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allogeneic hematopoietic stem cell transplantation

Hongshuang Yu, Yuanyuan Tian, Ying Wang, Shin Mineishi, Yi Zhang
Graft-vs.-host disease (GVHD) remains a significant cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Significant progresses have been made in defining the dichotomous role of dendritic cells (DCs) in the development of GVHD. Host-derived DCs are important to elicit allogeneic T cell responses, whereas certain donor-types of DCs derived from newly engrafted hematopoietic stem/progenitor cells (HSPCs) can amply this graft-vs.-host reaction. In contrast, some DCs also play non-redundant roles in mediating immune tolerance...
2019: Frontiers in Immunology
Arghavan Etebarian, Hamid Mirshamsi, Hanieh Sadat Sadeghi, Fatemeh Hemmati
Acute myeloid leukemia is a bone marrow malignancy in which blasts count increases by more than 20% in the bone marrow. Allogeneic hematopoietic stem cell transplantation (alloHCT) is a treatment option for these patients with high risk of graft versus host disease (GVHD) development. Chronic GVHD (cGVHD) often mimics a variety of autoimmune conditions such as systemic lupus erythematous or systemic sclerosis. Sclerotic cGVHD has a wide spectrum of oral manifestations, including mucosal atrophy, microstomia, and hyposalivation...
2019: Quintessence International
Dao-Xing Deng, Hong-Hu Zhu, Yan-Rong Liu, Ying-Jun Chang, Guo-Rui Ruan, Jin-Song Jia, Hao Jiang, Qian Jiang, Xiao-Su Zhao, Xiao-Jun Huang
Acute myeloid leukemia (AML) patients with biallelic CEBPA (bi CEBPA) mutations are considered prognostically favorable, but 38-58% of them still relapse. Therefore, recognizing patients with a high risk of relapse is important. We retrospectively analyzed 83 bi CEBPA AML. Minimal residual disease (MRD) was detected by multiparameter flow cytometry (MFC). Patients with MRD positivity during consolidation chemotherapy had inferior 3-year CIR (55% vs. 36.7%; p = .037) and RFS (45% vs. 63.3%; p = .037) than those with MRD negativity...
February 18, 2019: Leukemia & Lymphoma
Madeleine Taylor, Shaukat Khan, Molly Stapleton, Jianmin Wang, Jing Chen, Robert Wynn, Hiromasa Yabe, Yasutsugu Chinen, Jaap Jan Boelens, Robert W Mason, Francyne Kubaski, Dafne D G Horovitz, Anneliese L Barth, Marta Serafini, Maria Ester Bernardo, Hironori Kobayashi, Kenji E Orii, Yasuyuki Suzuki, Tadao Orii, Shunji Tomatsu
Allogenic hematopoietic stem cell transplantation (HSCT) has been shown to be a treatment option for a selected group of patients with mucopolysaccharidoses (MPS) (MPS I, II, IVA, VI, and VII). Early diagnosis and timely referral to an expert in MPS are critical, followed by a complete examination and evaluation with a multidisciplinary team, including a transplant physician. Treatment recommendations for MPS are based on multiple factors such as biological, sociological, and financial effects. These include type of MPS, clinical severity, prognosis, present clinical signs and symptoms (disease stage), age at onset, the rate of progression, family significances and expectations, financial burdens, feasibility, availability, risks and benefits with available therapies such as HSCT, enzyme replacement therapy (ERT), surgical interventions, and other supportive care...
February 14, 2019: Biology of Blood and Marrow Transplantation
Amandine Fayard, Elisabeth Daguenet, Didier Blaise, Patrice Chevallier, Hélène Labussière, Ana Berceanu, Ibrahim Yakoub-Agha, Gérard Socié, Amandine Charbonnier, Felipe Suarez, Anne Huynh, Mélanie Mercier, Claude-Eric Bulabois, Bruno Lioure, Sylvain Chantepie, Yves Beguin, Jean-Henri Bourhis, Jean-Valère Malfuson, Laurence Clément, Régis Peffault de la Tour, Jérôme Cornillon
Several approaches have been developed to overcome historical barriers associated with poor outcomes in the setting of HLA-haploidentical allogeneic transplantation (HaploSCT). Here, we examine the outcome of patients with various hematological disorders undergoing HaploSCT with high-dose, post-transplantation cyclophosphamide. We performed a retrospective study on 381 patients from 30 centers between January 2013 and December 2015. At the last follow-up, a total of 1058 infectious episodes were diagnosed, affecting 90...
February 15, 2019: Bone Marrow Transplantation
Diego Adrianzen Herrera, Noah Kornblum, Ana Acuna-Villaorduna, R Alejandro Sica, Urvi Shah, Moya Butler, Nivetha Vishnuvardhan, Nishi Shah, Lizamarie Bachier-Rodriguez, Olga Derman, Aditi Shastri, Ioannis Mantzaris, Amit K Verma, Ira Braunchweig, Murali Janakiram
In the United States (U.S.), Adult T-Cell Lymphoma-Leukemia (ATLL) carries a dismal prognosis and mainly affects immigrants from Human T-Cell Lymphotropic Virus 1 (HTLV-1) endemic areas. Allogeneic Hematopoietic Stem Cell Transplant (alloHSCT) can be effective and is recommended as an upfront treatment in the NCCN guidelines. We studied the barriers to alloHSCT in one of the largest ATLL populations in the U.S. Comprehensive chart and donor registry reviews were conducted for 88 ATLL patients treated at Montefiore Medical Center from 2003 to 2018...
February 12, 2019: Biology of Blood and Marrow Transplantation
Sinem Namdaroglu, Ali Hakan Kaya, Hikmettullah Batgi, Omur Kayikci, Mehmet Sinan Dal, Dicle Iskender, Merih Kizil Cakar, Emre Tekgunduz, Fevzi Altuntas
Post-transplant cyclophosphamide has become a promising medical option after allogeneic HSCT. In this study we aimed to evaluate the efficacy of cyclophosphamide and cyclosporine combination in acute and chronic graft-versus-host disease (GvHD) prophylaxis in acute myeloid leukemia (AML) cases scheduled for allogeneic hematopoietic stem cell transplantation (allo-HSCT). Retrospective analysis of data from 40 cases who underwent allogeneic HSCT under GvHD prophylaxis with cyclophosphamide and cyclosporine combination between April 2016 and August 2017 was made...
February 14, 2019: Scientific Reports
K Steinerová, P Jindra, D Lysák, M Karas
BACKGROUND: Allogeneic hematopoietic stem cell transplantation is one of the therapeutic options for patients with relapsed or refractory classic Hodgkins lymphoma (cHL). In the case of dis-ease relapse after transplant, other treatment options are still limited (for example donor lymphocyte infusion, and chemother-apy with brentuximab, bendamustine, or other agents) with uncertain outcomes in terms of patient tolerance and long-term dis-ease remission. One way to achieve remission is administration of the PD-1 inhibitor nivolumab, a PD-1 checkpoint inhibitor...
2019: Klinická Onkologie: Casopis Ceské a Slovenské Onkologické Spolecnosti
Armando Ganoza, George V Mazariegos, Ajai Khanna
PURPOSE OF REVIEW: Over the past decades, visceral transplantation has become the standard of care for patients with irreversible intestinal failure who suffer complications of total parenteral nutrition (TPN). Graft-versus-host disease (GVHD) after solid organ transplantation is a rare but often fatal complication with high mortality. GVHD after intestinal transplantation, given the large lymphoid content of the graft, is more frequent compared with other solid organs. It is a complex condition that may have varied clinical presentations...
February 11, 2019: Current Opinion in Organ Transplantation
Elifcan Aladağ, İbrahim Celalettin Haznedaroğlu
With an annual incidence of 1-2 in a million, Ph*(+) chronic myeloid leukemia (CML) is a clonal hematopoietic stem cell disease that makes myeloid neoplastic cells breed out of control. This BCR-ABL(+) myeloproliferative disease makes up about 15%-20% of all leukemia cases in adults. CML is seen more in males than females, with a rate of three to two. However, it does not show differences in prevalence in terms of age. CML consists of three clinical phases. The first one is the chronic phase, defined by rising white blood cell levels and also by myeloid proliferation and bone marrow maturation...
February 11, 2019: Turkish Journal of Medical Sciences
Nico Gagelmann, Markus Ditschkowski, Rashit Bogdanov, Swan Bredin, Marie Robin, Bruno Cassinat, Rabia Shahswar, Felicitas Thol, Michael Heuser, Gerard Socié, Dietrich Beelen, Ioanna Triviai, Anita Badbaran, Nicolaus Kröger
Allogeneic hematopoietic stem cell transplantation is curative in myelofibrosis and current prognostic scoring systems aim to select patients for transplantation. Here, we aimed to develop a prognostic score to determine prognosis after transplantation itself using clinical, molecular and transplant-specific information of a total of 361 myelofibrosis patients. Of these, 205 patients were used as a training cohort to create a clinical-molecular myelofibrosis transplant scoring system (MTSS), which was then externally validated in a cohort of 156 patients...
February 13, 2019: Blood
Krzysztof Giannopoulos
The modest successes of targeted therapies along with the curative effects of allogeneic hematopoietic stem cell transplantation (alloHSCT) in acute myeloid leukemia (AML) stimulate the development of new immunotherapies. One of the promising methods of immunotherapy is the activation of immune response by the targeting of negative control checkpoints. The two best-known inhibitory immune checkpoints are cytotoxic T-lymphocyte antigen-4 (CTLA-4) and the programmed cell death protein 1 receptor (PD-1). In AML, PD-1 expression is observed in T-cell subpopulations, including T regulatory lymphocytes...
February 12, 2019: Journal of Clinical Medicine
Nicola Gökbuget, Hervé Dombret, Sebastian Giebel, Monika Bruggemann, Michael Doubek, Robin Foà, Dieter Hoelzer, Christopher Kim, Giovanni Martinelli, Elena Parovichnikova, Alessandro Rambaldi, Josep-Maria Ribera, Marieke Schoonen, Julia M Stieglmaier, Gerhard Zugmaier, Renato Bassan
OBJECTIVES: Detectable minimal residual disease (MRD) after therapy for acute lymphoblastic leukemia (ALL) is the strongest predictor of hematologic relapse. This study evaluated outcomes of patients with B-cell precursor ALL with MRD of ≥10-4 Methods: Study population was from ALL study groups in Europe managed in national study protocols 2000-2014. MRD was measured by polymerase chain reaction or flow cytometry. Patients were age ≥15 years at initial ALL diagnosis. Patients were excluded if exposed to blinatumomab within 18 months of baseline or prior alloHSCT...
December 2019: Hematology (Amsterdam, Netherlands)
Kenneth A Fowler, Viktoria Vasilieva, Ekaterina Ivanova, Olga Rimkevich, Andrey Sokolov, Svetlana Abbasova, Eldar Kim, James M Coghill
Acute graft-versus-host disease (aGVHD) remains a barrier to the success of allogeneic hematopoietic stem cell transplantation (HSCT). Previously, we demonstrated that CC-Chemokine Receptor 7 (CCR7) is critical for aGVHD pathogenesis but dispensable for beneficial graft-versus-leukemia (GVL) responses. As a result, we evaluated a fully human anti-CCR7 blocking antibody as a new approach to prevent aGVHD in preclinical models. Here we report that antibody R707 is able to block human CCR7 signaling and function in vitro in response to its two natural ligands...
February 12, 2019: American Journal of Transplantation
Hong Xu, Ziqiang Zhu, Yiming Huang, Suzanne T Ildstad
BACKGROUND: Mobilization of hematopoietic stem cells (HSC) has become the preferred approach for HSC transplantation. AMD3100, a competitive inhibitor of C-X-C motif chemokine receptor-4 (CXCR4), has been found to be a rapid mobilizing agent. The present study evaluated approaches to optimize the product collected. METHODS: Mobilized peripheral blood mononuclear cells (mPBMC) from B6 mice were transplanted to recipient BALB/c mice conditioned with ablative or nonmyeloablative approaches...
February 4, 2019: Transplantation
Asama Lekbua, Jodie Ouahed, Amy E O'Connell, Stacy A Kahn, Jeffrey D Goldsmith, Toshihiko Imamura, Christine N Duncan, Judith R Kelsen, Elizabeth Worthey, Scott B Snapper, Samir Softic
Very early onset inflammatory bowel disease (VEO-IBD) represents a diagnostic and treatment challenge. Here we present a case of VEO-IBD secondary to a mutation in BIRC4 gene, which encodes X-linked inhibitor of apoptosis protein (XIAP), in a 17 month-old male with severe failure to thrive, intractable diarrhea and hepatosplenomegaly. Endoscopy and histology identified only mild duodenitis and ileitis, but severe pancolitis with crypt abscesses and epithelium apoptosis. Minimal improvement in symptoms was achieved with total parenteral nutrition (TPN), intravenous (IV) corticosteroids and tacrolimus, whereas induction and maintenance therapy with adalimumab led to complete remission...
February 7, 2019: Journal of Pediatric Gastroenterology and Nutrition
Xiao-Ning Gao, Ji Lin, Li-Jun Wang, Fei Li, Hong-Hua Li, Shu-Hong Wang, Wen-Rong Huang, Chun-Ji Gao, Li Yu, Dai-Hong Liu
Donor lymphocyte infusion (DLI) might be used prophylactically to reduce relapse after allogeneic hematopoietic stem cell transplantation for very high-risk leukemia/lymphoma without effective targeted therapy. To compare the safety and efficacy of prophylactic DLI for prevention of relapse after allogeneic peripheral blood stem cell transplantation from haploidentical donors (HID-SCT) and matched-sibling donors (MSD-SCT) in patients with very high-risk acute myeloid leukemia (AML), we performed a retrospective analysis in a cohort of 21 HID-SCT and 13 MSD-SCT recipients, displaying similar baseline characteristics except for donor's gender distribution...
February 12, 2019: Annals of Hematology
Nils Waldhüter, Wolfgang Köhler, Philipp G Hemmati, Christian Jehn, Rudolf Peceny, Giang L Vuong, Renate Arnold, Jörn-Sven Kühl
The adult cerebral form of X-linked adrenoleukodystrophy (ACALD), an acute inflammatory demyelinating disease, results in a rapidly progressive neurodegeneration, typically leading to severe disability or death within a few years after onset. We have treated 15 men who had developed ACALD with allogeneic hematopoietic stem cell transplantation (HSCT) from matched donors after myeloablative conditioning with busulfan and cyclophosphamide. All patients engrafted and 11 survived (estimated survival 73 ± 11%), 8 with stable cognition and 7 of them with stable motor function (estimated event-free survival 36 ± 17%)...
February 11, 2019: Journal of Inherited Metabolic Disease
Shivani Patel, Venkateswaran Vellaichamy Swaminathan, V Sr Mythili, M Sr Venkatadesikalu, Meena Sivasankaran, Dhaarani Jayaraman, R Balasubramaniam, Ramya Uppuluri, Revathi Raj
OBJECTIVE: To compare quality of life of children with thalassemia major who have undergone stem cell transplantation with those on regular transfusion. METHODS: The study included 40 children who underwent transplantation and 40 children and 20 adults on regular transfusion and iron chelation therapy. The quality of life assessment was done using the Pediatric Quality of Life Inventory 4.0 Generic Core Scale. RESULTS: The mean total summary score, psychosocial summary score and physical score was 92, 91 and 92...
December 15, 2018: Indian Pediatrics
Wendy W Pang, Agnieszka Czechowicz, Aaron C Logan, Rashmi Bhardwaj, Jessica Poyser, Christopher Y Park, Irving L Weissman, Judith A Shizuru
The myelodysplastic syndromes (MDS) represent a group of clonal disorders that result in ineffective hematopoiesis and are associated with an increased risk of transformation into acute leukemia. MDS arises from hematopoietic stem cells (HSCs); therefore, successful elimination of MDS HSCs is an important part of any curative therapy. However, current treatment options, including allogeneic hematopoietic cell transplantation (HCT), often fail to ablate disease-initiating MDS HSCs, and thus have low curative potential and high relapse rates...
February 11, 2019: Blood
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