Acta Haematologica最新文献

{"title":"Beyond Cellular Therapies: The Expanding Role of Antibody-Driven Immunotherapy in Pediatric Acute Lymphoblastic Leukemia.","authors":"David McCall, Samanta Catueno, Ramya Ramakrishnan, Priti Tewari, Irtiza Sheikh, Amber Gibson, Cesar A Nuñez, Miriam B Garcia, Branko Cuglievan","doi":"10.1159/000546249","DOIUrl":"10.1159/000546249","url":null,"abstract":"<p><strong>Background: </strong>The integration of novel antibody-mediated targeted therapies into both relapsed/refractory (R/R) and frontline pediatric acute lymphoblastic leukemia (ALL) treatment protocols has led to critical advancements in the field. Current research efforts focus on optimizing targeted therapies to enhance precision and efficacy while minimizing toxicity by reducing chemotherapy. A notable example is the addition of blinatumomab, demonstrating superiority over conventional chemotherapy, with an 8% increase in disease-free survival at an interim analysis, reaching 96%. Inotuzumab ozogamicin (InO) has also shown promise, achieving nearly a 70% complete response rate in pediatric R/R B-cell ALL (B-ALL) trials. Additionally, daratumumab in T-cell ALL (T-ALL) and chimeric antigen receptor T-cell therapies, particularly CD19-directed (B-ALL) and CD7-directed (T-ALL) strategies, are under active investigation.</p><p><strong>Summary: </strong>This review will provide an overview of targeted antibody-mediated immunotherapies in both B-ALL and T-ALL, with a focus on their pediatric applications, supporting data, and future prospects.</p><p><strong>Key messages: </strong>The next cycle of frontline trials in pediatric ALL will incorporate more immunotherapy with reduction of chemotherapy. Subsequent trials will utilize more concurrent chemoimmunotherapy blocks as precision testing and risk-adapted therapy will continue to develop. These advancements reflect a paradigm shift toward more precise, less toxic treatment strategies in pediatric ALL.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-16"},"PeriodicalIF":1.7,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143956843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Favorable Rates of Cardiovascular Events with Stringent Cardiovascular Monitoring and a Lowered Dose of Ponatinib as Second-Line Treatment in Chronic Phase Chronic Myeloid Leukemia Patients Failing or Intolerant to First-Line Second-Generation Tyrosine Kinase Inhibitor Treatment: Results of the Prospective PONS Trial.","authors":"Philipp le Coutre, Andreas Burchert, Susanne Saußele, Thekla Schwarzer, Sebastian Stintzing, Uwe Pelzer, Lars Bullinger, Ahmet Elmaagacli, Christian Jehn, Frank Stegelmann, Andreas Hochhaus, Thomas Ernst, Joachim R Göthert","doi":"10.1159/000545826","DOIUrl":"10.1159/000545826","url":null,"abstract":"<p><strong>Introduction: </strong>In chronic myeloid leukemia patients, second-line treatment requires careful consideration of response and tolerability. As most patients need a more efficient tyrosine kinase inhibitor, ponatinib at a lowered dose should be evaluated in this setting.</p><p><strong>Methods: </strong>We studied a lowered dose of 30 mg ponatinib in second line in patients selected toward a low cardiovascular risk. In 22 screened patients, ponatinib was started in 18 patients previously treated with imatinib (n = 3), dasatinib (n = 9), or nilotinib (n = 6). Patients were frequently monitored for cardiovascular toxicities by testing of blood pressure, vital signs, ankle brachial index or duplex, oral glucose tolerance test, echocardiography, ECG, and fundoscopy. The study protocol allowed dose reductions in patients achieving MMR. Both previously resistant or intolerant patients were recruited.</p><p><strong>Results: </strong>No serious cardiovascular events were observed, and low-grade cardiovascular toxicity was negligible. By 12 months, 13 patients (92.9%) were in complete hematologic remission, 10 patients (55.6%) were in MMR, and 5 patients (27.8%) were in MR4. Most importantly, we demonstrated that thorough monitoring of cardiovascular risk is feasible.</p><p><strong>Conclusions: </strong>We demonstrated that a lowered dose of 30 mg ponatinib in selected patients can be maintained without serious cardiovascular complications, provided cardiovascular risk monitoring is performed. In our patient cohort, this approach resulted in favorable response rates.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-8"},"PeriodicalIF":1.7,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143958017","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Dose Reduced Induction Therapy (A)-AIDA in Patients with Acute Myeloid Leukemia, Being Unfit for Standard 7+3 Chemotherapy and Literature Review of Acute Myeloid Leukemia Treatment in Unfit/Older AML Patients.","authors":"Karin Mayer, Leonie Birk, Katjana Schwab, Ingo G H Schmidt-Wolf, Marie von Lilienfeld-Toal, Peter Brossart, Axel Glasmacher, Corinna Hahn-Ast","doi":"10.1159/000545792","DOIUrl":"10.1159/000545792","url":null,"abstract":"<p><strong>Introduction: </strong>The standard induction chemotherapy in newly diagnosed acute myeloid leukemia (AML) is 7 days cytarabine and 3 days daunorubicin. However, older and frail patients cannot be treated intensively. Before hypomethylating agents (HMA) plus venetoclax (VEN) was established we formulated a more tolerable induction therapy for these patients using continuous infusions of reduced doses of cytarabine (A) and idarubicin (IDA) in combination with ATRA (A), the (A)-AIDA regimen and trying to reach noteworthy and relevant remission rates with acceptable toxicities in this special population.</p><p><strong>Methods: </strong>Between 1998 and 2015 older/frail patients with newly diagnosed or relapsed AML received (A)-AIDA. Treatment consisted of cytarabine 100 mg/m2/d d1-5 and IDA 5 mg/m2/d d1-5 as continuous infusion. Since 2003 ATRA (45 mg/m2/d d4-6, 15 mg/m2/d d7-28) was added in some patients.</p><p><strong>Results: </strong>154 patients received (A)-AIDA, median age was 71 years. In 40% the AML was secondary, 15.6% had relapsed AML. The complete remission rate was 41%, 7% reached a partial remission, the median overall survival was 7 months. No significant differences of remission rates regarding various known risk factors for outcome with (A)-AIDA were detected.</p><p><strong>Conlusion: </strong>Recent studies established HMA/VEN as first-line treatment for older/frail AML patients. Because (A)-AIDA is equally effective in patients with secondary or relapsed AML or ECOG >1, this regimen is an interesting treatment option for patients uneligible to HMA/VEN regimens.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-13"},"PeriodicalIF":1.7,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143954978","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Emicizumab and Acquired Hemophilia A Secondary to Multiple Myeloma.","authors":"Florence Cuschera, Marie-Christiane Vekemans, Cedric Hermans","doi":"10.1159/000545442","DOIUrl":"10.1159/000545442","url":null,"abstract":"<p><strong>Introduction: </strong>Acquired hemophilia A (AHA) is a severe bleeding disorder, sometimes linked to plasma cell dyscrasias. In this context, emicizumab, a bispecific antibody, provides stable hemostasis by mimicking factor VIII (FVIII), offering convenience and flexibility compared to bypassing agents. Moreover, targeted anti-myeloma therapy directly addresses the underlying plasma cell disorder, potentially achieving better and more durable control of AHA than conventional immunosuppressive therapy, while reducing its associated adverse effects.</p><p><strong>Case presentation: </strong>We describe the successful management of AHA secondary to multiple myeloma (MM) using emicizumab and targeted anti-myeloma therapy. The patient initially responded to bortezomib-dexamethasone but required teclistamab due to disease progression. Emicizumab maintained hemostatic stability, allowing time for effective MM management.</p><p><strong>Conclusion: </strong>Emicizumab, in conjunction with targeted myeloma treatment, represents a promising strategy to improve outcomes for AHA patients with MM. This approach is particularly advantageous for older patients with multiple comorbidities, who face elevated risks of thrombotic, bleeding, and infectious complications.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-7"},"PeriodicalIF":1.7,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143959199","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Concordance between Tympanic and Enteral Temperature for Early Fever Detection in Hematologic Intensive Care Unit Patients: A Prospective Study.","authors":"Florian Bouclet, Juliette Pénichoux, Sorina-Dana Mihailescu, Stéphanie Seris, Lucie Burel, Doriane Richard, Sabine Collard, Emmanuelle Jennet, Elisabeth Bertrand, Mustafa Alani, Sydney Dubois, Anne-Lise Ménard, Vincent Camus, Aspasia Stamatoullas, Pascal Lenain, Nathalie Contentin, Stéphane Leprêtre, Hervé Tilly, Emilie Lemasle, Hélène Lanic, Mikael Daouphars, Louis-Ferdinand Pépin, Sébastien Moussay, Fabrice Jardin","doi":"10.1159/000544693","DOIUrl":"10.1159/000544693","url":null,"abstract":"<p><strong>Introduction: </strong>Early detection of fever is crucial in neutropenic patients. Lack of precision in body temperature measurement can occur with peripheral site measurements. Furthermore, intermittent temperature monitoring may result in a delay in fever detection.</p><p><strong>Methods: </strong>We conducted a prospective study in hematologic intensive care unit (HICU) patients receiving autologous stem cell transplant or intensive chemotherapy in order to compare tympanic and enteral temperature measured by an ingested electronic pill (BodyCap®, France).</p><p><strong>Results: </strong>Twenty-six patients ingested at least one capsule with a total of 218 days of tympanic and enteral simultaneous measurement. In 12% of these measurements, we identified a difference over 0.5°C between the left ear versus right ear measurements. Enteral temperature was usually higher than the tympanic one (p < 0.0001) with 14% of the measurements that were discordant with higher enteral temperature and 1.9% that were discordant with higher tympanic temperature. Febrile episodes were detected with the ingestible pill on average almost 24 h earlier than with the tympanic measurement. Early capsule evacuation occurred in 20.4% of the cases, mainly because of diarrhea, a frequent adverse event in these patients. Patients were satisfied with capsule ingestion according to a questionnaire.</p><p><strong>Conclusion: </strong>In this prospective trial (TEMPET), we demonstrated that enteral temperature measured by an ingested pill is feasible in HICU and detects fever earlier than tympanic routine measurements. Digestive symptoms related to hematological disease and/or treatments are limiting factors for its generalized usage.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-12"},"PeriodicalIF":1.7,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143957940","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Epiglottitis in Patients Treated for Acute Leukemia: Case Series and Systematic Review of the Literature.","authors":"Adi Sherban, Avraham Frisch, Alon Rozenthal, Shira Buchrits, Baha Atamna, Daniel Ben-Ner, Sivan Eden, Ala Atamna, Yishai Ofran, Pia Raanani, Ofir Wolach","doi":"10.1159/000545927","DOIUrl":"10.1159/000545927","url":null,"abstract":"<p><strong>Introduction: </strong>Acute epiglottitis is a medical emergency characterized by inflammation of supraglottic structures of the larynx and epiglottis. The clinical characteristics and course of this complication in acute leukemia (AL) are largely unknown. We present a case series and a systematic review of the literature of adult patients with AL and acute epiglottitis.</p><p><strong>Case presentation: </strong>Four patients with acute myeloid leukemia (age 38-61 years) were diagnosed with acute epiglottitis during their intensive treatment course. One patient presented with epiglottitis at the end of induction, two at the end of consolidation therapy, and 1 patient presented with a later event. Three patients were in grade 4 neutropenia during infection onset (median aplasia duration to event 9 [range 4-14] days). All patients presented with neck pain and dysphagia and 2 patients also had dyspnea at presentation. Urgent invasive airway protection was required in 3 patients. Two patients died due to this complication, one suffered anoxic brain injury. A systematic literature review identified 8 additional cases of epiglottitis.</p><p><strong>Conclusion: </strong>Acute epiglottis is a rare but significant medical emergency with unique challenges in the setting of AL that is caused by atypical infectious pathogens. Early detection and a multidisciplinary therapeutic effort are crucial to improve patient outcome.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":1.7,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12240564/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144054035","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Tucidinostat Combined with Bortezomib, Liposomal Doxorubicin, and Dexamethasone in Multiple Myeloma Treatment.","authors":"Jingyi Bi, Xuelin Dou, Ruqi Liang, Lei Wen, Yang Liu, Xiaoguang Lei, Liru Wang, Huixia Guo, Xiaojun Huang, Mingdi Wang, Jin Lu","doi":"10.1159/000545709","DOIUrl":"10.1159/000545709","url":null,"abstract":"<p><strong>Introduction: </strong>We conducted a single-arm, open-label dose-exploration study to evaluate the safety and efficacy of the histone deacetylase inhibitor tucidinostat combined with bortezomib, liposomal doxorubicin, and dexamethasone (C-PDD) in treating relapsed and refractory multiple myeloma (RRMM) patients.</p><p><strong>Methods: </strong>Eighteen patients were enrolled from August 2020 to May 2021, receiving 21-day cycles of C-PDD.</p><p><strong>Results: </strong>Eighteen cases were analysed, with a median prior treatment line of 2 (range: 1-4). The median number of completed treatment cycles was 4 (range: 1-8). The overall response rate was 57%, including 14% complete response, 14% very good partial response, and 29% partial response. Both bortezomib-sensitive and refractory groups had a response rate of 57%. The response rate was 100% in patients with extramedullary extraosseous involvement. The median follow-up was 42 months (range: 3-44), with median progression-free survival of 7 months and median overall survival of 24.5 months. Grade 3-4 haematologic adverse events included thrombocytopaenia (50%), neutropenia (33%), and anaemia (33%). Non-haematologic adverse events were mostly grade 1-2, with 1 case of grade 3 peripheral sensory neuropathy.</p><p><strong>Conclusion: </strong>The C-PDD regimen showed efficacy in RRMM, including bortezomib-refractory disease and EME patients. The optimal dose and combination need to be explored in the future.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":1.7,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143964079","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Streamline: Retrospective Cohort Study of FMS-Like Tyrosine Kinase 3-Mutated Acute Myeloid Leukemia - Real-World Treatment Patterns and Clinical Outcomes of Patients in First Relapse or Refractory Diagnosis.","authors":"Amer M Zeidan, Rebekah Yu, Yuexi Wang, Ziyu Lan, David L Grinblatt, Dina Elsouda, James Spalding, Alana Block, Maelys Touya, Mark S Walker, Bhavik J Pandya","doi":"10.1159/000545384","DOIUrl":"10.1159/000545384","url":null,"abstract":"<p><strong>Introduction: </strong>Recent advances in genomic research have expanded the treatment landscape for acute myeloid leukemia (AML). This study examined treatment patterns and clinical outcomes among relapsed/refractory (R/R) FMS-like tyrosine kinase 3 (FLT3)-mutated AML patients.</p><p><strong>Methods: </strong>This retrospective longitudinal study included patients with confirmed AML diagnosis, FLT3 mutation, and 1st R/R event from 1/1/2015 to 1/31/2023 in the ConcertAI Oncology Dataset. Treatment patterns, FLT3 testing rates, real-world overall survival (rwOS), and real-world time to next treatment (rwTTNT) were studied.</p><p><strong>Results: </strong>Among the 336 treated patients, 50.6% received FLT3-tyrosine kinase inhibitors (FLT3-TKIs) as first treatment after R/R event, of which 51.8% received gilteritinib. High-intensity chemotherapy used as first treatment after R/R event decreased from 67.9% in 2015 to 20.0% in 2022, while FLT3-TKI utilization rose to 50% over the same period. Among the 246 patients tested for FLT3 at initial AML diagnosis, only 36% were retested at 1st R/R event. Median rwOS and rwTTNT among FLT3-TKI patients were 12.4 months and 2.9 months, respectively.</p><p><strong>Conclusion: </strong>This study reveals a trend toward increasing FLT3-TKI use and highlights the need for repeated FLT3 testing among R/R AML patients. Real-world evidence is vital in understanding R/R AML patient care amidst emerging therapies.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-12"},"PeriodicalIF":1.7,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12112888/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143960332","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Global, Regional, and National Burden of Leukemia (1990-2021): A Systematic Analysis for the Global Burden of Disease Study 2021.","authors":"Xue Yao, Hui Wang, Linhua Yang","doi":"10.1159/000545724","DOIUrl":"10.1159/000545724","url":null,"abstract":"<p><strong>Introduction: </strong>Leukemia is a group of diseases caused by malignant hematopoietic stem cell clones. Leukemia ranks 13th in incidence and 10th in mortality, according to the Global Cancer Statistics 2022.</p><p><strong>Methods: </strong>We computed the prevalence, incidence, mortality, and disability-adjusted life years (DALYs) using the Global Burden of Disease (GBD) 2021 data. We rigorously tested the time trend from 1990 to 2021 using Joinpoint regression analysis. This method facilitates the calculation of annual percentage change (APC), average annual percentage change, and 95% confidence interval (CI).</p><p><strong>Results: </strong>In 2021, the age-standardized prevalence rate (ASPR), age-standardized incidence rate (ASIR), age-standardized mortality rate (ASMR), and age-standardized DALYs rate of leukemia were 21.07 per 100,000 people (95% CI: 17.65-23.61), 5.63 per 100,000 people (95% CI: 4.83-6.17), 3.89 per 100,000 people (95% CI: 3.34-4.25), and 136.94 per 100,000 people (95% CI: 111.89-153.71), respectively. These figures have been obtained globally. Gender comparisons show that men have a higher burden of disease. The 90-94 age-group has the highest global prevalence of leukemia, with the prevalence increasing with age. Age-standardized DALYs rates, ASMR, and ASIR demonstrated a general declining trend from 1990 to 2021. The results of the Joinpoint analysis showed that between 2019 and 2021, there was a drop in the global ASPR (APC = -2.68%; 95% CI: -4.76% to -0.54%; p = 0.017), ASIR (APC = -2.46%; 95% CI: -3.33% to -1.59%; p < 0.001), and ASMR (APC = -1.87%; 95% CI: -2.75% to -0.99%; p < 0.001).</p><p><strong>Conclusion: </strong>The results indicated an overall decrease in the burden of leukemia. These results provide important epidemiological data for the development of novel treatments.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-18"},"PeriodicalIF":1.7,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143957863","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Recombinant Myeloid Hematopoietic Growth Factors and Clinical Stimulation of Acute Myeloid Leukemia Cells: A Narrative Review.","authors":"Jason S Gilbert, Daniel A Pollyea, Thomas J Walsh, Andreas H Groll, Hillard M Lazarus","doi":"10.1159/000545588","DOIUrl":"10.1159/000545588","url":null,"abstract":"<p><strong>Background: </strong>The advent of molecularly cloned hematopoietic growth factors (rhuG-CSF and rhuGM-CSF) enhanced the safety in treating acute myeloid leukemia (AML) by reducing the duration of neutropenia and thus decreasing the risks for infection. However, early in vitro studies demonstrated leukemia cell proliferation in response to these agents, raising reasonable concerns related to whether these factors can cause or contribute to relapse or progression of AML.</p><p><strong>Summary: </strong>Clinical studies using recombinant myeloid hematopoietic growth factors have supported their safety, as there is little or no clear evidence for an association with an increased risk of relapse in AML in patients, regardless of the hematopoietic growth factor used, or whether the setting of AML is newly diagnosed or relapsed/refractory. One exception may be in the pediatric population, though this effect might reflect a different isoform of the G-CSF receptor expressed on the AML cells, as this truncated receptor is also seen in severe congenital neutropenia and aplastic anemia and underlies the increased myeloid malignancies that develop in these diseases after long-term exposure to growth factors.</p><p><strong>Key messages: </strong>The current data support the use of rhuG-CSF and rhuGM-CSF in most patient populations with AML. Future studies should explore the factors influencing hematopoietic growth factor sensitivity in AML subpopulations to guide therapeutic decisions.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-9"},"PeriodicalIF":1.7,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143810112","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}