About 3,398 results

ALLMedicine™ Bone Marrow Failure Center

Research & Reviews  1,167 results

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Jun 24th, 2022 - OUTLINE: CONDITIONING REGIMEN: Patients receive treosulfan intravenously (IV) over 120 minutes on days -6 to -4, fludarabine phosphate IV over 60 minutes on days -6 to -2, and lapine T-lymphocyte immune globulin (rATG) IV over 4-6 hours on days -4...

Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)

Jun 24th, 2022 - Severe aplastic anemia (SAA) is a life-threatening bone marrow failure characterized by pancytopenia and a hypocellular marrow. Allogeneic bone marrow transplantation is curative in younger patients, but older age and/or lack of a suitable donor h...

Eltrombopag for People With Fanconi Anemia

Jun 24th, 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

Jun 24th, 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

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...

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Guidelines  1 results

Recommendations on hematopoietic stem cell transplantation for inherited bone marrow fa...
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  5 results see all →

Clinicaltrials.gov  121 results

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused W...

Jun 24th, 2022 - Allogeneic hematopoietic stem cell transplantation (aHSCT) can cure patients with a variety of bone marrow failure syndromes (BMFS) including severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH) or myelodysplastic syndrome (MDS)...

Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy

Jun 24th, 2022 - 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...

Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy

Jun 24th, 2022 - Severe aplastic anemia (SAA) is a form of bone marrow failure and in most cases is the result of a cytotoxic T cell attack on the marrow stem cell. It is effectively treated in most patients with either immunosuppressive treatment (IST) or upfront...

Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7 Abnormalities After Immunosuppressive Therapy

Jun 24th, 2022 - Severe aplastic anemia (SAA) is a form of bone marrow failure in most cases is the result of a cytotoxic T cell attack on the marrow stem cell. It is effectively treated in most patients with either immunosuppressive treatment (IST) or allogeneic ...

Natural History Study of GATA2 Deficiency and Related Disorders

Jun 24th, 2022 - Mutations in GATA2, a critical hematopoietic transcription factor, underlie a complex congenital disorder characterized by immunodeficiency, bone marrow failure, and lymphatic/vascular dysfunction. Patients with GATA2 deficiency may suffer from a ...

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News  63 results

Pacritinib Paves a “Unique Lane” of Treatment for Select Myelofibrosis

Apr 5th, 2022 - Patients who develop post–essential thrombocythemia myelofibrosis (PET-MF) or post–polycythemia vera myelofibrosis (PPV-MF), which are associated with significant symptom burden and bone marrow failure, have limited therapies available for effecti...

Isolated Nodule and Generalized Lymphadenopathy
Yahya Daneshbod, MD, Michael Greas, MD et. al.

Mar 8th, 2022 - The Diagnosis: Blastic Plasmacytoid Dendritic Cell Neoplasm A diagnosis of blastic plasmacytoid dendritic cell neoplasm (BPDCN) was rendered. Subsequent needle core biopsy of a left axillary lymph node as well as bone marrow aspiration and biopsy.

An Introduction to Myeloproliferative Neoplasms

Feb 17th, 2022 - Ruben Mesa, MD:Hello and welcome to this OncLive® Peer Exchange titled Myeloproliferative Neoplasms. I’m Dr Ruben Mesa, the executive director of the Mays Cancer Center at UT Health San Antonio MD Anderson. Joining me in today’s discussions are my...

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...

Umbilical Cord Blood Stem Cells: FAQs

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’ - ...

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Patient Education  2 results see all →