About: Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel     Goto   Sponge   NotDistinct   Permalink

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Description
  • Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in the world. The outlook for affected individuals has improved in recent years due to advances in medical management in the prevention and treatment of complications. However, hematopoietic stem cell transplantation is still the only available curative option. The use of hematopoietic stem cell transplantation has been increasing, and outcomes today have substantially improved compared with the past three decades. Current experience world-wide is that more than 90% of patients now survive hematopoietic stem cell transplantation and disease-free survival is around 80%. However, only a few controlled trials have been reported, and decisions on patient selection for hematopoietic stem cell transplantation and transplant management remain principally dependent on data from retrospective analyses and on the clinical experience of the transplant centers. This consensus document from the European Blood and Marrow Transplantation Inborn Error Working Party and the Paediatric Diseases Working Party aims to report new data and provide consensus-based recommendations on indications for hematopoietic stem cell transplantation and transplant management.
  • Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in the world. The outlook for affected individuals has improved in recent years due to advances in medical management in the prevention and treatment of complications. However, hematopoietic stem cell transplantation is still the only available curative option. The use of hematopoietic stem cell transplantation has been increasing, and outcomes today have substantially improved compared with the past three decades. Current experience world-wide is that more than 90% of patients now survive hematopoietic stem cell transplantation and disease-free survival is around 80%. However, only a few controlled trials have been reported, and decisions on patient selection for hematopoietic stem cell transplantation and transplant management remain principally dependent on data from retrospective analyses and on the clinical experience of the transplant centers. This consensus document from the European Blood and Marrow Transplantation Inborn Error Working Party and the Paediatric Diseases Working Party aims to report new data and provide consensus-based recommendations on indications for hematopoietic stem cell transplantation and transplant management. (en)
Title
  • Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel
  • Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel (en)
skos:prefLabel
  • Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel
  • Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel (en)
skos:notation
  • RIV/00216208:11130/14:10292968!RIV15-MSM-11130___
http://linked.open...avai/riv/aktivita
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  • 18929
http://linked.open...ai/riv/idVysledku
  • RIV/00216208:11130/14:10292968
http://linked.open...riv/jazykVysledku
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  • preparative regimen; mixed chimerism; conditioning regimen; allogeneic transplantation; long-term; peripheral-blood; beta-thalassemia; iron chelation-therapy; cord blood transplantation; bone-marrow-transplantation (en)
http://linked.open.../riv/klicoveSlovo
http://linked.open...odStatuVydavatele
  • IT - Italská republika
http://linked.open...ontrolniKodProRIV
  • [C9082AB15FB5]
http://linked.open...i/riv/nazevZdroje
  • Haematologica
http://linked.open...in/vavai/riv/obor
http://linked.open...ichTvurcuVysledku
http://linked.open...cetTvurcuVysledku
http://linked.open...UplatneniVysledku
http://linked.open...v/svazekPeriodika
  • 99
http://linked.open...iv/tvurceVysledku
  • Sedláček, Petr
  • Gluckman, Eliane
  • Locatelli, Franco
  • Rocha, Vanderson
  • Minkov, Milen
  • Peters, Christina
  • Angelucci, Emanuele
  • Baronciani, Donatella
  • Bernaudin, Francoise
  • Bonanomi, Sonia
  • Cappellini, Maria Domenica
  • Cavazzana, Marina
  • Dalle, Jean-Hugues
  • Di Bartolomeo, Paolo
  • Diaz de Heredia, Cristina
  • Dickerhoff, Roswitha
  • Giardini, Claudio
  • Hussein, Ayad Achmed
  • Kamani, Naynesh
  • Matthes-Martin, Susanne
  • Smiers, Frans
  • Thuret, Isabelle
  • Yaniv, Isaac
http://linked.open...ain/vavai/riv/wos
  • 000336257500008
issn
  • 0390-6078
number of pages
http://bibframe.org/vocab/doi
  • 10.3324/haematol.2013.099747
http://localhost/t...ganizacniJednotka
  • 11130
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