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About 2,553 results

ALLMedicine™ Bone Marrow Failure Center

Research & Reviews  942 results

LNK (SH2B3) Inhibition Expands Healthy and Fanconi Anemia Human Hematopoietic Stem and ...
https://doi.org/10.1182/bloodadvances.2021004205
Blood Advances; Holdreith N, Lee GY et. al.

Nov 30th, 2021 - Hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for a variety of hematological diseases. Allogenic HSCT requires hematopoietic stem cells (HSCs) from matched donors and comes with cytotoxicity and mortality. Rece...

miR‑30a‑5p induces the adipogenic differentiation of bone marrow mesenchymal stem cells...
https://doi.org/10.3892/mmr.2021.12543
Molecular Medicine Reports; Wang E, Zhang Y et. al.

Nov 26th, 2021 - Aplastic anemia (AA) is a bone marrow failure syndrome with high morbidity and mortality. Bone marrow (BM)‑mesenchymal stem cells (MSCs) are the main components of the BM microenvironment, and dysregulation of BM‑MSC adipogenic differentiation is ...

Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
https://clinicaltrials.gov/ct2/show/NCT02979873

Nov 22nd, 2021 - Most acquired aplastic anemia ensues from immune-mediated destruction of hematopoietic stem and progenitor cells. Immunosuppression is the definitive treatment of patients with acquired aplastic anemia who are not candidates for immediate hematopo...

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

Nov 17th, 2021 - 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 ...

Gain-of-Function Mutations in RPA1 Cause a Syndrome with Short Telomeres and Somatic Ge...
https://doi.org/10.1182/blood.2021011980
Blood Sharma R, Sahoo SS et. al.

Nov 13th, 2021 - Human telomere biology disorders (TBD)/short telomere syndromes (STS) are heterogeneous disorders caused by inherited loss-of-function mutations in telomere-associated genes. Here, we identify three germline heterozygous missense variants in RPA1 ...

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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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Drugs  4 results see all →

Clinicaltrials.gov  37 results

Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
https://clinicaltrials.gov/ct2/show/NCT02979873

Nov 22nd, 2021 - Most acquired aplastic anemia ensues from immune-mediated destruction of hematopoietic stem and progenitor cells. Immunosuppression is the definitive treatment of patients with acquired aplastic anemia who are not candidates for immediate hematopo...

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

Nov 17th, 2021 - 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 ...

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

Nov 10th, 2021 - 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...

Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
https://clinicaltrials.gov/ct2/show/NCT03047746

Sep 17th, 2021 - This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in pat...

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  36 results

A New Era of Precision Medicine Takes Shape in Prostate Cancer
https://www.onclive.com/view/a-new-era-of-precision-medicine-takes-shape-in-prostate-cancer

Oct 26th, 2021 - Prostate cancer is a highly hetero-geneous disease, with individual tumor cells having distinct genomic and pheno-typic characteristics, resulting in a great deal of variability in patients’ disease course. Although some pros-tate cancers are slow...

Bringing Epigenetic Therapies From Bench to Bedside
https://www.onclive.com/view/bringing-epigenetic-therapies-from-bench-to-bedside

Oct 6th, 2021 - Ronan T. Swords, MD, PhD Pap Corps Endowed Professor in Leukemia Sylvester Comprehensive Cancer Center University of Miami Miller School of Medicine Miami, FL Although there have been advances in therapeutics and supportive care, the...

Umbilical Cord Blood Stem Cells: FAQs
https://www.medscape.com/viewarticle/959130

Sep 21st, 2021 - WHAT Is Cord Blood? Umbilical cord blood remains in the placenta and umbilical cord after a baby is born. This blood is rich in stem cells, which may be able to help cure some life threatening diseases. Stem cells – also known as ‘master cells’ - ...

First-Line Therapy Options in Hairy Cell Leukemia
https://www.onclive.com/view/first-line-therapy-options-in-hairy-cell-leukemia

Apr 2nd, 2021 - Transcript: Robert J. Kreitman, MD: Hairy cell leukemia is a rare chronic B-cell leukemia that accounts for 2% of all leukemias, with approximately 1200 new cases reported in the United States each year. Major advances in the treatment and unde...

Heterogeneity and Presentation of MDS
https://www.onclive.com/view/heterogeneity-and-presentation-of-mds

Apr 2nd, 2021 - Amy DeZern, MD, MHS: Hello, and welcome to this OncLive® Insights®, Treatment Options in Myelodysplastic Syndromes. I am Amy DeZern from Johns Hopkins University School of Medicine in Baltimore, Maryland. Joining me is Rami Komrokji from H. Lee Mo...

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Patient Education  1 results see all →