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  1. HLA-Compatible Related or Unrelated Donors With CD34+ Enriched, T-cell Depleted Peripheral Blood Stem Cells Isolated by the CliniMACS System in the Treatment of Patients With Hematologic Malignancies

    ... is indicated (e.g. hemophagocytic lymphohistiocytosis; refractory aplastic anemia or congenital cytopenias; non-SCID ... Syndrome, CD40 ligand deficiency, or ALPS, as well as refractory autoimmune cytopenias, PNH, metabolic storage diseases or heavily ...

    Clinical Trial last updated 04/29/2016 - 3:18pm.

  2. Clinical Trials Report for August 2015

    ... Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia The main goal of this study is to ... Ipilimumab in Treating Patients With Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia The ...

    Page last updated 07/30/2015 - 8:45am.

  3. Eltrombopag now an approved therapy for certain patients with severe aplastic anemia

    ... ATG, and it is the first drug ever approved to treat refractory aplastic anemia. Once a day treatment with this oral, ... FDA has approved eltrombopag for the treatment of these refractory patients.  It is important to note that this new labeling ...

    Page last updated 08/27/2014 - 2:19pm.

  4. Biparental HLA Haplotype Disparate T-cell Depleted Transplants for Patients Lacking an HLACompatible Donor

    ... is indicated (e.g. hemophagocytic lymphohistiocytosis; refractory aplastic anemia or conjugated cytopenias; non-SCID ...

    Clinical Trial last updated 04/29/2016 - 1:59pm.

  5. 2014 Bone Marrow Failure Disease Symposia - Penn State College of Medicine

    ... : A new and effective Treatment option for patients with Refractory Aplastic Anemia who lack HLA matched donors ...

    Page last updated 10/29/2014 - 3:39pm.

  6. Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders

    ... the World Health Organization (WHO) system as RA, RARS, refractory cytopenia with multilineage dysplasia (RCMD), RCMD and ...

    Clinical Trial last updated 04/28/2016 - 2:45pm.

  7. Eltrombopag: Recent Discoveries on Potential New Applications for Aplastic Anemia

    ... rounds of immunosuppressive therapy. This is known as refractory SAA. When tested in this setting, it was found to not only ... because there are few therapies available to patients with refractory aplastic anemia.  Additionally, we thought it would be likely that ...

    Interview last updated 02/14/2014 - 2:01pm.

  8. Kathryn (Kate) Bauer, Retired MDS Patient Living Life to the Fullest Since Transplant

    ... thought was, "I can't even spell it!"  The stage was Refractory Anemia with Excess Blasts (RAEB-1) with one ...

    Patient Chronicle last updated 07/23/2015 - 9:38am.

  9. Moderate, Severe, and Very Severe Aplastic Anemia: How are treatment decisions made?

    ... but at this point it is only approved for patients with refractory aplastic anemia. This is when there is no response or success ... anemia returns. In our trials here at NIH, roughly half of refractory patients responded to eltrombopag. Now we have an option for those ...

    Interview last updated 10/14/2015 - 12:47pm.

  10. Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow

    ... AML in second or subsequent complete remission. MDS: refractory anemia with excess blasts (RAEB), or chronic ...

    Clinical Trial last updated 04/28/2016 - 2:08pm.