Pub Date : 2026-01-01DOI: 10.1016/j.lrr.2025.100556
Donia Bouhoula, Haifa Regaieg, Hela Abroug, Nesrine Ben Sayed, Yossra Ben Youssef
Pediatric acute myeloid leukemia outcomes in resource-limited regions remain understudied, particularly regarding how regional epidemiologic patterns interact with prognostic determinants. Our Tunisian cohort (n=68) reveals distinct pediatric AML features: older presentation (median 11 years), male predominance (63%), and unexpected AML2 subtype prevalence (35%). Cytogenetics showed 24% favorable-risk and 22% adverse-risk cases. Treatment outcomes correlated strongly with risk stratification, emphasizing the need for adapted diagnostic protocols in resource-conscious settings to optimize care pathways.
{"title":"Pediatric AML in Sousse, Tunisia: Epidemiologic patterns and hierarchical prognostic factors","authors":"Donia Bouhoula, Haifa Regaieg, Hela Abroug, Nesrine Ben Sayed, Yossra Ben Youssef","doi":"10.1016/j.lrr.2025.100556","DOIUrl":"10.1016/j.lrr.2025.100556","url":null,"abstract":"<div><div>Pediatric acute myeloid leukemia outcomes in resource-limited regions remain understudied, particularly regarding how regional epidemiologic patterns interact with prognostic determinants. Our Tunisian cohort (n=68) reveals distinct pediatric AML features: older presentation (median 11 years), male predominance (63%), and unexpected AML2 subtype prevalence (35%). Cytogenetics showed 24% favorable-risk and 22% adverse-risk cases. Treatment outcomes correlated strongly with risk stratification, emphasizing the need for adapted diagnostic protocols in resource-conscious settings to optimize care pathways.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"25 ","pages":"Article 100556"},"PeriodicalIF":0.9,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145938759","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}
To systematically evaluate the clinical efficacy and safety of decitabine (DAC) combined with CAG regimen in the treatment of elderly patients with acute myeloid leukemia (AML) using meta-analysis
Methods
The studies reported the clinical efficacy and safety of DAC combined with CAG regimen in the treatment of elderly AML patients were searched in Pubmed, Chinese National Knowledge Infrastructure (CNKI), Wanfang Database, and VIP database by computer. On the basis of the screening criteria, the non-conforming literature was eliminated and the final selected literature was analyzed. The methodological quality was assessed and the research data was extracted. Revman 5.3 software was used to assess the clinical efficacy and safety of DAC combined with CAG in the treatment of elderly AML patients, which were shown with forest map. The funnel plots were used to test publication bias.
Results
Thirteen studies involving 1133 elderly AML patients were included. Meta-analysis demonstrated that in comparison with CAG alone group, the CR rate (Z = 5.50, P < 0.001) and total effective rate (Z = 8.71, P < 0.001) of DAC combined with CAG group were higher, while there was no significant difference in PR rate between the two groups (Z = 1.59, P = 0.11). And the infection rate (Z = 3.56, P < 0.001) and fever rate (Z = 5.86, P < 0.001) of DAC combined with CAG group were increased. There were no significant differences in the rates of hematological adverse reactions (P = 0.14), gastrointestinal reactions (P = 0.05), alopecia (P = 0.39), heart injury (P = 0.55), liver and kidney injury (P = 0.74) and myelosuppression (P = 0.82) between the two groups.
Conclusion
Compared with CAG alone regimen, DAC combined with CAG regimen improves clinical efficacy in elderly AML patients, but increases risks of infection and fever-related adverse events.
{"title":"Meta-analysis of clinical efficacy and safety of decitabine combined with CAG regimen in the treatment of acute myeloid leukemia in the elderly","authors":"Yanxia He, Lili Zhang, Mengmeng Liu, Fuhua Zhang, Hui Gao","doi":"10.1016/j.lrr.2025.100559","DOIUrl":"10.1016/j.lrr.2025.100559","url":null,"abstract":"<div><h3>Objective</h3><div>To systematically evaluate the clinical efficacy and safety of decitabine (DAC) combined with CAG regimen in the treatment of elderly patients with acute myeloid leukemia (AML) using meta-analysis</div></div><div><h3>Methods</h3><div>The studies reported the clinical efficacy and safety of DAC combined with CAG regimen in the treatment of elderly AML patients were searched in Pubmed, Chinese National Knowledge Infrastructure (CNKI), Wanfang Database, and VIP database by computer. On the basis of the screening criteria, the non-conforming literature was eliminated and the final selected literature was analyzed. The methodological quality was assessed and the research data was extracted. Revman 5.3 software was used to assess the clinical efficacy and safety of DAC combined with CAG in the treatment of elderly AML patients, which were shown with forest map. The funnel plots were used to test publication bias.</div></div><div><h3>Results</h3><div>Thirteen studies involving 1133 elderly AML patients were included. Meta-analysis demonstrated that in comparison with CAG alone group, the CR rate (<em>Z</em> = 5.50, <em>P</em> < 0.001) and total effective rate (<em>Z</em> = 8.71, <em>P</em> < 0.001) of DAC combined with CAG group were higher, while there was no significant difference in PR rate between the two groups (<em>Z</em> = 1.59, <em>P</em> = 0.11). And the infection rate (<em>Z</em> = 3.56, <em>P</em> < 0.001) and fever rate (<em>Z</em> = 5.86, <em>P</em> < 0.001) of DAC combined with CAG group were increased. There were no significant differences in the rates of hematological adverse reactions (<em>P</em> = 0.14), gastrointestinal reactions (<em>P</em> = 0.05), alopecia (<em>P</em> = 0.39), heart injury (<em>P</em> = 0.55), liver and kidney injury (<em>P</em> = 0.74) and myelosuppression (<em>P</em> = 0.82) between the two groups.</div></div><div><h3>Conclusion</h3><div>Compared with CAG alone regimen, DAC combined with CAG regimen improves clinical efficacy in elderly AML patients, but increases risks of infection and fever-related adverse events.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"25 ","pages":"Article 100559"},"PeriodicalIF":0.9,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146077384","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}
Pub Date : 2026-01-01DOI: 10.1016/j.lrr.2025.100558
Kaiqiong Liao , Chong Guo , Min Zhang , Xiaobin Lv , Jinhua Yan
Objective
The objective of this research is to explore the anti-leukemic properties of CDK4/6 inhibitors when used alongside BET inhibitors for treating acute myeloid leukemia (AML), as well as to clarify the molecular mechanisms involved.
Methods
Cell viability was assessed using the CCK-8 assay following treatment of AML cells with varying doses of SHR6390, a CDK4/6 inhibitor, and OTX015, a BET inhibitor.The time- and dose-dependent inhibitory effects of these two drugs on AML cells were assessed, and the respective IC50 and combination index (CI) values after co-treatment were calculated. The effects of SHR6390 and OTX015 on the growth potential of AML cells were additionally examined through soft agar colony formation assays and flow cytometry. Furthermore, RNA sequencing and Western blot analysis were conducted on cells treated with both drugs. The aim of this study is to explore the mechanism by which SHR6390 and OTX015 synergistically inhibit the proliferation of AML cells.The anti-tumor activity of SHR6390 and/or OTX015 in AML xenograft mice was also investigated through animal experiments.
Results
1. Either the CDK4/6 inhibitor SHR6390 or the BRD4 inhibitor OTX015, or a combination of the two, were employed to hinder both the survival and proliferation of cell lines associated with acute myeloid leukemia, showing a synergistic effect. 2. The combined application of SHR6390 and OTX015 markedly suppresses the invasive and migratory capacities of acute myeloid leukemia cells.3. The use of both SHR6390 and OTX015 induces apoptosis in acute myeloid leukemia cells while also disrupting cell cycle progression, leading to a halt before DNA replication occurs. 4. SHR6390 and OTX015 hinder the proliferation of acute myeloid leukemia cells by targeting both the PI3K-AKT-mTOR and the Wnt-β-Catenin pathway. 5. SHR6390 and OTX015 Synergistically Inhibit the Growth of AML Xenografts In Vivo.
{"title":"The synergistic tumor suppressor effect of CDK4/6 inhibitors and BRD4 inhibitors in acute myeloid leukemia","authors":"Kaiqiong Liao , Chong Guo , Min Zhang , Xiaobin Lv , Jinhua Yan","doi":"10.1016/j.lrr.2025.100558","DOIUrl":"10.1016/j.lrr.2025.100558","url":null,"abstract":"<div><h3>Objective</h3><div>The objective of this research is to explore the anti-leukemic properties of CDK4/6 inhibitors when used alongside BET inhibitors for treating acute myeloid leukemia (AML), as well as to clarify the molecular mechanisms involved.</div></div><div><h3>Methods</h3><div>Cell viability was assessed using the CCK-8 assay following treatment of AML cells with varying doses of SHR6390, a CDK4/6 inhibitor, and OTX015, a BET inhibitor.The time- and dose-dependent inhibitory effects of these two drugs on AML cells were assessed, and the respective IC50 and combination index (CI) values after co-treatment were calculated. The effects of SHR6390 and OTX015 on the growth potential of AML cells were additionally examined through soft agar colony formation assays and flow cytometry. Furthermore, RNA sequencing and Western blot analysis were conducted on cells treated with both drugs. The aim of this study is to explore the mechanism by which SHR6390 and OTX015 synergistically inhibit the proliferation of AML cells.The anti-tumor activity of SHR6390 and/or OTX015 in AML xenograft mice was also investigated through animal experiments.</div></div><div><h3>Results</h3><div>1. Either the CDK4/6 inhibitor SHR6390 or the BRD4 inhibitor OTX015, or a combination of the two, were employed to hinder both the survival and proliferation of cell lines associated with acute myeloid leukemia, showing a synergistic effect. 2. The combined application of SHR6390 and OTX015 markedly suppresses the invasive and migratory capacities of acute myeloid leukemia cells.3. The use of both SHR6390 and OTX015 induces apoptosis in acute myeloid leukemia cells while also disrupting cell cycle progression, leading to a halt before DNA replication occurs. 4. SHR6390 and OTX015 hinder the proliferation of acute myeloid leukemia cells by targeting both the PI3K-AKT-mTOR and the Wnt-β-Catenin pathway. 5. SHR6390 and OTX015 Synergistically Inhibit the Growth of AML Xenografts In Vivo.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"25 ","pages":"Article 100558"},"PeriodicalIF":0.9,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145977084","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}
Pub Date : 2026-01-01DOI: 10.1016/j.lrr.2026.100563
Christian J. Puzo , Karl Hager , Michal Rose , Ellice Wong , Christopher A. Tormey , Alexa J. Siddon
This case details a patient presenting with a history of chronic leukocytosis with isolated eosinophilia, who was found to have a JAK2 G571S mutation by a next generation sequencing (NGS) panel for myeloproliferative neoplasm driver mutations. Adjunct NGS testing was performed on a sample of buccal cells to demonstrate this variant was a germline alteration, occurring in the absence of additional disease-causing mutations. The result caused the patient to be re-treated for a prior Strongyloides infection with ivermectin, which resolved his eosinophilia. This patient case highlights the likely benign polymorphic nature of the rare G571S JAK2 mutation that has been previously reported. Moreover, our results stress the importance of appropriate interpretation of rare variants of uncertain significance, namely that clinical decision making should be supported by adjunct genetic testing and with appropriate reference to each patient’s clinical context.
{"title":"Leukocytosis and a JAK2 mutation: The importance of expertise in somatic variant interpretation","authors":"Christian J. Puzo , Karl Hager , Michal Rose , Ellice Wong , Christopher A. Tormey , Alexa J. Siddon","doi":"10.1016/j.lrr.2026.100563","DOIUrl":"10.1016/j.lrr.2026.100563","url":null,"abstract":"<div><div>This case details a patient presenting with a history of chronic leukocytosis with isolated eosinophilia, who was found to have a <em>JAK2</em> G571S mutation by a next generation sequencing (NGS) panel for myeloproliferative neoplasm driver mutations. Adjunct NGS testing was performed on a sample of buccal cells to demonstrate this variant was a germline alteration, occurring in the absence of additional disease-causing mutations. The result caused the patient to be re-treated for a prior Strongyloides infection with ivermectin, which resolved his eosinophilia. This patient case highlights the likely benign polymorphic nature of the rare G571S JAK2 mutation that has been previously reported. Moreover, our results stress the importance of appropriate interpretation of rare variants of uncertain significance, namely that clinical decision making should be supported by adjunct genetic testing and with appropriate reference to each patient’s clinical context.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"25 ","pages":"Article 100563"},"PeriodicalIF":0.9,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146037337","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}
Pub Date : 2026-01-01DOI: 10.1016/j.lrr.2025.100557
Donia Bouhoula , Haifa Regaieg , Hela Abroug , Nesrine Ben Sayed , Yossra Ben Youssef
Treating pediatric acute myeloid leukemia in resource-limited settings presents unique therapeutic challenges. This study shares 19 years of experience from a Tunisian center, focusing on practical treatment outcomes. Our pediatric AML cohort (n=68) shows 50% initial remission rates (75% with idarubicin vs 31% novantrone) and 9% induction mortality. Despite salvage therapy (43% CR2), 5-year survival remained at 33%, constrained by high relapse (53%) and limited transplant access (21%). These results highlight critical needs: optimized frontline therapy and expanded salvage options for refractory cases.
{"title":"Long-term outcomes of pediatric AML in Tunisia: Lessons from 19 years of practice in Sousse","authors":"Donia Bouhoula , Haifa Regaieg , Hela Abroug , Nesrine Ben Sayed , Yossra Ben Youssef","doi":"10.1016/j.lrr.2025.100557","DOIUrl":"10.1016/j.lrr.2025.100557","url":null,"abstract":"<div><div>Treating pediatric acute myeloid leukemia in resource-limited settings presents unique therapeutic challenges. This study shares 19 years of experience from a Tunisian center, focusing on practical treatment outcomes. Our pediatric AML cohort (n=68) shows 50% initial remission rates (75% with idarubicin vs 31% novantrone) and 9% induction mortality. Despite salvage therapy (43% CR2), 5-year survival remained at 33%, constrained by high relapse (53%) and limited transplant access (21%). These results highlight critical needs: optimized frontline therapy and expanded salvage options for refractory cases.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"25 ","pages":"Article 100557"},"PeriodicalIF":0.9,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145938760","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}
Pub Date : 2026-01-01DOI: 10.1016/j.lrr.2026.100561
Jordan Rubenstein, Sarah M. Schwartz, Jay Vankawala, Michael Caplan, Steven Shea, Joseph G. Jurcic
Imatinib, a tyrosine kinase inhibitor used for chronic myeloid leukemia (CML) and gastrointestinal stromal tumors (GIST), is associated with myalgia and creatine kinase (CK) elevations, though severe rhabdomyolysis and myopathy are rare. We report an 83-year-old woman with CML who developed progressive proximal weakness, dark urine, and acute kidney injury after three years of imatinib therapy. Laboratory evaluation revealed CK >24,000 U/L, transaminitis, and myoglobinuria. MRI showed diffuse muscle and fascial edema, while autoimmune testing was negative. Imatinib and rosuvastatin were discontinued, and the patient was managed with intravenous fluids and supportive care. CK and renal function normalized within 10 days, with substantial recovery of strength. The strong temporal relationship between drug withdrawal and improvement implicates imatinib as the etiology. This case represents one of the most severe reported instances of imatinib-induced rhabdomyolysis. Early recognition and discontinuation are essential to prevent life-threatening sequelae.
{"title":"Imatinib-induced rhabdomyolysis: A case report","authors":"Jordan Rubenstein, Sarah M. Schwartz, Jay Vankawala, Michael Caplan, Steven Shea, Joseph G. Jurcic","doi":"10.1016/j.lrr.2026.100561","DOIUrl":"10.1016/j.lrr.2026.100561","url":null,"abstract":"<div><div>Imatinib, a tyrosine kinase inhibitor used for chronic myeloid leukemia (CML) and gastrointestinal stromal tumors (GIST), is associated with myalgia and creatine kinase (CK) elevations, though severe rhabdomyolysis and myopathy are rare. We report an 83-year-old woman with CML who developed progressive proximal weakness, dark urine, and acute kidney injury after three years of imatinib therapy. Laboratory evaluation revealed CK >24,000 U/L, transaminitis, and myoglobinuria. MRI showed diffuse muscle and fascial edema, while autoimmune testing was negative. Imatinib and rosuvastatin were discontinued, and the patient was managed with intravenous fluids and supportive care. CK and renal function normalized within 10 days, with substantial recovery of strength. The strong temporal relationship between drug withdrawal and improvement implicates imatinib as the etiology. This case represents one of the most severe reported instances of imatinib-induced rhabdomyolysis. Early recognition and discontinuation are essential to prevent life-threatening sequelae.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"25 ","pages":"Article 100561"},"PeriodicalIF":0.9,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145977082","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}
Pub Date : 2026-01-01DOI: 10.1016/j.lrr.2026.100562
Wenwen Zhang , Bin Luo , Zhuo Liu , Jie Mi , Yanping Yang , Yuan Li
This study analyzed FAERS data to evaluate pegaspargase safety in pediatric acute lymphoblastic leukemia treatment. Among 21,161,817 reports, 847 identified pegaspargase as the primary suspect drug, revealing adverse events (AEs) across 26 organ systems. Disproportionality analysis identified four previously unlisted AEs. Critically, pegaspargase-associated pancreatitis incidence showed significant age-dependence (χ²=8.219, p < 0.05). These findings address the knowledge gap in real-world, long-term pediatric safety profiles and provide crucial clinical references for safer medication use in children.
{"title":"Pancreatitis induced by Peg-aspargase in children: a real-world pharmacovigilance analysis based on FDA Adverse Event Reporting System (FAERS)","authors":"Wenwen Zhang , Bin Luo , Zhuo Liu , Jie Mi , Yanping Yang , Yuan Li","doi":"10.1016/j.lrr.2026.100562","DOIUrl":"10.1016/j.lrr.2026.100562","url":null,"abstract":"<div><div>This study analyzed FAERS data to evaluate pegaspargase safety in pediatric acute lymphoblastic leukemia treatment. Among 21,161,817 reports, 847 identified pegaspargase as the primary suspect drug, revealing adverse events (AEs) across 26 organ systems. Disproportionality analysis identified four previously unlisted AEs. Critically, pegaspargase-associated pancreatitis incidence showed significant age-dependence (χ²=8.219, <em>p</em> < 0.05). These findings address the knowledge gap in real-world, long-term pediatric safety profiles and provide crucial clinical references for safer medication use in children.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"25 ","pages":"Article 100562"},"PeriodicalIF":0.9,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145977079","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}
Pub Date : 2026-01-01DOI: 10.1016/j.lrr.2026.100564
Tristan Meier , Giovann Huynh , Ernesto Ayala , Han W. Tun , Madiha Iqbal
Richter’s syndrome (RS) is characterized by transformation of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) into an aggressive lymphoma. While significant treatment advances have been made in the management of CLL/SLL, current front-line management of RS is consistent with chemoimmunotherapy (CIT), a therapeutic approach adopted from the management of de-novo diffuse large B-cell lymphoma (DLBCL). In de-novo DLBCL, CIT alone can potentially cure a significant percentage of patients; however in RS, outcomes with CIT alone are particularly poor. Therefore there is a significant unmet need to improve the current standard of care for the management of RS. We present a case of a patient with newly diagnosed RS who was treated with a novel upfront combination of CIT and a Bruton tyrosine kinase inhibitor, resulting in excellent long-term outcomes. We also present accompanying literature review highlighting other anecdotal cases reported with similar therapeutic approach and discuss emerging treatment strategies in the management of RS.
{"title":"Bruton tyrosine kinase inhibitors in combination with chemoimmunotherapy is an effective treatment for patients with Richter’s syndrome","authors":"Tristan Meier , Giovann Huynh , Ernesto Ayala , Han W. Tun , Madiha Iqbal","doi":"10.1016/j.lrr.2026.100564","DOIUrl":"10.1016/j.lrr.2026.100564","url":null,"abstract":"<div><div>Richter’s syndrome (RS) is characterized by transformation of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) into an aggressive lymphoma. While significant treatment advances have been made in the management of CLL/SLL, current front-line management of RS is consistent with chemoimmunotherapy (CIT), a therapeutic approach adopted from the management of de-novo diffuse large B-cell lymphoma (DLBCL). In de-novo DLBCL, CIT alone can potentially cure a significant percentage of patients; however in RS, outcomes with CIT alone are particularly poor. Therefore there is a significant unmet need to improve the current standard of care for the management of RS. We present a case of a patient with newly diagnosed RS who was treated with a novel upfront combination of CIT and a Bruton tyrosine kinase inhibitor, resulting in excellent long-term outcomes. We also present accompanying literature review highlighting other anecdotal cases reported with similar therapeutic approach and discuss emerging treatment strategies in the management of RS.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"25 ","pages":"Article 100564"},"PeriodicalIF":0.9,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145977083","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}
Pub Date : 2026-01-01DOI: 10.1016/j.lrr.2026.100565
Ha Thanh Nguyen , Quoc Khanh Bach , Quoc Nhat Nguyen , Van Nam Nguyen , Thi Van Anh Nguyen , Hai Pham-The
Background
Hypomethylating agents such as decitabine represent a key treatment option for elderly acute myeloid leukemia (AML) patients who are unfit for intensive chemotherapy. However, real-world evidence from low- and middle-income countries (LMICs), including Vietnam, remains scarce. This study aimed to identify baseline clinical and laboratory predictors of 1-year overall survival (OS) in elderly Vietnamese AML patients treated with decitabine.
Methods
This prospective, single-center, observational study was conducted at the National Institute of Hematology and Blood Transfusion, Vietnam, from April 2023 to June 2025. Seventy newly diagnosed AML patients aged ≥ 60 years received decitabine. The primary outcome was 1-year OS. Baseline demographic, clinical, hematologic, and immunophenotypic variables were analyzed using univariate and multivariate Cox regression. Treatment response was descriptively evaluated in patients completing ≥4 cycles.
Results
The 1-year OS rate was 38.6 %, with a median survival of 276.0 days. Elevated bone marrow cell count (HR: 1.003, p = 0.002), fibrinogen (HR: 1.22, p = 0.044), and urea (HR: 1.23, p = 0.001) were independently associated with increased mortality. CD64 positivity (HR: 0.29, p = 0.029) and urban residence (HR: 0.43, p = 0.014) were protective. Among 39 patients completing ≥4 cycles, 48.7 % achieved a complete response. No significant survival difference was observed between responders and non-responders.
Conclusion
This first prospective real-world study in Vietnam identified accessible predictors of survival in elderly AML patients receiving decitabine. The findings may aid early risk stratification using simple baseline parameters in LMIC settings, where access to molecular diagnostics may be limited, and warrant multicenter validation.
对于不适合强化化疗的老年急性髓性白血病（AML）患者，地西他滨等聚甲基化药物是一种关键的治疗选择。然而，来自包括越南在内的低收入和中等收入国家（LMICs）的真实证据仍然很少。本研究旨在确定接受地西他滨治疗的越南老年AML患者1年总生存率（OS）的基线临床和实验室预测指标。该前瞻性、单中心、观察性研究于2023年4月至2025年6月在越南国家血液学和输血研究所进行。70例年龄≥60岁的新诊断AML患者接受地西他滨治疗。主要终点为1年OS。使用单因素和多因素Cox回归分析基线人口统计学、临床、血液学和免疫表型变量。完成≥4个周期的患者对治疗反应进行描述性评价。结果1年OS率为38.6%，中位生存期为276.0天。骨髓细胞计数（HR: 1.003, p = 0.002）、纤维蛋白原（HR: 1.22, p = 0.044）和尿素（HR: 1.23, p = 0.001）升高与死亡率升高独立相关。CD64阳性（HR: 0.29, p = 0.029）和城市居住（HR: 0.43, p = 0.014）具有保护作用。在完成≥4个周期的39例患者中，48.7%达到完全缓解。反应者和无反应者的生存期无显著差异。结论：越南的首次前瞻性现实世界研究确定了接受地西他滨治疗的老年AML患者的可获得的生存预测因子。这些发现可能有助于在低mic环境中使用简单的基线参数进行早期风险分层，在低mic环境中，分子诊断可能受到限制，并且需要多中心验证。
{"title":"Survival predictors in Vietnamese elderly AML patients treated with decitabine: Real-world evidence from a low- and middle-income country","authors":"Ha Thanh Nguyen , Quoc Khanh Bach , Quoc Nhat Nguyen , Van Nam Nguyen , Thi Van Anh Nguyen , Hai Pham-The","doi":"10.1016/j.lrr.2026.100565","DOIUrl":"10.1016/j.lrr.2026.100565","url":null,"abstract":"<div><h3>Background</h3><div>Hypomethylating agents such as decitabine represent a key treatment option for elderly acute myeloid leukemia (AML) patients who are unfit for intensive chemotherapy. However, real-world evidence from low- and middle-income countries (LMICs), including Vietnam, remains scarce. This study aimed to identify baseline clinical and laboratory predictors of 1-year overall survival (OS) in elderly Vietnamese AML patients treated with decitabine.</div></div><div><h3>Methods</h3><div>This prospective, single-center, observational study was conducted at the National Institute of Hematology and Blood Transfusion, Vietnam, from April 2023 to June 2025. Seventy newly diagnosed AML patients aged ≥ 60 years received decitabine. The primary outcome was 1-year OS. Baseline demographic, clinical, hematologic, and immunophenotypic variables were analyzed using univariate and multivariate Cox regression. Treatment response was descriptively evaluated in patients completing ≥4 cycles.</div></div><div><h3>Results</h3><div>The 1-year OS rate was 38.6 %, with a median survival of 276.0 days. Elevated bone marrow cell count (HR: 1.003, <em>p</em> = 0.002), fibrinogen (HR: 1.22, <em>p</em> = 0.044), and urea (HR: 1.23, <em>p</em> = 0.001) were independently associated with increased mortality. CD64 positivity (HR: 0.29, <em>p</em> = 0.029) and urban residence (HR: 0.43, <em>p</em> = 0.014) were protective. Among 39 patients completing ≥4 cycles, 48.7 % achieved a complete response. No significant survival difference was observed between responders and non-responders.</div></div><div><h3>Conclusion</h3><div>This first prospective real-world study in Vietnam identified accessible predictors of survival in elderly AML patients receiving decitabine. The findings may aid early risk stratification using simple baseline parameters in LMIC settings, where access to molecular diagnostics may be limited, and warrant multicenter validation.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"25 ","pages":"Article 100565"},"PeriodicalIF":0.9,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146037212","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}
Pub Date : 2026-01-01DOI: 10.1016/j.lrr.2026.100566
Vincent T. Taillefer , Marianne Emond , Marie-Hélène Leblanc , Stephane Doucet , Jean-Philippe Adam
Introduction
A combination of Gemcitabine, Dexamethasone and Cisplatin (GDP) with or without rituximab (±R) is an outpatient treatment widely used for relapsed or refractory (R/R) lymphoma. Limited data are available on stem cell mobilization following GDP treatment.
Method
A retrospective multicenter study was conducted from January 1st, 2014, to December 31st, 2020. Patients received GDP±R before autologous stem cell transplantation (ASCT) and were mobilized with either GDP±R or intermediate-dose cyclophosphamide (ID-CY). The primary objective was to determine the percentage of patients achieving ≥2 × 106 CD34+/kg, median CD34+ yield, and number of apheresis days. The secondary objective compared these results with ID-CY.
Results
Ninety-two patients (median age 54) were treated for diffuse large B-cell (40%), Hodgkin (36%), or follicular lymphoma (16%). Of these, 83 were mobilized with GDP±R and 9 with ID-CY. G-CSF was administered at 5 mcg/kg (61%) or 10 mcg/kg (37%) daily, typically starting on day 9, after two GDP cycles. A successful stem cell collection at the first attempt occurred in 96% of GDP±R patients, and 94% of patients proceeded to ASCT. No significant difference was found between GDP±R and ID-CY in terms of successful collection (96% vs. 100%, p=NS), but fewer hospitalizations occurred within 21 days of mobilization (1% vs. 22%, p = 0.02).
Conclusion
This study demonstrated the feasibility of performing a peripheral blood stem cell mobilization at day 15 following GDP±R. It is highly effective and represents a better option due to its simplicity of administration, low rate of hospitalization and low cost.
{"title":"Gemcitabine, dexamethasone and cisplatin with or without rituximab is highly effective as a mobilization regimen in relapsed or refractory lymphoma","authors":"Vincent T. Taillefer , Marianne Emond , Marie-Hélène Leblanc , Stephane Doucet , Jean-Philippe Adam","doi":"10.1016/j.lrr.2026.100566","DOIUrl":"10.1016/j.lrr.2026.100566","url":null,"abstract":"<div><h3>Introduction</h3><div>A combination of Gemcitabine, Dexamethasone and Cisplatin (GDP) with or without rituximab (±R) is an outpatient treatment widely used for relapsed or refractory (R/R) lymphoma. Limited data are available on stem cell mobilization following GDP treatment.</div></div><div><h3>Method</h3><div>A retrospective multicenter study was conducted from January 1st, 2014, to December 31st, 2020. Patients received GDP±R before autologous stem cell transplantation (ASCT) and were mobilized with either GDP±R or intermediate-dose cyclophosphamide (ID-CY). The primary objective was to determine the percentage of patients achieving ≥2 × 10<sup>6</sup> CD34<sup>+</sup>/kg, median CD34<sup>+</sup> yield, and number of apheresis days. The secondary objective compared these results with ID-CY.</div></div><div><h3>Results</h3><div>Ninety-two patients (median age 54) were treated for diffuse large B-cell (40%), Hodgkin (36%), or follicular lymphoma (16%). Of these, 83 were mobilized with GDP±R and 9 with ID-CY. G-CSF was administered at 5 mcg/kg (61%) or 10 mcg/kg (37%) daily, typically starting on day 9, after two GDP cycles. A successful stem cell collection at the first attempt occurred in 96% of GDP±R patients, and 94% of patients proceeded to ASCT. No significant difference was found between GDP±R and ID-CY in terms of successful collection (96% vs. 100%, <em>p</em>=NS), but fewer hospitalizations occurred within 21 days of mobilization (1% vs. 22%, <em>p</em> = 0.02).</div></div><div><h3>Conclusion</h3><div>This study demonstrated the feasibility of performing a peripheral blood stem cell mobilization at day 15 following GDP±R. It is highly effective and represents a better option due to its simplicity of administration, low rate of hospitalization and low cost.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"25 ","pages":"Article 100566"},"PeriodicalIF":0.9,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146077382","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}
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