Martina Tripcovici, Aditya Sharma, Brittni J Clopton, Daniel Kurtz, Ayman O Soubani
{"title":"Epidemiology and outcomes of hospitalized patients with lung cancer and acute respiratory failure: National inpatient database analysis.","authors":"Martina Tripcovici, Aditya Sharma, Brittni J Clopton, Daniel Kurtz, Ayman O Soubani","doi":"10.4103/hemoncstem.HEMONCSTEM-D-25-00025","DOIUrl":"https://doi.org/10.4103/hemoncstem.HEMONCSTEM-D-25-00025","url":null,"abstract":"<p><strong>Background: </strong>Acute respiratory failure (ARF) is common in lung cancer patients and could be related to tumor progression, effects of treatment, or comorbid illnesses. Data on the short-term outcomes of these patients, especially those requiring invasive mechanical ventilation (IMV), remain scarce.</p><p><strong>Methods: </strong>The National Inpatient Sample database (2016-2019) was used to identify lung cancer admissions using International Classification of Diseases, Tenth Revision, Clinical Modification (ICD-10-CM) codes. ARF and IMV subgroups within lung cancer hospitalizations were identified using ICD-10-CM codes. Baseline variables were analyzed using χ2 test for categorical data and adjusted Wald tests for continuous data. Results were reported as percentages and mean ± standard deviation.</p><p><strong>Results: </strong>We identified 581,805 (24.7%) admissions for lung cancer with ARF during the study period. All-cause inpatient mortality was six times higher in lung cancer admissions with ARF compared to the non-ARF cohort (18.50% vs. 3.19%; P < 0.001). Similarly, lung cancer admissions with ARF that required IMV had significantly higher mortality compared to the cohort that did not require IMV (44.86% vs. 12.59%; P < 0.001). Lung cancer admissions with ARF requiring IMV had longer stay in the hospital (11.19 vs. 6.69 days; P < 0.001). The total hospitalization cost was more than two times higher for lung cancer admissions with ARF requiring IMV ($40,024.2 vs. $16,260.5; P < 0.001).</p><p><strong>Conclusions: </strong>In this largest study to date, we provide insight into the incidence and outcomes of lung cancer admissions with ARF. Lung cancer admissions with ARF, especially those requiring IMV, were associated with worse outcomes, longer hospital stays, and significantly higher healthcare costs. Compared to historical data, the outcomes in this large database have improved.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145042769","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}
Eman A El-Bostany, Eman A Elghoroury, Eman H Thabet, Nehal Abdelhamid, Hanan M Hamed, Niveen Salama, Alaa A Rashad, Shereen H Abd El Aziz, Solaf Kamel, Eman Awadallah, Iman I Salama
{"title":"Impact of TMPRSS6 gene polymorphism on iron overload among children with sickle cell disease.","authors":"Eman A El-Bostany, Eman A Elghoroury, Eman H Thabet, Nehal Abdelhamid, Hanan M Hamed, Niveen Salama, Alaa A Rashad, Shereen H Abd El Aziz, Solaf Kamel, Eman Awadallah, Iman I Salama","doi":"10.4103/hemoncstem.HEMONCSTEM-D-25-00002","DOIUrl":"https://doi.org/10.4103/hemoncstem.HEMONCSTEM-D-25-00002","url":null,"abstract":"<p><strong>Objective/background: </strong>Iron overload is a significant problem in transfusion-dependent children with sickle cell disease (SCD). The molecular mechanisms underlying the progression to iron overload in SCD are poorly understood. Our aim is to investigate how TMPRSS6 single nucleotide polymorphisms (SNPs) affect iron status in children with SCD.</p><p><strong>Methods: </strong>A case-control study was conducted for 61 SCD children, aged 6-18 years, and a control group of 42 age-sex matched healthy children. Real time polymerase chain reaction (PCR) was performed to determine the SNPs of rs11704654 C/T, rs4820268 A/G and rs855791 A/G of TMPRSS6 gene and iron overload parameters were measured.</p><p><strong>Results: </strong>There was a significant protective association between SCD and the rs11704654 polymorphism (OR [95% CI)] = 0.36(0.15-0.86), P < 0.05. In contrast, a significant association between rs4820268, rs855791 polymorphisms and SCD risk was found, using the dominant genetic model (P < 0.05). The hepcidin gene expression and hepcidin/iron ratio were significantly higher in rs4820268 AG genotype compared to the minor homozygote GG genotype. Meanwhile, there was no significant difference between rs11704654, rs855791 genotypes and iron status parameters.</p><p><strong>Conclusion: </strong>TMPRSS6 gene polymorphism influences susceptibility to SCD and contributes to the regulation of iron status. The rs4820268 AG genotype provides protection against iron overload. Modulation of iron status by TMPRSS6 SNPs represents a promising target for future therapy.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145042764","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}
Ibrahim N Muhsen, Dietger Niederwieser, Laurent Garderet, Olaf Penack, Hildegard T Greinix, Riad El Fakih, Nour Ben Abdeljelil, Ibraheem Abosoudah, Sameer Alamoudi, Amal Albeihany, Saad Ahmed Al Daama, Mohammad Hamad Alshahrani, Salem Alshemmari, Murtadha Al-Khabori, Ahlam Almasari, Abdulhakim Al Rawas, Medhat Askar, Jean El-Cheikh, Mohammed-Amine Bekadja, Malek Benakli, Munira Borhany, Maria El Kababri, Khalid Halahleh, Amir Ali Hamidieh, Mahmoud Hammad, Ahmad Ibrahim, Solaf Kanfar, Mohamed Hamed Khalaf, Mohammed Marei, Muhammad Ayaz Mir, Dania Monagel, Asma Quessar, Rawad Rihani, Munira Shabbir-Moosajee, Marwan Shaheen, Almetwaly Mohamed Sultan, Mohammad Vaezi, Damiano Rondelli, Mickey Boon Chai Koh, Zina Peric, Yoshiko Atsuta, Naeem Chaudhri, Mahmoud Aljurf
{"title":"Patterns of Graft-Versus-Host Disease (GvHD) Prevention Practices in the Eastern Mediterranean (EM) Region: A Worldwide Network for Blood & Marrow Transplantation (WBMT) Global Study.","authors":"Ibrahim N Muhsen, Dietger Niederwieser, Laurent Garderet, Olaf Penack, Hildegard T Greinix, Riad El Fakih, Nour Ben Abdeljelil, Ibraheem Abosoudah, Sameer Alamoudi, Amal Albeihany, Saad Ahmed Al Daama, Mohammad Hamad Alshahrani, Salem Alshemmari, Murtadha Al-Khabori, Ahlam Almasari, Abdulhakim Al Rawas, Medhat Askar, Jean El-Cheikh, Mohammed-Amine Bekadja, Malek Benakli, Munira Borhany, Maria El Kababri, Khalid Halahleh, Amir Ali Hamidieh, Mahmoud Hammad, Ahmad Ibrahim, Solaf Kanfar, Mohamed Hamed Khalaf, Mohammed Marei, Muhammad Ayaz Mir, Dania Monagel, Asma Quessar, Rawad Rihani, Munira Shabbir-Moosajee, Marwan Shaheen, Almetwaly Mohamed Sultan, Mohammad Vaezi, Damiano Rondelli, Mickey Boon Chai Koh, Zina Peric, Yoshiko Atsuta, Naeem Chaudhri, Mahmoud Aljurf","doi":"10.4103/hemoncstem.hemoncstem-D-25-00004","DOIUrl":"https://doi.org/10.4103/hemoncstem.hemoncstem-D-25-00004","url":null,"abstract":"<p><strong>Background: </strong>Preventing graft vs. host disease (GvHD) after allogeneic hematopoietic stem cell transplant (allo-HSCT) is essential to improving the outcomes and quality of life of allo-HSCT recipients. Little is known about the trends and patterns of global GvHD prevention practices. Thus, the Worldwide Network for Blood & Marrow Transplantation global GvHD project aims to understand and describe the patterns of GvHD prevention and management worldwide. This article discusses GvHD prevention practices in the East Mediterranean (EM) region.</p><p><strong>Materials and methods: </strong>A questionnaire was distributed electronically to the EM region transplant centers and filled out by program directors or designees. Responses were received between December 2022 and June 2023. The questionnaire had 33 items, with 7 sections focusing on the different commonly used agents in GvHD practices (including calcineurin inhibitors, in vivo T-cell depletion, and methotrexate [MTX]).</p><p><strong>Results: </strong>Thirty responses from 26 institutions were received from 11 countries in the EM region. All programs perform matched-related donor (MRD) transplants, 29 perform haploidentical transplants, and 19 perform matched unrelated donor (MUD) transplants. Cyclosporine (CsA) with MTX was the preferred regimen in both myeloablative (79%) and reduced intensity conditioning (50%). CsA was more widely used compared to tacrolimus (Tac) (93% vs. 57%). The duration of calcineurin inhibitor use before initiating taper in recipients with malignant and non-malignant disorders was similar between CsA and Tac. All programs reported routine monitoring of calcineurin inhibitor levels. Twenty-nine programs reported using MTX, administering it over 3-4 days post-HSCT. The use of different in vivo T-cell depletion therapies was commonly reported, particularly anti-thymocyte globulin (ATG) and post-transplant cyclophosphamide (PTCy). ATG use was reported by 77% and 79% of programs for MRD and MUD HCT, respectively. Additionally, most programs (97%) reported using PTCy mainly for haploidentical transplants. Among centers using PTCy, most programs reported using a dose of 50 mg/kg, and the most common schedule was Days +3 and +4. However, 12 programs reported using lower doses of 25-40 mg/kg or spaced-out schedules (Days +3 and +5).</p><p><strong>Conclusion: </strong>This study describes the different practices of GvHD prophylaxis in the EM region. Our results show that allo-HSCT centers in the EM regions utilize most standard-of-care agents, and most practices are in alignment with evidence-based guidelines.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145042864","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":"Allogeneic hematopoietic cell transplantation as the standard of care for blastic plasmacytoid dendritic cell neoplasm in first complete remission.","authors":"Mohamed A Kharfan-Dabaja","doi":"10.4103/hemoncstem.HEMONCSTEM-D-25-00023","DOIUrl":"https://doi.org/10.4103/hemoncstem.HEMONCSTEM-D-25-00023","url":null,"abstract":"<p><p>Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic disorder derived from precursors of plasmacytoid dendritic cells, with a generally aggressive clinical course. Tagraxofusp, a first-in-class CD123-directed antibody conjugate comprising human interleukin-3 and truncated diphtheria toxin, represents a welcome addition to the treatment armamentarium for BPDCN. Allogeneic hematopoietic cell transplantation (allo-HCT) is a treatment modality with curative potential in patients with BPDCN, based on data derived from registry studies, as well as multicenter and single-center retrospective studies. Several studies and a systematic review/meta-analysis have shown superior survival when patients undergo allo-HCT in first complete remission (CR1). In younger or fit patients, a myeloablative conditioning (MAC) allo-HCT regimen is the preferred approach whenever an allograft is indicated. Incorporating total body irradiation within the MAC regimen has been shown to improve progression-free survival, disease-free survival, and overall survival. However, MAC regimens have limited applicability in older, frail, or less fit patients, for whom reduced-intensity conditioning is the most appropriate approach. Several questions remain unanswered-namely, the potential benefit of post-transplant consolidation or maintenance strategies to mitigate the risk of relapse or progression, and the role of next-generation sequencing as a prognostic tool and for better defining depth of remission and measurable residual disease, among others. We believe that further improvement in the prognosis of BPDCN will require large collaborative efforts, considering the rarity of this disease.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145042799","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}
Andrew D Trunk, Yanwen Chen, Aaron T Gerds, Akriti Jain, Sudipto Mukherjee, Sophia Balderman, Hetty Carraway, Betty K Hamilton, Ronald Sobecks, Matt Kalaycio, Craig Sauter, Claudio Brunstein
{"title":"Myeloablative and Reduced Intensity Allogeneic Transplant in Patients with Myeloproliferative Neoplasms.","authors":"Andrew D Trunk, Yanwen Chen, Aaron T Gerds, Akriti Jain, Sudipto Mukherjee, Sophia Balderman, Hetty Carraway, Betty K Hamilton, Ronald Sobecks, Matt Kalaycio, Craig Sauter, Claudio Brunstein","doi":"10.4103/hemoncstem.HEMONCSTEM-D-25-00008","DOIUrl":"https://doi.org/10.4103/hemoncstem.HEMONCSTEM-D-25-00008","url":null,"abstract":"<p><strong>Background: </strong>Allogeneic hematopoietic cell transplant (allo-HCT) is the only potentially curative treatment for myelofibrosis (MF) and chronic myelomonocytic leukemia (CMML). Older age, comorbidities, and often advanced disease make patient selection and optimal transplant timing challenging. This study sought to understand allo-HCT outcomes for these myeloproliferative neoplasms in a contemporary era, including molecular data, to define a uniform transplant approach.</p><p><strong>Methods: </strong>Retrospective analysis was performed on patients with MF or CMML who received allo-HCT at the Cleveland Clinic between January 1, 2010 and April 1, 2023. All donor types and graft sources were included. MF and CMML outcomes were analyzed separately.</p><p><strong>Results: </strong>Fifty-nine MF and 33 CMML patients were included. JAK2 V617F was detected in 57.6% of MF patients; only 34 (57.6%) had next-generation sequencing (NGS) performed. Most MF transplants were reduced intensity (RIC; 69.5%) and peripheral blood stem cell (PBSC; 91%). At median follow-up of 41 months, 28/59 (47.5%) MF patients were alive. MF patients who were JAK2+ with additional cytogenetic changes or concurrent mutations had better overall survival. In CMML, 69.7% had myeloid NGS, with ASXL1 identified in 51.9% of cases. Most transplants were RIC (66.7%) and PBSC (72.7%). At median follow-up of 46.8 mos, 13/33 (39.4%) patients were alive. Relapse accounted for 9/20 CMML deaths; 8 of these received RIC. Mutational signature did not significantly impact survival, though the presence of any cytogenetic aberrancy was associated with worse OS (12 mos, 95% CI, 7.13-NA vs. 24.2 mos, 9.6-NA; P = 0.19).</p><p><strong>Conclusion: </strong>For MF and CMML, older patients (≥65) and RIC transplants trended toward worse survival. Strategies to reduce relapse and optimize patient selection utilizing molecular and cytogenetic data should be considered.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145042866","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}
Ibrahim N Muhsen, Dietger Niederwieser, Laurent Garderet, Olaf Penack, Hildegard T Greinix, Riad El Fakih, Nour Ben Abdeljelil, Ibraheem Abosoudah, Sameer Alamoudi, Amal Albeihany, Saad Ahmed Al Daama, Mohammad Hamad Alshahrani, Salem Alshemmari, Murtadha Al-Khabori, Ahlam Almasari, Abdulhakim Al Rawas, Medhat Askar, Ali Bazarbachi, Mohammed-Amine Bekadja, Malek Benakli, Munira Borhany, Maria El Kababri, Khalid Halahleh, Amir Ali Hamidieh, Mahmoud Hammad, Ahmad Ibrahim, Solaf Kanfar, Mohamed Hamed Khalaf, Mohammed Marei, Muhammad Ayaz Mir, Dania Monagel, Asma Quessar, Rawad Rihani, Munira Shabbir-Moosajee, Marwan Shaheen, Almetwaly Mohamed Sultan, Mohammad Vaezi, Damiano Rondelli, Mickey Boon Chai Koh, Zina Peric, Yoshiko Atsuta, Naeem Chaudhri, Mahmoud Aljurf
{"title":"Acute and chronic graft-versus-host disease treatment and management in the Eastern Mediterranean region: A Worldwide Network for Blood and Marrow Transplantation survey.","authors":"Ibrahim N Muhsen, Dietger Niederwieser, Laurent Garderet, Olaf Penack, Hildegard T Greinix, Riad El Fakih, Nour Ben Abdeljelil, Ibraheem Abosoudah, Sameer Alamoudi, Amal Albeihany, Saad Ahmed Al Daama, Mohammad Hamad Alshahrani, Salem Alshemmari, Murtadha Al-Khabori, Ahlam Almasari, Abdulhakim Al Rawas, Medhat Askar, Ali Bazarbachi, Mohammed-Amine Bekadja, Malek Benakli, Munira Borhany, Maria El Kababri, Khalid Halahleh, Amir Ali Hamidieh, Mahmoud Hammad, Ahmad Ibrahim, Solaf Kanfar, Mohamed Hamed Khalaf, Mohammed Marei, Muhammad Ayaz Mir, Dania Monagel, Asma Quessar, Rawad Rihani, Munira Shabbir-Moosajee, Marwan Shaheen, Almetwaly Mohamed Sultan, Mohammad Vaezi, Damiano Rondelli, Mickey Boon Chai Koh, Zina Peric, Yoshiko Atsuta, Naeem Chaudhri, Mahmoud Aljurf","doi":"10.4103/hemoncstem.HEMONCSTEM-D-25-00005","DOIUrl":"10.4103/hemoncstem.HEMONCSTEM-D-25-00005","url":null,"abstract":"<p><strong>Background: </strong>The treatment of acute and chronic graft-versus-host disease (GvHD) remains a challenge, particularly in cases of steroid-refractory GvHD. The management of GvHD varies between institutions, and little is known regarding the practices in different regions of the world. Thus, the Worldwide Network for Blood and Marrow Transplantation has developed a questionnaire to understand the current practices of GvHD management in the Eastern Mediterranean (EM) region.</p><p><strong>Methodology: </strong>The questionnaire had 46 items and was distributed electronically to transplant centers in the EM region. Responses were received between December 2022 and June 2023. The questionnaire addressed the management of acute and chronic GvHD for both newly diagnosed and refractory cases.</p><p><strong>Results: </strong>The questionnaire was completed by 30 programs across 26 institutions located in 11 countries. For patients with newly diagnosed acute GvHD, most programs reported the use of systemic steroids for initial treatment, with doses selected based on the severity of the presentation: the equivalent of 1 mg/kg/day of prednisone for grade IIa and 2 mg/kg/day for grade IIb. In addition to steroids, most programs continued immunosuppressive therapy or reintroduced it if GvHD developed after its cessation. For patients who were refractory to steroids, ruxolitinib was the most frequently selected second-line treatment, chosen by 80% of the programs, followed by calcineurin inhibitors (47%), high-dose steroids (>2 mg/kg, 43%), mycophenolate mofetil (MMF, 40%), and extracorporeal photopheresis (ECP, 40%). On the other hand, for patients with newly diagnosed chronic GvHD, systemic steroids are used for the initial management of mild chronic GvHD not accessible by topical treatment and moderate to severe disease, with the most commonly used initial dose being the equivalent of 0.5 to 1 and >1 mg/kg/day of prednisone, respectively. More than two-thirds of the programs use another agent in addition to steroids in patients who develop moderate/severe chronic GvHD while off immunosuppressive therapy. For patients with steroid-refractory chronic GvHD, most programs selected multiple options in the second-line setting, with the most frequently selected options being ruxolitinib (77%), calcineurin inhibitors (68%), MMF (53%), imatinib (53%), ECP (50%), rituximab (47%), and ibrutinib (40%).</p><p><strong>Conclusion: </strong>Our results demonstrated that GvHD management practices in the EM region generally align with current guidelines. However, the results highlight that access to clinical trials and multidisciplinary support teams remains limited.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":" ","pages":"79-85"},"PeriodicalIF":0.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144334735","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}
Diego Medina Valencia, Natalia Builes, Alexis A Franco, Angela Trujillo, Laura Isabel Niño Quiroga, Mauricio Chaparro, Andrés Felipe Escobar-González, Eliana Manzi, Diana Muñoz-Caluce, Estefania Beltran, Angela Devia Zapata, Marcela Estupiñan
{"title":"Outcomes of Matched Sibling and Haploidentical Donors Hematopoietic Stem Cell Transplantation for Pediatric Severe Aplastic Anemia: A Retrospective Multicenter Study.","authors":"Diego Medina Valencia, Natalia Builes, Alexis A Franco, Angela Trujillo, Laura Isabel Niño Quiroga, Mauricio Chaparro, Andrés Felipe Escobar-González, Eliana Manzi, Diana Muñoz-Caluce, Estefania Beltran, Angela Devia Zapata, Marcela Estupiñan","doi":"10.4103/hemoncstem.HEMONCSTEM-D-24-00037","DOIUrl":"10.4103/hemoncstem.HEMONCSTEM-D-24-00037","url":null,"abstract":"<p><strong>Background: </strong>Acquired aplastic anemia (AA) is a life-threatening hematologic disorder characterized by bone marrow failure. This study evaluates clinical outcomes of matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT) and haploidentical HSCT (haplo-HSCT) in pediatric patients with severe (SAA) and very severe aplastic anemia (VSAA) in Colombia.</p><p><strong>Methods: </strong>A retrospective multicenter study was conducted in four high-complexity centers in Colombia between 2011 and 2021, including 59 pediatric patients with SAA/VSAA who underwent allo-HSCT. Eligible patients were divided into two groups: one group underwent MSD-HSCT and the other haplo-HSCT, either as first-line treatment or after immunosuppressive therapy (IST) failure. Clinical outcomes, graft failure, graft-versus-host disease (GVHD), and overall survival (OS) were analyzed.</p><p><strong>Results: </strong>A total of 59 patients with SAA/VSAA undergoing allo-HSCT were included, 29 undergoing MSD-HSCT and 30 undergoing haplo-HSCT. The 2-year OS for the cohort was 81%, with 85% for MSD-HSCT and 75% for haplo-HSCT. Modified Baltimore-based conditioning regimens in haplo-HSCT showed an OS of 83%. Primary graft failure occurred in two haplo-HSCT patients. Grades III-IV acute GVHD and moderate-severe chronic GVHD were more common in haplo-HSCT. Infection was the leading cause of posttransplant mortality.</p><p><strong>Conclusion: </strong>Pediatric SAA patients undergoing MSD-HSCT had better survival outcomes and less incidence of aGVHD compared to haplo-HSCT. Haplo-HSCT with modified Baltimore conditioning offers a viable alternative, particularly when an MSD is unavailable. Prospective studies are needed to confirm these findings.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":" ","pages":"72-78"},"PeriodicalIF":0.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144334736","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}
Sergei V Belokon, Valeriya A Kukarskaya, Ilya D Klabukov, Victoria A Shestakova, Sergei A Ivanov, Petr V Shegay, Andrei D Kaprin, Denis S Baranovskii
{"title":"A Critical Review of CAR-T Therapies for Glioblastoma: What's Wrong with the Current Attempts?","authors":"Sergei V Belokon, Valeriya A Kukarskaya, Ilya D Klabukov, Victoria A Shestakova, Sergei A Ivanov, Petr V Shegay, Andrei D Kaprin, Denis S Baranovskii","doi":"10.4103/hemoncstem.HEMONCSTEM-D-24-00040","DOIUrl":"10.4103/hemoncstem.HEMONCSTEM-D-24-00040","url":null,"abstract":"<p><p>Today, CAR-T therapy has been widely acknowledged as a \"gold standard\" treatment for certain hematologic diseases. There is a relatively small but enhancing body of clinical trials studying the effectiveness of CAR-T in treating glioblastoma, known as the most common and aggressive brain tumor in adults. Despite the promising findings, currently available data is still erratic. We aimed to overview the recent clinical attempts to apply CAR-T therapy as the treatment strategy for glioblastoma and highlight non-obvious problems occurring: flaws in the study design with suspicious inclusion criteria, absence of narrow nosologic focus, poor validation or even nonvalid imaging technologies and inconveniencing efficacy evaluation. We also discussed further upcoming advanced approaches for CAR-T cell manufacturing. We are convinced that our review could help to define the right place for CAR-T therapy in glioblastoma treatment strategy and would pave the way for future successful clinical trials.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":" ","pages":"35-47"},"PeriodicalIF":0.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144334767","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":"Relevant biomarkers in acute graft-versus-host disease secondary to transplantation of hematopoietic stem cells.","authors":"Juan-Pablo Manosalva, Pamela Quimbaya, Rosa-Helena Bustos, Ana-Maria Perdomo-Arciniegas, Ximena Bonilla, Henry Oliveros","doi":"10.4103/hemoncstem.hemoncstem-D-24-00003","DOIUrl":"10.4103/hemoncstem.hemoncstem-D-24-00003","url":null,"abstract":"<p><p>Acute graft-versus-host disease (aGVHD) is a serious complication of allogeneic hematopoietic stem cell transplantation. Specific biomolecules identified in samples from patients with aGVHD have been correlated with the diagnosis, prognosis, prediction, and treatment response of aGVHD. In this review, we summarize the most promising biomarkers with clinical relevance for aGVHD. Articles were identified through a literature search in PubMed, EMBASE, and Cochrane databases up to November 1, 2022. All identified papers were manually reviewed. We included studies written in English or other languages that investigated biomarkers in GVHD. A total of 3733 studies were identified, of which 32 met the inclusion criteria and were included in this scoping review. Twenty-one different biomarkers were reviewed, including 11 plasma biomarkers and 9 organ-specific biomarkers. Nearly all biomarkers had distinct roles in aGVHD pathophysiology, while some were combined into biomarker panels along with clinical responses. The study of biomarkers in aGVHD has significantly expanded in recent years, encompassing various aspects of the disease. Currently, biomarker panels have shown the most promising results in terms of sensitivity and specificity for diagnosis and prognosis. Biomarkers hold great potential as tools for the reproducible prediction of aGVHD onset, severity, and treatment outcomes, although they remain unproven in routine clinical practice.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":"18 2","pages":"58-71"},"PeriodicalIF":0.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144736548","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}
Louis Fabio Jonathan Jusni, Eric Ricardo Yonatan, Nicolas Daniel Widjanarko, Rio Gusta Notario Besri
{"title":"A Systematic Review and Meta-Analysis of the Efficacy and Safety of Lazertinib as First-Line Treatment for EGFR-Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC).","authors":"Louis Fabio Jonathan Jusni, Eric Ricardo Yonatan, Nicolas Daniel Widjanarko, Rio Gusta Notario Besri","doi":"10.4103/hemoncstem.HEMONCSTEM-D-24-00041","DOIUrl":"10.4103/hemoncstem.HEMONCSTEM-D-24-00041","url":null,"abstract":"<p><strong>Background and objective: </strong>Epidermal growth factor receptor (EGFR) mutations are a common driver of oncogenesis in non-small cell lung cancer (NSCLC). Lazertinib, a third-generation EGFR tyrosine kinase inhibitor (TKI), has shown promise as a first-line treatment for patients with locally advanced or metastatic EGFR-mutated NSCLC. However, the comparative efficacy and safety of lazertinib in this setting have not been thoroughly investigated. This study aims to evaluate the efficacy and safety of lazertinib for EGFR-mutated locally advanced NSCLC.</p><p><strong>Method: </strong>This study was conducted following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. We performed a search of PubMed, ProQuest, EBSCO, and ScienceDirect to identify eligible studies based on the PICOTS-SD criteria. The meta-analysis of overall survival (OS), progression-free survival (PFS), and adverse effects was performed using RevMan 5.4 software.</p><p><strong>Results: </strong>This review included a total of three randomized controlled trials. The pooled analysis revealed significant differences in PFS, with a hazard ratio (HR) of 0.44 (95% confidence interval [CI]: 0.37-0.53). However, OS showed no significant differences, with an HR of 0.82 (95% CI: 0.64-1.06). Paresthesia occurred significantly more frequently in the lazertinib group (odds ratio [OR]: 11.30; 95% CI: 7.30-17.49). In contrast, the incidence of diarrhea and increases in Alanine Transaminase (ALT) and Aspartate Aminotransferase (AST) levels were significantly higher in the gefitinib group, with ORs of 0.52 (95% CI: 0.39-0.70), 0.35 (95% CI: 0.25-0.50), and 0.32 (95% CI: 0.22-0.47), respectively.</p><p><strong>Conclusion: </strong>Lazertinib demonstrated a greater therapeutic benefit for patients with EGFR-mutated advanced NSCLC compared to first-generation EGFR-TKIs. Further research is needed to validate these findings.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":" ","pages":"48-57"},"PeriodicalIF":0.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144334734","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}