20 days. CMVIG was well tolerated among all patients, with no reported adverse reactions recorded.</p></div><div><h3>Conclusion</h3><p>This single-center analysis demonstrates the clinical efficacy of CMVIG in the pre-emptive management of CMV reactivation, achieving complete remission in all patients without necessitating additional antiviral therapy. Given the inherent neutropenic status of such patients, the use of CMVIG represents a","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2531137924001305/pdfft?md5=25bfc65b564c8773e6a0c00f173854d9&pid=1-s2.0-S2531137924001305-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140879027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"SEVERE CONGENİTAL NEUTROPENİA WİTH GLUCOSE-6-PHOSPHATASE CATALYTİC SUBUNİT 3 (G6PC3) DEFİCENCY OR DURSUN SYNDROME DİAGNOSED AT ADULTHOOD","authors":"Yunus CATMA ,&nbsp;Elif Sakci ,&nbsp;Tugba Kalayci ,&nbsp;Sevgi Kalayoglu Besisik","doi":"10.1016/j.htct.2024.04.041","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.041","url":null,"abstract":"<div><h3>Case report</h3><p>Severe congenital neutropenia is rare and usually diagnosed at childhood. G6PC3 deficiency emerge by mutation in glucose metabolism controlling genes as a syndromic variant. We here present a young adult case with unexplained neutropenia after kidney transplantation for FMF related AA amyloidosis. He had facial dismorphism, growth retardation, and atrial septal defect. Parents were relatives and he had recurrent infection history. Genetic screening revealed G6PC3 gene mutation in patient.</p></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2531137924001238/pdfft?md5=c0a66896dc9eac8ee3b520eeee5c0cb1&pid=1-s2.0-S2531137924001238-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140878541","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"EFFECT OF HEREDITARY THROMBOPHILIA ON ARTERIAL THROMBOSIS","authors":"Ferda Can ,&nbsp;Tansu Büyükgül ,&nbsp;Nuray Yılmaz Cakmak ,&nbsp;Ihsan Ates ,&nbsp;Vehap Topcu ,&nbsp;Said Furkan Yıldırım ,&nbsp;Sema Akıncı","doi":"10.1016/j.htct.2024.04.006","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.006","url":null,"abstract":"<div><h3>Objective</h3><p>Screening for hereditary thrombophilia is recommended for venous thrombosis, but there is conflicting information about the causal relation with arterial thrombosis. In this study, in order to clarify these conflicting results and recommendations, it was aimed to determine whether there is a relation between arterial thrombosis and hereditary thrombophilia tests, to determine whether the treatment plan changes according to the test results of patients with hereditary thrombophilia panel, and t</p></div><div><h3>Methodology</h3><p>In this single-centre, non-intervention, retrospective cohort study, 200 patients over the age of 18 who were performed hereditary thrombophilia tests by various clinics between 12/02/2019 and 01/07/2022 were included. The patients had no history of disease predisposing to thrombosis, no rheumatological disease, negative antiphospholipid antibodies, and arterial thrombosis. As a control group, 50 patients without arterial and venous thrombosis were included.</p></div><div><h3>Results</h3><p>When the patient group with arterial thrombosis was compared with the control group, no difference was found in the risk of thrombosis in terms of factor V Leiden, prothrombin, Factor XIII, MTHFR 677, MTHFR 1298, PAI-1 gene mutation (p=0.084, p=0.82, p=1, p=0.65, p=0.064, p=1, respectively). In our study, no significant difference was found in the increased risk of thrombosis in the detection of thrombophilic gene tests in arterial thrombosis compared with the control group.</p></div><div><h3>Conclusion</h3><p>In our study, thrombophilia gene panel screening was not considered necessary in patients with arterial thrombosis, and it was observed that factor V Leiden, prothrombin, Factor XIII, MTHFR 677, MTHFR 1298, PAI-1 gene mutations in the hereditary thrombophilia panel did not lead to an increased risk of arterial thrombosis. Hereditary thrombophilia testing is not recommended in patients with arterial thrombosis according to current guidelines.</p></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2531137924000889/pdfft?md5=8525269f47eda9fedea015df7249f6bc&pid=1-s2.0-S2531137924000889-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140878902","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"AUTOLOGOUS PERIPHERAL BLOOD STEM CELL TRANSPLANTATION IN PATIENTS WITH HIV-ASSOCIATED LYMPHOPROLYPHERATIVE DISORDER","authors":"Vera Kovalskaya,&nbsp;Natalya Falaleeva,&nbsp;Stanislav Shklyaev,&nbsp;Andrey Chelmakov,&nbsp;Ludmila Grivtsova","doi":"10.1016/j.htct.2024.04.008","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.008","url":null,"abstract":"<div><h3>Objective</h3><p>Autologous transplantation of bone marrow/peripheral blood stem cells in patients with HIV-associated lymphopoliferative disorder is a feasible and relatively safe therapeutic option. However, at the moment there are a number of unsolved problems, including optimal risk/benefit pre-transplant conditioning, taking into account drug-drug interactions and indications for hematopoietic stem cell transplantation.</p></div><div><h3>Methodology</h3><p>Since 2020 PBSCT has been performed in 15 patients with HIV in our center. The 12 (80%), had diagnosis of HIV-associated plasmablastic lymphoma (HIV-PBL), 3 patients (20%) were with HIV-associated Hodgkin' lymphoma (HIV-HL). All of the patients with HIV-PBL were transplanted after completion of a first-line treatment and achievement of at least a partial response. The pre-transplant conditioning was performed using BEAM-like regimens.</p></div><div><h3>Results</h3><p>Toxicity from organs and systems did not exceed grade 2 (moderate) mainly from the gastrointestinal tract, no need antiretroviral therapy in all of the cases. Median time to neutrophil engraftment was +12 days, while to platelet engraftment was +13 days. At the time of submitting the abstract all of the transplanted patients described above except one (lethal case due to progression of concomitants hepatitis C virus (HCV) infection) are in the state of remission.</p></div><div><h3>Conclusion</h3><p>Autologous transplantation of peripheral blood stem cells in patients with HIV is a feasible and relatively safe option with clear planning of the patient's treatment strategy from the first day of therapy and accompanying consideration of drug-drug interactions which is confirmed both by world literature and our Center's own experience.</p></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2531137924000907/pdfft?md5=ea7f7eea47110b35c2cb63c6ff6f6332&pid=1-s2.0-S2531137924000907-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140878904","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"GRANULOCYTE TRANSFUSION ACCELERATES RECOVERY FROM NEUTROPENIA IN PATIENTS WITH HEMATOLOGIC MALIGNANCIES","authors":"Ayşegül Çelik ,&nbsp;Ali Ünal ,&nbsp;Mustafa Baydar","doi":"10.1016/j.htct.2024.04.038","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.038","url":null,"abstract":"<div><h3>Objective</h3><p>Neutropenia is the most common and serious consequence of myelosuppressive chemotherapy in patients with hematologic malignancies.Granulocyte transfusions can restore granulocyte counts and thus theoretically reduce the risk of infection in such patients.In our study, we aimed to demonstrate the efficacy of granulocyte transfusion in neutropenic patients with hematologic malignancy despite recombinant myeloid growth factor therapy.</p></div><div><h3>Methodology</h3><p>In this retrospective study, 72 patients who were treated in our hematology clinic between 2016 and 2022 and who met the criteria of our study were included.</p><p>Demographic data, malignancy subtypes, chemotherapy regimens, number of neutropenic days, clinical outcome before and after granulocyte transfusion, and neutrophil count changes in blood parameters were analyzed.In the study, p-values less than 0.05 were considered significant. The analyses were analyzed with the SPSS 25.0 program.</p></div><div><h3>Results</h3><p>In our study, 56.9% of the patients were male, the most common diagnosis was AML with 65.3% and 91.7% Gram-/+ was the most common type of treatment.It was observed that 62.5% of the patients recovered from neutropenia after granulocyte transfusion and 37.5% did not recover or exited.It was observed that patients who were neutropenic before chemotherapy were more likely to recover from neutropenia after granulocyte transfusion (p=0.01) and had lower rates of recovery from neutropenia (p=0.04).</p></div><div><h3>Conclusion</h3><p>Considering the present results, granulocyte transfusion seems to accelerate the recovery from neutropenia in the sample we analyzed. In addition, the diagnosis of the patient, the type of chemotherapy received, and the time of granulocyte transfusion were evaluated as factors affecting the results. However, in light of the data obtained, we believe that prospective studies with a larger number of patients should be conducted to evaluate the consistency of our results.</p></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2531137924001202/pdfft?md5=a6cf24ab79e29fd4e4c096ef57f64a28&pid=1-s2.0-S2531137924001202-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140879028","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"MAINTENANCE LOW-DOSE CORTICOSTEROID THERAPY IN PATIENTS WITH CHRONIC ITP: SINGLE CENTER RESULTS","authors":"Birsen Sahip Yesiralioğlu,&nbsp;Hatice Ayağ,&nbsp;Müzeyyen Aslaner Ak,&nbsp;Şehmus Ertop","doi":"10.1016/j.htct.2024.04.009","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.009","url":null,"abstract":"<div><h3>Objective</h3><p>Immune thrombocytopenia (ITP) is an acquired, autoimmune disease affecting 2 to 4 individuals per 100,000 annually. ITP is characterized by an isolated platelet count of &lt;100 × 10³/μL. The diagnosis of primary ITP relies on excluding non-immune causes of thrombocytopenia (myelodysplastic syndrome, inherited thrombocytopenia) and secondary immune thrombocytopenia caused by other conditions such as autoimmune diseases (systemic lupus erythematosus), malignancies (chronic lymphocytic leukemia), infections (hepatitis C virus and HIV), and medications. The clinical manifestations of ITP range from entirely asymptomatic patients to increased petechiae-ecchymosis and rarely major or life-threatening bleeding. Corticosteroids are the first-line treatment. Initial treatment for ITP consists of methylprednisolone at a dose of 1mg/kg/day or dexamethasone administered at 40mg/day for 4 days, repeated every 14-28 days. While &gt;75% of adult patients respond to corticosteroids, only 20-30% remain in continuous remission after cessation.\"</p></div><div><h3>Methodology</h3><p>We have 114 registered patients with immune thrombocytopenia (ITP) in our clinic over the past 5 years. Among these patients, a subgroup of 45 who received high-dose corticosteroid treatment as first-line therapy, responded to corticosteroids, but subsequently experienced loss of response, was identified as corticosteroid-sensitive. This group was selected for follow-up with maintenance low-dose steroid (LDS) therapy for 1 year. Patients were treated with 4 mg of methylprednisolone for 4 days per month and followed up for 12 months. Among our patients, 18 achieved response with platelet levels &gt;30 × 10 ³ / μ L without signs of bleeding (see Table 1), while in 27 patients, additional corticosteroid doses were added or second-line treatment modalities such as splenectomy or eltrombopag were initiated due to platelet levels dropping below 30 × 10 ³ / μ L \"</p><p><strong>Results:</strong></p></div><div><h3>Conclusion</h3><p>The goal of treatment in a patient with ITP is not only to normalize platelet counts but also to achieve a level of platelets that can prevent clinically significant bleeding. Based on this premise, we have demonstrated that maintenance corticosteroid therapy at an acceptable cumulative dose can reliably maintain platelet levels within a safe range. We believe that this should be further supported by larger multicenter studies.\"</p></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2531137924000919/pdfft?md5=fab6ebb657562068536bc74f9cf949f9&pid=1-s2.0-S2531137924000919-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140878976","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"SHIFTING PARADIGMS: EXPLORING ENARODUSTAT FOR ANEMIA IN CHRONIC KIDNEY DISEASE IN A META ANALYSIS OF RANDOMIZED CONTROLLED TRIALS","authors":"Lokman Hekim Tanrıverdi ,&nbsp;Ahmet Sarıcı","doi":"10.1016/j.htct.2024.04.012","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.012","url":null,"abstract":"<div><h3>Objective</h3><p>Anemia commonly accompanies chronic kidney disease (CKD). Erythropoiesis-stimulating agents (ESAs), such as darbepoetin, are initiated for anemia in CKD. Additionally, hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitors have demonstrated efficacy in treating CKD-associated anemia. This meta-analysis aims to compare the efficacy, safety, and tolerability of enarodustat in anemic CKD patients.</p><p><strong>Case report</strong></p></div><div><h3>Methodology</h3><p>A systematic search of Cochrane CENTRAL, Ovid Medline R, PubMed, and Web of Science databases up to March 1, 2024, was conducted. Randomized controlled trials (RCTs) directly comparing enarodustat with darbepoetin were included. Data from four unique RCTs comprising an inverse variance-weighted random-effects model were utilized for the main analysis. Primary efficacy outcome measures included hemoglobin (Hb) change at weeks 4-6 and during follow-up, while primary safety outcomes focused on serious adverse events (SAEs). Subgroup analyses were performed based on dialysis status and prior use of ESA for the primary outcome.</p></div><div><h3>Results</h3><p>Four RCTs with 7 reports involving 586 patients were included in the main analysis. Enarodustat demonstrated superiority to control in terms of change in Hb levels at week 4-6 (RR 0.76, 95% CI 0.02 to 1.50, I2=96%, p=0.04) but non-inferiority during follow-up (MD 0.66, 95% CI -0.22 to 1.53, I2=91%, p=0.14). Enarodustat exhibited comparable effects for safety and tolerability parameters such as SAEs (RR 1.17, 95% CI 0.72 to 1.91, I2=0%, p=0.52), any adverse events (RR 0.95, 95% CI 0.82 to 1.08), any adverse events leading to discontinuation (RR 0.90, 95% CI 0.37 to 2.20), diarrhea (RR 1.50, 95% CI 0.05 to 43.15), hypertension (RR 0.89, 95% CI 0.43 to 1.84), and all-cause mortality (RR 0.63, 95% CI 0.08 to 5.08). Subgroup analysis by dialysis status revealed nonsignificant differences for change in Hb levels at week 4-6 and during follow-up, but comparator-based subgroup analysis demonstrated a significant difference only when comparing to placebo at week 4-6.</p></div><div><h3>Conclusion</h3><p>Enarodustat exhibits promise as a treatment option for anemia associated with CKD, demonstrating superiority to control in terms of Hb change at week 4-6 and non-inferiority during follow-up. Moreover, it demonstrates comparable safety and tolerability profiles to darbepoetin, making it a potential alternative in the management of CKD-related anemia.</p></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2531137924000944/pdfft?md5=36b36a4ccc7d2a59cb04a9275032cfca&pid=1-s2.0-S2531137924000944-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140878826","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"FLUDARABINE-INDUCED BRADYCARDIA IN ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION","authors":"Serhat Çelik ,&nbsp;Zeynep Tuğba Güven ,&nbsp;Abdullah Altınsoy ,&nbsp;Şaziye Esra Tubay ,&nbsp;Muzaffer Keklik ,&nbsp;Ali Ünal","doi":"10.1016/j.htct.2024.04.010","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.010","url":null,"abstract":"<div><h3>Objective</h3><p>Fludarabine, a purine analog, is getting more attention with the increasing use of reduced intensive conditioning regimens in allogeneic hematopoietic stem cell transplantation (allo-HSCT). Bradycardia was observed in only a few cases reported in the literature. In clinical practice, bradycardia can be asymptomatic or cause syncope and cardiac arrest. This study aimed to evaluate the bradycardia side effect of fludarabine used in allo-HSCT recipients and to increase awareness of this issue.</p></div><div><h3>Methodology</h3><p>This retrospective study included 73 patients who received fludarabine in the allo-HSCT conditioning regimen between January 2015 and January 2021. Patients with and without bradycardia were compared regarding demographic data, allo-HSCT characteristics, electrolyte values, fludarabine administration dose and duration, and survival. Univariate and multivariate analyzes were performed to evaluate independent predictors for fludarabine-induced bradycardia (FİB).</p></div><div><h3>Results</h3><p>Fludarabine doses were higher in the bradycardia group, but not statistically significant. Age was the only independent predictor of FİB (OR 0.93, 95% CI: 0.89-0.98, p =0.007). The median age in the group with bradycardia was 19 years younger than those without bradycardia (34 (19-49) vs 53 (19-69), p=0.005). In 11 (84.6%) of the patients who had bradycardia, bradycardia improved with the discontinuation of fludarabine alone, but atropine was administered in 2 (15.4%) patients.</p></div><div><h3>Conclusion</h3><p>Bradycardia was observed in 17.8% of our patients who used fludarabine in the conditioning regimen. Age was the only independent predictor of fludarabine-induced bradycardia; therefore, close heart rate monitoring is recommended during fludarabine administration, especially in younger patients. Although our results are promising, further studies evaluating the fludarabine intermediate fluoroadenosine are needed to support our results.</p></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2531137924000920/pdfft?md5=4375d34715c97e66b9b90fe7246a06b1&pid=1-s2.0-S2531137924000920-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140878977","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}