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JOURNAL ARTICLE
Laurent Garderet, Luuk Gras, Linda Koster, Liesbeth De Wreede, Rovira Montserrat, Laure Vincent, Roland Fenk, Kamaraj Karunanithi, Dries Deeren, Martin Kaufmann, Jürgen Kuball, Hakan Ozdogu, Maria Jesus Pascual Cascon, Jakob Passweg, Adam Rye, Urpu Salmenniemi, John Snowden, Charlotte Toftmann Hansen, Xavier Leleu, Lauris Gastaud, Joanna Drozd Sokolowska, Kavita Raj, Meral Beksac, Stefan Schönland, Patrick Hayden, Donal McLornan
There is little long-term outcome data on the efficacy of autologous hematopoietic stem cell transplantation (ASCT) in light chain deposition disease (LCDD). We identified 51 LCDD patients in the EBMT registry who had undergone upfront ASCT between 1995 and 2021. The median serum creatinine was 280 μmol/L and 45% required renal replacement therapy (RRT) at time of transplant. The melphalan dose was 100mg/m2 in 23%, 140mg/m2 in 55% and 200 mg/m2 in 21%. The rate of very good partial response or better improved from 41% pre-transplant to 66% at Day +100 post-ASCT...
March 28, 2024: Haematologica
#2
JOURNAL ARTICLE
Y Wu, B L Tang, K D Song, G Y Sun, T Z Pan, A J Huang, B B Yan, X Y Zhu
Objective: To evaluated the clinical efficacy of a reduced-intensity preconditioning regimen for single non-blood-related umbilical cord blood transplantation (sUCBT) in the treatment of severe aplastic anemia (SAA) . Methods: The clinical data of 63 patients with SAA who underwent sUCBT from January 2021 to July 2023 at the Department of Hematology of the First Affiliated Hospital of USTC were retrospectively analyzed. Fifty-two patients received total body irradiation/total bone marrow irradiation (TMI) combined with fludarabine or a cyclophosphamide- conditioning regimen (non-rATG group) , while 11 patients received rabbit anti-human thymocyte immunoglobulin (rATG) combined with TMI, fludarabine, or the cyclophosphamide-conditioning regimen (rATG group) ...
January 14, 2024: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
#3
JOURNAL ARTICLE
Hanadi Rafii, Fernanda Volt, Marc Bierings, Jean-Hugues Dalle, Mouhab Ayas, Rawad Rihani, Maura Faraci, Giuseppina de Simone, Henrik Sengeloev, Jakob Passweg, Marina Cavazzana, Regis Costello, Johan Maertens, Alessandra Biffi, Jan-Erik Johansson, Juan Montoro, Gabrielle Roth Guepin, Miguel Angel Diaz, Anne Sirvent, Chantal Kenzey, Monica M Rivera Franco, Barbara Cappelli, Graziana Maria Scigliuolo, Vanderson Rocha, Annalisa Ruggeri, Antonio Risitano, Regis Peffault De Latour, Eliane Gluckman
BACKGROUND: Hematopoietic cell transplant (HCT) remains the only available curative treatment for Fanconi Anemia (FA), with particularly favorable outcomes reported after matched sibling donor (MSD) transplant. OBJECTIVES: To describe outcomes, with a special focus on late complications, in FA patients who underwent umbilical cord blood transplantation (UCBT). STUDY DESIGN: Retrospective analysis of allogeneic UCBT for FA performed between 1988 and 2021 in European Society for Blood and Marrow Transplantation (EBMT) affiliated centers...
March 5, 2024: Transplantation and cellular therapy
#4
JOURNAL ARTICLE
Keiko Tsumita, Shinsuke Takagi, Yuki Asano-Mori, Otoya Watanabe, Michiho Shindo, Kyosuke Yamaguchi, Mitsuhiro Yuasa, Kosei Kageyama, Daisuke Kaji, Yuki Taya, Aya Nishida, Kazuya Ishiwata, Hisashi Yamamoto, Hideki Araoka, Go Yamamoto, Shigeyoshi Makino, Atsushi Wake, Naoyuki Uchida, Shuichi Taniguchi, Yukako Koike
The number of umbilical cord blood transplantation (U-CBT) procedures has been growing annually, but little research has been done on long-term immune recovery after U-CBT. Infection risk is high in U-CBT recipients, and this can be partially attributed to immature immunocompetent cells in umbilical cord blood. In this study, we analyzed lymphocyte subset (LST) number to determine the long-term recovery timeline. We included 36 U-CBT and 10 unrelated bone marrow transplantation (U-BMT) recipients who survived more than 2 years after transplantation, and followed them for up to 10 years post-transplant...
February 26, 2024: International Journal of Hematology
#5
JOURNAL ARTICLE
Maddalena Migliavacca, Federica Barzaghi, Claudia Fossati, Paola M V Rancoita, Michela Gabaldo, Francesca Dionisio, Stefania Giannelli, Federica Andrea Salerio, Francesca Ferrua, Francesca Tucci, Valeria Calbi, Vera Gallo, Salvatore Recupero, Giulia Consiglieri, Roberta Pajno, Maria Sambuco, Alessio Priolo, Chiara Ferri, Vittoria Garella, Ilaria Monti, Paolo Silvani, Silvia Darin, Miriam Casiraghi, Ambra Corti, Stefano Zancan, Margherita Levi, Daniela Cesana, Filippo Carlucci, Anna Pituch-Noworolska, Dalia AbdElaziz, Ulrich Baumann, Andrea Finocchi, Caterina Cancrini, Saverio Ladogana, Andrea Meinhardt, Isabelle Meyts, Davide Montin, Lucia Dora Notarangelo, Fulvio Porta, Marlène Pasquet, Carsten Speckmann, Polina Stepensky, Alberto Tommasini, Marco Rabusin, Zeynep Karakas, Miguel Galicchio, Lucia Leonardi, Marzia Duse, Sukru Nail Guner, Clelia Di Serio, Fabio Ciceri, Maria Ester Bernardo, Alessandro Aiuti, Maria Pia Cicalese
Adenosine deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34+ cell gene therapy (GT) following busulfan reduced-intensity conditioning is a promising therapeutic approach for ADA-SCID, but long-term data are warranted. Here we report an analysis on long-term safety and efficacy data of 43 patients with ADA-SCID who received retroviral ex vivo bone marrow-derived hematopoietic stem cell GT. Twenty-two individuals (median follow-up 15...
February 14, 2024: Nature Medicine
#6
JOURNAL ARTICLE
Edward Abadir, Jad Othman, John Kwan, David J Gottlieb, Glen A Kennedy, Ashish Bajel, Richard Doocey, Travis Perera, Anne-Marie Watson, Peter G Bardy, Matthew Greenwood, David J Curtis, Steven Tran, John Moore, Nada Hamad
There is a limited body of evidence for haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in older patients. Previous studies have used a high proportion of bone marrow-derived grafts and a variety of conditioning regimens. In Australia and New Zealand, haplo-HCST is predominantly performed using peripheral blood (PB) with universal use of post-transplantation cyclophosphamide (PTCy). To characterize the outcomes of older recipients undergoing haplo-HSCT for acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS)...
November 27, 2023: Transplantation and cellular therapy
#7
Matt S Zinter, Ruta Brazauskas, Joelle Strom, Stella Chen, Stephanie Bo-Subait, Akshay Sharma, Amer Beitinjaneh, Dimana Dimitrova, Greg Guilcher, Jaime Preussler, Kasiani Myers, Neel S Bhatt, Olle Ringden, Peiman Hematti, Robert J Hayashi, Sagar Patel, Satiro Nakamura De Oliveira, Seth Rotz, Sherif M Badawy, Taiga Nishihori, David Buchbinder, Betty Hamilton, Bipin Savani, Hélène Schoemans, Mohamed Sorror, Lena Winestone, Christine Duncan, Rachel Phelan, Christopher C Dvorak
BACKGROUND: Allogeneic hematopoietic cell transplantation (HCT) can be complicated by the development of organ toxicity and infection necessitating intensive care. Risk factors for intensive care admission are unclear due to heterogeneity across centers, and long-term outcome data after intensive care are sparse due to a historical paucity of survivors. METHODS: The Center for International Blood and Marrow Transplant Research (CIBMTR) was queried to identify patients age ≤21 years who underwent a 1 st allogeneic HCT between 2008-2014 in the United States or Canada...
August 5, 2023: medRxiv
#8
REVIEW
Paulo Sérgio da Silva Santos, Fabiana Caramori Noal Granzotto, Héliton Spindola Antunes, Emilze Mafra de Lima, Renata de Freitas Varanda, Karina Maccari, Leticia Mello Bezinelli, Walmyr Ribeiro Melo, Luiz Alberto Valente Soares Junior, Leandro Dorigan De Macedo, Fernanda de Paula Eduardo
Patients undergoing hematopoietic stem cell transplantation (HSCT) might present acute and late toxicities and the oral tissues are frequently affected. With the survival increasing, patients show late and long-term morbidities, and there is an important association between the general and the oral health. The first and second parts of this Consensus have showed the importance of the adequacy of oral health in the pre-HSCT, and the main alterations and oral care during the period of admission for HSCT. This third part aims to review specific themes of post-HSCT dental care, such as graft-versus-host disease (GVHD) and the pediatric patient...
June 6, 2023: Hematology, Transfusion and Cell Therapy
#9
JOURNAL ARTICLE
Patrycja Zielińska, Izabela Noster, Agata Wieczorkiewicz-Kabut, Krzysztof Białas, Anna Koclęga, Grzegorz Helbig
INTRODUCTION: Severe aplastic anemia (SAA) is rare but potentially fatal disorder characterized by hypocellular bone marrow resulting in pancytopenia. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) can be curable especially for young individuals. OBJECTIVES: The main objective of the study was to assess the safety of the procedure and identification of factors influencing long-term post-transplant outcome. PATIENTS AND METHODS: Using our institutional database the retrospective analysis of patients with SAA allotransplanted in years 2001-2021 was performed...
February 28, 2023: Polish Archives of Internal Medicine
#10
JOURNAL ARTICLE
Chenyu Lin, Gautam Sajeev, Patrick J Stiff, Claudio G Brunstein, Corey Cutler, Guillermo Sanz, Caroline A Lindemans, Andrew R Rezvani, Rabi Hanna, Liang Piu Koh, Richard T Maziarz, William Y K Hwang, Yan Song, Qing Liu, Rocio Manghani, Smitha Sivaraman, James Signorovitch, Mitchell E Horwitz, Anthony D Sung
BACKGROUND: Omidubicel is an advanced cell therapy derived from umbilical cord blood (UCB) for use in allogeneic hematopoietic cell transplantation (HCT). A recent randomized phase 3 clinical trial demonstrated faster engraftment, shorter hospitalizations, and lower rates of infections with omidubicel when compared to standard UCB transplantation in patients with high-risk hematologic malignancies (Horwitz et al., Blood 2021). Despite the proven clinical benefits of omidubicel, its impact on health-related quality of life (HRQL) from the patient's perspective has not been described...
September 27, 2022: Transplantation and cellular therapy
#11
JOURNAL ARTICLE
Sagar Jawale
Introduction: Type 1 diabetes is a dreadful autoimmune disease of childhood with incidence of 0.26/1000 children in India. To develop a new, cheap, and effective treatment for this disease, we invented an autologous Stem cell therapy for type 1 diabetes in which stem cells are transplanted into the Omental pouch, peritoneum, and blood. The Omental pouch stem cell operation in the therapy is reported for the first time in the medical literature. Materials and methods: Last 5 years I treated 21 patients of Type 1 diabetes with autologous stem cell therapy and in the same period, a group of 26 patients of Type 1 diabetes with conventional treatment of Insulin injections was put as a control group...
September 2022: Annals of Medicine and Surgery
#12
JOURNAL ARTICLE
Catherine J Lee, Tao Wang, Karen Chen, Mukta Arora, Ruta Brazauskas, Stephen R Spellman, Carrie Kitko, Margaret L MacMillan, Joseph A Pidala, Jeffery J Auletta, Sherif M Badawy, Neel Bhatt, Vijaya R Bhatt, Jean-Yves Cahn, Zachariah DeFilipp, Miguel A Diaz, Nosha Farhadfar, Shahinaz Gadalla, Robert P Gale, Hasan Hashem, Shahrukh Hashmi, Peiman Hematti, Sanghee Hong, Nasheed M Hossain, Yoshihiro Inamoto, Lazaros J Lekakis, Dipenkumar Modi, Sager Patel, Akshay Sharma, Scott Solomon, Daniel R Couriel
BACKGROUND: Chronic graft-versus-host disease (cGVHD) occurs in up to 25% of children following allogeneic hematopoietic cell transplantation (HCT) and continues to be a major cause of late morbidity and poor quality-of-life among long-term survivors of pediatric HCT. Late effects of HCT are well documented in this population and cGVHD has been reported as a risk factor for subsequent neoplasms (SN) and several non-malignant late effects. However, the correlation between cGVHD and late effects varies between studies...
July 18, 2022: Transplantation and cellular therapy
#13
JOURNAL ARTICLE
Saveria Capria, Silvia Maria Trisolini, Daniela Diverio, Clara Minotti, Massimo Breccia, Claudio Cartoni, Daniela Carmini, Maria Gozzer, Ursula La Rocca, Mahnaz Shafii Bafti, Maurizio Martelli
BACKGROUND: Autologous stem cell transplantation (ASCT) has gained growing consideration as a treatment option for favorable-risk acute myeloid leukemia (FR-AML) in first complete remission (CR1), compared with chemotherapy. MATERIALS AND METHODS: We report the long-term outcomes of 117 consecutive patients with FR-AML fit for intensive chemotherapy diagnosed in our center between 1999 and 2020, who underwent ASCT. RESULTS: Sixty-five of the 117 were eligible for intensive post-remission treatment, and 42 of those 65 received ASCT...
October 2022: International Journal of Hematology
#14
JOURNAL ARTICLE
Elif Ozturk, Mehmet Cihangir Catak, Ayca Kiykim, Dilek Baser, Sevgi Bilgic Eltan, Koray Yalcin, Nurhan Kasap, Ercan Nain, Alper Bulutoglu, Gamze Akgun, Yasemin Can, Asena Pinar Sefer, Royala Babayeva, Suar Caki-Kilic, Gulsun Tezcan Karasu, Akif Yesilipek, Ahmet Ozen, Elif Karakoc-Aydiner, Safa Baris
PURPOSE: Severe combined immunodeficiency (SCID) is one of the most severe forms of inborn errors of immunity characterized by absence or loss of function in T cells. The long-term outcomes of all forms of SCID have been evaluated in a limited number of studies. We aimed to evaluate the pre- and post-transplant manifestations of SCID patients and determine the factors affecting the survival of patients. METHODS: We included 54 SCID patients (classical SCID, Omenn syndrome, atypical SCID (AS)) in this study...
July 2022: Journal of Clinical Immunology
#15
REVIEW
Kyaw Hein, Nicholas Short, Elias Jabbour, Musa Yilmaz
Measurable (minimal) residual disease (MRD) status in acute lymphoblastic leukemia (ALL) has largely superseded the importance of traditional risk factors for ALL, such as baseline white blood cell count, cytogenetics, and immunophenotype, and has emerged as the most powerful independent prognostic predictor. The development of sensitive MRD techniques, such as multicolor flow cytometry (MFC), quantitative polymerase chain reaction (PCR), and next-generation sequencing (NGS), may further improve risk stratification and expand its impact in therapy...
2022: Blood and Lymphatic Cancer: Targets and Therapy
#16
JOURNAL ARTICLE
Leo Ruhnke, Friedrich Stölzel, Uta Oelschlägel, Malte von Bonin, Katja Sockel, Jan Moritz Middeke, Christoph Röllig, Korinna Jöhrens, Johannes Schetelig, Christian Thiede, Martin Bornhäuser
In patients who have undergone allogeneic hematopoietic cell transplantation (HCT), myeloid mixed donor chimerism (MC) is a risk factor for disease relapse. In contrast, several studies found favorable outcome in patients with lymphoid MC. Thus far, most studies evaluating MC focused on a short-term follow-up period. Here, we report the first case series of long-term survivors with MC. We screened 1,346 patients having undergone HCT for myeloid neoplasms at our center from 1996 to 2016; 443 patients with data on total peripheral blood mononuclear cells (PBMC)/CD4+ /CD34+ short tandem repeat (STR) donor chimerism (DC) and follow-up ≥24 months post-HCT were included...
2021: Frontiers in Oncology
#17
P J Hayden, C Roddie, P Bader, G W Basak, H Bonig, C Bonini, C Chabannon, F Ciceri, S Corbacioglu, R Ellard, F Sanchez-Guijo, U Jäger, M Hildebrandt, M Hudecek, M J Kersten, U Köhl, J Kuball, S Mielke, M Mohty, J Murray, A Nagler, J Rees, C Rioufol, R Saccardi, J A Snowden, J Styczynski, M Subklewe, C Thieblemont, M Topp, Á U Ispizua, D Chen, R Vrhovac, J G Gribben, N Kröger, H Einsele, I Yakoub-Agha
BACKGROUND: Several commercial and academic autologous chimeric antigen receptor T-cell (CAR-T) products targeting CD19 have been approved in Europe for relapsed/refractory B-cell acute lymphoblastic leukemia, high-grade B-cell lymphoma and mantle cell lymphoma. Products for other diseases such as multiple myeloma and follicular lymphoma are likely to be approved by the European Medicines Agency in the near future. DESIGN: The European Society for Blood and Marrow Transplantation (EBMT)-Joint Accreditation Committee of ISCT and EBMT (JACIE) and the European Haematology Association collaborated to draft best practice recommendations based on the current literature to support health care professionals in delivering consistent, high-quality care in this rapidly moving field...
March 2022: Annals of Oncology: Official Journal of the European Society for Medical Oncology
#18
JOURNAL ARTICLE
Radhika Gangaraju, Yanjun Chen, Lindsey Hageman, Jessica Wu, Liton Francisco, Michelle Kung, Daniel J Weisdorf, Stephen J Forman, Mukta Arora, Saro Armenian, Smita Bhatia
BACKGROUND: Allogeneic blood or marrow transplant (BMT) recipients are at risk for venous-thromboembolism (VTE) because of high-intensity therapeutic exposures, comorbidities and a pro-inflammatory state due to chronic graft vs. host disease (GvHD). The long-term risk of VTE in allogeneic BMT survivors remains unstudied. METHODS: Participants were drawn from the BMT Survivor Study (BMTSS), a retrospective cohort study that included patients who underwent transplantation between 1974 and 2014 and survived ≥2y after BMT...
August 30, 2021: Blood Advances
#19
JOURNAL ARTICLE
Joanna Cwykiel, Arkadiusz Jundzill, Aleksandra Klimczak, Maria Madajka-Niemeyer, Maria Siemionow
This study evaluated the efficacy of donor recipient chimeric cell (DRCC) therapy created by fusion of donor and recipient derived bone marrow cells (BMC) in chimerism and tolerance induction in a rat vascularized composite allograft (VCA) model. Twenty-four VCA (groin flaps) from MHC-mismatched ACI (RT1a ) donors were transplanted to Lewis (RT1l ) recipients. Rats were randomly divided into (n = 6/group): Group 1-untreated controls, Groups 2-7-day immunosuppression controls, Group 3-DRCC, and Group 4-DRCC with 7-day anti-αβTCR monoclonal antibody and cyclosporine A protocol...
May 10, 2021: Archivum Immunologiae et Therapiae Experimentalis
#20
JOURNAL ARTICLE
Gemma McErlean, Lisa Brice, Nicole Gilroy, Masura Kabir, Matt Greenwood, Stephen R Larsen, John Moore, David Gottlieb, Mark Hertzberg, Louisa Brown, Megan Hogg, Gillian Huang, Christopher Ward, Ian Kerridge
BACKGROUND: Allogenic blood and marrow transplant (allo-BMT) is an arduous treatment used increasingly for many life-threatening conditions. Recognition of the profound impacts of the long term and late effects is ever-growing, as is the healthcare workload (treatment burden) of survivorship. PURPOSE: To quantify the treatment burden of long-term survival following allo-BMT, regarding the range of health services, therapies and investigations accessed by survivors...
April 2022: Journal of Cancer Survivorship: Research and Practice
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