Literature DB >> 32035274

Priorities for Improving Outcomes for Nonmalignant Blood Diseases: A Report from the Blood and Marrow Transplant Clinical Trials Network.

John E Levine1, Joseph H Antin2, Carl E Allen3, Lauri M Burroughs4, Kenneth R Cooke5, Steven Devine6, Helen Heslop7, Ryotaro Nakamura8, Julie An Talano9, Gregory Yanik10, Nancy DiFronzo11.   

Abstract

Nonmalignant blood diseases such as bone marrow failure disorders, immune dysregulation disorders, and hemoglobinopathies often lead to shortened life spans and poor quality of life. Many of these diseases can be cured with allogeneic hematopoietic cell transplantation, but patients are often not offered the procedure because of perceived insufficient efficacy and/or excess toxicity. In 2018, the Blood and Marrow Transplant Clinical Trials Network convened a task force to identify the most urgently needed yet feasible clinical trials with potential to improve the outcomes for patients with nonmalignant diseases. This report summarizes the task force discussions and specifies the network plans for clinical trial development for nonmalignant blood diseases.
Copyright © 2020 American Society for Transplantation and Cellular Therapy. All rights reserved.

Entities:  

Keywords:  Bone marrow failure; Nonmalignant; Thalassemia; Transplantation

Year:  2020        PMID： 32035274      PMCID： PMC8062984          DOI： 10.1016/j.bbmt.2020.01.024

Source DB:  PubMed          Journal:  Biol Blood Marrow Transplant        ISSN： 1083-8791            Impact factor:   5.742


  61 in total

1.  Blood and Marrow Transplant Clinical Trials Network State of the Science Symposium 2007.

Authors:  James L M Ferrara; Claudio Anasetti; Edward Stadtmauer; Joseph Antin; John Wingard; Stephanie Lee; John Levine; Kirk Schultz; Frederick Appelbaum; Robert Negrin; Sergio Giralt; Christopher Bredeson; Helen Heslop; Mary Horowitz
Journal:  Biol Blood Marrow Transplant       Date:  2007-11       Impact factor: 5.742

2.  Haploidentical HSCT for hemoglobinopathies: improved outcomes with TCRαβ+/CD19+-depleted grafts.

Authors:  Javid Gaziev; Antonella Isgrò; Pietro Sodani; Katia Paciaroni; Gioia De Angelis; Marco Marziali; Michela Ribersani; Cecilia Alfieri; Alessandro Lanti; Tiziana Galluccio; Gaspare Adorno; Marco Andreani
Journal:  Blood Adv       Date:  2018-02-13

3.  Haematopoietic stem cell transplantation for Shwachman-Diamond disease: a study from the European Group for blood and marrow transplantation.

Authors:  Simone Cesaro; Rosi Oneto; Chiara Messina; Brenda E Gibson; Agnes Buzyn; Colin Steward; Eliane Gluckman; Robbert Bredius; Robbert Breddius; Marc Boogaerts; Christiane Vermylen; Paul Veys; Judith Marsh; Isabel Badell; Gerard Michel; Tayfun Güngör; Dietrich Niethammer; Pierre Bordigoni; Cecilia Oswald; Claudio Favre; Jakob Passweg; Giorgio Dini
Journal:  Br J Haematol       Date:  2005-10       Impact factor: 6.998

Review 4.  Population Screening for Hemoglobinopathies.

Authors:  H W Goonasekera; C S Paththinige; V H W Dissanayake
Journal:  Annu Rev Genomics Hum Genet       Date:  2018-05-11       Impact factor: 8.929

5.  Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.

Authors:  Shalini Shenoy; Mark C Walters; Alex Ngwube; Sandeep Soni; David Jacobsohn; Sonali Chaudhury; Michael Grimley; Kawah Chan; Ann Haight; Kimberley A Kasow; Suhag Parikh; Martin Andreansky; Jim Connelly; David Delgado; Kamar Godder; Gregory Hale; Michael Nieder; Michael A Pulsipher; Felicia Trachtenberg; Ellis Neufeld; Janet L Kwiatkowski; Alexis A Thompson
Journal:  Biol Blood Marrow Transplant       Date:  2018-01-31       Impact factor: 5.742

Review 6.  Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members.

Authors:  Barbara P Yawn; George R Buchanan; Araba N Afenyi-Annan; Samir K Ballas; Kathryn L Hassell; Andra H James; Lanetta Jordan; Sophie M Lanzkron; Richard Lottenberg; William J Savage; Paula J Tanabe; Russell E Ware; M Hassan Murad; Jonathan C Goldsmith; Eduardo Ortiz; Robinson Fulwood; Ann Horton; Joylene John-Sowah
Journal:  JAMA       Date:  2014-09-10       Impact factor: 56.272

Review 7.  Emapalumab: First Global Approval.

Authors:  Zaina T Al-Salama
Journal:  Drugs       Date:  2019-01       Impact factor: 11.431

8.  Risk factors for mortality in adult patients with sickle cell disease: a meta-analysis of studies in North America and Europe.

Authors:  Poulami Maitra; Melissa Caughey; Laura Robinson; Payal C Desai; Susan Jones; Mehdi Nouraie; Mark T Gladwin; Alan Hinderliter; Jianwen Cai; Kenneth I Ataga
Journal:  Haematologica       Date:  2017-01-19       Impact factor: 9.941

9.  Alpha-1 Antitrypsin Attenuates Acute Lung Allograft Injury in a Rat Lung Transplant Model.

Authors:  Amir M Emtiazjoo; Hanbo Hu; Li Lu; Mark L Brantly
Journal:  Transplant Direct       Date:  2019-05-29

10.  DNA alkylation and interstrand cross-linking by treosulfan.

Authors:  J A Hartley; C C O'Hare; J Baumgart
Journal:  Br J Cancer       Date:  1999-01       Impact factor: 7.640
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  2 in total

Review 1.  Blood and Marrow Transplant Clinical Trials Network State of the Science Symposium 2021: Looking Forward as the Network Celebrates its 20th Year.

Authors:  Helen E Heslop; Edward A Stadtmauer; John E Levine; Karen K Ballen; Yi-Bin Chen; Amy E DeZern; Mary Eapen; Mehdi Hamadani; Betty K Hamilton; Parameswaran Hari; Richard J Jones; Brent R Logan; Leslie S Kean; Eric S Leifer; Frederick L Locke; Richard T Maziarz; Eneida R Nemecek; Marcelo Pasquini; Rachel Phelan; Marcie L Riches; Bronwen E Shaw; Mark C Walters; Amy Foley; Steven M Devine; Mary M Horowitz
Journal:  Transplant Cell Ther       Date:  2021-08-27

2.  CXCR6+ and NKG2C+ Natural Killer Cells Are Distinct With Unique Phenotypic and Functional Attributes Following Bone Marrow Transplantation.

Authors:  Kevin Aviles-Padilla; Laura S Angelo; Dwight Fan; Silke Paust
Journal:  Front Immunol       Date:  2022-06-29       Impact factor: 8.786
  2 in total

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