Journal of hematologyPub Date : 2025-08-25eCollection Date: 2025-08-01DOI: 10.14740/jh2092
Amr Hanbali, Ahmed Kotb, Mostafa Saleh
{"title":"B-Cell Lymphoma 2 Inhibition in Acute Lymphoblastic Leukemia: Mechanisms, Resistance, and Emerging Combinations With Venetoclax.","authors":"Amr Hanbali, Ahmed Kotb, Mostafa Saleh","doi":"10.14740/jh2092","DOIUrl":"10.14740/jh2092","url":null,"abstract":"<p><p>Recent studies show that the B-cell lymphoma 2 (BCL-2) inhibitor venetoclax is a promising therapeutic drug for acute lymphoblastic leukemia (ALL), especially in high-risk subtypes including early T-cell precursor (ETP)-ALL, Philadelphia chromosome (Ph)-like B-cell ALL, and KMT2A-rearranged leukemia. The preclinical and early-phase clinical research shows that venetoclax-based combinations can increase apoptosis and improve response rates when used with chemotherapy or hypomethylating agents. The main challenge to venetoclax efficacy remains the resistance mechanisms that primarily involve myeloid cell leukemia-1 (MCL-1) and BCL-extra large (XL). This review provides an overview of the current state of venetoclax in ALL by discussing its mechanistic basis and clinical trial results as well as safety data and strategies to overcome resistance and enhance venetoclax-based treatments.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 4","pages":"193-201"},"PeriodicalIF":1.3,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12404105/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992617","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}
Journal of hematologyPub Date : 2025-08-25eCollection Date: 2025-08-01DOI: 10.14740/jh2103
Rowena Lee Ying Kwan, Ryan Mao Heng Lim, Jason Yongsheng Chan
{"title":"Clinical Patterns and Prognostic Outcomes of Asian Ocular Adnexal Marginal Zone Lymphoma.","authors":"Rowena Lee Ying Kwan, Ryan Mao Heng Lim, Jason Yongsheng Chan","doi":"10.14740/jh2103","DOIUrl":"10.14740/jh2103","url":null,"abstract":"<p><strong>Background: </strong>Ocular adnexal marginal zone lymphoma (OAMZL) is the most common subtype of primary ocular lymphoma and has been rising in incidence in Asian populations.</p><p><strong>Methods: </strong>We conducted a retrospective review of 95 patients diagnosed with OAMZL within a multi-ethnic cohort from Singapore. Clinical characteristics, survival outcomes including overall survival (OS) and progression-free survival (PFS), and maximum standardized uptake value (SUVmax) on staging F-18 fluorodeoxyglucose positron emission tomography/computed tomography (18-FDG-PET/CT) were investigated.</p><p><strong>Results: </strong>The cohort comprised 60 males and 35 females, with a median age of 58 years (25 - 88). Median follow-up was 92 months. The most common sites involved were the orbit (49.5%) and lacrimal gland (23.2%). Most patients presented with stage 1 disease (72.6%). Five-year OS and PFS for the whole cohort were 94.9% and 84.1%, respectively. Factors significantly associated with poorer OS included advanced (stage 2-4) disease (hazard ratio (HR) 6.26, 95% confidence interval (CI): 1.69 - 23.19, P = 0.0061), older age above 58 years (HR = 15.29, 95% CI: 4.47 - 52.3, P < 0.0001), and higher mucosa-associated lymphoid tissue International Prognostic Index (MALT-IPI) scores of 2 - 3 compared to low (0) and intermediate (1) scores (HR = 9.28, 95% CI: 1.24 - 69.11, P < 0.0001 and HR = 10.99, 95% CI: 1.34 - 89.94, P < 0.0001), respectively. Older age (HR = 2.41, 95% CI: 1.07 - 5.43, P = 0.0330) and advanced disease (HR = 2.47, 95% CI: 1.07 - 7.03, P = 0.0348) were significantly associated with poorer PFS. Median SUVmax of the lesions was 5.6 (2.1 - 9.6), with significantly higher values in advanced disease.</p><p><strong>Conclusions: </strong>Our study illustrates the favorable prognosis of OAMZL in an Asian cohort, although particular factors may portend worse survival outcomes.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 4","pages":"223-233"},"PeriodicalIF":1.3,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12404121/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992657","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}
Journal of hematologyPub Date : 2025-08-25eCollection Date: 2025-08-01DOI: 10.14740/jh2104
Muralidhar Idamakanti, Ala Ebaid, Rani Indrani Bijjam, Alexei Bakhirev
{"title":"Mast Cell Leukemia: Comprehensive Review of Literature With Current Insights and Updates on Management.","authors":"Muralidhar Idamakanti, Ala Ebaid, Rani Indrani Bijjam, Alexei Bakhirev","doi":"10.14740/jh2104","DOIUrl":"10.14740/jh2104","url":null,"abstract":"<p><p>Mast cell leukemia (MCL) is an exceedingly rare and aggressive variant of systemic mastocytosis (SM). MCL is classified as primary, occurring <i>de novo</i> without prior mast cell (MC) disorders or secondary, from a pre-existing SM, and acute aggressive form with C-findings that indicate organ damage or chronic indolent form without organ damage. Of the cases, 60-65% are aleukemic with < 10% circulating MCs in the peripheral blood, and the rest of the cases are leukemic with > 10% MCs. Diagnosis is typically confirmed by bone marrow biopsy revealing greater than 20% atypical or immature MCs in the smear. Specific MCL-targeted treatments are limited, and multiple treatment modalities used for SM and acute myeloid leukemia (AML) have been tried in MCL with limited success and variable survival benefit. The management has significantly advanced since the implication of the receptor tyrosine kinase type III (KIT) D816V mutation in the pathogenesis of SM and MCL. The two targeted therapies approved for MCL are midostaurin, a multikinase inhibitor, and avapritinib, a selective KIT D816V mutation-targeted tyrosine kinase inhibitor. Multiple drugs are being evaluated in clinical trials for managing MCL. MCL has a poor prognosis with a median overall survival (OS) of around 1.5 years. Further advancements and research are essential to develop treatments that may enhance median OS. In this article, we conducted a comprehensive yet simplified review of MCL, focusing primarily on its clinical manifestations and recent updates on management. We also identified the areas that require further research and emphasized the aggressive nature and poor prognosis associated with this disease.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 4","pages":"174-192"},"PeriodicalIF":1.3,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12404107/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992664","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}
Journal of hematologyPub Date : 2025-08-25eCollection Date: 2025-08-01DOI: 10.14740/jh2094
Jowan Al-Nusair, Mo'men Aldalal'ah, Mohammad Alqudah, Fakhri Al-Malkawi, Nora Al-Khateeb, Hasan Khasawneh, Omar El-Shatel, Obada Khayyat, Sarah H Alseid, Mahmoud Abdallah, Ola Soudah, Toni Pacioles, Muhammad Omer Jamil
{"title":"Efficacy and Safety of Momelotinib in Myelofibrosis: A Systematic Review and Meta-Analysis With a Focus on Anemia Outcomes.","authors":"Jowan Al-Nusair, Mo'men Aldalal'ah, Mohammad Alqudah, Fakhri Al-Malkawi, Nora Al-Khateeb, Hasan Khasawneh, Omar El-Shatel, Obada Khayyat, Sarah H Alseid, Mahmoud Abdallah, Ola Soudah, Toni Pacioles, Muhammad Omer Jamil","doi":"10.14740/jh2094","DOIUrl":"10.14740/jh2094","url":null,"abstract":"<p><strong>Background: </strong>Myelofibrosis (MF) can be primary (PMF) or secondary (SMF), with PMF driven by Janus kinases-signal transducer and activator of transcription proteins (JAK-STAT) pathway activation due to Janus kinase 2 (<i>JAK2</i>), the thrombopoietin receptor gene (myeloproliferative leukemia virus oncogene (<i>MPL</i>)), or calreticulin (<i>CALR</i>) mutations. Nearly 50% of PMF patients experience anemia (hemoglobin (Hb) < 10 g/dL), often worsened by JAK inhibitors like ruxolitinib and fedratinib. Momelotinib, an oral ACVR1, JAK1, and JAK2 inhibitor, improves anemia, symptoms, and splenomegaly, likely through hepcidin regulation. This review evaluates its efficacy and safety, with a focus on anemia.</p><p><strong>Methods: </strong>A systematic review and meta-analysis were conducted following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, including randomized controlled trials (RCTs) and clinical studies assessing momelotinib's efficacy and safety. Primary outcomes included spleen volume reduction (≥ 35%) and anemia response (transfusion independence). Secondary endpoints included symptom burden reduction and safety.</p><p><strong>Results: </strong>Six studies, including three RCTs, met inclusion criteria. Meta-analysis showed momelotinib was noninferior to ruxolitinib in spleen volume reduction but superior in anemia benefits, increasing transfusion independence (odds ratio (OR): 2.09; 95% confidence interval (CI): 1.53 - 2.85) and reducing transfusion dependence (OR: 0.62; 95% CI: 0.45 - 0.84). Symptom burden reduction was comparable to other JAK inhibitors. Common adverse events included dizziness (OR: 1.70; 95% CI: 1.05 - 2.74) and nausea (OR: 3.07; 95% CI: 1.82 - 5.18), with no significant increase in serious adverse events.</p><p><strong>Conclusions: </strong>Momelotinib improved anemia-related outcomes and quality of life in MF without increased adverse events. However, heterogeneity in control groups limited direct efficacy comparisons. Larger studies are needed to confirm its effectiveness and safety.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 4","pages":"214-222"},"PeriodicalIF":1.3,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12404109/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992653","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}
Journal of hematologyPub Date : 2025-08-07eCollection Date: 2025-08-01DOI: 10.14740/jh2078
Amer Al Homssi, Ryan Lokkesmoe, Ryan Powers, Benjamin Jung, Farheen Chunara, Aniko Szabo, Lisa Baumann Kreuziger
{"title":"Fixed-Dose Four-Factor Prothrombin Complex Concentrate for Reversal of Anticoagulation: Evaluation of Efficacy, Safety, and Cost Savings.","authors":"Amer Al Homssi, Ryan Lokkesmoe, Ryan Powers, Benjamin Jung, Farheen Chunara, Aniko Szabo, Lisa Baumann Kreuziger","doi":"10.14740/jh2078","DOIUrl":"10.14740/jh2078","url":null,"abstract":"<p><strong>Background: </strong>Four-factor prothrombin complex concentrate (4F-PCC) is used for warfarin reversal and off-label management of bleeding in patients taking direct oral anticoagulants (DOACs). Dosing strategies that optimize hemostatic efficacy and cost, such as fixed dosing of 4F-PCC, are still under evaluation. The objective of this study was to retrospectively evaluate the efficacy, safety, and cost savings of fixed-dosing of 4F-PCC (1,500 IU for warfarin, 2,000 IU for DOACs).</p><p><strong>Methods: </strong>Patients records from October 1, 2018, to April 30, 2021, at three hospitals within the Froedtert Health System were retrospectively reviewed for individuals who received fixed-dosing of 4F-PCC. Safety and efficacy were reflected in 30-day bleeding and thrombosis events, the need for repeat doses, and all-cause mortality. Cost savings were defined as the difference in the cost between the administration of fixed-dosing and the projected weight- and international normalized ratio (INR)-based dosing based on the package insert for warfarin reversal or 50 IU/kg in patients treated with DOACs.</p><p><strong>Results: </strong>A total of 592 patients received fixed-dosing of 4F-PCC during the prespecified period, of whom 541 received it for warfarin reversal (n = 414) or DOACs (n = 127) management in emergency settings. INR below 2 was achieved in 89% of patients on warfarin. Less than 5% in either group required repeat doses of 4F-PCC. Within 30 days, both groups had similar bleeding (12%) and thrombotic (5%) events. All-cause 30-day mortality rates in patients treated with warfarin and DOACs were 24% and 30%, respectively. The median cost savings of fixed-dosing per patient on warfarin and DOACs were $1,567 and $3,936, respectively, with annualized median hospital cost savings of $176,239 and $146,733, respectively.</p><p><strong>Conclusions: </strong>Fixed-dosing of 4F-PCC had significantly less cost than adjusted dose and is associated with similar rates of thrombosis and death compared to other studies.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 4","pages":"234-239"},"PeriodicalIF":1.3,"publicationDate":"2025-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12404115/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992671","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}
Journal of hematologyPub Date : 2025-07-08eCollection Date: 2025-08-01DOI: 10.14740/jh2086
Kristina Sevcik, Claire Jackson, Shelly M Williams, Scott A Koepsell, Aleh Bobr
{"title":"Novel Red Blood Cell Exchange Parameters for Treatment of Transfusion-Dependent Thalassemia Based on Experience of Five Patients.","authors":"Kristina Sevcik, Claire Jackson, Shelly M Williams, Scott A Koepsell, Aleh Bobr","doi":"10.14740/jh2086","DOIUrl":"10.14740/jh2086","url":null,"abstract":"<p><strong>Background: </strong>Thalassemias are inherited red blood cell disorders characterized by defective globin production, resulting in microcytic hypochromic anemia. Severe variants lead to transfusion dependence and consequent iron overload, often despite chelation therapy. The role of automated red blood cell exchange (RBCX) for transfusion-dependent thalassemia (TDT) is unclear and previously there was no specific apheresis parameters specific for thalassemia defined. We present our experience with patients with TDT treated with RBCX using higher hematocrit parameters specifically tailored for this condition.</p><p><strong>Methods: </strong>Five patients with TDT underwent chronic RBCX in place of simple transfusion with the primary goal of stabilizing iron overload. Novel parameters were established to satisfy the Thalassemia International Federation goal pre-transfusion hemoglobin of 9.5 g/dL, while targeting a post-transfusion hematocrit of 37-38%.</p><p><strong>Results: </strong>RBCX was well tolerated with infrequent occurrences of vasovagal reactions, asymptomatic hypotension, citrate side effects, and vascular access issues. The transfusion interval increased from an average of 3 weeks with simple transfusions to 5 weeks with RBCX. Despite an increase in average blood utilization, serum ferritin levels remained stable.</p><p><strong>Conclusion: </strong>RBCX with higher hematocrit parameters can be performed safely and efficiently in TDT patients. To our knowledge, this is the first report of TDT-specific RBCX parameters. Though blood utilization is higher with RBCX, it offers longer intervals between transfusions and has no increase in iron overload, improving quality of life for patients.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 4","pages":"210-213"},"PeriodicalIF":1.3,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12404126/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992651","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":"Does Time Matter From Diagnosis to Induction in Acute Myeloid Leukemia?","authors":"Osama Batayneh, Deevyashali Parekh, Margarita Vazquez Almonte, Cristina Hamacher, Angela Gupta, Danielle Passafiume, Michael Sandhu, Carina Hernandez, Safa Afridi, Sanjay Rao Gergal GopalakrishnaRao, Alyssa Cortese, Alexandra Goodman, Kelsey Christo, Josh Wallace, Teresa Gentile","doi":"10.14740/jh2075","DOIUrl":"10.14740/jh2075","url":null,"abstract":"<p><strong>Background: </strong>While molecular and cytogenetic testing may change prognosis and guide treatment intensity for patients with acute myeloid leukemia (AML), timing from diagnosis to treatment (TDT) on the other hand may impact treatment outcomes and survival. These considerations are sometimes at odds with each other given that molecular studies can take up to 2 weeks to result.</p><p><strong>Methods: </strong>A retrospective cohort analysis was conducted at SUNY Upstate University Hospital to examine the effect of TDT on complete remission (CR) and overall survival (OS). The subjects were at least 18 years old and diagnosed with AML and treated between January 2010 and June 2024. TDT was divided into three categories: chemotherapy induction within 1 - 5, 6 - 10, and 11+ days. Univariate Kaplan-Myer survival analysis and multivariate Cox regression model were performed.</p><p><strong>Results: </strong>A total of 187 patients were included, and 34% (n = 64) were younger than age 60. Patients were classified as 20% (n = 37) favorable, 36% (n = 67) intermediate, and 40% (n = 74) adverse risk, , while 4% risk stratification could not be completed due to missing data. Seventy-two percent (n = 134) had <i>de novo</i> AML. Chemotherapy induction began for 70% (n = 130) on days 1 - 5, 16% (n = 30) between days 6 - 10, and 14% (n = 27) on day 11 or after. The probability of achieving CR decreases for those who had induction 11+ days from diagnosis compared to those who had induction 1 to 5 days from diagnosis. This relationship was statistically significant (odds ratio = 0.32, 95% confidence interval (CI): 0.125 - 0.796; P = 0.003). However, no differences in OS and CR between TDT groups were seen when multivariate analysis was performed.</p><p><strong>Conclusion: </strong>Our retrospective study showed no difference in OS based on TDT groups, which supports clinicians' approach to await on comprehensive AML profiling for an optimal risk stratification at diagnosis and implementing best course of action.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 4","pages":"202-209"},"PeriodicalIF":1.3,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12404120/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992605","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}
Journal of hematologyPub Date : 2025-07-08eCollection Date: 2025-08-01DOI: 10.14740/jh2064
Jackson M Wahman, Rhoda X Hijazi, Elizabeth Duncan, Petra Rocic, Dominica Moussoki, Hosam G Abdelhady
{"title":"Beyond the Blood Cell: The Emerging Role of Cell-Free DNA in Transfusion Medicine.","authors":"Jackson M Wahman, Rhoda X Hijazi, Elizabeth Duncan, Petra Rocic, Dominica Moussoki, Hosam G Abdelhady","doi":"10.14740/jh2064","DOIUrl":"10.14740/jh2064","url":null,"abstract":"<p><p>Cell-free DNA (cfDNA) consists of fragmented nuclear and mitochondrial DNA circulating in the bloodstream, primarily originating from hematopoietic cells. While cfDNA analysis has transformed diagnostic medicine, its presence in transfused blood products introduces emerging clinical concerns. Donor-derived cfDNA may persist in transfusion recipients and contribute to transfusion-associated microchimerism, defined as the long-term presence of donor genetic material in recipient tissues or circulation. These fragments have potential to integrate into the host genome, modify DNA methylation and histone structure, and activate innate immune pathways such as Toll-like receptors. In addition to nuclear and mitochondrial sources, cfDNA in transfused blood may include environmental or dietary DNA acquired by donors, further influencing immune regulation. Current leukoreduction methods do not eliminate cfDNA or prevent microchimerism. This review synthesizes current evidence regarding the persistence, genomic integration, and immunologic impact of cfDNA in transfusion recipients. The findings highlight an urgent need for further investigation and refinement of blood processing practices to ensure transfusion safety and protect recipient health.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 4","pages":"165-173"},"PeriodicalIF":1.3,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12404111/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992676","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 of Short-Course High-Dose Oral Prednisolone in Rapid Platelet Recovery for Pediatric Acute Immune Thrombocytopenic Purpura: A Prospective Cohort Study.","authors":"Akshat Jhingan, Neha Goel, Amitabh Singh, Rani Gera, Nidhi Chopra, Sumit Mehndiratta, Monica Sharma","doi":"10.14740/jh2052","DOIUrl":"10.14740/jh2052","url":null,"abstract":"<p><strong>Background: </strong>Standard management of acute immune thrombocytopenic purpura (ITP) remains debated, with some advocating observation for mild cases, while others recommend pharmacological intervention to accelerate platelet recovery in children with severe thrombocytopenia (TCP) (platelet count < 20,000/mm<sup>3</sup>) or significant mucosal bleeds. Corticosteroids, particularly prednisolone (PDN), are a widely used first-line treatment due to their rapid immunosuppressive effect. This prospective cohort study evaluated the effectiveness of short-course high-dose PDN (4 mg/kg/day for 7 days) in treating children aged 1 - 12 years with newly diagnosed acute ITP. The study aimed to assess the clinical and hematological profile of these children and the mean time to platelet recovery.</p><p><strong>Methods: </strong>A total of 61 patients were enrolled, and their response to treatment was monitored at various intervals, including 48 h, 72 h, day 7, 1 month, and 3 months.</p><p><strong>Results: </strong>Results revealed a rapid platelet recovery in patients receiving high-dose PDN, with 83.6% of patients achieving platelet counts greater than 50,000/mm<sup>3</sup> by day 7. Additionally, 91.8% maintained platelet recovery at 1 month. The study also found that the age group 6 - 12 years had a higher risk of persistent ITP (24.5%), highlighting the importance of close monitoring in this demographic. While the treatment was generally well tolerated, some mild side effects like hypertension and glycosuria were observed.</p><p><strong>Conclusion: </strong>The study suggests that short-course high-dose PDN can be an effective first-line therapy for acute ITP. It promotes faster platelet recovery and reduces hospitalization time with minimal adverse effects.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 3","pages":"133-138"},"PeriodicalIF":1.3,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12151098/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144275090","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}