Article
Immunology
Lilli Leimi, Jessica R. Koski, Outi Kilpivaara, Kim Vettenranta, A. Inkeri Lokki, Seppo Meri
Summary: This study investigated genetic complement system variants in patients with documented endotheliopathy and found that variants in complement genes may be related to endotheliopathy and poor prognosis in these patients.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Pediatrics
Hilda Mekelenkamp, Teija Schroder, Eugenia Trigoso, Daphna Hutt, Jacques-Emmanuel Galimard, Anne Kozijn, Arnaud Dalissier, Marjola Gjergji, Sarah Liptrott, Michelle Kenyon, John Murray, Selim Corbacioglu, Peter Bader
Summary: This study investigated the place and cause of death of transplanted pediatric HSCT patients and the availability of specialized pediatric palliative care services (SPPCS) through database analysis and a survey of HSCT professionals. The majority of patients died in hospitals, and while professionals recognized the importance of SPPCS for HSCT patients, referral rates were low.
Article
Pharmacology & Pharmacy
Su-jie Jia, Ke-qin Gao, Pan-hao Huang, Ren Guo, Xiao-cong Zuo, Qing Xia, Shuang-yao Hu, Zhen Yu, Yue-liang Xie
Summary: Glucocorticoids reduced C-min/dose levels of VRC and different SNPs of CYP450 have varied effects on the C-min/dose ratio of VRC. Glucocorticoids and CYP2C19*17 mutants had a synergistic effect in reducing VRC C-min/dose. The clinical efficacy of VRC should be carefully monitored, especially in the presence of CYP2C19*17 mutants when combined with glucocorticoids.
FRONTIERS IN PHARMACOLOGY
(2021)
Article
Oncology
Veronika Schneider, Karin M. Cabanillas Stanchi, Karina Althaus, Sarah Schober, Sebastian Michaelis, Christian Seitz, Peter Lang, Rupert Handgretinger, Tamam Bakchoul, Stefanie Hammer, Michaela Doering
Summary: This study aimed to use thrombelastography to detect signs of hypofibrinolysis in pediatric patients undergoing hematopoietic stem cell transplantation (HSCT). The results showed prolonged clot lysis and reduced maximum lysis in patients with veno-occlusive disease (VOD) compared to other HSCT patients and a control group. Thrombelastography might serve as a parameter to support the diagnosis and follow-up of VOD.
JOURNAL OF CANCER RESEARCH AND CLINICAL ONCOLOGY
(2023)
Article
Pharmacology & Pharmacy
Jordan T. Brooks, Ron J. Keizer, Janel R. Long-Boyle, Sandhya Kharbanda, Christopher C. Dvorak, Brian D. Friend
Summary: This study aimed to develop a PopPK model for IV continuous infusion of tacrolimus in the pediatric and young adult HCT population, and implement the model in a Bayesian dosing tool to enable individualized dosing and optimize clinical utilization of tacrolimus.
FRONTIERS IN PHARMACOLOGY
(2021)
Review
Medicine, General & Internal
Mattia Algeri, Pietro Merli, Franco Locatelli, Daria Pagliara
Summary: Allogeneic hematopoietic stem cell transplantation (HSCT) provides a potentially curative treatment for high-risk or relapsed acute leukemia in children by combining intense preparative radio/chemotherapy with the graft-versus-leukemia (GvL) effect. Advances in donor typing, conditioning regimens, GvHD prophylaxis, and MRD-directed interventions have continuously improved transplant outcomes, expanding the potential application of allotransplantation to most patients. Simultaneously, refinements in chemotherapy protocols and targeted therapies may redefine the indications for HSCT in the future.
JOURNAL OF CLINICAL MEDICINE
(2021)
Article
Medicine, General & Internal
Jiayu Wang, Meng Yuan, Guanghua Zhu, Runhui Wu, Chenguang Jia, Bin Wang, Jie Zheng, Jie Ma, Maoquan Qin, Sidan Li
Summary: This study analyzed the immune reconstitution of pediatric patients with aplastic anemia after hematopoietic stem-cell transplantation (HSCT) and found that early rapid immune reconstitution is a good predictor of clinical prognosis in children with AA.
INTERNATIONAL JOURNAL OF MEDICAL SCIENCES
(2022)
Article
Virology
Estela Gimenez, Carolina Monzo, Eliseo Albert, Azahara Fuentes-Trillo, Enrique Seda, Jose Luis Pinana, Juan Carlos Hernandez Boluda, Carlos Solano, Javier Chaves, David Navarro
Summary: Monitoring alphatorquevirus (TTV) DNA in plasma may help assess immune competence after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The study found co-detection of TTV, TTMV, and TTMDV in more than two-thirds of patients in pre-transplant and post-engraftment plasma specimens. Targeting TTMV and TTMDV with quantitative real-time PCR assays may provide additional value in assessing immune competence in this clinical setting.
JOURNAL OF MEDICAL VIROLOGY
(2021)
Review
Cell & Tissue Engineering
Davod Pashoutan Sarvar, Hosein Effatpanah, Parvin Akbarzadehlaleh, Karim Shamsasenjan
Summary: The study highlights the important role of MSC-derived extracellular vesicles (MSC-EVs) in hematopoietic regulation and immune response after transplantation. MSC-EVs directly fuse with hematopoietic stem cell membranes, affecting their fate, and regulate the proliferation and differentiation of hematopoietic stem cells through the release of various bioactive factors.
STEM CELL RESEARCH & THERAPY
(2022)
Article
Oncology
Hyewon Shin, Mi-Sun Yum, Min-Jee Kim, Jin Kyung Suh, Ho Joon Im, Hyery Kim, Kyung-Nam Koh, Tae-Sung Ko
Summary: Most children with neurologic complications and neuroimaging abnormalities during HSCT had recovered. However, children with neurologic complications associated with infectious causes, malignant disease or severe brain MRI abnormalities should be more carefully monitored during HSCT.
Article
Surgery
Mattia Algeri, Enrico Velardi, Marco Spada, Federica Galaverna, Roberto Carta, Luciana Vinti, Giuseppe Palumbo, Stefania Gaspari, Andrea Pietrobattista, Emilia Boccieri, Marco Becilli, Paola Francalanci, Valentina Bertaina, Pietro Merli, Franco Locatelli
Summary: This report describes a case of operational tolerance in a patient who received combined liver transplantation and hematopoietic stem cell transplantation (HSCT) from different donors, without causing any sign of liver graft dysfunction.
AMERICAN JOURNAL OF TRANSPLANTATION
(2023)
Review
Immunology
Tirthankar Sen, Rajkumar P. Thummer
Summary: The human microbiota plays a significant role in organ transplantation outcomes and rejection risk, with complex interactions existing between different microbiotas and the human body.
FRONTIERS IN IMMUNOLOGY
(2022)
Review
Biotechnology & Applied Microbiology
Soheila Montazersaheb, Ali Ehsani, Ezzatollah Fathi, Raheleh Farahzadi, Ilja Vietor
Summary: Autophagy is a fundamental process that helps cells adapt to metabolic stress by degrading and recycling intracellular components. It plays a crucial role in various aspects of allogeneic hematopoietic stem cell transplantation (HSCT), including the survival and function of hematopoietic stem cells, T-cell differentiation, antigen presentation, and responsiveness to cytokine stimulation. However, the overall role of autophagy in HSCT remains unclear, highlighting the need for further understanding to improve transplant outcomes.
FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY
(2022)
Article
Immunology
Ashley M. Nelson, Alexandra A. Erdmann, Christopher L. Coe, Mark B. Juckett, Keayra Morris, Jennifer M. Knight, Peiman Hematti, Erin S. Costanzo
Summary: Increased synthesis and release of inflammatory signalling proteins are common among individuals with hematologic malignancies undergoing hematopoietic cell transplantation (HCT). This study examined the relationship between markers of inflammatory activity and depression symptoms following HCT. The results showed that patients with elevated levels of IL-6 and IL-10 reported more severe depression symptoms post-HCT, especially in terms of neurovegetative symptoms.
BRAIN BEHAVIOR AND IMMUNITY
(2023)
Article
Cell & Tissue Engineering
Rashin Mohseni, Pouya Mahdavi Sharif, Maryam Behfar, Mohammad Reza Modaresi, Rohola Shirzadi, Mahta Mardani, Leila Jafari, Fahimeh Jafari, Zeynab Nikfetrat, Amir Ali Hamidieh
Summary: This study aimed to assess the safety and effectiveness of adipose tissue-derived mesenchymal stem cells (AT-MSCs) for pediatric patients with bronchiolitis obliterans syndrome (BoS) after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The results showed that a single injection of AT-MSCs partially improved the symptoms of patients and the treatment was safe and tolerable.
STEM CELL RESEARCH & THERAPY
(2023)
Article
Oncology
Eward J. Melis, Jeannette E. F. Zwart-van Rijkom, Toine C. G. Egberts, Bart J. F. van den Bemt, Petronella O. Witteveen, Helga Gardarsdottir
Summary: This study assesses the association between patient satisfaction with information about oral anticancer agents and adherence. The results showed no significant relationship between patient satisfaction with information and adherence, despite the high level of satisfaction with the information. However, 35.5% of patients were found to be non-adherent based on refill data.
JOURNAL OF ONCOLOGY PHARMACY PRACTICE
(2023)
Review
Medicine, General & Internal
Lenneke Minjon, Juul W. Aarts, Els van den Ban, Toine C. G. Egberts, Eibert R. Heerdink
Summary: This study assessed the clarity and applicability of adverse drug reaction (ADR)-related monitoring instructions in clinical practice guidelines (CPGs) for children and adolescents treated with antipsychotic drugs. The results showed that the presentation of monitoring instructions was clear in different CPGs, but improvement in their applicability is necessary. There were variations in the parameters that needed to be monitored among CPGs.
Review
Computer Science, Information Systems
Britt W. M. van de Burgt, Arthur T. M. Wasylewicz, Bjorn Dullemond, Rene J. E. Grouls, Toine C. G. Egberts, Arthur Bouwman, Erik M. M. Korsten
Summary: This review examines the use of text mining and clinical decision support in clinical practice, discussing their potential benefits, implementation challenges, and barriers. The findings suggest that combining text mining and clinical decision support can improve diagnostic and therapeutic processes, leading to increased patient safety. However, there are still barriers to implementation that need to be addressed through further research.
JOURNAL OF THE AMERICAN MEDICAL INFORMATICS ASSOCIATION
(2023)
Article
Genetics & Heredity
Corine de Jong, Gerarda J. M. Herder, Simone W. A. van Haarlem, Femke S. van der Meer, Anne S. R. van Lindert, Alexandra ten Heuvel, Jan Brouwer, Toine C. G. Egberts, Vera H. M. Deneer
Summary: This study investigated the genetic risk factors for chemotherapy-induced peripheral neuropathy (CIPN) in non-small cell lung cancer (NSCLC) patients. It revealed that the GG genotype of TRPV1 gene (rs879207) is associated with an increased risk of severe neuropathy. Additionally, concomitant use of paclitaxel and GG genotype also showed a significant association with severe CIPN.
Review
Pharmacology & Pharmacy
Daan W. Huntjens, Jacob A. A. Dijkstra, Lisanne N. N. Verwiel, Mirjam Slijkhuis, Paul Elbers, Matthijs R. A. Welkers, Agnes I. I. Veldkamp, Marianne A. A. Kuijvenhoven, David C. C. de Leeuw, Heshu Abdullah-Koolmees, Maria T. T. Kuipers, Imke H. H. Bartelink
Summary: HSV and CMV infections pose a higher risk to severely immunocompromised patients. Antiviral therapy for these patients is challenging due to drug variability and comorbidities. This review explores the current knowledge of antiviral drug pharmacokinetics and pharmacodynamics, discusses treatment guidelines, and proposes interventions like drug resistance detection, therapeutic drug monitoring, introduction of new drugs, alternate drug use, and combination therapy.
Editorial Material
Hematology
Jaap Jan Boelens, Andromachi Scaradavou
Article
Biophysics
T. (Tim) Bognar, J. S. (Jurjen) Kingma, E. H. (Erin) Smeijsters, K. C. M. (Kim) van der Elst, C. T. M. (Klaartje) de Kanter, C. A. (Caroline) Lindemans, A. C. G. (Toine) Egberts, I. H. (Imke) Bartelink, A. (Arief) Lalmohamed
Summary: This observational study assessed the impact of therapeutic drug monitoring on busulfan exposure in pediatric patients undergoing hematopoietic cell transplantation. The results showed no significant difference in achieving therapeutic busulfan exposure between the two monitoring regimens, suggesting that day 1-guided dosing may be sufficient with subsequent monitoring if needed.
BONE MARROW TRANSPLANTATION
(2023)
Article
Pharmacology & Pharmacy
Renske C. F. Hebing, Imke H. Bartelink, Helen R. Gosselt, Sandra G. Heil, Mauritis C. F. J. de Rotte, Pascal H. P. de Jong, Mike T. Nurmohamed, Robert de Jonge, Ron A. A. Mathot
Summary: This study investigated the association between red blood cell methotrexate polyglutamates (MTX-PG) concentrations and response in rheumatoid arthritis patients starting methotrexate (MTX) therapy. The results showed that there was an association between RBC-MTX-PG concentrations and Disease Activity Score in 28 joints (DAS28). Based on the findings, it is suggested to increase the dose if MTX-PG concentration is below 9.15 nmol/L at month 1, continue with the same dose if the concentration is above 47 nmol/L, and consider other treatment options if the concentration is above 78 nmol/L from 3 months onwards.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(2023)
Article
Immunology
Marjon V. Verschueren, Talitha Dijs, Judith L. Gulikers, Ard van Veelen, Sander Croes, Lizza E. L. Hendriks, Adrianus A. J. Smit, Lourens T. Bloem, Antoine C. G. Egberts, Ewoudt M. W. van de Garde, Bas J. M. Peters
Summary: Real-world stage III NSCLC patients who received durvalumab after CRT had better outcomes than those who received CRT alone. Longer PFS in real-world versus trial may be due to follow-up differences.
Article
Medicine, General & Internal
K. Hakki Karagozoglu, Anissa Mahraoui, Joseph C. J. Bot, Seunghee Cha, Jean-Pierre T. F. Ho, Marco N. Helder, Henk S. Brand, Imke H. Bartelink, Arjan Vissink, Gary A. Weisman, Derk Hendrik Jan Jager
Summary: Through sialendoscopy, CEUSS was tested for its safety and feasibility in SS patients. The results showed that CEUSS is a safe and feasible treatment that has the potential to increase salivary secretion and reduce xerostomia in SS patients.
JOURNAL OF CLINICAL MEDICINE
(2023)
Editorial Material
Oncology
Medhat M. Said, Martinus P. G. Broen, Eleonora L. L. Swart, Imke H. H. Bartelink, Mathilde C. M. Kouwenhoven
Article
Hematology
Anouk A. M. T. Donners, Konrad van der Zwet, Carin M. A. Rademaker, Toine C. G. Egberts, Roger E. G. Schutgens, Kathelijn Fischer
Summary: This study evaluated the efficacy of entire-vial dosing of emicizumab in a real-world setting. The results demonstrated that this dosing method can achieve therapeutic plasma concentrations, effectively control bleeding, and reduce drug waste.
RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Biophysics
Alexandra M. Satty, Elizabeth Klein, Audrey Mauguen, Binni Kunvarjee, Jaap Jan Boelens, Maria Cancio, Kevin J. Curran, Nancy A. Kernan, Susan E. Prockop, Andromachi Scaradavou, Barbara Spitzer, Roni Tamari, Julianne Ruggiero, Joanne Torok-Castanza, Parinda A. Mehta, Richard J. O'Reilly, Farid Boulad
Summary: The study retrospectively evaluated the outcomes of patients with Fanconi anemia and MDS/AML who underwent allogeneic HCT with a T-cell depleted graft. The results demonstrated promising outcomes, particularly in a cohort of high-risk patients, with a high survival rate.
BONE MARROW TRANSPLANTATION
(2023)
Article
Medicine, General & Internal
Akshay Sharma, Jaap-Jan Boelens, Maria Cancio, Jane S. Hankins, Prafulla Bhad, Marjohn Azizy, Andrew Lewandowski, Xiaojun Zhao, Shripad Chitnis, Radhika Peddinti, Yan Zheng, Neena Kapoor, Fabio Ciceri, Timothy Maclachlan, Yi Yang, Yi Liu, Jianping Yuan, Ulrike Naumann, Vionnie W. C. Yu, Susan C. Stevenson, Serena De Vita, James L. LaBelle
Summary: Sickle cell disease is caused by a mutation in beta-globin subunit of adult hemoglobin leading to hemolysis and vaso-occlusion. Elevated fetal hemoglobin levels can protect against disease complications. CRISPR-Cas9 technology was used to increase fetal hemoglobin expression in red cells, showing clinical improvement in patients with sickle cell disease.
NEW ENGLAND JOURNAL OF MEDICINE
(2023)
Article
Hematology
Ellen Fraint, Sana Farooki, Elizabeth Klein, Audrey Mauguen, Susan E. Prockop, Andromachi Scaradavou, Kevin Curran, Maria Cancio, Barbara Spitzer, Jaap Jan Boelens, Joseph Oved, Andrew Harris, Richard J. O'Reilly, Nancy A. Kernan
Summary: Poor graft function is a serious complication after allogeneic stem cell transplantation. There are no standardized approaches to treatment and outcomes have been historically poor. This study reports on the use of CD34-selected stem cell boost for the treatment of poor graft function in pediatric patients, showing favorable outcomes in terms of hematopoietic recovery and long-term survival.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)