Last updated on April 2019

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 with a Reduced Dose of Non-Mobilized Donor T-Cells

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: Myelodysplastic Syndromes (MDS) | Severe Aplastic Anemia | Bone Marrow Failure | Adult | Pediatric | Aplastic Anemia
  • Age: Between 4 - 80 Years
  • Gender: Male or Female




  • Patients diagnosed with one of the following hematologic diseases which are associated with reasonable longevity, shown to be curable by allogeneic BMT but where concern for a high procedural mortality with conventional BMT may delay or prevent such treatment:
    • Paroxysmal nocturnal hemoglobinuria (PNH) associated with life-threatening thrombosis, and/or cytopenia, and/or transfusion dependence and/or recurrent and debilitating hemolytic crisis
    • Severe aplastic anemia (SAA) or pure red cell aplasia (PRCA [acquired or congenital]) associated with transfusion dependence and/or neutropenia in patients who are not candidates for, or who have failed immunosuppressive therapy
    • Refractory anemia (RA) or RARS MDS patients who have associated transfusion dependence and/or neutropenia.
  • Ages 4 to 80 (both inclusive), and weight >18kg
  • Availability of HLA identical or single HLA locus mismatched family donor or 10/10 matched unrelated donor at the allelic level (HLA alleles A, B, C, DR, and DQ).
  • 9/10 donors where all the HLA sequences have the same antigen/peptide binding domains in key exons to the patient. This can result in identical protein sequences between patient and donor. Allele mismatches in p and g groups can be considered acceptable due to the exact matching which exists in the binding domains.
  • Telomere Length Testing
  • Germline/Inherited gene panel in patients where a suspicion for a familial bone marrow failure syndrome (BMFS) exists, hTERC and hTERT, GATA2 mutation testing will be performed on protocol 04-H-0012 or performed elsewhere prior to enrolling on 04-H-0012.

Related Donor:

  • HLA identical or single HLA mismatched family donor
  • Age greater than or equal to 4 and less than or equal to 80 years old
  • Weight > 18 kg
  • If there is a suspicion of familial BMFS in the recipient, then the donor must have underong genetic testing for genes associated with BMFS -performed at a CLIA-certified laboratory, prior to enrolling in this protocol.



Recipient: any of the following

  • Major anticipated illness or organ failure incompatible with survival from PBSC transplant
  • Diffusion capacity of carbon monoxide (DLCO) <40% predicted (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed) using DL Adj and DL/VA/Adj.
  • Left ventricular ejection fraction <40% (evaluated by ECHO) or <30% (evaluated by MUGA)
  • Serum creatinine greater than 2.5mg/dl or creatinine clearance less than 50 ml/min by 24 hr urine collection
  • Serum bilirubin greater than 4 mg/dl, transaminases greater than 5 times the upper limit of normal
  • Pregnant or lactating
  • Fanconi s anemia
  • ECOG performance status of 3 or more (See Bone & Marrow Transplant Consortium Supportive Care Guidelines for HSCT Recipients)
  • Other malignant diseases liable to relapse or progress within 5 years, with the exception of a separate hematologic malignancy where allogeneic stem cell transplant has been shown to be potentially curative.
  • Presence of an active infection not adequately responding to appropriate therapy.
  • Inability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects age 8 -17 years with formal consent being obtained from parents or legal guardian.

Related Donor: any of the following

  • Pregnant or lactating
  • Unfit to receive filgrastim (G-CSF) or undergo apheresis (history of stroke, MI, unstable angina, uncontrolled hypertension, severe heart disease or palpable spleen)
  • HIV positive (donors who are positive for HBV, HCV or HTLV-I/II, T.cruzi [Chagas] may be used at the discretion of the investigator following counseling and approval from the recipient)
  • Sickling hemoglobinopathies including HbSS or HbSC. Donors with HbAS are acceptable
  • Inability of donor or guardian of donor to comprehend the investigational nature of the study and provide informed consent.
  • Screening test positive for Chagas disease (Trypanosoma cruzi /T. cruzi/trypanosomiasis) confirmed by the Center for Disease Control (CDC).



Unrelated Donor

  • The NMDP unrelated donor inclusion criteria will be used as outlined in document (link).
  • Donor eligibility will be completed per NMDP standards and in accordance with most recent and stringent FDA guidelines.

Recruitment Status: Open

Brief Description Eligibility Contact Research Team

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