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About 653 results

ALLMedicine™ Diamond-blackfan Anemia Center

Research & Reviews  168 results

Cancer in Inherited Bone Marrow Failure Syndromes
https://clinicaltrials.gov/ct2/show/NCT00027274

Jun 24th, 2022 - Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders. Pathogenic variant(s) in IBMFS genes are relevant to carcinogenesis in sporadic ca...

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
https://clinicaltrials.gov/ct2/show/NCT04269889

Jun 24th, 2022 - Diamond-Blackfan anemia (DBA) is a heritable bone marrow failure (BMF) syndrome characterized by selective erythroid defects typically presenting within the first year of life as a normochromic, macrocytic anemia with reticulocytopenia. More than ...

Recent advances in hematopoietic cell transplantation for inherited bone marrow failure...
https://doi.org/10.1007/s12185-022-03362-4 10.3324/haematol.2010.025619 10.1007/s12185-012-1249-9 10.1016/j.blre.2010.03.002 10.1182/blood-2017-09-806489 10.1038/gim.2016.197 10.1111/cas.14986 10.1038/ng.3569 10.1126/sciadv.abd7197 10.3324/haematol.2017.178111 10.1182/bloodadvances.2019000839 10.1182/blood-2013-09-427740 10.1007/s12185-012-1079-9 10.1182/blood-2013-01-479733 10.1182/blood-2015-02-626002 10.1016/j.bbmt.2017.11.031 10.1007/s12185-020-02991-x 10.1056/NEJM198910263211707 10.1016/j.bbmt.2007.05.015 10.1002/ajh.26135 10.1182/bloodadvances.2020003707 10.1002/pbc.29440 10.1002/ajh.25810 10.1111/bjh.17418 10.1016/j.bbmt.2016.03.007 10.1182/blood-2005-07-2622 10.1007/s12185-015-1861-6 10.1182/asheducation-2007.1.29 10.1074/jbc.REV120.014017 10.1111/bjh.15495 10.3324/haematol.2010.027839 10.1038/bmt.2010.65 10.1111/j.1399-3046.2010.01431.x 10.1016/j.bbmt.2013.05.021 10.1016/j.bbmt.2016.03.001 10.1182/blood-2008-09-178871 10.1111/bjh.15493 10.1056/NEJMoa1515319 10.1136/bmjresp-2021-001127 10.1182/blood-2011-08-375972 10.1007/s12328-020-01176-7 10.1182/blood.2019000947 10.1111/bjh.15701 10.1111/bjh.12787 10.1182/bloodadvances.2019001210 10.1038/s41409-020-01056-1 10.1016/j.jtct.2020.12.024 10.1111/j.1365-2141.2009.07888.x 10.1111/j.1365-2141.2010.08216.x 10.1038/s41409-020-0800-1 10.1182/blood-2015-02-628859 10.1016/S2352-3026(19)30206-6 10.1016/j.jpeds.2019.12.041 10.1016/j.jpeds.2013.11.039 10.1038/ng1062 10.1038/s41409-020-0863-z 10.1016/j.bbmt.2020.04.029 10.1111/j.1365-2141.2005.05819.x 10.3324/haematol.11425 10.1016/j.bbmt.2013.12.099 10.1016/j.jtct.2021.10.009 10.1182/blood-2017-12-822262 10.1038/s41591-021-01511-6 10.1038/s41467-017-01590-5 10.1016/j.bbmt.2019.07.007 10.1182/bloodadvances.2017012682
International Journal of Hematology; Sakaguchi H, Yoshida N

May 29th, 2022 - Inherited bone marrow failure syndromes (IBMFSs) are a group of rare genetic disorders characterized by bone marrow failure with unique phenotypes and predisposition to cancer. Classical IBMFSs primarily include Fanconi anemia with impaired DNA da...

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
https://clinicaltrials.gov/ct2/show/NCT03653338

May 6th, 2022 - CD3/CD19 depletion of mismatched donor grafts in the setting of reduced intensity, immune-ablative conditioning for patients with sickle cell disease and other transfusion-dependent anemias should sufficiently achieve engraftment while decreasing ...

Observational Study for Patients With Hemoglobinopathies and Rare Inherited Anemia and Covid 19
https://clinicaltrials.gov/ct2/show/NCT04746066

May 5th, 2022 - The COVID-19 pandemic is causing many deaths around the world, putting a strain on health services. Patients with pre-existing chronic diseases are most affected by SARS-COV2 infection. Italy is one of the countries most involved. Thalassaemia, dr...

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Drugs  74 results see all →

Clinicaltrials.gov  12 results

Cancer in Inherited Bone Marrow Failure Syndromes
https://clinicaltrials.gov/ct2/show/NCT00027274

Jun 24th, 2022 - Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders. Pathogenic variant(s) in IBMFS genes are relevant to carcinogenesis in sporadic ca...

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
https://clinicaltrials.gov/ct2/show/NCT04269889

Jun 24th, 2022 - Diamond-Blackfan anemia (DBA) is a heritable bone marrow failure (BMF) syndrome characterized by selective erythroid defects typically presenting within the first year of life as a normochromic, macrocytic anemia with reticulocytopenia. More than ...

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
https://clinicaltrials.gov/ct2/show/NCT03653338

May 6th, 2022 - CD3/CD19 depletion of mismatched donor grafts in the setting of reduced intensity, immune-ablative conditioning for patients with sickle cell disease and other transfusion-dependent anemias should sufficiently achieve engraftment while decreasing ...

Observational Study for Patients With Hemoglobinopathies and Rare Inherited Anemia and Covid 19
https://clinicaltrials.gov/ct2/show/NCT04746066

May 5th, 2022 - The COVID-19 pandemic is causing many deaths around the world, putting a strain on health services. Patients with pre-existing chronic diseases are most affected by SARS-COV2 infection. Italy is one of the countries most involved. Thalassaemia, dr...

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
https://clinicaltrials.gov/ct2/show/NCT01966367

Jun 19th, 2020 - Graft-versus-host disease (GVHD) is a condition that results from a reaction of transplanted donor T-lymphocytes against the body and organs of the patient receiving the transplanted cells. There are two forms: acute (early) and chronic (late). Ac...

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News  7 results

Posttransplant Cyclophosphamide for GVHD
https://www.onclive.com/view/posttransplant-cyclophosphamide-for-gvhd

Apr 2nd, 2021 - Transcript: Yi-Bin A. Chen, MD: The use of posttransplant cyclophosphamide-based regimens do prevent graft-versus-host disease. It’s one of the biggest advances in transplant over the past 2 decades. Popularized by our colleagues at The Johns ...

Study of DBA provides new insight into hematopoiesis
https://www.mdedge.com/hematology-oncology/article/185013/anemia/study-dba-provides-new-insight-hematopoiesis
HT Staff

Apr 13th, 2018 - Red blood cells In studying Diamond-Blackfan anemia (DBA), researchers have found evidence to suggest that ribosome levels play a key role in hematopoiesis. The researchers found that a reduction in ribosomes is responsible for the disruption in r.

T-cell product improves outcomes of haplo-HSCT
https://www.mdedge.com/hematology-oncology/article/185594/anemia/t-cell-product-improves-outcomes-haplo-hsct
Jen Smith

Jun 29th, 2017 - Photo by Chad McNeeley HSCT preparation MADRID—Updated results of a phase 1/2 study suggest the T-cell product BPX-501 lowers the risks associated with haploidentical hematopoietic stem cell transplant (haplo-HSCT). In this ongoing study, research.

Inflammation may predict transformation to AML
https://www.mdedge.com/hematology-oncology/article/188849/anemia/inflammation-may-predict-transformation-aml
HT Staff

Oct 5th, 2016 - Micrograph showing MDS Inflammatory signaling in mesenchymal niche cells can be used to predict the transformation from pre-leukemic syndrome to acute myeloid leukemia (AML), according to preclinical research published in Cell Stem Cell. “This dis.

Model recapitulates cancer susceptibility in DBA
https://www.mdedge.com/hematology-oncology/article/187539/anemia/model-recapitulates-cancer-susceptibility-dba
HT Staff

Oct 20th, 2015 - Lab mouse Researchers say they’ve created the first animal model that recapitulates the predisposition to cancer observed in patients with Diamond-Blackfan anemia (DBA). DBA is caused by mutations in ribosomal genes such as RPL11, so the researche.

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