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About 233 results

ALLMedicine™ Inherited Bone Marrow Failure Syndromes Center

Research & Reviews  86 results

Eltrombopag for People With Fanconi Anemia
https://clinicaltrials.gov/ct2/show/NCT03206086

May 13th, 2022 - Fanconi anemia (FA) is a rare genetic disease that often presents as a bone marrow failure (BMF) syndrome but also can affect any other organ. Etiologically, loss of function mutations in more than 21 different gene members of the FA core complex ...

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
https://clinicaltrials.gov/ct2/show/NCT05012111

May 13th, 2022 - Study Description: This study will allow for the long term follow up of patients with acquired and inherited bone marrow failure, both treated and untreated. Objectives: Primary Objective The primary objective is to characterize disease and treatm...

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

May 13th, 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...

Cell senescence and malignant transformation in the inherited bone marrow failure syndr...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9062194
Seminars in Hematology; Groarke EM, Calado RT et. al.

May 2nd, 2022 - Fanconi anemia, telomeropathies and ribosomopathies are members of the inherited bone marrow failure syndromes, rare genetic disorders that lead to failure of hematopoiesis, developmental abnormalities, and cancer predisposition. While each disord...

Syndromes predisposing to leukemia are a major cause of inherited cytopenias in children.
https://doi.org/10.3324/haematol.2021.280116
Haematologica Gilad O, Dgany O et. al.

Mar 18th, 2022 - Prolonged cytopenias are a non-specific sign with a wide differential diagnosis. Among inherited disorders, cytopenias predisposing to leukemia require a timely and accurate diagnosis to ensure appropriate medical management, including adequate mo...

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Guidelines  1 results

Recommendations on hematopoietic stem cell transplantation for inherited bone marrow fa...
https://doi.org/10.1038/bmt.2015.117
Bone Marrow Transplantation; Peffault de Latour R, Peters C et. al.

Jun 9th, 2015 - Allogeneic hematopoietic stem cell transplantation (HSCT) offers the potential to cure patients with an inherited bone marrow failure syndrome (IBMFS). However, the procedure involves the risk of treatment-related mortality and may be associated w...

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Clinicaltrials.gov  4 results

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

May 13th, 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...

Eltrombopag for People With Fanconi Anemia
https://clinicaltrials.gov/ct2/show/NCT03206086

May 13th, 2022 - Fanconi anemia (FA) is a rare genetic disease that often presents as a bone marrow failure (BMF) syndrome but also can affect any other organ. Etiologically, loss of function mutations in more than 21 different gene members of the FA core complex ...

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
https://clinicaltrials.gov/ct2/show/NCT05012111

May 13th, 2022 - Study Description: This study will allow for the long term follow up of patients with acquired and inherited bone marrow failure, both treated and untreated. Objectives: Primary Objective The primary objective is to characterize disease and treatm...

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
https://clinicaltrials.gov/ct2/show/NCT03513328

Jul 12th, 2021 - Hematopoietic stem cell transplantation is the only curative choice for a number of inherited bone marrow failure syndromes, hemoglobinopathies, metabolic disorders and primary immune deficiencies. While survival of these patients is typically bet...

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News  2 results

Study links gene dysfunction to Fanconi anemia, AML
https://www.mdedge.com/hematology-oncology/article/186620/anemia/study-links-gene-dysfunction-fanconi-anemia-aml
HT Staff

Aug 26th, 2014 - DNA repair Credit: Tom Ellenberger Researchers say they’ve discovered “an intimate link” between RUNX genes and Fanconi anemia, a finding that also has implications for treating acute myeloid leukemia (AML). The group found that RUNX1 and RUNX3 in.

Topics in Pediatric Leukemia -- Fanconi's Anemia: New Insights
https://www.medscape.com/viewarticle/499947

Introduction The inherited bone marrow failure syndromes in children comprise a number of rare congenital disorders in which the bone marrow is unable to produce blood cells; this results in a deficiency of 1 or more cell lines. Studies on the pat...

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