keyword
https://read.qxmd.com/read/38640348/final-phase-i-substudy-results-of-ivosidenib-in-patients-with-mutant-idh1-relapsed-refractory-myelodysplastic-syndrome
#1
JOURNAL ARTICLE
Courtney D D DiNardo, Gail J Roboz, Justin M Watts, Yazan F Madanat, Gabrielle T Prince, Praneeth Baratam, Stéphane de Botton, Anthony S Stein, James M Foran, Martha L Arellano, David A Sallman, Mohammad Hossain, Dylan M Marchione, Xiaofei Bai, Prapti A Patel, Stephanie M Kapsalis, Guillermo Garcia-Manero, Amir T Fathi
Ivosidenib is a first-in-class mutant isocitrate dehydrogenase 1 (mIDH1) inhibitor and has shown efficacy and tolerability in patients with advanced mIDH1 hematologic malignancies, leading to approval in front-line and relapsed/refractory (R/R) mIDH1 AML populations. We report final data from a phase I single-arm substudy (NCT02074839) of patients with R/R mIDH1 MDS following failure of standard-of-care therapies. Oral ivosidenib was taken once daily on days 1-28 in 28-day cycles. Primary objectives were to determine safety, tolerability, and clinical activity...
April 19, 2024: Blood Advances
https://read.qxmd.com/read/38639895/characterization-of-potential-spermatogonia-biomarker-genes-in-the-european-eel-anguilla-anguilla
#2
JOURNAL ARTICLE
Marta Blanes-García, Zoran Marinović, Juan Germán Herranz-Jusdado, Xuan Xie, Leonor Ferrão, Victor Gallego, Luz Pérez, Abdul Rasheed Baloch, Ákos Horváth, Martin Pšenička, Juan F Asturiano, Marina Morini
Identification of specific molecular markers for spermatogonial stem cells in teleost is crucial for enhancing the efficacy of reproductive biotechnologies in aquaculture, such as transplantation and surrogate production in fishes. Since it is not yet possible to distinguish spermatogonial stem cells of European eel (Anguilla anguilla) using specific molecular markers, we isolated spermatogonial cells from immature European eels to find these potential markers. We attempted this by studying three candidate genes: vasa, nanos2, and dnd1...
April 19, 2024: Fish Physiology and Biochemistry
https://read.qxmd.com/read/38639861/hematopoietic-stem-cell-transplantation-for-storage-disorders-present-status
#3
REVIEW
Soumalya Chakraborty, Aditya Kumar Gupta, Neerja Gupta, Jagdish Prasad Meena, Rachna Seth, Madhulika Kabra
Storage disorders are a group of inborn errors of metabolism caused by the defective activity of lysosomal enzymes or transporters. All of these disorders have multisystem involvement with variable degrees of neurological features. Neurological manifestations are one of the most difficult aspects of treatment concerning these diseases. The available treatment modalities for some of these disorders include enzyme replacement therapy, substrate reduction therapy, hematopoietic stem cell transplantation (HSCT) and the upcoming gene therapies...
April 19, 2024: Indian Journal of Pediatrics
https://read.qxmd.com/read/38639671/allogeneic-transplantation-of-bone-marrow-versus-peripheral-blood-stem-cells-from-hla-identical-sibling-donors-for-hematological-malignancies-in-6064-adults-from-2003-to-2020-different-impacts-on-survival-according-to-time-period
#4
JOURNAL ARTICLE
Takaaki Konuma, Kotaro Miyao, Hideki Nakasone, Fumihiko Ouchi, Takahiro Fukuda, Masatsugu Tanaka, Yukiyasu Ozawa, Shuichi Ota, Toshiro Kawakita, Naoyuki Uchida, Masashi Sawa, Yuta Katayama, Nobuhiro Hiramoto, Tetsuya Eto, Tatsuo Ichinohe, Yoshiko Atsuta, Junya Kanda
BACKGROUND: Mobilized peripheral blood stem cells (PBSC) have been widely used instead of bone marrow (BM) as the graft source for allogeneic hematopoietic cell transplantation (HCT). Although early studies demonstrated no significant differences in survival between PBSC transplantation (PBSCT) and BM transplantation (BMT) from human leukocyte antigen (HLA)-identical sibling donors to adults with hematological malignancies, recent results have been unclear. OBJECTIVE: The objective of this retrospective study was to compare overall survival (OS), relapse, non-relapse mortality (NRM), hematopoietic recovery and graft-versus-host disease (GVHD) between PBSCT and BMT according to the time period of HCT (2003-2008, 2009-2014, or 2015-2020)...
March 30, 2024: Cytotherapy
https://read.qxmd.com/read/38639421/germline-assessment-for-allohsct-candidates-over-50%C3%A2-years-a-fast-track-screening-in-myeloid-neoplasms
#5
JOURNAL ARTICLE
Sara Torres-Esquius, Francisco Beas, Tzu Hua Chen-Liang, Helena Pomares, Marta Santiago, Nicolás Díaz Varela, Alessandro Liquori, Francisca Hernandez, Blanca Xicoy, Lourdes Hermosín, Montserrat Arnan, Bárbara Tazón-Vega, Adoración Blanco, José Cervera, María Diez-Campelo, María Luisa Lozano, David Valcárcel, Francesc Bosch, Maria Julia Montoro, Andrés Jerez
Patients aged 50 or above diagnosed with myeloid neoplasms (MNs) are typically not candidates for germline testing. However, approximately 8% carry pathogenic germline variants. Allogeneic haematopoietic stem cell transplantation (alloHSCT) remains an option for those aged over 50; neglecting germline testing could mask the risk for relative donor cell-derived MN. We propose a germline-augmented somatic panel (GASP), combining MN predisposition genes with a myeloid somatic panel for timely germline variant identification when initial testing is not indicated...
April 19, 2024: British Journal of Haematology
https://read.qxmd.com/read/38639015/an-update-on-stem-cell-therapy-for-stroke-patients-where-are-we-now
#6
REVIEW
Jonah Gordon, Cesar V Borlongan
With a foundation built upon initial work from the 1980s demonstrating graft viability in cerebral ischemia, stem cell transplantation has shown immense promise in promoting survival, enhancing neuroprotection and inducing neuroregeneration, while mitigating both histological and behavioral deficits that frequently accompany ischemic stroke. These findings have led to a number of clinical trials that have thoroughly supported a strong safety profile for stem cell therapy in patients but have generated variable efficacy...
April 19, 2024: Journal of Cerebral Blood Flow and Metabolism
https://read.qxmd.com/read/38638843/daratumumab%C3%A2-resistant-multiple-myeloma-with-extramedullary-disease-successfully-treated-with-combination-elotuzumab-pomalidomide-and-dexamethasone-a-case-report
#7
Masataka Sakashita, Naohi Sahara, Jun Aoki, Takashi Matsunaga, Seiichiro Kobayashi, Shinsuke Kitahara, Tomoki Fujii, Nobuhiro Ohno
Despite the emergence of monoclonal antibodies, the prognosis of patients with multiple myeloma (MM) with extramedullary disease remains poor. The present report describes a rare case of daratumumab-refractory MM that was successfully treated with elotuzumab, pomalidomide and dexamethasone. A 66-year-old male patient diagnosed with MM was treated with bortezomib, lenalidomide and dexamethasone, followed by high-dose chemotherapy and autologous stem cell transplantation. Thereafter, the patient was treated with lenalidomide and dexamethasone as maintenance therapy...
June 2024: Oncology Letters
https://read.qxmd.com/read/38638371/analysis-of-factors-affecting-hematopoietic-stem-cell-mobilization-efficiency-and-early-hematopoietic-reconstruction-indicators-during-autologous-peripheral-blood-hematopoietic-stem-cell-transplantation
#8
REVIEW
Hao Shi, Yaya Duan, Xinting Bu
Purpose  To analyze the factors affecting the mobilization efficiency of hematopoietic stem cells and hematopoietic reconstruction indicators during autologous peripheral hematopoietic stem cell transplantation. Methods  The clinical data of 54 patients who underwent autologous peripheral blood hematopoietic stem cell mobilization and transplantation at Xuzhou Central Hospital from May 2016 to April 2023 were retrospectively analyzed. The gender, age, disease type, mobilization regimen, number of chemotherapy sessions, G-CSF (granulocyte colony-stimulating factor) dosage, and platelet number at the time of collection were also collected...
June 2024: Global medical genetics
https://read.qxmd.com/read/38637875/pleuroparenchymal-fibroelastosis-as-a-late-complication-of-childhood-cancer-therapy-a-case-series
#9
JOURNAL ARTICLE
Priya H Marathe, Valeria Santibanez, Paul A Meyers, Maria L Padilla, Danielle N Friedman
Pleuroparenchymal fibroelastosis (PPFE) is a rare interstitial pneumonia with distinct clinicopathologic features. It has been associated with exposure to hematopoietic stem cell transplantation (HSCT) and classical alkylating agents. Here, we highlight PPFE as a late complication of childhood cancer therapy by describing the cases of four survivors of childhood cancer with a diagnosis of treatment-related PPFE. All patients received high-dose alkylating agents. PPFE should be considered in the differential diagnosis of restrictive lung disease in patients with history of exposure to alkylating agents or HSCT...
April 18, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38637559/caspase-8-deletion-causes-infection-inflammation-induced-bone-marrow-failure-and-mds-like-disease-in-mice
#10
JOURNAL ARTICLE
Shanhui Liu, Kanak Joshi, Lei Zhang, Wenyan Li, Ryan Mack, Austin Runde, Patrick A Hagen, Kevin Barton, Peter Breslin, Hong-Long Ji, Ameet R Kini, Zhiping Wang, Jiwang Zhang
Myelodysplastic syndromes (MDS) are a heterogeneous group of pre-leukemic hematopoietic disorders characterized by cytopenia in peripheral blood due to ineffective hematopoiesis and normo- or hypercellularity and morphologic dysplasia in bone marrow (BM). An inflammatory BM microenvironment and programmed cell death of hematopoietic stem/progenitor cells (HSPCs) are thought to be the major causes of ineffective hematopoiesis in MDS. Pyroptosis, apoptosis and necroptosis (collectively, PANoptosis) are observed in BM tissues of MDS patients, suggesting an important role of PANoptosis in MDS pathogenesis...
April 18, 2024: Cell Death & Disease
https://read.qxmd.com/read/38637498/antiviral-cellular-therapy-for-enhancing-t-cell-reconstitution-before-or-after-hematopoietic-stem-cell-transplantation-aces-a-two-arm-open-label-phase-ii-interventional-trial-of-pediatric-patients-with-risk-factor-assessment
#11
JOURNAL ARTICLE
Michael D Keller, Patrick J Hanley, Yueh-Yun Chi, Paibel Aguayo-Hiraldo, Christopher C Dvorak, Michael R Verneris, Donald B Kohn, Sung-Yun Pai, Blachy J Dávila Saldaña, Benjamin Hanisch, Troy C Quigg, Roberta H Adams, Ann Dahlberg, Shanmuganathan Chandrakasan, Hasibul Hasan, Jemily Malvar, Mariah A Jensen-Wachspress, Christopher A Lazarski, Gelina Sani, John M Idso, Haili Lang, Pamela Chansky, Chase D McCann, Jay Tanna, Allistair A Abraham, Jennifer L Webb, Abeer Shibli, Amy K Keating, Prakash Satwani, Pawel Muranski, Erin Hall, Michael J Eckrich, Evan Shereck, Holly Miller, Ewelina Mamcarz, Rajni Agarwal, Satiro N De Oliveira, Mark T Vander Lugt, Christen L Ebens, Victor M Aquino, Jeffrey J Bednarski, Julia Chu, Suhag Parikh, Jennifer Whangbo, Michail Lionakis, Elias T Zambidis, Elizabeth Gourdine, Catherine M Bollard, Michael A Pulsipher
Viral infections remain a major risk in immunocompromised pediatric patients, and virus-specific T cell (VST) therapy has been successful for treatment of refractory viral infections in prior studies. We performed a phase II multicenter study (NCT03475212) for the treatment of pediatric patients with inborn errors of immunity and/or post allogeneic hematopoietic stem cell transplant with refractory viral infections using partially-HLA matched VSTs targeting cytomegalovirus, Epstein-Barr virus, or adenovirus...
April 18, 2024: Nature Communications
https://read.qxmd.com/read/38637333/psychological-and-physical-side-effects-during-g-csf-mobilization-in-related-donors-of-allo-hct
#12
JOURNAL ARTICLE
Lingxia Cai, Diange Jin, Jianbo Lai, Lin Li, Yi Luo, Jimin Shi, Xiaoyu Lai, Lizhen Liu, Yanmin Zhao, Jian Yu, Yunfei Qiu, Kaixia Song, Fangquan Yu, Qinna Guo, Aiyun Jin, He Huang, Shuyi Ding, Yishan Ye
The psychological side effects of granulocyte colony-stimulating factor mobilization in related donors of allogeneic hematopoietic cell transplantation (allo-HCT) and impacts of psychological/physical side effects on harvest outcomes remain largely unknown. We prospectively analyzed 349 consecutive related peripheral blood stem cell (PBSC) donors for allo-HCT at the First Affiliated Hospital, Zhejiang University, School of Medicine from March 2021 to August 2023. Higher baseline peripheral blood white blood cell counts (p = 0...
April 19, 2024: Annals of Hematology
https://read.qxmd.com/read/38637310/posaconazole-in-paediatric-malignancy-and-haematopoietic-stem-cell-transplant-dosing-to-achieve-therapeutic-concentration
#13
JOURNAL ARTICLE
Heather Weerdenburg, Hannah Walker, Nigel Curtis, Stephen Duffull, Gabrielle Haeusler, Theresa Cole, Amanda Gwee
OBJECTIVES: Posaconazole is increasingly used for the treatment and prophylaxis of invasive fungal infections in immunocompromised children. We aimed to review evidence for paediatric posaconazole dosing regimens focusing on attainment of target concentrations and frequency of adverse effects. METHODS: In May 2023, the Cochrane, Embase, MEDLINE and PubMed databases were searched for articles reporting posaconazole dosing in children with malignancy or post-haematopoietic stem cell transplantation...
April 18, 2024: Journal of Antimicrobial Chemotherapy
https://read.qxmd.com/read/38636998/-senolytic-effects-on-dental-pulp-stem-cell-s-proliferation-and-differentiation-during-long-term-expansion
#14
JOURNAL ARTICLE
G Y Wang, L Liao, W D Tian
Objective: To investigate the impact of intermittent senescent cell clearance on the proliferation and differentiation of dental pulp stem cells (DPSC) in long-term, large-scale expansion, and to explore strategies for maintaining the youthful state of DPSC in vitro. Methods: Human-derived dental pulp stem cells were isolated from healthy permanent teeth extracted for orthodontic or impeding eruption reasons, provided by the Department of Oral and Maxillofacial Surgery at West China Hospital of Stomatology, Sichuan University...
April 18, 2024: Zhonghua Kou Qiang Yi Xue za Zhi, Zhonghua Kouqiang Yixue Zazhi, Chinese Journal of Stomatology
https://read.qxmd.com/read/38636307/extracorporeal-membrane-oxygenation-ecmo-during-aplasia-a-bridge-towards-myopericarditis-recovery-after-autologous-hematopoietic-stem-cell-transplant-for-systemic-sclerosis-and-recent-coronarovirus-disease-covid-19-vaccination
#15
JOURNAL ARTICLE
Carlotta Cacciatore, Mathilde Baudet, Estelle Jean, Simona Presente, Marylou Para, Romain Sonneville, Dimitri Arangalage, Nassim Ait Abdallah, Flore Sicre de Fontbrune, Pedro Henrique Prata, Benjamin Crichi, Baptiste Hervier, Nathalie Parquet, Gilles Soulat, Elie Mousseaux, Richard K Burt, Dominique Farge
Systemic sclerosis (SSc) is a rare autoimmune disease (AD), characterised by early diffuse vasculopathy, activation of the immune response and progressive skin and internal organ fibrosis. In severe progressive diffuse SSc (dSSc), autologous hematopoietic stem cell transplantation (aHSCT) improves survival, despite its own risk of complications and transplant related mortality (TRM). We present herein the case of a dSSc patient undergoing aHSCT with low dose cyclophosphamide conditioning and sudden acute myopericarditis and cardiogenic shock, four weeks after a second mRNA SARS-CoV-2 vaccine (Pfizer) injection...
April 9, 2024: Current Research in Translational Medicine
https://read.qxmd.com/read/38635938/clonal-hematopoiesis-and-therapy-related-myeloid-neoplasms-after-autologous-transplant-for-hodgkin-lymphoma
#16
JOURNAL ARTICLE
Chengcheng Yan, Melissa A Richard, Christopher J Gibson, Jianbo He, Alysia Bosworth, David K Crossman, Purnima Singh, Lindsey Hageman, Rashi Kalra, Saro H Armenian, Julie Vose, Daniel J Weisdorf, Benjamin L Ebert, Yutaka Yasui, Stephen J Forman, Ravi Bhatia, Smita Bhatia
PURPOSE: Therapy-related myeloid neoplasm (t-MN) is a life-threatening complication of autologous peripheral blood stem cell transplantation (aPBSCT) for Hodgkin lymphoma (HL). Although previous studies have reported an association between clonal hematopoiesis (CH) in the infused PBSC product and subsequent post-aPBSCT risk of t-MN in patients with non-HL, information about patients with HL treated with aPBSCT is not available. METHODS: We constructed a retrospective cohort of 321 patients with HL transplanted at a median age of 34 years (range, 18-71)...
April 18, 2024: Journal of Clinical Oncology
https://read.qxmd.com/read/38635290/coadministration-of-fluconazole-to-boost-subtherapeutic-sirolimus-concentrations-a-case-report
#17
JOURNAL ARTICLE
Camilo Scherkl, Andreas D Meid, Sven E Cuntz, Laura Classen, Johanna Weiss, David Czock, Walter E Haefeli
Individual sirolimus whole blood concentrations are highly variable, critically influenced by the concomitant use of cytochrome P450 (CYP) 3A inducers or inhibitors, and also modulated by food. Therapeutic drug monitoring is therefore recommended, especially at treatment start or in circumstances that can influence sirolimus exposure. In this case report, we highlight the challenge of achieving therapeutic sirolimus concentrations and present pragmatic solutions with regimen adaptions, pharmacokinetic enhancement (use of a drug-drug interaction), concentration monitoring, and subsequent modeling of population pharmacokinetics to support treatment decisions...
June 2024: Pharmacology Research & Perspectives
https://read.qxmd.com/read/38634859/deep-learning-based-optimization-of-field-geometry-for-total-marrow-irradiation-delivered-with-volumetric-modulated-arc-therapy
#18
JOURNAL ARTICLE
Nicola Lambri, Giorgio Longari, Daniele Loiacono, Ricardo Coimbra Brioso, Leonardo Crespi, Carmela Galdieri, Francesca Lobefalo, Giacomo Reggiori, Roberto Rusconi, Stefano Tomatis, Luisa Bellu, Stefania Bramanti, Elena Clerici, Chiara De Philippis, Damiano Dei, Pierina Navarria, Carmelo Carlo-Stella, Ciro Franzese, Marta Scorsetti, Pietro Mancosu
BACKGROUND: Total marrow (lymphoid) irradiation (TMI/TMLI) is a radiotherapy treatment used to selectively target the bone marrow and lymph nodes in conditioning regimens for allogeneic hematopoietic stem cell transplantation. A complex field geometry is needed to cover the large planning target volume (PTV) of TMI/TMLI with volumetric modulated arc therapy (VMAT). Five isocenters and ten overlapping fields are needed for the upper body, while, for patients with large anatomical conformation, two specific isocenters are placed on the arms...
April 18, 2024: Medical Physics
https://read.qxmd.com/read/38634819/-correction-to-the-essence-of-quiescence-by-peter-quesenberry-et-al-stem-cells-dev-2024-33-7-8-149-152-doi-10-1089-scd-2024-0032
#19
(no author information available yet)
No abstract text is available yet for this article.
April 17, 2024: Stem Cells and Development
https://read.qxmd.com/read/38634440/overcoming-methodological-challenges-for-advancing-stem-cell-therapies-in-parkinson-s-disease
#20
REVIEW
Stephen Polgar, David I Finkelstein, Leila Karimi
The quest for new and improved therapies for Parkinson's disease (PD) remains of paramount importance, despite previous trial failures. There is a current debate regarding the potential of stem cell research as a therapeutic approach for PD. The studies of dopaminergic fetal stem cells for PD treatment, their design, and the results of the initial surgical placebo-controlled trials were reviewed in this study. Some of the fundamental methodological challenges and possible strategies to resolve them were proposed...
2024: Cell Transplantation
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