Hematology, Transfusion and Cell Therapy最新文献

{"title":"LYSOZYME-INDUCED NEPHROPATHY IN CMML: A RARE BUT SIGNIFICANT COMPLICATION REQUIRING EARLY RECOGNITION AND INTERVENTION","authors":"Alfadil Haroon ,&nbsp;Mohamed Kindawi ,&nbsp;Ahmed S Alotaibi ,&nbsp;Ali Debsan Alahmari ,&nbsp;Hazzaa Alzahrani ,&nbsp;Mansour Alfayez ,&nbsp;syed Osman Ahmed","doi":"10.1016/j.htct.2025.103900","DOIUrl":"10.1016/j.htct.2025.103900","url":null,"abstract":"<div><h3>Background</h3><div>CMML is a clonal hematopoietic disorder with features of myelodysplasia and myeloproliferation, characterized by monocytosis. Monocytes secrete lysozyme, a cationic protein filtered by the glomerulus and reabsorbed in proximal tubules. Excess lysozyme accumulation causes tubular injury, leading to Lysozyme-Induced Nephropathy (LyN).</div></div><div><h3>Case 1</h3><div>A 72-year-old male with CMML that transformed into AML with a TP53 mutation. Laboratory results showed WBC 9 × 10⁹/L, Hb 80 g/Platelets 139 × 10⁹/L, and creatinine 162 µmoL/L. Additional findings included Na 129 mmoL/L, Cl 97 mmoL/L, Mg 0.51 mmoL/L, PO4 1.50 mmoLL, and 24-hour urine protein of 1.20 g/L. LyN was suspected and confirmed with a lysozyme level of 117 mcg/mL (2.7‒9.4). He was treated with Azacitidine and Venetoclax, achieving CR and normal renal function.</div></div><div><h3>Case 2</h3><div>A 60-year-old male with CMML initially presented with a creatinine level of 139 µmoL/L, Na 130 mmoL/L, K 2.6 mmoL/L, normal Magnesium, and a lysozyme level of 153 mcg/mL. His creatinine normalized with prednisone. He progressed to AML after six cycles of Azacitidine and later received four cycles of Venetoclax, achieving CR. Unfortunately, he passed away 18-months post-diagnosis due to pneumonia and pulmonary hemorrhage.</div></div><div><h3>Case 3</h3><div>A 57-year-old female with CMML transformed into AML underwent MSD SCT but relapsed after five months. She initially achieved CR with Aza-Ven, followed by DLI, but relapsed again after four years. She was unresponsive to Aza-Ven but improved with palliative cytarabine. Initially, she had AKI due to TLS, which improved with chemotherapy. During her last relapse, she had creatinine of 154 µmoL/L, lysozyme levels 124 mcg/mL and electrolyte imbalances. Her renal function significantly improved after cytarabine injections.</div></div><div><h3>Discussion/Conclusion</h3><div>Most frequent renal complications in CMML are LyN (56%) and renal infiltration by the CMML with incidence of AKI (34.9%) and CKD (7.6%). LyN is a rare and poorly understood complication of CMML. Filtered lysozyme accumulates in the renal cortex, causing severe hypokalemia via kaliuresis or direct tubular injury, potentially leading to kidney failure. In our patient CMML treatment, hydration and steroids restored kidney function. LyN in CMML necessitates early recognition and intervention to improve renal outcomes. Further research is needed to optimize treatment strategies.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103900"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534301","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":"CLINICO-BIOLOGICAL PROFILE AND MANAGEMENT OF CHRONIC LYMPHOCYTIC LEUKEMIA","authors":"Vasile Musteata ,&nbsp;Vasile Cepraga ,&nbsp;Larisa Musteata","doi":"10.1016/j.htct.2025.103890","DOIUrl":"10.1016/j.htct.2025.103890","url":null,"abstract":"<div><h3>Objective</h3><div>Nearly 60%‒70% of patients with Chronic Lymphocytic Leukemia (CLL) are oligosymptomatic at diagnosis. The objective of the study was highlighting the clinical evolution and hematological patterns, as well as the assessment of short- and long-term results of treatment of patients with CLL.</div></div><div><h3>Methodology</h3><div>We realized a prospective and cohort study. The clinical-hematological features of CLL, the short- and long-term results of therapeutic management were studied in 62 patients, who were treated and followed up in the Institute of Oncology of Moldova between 2019‒2024. The study was related to the outpatient and hospitalized care. The diagnosis was proved according to the IWCLL criteria based on the complete blood count with the detection of lymphocytosis ≥ 5 × 10^9/l, bone marrow aspiration with lymphocytic infiltration ≥ 30% and immunophenotyping. The study was carried out on a basis of the data collected from the outpatient records and from the observation sheets of the patients according to the questionnaire drafted for the achievement of the settled objective. All patients were staged according to Binet and RAI Classifications.</div></div><div><h3>Results</h3><div>There were 25 (40.3%) males and 37 (59.7%) females in the study group. The age of the analyzed group was between 53 and 87-years (average age – 55.2-years). Forty-two (67.7%) patients with CLL belonged to the age category of 60‒79 years. The ECOG-WHO score at diagnosis was 2-3. Most of the patients (34% or 54.8%) were referred to hematologist in stage A. Twenty-three (37.1%) patients were diagnosed in stage B and 5 (8.1%) – in stage C. Nine (39.1%) cases of autoimmune hemolytic anemia and 8 (34.8%) cases of metaplastic anemia were revealed in stage B. Leukocytosis varied between 88.7‒325.0 × 10ꝰ/l (average value – 161.2 × 10ꝰ/l). Lymphocyte count ranged between 81%‒97% (average value 89%). Bone marrow aspiration in stages A and B revealed lymphocyte expansion of 33%‒91%. The respiratory bacterial infections turned out to be frequently diagnosed (29 patients, or 46.8%): acute pneumonia in 10 (16.1%), acute bronchitis in 7 (11.3%), relapse of chronic bronchitis in 11 (17.7%), and tuberculosis in 1 (1.7%) patient. The patients with progressive stage A, stage B and C disease received combined immuno-chemotherapy. Under the antineoplastic treatment, the ECOG-WHO score improved to 0‒1. Overall survival over 3 and 5 ears was 100%.</div></div><div><h3>Conclusion</h3><div>Our prospective study of CLL proved a predominance of female gender, patients of 60‒79 years old and stage A at diagnosis. The prognosis emerged to be relatively favorable, with the overall survival rates sustained at 100% within 3 and 5 years.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103890"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534472","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":"A dream or reality: Consideration of ‘bloodless’ hematopoietic stem cell transplants for Jehovah’s witness patients","authors":"Michael MacNeill ,&nbsp;Adrienne Fulford ,&nbsp;Deanna Caldwell ,&nbsp;Uday Deotare","doi":"10.1016/j.htct.2025.103958","DOIUrl":"10.1016/j.htct.2025.103958","url":null,"abstract":"<div><h3>Background</h3><div>Hematopoietic stem cell transplantation (HSCT) is an important part of treatment for many hematologic conditions. The high-dose chemotherapy used in HSCTs puts patients at risk of significant cytopenias which often necessitate blood product transfusions. Certain populations, including Jehovah’s Witnesses, are unable to receive blood product transfusions during their transplant and thus, in the past, they have been seen as unsuitable candidates for transplantations. However, there has been growing evidence of the safety and efficacy of so-called “bloodless” HSCT protocols.</div></div><div><h3>Methods</h3><div>The most recent and relevant literature on “bloodless” transplants were identified through Embase, MEDLINE, and PubMed, and analyzed to construct a “bloodless” HSCT protocol at a Canadian centre. Since 2021, the regimen was utilized for four autologous transplantations in three different Jehovah’s Witness patients.</div></div><div><h3>Results</h3><div>None of the patients had a significant bleeding event nor a hemoglobin nadir below 8.0 g/dL. Minor bleeding events, predominantly mucositis, resolved with site-specific management. No patient had significant thrombocytopenia, and all the cell lines of patients had normalized without transfusions by the time of discharge. All patients were hospitalized for &lt;30 days, similar to the experience of the centre with “regular” autologous transplants.</div></div><div><h3>Conclusion</h3><div>Careful planning and tailored regimens support the achievability of “bloodless” HSCTs in patients, such as Jehovah’s Witnesses, allowing practitioners to provide care to a previously excluded group and minimize the use of blood products in all HSCT patients.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 3","pages":"Article 103958"},"PeriodicalIF":1.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144739118","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":"Digital polymerase chain reaction enhances analysis of the cKIT D816V mutation in systemic mastocytosis patients: Clinical insights","authors":"Alexandre Eiji Kayano,&nbsp;Luan Lima Marchi,&nbsp;Luciana Nardinelli,&nbsp;Evelin Alves Ramos,&nbsp;Eliane Aparecida Rosseto,&nbsp;Vanderson Rocha,&nbsp;Fernanda Salles Seguro,&nbsp;Elvira Deolinda Rodrigues Pereira Velloso","doi":"10.1016/j.htct.2025.103938","DOIUrl":"10.1016/j.htct.2025.103938","url":null,"abstract":"","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 3","pages":"Article 103938"},"PeriodicalIF":1.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144739119","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":"A CASE OF FAMILIAL PORFIRIA","authors":"Laman Hamidova,&nbsp;Oktay Abdullayev,&nbsp;Zari Balaşova,&nbsp;Farid Mammadov","doi":"10.1016/j.htct.2025.103898","DOIUrl":"10.1016/j.htct.2025.103898","url":null,"abstract":"<div><h3>Introduction</h3><div>Porphyria is a group of metabolic diseases caused by hereditary defects in the enzymatic system of heme biosynthesis. We present three cases of familial porphyria in two sisters (50 and 45-years-old) and a brother (39-years-old). The clinical symptoms of these patients were analyzed, and it was found that all patients have the same symptoms of the disease since childhood: constantly dark urine, burn-like changes on the skin of the face and ears, long-lasting wounds, moderate splenomegaly, and deformity of the joints on the fingers.</div></div><div><h3>Clinical case</h3><div>Here, we present the some clinical and laboratory data one of the three patients. This patient was initially diagnosed with scleroderma, but due to the splenomegaly, she was referred to a hematologist to clarify the diagnosis. It was found that since childhood she had black urine, poorly healing wounds, and burn-like changes on the face and on the hands, which most often appeared after exposure to the sun. A physical examination revealed splenomegaly (+2‒3 cm). The urine was initially intensely yellow-colored ‒ when exposed to bright sunlight, the color changed to a dark yellow color. Total porphyrins in daily urine were 1.93 mg/L (norm 0‒0.15 mg/L), uroporphyrinogen in single probe urine is 13 mg/L (norm 0‒2 mg/L), and δ-aminolevulinic acid is 16.7 mg/L (norm 0.1‒4.5 mg/L). The patient’s sister and brother also were examined for quantitative tests for porphyrins in the urine. Both had similar porphyrin samples in their urine: total porphyrins in single probe urine – 1.23‒1.3 mg/L, uroporphyrinogen in single probe urine – 12‒13 mg/L, δ-aminolevulinic acid – 17.1 mg/L.</div></div><div><h3>Conclusions</h3><div>Increased excretion of porphyrin precursors is one of the permanent signs of the disease and is observed not only during acute manifestations of the disease but also during the period of remissions, among the people with the latent form of the disease. Analyzing the results of our discussions, we can draw the following conclusions. Porphyria is a rare disease with very variable clinical symptoms, which causes certain difficulties in its timely diagnosis. To verify the diagnosis, even in the presence of a characteristic clinical picture, it is necessary to study the excretory profile of porphyrin metabolism with quantification of porphyrin precursors and fractions, as well as a comprehensive genetic examination.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103898"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534277","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 DIFFUSE LARGE B-CELL LYMPHOMA IN OLDER ADULTS TREATED IN RESOURCE-CONSTRAINED SETTINGS","authors":"Natasha Ali ,&nbsp;Raheel Iftikhar ,&nbsp;Zeeshan Khan ,&nbsp;Usman Ahmed ,&nbsp;Humera Mahmood ,&nbsp;Zeba Aziz","doi":"10.1016/j.htct.2025.103893","DOIUrl":"10.1016/j.htct.2025.103893","url":null,"abstract":"<div><h3>Objective</h3><div>Treating Diffuse Large B-Cell Lymphoma (DLBCL) in elderly patients is challenging. There is limited data available from Low- and Middle-Income Countries (LMICs) on elderly DLBCL. We analyzed the presentations and survival outcomes of patients with DLBCL according to their socioeconomic status.</div></div><div><h3>Methodology</h3><div>This was a multicenter retrospective study conducted from 2015 to 2023. We included 176 patients aged 60 years or older. The variables examined were age, gender, subtype, resource environment and treatment received. Kaplan-Meier curve was created for the entire patient cohort; t-test was utilized to compare means, Disease-Free Survival (DFS) and Overall Survival (OS), with a significance level of p &lt; 0.05. Analysis was performed using SPSS version 29.</div></div><div><h3>Results</h3><div>The median age was 66 years (range: 60–89 years). Ninety-three (57%) patients were treated in limited resource settings, while 43% had enhanced resources. ECOG performance scores between 2 and 3 were present in 71%. Median IPI score was 3. RCHOP regimen was administered to 51% (n = 81) patients, and CHOP regimen to 20% (n = 32) patients. In 21% (n = 38) salvage treatment was given due to relapsed/refractory disease. None of the patients in this group received consolidation with autologous stem cell transplant. The entire cohort’s OS was 12-months, while DFS was 8-months. OS (33.9% vs. 8.2%; p = 0.00) and DFS (29% vs. 5.9%; p = 0.00) were better in patients with enhanced resources. The median DFS of patients treated in enhanced settings was 1.3-years versus 0.4-years in limited resource settings (p &lt; 0.0001)</div></div><div><h3>Conclusion</h3><div>Survival rates were lower for patients receiving treatment in resource-limited settings. Outcomes can be improved with early referral and inclusion of Rituximab. Enhanced geriatric assessments along with better supportive care is essential.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103893"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534475","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":"ASSESSMENT OF INTERPHASE FLUORESCENCE IN SITU HYBRIDIZATION (FISH) TEST IN A PATIENT WITH MULTIPLE MYELOMA: EXPERIENCE OF OUR MEDICAL GENETICS DEPARTMENT","authors":"Fatma Turki ,&nbsp;Imen Rezgui ,&nbsp;Faten Kallel ,&nbsp;Moez Mdhaffer ,&nbsp;Hassen Kamoun ,&nbsp;Rim Frikha","doi":"10.1016/j.htct.2025.103904","DOIUrl":"10.1016/j.htct.2025.103904","url":null,"abstract":"<div><h3>Objective</h3><div>Multiple Myeloma (MM) is an orphan disorder of end stage plasma cells with acquired genetic abnormalities of clinical importance not captured by conventional cytogenetic analysis because of the low proliferation of malignant plasma cells. Thus, interphase Fluorescence In Situ Hybridization (FISH), performed on sorted plasma cells detected abnormalities independently of a proliferative and infiltrative index. The purpose of this study was to explore, for the first time in our Medical Genetics department the molecular genetics features in a Tunisian patient with multiple myeloma. A 35-year-old Tunisian man, followed-up for MM since two years and received VTD chemotherapy protocol (bortézomib, thalidomide et dexaméthasone). Actually, as part of evaluation of his disease, and in the presence of infectious syndrome, the MM’s relapse is suspected. Magnetic cell separation of PCs was performed using the Whole Blood CD138 MicroBeads, Whole Blood Column Kit, and the QuadroMACS Separation Unit (Miltenyi Biotec) according to the manufacturer's protocol. Slides were pretreated according to the manufacture's protocol. The FISH probes used in this study included IGH/FGFR3(4p16/ 14q32; DC.DF)/vysis, TP53/CEP 17(17p11.1-q11.1/ 17p13.1) FISH probe, Vysis.</div></div><div><h3>Results</h3><div>Revealed the presence of three signals of IGH in 75% of nuclei and one signal of TP53 in 96% of nuclei. These results demonstrated the deletion of the short arm of chromosome 17 (del(17p)) and the absence of t(4;14). However, the presence of three signals of IGH indicated either the IGH amplification or the IGH rearrangement involving other partner chromosomes. These results were consistent with patient’s relapse. The t(4;14) and del (17p) are high-risk markers associated with adverse prognosis. Patients with these genomic aberrations should be treated with targeted therapy. The detection of the 1q21 ‘gain could be considered in further studies because it is the most frequent structural abnormality, observed in 35%–40% of the patients with MM which is an independent poor prognostic factor.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103904"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534483","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":"DIFFUSE LARGE CELL LYMPHOMAS WITH RESTRICTED SOURCES","authors":"Suzana Krasniqi","doi":"10.1016/j.htct.2025.103865","DOIUrl":"10.1016/j.htct.2025.103865","url":null,"abstract":"<div><h3>Context</h3><div>Non-Hodgkin Lymphoma (NHL) encompasses a range of blood cancers originating in the lymphatic system, with Diffuse Large B-Cell Lymphoma (DLBCL) being the most prevalent type. In Kosovo, a country with limited healthcare resources, managing NHL remains a significant challenge. In 2024, Kosovo reported 60 cases of lymphoma, of which 53 were classified as NHL, and 24 of those were identified as DLBCL. The healthcare system faces numerous obstacles, particularly in providing specialized treatments such as Bone Marrow Transplantation (BMT), which is not available domestically. These limitations impact the diagnosis, management, and outcomes for DLBCL patients.</div></div><div><h3>Aim</h3><div>This study aims to explore the prevalence of DLBCL among NHL cases in Kosovo, identify the healthcare challenges posed by limited resources, and highlight the critical issue of the unavailability of bone marrow transplantation as part of lymphoma treatment.</div></div><div><h3>Methods</h3><div>Data from the Kosovo National Cancer Registry for 2024 shows that out of 60 lymphoma cases, 53 were NHL, and 24 were DLBCL. This retrospective study assesses the diagnostic, treatment, and follow-up data of these patients, focusing on challenges in managing DLBCL, especially the lack of bone marrow transplant services.</div></div><div><h3>Discussion</h3><div>Kosovo’s healthcare infrastructure is underdeveloped in terms of both diagnostic tools and treatment options for cancers like DLBCL. Early diagnosis, which is crucial for the successful treatment of DLBCL, is often delayed due to the lack of advanced imaging and molecular diagnostic techniques. Furthermore, chemotherapy regimens ‒ standard treatments for DLBCL ‒ are often delayed because of limited access to essential drugs, inadequate oncology training, and logistical issues in the healthcare system. A major issue is the absence of Bone Marrow Transplantation (BMT) services in Kosovo. BMT, a critical treatment for certain aggressive cases of DLBCL, is not available within the country, forcing patients to seek treatment abroad. This process is expensive, and many patients face financial barriers to accessing this life-saving procedure. For those who cannot afford treatment outside Kosovo, the lack of BMT options often leads to poorer outcomes, particularly for patients with relapsed or refractory DLBCL. Additionally, limited access to second-line treatments such as immunotherapy and targeted therapies exacerbates the situation. The healthcare system struggles with a shortage of specialized medical personnel and advanced cancer care facilities. The lack of access to BMT and modern therapies limits the treatment options available for patients who fail to respond to first-line chemotherapy, resulting in a worse prognosis.</div></div><div><h3>Conclusion</h3><div>Kosovo faces significant challenges in managing Diffuse Large B-Cell Lymphoma due to limited healthcare resources, particularly the unavai","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103865"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534551","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":"HOW I TREAT PH+ ACUTE LYMPHOBLASTIC LEUKEMIA","authors":"Robin Foà","doi":"10.1016/j.htct.2025.103869","DOIUrl":"10.1016/j.htct.2025.103869","url":null,"abstract":"<div><div>The greatest improvements in the management of Acute Lymphoblastic Leukemia (ALL) have been witnessed in Ph+ALL patients. The advancements have stemmed from an always more precise genetic characterization at presentation, the use of tailored treatment, the precise monitoring of minima/Measurable Residual Disease (MRD) and, finally, by the inclusion of immunotherapy in the frontline treatment. Prior to the advent of Tyrosine Kinase Inhibitors (TKIs), Ph+ALL was the hematologic malignancy with the worse outcome. The frontline use of TKIs has changed the natural history of the disease. Since year 2000 in Italy all patients enrolled in the GIMEMA multicenter protocols have been treated in induction with a TKI alone (plus steroids) and no systemic chemotherapy. The subsequent advancement has been brought by the addition of the bispecific monoclonal antibody blinatumomab as consolidation, always in the absence of systemic chemotherapy. The results of the GIMEMA LAL2116 (D-ALBA) trial for patients of all ages showed high rates of molecular response following an induction/consolidation treatment with dasatinib and blinatumomab. At 53-months, survival rates of 75%‒80% were recorded, with 50% of patients being managed only with a TKI and blinatumomab, without chemotherapy and transplant. Most MRD+ patients were allografted. IKZF-plus patients have a less favorable outcome and should be identified at diagnosis. When possible, they should undergo an allogeneic transplant. In the subsequent phase 3 GIMEMA ALL2820 trial, patients enrolled in the experimental arm and treated with ponatinib followed by blinatumomab showed even higher rates of molecular response, with estimated OS and DFS of 94.9% and 95.6% at 12-months. Of interest, the combination of dasatinib and ponatinib plus blinatumomab, in the absence of systemic chemotherapy, is associated with a marked host immune activation. The MDACC group also reported the effectiveness of ponatinib combined with blinatumomab, though the combination was associated with greater toxicity. For a review on the treatment of adult Ph+ALL see Chiaretti &amp; Foà. The GIMEMA ALL2820 trial will conclusively show how many patients can be spared systemic chemotherapy and transplant. At the interim analysis, only 10% of patients enrolled in the ponatinib + blinatumomab arm have so far undergone a transplant. I have been asked to cover ‘How I Treat Ph+ALL’, which more appropriately should be ‘How Should I Treat Ph+ LL’ Based on the 25-year experience gathered through the GIMEMA trials, the optimal algorithm should be: i) Identify the presence of the BCR/ABL gene lesion within one week from diagnosis; ii) During this time treat patients with steroids; iii) Start induction with dasatinib or ponatinib plus steroids, with no systemic chemotherapy; iv) CNS prophylaxis should be carried out; v) MRD should be monitored molecularly at given timepoints; vi) After induction, all patients should be consolidated with multiple cycle","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103869"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534610","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":"INVESTIGATION OF POSTURAL CONTROL IN CHILDREN WITH ACUTE LYMPHOBLASTIC LEUKEMIA","authors":"Fulya Ipek-Erdem ,&nbsp;Sena Sonkaya ,&nbsp;Arzu Genç ,&nbsp;Şebnem Yılmaz","doi":"10.1016/j.htct.2025.103884","DOIUrl":"10.1016/j.htct.2025.103884","url":null,"abstract":"<div><h3>Objective</h3><div>Children with leukemia may face balance impairments due to somatosensory, motor, muscular, and cognitive deficits that can persist into adulthood and increase fall risk. This study aimed to evaluate postural control in children with Acute Lymphoblastic Leukemia (ALL) undergoing consolidation therapy by comparing their performance with normative data to identify potential treatment-related impairments in sensory integration and balance.</div></div><div><h3>Methodology</h3><div>Thirteen children with ALL were recruited at Dokuz Eylül University, Faculty of Physiotherapy and Rehabilitation in Turkey, and divided into two age groups: 6–7 years (n = 9) and 8–9 years (n = 4). Static balance was evaluated using the modified Clinical Test for Sensory Interaction on Balance (mCTSIB) with the Balance Master system. The test assessed postural control under four conditions: Eyes Open-firm surface (FirmEO), Eyes Closed-firm surface (FirmEC), eyes open-unstable (foam) surface (FoamEO), and eyes closed-unstable (foam) surface (FoamEC). The center of gravity's average sway speed (°/s) was measured for each condition, with higher values indicating reduced balance capability. Normative data for each condition were obtained from previous studies on healthy children.</div></div><div><h3>Results</h3><div>In the 6–7 years group, sway speeds during FirmEO and FirmEC were 0.92 s and 0.97 s, respectively, compared to norms of 0.70s and 0.92s. Under foam conditions, FoamEO reached 1.31s (norm: 1.20s), while FoamEC was 1.81s, nearly identical to the normative 1.80s. In the 8–9 years group, FirmEO was 0.55s (norm: 0.40s) and FirmEC was 0.65s (norm: 0.53s). FoamEO measured 0.82s (norm: 0.89s), whereas FoamEC was 1.70s (norm: 1.47s). Overall, these results suggest that children with ALL generally exhibit elevated sway speeds ‒ particularly under firm conditions ‒ implying impaired postural control and potential challenges in sensory integration.</div></div><div><h3>Conclusion</h3><div>Our findings demonstrate that postural control is compromised in children with ALL undergoing consolidation therapy. Elevated sway speeds on firm surfaces suggest diminished balance performance, while the mixed results on foam conditions highlight difficulties with sensory integration. These preliminary observations underscore the need for targeted interventions and further research with larger samples to clarify the mechanisms behind these deficits.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103884"},"PeriodicalIF":1.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144534303","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}