Hematology, Transfusion and Cell Therapy最新文献

{"title":"EFFICACY OF ROXADUSTAT IN CHRONIC KIDNEY DISEASE PATIENTS NOT ON DIALYSIS WITH ANEMIA: SYSTEMATIC REVIEW AND META-ANALYSIS OF RANDOMIZED CONTROLLED TRIALS","authors":"Lokman Hekim Tanriverdi ,&nbsp;Ayşe Uysal ,&nbsp;Arzu Akyay ,&nbsp;Yurday Öncül ,&nbsp;Ahmet Sarici","doi":"10.1016/j.htct.2025.103924","DOIUrl":"10.1016/j.htct.2025.103924","url":null,"abstract":"<div><h3>Objective</h3><div>Anemia is a common complication in patients with Chronic Kidney Disease (CKD), particularly in those not receiving dialysis. Roxadustat, a Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitor (HIF-PHI), has been investigated as a therapeutic option for anemia management in this population. This study aimed to evaluate the efficacy of Roxadustat compared to control interventions in Non-Dialysis-Dependent CKD (NDD-CKD) patients.</div></div><div><h3>Methodology</h3><div>A comprehensive literature search was conducted in Cochrane CENTRAL, Ovid Medline, PubMed, and Web of Science up to December 14, 2024. Randomized Controlled Trials (RCTs) directly comparing Roxadustat with a control group were included. Data were pooled using an inverse variance-weighted random-effects model. The primary efficacy outcome was the change in Hemoglobin (Hb) levels at weeks 24–28 and during follow-up. Subgroup analyses were performed based on the type of control intervention (Erythropoiesis-Stimulating Agents [ESAs] vs. placebo) and prior ESA use.</div></div><div><h3>Results</h3><div>A total of six RCTs, including 5,330 patients, from 520 unique records from the databases were included. Roxadustat significantly increased Hb levels during follow-up compared to the control group (Mean Difference [MD = 1.21 g/dL], 95% confidence interval [95% CI 0.45 to 1.97], I² = 99%, p = 0.0017). However, at weeks 24–28, the increase in Hb levels was not statistically significant (MD = 0.86 g/dL, 95% CI -0.11 to 1.83, I² = 99.4%, p = 0.0833). Iron-related parameters showed mixed results. Roxadustat was associated with a significant reduction in ferritin levels (MD = -38.54 ng/mL, 95% CI -68.21 to -8.87, I² = 84.1%, p = 0.0109). Conversely, Total Iron-Binding Capacity (TIBC) was significantly increased with Roxadustat treatment (MD = 20.33 μg/dL, 95% CI 1.15 to 39.51, I² = 98.5%, p = 0.0377). No significant difference was observed in serum iron (MD = 3.1 μg/dL, 95% CI -0.39 to 6.6, I² = 93.1%, p = 0.0820) and Transferrin Saturation (TSAT) levels (MD = -1.08%, 95% CI -2.42 to 0.26, I² = 40.1%, p = 0.1151) between the two groups. Subgroup analyses revealed that in placebo-controlled trials, Roxadustat significantly increased Hb levels at both weeks 24–28 and during follow-up. However, in trials comparing Roxadustat with ESAs, the changes in Hb levels were not significant at either time point.</div></div><div><h3>Conclusion</h3><div>Roxadustat reduced ferritin but increased TIBC without significantly affecting free iron and TSAT levels compared to the control group in patients with NDD-CKD.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103924"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534312","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"LOW DOSE CYTARABINE PLUS SORAFENIB IN AN ELDERLY PATIENT WITH ACUTE MYELOID LEUKEMIA","authors":"Tural Mahmudov","doi":"10.1016/j.htct.2025.103886","DOIUrl":"10.1016/j.htct.2025.103886","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Introduction&lt;/h3&gt;&lt;div&gt;Acute Myeloid Leukemia (AML) is the most common acute leukemia in adults and is generally associated with a poor prognosis. The failure of therapeutic approaches in AML treatment is attributed to various clinical characteristics of patients, disease biology, and treatment intensity. Mutations in the Fms-Like Tyrosine kinase-3 (FLT3) receptor have been reported in approximately one-third of AML cases. The most common FLT3 mutation is Internal Tandem Duplication (ITD), which has been identified in approximately 25% of adult AML patients and in 3%–5% of newly diagnosed Myelodysplastic Syndromes (MDS). FLT3-ITD is associated with high White Blood Cell (WBC) counts, elevated Lactate Dehydrogenase (LDH) levels, increased percentages of blast cells in the blood and bone marrow, and poor clinical outcomes. However, it does not appear to significantly affect the ability of adult patients to achieve Complete Remission (CR).&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Objective&lt;/h3&gt;&lt;div&gt;This case report presents the response of a 71-year-old AML patient to low-dose Cytarabine (Ara-C) combined with sorafenib treatment.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Case presentation&lt;/h3&gt;&lt;div&gt;A 71-year-old female patient presented in February 2024 with complaints of excessive thirst, fatigue, weakness, and loss of appetite. At diagnosis, leukocytosis, anemia, and thrombocytopenia were observed. Peripheral blood smear analysis revealed blast cell infiltration, and immunophenotypic studies identified markers consistent with the AML M5 subtype: CD34+/− (3.4%), CD123+, CD33+, CD13+, CD14+, CD36+, CD64+, HLA-DR+, and cMPO+. Conventional karyotyping was normal, whereas molecular analysis detected FLT3-ITD (51%, 27 bp mutant). Given the patient’s overall health status, a treatment regimen of low-dose Ara-C (20 mg BID on days 1–10) and sorafenib (400 mg on days 11–28) was initiated. Due to hematologic toxicity, dose reductions were necessary during treatment. After four cycles, bone marrow aspiration revealed a blast percentage of 0.8%, FLT3-ITD mutation was no longer detectable, and Minimal Residual Disease (MRD) negativity was achieved, confirming Complete Remission (CR). This treatment protocol was selected based on the patient's clinical condition, leading to a successful outcome.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;div&gt;FLT3-ITD-positive AML patients may benefit from low-dose Ara-C and sorafenib therapy, particularly when carefully selected based on clinical criteria. However, further comprehensive randomized prospective studies are required to better evaluate the efficacy and safety of this approach.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Discussion&lt;/h3&gt;&lt;div&gt;This case highlights the efficacy of low-dose cytarabine and sorafenib combination therapy in an elderly AML patient with FLT3-ITD mutation. FLT3-ITD mutation is a well-established marker of poor prognosis in AML and is associated with resistance to conventional therapies. In elderly AML patients, treatment decisions are often challenging due to com","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103886"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534468","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"EXPERIENCE WITH TOTALLY IMPLANTABLE VENOUS ACCESS PORTS IN PATIENTS WITH HEMATOLOGICAL MALIGNANCIES","authors":"Erkan Bilgin,&nbsp;Ahmet Bayrak","doi":"10.1016/j.htct.2025.103888","DOIUrl":"10.1016/j.htct.2025.103888","url":null,"abstract":"<div><h3>Objective</h3><div>Totally Implantable Venous Access Ports (TIVAP) are widely preferred for oncology patients requiring chemotherapy.[1] The most common cause of port removal in hematological malignancy patients is infection, followed by dysfunction.[2,3] The aim of this study is to evaluate single center experience of TIVAP in patients with hematologic oncology.</div></div><div><h3>Methodology</h3><div>126 patients who were followed up in the hematology-oncology department of the tertiary oncology hospital and who had port catheters inserted in the interventional radiology unit between January 2019 ‒ January 2022 was evaluated retrospectively. Post-procedure control radiographs of the patients were evaluated for early complications and localization suitability. Additionally, patients’ port catheter follow-ups were evaluated for infection and dysfunction.</div></div><div><h3>Results</h3><div>68 (54%) of the patients were male and 58 (46%) were female. The age range of the patients was 18‒65, and the average age was calculated as 41.3. 60 (48%) patients were followed up with a diagnosis of acute myeloid leukemia, 30 (24%) with acute lymphocytic leukemia, 24 (19%) with non-Hodgkin lymphoma, 10 (8%) with Hodgkin lymphoma, and 2 (1%) with multiple myeloma. In the control radiograph examination taken after the procedure, the port location was appropriate in 124 patients and the port catheter ended in the right ventricle in 2 patients. No early complications were observed in any of the patients. Port catheter dysfunction was observed in 8 patients (6%) during follow-up. The average duration of dysfunction development was calculated as 17.25 weeks. Port infection developed in 14 patients (11%) and the average time to develop port infection was calculated as 3.4 weeks. Port infection rates are higher in hematological malignancy patients compared to other malignancy patient groups.</div></div><div><h3>Conclusion</h3><div>Although the use of port catheters is common in patients with hematological malignancy, caution should be exercised in terms of possible port infection.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103888"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534470","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"TWO CASES OF PRIMARY AMYLOIDOSIS PRESENTING WITH VERTEBRAL AMYLOIDOMA","authors":"Ebru Kavak Yavuz,&nbsp;Vehbi Demircan,&nbsp;Abdullah Karakus,&nbsp;Orhan Ayyildiz","doi":"10.1016/j.htct.2025.103928","DOIUrl":"10.1016/j.htct.2025.103928","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Objective&lt;/h3&gt;&lt;div&gt;Amyloidosis is a rare disease in which symptoms occur due to protein misfolding and the accumulation of amyloid fibrils in organs and tissues. 20 types of amyloid proteins have been identified. AL (Amyloid Light chain), AA (Amyloid Associated), Aβ (Amyloid beta) constitute the major types. AL type contains free immunoglobulin light chain amino terminals formed by plasma cells. AA amyloidosis occurs due to the accumulation of Serum Amyloid A (SAA) in tissues due to severe and long-term infection or inflammation. AL type amyloidosis accompanies approximately 10% of multiple myeloma. The frequency of AL amyloidosis is between 3 and 12 per million. It is more common in men and occurs at an average age of 63. AL amyloidosis can affect various tissues. The involvement of AL amyloidosis is in the heart, kidney, liver, nervous system and very rarely in the vertebral area.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Case 1&lt;/h3&gt;&lt;div&gt;A 56-year-old male patient was admitted to the hospital with complaints of low back pain, leg pain, numbness, and inability to walk. After examinations, a compression fracture was detected that caused height loss at L5, and he underwent surgery by a neurosurgeon. After the operation, fixators were placed at L3, L4, and S1. AL amyloidosis was detected as a result of tissue biopsy taken after surgery. Serum free kappa and lambda levels were studied. Lambda light chain was found to be 41.4 mg/dL (8.3‒27 mg/dL) high. Other examination results were urea: 53 mg/dL (17‒43 mg/dL), creatinine: 0.95 mg/dL (0.67‒1.17 mg/dL), calcium 8.8 mg/dL (8.8‒10.6 mg/dL), INR = 1.6, PT = 18.1 sec (10‒14 sec), APTT 35.6 sec (21‒29 sec), Hbg: 9g/dL (12.9‒14.2 g/dL), MCV = 78fL (81‒96 fL), platelet: 220 10^3/UL (155‒366 10^3/U/L), complete urine test was normal, there was no proteinuria. Bone marrow biopsy showed 5%‒7% plasma cells. NTpro BNP was 153 pg/mL (&lt; 100 pg/mL), Echocardiography (ECHO) showed the left atrium wider than normal, other heart chambers were of normal width and their wall thicknesses increased. It was evaluated as hypertrophic cardiomyopathy. Ejection Fraction (EF) was measured as 60% with preserved systolic functions. The patient was started on VCD (cyclophosphamide, bortezomib, dexamethasone) and daratumumab treatment with the diagnosis of primary amyloidosis. The patient developed a hematoma in the surgical area, factor levels were studied, factor 10 level was found to be 10% low. Upon numbness, Electromyomography (EMG) was performed on bilateral lower extremities. EMG results showed low bilateral tibial nerve amplitudes. The patient's complaints of loss of strength in the legs and inability to walk began to improve after treatment.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Case 2&lt;/h3&gt;&lt;div&gt;A 60-year-old female patient with diabetes mellitus and coronary artery disease presented with complaints of back pain and difficulty walking. As a result of the vertebral MRI examination performed for the complaints, a lytic mass lesion of 27 × 13 mm in size,","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103928"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534486","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"HISTORY OF BLOOD TRANSFUSION","authors":"Tanju Atamer","doi":"10.1016/j.htct.2025.103872","DOIUrl":"10.1016/j.htct.2025.103872","url":null,"abstract":"<div><div>It is not easy to cover the history of transfusion in all its aspects. In the era when blood transfusion was first tried, the indications for blood transfusion were also very different: mental illness, gaining strength, rejuvenation, etc. Blood transfusion process has gone through many dangerous and difficult stages, from obtaining the blood to its safe application in peace, in war, and in the laboratory. This presentation focuses on some stages and developments in the application of blood transfusion. The main developments are summarized in Table 1. The main problems encountered in the history of transfusion can be summarized as follows:</div><div> <!-->- Blood clotting and preservation could be prevented by the use of 0.2% sodium citrate and dextrose.</div><div> <!-->- Severe transfusion reactions could mostly be prevented after the ABO blood groups were identified.</div><div> <!-->- Infection problems that could be partially controlled with antiseptic agents.</div><div>In this presentation, various obstacles encountered in the history of blood transfusion and developments regarding their solutions are presented.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103872"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534539","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"MDS DIAGNOSIS? STILL BY BONE MARROW EXAMINATION?","authors":"Moshe Mittelman","doi":"10.1016/j.htct.2025.103868","DOIUrl":"10.1016/j.htct.2025.103868","url":null,"abstract":"<div><div>The Myelodysplastic Syndromes (MDS) are a heterogenous group of clonal Bone Marrow (BM) stem cell myeloid neoplasms, characterized by BM dysplasia, macrocytic anemia or cytopenia with a tendency for leukemic transformation. The suspicion of MDS is raised by a typical but not specific clinical picture and routine laboratory findings, but the gold standard for the diagnosis of MDS is still BM examination with the presence of uni- or multi-lineage dysplasia and blast percentage, together with exclusion of other reasons. Cytogenetics is also a part of the diagnostic process. Flow cytometry and genetics are helpful but are not always mandatory for the diagnosis of MDS. We will summarize the current steps in the diagnostic approach for a patient suspected of having MDS. I will also describe new concepts that use non-invasive diagnostic technologies, especially digital methods as well as peripheral blood genetics. The hope is that one day these will mature, be introduced into clinical practice, and perhaps in many cases even replace the invasive BM biopsy.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103868"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534609","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CASES OF PRIMARY CUTANEOUS ANAPLASTIC LARGE CELL LYMPHOMA TREATED WITH SYSTEMIC OR LOCAL THERAPY","authors":"Müzeyyen Aslı Ergözoplu,&nbsp;Berksoy Şahin,&nbsp;Ertuğrul Bayram,&nbsp;Esra Gökçe","doi":"10.1016/j.htct.2025.103896","DOIUrl":"10.1016/j.htct.2025.103896","url":null,"abstract":"<div><h3>Introduction</h3><div>Primary Cutaneous Anaplastic Large Cell Lymphoma (PC-ALCL) is a CD30+ peripheral T-cell lymphoproliferative disorder without systemic involvement. It accounts for approximately 8% of cutaneous lymphoma cases. Most patients with PC-ALCL present with slow-growing, solitary or grouped skin nodules, and in some cases, regional lymph node involvement is observed.</div></div><div><h3>Case-1</h3><div>A 61-year-old male patient presented to our clinic with swelling and edema of the right lower lip, along with a 57 cm draining ulcerative skin lesion in the suprasternal region. The patient had previously received six cycles of treatment for T-cell lymphoma at an outside center. A PET-CT scan identified a soft tissue lesion in the skin/subcutaneous tissue at the level of the right thyroid lobe and isthmus, measuring 57*15*52 mm, with a maximum SUV of 34. Biopsies taken from the lower lip and suprasternal skin were reported as primary cutaneous anaplastic large cell lymphoma. CD30 expression was found to be 95%. The patient was started on brentuximab vedotin along with the GDP protocol. After two cycles of treatment, improvement in the skin lesions was observed.</div></div><div><h3>Case-2</h3><div>A 61-year-old male patient presented to our clinic with a complaint of a lesion on the anterior surface of the right tibia, measuring approximately 10*10 cm. The biopsy taken from the lesion was reported as CD30+ primary cutaneous anaplastic large cell lymphoma. A PET-CT scan revealed moderately hypermetabolic lymph nodes with thick cortices in the right iliac and inguinal lymphatic chains. Radiotherapy was applied to the area of the primary lesion and the regional lymph nodes, and improvement in the skin lesions was observed with treatment.</div></div><div><h3>Discussion</h3><div>We aimed to discuss the outcomes of applying localized radiotherapy or systemic treatment to two patients with PC-ALCL who presented to our clinic: one with a relapse and the other with a new diagnosis. Brentuximab vedotin is effective in this disease. Since disease control could not be fully achieved with localized radiotherapy alone, we believe that the combination of systemic therapy and radiotherapy may be an important treatment option for these patients. Further large-scale case series are needed to guide treatment.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103896"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534477","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CYTOGENETIC FEATURES OF B-CELL ACUTE LYMPHOBLASTIC LEUKEMIA WITH INTRACHROMOSOMAL AMPLIFICATION OF CHROMOSOME 21","authors":"Jiantuo Liu,&nbsp;Hongrui Li,&nbsp;Li Jiang,&nbsp;Ping Zhang,&nbsp;Shanlin Zheng,&nbsp;Xuekui Qiu,&nbsp;Haiqin Zhang","doi":"10.1016/j.htct.2025.103881","DOIUrl":"10.1016/j.htct.2025.103881","url":null,"abstract":"<div><h3>Objective</h3><div>To investigate the cytogenetic features and prognosis of B-ALL with iAMP21.</div></div><div><h3>Methodology</h3><div>Retrospective analysis of data from 15 children diagnosed with B-ALL and iAMP21 seen in the Department of Hematology, Union Hospital, Tongji Medical College HUST from 2021 to 2024. Screened patients ranged in age from 1 month to 18-years. All were diagnosed with B-ALL using standard morphological and immunophenotypic criteria. Patients in this study were classified as iAMP21 using the criteria of 5 or more RUNX1 signals per cell and by a ratio of RUNX1 to tel 21q signals exceeding 1.</div></div><div><h3>Results</h3><div>In this study, 15 patients demonstrated 5 or more RUNX1 signals per interphase nucleus. Compared to RUNX1, all cases showed fewer subtelomeric signals in interphase cells, ranging from 1 to 5. The accurate interphase distinction of iAMP21 was made by calculating the ratio of RUNX1 to subtelomeric signals; the minimum and maximum values were 1.67 and more than 10, respectively. Metaphase FISH data were recorded if available. Of the cases, 5 of 15 demonstrated 3 or more extra copies of RUNX1 on a single abnormal chromosome 21 with the morphology of mar,r(21)c and add(21)c. Using MLPA, 4 of 15 cases showed gene deletion, including IKZF1, CDKN2A/B, ETV6, BTG1, and RB1. All cases received chemotherapy according to CCCG-ALL-2020. Induction regimens included a combination of vincristine, prednisone, and pegaspargase with daunorubicin. High-Dose Methotrexate (HD-MTX) and 6-Mercaptopurine (6-MP) were incorporated into consolidation regimens. Maintenance regimens were based on a backbone of daily 6-MP and weekly methotrexate with periodic vincristine and prednisone. 14 achieved remissions, and MRD remained negative; 1 did not achieve remission, and MRD was &gt; 0.01%.</div></div><div><h3>Conclusion</h3><div>iAMP21 is a primary cytogenetic abnormality located in the same region of amplification as the RUNX1 gene and a common region of deletion at the telomere. Some patients display other genetic abnormalities in addition to iAMP21. iAMP21 is associated with inferior outcomes, and patients with this abnormality require more intensive therapy.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103881"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534550","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"THE ROAD NOT-TAKEN: EXPLORING NON-TRANSPLANT OPTIONS IN DE NOVO PHILADELPHIA - POSITIVE ACUTE MYELOID LEUKEMIA","authors":"Mohamed Sharif,&nbsp;Mansour Alfayez","doi":"10.1016/j.htct.2025.103919","DOIUrl":"10.1016/j.htct.2025.103919","url":null,"abstract":"<div><h3>Background</h3><div>Philadelphia chromosome-positive acute myeloid leukemia (Ph+AML) is a rare and aggressive subtype of AML, characterized by the BCR::ABL1 fusion gene. It has historically been considered a high-risk leukemia, with allogeneic Hematopoietic Cell Transplant (HCT) recommended in the first remission. However, the emergence of targeted therapies, particularly potent Tyrosine Kinase Inhibitors (TKIs), has led to reconsideration of this approach. Drawing from advances in Philadelphia-positive acute lymphoblastic leukemia (Ph+ALL), where HCT omission has been explored successfully, this study examines whether a similar strategy can be applied in select Ph+AML cases.</div></div><div><h3>Objective</h3><div>To evaluate the feasibility of a non-HCT approach in Ph+AML by analyzing a case where transplant was omitted, and the patient achieved sustained remission.</div></div><div><h3>Methods</h3><div>We present a case of a 30-year-old male diagnosed with de novo Ph+AML, identified through cytogenetics and molecular testing. The patient received induction chemotherapy with cytarabine and anthracycline (3+7) along with a TKI. Due to complications, his initial TKI was switched to ponatinib, and consolidation therapy consisted of azacytidine, venetoclax, and ponatinib. Disease monitoring was performed using quantitative Polymerase Chain Reaction (qPCR) for BCR::ABL1 and Next-Generation Sequencing (NGS).</div></div><div><h3>Results</h3><div>The patient achieved a complete Molecular Response (MR 4.5) after the first cycle of consolidation therapy. Over 12-cycles of treatment, he maintained MRD negativity without emerging mutations. At 30-months post-diagnosis, he remains in sustained remission without undergoing HCT. Key factors that may have contributed to his favorable outcome include the absence of additional cytogenetic abnormalities, early achievement of deep molecular remission, and the use of a third generation TKI with activity against T315I mutations.</div></div><div><h3>Conclusion</h3><div>This case suggests that a transplant-free approach may be a viable option for select patients with Ph+AML. While HCT remains the standard of care, the use of targeted therapies such as ponatinib in combination with venetoclax and azacytidine may provide an alternative pathway to long-term remission. Further studies are needed to validate patient selection criteria and assess long-term efficacy and safety of this approach.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103919"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534562","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"OUTCOMES OF ALLOGENEIC STEM CELL TRANSPLANTATION IN PATIENTS WITH ACUTE MYELOID LEUKEMIA: A SINGLE-CENTER EXPERIENCE","authors":"Yakup Unsal ,&nbsp;Muhammed Murati ,&nbsp;Guler Delibalta ,&nbsp;Serdar Bedii Omay","doi":"10.1016/j.htct.2025.103887","DOIUrl":"10.1016/j.htct.2025.103887","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Objective&lt;/h3&gt;&lt;div&gt;The aim of this study is to evaluate the allogeneic hematopoietic stem cell transplantation data performed in our transplant center for patients diagnosed with Acute Myeloid Leukemia (AML).&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Materials and methods&lt;/h3&gt;&lt;div&gt;Between 2016 and 2024, a retrospective evaluation was conducted on 176 patients who underwent allogeneic hematopoietic stem cell transplantation at the Adult Stem Cell Transplant Unit of Private Emsey Hospital due to AML diagnosis.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;div&gt;The retrospective analysis of AML patients who underwent allogeneic transplantation revealed an average age of 43.2 years. A total of 130 patients received a related donor transplant, while 46 underwent unrelated donor transplantation. The Turkish National Bone Marrow Donor Bank (TÜRKÖK) was the only source for unrelated donors. HLA allele mismatch (1 allele) was present in 29 donors, and 80 transplants were performed between different genders. The average time for neutrophil engraftment was 17.7 days, and for platelet engraftment, it was 19.5 days. The 100-day mortality rate was determined to be 20%.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Conclusion&lt;/h3&gt;&lt;div&gt;AML is a hematologic malignancy that can be treated with allogeneic stem cell transplantation. Unrelated donor transplantation is a critical option for patients without a suitable family donor who require allogeneic transplantation. Our center’s AML transplant patient demographic data aligns with the findings in the literature.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Case report&lt;/h3&gt;&lt;div&gt;Acute Myeloid Leukemia (AML) is the most common clonal hematopoietic stem cell disorder in adults. Prognosis is assessed based on age, gender, performance status, and cytogenetic mutations. Hematopoietic Stem Cell Transplantation (HSCT) is a widely used treatment method, particularly for hematological malignancies. Allogeneic HSCT offers curative potential for many hematologic malignancies. Transplantation is performed in AML patients to reduce the risk of relapse. A fully HLA-matched related donor is the preferred choice for allogeneic HSCT. However, only about 25% of patients have a fully HLA-matched related donor. In such cases, an allogeneic transplant from an unrelated HLA-matched donor may be performed. This study presents data on allogeneic HSCT procedures performed in our transplant center for AML patients.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Methodology&lt;/h3&gt;&lt;div&gt;A retrospective analysis was conducted on allogeneic hematopoietic stem cell transplantations performed between 2016 and 2024 in the Adult Stem Cell Transplant Unit of Private Emsey Hospital for AML patients.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;div&gt;A total of 176 AML patients who underwent allogeneic transplantation were retrospectively analyzed. The median patient age was 43.2 years (range: 16–72), with 53% (n = 94) being male and 47% (n = 82) female. Two patients (1%) had previously undergone autologous stem cell transplantation, and seven patients (4%) had a history o","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103887"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534469","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}